NMDP℠ Demonstrates Scientific Leadership in Expanding Patient Access to Lifesaving Cell Therapy at ASH 2025

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NMDP℠ Demonstrates Scientific Leadership in Expanding Patient Access to Lifesaving Cell Therapy at ASH 2025

NMDP℠ Demonstrates Scientific Leadership in Expanding Patient Access to Lifesaving Cell Therapy at ASH 2025

New results from the Phase II ACCESS trial represent a significant milestone in solving one of transplant medicine’s biggest challenges: finding a donor for every patient in need of a blood stem cell transplant

One-year overall survival exceeds 80% among trial participants receiving 4/8-7/8 mismatched unrelated donor transplant

Ninety-nine percent of patients with common blood cancers can find a suitable donor and safely and effectively access transplant

MINNEAPOLIS–(BUSINESS WIRE)–New research from NMDP, a global nonprofit leader in cell therapy, and conducted by CIBMTR® (Center for International Blood and Marrow Transplant Research®) at the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla., demonstrates that patients receiving allogeneic transplantation using more deeply mismatched unrelated donors (MMUD), followed by post-transplant chemotherapy, can achieve outcomes comparable to those receiving transplants from more closely, but still not fully, matched donors. The data also show that virtually every patient searching international registries now has a greater than 99% likelihood of identifying a suitable blood stem cell donor. Notably, ASH selected this abstract for presentation at its 2026 Highlights of ASH® program in January.

While exact donor-patient genetic matching was historically considered essential for preventing life-threatening complications after transplant, results from the NMDP-sponsored ACCESS study [#936] showed strong, consistent outcomes among adults receiving donor grafts across all mismatch levels (4/8–7/8). At one-year post-transplant, overall survival (OS) exceeded 80% across all match levels, achieving a significant 86% for <7/8 mismatches and 79% for 7/8 mismatches.

“We are fundamentally changing what’s possible in transplant medicine and creating a new standard of care for curing common blood cancers,” said Steven M. Devine, M.D., chief medical officer, NMDP, and executive lead, CIBMTR. “For the first time, we have clear evidence that patients can safely receive a range of mismatched unrelated donor grafts and achieve survival outcomes on par with those from fully matched donors—this is a giant leap forward for transplant science and medicine.”

The most important factor that determines donor-recipient compatibility in unrelated donor transplantation is matching at human leukocyte antigens (HLA), the proteins that help the immune system distinguish its cells from another’s. When HLA mismatch occurs, the donor’s immune system can recognize the patient’s tissues as foreign, triggering graft-versus-host disease (GVHD), a potentially life-threatening complication.

Historically, achieving an 8/8 HLA match resulted in the best transplant outcomes, while using donors with fewer matching alleles (<7/8) was associated with poor survival and higher rates of GVHD. However, NMDP research published in the Journal of Clinical Oncology in June demonstrated that patients receiving <7/8 matched graft achieved outcomes comparable to, or even exceeding, historical 8/8 benchmarks of 75% survival—supporting broader donor eligibility.

Finding a fully or closely matched donor remains a major barrier for many patients, as HLA markers are inherited. If there are no suitable matches within the family, physicians must search international registries, now totaling more than 42 million potential donors and more than 760,000 cord blood units. Unfortunately, patients of non-European ancestry have historically had much lower chances of finding a suitable donor.¹

ACCESS trial demonstrates safe and effective use of MMUDs

Presenting author Antonio Jimenez-Jimenez, M.D. will deliver the oral presentation on Monday, Dec. 8, at 4 p.m. EST sharing detailed results from ACCESS (NCT04904588). This prospective, multicenter trial evaluated 268 adult patients with common blood cancers who received 4/8 to 7/8 HLA-MMUD peripheral blood stem cell (PBSC) grafts with post-transplant cyclophosphamide (PTCy). While prior studies explored PTCy for GVHD prevention in related individuals with HLA mismatches, the ACCESS trial is the first to evaluate this approach in patients receiving PBSC transplants from unrelated donors with as few as 4/8 HLA matches. Notably, 61% of participants in the <7/8 cohort self-identified as other than non-Hispanic White.

“These results challenge long-held assumptions about the risks of HLA mismatching and demonstrate that PTCy-based regimens can safely extend donor eligibility to nearly all patients in need of transplant, including those from varied backgrounds who are most in need of a suitably matched donor,” said Dr. Jimenez-Jimenez, associate professor of medicine in the division of Transplantation & Cellular Therapy at Sylvester Comprehensive Cancer Center, at the University of Miami Miller School of Medicine.

Non-relapse mortality (7/8=13.7%; <7/8=8.4%), relapse (7/8=17.1%; <7/8=22.8%), and moderate-to-severe chronic GVHD (7/8=11.3%; <7/8=7.7%) rates were similarly favorable between cohorts, regardless of conditioning regimen.

An additional CIBMTR analysis included in the presentation depicted the ACCESS results within the context of current donor availability on international registries. For patients highly unlikely to find an 8/8 match, allowing a <7/8 match offers a significantly higher pool of potential donors available, even if these patients had ethnically diverse backgrounds. Sixty-two percent of patients included in this five year analysis were ethnically diverse, and the median number of available donors increased from two at the 7/8 level to 83 at the 6/8 level, illustrating the profound impact of broadening acceptable match levels.

Donor for All: Research advances outcomes and access for all patients

NMDP’s Donor for All initiative unites clinical research, data science and operational innovation to close long-standing gaps in access for patients, especially for those of diverse ancestry. The ACCESS, OPTIMIZE (NCT06001385) and ACCELERATE (NCT06859424) trials are key Phase II trials at the core of the Donor for All initiative; additional ACCESS findings and in-progress updates from ACCELERATE will be presented at ASH, including:

Results from the reduced-intensity conditioning (RIC) cohort [#4232] of the ACCESS study in a poster presentation. Findings demonstrated one-year OS at 79.6% and relatively low rates of non-relapse mortality (12.5%), chronic GVHD (15.6%) and severe GVHD (3.7%) across HLA match levels. Results confirmed that PTCy-based MMUD transplantation delivers consistent and reproducible outcomes across conditioning intensities and centers, further supporting its use as a safe and scalable approach to expand donor access.
A poster presentation of the ACCESS PRO (“Living the Recovery”) [#6042] will also show findings that complemented these positive clinical outcomes. The study examined patient-reported outcomes to better understand survivorship and quality of life one year after transplant. Among 268 patients, self-reported quality-of-life scores, physical function and fatigue returned to or exceeded baseline levels, with overall results comparable to population norms. Financial well-being also remained stable from baseline to one year. Patients who experienced moderate or severe chronic GVHD reported greater symptom burden and reduced physical function, underscoring the importance of continued efforts to reduce GVHD incidence and strengthen supportive care strategies.
Another poster presentation will show the latest from the in-progress ACCELERATE trial, [#6035] which represents the next step in optimizing transplant. This multicenter platform protocol will evaluate multiple PTCy-based GVHD prevention regimens—testing reduced-dose PTCy with novel agents such as abatacept and ruxolitinib—across up to 60 U.S. centers. The goal: to refine efficacy, minimize toxicity and further improve long-term outcomes for patients receiving mismatched donor transplants. ACCELERATE is still enrolling at sites across the U.S.

“These collective studies move us closer to a future where donor match limitations no longer determine who receives a cure,” said Dr. Devine. “With donor matching no longer as restrictive, clinicians can now prioritize other factors, such as donor age and cell quality, to optimize outcomes and further individualize transplant care. Through our Donor for All initiative, NMDP is combining rigorous science with a commitment to equity so that every searching patient, regardless of ancestry, can find their cure.”

Additional studies presented at ASH 2025

Other research presented by NMDP and CIBMTR at ASH further support the organizations’ aim to make safe and effective cell therapy available to every patient in need.

In an oral presentation to be presented Sunday, Dec. 7 at 10:30 a.m., Kai Yu, Ph.D., Fralin Biomedical Research Institute, Virginia Tech FBRI Cancer Research Center, will share results from the MEASURE Genome Atlas study [#455], which sought to advance understanding of the genomic landscape of acute myeloid leukemia (AML). Using whole genome sequencing (WGS) from 255 adult patients across 18 U.S. cancer centers, the study demonstrated that WGS can function as a single, comprehensive diagnostic platform for AML—reproducing 93% of standard prognostic markers while identifying additional alterations, including KMT2A partial tandem duplications and cryptic gene fusions missed by traditional testing. This comprehensive atlas of adult AML genomics provides novel insights into disease biology, creates an evidentiary basis to support clinical testing improvements, and is a resource for both diagnostic and drug development. NMDP supported this study, with CIBMTR facilitating the research.

ReAlta Announces Promising Phase 2 Data Demonstrating Rapid Clinical Responses and Target Engagement in Steroid-Refractory Acute GvHD at the ASH 2025 Annual Meeting

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ReAlta Announces Promising Phase 2 Data Demonstrating Rapid Clinical Responses and Target Engagement in Steroid-Refractory Acute GvHD at the ASH 2025 Annual Meeting

ReAlta Announces Promising Phase 2 Data Demonstrating Rapid Clinical Responses and Target Engagement in Steroid-Refractory Acute GvHD at the ASH 2025 Annual Meeting

Pegtarazimod demonstrates significant clinical improvement and reduction in inflammatory markers in patients with lower gastrointestinal acute GvHD

NORFOLK, Va.–(BUSINESS WIRE)–ReAlta Life Sciences, Inc. (“ReAlta” or the “Company”), a clinical-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address rare and acute inflammatory diseases, today announced a presentation of interim Phase 2 clinical data on RLS-0071 (pegtarazimod) for the treatment of acute graft-versus-host disease (aGvHD) at the 67th American Society of Hematology (ASH) Annual Meeting.

The new findings will be presented in a poster titled “Anti-inflammatory drug pegtarazimod (RLS-0071) demonstrates clinical improvement in lower gastrointestinal acute graft-versus-host disease (aGvHD) and target engagement of key inflammatory marker myeloperoxidase (MPO).” The data highlight interim results from ReAlta’s Phase 2 clinical trial, an open-label, prospective, dose-escalation study evaluating pegtarazimod in hospitalized patients with steroid-refractory aGvHD, underscoring the program’s continued clinical momentum.

The study design features multiple cohorts exploring different dosing strategies, including a treatment optimization cohort of 6 patients receiving 10mg/kg of pegtarazimod with ruxolitinib for 7 days. Preliminary efficacy of pegtarazimod was evaluated by clinical response in the lower GI, skin and liver, and characterized as Grade II, III or IV, per Mount Sinai Acute GVHD International Consortium (MAGIC) guidelines. Plasma levels of MPO were evaluated as a pharmacodynamic (PD) measurement of drug activity in a qualified assay.

Key Highlights from ASH 2025 Poster Presentation:

Clinical MAGIC Stage improvement observed for 6 of 6 patients with steroid-refractory aGvHD, with 4 of 5 participants demonstrating lower GI MAGIC Stage improvement of 1, 2, or 3 stages over 7 days of treatment
Median plasma MPO levels decreased by 68% (p = 0.02), suggesting modulation of neutrophil-driven inflammatory mechanisms
Safety profile characterized by no dose-limiting toxicities or serious adverse events attributed to the study drug

“Pegtarazimod treatment shows compelling clinical improvements for patients with steroid-refractory aGvHD, particularly in lower gastrointestinal involvement,” said Robert Zeiser, M.D., from the Medical Center, University of Freiburg and lead author of the study. “The statistically significant decrease in plasma myeloperoxidase levels suggests a direct pathway to reducing tissue damage and potentially improving outcomes for patients with this life-threatening condition.”

Kenji Cunnion, M.D., Chief Medical Officer of ReAlta, said, “This data demonstrates pegtarazimod’s unique ability to target multiple inflammatory pathways simultaneously, particularly in lower gastrointestinal aGvHD, a condition with historically poor patient outcomes. By selectively inhibiting complement activation and neutrophil-driven mechanisms, we are exploring a novel therapeutic approach that could transform the clinical management of this complex and life-threatening disease.”

About Pegtarazimod (RLS-0071)

Pegtarazimod (RLS-0071) is a pipeline-in-a-product that represents a paradigm shift in treating immune-mediated inflammatory disease. Pegtarazimod inhibits C1 and MBL activation within the classical and lectin pathways to block the initiation of the inflammatory cascade at its earliest stages, while simultaneously blocking myeloperoxidase (MPO) in order to prevent the activation of toxic reactive oxidative species (ROS) and formation of neutrophil extracellular traps (NETs) that are known to accelerate tissue damage.

With demonstrated biomarker validation across neurology, immunology and pulmonology, pegtarazimod is currently advancing through multiple Phase 2 clinical trials, including ongoing studies in newborns with moderate to severe hypoxic ischemic encephalopathy (HIE) and patients with steroid-refractory acute graft-versus-host disease (aGvHD), along with a recently completed Phase 2 proof-of-concept study in hospitalized patients with acute exacerbations of chronic obstructive pulmonary disease (COPD).

ReAlta has secured multiple FDA Orphan Drug and Fast Track Designations for pegtarazimod in HIE and aGvHD, as well as European Medicines Agency Orphan Drug Designations for both indications, positioning ReAlta to deliver transformative outcomes for patients facing life-threatening inflammatory conditions with no adequate treatment options.

About ReAlta Life Sciences

ReAlta Life Sciences is clinical-stage biopharmaceutical company redefining the treatment possibilities for patients with devastating and historically untreatable inflammation-driven diseases by developing first-in-class tailored peptides designed to achieve life-changing outcomes. The company’s lead candidate, pegtarazimod (RLS-0071) is a next-generation dual-targeting intervention that represents a potential solution that rebalances the body’s inflammatory response and prevents tissue damage by selectively blocking both complement- and neutrophil-mediated pathways at the outset, disrupting inflammation at the core. To learn more about ReAlta, visit https://realtalifesciences.com and follow us on LinkedIn.

Contacts

Investors
John Rickman

Chief Financial Officer

jrickman@realtals.com

Media
Harrison Wong

ICR Healthcare

ReAltaPR@icrhealthcare.com

Genetix Participates in the American Society of Hematology’s (ASH) Exclusive Press Program & Shares Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation

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Genetix Participates in the American Society of Hematology’s (ASH) Exclusive Press Program & Shares Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation

Genetix Participates in the American Society of Hematology’s (ASH) Exclusive Press Program & Shares Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation

Data presented was selected for ASH’s exclusive program to highlight the most impactful research submitted for this year’s meeting
Real-world data reinforces the value of durable, one-time gene therapy
369 patients enrolled and 115 treated since FDA approval of ZYNTEGLO™ and LYFGENIA™
Lessons learned from ZYNTEGLO launch informed Genetix’s revamped commercial strategy and ongoing infrastructure expansion driving LYFGENIA adoption

SOMERVILLE, Mass.–(BUSINESS WIRE)–Genetix Biotherapeutics Inc. today announced their participation in the American Society of Hematology’s (ASH) exclusive press program ahead of the 67^th ASH annual meeting in Orlando, Florida (December 6-9, 2025). Genetix’s abstract Accelerating access to gene therapy: Lessons from commercial implementation in sickle cell disease and transfusion-dependent thalassemia was selected by the ASH Program Committee as one of the meeting’s most impactful datasets submitted this year.

“We’re honored to be selected for ASH’s exclusive program, a recognition that underscores the significance of our data, which is the first to analyze commercial gene therapy adoption for the treatment of hemoglobinopathies,” said Joanne Lager, MD, Chief Medical Officer. “These findings from patients prescribed ZYNTEGLO and LYFGENIA demonstrate the growing demand and scalability of our durable one-time therapies, setting a new benchmark for delivering commercial gene therapy to patients seeking curative therapies. Building on lessons learned from our first launch of ZYNTEGLO, we’ve accelerated access to LYFGENIA for sickle cell patients, shortening timelines and expanding treatment availability. While initial progress has been made, the vast majority of the thousands of individuals who would benefit from receiving LYFGENIA have not yet been treated. Genetix remains determined and committed to invest in its commercial infrastructure throughout the U.S. to further expand access to LYFGENIA to those in need.”

Since FDA approval of ZYNTEGLO (August, 2022) and LYFGENIA (December, 2023), nearly 370 patients have initiated the treatment journey of which 115 have been treated as of mid-November. Lessons learned from the ZYNTEGLO launch along with Genetix’s revamped commercial strategy and ongoing expansion of infrastructure is enabling faster, broader access to LYFGENIA for patients with sickle cell disease.

LYFGENIA Commercial Launch Metrics Compared to ZYNTEGLO

2x faster lead time from FDA approval to first patient enrollment
167% faster enrollment rates in the first quarter post approval
6x more treatment center activations in the first quarter post approval

Genetix will present the full dataset in an oral presentation at the annual ASH meeting. See details below.

Abstract Title: Accelerating Access to Gene Therapy: Lessons from Commercial Implementation in Sickle Cell Disease and Transfusion-Dependent Thalassemia (Abstract #948)

Date & Time: Monday, December 8^th at 4:00pm EST

Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite.

About LYFGENIA™ (lovotibeglogene autotemcel)

LYFGENIA is a one-time ex-vivo lentiviral vector (LVV) gene addition therapy approved for eligible patients with sickle cell disease, in which a functional β-globin gene is added to patients’ own hematopoietic (blood) stem and progenitor cells (HSPCs). This addition results in the production of adult hemoglobin with anti-sickling properties (HbA^T87Q) which has a similar oxygen-binding affinity to wild-type HbA. LYFGENIA has been shown to limit sickling of red blood cells and reduce or eliminate vaso-occlusive events (VOEs).

LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of VOEs.

Limitations of Use

Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions.

IMPORTANT SAFETY INFORMATION FOR LYFGENIA (lovotibeglogene autotemcel)

Boxed WARNING: HEMATOLOGIC MALIGNANCY

Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted.

Hematologic Malignancy

Hematologic malignancy has occurred in patients treated with LYFGENIA (Study 1, Group A). At the time of initial product approval, two patients treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A) developed acute myeloid leukemia (AML). One patient with α-thalassemia trait (Study 1, Group C) has been diagnosed with myelodysplastic syndrome (MDS).

The additional hematopoietic stress associated with mobilization, conditioning, and infusion of LYFGENIA, including the need to regenerate the hematopoietic system, may increase the risk of a hematologic malignancy. Patients with sickle cell disease have an increased risk of hematologic malignancy as compared to the general population.

Patients treated with LYFGENIA may develop hematologic malignancies and should have lifelong monitoring. Monitor for hematologic malignancies with a complete blood count (with differential) at least every 6 months for at least 15 years after treatment with LYFGENIA, and integration site analysis at Months 6, 12, and as warranted.

In the event that a malignancy occurs, contact Genetix Biotherapeutics at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for testing.

Post-Marketing Long Term Follow-Up Study: Patients who intend to receive treatment with LYFGENIA are encouraged to enroll in the study, as available, to assess the long-term safety of LYFGENIA and the risk of malignancies occurring after treatment with LYFGENIA by calling Genetix Biotherapeutics at 1-833-999-6378. The study includes monitoring (at pre-specified intervals) for clonal expansion.

Delayed Platelet Engraftment

Delayed platelet engraftment has been observed with LYFGENIA. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia. Two patients (4%) required more than 100 days post treatment with LYFGENIA to achieve platelet engraftment.

Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise.

Neutrophil Engraftment Failure

There is a potential risk of neutrophil engraftment failure after treatment with LYFGENIA. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 109 cells/L obtained on different days by Day 43 after infusion of LYFGENIA. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with LYFGENIA, provide rescue treatment with the back-up collection of CD34+ cells.

Insertional Oncogenesis

There is a potential risk of lentiviral vector-mediated insertional oncogenesis after treatment with LYFGENIA.

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of LYFGENIA. The dimethyl sulfoxide (DMSO) or dextran 40 in LYFGENIA may cause hypersensitivity reactions, including anaphylaxis.

Anti-retroviral Use

Patients should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization and until all cycles of apheresis are completed. There are some long-acting anti-retroviral medications that may require a longer duration of discontinuation for elimination of the medication. If a patient is taking anti-retrovirals for HIV prophylaxis, confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells.

Hydroxyurea Use

Patients should not take hydroxyurea for at least 2 months prior to mobilization and until all cycles of apheresis are completed. If hydroxyurea is administered between mobilization and conditioning, discontinue 2 days prior to initiation of conditioning.

Iron Chelation

Drug-drug interactions between iron chelators and the mobilization process and myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of mobilization or conditioning. Do not administer myelosuppressive iron chelators (e.g., deferiprone) for 6 months post-treatment with LYFGENIA. Non-myelosuppressive iron chelation should be restarted no sooner than 3 months after LYFGENIA infusion. Phlebotomy can be used in lieu of iron chelation, when appropriate.

Interference with PCR-based Testing

Patients who have received LYFGENIA are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a possible false-positive PCR assay test result for HIV. Therefore, patients who have received LYFGENIA should not be screened for HIV infection using a PCR-based assay.

Adverse Reactions

The most common adverse reactions ≥ Grade 3 (incidence ≥ 20%) were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia.

Three patients died during LYFGENIA clinical trials; one from sudden cardiac death due to underlying disease and two from acute myeloid leukemia who were treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A).

Pregnancy/Lactation

Advise patients of the risks associated with myeloablative conditioning agents, including on pregnancy and fertility.

LYFGENIA should not be administered to women who are pregnant, and pregnancy after LYFGENIA infusion should be discussed with the treating physician.

LYFGENIA is not recommended for women who are breastfeeding, and breastfeeding after LYFGENIA infusion should be discussed with the treating physician.

Females and Males of Reproductive Potential

A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before LYFGENIA administration.

Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of LYFGENIA.

Advise patients of the options for fertility preservation.

Please see full Prescribing Information, including Boxed WARNING and Medication Guide for LYFGENIA.

About ZYNTEGLO™ (betibeglogene autotemcel)

ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene addition therapy approved for eligible patients with transfusion-dependent beta-thalassemia who require regular red blood cell (RBC) transfusions, in which functional copies of a modified form of the beta-globin gene (β^A-T87Q-globin gene) is added into a patient’s own HSPCs. This addition results in the production of adult hemoglobin (HbA^T87Q) enabling total hemoglobin to reach normal or near normal levels. ZYNTEGLO has been shown to eliminate the need for regular RBC transfusions.

ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions.

IMPORTANT SAFETY INFORMATION FOR ZYNTEGLO (betibeglogene autotemcel)

Delayed Platelet Engraftment

Delayed platelet engraftment has been observed with ZYNTEGLO treatment. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia; 15% of patients had ≥ Grade 3 decreased platelets on or after Day 100.

Risk of Neutrophil Engraftment Failure

There is a potential risk of neutrophil engraftment failure after treatment with ZYNTEGLO. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 500 cells/microliter obtained on different days by Day 43 after infusion of ZYNTEGLO. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with ZYNTEGLO, provide rescue treatment with the back-up collection of CD34+ cells.

Risk of Insertional Oncogenesis

There is a potential risk of lentiviral vector (LVV)-mediated insertional oncogenesis after treatment with ZYNTEGLO.

Patients treated with ZYNTEGLO may develop hematologic malignancies and should be monitored lifelong. Monitor for hematologic malignancies with a complete blood count (with differential) at Month 6 and Month 12 and then at least annually for at least 15 years after treatment with ZYNTEGLO, and integration site analysis at Months 6, 12, and as warranted.

In the event that a malignancy occurs, contact Genetix Biotherapeutics at 1 833-999-6378 for reporting and to obtain instructions on collection of samples for testing.

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of ZYNTEGLO. The dimethyl sulfoxide (DMSO) in ZYNTEGLO may cause hypersensitivity reactions, including anaphylaxis.

Anti-retroviral and Hydroxyurea Use

Patients should not take prophylactic HIV anti-retroviral medications or hydroxyurea for at least one month prior to mobilization, or for the expected duration for elimination of the medications, and until all cycles of apheresis are completed. If a patient requires anti-retrovirals for HIV prophylaxis, then confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells.

Interference with Serology Testing

Patients who have received ZYNTEGLO are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a false-positive test for HIV. Therefore, patients who have received ZYNTEGLO should not be screened for HIV infection using a PCR-based assay.

Adverse Reactions

The most common non-laboratory adverse reactions (≥20%) were mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus. The most common Grade 3 or 4 laboratory abnormalities (>50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.

Drug Interactions

Drug-drug interactions between iron chelators and the myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of conditioning. The prescribing information for the iron chelator(s) and the myeloablative conditioning agent should be consulted for the recommendations regarding co-administration with CYP3A substrates.

Some iron chelators are myelosuppressive. After ZYNTEGLO infusion, avoid use of these iron chelators for 6 months. If iron chelation is needed, consider administration of non-myelosuppressive iron chelators. Phlebotomy can be used in lieu of iron chelation, when appropriate.

Pregnancy/Lactation

Advise patients of the risks associated with conditioning agents, including on pregnancy and fertility.

ZYNTEGLO should not be administered to women who are pregnant, and pregnancy after ZYNTEGLO infusion should be discussed with the treating physician.

ZYNTEGLO is not recommended for women who are breastfeeding, and breastfeeding after ZYNTEGLO infusion should be discussed with the treating physician.

Females and Males of Reproductive Potential

A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before ZYNTEGLO administration.

Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of ZYNTEGLO.

Advise patients of the option to cryopreserve semen or ova before treatment if appropriate.

Please see full Prescribing Information and Patient Information for ZYNTEGLO.

About Genetix Biotherapeutics

Genetix Biotherapeutics Inc. is a privately-held, commercial-stage biotechnology company dedicated to delivering genetic therapies for patients with severe rare diseases. Backed by more than 30 years of pioneering genetic therapy innovation, the company has FDA-approved treatments for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy that directly treat the underlying cause of disease. Genetix is committed to commercial execution, scaling patient access, and improving the treatment experience for patients and providers. Genetix is headquartered in Somerville, Massachusetts.

Contacts

For media inquiries:
info@GenetixBioTx.com

Website: www.GenetixBioTx.com

Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions

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Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions

Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions

– Data from pivotal studies of CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrates the transformative potential of the therapy in younger patients –

– Efficacy and safety data in children 5-11 years are consistent with the durable and positive benefit/risk profile established from clinical studies in patients 12 years of age and older –

– Vertex expects to initiate global regulatory submissions for CASGEVY in children 5-11 years in 1H 2026 –

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical benefits of CASGEVY^® (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, including the first presentation of clinical data from pivotal studies in children ages 5-11 years, and longer-term data from the pivotal studies of people with severe SCD and TDT ages 12 years and older, will be presented at the American Society of Hematology (ASH) Annual Meeting. CASGEVY is currently approved for eligible people ages 12 years and older with SCD or TDT in the United States, Great Britain, the European Union, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Kuwait, Qatar, Canada, Switzerland and the United Arab Emirates.

“These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the transformative potential of CASGEVY,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With dosing completed in the 5-11 age group and the Commissioner’s National Priority Voucher for CASGEVY in this population in hand, we are excited to begin global regulatory filings in the first half of next year and bring this potentially transformative therapy to eligible children as soon as possible.”

“As an investigator in the clinical program for patients 12 years and older and after having real-world experience with CASGEVY as an early commercial treatment center, I have seen firsthand the transformative impact this therapy has had on older patients with SCD or TDT. I am excited to hopefully be able to offer this option to my younger patients soon, early in life, before some of the most devastating impacts of these diseases begin,” said Haydar Frangoul, M.D., M.S., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute and HCA Healthcare’s TriStar Centennial Children’s Hospital, Member of Vertex’s SCD Program Steering Committee, and presenting author of the 5-11 years old CASGEVY data at ASH.

First presentation of data in children ages 5-11 years treated with CASGEVY

In children with SCD, 11 patients have been dosed with CASGEVY in the Phase 3 CLIMB-151 clinical study, and all (4/4) patients with sufficient follow-up achieved the primary endpoint of being free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12).
- No patient experienced a VOC following infusion with CASGEVY, with the longest duration of VOC-free of approximately two years (range 3.2–24.1 months).
In children with TDT, 13 patients have been dosed with CASGEVY in the Phase 3 CLIMB-141 clinical study, and all (6/6) patients with sufficient follow-up achieved the primary endpoint of transfusion independence for at least 12 consecutive months while maintaining a weighted average hemoglobin (Hb) of at least 9 g/dL (TI12).
- Following CASGEVY infusion, 12/13 are transfusion free, with the longest duration of transfusion free just under two years (range 2.3–22.5 months).
- One patient died from pneumonia in the setting of multi-organ failure due to severe veno-occlusive disease related to the busulfan conditioning.
The safety profile of CASGEVY in younger patients is consistent with myeloablative conditioning and autologous transplant in both SCD and TDT, as established in clinical studies in older patients.
Consistent with studies in older patients, children treated with CASGEVY have durable increases in fetal hemoglobin (HbF) and stable allelic editing.

Longer-term data for people with SCD and TDT ages 12 years and older treated with CASGEVY

New longer-term data from the pivotal clinical studies of CASGEVY in people 12 years and older will also be presented at ASH. These data, as of April 2025, continue to demonstrate the transformative, durable clinical benefits that CASGEVY provides to people living with SCD or TDT. In SCD, 100% of patients (45/45) achieved VF12 in either CLIMB-121 or the long-term follow-up study CLIMB-131, with a mean duration of VOC-free for 35.3 months (range 12.9–67.7 months). In TDT, 98.2% (55/56) achieved TI12 in either CLIMB-111 or CLIMB-131 with a mean duration of transfusion independence of 41.4 months (range 13–72.3 months). The safety profile remained consistent with myeloablative conditioning and autologous transplant in both SCD and TDT.

About Sickle Cell Disease (SCD)

SCD is a debilitating, progressive and life-shortening disease. It is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires a lifetime of treatment and results in a reduced life expectancy. In the U.S., the median age of death for patients living with SCD is approximately 45 years. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the U.S. of managing SCD for patients with recurrent VOCs is estimated between $4 and $6 million.

About Transfusion-Dependent Beta Thalassemia (TDT)

TDT is a serious, life-threatening genetic disease. It requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In the U.S., the median age of death for patients living with TDT is 37 years. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the U.S. of managing TDT are estimated between $5 and $5.7 million.

About CASGEVY^® (exagamglogene autotemcel)

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.

The use of CASGEVY in children ages 5-11 years is investigational.

About the CLIMB Studies

The Phase 1/2/3 open-label studies, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12-35 years with TDT or with SCD and recurrent VOCs. Patients will be followed for approximately two years after CASGEVY infusion in these studies. CLIMB-141 and CLIMB-151 are ongoing Phase 3 open-label studies, designed to assess the safety and efficacy of a single dose of exagamglogene autotemcel in patients ages 2-11 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5-11-years-old cohort in both studies with the plan to extend to ages 2-4 years.

Each patient will be asked to participate in the ongoing long-term, open-label study, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients with up to 15 years of follow up after CASGEVY infusion.

Next steps for CASGEVY in children ages 5-11 years

Enrollment and dosing are complete for the 5-11 years cohort in both studies. Vertex expects to initiate global regulatory filings for this age group, including a supplemental Biologics License Application (sBLA) in the U.S., in the first half of next year. Vertex recently received a Commissioner’s National Priority Voucher for CASGEVY in the 5-11 years age group from the U.S. Food and Drug Administration to accelerate the review of the sBLA once submitted. Products under the program will be subject to a 1–2-month review clock from the start of FDA’s review and will also benefit from enhanced communication opportunities with the agency.

U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY

WHAT IS CASGEVY?

CASGEVY is a one-time therapy used to treat people ages 12 years and older with:

sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs
beta thalassemia (β-thalassemia) who need regular blood transfusions

CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about CASGEVY?

After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels.

Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of platelet cells:
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood

Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of white blood cells:
- fever
- chills
- infections

You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects.

How will I receive CASGEVY?

Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment.

After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment.

STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week.

During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY.

STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider.

STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY.

STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time.

After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home.

What should I avoid after receiving CASGEVY?

Do not donate blood, organs, tissues, or cells at any time in the future

What are the possible or reasonably likely side effects of CASGEVY?

The most common side effects of CASGEVY include:

Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding
Low levels of white blood cells, which may make you more susceptible to infection

Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms:

fever
chills
infections
severe headache
abnormal bruising
prolonged bleeding
bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood

These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

General information about the safe and effective use of CASGEVY

Talk to your healthcare provider about any health concerns.

Please see full Prescribing Information including Patient Information for CASGEVY.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 16 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X.

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Carmen Bozic, M.D., and Haydar Frangoul, M.D., M.S., and statements regarding expectations for the clinical benefits of CASGEVY, plans to initiate global regulatory submissions for children 5-11, including in the U.S., in the first half of 2026, expectations that the use of a Priority Voucher will accelerate the review of the sBLA, expectations for the design of the CLIMB studies, including plans to follow patients after infusion, expectations that each patient will be asked to participate in the CLIMB-131 study and expectations that the studies will be extended to children 2-4 years of age. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s research and development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, that the company may be unable to make the anticipated regulatory submissions on the expected timeline, or at all, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company’s website at www.vrtx.com. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contacts

Vertex Pharmaceuticals Incorporated

Investors:

InvestorInfo@vrtx.com or

+1 617-341-6108

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Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting

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Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting

Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting

—Long-term Phase 1/2a and open label extension (OLE) data for zorevunersen on top of standard of care anti-seizure medicines (ASMs) demonstrate durable seizure reductions, including increases in seizure-free days, in addition to improvements in cognition, behavior and quality of life—

—Propensity score weighted analysis comparing the effects of zorevunersen to natural history showed reductions in seizures and improvements in cognition and behavior with dose levels and timepoints similar to and consistent with the ongoing Phase 3 EMPEROR study—

—Analysis of electroencephalogram (EEG) supports a disease-modifying mechanism of action—

BEDFORD, Mass. & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB) today announced data presentations that further support the potential of zorevunersen, an investigational antisense oligonucleotide, as a disease-modifying medicine for Dravet syndrome. These data were presented at the 2025 American Epilepsy Society (AES) Annual Meeting in Atlanta, Georgia.

Long-term results from the ongoing Phase 1/2a and open label extension (OLE) studies demonstrated durable seizure reductions, including increases in seizure-free days, in addition to improvements in cognition, behavior and quality of life in patients treated with zorevunersen on top of standard of care anti-seizure medicines (ASMs). A new propensity score weighted analysis provides the first direct comparison between patients with Dravet syndrome treated with zorevunersen and a matched cohort from the BUTTERFLY natural history study and showed reductions in seizures and improvements in cognition and behavior with dose levels and timepoints similar to and consistent with the ongoing Phase 3 EMPEROR study.

“Over the last four years we have gained an increasing appreciation for the potential to change the course of Dravet syndrome with new disease-modifying medicines. Open-label data from the zorevunersen clinical studies have been highly encouraging, bringing hope and anticipation to the Dravet community,” said M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center. “In addition, the new propensity score weighted analysis comparing zorevunersen treatment to patients treated with the current standard of care gives us further context for the improvements we’re seeing in patients living with this devastating disease.”

Propensity Score Weighted Analysis

A propensity score weighted analysis, designed to mimic randomization by adjusting for baseline differences between treated and untreated (natural history) patient groups, showed patients receiving two loading doses of zorevunersen (70mg) experienced statistically significant reductions in major motor seizure frequency at six months compared to natural history. Six months is consistent with the Week 28 Phase 3 primary endpoint measuring the effects of zorevunersen on seizure frequency. With continued dosing at 45mg, improvements in five key assessments of cognition and behavior as measured by Vineland-3 were shown at 18 months, with several reaching statistical significance. At the 18-month timepoint, cumulative dosing is similar to and consistent with a key secondary endpoint in the Phase 3 EMPEROR study. Durable effects were demonstrated through 24 months, the longest evaluable timepoint in the BUTTERFLY natural history study.

“Data from this analysis, which allows us to match patients treated with zorevunersen directly to natural history, add to a growing body of information shaping our understanding of Dravet syndrome and the effects of zorevunersen over time,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “As we continue to progress our Phase 3 EMPEROR program and await safety and efficacy data from this larger, sham-controlled study, results presented at AES give us additional confidence in what zorevunersen may one day offer to patients and their families.”

“In addition to these important efficacy findings, we are also encouraged by the accumulating long-term safety data, with some patients treated with up to 15 doses over more than four years,” said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. “The totality of clinical data, in addition to the new EEG findings, demonstrates zorevunersen’s potential to restore protein function and address the underlying cause of Dravet syndrome to improve debilitating symptoms like seizures and cognitive impairment.”

Electroencephalogram (EEG) Results

Data from an analysis of EEGs performed in patients treated with zorevunersen highlighted the dose-dependent effects of zorevunersen in decreasing abnormal brain activity that is persistently higher in patients with Dravet syndrome. The analysis also showed that a reduction in abnormal EEG activity in the brain was associated with an increased probability of achieving a meaningful reduction in seizure frequency.

Summary of Zorevunersen Safety Data

Eighty-one patients received at least one dose of zorevunersen and have been evaluated for safety, and more than 800 doses have been administered to date. Zorevunersen has been generally well tolerated across the Phase 1/2a and OLE studies. Study drug related treatment emergent adverse events (TEAEs) were observed in 30% (24/81) and 53% (40/75) of patients treated in the Phase 1/2a and OLE studies, respectively. The most common study drug related TEAE was CSF protein elevations reported in 14% (11/81) of patients in the Phase 1/2a studies and 45% (33/75) of patients in the OLE studies. CSF protein elevations (>50 mg/dL) occurred in 42% (34/81) of patients in the Phase 1/2a studies and 86% (62/72) of patients in the OLE studies. No related clinical manifestations have been observed although one patient discontinued treatment due to elevated CSF protein levels. Treatment-emergent serious adverse events (TESAEs) were reported in 22% (18/81) and 29% (22/75) of patients in the Phase 1/2a and OLE studies, respectively, all of which were assessed to be unrelated to zorevunersen except one patient who experienced SUSARs. Three patient deaths have been reported across the Phase 1/2a and OLE studies, two from sudden unexpected death in epilepsy (SUDEP) and one from malnutrition. All were unrelated to zorevunersen.

Details of the AES Presentations:

Title: Zorevunersen Continues to Demonstrate Potential as a Disease-modifying Therapy in Long-term Open-label Extension Studies of Patients with Dravet Syndrome

Oral Presentation Date & Time: Friday, December 5, 3:30-5:55 PM EST

Oral Presenter: M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center

Title: Zorevunersen Demonstrates Disease-modifying Potential in Patients with Dravet Syndrome with Increases in Seizure-free Days, Improvements in Quality of Life, and Benefits in Overall Clinical Status

Poster Presentation Date & Time: Saturday, December 6, 12:00-2:00 PM EST

Poster Presenter: Kelly Knupp, M.D., MSCS, Professor of Pediatrics and Neurology at the University of Colorado Anschutz and the Dravet Program Director and Epilepsy Program Lead at Children’s Hospital Colorado

Poster Number: 1.379

Title: Zorevunersen Continues to Demonstrate Potential as a Disease-modifying Therapy in Long-term Open-label Extension Studies of Patients with Dravet Syndrome

Poster Presentation Date & Time: Sunday, December 7, 12:00-2:00 PM EST

Poster Presenter: M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center

Poster Number: 2.341

Title: Spectral Electroencephalogram Abnormalities Across Development in Patients with Dravet Syndrome

Poster Presentation Date & Time: Sunday, December 7, 12:00-2:00 PM EST

Poster Presenter: Pieter van Mierlo, Founder and Chief Scientific Officer, Epilog, Clouds of Care, Associate Professor, Ghent University

Poster Number: 2.432

Title: Electrophysiological Improvements in Patients with Dravet Syndrome Following Treatment with Zorevunersen, an Investigational Antisense Oligonucleotide

Poster Presentation Date & Time: Monday, December 8, 12:00-1:45 PM EST

Poster Presenter: Nigel Colenbier, Senior Data Scientist, Epilog, Clouds of Care

Poster Number: 3.489

About Dravet Syndrome

Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. Even when treated with the best available anti-seizure medicines (ASMs), up to 57 percent of patients with Dravet syndrome do not achieve ≥50 percent reduction in seizure frequency. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent of children and adolescents with Dravet syndrome die before adulthood due to SUDEP, prolonged seizures, seizure-related accidents or infections¹. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan.²There are no approved disease-modifying therapies for people living with Dravet syndrome.

About Zorevunersen

Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.

About the EMPEROR Study

The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety, and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain-of-function. The trial is currently enrolling patients within the United States and is expected to enroll participants across Japan, United Kingdom and European Union, with participants being randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. Following the completion of the study, eligible participants will be offered ongoing treatment with zorevunersen as part of an OLE study. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), and the Bayley Scales of Infant Development (BSID-IV). For more information, visit https://www.emperorstudy.com/.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.

About Biogen

Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Facebook, LinkedIn, X, YouTube.

Stoke Therapeutics Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the potential benefits, safety and efficacy of zorevunersen; the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations; and the participation of scientists associated with Stoke making presentations at AES 2025 and the presentation of data at AES 2025. Statements including words such as “plan,” “potential,” “will,” “continue,” “expect,” or similar words and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke’s results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stokes ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, Stoke would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke’s ability to protect its intellectual property; Stoke’s ability to fund development activities and achieve development goals to mid-2028; and the other risks and uncertainties described under the heading “Risk Factors” in its Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Biogen Safe Harbor

This news release contains forward-looking statements, including, among others, relating to: the potential clinical effects of zorevunersen; the potential for zorevunersen to improve outcomes and for patients of Dravet syndrome; the potential benefits, safety and efficacy of zorevunersen and continued treatment with zorevunersen; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of Dravet syndrome; the anticipated benefits, risks and potential of Biogen’s collaboration arrangements with Stoke Therapeutics; the potential of Biogen’s commercial business and pipeline programs, including zorevunersen; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would” or the negative of these words or other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements.

These forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to differ materially from those stated or implied in this document, including, among others, uncertainty of our long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans, prospects and timing of actions relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third-parties, intellectual property, competitive and market challenges and regulatory compliance; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; and the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission, which are available on the SEC’s website at www.sec.gov.

These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.

Digital Media Disclosure

From time to time, we have used, or expect in the future to use, our investor relations website (investors.biogen.com), the Biogen LinkedIn account (linkedin.com/company/biogen-) and the Biogen X account (https://x.com/biogen) as a means of disclosing information to the public in a broad, non-exclusionary manner, including for purposes of the SEC’s Regulation Fair Disclosure (Reg FD). Accordingly, investors should monitor our investor relations website and these social media channels in addition to our press releases, SEC filings, public conference calls and websites, as the information posted on them could be material to investors.

References:

Symonds, J. et al. Early childhood epilepsies: epidemiology, classification, aetiology, and socio-economic determinants. Brain. 2021;144(9):2879-2891.
Based on Stoke Therapeutics’ preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality.

Contacts

Stoke Media & Investor Contacts:
Susan Willson

Vice President, Corporate Communications

swillson@stoketherapeutics.com
415-509-8202

Doug Snow

Director, Communications & Investor Relations

IR@stoketherapeutics.com
508-642-6485

Biogen Media Contact:
Madeleine Shin

Public.affairs@biogen.com
+ 1 781 464 3260

Biogen Investor Contact:
Tim Power

IR@biogen.com
+1 781 464 2442

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Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial

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Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial

Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial

– AMX0114 was generally well-tolerated, with no treatment-related serious adverse events

– Amylyx will proceed with opening enrollment of second cohort

– Data are being presented at the 36^th International Symposium on ALS/MND

CAMBRIDGE, Mass.–(BUSINESS WIRE)–$AMLX—Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the presentation of early safety and tolerability data from its Phase 1 LUMINA trial of AMX0114 and results from ongoing work characterizing biomarkers of AMX0114 target engagement at the 36^th International Symposium on ALS/MND (MNDA) held from December 5-7 in San Diego, California. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in cohort 1 (n=12), with no treatment-related serious adverse events (SAEs). Based on these data, Amylyx expects to begin enrolling the second cohort of participants in Canada later this month and in the U.S. in January.

“LUMINA is a first-in-human study, and we are encouraged by these data as we continue to advance AMX0114 as a potential treatment for this rapidly progressive disease with high unmet need. The safety and tolerability analysis allows LUMINA to proceed with its next cohort of participants, which is critical given that this community has no time to wait,” said Sabrina Paganoni, MD, PhD, principal investigator of the LUMINA trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, and member of the Scientific Advisory Board of the Network of Excellence for ALS (NEALS).

The LUMINA trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 – an investigational, potent antisense oligonucleotide (ASO) targeting calpain-2 – in people living with amyotrophic lateral sclerosis (ALS). LUMINA is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS and assessing both novel and broadly researched ALS biomarkers, including change from baseline in neurofilament light chain (NfL) levels. Amylyx expects biomarker data from the first cohort of LUMINA participants (n=12) in the first half of 2026.

“We appreciate the partnership with LUMINA sites and participants to achieve complete enrollment of the first cohort. In addition, we are pleased that to date no drug-related SAEs or dose-limiting toxicities were observed, which represent important early steps in this study,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “AMX0114 is designed to inhibit calpain-2, a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS. Preclinical studies have demonstrated that treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in calpain-2 protein levels, translating to improved neuronal survival and reductions in extracellular NfL levels. We look forward to presenting cohort 1 biomarker data at a medical meeting in the first half of next year.”

The MNDA presentation details are as follows:

Friday, December 5, 2025, Session A, 5:30-7pm Pacific Time:

Title: A Phase 1, Multicenter, Randomized, Placebo-Controlled Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AMX0114 in Amyotrophic Lateral Sclerosis (LUMINA)

Presenting Author: Sabrina Paganoni, MD, PhD, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Spaulding Rehabilitation Hospital, Harvard Medical School

Poster Number: CLT-14

Saturday, December 6, 2025, Session 6B: Biomarkers, 10:50-11:10am Pacific Time:

Title: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS

Presenting Author: Robert Bowser, PhD, Barrow Neurological Institute and nVector, Inc.

For MNDA conference information, visit https://symposium.mndassociation.org/. Abstracts have been published as an online open access supplement of Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. The presentations will be available on the “Events & Presentations” tab of the Amylyx website.

About AMX0114

AMX0114 is an investigational antisense oligonucleotide (ASO) with U.S. Food and Drug Administration (FDA) Fast Track designation for the potential treatment of ALS. AMX0114 targets calpain-2 (CAPN2), a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in cohort 1 (n=12), with no treatment-related serious adverse events (SAEs).

About LUMINA

The Phase 1 LUMINA clinical trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS. LUMINA will also assess change from baseline in calpain-2 levels, neurofilament light (NfL) levels, and other pharmacodynamic biomarkers of ALS. LUMINA is anticipated to enroll approximately 48 adult participants. Participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks for a total of up to 4 doses. Study sites are active in Canada and the United States, and the first cohort has been fully enrolled. Amylyx expects to begin enrolling the second cohort of participants in Canada in December and in the U.S. in January.

About ALS

Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. One of the ways ALS progresses is through axonal degeneration, which disrupts neural connectivity and contributes significantly to disease pathology. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history.

About Amylyx Pharmaceuticals

At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’ expectations regarding: the potential for AMX0114 as a treatment for ALS, the expected timeline for enrollment of future cohorts, and the expected timeline for data readout. Any forward-looking statements in this press release and related comments in the Company’s earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities; Amylyx’ ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; the risk that early-stage results may not reflect later-stage results; Amylyx’ ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’ operations, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Contacts

Media
Amylyx Media Team

(857) 799-7274

amylyxmediateam@amylyx.com

Investors
Lindsey Allen

(857) 320-6244

Investors@amylyx.com

Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity

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Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity

Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity

Data to be Presented During an Oral Platform Presentation at the American Epilepsy Society Annual Meeting

SAN DIEGO–(BUSINESS WIRE)–Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced preclinical data related to the company’s co-lead candidate ABS-1230, a selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. The data are being presented today, December 5, in a platform presentation by Actio CSO David Breckenridge, Ph.D., from 3:30-5:45 p.m. at the American Epilepsy Society (AES) Annual Meeting in Atlanta.

The preclinical data support the therapeutic potential of ABS-1230 to reduce seizures by selectively inhibiting mutations of the KCNT1 gene, which cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy. Actio is currently advancing ABS-1230 in a Phase 1a healthy volunteer clinical trial and plans to initiate a Phase 1b/2a clinical trial in patients with KCNT1-related epilepsy in 2026.

“Development of treatments for KCNT1-related epilepsy has been limited by the large number of distinct pathogenic mutations, many of which are resistant to known inhibitors,” said David Goldstein, Ph.D., CEO of Actio. “In our preclinical work, ABS-1230 has consistently demonstrated its ability to potently and selectively inhibit KCNT1 and pathogenic mutations leading to notable reductions in seizures in a mouse model. We’re pleased to share these data today, which support ABS-1230 as a targeted therapy with the potential to address the immense burden and unmet need for patients with KCNT1-related epilepsy. Our Phase 1a trial of ABS-1230 in healthy volunteers is ongoing, and we look forward to reporting data from that study in early 2026 and advancing into a first-in-patient trial next year.”

Presentation Highlights

In preclinical human and mouse models, administration of ABS-1230 led to:

Potent and selective inhibition of KCNT1 and known pathogenic mutations
High oral bioavailability and brain penetration across species
Significant dose-dependent reduction of seizures in a KCNT1 mouse model relative to vehicle control treatment

About ABS-1230

ABS-1230 is designed to be a potent and selective orally available small molecule inhibitor of the KCNT1 ion channel and is initially in clinical development for the treatment of KCNT1-related epilepsy. The KCNT1 gene provides instructions for making potassium channels that allow potassium ions to flux out of cells, especially neurons in the brain. Gain-of-function mutations in the KCNT1 gene are the known genetic cause of KCNT1-related epilepsy — a rare, severe and often fatal form of pediatric epilepsy. The company has initiated a Phase 1a trial of ABS-1230 in healthy adult volunteers. Actio is also conducting preclinical studies to evaluate the therapeutic potential of ABS-1230 in more prevalent genetic epilepsies with related underlying biology. The FDA has granted ABS-1230 Rare Pediatric Disease, Fast Track and Orphan Drug designations for the treatment of KCNT1-related epilepsy.

About KCNT1-related Epilepsy

KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence Actio estimates to be 2,500 patients. KCNT1-related epilepsy patients typically have a normal prenatal course before developing sporadic, asynchronous focal seizures. Seizure onset is often in the first six months of life, and the median age of onset is approximately 3.5 weeks. Seizure frequency tends to increase over time and can reach up to 50 seizures per day (15-20 per day on average). Other common symptoms and complications of KCNT1-related epilepsy include developmental delays and profound cognitive impairments and neurological deficits. There are currently no approved therapies for KCNT1-related epilepsy.

About Actio Biosciences

Actio Biosciences is a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines. The company applies deep expertise in Mendelian genetics, drug discovery and data science to identify targets whose biology underlies rare diseases with high unmet need and holds promise for the treatment of more prevalent indications. By precisely targeting the root causes of rare diseases, Actio generates biological insights to enable expansion of development into more prevalent indications.

The company is advancing two lead clinical-stage programs, ABS-1230, initially being developed for the treatment of KCNT1-related epilepsy, and ABS-0871, initially being developed for the treatment of Charcot-Marie-Tooth disease type 2C including other TRPV4-related neuromuscular disorders (collectively, CMT2C). Actio plans to pursue development of both lead programs in more prevalent indications such as additional genetic epilepsies for ABS-1230 and overactive bladder for ABS-0871. In addition to Actio’s two lead programs, Actio has also launched a third program for a rare genetic epilepsy. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.

Contacts

Contact
Katie Engleman, 1AB

katie@1abmedia.com

Investor Contact:
Renee Leck, THRUST

renee@thrustsc.com

Zoetis Receives Health Canada Approval for Portela™(relfovetmab injection) to Alleviate Pain Associated with Osteoarthritis in Cats

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Zoetis Receives Health Canada Approval for Portela™(relfovetmab injection) to Alleviate Pain Associated with Osteoarthritis in Cats

Zoetis Receives Health Canada Approval for Portela™(relfovetmab injection) to Alleviate Pain Associated with Osteoarthritis in Cats

PARSIPPANY, N.J.–(BUSINESS WIRE)–$ZTS #animalhealth—Zoetis Inc. today announced that Health Canada has approved Portela^™ (relfovetmab injection) for the alleviation of pain associated with osteoarthritis (OA) in cats. Designed to provide three months of OA pain relief with a single injection, Portela targets anti-nerve growth factor (NGF), which is a key mediator of OA pain and inflammation.

In a nine-month field trial in Europe, Portela demonstrated effectiveness in alleviating OA pain and was found to be well tolerated, including by cats identified with kidney disease at IRIS stage 1, 2 or 3¹. Portela is also approved in the European Union (EU), and Zoetis anticipates making Portela commercially available in Canada and the EU in 2026.

“Health Canada’s approval of Portela marks a significant step forward in managing osteoarthritis-related pain in cats,” said Rob Polzer, Ph.D., Executive Vice President and President, Research and Development at Zoetis. “Thanks to Portela’s long-lasting antibody and its unique binding site to NGF, veterinarians and cat owners in Canada now have a new, innovative way to address osteoarthritis pain. This achievement underscores our ongoing dedication to developing new therapies that improve the health and quality of life for companion animals.”

Osteoarthritis as a Significant Unmet Medical Need

OA is a common, chronic and progressive joint disease characterized by the inflammation and breakdown of joints, leading to pain and mobility issues. Although the disease cannot be cured, much can be done to control the associated pain and improve quality of life. Even with up to 40% of all cats having clinical signs of OA² ³, pet owners often overlook these signs, and as a result only 18% of affected cats are diagnosed with OA pain by veterinary professionals⁴. Identifying and treating OA pain is important because chronic pain has a negative impact on many aspects of a cat’s health. In addition to gait and movement, chronic pain impacts sleep, relationships, and cognition⁵. Portela now provides a convenient, long-acting therapy to reduce OA pain – particularly beneficial for cats that are difficult to medicate regularly.

Building on a Legacy of Innovation

Zoetis continues to advance care for animals around the globe with a robust pipeline fueled by lifecycle innovation, geographic expansion and new product innovation. Portela joins Solensia^® (frunevetmab injection) in the company’s growing portfolio of OA pain products for cats. Like Solensia, Portela is a monoclonal antibody that targets nerve growth factor (NGF); however, Portela is designed to alleviate pain associated with OA for a longer period of time by binding to a different site on NGF.

“Portela is a new therapy designed to support long-term comfort and mobility for cats—with the added benefit that pet owners only need to bring their cats in for an injection once every three months. This is a testament to the value we place at Zoetis on meaningful innovation in feline medicine, solving for the unmet needs of our patients and their families,” said Richard Goldstein, DVM, DACVIM, DECVIM-CA, Global Chief Medical Officer and Head of Medical Affairs at Zoetis. “We look forward to providing veterinarians with another innovative tool, adding to our wide feline portfolio, to help them improve the quality of life for the cats in their care.”

About Portela

Portela (relfovetmab injection) is a monoclonal antibody therapy that binds to nerve growth factor to reduce NGF’s effects. Portela is indicated for the alleviation of pain associated with osteoarthritis in cats. Portela is administered subcutaneously once every three months. Portela should not be used in cats with known hypersensitivity to relfovetmab, or to any of the excipients; or in breeding, pregnant or lactating cats, or in cats less than 12 months of age. The following clinical signs have been reported in cats receiving Portela: pain upon injection, skin infection, dermatitis, hair loss, and anorexia.

About Zoetis

As the world’s leading animal health company, Zoetis is driven by a singular purpose: to nurture our world and humankind by advancing care for animals. After innovating ways to predict, prevent, detect, and treat animal illness for more than 70 years, Zoetis continues to stand by those raising and caring for animals worldwide – from veterinarians and pet owners to livestock producers. The company’s leading portfolio and pipeline of medicines, vaccines, diagnostics and technologies make a difference in over 100 countries. A Fortune 500 company, Zoetis generated revenue of $9.3 billion in 2024 with approximately 13,800 employees. For more information, visit www.zoetis.com.

DISCLOSURE NOTICES

Forward-Looking Statements: This press release contains forward-looking statements which reflect the current views of Zoetis with respect to: our business plans or prospects; expectations regarding products, product approvals or licenses, products under development; and other future events. These statements are not guarantees of future performance or actions. Forward-looking statements are subject to risks and uncertainties. If one or more of these risks or uncertainties materialize, or if management’s underlying assumptions prove to be incorrect, actual results may differ materially from those contemplated by a forward-looking statement. Forward-looking statements speak only as of the date on which they are made. Zoetis expressly disclaims any obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. A further list and description of risks, uncertainties and other matters can be found in our most recent Annual Report on Form 10-K, including in the sections thereof captioned “Forward-Looking Statements and Factors That May Affect Future Results” and “Item 1A. Risk Factors,” in our Quarterly Reports on Form 10-Q and in our Current Reports on Form 8-K. These filings and subsequent filings are available online at www.sec.gov, www.zoetis.com, or on request from Zoetis.

All trademarks are the property of Zoetis Services LLC or a related company or a licensor unless otherwise noted.

IRIS stages of kidney disease. Accessed August 21, 2025. https://static1.squarespace.com/static/666b9ecb4064a156963b4162/t/66a6dbc90ca6986e1b5c06bd/1722211273243/2_IRIS_Staging_of_CKD_2023.pdf
Enomoto M, Mantyh PW, Murrell J, Innes JF, Lascelles BDX. Anti-nerve growth factor monoclonal antibodies for the control of pain in dogs and cats. Vet Rec. 2019;184(1):23.
L.I. Slingerland, H.A.W. Hazewinkel, B.P. Meij, Ph. Picavet, G. Voorhout, Cross-sectional study of the prevalence and clinical features of osteoarthritis in 100 cats. The Veterinary Journal, Volume 187, Issue 3, 2011, Pages 304-309.
Solensia Veterinarian A&U MR (MARC 2025)
Lascelles BDX, Brown DC, Conzemius MG, Gill M, Oshinsky ML, Sharkey M. Measurement of chronic pain in companion animals: discussions from the Pain in Animals Workshop (PAW) 2017. Vet J. 2019;250(8):71-78.

ZTS-COR

ZTS-IR

ZTS-CA

ZTS-PS

Contacts

Media Contacts:

Jennifer Albano

862-399-0810 (o)

jennifer.albano@zoetis.com

Laura Panza

973-975-5176 (o)

laura.panza@zoetis.com

Investor Contacts:

Steve Frank

973-822-7141 (o)

steve.frank@zoetis.com

Nick Soonthornchai

973-443-2792 (o)

nick.soonthornchai@zoetis.com

Guardant Health to Present 14 Abstracts Demonstrating the Power of Multiomic Liquid Biopsy Tests in Predicting Outcomes and Tailoring Treatment for Early and Metastatic Breast Cancer at the 2025 San Antonio Breast Cancer Symposium

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Guardant Health to Present 14 Abstracts Demonstrating the Power of Multiomic Liquid Biopsy Tests in Predicting Outcomes and Tailoring Treatment for Early and Metastatic Breast Cancer at the 2025 San Antonio Breast Cancer Symposium

Guardant Health to Present 14 Abstracts Demonstrating the Power of Multiomic Liquid Biopsy Tests in Predicting Outcomes and Tailoring Treatment for Early and Metastatic Breast Cancer at the 2025 San Antonio Breast Cancer Symposium

Presentations highlight advancements in cancer recurrence detection with Guardant Reveal and real-time treatment response monitoring with Guardant360 Liquid

PALO ALTO, Calif.–(BUSINESS WIRE)–$GH–Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, will present a total of 14 abstracts with its research collaborators from multiple studies demonstrating the value of its tissue-free liquid biopsy tests at the 2025 San Antonio Breast Cancer Symposium (SABCS), taking place Dec. 9–12, 2025.

Oral presentations will highlight results generated with Guardant Reveal, the company’s tissue-free test for detecting minimal residual disease (MRD) and Guardant360 Liquid, the company’s comprehensive multiomic profiling test, both leveraging Guardant’s proprietary epigenomic technology powered by the Guardant Infinity Smart Platform. Collectively, these studies demonstrate how blood-based testing can uncover risk, guide treatment selection, and influence long-term patient management across the breast cancer continuum, from early-stage disease through metastatic settings.

Guardant’s MRD presentations reinforce the value of tissue-free circulating tumor DNA (ctDNA) testing for both neoadjuvant treatment response assessment and post-treatment surveillance in early-stage breast cancer. In a retrospective analysis of HER2-positive patients, ctDNA measured with Guardant Reveal demonstrated strong prognostic significance in the neoadjuvant setting, showing a correlation with pathological complete response and three-year invasive disease-free survival. In an independent study of triple-negative breast cancer patients, Guardant Reveal accurately predicted relapse risk and detected ctDNA more frequently and earlier than orthogonal methods.

Together, these findings demonstrate the validity of Guardant Reveal as a reliable approach for ctDNA detection and monitoring in both neoadjuvant and surveillance settings.

“Breast cancer care increasingly depends on understanding tumor biology at every stage of disease, and Guardant’s data continues to reaffirm the growing value of multiomic liquid biopsy as a powerful, non-invasive tool to equip providers with the data necessary to tailor therapy and treatment,” said Dr. Craig Eagle, Chief Medical Officer at Guardant Health. “We look forward to sharing our findings demonstrating the power of blood in helping provide clinicians with deeper clarity and more confident decision-making for their patients.”

Other data highlights to be presented include:

Data showcasing the clinical utility of Guardant Reveal in predicting outcomes in patients with early-stage HER2-positive breast cancer. In this analysis, early ctDNA clearance throughout treatment was linked to better outcomes, underscoring ctDNA as a sensitive marker of treatment response and highlighting the value of tissue-free testing for real-time response monitoring.
Data supporting the clinical value potential of non-invasive, methylation-based, continuous breast cancer subtype monitoring by Guardant360 Liquid to uncover tumor evolution and heterogeneity that may guide more precise treatment decisions. In this analysis, Guardant360 Liquid showed agreement to tissue-based monitoring and replicated previously reported accuracy.
Data showcasing Guardant360 Liquid methylation-based breast cancer subtyping feature as a noninvasive liquid tool to dynamically reassess ER, HER2 and triple negative status in patients who are resistant to ER therapy but don’t carry an ESR1 mutation. In this study, Guardant360 Liquid predicted real-world outcomes in previously AI-treated patients receiving subsequent lines of therapy.

Guardant-Led Research at SABCS 2025

Presentation	Title	Time / Location
Wednesday, December 10
PD2-09	Molecular Evolution in Early and Late-Stage Tumors	7:00 – 8:30am CST / 301 ABC
GS1-07	Circulating tumor DNA (ctDNA) in human epidermal growth factor receptor	9:30am – 12:00pm / Hall 1
PS1-12-05	Liquid biopsy–based molecular profiling using GUARDANT360 CDx at progression on CDK4/6i+ET: findings from the AGO-B CAPTOR study	12:30pm – 2:00pm / Exhibit Hall
PS1-11-09	Real-world prevalence of estrogen receptor (ER) 1 mutations (ESR1m) among patients with ER+/ human epidermal growth factor receptor 2 (HER2)− metastatic breast cancer (MBC) after first-line (1L) treatment with endocrine therapy (ET) and/or cyclin dependent kinase 4/6 inhibitors (CDK4/6i)	12:30pm – 2:00pm / Exhibit Hall
RF3-05	Tissue-free circulating tumor DNA detection in patients with early triple negative breast cancer from the c-TRAK-TN trial	12:30pm – 2:00pm / Exhibit Hall
PS2-07-02	Tissue-free epigenomic circulating tumor DNA (ctDNA) analysis pre- and post-surgery in early breast cancer: clinical features and prognostic utility	4:30pm – 5:30pm / Stars at Night
PS2-07-18	Prognostic effects of methylation-based HR and HER2 assessments by liquid biopsy in ESR1 mutation negative advanced breast cancer	4:30pm – 5:30pm / Stars at Night
PS2-07-16	Clinicogenomic characterization and ctDNA detection of ESR1 fusion positive metastatic breast cancer	5:00pm – 6:30pm / Exhibit Hall
PS2-09-21	Real-world clinical outcomes and genomic insights for patients with PIK3CA-mutant metastatic breast cancer following progression on CDK4/6 inhibitor therapy	5:00pm – 6:30pm / Exhibit Hall
PS2-08-17	Prevalence and clinical significance of exon 6 ESR1 gene fusions in advanced breast cancer after disease progression on aromatase inhibitors	5:00pm – 6:30pm / Exhibit Hall
PS2-10-19	Circulating tumor DNA (ctDNA) as a strong prognostic biomarker of minimal residual disease (MRD) using a tissue-free, epigenomic assay in early-stage breast cancer.	5:00pm – 6:30pm / Exhibit Hall
Thursday, December 11
PD9-03	Comprehensive genomic profiling and clinically targetable mutations of metastatic invasive lobular and no special type breast cancer	7:00am – 8:30am / Hemisfair Ballroom
PS4-01-11	Exploration of racial differences in variants of uncertain significance (VUS), ESR1 and PIK3CA mutation frequency, matched therapy use, and outcomes in metastatic breast cancer (mBC)	7:00am – 8:30am / Hemisfair Ballroom
PS4-02-10	Agreement of cell-free DNA methylation-based molecular breast subtyping and tissue subtyping in hormone receptor positive metastatic breast cancer: a clinical cohort analysis	5:00pm – 6:30pm / Exhibit Hall

The full abstracts for Guardant Health and a list of all abstracts being presented at SABCS 2025 can be found on the SABCS website.

For information and updates from the conference, visit booth #942 and follow Guardant Health on LinkedIn, X (Twitter) and Facebook.

About Guardant Health

Guardant Health is a leading precision oncology company focused on guarding wellness and giving every person more time free from cancer. Founded in 2012, Guardant is transforming patient care and accelerating new cancer therapies by providing critical insights into what drives disease through its advanced blood and tissue tests, real-world data and AI analytics. Guardant tests help improve outcomes across all stages of care, including screening to find cancer early, monitoring for recurrence in early-stage cancer, and treatment selection for patients with advanced cancer. For more information, visit guardanthealth.com and follow the company on LinkedIn, X (Twitter) and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of federal securities laws, including statements regarding the potential utilities, values, benefits and advantages of Guardant Health’s liquid biopsy tests or assays, which involve risks and uncertainties that could cause the actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect Guardant Health’s financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the captions “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operation” and elsewhere in its Annual Report on Form 10-K for the year ended December 31, 2024, and any current and periodic reports filed with or furnished to the Securities and Exchange Commission thereafter. The forward-looking statements in this press release are based on information available to Guardant Health as of the date hereof, and Guardant Health disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing Guardant Health’s views as of any date subsequent to the date of this press release.

Contacts

Investor Contact:
Zarak Khurshid

investors@guardanthealth.com

Media Contact:
Meaghan Smith

press@guardanthealth.com

Natera Acquires Foresight Diagnostics

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Natera Acquires Foresight Diagnostics

Natera Acquires Foresight Diagnostics

Expands Natera’s lead in solid tumor MRD, acquiring ultrasensitive phased variant technology with LOD95 of 0.3 parts per million (ppm) and detection below 0.1 ppm¹

Cutting-edge IP adds to Natera’s portfolio of >500 issued or pending patents

Accelerates Natera’s expansion into lymphoma, where Foresight has developed a strong clinical position

AUSTIN, Texas & BOULDER, Colo.–(BUSINESS WIRE)–Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, and Foresight Diagnostics, a leader in ultrasensitive molecular residual disease (MRD) detection, today announced that Natera has completed a transaction to acquire Foresight.

Foresight is a cancer diagnostics company and CLIA-registered laboratory. The company’s circulating tumor DNA (ctDNA)-based MRD tests leverage its patented PhasED-Seq™ technology, targeting phased variants. With this technology, Foresight has reported performance with LOD95 of 0.3 parts per million and detection below 0.1 ppm¹.

Foresight was founded by Stanford University physicians and scientists, Maximilian Diehn, M.D., Ph.D., Ash Alizadeh, M.D., Ph.D., and David Kurtz, M.D., Ph.D., together with Jake Chabon, Ph.D., Foresight’s chief scientific officer and chief executive officer. The company has authored more than 40 scientific publications and presentations and partnered with more than 30 biopharma and academic researchers.

Strategic Rationale

The transaction combines Natera’s leading commercial and operational infrastructure for the delivery of personalized MRD testing with Foresight’s unique phased variant technology and leadership in lymphoma. It builds on Natera’s broad intellectual property portfolio for tumor-informed and personalized MRD products including in phased variants, and promises to accelerate MRD adoption in lymphoma and other solid tumor types.

Signatera™ platform with phased variants: The integration of phased variants into the Signatera platform will further differentiate and strengthen test performance across solid tumors. This enhanced version is available immediately for research use for biopharma and academic partners and is expected to be launched for clinical use in 2026.
Leadership in lymphoma: The transaction builds on Foresight’s clinical research momentum in B-cell lymphomas, a large patient population with more than 75,000 new cases annually in the U.S.² Earlier this year, Foresight data provided the foundation for the inclusion of ctDNA MRD into the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines for diffuse large B-cell lymphoma. Additionally, Foresight’s CLARITY™ MRD assay for lymphoma is being used in three prospective MRD-driven clinical trials informing treatment decisions for patients. Foresight CLARITY joins Natera’s extensive MRD product portfolio and will continue to support clinical trials, translational research and future applications.

At the American Society of Hematology (ASH) Annual Meeting beginning on December 6, Natera and Foresight will have a total of 15 abstracts featuring Signatera and PhasED-Seq, including seven oral presentations.

Transaction Terms

Natera has closed the acquisition of Foresight in an all-stock transaction consisting of a $275 million upfront with an additional $175 million in earnouts tied to the achievement of revenue- and reimbursement-based milestones.

“This acquisition reinforces Natera’s position at the forefront of precision oncology,” said Steve Chapman, chief executive officer of Natera. “Foresight’s phased variant technology and leadership in lymphoma complement Natera’s strong capabilities in personalized MRD testing, improving the value we can deliver to patients, clinicians, biopharma partners and the broader healthcare system.”

“Foresight’s mission has always been to improve the lives of cancer patients worldwide through innovative diagnostics,” said Chabon. “As we join Natera, I’m deeply grateful to our employees, partners and investors who have helped bring us to this moment. Together, we can realize this mission on a far greater scale, accelerating the pace of discovery across both hematologic and solid tumors.”

Gibson, Dunn & Crutcher LLP is serving as legal counsel to Natera. Wilson Sonsini Goodrich & Rosati is serving as legal counsel to Foresight, while Centerview Partners LLC is acting as its financial advisor.

References

Cabel L, Jeon YJ, Parikh AR, et al. Ultrasensitive Phased Variant Detection Enables Improved ctDNA-Based MRD Assessment Across Solid Tumors. Presented at: European Society for Medical Oncology (ESMO) Annual Meeting; 2024.
American Cancer Society. Types of B-cell Lymphoma. American Cancer Society. https://www.cancer.org/cancer/types/non-hodgkin-lymphoma/about/b-cell-lymphoma.html. Accessed November 26, 2025.

About Natera

Natera™ is a global leader in cell-free DNA and precision medicine, dedicated to oncology, women’s health, and organ health. We aim to make personalized genetic testing and diagnostics part of the standard-of-care to protect health and inform earlier, more targeted interventions that help lead to longer, healthier lives. Natera’s tests are supported by more than 325 peer-reviewed publications that demonstrate excellent performance. Natera operates ISO 13485-certified and CAP-accredited laboratories certified under the Clinical Laboratory Improvement Amendments (CLIA) in Austin, Texas, and San Carlos, California. For more information, visit www.natera.com.

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements and are not a representation that Natera’s plans, estimates, or expectations will be achieved. These forward-looking statements, including those regarding the expected benefits of the Foresight acquisition, integration of technologies, clinical and commercial opportunities, and future performance, represent Natera’s expectations as of the date of this press release, and are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including the ability to integrate Foresight’s business, realize anticipated benefits, achieve expected clinical or commercial outcomes, obtain or maintain reimbursement coverage, and respond to competitive or regulatory developments. Additional risks and uncertainties are discussed in greater detail in “Risk Factors” in Natera’s recent filings on Forms 10-K and 10-Q and in other filings Natera makes with the SEC from time to time. These documents are available at www.natera.com/investors and www.sec.gov.

Contacts

Investor Relations: Mike Brophy, CFO, Natera, Inc., investor@natera.com
Media: Lesley Bogdanow, VP of Corporate Communications, Natera, Inc., pr@natera.com

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Natera Acquires Foresight Diagnostics