Earlier use of CARVYKTI®▼ (ciltacabtagene autoleucel) demonstrated lasting treatment-free remissions at 2.5 years in patients with relapsed or refractory multiple myeloma




Earlier use of CARVYKTI®▼ (ciltacabtagene autoleucel) demonstrated lasting treatment-free remissions at 2.5 years in patients with relapsed or refractory multiple myeloma

Follow-up data from CARTITUDE-4 show at least 80 percent of as-treated standard-risk patients remained progression- and treatment-free following a single infusion as early as second line¹

Data suggest stronger immune fitness in earlier lines may be associated with longer progression-free survival²

BEERSE, BELGIUM, Dec. 06, 2025 (GLOBE NEWSWIRE) — Johnson & Johnson today announced updated results from the Phase 3 CARTITUDE-4 study supporting durable treatment-free remissions as early as second line treatment with CARVYKTI^®▼ (ciltacabtagene autoleucel; cilta-cel).¹ In 80 percent of as-treated patients with relapsed or refractory multiple myeloma (RRMM) and standard-risk cytogenetics who were treated with cilta-cel as early as first relapse, the disease did not progress and no further treatment was required at 2.5 years (30 months).¹ These results add to the body of clinical and real-world experience established across more than 9,000 patients treated with cilta-cel globally.³

Additional translational analyses demonstrated that patients receiving cilta-cel in earlier lines had improved immune fitness, which suggests a correlation with longer progression-free survival (PFS).² These data (Abstracts #92, #94) were featured in oral presentations at the 2025 American Society of Hematology (ASH) Annual Meeting.^1,2

“These data suggest that a single infusion of cilta-cel for standard-risk patients may provide additional benefit to patients as early as second line of therapy,” said Luciano J. Costa, M.D., Ph.D., Professor of Medicine at the University of Alabama and principal investigator of the CARTITUDE-4 study.* “Treating patients with multiple myeloma after first relapse offers the opportunity to achieve deeper and more durable responses, shifting the treatment paradigm closer to the possibility of long-term remission and, ultimately, cure.”

“Our goal is to treat patients as early as possible, when they have the best chance for lasting remission,” said Jordan Schecter, M.D., Vice President, Research & Development, Multiple Myeloma, Johnson & Johnson Innovative Medicine. “With more than 9,000 patients treated globally, cilta-cel has demonstrated robust efficacy as soon as first relapse and is the only CAR-T to significantly extend overall survival versus standard therapies.”

In the analysis of CARTITUDE-4 data, 176 patients received cilta-cel as early as second line and 59 of those patients had standard-risk cytogenetics.^1,4 At a median follow-up of 33.6 months, the 30-month PFS rate among the standard-risk patients in the as-treated population appeared to plateau at 80.5 percent (95 percent confidence interval [CI], 67.2–88.8) following a single infusion of cilta-cel.^1,4 Notably, all 26 patients (100 percent) from this group who achieved minimal residual disease (MRD)-negative complete response at 12 months following cilta-cel infusion remained progression-free at 30 months.¹

With an additional median follow-up, the safety profile in CARTITUDE-4 was consistent with the known safety profile of cilta-cel, where 97 percent of patients in both the cilta-cel and standard of care treatment arms experienced Grade 3/4 treatment-emergent adverse events (TEAEs), with cytopenia being the most common.^1,5 In patients with standard-risk cytogenetics, non-haematologic serious adverse events occurred in 52.5 percent, 28.8 percent experienced Grade 3/4 infections, 74.6 percent experienced cytokine release syndrome (CRS), 1.7 percent experienced immune effector cell (IEC)-associated neurotoxicity syndrome (ICANS) and 6.8 percent cranial nerve palsy (CNP).¹ There were no cases of IEC-parkinsonism and 10.2 percent non-relapse mortality after one year.¹

Additionally, a translational analysis evaluated the relationship between immune biomarkers and PFS in patients treated with cilta-cel in CARTITUDE-1 and CARTITUDE-4.² Using cilta-cel after one or two prior lines of therapy demonstrated stronger immune fitness versus patients with three or more prior lines of therapy, characterised by increased baseline CD4⁺ naïve T-cells (a type of immune cell that has not encountered an antigen) in peripheral blood.² Bone marrow tumour analyses from patients treated with cilta-cel in CARTITUDE-4 also demonstrated a more immune-activated profile in patients treated after one prior line of therapy versus three.² These biomarker data identify potential immunologic factors associated with longer PFS and support the improved survival outcomes exhibited with cilta-cel for patients treated as early as second line.²

“Our mission in multiple myeloma is to deliver transformational innovations that fundamentally shift the trajectory of disease and extend and improve patients’ lives,” said Ester in ‘t Groen, EMEA Therapeutic Area Head Haematology, Johnson & Johnson Innovative Medicine. “The data presented at ASH add to the growing body of evidence confirming the potential of cilta-cel to do just that. The important correlation between immune fitness and long-term progression-free survival reinforces our commitment to advancing this therapy earlier in the treatment pathway, where it has the greatest opportunity to maximise deeper, more durable outcomes for patients.”

As cilta-cel use has broadened across academic centres and community practices, Johnson & Johnson continues to collect and analyse clinical and real-world data to further characterise long-term remission outcomes and safety trends. This comprehensive experience across diverse patient populations provides an important foundation for expanding use into earlier treatment settings.

About CARTITUDE-1
CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicentre study that evaluated the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma (RRMM), 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory, meaning their cancer did not or no longer responds to an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody.^6,7

The primary objective of the Phase 1b portion of the study, involving 29 patients, was to characterise the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2).⁶ Based on the safety profile observed in this portion of the study, outpatient dosing is being evaluated in additional CARTITUDE studies. The Phase 2 portion of the study is evaluating the efficacy of cilta-cel with overall response as the primary endpoint.⁶ The study involved patients with heavily pretreated RRMM who historically have an expected median progression-free survival (PFS) of <6 months and median overall survival (OS) of ~1 year.⁶

About CARTITUDE-4
CARTITUDE-4 (NCT04181827) is the first international, randomised, open-label Phase 3 study evaluating the efficacy and safety of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in adult patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, including a PI and an IMiD.⁸ Patients were randomised to receive either a sequence of apheresis, bridging therapy, lymphodepletion and cilta-cel (n=208) or standard of care (SOC), which included PVd or DPd (n=211).⁹ The primary outcome measure for the study is PFS, defined as the time from the date of randomisation to the date of first documented disease progression, as defined in the International Myeloma Working Group (IMWG) criteria, or death due to any cause.⁸ Safety, OS, minimal residual disease (MRD) negativity rate and overall response rate are secondary endpoints.⁸

About Cilta-cel 
Cilta-cel is a B-cell maturation antigen (BCMA)-directed, genetically modified autologous T-cell immunotherapy that involves reprogramming a patient’s own T-cells with a transgene encoding chimeric antigen receptor (CAR) that directs the CAR-positive T-cells to eliminate cells that express BCMA.⁹ BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.¹⁰ The cilta-cel CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA.⁹ Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.¹¹ 

In April 2024, the European Commission (EC) approved an indication extension for cilta-cel for the treatment of adults with RRMM who have received at least one prior therapy, including an iMiD and a PI, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide. In April 2024, cilta-cel was approved in the U.S. for the second line treatment of adult patients with relapsed or refractory myeloma who have received at least one prior line of therapy including a PI, an iMiD, and who are refractory to lenalidomide. 

In December 2017, Janssen Biotech, Inc., a Johnson & Johnson company, entered into an exclusive worldwide licence and collaboration agreement with Legend Biotech USA, Inc., to develop and commercialise cilta-cel.¹²

For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using cilta-cel please refer to the Summary of Product Characteristics.⁹ ▼In line with European Medicines Agency (EMA) regulations for new medicines and those given conditional approval, cilta-cel is subject to additional monitoring.⁹  

About Multiple Myeloma
Multiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.^13,14 In multiple myeloma, these plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, often causing bone destruction and other serious complications.^13,14 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died.¹⁵ Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy while remissions become progressively shorter.^16,17,18 Whilst some people diagnosed with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney problems.^19,20

About Johnson & Johnson  
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.

Learn more at www.innovativemedicine.jnj.com/emea. Follow us at www.linkedin.com/company/jnj-innovative-medicine-emea.

Cautions Concerning Forward-Looking Statements  
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of cilta-cel. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov/, http://www.jnj.com/ or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments.

^* Luciano J. Costa, M.D., Ph.D. Professor of Medicine at the University of Alabama and principal investigator of the CARTITUDE-4 study, has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work.

¹ Costa L, et al. Long-Term Progression-Free Survival Benefit With Ciltacabtagene Autoleucel in Standard-Risk Relapsed/Refractory Multiple Myeloma. Oral presentation. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
² Parekh S Li K, et al. Earlier use of Ciltacabtagene Autoleucel (Cilta-cel) is Associated With Better Immune Fitness and Stronger Immune Effects as Shown by Correlative Analysis of Peripheral Blood and the Bone Marrow Tumor Microenvironment (TME) From the CARTITUDE-4 Study. Oral presentation. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
³ Legend Biotech. Legend Biotech announces 9 presentations at the 67th American Society of Hematology Annual Meeting. Available at: https://investors.legendbiotech.com/news-releases/news-release-details/legend-biotech-announces-10-presentations-67th-american-society. Last accessed: December 2025.
⁴ Costa L, et al. Long-Term Progression-Free Survival Benefit With Ciltacabtagene Autoleucel in Standard-Risk Relapsed/Refractory Multiple Myeloma. Abstract. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
⁵ Mateos MV, et al. Overall Survival (OS) With Ciltacabtagene Autoleucel (Cilta-cel) Versus Standard of Care (SoC) in Lenalidomide (Len)-Refractory Multiple Myeloma (MM): Phase 3 CARTITUDE-4 Study Update. International Myeloma Society 2024 Annual Meeting. September 2024.
⁶ ClinicalTrials.gov. A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma (CARTITUDE-1). Available at: Study Details | A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma | ClinicalTrials.gov. Last accessed: December 2025.
⁷ Lin Y, et al. CARTITUDE-1 final results: Phase 1b/2 study of ciltacabtagene autoleucel in heavily pretreated patients with relapsed/refractory multiple myeloma. Oral presentation. American Society of Clinical Oncology (ASCO) Annual Meeting 2023.
⁸ ClinicalTrials.gov. A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma (CARTITUDE4). Available at: https://clinicaltrials.gov/study/NCT04181827. Last accessed: December 2025.
⁹ European Medicines Agency. CARVYKTI (ciltacabtagene autoleucel) Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/carvykti-epar-product-information_en.pdf. Last accessed: December 2025.
¹⁰ Cho SF, et al. Targeting B Cell Maturation Antigen (BCMA) in Multiple Myeloma: Potential Uses of BCMA-Based Immunotherapy. Front Immunol 2018;10(9):1821.
¹¹ Tai YT, et al. Targeting B-cell maturation antigen in multiple myeloma. Immunotherapy 2015;7(11):1187-1199. 
¹² Johnson & Johnson.com. Janssen Enters Worldwide Collaboration and License Agreement with Chinese Company Legend Biotech to Develop Investigational CAR-T Anti-Cancer Therapy. Available at: https://www.jnj.com/media-center/press-releases/janssen-enters-worldwide-collaboration-and-license-agreement-with-chinese-company-legend-biotech-to-develop-investigational-car-t-anti-cancer-therapy. Last accessed: December 2025.
¹³ Abdi J, et al. Drug resistance in multiple myeloma: latest findings on molecular mechanisms. Oncotarget 2013;4(12):2186-2207.
¹⁴ American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction. Last accessed: December 2025. 
¹⁵ ECIS. European Cancer Information System. Estimates of cancer incidence and mortality in 2022, by country. Multiple myeloma. Available at: https://ecis.jrc.ec.europa.eu/explorer.php?$0-0$1-All$2-All$4-1,2$3-51$6-0,85$5-2022,2022$7-7$CEstByCountry$X0_8-3$X0_19-AE27$X0_20-No$CEstBySexByCountry$X1_8-3$X1_19-AE27$X1_-1-1$CEstByIndiByCountry$X2_8-3$X2_19-AE27$X2_20-No$CEstRelative$X3_8-3$X3_9-AE27$X3_19-AE27$CEstByCountryTable$X4_19-AE27. Last accessed: December 2025.
¹⁶ Bhatt P, et al. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347.
¹⁷ Hernández-Rivas JÁ, et al. The changing landscape of relapsed and/or refractory multiple myeloma (MM): fundamentals and controversies. Biomark Res. 2022;10(1):1-23.
¹⁸ Gavriatopoulou M, et al. Metabolic Disorders in Multiple Myeloma. Int J Mol Sci. 2021;22(21):11430.
¹⁹ American Cancer Society. What is Multiple Myeloma? https://www.cancer.org/cancer/multiple-myeloma/about/what-is-multiple-myeloma.html. Last accessed: December 2025.
²⁰ American Cancer Society. Multiple Myeloma Early Detection, Diagnosis, and Staging. https://www.cancer.org/cancer/types/multiple-myeloma/detection-diagnosis-staging/detection.html. Last accessed: December 2025.

December 2025
CP-554345

CONTACT: Media contact:
Jenni Mildon 
jmildon@its.jnj.com  
+44 7920 418 552 

Investor contact:
Lauren Johnson
investor-relations@its.jnj.com

Cogent Biosciences Presents Full SUMMIT Results of Bezuclastinib in Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM) at the 67th Annual Meeting of the American Society of Hematology (ASH)




Cogent Biosciences Presents Full SUMMIT Results of Bezuclastinib in Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM) at the 67th Annual Meeting of the American Society of Hematology (ASH)

— Bezuclastinib achieves clear clinical benefit across all symptom domains including significant improvements on 11 individual symptoms plus the most severe symptom at baseline —

— Bezuclastinib demonstrates that reducing objective measures of disease, including serum tryptase, correlates with improvement in symptom severity; the first time this has been shown in NonAdvSM patients —

— New 48-week data demonstrate a clear, continued deepening of symptomatic improvement over time —

— Bezuclastinib demonstrated a favorable safety and tolerability profile supporting chronic use — 

— Granted Breakthrough Therapy Designation for bezuclastinib in October 2025; New Drug Application (NDA) on track for submission in December 2025 — 

— Cogent to host investor conference call and webcast on Monday, December 8, at 8:00 a.m. ET — 

WALTHAM, Mass. and BOULDER, Colo., Dec. 06, 2025 (GLOBE NEWSWIRE) — Cogent Biosciences, Inc. (NASDAQ: COGT) today announced complete results from the registration-directed Part 2 of the SUMMIT clinical trial of bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM). As previously reported, bezuclastinib demonstrated clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints. New results further highlight the benefit of bezuclastinib on patient-reported symptoms and objective measures of mast cell burden and demonstrate significant correlation between improvement in disease pathology and patient-reported symptom severity. 

“We are excited to present additional data from the SUMMIT trial that support our conviction that bezuclastinib will be the best-in-class treatment option for patients with nonadvanced systemic mastocytosis,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “We remain on track to submit our first New Drug Application for bezuclastinib in NonAdvSM with the FDA this month and are encouraged by the increased interest in our Expanded Access Program.”

“Nonadvanced systemic mastocytosis patients currently have very limited treatment options, and the benefit bezuclastinib demonstrated in the SUMMIT trial across measures of disease pathology and symptomatic improvement is very exciting for this patient population,” said Lindsay Rein, MD, Associate Professor of Medicine in the Division of Hematologic Malignancies and Cellular Therapy, Duke University. “The SUMMIT trial results match my clinical experience using bezuclastinib with NonAdvSM patients, delivering rapid and deep improvement in symptom control and objective measures of disease without tolerability challenges.”

SUMMIT Trial Data 

In the registration-directed Part 2 of the SUMMIT clinical trial, 118 patients received bezuclastinib once daily plus best supportive care (BSC) and 60 patients received placebo plus BSC. The study included adults with a NonAdvSM diagnosis confirmed by central pathology review, and moderate-to-severe symptom burden despite an optimized regimen of BSC. 

Following completion of the 24-week treatment period, patients had the option to receive bezuclastinib in an open-label extension study. Baseline patient demographics were balanced between treatment arms and reflected significant disease burden. Disease symptoms were assessed using the Mastocytosis Symptom Severity Daily Diary (MS2D2).

Bezuclastinib delivered clinically meaningful and statistically significant symptomatic improvement

Across several additional key secondary endpoints, bezuclastinib demonstrated rapid, deep and sustained improvement on objective disease markers of mast cell burden. At week 24, 87.4% of patients achieved ≥50% reduction in serum tryptase levels, 75.6% of patients demonstrated ≥50% reduction in bone marrow mast cells or clearance of aggregates and 85.7% of patients achieved ≥50% reduction in KIT D816V variant allele frequency or undetectable, each of which was statistically significant when compared to placebo. Additional pathobiology data from SUMMIT patients will be shared in an oral presentation on Monday, December 8^th at ASH.

SUMMIT Subgroups

As part of the SUMMIT study, patients with Smoldering Systemic Mastocytosis (n=8 bezuclastinib arm, n=4 placebo arm) and patients who had previously been treated with avapritinib (n=11 bezuclastinib arm, n=3 placebo arm) were enrolled. Patients treated with bezuclastinib in these subgroups showed a mean change in TSS of -35.6 and -21.6, respectively. The response in objective measures of disease burden in these patients was consistent with results from the broader SUMMIT population, as were their related adverse events and overall tolerability.

Safety Data 

As previously reported on July 7, 2025, the majority of treatment emergent adverse events (TEAEs) (98.3% in bezuclastinib arm vs. 88.3% in placebo arm) were of low grade. The most frequent TEAEs reported on bezuclastinib treatment were hair color change (69.5% bezuclastinib vs. 5.0% placebo), altered taste (23.7% bezuclastinib vs. 0% placebo), nausea (22.0% bezuclastinib vs. 13.3% placebo) and ALT/AST elevations (22.0% bezuclastinib vs. 6.6% placebo; ≥Gr 3, 5.9% vs. 0%). Serious AEs occurred in 4.2% of patients treated with bezuclastinib, compared to 5.0% of patients treated with placebo. Discontinuations due to treatment-related AEs occurred in 5.9% of patients treated with bezuclastinib, all due to ALT/AST elevations and all patients fully resolved. There were no hepatic AEs reported in any patient other than transient and manageable lab abnormalities.

SUMMIT Long Term Follow-up

Data from longer term follow-up in patients participating in the SUMMIT trial are expected to be presented at an upcoming scientific meeting in Q1 2026. Preliminary 48-week data will be shared during the investor call scheduled for Monday, December 8^th.

Webcast Information  

Cogent will host a live webcast on Monday, December 8, at 8:00 a.m. ET to discuss these additional data from SUMMIT in NonAdvSM. The live event will be available on the Investors & Media page of Cogent’s website at investors.cogentbio.com. A replay of the webcast will be available approximately two hours after the completion of the event and will be archived for up to 30 days.

About Cogent Biosciences, Inc.
Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. The company also has an ongoing Phase 1 study of its novel internally discovered FGFR2/3 inhibitor. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Kα, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at www.cogentbio.com. Follow Cogent Biosciences on social media: X (formerly known as Twitter) and LinkedIn. Information that may be important to investors will be routinely posted on our website and X.

Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: plans to submit an NDA to the FDA for bezuclastinib in patients with NonAdvSM in December 2025; the company’s belief that bezuclastinib will be the best-in-class treatment option for NonAdvSM patients and plans to present data from longer term follow-up in patients participating in the SUMMIT trial at an upcoming scientific meeting in Q1 2026. The use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” and similar words or expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption “Risk Factors” in Cogent’s most recent Annual Report on Form 10-K filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. 

Contact: 
Christi Waarich 
Senior Director, Investor Relations 
christi.waarich@cogentbio.com 
617-830-1653    

Nurix Therapeutics Presents New Data Demonstrating Durable, Deepening Responses in Phase 1 Trial of Bexobrutideg (NX-5948) in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition




Nurix Therapeutics Presents New Data Demonstrating Durable, Deepening Responses in Phase 1 Trial of Bexobrutideg (NX-5948) in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition

Objective response rate (ORR) of 83% including two complete responses in CLL patients in Phase 1a study with median progression free survival (PFS) of 22.1 months across all doses tested

Emerging data from randomized Phase 1b cohorts points to higher ORR and longer progression free survival at the 600 mg recommended Phase 2 dose (RP2D) compared to the 200 mg dose

Bexobrutideg was well tolerated with a consistent safety profile between the 600 mg RP2D and the overall study population

Phase 2 clinical trial of bexobrutideg (DAYBreak-CLL-201) currently enrolling globally

Company will host a webcast to discuss the data on Monday, December 8, 2025, at 8:15 p.m. ET

BRISBANE, Calif., Dec. 06, 2025 (GLOBE NEWSWIRE) — Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines in oncology and autoimmune disease, today announced new clinical data from the Company’s ongoing Phase 1a/1b NX-5948-301 study of bexobrutideg (NX-5948) in patients with relapsed or refractory B-cell malignancies. These data will be presented in an oral session at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, FL, on December 6, 2025, at 9:45 a.m. ET, by Zulfa Omer, M.D., Assistant Professor of Internal Medicine at the University of Cincinnati and a principal investigator in the study.

“The clinical activity and durability observed with bexobrutideg in this study are highly encouraging for patients with relapsed or refractory CLL/SLL, many of whom have limited treatment options,” said Dr. Omer. “The responses we are seeing across heavily pretreated patients, including those with prior exposure to both covalent and non-covalent BTK inhibitors and BCL-2 inhibitors, support continued evaluation of bexobrutideg as a therapeutic approach for patients with relapsed or refractory CLL/SLL and ultimately earlier line patients.”

The new and updated data from the Phase 1a/1b study (NX-5948-301) in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) include safety findings across all patients, safety findings for patients treated at the RP2D of 600 mg once daily, updated Phase 1a results with extended follow-up, and emerging efficacy results from the randomized Phase 1b cohort 1 comparing 200 mg and 600 mg once-daily dosing. Collectively, these results provide a maturing clinical picture of bexobrutideg’s efficacy, durability, and tolerability, which form the foundation for Nurix’s advancing pivotal clinical program.

“We are excited to share this important data update for bexobrutideg, which continues to demonstrate compelling efficacy and durability for patients with relapsed or refractory CLL/SLL” said Paula O’Connor, M.D., chief medical officer of Nurix. “Advancing the 600 mg dose into our pivotal DAYBreak program reflects our conviction that this regimen offers patients the greatest opportunity for sustained clinical benefit, supported by a favorable safety profile.”

Data presented at the 2025 ASH Annual Meeting include baseline demographics and safety findings for all patients with CLL/SLL in the ongoing Phase 1a/1b study (n=126) and safety findings for patients treated at the RP2D of 600 mg (n=70). Efficacy results are presented for patients treated with bexobrutideg at doses ranging from 50 mg to 600 mg in the Phase 1a study (n=48) and for patients in the Phase 1b cohort 1, who were randomized and treated with either a 200 mg or 600 mg dose (n=42) in accordance with FDA’s Project Optimus.

Phase 1a/1b demographics and safety findings
Overall, the heavily pretreated Phase 1a/1b population had received a median of three prior lines of therapy (range = 1–17) including prior BTK inhibitors (85.7%), prior BCL-2 inhibitors (61.9%), and prior non-covalent BTK inhibitors (27.0%). The Phase 1a population was more heavily pretreated with a median of four prior lines of therapy (range = 2-12) including prior BTK inhibitors (97.9%), prior BCL-2 inhibitors (83.3%), and prior non-covalent BTK inhibitors (27.1%). At baseline, many patients had mutations associated with BTK inhibitor resistance, including mutations in BTK (39.6% overall, 38.3% in the Phase 1a population) and PLCG2 (8.1% overall, 14.9% in the Phase 1a population). Poor prognostic features were common, including TP53 mutations (39.6% overall, 44.7% in the Phase 1a population). Of the five patients (4.0%) in the trial who had central nervous system (CNS) involvement, all five were in the Phase 1a population.

Bexobrutideg was well tolerated across all dose levels evaluated, consistent with prior disclosures. The treatment emergent adverse event (TEAE) profile was similar between the RP2D of 600 mg and the overall study population with the most common treatment emergent adverse events being purpura/contusion, neutropenia, and petechiae. There were no dose-limiting toxicities, no systemic fungal infections or Grade 4 infections of any kind, and a single event of new onset atrial fibrillation was consistent with the rate in the age-matched general population.

Phase 1a efficacy update (n=48)
The updated Phase 1a dataset includes patients treated at starting dose levels ranging from 50 mg to 600 mg once daily with a median follow-up of 19.0 months (range = 13.5 – 32.3). Among the 47 efficacy evaluable patients, the objective response rate (ORR) was 83.0% including two patients (4.3%) with a complete response, an improvement from earlier disclosures due to additional follow-up and deepening of response. Overall, the disease control rate (DCR) was 95.7%. Importantly, the median progression-free survival was 22.1 months, and the median duration of response (DOR) was 20.1 months. Responses were observed across clinically challenging subgroups including patients who had progressed on prior BTK inhibitors, patients who were double-exposed to both BTK inhibitors and BCL-2 inhibitors, patients who had received prior non-covalent BTK inhibitors, patients with baselines mutations associated with BTK inhibitor resistance including non-C481 BTK mutations, and patients with high-risk molecular features such as TP53 mutations. Meaningful reductions in lymph node burden were also observed independent of baseline mutations associated with BTK inhibitor resistance and poor prognosis.

Phase 1b Cohort 1: Randomized evaluation of 200 mg vs 600 mg once daily (n=42)
In the randomized Phase 1b cohort, 42 patients were assigned to receive either 200 mg (n = 22) or 600 mg (n = 20) once daily. Among the 37 efficacy evaluable patients, preliminary data showed the 600 mg dose with an ORR of 83.3% compared to 73.7% for the 200 mg dose. With a median follow up of 9.8 months, the preliminary PFS curves suggest longer progression free survival for the 600 mg group compared to the 200 mg group.

Across Phase 1a and Phase 1b, the totality of clinical data supports 600 mg once daily as the optimal dose for further development. At this dose level, bexobrutideg demonstrated the strongest clinical activity observed to date, including higher response rates and a favorable trend toward longer progression-free survival in the randomized Phase 1b cohort. Importantly, the 600 mg dose maintained a tolerable safety profile comparable to the overall study population, with no dose-limiting toxicities, no systemic fungal infections, and no Grade 4 infections reported. Taken together, in accordance with FDA’s Project Optimus, these results provide a robust foundation for advancing 600 mg as the recommended Phase 2 dose and for the ongoing pivotal DAYBreak development program.

“These exciting, positive results reinforce the potential for bexobrutideg to be best-in-class and form a strong foundation to support our pivotal development program,” said Arthur T. Sands, M.D., Ph.D., president and chief executive officer, Nurix. “Nurix has entered this next phase of clinical development with momentum and a commitment to deliver a transformative new medicine for patients with B-cell malignancies.”

Webcast Details
Date and time: Monday, December 8, 2025, 8:15 p.m. ET
Access Details: The live webcast and subsequent archived replay will be available in the Investors section of the Nurix website under Events.

About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, highly selective small molecule degrader of BTK currently being evaluated in the DAYBreak CLL-201 clinical trial (NCT07221500), a pivotal single-arm Phase 2 study of bexobrutideg in patients with relapsed or refractory chronic lymphocytic leukemia. Nurix also continues enrollment in the NX-5948-301 Phase 1a/1b clinical trial (NCT05131022) of bexobrutideg in patients with relapsed or refractory B cell malignancies. Additional information on the ongoing clinical trials can be accessed at clinicaltrials.gov.

About Nurix Therapeutics, Inc.
Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, the next frontier in innovative drug design aimed at improving treatment options for patients with cancer and autoimmune diseases. Nurix’s wholly owned, clinical stage pipeline includes degraders of Bruton’s tyrosine kinase (BTK), a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B (CBL-B), an E3 ligase that regulates activation of multiple immune cell types including T cells and NK cells. Nurix also is advancing multiple potentially first-in-class or best-in-class degraders and degrader antibody conjugates (DACs) in its preclinical pipeline. Nurix’s partnered drug discovery pipeline consists of a preclinical stage degrader of STAT6 in collaboration with Sanofi, and a clinical stage degrader of IRAK4 in collaboration with Gilead, as well as multiple additional programs under collaboration agreements with Gilead Sciences, Inc., Sanofi S.A. and Pfizer Inc., within which Nurix retains certain options for co-development, co-commercialization and profit sharing in the United States for multiple drug candidates. Powered by a fully AI-integrated discovery engine capable of tackling any protein class, and coupled with unparalleled ligase expertise, Nurix’s dedicated team has built a formidable advantage in translating the science of targeted protein degradation into clinical advancements. Nurix aims to establish degrader-based treatments at the forefront of patient care, writing medicine’s next chapter with a new script to outmatch disease. Nurix is headquartered in Brisbane, California. For additional information visit http://www.nurixtx.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and other federal securities laws. When or if used in this press release, the words “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “outlook,” “plan,” “predict,” “should,” “will,” and similar expressions and their variants, as they relate to Nurix, may identify forward-looking statements. All statements that reflect Nurix’s expectations, assumptions or projections about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements regarding: the tolerability, safety profile, therapeutic potential and other advantages of bexobrutideg; the potential role of bexobrutideg in the treatment of patients with CLL and SLL, and Nurix’s plans and expectations for the development of bexobrutideg. Forward-looking statements reflect Nurix’s current beliefs, expectations, and assumptions. Although Nurix believes the expectations and assumptions reflected in such forward-looking statements are reasonable, Nurix can give no assurance that they will prove to be correct. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and changes in circumstances that are difficult to predict, which could cause Nurix’s actual activities and results to differ materially from those expressed in any forward-looking statement. Such risks and uncertainties include, but are not limited to: (i) the risks inherent in the drug development process, including the unexpected emergence of adverse events or other undesirable side effects during clinical development; (ii) uncertainties related to the timing and results of clinical trials; (iii) whether Nurix will be able to fund its research and development activities and achieve its research and development goals; (iv) the impact of economic and market conditions and global and regional events on Nurix’s business, clinical trials, financial condition, liquidity and results of operations; (v) whether Nurix will be able to protect intellectual property and (vi) other risks and uncertainties described under the heading “Risk Factors” in Nurix’s Quarterly Report on Form 10-Q for the fiscal period ended August 31, 2025, and other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. The statements in this press release speak only as of the date of this press release, even if subsequently made available by Nurix on its website or otherwise. Nurix disclaims any intention or obligation to update publicly any forward-looking statements, whether in response to new information, future events, or otherwise, except as required by applicable law.

Contacts:

Investors
Kris Fortner
Nurix Therapeutics, Inc.
Kfortner@nurixtx.com

Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
lwolffe@wheelhouselsa.com

Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
areynolds@wheelhouselsa.com

Bicara Therapeutics’ Preliminary Phase 1b Expansion Cohort Data Evaluating 750mg of Ficerafusp Alfa Weekly Plus Pembrolizumab Advances Pivotal Study Dose Selection on Track for First Quarter 2026




Bicara Therapeutics’ Preliminary Phase 1b Expansion Cohort Data Evaluating 750mg of Ficerafusp Alfa Weekly Plus Pembrolizumab Advances Pivotal Study Dose Selection on Track for First Quarter 2026

Ficerafusp alfa 750mg QW in combination with pembrolizumab demonstrates consistent overall response rate and safety profile comparable to 1500mg QW dose, further derisking pivotal FORTIFI-HN01 study interim analysis

Totality of data demonstrates that greater TGF-β inhibition, observed at 1500mg of ficerafusp alfa, drives deeper tumor responses that translate to more durable outcomes for patients

Pivotal FORTIFI-HN01 optimal dose declaration expected in first quarter 2026

Company to host conference call and webcast today at 9:00 a.m. ET

BOSTON, Dec. 06, 2025 (GLOBE NEWSWIRE) —  Bicara Therapeutics Inc. (Nasdaq: BCAX), a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors, today presented preliminary data from a Phase 1b expansion cohort evaluating 750 mg of ficerafusp alfa weekly (QW) in combination with pembrolizumab in first-line (1L) human papillomavirus (HPV)-negative recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). The data were highlighted in an oral presentation by Deborah Wong, MD, PhD of UCLA Medical Center at the European Society for Medical Oncology (ESMO) Asia Congress and will be discussed on a company conference call and webcast today, December 6, at 9:00 a.m. ET.

“Inadequate tumor penetration remains a major barrier in treating solid tumors such as R/M HNSCC,” said Claire Mazumdar, PhD, MBA, Chief Executive Officer of Bicara Therapeutics. “Ficerafusp alfa, the first and only bifunctional EGFR-directed antibody x TGF-β ligand trap, was purposefully designed to deliver deep and durable responses with the potential to meaningfully extend overall survival for patients. The data presented today mark an important advancement in our dose-optimization strategy, reinforce our confidence in the interim overall response rate analysis as the foundation for pursuing accelerated approval in the FORTIFI-HN01 pivotal trial, and further elucidate the relative contribution of TGF- β in driving deep and durable tumor responses. We have made significant progress in the FORTIFI-HN01 trial this year and are on track to declare an optimal dose in the first quarter of 2026.”

Phase 1/1b expansion cohort data presented at ESMO Asia show that 750mg ficerafusp alfa in combination with pembrolizumab was generally well-tolerated, with a safety profile consistent with the known safety profile of ficerafusp alfa plus pembrolizumab in R/M HNSCC. At a preliminary duration of follow-up, 750 mg of ficerafusp alfa demonstrated a 57% confirmed overall response rate, with 10% of patients achieving a completed response, and 29% of responders demonstrating deep responses of at least 80% tumor shrinkage.

New biomarker data to be presented during Bicara’s corporate call and webcast show that 1500mg of ficerafusp alfa yielded a greater increase TGF-β inhibition within the tumor microenvironment and greater immune activation, compared to 750mg of ficerafusp alfa. The increased TGF-β inhibition in the tumor translated to greater depth of clinical responses at 24 weeks. The median depth of response was 82% at the 1500mg dose vs. 63% at the 750mg dose, and 64% of responders at 1500mg achieved a deep response, compared to 27% of responders at the 750mg dose.

The totality of the data suggests that a higher dose of ficerafusp alfa with greater TGF-β inhibition and immune activation drives deeper tumor responses that translate to more durable outcomes for patients.

Bicara plans to declare the optimal biologic dose for use in the pivotal FORTIFI-HN01 study in the first quarter of 2026.

Conference Call and Webcast Details
Bicara Therapeutics will host a conference call and webcast today December 6, 2025 at 9:00 a.m. ET. Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and a unique PIN which will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of Bicara’s website under Events and Presentations. An archived replay will also be available for 30 days following the webcast.

About Head and Neck Squamous Cell Carcinoma
Head and neck squamous cell carcinomas (HNSCCs) develop from the mucosal epithelium in the oral cavity, pharynx and larynx and are the most common malignancies that arise in the head and neck. HNSCC is one of the most common cancers in the United States and globally with a rising incidence anticipated to reach one million new global cases annually by 2030. Ten percent of HNSCC patients are diagnosed with metastatic disease and up to 30% develop a recurrence or metastases over time after receiving initial treatment for advanced HNSCC.

Most cases of HNSCC are thought to result from accumulated mutations caused by carcinogenic exposures such as tobacco smoke or HPV infection. Approximately 80% of patients with R/M HNSCC are HPV-negative. These HPV-negative tumors often exhibit a recurrence pattern that is primarily local and are associated with severe morbidities, including fatal tumor bleeding, intense pain, difficulty swallowing, significant weight loss, and cachexia. This highlights a critical unmet need for therapies that have the potential to deliver durable anti-tumor responses, ultimately leading to meaningful improvements in patients’ quality of life.

About Ficerafusp Alfa
Ficerafusp alfa is a first-in-class bifunctional antibody designed to drive tumor penetration by breaking barriers in the tumor microenvironment that have challenged the treatment of multiple solid tumor cancers. Specifically, ficerafusp alfa combines two clinically validated targets: an epidermal growth factor receptor (EGFR) directed monoclonal antibody with a domain that binds to human transforming growth factor beta (TGF-β). Through this targeted mechanism, ficerafusp alfa reverses the fibrotic and immune-excluded tumor microenvironment driven by TGF-β signaling to enable tumor penetration that drives deep and durable responses. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ficerafusp alfa in combination with pembrolizumab for the first line (1L) treatment of patients with metastatic or with unresectable, recurrent (R/M) head and neck squamous cell carcinoma (HNSCC) whose tumors express programmed death-ligand 1 with combined positive score (CPS) ≥1, excluding human papillomavirus (HPV)-positive oropharyngeal squamous cell carcinoma.

Ficerafusp alfa is currently being evaluated in FORTIFI-HN01, a pivotal Phase 2/3 clinical trial in patients with 1L R/M HNSCC.

About Bicara Therapeutics
Bicara Therapeutics is a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors. Bicara’s lead program, ficerafusp alfa, is a first-in-class bifunctional antibody designed to drive tumor penetration by breaking barriers in the tumor microenvironment that have challenged the treatment of multiple solid tumor cancers. Specifically, ficerafusp alfa combines two clinically validated targets: an epidermal growth factor receptor (EGFR) directed monoclonal antibody with a domain that binds to human transforming growth factor beta (TGF-β). Through this targeted mechanism, ficerafusp alfa reverses the fibrotic and immune-excluded tumor microenvironment driven by TGF-β signaling to enable tumor penetration that drives deep and durable responses. Ficerafusp alfa is being developed in head and neck squamous cell carcinoma, where there remains a significant unmet need, as well as other solid tumor types. For more information, please visit www.bicara.com or follow us on LinkedIn and X.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “may,” “might,” “will,” “could,” “would,” “should,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and similar words or expressions, or the negative thereof, are intended to identify forward-looking statements, although not all contain identifying words. Any statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, express or implied statements regarding Bicara’s clinical development of ficerafusp alfa in combination with pembrolizumab and presentation of early data from a Phase 1b expansion cohort evaluating 750 mg of ficerafusp alfa weekly (QW) in combination with pembrolizumab in first-line (1L) human papillomavirus (HPV)-negative recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC), the expected therapeutic potential and clinical benefits of ficerafusp alfa, including potential efficacy and tolerability, and Bicara’s optimal biological dose selection plans. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to uncertainties inherent in the development of product candidates, including the conduct of research activities and the conduct of clinical trials; uncertainties as to the availability and timing of results and data from clinical trials; whether results from prior preclinical studies, preliminary or interim data from earlier stage clinical trials will be predictive of the results of subsequent preclinical studies and clinical trials; regulatory developments in the United States and foreign countries; whether Bicara’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Bicara’s filings with the Securities and Exchange Commission (SEC), including its Annual Report on Form 10-K for the year ended December 31, 2024, its Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 and any subsequent filings Bicara makes with the SEC. In addition, any forward-looking statements represent Bicara’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Bicara explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Contacts

Investors
Jenna Cohen
IR@bicara.com

Media
Amanda Lazaro
1AB
Amanda@1abmedia.com

Aptose’s Tuspetinib Triple Drug Therapy Featured at the 2025 ASH Annual Meeting; High Rate of Frontline Clinical Responses Continues Across AML Populations




Aptose’s Tuspetinib Triple Drug Therapy Featured at the 2025 ASH Annual Meeting; High Rate of Frontline Clinical Responses Continues Across AML Populations

• TUS+VEN+AZA triplet frontline therapy demonstrates high rates of efficacy and MRD-negative remissions in newly diagnosed AML patients with diverse mutations
• Safety continues to be a notable hallmark of TUS-based therapies
• 100% response rate (CR/CRh) at the two higher dose levels (80 and 120 mg TUS dose)
• CR/CRh observed in FLT3 wildtype subjects, representing ~70% of AML patients
• CR/CRh observed in AML with TP53/complex karyotype, RAS, and MDS-related mutations

SAN DIEGO and TORONTO, Dec. 06, 2025 (GLOBE NEWSWIRE) — Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated targeted agents to treat hematologic malignancies, today featured clinical data for its lead compound tuspetinib (TUS) combined with standard dosing of venetoclax (VEN) and azacitidine (AZA) in a poster presentation at the 67^th American Society of Hematology (ASH) Annual Meeting in Orlando, FL. Updated data from patients in the TUSCANY trial across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, reveal promising clinical safety and antileukemic activity and support the use of TUS in combination with standard of care treatment across a broad range of AML populations, including those with adverse mutations regardless of FLT3 mutation status.  

Poster title: “TUSCANY study demonstrates safety and efficacy of tuspetinib plus standard of care venetoclax and azacitidine in patients with newly diagnosed AML ineligible for induction chemotherapy”

Key Findings and Messages:

• In newly diagnosed AML patients, TUS+VEN+AZA shows promising safety, tolerability and resilient efficacy, including MRD-negative remissions across a broad mutational spectrum
• High-quality clinical responses (CR/CRh):
  • 90% across 40, 80 and 120 mg dose levels
  • 100% at the higher 80 mg and 120 mg dose levels
  • Observed in FLT3-WT, FLT3-ITD, and NPM1c genetic subgroups
  • Observed in biallelic TP53/complex karyotype and RAS adverse genetic subgroups
  • Observed in AML with MDS-related mutations
• MRD negativity: 78% by central flow cytometry in responding subjects
• TUS targets VEN resistance mechanisms; inhibits kinase-driven abnormal signaling
• Two subjects transitioned to stem cell transplantation and both returned for TUS maintenance
• TUS+VEN+AZA triplet therapy was well tolerated with no dose-limiting toxicities (DLTs) across all evaluable TUS dose levels
  • No DLTs including no prolonged myelosuppression for subjects in remission in Cycle 1
  • No drug-related deaths, differentiation syndrome, QTc prolongation, or CPK elevation reported
  • 8/10 evaluable subjects experienced red cell and platelet transfusion independence for > 8 weeks after their best response
  • Febrile neutropenia was reported in 2 subjects (16.7%), with 1 subject related to TUS
• At the recently enrolled 160 mg dose level, preliminary findings show patients achieving early blast clearance with MRD-negativity and formal responses in the first few weeks of treatment (not included in poster data cut).

“Tuspetinib, as part of a triple drug therapy, continues to perform well, achieving 100% clinical response in the two higher doses we have evaluated to date,” said Rafael Bejar, MD, PhD, Chief Medical Officer at Aptose. “We recently commenced treating patients at the highest dose level of 160 mg TUS and have already achieved early responses. With no dose-limiting toxicities and activity across diverse mutations, TUS+VEN+AZA targets AML’s greatest unmet needs and largest populations.”

The ASH poster presentation is available here.

About Tuspetinib

Aptose’s lead compound tuspetinib is a convenient once daily oral agent that potently targets SYK, mutated and wild type forms of FLT3, mutated KIT, JAK1/2, and RSK2 kinases, while avoiding many typical toxicity concerns observed with other agents. The ongoing TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine and venetoclax in newly diagnosed patients with AML who are ineligible to receive induction chemotherapy. Data from the first three dose cohorts demonstrate safety, CRs and minimal residual disease (MRD) negativity across patients with diverse mutations. The early data showed that 9 out of 10 patients responded to the TUS triplet therapy, with 100% complete remission (CR/CRh) achieved in the 80mg and 120mg cohorts. Notably, patients with difficult-to-treat mutations in TP53, RAS and FLT3 genes also achieved a 100% CR/CRh rate.

About Aptose

Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company’s small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies without overlapping toxicities. The Company’s lead clinical-stage compound tuspetinib (TUS), is an oral kinase inhibitor that has demonstrated activity as monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com.

Forward Looking Statements

This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements relating to the therapeutic potential of tuspetinib, its clinical development and safety profile including its tolerability and resilient efficacy, as well as statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to obtain the capital required for research and operations and to continue as a going concern; the inherent risks in early stage drug development including demonstrating efficacy; development time/cost and the regulatory approval process; the progress of our clinical trials; our ability to find and enter into agreements with potential partners; our ability to attract and retain key personnel; changing market conditions; inability of new manufacturers to produce acceptable batches of GMP in sufficient quantities; unexpected manufacturing defects; and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission.

Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled “Risk Factors” in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein.

For further information, please contact:

Aptose Biosciences Inc.                        
Susan Pietropaolo                        
Corporate Communications & Investor Relations                        
201-923-2049                        
spietropaolo@aptose.com

Disc Medicine Presents Positive Initial Data from RALLY-MF Phase 2 Trial in Patients with Myelofibrosis (MF) and Anemia at the 67th American Society of Hematology (ASH) Annual Meeting




Disc Medicine Presents Positive Initial Data from RALLY-MF Phase 2 Trial in Patients with Myelofibrosis (MF) and Anemia at the 67th American Society of Hematology (ASH) Annual Meeting

• Demonstrated meaningful overall anemia responses across all patient subgroups, regardless of baseline transfusion status 
• Anemia response was seen independent of concomitant JAK inhibitor therapy use

WATERTOWN, Mass., Dec. 06, 2025 (GLOBE NEWSWIRE) — Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today presented positive initial data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF at the ASH Annual Meeting in Orlando, FL. The data demonstrated that treatment with DISC-0974 resulted in substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types.

“We are excited that we continue to see robust hematologic responses to DISC-0974 regardless of background JAK inhibitor therapy,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. “It is also encouraging to see these hematologic improvements translating into reduced transfusion burden and fatigue. We look forward to advancing this program with the goal of addressing a significant unmet need as anemia is one of the key manifestations of MF and there is currently no therapy approved to treat anemia in this population.”

This ongoing Phase 2 open-label study had enrolled 47 adult patients with MF and anemia as of the data cutoff date of October 16, including 34 patients with sufficient follow up to be included in the responder analysis (non-transfusion dependent receiving no transfusions (nTD, n=24), transfusion dependent with low transfusion burden (TD Low, n=7) and transfusion dependent with high transfusion burden (TD High, n=3)). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=18) and not receiving JAK inhibitor therapy (n=16). DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments. Initial results demonstrated:

• Consistent, substantial decreases in hepcidin reaching >75% reduction from baseline and corresponding increases in serum iron
• 63% of baseline nTD patients achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks (overall response) and 50% had an increase of ≥1.5 g/dL for ≥12 weeks (major response)
• 71% of TD Low patients achieved transfusion independence (TI, major response) over a 16-week period
• 67% of TD High patients with at least 85 days on study achieved a ≥50% reduction in transfusion requirement (overall response)
  • Initial data for additional n=3 TD High patients trending towards major response of TI >12 weeks
• 50% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response
• Dosing with DISC-0974 was associated with improvements in FACIT-Fatigue scores in nTD and TD Low participants
• DISC-0974 was generally well-tolerated. Diarrhea and urinary tract infections, neither considered serious, were the only adverse events (AE) that were considered related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974.
• Additional data to be shared in H2 2026

Disc also shared a poster overviewing the trial design for the ongoing Phase 2 study of the anti-TMPRSS6 antibody DISC-3405 in polycythemia vera requiring frequent phlebotomy.

Management will host a call during the ASH meeting to review highlights of the presented data and plans for next steps in development on Sunday, December 7 at 7:30am EST. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).

About Disc Medicine

Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.

Disc Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its RALLY-MF Phase 2 clinical trial of DISC-0974 in patients with MF and anemia, including the results thereof and the projected timeline for the presentation of additional data. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of Disc’s Annual Report on Form 10-K for the year ended December 31, 2024, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.

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Deerfield Group
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Precision AQ
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Toripalimab Presents Long-Term Survival Benefits as 1st-line Treatment for Advanced Nasopharyngeal Carcinoma and Esophageal Squamous Cell Carcinoma Patients




Toripalimab Presents Long-Term Survival Benefits as 1st-line Treatment for Advanced Nasopharyngeal Carcinoma and Esophageal Squamous Cell Carcinoma Patients

• Long-term OS follow-up analysis of JUPITER-02 demonstrates significantly better and clinically meaningful improvement with toripalimab plus chemotherapy as 1^st-line treatment for R/M NPC, achieving mOS of 64.8 months and a 5-year OS rate of 52.3%.
• Torpalimab plus chemotherapy has been approved in over 40 countries, representing the new standard of care for the 1^st line treatment of R/M NPC.
• The final OS analysis of JUPITER-06 confirms the sustained survival benefit with toripalimab plus chemotherapy in advanced ESCC, showing a mOS of 17.7 months and a 3-year OS rate of 29.7%.
• Results from JUPITER-02 and JUPITER-06 were orally presented at ESMO Asia 2025.

SHANGHAI, Dec. 05, 2025 (GLOBE NEWSWIRE) — Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, announced new and promising long-term survival results from JUPITER-02 (NCT03581786) and JUPITER-06 (NCT03829969) at the European Society for Medical Oncology (ESMO) ASIA Congress 2025. JUPITER-02 and JUPITER-06 evaluated toripalimab, an anti-PD-1 antibody developed by Junshi Biosciences, in combination with chemotherapy as first-line treatment for recurrent or metastatic (R/M) nasopharyngeal carcinoma (NPC) in JUPITER-02 and advanced or metastatic esophageal squamous cell carcinoma (ESCC) in JUPITER-06.

Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences and TopAlliance Biosciences, said: “At ESMO ASIA, we were excited to share the latest long-term survival follow-up data from the Phase 3 JUPITER-02 and JUPITER-06 trials. These remarkable results set new records for the longest survival benefit achieved with immuno-oncology (I-O) therapy in certain NPC and ESCC populations, further cementing toripalimab’s global leadership in immunotherapy for advanced NPC and ESCC. It’s so encouraging to see toripalimab now approved in over 40 countries and regions worldwide, giving more patients access to the new standard of care. We extend our deepest gratitude to the patients, their families, investigators and the R&D teams who made these landmark studies possible.”

JUPITER-02: Study Design and Long-term Overall Survival Follow-up Analysis

JUPITER-02, the first global multicenter, double-blind, randomized Phase 3 trial in immunotherapy for NPC, has reported breakthrough results at multiple international congresses. Its interim and final overall survival (OS) analyses were published in Nature Medicine and the Journal of the American Medical Association (JAMA), respectively, making it the first NPC immunotherapy study featured in JAMA. The JUPITER-02 5-year OS follow-up data was presented at the ESMO Asia 2025, and it demonstrated sustained clinical benefits of toripalimab plus chemotherapy in R/M NPC.

The JUPITER-02 trial enrolled 289 chemotherapy-naïve patients with R/M NPC, randomizing them 1:1 to receive either toripalimab 240 mg (n=146) or placebo (n=143) combined with gemcitabine/cisplatin (GP) chemotherapy every 3 weeks (Q3W) for ≤6 cycles, followed by toripalimab or placebo monotherapy (Q3W) until disease progression, unacceptable toxicity, or 2-year treatment completion. Stratification factors included baseline ECOG status (0 vs. 1) and disease extent (recurrent vs. primary metastatic). The primary endpoint was Blinded Independent Review Committee (BIRC)-assessed progression-free survival (PFS) per RECIST v1.1 in the intent-to-treat (ITT) population. The secondary endpoints included OS (key secondary), investigator-assessed PFS, objective response rate (ORR), safety, etc.

By the data cut-off date, June 24, 2025, the toripalimab arm had demonstrated consistently significant survival improvements with the final OS analysis. Patients on toripalimab achieved a median OS (mOS) of 64.8 months compared to the 33.7 months seen with placebo. This 31.1-month extension in median survival also meant a 39% reduction in death risk (HR=0.62; 95%CI: 0.45-0.85; p=0.0027), with a 5-year OS rate of 52.3% vs. 33.9%. PD-L1 expression subgroup analyses revealed consistent OS improvements regardless of PD-L1 status.

As the first trial to confirm survival benefits with first-line immunotherapy for NPC, JUPITER-02 has now established a transformative global standard for R/M NPC treatment. Toripalimab plus GP chemotherapy is now approved in over 40 countries and endorsed by 3 major international guidelines (CSCO, NCCN, and ESMO), establishing a new benchmark for first-line R/M NPC therapy worldwide.

JUPITER-06: Study Design and Final Analysis

JUPITER-06 is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial designed to evaluate the efficacy and safety of toripalimab combined with TP (paclitaxel + cisplatin) chemotherapy versus placebo plus TP chemotherapy as first-line treatment for advanced or metastatic ESCC. The findings from JUPITER-06 have been presented at major international congresses and published in top-tier journals, including Cancer Cell and the Journal of Clinical Oncology (JCO). Results from the JUPITER-06 final analysis and biomarker evaluation were presented at the ESMO ASIA 2025.

The JUPITER-06 trial enrolled 514 systemic treatment-naïve patients with advanced or metastatic ESCC, randomly assigning 1:1 to receive either toripalimab 240 mg plus TP chemotherapy (n=257) or placebo plus TP chemotherapy (n=257) Q3W for ≤6 cycles, followed by toripalimab or placebo monotherapy Q3W. With stratification by baseline ECOG status (0 vs. 1) and prior radiotherapy history (yes vs. no), the study featured dual primary endpoints of PFS and OS assessed by BICR per RECIST v1.1, alongside secondary endpoints including investigator-assessed PFS, ORR, safety, etc.

By the data cut-off date, February 23, 2023 (median follow-up: 14.2 months), the pre-specified final OS analysis of JUPITER-06 demonstrated that according to the BICR, the toripalimab arm achieved a mOS of 17.7 months versus 12.9 months in the placebo arm, with a 28% reduction in death risk (HR=0.72, 95% CI: 0.58–0.88; P=0.002). The 2-year and 3-year OS rates are 39.1% vs. 27.1% and 29.7% vs. 19.9%, respectively.

Key subgroup analyses showed consistent OS benefits with toripalimab plus chemotherapy across all subgroups. Specifically, the PD-L1 expression subgroup analysis revealed improved OS in the toripalimab arm regardless of PD-L1 expression status.

Long-term follow-up revealed no new safety signals. Treatment-emergent adverse events (TEAEs) related to study treatment were comparable between the toripalimab arm and the placebo arm, with both groups showing a 97.3% incidence rate.

Furthermore, the investigators conducted translational research that, for the very first time, systematically identified predictive biomarkers for the “anti-PD-1 antibody + chemotherapy” combination. The results helped establish the world’s first Esophageal Genomic Immunophenotype Classification (EGIC). This new framework prospectively pinpoints potential precision-targeted strategies for patients who respond poorly to chemo-immunotherapy, paving a practice-changing path for guiding more individualized clinical therapy and developing future combination approaches.

Up till now, toripalimab has gained approval for first-line treatment of advanced ESCC in China, EU, and multiple countries. Notably, it stands as Europe’s first anti-PD-1 antibody approved for 1L advanced ESCC regardless of PD-L1 expression status.

About Toripalimab

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and to induce PD-1 receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells.

More than forty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Europe and Southeast Asia. Ongoing or completed pivotal clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types, including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney, and skin.

In the Chinese mainland, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI^®). Currently, there are twelve approved indications for toripalimab in the Chinese mainland:

1. unresectable or metastatic melanoma after failure of standard systemic therapy;
2. recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least two lines of prior systemic therapy;
3. locally advanced or metastatic urothelial carcinoma that failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy;
4. in combination with cisplatin and gemcitabine as the first-line treatment for patients with locally recurrent or metastatic NPC;
5. in combination with paclitaxel and cisplatin in first-line treatment of patients with unresectable locally advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (ESCC);
6. in combination with pemetrexed and platinum as the first-line treatment in EGFR mutation-negative and ALK mutation-negative, unresectable, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC);
7. in combination with chemotherapy as perioperative treatment and subsequently with monotherapy as adjuvant therapy for the treatment of adult patients with resectable stage IIIA-IIIB NSCLC;
8. in combination with axitinib for the first-line treatment of patients with medium to high risk unresectable or metastatic renal cell carcinoma (RCC);
9. in combination with etoposide plus platinum for the first-line treatment of extensive-stage small cell lung cancer (ES-SCLC);
10. in combination with paclitaxel for injection (albumin-bound) for the first-line treatment of recurrent or metastatic triple-negative breast cancer (TNBC);
11. in combination with bevacizumab for the first-line treatment of unresectable or metastatic hepatocellular carcinoma (HCC) patients;
12. first-line treatment for unresectable or metastatic melanoma.

The first 10 indications have been included in the National Reimbursement Drug List (NRDL) (2024 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for the treatment of melanoma, perioperative treatment of NSCLC, treatment of RCC and treatment of TNBC. Toripalimab for the treatment of advanced NPC and ESCC was approved in Hong Kong SAR, China.

Internationally, toripalimab has been approved for marketing in the United States, the European Union, India, the UK, Jordan, Australia, Singapore, United Arab Emirates, Kuwait, Pakistan, Canada and other countries and regions. In addition, toripalimab BLAs are under review in many countries or regions around the globe.

About Junshi Biosciences

Founded in December 2012, Junshi Biosciences (HKEX: 1877; SSE: 688180) is an innovation-driven biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapeutics. The company has established a diversified R&D pipeline comprising over 50 drug candidates, with five therapeutic focus areas covering cancer, autoimmune, metabolic, neurological, and infectious diseases. Five of the company’s products have received approvals in China and international markets, one of which is toripalimab, China’s first domestically produced and independently developed anti-PD-1 monoclonal antibody. Toripalimab has been approved in over 40 countries and regions including China, the US, and Europe. During the COVID-19 pandemic, Junshi Biosciences actively shouldered the social responsibilities of a Chinese pharmaceutical company through its involvement in developing etesevimab, MINDEWEI^®, and other novel therapies for the prevention and treatment of COVID-19.

With a mission of “providing patients with world-class, trustworthy, affordable, and innovative drugs,” Junshi Biosciences is “In China, For Global.” At present, the company boasts approximately 2,500 employees in the United States (Maryland) and China (Shanghai, Suzhou, Beijing, Guangzhou, etc.). For more information, please visit: http://www.junshipharma.com.

Junshi Biosciences Contact Information

IR Team:

Junshi Biosciences

info@junshipharma.com

+ 86 021-6105 8800

PR Team:

Junshi Biosciences

Zhi Li

zhi_li@junshipharma.com

+ 86 021-6105 8800

Cerevance Showcases Positive Phase 2 Results Demonstrating Significant Reduction in OFF Time with Solengepras as an Adjunctive Treatment in Parkinson’s Disease




Cerevance Showcases Positive Phase 2 Results Demonstrating Significant Reduction in OFF Time with Solengepras as an Adjunctive Treatment in Parkinson’s Disease

• Phase 2 results selected as one of only 10 featured presentations in the Poster Tour at the 2025 Parkinson Study Group (PSG) Annual Meeting
• Pivotal Phase 3 ARISE trial evaluating solengepras as an adjunctive treatment in Parkinson’s disease is underway and actively enrolling patients

BOSTON, Dec. 05, 2025 (GLOBE NEWSWIRE) — Cerevance, a clinical-stage biopharmaceutical company advancing targeted therapies for neurodegenerative diseases and obesity, today presented positive Phase 2 results of solengepras, a novel, non-dopaminergic GPR6 inhibitor in development as an adjunctive treatment for Parkinson’s disease. The findings demonstrated that four weeks of once-daily treatment with solengepras significantly reduced OFF time (periods of symptomatic worsening) by decreasing both the number and duration of OFF episodes, including morning OFF time.

The data was presented today by Harini Sarva, M.D., trial investigator and associate professor of clinical neurology at Weill Cornell Medicine in New York, during the 2025 Parkinson Study Group (PSG) Annual Meeting in San Diego. The presentation was selected as one of only 10 featured posters in this year’s Poster Tour.

“More than 90% of individuals with Parkinson’s disease who have received carbidopa/levodopa therapy for over 5 years experience motor fluctuations, including daily OFF time,” said Dr. Sarva. “These OFF episodes, which can cause a return of Parkinson’s motor symptoms, can be very disabling. The significant and clinically meaningful reductions in OFF time observed with solengepras highlight the potential of this once-daily, oral treatment to improve quality of life. Remarkably, the magnitude of benefit is clinically meaningful and may provide patients with a non-invasive alternative to other therapies.”

Phase 2 Results

The randomized, double-blind Phase 2 study evaluated solengepras as an adjunctive treatment in adults with Parkinson’s disease experiencing motor fluctuations. Key findings include:

• Primary efficacy endpoint: Solengepras 150mg achieved a statistically significant 1.3-hour reduction in average daily OFF time versus placebo after 27 days of treatment (p=0.02).
• In a subgroup of patients with 3 or more hours of baseline OFF time (88% of study participants), a normalized regulatory analysis showed that solengepras 150 mg:
  • Reduced OFF time by 1.78-hours versus placebo at Day 27 (p=0.0045).
  • Improved total daily OFF time by 34.7% from baseline versus placebo (p=0.0026).
  • Decreased the number of OFF periods per day by 0.98 episodes versus placebo (p=0.0026).
  • Reduced the duration of each OFF period by 26 minutes versus placebo (p=0.0958).

Solengepras was well tolerated, with a low incidence of dopaminergic adverse events.

“Despite being the fastest-growing neurological disorder worldwide, Parkinson’s disease has seen limited therapeutic innovation in the past 25 years,” said Craig Thompson, chief executive officer of Cerevance. “These data reinforce solengepras’ potential as a differentiated, non-dopaminergic approach. With the Phase 3 ARISE trial now underway, we are one step closer to bringing this treatment option to patients in need.”

Solengepras is being evaluated in the global, Phase 3 ARISE trial as a once-daily, oral treatment for use as an adjunctive therapy to levodopa and other anti-Parkinsonian medications. Topline data are expected in 2026.

About Solengepras (CVN424)
Solengepras is designed to provide a potentially novel approach to the treatment of Parkinson’s disease. Unlike dopaminergic therapies, which primarily act by replenishing, enhancing, or mimicking dopamine, solengepras is designed to selectively address the indirect pathway by modulating the GPR6 receptor. By inhibiting GPR6, solengepras aims to restore both motor and non-motor function without directly affecting dopamine levels or signaling, improving the relative balance between the direct and indirect pathways, and potentially reducing the risk of common side effects associated with dopaminergic therapies, such as dyskinesias and motor fluctuations. Solengepras is currently being evaluated as a once-daily, oral treatment for use as an adjunctive therapy to levodopa and other anti-Parkinsonian medications in the Phase 3 ARISE trial.

About the Pivotal Phase 3 ARISE Trial
The multicenter, randomized, double-blind, placebo-controlled Phase 3 ARISE trial is evaluating the efficacy and safety of solengepras as an adjunctive therapy to levodopa and other background Parkinson’s disease medications. The trial is enrolling approximately 330 patients with Parkinson’s disease age 30 and older with motor fluctuations who have an average of three or more hours of total OFF time per day. Study participants are randomized to solengepras 75 mg or 150 mg or placebo once daily for 12 weeks. The primary endpoint is the change from baseline to week 12 in average daily OFF time for solengepras 150 mg versus placebo. Secondary objectives include assessing safety and tolerability and further characterizing the effect of solengepras on other motor symptoms (e.g., ON time), non-motor symptoms (e.g., daytime sleepiness), cognitive function, and several quality-of-life measures (e.g., Movement Disorder Society-UPDRS, PD Questionnaire 39, Clinical Global Impression scale, Patient Global Impression scale). ARISE is being conducted globally at sites in the United States and Europe.

About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disorder that is characterized by both motor symptoms, such as tremor, rigidity, and bradykinesia/akinesia, and non-motor symptoms, such as mood changes, apathy, and cognitive deficits. Globally, Parkinson’s disease is the fastest growing neurological disorder, affecting more than 10 million people worldwide and approximately 1 million people in the United States. The current standard of care has primarily relied on dopaminergic therapies, such as levodopa, which lose effectiveness over time and are associated with side effects that result in challenging risk-benefit profiles.

About Cerevance
Cerevance is focused on advancing cell type-specific therapies for the treatment of neurodegenerative diseases and obesity. Our proprietary platform, Nuclear Enriched Transcript Sort sequencing (NETSseq), allows us to identify targets that are expressed at very low levels, that are present in rare cell types, or that change over time as a disease progresses. Our most advanced investigational treatment, solengepras, is currently in Phase 3 development and has the potential to be a first-in-class, oral non-dopaminergic therapy for both motor and non-motor symptoms of Parkinson’s disease. Our second investigational treatment, CVN293, is a highly selective investigational oral inhibitor targeting potassium two pore domain channel subfamily K member 13 (KCNK13). CVN293 represents a potentially novel intervention point for neurodegenerative disorders and obesity. For more information, please visit www.cerevance.com and follow us on LinkedIn and X.

Contacts 

Cerevance: 
Johnna Simões, ir@cerevance.com

Media:
April Dovorany, adovorany@realchemistry.com

ORIC® Pharmaceuticals Presents Potential Best-in-Class Profile for Enozertinib with Robust Systemic and CNS Activity in 1L and 2L NSCLC Patients with EGFR Exon 20 Mutations at the ESMO Asia Congress 2025




ORIC® Pharmaceuticals Presents Potential Best-in-Class Profile for Enozertinib with Robust Systemic and CNS Activity in 1L and 2L NSCLC Patients with EGFR Exon 20 Mutations at the ESMO Asia Congress 2025

Highly differentiated 1L preliminary systemic activity of 67% ORR and 100% intracranial ORR (by BICR-RANO), including in patients with active brain metastases

45% ORR in 2L patients exceeds competitor benchmarks

Competitive safety profile, with no significant off-target toxicity and manageable on-target toxicity, resulting in low discontinuation rate

Enrollment and follow-up continue in 1L patients at selected dose of 80 mg once daily,
with next update expected mid-2026 ahead of initiation of potential Phase 3 trial

Company to host a conference call and webcast on Saturday, December 6, 2025, at 8:00 pm ET

SOUTH SAN FRANCISCO, Calif. and SAN DIEGO, Dec. 05, 2025 (GLOBE NEWSWIRE) — ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, announced data from a Phase 1b trial of enozertinib (ORIC-114) at the ESMO Asia Congress 2025. Data in treatment-naïve and in previously treated NSCLC patients with EGFR exon 20 mutations were presented at an oral session, and the presentation can be found in the publication section of ORIC’s website here.

“Enozertinib was purposefully designed to be highly brain-penetrant and exquisitely selective in order to address the limitations of available therapies and potentially drive differentiated durability. These data provide clinical support for this design approach, demonstrating strong systemic and CNS activity in NSCLC patients with EGFR exon 20 mutations,” said Pratik S. Multani, M.D., chief medical officer. “The profile we have seen with enozertinib compares favorably to other approved and investigational therapies and continues to support enozertinib’s best-in-class potential.”

Enozertinib Phase 1b Trial Design
Enozertinib is being evaluated in a Phase 1b trial in patients with locally advanced or metastatic NSCLC with EGFR exon 20 mutations. Notably, enrollment allows patients with active untreated brain metastases. Prior therapy in 2L patients consisted of platinum-based chemotherapy. The primary endpoint of the trial is to determine the recommended Phase 2 dose (RP2D), and secondary endpoints include investigator-assessed objective response rate (ORR), CNS response rate by blinded independent central review (BICR) using response assessment in neuro-oncology (RANO) criteria in treatment-naïve patients, disease control rate (DCR), and safety.

2L NSCLC Patients with EGFR Exon 20 Mutations
As of the August 29, 2025 cutoff date, 45 2L patients were dosed — 24 patients received 80 mg QD oral enozertinib and 21 patients received 120 mg QD. Brain metastases were present in 38% of patients at baseline, including those with active brain metastases.

Preliminary Safety Analysis
Enozertinib was well tolerated with mostly Grade 1 or 2 treatment-related adverse events (TRAEs) and no significant off-target toxicities. Most frequent TRAEs included diarrhea, paronychia, and stomatitis. Only 3 patients discontinued treatment due to TRAEs and higher rates of dose reductions occurred in the 120 mg cohort compared to the 80 mg cohort.

Preliminary Activity Analysis
In the 20 efficacy evaluable patients in the 80 mg cohort, enozertinib demonstrated strong systemic and CNS antitumor activity.

• 45% confirmed ORR and 100% DCR, with comparable rates in patients with brain metastases at baseline
• As of the data cutoff (at a median follow-up of over 30 weeks), 67% of responders remained on treatment

1L NSCLC Patients with EGFR Exon 20 Mutations
As of the August 29, 2025 cutoff date, 33 1L patients were dosed — an initial cohort of 15 patients received 120 mg QD oral enozertinib and a subsequent cohort of 18 patients received 80 mg QD. Brain metastases were present in 39% of patients at baseline, including those with active brain metastases.

Preliminary Safety Analysis
Enozertinib was well tolerated with mostly Grade 1 or 2 TRAEs and no significant off-target toxicities. Most frequent TRAEs included diarrhea, paronychia, and stomatitis. Only 2 patients discontinued treatment due to TRAEs.

The initial cohort of efficacy-evaluable patients was treated at 120 mg QD and 80% of these patients underwent early dose reductions due to TRAEs, such that most of these patients received an effective dose of 80 mg QD. The subsequent cohort of patients was treated at 80 mg QD, for which follow-up is still in progress.

Preliminary Activity Analysis
In the 15 efficacy-evaluable patients in the 120 mg cohort, the majority of which received an effective dose of 80 mg QD, enozertinib demonstrated strong systemic and CNS antitumor activity.

• 67% best ORR (60% confirmed ORR) and 93% DCR
• 100% confirmed intracranial ORR (by BICR-RANO) in patients with measurable CNS disease
• In 4 patients with non-measurable CNS disease, 2 achieved confirmed intracranial complete responses, both with active untreated brain metastases
• As of the data cutoff (at a median follow-up of 33 weeks), 80% of responders remained on treatment

Next Steps
Based on these data, 80 mg QD oral enozertinib has been selected as the dose for potential Phase 3 development. Enrollment and follow-up continues in 1L EGFR exon 20 patients with the next update expected in mid-2026, ahead of initiation of a potential Phase 3 trial.

Conference Call and Webcast Details
In conjunction with the ESMO Asia Congress, ORIC will host a conference call and webcast on Saturday, December 6, 2025, at 8:00 pm ET. To join the conference call via phone and participate in the live Q&A session, please pre-register online here to receive a telephone number and unique passcode required to enter the call. A live webcast and audio archive of the conference call will be available through the investor section of ORIC’s website at www.oricpharma.com. The webcast will be available for replay for 90 days following the presentation.

About ORIC Pharmaceuticals, Inc.
ORIC Pharmaceuticals is a clinical stage biopharmaceutical company dedicated to improving patients’ lives by Overcoming Resistance In Cancer. ORIC’s clinical stage product candidates include (1) ORIC-944, an allosteric inhibitor of the polycomb repressive complex 2 (PRC2) via the EED subunit, being developed for prostate cancer, and (2) enozertinib (ORIC-114), a brain-penetrant inhibitor that selectively targets EGFR exon 20, EGFR atypical, and HER2 exon 20 mutations, being developed across multiple genetically defined cancers. ORIC has offices in South San Francisco and San Diego, California. For more information, please go to www.oricpharma.com, and follow us on X or LinkedIn.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the continued clinical development of enozertinib; statements regarding the potential best-in-class activity or properties of enozertinib, including antitumor activity that exceeds competitor benchmarks; clinical outcomes from studies of enozertinib, which may materially change as patient enrollment continues or more patient data become available; the development plans and timelines for enozertinib; the potential advantages of enozertinib; plans underlying ORIC’s clinical trials and development; next steps and anticipated program milestones, including timing of program and data updates; and statements by the company’s chief medical officer. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon ORIC’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company; ORIC’s ability to develop, initiate or complete preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates; changes in ORIC’s plans to develop and commercialize its product candidates; the potential for clinical trials of enozertinib or any other product candidates to differ from preclinical, initial, interim, preliminary or expected results; negative impacts of health emergencies, economic instability or international conflicts on ORIC’s operations, including clinical trials; the risk of the occurrence of any event, change or other circumstance that could give rise to the termination of ORIC’s license and collaboration agreements or its clinical trial collaboration and supply agreements; the potential market for ORIC’s product candidates, and the progress and success of competing therapeutics currently available or in development; ORIC’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; ORIC’s reliance on third parties, including contract manufacturers and contract research organizations; ORIC’s ability to obtain and maintain intellectual property protection for its product candidates; the loss of key scientific or management personnel; competition in the industry in which ORIC operates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in ORIC’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the SEC) on November 13, 2025, and ORIC’s future reports to be filed with the SEC. These forward-looking statements are made as of the date of this press release, and ORIC assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law.

Contact:
Dominic Piscitelli, Chief Financial Officer
dominic.piscitelli@oricpharma.com
info@oricpharma.com

Clearmind Medicine Announces Receipt of Nasdaq Minimum Bid Price Notification




Clearmind Medicine Announces Receipt of Nasdaq Minimum Bid Price Notification

Vancouver, Canada, Dec. 05, 2025 (GLOBE NEWSWIRE) — Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“Clearmind” or the “Company”), a clinical-stage biotech company focused on discovery and development of novel neuroplastogen-derived therapeutics to solve major under-treated health problems, announced today that on December 4, 2025, the Company received a written notice from the Nasdaq Stock Market LLC (“Nasdaq”) indicating that the Company was not in compliance with Nasdaq Listing Rule 5550(a)(2), as the Company’s closing bid price for its common shares was below $1.00 per share for the last 30 consecutive business days.

Pursuant to Nasdaq Listing Rule 5810(c)(3)(A), the Company has been granted a 180-calendar day compliance period, or until June 2, 2026, to regain compliance with the minimum bid price requirement. During the compliance period, the Company’s common shares will continue to be listed and traded on the Nasdaq Stock Market under the symbol “CMND”.. To regain compliance, the closing bid price of the Company’s common shares must meet or exceed $1.00 per share for at least 10 consecutive business days during the 180-calendar day compliance period.

If the Company is not in compliance by June 2, 2026, the Company may be afforded a second 180-calendar day compliance period. To qualify for this additional time, the Company will be required to meet the continued listing requirement for market value of publicly held shares and all other initial listing standards for the Nasdaq Capital Market with the exception of the minimum bid price requirement and will need to provide written notice of its intention to cure the deficiency during the second compliance period. If the Company does not regain compliance within the allotted compliance period(s), including any extensions that may be granted by Nasdaq, Nasdaq will provide notice that the Company’s common shares will be subject to delisting.

The Company intends to monitor the closing bid price of its common shares between now June 2, 2026, and will consider available options to resolve the Company’s noncompliance with the minimum bid price requirement as may be necessary. There can be no assurance that the Company will be able to regain compliance with the minimum bid price requirement or will otherwise be in compliance with other Nasdaq listing criteria.

About Clearmind Medicine Inc.

Clearmind is a clinical-stage psychedelic pharmaceutical biotech company focused on the discovery and development of novel psychedelic-derived therapeutics to solve widespread and underserved health problems, including alcohol use disorder. Its primary objective is to research and develop psychedelic-based compounds and attempt to commercialize them as regulated medicines, foods or supplements.

The Company’s intellectual portfolio currently consists of nineteen patent families including 31 granted patents. The Company intends to seek additional patents for its compounds whenever warranted and will remain opportunistic regarding the acquisition of additional intellectual property to build its portfolio.

Shares of Clearmind are listed for trading on Nasdaq under the symbol “CMND” and the Frankfurt Stock Exchange under the symbol “CWY0.”

For further information visit: https://www.clearmindmedicine.com or contact:

Investor Relations
invest@clearmindmedicine.com

Telephone: (604) 260-1566
US: CMND@crescendo-ir.com

General Inquiries
Info@Clearmindmedicine.com
www.Clearmindmedicine.com

Forward-Looking Statements:

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act and other securities laws. Words such as “expects,” “anticipates,” “intends,” “plans,” “believes,” “seeks,” “estimates” and similar expressions or variations of such words are intended to identify forward-looking statements. For example, the Company is using forward-looking statements when it discusses regaining compliance with Nasdaq’s continued listing requirements, and timing and effect thereof. Forward-looking statements are not historical facts, and are based upon management’s current expectations, beliefs and projections, many of which, by their nature, are inherently uncertain. Such expectations, beliefs and projections are expressed in good faith. However, there can be no assurance that management’s expectations, beliefs and projections will be achieved, and actual results may differ materially from what is expressed in or indicated by the forward-looking statements. Forward-looking statements are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in the forward-looking statements. For a more detailed description of the risks and uncertainties affecting the Company, reference is made to the Company’s reports filed from time to time with the Securities and Exchange Commission (“SEC”), including, but not limited to, the risks detailed in the Company’s annual report on Form 20-F for the fiscal year ended October 31, 2024 and subsequent filings with the SEC. Forward-looking statements speak only as of the date the statements are made. The Company assumes no obligation to update forward-looking statements to reflect actual results, subsequent events or circumstances, changes in assumptions or changes in other factors affecting forward-looking information except to the extent required by applicable securities laws. If the Company does update one or more forward-looking statements, no inference should be drawn that the Company will make additional updates with respect thereto or with respect to other forward-looking statements. References and links to websites have been provided as a convenience, and the information contained on such websites is not incorporated by reference into this press release. Clearmind is not responsible for the contents of third-party websites.