Vertex Presents Updated Phase 1/2 Data From RUBY-3 Study That Continue to Demonstrate Best-in-Class Potential for Povetacicept in Adults with IgA Nephropathy and Primary Membranous Nephropathy at American Society of Nephrology Kidney Week




Vertex Presents Updated Phase 1/2 Data From RUBY-3 Study That Continue to Demonstrate Best-in-Class Potential for Povetacicept in Adults with IgA Nephropathy and Primary Membranous Nephropathy at American Society of Nephrology Kidney Week

– 48-week data show a 64% decrease from baseline in proteinuria in IgA nephropathy, 82% decrease from baseline in proteinuria in primary membranous nephropathy, and stabilization of estimated glomerular filtration rate across both diseases –

– Vertex on track to initiate rolling submission of Biologics License Application for potential accelerated approval to the U.S. Food and Drug Administration this year; full enrollment completed for Phase 3 RAINIER trial in IgA nephropathy –

– Povetacicept in primary membranous nephropathy granted Fast Track Designation by the U.S. Food and Drug Administration and Phase 2/3 pivotal trial initiated –

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced updated data for povetacicept (pove) in IgA nephropathy (IgAN) and primary membranous nephropathy (pMN) from the ongoing RUBY-3 trial at the American Society of Nephrology (ASN) Kidney Week 2025 in Houston, Texas. Pove is an investigational recombinant fusion protein therapeutic and dual inhibitor of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines. Pove is the only BAFF+APRIL inhibitor in pivotal trials for multiple kidney diseases.

Results were presented today as a late-breaking oral presentation (SA-OR091) and included interim data from the open-label Phase 1/2 RUBY-3 trial, where adults with IgAN and pMN received pove subcutaneously every 4 weeks. The analysis included 21 participants with IgAN and 10 participants with pMN treated with pove at the 80mg dose, of which 17 participants and 5 participants, respectively, completed the Week 48 study visit.

Results in IgAN

In IgAN, key efficacy findings for the pove 80mg cohort at 48 weeks showed a 64% decrease from baseline in mean 24-hour urine protein to creatinine ratio (UPCR), estimated glomerular filtration rate (eGFR) stabilization with change from baseline in eGFR (mean±SE) of 3.3±3.1 mL/min/1.73m², 90% (9/10) of participants achieving hematuria resolution (defined as a decrease to negative or small levels of urine blood in participants with baseline levels of urine blood of moderate or large), and 53% of participants achieving clinical remission (defined as UPCR <0.5 g/g, negative hematuria, and <25% reduction in eGFR vs. baseline).

Results in pMN

In pMN, key efficacy findings for the pove 80mg cohort at 48 weeks showed an 82% decrease from baseline in mean 24-hour UPCR, eGFR stabilization with change from baseline in eGFR (mean±SE) of -0.3±3.4 mL/min/1.73m², and 40% of participants achieving complete clinical remission (defined as UPCR <0.5 g/g).

Pove was generally safe and well tolerated with adverse events (AEs) that were mostly mild or moderate in severity. There were no serious adverse events related to povetacicept. The safety data is consistent with previous interim analyses, and the safety profile is similar between the IgAN and pMN cohorts.

“These impressive data demonstrate the viability of BAFF+APRIL inhibition to transform the treatment of serious kidney diseases such as IgAN and pMN, important areas of high unmet need,” said RUBY-3 Principal Investigator James Tumlin, M.D., Professor, Department of Medicine, Emory University School of Medicine, and Director of Clinical Research at Georgia Nephrology. “In IgAN, it is especially encouraging to see that at 48 weeks of follow up, a full two-thirds of the participants treated with pove achieved a complete response as defined by UPCR <0.5 g/g, which is in line with the most recent KDIGO guidelines.”

“The exceptionally fast pace of enrollment for the Phase 3 RAINIER trial demonstrates the unmet demand to find effective interventions in IgAN,” said RAINIER Steering Committee Member Richard Lafayette, M.D., Professor of Medicine (Nephrology) at the Stanford University Medical Center. “People living with IgAN and pMN indeed need additional disease-modifying treatments that address the specific drivers of nephron loss and will provide lasting disease control. Pove’s data from RUBY-3 in two serious kidney diseases support the utility of a dual BAFF+APRIL approach, and we are increasingly excited awaiting the RAINIER Phase 3 results.”

Next Steps for Pove Development

Vertex recently announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for pove in IgAN, and the Company expects to submit the first module of the Biologics License Application (BLA) rolling submission this year for potential accelerated approval. Vertex has notified the FDA of its intent to use a priority review voucher to expedite the review of the pove BLA in IgAN from ten months to six months. The Phase 3 RAINIER study is now fully enrolled.

Vertex also received Fast Track Designation from the FDA for pove in pMN, and recruitment for the pivotal Phase 2/3 OLYMPUS trial is currently underway. pMN is the second indication in which pove has demonstrated best-in-class potential.

Investor Event

Vertex will host an investor event at 7:00 p.m. CST (8:00 p.m. EST) in Houston to discuss the updated data for pove in IgAN and pMN and other highlights across its kidney disease portfolio. A live webcast of the presentation and Q&A portions can be accessed through the Investor Relations section of Vertex’s website at https://investors.vrtx.com/. An archived webcast will be available on the company’s website.

Visit news.vrtx.com/asn-kidney-week for more information about Vertex’s presence at ASN Kidney Week 2025.

About Povetacicept (Pove)

Pove is a dual inhibitor of the BAFF and APRIL cytokines, which promote B cell activation, differentiation and/or survival, and provides B cell control by inhibiting the ability of BAFF and APRIL to drive the pathogenesis of multiple autoimmune diseases. Due to its engineered TACI domain, pove has demonstrated greater binding affinity, potency and/or tissue penetration compared to other APRIL, BAFF, and dual BAFF+APRIL inhibitors in preclinical studies. This preclinical data, combined with clinical data observed to date and convenient dosing and administration, give pove best-in-class potential across a number of serious autoimmune diseases driven by uncontrolled B cells. Pove is an investigational agent and has not been approved by health authorities globally.

About IgA Nephropathy (IgAN)

IgAN is a serious, progressive, life-threatening kidney disease driven by uncontrolled autoreactive B cell activity and is the most common cause of primary glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan and approximately 750,000 diagnosed patients in China. IgAN results from the deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years of diagnosis. There are no approved therapies that specifically target the underlying cause of IgAN.

About Primary Membranous Nephropathy (pMN)

pMN is a rare and serious autoimmune glomerular disease, which is driven by uncontrolled autoreactive B cell activity, resulting in autoantibody production against glomerular antigens including protein phospholipase A2 receptor (PLA2R). pMN affects approximately 150,000 people in the United States and Europe. Over-production of these autoantibodies against glomerular antigens results in kidney damage, fibrosis, and renal failure. There are no therapies specifically approved for the treatment of pMN.

About Fast Track Designation

Fast Track Designation is a program administered by the FDA to expedite the development and review of new drugs and biologics that treat serious or life-threatening conditions and have the potential to fill unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs.

About Breakthrough Therapy Designation

The FDA’s BTD is intended to expedite development and review of medicines that aim to address a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing treatments on one or more clinically significant endpoints. BTD was granted for pove in IgAN based on data from the Phase 2 RUBY-3 clinical trial.

About RUBY-3

RUBY-3 is an ongoing, multiple-ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, pMN, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis, where povetacicept is being administered subcutaneously for up to 104 weeks.

About RAINIER

RAINIER is a global Phase 3 randomized, placebo-controlled pivotal trial of pove 80 mg administered subcutaneously every four weeks vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating the percent change from baseline in urine protein to creatinine ratio (UPCR) for the pove arm vs. placebo after a pre-specified number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total estimated glomerular filtration rate (eGFR) slope through Week 104. The RAINIER study design was presented as a poster (FR-PO0813) during ASN Kidney Week.

About OLYMPUS

OLYMPUS is a global Phase 2/3 adaptive, randomized, active-controlled pivotal trial of pove in approximately 176 patients with pMN. In the Phase 2 portion, participants will be randomized to receive one of two different doses of pove and after the last participant completes 12 weeks of treatment, the Phase 3 dose will be selected. In the Phase 3 portion, participants will be randomized to receive either the selected dose of pove or a calcineurin inhibitor. Final analysis will occur at two years of treatment, with a primary endpoint of proportion of participants with complete clinical remission at Week 104.

About Vertex

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

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

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by James Tumlin M.D., and Richard Lafayette, M.D., and statements about the expectations for the Company’s BLA submission for pove in IgAN for potential accelerated approval in the U.S., including expectations for the timing of the submission of the first module and the completion of the full BLA submission, expectations for pove’s best-in-class potential in IgAN and pMN and pipeline-in-a-product potential across a range of diseases, clinical status of and expectations for the OLYMPUS Phase 2/3 trial in pMN, plans for an investor event to discuss updated data for pove in IgAN and pMN and other highlights across the Company’s kidney disease portfolio, expectations for the RAINIER study design and data expectations, including timing of data availability, and expectations that the Company will seek accelerated approval in the U.S., if the RAINIER interim analysis is positive. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that clinical trial data might not be available on the expected timeline, that data from the company’s research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks, that the company may be unable to make the anticipated regulatory submissions on the expected timeline, or at all, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company’s website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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New Post Hoc Analysis from the HELIOS-B Phase 3 Study Shows Vutrisiran Improved Measures of Heart Structure and Function in Patients with ATTR-CM




New Post Hoc Analysis from the HELIOS-B Phase 3 Study Shows Vutrisiran Improved Measures of Heart Structure and Function in Patients with ATTR-CM

− Analyses Presented at the American Heart Association Scientific Sessions 2025 Underscore Vutrisiran’s Differentiated Profile –

− Cardiovascular Magnetic Resonance (CMR) and Echocardiographic Analyses Demonstrate that Treatment with Vutrisiran Resulted in Significant Changes on Multiple Functional and Structural Cardiac Parameters –

– In a Cohort of HELIOS-B Patients, CMR Imaging Showed Amyloid Regression in 22% of Vutrisiran Treated Patients with No Regression Found in Patients Who Received Placebo –

– Treatment with Vutrisiran Preserved Kidney Function in HELIOS-B Patients, and Reduced Risk of Death and Cardiovascular Events in Patients with Advanced Chronic Kidney Disease –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results from new post hoc analyses of the HELIOS-B Phase 3 study of AMVUTTRA^® (vutrisiran), an RNAi therapeutic approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Data from cardiovascular magnetic resonance (CMR) and echocardiographic imaging as well as renal function analyses were presented at the American Heart Association (AHA) Scientific Sessions 2025 in New Orleans, Louisiana.

As a multisystem disease, ATTR-CM can impact the health and function of organs throughout the body. These analyses evaluated the effect of vutrisiran on critical measures of heart function and structure as well as kidney health, providing deeper insight into its potential impact on key organs affected by the disease.

HELIOS-B Analyses: Cardiac Structure, Function, and Amyloid Burden

In ATTR-CM, amyloid deposits in the heart lead to thickening and stiffening of the ventricles, affecting how well the heart functions and increasing the risk of heart failure and death. Advanced imaging techniques offer insights into structural and functional changes, capturing the overall burden of amyloid deposits and their impact on cardiac function.

• A CMR analysis evaluated a retrospectively identified cohort of HELIOS-B patients from the National Amyloidosis Centre in London who underwent serial CMR scans as part of their routine clinical care at baseline (N=43), Year 1 (N=39), Year 2 (N=26), and Year 3 (N=17).
  • A mixed model analysis that pooled 24- and 36-month data found that treatment with vutrisiran monotherapy was associated with nominally statistically significant and directionally favorable changes in multiple measures of cardiac structure, function, and amyloid burden, including improvements in left and right ventricular ejection fractions as well as stroke volumes and left ventricular mass, compared to placebo.
  • Treatment with vutrisiran also reduced extracellular volume (ECV), which is thought to reflect amyloid buildup in the heart. At Year 3, amyloid regression, as assessed by ECV, was observed in 22% of patients treated with vutrisiran, while no patients who received placebo showed regression; conversely, progression occurred in 63% of patients who received placebo compared to 11% of patients treated with vutrisiran.
  • At Year 3, patients treated with vutrisiran exhibited an absolute mean (standard deviation) reduction in ECV of -0.10% (± 4.72) versus an increase of 7.86% (± 5.67) in the placebo group (p=0.006).
• To further assess cardiac function in patients with ATTR-CM, echocardiographic parameters including left atrial strain (LAS) and right ventricular free wall strain (RVFWS) were evaluated in analyses of the overall HELIOS-B population.
  • The LAS analysis demonstrated that measures of LAS were associated with baseline disease severity, mortality, and cardiovascular (CV) events, and that patients treated with vutrisiran experienced less worsening in LAS at 30 months, compared to those receiving placebo.
  • The RVFWS analysis showed that worse RVFWS was associated with higher risk of adverse outcomes and that treatment with vutrisiran stabilized RVFWS at 30 months, compared to placebo.

“As ATTR-CM progressively damages the heart muscle, making it thicker, stiffer, and less capable of pumping blood effectively, patients experience worsening symptoms, quality of life, and outcomes,” said Marianna Fontana, M.D., Ph.D., HELIOS-B investigator, Professor of Cardiology, University College London, National Amyloidosis Center, Royal Free Hospital, London. “The HELIOS-B CMR analysis provides compelling initial evidence that treatment with vutrisiran can counteract these changes in some patients, as shown by favorable impacts on measures of cardiac structure and function as well as regression of amyloid burden. These results suggest that vutrisiran has the potential not only to slow disease progression, but to reverse some of the structural damage caused by amyloid deposition in the heart in some patients.”

HELIOS-B Analysis: Renal Function and CV Outcomes in Patients with Severe Chronic Kidney Disease (CKD)

Kidney involvement is common in patients with ATTR-CM and is associated with disease progression along with worse patient outcomes as amyloid deposits in the kidneys can lead to a decline in kidney function, indicated by a decreasing estimated glomerular filtration rate (eGFR).

• A post hoc analysis of the HELIOS-B study evaluated the impact of vutrisiran on renal function and assessed the efficacy and safety of vutrisiran in patients who advanced to severe CKD during the double-blind period. Outcomes, including the composite endpoint of all-cause mortality (ACM) and recurrent CV events, as well as safety, were assessed in patients who advanced to CKD Stage 4 or greater (eGFR < 30 mL/min/1.73 m²) in the overall population, vutrisiran monotherapy population, and baseline tafamidis treatment group.
  • In the overall population, treatment with vutrisiran resulted in fewer patients experiencing ≥40% decreases in eGFR from baseline during the double-blind period, compared to placebo (12.7% vs 21.2%).
  • In patients in the overall population who progressed to CKD Stage 4 or greater during the double-blind period, treatment with vutrisiran reduced the risk of ACM and CV events (hazard ratio [HR] 0.47; 95% confidence interval [CI]: 0.26–0.85), compared to placebo.
  • In both analyses, results observed in patients who progressed to CKD Stage 4 or greater were consistent across the overall population, vutrisiran monotherapy, and baseline tafamidis treatment groups, and were also consistent with vutrisiran’s established efficacy profile from the primary HELIOS-B analysis.
  • The safety profile of vutrisiran in patients who advanced to CKD Stage 4 or greater was comparable with that established in the overall population of the HELIOS-B study.

“The new HELIOS-B analyses add depth to the growing body of evidence supporting AMVUTTRA’s first-line potential in ATTR-CM treatment,” said John Vest, M.D., Senior Vice President, TTR Global Clinical Lead, Alnylam. “From heart structure and function to renal health, the benefits observed are consistent across a wide range of measures and analyses, underscoring the potential for protection of the multiple organs impacted by this systemic disease. These findings build on the broadening dataset from HELIOS-B and continue to support the potential of AMVUTTRA to provide meaningful clinical benefit for patients living with this rapidly progressive multisystem disease.”

Data from the HELIOS-B study supported the approvals of AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM in adults in the United States (US), Brazil, European Union (EU), Japan, United Arab Emirates (UAE), United Kingdom (UK), and Switzerland. AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of transthyretin, addressing the disease at its source, with four subcutaneous doses per year. Collectively, AMVUTTRA has more than 8,000 patient-years of experience worldwide and is the first RNAi therapeutic approved for the treatment of both ATTR-CM and hATTR-PN in adults.

For additional information on Alnylam’s presentations in ATTR-CM at the AHA 2025 Scientific Sessions, please visit Capella.

Indications and Important Safety Information

Indications Approved by the U.S. FDA

AMVUTTRA^® (vutrisiran) is indicated for the treatment of the:

• cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
• polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA treatment leads to a decrease in serum vitamin A levels.

Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

In a study of patients with ATTR-CM, no new safety issues were identified.

For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025)

About AMVUTTRA

AMVUTTRA^® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, EU, UK, Brazil, Japan, Switzerland, and UAE. In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR).

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.^1-4

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.⁵ Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.⁶ By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made.⁵ This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products include AMVUTTRA^® (vutrisiran), ONPATTRO^® (patisiran), GIVLAARI^® (givosiran), and OXLUMO^® (lumasiran), which are being developed and commercialized by Alnylam, and Leqvio^® (inclisiran) and Qfitlia^™ (fitusiran), which are being developed and commercialized by Alnylam’s partners, Novartis and Sanofi, respectively. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran as a treatment for ATTR-CM, including vutrisiran’s impact on measures of cardiac structure and function; the potential for treatment with AMVUTTRA to result in regression of amyloid burden in some patients; the potential of AMVUTTRA to slow disease progression and to reverse some of the structural damage caused by amyloid deposition in the heart in some patients; the potential impact of vutrisiran on key organs affected by ATTR-CM; AMVUTTRA’s potential to be a first-line treatment for ATTR-CM; AMVUTTRA’s potential to protect multiple organs impacted by ATTR-CM and to deliver meaningful clinical benefit for patients living with ATTR-CM; and Alnylam’s ability to execute on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P⁵x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; the possibility of unfavorable new clinical data and further analyses of existing clinical data; interim and preliminary data; the possibility that clinical data are subject to differing interpretations and assessments by regulatory agencies; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

References

¹ Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.

² Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.

³ Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.

⁴ Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

⁵ Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.

⁶ Zamore P. Cell. 2006;127(5):1083-1086.

Contacts

Alnylam Pharmaceuticals, Inc.

Christine Akinc

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Josh Brodsky

(Investors)

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Merck’s Enlicitide Decanoate, an Investigational Oral PCSK9 Inhibitor, Significantly Reduced LDL-C in Phase 3 CORALreef Lipids Trial




Merck’s Enlicitide Decanoate, an Investigational Oral PCSK9 Inhibitor, Significantly Reduced LDL-C in Phase 3 CORALreef Lipids Trial

Enlicitide, designed to deliver antibody-like efficacy, has the potential to be the first approved oral PCSK9 inhibitor to lower LDL-C with a safety profile comparable to placebo

Enlicitide may help address unmet needs in ASCVD, a key driver of the ongoing cardiovascular (CV) epidemic

RAHWAY, N.J.–(BUSINESS WIRE)–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the first presentation of results from the pivotal Phase 3 CORALreef Lipids trial demonstrating that treatment with enlicitide decanoate, an investigational, once-daily oral proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, resulted in a statistically significant and clinically meaningful reduction in low-density lipoprotein cholesterol (LDL-C) of 55.8% (primary analysis; 95% CI: -60.9, -50.7; p<0.001) and of 59.7% in a post-hoc reanalysis (95% CI: -62.3, -57.1; p<0.001) compared to placebo at week 24. These late-breaking data will be presented for the first time today at the American Heart Association (AHA) Scientific Sessions 2025 (Abstract #4391578) and were selected for the Late-Breaking Science News Briefing.

In CORALreef Lipids, adults with or at-risk for atherosclerotic cardiovascular disease (ASCVD) on background lipid-lowering therapies or a documented statin intolerance who received once-daily oral enlicitide had statistically significant and clinically meaningful reductions in LDL-C at week 24 (primary endpoint) and statistically significant and sustained reductions in LDL-C through one year (week 52). Enlicitide demonstrated statistically significant reductions in secondary endpoints including non-high-density lipoprotein cholesterol (non-HDL-C), apolipoprotein B (ApoB) and lipoprotein(a) (Lp(a)) at week 24. The overall safety profile was comparable to placebo. High adherence with study intervention (97%) and dosing instructions (≥97%) were observed across treatment groups.

“Enlicitide demonstrated impressive LDL-C reductions with placebo-like safety in the CORALreef Lipids study, underscoring the practice-changing potential of an oral PCSK9 inhibitor,” said Dr. Ann Marie Navar, a lead author of the study and Associate Professor of Medicine in the Division of Cardiology at UT Southwestern Medical Center. “Despite the availability of lipid-lowering therapies such as statins and injectable PCSK9 inhibitors, the majority of patients with atherosclerotic cardiovascular disease do not reach their LDL-C goal. Enlicitide has the potential to help close gaps in achievement of lipid goals in patients with and at risk for cardiovascular events and ultimately help address the ongoing CV epidemic.”

“Enlicitide was designed to deliver PCSK9 antibody-like efficacy and specificity in an easy-to-use pill,” said Dr. Dean Y. Li, president, Merck Research Laboratories. “Enlicitide, if approved, adds to physicians’ armamentarium to lower LDL-C. This moment is the result of Merck’s legacy and commitment to researching ways to help improve ASCVD outcomes for millions worldwide and our strength in medicinal chemistry using our novel macrocyclic peptide platform. Cardiovascular disease is the leading cause of death globally, and we look forward to bringing a potential new option to help address the CV epidemic.”

At one year, enlicitide showed a sustained statistically significant reduction in LDL-C of 47.6% (primary analysis; 95% CI: -52.7, -42.5; p<0.001) and of 52.4% (post-hoc reanalysis; 95% CI: -55.1, -49.7; p<0.001) compared to placebo. At week 24, enlicitide demonstrated reductions in non-HDL-C of 53.4% (95% CI: -55.5, -51.2; p<0.001), ApoB of 50.3% (95% CI: -52.1, -48.5; p<0.001) and Lp(a) of 28.2% (95% CI: -30.3, -26.0; p<0.001) compared to placebo. The study also showed that 67.5% of patients treated with enlicitide achieved the rigorous prespecified goal of at least 50% reduction in LDL-C along with an LDL-C <55 mg/dL (1.42 mmol/L) compared to 1.2% in the placebo arm at week 24.

Enlicitide had a safety profile similar to placebo. There were no apparent differences between the enlicitide and placebo groups in the incidence of any adverse events (AEs), serious AEs or deaths. Discontinuations due to AEs were low and similar between enlicitide (3.1%) and placebo (4.1%).

Merck plans to share data from this trial, along with data from CORALreef HeFH and CORALreef AddOn with regulatory authorities worldwide.

About CORALreef Lipids

CORALreef Lipids (NCT05952856) is a Phase 3 randomized, double-blind, placebo-controlled study designed to evaluate the efficacy, safety and tolerability of enlicitide decanoate in adults with hypercholesterolemia and a history of a major atherosclerotic cardiovascular disease (ASCVD) event or increased risk for a first event. Participants were required to be treated with stable lipid-lowering therapies including at least a moderate or high intensity statin (or have documented statin intolerance). The study enrolled 2,912 participants who were randomized 2:1 to receive either 20mg of once-daily oral enlicitide (n=1,942) or placebo (n=970). The primary endpoints were mean percent change in LDL-C from baseline at week 24 versus placebo, number of participants with one or more AEs, and number of participants who discontinued study drug due to an AE. Multiplicity-controlled secondary efficacy endpoints included: mean percent change from baseline in LDL-C at week 52, mean percent change from baseline in other key atherogenic lipids at week 24 (non-HDL-C, ApoB and Lp(a)).

A post-hoc reanalysis was conducted to revise missing data handling rules that resulted in 5 biologically impossible baseline values being included in the primary analysis. The intent of the reanalysis was to provide a more clinically accurate estimate of the treatment effect.

CORALreef Lipids is the largest completed Phase 3 study evaluating enlicitide in a broad range of participants with elevated LDL-C.

About enlicitide and PCSK9

Enlicitide has the potential to be the first FDA approved oral PCSK9 inhibitor. It is designed to lower LDL-C via the same biological mechanism as currently approved monoclonal antibody, injectable PCSK9 inhibitors but in a daily pill form. Enlicitide is a novel small molecule macrocyclic peptide candidate that binds to PCSK9 and inhibits the interaction of PCSK9 with LDL receptors.

PCSK9 plays a key role in cholesterol homeostasis by regulating levels of the LDL receptor, which is responsible for the uptake of cholesterol into cells. Inhibition of PCSK9 is designed to prevent the interaction of PCSK9 with LDL receptors. This results in greater numbers of LDL receptors available on the cell surface to remove LDL cholesterol from the blood.

About CORALreef Clinical Trial Program

The efficacy and safety profile of enlicitide is being evaluated through the comprehensive CORALreef Clinical Trial program evaluating over 19,000 participants who have hypercholesterolemia. As previously announced, enlicitide demonstrated statistically significant and clinically meaningful reductions in LDL-C in three pivotal Phase 3 studies: CORALreef Lipids (NCT05952856), CORALreef HeFH (NCT05952869) and CORALreef AddOn (NCT06450366). Enlicitide is continuing to be evaluated in the large cardiovascular outcomes trial, CORALreef Outcomes (NCT06008756), which has completed enrollment with over 14,500 participants. Additional CORALreef clinical trials include CORALreef Extension (NCT06492291), CORALreef Pediatric (NCT07058077) and CORALreef Combination (NCT07216482).

About hypercholesterolemia

Hypercholesterolemia, a type of hyperlipidemia, is a disorder in which there are elevated LDL-C levels in the blood. It affects approximately 86 million adults in the U.S. and is a major risk factor for ASCVD. Nearly 70% of people with ASCVD who are treated with lipid-lowering therapies do not reach target LDL cholesterol levels. High LDL-C, if left untreated, can lead to ASCVD events such as heart attacks and strokes.

About the CV epidemic and atherosclerotic cardiovascular disease

The silent CV epidemic is the leading cause of deaths globally, contributing to the majority of heart attacks and strokes, and deaths related to CV continue to rise. ASCVD accounts for 85% of CV deaths. It is caused by the buildup of plaque within the arteries, leading to narrowed or blocked blood vessels that can result in serious CV events such as heart attacks and strokes as well as coronary artery disease, peripheral artery disease and cerebrovascular disease.

Merck’s focus on cardiovascular disease

Merck has a long history of developing treatments for cardiovascular disease. Nearly 70 years ago, we introduced our first cardiovascular therapy—and our scientific efforts to understand and treat cardiovascular-related disorders have continued. Cardiovascular disease continues to be one of the most serious health challenges of the 21st century and is the leading cause of death worldwide. Approximately 18 million people across the globe die from cardiovascular disease every year; in the United States, one person dies every 36 seconds from cardiovascular disease.

Advancements in the treatment of cardiovascular disease can make a critical difference for patients and health systems around the world. At Merck, we strive for scientific excellence and innovation in all stages of research, from discovery through approval and life cycle management. We work with experts throughout the cardiovascular and pulmonary community to advance research that can help improve the lives of patients globally.

Information for other currently enrolling cardiovascular studies can be found by visiting: https://www.merckclinicaltrials.com/cardiovascular.

About Merck

At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA

This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov). 

Contacts

Media Contacts:

Julie Cunningham

(617) 519-6264

Justine Moore

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Investor Contacts:

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Ayn Wisler

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Groundbreaking pivotal study results of olezarsen for severe hypertriglyceridemia (sHTG) presented as a late breaker at AHA Scientific Sessions




Groundbreaking pivotal study results of olezarsen for severe hypertriglyceridemia (sHTG) presented as a late breaker at AHA Scientific Sessions

– Up to 72% placebo-adjusted mean reduction in fasting triglyceride levels at six months, with reductions sustained through 12 months –

– 86% of olezarsen-treated patients achieved triglyceride levels less than 500 mg/dL, below the risk threshold for acute pancreatitis –

– First and only investigational treatment for sHTG to significantly reduce acute pancreatitis events –

– Data simultaneously published in The New England Journal of Medicine –

– Ionis to host webcast today at 3:00 p.m. ET –

CARLSBAD, Calif.–(BUSINESS WIRE)–Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). The studies met the primary endpoint, with olezarsen achieving a highly statistically significant placebo-adjusted mean reduction in fasting triglyceride (TG) levels of up to 72% at six months. The reductions were sustained through 12 months. Olezarsen showed a highly statistically significant 85% reduction in acute pancreatitis events, the first and only time achieved in sHTG. Additionally, 86% of olezarsen-treated patients achieved triglyceride levels less than 500 mg/dL, below the risk threshold for acute pancreatitis. Olezarsen demonstrated favorable safety and tolerability.

These data were presented today during a late-breaking session at the American Heart Association (AHA) Scientific Sessions, taking place November 7-10 in New Orleans, and simultaneously published in The New England Journal of Medicine.

“CORE and CORE2 are the first studies to show a significant reduction in acute pancreatitis events in sHTG, with most patients on olezarsen achieving triglyceride levels below the risk threshold for these potentially life-threatening episodes,” said Nicholas Marston, M.D., M.P.H, presenting author, cardiologist, Brigham and Women’s Hospital, Harvard Medical School. “As a lipid specialist who takes care of sHTG patients, I have seen the major consequences of acute pancreatitis, including cases with recurrent events requiring frequent hospitalizations. Given the modest effects of conventional therapies, these impactful data are a welcome advance and underscore the potential of olezarsen to transform the way we treat sHTG.”

Nearly 1,100 patients were enrolled in the CORE and CORE2 studies, which is the largest pivotal program ever conducted in sHTG, and patients were required to be on standard of care lipid-lowering therapy. The CORE and CORE2 studies met the primary endpoint across doses, with olezarsen demonstrating an up to 72% (p<0.001) placebo-adjusted mean reduction in fasting triglyceride levels at six months. The reductions were sustained through 12 months. Additionally, among patients with baseline levels above these thresholds at 12 months:

• TGs <880 mg/dL: 89% and 88% of patients on olezarsen 50 mg and 80 mg, respectively, achieved triglyceride levels less than 880 mg/dL, the level associated with the highest risk of acute pancreatitis.
• TGs <500 mg/dL: 86% of patients on olezarsen 50 mg and 80 mg achieved triglyceride levels less than 500 mg/dL, below the risk threshold for sHTG and acute pancreatitis.
• TGs <150 mg/dL: 34% and 54% of patients on olezarsen 50 mg and 80 mg, respectively, achieved normal triglyceride levels less than 150 mg/dL.

Olezarsen demonstrated a highly statistically significant 85% reduction in adjudicated acute pancreatitis events at 12 months (p<0.001). These results were based on a total of 22 events in 17 patients in the placebo group, compared to seven events in five patients in the olezarsen group.

• In an overall pooled analysis of the number of patients needed to treat (NNT), treating 20 patients with olezarsen is estimated to prevent one acute pancreatitis event over one year.
• In the highest risk group, patients with triglyceride levels greater than or equal to 880 mg/dL and a history of acute pancreatitis, treating four patients is estimated to prevent one event over one year.

Olezarsen also showed an overall favorable lipid profile, with significant reductions in the secondary endpoints of apoC-III, remnant cholesterol and non-HDL-C.

“Building on olezarsen’s success in treating familial chylomicronemia syndrome, a rare form of sHTG, these groundbreaking results position us to reach a significantly larger patient population who remain at risk of dangerous acute pancreatitis attacks,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “Olezarsen will be one of two independent launches for Ionis in 2026, our first in a broad population if approved, and is a powerful example of how we are turning groundbreaking science into meaningful medicines that have the potential to change lives.”

Olezarsen demonstrated a favorable safety and tolerability profile in the CORE and CORE2 studies. Adverse events were balanced across treatment arms (75% olezarsen 50 mg; 76% olezarsen 80 mg; 75% placebo). Serious adverse events occurred less frequently in the olezarsen group compared to placebo (9% olezarsen 50 mg; 11% olezarsen 80 mg; 14% placebo). The most common treatment-emergent events were injection site reactions, which were mostly mild and occurred more frequently with olezarsen (10% olezarsen 50 mg; 17% olezarsen 80 mg; 1% placebo).

Several additional parameters were generally consistent with previous study results. At the 80 mg dose, asymptomatic increases in liver enzymes ≥3 times the upper limit of normal occurred in 7% of patients compared to 2% in the placebo group. These were not associated with clinical complications and generally resolved with continued dosing. No cases met the criteria for Hy’s law. Consistent with previously reported results with apoC-III-targeting therapies, small absolute mean elevations in liver fat (2.28% olezarsen 50 mg; 4.18% olezarsen 80 mg; 0.14% placebo) and hemoglobin A1c (HbA1c) (0.25% olezarsen 50 mg; 0.24% olezarsen 80 mg; placebo-adjusted) were observed. Increases in liver fat were not correlated with transaminase elevations and were not associated with clinical sequelae. There were no imbalances in HbA1c in non-diabetic patients.

Ionis is on track to submit a supplemental new drug application for both the 50 mg and 80 mg doses to the FDA by the end of the year. An open-label extension (OLE) study of olezarsen for sHTG is ongoing. More than 90% of patients who completed CORE and CORE2 chose to continue into the OLE.

Webcast

Ionis will host a webcast to discuss the results from the CORE and CORE2 studies on Saturday, November 8 at 3:00 p.m. ET. Interested parties may access the webcast here. A webcast replay will be available for a limited time.

About the CORE and CORE2 Studies

CORE (NCT05079919; n=617) and CORE2 (NCT05552326; n=446), conducted with The TIMI Study Group, are Phase 3 global, multicenter, randomized, double-blind, placebo-controlled trials investigating the safety and efficacy of olezarsen for severe hypertriglyceridemia (sHTG). Participants aged 18 and older with triglyceride levels ≥500 mg/dL were enrolled. Participants were required to be on standard of care therapies for elevated triglycerides. At baseline, 47% and 37% of participants had baseline fasting triglycerides ≥880 mg/dL in CORE and CORE2, respectively. Participants were randomized to receive 50 mg or 80 mg of olezarsen or placebo every 4 weeks via subcutaneous injection for 12 months. The primary endpoint is the percent change from baseline in fasting triglycerides at six months compared to placebo.

About Severe Hypertriglyceridemia

Severe hypertriglyceridemia (sHTG) is defined by severely high triglycerides (≥500 mg/dL) and characterized by an increased risk of acute pancreatitis and other morbidities. Considered a medical emergency, acute pancreatitis causes debilitating abdominal pain that often requires prolonged hospitalization, can lead to permanent organ damage and can become life-threatening. Preventing the first attack is key. In people with a history of acute pancreatitis episodes, the risk of future attacks is even greater. Current standard of care therapies for sHTG and lifestyle modifications (such as diet and exercise) do not sufficiently or consistently lower triglyceride levels or reduce the risks of sHTG in all patients. Approximately 3 million people are living with sHTG in the U.S., including more than 1 million who are considered high risk. High-risk sHTG includes those with triglycerides ≥880 mg/dL or triglycerides ≥500 mg/dL and a history of acute pancreatitis or other comorbidities.

About Olezarsen

Olezarsen is an investigational RNA-targeted medicine being evaluated for the treatment of sHTG. Olezarsen is designed to lower the body’s production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. Olezarsen is approved in the U.S. and the European Union as TRYNGOLZA® for adults with familial chylomicronemia syndrome (FCS).

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiometabolic disease and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.

Ionis Forward-looking Statements

This press release includes forward-looking statements regarding Ionis’ business, the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies, and our expectations regarding development and regulatory milestones. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis’ programs are described in additional detail in Ionis’ annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, “Ionis,” “Company,” “we,” “our” and “us” all refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals^® and TRYNGOLZA^® are trademarks of Ionis Pharmaceuticals, Inc.

Contacts

Ionis Investor Contact:
D. Wade Walke, Ph.D.

IR@ionis.com 760-603-2331

Ionis Media Contact:
Hayley Soffer

media@ionis.com 760-603-4679

New Data Show Early and Consistent Response to VTAMA® (tapinarof) Cream, 1%, in Children Aged 2+ with Atopic Dermatitis, Including Those With Associated Comorbidities




New Data Show Early and Consistent Response to VTAMA® (tapinarof) Cream, 1%, in Children Aged 2+ with Atopic Dermatitis, Including Those With Associated Comorbidities

• Sub-analysis of children with atopic dermatitis aged 2-17 in pivotal Phase 3 trials revealed early and clinically meaningful improvements in vIGA-AD^™ and EASI-75 (skin clearance and severity), POEM (patient-reported outcomes) and PP-NRS (itch), regardless of comorbidity status, at week 8

JERSEY CITY, N.J.–(BUSINESS WIRE)–Organon (NYSE: OGN), a global independent healthcare company with a focus on women’s health, will present results from a sub-analysis of pooled data from the Phase 3 ADORING 1 and ADORING 2 pivotal trials evaluating VTAMA cream versus vehicle at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Orlando, Florida, on November 8, 2025. The new data demonstrate that VTAMA cream provided early and consistent response for children aged 2-17 with atopic dermatitis (AD), with or without atopic comorbidities such as asthma, allergic rhinitis and food allergies.

“As many children with atopic dermatitis may also be living with potential comorbidities such as allergies and asthma that may add to their disease burden,¹ it’s important to understand the effects of approved treatments on this population,” said Dr. Luz Fonacier, Professor of Medicine, Section Head of Allergy and Training Program Director, NYU Grossman Long Island School of Medicine. “These reassuring data show tapinarof cream provided early relief, including on bothersome symptoms such as itch, for children as young as 2 years of age with and without comorbidities.”

In the ADORING 1 and ADORING 2 pivotal trials, adults and children (≥2 years; N=813) with moderate to severe AD were randomized to VTAMA cream or vehicle once daily for 8 weeks. The results presented at ACAAI 2025 are from a sub-analysis of the 654 children in the trials aged 2-17, with and without comorbidities associated with AD. The data highlight improvements in clinically relevant patient-reported outcomes, Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD^™) response, and Eczema Area and Severity Index (EASI) scores in children aged 2-17 years, regardless of comorbidities.

Notable findings include:

• Early improvements in skin clearance were observed, with significant differences in vIGA-AD response rates as early as week 1 and maintained through week 8 in children with or without atopic comorbidities (with comorbidities: 42.3% with VTAMA cream vs. 11.8% with vehicle, p<0.0001; without comorbidities: 49.5% vs. 14.8%, respectively, p<0.0001).
• Improvement in eczema severity, as measured by EASI scores, was observed as early as week 2 and sustained through week 8 (with comorbidities: 54.5% with VTAMA cream vs. 21.8% with vehicle, p<0.0001; without comorbidities: 63.1% vs. 20.4%, respectively, p<0.0001).
• Improvements in patient-reported outcomes, including sleep, as measured by total mean Patient-Oriented Eczema Measure (POEM) and mean POEM sleep scores (based on a single question within the POEM and analyzed separately from the total POEM score), were noted as early as week 1 and maintained through week 8 (total POEM with comorbidities: 6.9 with VTAMA cream vs. 12.0 with vehicle, p<0.0001; total POEM without comorbidities: 6.7 vs. 11.9, respectively, p<0.0001; POEM sleep with comorbidities: 0.9 with VTAMA cream vs. 1.4 with vehicle, p=0.0003; POEM sleep without comorbidities: 0.6 vs. 1.4, respectively, p<0.0001).
• Clinically meaningful improvements in itch (a ≥4-point Peak Pruritus- Numeric Rating Scale [PP-NRS] response) were observed at week 2, based on responder analysis, with continued improvement through week 8 (with comorbidities: 55.6% with VTAMA cream vs. 36.3% with vehicle, p=0.0043; without comorbidities: 63.3% vs. 29.2%, respectively, p<0.0001).

Consistent with the prescribing information, the most frequently reported treatment-emergent adverse events in the sub-analysis of children aged 2-17 in the ADORING 1 and ADORING 2 trials were folliculitis (7.8%), upper respiratory tract infection (4.6%) and headache (3.7%).

“We are proud to announce data from this new sub-analysis demonstrating early and consistent skin clearance with VTAMA cream, as well as other clinically meaningful improvements in key atopic dermatitis measures in children 2 years and older, regardless of comorbidity status,” said Rafael Chaves Cardona, MD, Head of U.S. Medical Affairs and Outcomes Research, Organon. “This study represents the largest pediatric data set to date for VTAMA, and when added to the wealth of data from the pivotal trials, as well as the ADORING 3 open-label, long-term extension trial, these findings support Organon’s commitment to offering treatment options with a favorable safety and efficacy profile for children 2 years and older.”

In December 2024, the U.S. Food and Drug Administration (FDA) approved VTAMA cream for the topical treatment of AD in adults and pediatric patients 2 years of age and older. VTAMA cream was also approved by the FDA in May 2022, for the topical treatment of plaque psoriasis in adults.²

About the Phase 3 Program for VTAMA cream in Atopic Dermatitis

ADORING was the Phase 3 AD clinical development program for VTAMA cream, consisting of two 8-week pivotal trials, ADORING 1 (NCT05014568) and ADORING 2 (NCT05032859), as well as ADORING 3 (NCT05142774), a 48-week, open-label, long-term extension trial.²

INDICATIONS: VTAMA^® (tapinarof) cream, 1% is an aryl hydrocarbon receptor (AhR) agonist indicated for:

• the topical treatment of plaque psoriasis in adults
• the topical treatment of atopic dermatitis in adults and pediatric patients 2 years of age and older

SELECTED SAFETY INFORMATION

Adverse Events: In plaque psoriasis, the most common adverse reactions (incidence ≥1%) were: folliculitis, nasopharyngitis, contact dermatitis, headache, pruritus, and influenza.

Adverse Events: In atopic dermatitis, the most common adverse reactions (incidence ≥1%) were: upper respiratory tract infection, folliculitis, lower respiratory tract infection, headache, asthma, vomiting, ear infection, pain in extremity, and abdominal pain.

Before prescribing VTAMA cream, please read the Prescribing Information.

For more information about VTAMA (tapinarof) cream, 1%, visit www.vtamahcp.com.

About Atopic Dermatitis (AD)

AD, commonly referred to as eczema, is one of the most prevalent inflammatory skin diseases, affecting an estimated 26 million people in the U.S. alone and up to 10% of adults worldwide.^3,4 AD occurs most frequently in children, affecting up to 20% worldwide, including nearly 10 million children in the US.^4,5, The disease results in itchy, red, swollen, and cracked skin, often on the folds of the arms, back of the knees, hands, face, and neck.^3, Itching is an especially bothersome symptom for those with AD, and tends to worsen at night.⁴ Around 90% of children with eczema also have another related condition such as allergies or asthma, according to international survey data.¹

About Organon

Organon (NYSE: OGN) is a global healthcare company with a mission to deliver impactful medicines and solutions for a healthier every day. With a portfolio of over 70 products across Women’s Health and General Medicines, which includes biosimilars, Organon focuses on addressing health needs that uniquely, disproportionately, or differently affect women, while expanding access to essential treatments in over 140 markets.

Headquartered in Jersey City, New Jersey, Organon is committed to advancing access, affordability, and innovation in healthcare. Learn more at www.organon.com and follow us on LinkedIn, Instagram, X, YouTube, TikTok, and Facebook.

Cautionary Note Regarding Forward-Looking Statements

Except for historical information, this press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the US Private Securities Litigation Reform Act of 1995, including, but not limited to, statements about Organon’s expectations about the potential impact of VTAMA as a treatment option for AD. Forward-looking statements may be identified by words such as “potential,” “mission,” “expects,” “will,” or words of similar meaning. These statements are based upon the current beliefs and expectations of Organon’s management and are subject to significant risks and uncertainties. If underlying assumptions prove inaccurate, or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Factors that could cause results to differ materially from those described in the forward-looking statements can be found in Organon’s filings with the SEC, including Organon’s most recent Annual Report on Form 10-K (as amended), Quarterly Reports on Form 10-Q (as amended), Current Reports on Form 8-K, and other SEC filings, available at the SEC’s Internet site (www.sec.gov). Organon undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.

© 2025 Organon group of companies. All rights reserved. vIGA-AD is the trademark of Eli Lilly and Co. US-VTA-113019 11/25

¹ Weidinger S, Simpson EL, Silverberg JI, et al. Burden of atopic dermatitis in paediatric patients: an international cross-sectional study. Br J Dermatol. 2024;190(6):846-857. doi:10.1093/bjd/ljad449

² VTAMA (tapinarof) cream, 1%, Prescribing Information. Organon; Revised May 2025.

³ Atopic dermatitis. National Institute of Arthritis and Musculoskeletal and Skin Diseases. November 2022. Accessed August 19, 2025. https://www.niams.nih.gov/health-topics/atopic-dermatitis
⁴ Eczema stats. National Eczema Association. Accessed June 5, 2025. https://nationaleczema.org/research/eczema-facts/
⁵ Global Report on Atopic Dermatitis 2022. International League of Dermatological Societies; 2022. Accessed February 25, 2025. https://www.eczemacouncil.org/assets/docs/global-report-on-atopic-dermatitis-2022.pdf

Contacts

Media Contacts:

Felicia Bisaro

(646) 703-1807

Investor Contact:

Jennifer Halchak

(201) 275-2711

Biomunex to Present Preclinical Data of its MAIT Engager Platform in Immuno-Oncology at 2025 SITC Annual Meeting




Biomunex to Present Preclinical Data of its MAIT Engager Platform in Immuno-Oncology at 2025 SITC Annual Meeting

• Dr. Simon Plyte, Biomunex’s CSO, has been invited to present MAIT engagers preclinical data during an oral communication at 2025 SITC Annual meeting

• With a very attractive safety and efficacy profile, MAIT engagers have a major potential to fuel an innovative portfolio of drug candidates in oncology

• Biomunex’s ambition is to develop the next generation of immunotherapies in oncology: MAIT engagers, bispecific antibodies capable of identifying, mobilizing, and activating MAIT cells, a subpopulation of T cells, to kill cancer cells.

PARIS & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biomunex Pharmaceuticals, a French biopharmaceutical company specializing in the development of next generation immunotherapies based on the discovery and development of bispecific and multispecific antibodies, today announces presentation of preclinical data from its MAIT engager platform at the Society for Immunotherapy of Cancer’s (“SITC”) 40^th Annual Meeting being held in National Harbor, USA, from November 5th-9th, 2025.

MAIT engagers: a new class of bispecific antibodies for cancer treatment

Invited to present an oral communication on November 8^th, Dr Simon Plyte, Biomunex CSO (Chief Scientific Officer), will make a presentation entitled: “The MAIT Engager platform : rapid generation of several MAIT T cell engagers with significantly improved safety profile and large therapeutic window (Abstract 1197).” He is also presenting a Poster regarding the topic on November 7^th.

Biomunex is developing a large portfolio of MAIT engager drug candidates, a new therapeutic class in immuno-oncology, that is differentiated from classical TCEs (“T cell engagers”). MAIT engagers are bispecific antibodies that identify, mobilize and bridge MAIT cells (Mucosal-Associated Invariant T cells) to cancer cells resulting in MAIT activation and directed killing of tumors; MAIT engagers could become a breakthrough approach in the treatment of many cancers, particularly in solid tumors.

The presentation at SITC Annual meeting will focus on the MAIT engager differentiation from classical CD3+ TCEs and highlight the superior safety of MAIT engagers. MAIT engagers are expected to overcome some of the limitations of current CD3+ TCE therapies, including activation of regulatory T cells (Tregs) and cytokine release syndrome, serious side effects that are difficult to manage for cancer patients.

The Biomunex platform enables rapid generation of several MAIT engagers, that are not only as potent at classical CD3+ T cell engagers but also bring significantly improved safety profile and have the potential to provide a larger therapeutic window in specific tumor types. Moreover, MAIT engagers can effectively induce the “SPARK effect”, enabling long-term durable anti-cancer response.

Based on preclinical data, Biomunex expects the first MAIT engager to enter clinical trials soon. The MAIT engager platform paves the way for a series of innovative new immunotherapeutics targeting many cancers. “MAIT engagers are an attractive approach for the treatment of cancer with breakthrough potential for redefining the standard of care for solid tumors,” comments Dr. Simon Plyte, Biomunex’s CSO. “Presenting those preclinical data as an oral communication and a poster during the 40^th SITC meeting is a unique opportunity in sharing the scientific and medical potential of this innovative approach with the scientific community”.

Biomunex accelerates development of its disruptive immunotherapies pipeline

Biomunex is becoming a major innovative player in oncology. Bi- and multi-specific antibodies and T cell engagers are attracting considerable interest from pharmaceutical companies, with the recent signing of many licensing and partnership agreements, including for Biomunex. “The last few months have marked the beginning of a new chapter in Biomunex’s development with the signing of a major agreement with Ipsen, following other licensing or partnership deals, demonstrating the team’s ability to develop drugs and technologies of interest for the pharmaceutical industry, such as the MAIT engagers presented at 2025 SITC Annual meeting, to treat cancer patients, but also to establish leading licensing agreements and partnerships with the industry,” comments Dr. Pierre-Emmanuel Gerard, Biomunex’s founder and President.

In addition to BMX-502 licensed to Ipsen, Biomunex is developing a portfolio of MAIT engager drug candidates and innovative new therapeutic approaches with high potential, based on its proprietary “Plug-and-Play” BiXAb® platform. This technology enables the generation of breakthrough immunotherapies faster than most other platforms in the field, with excellent drug-like properties and high industrial yield. Biomunex’s first BiXAb drug candidate is currently in Phase 1 clinical development in partnership, as a monotherapy or in combination, in patients with certain solid tumors or hematological malignancies.

***

Details about Biomunex’ presentations at 2025 SITC Annual Meeting

Abstract Title: “The MAIT Engager platform – rapid generation of several MAIT T cell engagers with significantly improved safety profile and large therapeutic window”

Abstract Number: 1197 – Ranked amongst 150 best abstracts.

Poster presentation

Friday 7th Nov, 5:35–7 p.m. ET

Poster Reception | Gaylord National Resort and Convention Center – Lower Level Atrium – Prince George’s ABC

Oral presentation

Saturday 8th Nov, 1:08-1:16 p.m. ET

Concurrent Session 205a: Rapid Oral Abstract Session – Basic

Maryland Ballroom AB

About Biomunex Pharmaceuticals : www.biomunex.com

Biomunex Pharmaceuticals is a biopharmaceutical company based in Paris, France, and Cambridge, MA, USA, specializing in the discovery and development of innovative therapeutic approaches based on solid data and biological and clinical evidence to address unmet medical needs in oncology.

Biomunex has created and developed BiXAb®, a robust, next-generation, plug-and-play technology platform for bi- and multi-specific antibodies, using a proprietary in silico modeling approach, based on a very robust intellectual property and patent portfolio.

The BiXAb platform, which enables the generation of bispecific antibodies from any pair of monoclonal antibodies in a simple, rapid, and cost-effective manner, has been validated through licensing agreements and collaborations with the pharmaceutical and biotechnology industry, including Sanofi, Onward Therapeutics, and most recently Ipsen.

Biomunex is the first company in the world to develop a cancer immunotherapy approach that uses bispecific antibodies from its BiXAb platform to specifically target, engage, and redirect MAIT cells, a subpopulation of T cells naturally present throughout the body, particularly in mucosal and barrier tissues, to kill cancer cells for the treatment of solid tumors.

Contacts

Media:

Biomunex Pharmaceuticals
NewCap Agency – Nicolas Merigeau

nmerigeau@newcap.fr
+33 (0)1 44 71 94 98

Evinova China and Harbour BioMed Announce Strategic AI Collaboration to Accelerate AI-Enabled Drug Development




Evinova China and Harbour BioMed Announce Strategic AI Collaboration to Accelerate AI-Enabled Drug Development

SHANGHAI–(BUSINESS WIRE)–Yesterday, during the 8th China International Import Expo (“CIIE”), Evinova, a global health-tech company accelerating the delivery of better health outcomes by propelling the life sciences sector forward in digital health, and Harbour BioMed (HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology and oncology, announced a strategic collaboration in artificial intelligence (AI).

Under the terms of the collaboration, Evinova China and Harbour BioMed will jointly apply AI and digital technologies to enhance the efficiency of innovative biologics development. Building on their own strengths, the two companies aim to build an open ecosystem for AI-driven drug R&D.

Nate Zhang, General Manager of Evinova China, stated, “Evinova leverages the digital transformation insights of top global pharmaceutical companies to design our AI-powered clinical development solutions and digital strategy consulting services, so that we can accelerate the delivery of better health outcomes. To realize our mission, Evinova China needs to partner with innovative companies like Harbour BioMed, which are rooted in China while harboring global ambitions. Together, through our world-class AI technology platforms and deep therapeutic expertise, we can help take China-originated breakthrough assets from the laboratory to the world.”

Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, stated, “We are pleased to collaborate with Evinova to advance the application of AI in biotherapeutic development. Harbour BioMed has built a robust and differentiated product pipeline based on our industry-leading Harbour Mice® technology platform. Through this collaboration, we look forward to applying AI to improving clinical study efficiency and accelerating the delivery of innovative therapies to patients around the world.”

About Evinova

Evinova is a global health-tech business, accelerating the delivery of better health outcomes by propelling the life sciences sector forward in digital health, from the inside. Through our application of science-based expertise, evidence-led rigour, and human experience-driven insight, our digital solutions are deliberately designed so that everyone can reach better health outcomes together. Evinova is a separate health-tech business within the AstraZeneca Group. Please visit evinova.com or follow the company on social media @Evinova.

About Harbour BioMed

Harbour BioMed (HKEX: 02142) is a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology and oncology. The company is building a robust and differentiated pipeline through internal R&D capabilities, strategic global collaborations in co-discovery and co-development, and selective acquisitions.

Harbour BioMed’s proprietary antibody technology platform, Harbour Mice®, generates fully human monoclonal antibodies in both the conventional two heavy and two light chain (H2L2) format and the heavy chain-only (HCAb) format. Building upon HCAb antibodies, the HCAb-based immune cell engagers (HBICE®) bispecific antibody technology enables tumor-killing effects that traditional combination therapies cannot achieve. Additionally, the HCAb-based bispecific immune cell antagonist (HBICA^TM) technology empowers the development of innovative biologics for immunological and inflammatory diseases. By integrating Harbour Mice®, HBICE®, and HBICA^TM with a single B-cell cloning platform, Harbour BioMed has built a highly efficient and distinctive antibody discovery engine for developing next-generation therapeutic antibodies. For more information, please visit www.harbourbiomed.com.

Contacts

Media Contact:
Heather Bonsiero

Head of Communications and Marketing

Evinova

heather.bonsiero@evinova.com

Fresenius Kabi Issues Voluntary Nationwide Recall of Three Lots of Famotidine Injection, USP, 20 mg per 2 mL (10 mg per mL), 2 mL Fill in a 2 mL Vial Due to Out-of-Specification Endotoxin Results in Certain Reserve Samples




Fresenius Kabi Issues Voluntary Nationwide Recall of Three Lots of Famotidine Injection, USP, 20 mg per 2 mL (10 mg per mL), 2 mL Fill in a 2 mL Vial Due to Out-of-Specification Endotoxin Results in Certain Reserve Samples

LAKE ZURICH, Ill.–(BUSINESS WIRE)–#CommittedToLife–Fresenius Kabi, part of the global healthcare company Fresenius, and a leading provider of essential medicines and medical technologies is voluntarily recalling three lots (numbers 6133156, 6133194, 6133388) of Famotidine Injection, USP, 20 mg per 2 mL (10 mg per mL), 2 mL Fill in a 2 mL vial. This recall is being performed to the user level in the United States.

The product is being recalled due to out-of-specification (OOS) endotoxin results of certain reserve samples from a single lot. Based upon the investigation, two additional lots were also included in the recall as a precautionary measure.

Elevated endotoxin levels can precipitate severe systemic reactions such as sepsis and septic shock. Severe responses may include inflammatory and life-threatening immune responses and death. Non-serious adverse event reports potentially associated with the OOS have been received for one lot. These non-serious adverse events included chills, change in mental status, change in respiratory status, fever, increase in body temperature, shivering and shaking. To date, no adverse event reports have been received for the second and third lots.

Famotidine Injection is indicated in some hospitalized patients with pathological hypersecretory conditions or intractable ulcers, or as an alternative to the oral dosage forms for short term use in patients who are unable to take oral medication for the following conditions:

1. Short term treatment of active duodenal ulcer.
2. Maintenance therapy for duodenal ulcer patients at reduced dosage after healing of an active ulcer.
3. Short term treatment of active benign gastric ulcer.
4. Short term treatment of gastroesophageal reflux disease (GERD).
5. Treatment of pathological hypersecretory conditions.

Fresenius Kabi is notifying its distributors and customers and is arranging for return of the recalled product. If health care facilities have any of the affected lots, they are to immediately discontinue distributing, dispensing or using the lots and return all units to Fresenius Kabi. Distributors are instructed to immediately notify their customers that have been shipped or may have been shipped, the product involved in this recall.

Consumers with questions regarding this recall can contact Fresenius Kabi USA Quality Assurance at 1-866-716-2459, Monday through Friday, during the hours of 8:00 a.m. to 5:00 p.m. Central Standard Time. Patients should contact their physician or health care provider if they have experienced any problems that may be related to receiving this drug product.

Adverse reactions or quality problems experienced with the use of this product may be reported to Fresenius Kabi Medical Affairs or Vigilance departments at 1-800-551-7176, Monday through Friday, during the hours of 8:00 a.m. to 5:00 p.m. Central Standard Time, or send an e-mail to either productcomplaint.USA@fresenius-kabi.com or adverse.events.USA@fresenius-kabi.com.

Adverse reactions or quality problems experienced with the use of this product may be reported to the FDA’s MedWatch Adverse Event Reporting program either online, by regular mail or by fax.

• Complete and submit the report Online: www.fda.gov/medwatch/report.htm
• Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178.

This recall is being conducted with the knowledge of the U.S. Food and Drug Administration.

About Fresenius Kabi

As a global healthcare company, Fresenius Kabi is Committed to Life. The company’s products, technologies, and services are used for the therapy and care of patients with critical and chronic conditions. With more than 41,000 employees and present in more than 100 countries, Fresenius Kabi’s expansive product portfolio focuses on providing access to high-quality and lifesaving medicines and technologies.

Contacts

Media Contact

Matt Kuhn

847-220-3033

City of Hope Appoints Leading Lung Cancer Expert Dr. Christine M. Lovly to Head National Thoracic Oncology Program




City of Hope Appoints Leading Lung Cancer Expert Dr. Christine M. Lovly to Head National Thoracic Oncology Program

• Dr. Lovly brings nearly two decades of experience in clinical care, research and academic leadership to her new role.
• Her work has advanced personalized therapies that improve outcomes and quality of life for people with lung cancer.

LOS ANGELES–(BUSINESS WIRE)–City of Hope^®, one of the largest and most advanced cancer research and treatment organizations in the United States with its National Medical Center ranked among the nation’s top cancer centers by U.S. News & World Report, today announced that internationally recognized physician-scientist Christine M. Lovly, M.D., Ph.D., F.A.S.C.O., will spearhead the development of its new national thoracic oncology program, furthering City of Hope’s mission to deliver exceptional multidisciplinary care and transformative research for patients with lung cancer. Dr. Lovly’s appointment is effective Jan. 1.

Dr. Lovly will be division chief of thoracic medical oncology, professor in the Department of Medical Oncology & Therapeutics Research at City of Hope National Medical Center and will hold the Dr. Norman and Melinda Payson Professorship in Medical Oncology.

She brings nearly two decades of experience in clinical care, translational research and academic leadership. She joins City of Hope at a critical time in the fight against lung cancer, as rates of the disease continue to rise — especially among women, younger people and nonsmokers.

Her pioneering work has shaped the modern landscape of lung cancer care, leading to more effective therapies tailored to the genetic makeup of individual tumors. Dr. Lovly is a leading authority on the molecular dynamics of targeted therapy response and resistance, especially in lung cancer subtypes characterized by EGFR and ALK alterations.

“Dr. Christine Lovly is a widely recognized expert whose groundbreaking research and unwavering commitment to patient-centered care make her an extraordinary addition to City of Hope. Her expertise in precision medicine and translational science will be instrumental in shaping our national thoracic oncology program and accelerating progress for patients with lung cancer across the country,” said Marcel van den Brink, M.D., Ph.D., president, City of Hope Los Angeles and City of Hope National Medical Center, and Deana and Steve Campbell Chief Physician Executive Distinguished Chair in Honor of Alexandra Levine, M.D.

Dr. Lovly will be responsible for advancing multidisciplinary clinical care, building and mentoring a world-class faculty, expanding translational and clinical research, and fostering strategic partnerships to further solidify City of Hope as a national leader in lung cancer innovation and patient outcomes. Dr. Lovly’s arrival marks an exciting chapter for City of Hope. Her collaborative, innovative approach will further elevate City of Hope’s lung cancer program and strengthen its systemwide commitment to excellence.

Her research portfolio includes ongoing projects focused on biomarkers, drug development and residual disease. She integrates data science into her research, helping create predictive algorithms to uncover potent drug interactions with the potential to improve efficacy and lessen side effects. This body of work resulted in Dr. Lovly receiving the 2025 William J. Darby Award “for translational research that has changed the practice of medicine worldwide.”

Today the LUNGevity Foundation presented Dr. Lovly with the Face of Hope Award, which is given to individuals who recognize the needs of those living with lung cancer and is actively making a difference on their behalf. Past recipients include Richard Pazdur, M.D., director of the U.S. Food and Drug Administration’s Oncology Center of Excellence, and Robert Winn, M.D., president of the Association of American Cancer Institutes.

“I believe the future of lung cancer care lies in precision medicine and team-based collaboration, and City of Hope is the ideal place to bring that vision to life,” Dr. Lovly said. “I look forward to working alongside world-class clinicians and researchers to build a program that truly serves patients and their families.”

Dr. Lovly comes to City of Hope from Vanderbilt University Medical Center, where she was a tenured faculty member and held a joint appointment at the Veterans Affairs Medical Center. She is an elected member of the American Society for Clinical Investigation and serves on numerous editorial boards, including Cancer Discovery, Clinical Cancer Research and JCO Precision Oncology. Dr. Lovly holds leadership roles in multiple national and international organizations, including as a current member of the American Association for Cancer Research (AACR) Board of Directors.

Dr. Lovly contributes to the scientific leadership boards of LUNGevity Foundation, GO2 Foundation for Lung Cancer Research and Lung Cancer Research Foundation. Additionally, she plays a key role in shaping national standards of care through her service on the National Comprehensive Cancer Network (NCCN) guidelines panel for non-small cell lung cancer.

About City of Hope

City of Hope’s mission is to make hope a reality for all touched by cancer and diabetes. Founded in 1913, City of Hope has grown into one of the largest and most advanced cancer research and treatment organizations in the United States, and one of the leading research centers for diabetes and other life-threatening illnesses. City of Hope research has been the basis for numerous breakthrough cancer medicines, as well as human synthetic insulin and monoclonal antibodies. With an independent, National Cancer Institute-designated comprehensive cancer center that is ranked among the nation’s top cancer centers by U.S. News & World Report at its core, City of Hope’s uniquely integrated model spans cancer care, research and development, academics and training, and a broad philanthropy program that powers its work. City of Hope’s growing national system includes its Los Angeles campus, a network of clinical care locations across Southern California, a new cancer center in Orange County, California, and cancer treatment centers and outpatient facilities in the Atlanta, Chicago and Phoenix areas. City of Hope’s affiliated group of organizations includes Translational Genomics Research Institute and AccessHope™. For more information about City of Hope, follow us on Facebook, X, YouTube, Instagram and LinkedIn.

Contacts

Zen Logsdon

626-409-9367

zlogsdon@coh.org

FillPoint Health Forms Collaborative Partnership with Noble, an Aptar Pharma Company, to Enhance Patient Experience and Drive Treatment Adherence




FillPoint Health Forms Collaborative Partnership with Noble, an Aptar Pharma Company, to Enhance Patient Experience and Drive Treatment Adherence

DUBLIN, Ohio–(BUSINESS WIRE)–FillPoint Health, a Lyceum Health company and URAC/ACHC-accredited specialty pharmacy and MSO, announced today a strategic partnership with Noble, an Aptar Pharma company and global leader in drug delivery training and onboarding solutions. This collaboration aims to enhance the patient experience through provider engagement and pharmaceutical partnerships, ensuring proper device use, improved adherence, and better clinical outcomes.

Under this partnership, FillPoint Health and Noble will jointly develop and implement education and support programs that integrate Noble’s industry-leading training devices, patient onboarding tools, and education platforms into FillPoint’s provider-driven specialty pharmacy model. The collaboration will focus on bridging the gap between pharmaceutical manufacturers, healthcare providers, and patients, creating a consistent, high-touch experience from prescription to administration.

“Our partnership with Noble represents a critical step in advancing how specialty therapies are delivered and supported at the point of care,” said Michael Baldzicki, Chief Revenue Officer of FillPoint Health. “By combining Noble’s proven patient training expertise with FillPoint’s provider-centric pharmacy model, we can help patients start therapy with confidence, use their medications correctly, and stay adherent over time—ultimately improving outcomes and satisfaction.”

The collaboration aligns with FillPoint Health’s broader mission of empowering providers and patients through technology, education, and integrated care models. As part of Lyceum Health’s network, FillPoint Health partners with pharmaceutical manufacturers and specialty providers to deliver fair market value (FMV)-based services, risk-of-loss pharmacy models, and real-world data insights that enhance access, quality, and efficiency in specialty care.

“We’re excited to partner with FillPoint Health to further our shared vision of improving patient engagement and outcomes,” said Craig Baker, President, Noble – Aptar Pharma. “Together, we will leverage our complementary strengths to ensure patients are fully supported in their treatment journey, from training to adherence.”

The partnership will initially focus on key therapeutic areas—including cardiology, dermatology, neurology, and rare diseases—with plans to expand into additional specialty categories and manufacturer collaborations throughout 2026.

About FillPoint Health, A Lyceum Health Company

FillPoint Health is a URAC- and ACHC-accredited specialty pharmacy and MSO focused on enhancing specialty patient care through integrated provider, payer, and pharmaceutical partnerships. FillPoint operates as part of Lyceum Health, a group of companies that includes RxNexus, a digital patient-access platform, and NewPoint-Rx, a physician-dispense division enabling compliant, FMV-based, in-office dispensing. For more info, please visit fillpointhealth.com and lyceumhealth.com.

About Noble, An Aptar Pharma Company

Noble is an Aptar Pharma company and part of AptarGroup, Inc., a global leader in drug and consumer product dosing, dispensing and protection technologies. Noble is a global leader in the development of patient-centric onboarding and drug delivery training solutions designed to improve the patient experience and drive adherence. Noble partners with pharmaceutical and biotechnology companies worldwide to create innovative, user-friendly training and support programs that empower patients to start and stay on therapy. For more information, please visit gonoble.com and www.aptar.com.

Contacts

Media Contacts:
FillPoint Health, A Lyceum Health Company:

info@fillpointhealth.com
www.lyceumhealth.com

Noble, An Aptar Pharma Company:

Ciara Jackson

Aptar Pharma

ciara.jackson@aptar.com
+49 151 1951 6502