• Designation is based on results from the progressive pulmonary fibrosis cohort of the Phase 2 study assessing the safety and efficacy of BMS-986278 treatment versus placebo

PRINCETON, N.J. / Oct 24, 2023 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for BMS-986278, a potential first-in-class, oral, lysophosphatidic acid receptor 1 (LPA₁) antagonist, for the treatment of progressive pulmonary fibrosis (PPF), a devastating, life-threatening illness. Currently, there is only one therapy approved for the treatment of PPF.

The Breakthrough Therapy Designation is based on results from the global, randomized Phase 2 study that assessed the safety and efficacy of BMS-986278 treatment versus placebo in people living with idiopathic pulmonary fibrosis (IPF) and PPF. Stable background use of antifibrotics in the IPF cohort and/or select immunosuppressives in the PPF cohort were allowed. Results from the PPF cohort showed that 26 weeks of treatment with a twice-daily 60 mg dose of BMS-986278 resulted in a 69% relative reduction in the rate of decline in percent predicted forced vital capacity versus placebo. Treatment effect was consistent with or without background therapy and BMS-986278 was well tolerated, with rates of adverse events similar to placebo and low discontinuation rates. These findings were presented at the European Respiratory Society (ERS) 2023 International Congress in September 2023.

Breakthrough Therapy Designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening diseases with preliminary clinical evidence that the investigational therapy may offer substantial improvement on at least one clinically significant endpoint over available therapies.

“People living with pulmonary fibrosis face deteriorating lung function, worsening respiratory symptoms and reduced quality of life, which can ultimately lead to respiratory failure and death,” said Roland Chen, MD, senior vice president and head, Immunology, Cardiovascular and Neuroscience Development, Bristol Myers Squibb. “The FDA’s Breakthrough Therapy Designation underscores the potential of BMS-986278 as an innovative, first-in-class treatment that may redefine the standard of care for progressive pulmonary fibrosis.”

In addition to this Breakthrough Therapy Designation for PPF, the U.S. FDA has also previously granted BMS-986278 fast-track designation and orphan drug designation for the treatment of IPF. Bristol Myers Squibb is continuing the development of BMS-986278 with the global Phase 3 ALOFT program for PPF (NCT06025578) and IPF (NCT06003426).

About BMS-986278

BMS-986278 is a potential first-in-class, oral, small molecule lysophosphatidic acid receptor 1 (LPA₁) antagonist currently being evaluated as a novel antifibrotic treatment for patients with idiopathic pulmonary fibrosis and progressive pulmonary fibrosis. Increased LPA levels and activation of LPA₁ are involved in the pathogenesis of pulmonary fibrosis. A preclinical in vitro and in vivo study found that antagonizing LPA₁ may be beneficial in treating lung injury and fibrosis.

About the BMS-986278 Phase 2 Study

The Phase 2 study was a global, randomized study in which parallel cohorts of patients with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) received 30 mg or 60 mg of BMS-986278 or matched placebo orally twice-daily. The study consisted of a 26-week placebo-controlled treatment period, an optional 26-week active treatment extension period and a 4-week post-treatment follow-up period. Patients were permitted to take background antifibrotics in the IPF cohort and background antifibrotics and/or immunosuppressants in the PPF cohort. The primary endpoint was rate of change in percent predicted forced vital capacity (ppFVC) from baseline to Week 26 in the IPF cohort. ppFVC compares the observed FVC to that which is expected for a healthy person of the same age, gender, race and height. Rate of change in ppFVC from baseline through Week 26 in the PPF cohort was a key secondary endpoint. Patients who met prespecified blood pressure reduction criteria were to receive a dose reduction to 10 mg of BMS-986278 or matching placebo twice-daily.

More information can be found on www.clinicaltrials.gov (NCT04308681).

About Pulmonary Fibrosis

Pulmonary fibrosis is a chronic, life-threatening interstitial lung disease (ILD) that occurs when lung tissue becomes damaged and scarred, impacting how lungs function. Progressive pulmonary fibrosis (PPF) is the preferred term to describe patients who have an ILD with a progressive fibrotic phenotype. Idiopathic pulmonary fibrosis (IPF) is the most common type of progressive fibrosing ILD. As an idiopathic disease, there is no identifiable cause, and as of 2021, more than 700,000 adults are living with IPF globally.

Many people living with PPF and IPF are physically impaired, experience a progressive decline in lung function, have difficulty performing simple daily activities due to breathlessness and require continuous supplemental oxygen to ease the burden of normal breathing.

IPF is a fatal disease with a median survival time of 3-5 years following diagnosis and 5-year survival rate of approximately 45%; PPF has shown a similar prognosis. Innovation in treatment has been limited with few new therapies approved in nearly 10 years.

Bristol Myers Squibb: Pioneering Paths Forward in Immunology to Transform Patients’ Lives

Bristol Myers Squibb is inspired by a single vision – transforming patients’ lives through science. For people living with immune-mediated diseases, the debilitating reality of enduring chronic symptoms and disease progression can take a toll on their physical, emotional and social well-being, making simple tasks and daily life a challenge. Driven by our deep understanding of the immune system that spans over 20 years of experience, and our passion to help patients, the company continues to pursue pathbreaking science with the goal of delivering meaningful solutions that address unmet needs in rheumatology, gastroenterology, dermatology and pulmonology. We follow the science, aiming to tailor therapies to individual needs, improve outcomes and expand treatment options by working to identify mechanisms with the potential to achieve long-term remission – and perhaps even cures – in the future. By building partnerships with researchers, patients and caregivers to deliver innovative treatments, Bristol Myers Squibb strives to elevate patient care to new standards and deliver what matters most – the promise of living a better life.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that future study results may not be consistent with the results to date, that BMS-986278 may not achieve its primary study endpoints or receive regulatory approval for the indication described in this release in the currently anticipated timeline or at all and, if approved, whether such product candidate for such indication described in this release will be commercially successful. It should also be noted that Breakthrough Therapy Designation does not change the standards for FDA approval. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2022, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.