Health Canada Approves REBLOZYL® (luspatercept), New Class of Treatment for Adult Patients Living With Myelodysplastic Syndromes

Rhea-AI Summary

Bristol Myers Squibb Canada and Acceleron Pharma announced that Health Canada has approved REBLOZYL^® (luspatercept) for treating adult patients with transfusion-dependent anemia due to myelodysplastic syndromes (MDS) who require at least two RBC units over eight weeks.

This marks REBLOZYL^® as the first and only approved erythroid maturation agent in Canada. The approval is based on the successful results of the phase 3 MEDALIST study, where 38% of patients treated achieved transfusion independence compared to 13% on placebo.

Positive

• Health Canada's approval of REBLOZYL^® addresses an unmet need in treating MDS-related anemia.
• REBLOZYL^® has shown a 38% transfusion independence rate in the MEDALIST trial, significantly higher than the 13% for placebo.

Negative

• None.

Bristol Myers Squibb Canada (BMS) and Acceleron Pharma Inc. announced today that Health Canada has approved REBLOZYL^® (luspatercept for injection) for the treatment of adult patients with transfusion-dependent anemia requiring at least two RBC red blood cell (RBC) units over 8 weeks resulting from very low-to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts and who have failed or are not suitable for erythropoietin-based therapy.¹ REBLOZYL^® represents a new class of treatment for eligible patients as the first and only approved erythroid maturation agent in Canada.¹

“Those living with MDS are often dependent on regular RBC transfusions. While these transfusions help manage symptoms of anemia, transfusion dependency is very burdensome for patients,” said Dr. Rena Buckstein, MD, FRCPC, Haematologist, associate professor and Head of the Hematology Disease Site Group at Odette Cancer Centre, Sunnybrook Health Sciences Centre. “Treatment options to remedy transfusion dependence are limited once first line therapy fails, so REBLOZYL^®, as the only erythroid maturation agent in Canada, provides an important MDS treatment to achieve RBC transfusion independence in a subgroup of patients.”

MDS are a group of cancers which cause limitations in the bone marrow’s ability to produce healthy and mature RBCs,² and typically occur among older people, and in men more than in women.²

“MDS can significantly impact quality of life; especially for those who are dependent on regular RBC transfusions,” said Cindy Anthony, Executive Director, Aplastic Anemia & Myelodysplasia Association of Canada (AAMAC). “We’re pleased to see an alternative to transfusions available which can help alleviate some of the burden of the disease and support patients.”

REBLOZYL^® helps to regulate late-stage RBC maturation in order to potentially reduce the need for RBC transfusions.¹

“Bristol Myers Squibb is thrilled to provide Canadians with an additional option beyond RBC transfusions to treat MDS-associated anemia,” said Al Reba, General Manager, Bristol Myers Squibb Canada. “This first-in-class treatment is an example of Bristol Myers Squibb’s commitment to develop innovative therapies for Canadians living with severe blood disorders.”

“Working with our partners at Bristol Myers Squibb, we are excited to help address the needs of Canadians living with MDS,” said Habib Dable, President and Chief Executive Officer of Acceleron. “By targeting the ineffective erythropoiesis associated with MDS, REBLOZYL^® represents an important addition for the treatment of patients living with this condition.”

Health Canada’s approval of REBLOZYL^® is based upon findings from the phase 3, double-blind, randomized, placebo-controlled MEDALIST study, which involved patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) requiring regular RBC transfusions (>2 RBC units per 8 weeks).¹ The patients were randomized 2:1 to REBLOZYL^® or placebo.¹ In the trial, results demonstrated significantly greater percentage of patients treated with REBLOZYL^® achieving transfusion independence for eight weeks or longer during the first 24 weeks of the trial as compared to placebo (38% vs. 13%, P<0.001) at primary endpoint.¹

About Bristol Myers Squibb Canada
Bristol Myers Squibb Canada Co. is an indirect wholly-owned subsidiary of Bristol Myers Squibb Company, 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 global operations, visit www.bms.com. Bristol Myers Squibb Canada Co. delivers innovative medicines for serious diseases to Canadian patients in the areas of cardiovascular health, oncology, and immunoscience. Bristol Myers Squibb Canada Co. employs close to 400 people across the country. For more information, please visit www.bms.com/ca.

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.

About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron’s leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research, development, and commercialization efforts in pulmonary and hematologic diseases. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH), having reported positive topline results of the PULSAR Phase 2 trial. The Company is currently planning multiple Phase 3 trials with the potential to support its long-term vision of establishing sotatercept as a backbone therapy for patients with PAH at all stages of the disease. Acceleron is also investigating the potential of its early-stage pulmonary candidate, ACE-1334, which it plans to advance into a Phase 1b/2 trial in systemic sclerosis-associated interstitial lung disease (SSc-ILD) this year.

In hematology, REBLOZYL^® (luspatercept-aamt) is the first and only erythroid maturation agent approved in the United States, Europe, and Canada for the treatment of anemia in certain blood disorders. REBLOZYL^® is part of a global collaboration partnership with Bristol Myers Squibb. The Companies co-promote REBLOZYL^® in the United States and are also developing luspatercept for the treatment of anemia in patient populations of myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.

For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

Bristol Myers Squibb 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 historical performance and 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, whether REBLOZYL (luspatercept) for the additional indication described in this release will be commercially successful and that continued approval of such product candidate for such additional indication described in this release may be contingent upon verification and description of clinical benefit in confirmatory trials. 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 Canada or Bristol-Myers Squibb Company’s (Bristol Myers Squibb) 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, 2020, 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, neither Bristol Myers Squibb Canada nor Bristol Myers Squibb undertakes an obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

Acceleron Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements about Acceleron’s strategy, future plans and prospects, including statements regarding the development and commercialization of REBLOZYL and the potential of REBLOZYL as a therapeutic drug. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that REBLOZYL may not be commercially successful in the additional indication described in this press release, and that continued approval of REBLOZYL for such additional indication described in this press release may be contingent upon verification and description of clinical benefit in confirmatory trials. These and other risks and uncertainties are identified under the heading “Risk Factors” included in Acceleron’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future.

The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

References:

1. REBLOZYL^® Canada Product Monograph. February 11, 2021.
2. Canadian Cancer Society. Myelodysplastic Syndromes. Available at: https://www.cancer.ca/en/cancer-information/cancer-type/leukemia/leukemia/myelodysplastic-syndromes/?region=on#risk_factors. Accessed January 29, 2021.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210216005710/en/

FAQ

What is REBLOZYL and what is its approval status in Canada?

REBLOZYL (luspatercept) has been approved by Health Canada for treating adult patients with transfusion-dependent anemia resulting from myelodysplastic syndromes.

What were the results of the MEDALIST study for REBLOZYL?

In the MEDALIST study, 38% of patients treated with REBLOZYL achieved transfusion independence for eight weeks or longer, compared to 13% in the placebo group.

When was REBLOZYL approved by Health Canada?

Health Canada approved REBLOZYL on February 11, 2021.

What is the significance of REBLOZYL for patients with MDS?

REBLOZYL provides a new treatment option for MDS patients who are dependent on red blood cell transfusions and have not responded to erythropoietin-based therapy.

Who collaborated on the development of REBLOZYL?

REBLOZYL was developed through a collaboration between Bristol Myers Squibb and Acceleron Pharma.