On May 11, 2020 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Acceleron Pharma, MAY 11, 2020, View Source [SID1234557505]).
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"Acceleron is off to a strong start in 2020, propelled by one of the most eventful time periods in the Company’s history," said Habib Dable, President and Chief Executive Officer of Acceleron. "The breadth and depth of our hematology program has been on full display, owing to the publication of the MEDALIST and BELIEVE Phase 3 luspatercept trial results in the prestigious New England Journal of Medicine and the recent FDA approval of REBLOZYL for the treatment of anemia in a population of patients with lower-risk MDS. We’re thrilled that this first-in-class erythroid maturation agent, having received approvals in two distinct indications over a span of just five months, is now available in the US to patients who have long needed a new option for treating their chronic anemias."
Added Mr. Dable: "Our scientific expertise in leveraging the therapeutic potential of the TGF-beta superfamily of proteins is delivering equally exciting results in pulmonary disease. With great enthusiasm, we announced that the PULSAR Phase 2 trial of sotatercept in patients with PAH met its primary and key secondary endpoints-underscoring our belief that sotatercept could eventually alter the current treatment paradigm. Following the results, sotatercept was granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. We look forward to presenting topline results from PULSAR in a virtual meeting of the ATS as well as interactions with health authorities as we prepare for future Phase 3 development of sotatercept on our path to potential global registration."
Program Highlights
Hematology
REBLOZYL (luspatercept-aamt): Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
REBLOZYL is the first and only U.S. FDA approved erythroid maturation agent designed to promote red blood cell production through a novel mechanism. Luspatercept-aamt is being developed to treat anemia in patients with beta-thalassemia, MDS, and MF. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.
In April, Acceleron and partner Bristol Myers Squibb announced the FDA approved REBLOZYL for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
The New England Journal of Medicine published results from the pivotal Phase 3 MEDALIST and BELIEVE trials.
In April, REBLOZYL received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the treatment of adult patients with:
transfusion-dependent anemia due to very low-, low- and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy; and
transfusion-dependent anemia associated with beta thalassemia
Bristol Myers Squibb expects to initiate the Phase 3 INDEPENDENCE trial in patients with MF on concomitant JAK 2 inhibitor therapy who require RBC transfusions by year-end 2020.
The BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia is ongoing, with topline results expected by year-end 2020 or early 2021.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve, lower-risk MDS, with topline results expected in 2022.
Pulmonary
Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.
In January, Acceleron reported that the PULSAR Phase 2 trial in patients with PAH met its primary and key secondary endpoints. Sotatercept was generally well tolerated in the trial and adverse events observed were generally consistent with previously published data on sotatercept in other diseases.
PULSAR Phase 2 topline results are expected to be presented in an American Thoracic Society (ATS) 2020 web-based session by the end of June 2020.
Acceleron plans to host a webcast and conference call for investors and analysts following the ATS 2020 virtual presentation to discuss highlights from the PULSAR trial results.
In April, sotatercept was granted Breakthrough Therapy designation by the FDA for the treatment of patients with PAH (World Health Organization Group 1).
The EMA recently granted Priority Medicines (PRIME) designation to sotatercept for the treatment of patients with PAH.
Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.
Financial Results
Cash Position – Cash, cash equivalents and investments as of March 31, 2020 were $415.6 million, as compared to $453.8 million as of December 31, 2019. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from REBLOZYL sales.
Revenue – Revenue for the first quarter of 2020 was $4.3 million, which includes $2.8 million of cost share revenue, and $1.5 million of royalty revenue from net U.S. sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
Costs and Expenses – Total costs and expenses for the first quarter of 2020 were $55.9 million. This includes R&D expenses of $37.7 million and SG&A expenses of $18.3 million.
Net Loss – The Company’s net loss for the first quarter of 2020 was $50.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2020 financial results and provide an update on recent corporate activities on May 11, 2020, at 5:00 p.m. EDT.
The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2020 Earnings Call."
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.