On February 25, 2021 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 fourth quarter and full year ended December 31, 2020 (Press release, Acceleron Pharma, FEB 25, 2021, View Source [SID1234575641]).

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"2020 was a very productive year for Acceleron as we generated important results in our lead pulmonary program to advance sotatercept into a registrational Phase 3 trial and executed on a successful first full year of the commercial launch of REBLOZYL in the United States," said Habib Dable, President and Chief Executive Officer of Acceleron. "I would like to acknowledge our team’s strong commitment to our company and mission as these important milestones along with many others were achieved despite the many challenges resulting from the ongoing global pandemic. Throughout the year, we presented updates from our two Phase 2 trials with our lead pulmonary program, supporting our long-term vision of establishing sotatercept as a backbone therapy for patients with pulmonary arterial hypertension across all stages of the disease."

Added Mr. Dable: "As we continue to grow our capabilities in rare pulmonary disease, we are also advancing ACE-1334 into a Phase1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease later this year. We look forward to hosting a research and development day to provide additional details on our long-term vision and programs in rare pulmonary disease in the middle of 2021."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)

Sotatercept is an investigational reverse-remodeling 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. The PULSAR Phase 2 trial evaluating sotatercept in combination with approved PAH-specific medicines in patients with PAH achieved its primary endpoint of improvement in pulmonary vascular resistance and its key secondary endpoint of improvement in 6-minute walk distance.

In November, the Company presented the 24-week echocardiography results from the PULSAR Phase 2 trial at the virtual 2020 American Heart Association (AHA) Scientific Sessions, which earned the AHA’s "Cardiopulmonary Best Abstract" award. In addition, preliminary interim results from the SPECTRA Phase 2 trial were presented at the congress.
In December, the European Commission (EC) granted Orphan Drug designation to sotatercept for the treatment of patients with PAH.
In December, Acceleron initiated its registrational STELLAR Phase 3 trial in patients with PAH.
The Company expects to initiate the HYPERION (early intervention) Phase 3 trial and the ZENITH (WHO Functional Class IV) Phase 3 trial in expanded PAH populations in the middle of 2021.
Results from the open-label extension period of the PULSAR Phase 2 trial and additional results from the SPECTRA Phase 2 trial are expected in the first half of 2021.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis. ACE-1334 recently completed an ascending-dose Phase 1 clinical trial in healthy volunteers.

In December, the United States Food and Drug Administration (FDA) granted Orphan Drug designation to ACE-1334 for the treatment of patients with systemic sclerosis.
Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD in 2021.
Hematology

REBLOZYL (luspatercept-aamt):

REBLOZYL is the first and only approved erythroid maturation agent designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

The Company recognized approximately $23.0 million in royalty revenue from approximately $115 million in net sales of REBLOZYL in the fourth quarter of 2020. This compares with approximately $19.3 million in royalty revenue from approximately $96 million in net sales of REBLOZYL in the third quarter of 2020.
For the full year 2020, Acceleron recognized approximately $54.8 million in royalty revenue from approximately $274 million in net sales of REBLOZYL.
In February 2021, Acceleron and partner Bristol Myers Squibb announced that Health Canada approved REBLOZYL for the treatment of adult patients with transfusion-dependent anemia requiring at least two RBC red blood cell 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.
Six clinical abstracts on REBLOZYL were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition held virtually in December 2020.
The INDEPENDENCE Phase 3 trial is initiating in patients with myelofibrosis.
The Companies expect to present results from the BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia in the first half of 2021.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Corporate Highlights

Acceleron is planning to host a research and development day in the middle of 2021 to provide a deep dive into all of the Company’s programs.
Financial Results

Cash Position – Cash, cash equivalents and investments as of December 31, 2020 were $857.5 million. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the fourth quarter of 2020 was $25.9 million, which includes $2.9 million of cost share revenue and $23.0 million of royalty revenue from net sales of REBLOZYL. Revenue for the full year was $92.5 million, which includes $12.7 million of cost share revenue, $54.8 million of royalty revenue from net sales of REBLOZYL, and the recognition of a $25.0 million regulatory-based milestone for the approval of REBLOZYL in Europe. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
Costs and Expenses – Total costs and expenses for the fourth quarter of 2020 were $83.5 million. This includes R&D expenses of $57.3 million and SG&A expenses of $26.2 million. Total costs and expenses for the full year were $259.8 million. This includes R&D expenses of $173.9 million and SG&A expenses of $85.9 million.
Net Loss – The Company’s net loss for the fourth quarter of 2020 was $57.4 million. The Company’s net loss for the year ended December 31, 2020 was $166.0 million.
Conference Call and Webcast

The Company will host a webcast and conference call to discuss its fourth quarter and full year 2020 financial results on February 25, 2021, at 5:00 p.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #4282162.

An archived version of the webcast will be available for replay on the Company’s website for approximately one year.