On August 5, 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 second quarter ended June 30, 2021 (Press release, Acceleron Pharma, AUG 5, 2021, View Source [SID1234585867]).

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"We were very pleased to highlight clinical updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept reported at the annual ATS medical meeting and outline our plans for future long-term growth in rare pulmonary diseases at our Research & Development Day during the second quarter," said Habib Dable, President and Chief Executive Officer of Acceleron. "Beyond our sotatercept clinical program in pulmonary arterial hypertension, most recently, we announced plans to expand the development of sotatercept into a Phase 2 trial in patients with Group 2 pulmonary hypertension and develop ACE-1334 in systemic sclerosis-associated interstitial lung disease. To date, our pulmonary pipeline has grown to include seven ongoing and planned clinical trials, showcasing our long-term commitment to becoming a global leader in rare pulmonary disease."

Added Mr. Dable: "With respect to our hematology program, along with our commercial partner Bristol-Myers Squibb Company (Bristol Myers Squibb), we continue to be pleased with product uptake. Our joint commercial teams are presently focused on expanding the reach to appropriate patients earlier in their MDS journey—where there is particular demand—along with the optimal dose of REBLOZYL to maximize patient benefit and increased duration of treatment in this population to drive further growth this year and beyond. In June, we presented results from the BEYOND Phase 2 trial of luspatercept during the Presidential Symposium at EHA (Free EHA Whitepaper). The study achieved its primary endpoint of an increase in hemoglobin of at least 1 gram per deciliter in the luspatercept treated group compared to placebo for the treatment of anemia in adults with non-transfusion dependent beta-thalassemia, supporting the rationale for its potential development in additional patient groups."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Hypertension
Sotatercept acts as an investigational reverse-remodeling agent proposed to rebalance TGF-beta superfamily signaling. In preclinical models of PAH, sotatercept reversed pulmonary arterial wall and right ventricular remodeling that are hallmarks of the disease.

In May, Acceleron presented updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept in patients with PAH at the American Thoracic Society (ATS) 2021 International Conference.
Enrollment is ongoing in the registrational STELLAR Phase 3 trial in patients with PAH.
Study start up activities are underway for the HYPERION and ZENITH Phase 3 trials of sotatercept in expanded PAH patient populations.
Acceleron expects to initiate the CADENCE Phase 2 trial in patients with pulmonary hypertension with left heart disease this year.
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.

Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD by year-end 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.

Acceleron recognized approximately $25.6 million in royalty revenue from approximately $128 million in net sales of REBLOZYL in the second quarter of 2021. This compares with approximately $22.4 million in royalty revenue from approximately $112 million in net sales of REBLOZYL in the first quarter of 2021.
In June, the Companies presented results from multiple abstracts on luspatercept at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress.
Results from the BEYOND Phase 2 trial of luspatercept in adult patients with non-transfusion dependent beta-thalassemia were presented during the Presidential Symposium, which honors the top six research papers submitted for presentation at the meeting.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Enrollment is ongoing in the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
Corporate Highlights

Acceleron published its inaugural Environmental, Social, and Governance (ESG) report in June, which provides a comprehensive summary of the Company’s broad range of ESG initiatives. Acceleron has adopted the accounting standards for the biotechnology and pharmaceuticals industry from the Sustainability Accounting Standards Board (SASB) to help develop and prioritize areas for inclusion in its ESG report. The full ESG report can be found on Acceleron’s Investor Relations website by clicking here.
On June 22, 2021, members of the Acceleron leadership team, along with external experts, reviewed the Company’s numerous ongoing and planned trials in rare pulmonary diseases, and highlighted Acceleron’s vision and strategy for long-term growth. An archived recording of the video webcast of the presentations and question and answer sessions is accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com.
Financial Results

Cash Position – Cash, cash equivalents and investments as of June 30, 2021 were $712.5 million, compared with $857.5 million as of December 31, 2020. 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 second quarter of 2021 was $27.9 million, which includes $2.3 million of cost share revenue and $25.6 million of royalty revenue from net sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
R&D Expenses – GAAP R&D expenses were $56.1 million for the second quarter of 2021. Non-GAAP R&D expenses were $49.3 million for the second quarter of 2021, excluding $5.9 million and $0.9 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $35.5 million for the second quarter of 2021. Non-GAAP SG&A expenses were $27.8 million for the second quarter of 2021, excluding $7.6 million and $0.1 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Net Loss – The Company’s GAAP net loss for the second quarter of 2021 was $63.5 million, or $1.05 per share. Non-GAAP adjusted net loss for the second quarter was $49.0 million, or $0.81 per share, excluding $13.5 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Non-GAAP Financial Measures

Acceleron supplements its results of operations prepared in accordance with U.S. generally accepted accounting principles, or GAAP, with certain non-GAAP financial measures, including non-GAAP R&D expense, non-GAAP SG&A expense, adjusted net loss and adjusted net loss per share, that exclude stock-based compensation expense and depreciation and amortization expense. These results should not be viewed as a substitute for the Company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures provide investors with additional insight into underlying trends of the Company’s ongoing business, and are important in comparing current results with prior period results. Investors are cautioned that there are material limitations associated with the use of non-GAAP financial measures. In addition, other companies may report similarly titled non-GAAP measures, but calculate them differently, which reduces their usefulness as a comparative measure. In the reconciliation tables below, Acceleron presents these non-GAAP financial measures reconciled to their comparable GAAP financial measures.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its second quarter 2021 financial results on August 5, 2021, 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 www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #5736809.

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