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, today provided a corporate update and reported financial results for the first quarter ended March 31, 2020.
“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 U.S. 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.
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.
Financial Results
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.
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 commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.
For more information, please visit acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.
ACCELERON PHARMA INC. |
||||||||
CONDENSED CONSOLIDATED BALANCE SHEET |
||||||||
(Amounts in thousands) |
||||||||
(unaudited) |
||||||||
|
March 31, 2020 |
|
December 31, 2019 |
|||||
Cash and cash equivalents |
$ |
277,486 |
|
|
$ |
237,677 |
|
|
Short and long-term investments |
138,077 |
|
|
216,169 |
|
|||
Operating lease - right of use asset, net |
22,531 |
|
|
23,908 |
|
|||
Other assets |
26,068 |
|
|
27,152 |
|
|||
Total assets |
$ |
464,162 |
|
|
$ |
504,906 |
|
|
|
|
|
|
|||||
Operating lease liability - right of use, short-term and long-term |
$ |
24,918 |
|
|
$ |
26,384 |
|
|
Warrants to purchase common stock |
3,304 |
|
|
1,856 |
|
|||
Other liabilities |
22,118 |
|
|
27,190 |
|
|||
Total liabilities |
50,340 |
|
|
55,430 |
|
|||
Total stockholders’ equity |
413,822 |
|
|
449,476 |
|
|||
Total liabilities and stockholders’ equity |
$ |
464,162 |
|
|
$ |
504,906 |
|
ACCELERON PHARMA INC. |
||||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
||||||||
(Amounts in thousands except per share data) |
||||||||
(unaudited) |
||||||||
|
Three Months Ended March 31, 2020 |
|||||||
|
2020 |
|
2019 |
|||||
Revenue: |
|
|
|
|||||
Collaboration revenue: |
$ |
4,344 |
|
|
$ |
2,780 |
|
|
Costs and expenses: |
|
|
|
|||||
Research and development |
37,663 |
|
|
32,771 |
|
|||
Selling, general and administrative |
18,253 |
|
|
10,814 |
|
|||
Total costs and expenses |
55,916 |
|
|
43,585 |
|
|||
Loss from operations |
(51,572 |
) |
|
(40,805 |
) |
|||
Other income, net |
648 |
|
|
2,772 |
|
|||
Loss before income taxes |
(50,924 |
) |
|
(38,033 |
) |
|||
Income tax provision |
(15 |
) |
|
(20 |
) |
|||
Net loss |
$ |
(50,939 |
) |
|
$ |
(38,053 |
) |
|
|
|
|
|
|||||
Net loss per share- basic and diluted |
$ |
(0.95 |
) |
|
$ |
(0.74 |
) |
|
|
|
|
|
|||||
Weighted-average number of common shares used in computing net loss per share- basic and diluted |
53,361 |
|
|
51,126 |
|
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about the Company's strategy, future plans and prospects, including statements regarding the development and commercialization of the Company's compounds, the timeline for clinical development and regulatory approval of the Company's compounds, the expected timing for reporting of data from ongoing clinical trials, the Company's future cash position and the potential of REBLOZYL® (luspatercept-aamt) 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 preclinical testing of the Company's compounds and data from clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of the Company’s compounds in one indication or country may not be predictive of approval in another indication or country, that the development of the Company’s compounds will take longer and/or cost more than planned or accelerate faster than currently expected, that the Company or its collaboration partner, Bristol-Myers Squibb Corporation (“BMS”), will be unable to successfully complete the clinical development of the Company’s compounds, that the Company or BMS may be delayed in initiating, enrolling or completing any clinical trials, and that the Company’s compounds will not receive regulatory approval or become commercially successful products. These and other risks and uncertainties are identified under the heading “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that the Company 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 the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200511005852/en/
Acceleron Pharma Inc.
Investors:
Todd James, 617-649-9393
Senior Vice President, Corporate Affairs and Investor Relations
Media:
Matt Fearer, 617-301-9557
Director, Corporate Communications