Acceleron Pharma Inc.
Nov 7, 2017

Acceleron Pharma Reports Third Quarter 2017 Operational and Financial Results

-Presented vision and strategic focus in hematological, neuromuscular, and pulmonary diseases at September R&D Day-

-Obtained the rights to fund, develop, and lead global commercialization of sotatercept in pulmonary arterial hypertension; robust preclinical results show potential for first-in-class disease modifying approach-

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today provided a corporate update and reported financial results for the third quarter ended September 30, 2017.

"As we think about and plan to execute on our long-term vision and strategy, there were several significant corporate events in the third quarter. At our September R&D day, we outlined our core research and development focus in three disease areas of high unmet medical need: hematology, neuromuscular, and pulmonary disease. We announced that we gained rights to sotatercept, an internally discovered Phase 2 asset, for the development in pulmonary arterial hypertension. We also completed a successful equity offering that will provide sufficient funding through key inflection points in each of our clinical programs," said Habib Dable, President and Chief Executive Officer of Acceleron. "We and our partner Celgene continue to invest heavily in our luspatercept development plan with seven clinical trials expected to be ongoing in 2018. In neuromuscular diseases, ACE-083 continues to advance in the Phase 2 trials in FSHD and CMT, and we remain on track to launch a Phase 2 trial with sotatercept in the first half of 2018 as we work to grow our pulmonary franchise, and ultimately deliver transformative treatment options to patients in need."

Development Program Highlights



Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis

Luspatercept is designed to treat chronic anemia and reduce red blood cell (RBC) transfusion burden in adults with rare blood disorders. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

Neuromuscular Disease


Facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease

ACE-083 is a locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.


ACE-2494 is a protein therapeutic designed to have a systemic effect on muscle mass and strength throughout the body by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

Pulmonary Disease


Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the TGF-beta protein superfamily involved in remodeling and regeneration of a variety of different tissues, including the vasculature and fibrotic tissue.

Key Corporate Priorities





Financial Results

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its third quarter 2017 financial results and provide an update on recent clinical development and corporate activities on November 7, 2017, at 8:00 a.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company's website at Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron Third Quarter 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 Cambridge-based, clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and ACE-2494, and a pulmonary program with a Phase 2 trial of sotatercept planned in pulmonary arterial hypertension.

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

(Amounts in thousands)
    September 30, 2017   December 31, 2016
Cash and cash equivalents $ 238,959   $ 20,950
Short and long-term investments 127,638 213,432
Other assets 14,680 13,265
Total assets $ 381,277 $ 247,647
Deferred revenue $ 3,838 $ 4,245
Warrants to purchase common stock 1,927 1,244
Other liabilities 16,971 16,561
Total liabilities 22,736 22,050
Total stockholders' equity 358,541 225,597
Total liabilities and stockholders' equity $ 381,277 $ 247,647
(Amounts in thousands except per share data)

Three Months Ended
September 30,


Nine Months Ended
September 30,

2017   2016 2017   2016
Collaboration revenue $ 3,014 $ 3,005 $ 9,776 $ 24,402
Costs and expenses:
Research and development 21,059 17,102 64,387 49,492
General and administrative 7,533     6,411   26,735     19,029  
Total costs and expenses 28,592   23,513   91,122   68,521  
Loss from operations (25,578 ) (20,508 ) (81,346 ) (44,119 )
Total other income (expense) net 86     (282 ) 791     6,374  
Loss before income taxes (25,492 ) (20,790 ) (80,555 ) (37,745 )
Income tax benefit 41     20   29     20  
Net loss applicable to common stockholders - basic and diluted $ (25,451 )   $ (20,770 ) $ (80,526 )   $ (37,725 )

Net loss per share applicable to common stockholders - basic and

$ (0.65 ) $ (0.55 ) $ (2.08 ) $ (1.01 )

Weighted-average number of common shares used in computing net
loss per share applicable to common stockholders

39,361 37,616 38,804 37,268

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 of the Company's compounds, the timeline for clinical development and regulatory approval of the Company's compounds and the expected timing for reporting of data from ongoing clinical trials. 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 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 ongoing or later clinical trials, that the development of the Company's compounds will take longer and/or cost more than planned, that the Company or its collaboration partner, Celgene, will be unable to successfully complete the clinical development of the Company's compounds, that the Company or Celgene 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, 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.

Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
BMC Communications
Brad Miles, 646-513-3125

Source: Acceleron Pharma

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