Acceleron Pharma Inc.
Aug 3, 2017

Acceleron Pharma Reports Second Quarter 2017 Operational and Financial Results

- Completed full enrollment of luspatercept MEDALIST and BELIEVE Phase 3 trials -

- Presented updated luspatercept Phase 2 results in patients with MDS and beta-thalassemia receiving treatment for up to two years -

- Treated first patient in ACE-083 Phase 2 study in Charcot-Marie-Tooth disease -

- Upcoming R&D Day on September 19th to highlight Acceleron's pipeline and strategic focus -

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, today provided a corporate update and reported financial results for the second quarter ended June 30, 2017.

"The second quarter, and 2017 as a whole, have been marked by tremendous progress and operational execution across our hematology and neuromuscular programs," said Habib Dable, President and Chief Executive Officer of Acceleron. "Over 560 patients were enrolled ahead of estimated timing in our MEDALIST and BELIEVE Phase 3 trials combined, and our partner Celgene continues to invest in new opportunities for luspatercept. Our recent Phase 2 luspatercept results continue to support our confidence in achieving long-term, clinically meaningful outcomes for patients. In addition, our wholly-owned muscle compound ACE-083 has recently expanded its Phase 2 development program into a second neuromuscular disease. We look forward to outlining our long-term vision and growth strategy at our upcoming R&D day in September."

Development Program Highlights

Hematology - Luspatercept

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

Luspatercept is designed to treat the condition of 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 - ACE-083

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.

Preclinical Research

Acceleron continues its research on several molecules targeting musculoskeletal diseases, fibrotic disorders and other serious diseases.

Key Corporate Priorities

Luspatercept

ACE-083

ACE-2494

Research & Development

Financial Results

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its second quarter 2017 financial results and provide an update on recent clinical development and corporate activities on August 3, 2017, 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. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron Second 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 clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases. Its pioneering research platform leverages the powerful biology behind the body's ability to rebuild and repair its own cells and tissues. The Company's lead therapeutic candidate, luspatercept, is being evaluated in Phase 3 studies for the treatment of the hematologic diseases myelodysplastic syndromes (MDS) and beta-thalassemia under a global partnership with Celgene. Acceleron is also advancing its ACE-083 clinical program in the field of neuromuscular disease, and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases.

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

         

ACCELERON PHARMA INC.

CONDENSED CONSOLIDATED BALANCE SHEET

(Amounts in thousands)

(unaudited)

 
June 30, 2017     December 31, 2016
Cash and cash equivalents $ 30,726 $ 20,950
Short and long-term investments 163,312 213,432
Other assets 14,425   13,265
Total assets $ 208,463   $ 247,647
 
Deferred revenue $ 3,973 $ 4,245
Warrants to purchase common stock 1,516 1,244
Other liabilities 14,442   16,561
Total liabilities 19,931   22,050
Total stockholders' equity 188,532   225,597
Total liabilities and stockholders' equity $ 208,463   $ 247,647
 
         

ACCELERON PHARMA INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(Amounts in thousands except per share data)

(unaudited)

 

Three Months Ended
June 30

Six Months Ended
June 30

2017     2016 2017     2016
Revenue:        
Collaboration revenue $ 3,057 $ 3,195 $ 6,762 $ 21,396
Costs and expenses:
Research and development 21,598 16,138 43,327 32,390
General and administrative 11,370       6,712   19,203       12,618  
Total costs and expenses 32,968   22,850   62,530   45,008  
Loss from operations (29,911 ) (19,655 ) (55,768 ) (23,612 )
Total other income (expense) net 248       (2,361 ) 705       6,656  
Loss before income taxes (29,663 ) (22,016 ) (55,063 ) (16,956 )
Income tax provision (6 )       (12 )      
Net loss applicable to common stockholders - basic and diluted $ (29,669 )     $ (22,016 ) $ (55,075 )     $ (16,956 )
 
Net loss per share applicable to common stockholders - basic and diluted $ (0.77 ) $ (0.59 ) $ (1.43 ) $ (0.46 )
 
Weighted-average number of common shares used in computing net (loss) income per share applicable to common stockholders 38,631 37,272 38,515 37,092
 

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," "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
or
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
or
Media:
BMC Communications
Brad Miles, 646-513-3125

Source: Acceleron Pharma

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