Acceleron Pharma Inc.
Nov 3, 2016

Acceleron Pharma Reports Third Quarter 2016 Financial and Operational Results

- Appointed Habib Dable as President and CEO effective December 1, 2016 -

- Five upcoming presentations at American Society of Hematology Annual Meeting in December -

- Advancing ACE-083 into Phase 2 study for facioscapulohumeral muscular dystrophy (FSHD) -

- Maintained strong balance sheet with $251 million in cash to fund projected operating requirements into the second half of 2019 -

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 third quarter ended September 30, 2016.

"In the third quarter, we continued to make strong progress across our pipeline of clinical and preclinical programs," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Enrollment continues to go well in both of the ongoing luspatercept Phase 3 studies in patients with myelodysplastic syndromes and beta-thalassemia. We look forward to providing new and updated preliminary results from several Phase 2 studies at the upcoming American Society of Hematology annual meeting in December. Within our muscle program, we're on track to initiate the ACE-083 Phase 2 study in patients with facioscapulohumeral muscular dystrophy before year-end."

Continued Dr. Knopf, "I would also like to extend a warm welcome to the Company's newly appointed Chief Executive Officer, Habib Dable, who will assume this role on December 1st. Habib's global pharmaceutical operational and commercial experience will serve Acceleron well as we advance toward becoming a commercial-stage company."


Development Programs


Neuromuscular Disease


Preclinical Research and Development

Other Corporate Updates

Upcoming Events in Q4 2016

Financial Results

Conference Call and Webcast

The Company will host a live conference call and webcast to discuss its third quarter 2016 financial results and provide a corporate update on November 3, 2016, at 5:00 p.m. EDT. Participants can access the live conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron Earnings Call."

The live webcast can be accessed on the Investors page of the Company's website at

A replay of the webcast will be available approximately two hours after the event on the Company's website.

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. This approach to drug discovery has generated four therapeutic candidates that are currently in clinical trials. 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 Corp. Acceleron is also advancing clinical programs in the fields of oncology and neuromuscular diseases and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases.

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




(Amounts in thousands)


September 30, 2016 December 31, 2015
Cash and cash equivalents $ 31,016 $ 27,783
Short and long-term investments 220,007 108,198
Other assets 11,862   10,356
Total assets $ 262,885   $ 146,337
Deferred revenue 4,380 4,794
Warrants to purchase common stock 12,161 17,187
Other liabilities 16,797   15,093
Total liabilities 33,338   37,074
Total stockholders' equity 229,547   109,263
Total liabilities and stockholders' equity $ 262,885   $ 146,337



(Amounts in thousands except per share data)


Three Months Ended September 30, Nine Months Ended September 30,
2016   2015 2016   2015
Collaboration revenue:
Collaboration revenue $ 3,005 $ 4,155 $ 24,402 $ 14,294
Costs and expenses:
Research and development 17,102 13,335 49,492 42,261
General and administrative 6,411     5,433   19,029     14,796  
Total costs and expenses 23,513 18,768 68,521 57,057
Loss from operations (20,508 ) (14,613 ) (44,119 ) (42,763 )
Total other (expense) income, net (282 ) 2,755   6,374   5,951  
Loss before income taxes (20,790 ) (11,858 ) (37,745 ) (36,812 )
Income tax benefit 20       20      
Net loss applicable to common stockholders—basic and diluted $ (20,770 )   $ (11,858 ) $ (37,725 )   $ (36,812 )
Net loss per share applicable to common stockholders - basic and diluted $ (0.55 ) $ (0.36 ) $ (1.01 ) $ (1.12 )
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders - basic and diluted 37,616 33,097 37,268 32,869

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, including sotatercept, luspatercept, dalantercept, ACE-083, ACE-2494, the Company's IntelliTrap™ drug discovery platform, and the Company's TGF-beta superfamily program generally, the timeline for clinical development and regulatory approval of the Company's compounds, the expected timing for the reporting of data from ongoing trials, and the structure of the Company's planned or pending clinical trials. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "project," "should," "strategy," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the Company's cash, cash equivalents and investments will be insufficient to fund operations into the second half of 2019, 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 data may not be available when the Company expects it to be, that the Company or its collaboration partner, Celgene, will be unable to successfully complete the clinical development of the Company's compounds, that the development of the Company's compounds will take longer or cost more than planned, that the Company or Celgene may be delayed in initiating or completing any clinical trials, that the Company's drug discovery activities may not yield drug candidates for which the Company can commence clinical trials at the rate at which the Company currently anticipates or at all, and that the Company's compounds will not receive regulatory approval or become commercially successful products.

Other risks and uncertainties include those identified under the heading "Risk Factors" included in the Company's Annual Report on Form 10-K which was filed with the Securities and Exchange Commission (SEC) on February 25, 2016, 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 reflect the Company's current views 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
Senior Director, Investor Relations and Corporate Communications
BMC Communications
Brad Miles, 646-513-3125

Source: Acceleron Pharma

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