Acceleron Pharma Inc.
Mar 1, 2017

Acceleron Reports Fourth Quarter and Year Ended 2016 Operational and Financial Results

- Luspatercept Phase 3 trials to complete enrollment in both the MDS ("MEDALIST") and beta-thalassemia ("BELIEVE") trials in 2H 2017 -

- Presented data from five abstracts on luspatercept and sotatercept at the American Society of Hematology Annual Meeting in December -

- Treated first patient in ACE-083 Phase 2 study for facioscapulohumeral muscular dystrophy -

- Outlined plans to initiate several new clinical trials in 2017 -

- Maintained strong balance sheet with $234.4 million in cash and investments 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 fourth quarter and year ended December 31, 2016.

"Acceleron made tremendous progress in 2016 across our entire portfolio of potentially first- and best-in-class therapies for patients with serious hematologic, neuromuscular and fibrotic diseases without effective treatments," said Habib Dable, President and Chief Executive Officer of Acceleron. "The pace of recruitment continues to accelerate in both of our ongoing Phase 3 luspatercept trials, and we are highly confident that Celgene will complete enrollment in the second half of 2017. Furthermore, we and Celgene plan to expand the program by initiating new Phase 2 trials in non-transfusion dependent beta-thalassemia and myelofibrosis patient populations highlighting our confidence in the blockbuster potential of luspatercept. With encouraging preliminary results and multiple ongoing studies across all of our programs, we are well positioned for a transformational year in 2017."



Luspatercept in myelodysplastic syndromes (MDS)

Luspatercept is being developed to treat anemia and reduce or eliminate the need for red blood cell transfusions in patients with MDS, a rare disorder in which bone marrow fails to produce enough healthy blood cells. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

Luspatercept in beta-thalassemia

Luspatercept is designed to treat severe, chronic anemia and reduce RBC transfusion-dependence in adults with beta-thalassemia, a rare genetic disorder that reduces the production of hemoglobin. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

Luspatercept and sotatercept in myelofibrosis

Myelofibrosis is a malignant disease which results in fibrotic bone marrow, multiple cytopenias and severe anemia often requiring RBC transfusions. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

Neuromuscular Disease

ACE-083 in facioscapulohumeral muscular dystrophy (FSHD)

ACE-083 is designed to increase muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss.


Dalantercept in Advanced Renal Cell Carcinoma

Dalantercept is being developed in combination with axitinib to further inhibit tumor angiogenesis.

Preclinical Research

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









Conference Call and Webcast Information

The Company will host a live conference call and webcast to discuss its fourth quarter and full year financial results for 2016 and provide a corporate update on Wednesday, March 1, 2017, at 8:00 AM EST. 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 archived on the Company's website and accessible 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. 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.

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(Amounts in thousands)


31, 2016

31, 2015

Cash and cash equivalents $ 20,950 $ 27,783
Short and long-term investments 213,432 108,198
Other assets 13,265   10,356  
Total assets $ 247,647   $ 146,337  
Deferred revenue $ 4,245 $ 4,794
Warrants to purchase common stock 1,244 17,187
Other liabilities 16,561   15,093  
Total liabilities 22,050 37,074
Total stockholders' equity 225,597   109,263  
Total liabilities and stockholders' equity $ 247,647   $ 146,337  


(Amounts in thousands except per share data)

Three Months Ended December 31, Year Ended December 31,
2016   2015 2016   2015
Collaboration revenue $ 3,369 $ 3,804 $ 27,771 $ 18,097
Costs and expenses:
Research and development 19,088 16,146 68,580 58,404
General and administrative 6,267   5,773   25,297   20,572  
Total costs and expenses 25,355 21,919 93,877 78,976
Loss from operations (21,986 ) (18,115 ) (66,106 ) (60,879 )
Other income (expense), net 2,742   (8,967 ) 9,116   (3,015 )
Loss before income taxes (19,244 ) (27,082 ) (56,990 ) (63,894 )
Income tax provision (44 )   (24 )  
Net loss $ (19,288 ) $ (27,082 ) $ (57,014 ) $ (63,894 )
Other comprehensive loss:
Net unrealized holding losses on short-term and long-term investments during the period (83 ) (199 ) (205 ) (220 )
Comprehensive loss $ (19,371 ) $ (27,281 ) $ (57,219 ) $ (64,114 )
Net loss per share - basic and diluted $ (0.51 ) $ (0.81 ) $ (1.52 ) $ (1.92 )
Weighted-average number of common shares used in computing net loss per share - basic and diluted 37,914 33,268 37,430 33,303

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," "appear," "believe," "continue," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "predict," "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.

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, enrolling 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 March 1, 2017, 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.

Source: Acceleron Pharma

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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