Market Overview

Acceleron Reports Second Quarter 2018 Operating and Financial Results


– MEDALIST and BELIEVE Phase 3 trials met all primary and key
secondary endpoints –

– ACE-083 Part 2 of the Phase 2 trials in facioscapulohumeral
muscular dystrophy and Charcot-Marie-Tooth disease are underway –

– PULSAR Phase 2 trial initiated with sotatercept in pulmonary
arterial hypertension –

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 second quarter ended June 30, 2018.

"We have had a very successful start to 2018, and we look forward to
carrying this momentum forward throughout the rest of the year and
beyond. We are extremely pleased that luspatercept, our lead product
candidate and first-in-class erythroid maturation agent, achieved
positive Phase 3 results in both the MEDALIST and BELIEVE trials,
confirming our confidence in its clinical profile in myelodysplastic
syndromes and beta-thalassemia," said Habib Dable, President and Chief
Executive Officer of Acceleron. "We and our collaboration partner,
Celgene, look forward to presenting the Phase 3 data at an upcoming
medical congress and are focused on the execution of key regulatory
activities, including US and EU application submissions in the first
half of 2019. We believe luspatercept is a potential platform treatment
to transform the lives of patients suffering from a range of hematologic
diseases associated with anemia."

Added Mr. Dable: "Our neuromuscular and pulmonary programs achieved
critical milestones, putting us in a position for important Phase 2
readouts for ACE-083 and sotatercept in 2019 and 2020, respectively."

Development Program Highlights



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

Luspatercept is a first-in-class erythroid maturation agent (EMA)
designed to treat the late-stage erythroid maturation defect that
results in chronic anemia and the need for regular red blood cell
transfusions in adults with serious hematologic diseases. Luspatercept
is part of the global collaboration between Acceleron and Celgene.

  • The MEDALIST and BELIEVE Phase 3 trials in patients with lower-risk
    MDS and transfusion-dependent beta-thalassemia, respectively, met all
    primary and key secondary endpoints.
    • Data will be submitted to a future medical meeting for
      presentation in late 2018.
    • Acceleron and Celgene plan to submit regulatory applications in
      the United States and Europe in the first half of 2019.
  • Updated results from the ongoing Phase 2 trials in MDS and
    beta-thalassemia were presented at the 2018 American Society of
    Clinical Oncology (ASCO) Annual Meeting and the 23rd
    Congress of the European Hematology Association (EHA) in June 2018.
  • The initiation of the COMMANDS Phase 3 trial in patients with
    lower-risk MDS who are treatment naïve is planned for the third
    quarter of 2018.
  • Enrollment and treatment are ongoing in the BEYOND Phase 2 trial in
    non-transfusion-dependent beta-thalassemia and the Phase 2 trial in MF.

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 focal muscle weakness. ACE-083 utilizes the
"Myostatin+" approach to inhibit multiple TGF-beta ligands.

  • Preliminary results from Part 1 of the ACE-083 Phase 2 trial in
    patients with CMT disease were highlighted in oral and poster
    presentations at the 2018 Peripheral Nerve Society (PNS) Annual
    Meeting in July.
  • Part 2 of the Phase 2 trial in patients with CMT was recently
    initiated with preliminary results expected by the end of 2019.
  • The Company plans to present results from all Part 1 dose cohorts in
    the FSHD Phase 2 trial in October 2018.
  • Enrollment and treatment are ongoing in Part 2 of the Phase 2 FSHD
    trial with preliminary results expected in the second half of 2019.
  • ACE-083 received FDA Fast Track status and Orphan Drug designation in


ACE-2494 is designed to have a systemic effect on muscle mass and
strength for diseases that cause muscle weakness throughout the body.
ACE-2494 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta

  • Enrollment and treatment are ongoing in the Phase 1 healthy volunteer
    trial with preliminary results expected in the first half of 2019.

Pulmonary Disease


Pulmonary Arterial Hypertension (PAH)

Sotatercept acts as a ligand trap for members of the TGF-beta
superfamily directly involved in the BMP signaling pathway, which is
proven critical for maintaining healthy pulmonary vasculature. In
multiple preclinical studies in PAH, sotatercept significantly decreased
pulmonary vessel muscularization, improved pulmonary arterial pressures,
and decreased indicators of right heart failure.

  • The Company initiated the PULSAR Phase 2 trial in patients with PAH
    with preliminary results expected in the first half of 2020.
  • The Company plans to initiate an exploratory imaging study in Q1 2019
    to provide additional understanding of endpoints in anticipation of a
    potential pivotal trial in the future.
  • In November 2018, the Company will host a PAH Research and Development
    Deep Dive event in New York City.
    • The event will include internal and external expert presentations
      to discuss disease background, the current treatment landscape,
      key disease pathways including BMP signaling, Acceleron's clinical
      development activities, and the latest sotatercept preclinical

Corporate Highlights

  • Robert K. Zeldin, M.D., was appointed Chief Medical Officer (CMO). He
    brings 20 years of industry experience and joined from Ablynx where he
    served as Chief Medical Officer. Prior to Ablynx, Dr. Zeldin served in
    senior roles at Novartis, Merck, and the FDA Center for Biologics
    Evaluation and Research.
  • Janethe Pena, M.D., Ph.D., recently joined the Company as Vice
    President of Pulmonary to lead the company's clinical development
    efforts in this area. Dr. Pena most recently served as Vice
    President and Group Head of Pulmonology Clinical Development at Bayer
    Pharmaceuticals. At Bayer, she was responsible for the pulmonary
    portfolio, including leading clinical trials with riociguat (Adempas®)
    in different pulmonary hypertension indications and life cycle
    management for the program.

Financial Results

  • Cash position – Cash, cash equivalents and investments as of
    June 30, 2018 were $332.3 million. As of December 31, 2017, the
    Company had cash, cash equivalents and investments of $372.9 million.
    The Company believes that existing cash, cash equivalents and
    investments will be sufficient to fund projected operating
    requirements into 2021.
  • Revenue – Collaboration revenue for the second quarter was $3.7
    million. The revenue is all from Acceleron's partnership with Celgene
    and is primarily related to expenses incurred by the Company in
    support of luspatercept.
  • Costs and expenses – Total costs and expenses for the
    second quarter were $33.6 million. This includes R&D expenses of $25.9
    million and G&A expenses of $7.7 million.
  • Net loss – The Company's net loss for the second quarter
    ended June 30, 2018 was $28.9 million.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its
second quarter financial results for 2018 and provide an update on
recent corporate activities on August 2, 2018, 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
Individuals can participate in the conference call by dialing
877-312-5848 (domestic) or 253-237-1155 (international) and referring to
the "Acceleron Second Quarter 2018 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 Phase 2 pulmonary program with sotatercept in pulmonary
arterial hypertension.

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

(Amounts in thousands)

    June 30, 2018    

  December 31, 2017
Cash and cash equivalents $ 79,592   $ 100,150
Short and long-term investments 252,666 272,800
Other assets 19,283   16,227
Total assets $ 351,541   $ 389,177
Deferred revenue $ $ 3,702
Warrants to purchase common stock 1,689 2,236
Other liabilities 16,743   18,021
Total liabilities 18,432 23,960
Total stockholders' equity 333,109     365,217
Total liabilities and stockholders' equity $ 351,541   $ 389,177
(Amounts in thousands except per share data)
  Three Months Ended June 30,   Six Months Ended June 30,
2018   2017 2018   2017
Collaboration revenue $ 3,685 $ 3,057 $ 6,917 $ 6,762
Costs and expenses:
Research and development 25,933 21,598 49,363 43,327
General and administrative 7,658     11,370   15,099     19,203  
Total costs and expenses 33,591   32,968   64,462   62,530  
Loss from operations (29,906 ) (29,911 ) (57,545 ) (55,768 )
Total other income, net 979     248   2,410     705  
Loss before income taxes (28,927 ) (29,663 ) (55,135 ) (55,063 )
Income tax provision (11 )   (6 ) (21 )   (12 )
Net loss applicable to common stockholders- basic and diluted $ (28,938 )   $ (29,669 ) $ (55,156 )   $ (55,075 )
Net loss per share applicable to common stockholders- basic and
$ (0.63 ) $ (0.77 ) $ (1.21 ) $ (1.43 )
Weighted-average number of common shares used in computing net loss
per share applicable to common stockholders
45,789 38,631 45,654 38,515

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 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 ongoing or later clinical
trials, that the results of any clinical trial may not be predictive of
the results or success of other clinical trials of the same product
candidate, that the development of the Company's compounds will take
longer and/or cost more than planned, that the Company will be unable to
successfully complete the clinical development of the Company's
compounds, that the Company 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.

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