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 –
“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
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
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.
Celgeneplan to submit regulatory applications in the United Statesand Europein 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 Congressof 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.
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
- 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 FSHD.
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 ligands.
- Enrollment and treatment are ongoing in the Phase 1 healthy volunteer trial with preliminary results expected in the first half of 2019.
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.
November 2018, the Company will host a PAH Researchand 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 results.
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 asVice President and Group Head of Pulmonology Clinical Developmentat 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.
- 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
Celgeneand 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 $28.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its
second quarter financial results for 2018 and provide an update on
recent corporate activities on
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the “Acceleron Second Quarter 2018 Earnings Call.”
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED BALANCE SHEET|
|(Amounts in thousands)|
June 30, 2018
|December 31, 2017|
|Cash and cash equivalents||$||79,592||$||100,150|
|Short and long-term investments||252,666||272,800|
|Warrants to purchase common stock||1,689||2,236|
|Total stockholders’ equity||333,109||365,217|
|Total liabilities and stockholders’ equity||$||351,541||$||389,177|
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands except per share data)|
|Three Months Ended June 30,||Six Months Ended June 30,|
|Costs and expenses:|
|Research and development||25,933||21,598||49,363||43,327|
|General and administrative||7,658||11,370||15,099||19,203|
|Total costs and expenses||33,591||32,968||64,462||62,530|
|Loss from operations||(29,906||)||(29,911||)||(57,545||)||(55,768||)|
|Total other income, net||979||248||2,410||705|
|Loss before income taxes||(28,927||)||(29,663||)||(55,135||)||(55,063||)|
|Income tax provision||(11||)||(6||)||(21||)||(12||)|
|Net loss applicable to common stockholders- basic and diluted||$||(28,938||)||$||(29,669||)||$||(55,156||)||$||(55,075||)|
|Net loss per share applicable to common stockholders- basic and diluted||$||(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
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
Matt Fearer, 617-301-9557
Director, Corporate Communications