Acceleron Reports First Quarter 2019 Operating and Financial Results
– Luspatercept Biologics License Application (BLA) and Marketing
Authorization Application (MAA) submitted in
– Part 2 results from the ACE-083 Phase 2 trials in facioscapulohumeral muscular dystrophy (FSHD) expected in the second half of 2019 and Charcot-Marie-Tooth disease (CMT) anticipated in Q1 2020 –
– PULSAR and SPECTRA Phase 2 trials of sotatercept in pulmonary arterial hypertension (PAH) on track with topline results expected from PULSAR in 1H 2020 –
“With the submission of marketing applications for luspatercept in the
U.S. and E.U. last month, we and our global collaboration partner,
Development Program Highlights
Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid
maturation agent (EMA) designed to address a 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
Celgenerecently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration( FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency(EMA) for luspatercept in patients with MDS- and beta-thalassemia-associated anemia based on the safety and efficacy results of the pivotal Phase 3 studies MEDALIST and BELIEVE.
- The Companies expect to announce preliminary topline results from the Phase 2 trial of luspatercept in patients with MF in the second half of 2019.
- Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia, with preliminary results expected from the BEYOND trial in 2020.
ACE-083: Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)
ACE-083 is an investigational 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 superfamily ligands involved in muscle formation.
Previously presented results from Part 1 of the Phase 2 trial
evaluating ACE-083 in patients with FSHD were highlighted in an encore
presentation at the
Muscular Dystrophy Association (MDA) Clinical & Scientific Conferencein April 2019.
- Preliminary results from Part 2 of the Phase 2 trial in patients with FSHD are expected in the second half of 2019.
Previously presented results from Part 1 of the Phase 2 trial
evaluating ACE-083 in patients with CMT will be highlighted in a
platform presentation at the
American Academy of Neurology(AAN) 71st Annual Meeting on May 10, 2019.
- Enrollment is ongoing in Part 2 of the Phase 2 trial in patients with CMT, with preliminary results expected in the first quarter of 2020.
Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure.
- Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with topline results expected in the first half of 2020.
- Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.
A preclinical abstract of sotatercept in PAH has been accepted for
presentation at the
American Thoracic Society(ATS) 2019 International Conferenceon May 21, 2019.
- Cash Position – Cash, cash equivalents and investments as of
March 31, 2019were $513.1 million. As of December 31, 2018, the Company had cash, cash equivalents and investments of $291.3 million. Based on the Company's current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales.
- Revenue – Collaboration revenue for the first quarter was
$2.8 million. The revenue is all from the Company'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
first quarter were
$43.6 million. This includes R&D expenses of $32.8 millionand G&A expenses of $10.8 million.
- Net loss – The Company's net loss for the first quarter
March 31, 2019was $38.1 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first
quarter 2019 financial results and provide an update on recent corporate
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the “Acceleron First Quarter 2019 Earnings Call.”
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
ACCELERON PHARMA INC.
|March 31, 2019||December 31, 2018|
|Cash and cash equivalents||$||214,490||$||144,052|
|Short and long-term investments||298,615||147,260|
|Operating lease assets||27,817||—|
|Short-term and long-term operating lease liabilities||$||30,455||$||—|
|Warrants to purchase common stock||1,607||1,491|
|Total stockholders’ equity||510,535||292,037|
|Total liabilities and stockholders’ equity||$||560,330||$||314,821|
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands except per share data)|
|Three Months Ended March 31,|
|Costs and expenses:|
|Research and development||32,771||23,431|
|General and administrative||10,814||7,441|
|Total costs and expenses||43,585||30,872|
|Loss from operations||(40,805||)||(27,640||)|
|Total other income, net||2,772||1,431|
|Loss before income taxes||(38,033||)||(26,209||)|
|Income tax provision||(20||)||(10||)|
Net loss applicable to common stockholders - basic and diluted
Net loss per share applicable to common stockholders - basic and diluted
|Weighted-average number of common shares used in computing net loss per share applicable to common stockholders||51,126||45,516|
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 or its
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
Matt Fearer, 617-301-9557
Director, Corporate Communications