Acceleron Reports First Quarter 2018 Operating and Financial Results
– Top-line results from the MEDALIST and BELIEVE Phase 3 trials of luspatercept expected in mid-2018 –
– ACE-083 advances into Part 2 of the ongoing Phase 2 trial in facioscapulohumeral muscular dystrophy –
– PULSAR Phase 2 trial with sotatercept in pulmonary arterial hypertension on track to initiate in Q2 2018 –
“2018 is an important year for our Company, as we prepare for the
upcoming top-line results from our MEDALIST and BELIEVE Phase 3 trials
with luspatercept expected mid-year. We and
Added Mr. Dable: “Our team continues to make meaningful progress in the advancement of our wholly-owned clinical programs. In neuromuscular disease, we presented preliminary results from our Phase 2 trial of ACE-083 in FSHD for the first time and recently initiated Part 2 of the trial. In pulmonary, we expect to initiate our first Phase 2 trial with sotatercept in pulmonary arterial hypertension in the second quarter.”
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 red blood cell (
- Top-line results from the MEDALIST and BELIEVE Phase 3 trials in MDS and beta-thalassemia, respectively, are expected in mid-2018.
- The initiation of the COMMANDS Phase 3 trial in first-line, lower-risk MDS patients is planned for the first half 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.
The Company expects to provide updates from the ongoing long-term
Phase 2 extension trials in MDS and beta-thalassemia at the 2018
American Society of Clinical Oncology( ASCO) Annual Meeting and 23rd Congressof the European Hematology Association(EHA) in June.
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 dose cohorts 1 and 2 in Part 1 of the ACE-083
Phase 2 trial in patients with FSHD were highlighted in a “Best of
Neuromuscular Disease” clinical session at the
American Academy of Neurology(AAN) 70th Annual Meeting on April 26, 2018.
- The Company plans to present additional preliminary results from all Part 1 dose cohorts during the second half of 2018.
- Part 2 of the Phase 2 trial was recently initiated and preliminary results are expected in the second half of 2019.
- ACE-083 received FDA Fast Track designation in FSHD.
Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in
patients with CMT disease.
- The Company plans to present preliminary Part 1 results in the second half of 2018 and to initiate Part 2 of the Phase 2 trial by the end of 2018.
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.
- Preliminary results from the Phase 1 trial are expected in the first half of 2019.
Pulmonary Arterial Hypertension (PAH)
Sotatercept is a ligand trap for members in the TGF-beta superfamily involved in remodeling a variety of different tissues, including the vasculature and fibrosis. In multiple preclinical studies in PAH, sotatercept decreased vessel muscularization, improved pulmonary arterial pressures, and decreased indicators of right heart failure.
- The Company expects to initiate the PULSAR Phase 2 trial during the second quarter of 2018.
- Preliminary results from the PULSAR Phase 2 trial are expected in the first half of 2020.
- Cash position – Cash, cash equivalents and investments as of
March 31, 2018 were
$353.3 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 first quarter was $3.2
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
first quarter were
$30.8 million. This includes R&D expenses of $23.4 million and G&A expenses of $7.4 million.
- Net loss – The Company's net loss for the first quarter ended March 31, 2018 was $26.2 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first
quarter financial results for 2018 and provide an update on recent
corporate activities on
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the “Acceleron First Quarter 2018 Earnings Call.”
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED BALANCE SHEET|
|(Amounts in thousands)|
|March 31, 2018||December 31, 2017|
|Cash and cash equivalents||$||69,736||$||100,150|
|Short and long-term investments||283,607||272,800|
|Warrants to purchase common stock||1,320||2,236|
|Total stockholders’ equity||353,780||365,217|
|Total liabilities and stockholders’ equity||$||370,034||$||389,177|
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands except per share data)|
|Three Months Ended March 31,|
|Costs and expenses:|
|Research and development||23,431||21,727|
|General and administrative||7,441||7,836|
|Total costs and expenses||30,872||29,563|
|Loss from operations||(27,640||)||(25,858||)|
|Total other income, net||1,431||457|
|Loss before income taxes||(26,209||)||(25,401||)|
|Income tax provision||(10||)||(6||)|
|Net loss applicable to common stockholders- basic and diluted||$||(26,219||)||$||(25,407||)|
|Net loss per share applicable to common stockholders- basic and diluted||$||(0.58||)||$||(0.66||)|
|Weighted-average number of common shares used in computing net loss per share applicable to common stockholders||45,516||38,404|
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 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
development of the Company's compounds will take longer and/or cost more
than planned, that the Company or its collaboration partner,
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
Brad Miles, 646-513-3125