Acceleron Reports Fourth Quarter and Full Year 2017 Operating and Financial Results
– Top-line results from the MEDALIST and BELIEVE Phase 3 trials of luspatercept are expected in mid-2018 –
– Plans to advance neuromuscular agent ACE-083 into Part 2 of the ongoing Phase 2 trials in facioscapulohumeral dystrophy and Charcot-Marie-Tooth disease –
– Expanded pipeline with sotatercept in pulmonary disease; on track to initiate a Phase 2 trial in pulmonary arterial hypertension in 1H 2018 –
“2017 was an important year for Acceleron as we laid out our corporate
strategy and vision for future growth. We are committed to building
therapeutic area leadership and advancing our clinical programs in three
areas of high unmet medical need in hematologic, neuromuscular, and
pulmonary disease,” said
Development Program Highlights
Myelodysplastic Syndromes (MDS),
Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is designed to treat chronic anemia and reduce 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.
- The BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia was recently initiated and enrollment is ongoing in the Phase 2 trial in MF.
Updated results from the ongoing Phase 2 trial in lower-risk MDS
patients were presented at the
American Society of Hematology(ASH) 2017 annual meeting. Multiple patients are now nearing three years on treatment.
Facioscapulohumeral Muscular Dystrophy (FSHD)
and Charcot-Marie-Tooth (CMT) disease
ACE-083 is a locally-acting therapeutic, based on the naturally-occurring protein follistatin, designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause debilitating 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 demonstrated mean total muscle
volume increases of over 12% in the tibialis anterior and biceps
brachii muscle cohorts. In addition, both of these cohorts
demonstrated a reduction in fat fraction.
- Part 1 of the trial is fully enrolled with treatment ongoing in dose cohort 3; the Company plans to begin Part 2 of the Phase 2 trial in the second quarter of this year.
- 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 throughout the body by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.
- The Phase 1 clinical trial has been initiated and preliminary results from this healthy volunteer study are expected in the first half of 2019.
Pulmonary Arterial Hypertension (PAH)
Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the TGF-beta protein superfamily involved in the remodeling of a variety of different tissues, including the vasculature and fibrotic tissue.
Preclinical results were presented at the
American Heart Association2017 Scientific Sessions demonstrating that sotatercept decreased vessel muscularization, improved pulmonary arterial pressures, and decreased indicators of right heart failure.
- Clinical activities are underway to initiate the planned Phase 2 trial in PAH in the first half of 2018.
- The Company plans to host an educational webinar to discuss the Phase 2 trial design in the first half of 2018.
- Cash Position – Cash, cash equivalents and investments as of
December 31, 2017 were
$372.9 million. As of December 31, 2016the Company had cash, cash equivalents and investments of $234.4 million. Cash, cash equivalents and investments include $215.8 millionof net proceeds from a follow-on public offering of common stock in 2017. We believe that our existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into 2021.
- Revenue – Collaboration revenue for the year was
$13.5 million. The revenue is all from our Celgenepartnership and is primarily due to cost sharing revenue of $12.9 millionrelated to expenses incurred by the Company in support of our partnered programs.
- Costs and expenses – Total costs and expenses for the year were
$123.5 million. This includes R&D expenses of $89.7 millionand G&A expenses of $33.7 million.
- Net Loss – The Company’s net loss for the year ended
December 31, 2017 was
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its
fourth quarter and full year financial results for 2017 and provide an
update on recent corporate activities on
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the “Acceleron Fourth Quarter and Full Year 2017 Earnings Call”.
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED BALANCE SHEET
(Amounts in thousands)
|December 31, 2017||December 31, 2016|
|Cash and cash equivalents||$||100,150||$||20,950|
|Short and long-term investments||272,800||213,432|
|Warrants to purchase common stock||2,236||1,244|
|Total stockholders’ equity||365,217||225,597|
|Total liabilities and stockholders’ equity||$||389,177||$||247,647|
ACCELERON PHARMA INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(Amounts in thousands except per share data)
|Three Months Ended December 31,||Year Ended December 31,|
|Costs and expenses:|
|Research and development||25,339||19,088||89,726||68,580|
|General and administrative||7,002||6,267||33,738||25,297|
|Total costs and expenses||32,341||25,355||123,464||93,877|
|Loss from operations||(28,636||)||(21,986||)||(109,983||)||(66,106||)|
|Total other income, net||770||2,742||1,561||9,116|
|Loss before income taxes||(27,866||)||(19,244||)||(108,422||)||(56,990||)|
|Income tax provision||(60||)||(44||)||(32||)||(24||)|
|Net loss per share - basic and diluted||$||(0.62||)||$||(0.51||)||$||(2.68||)||$||(1.52||)|
|Weighted-average number of common shares used in computing net loss per share||45,217||37,914||40,420||37,430|
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.
For Acceleron Pharma Inc.
Todd James, 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