Acceleron Reports Third Quarter 2018 Operating and Financial Results
– MEDALIST and BELIEVE Phase 3 trial results in myelodysplastic
syndromes (MDS) and beta-thalassemia, respectively, are expected to be
presented at the 60th
– COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve has been initiated –
– Part 1 results from the ACE-083 Phase 2 trial in
facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth
disease (CMT) were presented at the
– Pulmonary Arterial Hypertension (PAH) R&D Deep Dive Event scheduled
“2018 is proving to be a pivotal year for Acceleron, and with several
key milestones approaching, we are well-positioned for future growth.
Along with our global collaboration partner,
Development Program Highlights
Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is a 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 Acceleron and
The MEDALIST and BELIEVE Phase 3 trial results in patients with
lower-risk MDS and transfusion-dependent beta-thalassemia,
respectively, are expected to be presented at the 60th ASH
Annual Meeting and Exposition in
Celgeneplan to submit regulatory applications for both MDS and beta-thalassemia in the United Statesand Europein the first half of 2019.
- Acceleron and
- The COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve has been initiated.
- Enrollment is ongoing in the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia as well as the Phase 2 trial in patients with MF.
Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)
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 involved in muscle formation.
Final results from Part 1 of each Phase 2 trial with ACE-083 in
patients with FSHD and CMT, respectively, were presented at the 2018
World Muscle Society(WMS) Annual Meeting.
- Enrollment is ongoing in Part 2 of the Phase 2 FSHD trial, with preliminary results expected in the second half of 2019.
- Enrollment is ongoing in Part 2 of the Phase 2 CMT trial, with preliminary results expected by the end of 2019.
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 involved in muscle formation.
- Enrollment is 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 that control the BMPRII signaling pathway, which is 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.
Multiple preclinical abstracts for sotatercept in PAH have been
accepted for presentation at the American Heart Association Scientific
November 10-12, 2018.
November 16, 2018, the Company will host a PAH Researchand Development Deep Dive event in New York City.
- Enrollment is ongoing in 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 study, called SPECTRA, in Q1 2019 to provide further understanding of sotatercept’s impact on the disease.
- Cash position – Cash, cash equivalents and investments as of
September 30, 2018were $319.8 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 third quarter was
$3.3 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
third quarter were
$33.4 million. This includes R&D expenses of $24.7 millionand G&A expenses of $8.7 million.
- Net loss – The Company's net loss for the third quarter
September 30, 2018was $29.0 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its third
quarter financial results for 2018 and provide an update on recent
corporate activities on
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the “Acceleron Third Quarter 2018 Earnings Call.”
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED BALANCE SHEET|
|(Amounts in thousands)|
|September 30, 2018||December 31, 2017|
|Cash and cash equivalents||$||112,427||$||100,150|
|Short and long-term investments||207,380||272,800|
|Warrants to purchase common stock||2,031||2,236|
|Total stockholders’ equity||319,112||365,217|
|Total liabilities and stockholders’ equity||$||338,117||$||389,177|
|ACCELERON PHARMA INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands except per share data)|
Three Months Ended
Nine Months Ended
|Costs and expenses:|
|Research and development||24,667||21,059||74,027||64,387|
|General and administrative||8,653||7,533||23,756||26,735|
|Total costs and expenses||33,320||28,592||97,783||91,122|
|Loss from operations||(30,062||)||(25,578||)||(87,608||)||(81,346||)|
|Total other income, net||1,071||86||3,481||791|
|Loss before income taxes||(28,991||)||(25,492||)||(84,127||)||(80,555||)|
|Income tax benefit (provision)||12||41||(9||)||29|
|Net loss applicable to common stockholders- basic and diluted||$||(28,979||)||$||(25,451||)||$||(84,136||)||$||(80,526||)|
|Net loss per share applicable to common stockholders- basic and diluted||$||(0.63||)||$||(0.65||)||$||(1.84||)||$||(2.08||)|
Weighted-average number of common shares used in computing
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
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
Matt Fearer, 617-301-9557
Director, Corporate Communications