Analyst Conference Summary


Acceleron Pharma

conference date: May 9, 2019 @ 2:00 PM Pacific Time
for quarter ending: March 31, 2019 (Q1, first quarter)

Forward-looking statements

Overview: Luspatercept data looks good, Celgene has submitted the BLA to the FDA to grant approval.

Basic data (GAAP):

Revenue was $2.8 million, down sequentially from $3.8 million, and down from $3.2 million year-earlier. All revenue is from collaborations.

Net income was negative $38.0 million, down sequentially from negative $34.7 million, and down from negative $26.2 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.74, up sequentially from negative $0.75, and down from negative $0.58 year-earlier.


Believes cash is sufficient to support the company until significant luspatercept royalties come in.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "With the submission of marketing applications for luspatercept in the U.S. and E.U. last month, we and our global collaboration partner, Celgene, are excited about the potential to bring a new therapy to patients with myelodysplastic syndromes and beta-thalassemia within the next year. At the same time, our pulmonary program remains on track, with enrollment ongoing in two Phase 2 trials of sotatercept in PAH, and we are anticipating topline results from the placebo-controlled part of the Phase 2 trial of our locally-acting muscle agent, ACE-083, in FSHD during the second half of this year."

All revenue was from collaboration partner Celgene, mainly from cost-sharing.

Celgene funds 100% of expenses in the partnered luspatercept programs, and royalties will be in the low to mid 20% range. Believes sales in the first two indications could reach $2 billion annually, resulting in about $400 million per year in royalties.

LuspaterceptBeta-thalassemia BELIEVE study and MDS (myelodysplastic syndromes) MEDALIST trial reported positive top-line Phase 3 data, which should qualify for FDA approval for treatment of anemia, at ASH in December. BLA submission was made in April. With Celgene, a third Phase 3 trial, versus standard of care in first line, lower risk MDS patients continues. A Phase 2 trial in beta-thalassemia started in Q1 2018; it is 48-weeks and double-blind. A phase 2 trial started in myelofibrosis will be open-label and 24 weeks. Luspatercept helps treat chronic anemia associated with the indicated diseases and is partnered with Celgene.

ACE-083 Phase 2 study for FSHD (facioscapulohumeral muscular dystrophy) continued enrollment and more data was presented in October 2018. Preliminary full trial results expected in 2H 2019. Also enrolling a Phase 2 trial for Charcot-Marie-Tooth disease, with data also presented at WMS.

ACE-2494 Phase 1 systemic muscle trial has begun; preliminary results had side effects that were unacceptable, so we terminated the study.

Sotatercept in PAH (pulmonary arterial hypertension) Phase 2 trial (PULSAR) started with preliminary results expected in 2020. Positive Phase 2 results in myelofibrosis were presented at ASH. Acceleron has full rights.

See also Acceleron pipeline.

Cash and equivalents ended at $513 million, up sequentially from $291 million. No debt. Raised $248 million with a stock sale in Q1 2019. Believes has sufficient cash to operate into 2021.

$32.8 million was spent on R&D and $10.8 million on general and administration. Loss from operations was $40.8 million. Other income $2.8 million.


Support for 8 week dosing? The extension study for CMT and FSHD are designed for a number of secondary endpoints showing function. How do we optimize 083? The most important thing is to build healthy muscle. But convenience would also be optimal. We are testing 4 week and 8 week dosing.

2494? We terminated that study. We are not pursuing a backup candidate at this point. We are looking for the 083 data.

We expect no significant expense reduction from terminating 2494. Because of other trials expenses will be going up this year.

Luspatercept in PAH? The primary endpoint is pulmonary vascular resistance. Secondary is the six minute walk, looking for 30 meters.

Any interactions with BMS yet? We see from the sidelines that Bristol sees luspatercept as a very important part of their portfolio. We are very pleased with all of the efforts, but we have not had direct discussions with BMS. Under standard review we would get a result in the US in April 2020, in the EU in 2H 2020.

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Disclaimer: My analyst call summaries may include both our condensations of statements made by company representatives and my own analysis. They are not covered by any warranty. I cannot guarantee anything said by company representatives is true. I try not to make errors, but it is possible. This is investment journalism, not financial advice.

Copyright 2019 William P. Meyers