Analyst Conference Summary

Biotechnology

conference date: August 2, 2018 @ 2:00 PM Pacific Time
for quarter ending: June 30, 2018 (Q2, second quarter)

Forward-looking statements

Overview: Acceleron, with Celgene, had positive Phase 3 luspatercept results for anemia due to both myelodysplastic syndromes and beta-thalassemia.

Basic data (GAAP):

Revenue was $3.7 million, up sequentially from $3.2 million, and up from $3.1 million year-earlier. All revenue is from collaborations.

Net income was negative $28.9 million, down sequentially from negative $26.2 million, and up from negative $29.7 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.63, down sequentially from negative $0.58, and up from negative $0.77 year-earlier.

Guidance:

none

Conference Highlights:

Habib Dable, CEO of Acceleron said: "We and our collaboration partner, Celgene, look forward to presenting the Phase 3 data at an upcoming medical congress and are focused on the execution of key regulatory activities, including US and EU application submissions in the first half of 2019. We believe luspatercept is a potential platform treatment to transform the lives of patients suffering from a range of hematologic diseases associated with anemia. Our neuromuscular and pulmonary programs achieved critical milestones, putting us in a position for important Phase 2 readouts for ACE-083 and sotatercept in 2019 and 2020, respectively."

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. There was no commercial revenue in Q2.

LuspaterceptBeta-thalassemia BELIEVE study and MDS (myelodysplastic syndromes) MEDALIST trial reported positive top-line Phase 3 data in the quarter, which should qualify for FDA approval. Regulatory applications will be made in the first half of 2019. With Celgene, a third Phase 3 trial, versus standard of care in first line, lower risk MDS patients should start in first half of 2018. 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 will be presented in October 2018. Preliminary full trial results expected in 2H 2019. Received Fast Track designation. Preliminary data from cohorts were presented in April 2018, with more results expected later this year. Also enrolling a Phase 2 trial for Charcot-Marie-Tooth disease, with preliminary results presented in July.

ACE-2494 Phase 1 systemic muscle trial has begun; preliminary results possible in first half of 2019.

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 $332 million, down sequentially from $353.3 million. No debt. Believes has sufficient cash to operate into 2021.

$26 million was spent on R&D and $8 million on general and administration. Loss from operations was $30 million. Other income $1 million.

Q&A:

Strategy changes on success? We are staying with our commitments and investments. But as we gain confidence in the portfolio we are gaining insights into the diseases. So positioning well to become a partner of choice, and thinking of external opportunities.

Timeline for filings? We will request priority review.

FSHD and CMT findings on strength and function? Part 2 will be looking at strength and function.

$185 million in outstanding milestones. Next is $25 million on acceptance of the filing.

Muscle function v. myostatin targetted competition? ACE083 is the only locally directed therapy. No systemic program has ever shown anywhere near the effectiveness we have shown. Versus Regeneron, our therapy mops up additional ligands that are important in the pathway.

MDS awareness, need? Yes, with Celgene we will be working to raise awareness of MDS and treatment

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