Analyst Conference Summary

Biotechnology

conference date: February 27, 2018 @ 2:00 PM Pacific Time
for quarter ending: December 31, 2017 (Q4, fourth quarter)

Forward-looking statements

Overview: Acceleron remains on track to deliver Phase 3 Luspatercept results in mid-2018. Has plenty of cash to get to commercial operations.

Basic data (GAAP):

Revenue was $3.7 million, up sequentially from $3.0 million, and up from $3.4 million year-earlier. All revenue is from collaborations.

Net income was negative $27.9 million, down sequentially from negative $24.5 million, and down from negative $19.3 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.62, up sequentially from negative $0.65, and down from negative $0.51 year-earlier.

Guidance:

Cash should last into 2021.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "In hematology, we look forward to announcing top-line results from our MEDALIST and BELIEVE Phase 3 trials of luspatercept in mid-2018. We and Celgene continue to drive preparations and key activities for regulatory and commercial readiness. In neuromuscular, we are advancing ACE-083, our locally acting ‘Myostatin+’ agent, through Phase 2 trials in two distinct neuromuscular diseases. In pulmonary, we plan to initiate a Phase 2 study with sotatercept in pulmonary arterial hypertension."

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, if the programs are successful.

The Phase 2 study of ACE-083 in Charcot-Marie-Tooth disease continued.

Luspatercept Phase 3 trials completed enrollment: beta-thalassemia BELIEVE study and MDS (myelodysplastic syndromes) MEDALIST trial. Both trials should report top-line data in mid-2018. 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. More positive data was presented at ASH in December. 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 initial data from cohorts 1 and 2 was positive.. Data from all dose cohorts should be available in 2018. Also enrolling a Phase 2 trial for Charcot-Marie-Tooth disease. Preliminary data could be available in 2H 2018.

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

Sotatercept preclinical results in PAH (pulmonary arterial hypertension) was presented on November 14, 2017. Positive Phase 2 results in myelofibrosis were presented at ASH. A Phase 2 trial in PAH will begin in the first half of 2018. Has full rights, which were regained from Celgene.

See also Acceleron pipeline.

Cash and equivalents ended at $373 million, up sequentially from $367 million. No debt. Believes has sufficient cash to operate through 2021.

$25.3 million was spent on R&D and $7.0 million on general and administration. Loss from operations was $28.6 million. Other income $0.8 million.

Acceleron's goal is to have 3 programs in Phase 3 trials by 2020.

Sees Luspatercept as a multi-billion dollar opportunity.

Q&A:

Recent business development activity in MF? Myelofibrosis acquisition by Celgene shows their commitment to the space. Patients have both disease and drug induced anemia. We are not changing our MF development plan.

Luspatercept RS+ vs. RS- ? RS+ patients take longer to progress, so the incidence pool is weighted to RS-, about 2/3 of pool.

CMP trial data release timeline? In 2H we will give preliminary results for FSHD and CMP. So no change in guidance.

2494 muscle measured? Looking using MRI scans in lower extremity, particularly the thigh muscle. We expect to see substantial increases in muscle volume. 5% is what is needed to see strength and function increases.

2494 indications? Details were at R&D presentation. Have not picked a Phase 2 target, but possibilities are DMD, ALS, SMA, and FSHD.

Luspatercept Phase 2 follow ups help show duration and quality of response, which will help in competition with ESA agents.

083 competitive landscape? It is a locally acting agent, the competitors are very different, for very different diseases, with systemic approaches, including Regeneron's. We believe there is value in our having both a local and a systemic agent. We believe our agents could be combined with myostatin agents.

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