Analyst Conference Summary

Biotechnology

conference date: May 8, 2018 @ 2:00 PM Pacific Time
for quarter ending: March 31, 2018 (Q1, first 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 $3.2 million, down sequentially from $3.7 million, and down from $3.7 million year-earlier. All revenue is from collaborations.

Net income was negative $26.2 million, up sequentially from negative $27.9 million, and down from negative $25.4 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.58, up sequentially from negative $0.62, and up from negative $0.66 year-earlier.

Guidance:

Cash should last into 2021.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "We are currently targeting five indications across multiple clinical trials and are evaluating additional lifecycle management prospects. Our wholly-owned neuromuscular franchise is uniquely positioned with both a local and a systemic muscle agent, that utilize our Myostatin+ approach to inhibit multiple TGF-beta proteins. In pulmonary disease, we are very close to initiating our first Phase 2 trial with sotatercept in pulmonary arterial hypertension or PAH. We continue to prepare for key upcoming luspatercept activities and remain fully committed to moving our clinical programs in to late stage development."

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. 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. 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. 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 Q2 of 2018, with preliminary results in 2020. Has full rights, which were regained from Celgene.

See also Acceleron pipeline.

Cash and equivalents ended at $353.3 million, down sequentially from $373 million. No debt. Believes has sufficient cash to operate through 2021.

$23.4 million was spent on R&D and $7.4 million on general and administration. Loss from operations was $27.6 million. Other income $1.4 million.

Sees Luspatercept as a multi-billion dollar opportunity.

Q&A:

Rational for change to six month treatment period for FSHD? We completed animal studies that extended our safety bracket. So we can prove safety and effectiveness over a longer time.

AAN safety data, could population be changed for part 2? We don't think the population was heterogeneous. We see it as homogeneous.

Medalist trial changes from Phase 2, could they increase or decrease response rates? We have tried to minimize differences between the phases. Revlimid study looked at a similar population, design similar to Medalist, but we are looking to 2 transfusions over previous 8 weeks. Our Phase 2 had a 39% efficacy rate, in 8 weeks. In Medalist we are looking at a longer number of weeks. "Things tend to drift downward from Phase 2 to Phase 3," so we would see 26.9% to 39% as a win.

Any safety concerns in Phase 2 that could be a problem in Phase 3? We feel good about the safety profile, which has been stable. We have over 1000 patient-months of data.

FSHD regulatory process? Plan is to talk to FDA after Phase 2, then go ahead with the Phase 3 trial. If the Phase 2 trial is incredibly positive, that could change.

Commercial preparation for luspatercept? We work closely with Celgene, patient groups, doctors and payers. We intend to co-promote in North America.

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