Analyst Conference Summary

Biotechnology

conference date: October 30, 2018 @ 1:30 PM Pacific Time
for quarter ending: September 30, 2018 (Q3, third quarter)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $3.3 million, down sequentially from $3.7 million, but up from $3.0 million year-earlier. All revenue is from collaborations.

Net income was negative $29.0 million, down sequentially from negative $28.9 million, and up from negative $25,5 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.63, flat sequentially from negative $0.63, and up from negative $0.65 year-earlier.

Guidance:

none

Conference Highlights:

Habib Dable, CEO of Acceleron said: "Along with our global collaboration partner, Celgene, we expect to share results from the MEDALIST and BELIEVE Phase 3 trials of luspatercept with the global hematology community at the upcoming ASH meeting in December. With the recent initiation of the COMMANDS Phase 3 trial, we now have ongoing clinical trials with luspatercept in three additional patient populations. We remain committed to further exploring luspatercept’s potential to be a platform treatment for a range of anemias. Our neuromuscular and pulmonary teams have done a tremendous job in getting multiple Phase 2 trials underway for ACE-083 in patients with FSHD and CMT, and sotatercept in patients with PAH."

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 which should qualify for FDA approval for treatment of anemia. Full data should be released at ASH in December. 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 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 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. On November 16 Acceleron will host a PAH deep dive event.

See also Acceleron pipeline.

Cash and equivalents ended at $320 million, down sequentially from $332 million. No debt. Believes has sufficient cash to operate into 2021.

$24.7 million was spent on R&D and $8.7 million on general and administration. Loss from operations was $30.1 million. Other income $1.1 million.

Q&A:

Duration data at ASH? Data at ASH 2017, duration was 19 months. Medalist data duration won't be that long.

ACE-2494 enrollment? Original design had single ascending, then multiple. Redesigned to a higher single ascending dose, 3 mg/kg, with multiple dose study in the future.

ACE-2494 data to be released? Primary focus is on safety, but doing assessments by MRI in lower extremity. We have not made a choice yet of which diseases to treat in Phase 2.

Luspatercept potential beyond currently tested indications? The Phase 3 anemia results were very positive in distinct diseases, which gives us some confidence for other anemia indications.

Acceleron's strategy is to invest in the Phase 2 trials so that they are meaningful and the Phase 3 trials are confirmatory.

Assuming a standard review process, if we have our planned submissions to the FDA and EM in first half of 2019, we could have a commercial launch in the first half of 2020.

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