Analyst Conference Summary

Biotechnology

conference date: November 6, 2019 @ 7:00 AM Pacific Time
for quarter ending: September 30, 2019 (Q3, third quarter)

Forward-looking statements

Overview: The FDA target action date for the beta-thalassemia indication is December 4, 2019, and for the MDS is April 4, 2020.

Basic data (GAAP):

Revenue was $4.2 million, down sequentially from $27.7 million, and up from $3.3 million year-earlier. All revenue is from collaborations.

Net income was negative $45.3 million, down sequentially from negative $17.9 million, and down from negative $29.0 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.86, down sequentially from negative $0.34, and down from negative $0.63 year-earlier.

Guidance:

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

Conference Highlights:

Habib Dable, CEO of Acceleron said: "We are close to achieving a major company milestone-the potential approval for the first Acceleron-discovered medicine. Alongside our global collaboration partner, Celgene, we are preparing for luspatercept's potential commercial launch in the U.S. We also await the FDA decision on our BLA for the MDS indication in April 2020 and the European Medicines Agency decision on the MAA for both indications, which is expected in the second half of 2020. We continue to advance our ongoing clinical trials in first-line lower-risk MDS-, non-transfusion-dependent beta-thalassemia- and myelofibrosis-associated anemia. In addition, we are looking forward to presenting luspatercept updates on our Phase 3 MEDALIST and BELIEVE trials, as well as interim results from the ongoing Phase 2 myelofibrosis trial at the upcoming ASH meeting in December. We anticipate reporting results in the first quarter of 2020 for both our PULSAR Phase 2 sotatercept trial in patients with PAH, and our ACE-083 trial in patients with CMT."

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

Luspatercept programs, and royalties from Celgene 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.

Luspatercept Beta-thalassemia and MDS (myelodysplastic syndromes) BLA submissions to FDA were made in April. Beta-thalassemia has priority review with a PDUFA date of December 4, 2019, while MDS has a April 4, 2020 PDUFA date. The EU decision is expected in the second half of 2020 for beta-thalassemia associated anemias. A third Phase 3 trial, versus standard of care in first line, lower risk MDS patients continues. A phase 2 trial for myelofibrosis will be open-label and 24 weeks, with results in 2019. Luspatercept helps treat chronic anemia associated with the indicated diseases. New data presentations will be made at ASH on December 9, 2019.

ACE-083 Phase 2 study for FSHD (facioscapulohumeral muscular dystrophy) discontinued enrollment following negative results in Q3 2019. But still enrolling a Phase 2 trial for Charcot-Marie-Tooth disease, with topline data expected in 1H 2020.

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) completed enrollment with preliminary results expected in Q1 2020. Acceleron has full rights.

See also Acceleron pipeline.

Cash and equivalents ended at $468 million, down sequentially from $501 million. No debt. Believes has sufficient cash to operate into 2021.

$37.6 million was spent on R&D and $15.5 million on general and administration. Total op ex was $53.1 million. Loss from operations was $48.9 million. Other income $3.5 million.

Q&A Summary:

Phase 2 myelofibrosis data difference in cohort durability? We did see 32% and 53% efficacy rates, but cannot give details until ASH presentation. Based on hurdle rates, we are excited about this indication.

Advisory committee? There is always potential for that.

The Rux (Jakafi) combination myelofibrosis cohort, the most important one, we are pleased with the results, more at ASH. There are two potential monotherapy types, newly diagnosed who have not had rux, and those who have failed rux. The rux population is the larger addressable population in this space.

More questions that won't be answered until the ASH presentations.

Potential sotatercept combinations? We have presented preclinical data. In the trial we are admitting patients who are on other medications. We think it will be additive to vaso-dialators, but have not presented any clinical data on that yet.

Pediatric studies? Beta-thalassemia is hereditary, so it shows up as early as 2 years of age. So we are initiating a pediatric study across age groups with 46 subjects.

FSHD vs. CMT? One was a myopathy, one was a neuropathy. Even in the discontinued FSHD group we saw increases in muscle mass. They are very different diseases. We are cautiously optimistic. We do not have any other neuromuscular products planned for the pipeline.

Sales plans with Celgene? Celgene has leadership in haematology. We have a team of about 20 to optimize the high target accounts.

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