Analyst Conference Summary

Biotechnology

conference date: August 3, 2017 @ 2:00 PM Pacific Time
for quarter ending: June 30, 2017 (Q2, second quarter)

Forward-looking statements

Overview: Clinical development continues.

Basic data (GAAP):

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

Net income was negative $29.7 million, down sequentially from negative $25.4 million, and down from negative $22.0 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.77, down sequentially from negative$0.66, and down from negative $0.59 year-earlier.

Guidance:

Not given.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "Over 560 patients were enrolled ahead of estimated timing in our MEDALIST and BELIEVE Phase 3 trials combined, and our partner Celgene continues to invest in new opportunities for luspatercept. Our recent Phase 2 luspatercept results continue to support our confidence in achieving long-term, clinically meaningful outcomes for patients. In addition, our wholly-owned muscle compound ACE-083 has recently expanded its Phase 2 development program into a second neuromuscular disease."

All GAAP revenue was from collaboration partner Celgene.

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.

On August 1 Acceleron announced in began the Phase 2 study of ACE-083 in Charcot-Marie-Tooth disease.

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 early 2018. A Phase 2 trial for myelofibrosis was initiated. Updated positive Phase 2 data was presented. Luspatercept helps treat chronic anemia associated with the indicated diseases.

A phase 2 trial for first-line MDS patients also continued, with positive interim results reported at the International Symposium on MDS. The slide deck has several slides illustrating details of the data. While responses were basically positive, they were stronger in ESA-naive patients and in RS negative patients. See discussion in Q&A below.

ACE-083 Phase 2 study for facioscapulohumeral muscular dystrophy continued enrollment and should report initial data by year-end. Data from all dose cohorts should be available in 2018.

The dalantercept (+axitinib) Phase 2 study for advanced renal cell carcinoma completed enrollment, with top line results expected this quarter. We did not get the data in time for ASCO, so results will just be in a press release.

ACE-2494 will begin its first Phase 1 trail in 2017. This would be the first IntelliTrap platform agent to go to trial.

See also Acceleron pipeline.

Cash and equivalents ended at $194 million, down sequentially from $213.2 million. No debt. Believes has sufficient cash to operate through second half of 2019.

$21.6 million was spent on R&D and $11.4 million on general and administration. Loss from operations was $29.9 million. Other income $0.2 million.

Acceleron's goal is to have FDA approvals in 5 indications by 2020, and at that time to have 8 candidates in clinical trials. Hopes to be cash flow positive in 2020.

Acceleron plans to introduce a new internally discovered compound into clinical trials every 12 to 18 months. There are currently 6 preclinical programs that could enter clinical trials, all targeting diseases with high unmet medical needs. Focus is on muscle diseases and fibrosis. IntelliTrap platform is being used to generate new potential candidates.

R&D Day will be on September 19.

Q&A:

ACE-083 first cohort expectations? We have three dose escalating cohorts, looking just at muscle growth. We need to demonstrate strength and function, which we would attempt in second round, but we should have some data for the first cohort. Before the end of this year.

Luspatercept value proposition newer indications vs. competition? In the newer indications, like first line MDS competition with ESAs which have $500 to $600 million use off-label in U.S. alone. If we prove superiority the pricing risk declines. We want to differentiate from ESAs in this setting, and to be on-label.

Priority for acquiring external assets? TGF beta has always been a foundation internally. External business development could include finding partners for our internal pipeline. When we have made enough progress we could look at external asset acquisition to expand, but this is very early thinking.

Systemic muscle program, other Phase 2 indications? We are thinking about several rare diseases where we could develop in house. We will say more on R&D day. We would begin by studying healthy volunteers, and decide when we get that data.

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