Analyst Conference Summary

Biotechnology

conference date: November 7, 2017 @ 5:00 AM Pacific Time
for quarter ending: September 30, 2017 (Q3, third 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 $3.0 million, down sequentially from $3.1 million, and flat from $3.0 million year-earlier. All revenue is from collaborations.

Net income was negative $24.5 million, up sequentially from negative $29.7 million, and down from negative $20.8 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.65, up sequentially from negative$0.77, and down from negative $0.55 year-earlier.

Guidance:

Not given.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "there were several significant corporate events in the third quarter. At our September R&D day, we outlined our core research and development focus in three disease areas of high unmet medical need: hematology, neuromuscular, and pulmonary disease. We announced that we gained rights to sotatercept, an internally discovered Phase 2 asset, for the development in pulmonary arterial hypertension. We also completed a successful equity offering that will provide sufficient funding through key inflection points in each of our clinical programs. We and our partner Celgene continue to invest heavily in our luspatercept development plan with seven clinical trials expected to be ongoing in 2018. In neuromuscular diseases, ACE-083 continues to advance in the Phase 2 trials in FSHD and CMT, and we remain on track to launch a Phase 2 trial with sotatercept in the first half of 2018 as we work to grow our pulmonary franchise, and ultimately deliver transformative treatment options to patients in need."

All 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 it 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 should enroll its first patient before the end of 2017. A Phase 2 trial in beta-thalassemia is planned to begin soon. Updated positive Phase 2 data for lower-risk MDS was presented. More data will be presented at ASH in December. Luspatercept helps treat chronic anemia associated with the indicated diseases.

ACE-083 Phase 2 study for FSHD (facioscapulohumeral muscular dystrophy) continued enrollment and should report initial data by year-end. Data from all dose cohorts should be available in 2018. Also enrolling a Phase 2 trial for Charcot-Marie-Tooth disease.

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

Sotatercept preclinical results in PAH (pulmonary arterial hypertension) will be presented on November 14, 2017. A Phase 2 trial in PAH will begin in the first half of 2018.

See also Acceleron pipeline.

Cash and equivalents ended at $367 million, up sequentially from $194 million. Raised $216 million in the quarter with a stock offering. No debt. Believes has sufficient cash to operate through 2021.

$21.1 million was spent on R&D and $7.5 million on general and administration. Loss from operations was $25.6 million. Other income $0 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:

Updated Sotatercept data to be presented at ASH? Investigator initiated trial in MF, we have no expectation that the dosing timing would be different in that population. It is early data, but robust.

Frontline COMMAND study design? Will give an update when we are closing to starting the trial.

ACE-083 data timeline? Plan is to release the data in January, there are no conferences then, so we would detail it at a muscle conference later in the year.

NTD group? Beta-thalassemia patients divide into transfusion dependent and non-transfusion (NTD), but the nons tend to be sicker and suffer more complications.

FSHD, what will be disclosed in January? We had been planning for data at the end of the year, but the cleanup of the data took us to the end of December. Everything is on track, enrollment is good. Disclosure is the right dose guided by muscle mass. We will release strength and function, but the numbers will be too small to mean much. Patient advocacy groups have helped get a brisk enrollment. It is genetic, so families have multiple members with it. We think at least a 5% increase in muscle volume is needed.

Sotatercept preclinical data color? At American Heart, preclinical data will inform moving forward with the Phase 2 trial. Endpoints are pulmonary vasculature & the like. It has the potential to be disease modifying. The approved drugs are dilators.

ACE-083, time to functional benefits? CMT and FSHD studies are in two parts. First is dose finding, mainly looking at muscle mass. Goal is to start part 2 in FDHD in the second half of 2018, placebo controlled, and with functional endpoints like strength. We could see some improvements in 6 to 8 weeks.

Powering of COMMAND trial vs. ESAs? We are not giving details on the study yet. In the frontline setting it is important to look at ESAs with a value-benefit model that will be embraced by all stakeholders, including the payers. We hope we are looking at an on-label superior result over ESAs. We will recruit both RS positive and negative patients.

Pricing, parity to ESAs? We think we can design a study to prove superiority to ESAs. $50,000 to $100,000 would be our current pricing estimate.

Yes there could be a quality of life scales in the COMMAND trial. It is pretty straightforward in anemia. We did this in our Phase 2 study.

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