Analyst Conference Summary

Biotechnology

conference date: May 8, 2017 @ 5:00 AM Pacific Time
for quarter ending: March 31, 2017 (Q1, first quarter)

Forward-looking statements

Overview: Most advanced product is now in Phase 3 trials. New positive Phase 2 results for Luspatercept for improving red blood cell response for first-line, lower risk MDS (myelodyspatic syndromes) provide further encouragement.

Basic data (GAAP):

Revenue was $3.7 million, up sequentially from $3.4 million, and down from $18.2 million year-earlier. All revenue is from collaborations.

Net income was negative $25.4 million, down sequentially from negative $19.3 million, and down from $5.3 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.66, down sequentially from negative$0.51, and up from $0.13 year-earlier.

Guidance:

Not given.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "Our luspatercept program in MDS and beta-thalassemia continues to build momentum, as we look to complete enrollment of the MEDALIST and BELIEVE Phase 3 studies ahead of schedule, and expand into additional patient populations where there are limited treatment options, including first-line, lower-risk MDS and myelofibrosis. As we continue to explore further expansion opportunities for luspatercept, we are also preparing to expand our wholly-owned pipeline into a second neuromuscular disease, with the expected initiation by mid-year of a Phase 2 study with ACE-083 in patients with Charcot-Marie-Tooth disease."

All GAAP revenue was from collaboration partners.

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.

Luspatercept Phase 3 trials continued enrollment: beta-thalassemia study and MDS (myelodysplastic syndromes). Both trials are expected to complete enrollment in Q2 2017 and should report top-line dta 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 is also expected to start by year-end.

A phase 2 trial for first-line MDS patients also continued, with positive interim results reported at the Internation 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. A separate Phase 2 trial for Charcot-Marie-Tooth disease is planned for mid-2017.

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 $213.2 million, down sequentially from $234.4 million. Liabilities included $1.3 million in warrants. Believes has sufficient cash to operate through 2019.

$21.7 million was spent on R&D and $7.8 million on general and administration. Loss from operations was $25.9 million. Other income $0.5 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.

Q&A:

Proportion of lower-risk MDS patients with baseline EPO less than 500? 70% to 80%.

Repeated transfusions on those who fail? Average age of onset in the study was 72, many patients have comorbidities. The extra transfusions could be related to that. One had GI bleeding unrelated to our drug. We have not seen any dropoff in the efficacy of the drug over time.

Weight-based vs. standard dosing? We have evidence supporting weight-based dosing.

Frontline MDS patients, will there be RS- patients enrolled? Cutoff based on EPO levels? It sets us up for going head-to-head with ESA therapies. Data shows robust response rate for majority of patients. Specifics of study design will be released later this year.

Safety in the Phase 2 trial reported? Very well tolerated drug. Small number of level 3 adverse events did not reach serious level. Given the age and comorbidities, these events could occur in the population even without the drug.

RS- and high EPO baseline lack of response, hypothesis about? It reflects the stage of their design, where their marrow is exhausted. It is not surprising.

RS- with high EPO, could this issue read through to myelofibrosis? Their marrows are highly reactive, that is the disease, so we got a high degree of response even in advanced MF.

AS-083 for Charcot-Marie-Tooth disease? AS-083 is a broader acting agent than myostatin inhibitors. In this disease the muscle remains healthy until there is a prolonged period of nerve impairment. We could see an increase in strength as well as muscle volume. There will be many tests of muscle function, including walking and balance tests.

EPO effect, is it stepwise or gradual? It tends to be more of a dropoff, at about 500 level.

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