Analyst Conference Summary

Biotechnology

conference date: February 25, 2016 @ 5:00 AM Pacific Time
for quarter ending: December 31, 2015 (Q4, fourth quarter 2015)

Forward-looking statements

Overview: Most advanced product is now in Phase 3 trials.

Basic data (GAAP):

Revenue was $3.8 million, sequentially from $4.2 million, and up from $3.7 million year-earlier.

Net Income was negative $27.3 million, down sequentially from negative $11.9 million, down from negative $17.6 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.81, down sequentially from negative $0.36, down from negative $0.55 year-earlier.

Guidance:

Not given.

Conference Highlights:

Hopes to launch two pivotal Phase 3 programs with Celgene before the end of the year. Reported significant clinical data. Introduced the IntelliTrap™ platform to discover selective and novel compounds targeting the transforming growth factor-beta superfamily of proteins.

All revenue was from collaboration partners.

Given the good data from the luspatercept Phase 2 trials for MDS (myelodysplastic syndromes) and beta-thalassemia, with partners Celgene, the Phase 2 trials have long-term extensions and cohort additions. Phase 3 trials, BELIEVE for beta-thalassemia (300 patients) and MEDALIST for MDS (210 patients) have begun. Both indications received fast track designation from the FDA.

Phase 1 data of ACE-083 showed it increased muscle volume, force, and power in healthy subjects. Data was "unprecedented" and will be released later this year. A Phase 2 trial is scheduled for mid-2016 for FSHD muscular dystrophy.

Preclinical mouse studies of ACE-2494 for muscle mass and strength were positive. Clinical studies could begin before the end of 2016. This is the first molecule from the IntelliTrap platform.

Phase 2, part 1 data of dalantercept with axitinib for renal cell carcinoma showed a 39% progression free survival rate vs. 25% for axitinib alone. Part 2 is enrolling with increased investigator interest due to the part 1 results. Data could be reported in 2016. Granted Fast Track Designation.

Celgene and Acceleron will shift sotatercept from dialysis setting to the pre-dialysis kidney disease setting. Design changes will be discussed with regulators in 2016. The number of pre-dialysis patients is much larger and the reimbursement environment is "preferred."

Sotatercept preliminary Phase 2a data for end-stage kidney disease patients was released in October. Celgene is assessing the therapy for pre-dialysis chronic kidney disease.

In December luspatercept Phase 2 data and

See also Acceleron pipeline.

Cash and equivalents ended at $136 million. Liabilities included $17.2 million in warrants to purchase common stock.

After the quarter ended Acceleron raised $140 million net through a stock sale.

Acceleron believes its cash should be sufficient into the second half of 2017.

Acceleron plans to collaborate on dalantercept to fund the Phase 3 program.

$16.1 million was spent on R&D and $5.8 million on general and administration. Loss from operations was $18.1 million. Other expense $9.0 million. Unrealized losses on investments were $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.

Q&A:

How many unnamed compounds are included in your positive cash flow statement? It assumes we fully fund all of the new products that enter the clinic.

Sotatercept shift? Not really any shift. We are moving back our talks with regulators to later this year.

Full enrollment for Phase 3 trials? We run the luspatercept Phase 2 trials, and aim for transparency on their progress. For Phase 3, Celgene is running both of them, and generally does not give updates on the enrollment status of trials. So we can't say more than what we've already said.

Dalantercept data timeline? For HCC we concluded the trial and decided it did not warrant further development. But in RCC we saw very robust activity. We should have an RCC update by the end of this year.

No Celgene milestone payment in Q4? We don't give quarterly guidance. We have the milestone payment in our long term cash guidance.

ACE-083 just muscle size, or other data? We expect to have data this year of changes in muscle volume.

Dalantercept talks with FDA about RCC? Once we have the Phase 2 data later this year we will use it to plan a Phase 3 study.

Expenses going forward? We continue to run extension studies with Celgene that are reimbursed at 100%. Our burn rate will grow a bit as we move more therapies into the clinic. We will use cash efficiently.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: