Acceleron Pharma Inc (NASDAQ:XLRN) just announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation to ACE-083, the Company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease (CMT).
“We’re pleased that the FDA has granted this designation for ACE-083,” said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. “Patients with CMT currently have no approved therapies. To date, results from our Phase 2 trials have shown that patients treated with ACE-083 experience robust increases in muscle volume. If our ongoing clinical studies show that ACE-083 also improves functional outcomes and confirm the favorable safety profile observed thus far, the Fast Track process could help us work with the FDA to deliver it to patients as quickly as possible.”
Acceleron Pharma Inc (NASDAQ:XLRN) frames itself as a clinical stage biopharmaceutical company that focuses on the discovery, development, and commercialization of therapeutics to treat serious and rare diseases.
Its therapeutic candidates include luspatercept, which is in Phase III clinical trials for the treatment of myelodysplastic syndromes and beta-thalassemia; Phase II clinical trials for the treatment of patients with transfusion-dependent beta-thalassemia and myelofibrosis.
The company is also developing and sotatercept for the treatment of patients with pulmonary arterial hypertension.
In addition, the company is developing ACE-083, a neuromuscular candidate that is in Phase II clinical trials for the treatment of patients with facioscapulohumeral dystrophy and Charcot-Marie-Tooth disease; and ACE-2494, which is in Phase I clinical trials for the treatment of systemic muscle disorders. It has a collaboration, license, and option agreement with Celgene Corporation.
ACE-083, a locally-acting therapeutic candidate based on the naturally-occurring protein follistatin, utilizes the Myostatin+ approach to inhibit multiple TGF-beta superfamily ligands. It is designed to have a concentrated effect along targeted muscles to maximize growth and strength selectively in the muscles into which the drug is administered. Acceleron is developing ACE-083 for disorders such as CMT and FSHD, in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life.
The company was formerly known as Phoenix Pharma, Inc. Acceleron Pharma Inc. was founded in 2003 and is headquartered in Cambridge, Massachusetts.
As we discussed earlier, XLRN just announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation to ACE-083, the Company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease (CMT).
The stock is up on the news. Overall, shares of XLRN have rallied about 5% over the past week. That sets up an interesting context for the action tomorrow.
According to the release, ACE-083 is currently being evaluated in two Phase 2 trials: one in CMT and one in facioscapulohumeral muscular dystrophy (FSHD). Preliminary results from Part 2 of the trials are expected by year end 2019 for CMT and in the second half of 2019 for FSHD. Earlier this year, the FDA granted Fast Track and Orphan Drug designations for ACE-083 for the treatment of patients with FSHD.
Acceleron Pharma Inc (NASDAQ:XLRN) generated sales of $3.3M, according to information released in the company’s most recent quarterly financial report. That adds up to a sequential quarter-over-quarter growth rate of -11.6% on the top line. In addition, the company has a strong balance sheet, with cash levels far exceeding current liabilities ($308M against $14.7M).