Sarepta Therapeutics (SRPT) Posts Wider Q4 Loss; NDA Submission for Eteplirsen Planned for Mid-Year 2015

Sarepta Therapeutics (NASDAQ: SRPT) reported Q4 EPS of ($0.94), $0.12 worse than the analyst estimate of ($0.82). Revenue for the quarter came in at $27 thousand versus the consensus estimate of $1.56 million.

• Cash and Other Investments of $211 Million at Year End 2014.
• NDA submission for eteplirsen planned for mid-year 2015 -
• Plan to discuss new data and NDA submission with FDA in 2nd Quarter

“Last month we released 168 week data from our Phase IIb study of eteplirsen. We believe the long-term safety of our drug with the continued ambulation and stability of pulmonary function in these boys are encouraging considering the length of follow up and the advancing age of the boys,” said Chris Garabedian, president and chief executive officer. “We are actively collecting and analyzing the additional datasets requested by the FDA and plan to meet with the FDA in the second quarter to ensure that we are aligned with their expectations for an acceptable NDA filing. We continue to work towards a complete NDA submission by mid-year 2015.”

Recent Corporate Developments

Duchenne Muscular Dystrophy Program

--Presented 168 week data from Study 202, a long-term Phase IIb extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through more than three years of treatment showed continued stability of respiratory muscle function, as assessed by pulmonary function tests. Results of the 6 minute walk test showed continued ambulation across all patients evaluable on the test, however all patients showed a decline in distance walked on this measure since the week 144 timepoint.

--Announced dosing in an open-label study, 4658-204 (Study 204), of eteplirsen in non-ambulant patients with DMD. The study, which is being conducted at several sites across the United States and is designed to evaluate the safety of eteplirsen over 96 weeks of dosing, will include approximately 20 patients who have genotypes amenable to exon 51 skipping.

--Announced dosing in an open-label confirmatory study, 4658-301 (PROMOVI), of eteplirsen in ambulant patients with DMD. The study, which is being conducted at 39 sites across the United States and is designed to evaluate the efficacy and safety of eteplirsen in patients over 48 weeks of dosing, will include up to 80 boys, aged 7-16 years, with genotypes amenable to exon 51 skipping.

--Announced dosing in first European Phase I/II Study of SRP-4053 to assess the safety, tolerability, efficacy, and pharmacokinetics in DMD patients with genotypes amenable to exon 53 skipping. The study is being conducted at four sites in Europe under a consortium agreement between Sarepta and various European hospitals, institutions, and scientists established to conduct the study and which is funded in part by the European Union’s Seventh Programme for research, technological development, and demonstration under grant agreement No. 305370.

Infectious Diseases Program-

--Published results from a study done in collaboration with the U.S. Army Medical Research Institute of Infectious Diseases (USAMRIID) on the efficacy of Sarepta’s PMO, AVI-7537, against the Ebola virus in Rhesus monkeys. The study demonstrated 75% effectiveness across the infected population treated with AVI-7537 compared to 0% survival in the control group.

For earnings history and earnings-related data on Sarepta Therapeutics (SRPT) click here.

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