The Burrill Report

Sarepta Therapeutics plummeted 64 percent after disclosing that the U.S. Food and Drug Administration said the biotech’s New Drug Application submission and confirmatory clinical study for eteplirsen as a treatment for Duchenne muscular dystrophy is premature. The FDA’s decision was due in part to the recent failed late-stage clinical study of a competitive product, drisapersen, being developed by Prosensa and GlaxoSmithKline. The FDA also voiced doubt about both the dystrophin biomarker and the use of the 6-minute walk test to support clinical efficacy in Sarepta’s mid-stage study of eteplirsen. “We are very disappointed with the FDA’s decision to reconsider their openness to a potential NDA filing based on our current data and the resultant impact this change may have on our efforts to achieve an earlier approval of eteplirsen,” said Chris Garabedian, president and CEO of Sarepta. The FDA’s decision could delay approval of the exon-skipping drug by at least two years, Garabedian said in a conference call to discuss the news. He said the company will work with the FDA on an acceptable confirmatory study design and seek to address the agency’s concerns regarding a potential NDA filing based on its current clinical endpoints. The FDA’s request to discuss different clinical endpoints, combined endpoints, and different DMD subpopulations for a confirmatory clinical study, along with its questions about dystrophin as a biomarker and the need for a placebo-controlled study, will delay initiation of the confirmatory study of eteplirsen until at least the second quarter of 2014, Sarepta says.

Aegerion Pharmaceuticals received a warning letter from the U.S. Food and Drug Administration after CEO Marc Beer suggested that the company’s Juxtapid could prevent heart attacks and strokes and extend the lives of patients suffering from the rare disease homozygous familial hypercholesterolemia (HoFH). Beer made the statements in two separate interviews on CNBC’s television news show “Fast Money.” Beer made statements such as, “this product has the potential of taking a patient that would die at 30 and allow them to meet their grandkids,” and in another interview, “these patients are going to die of a cardiac event, either a stroke or a heart attack, if we don’t have them on therapy.” The FDA approved Juxtapid only to reduce LDL cholesterol levels in patients suffering from HoFH. The agency noted in the warning letter that the drug comes with serious risks, including a boxed warning regarding the risk of liver toxicity. The FDA warned that Beer’s statements are evidence that Juxtapid is intended for new uses for which it is not approved and demanded a response by November 22 that includes a plan of action to correct any misimpressions about the drug’s approved use. Aegerion issued a statement saying that it would comply with the FDA and take quick action to respond to its letter. Juxtapid therapy costs $250,000 a year and competes with Kynamro, marketed by Isis Pharmaceuticals and Sanofi’s Genzyme.

Specialty drugmaker Transcept Pharmaceuticals is cutting its workforce by 43 percent and restructuring its Board of Directors to conserve cash and be more attractive to a potential buyer. The changes are expected to save the company about $1 million annually and are expected to take place at the end of 2013. The company will be left with just eight employees. In September Transcept rejected a $4 per share bid by Retrophin to acquire the company. Transcept has been in trouble for some time. It has had a difficult time increasing sales of its one approved product, the sleep aid Intermezzo, which competes with market leader Ambien. Partner Purdue Pharmaceuticals has stopped marketing the product, and Transcept is trying to regain commercialization and development rights from Purdue. Last year, it stopped development of an experimental therapy after it failed a mid-stage study to treat obsessive compulsive disorder. Transcept’s lead development candidate, TO-2070, is a novel, rapidly absorbed treatment for acute migraine, which was recently licensed from Japanese drugmaker Shin Nippon Biomedical Laboratories.

Dendreon reported third quarter 2013 results and announced another restructuring and cost reduction plan to remove more than $125 million in cash operating expenses from the company’s 2013 run rate, representing a reduction of approximately 20 percent. Reductions will come from all expense categories and leave the biotech with about 820 employees, down from more than 2,000 employees at its peak. The company had about 1,050 employees as of February 2013. In late October, Bloomberg reported that the company was looking for a buyer. Dendreon has been unable to boost sales of its prostate cancer vaccine Provenge, which were just $68 million in the third quarter of 2013, compared to $77.9 million in the third quarter of 2012.

During a slide presentation at the 2013 Credit Suisse Annual Health Care Conference, Theravance CEO Rick Winningham said its experimental drug TD-9855 did not meet the primary efficacy endpoint of symptom reduction in a mid-stage study. Winningham said the company will not it program in ADHD. TD-9855 is currently being evaluated in an ongoing mid-stage study in patients with fibromyalgia, results of which are expected during the first half of 2014. TD-9855 is an investigational norepinephrine and serotonin reuptake inhibitor for the treatment of central nervous system conditions such as chronic pain.

November 15, 2013
http://www.burrillreport.com/article-sarepta_plummets_after_fda_says_nda_filing_for_eteplirsen_premature.html