Sarepta Therapeutics (NASDAQ: SRPT), an RNA-based therapeutics company that develops drugs to treat serious, life-threatening, rare and infectious diseases, announced at the Action Duchenne meeting in London that patients treated with eteplirsen continue to do well.
Benefits to Patients
Sarepta’s Chief Medical Officer, Dr. Ed Kaye, was speaking about the results from the phase II study at the meeting. Dr Kaye said that patients are doing well on the therapy with no reductions in function, no missed infusions and no discontinuations. Additionally, the studies have revealed that nNOS, an important muscle cell marker, was reconstructed, providing further validation of eteplirsen. Moreover, eteplirsen is cleared via the kidneys without being metabolized and does not show any signs of toxicity. Another advantage of eteplirsen is the intravenous mode of delivery that reduces the severity of injection site reactions when compared with drisapersen.
The main competition for Sarepta’s eteplirsen is GlaxoSmithKline’s (NYSE: GSK) drisapersen. However, Glaxo said at the meeting that the results of the phase 2 study for drisapersen will not be released until the end of 2013. Moreover, Glaxo is targeting approval of drisapersen only in 2014-2015. Glaxo’s study had to place patients on an eight-weeks on, four-weeks off regimen to reduce side effects such as proteinura, thrombocytopenia and injection site reactions.
Sarepta is facing legal hurdles as Glaxo has blocked Sarepta from studying exon 51 drugs in the UK. However, other EU nations are adopting a more flexible approach. Sarepta is ready to compensate Glaxo with royalties, but Glaxo is unwilling to take up the offer. Additionally, reports indicate that Prosensa might try to block Sarepta’s exon 51 studies in the U.S. However, exons beyond 51 to 46 are available to Sarepta globally. Another positive of Sarepta’s side is that if patients benefit from eteplirsen, the law is more likely to favor Sarepta, especially if Sarepta is willing to compensate Glaxo financially.
Sarepta uses RNA-based therapeutics to help develop drugs that treat life threatening and infectious diseases. Sarepta is currently working on eteplirsen, a clinical candidate to treat Duchenne muscular dystrophy. Sarepta has a team of scientists and commercialization specialists to establish the company as a biotechnology leader. Sarepta’s technology has the ability to target both messenger RNA and precursor messenger RNA. Sarepta uses antisense technology to develop drugs for various infectious and genetic diseases.
Shares of Sarepta rose by 19.2% to $27.12 with nearly 4 million shares changing hands. Sarepta shares were the second most advanced shares on the NASDAQ on Monday.
While Sarepta shares went up substantially on Monday, other companies saw a decline in their share prices. For instance, Clovis Oncology (NASDAQ: CLVS) saw a 41.8% fall to $12.50 with 2.7 million shares traded. Nektar Therapeutics (NASDAQ: NKTR) declined to $7.04 after losing 13.6% of its value. Nearly 8 million Nektar shares were traded on Monday. Another pharmaceuticals company that declined was Achillion Pharmaceuticals (NASDAQ: ACHN) that lost 12.3% to end Monday’s trading session at $7.90 with 5.2 million shares traded.