Sarepta Therapeutics (SRPT) establishes a multi-year multi-product partnership with Colorado-based Flagship Biosciences LLC to develop automated tissue-based quantitative tests related to muscular dystrophy that will support the advancement of Sarepta's product pipeline.
There is a significant need for the ability to quantify dystrophin levels, for example, since the absence of the protein causes Duchenne muscular dystrophy. Measuring the specific levels of the biomarker in biopsy samples would be the most effective way to assess a therapy's (eteplirsen) efficacy.
Financial details of the partnership are not disclosed.
Shares of Sarepta Therapeutics (SRPT +13.3%) jump on increased volume after FDA Center for Drug Evaluation and Research chief Janet Woodcock stated that the agency is exploring all "potential pathways" to approve new drugs to treat Duchenne muscular dystrophy. She also specifically mentioned the possibility that agency would place Sarepta's eteplirsen in its accelerated approval program.
SRPT longs have had a rough ride since the October 7, 2013 peak of $55.61. Shares cratered to a low of $12.12 on November 11 after the FDA said that the company's NDA was premature due to a perceived lack of efficacy of the dystrophin biomarker. Shares climbed back to $40.00 on April 21, 2014 before dropping again.
Shares of Sarepta Therapeutics (SRPT) are down 14% premarket on robust volume in response to the company's clinical update on eteplirsen in patients with Duchenne muscular dystrophy. At week 144, the decline in walking ability was greater than the decline measured at 74 weeks implying that the drug's effectiveness may be declining. The average decline in walking ability as measured by the 6-minute walk test (6MWT) was 2.2 meters at 74 weeks compared to an average decline of 33.2 meters (n=6) at 144 weeks.
SRPT longs have had quite a bit of excitement since Q4 of last year. Shares peaked at $55.61 on October 7 and bottomed at $12.12 on November 13. Prices rebounded to $40 by April 21 only to retreat and start another down trend.
The FDA grants Fast Track designation to Akashi Therapeutics' lead product candidate HT-100 (delayed-release halofuginone) as a potential treatment to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne Muscular Dystrophy (DMD). The agency has also designated HT-100 an Orphan Drug. The product candidate is currently being evaluated in a Phase 1b/2a clinical trial.
Analysts say that Shire's (SHPG) attractive pipeline and tax inversion-stoking location make it a prime acquisition target. For example, Allergan (AGN) approached the company in April about a potential deal. Its best defense against an unwanted takeover may be to go on the offensive and make an acquisition itself. This would seem to be a good strategy since CEO Flemming Ornskov likes to acquire. He has presided over at least six acquisitions since taking the helm.
Cenkos Securities' Navid Malik perceives that Sarepta Therapeutics (SRPT), Prosensa (RNA) and ThromboGenics are attractive targets. So is NPS Pharmaceuticals (NPSP), but its CEO says the two firms aren't talking.