Big names associated with the raise. $100 million only about 2.5 million shares. Company needs sufficient capital. Positive imo
SRPT should go higher too
Sarepta Therapeutics (SRPT) surged 39% yesterday after the FDA’s surprise decision to consider an early application for its cerebral palsy drug. And today its shares keep climbing.
Wedbush’s Christopher Marai and team outline the bull’s case:
We believe that the FDA’s guidance paves the way for accelerated approval (AA) of eteplirsen. The FDA guidance appears to us to offer a pragmatic approach to evaluating the safety and efficacy of compounds for the treatment of DMD, where endpoints are difficult, patients are few and no drugs currently exist…
Importantly, [Sarepta] is likely to support AA with a small, 60-80 patient confirmatory trial of eteplirsen with a historical control group. The FDA has requested additional safety and dystrophin data from two small trials (n=~20) in younger patients and non-ambulatory patients. Finally, the FDA has proposed that [Sarepta] run a randomized placebo-controlled trial of a follow-on compound to support a correlation between dystrophin production and clinical outcomes. We believe this indicates FDA’s willingness to relax requirements for the rarest exons.
FBR Capital Markets’ William Tanner worries that Sarepta’s move is just a massive short squeeze:
FDA guidance that Sarepta may be able to file an NDA for eteplirsen is a positive surprise (and painful for the shorts) but we believe the news only marginally de-risks the program. Given the side effect profile observed thus far, we would be surprised if safety issues arose that might derail approval as we suspect regulators will view any safety risk in the context of the fatal nature of the disease. We view evidence of clinical benefit as more tenuous in nature (mainly a function of the small “n” in the 201/202 trial) and believe more convincing proof will be needed for full approval. The FDA’s new-found flexibility in evaluating eteplirsen is reasonable to us and seems to be the only tenable tack if rancorous public criticism is to be avoided. After the initial short squeeze has abated, we question the extent of follow through and so we are maintaining a Market Perform rating on the stock.
Shares of Sarepta have gained 11% to $37.86 at 1:18 p.m. today.
in their disease, like Jett. This is a tremendous opportunity for our community – an opportunity to truly develop the tools we need to move drugs out faster. Have no doubt – NO ONE got dosed with “drug” yesterday – the community got dosed with a clearer path for all drugs…and an opportunity to find out the answers to the many questions that have slowed us down for so long. This is just the beginning, not the closure of victory.
Posted by Christine on April 22, 2014
The news of a clearer road came yesterday. The press release hit around 6:55am. Jenn McNary and myself were ready. Our communication with the FDA had stalled the week before, not once – but twice. We felt things moving. It was also Easter weekend – as a Christian, I spent the weekend reflecting and reading about Sarepta (Zarepath.) The symbolism was overwhelming to me. Prayer, Petition and Prophecy.
The news was encouraging and reason to celebrate.
The reasons for a clearer road ahead are simple. The FDA gave guidance on good science, not sympathy from parents/advocates. There have been no side effects from the drug. Those in the trial – are stable, recent 120 week pulmonary data showed respiratory stabilization (never seen in Duchenne before), and seven of the world experts in Duchenne met with the FDA, answered their questions and spoke in a unified voice – this drug deserves a chance to be reviewed.
Lastly, our reason to celebrate was about more than eteplirsen. Communication and advocacy cleared the way and demanded that the “process” work the way it was intended and newly legislated by FDASIA – that was the reason to celebrate yesterday. Understanding how the agency views potential therapeutics in Duchenne, and what the guidelines look like when considering approval for drugs in Duchenne is a VICTORY – albeit small right now, but still a victory. It opens the door of possibilities for drugs in the pipeline and compounds currently in trial. Without the guidance given during this first historical event – other potential therapeutics could be held up by regulators longer than they need to be…industry, the community and organizations now an opportunity to build off this framework, in real time, with real Duchenne patients – not theoretical or speculative patients. This allows natural history, bio-markers and endpoints the chance to be clearly defined for our young ones, our ambulatory kiddos – and those more progress
More upgrades this morning
Great, so let's conduct another lengthy trial just to confirm what we pretty much know. Who cares about the children who will be come sicker and/or will die?
but again, they can put all of the disclaimers on the drug they want. that does not negate he fact hat gsk gave up entirely on the drug and the fda did a 180 on eteplirsen because they did not like drisp's results and the company even revealed (prosensa) mediocre reults . kind of hard to justify approving drisp over eteplirsen
we keep talking about hamburg being married to someone who has a big interest in gsk and that may influence the fda to approve drisp. remember though, gsk completel gave up on drisp. gsk is no longer in the picture