DMD Data by Giant Pharma Pfizer Kept Sarepta In the Driver Seat with Superior Drug

Pfizer (PFE) presented its first set of data (n=6) from the mini-dystrophin gene therapy for DMD (PF-06939926) at the PPMD conference on June 28th. Much speculations surrounded Pfizer’s decision to report the dystrophin quantification using mass-spec. Sarepta (SRPT) has used WB-western blot since 2015 during the Eteplirsen FDA approval process. Sarepta has also used it in […]

May 14 Biotech Update

I am not sure who had a more difficult day yesterday. You all for having to watch another market meltdown over China fears (and others) or me who had to help chaperone a middle school trip to New Orleans. In any case, we have all survived and the market seems to be bouncing this morning. […]

February 7 Biotech Update

We start off a little weak this morning, which is not completely unexpected given that we are at the top end of the recent range. Obviously it would be nice to see a breakout above the range to start another leg higher but range bound trading after the recent run is not bad at this […]

March 16 Biotech Update

The sector did not really have an encouraging day yesterday. Instead of broadening out the rally, the previous leaders slipped and nothing picked up the slack. There was a chance for the large caps to move the rally forward but they did not do so. Of course, no one should worry too much about a […]

March 15 Biotech Update

We might be seeing a little of the broadening rally that I talked about as the XBI seems to be modestly underperforming the IBB the past couple days. The move in the sector was mainly concentrated in the XBI (more heavily weighted towards the SMID) versus the IBB (more heavily weighted towards the large caps) […]

SRPT Was and Still The DMD Gene Therapy Leader

Today after the market close, Solid Biosciences (SLDB) announced it has received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold. The news sent SLDB share price down to $11.90 or […]

February 9 Biotech Update

The broader market looks heavy and it appears that if we end the day red that the weekly losses will be accelerating. Markets do not bottom on a Friday, so if we cannot have a real move higher today, then we will likely be lower again to start next week. We are obviously higher to […]

Dr. James Wilson Paper About Safety Concerns, Is Not Sarepta’s Concern!

Today morning, most of the gene-therapy stocks including SLDB, AVXS, ONCE, RGNX, BOLD, ABEO and even SRPT Stumbled following Dr. James Wilson paper published online “Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN” (link to the paper) highlighting the toxicity in NHP- non-human primate study. […]

January 30 Biotech Update

The sector remains strong. The market has weakened a little but the sector was still holding on. If the broader market weakness continues, it will eventually drag down the sector but that has not happened yet. Of course, a bit of a pullback is well overdue and will happen eventually but the trigger and timing […]

January 26 Biotech Update

The market is relentless. Just as it is important to buy when the market seems to be going down forever, it is also important to sell on these sort of rips higher. The selling at the high levels is always more difficult for me, so I want to spend some time today talking about potential […]