Which brings us full circle to the current (at least second, by our count) delay in starting AVXS' intrathecal trial targeting Type 2 & 3 SMA patients. And this is what we believe the press release was really about (and why AVXS wanted to sneak it out before Christmas when most of the Street was away from their desks), despite the comically irrelevant title. AVXS can talk about cGMP all it wants, but without filing a new IND, it cannot start its trial. No IND filing, no Phase 1 trial, it's that simple. Simultaneously, we find zero evidence that AVXS's current IND will allow intrathecal administration. We believe AVXS can't start the trial without filing the intrathecal IND - this is consistent with literally hundreds of other IND/Phase 1 trials submitted to the FDA over the years. Changing from intravenous to intrathecal is clearly a change in the route of administration. Specifically, the proposed AVXS-101 intrathecal trial satisfies one of the most important criteria for why a company needs to file an IND: there is a change in the approved route of administration or dosage level. We believe this new route of administration will require a new IND application, and that this is the real reason for yet another trial delay from AVXS, not the manufacturing issue they wrote about in the press release. AVXS is apparently now attempting to move to an intrathecal route of administration. This trial used an intravenous route of administration. The FDA reviews the IND filing to determine safety before giving the green light.Īs we will discuss later, the NCH submitted the IND for the initial Phase 1 trial of AVXS-101. A biotech or pharmaceutical company submits an IND to the FDA to gain permission to begin a Phase 1 trial. We suspect a lot of AVXS bulls don't even know what an IND filing is, so let us clarify: IND stands for Investigational New Drug. The continued delay in starting the trial is a significant setback for AVXS bull The implied IND delay, which we believe means AVXS cannot start its Phase 1 trial and which hints at a potential ongoing problem with the FDA, is the real story to emerge from this PR, in our eyes, and is the subject of much of our report. We will discuss cGMP and the challenges AVXS faces in manufacturing its product at commercial scale later in this report, but the main gist of our argument is that cGMP is a relative non-issue, and that by framing the press release this way, AVXS has by our reading implicitly guided the market that their intrathecal route of administration IND filing for SMA Type 2 & 3 will be delayed yet again. Ostensibly, this press release is about cGMP manufacturing of AVXS-101 - that is what the title of the press release mentions and discussion of cGMP for AVXS-101 makes up the majority of the content of the press release. "The company now expects to initiate the planned study of AVXS-101 in SMA Type 2 patients in Q2 2017." "he company has made the strategic decision to use the intended commercial GMP derived product in all future studies of the company's proprietary gene therapy candidate, AVXS-101, including the planned spinal muscular atrophy (SMA) Type 2 trial."Īnd then nestled away a little lower in the PR without acknowledging the timeline of their planned study in SMA Type 2 Patients was just delayed from the guidance they gave as recently as November, AVXS had this gem: The press release was titled AveXis to Use Intended Commercial GMP Product in SMA Type 2 Study. We're less accustomed to seeing $1.6 billion market cap stocks play these games, but truth be told, we believe AVXS is a penny stock in sheep's clothing, so what's a billion-six among friends? At Mako Research, we're accustomed to penny stocks trying to sneak out bad news in light pre-holiday trading and generally attempting to spin bad news as good. New Event #1: We Believe AVXS Just Delayed the Filing of Their IND for Type 2 & 3 SMAĪfter the close on Wednesday, December 21st, AVXS issued a press release that more closely resembles a bad game of verbal twister than an actual corporate PR. This follow up report will discuss these new events and why the worst case scenario has just begun for AVXS. Since our first report just over a month ago that fully debunked the bull case on AveXis ( NASDAQ: AVXS), new events have transpired that have further strengthened the bear case on the stock.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |