What's worse is his obsession with Beckers' patients and their dystrophin levels. There is probably less intense research and data available on Becker's, but he insists on using it as a comparison to dispute Etep's dystrophin measures.
It is clear from the briefing documents that the required basis for approval is The Perfect Trial - a placebo-controlled trial in which each boy is the exact same age, size and state of disease progression. Despite the fact that is impossible to achieve, that doesn't prevent the FDA from explaining away any signs of benefit in the data as a result of variability in the patient population or bias in how the data is analyzed. The 141M difference in average 6MWT (256 vs 114) at 36 months is explained by a theoretical potential bias in using historical control data. There is no assessment of how the control group was selected by SRPT or any specific biases identified between the two groups, just the fact that there is a theoretical potential bias is sufficient to dismiss the measured clinical impact of 3 years of treatment.
The FDA supposedly recognized the limitations and ethics involved in the trial design and and resulting clinical evidence in a rare disease when they approved the design of the confirmatory trial, but based on their criticism of their Phase II data, it is not clear whether the Phase III trial results will not be dismissed due to similar issues. There is no evidence that the position advocated by the USC letter - i.e. the best way to validate the clinical efficacy is to get as many kids on Etep as possible and assess them over the long-term - is being considered at all by the FDA. Unless a miracle drug comes along that actually does reverse the disease and get kids out of wheelchairs, these DMD boys are doomed, because no company is capable of designing and executing a trial that would eliminate all the theoretical biases and variability that could influence the data.
That is the problem with today's society - people have no shame. This guy raises issues with the data and trial that have already been resolved/explained, leaving him with this distaste for spending his "precious" tax dollars. If that is his concern, he should be writing letters about how the big pharma companies continue to screw the taxpayers by raising prices on blockbuster drugs, none of which are the only hope in treating a deadly children's disease. Embarrassment used to keep people from writing garbage like this - he is obviously a paid shill or has no family or friends to point out what a tool he is being.
As anxiety filled this AdCom will be for Exon 51 boys, it is even more so for boys with other Exons. If SRPT is denied AA for Exon 51, it is a 1-2 year setback for those boys, but that could translate into a 5+ delay for the other Exons. Without AA approval, SRPT will not have the resources to pursue the "lesser" Exons and it may not be economically feasible at all if the FDA does not allow some sort of "class approval" program to develop and instead forces larger Phase-III-type trials for all the different Exons.
thig - I hate to believe in that the FDA would act in such a vindictive or profiteering manner, but it is difficult to comprehend otherwise. How else do you explain an organization that claims to be working in a collaborative effort and then sabotages the company's NDA with false accusations.
xt - this post just proves how uninformed you are. Nobody disputes that steroids and PT will impact the progression of DMD, the "horse manure" is Farkas' blind speculation that the boys in the trial received higher levels of both - he had no evidence for that conjecture and the parents and doctors of the boys vehemently denied it.
That is the most disturbing aspect of the initial BDs - the FDA claims to have open communications with the company and parents in their efforts to bring a treatment to boys with DMD, but numerous points in the BD that were ultimately refuted could have been addressed with a simple phone call to the company. Instead, the FDA blindly made assertions about the boys and the tests that simply were false. If this didn't involve getting a treatment to dying boys, it would be another laughable case of govt bureaucracy gone amok.
berry - has he actually stated publicly that he believes very few new drugs work? That is an amazingly #$%$ statement from someone in the position to be a gate-keeper for live-saving drugs.
I agree with the fact that the new briefing docs will not be a "love fest" - has the FDA ever issued briefing docs that were overwhelmingly positive - if there were no issues to resolve, they would not even be having an AdCom. However, the idea that Farkas will "defend his position admirably" is equally ridiculous - many of his positions have been called out by the company, the parents and the doctors as being completely false or mis-guided - only a psychopath would try to dispute what experts have defined as reality. Practically, the new BD are likely to raise questions or express concern over the validity of certain test results, instead of an in-the-face attack that the results are meaningless, as the first BD's did. Anybody that believes that the FDA is going to express any positive views in the BDs (other than safety, which I'm sure they will still find an avenue to question the long-term safety risks) is kidding themselves.
I thought you were talking about partnerships for some of their new GPCR or early pipeline drugs - he was talking about marketing deals for Omidria. I'd like to think that they can generate significant sales for Omidria outside the US, but remember that it has been approved in Europe since August of last year - Omidria should not be a difficult drug to market and distribute, so the fact that it is taking the better part of a year to get any kind of agreement in place would indicate that the opportunity must not be that significant?
I have been listening to Dr. D talk up the GPCR platform for the past 4 years - he presents it as if they have the rights to whole classes of new drugs that could eclipse all the currently existing drugs and yet they have not licensed a single molecule or patent position in their entire portfolio. He needs to either prove there is value in all this GPCR work or stop talking about it, let alone wasting company resources on the research.
a "bad reputation on Wall Street"? The company has a market cap of less than $10M and one analyst following the company - I don't think this company is a blip on any Wall St radar screen. When it comes to growth, they grew revenues 25% last year - if they can match that growth rate the next couple of years, they will have a viable company.
That is not a change in the forecast, but I think he wanted to clarify the distinction for the presentation - i.e. Omidria is already generating cash above all its direct manufacturing, marketing and distribution costs and by the end of the year the revenue from Omidria will make the entire company cash flow positive - not bad for a drug that has only been on the market for a year.
I guess we should be happy they didn't pre-announce a revenue miss - a few companies that were at Needham already did. I hope that the fact that the CEO reiterated their goal of becoming cash flow positive this year means that Omidria sales for the quarter were on track?
Like most biotechs, CBMX is in a race to get to breakeven before the cash runs out. If they can get there, or get close enough to possibly get a bank loan, then dilution might be avoided. If not, they will have to go through another round of dilution. At this price, the risk/reward is tempting, but it is hard to gauge what the long-term potential for revenue will be - test volumes have been increasing consistently, but pricing on the tests has been mixed?
Oh joy, after talking about international sales for several years, they may actually have some in the second half - great execution! I was baffled by your labeling of the European market as "socialist" - what do you think the Asian healthcare markets are?
This conversation reminds me of how CG was bashed by many posters because of the supposedly aggressive posture he was taking with the FDA. I shouldn't have to remind people that the FDA opened the door for AA on their own and despite clear calls from the scientific and medical communities that Etep was producing dystrophin, providing clinical benefit and was ultra-safe, the FDA threw the AA process into reverse because of their irrational decision to assign some level of equivalency between Etep and drisa and their inability to assess the validity of the various dystrophin measures. The fact that there was little to no pressure on the FDA from congress or the scientific community with respect to drisa is sufficient to dispute AF's rantings. Congressional pressure is warranted here, just as pressure from CG was warranted, because the FDA is failing to do its job, while innocent boys are wasting away.
The EU market for Galafold is big enough, but investors don't buy into it - too much hassle dealing with the various govt agencies for reimbursement and pricing adds a layer of risk, so investors will adopt a wait-and-see approach. I think Galafold will be very successful in the EU - it way take a few years to fully ramp up and by then, FOLD will have its ERTs hitting the market, at which point this will be a $30+ stock.
I have never seen a significant pop from a biotech upon European approval - most biotech investors think until you have US approval, it is not worth touching. For a lot of drugs and indications that is probably true, but the EU is more progressive and accepting of drug prices for rare diseases, particularly ones that are "personalized" (e.g. linked to a genetic test) and can improve the patient's quality of life. Whereas a lot of drugs have pricing in the EU that is half or less what can be charged in the US, I believe the pricing of drugs for rare diseases have more parity, which is why 70% of the potential $ market for Galafold is outside the US. Unfortunately, we will likely have to wait for meaningful revenues kick in before the "market" takes notice?
You messed up the computation - the previous post notes the JPM estimate of $350M-$400M worldwide potential for Galafold with 70% ex-US - that translates to $245M-$280M outside the US. The $350M-$400M already accounts for the amenable mutation allocation - the entire Fabry ERT market is currently $1B+. Crowley has talked about how Fabry is under-diagnosed and under-treated, as many patients avoid the ERT infusions until their disease progresses, so Galafold may be able to capture a larger population than is currently represented by the ERT patients.
Reminds me of one of my first biotech investments - UTHR was waiting on the approval of Remodulin in mid-2002 and I had bought a few thousand shares at a split adjusted cost of ~$5 per share and it barely moved when approval came, so I unloaded it. Within 3 years it was trading over $30 and today it is over $110 - it peaked at $190 before the biotech market crashed the past few months. I think FOLD is a long-term keeper - they will have two drugs approved by mid-next year (SD-101 and Migalistat) and proof of concept on their ERT program, so I think we will be at $20 by the middle of next year and $50 within 5 years, assuming their ERT drugs are successful.
Looks like your wish came true - with 40% of the float short, I would have though this would have spiked into the double digits? I guess the news was not unexpected?