There is a 50/50 chance of Catalyst succeeding eventually. Near term maybe still bearish.
1st pt: I did a check on 3,4 DAP on the FDA orphan drug database and both catalyst and jacobus's 3,4 DAP drugs have orphan drug designation. The crucial orphan drug approval status has not been given to either. (Note: some people said catalyst misled investors but that is not the case. read their website, they only said firdapse has orphan drug designation.)
2nd pt: Checking the website for clinical trials, catalyst's trial is phase 3 and jacobus's trial is phase 2. However, both trials looked similar (30 pple, double blinded, drug taper to placebo for 50%, walking test..etc. there are some difference which I hope some biotech experts can help answer..). My point here is we cannot determine who can succeed based on the clinical trial alone. Remember Onyx's krypolis is phase 2 only and got FDA approval status for some indications (2nd, 3rd line) so once jacobus's trial finishes, I believe they have a chance to get the FDA approval. Jacobus's trial is ahead by about 2-3 mths but catalyst got the breakthrough drug designation (i believe is similar to FDA fast track status) which works to their advantage in the eventual approval timeline (usually 6mths instead of 1 year).
3rd pt: The manufacturing capability of jacobus and catalyst. Catalyst should be well supported by biomarin's expertise in this area while jacobus is unknown yet (somebody noted jacobus had been served warning letters by FDA before). FDA drug approval also take note of the manufacturing process and if that is not up to par, may delay approval. While reading the clinical trials, I had a sense that catalyst's trial is more well designed to show improvement and superiority while jacobus is more ad-hoc (invitation only,later expanded to all).
In conclusion, I believe jacobus may be able to show a topline result earlier (2014 april - may), resulting in another drop but should win the race for approval eventually.
Sam, based on all the known information, Catalyst does appear to be in the drivers seat in terms of clearing the FDA ahead of Jacobus and gaining orphan drug exclusivity. Hard to debate against this scenario without additional facts to back any case to the contrary.
HOWEVER, like myself and other investors that have recently started to take a closer look, the bigger question is more centered around realistic commercial potential (no WS hype) and how to value CPRX "assuming" confirmation of orphan drug exclusivity in NA.
I'm not at all convinced or buying into some of the peak estimates that have been thrown out there for public consumption. Granted, the NA market could be a bit more lucrative in comparison to the EU but the sales figures generated by BMRN are absolutely underwhelming relative to the sky high or pie in the sky estimates for NA. In addition, factoring in the size and financial strength of BioMarin still unable to increase sales anywhere near 30M annually indicates the questionable, at best, likelihood of success for a tiny little company with no sales/support infrastructure or marketing experience.
The above leads into additional puzzlement. CPRX is LOADED with licensee payout obligations in the form of royalty and milestone payouts from future sales. This includes cash considerations to BioMarin, Huxley Pharma shareholders as part of the 48M buy out agreement in '09 I believe, and an undisclosed Licensor of Firdapse that CPRX is also contractually obligated to pay cash considerations based on sales.
Whats left for CPRX and shareholders? Next question is if CPRX is capable of in-house sales/marketing? If so, how much cash will they need to do it? If not, how much will it cost them in terms of percentage of sales to attract and land a partnership if that option is even possible.
To be con't
hmm my post disappeared again. maybe bcos of the article link. did some research and found this article named: kv-pharma-sues-fda-over-makena-compounding . google it and check out the article and discuss.
Next, why did BioMarin, mid trial, just throw in the towel and off load this asset to such a small company for literally NOTHING if the potential in NA is viewed as significant by the street shills? If the optimistic view is even close to accurate, why did no cash rich entity step up and offer 20, 30, 40M+ in cash to grab Firdapse NA out from under Catalyst?
What possible sound business strategy is behind the idea of licensing out a high probability of approval asset with the intention of a future buy out of the licensee in it's entirety and especially at a premium?
Bottom line Sam is that the street pump was in motion to move the stock based on the narrow focus of FDA approval with the fundamental truths distraction buffers working overtime.
The difference between a shorter term trade that rides a manufactured wave of optimism and an investment for the longer haul.
@pfsiow78: You are talking non-sense on POINT # 2. What's your ground of claim that Jacobus Phase II outcome would be ENOUGH to get FDA approval? If that was the case, WHY FDA asked CPRX to go with Phase III in the first place? 3,4 DAP is no way superior than Firdpase. Then how could you come up with this IMAGINARY conclusion?
If you read my post carefully, I am leaning to a catalyst success but jacobus has obviously made plans to disrupt catalyst's plans on getting firdapse on orphan drug approval status.
My reasoning is that jacobus will not spend money to conduct the trial if they want to go to phase iii since that will mean catalyst will get the approval first and jacobus's program will be finished. In my opinion, jacobus will try with phase ii data and they shown that by expanding the candidate program.
hmm.. I did not conclude the outcome will be enough but they do have a chance to submit for approval. Not all drugs need to go to phase iii to get approval but not going to phase iii is obviously a danger to approval. Note the designs of the jacobus phase ii and catalyst phase iii is quite similar but jacobus phase ii's design is weaker in my opinion. I do own biomarin so I hope catalyst will be successful too.
You are confused.
Jacobus is over, done, kaput.
Why else did they resort to a hit article on their competitor?
1. Why was Jacobus cherry picking their patients?
2. Why would Jacobus spend money to prove a drug they give away for free?
3. After catalyst gets FDA approval, Jacobus can continue to prove a possibly less stable and possibly inferiorly produced product for 2nd and 3rd line and give it away for free, but why?
4. Jacobus' trial (not including selection criteria) is about 1+ yr behind Catalyst.
5. The reason Catalyst got the Breakthrough Therapy designation is because they are the one who broke through proving to the FDA that their version is safe and probably efficacious.
6. Jacobus lost. The game is over. That is why they are attacking their opponent publicly in more than one venue.
7. Why would they pay money for trials to give something away for free unless it wasn't really free? What advantage do they enjoy by giving it away for "free"?
8. With the lack oversight making their drugs and warnings, it would seem they are overburdened at any rate.
9. Catalyst is a year ahead, has the breakthrough therapy status, has the approval for phase 3, has superior patient criteria, has a possibly superior and more stable version, and has already won the race.
10. You didn't think this through.
Let me emphasise on pt 2. Once any company gets approval, then it is over for the other company. FDA will only award orphan drug approval status to one drug unless the other drug can exhibit superiority to the other one. Both drugs being 3,4 DAP, there is basically no difference. Even if one company decides to test for superiority, that will mean a newly designed phase 3 test which means 1-2 years more.