This article is intended to provide investors who are studying Catalyst Pharmaceutical Partners Inc. (CPRX) with more information on the upcoming results of Vigabatrin or CPP-109. According to the company website:
CPP-109 is Catalyst's designation for Vigabatrin, a potential treatment for cocaine and other drug addictions. The FDA has accepted Catalyst's Investigation New Drug application to conduct clinical trials in the United States of CPP-109 for addictions (initially cocaine and methamphetamine) and has granted Fast Track status to CPP-109. Vigabatrin, also known as gamma vinyl-GABA or GVG, is marketed as Sabril® outside of North America by Sanofi-Aventis and within North America by Lundbeck Inc. Sabril is available in tablet and powder forms and used in combination therapy for specific types of epilepsy and alone in the management of infantile spasms (West Syndrome).
I am providing this information to help those who have been asking why Catalyst may be a great investment. For example, some seem perplexed that I left Astex Pharmaceuticals (ASTX) and before that Zalicus Inc. (ZLCS) to dine at the table of Catalyst Pharma. I hope to explain my rationale by highlighting the new information that I am learning in this follow-up article.
1. As background, Catalyst Pharma has informed investors it expects to publish news on CPP-109 by the "end of September." That news shortened the original timeline from Q1 2013 by nearly three months. The question of the hour is, Why? Has something bad happened? Or has something great happened?
The company and CEO stated in the July press release:
Catalyst Pharmaceutical Partners, Inc. today announced that it expects to report top-line results from its CPP-109 (Vigabatrin) Phase II(b) trial around the end of September 2012 versus the previous guidance of early in the first quarter of 2013.
After discussions with our collaborators, the National Institute on Drug Abuse (NIDA) and the Department of Veterans Affairs Cooperative Studies Program (VACSP), and our statistical and regulatory consultants, we have been able to work through the complexities of modifying our statistical analysis plan. This will enable us to report top-line trial results about four months earlier than previously expected.
But at the close of the press release, Catalyst's CEO makes a rather bullish comment. I assume these things are reviewed by lawyers:
"We are pleased that we will be able to report the top-line results of our CPP-109 Phase II(b) clinical trial sooner than expected," said Patrick J. McEnany, Chief Executive Officer of Catalyst. "We are committed to bringing safe and effective breakthrough products for the treatment of cocaine addiction to the market as quickly as possible, and the earlier reporting of these data is consistent with that philosophy."
Now don't take my opinion as fact, but I interpret McEnany's comment as bullish. I also interpret it as suggesting that the early release of data is because the Vigabatrin is being fast-tracked and so the greater good (addiction sufferers) is to move Vigabatrin forward as quickly as possible. After all, if the clinical trial was a bust and was halted for that reason, he would have stated that, but he didn't.
In addition, for investors' knowledge, Vigabatrin (or Sabril) has an exclusivity expiration date of August 21, 2014, as a new chemical entity and August 21, 2016, for orphan drug exclusivity which explains why Catalyst intends to file a 505(b)(2) since it is already approved for infantile spasm. A phase III trial may not be required as the FDA may use phase II data for a 505(b)(2) for a second medicament indication (cocaine addiction). Therefore, it appears something very positive happened that has allowed Catalyst to release the data early. Frankly, I find it very hard to understand how "earlier reporting of the data" can be construed as a negative comment.
But here's what may have happened.
2. According to a research study by the Institute of Ophthalmology and Visual Science, New Jersey Medical School - UMDNJ, Newark, New Jersey, Vigabatrin in adult cocaine addicts did not cause ocular problems. Ocular side effects have been a major concern. This research study was published this summer:
Visual acuity decrease was detected in 1 eye of a subject receiving placebo and in none receiving Vigabatrin. Posttreatment reduction in TVS more than 15 dB in 5 or more adjacent visual field location points combined with reduction in TVS greater than 33% in 1 or more of the rings was detected in 2 of 54 subjects (3.7%) from the Vigabatrin group and in 1 of 49 subjects (2%) from the placebo group (P = .9, NS). None of the PVF changes were bilateral or concentric.CONCLUSIONS:
Short-term use of Vigabatrin did not cause a decrease in visual acuity or significant peripheral visual field changes in cocaine abusers.
I view the above study as critical evidence favoring Vigabatrin's safety profile. I find the release of the above data that is concurrent with Catalyst's shortened timeline rather predictive. As a 505(b)(2), general information can be used as part of the filing (See: FDA presentation). And yes, it certainly seems this is the case since Catalyst Pharma issued its own press release citing the above scientific report stating:
...CPP-109 (Vigabatrin) describing ocular safety results obtained with CPP-109 (Vigabatrin) was published in the June 14, 2012 web edition of the American Journal of Ophthalmology [AJO]. These results were obtained from the Company's previously completed Phase II(a), randomized, double-blind, placebo-controlled trial in cocaine addicted subjects.The paper is entitled, "Visual Field and Ocular Safety during Short-Term Vigabatrin Treatment in Cocaine Abusers" by Tamara L. Berezina, Albert S. Khouri, M. Douglas Winship and Robert D. Fechtner.
The above study followed by Catalyst's own announcement strongly suggests that the FDA will be accept these additional study results. I can only interpret this as another sign of a favorable outcome. However, SA readers are strongly advised to arrive at their own conclusion. The above events do still appear to be a very positive sign though the final efficacy results remain unknown.
3. Investors who want to really want an in-depth scientific argument for CPP-109's potential effectiveness against cocaine should read the herein referenced article (The full article is available for free). I offer a brief snippet:
Based on this, we believe that GVG-elevated GABA levels in brain reward circuitry might directly counteract the actions of cocaine, opiates or DA on GABAergic neurons, thereby antagonizing cocaine-induced reinstatement of drug-seeking behavior. The role of an increase in extracellular GABA levels in the NAc remains to be determined. One possibility is that an increase in NAc GABA levels may hyperpolarize NAc medium spiny (GABAergic) neurons, and therefore desensitize NAc neuronal responses to cocaine-induced increases in NAc DA and inhibit cocaine-triggered reinstatement.
In other words, these scientists argue their case for why Vigabatrin may be an effective agent in helping those who are trying to defeat cocaine addiction. Catalyst's own study will offer definitive proof assuming it is successful. Investors are again reminded the very opposite outcome (failure) is also possible. That stated, I don't want to diminish the information that favors Vigabatrin's safe use for adult cocaine addicts.
As a 505(b)(2), Catalyst is working with the FDA, citing relevant and concurrent research such as the one cited in this article. As a fast-tracked drug candidate, this may explain how Catalyst was able to amend its statistical plans to satisfy the FDA and to move towards filing a rolling 505(b)(2)/NDA in 2013-14. Again, as a second drug indication a phase III clinical trial will most likely not be required; per Catalyst, its current plan is to move towards the NDA preparation/filing.
Finally, late Friday Mr. Jim Cramer of CNBC pointed his viewers to Dynavax Technologies Corporation (DVAX). Just for a moment I thought he was going to say, "Catalyst Pharmaceuticals!" I mention this because Mr. Cramer pointed out that Dynavax has a key stock-moving catalyst pending as does Catalyst. Thankfully, he reminded his viewers of the inherent risk even saying while the stock could double, it could also be cut in half just as quickly. The same holds true for any stock be it Zalicus Inc. that recently plunged on Synavive's phase IIB failure or Peregrine's (PHMM) momentary fun in the sun citing successful phase II data for a cancer drug. Biotech trading is risky which is why:
- I prefer taking a modest profit than being greedy and ending up a pig who got slaughtered.
- I know market forces and a consortium of big-players are much, much, much bigger than me so when I see a favorite company taking a hit, I may ring the register rather than risk my profits.
- I invest to make money and spend a great deal of time studying biotechs to achieve my personal goals.
- I think taking a loss (no matter the size) and moving my money elsewhere to get back to even is better than being a bag-holder.
- I am constantly questioning my own decision-making which means I can reverse an earlier decision in a moment's notice when it comes to buying and/or selling a stock.
Additional disclosure: Investors buy and/or sell at their own risk. I declare that I may trade (buy and/or sell) any stock at any time mentioned in this article. For me "long" is until I sell. I do not "short" stocks.