Catalyst Pharmaceutical Partners Granted Orphan Medicinal Product Designation in European Union for CPP-115 for the Treatment of West Syndrome

Catalyst Pharmaceutical Partners, Inc.CPRX +2.80% today announced that the European Commission (EC) has granted orphan medicinal product designation for the company's investigational drug, CPP-115, a novel GABA aminotransferase inhibitor, for the treatment of West Syndrome (infantile spasms). The EC designation is based on the recommendation by the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) after their review of all relevant preclinical data for CPP-115, which showed that it may have a longer duration of action, improved benefits and fewer retinal side effects than the existing first-line treatment."We have hit another major milestone in our efforts to advance this clinically important treatment for patients around the world," said Patrick J. McEnany, Chief Executive Officer of Catalyst. "Obtaining an orphan designation for CPP-115 in the European Union (EU) is an important step that builds upon our recent progress with CPP-115, including the grant of Fast Track development program designation by the U.S. Food and Drug Administration (FDA) for cocaine dependency and commencement of a first-in-man Phase I(a) safety study.""Our orphan designations in the EU and U.S. support our global development strategy for CPP-115 and our goal of providing improved therapies for infants and children with West Syndrome and infantile spasms," said Steven R. Miller, Ph.D., Catalyst's Chief Operating Officer and Chief Scientific Officer. "Our preclinical experience with CPP-115 to-date has demonstrated its potential in treating infantile spasms (West Syndrome) with greater safety and efficacy than existing therapies. Currently, there are limited treatment options for this serious pediatric disease, all of which have significant side effects. We hope to offer providers and their patients a more effective and safer therapy than is currently available."Orphan medicinal product designation is granted by the EC, in conjunction with the EMA's COMP, to promote the development of products to treat life-threatening or very serious conditions that are rare and affect not more than 5 in 10,000 persons in the EU. Key benefits include 10 years of market exclusivity if CPP-115 is approved for the treatment of West Syndrome, as well as possible EU-funded research, protocol assistance and fee reductions for centralized activities like a Marketing Authorization Application (MAA).Similarly, orphan drug designation is granted by the FDA Office of Orphan Drug Products to promote the development of drugs and biologics for the treatment of rare diseases and disorders that affect fewer than 200,000 persons in the United States. The key benefit includes a 7-year period of market exclusivity if CPP-115 is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication. Other potential benefits include tax credits for clinical research costs, annual grant funding, clinical trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.Catalyst filed its application for orphan medicinal product designation in the EU through Catalent Pharma Solutions, Ltd., its representative in Europe. The approved grant of orphan medicinal product designation will be held in the representative's name until Catalyst instructs transfer of it to Catalyst or to another biopharmaceutical company with a presence in the EU.

Catalyst Pharmaceutical to sell 2.26M shares

Catalyst Pharmaceutical Partners (NASDAQ: CPRX) plans to sell 2.26 million shares to help fund non-clinical studies of a potential product to help with pain management and treat addictions and epilepsy. The Coral Gables-based company said the price per share will be $1.12, which was the Monday closing price. Catalyst expects to receive net proceeds of $2.2 million after the offering closes on or about March 11. Roth Capital Partners acted as manager for the offering. Catalyst expects to use the net proceeds to complete the studies necessary to file an investigational new drug application with the FDA for CPP-115, to fund a Phase I human clinical trial of CPP-115 and for general corporate purposes. Catalyst's website contains technical descriptions of CPP-115, which was discovered by Northwestern University and is being developed under a licensing agreement. Northwestern's Dr. Richard P. Silverman led the team of scientists that developed CPP-115, the website says. Silverman holds 41 patents and is the inventor of pregabalin, which is sold under the brand name Lyrica and had $3 billion in sales in 2009. Lyrica, a product of Pfizer Inc. (NYSE: PFE), is used to treat diabetic nerve pain, pain after shingles, fibromyalgia and partial onset seizures in adults with epilepsy who take one or more drugs for seizures, according to its website. In 2010, Catalyst reported completing a series of non-clinical studies for CPP-115 that were designed to demonstrate safety characteristics and efficacy in animals models for epilepsy and cocaine addiction. Catalyst's largest institutional shareholders are mutual fund companies Federated Investors, which has a 12.85 percent stake, and FMR LLC (Fidelity), which has a 6.44 percent stake, according to Yahoo Finance.
Read more: Catalyst Pharmaceutical to sell 2.26M shares | South Florida Business Journal

Dr. Richard B. Silverman Joins Catalyst Pharmaceutical Partners' Scientific Advisory Board

Catalyst Pharmaceutical Partners,today announced that Richard B. Silverman, Ph.D. has joined its Scientific Advisory Board. Highly accomplished in medicinal chemistry and rational drug design, Dr. Silverman is the inventor of Pfizer's $3 billion pain medication, Lyrica(R). He is also the inventor of CPP-115, a GABA aminotransferase inhibitor, which recently demonstrated positive safety and efficacy results in a number of non-clinical studies that Catalyst reported on November 1, 2010.

Patrick J. McEnany, Catalyst's Chief Executive Officer, stated, "Dr. Silverman brings a wealth of knowledge in the field of molecular design of pharmacotherapies. We believe that Dr. Silverman's presence on our Scientific Advisory Board will help us advance the development of CPP-115 to treat substance abuse and other diseases of the central nervous system, including epilepsy and neuropathic pain."

Steven R. Miller, Ph.D., Catalyst's Chief Scientific Officer, stated, "Dr. Silverman is a renowned expert in the field of rational drug design, on the biochemistry of GABA aminotransferase, and on CPP-115. His insights into the mechanism of action of CPP-115 will be very helpful in the design of future non-clinical and clinical studies, and I expect his interpretation of results from those studies to be equally useful to Catalyst. Dr. Silverman will be a valuable addition to our scientific team, particularly with regard to the continuing development of CPP-115."

Dr. Silverman is the John Evans Professor of Chemistry at Northwestern University and holds 42 patents. He is the recipient of numerous awards, most recently the 2009 Perkin Medal and the 2011 E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances from the American Chemical Society. In addition, Dr. Silverman has published over 285 peer reviewed articles and has written four books over his 34 year career in academia.

"I am delighted that CPP-115 has shown such outstanding biological and pharmacological properties and am excited that Catalyst Pharmaceutical Partners is shepherding it through the maze of scientific tests toward FDA approval for addiction and epilepsy," said Dr. Silverman. "The Northwestern University-Catalyst Pharmaceutical Partners partnership has been a highly rewarding collaboration."

Catalyst Pharmaceutical Partners Reports Positive Non-Clinical Safety and Efficacy Results for CPP-115

Catalyst Pharmaceutical Partners, Inc. today announced positive results from a series of CPP-115 preclinical safety and efficacy evaluations. CPP-115 was found to have a significantly improved retinal safety profile compared to vigabatrin. Additionally, the compound was found to be orally absorbed, not metabolized, and generally safe as determined in a battery of critical initial safety evaluations. Finally, CPP-115 induced significant responses in accepted animal models supporting potential efficacy as a treatment of both epilepsy and stimulant addiction.

Catalyst's initial evaluations compared CPP-115, vigabatrin and a placebo in an animal model optimized to assess visual safety. In this evaluation, CPP-115 was found to be significantly safer than vigabatrin, the only other known and commercially available GABA-aminotransferase inhibitor.

"The positive results from these studies represent a significant milestone for our company in that the responses in the animal models are strongly predictive of therapeutic benefit in humans," stated Patrick J. McEnany, Catalyst's Chief Executive Officer. "We are also extremely pleased that CPP-115 demonstrated a major improvement in visual safety compared to vigabatrin. The potential risk of peripheral vision loss from vigabatrin has been an important concern among patients and their physicians. Based on the strength of this and the other data found in these safety and efficacy studies, the company considers CPP-115 to be the leading candidate as a next generation treatment for addiction and epilepsy. We expect to initiate the remaining studies necessary to file an IND in the third quarter of next year."

"The effect of CPP-115 on retinal electrophysiological (ERG) responses showed significantly smaller changes from baseline than the effect of vigabatrin after both 45 and 90 days of drug exposure," said Cheryl M. Craft, Ph.D., the Mary D. Allen Chair in Vision Research at the Doheny Eye Institute and Professor of Ophthalmology and Cell & Neurobiology, Keck School of Medicine of the University of Southern California. "The observed vigabatrin treatment changes are similar to past reports of ERG deficits in individuals and in animal models, including our own published work. Therefore, these cumulative data from the current study support the hypothesis that CPP-115 caused substantially less retinal functional deficits than vigabatrin, which suggests this drug is likely to have an improved retinal safety profile compared to vigabatrin."

Catalyst Pharmaceutical Partners Announces Top-Line Results of CPP-109 Phase II Trial for Cocaine Addiction

CORAL GABLES, Fla., - Catalyst Pharmaceutical Partners, Inc. (Nasdaq: CPRX) today announced top-line results from its U.S. Phase II clinical trial to treat cocaine addiction. The data from the trial showed that CPP-109 did not demonstrate statistical significance in the primary endpoint -- that a significantly larger proportion of CPP-109-treated subjects than placebo-treated subjects were cocaine-free during the last two weeks of the treatment period (Weeks 11 and 12). The clinical trial did not reveal any unexpected "serious" adverse events.

Complete analyses of the clinical trial data (secondary clinical end-points and safety data) are ongoing. Based on the results of these analyses, Catalyst will evaluate what measures, if any, could be applied to improve the outcome of future studies and will also determine next steps to be taken regarding the development of CPP-109 for the treatment of cocaine addiction. In addition, Catalyst intends to investigate the reasons for the disparity between these trial results and previously published clinical and non-clinical results evaluating vigabatrin as a treatment for cocaine addiction. Catalyst expects to complete the analyses during the third quarter after fully unblinding the trial data and will then formulate future development plans.

Patrick J. McEnany, Catalyst's Chairman, President and Chief Executive Officer, commented, "We are obviously disappointed in the cocaine trial results, given the positive results of three prior human trials and numerous animal studies; however, we are not ready to abandon our view that CPP-109 has the potential for use in treating cocaine addiction, which still represents a significant unmet medical need. We will carefully study the results of our trial and assess the appropriate path for conducting future clinical trials evaluating CPP-109 for the treatment of cocaine addiction. We also expect to have the results of our methamphetamine proof-of-concept study during the third quarter, which will provide us with data that we can use to assess the next steps in our strategy."

Mr. McEnany continued, "As previously reported, we have sufficient cash to complete the analysis of the results from our cocaine trial, to complete our methamphetamine proof-of-concept study and to continue our operations through the end of 2010 without additional funding. As a result, we believe that we have sufficient financial and human resources to pursue a variety of strategies, which we will discuss in detail as they are developed."

Catalyst Pharmaceutical Q1 loss deepens

Catalyst Pharmaceutical Partners’ first quarter loss deepened to $3 million from a $1.6 million loss in the same period a year ago.

The Coral Gables-based company (NASDAQ: CPRX) had no revenue, as its drug candidates are in development. However, Catalyst said its $8.9 million in cash and equivalents as of March 31 should carry it through clinical trials and support operations through the end of 2010.

Catalyst CEO Patrick J. McEnany said that he expects top-line results from its clinical trial for drug candidate CPP-109 to treat cocaine addition by the end of June. Should the results turn out positive, the company would meet with regulators and begin planning a final-stage Phase III clinical trial.

Meanwhile, Catalyst expects to learn the results of its clinical trial for CPP-109 to treat methamphetamine addiction during the third quarter, McEnany said.

“We are actively exploring opportunities to obtain government funding from agencies that operate under the National Institutes of Health umbrella to further our development plans, which include developing large-scale clinical trials using CPP-109 to treat cocaine and methamphetamine addiction, as well as pilot studies for other addictions including alcohol and nicotine dependence,” McEnany said in a news release.

Catalyst shares were down 7 cents to $2.10 in afternoon trading. The 52-week high was $4.52 on Aug. 26. The 52-week low was $1.06 on March 11.