This Day On The Street
Continue to site
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

Catalyst Pharmaceutical Partners Provides Update On Research And Development Pipeline

CORAL GABLES, Fla., Feb. 11, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neurological diseases and disorders, today provided an update on its research and development pipeline.

"We are providing this information today to update our shareholders, patients, physicians, key opinion leaders and the financial community on our drug development activities. We are primarily focused on rapidly advancing the development of Firdapse TM for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), which is our lead product candidate," said Patrick J. McEnany, Chief Executive Officer of Catalyst.

Portfolio update


In October 2012, Catalyst acquired the North American rights to Firdapse, a proprietary form of amifampridine phosphate (3-4 diaminopyridine or 3-4 DAP), from BioMarin Pharmaceutical Inc. ("BioMarin"). Firdapse was approved in December 2009 by the European Medicines Agency for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare and sometimes fatal autoimmune disease characterized by muscle weakness. Firdapse has been granted orphan drug designation by the U.S. Food & Drug Administration, (FDA) for the treatment of LEMS, making the product eligible to obtain seven-year marketing exclusivity, if Catalyst is the first pharmaceutical company to obtain approval of an NDA for its formulation of amifampridine.

As part of its license agreement with BioMarin, Catalyst is taking over the sponsorship of their ongoing Phase III clinical trial evaluating amifampridine phosphate for the treatment of LEMS. The trial:
  • is designed as a randomized double-blind, placebo-controlled discontinuation trial as recommended by FDA to BioMarin;
  • has a goal to enroll approximately 30 LEMS patients (approximately one third enrolled currently);
  • currently has 7 active sites (expected to be increased to approximately 25 in the near future);
  • has defined as a primary endpoint-change in muscle strength during the 2-week, double-blind discontinuation period as determined using a validated questionnaire (Quantitative Myasthenia Gravis score); and
  • has defined as a secondary endpoint-change in walking speed (timed 25-foot walk test) during the discontinuation period.

For further details on this trial, please go to: ; Search "amifampridine phosphate".

With respect to the trial, Catalyst expects:
  • to complete enrollment by the end of 2013; and
  • to report top-line results from the double-blind portion of this clinical trial during the second quarter of 2014.

Assuming positive results are obtained from the trial, Catalyst hopes:
  • to file an NDA for Firdapse in the first quarter of 2015;
  • to obtain approval from the FDA of such NDA by the end of 2015; and
  • to commercially launch this product sometime in the first half of 2016.

Firdapse may also be an effective treatment for other neuromuscular orphan indications:
  • Congenital Myasthenic Syndrome; and
  • Myasthenia Gravis.

Catalyst believes Firdapse can achieve peak annual revenues from sales in the United States of approximately $100 million.


On August 27, 2009, Catalyst entered into a license agreement with Northwestern University (Northwestern), under which it acquired worldwide rights to commercialize new GABA aminotransferase inhibitors and derivatives of vigabatrin which were discovered and patented by Northwestern. Catalyst has designated the lead compound to be developed under this license as CPP-115. CPP-115 has been granted orphan drug designation by the FDA for the treatment of infantile spasms and orphan medicinal product designation in the European Union (EU) for West's syndrome (a form of infantile spasms). This means this product will be eligible to obtain the seven-year and ten-year marketing exclusivities available from the FDA and the EU, respectively, if Catalyst is the first pharmaceutical company to obtain approval of an NDA/MAA for CPP-115.

1 of 3

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

David Peltier identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
AAPL $94.99 0.00%
FB $99.54 0.00%
GOOG $678.11 0.00%
TSLA $148.25 0.00%
YHOO $26.82 0.00%


Chart of I:DJI
DOW 16,014.38 -12.67 -0.08%
S&P 500 1,852.21 -1.23 -0.07%
NASDAQ 4,268.7630 -14.99 -0.35%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs