Catalyst Pharmaceuticals

Catalyst Pharmaceutical Partners, Inc.
Public company
Traded as NASDAQ: CPRX
Industry Biotechnology
Headquarters Coral Gables, FL, USA
Key people
 Patrick J. McEnany, Co-Founder, Chairman, President and Chief Executive Officer
Products in development
Revenue US$ 0 million (2014)[1]
Decrease US$ -15.5 million (2014)[1]
Total assets Increase US$ 43.9 million (2014)[1]
Total equity Increase US$ 35.2 million (2014)[1]
Number of employees
 20 as of March 20, 2015[1]
Website www.catalystpharma.com
Footnotes / references
[2][3]

Catalyst Pharmaceuticals is a biopharmaceutical company based in Coral Gables, Florida. The company is developing therapeutics for rare neurological diseases, including Firdapse (3,4-diaminopyridine phosphate, a potassium channel blocker) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) and CPP-115 (a GABA transaminase) for the treatment of infantile spasms.[4][5]

History

Founded in 2002, Catalyst Pharmaceuticals competed an IPO in 2006.[6] Catalyst focused primarily on developing therapies to prevent addiction until 2012.[4]

Business Development

In 2009, Catalyst Pharmaceuticals in-licensed worldwide rights to a family of GABA inhibitors including CPP-115 from Northwestern University. [7][8] Catalyst Pharmaceuticals in-licensed the right to develop Firdapse for the North American market from BioMarin in 2012.[9]

Product Candidates

Catalyst Pharmaceuticals obtained a breakthrough therapy designation for Firdapse from the FDA for the development of a treatment of patients suffering from LEMS in 2013.[10][11] Catalyst Pharmaceuticals has evaluated Firdapse for safety and efficacy in clinical trials.[12][13][14][15][16][17] Although not approved by the FDA as of 2015, Firdapse is available to eligible LEMS patients through an Expanded Access Program.[18][19][20] Firdapse is also being evaluated as a potential therapy for patients with congenital myasthenic syndromes, for which it has obtained an orphan drug designation from the FDA.[21][22][23]

Catalyst Pharmaceuticals is also developing CPP-115 as a treatment for infantile spasms.[24][25]

Controversies

The development of Firdapse by Catalyst Pharmaceuticals has brought attention to orphan drug policies that grant market exclusivity as an incentive for companies to develop therapies for small numbers of patients.[4][26][27]

References

  1. 1 2 3 4 5 "Catalyst Pharmaceuticals 2014 Annual Report Form (10-K)" (XBRL. Retrieved 2015-03-05). United States Securities and Exchange Commission.
  2. ↑ Catalyst Pharmaceuticals (CPRX) stock information via Wikinvest.
  3. ↑ Catalyst Pharmaceuticals (CPRX) annual SEC balance sheet filing via Wikinvest.
  4. 1 2 3 Mann Jr., Joseph A. “The big gamble: Catalyst Pharmaceuticals of Coral Gables bets on new drug for rare disease”, Miami Herald, 18 July 2015. Retrieved on 26 August 2015.
  5. ↑ Nehamas, Nicholas. “Catalyst Pharmaceuticals reports second-quarter net loss, plans to submit new drug for FDA approval”, “Miami Herald”, 11 August 2015. Retrieved on 26 August 2015.
  6. ↑ "Catalyst Pharmaceuticals Registration Statement Form S-1" (XBRL. Retrieved 2015-09-08). United States Securities and Exchange Commission.
  7. ↑ Hawker D. and R. Silverman. “Synthesis and evaluation of novel heteroaromatic substrates of GABA Aminotransferase”, Bioorganic & Medicinal Chemistry, 1 October 2012. Retrieved on 8 September 2015.
  8. ↑ Brian Bandell. “Catalyst Pharmaceutical signs licensing deal with Northwestern” “South Florida Business Journal”, 31 August 2009. Retrieved on 8 September 2015.
  9. ↑ “Catalyst Acquires Late-Stage Orphan Drug from BioMarin, “Genetic Engineering and Biotechnology News, 1 November 2012. Retrieved on August 27, 2015.
  10. ↑ Burrill and Co. “First eight months of 2013 boast strong M&A, financing totals”, “Drug Discovery News”, October 2013. Retrieved on 26 August 2015.
  11. ↑ “Firdapse amifampridine regulatory update”, “BioCentury” 17 August 2015. Retrieved on 3 September 2015. (subscription required)
  12. ↑ Manocha, Vrinda. “Catalyst Pharma says autoimmune disorder drug safe for heart”, Reuters, 8 January 2014. Retrieved on 26 August 2015.
  13. ↑ Powers, Marie. “Firdapse cardiac safety study hits endpoint; LEMS data next” BioWorld, 9 January, 2014. Retrieved on 26 August 2015. (subscription required)
  14. ↑ BRIEF-Catalyst Pharmaceuticals announces positive top-line PHASE 3 data from Firdapse clinical trial, Reuters, 29 September 2014. Retrieved on 26 August 2015.
  15. ↑ “Catalyst's Firdapse meets in LEMS Phase III” BioCentury, 29 September 2014. Retrieved on 26 August 2015. (subscription required)
  16. ↑ Oh, Shin et al. “Amifampridine phosphate (FirdapseTM) is safe and effective in a pivotal Phase 3 trial in LEMS patients“, “Neurology” April 24, 2015. Retrieved on 27 August 2015.
  17. ↑ “LEMS: Firdapse Study Shows Encouraging Results” Muscular Dystrophy Association 29 September 2014. Retrieved on 26 August 2015.
  18. ↑ Radke, James. “Catalyst Using the Expanded Access Program to Conduct Phase IV Study with LEMS Patients”, Rare Disease Report, 30 October 2014. Retrieved on 25 August 2015.
  19. ↑ Levinsohn, Ben. “Five Biotech Stocks for the Next Six Months”, “Barron’s Stocks to Watch blog” 17 June 2015. Retrieved on 27 August 2015.
  20. ↑ Young, Donna. "Catalyst steps up to provide LEMS patients expanded Firdapse access", “SCRIP Intelligence”, 24 April 2014. Retrieved on 28 September 2014. (subscription required)
  21. ↑ “Orphan Drug Designations and Approvals”, US Food and Drug Administration, 3 March 2015. Retrieved on 26 August 2015.
  22. ↑ Ampel, Celia. “Catalyst Pharma stock rises 5% after firm hits FDA milestone”, “South Florida Business Journal”, 5 March 2015. Retrieved on 26 August 2015.
  23. ↑ Mazell, Shawnay et al. “Clinical and Jitter Changes During Oral Amifampridine Phosphate Firdapse Treatment in Congenital Myasthenic Syndrome”, American Association of Neuromuscular & Electrodiagnostic Medicine 2015 Annual Meeting Abstract 169, 1 June 2015. Retrieved on 8 September 2015.
  24. ↑ Briggs SW, et al. “CPP-115, a vigabatrin analogue, decreases spasms in the multiple-hit rat model of infantile spasms”, Epilepsia. January 2014. Retrieved on 8 September 2015.
  25. ↑ “Orphan Drug Designations and Approvals”, US Food and Drug Administration, 15 September 2015. Retrieved on 8 September 2015.
  26. ↑ Drummond, Michael and Towse, Adrian. “Orphan drugs policies: a suitable case for treatment”, “The European Journal of Health Economics”, May 2014. Retrieved on 3 September 2015.
  27. ↑ Lowe, Derek. "Market Exclusivity Is Sometimes Too Much", Seeking Alpha, 27 April 2015. Retrieved on 8 September 2015.
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