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Catalyst Pharma (CPRX) Focused on U.S. Approval for Firdapse

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We issued an updated research report on Catalyst Pharmaceuticals, Inc. (CPRX - Free Report) on Mar 22.

Catalyst’s share price has increased significantly by 60% year to date, compared with the Zacks classified Medical Drugs industry's gain of 2.1%.

Catalyst has in-licensed rights to Firdapse from BioMarin Pharmaceutical Inc. (BMRN - Free Report) for the development and commercialization of the product in the U.S. Currently, Firdapse has been approved in the EU for the symptomatic treatment of LEMS in adults. It also enjoys Orphan Drug and Breakthrough Therapy status in the U.S. for the treatment of LEMS. However, Firdapse is not yet approved in the U.S. In 2016, the company had received a refusal to file letter from the FDA in connection with the NDA for Firdapse. The agency had determined that the company’s NDA was insufficient after a preliminary review. Consequently, Catalyst is required to conduct additional studies as per the agency’s requirements.

The company is conducting a second phase III study (LMS-003) on Firdapse for LEMS (LMS-003) in order to gain approval in the U.S. It reached an agreement with the FDA under a Special Protocol Assessment (SPA) for protocol design, clinical endpoints and statistical analysis in Oct 2016. Top-line data from the study is anticipated in the second half of 2017. A positive outcome from the study is expected to allow the company to resubmit the new drug application (NDA) for Firdapse for the treatment of LEMS in the second half of 2017. Moving ahead, we believe that this approval will give a huge boost to the company.

Furthermore, Catalyst is working on developing Firdapse for additional indications. It is conducting an investigator-sponsored phase II/III study on Firdapse for the symptomatic treatment of MuSK-antibody positive MG.  In Mar 2017, the company reported top-line data from the phase II/III trial that showed statistically significant results. According to the investigators, this showed a large clinical benefit to patients. Catalyst’s efforts to develop Firdapse appear encouraging given the significant commercial potential in the target markets.

In addition, the company has an early-stage candidate, CPP-115 (infantile spasms and other neurological indications), in its pipeline. Further, it is also working on a generic version of Sabril (vigabatrin), which is marketed by Lundbeck Inc.in the U.S. for the treatment of infantile spasms and complex partial seizures.

However, the number of candidates in Catalyst’s pipeline is limited. In fact, the company is highly dependent on Firdapse for growth and therefore its U.S. approval is important for the company’s success. Any further delays might prove be detrimental to the company’s growth prospects.

Also, Catalyst is looking to develop CPP-115 for the treatment of epilepsy. We note that the market for epilepsy treatments is highly competitive and crowded, with products like Pfizer Inc.’s (PFE - Free Report) Neurontin and Lyrica, Johnson & Johnson’s (JNJ - Free Report) Topamax, UCB’s Keppra, AbbVie’s Depakote and Novartis’ Trileptal.

 

Zacks Rank & Stocks to Consider

Catalyst currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here

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