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Catalyst Enrolls First Patient in Phase III Firdapse Study

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Catalyst Pharmaceuticals, Inc. (CPRX - Free Report) , enroled the first patient in  its phase III clinical trial (MSK-002) to evaluate the efficacy and safety of Firdapse (amifampridine phosphate) in patients with MuSK antibody positive Myasthenia Gravis (MuSK-MG).

In August 2017, Catalyst announced that it had reached an agreement with the FDA under a Special Protocol Assessment for the protocol design, clinical endpoints, and statistical analysis approach in the phase III trial. Firdapse received Orphan Drug status in the United States for the treatment myasthenia gravis (“MG”).

Shares of the company increased 53% over a year compared with the industry’s gain of 7.9%.


Firdapse is being developed for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (“LEMS”). In Apr 2018, the company filed a new drug application (NDA) for Firdapse.

Firdapse is currently approved in the EU for the symptomatic treatment of LEMS in adults. However, the drug is not yet approved in the United States.  In January 2017, it held a Type C meeting with the FDA wherein Catalyst was informed, based on the briefing documents, that the company’s proposed NDA should be sufficient for resubmission. In November 2017, the company announced positive top-line results from its second phase III study of Firdapse in patients with LEMS.

Catalyst obtained in-licensed rights for Firdapse from BioMarin Pharmaceutical (BMRN - Free Report) in 2012 for the development and commercialization of the product in the United States. Also, Firdapse enjoys Orphan Drug and Breakthrough Therapy status in the United States for the treatment of LEMS. 

Therefore, approval of Firdapse in these indications will be a huge boost for the company given its commercial potential in the target markets.


Zacks Rank and Stocks to Consider

Catalyst carries a Zacks Rank #3 (Hold).

A few better-ranked stocks from the same space worth considering are Ligand Pharmaceuticals (LGND - Free Report) and Protagonist Therapeutics (PTGX - Free Report) . Both of them sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Ligand’s earnings per share estimates have moved up $3.78 to $4.40 from $4.75 to $5.32 for 2018 and 2019, respectively, over the last 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with an average beat of 24.88%. The company’s shares have rallied 60.1% over a year.

Protagonist’s loss estimates narrowed from $1.30 to 66 cents for 2018 and from $1.99 to $1.26 for 2019, over the last 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with an average beat of 24.95%.

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