In The Spotlight: Catalyst Pharmaceuticals

(RTTNews) - Shares of Catalyst Pharmaceuticals Inc. (CPRX) are up an impressive 179% year-to-date while the iShares NASDAQ Biotechnology Index ETF (IBB) has gained just 17% during the same period.

Catalyst Pharma is focused on developing drugs for rare debilitating neuromuscular and neurological diseases.

The Company's lead drug candidate is Firdapse, which is under phase III testing in patients with Lambert-Eaton Myasthenic Syndrome.

Catalyst licensed North American Rights to Firdapse from BioMarin Pharmaceutical Inc. (BMRN) in October 2012. In return, BioMarin made a $5 million investment in Catalyst.

Lambert-Eaton Myasthenic Syndrome, or LEMS in short, is an autoimmune disorder, in which the part of the neuromuscular junction is damaged due to attack from antibodies. This condition is characterized by muscle weakness, a tingling sensation in the affected areas, fatigue, and dry mouth. (Source: NIH).

Based on positive results from a phase III trial of Firdapse in 38 patients, the Company had sought approval of the drug candidate in the U.S. in 2015. But after a preliminary review, the FDA determined that the NDA for Firdapse was not sufficiently complete and even refused to accept the application. The Company was asked by the regulatory agency to submit positive results from an additional adequate and well-controlled study in patients with LEMS.

Accordingly, Catalyst Pharma initiated the second phase III trial of Firdapse in LEMS last December. The study, which is being conducted under FDA's Special Protocol Assessment, reached target enrollment of 26 patients last month. Top-line results from this trial of Firdapse in LEMS are expected in early December 2017.

As required by the FDA, the Company has also completed preclinical abuse liability studies for "Assessment of Abuse Potential of Drugs".

If all goes well as planned, the Company expects to resubmit the NDA for Firdapse in the first quarter of 2018.

In the European Union, Firdapse received the regulatory nod in December 2009, and has been marketed there by BioMarin since April 2010. In Europe, the drug is reportedly set to cost $60,000 a patient /year.

Firdapse fetched revenue of $14.05 million in the first nine months of 2017, and annual revenue of $18 million for BioMarin in 2016.

A privately held company Jacobus Pharmaceutical is providing 3,4-Diaminopyridine base (3,4-DAP), the same ingredient as in Catalyst's Firdapse, for LEMS patients for free for over 20 years through the FDA's Expanded Access (compassionate use) program. However, this medication is not approved in the U.S.

Catalyst Pharma is also exploring Firdapse in the indications of MuSK antibody positive Myasthenia Gravis and Congenital Myasthenic Syndromes.

Myasthenia gravis is a neuromuscular autoimmune disorder that affects voluntary muscle groups, say muscles of the face, hands, eyes, arms and legs. Some of the symptoms of this rare debilitating neurological disorder are a drooping eyelid, blurred or double vision, difficulty in chewing, swallowing and talking, weakness in the arms and legs, fatigue and shortness of breath. MuSK antibody positive myasthenia gravis, or MuSK-MG, is a particularly severe form of myasthenia gravis that affects about 3,000 to 4,800 patients in the U.S.

In March of this year, the Company reported positive top-line results from an investigator-sponsored trial of Firdapse in MuSK-MG, demonstrating significant symptomatic relief for the patients.

The Company expects to commence enrollment in a phase III trial of Firdapse in patients with MuSK antibody positive myasthenia gravis in the first quarter of 2018. The trial will enroll 60 subjects diagnosed with MuSK-MG.

Congenital myasthenic syndromes (CMS) are a diverse group of disorders that are also characterized by muscle weakness.

A phase III trial of Firdapse for the treatment of Congenital Myasthenic Syndromes (CMS), dubbed CMS-001, is underway, and top-line results from this trial are expected in the first half of 2018.

Financial Data:

For the third quarter ended September 30, 2017, Catalyst's net loss was $4.18 million or $0.05 per share, compared to a net loss of $3.95 million or $0.05 per share for the same period in 2016. No revenues have been generated till date.

The Company ended September 30, 2017 with zero debt and cash of $33.9 million.

CPRX closed Friday's (Nov.17) trading at $2.93, up 0.69%.

Given the fact that a couple of milestones are set to unfold through the coming months, Catalyst Pharma is a stock is worth a look.