Magazine Article | January 1, 2016

Companies To Watch: Catalyst Pharmaceuticals

Source: Life Science Leader

By Wayne Koberstein, Executive Editor, Life Science Leader magazine
Follow Me On Twitter @WayneKoberstein

Reemerging with a licensed drug for a rare neuromuscular disease — and treading carefully to set its price

Catalyst Pharmaceuticals is developing drugs in the neuromuscular disease area, with Firdapse (amifampridine phosphate) as its lead candidate. Firdapse treats Lambert-Eaton Myasthenic Syndrome (LEMS), a rare condition in which the immune system attacks calcium channels in neuronal junctions, causing muscle weakness. Firdapse is a potassium-channel blocker that counters that condition by increasing calciumchannel flow. Catalyst is also developing CPP- 115, a GABA-aminotransferase inhibitor, for the treatment of infantile spasms and Tourette’s disorder.

This month, I depart from one of the chief criteria for Companies to Watch — the subject company will have had little or no press coverage to date — but, I believe, with good reason. As reported extensively in the July 19, 2015, Miami Herald, Catalyst epitomizes what so many companies now face with new, possibly breakthrough or essential drugs they in-license or acquire the rights to develop: Do they price them at a premium and risk limited patient access (and a public uproar), or price them as low as possible to ensure maximum access? I spoke with Catalyst President and CEO Pat McEnany and CSO Steven Miller about the unique factors each company in that situation must consider before making a pricing decision.

Examples for comparison: Gilead did buy its Hep C blockbusters but took on a great deal of risk and expense to develop and market them. Turing merely bought an essential but inexpensive drug for a tiny market and promptly inflated the price by several orders of magnitude. Catalyst has an approach closer to Gilead’s — taking on the risk of developing a drug not approved in the United States for any indication, while carefully contemplating the price should the drug gain FDA approval as hoped in 2016.

After Catalyst’s initial lead candidate, a drug for cocaine and methamphetamine addiction, washed out of clinical trials in 2012, the company happened upon the opportunity to acquire the U.S. rights to Firdapse, for treating LEMS and congenital myasthenic syndromes (CMS), from BioMarin, which was already marketing the product in Europe. But that did not ensure a painless transition; the FDA required Catalyst and BioMarin to conduct 57 nonclinical studies and six clinical studies, including a Phase 3 trial, and requested that Catalyst file a rolling submission of the NDA (new drug application).

Compound pharmacies have made the basic drug, 3, 4 diaminopyridine (3, 4 DAP), available to patients at relatively low cost but in a free-base (possibly less stable and soluble) formulation, and perhaps with uncertain supply and quality. But the company has tested the waters with its LEMS constituents — patients, physicians, and payers — at medical meetings such as the AANEM (American Association of Neuromuscular Electrodiagnostic Medicine) meeting and in discussions with patient associations, including NORD (National Organization for Rare Disorders). McEnany describes some of the feedback:

“There were about 150 neuromuscular physicians who attended our sponsored industry forum at AANEM, and they found it to be very well-prepared and informative regarding LEMS and CMS. Our research with payers and physicians is ongoing. We have had an opportunity to pressure-test several price points without any significant pushback from payers.” At NORD, he says, the company met with LEMS patients and KOLs, a rare gathering for a rare disease. “Interestingly, all of the patients we met were initially misdiagnosed.”

Does McEnany agree that companies making pricing decisions for rare disease or specialty drugs face a trade-off between profits and patient access? “I don’t believe a drug’s price limits access as long as value for that treatment can be demonstrated,” he answers. “This is particularly true for orphan diseases such as LEMS. Our goal has always been to make sure all patients with LEMS and CMS have access to Firdapse.”

How will we know, if and when Catalyst sets a price for Firdapse, that it matches those criteria? This is one company we will have to watch.

Vital Statistics

Employees: 18
Headquarters: Coral Gables, FL



VC (or private rounds)


Other public financings

Lead institutional investors: BioMarin, Baker Bros., Consonance Capital, Federated Global, Broadfin Capital, Franklin Advisers

Research partnership funding

Previously, National Institute on Drug Abuse ~ $10M for cocaine addiction trials

Other partners

BioMarin invested, owns 8.05% in Catalyst from its sale of North American license for Firdapse to treat Lambert-Eaton Myasthenic Syndrome

Acquired worldwide license for CPP-115 from Northwestern University.

Latest Updates

December 2015: Completed FDA NDA filing for Firdapse

Initiated proof-of-concept clinical trial of Firdapse in patients with anti-MuSK antibody myasthenia gravis

Data from safety/tolerability study for CPP-115