Catalyst Pharmaceuticals Is a Rare Bird in the Gables 

 Success in a Unique Niche

Working for decades as an investment banker in Coral Gables, Patrick J. McEnany never expected to start his own pharmaceutical company listed on Wall Street. But McEnany now leads Gables-based Catalyst Pharmaceuticals, a Nasdaq-traded venture that recently earned U.S. approval for a drug to treat a rare nerve-muscle disease. Catalyst now has sales topping $100 million a year and is licensing the drug in Japan. “Our strategy is to grow globally with this drug and to bring on other products to treat rare diseases,” says the CEO and longtime Gables resident. 

McEnany’s path to entrepreneurship began accidentally in 1990. The investment bank where he worked had problems with a company it had backed, Miami Lakes-based Royce Laboratories. McEnany stepped in to run the troubled generic-drug maker. In seven years, he built up Royce’s sales to more than $100 million annually, then arranged the company’s sale. 

By then, McEnany knew the pharmaceutical industry well, so he looked for new projects, and saw opportunities in treatments for rare diseases that affect small numbers of patients. In 2002, he and a partner founded Catalyst. First, they sought FDA (Food and Drug Administration) approval for a drug to treat patients addicted to cocaine and other stimulants. But clinical trials flubbed. 

Next, they bought rights from a California firm, Biomarin, for a drug with potential to treat LEMS (Lambert-Eaton Myasthenic Syndrome), a disorder that afflicts some 3,000 people nationwide, prompting tumors and fatigue, often misdiagnosed as lupus or multiple sclerosis. 

In 2006, Catalyst sold stock to help fund clinical studies, legal fees and other expenses for regulatory approvals. In 2018, the FDA finally authorized the drug, Firdapse, to treat LEMS in adults. In 2021, Catalyst licensed Firdapse in Japan. Now, it’s working to sign other licensing deals and expand its drug portfolio. Prospects are so bright that Andrew Fein, a senior biotech analyst at H.C. Wain- wright, suggests a “buy” on Catalyst stock. He says, “every step of the way they’ve delivered on what they said they would.” 

For LEMS sufferers, Catalyst’s drug offers hope. But for U.S. insurers, it’s pricey: nearly $400,000 per year, per patient. Catalyst offers programs to give the drug free to patients with little or no insurance. Otherwise, insurers cover costs, with patients paying just $25 a year out-of-pocket, says McEnany. 

High prices for drugs for rare diseases – up to $2 million per year per patient – have sparked debate, as Washington seeks to curb rising health care costs. But McEnany sees a “systemic issue” in the growing costs for universities, labs, lawyers and others in the tough process for FDA approvals. “I share your concern as a consumer,” he says. “But patients that have these rare diseases want a treatment, and that costs money.” He says Biomarin and Catalyst invested more than $100 million and over a decade to secure FDA approval for Firdapse. That authorization also faced a costly court challenge from a rival drug maker, which Catalyst won. 

Based in a city little known for pharmaceuticals, McEnany faces hurdles recruiting talent. Catalyst employs some 35 people at 355 Alhambra Circle, plus 50 more nationwide. Fortunately, hiring is becoming easier in today’s Zoom era. Catalyst now allows even senior staff to work from home in New Jersey or other pharma hubs, and visit Coral Gables as needed.