May 17, 2016
By Alex Keown, BioSpace.com Breaking News Staff
CORAL GABLES, Fla. -- Catalyst Pharmaceuticals is slashing its workforce by about 30 percent in an effort to conserve capital and save its investigational drug program for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS).
In February, the U.S. Food and Drug Administration issued Catalyst a “Refusal to File” letter for its New Drug Application for Firdapse. The FDA found that Catalyst’s application, which was submitted in December 2015, was not sufficiently complete and requested additional supporting information. The letter did not provide comment on the acceptability of the submitted clinical data, nor was any judgment made on the efficacy or safety of Firdapse. Catalyst previously received Orphan Drug Designation for Firdapse to treat LEMS and CMS, and Breakthrough Therapy Designation for Firdapse to treat LEMS. In April, the FDA said Catalyst must run an additional Phase III trial, as well as additional toxicology studies for Firdapse.
Catalyst’s shares are down more than 4 percent this morning, trading at 58 cents per share.
Catalyst Pharmaceuticals markets a LEMS treatment in Europe that it licensed from BioMarin Pharmaceuticals . Catalyst has eyed the U.S. market for some time.
Catalyst said its workforce reduction will take effect immediately and will primarily impact the company’s commercial team, Catalyst said in a statement issued this morning. With the reduction, Catalyst said it believes that barring any other unforeseen issue, the company will have enough capital to complete development and refile an NDA for Firdapse for both LEMS and CMS.
“I would like to express my sincere appreciation to those employees affected by this difficult but necessary action. This is a loss to our Catalyst family of talented and dedicated individuals who have worked with integrity and passion towards improving the lives of people living with rare diseases,” Patrick J. McEnany, chairman and chief executive officer of Catalyst said in a statement. “The decision to reduce the Company’s workforce has been extremely difficult, but we believe that it is a necessary step to better align our resources and enable us to achieve our goal of bringing Firdapse to market for patients with LEMS and CMS.”
Lambert-Eaton myasthenic syndrome is an autoimmune disease where the attack occurs at the connection between nerve and muscle and interferes with the ability of nerve cells to send signals to muscle cells, according to information on the Muscular Dystrophy Association website. Patients with LEMS typically deal with fatigue, muscle pain and stiffness, with weakness in the lower body. More than half of LEMS cases are the result of underlying illnesses, particularly cancers. However, there are a large percentage of patients diagnosed that have no root cause for the disease.
Some LEMS patients in the U.S. have access to a drug provided by tiny Jacobus Pharmaceuticals, a family-owned company in New Jersey. For years Jacobus has given the drug away to about 200 patients, but if Catalyst receives approval, that particular market for Jacobus will likely be shut down. Jacobus has given the LEMS treatment away as part of a belief in being responsible to those with unmet medical needs. The company provided the drug, 3-4 Dap, for free through a federal program called compassionate use. That program allowed Jacobus to sidestep some of the regulatory hurdles associated with marketing a drug.
Catalyst believes its LEMS treatment will be able to reach a greater population than the 200 patients that Jacbobus’ treatment helps. In a previous BioSpace story, Catalyst said its LEMS drug could reach about 3,000 patients.
