Catalyst Pharmaceuticals Announces Publication of Clinical Data from the Investigator-Sponsored Phase IIb Study Evaluating Firdapse® for the Treatment of MuSK Antibody Positive Myasthenia Gravis
The positive topline results from this trial were previously announced, but the full results of safety, efficacy and other clinical data are now available online. The MSK-001 trial was a randomized, double-blind, placebo-controlled, double crossover design. The co-primary endpoints were statistically met (QMG score: p=0.0003 and MG-Activities of Daily Living Profile Score: p=0.0006) as well as the secondary endpoints. The study provided evidence that amifampridine phosphate was safe and effective in treating MuSK-MG patients. The study was conducted under the supervision of
These data are more fully described in the manuscript entitled, “Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study”, which was recently published online in SAGE Open Medicine. The article can be accessed at: https://journals.sagepub.com/doi/pdf/10.1177/2050312118819013 .
“The results of the pilot study are very encouraging, such that our multi-center pivotal Phase 3 trial evaluating Firdapse for the treatment of MuSK-MG is currently underway. Our pivotal trial is being conducted under an FDA Special Protocol Assessment (SPA),” said
About MuSK antibody positive Myasthenia Gravis (MG)
About 15% of MG patients test negative for the acetylcholine receptor antibody. These patients have seronegative (SN) MG. Approximately 40-50% of these patients with SNMG (equating to an estimate of approximately 4,500 patients in
Firdapse is currently being evaluated in clinical trials for the treatment of CMS, MuSK-MG and SMA type 3 and has received Orphan Drug Designation from the
About Firdapse® (amifampridine)
Firdapse® (amifampridine) 10 mg tablets is an oral, nonspecific, voltage-dependent, potassium (K+) channel blocker that causes depolarization of the presynaptic membrane and slows or inhibits repolarization. This action results in the opening of slow voltage-dependent calcium (Ca2+) channels, allowing for a subsequent influx of Ca2+. In turn, it induces the exocytosis of synaptic vesicles containing Acetylcholine (ACh) to release more ACh into the synaptic cleft, enhancing neuromuscular transmission, and providing for improved muscle function. Firdapse is approved in the U.S. and the
Important Safety Information
FIRDAPSE is contraindicated in patients with:
- A history of seizures
- Hypersensitivity to amifampridine phosphate or another aminopyridine
WARNINGS AND PRECAUTIONS
Seizures: FIRDAPSE can cause seizures. Consider discontinuation or dose reduction of FIRDAPSE in patients who have a seizure while on treatment. FIRDAPSE is contraindicated in patients with a history of seizures.
Hypersensitivity: If a hypersensitivity reaction such as anaphylaxis occurs, FIRDAPSE should be discontinued and appropriate therapy initiated.
The most common (> 10%) adverse reactions are: paresthesia, upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension, and muscle spasms.
To report SUSPECTED ADVERSE REACTIONS, contact
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Catalyst will be successful in commercializing Firdapse (ii) whether, even if Catalyst is successful in commercializing Firdapse, Catalyst will become profitable, (iii) whether Catalyst’s ongoing clinical trials evaluating Firdapse for the treatment of CMS, MuSK-MG and SMA type 3 will be successful; (iv) whether Firdapse will ever be approved for the treatment of CMS, MuSK-MG, SMA type 3, or any other disease, and (v) those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2017 and its other filings with the
Brian KorbSolebury Trout (646) 378-2923 firstname.lastname@example.org Company Contact Patrick J. McEnany Catalyst PharmaceuticalsChief Executive Officer (305) 420-3200 email@example.com Media Contact David Schull Russo Partners(212) 845-4271 firstname.lastname@example.org
Source: Catalyst Pharmaceuticals, Inc.