Catalyst Pharmaceuticals (NASDAQ:CPRX) highlighted continued growth in its rare disease portfolio and outlined efforts to expand patient identification and penetration for its lead product during a recent discussion with Barclays life sciences analyst Glen Santangelo. Chief Executive Officer Rich Daly also reviewed early commercial progress for Agamree in Duchenne muscular dystrophy (DMD), provided an update on Fycompa following generic competition, and discussed capital allocation priorities, including business development and share repurchases.
Portfolio overview: Firdapse, Agamree, and Fycompa
Daly described Catalyst as a rare disease company focused on “ultra rare” conditions, with three commercial products at different life cycle stages:
- Firdapse, a muscle-activating drug for Lambert-Eaton myasthenic syndrome (LEMS), on the market for about eight years and described as having grown 15%–20% annually.
- Agamree, a corticosteroid for Duchenne muscular dystrophy, launched in March 2024 and positioned as a differentiated steroid with what Daly called an “interesting clinical profile.”
- Fycompa, an epilepsy drug acquired from Eisai, which has faced generic competition beginning around mid-year and is declining in revenue as expected, according to management.
2025 momentum and operating initiatives
In parallel, Catalyst increased focus on cancer-associated LEMS. Daly said the company worked with a national lab to add a voltage-gated calcium channel (VGCC) test to broader testing panels and collaborated with the National Comprehensive Cancer Network (NCCN) to include Firdapse in guidelines for cancer-associated LEMS. Catalyst is also engaging group purchasing organizations (GPOs), which Daly said control approximately 80%–90% of community-based oncology practices and tend to follow NCCN guidance closely.
For Agamree, Daly said the launch “went really well” and that patients were coming from both generic prednisone and Emflaza (or generic Emflaza), with a smaller portion coming from therapy-naïve patients. He also noted that issues surrounding the gene therapy Elevidys, particularly the need for immunosuppressive dosing after a label expansion mid-year, created delays in switching some patients from prednisone or Emflaza to Agamree, though he said the brand “weathered” that period well.
Firdapse: penetration opportunity and IP update
Looking ahead, Santangelo cited Catalyst’s 2026 Firdapse revenue guidance of $435 million to $450 million, implying 21%–26% growth. Daly argued that penetration remains relatively low in both key segments. He said idiopathic LEMS is often misdiagnosed as generalized myasthenia gravis and that the average diagnostic journey for a LEMS patient is four to six years. In oncology-associated LEMS, he said LEMS can be an early indicator of small cell lung cancer, and he emphasized the importance of improving identification through testing and guideline-driven care pathways.
Daly provided the company’s view of the diagnosis pool, saying that in a typical year there may be about 150 idiopathic patients who could be diagnosed and about 900 on the cancer-associated side. As of the discussion, he said Firdapse penetration was about 30% in idiopathic LEMS and less than 10% in cancer-associated LEMS.
On intellectual property, Daly said Catalyst has three first filers and has settled with two of them—Lupin and Teva. He said the Teva settlement, reached in January of the prior year, sets a date of February 2035, and he characterized Teva as a highly sophisticated generic manufacturer. The remaining litigant is Hetero, with a trial that had been scheduled to start March 23, though Daly said the timing could be affected by potential court scheduling conflicts involving a criminal case. He added that Catalyst remains open to discussions but is prepared to defend its IP at trial.
Agamree: commercial execution and clinical studies
Santangelo noted Agamree’s 68% growth in the fourth quarter and asked about the durability of trends into 2026, given revenue guidance of $140 million to $150 million. Daly emphasized the market’s concentration: about 100 centers treat more than 90% of DMD patients, and about 45 centers account for roughly 80% of treatment. He said Catalyst has 12 regional account managers covering these centers, and that the commercial team has achieved use in 100% of the top 45 centers and roughly 90% of the top 100.
Daly reiterated the foundational role of steroids in DMD, stating that early steroid use can extend ambulation by roughly two to two-and-a-half years. He also detailed key long-term steroid concerns, including behavioral issues, stunted growth, weaker bones and fractures, cataracts, and cardiovascular disease. He referenced new data presented at the Muscular Dystrophy Association meeting from a GUARDIAN study versus standard-of-care steroids, describing potentially favorable comparisons for Agamree on fractures, height, and cataracts, while noting the company cannot promote data not included in the label.
He also discussed Catalyst’s ongoing SUMMIT study, an open-label trial planned to enroll about 250 patients and compare Agamree with standard of care across endpoints including behavior, bone health, stature, fractures, cataracts, and cardiovascular measures. Daly said the GUARDIAN findings increased management’s confidence in the SUMMIT effort.
On payer dynamics, Daly said both Firdapse and Agamree have about 90% approval coverage and that the company is not seeing significant payer pushback.
Fycompa, capital allocation, and 2026 outlook
For Fycompa, Daly said generic entry has led to expected revenue declines, with 2026 guidance of $40 million to $45 million versus $115 million previously referenced by Santangelo. Daly said promotion has been removed, leaving mostly website and social media activity, while noting epilepsy can be “sticky” with patients staying on therapy longer than in some categories.
Turning to capital allocation, Daly said the company has more than $700 million in cash, no funded debt, and continues to generate cash each quarter. He said Catalyst wants to “put the money to work” via acquisitions and is now willing to consider assets that are closer to market even if in Phase 3. Catalyst’s target profile is a differentiated rare disease product, with a peak opportunity around $500 million, along with alignment on product vision when licensing. Daly said a $200 million share repurchase authorization, running through December 2026, does not impede business development and that the company could also use debt if needed.
Asked about overall 2026 revenue guidance of $615 million to $645 million (4.5%–9.5% growth), Daly attributed the slower top-line growth rate primarily to Fycompa’s decline, while reiterating expectations for continued Firdapse growth and improved Agamree momentum as the company moves past prior-year “choppiness” tied to the gene therapy landscape. He said the company expects operating expenses to be relatively flat and flagged a planned focus on cancer-associated LEMS in the budget, as well as R&D spending flexibility tied to the SUMMIT study and potential lifecycle management opportunities for Agamree in other rare disease settings where steroids are used.
About Catalyst Pharmaceuticals (NASDAQ:CPRX)
Catalyst Pharmaceuticals, Inc is a biopharmaceutical company focused on the development and commercialization of therapies for patients living with rare neuromuscular, neurological and metabolic diseases. The company’s mission centers on delivering safe and effective treatments that address unmet medical needs in small patient populations.
The company’s lead product is Firdapse® (amifampridine phosphate), a treatment approved by the U.S. Food and Drug Administration for adults with Lambert-Eaton Myasthenic Syndrome (LEMS).
