CATALYST PHARMACEUTICALS, INC.
CPRX evidence brief
What we can confirm
- BioSniper Score:
- 7.9 / 10
- Market Cap:
- $3.85B
- Lead Asset:
- AGAMREE® (Approved)
What it means
What to watch
Prepared research question
What matters most for CPRX—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · CPRX
Catalyst Pharmaceuticals, Inc. (CPRX) is a commercial-stage biopharmaceutical company focused on rare diseases, with approved products FIRDAPSE and AGAMREE, and a strong cash position of $755.9M as of 2026-03-31 [sec].
Key risk: Product concentration
BioSniper Score
Based on 5 of 6 signals
Financial data as of Mar 31, 2026.
Tracking 3 pipeline candidates · 5 clinical trials · 2 catalysts · 4 FDA decisions for CATALYST PHARMACEUTICALS, INC..
Catalyst Pharmaceuticals, Inc. (CPRX) is a commercial-stage biopharmaceutical company focused on rare diseases, with approved products FIRDAPSE and AGAMREE, and a strong cash position of $755.9M as of 2026-03-31 [sec].
- Cash & equivalents of $755.9M, total revenue of $149.4M, net income of $63.7M, and shareholders' equity of $1,012.7M as of 2026-03-31 [sec].
- FIRDAPSE (amifampridine) is approved for Lambert-Eaton myasthenic syndrome; a Phase 2 trial in spinal muscular atrophy showed statistically significant improvement in HFMSE (mean difference 0.792; p=0.0083) [catalyst].
- AGAMREE (vamorolone) is approved for Duchenne muscular dystrophy; a Phase 1 study topline results were announced on June 30, 2026 [sec · filed 2026-06-30].
- Executive compensation goals for 2025 include total revenues of at least $573.5M, with FIRDAPSE net revenues of at least $362.9M and AGAMREE net product revenues of at least $122.1M [sec · filed 2026-04-30].
- The company has a patent expiry for AMIFAMPRIDINE PHOSPHATE on 2037-02-25 and for VAMOROLONE on 2040-07-16 [catalyst].
What works
- Strong financial position with $755.9M cash and $1,012.7M shareholders' equity as of 2026-03-31, providing runway for operations and potential acquisitions [sec].
- Two approved rare-disease products (FIRDAPSE and AGAMREE) generating revenue, with a 2025 revenue target of at least $573.5M [sec · filed 2026-04-30].
- Positive Phase 2 data for amifampridine in spinal muscular atrophy, showing statistically significant improvement in motor function [catalyst].
- Long patent protection extending to 2037 and 2040 for key products, providing extended market exclusivity [catalyst].
What to weigh
- Reliance on a small number of products (FIRDAPSE and AGAMREE) for the majority of revenue, creating concentration risk [sec · filed 2026-04-30].
- Forward-looking statements caution about risks including changes in business, legal proceedings, and stock price effects related to transactions [sec · filed 2026-06-23].
- Executive compensation targets imply significant revenue growth expectations, which may not be achieved [sec · filed 2026-04-30].
- Patent expirations in 2037 and 2040 could eventually expose products to generic competition [catalyst].
From the filings
Quoted directly from source documents.
Phase 2 amifampridine SMA trial result
“Amifampridine treatment led to a statistically significant improvement in HFMSE (mean difference 0.792; 95% CI from 0.22 to 1.37; p = 0.0083), compared to placebo”
Catalysts · November 30, 2022
2025 revenue targets for executive bonuses
“Achievement of total revenues in 2025 of at least $573.5 million, including FIRDAPSE®net revenues for 2025 of at least $362.9 million and AGAMREE®net product revenues for 2025 of at least $122.1 million”
SEC filings · April 30, 2026
Catalyst timeline
- 2037-02-25 · Patent_Expiry · AMIFAMPRIDINE PHOSPHATE — Patent expiry for NDA208078; potential generic competition may impact FIRDAPSE revenue.
- 2040-07-16 · Patent_Expiry · VAMOROLONE — Patent expiry for NDA215239; potential generic competition may impact AGAMREE revenue.
Risks & what to watch (3)›
Key risks
- Product concentration — Revenue heavily dependent on FIRDAPSE and AGAMREE; any disruption could significantly impact financials [sec · filed 2026-04-30].
- Patent expiration — AMIFAMPRIDINE PHOSPHATE patent expires 2037-02-25; VAMOROLONE patent expires 2040-07-16, opening door to generics [catalyst].
- Transaction-related risks — Forward-looking statements note risks from legal proceedings, regulatory actions, and stock price effects related to transactions [sec · filed 2026-06-23].
What to watch
- Achievement of 2025 revenue targets for FIRDAPSE ($362.9M) and AGAMREE ($122.1M) as indicators of commercial execution [sec · filed 2026-04-30].
- Progress of the Phase 2 trial of Visugroma (started 2025-08-01) and any subsequent readouts [catalyst].
- Potential acquisition of a 'significant company or de-risked late-stage orphan asset' as part of 2025 corporate objectives [sec · filed 2026-04-30].
- Patent expiry timelines for AMIFAMPRIDINE PHOSPHATE (2037) and VAMOROLONE (2040) and any generic entry preparations [catalyst].
Sources: Catalysts · SEC filings · Generated October 4, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 5 of 6 signals · derived from public data
Profitable (positive operating cash flow)
D/E 0.13 · 4% dilution
Lead: Approved
3 candidates
2 upcoming, next in ~3787d
No insider transactions in the last 180 days
Catalysts
Pipeline
Regulatory
Financials
Short Interest
as of May 20, 2026- % of float
- 7.9%
- Days to cover
- 4.3
- Shares short
- 7,838,703
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
CPRX short interestGovernance
Frequently asked questions
What is the AI investment thesis for CATALYST PHARMACEUTICALS, INC.?
Catalyst Pharmaceuticals, Inc. (CPRX) is a commercial-stage biopharmaceutical company focused on rare diseases, with approved products FIRDAPSE and AGAMREE, and a strong cash position of $755.9M as of 2026-03-31 [sec].
What is CATALYST PHARMACEUTICALS, INC.'s lead drug candidate?
CATALYST PHARMACEUTICALS, INC.'s most advanced tracked candidate is AGAMREE® (Approved) for treatment of Duchenne muscular dystrophy (DMD).
What is CATALYST PHARMACEUTICALS, INC.'s next catalyst?
CATALYST PHARMACEUTICALS, INC.'s next tracked catalyst is a Patent_Expiry event — "Patent_Expiry: NDA208078" — expected February 25, 2037.
Who is the CEO of CATALYST PHARMACEUTICALS, INC.?
Mr. Richard John Daly M.B.A. is the chief executive officer of CATALYST PHARMACEUTICALS, INC. (CPRX).
Where is CATALYST PHARMACEUTICALS, INC. headquartered?
CATALYST PHARMACEUTICALS, INC. (CPRX) is headquartered in Coral Gables, FL, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.