CPRX

• 2025 was a strong year with record revenues, driven by execution of strategy to maximize commercial portfolio value and personalized support for rare disease patients. • Ferdaps, the only evidence-based FDA-approved therapy for Lambert-Eaton myasthenic syndrome (LEMS), had growth in idiopathic LEMS and cancer-associated LEMS markets. • Agamri, a differentiated corticosteroid medication for Duchenne muscular dystrophy (DMD), had strong growth with expansion of center of excellence penetration and ongoing summit study. • Business development engine conducted over 100 assessments in 2025, with about 90% inbound, underscoring reputation as a proven leader. • Commercial team executed well for Ferdaps in lead generation, conversion efficiency, and patient retention; for Agamri in expanding center penetration and reducing discontinuations; for Ficompa despite generic competition.

• 2026 total revenue is guided to be between $615 million and $645 million. • Ferdaps guidance is $435 to $450 million, reflecting a 21.4% to 25.6% increase. • Agamri guidance is $140 to $150 million, forecasting a 19.6% to 28.1% growth. • Ficompa is forecasted to be $40 million to $45 million in 2026, with expected revenue erosion from generic competition but still meaningful. • 2025 total revenues were $589 million, up 19.8% year over year, and net income before income taxes increased significantly.

For the full year 2025, total revenues grew by 19.8% year over year to $589 million. Full year net product revenue reached $588.8 million, a 20.3% increase over 2024. Ferdaps guidance for 2026 is $435 to $450 million, representing a 21.4% to 25.6% increase. Agamri guidance is $140 to $150 million, forecasting a 19.6% to 28.1% growth. Ficompa is forecasted to be $40 million to $45 million in 2026. In 2025, Ferdaps revenue was $358.4 million, an increase of 17% for the full year. Agamri delivered 154.3% year-over-year growth with 2025 revenues of $117.1 million. Ficompa had net revenue of $113.3 million in 2025 despite generic competition.

• Risks associated with forward-looking statements, as actual results may vary from expectations. • Uncertainties in market opportunities for product growth. • Impact of generic competition on Ficompa's revenue. • Regulatory uncertainties in legal suits related to Ferdaps' IP. • Risks in business development initiatives such as aligning with potential partners and navigating regulatory environments.

Q: Speak to the drivers that underpin the growth implied within your 2026 guidance for Ferdaps and Agamri.

A: For Ferdaps, drivers include over 600 patient leads in LEMS diagnostic journey, 21% growth in BGCC testing, and pharmacy program reducing patient discontinuations. For Agamri, drivers include broad adoption in top DMD centers, reduced discontinuations, and deepening market penetration through initiatives like the summit study.

Q: How does the summit open label expansion affect the forecast for Agamri going forward?

A: The summit trial enrolling patients will generate data to evaluate long-term potential benefits, with positive data from Santera's studies supporting potential growth.

Q: Color on VGCC testing and its impact on Ferdaps sales.

A: VGCC testing is up 21% year over year, with significant opportunity remaining in oncologists' ordering of tests, which is an indicator of potential conversion to Ferdaps therapy.

Q: How does the phase 1A readout on establishing translational dosing impact Agamri's plans?

A: The phase 1A readout on translational dosing may impact life cycle management and potential additional indications for Agamri, with results influencing decisions on future opportunities.