Catalyst Pharmaceuticals Inc

Angelini Pharma has completed its acquisition of Catalyst Pharmaceuticals, integrating Catalyst’s portfolio and commercial infrastructure into its operations. The deal values Catalyst at approximately USD 4.1 billion, or USD 31.50 per share in cash, and gives Angelini Pharma a direct presence in the U.S. market while strengthening its global leadership in brain health and rare diseases. Funds managed by Blackstone are investing 1 billion euros in preferred equity, and CDP Equity has approved a roughly 1 billion euro capital increase to acquire a 23.5 percent stake in Angelini Pharma’s common equity. Catalyst’s common stock has ceased trading on the Nasdaq Global Market following the transaction. The financing was supported by a pool of 14 financial institutions led by BNP Paribas as sole global coordinator and underwriter.

Catalyst Pharmaceuticals reported positive top-line results from a phase I study of Agamree in healthy adults, suggesting the drug may deliver corticosteroid benefits without clinically meaningful immunosuppression at approved doses, potentially opening the door to additional chronic inflammatory rare disease indications. The study compared Agamree with PTC Therapeutics' Emflaza and found comparable glucocorticoid activity but less pronounced immunosuppressive biomarker effects with Agamree. Clinically relevant immunosuppression was observed only at the highest tested dose of 40 mg/kg, which exceeds currently approved dosing levels. The findings strengthen Catalyst's efforts to broaden Agamree's label beyond its current approval for Duchenne muscular dystrophy. The company is also set to be acquired by Angelini Pharma in a deal valued at approximately $4.1 billion, expected to close in the third quarter of 2026.

Santhera Pharmaceuticals noted topline results from a two-part Phase 1 clinical study of AGAMREE® conducted by its North American licensing partner, Catalyst Pharmaceuticals. The study suggests AGAMREE® delivers glucocorticoid and anti-inflammatory activity while avoiding significant immunosuppressive effects, supporting its potential use across a broad range of chronic inflammatory rare diseases. In healthy adult volunteers, both AGAMREE® and deflazacort showed expected glucocorticoid receptor activity and comparable cortisol suppression at clinical doses, but AGAMREE® had less pronounced immunosuppressive biomarker effects. Clinically relevant immunosuppressive effects were observed only at the highest dose level, above currently approved dosing for Duchenne muscular dystrophy. Under the 2023 license agreement, Catalyst holds North American commercialization rights for all indications, while Santhera retains a right of first negotiation for new indications outside North America and is eligible for sales-based milestones and royalties.

Catalyst Pharmaceuticals announced topline results from a two-part Phase 1 study showing vamorolone delivers glucocorticoid and anti-inflammatory activity without evidence of significant immunosuppressive effects at clinical doses. In Part A, 24 healthy adults received single doses of vamorolone 300 mg and deflazacort 0.9 mg/kg, with both demonstrating expected cortisol suppression and comparable onset, but vamorolone showed less pronounced immunosuppressive biomarker effects. In Part B, 36 healthy volunteers received vamorolone at 9, 27, or 40 mg/kg once daily for seven days; clinically relevant immunosuppressive effects were observed only at the highest dose of 40 mg/kg/day, which is above currently approved dosing. The findings support vamorolone's potential use across a broad range of chronic inflammatory rare diseases.