Companies in #Paroxysmal Nocturnal Hemoglobinuria (PNH)

Apellis Pharmaceuticals is a commercial-stage biopharmaceutical company developing targeted C3 therapies to inhibit the complement cascade in diseases of hematology, ophthalmology, and nephrology. The company has two FDA-approved C3 inhibitors: Empaveli (pegcetacoplan) for paroxysmal nocturnal hemoglobinuria (PNH), and Syfovre for geographic atrophy (GA) secondary to age-related macular degeneration.

Jasper Therapeutics is a clinical-stage biotechnology company developing therapeutics for mast-cell-driven diseases, chronic spontaneous urticaria, chronic inducible urticaria, and severe combined immunodeficiency (SCID). Following the July 16, 2026 all-stock acquisition of Kira Pharmaceuticals and concurrent $132M private placement of non-voting convertible preferred stock, the combined company is positioned around three core assets: KP-104 (a dual-complement inhibitor for paroxysmal nocturnal hemoglobinuria and high-unmet-need nephrology disorders), briquilimab (an anti-KIT monoclonal antibody with broad utility across transplant and immunologic indications), and KP-701 (a dual-acting anti-CD79BxCD32B mAb for autoantibody-mediated disorders). The combined cash position is expected to fund operations through H2 2028.

Kira Pharmaceuticals is a privately-held, clinical-stage biotechnology company pioneering a world free from complement-mediated disease. Founded in 2017 and headquartered in Cambridge, Massachusetts with research operations in Suzhou and Shanghai, China, the company uses its proprietary LOGIC platform (Lead identification, Optimization, Attribute Generation, In-vivo Confirmation) to design complement-pathway therapeutics. Its lead asset KP104 is a bifunctional C5 monoclonal antibody / Factor H fusion protein that simultaneously blocks the terminal and alternative complement pathways; KP104 is currently in Phase 2 trials for paroxysmal nocturnal hemoglobinuria (PNH), IgA nephropathy (IgAN), and complement 3 glomerulopathy (C3G). On July 16, 2026, Kira Pharmaceuticals announced an all-stock merger with Jasper Therapeutics combined with a $132M private placement of non-voting convertible preferred stock, positioning KP-104 alongside Jasper's briquilimab in the combined company's three-asset pipeline.