U.S. FDA Grants Orphan Drug Designation to Gamgertamig for the Treatment of Pemphigus

Mechelen, Belgium; 24 August 2026, 22.01 CET; regulated information – Lakefront Biotherapeutics NV (Euronext & NASDAQ: LKFT) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to gamgertamig for the treatment of pemphigus. Gamgertamig was previously granted both Fast Track and Orphan Drug Designation by the U.S. FDA for the treatment of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP).

Gamgertamig is being developed by Lakefront in collaboration with Gilead Sciences. Ongoing clinical trials in autoimmune diseases are actively enrolling, and expansion of the clinical trials program is anticipated in 2027.

• Phase 1b study with gamgertamig in patients with autoimmune cytopenias (including ITP and AIHA) (NCT07083960), active in Australia and the United States.
• Phase 1b study with gamgertamig in seropositive autoimmune diseases (including idiopathic inflammatory myositis, Sjögren’s disease, pemphigus vulgaris (PV), and pemphigus foliaceus (PF)) (NCT07229144) active in Australia, New Zealand, Czech Republic, and Japan.
• Expect to initiate proof-of-concept basket studies assessing gamgertamig in additional autoimmune indications.

“We’re excited by the U.S. FDA’s award of Orphan Drug Designation for gamgertamig for the treatment of pemphigus as we continue to advance it for patients suffering with this debilitating and potentially life-threatening autoantibody-driven disease,” said Eric Hedrick MD, Chief Medical Officer of Lakefront. “I continue to believe gamgertamig has the potential to represent a transformative medicine in autoimmune diseases.”

About Gamgertamig

Gamgertamig is an investigational BCMAxCD3 bispecific T cell engager for the treatment of autoantibodies driven immune-mediated disease. Gamgertamig has been granted Orphan Drug Designation and Fast Track Designation by the U.S. FDA for certain autoimmune diseases. Gamgertamig is currently in Phase 2 studies and is expected to enter registrational studies in 2027. Gamgertamig is in-licensed from Keymed Biosciences, which owns the rights to develop the program in Greater China.

About Lakefront Biotherapeutics

Lakefront Biotherapeutics is a biotechnology company dedicated to building a differentiated pipeline of medicines for patients with serious diseases in areas of high unmet need. The Company has established a clinical‑stage portfolio in immunology and inflammation, anchored by gamgertamig, a potential first‑in‑class and best-in-class BCMAxCD3 T‑cell engager for autoimmune diseases. Backed by deep deal‑making expertise, operational flexibility, and a strong capital position, Lakefront identifies, acquires, and advances high‑quality assets with clear potential to deliver meaningful patient impact and long‑term shareholder value. For more information, visit https://www.lakefrontbio.com or follow us on LinkedIn or X.

For further information, contact Lakefront Biotherapeutics:

Investor Relations Sherri Spear +1 412 522 6418 [email protected]