Lakefront Biotherapeutics NV (LKFT), on Monday announced that the U.S. FDA has granted Orphan Drug Designation to Gamgertamig for the treatment of Pemphigus.
Pemphigus is a rare autoimmune blistering disorder affecting the skin and mucous membranes. In the U.S., its prevalence is estimated at approximately 5.2 cases per 100,000 adults (0.005%), with prevalence increasing with age. Although there is no cure, treatment options include corticosteroids, immunosuppressants and biologic therapies such as rituximab, while IVIG and plasmapheresis may be used in selected severe cases. (Source: National Institute of Health)
Gamgertamig is an investigational BCMAxCD3 bispecific T cell engager designed for the treatment of autoantibody-driven immune-mediated diseases, including pemphigus vulgaris and pemphigus foliaceus. It is designed to direct T cells against BCMA-expressing plasma cells and B-cell populations that contribute to autoantibody production.
The drug is being developed by Lakefront in collaboration with Gilead Sciences. The U.S. FDA previously granted gamgertamig both Fast Track and Orphan Drug Designations for the treatment of autoimmune hemolytic anaemia (AIHA) and immune thrombocytopenia (ITP).
Fast Track designation facilitates the development and expedited review of drugs addressing serious conditions with unmet medical needs, enabling more frequent FDA interactions and potential eligibility for accelerated approval and priority review.
Gamgertamig Pipeline
Gamgertamig is currently being evaluated in a Phase 1b study in patients with autoimmune cytopenias, including AIHA and ITP, in Australia and the U.S.
The drug is also being studied in a Phase1b trial in seropositive autoimmune diseases, including idiopathic inflammatory myositis, Sjögren's disease, phemphigus vulgaris and foliaceus, across Australia, New Zealand, the Czech Republic and Japan.
As a milestone ahead, gamgertamig is currently in Phase 2 development study for autoimmune diseases, including AIHA and ITP, and expects to enter registrational studies in 2027.
Lakefront is also planning to initiate proof-of-concept basket studies for additional autoimmune indications in 2027.
LKFT has traded between $25.68 and $37.78 over the last year.
The stock closed Monday at $29.04, up 0.83%.
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