Potential therapy for rare blood disorders acquired by companies
Gamgertamig targets forms of AIHA, including cold agglutinin disease
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Gilead Sciences and Lakefront Biotherapeutics have acquired Ouro Medicines and its investigational therapy, gamgertamig, for cold agglutinin disease (CAD) and other forms of autoimmune hemolytic anemia (AIHA). Gamgertamig is also being developed as a treatment for several other autoimmune diseases.
Under the deal, valued at up to $2.175 billion, gamgertamig will be the foundation of Lakefront’s pipeline, while Gilead will retain the therapy’s commercialization rights around the world, the companies announced in a joint press release. Gilead will pay Lakefront royalties on net sales should the therapy be approved.
Keymed Biosciences, gamgertamig’s original developer, owns the rights to develop the therapy in Greater China.
Per the agreement, Lakefront (formerly known as Galapagos) will continue to oversee ongoing and planned early-stage clinical studies of gamgertamig, while Gilead will lead later-stage development.
The companies expect to launch registrational clinical trials of gamgertamig as early as 2027. These are the large trials typically designed to support applications for regulatory approval.
Gamgertamin aims to harness immune system to eliminate B-cells
AIHA comprises a group of rare autoimmune diseases in which self-reactive antibodies attack red blood cells, ultimately triggering the cells’ destruction. This can lead to anemia, a shortage of red blood cells, and symptoms such as fatigue, weakness, shortness of breath, and dizziness. In CAD, this attack occurs at low temperatures.
Also known as OM336, gamgertamin is a bispecific antibody designed to harness the body’s immune system to eliminate B-cells, the immune cells responsible for producing antibodies, including those that drive AIHA and other autoimmune diseases.
Given as a subcutaneous, or under-the-skin, injection, the therapy simultaneously binds to the CD3 protein on immune T-cells and to BCMA, a receptor protein commonly found on B-cells. By bringing these cells into close contact, gamgertamig directs T-cells to destroy B-cells, reducing levels of harmful, self-reactive antibodies.
According to the companies, the therapy enables rapid and deep depletion of these antibody-producing cells after a limited treatment course, with the goal of providing durable disease control without the risks associated with broad immune system suppression.
Gamgertamig received orphan drug and fast track designations in the U.S. for the treatment of AIHA and immune thrombocytopenia (ITP), an autoimmune disease that targets platelets, the cell fragments responsible for blood clotting. These statuses are meant to speed the clinical development and regulatory review of potential therapies for rare or serious conditions with unmet medical needs.
The therapy is currently being evaluated in a Phase 1b clinical trial (NCT07083960) involving up to 32 adults with hard-to-treat AIHA (including CAD), ITP, or both. Enrollment may still be ongoing at sites in the U.S. and Australia.
The study’s main goal is to assess gamgertamig’s safety and tolerability over 12 weeks (about three months), while also examining how the therapy moves into, through, and out of the body and whether it triggers immune responses against itself.
Gamgertamig is also being evaluated as a potential treatment for Sjögren’s disease and idiopathic inflammatory myopathies — two autoimmune disorders.
Helen Bohl
Being treated for WM with Brukinsa but bloods not improving and feel I’m more likely to have primary CAD fatigue weakness shortage of breath and dizziness my symptoms..
Cheers Helen