Graves' Disease and Graft-versus-Host Disease: Pipeline Therapeutics Update

Key Insights

• Several companies are actively developing novel therapies for Graves' Disease, targeting mechanisms like IGF-1R and FcRn.
• Clinical trials are underway for Batoclimab (Phase III), VRDN-001 (Phase III), and Linsitinib (Phase II/III) to address Graves' Disease.
• The Graft-versus-Host Disease pipeline includes over 65 drugs in development, with companies like MaaT Pharma and Syndax Pharmaceutical leading the way.

Several pharmaceutical companies are focusing on developing innovative treatments for Graves' Disease and Graft-versus-Host Disease (GVHD), with multiple drugs in various stages of clinical trials.

Graves' Disease Therapeutics

Graves' Disease, an autoimmune disorder affecting the thyroid gland, is seeing advancements in its treatment landscape. Immunovant Sciences GmbH is developing Batoclimab, a fully human monoclonal antibody targeting the neonatal Fc receptor (FcRn). Batoclimab is currently in Phase III trials. By blocking FcRn, batoclimab enhances the degradation of IgG antibodies, which are implicated in autoimmune diseases.

Viridian Therapeutics is also advancing VRDN-001, a monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). VRDN-001 is in Phase III trials (THRIVE) for active thyroid eye disease (TED) and Phase II trials for chronic TED. Preliminary data has demonstrated reductions in proptosis and improvements in clinical activity scores (CAS). Sling Therapeutics is investigating Linsitinib, an oral small molecule inhibiting IGF-1R, currently in Phase II/III trials.

Graft-versus-Host Disease Therapeutics

Graft-versus-host disease (GVHD) occurs when immune cells from a donor graft recognize the recipient's body as foreign and attack it. MaaT Pharma is developing MaaT013, a microbiome bio therapeutic consisting of allogeneic, full-ecosystem pooled intestinal microbiota. MaaT013 is in Phase III development and aims to restore the symbiotic relationship between the patient's gut microbiome and immune system. Medac is also developing MC0518 (Obnitix), a mesenchymal stromal cell preparation, currently in Phase III trials.

Syndax Pharmaceutical is working on axatilimab (SNDX-6352), an antibody targeting the colony-stimulating factor 1 receptor (CSF-1R). An ongoing Phase II study (AGAVE-201) is assessing the efficacy and safety of axatilimab in patients with chronic GVHD. AltruBio is in Phase I clinical trials with Neihulizumab/ALTB-168, a humanized antibody inducing apoptosis of activated T cells.

These emerging therapies represent potential advancements in treating Graves' Disease and GVHD, addressing unmet medical needs and offering new options for patients.