Biohaven protein-degrader drug falls short of investor expectations in early-study test

Biohaven Pharmaceuticals said Wednesday that a new type of experimental medicine reduced levels of a disease-causing immune molecule by up to 37% in an early-stage study of human volunteers — a result that the company called “positive” as a proof of concept but that also fell short of investor expectations.

The drug, called BHV-1300, belongs to a new class of antibody medicines that shuttle harmful proteins to the liver so they can be removed from the body. BHV-1300 is specifically designed to reduce levels of an autoantibody called IgG that is implicated in rheumatoid arthritis, myasthenia gravis, and other autoimmune disorders.

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In its Phase 1 study, Biohaven tested four escalating doses of BHV-1300 in healthy volunteers, showing a reduction in levels of IgG of 5%, 15%, 30%, and 37%, respectively, the company said. The reductions in IgG levels, which occurred relatively quickly in just four to five days, did not cause significant, adverse changes in liver enzyme levels, Biohaven added — an important safety check given the mechanism of the drug.

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