Wave Life Sciences Shelves its Huntington Drugs

Wave Life Sciences, a biotech company, based in Massachusetts, is shelving two of its most advanced drugs. This decision followed after the clinical testing found neither had a big enough effect on the mutated proteins that cause Huntington’s disease. Five doses of Wave’s drugs were evaluated in the tests. The drugs are known as WVE-120101 and WVE-120102.

On Monday, high-level results showed the drugs did not perform significantly better than the placebo. There was no evidence of the dose levels affected and how patients responded to treatment. An extension of the study WVE-120102 saw some modest reductions in the disease-causing proteins. Wave Life Sciences said that the results were inconsistent over the course of the trial.

With the winding down of two lead programs, Wave hopes that another experimental drug will fare better. The drug, WVE-003, has a different chemical backbone than that its predecessors. Wave believes that it will target a specific mutation in the gene responsible for making the Huntington protein. Sometime this year, the company hopes to begin dosing patients with the early-stage study of WVE-003.

Wave Life Sciences takes short pieces of genetic material called oligonucleotides to design its drugs and modifies them chemically to stop the production of harmful proteins. The approach is not new, but Wave claims to put its own spin on it. For decades, researchers have been exploring the therapeutic potential of oligonucleotides. In recent years, researchers have also watched the work turn into approved treatments for several inherited. However, victories have not come without setbacks.