Today, a brief rundown of news from Immunovant and Alto Neuroscience, as well as updates from Oric Pharmaceuticals, Korsana Biosciences and a couple genetic medicine developers you may have missed.
Shares for Immunovant, a Roivant spinout, fell Thursday after the company announced its experimental therapy for thyroid eye disease failed two late-stage trials. The company said batoclimab, its drug in development with partner HanAllBiopharma, did not meet the Phase 3 trials’ key goals, which centered on a reduction in eye swelling for adults with active thyroid disease. Immunovant said safety data was consistent with previous findings, and no new safety signals were identified. The company did not share detailed results. The partners said they will review plans for their candidate and provide updates at a later date. — Delilah Alvarado
An experimental drug from Alto Neuroscience has failed a so-called proof-of-concept study evaluating it as a treatment for the cognitive impairment associated with schizophrenia. The drug, known as ALTO-101, is meant to inhibit the “PDE4” enzyme. Alto’s study found ALTO-101 was not significantly better than a placebo when looking effects on brain activity and cognitive function. The company said has developed a modified-release, once-daily oral formulation of ALTO-101 and plans to explore partnering opportunities for it. Alto shares were subsequently down more than 7% in late morning trading Thursday. Andrew Tsai, an analyst at Jefferies, wrote that Wall Street had low expectations for this trial and is more focused on two Alto drugs for depression that have upcoming readouts. — Jacob Bell
Updated data published in the New England Journal of Medicine from Beam Therapeutics show that risto-cel, a gene editing treatment that it's studying for sickle cell disease, eliminated severe pain crises among 31 patients enrolled in the trial. The gene therapy company said Wednesday its treatment, which relies on a technology called base editing to change the individual letters in DNA, also helped restore the shape of red blood cells, resolve anemia and reduce the breakdown of red blood cells. Patients were followed anywhere from a little under two weeks to more than 20 months. Beam previously reported a patient death in its study of risto-cel, then known as BEAM-101, that investigators found to be related to busulfan, a chemotherapy drug used to condition patients for stem cell transplants. Late last month, the company also reported positive data from its study of a gene editing treatment for alpha-1 antitrypsin deficiency. — Gwendolyn Wu
Cell therapy developer Orca Bio said Wednesday that the Food and Drug Administration has delayed the review of its blood cancer drug Orca-T. Originally slated for this month, federal regulators now have until July 6 to return a decision on its allogeneic T cell therapy. In a Phase 3 study last year, the California biotechnology firm said that its treatment helped three-quarters of trial participants with a variety of hematological cancers avoid a serious side effect called graft-versus-host disease, compared to just 38% of patients given an ordinary stem cell transplant. Orca recently submitted additional information on its manufacturing processes, but has not been asked for additional clinical data, according to the company. The FDA has extended, or missed, deadlines for numerous other drugs over the past year. — Gwendolyn Wu
Shares of Oric Pharmaceuticals fell more than 40% Wednesday after the California-based drug developer provided updates on its most advanced research program. Oric has been testing “rinzimetostat” — an experimental medicine meant to block a cancer-linked enzyme called EZH2 — in an early-stage clinical trial of patients with metastatic castration-resistant prostate cancer who were previously treated with a median of two prior lines of therapy, including Johnson & Johnson’s Zytiga. Oric said based on data from the trial, it has decided to run a larger Phase 3 study to evaluate a 400 milligram dose of its drug in combination with Bayer’s Nubeqa. Jefferies analyst Maury Raycroft wrote in a client note that some investors believe J&J’s Erleada would have been a strategically better combination choice. Raycroft, though, argues that the evidence Oric has gathered so far shows it has a competitive drug. The stock sell off is therefore an overreaction, the analyst wrote. Oric shares rebounded around 10%, to $8.30 apiece, by mid-morning Thursday. — Jacob Bell
Korsana Biosciences on Wednesday announced a reverse merger with Cyclerion Therapeutics. The combined company will trade on Nasdaq under the ticker “KRSA” and focus on advancing Korsana’s potential treatments for neurodegenerative diseases. The most advanced of those treatments, codenamed KRSA-028, is a shuttled monoclonal antibody for Alzheimer’s disease that targets the protein amyloid beta. Alongside the merger, Korsana secured commitments for an oversubscribed private investment that’s expected to total $380 million in gross proceeds. Fairmount and Venrock Healthcare Capital Partners led the investment. — Jacob Bell