Shares of Disc Medicine Inc. (NASDAQ:IRON) were trading at $25.74, down $36.52, or 58%, after the Watertown, Mass.-based company disclosed top-line data from the phase II Aurora study with glycine transporter 1 inhibitor bitopertin in erythropoietic protoporphyria (EPP). The study met its primary endpoint, turning up a dose-dependent, statistically significant reductions in protoporphyrin IX compared to placebo in the 20-mg and 60-mg dose groups. On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients showed a positive response consistent with phase II Beacon results in EPP, but the data fell short of statistical significance, which the firm blamed on strong placebo results.

Rx, med-tech industry groups warn of impact of Gilead R&D suits

The U.S. biopharma and med-tech industries are adding their voice to that of Gilead Sciences Inc. in urging the California Supreme Court to review the Gilead Tenofovir Cases, which challenge the drug company’s HIV pipeline development decisions. Earlier this year, California’s First District Court of Appeal said thousands of state negligence suits could continue against Gilead, which the plaintiffs contended “breached its duty of reasonable care by postponing, solely to maximize profit, its effort to commercialize TAF [tenofovir alafenamide fumarate] as a treatment for HIV/AIDS while continuing to market a medication with serious side effects that it knew TAF would have enabled patients to avoid.” An amicus brief filed by both drug and device industry groups stressed the importance of the case and its potential impact on innovation in the life sciences. “Decisions regarding whether to continue research on a life sciences product in its early stages – or whether to start that research at all – will be greatly impacted if liability can be imposed if the company makes the wrong prediction,” according to the brief.

Alexion’s Voydeya gets second PNH approval, preps for another

Voydeya (danicopan), from Alexion, Astrazeneca Rare Disease, racked up its second global approval as the U.S. FDA greenlit it as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). Patients must already be receiving the standard of care, Ultomiris (ravulizumab-cwvz) or Soliris (eculizumab). The approval was based on the pivotal phase III Alpha study, which met its primary endpoint in changing hemoglobin levels and all the key secondary endpoints, which included transfusion avoidance and changes in patients’ fatigue levels. In January, Japan's MHLW approved Voydeya in combination with C5 inhibitor therapy for treating PNH. The oral factor D inhibitor received in February the EMA's Committee for Medicinal Products for Human Use recommendation for marketing authorization for residual hemolytic anemia in PNH patients.

BMS’ Zeposia falls in first of two phase III trials in Crohn’s 

Bristol Myers Squibb Co. reported disappointing data from the initial analysis of its Yellowstone study, the first of two phase III trials testing S1P modulator Zeposia (ozanimod) in patients with Crohn’s disease. Results showed the study failed to meet the primary endpoint, defined as clinical remission as measured by the Crohn’s Disease Activity Index at week 12. BMS, which noted no S1P modulators to date have been successful in late-stage Crohn’s disease studies, said it will conduct a full evaluation for a future presentation of detailed data.

China accepts Sperogenix NDA filed for rare muscle disorder drug

Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26. Sperogenix in-licensed exclusive rights to vamorolone in China, Hong Kong, Macau and Taiwan in January 2022 from Pratteln, Switzerland’s Santhera Pharmaceuticals Holding AG in a deal worth up to $124 million.

Orion Holdings completes acquisition of Ligachem Biosciences

South Korean confectionary giant Orion Holdings Inc. has completed the acquisition of antibody-drug conjugate (ADC) developer Ligachem Biosciences Inc., which recently changed its name from Legochem Bioscience Inc. Ligachem, derived from the Latin prefix “liga“ to mean “bind” or “tie,” helps highlight the biotech’s focus on developing novel ADC drugs and its strength in medicinal chemistry, the company said in a statement on April 1. Ligachem first announced plans of Orion’s acquisition in January 2024. Combined, the completed ₩548.7 billion (US$412.11 million) deal for 9.36 million shares makes Orion the controlling shareholder of Ligachem with a 25.73% stake.

Holiday notice

BioWorld's offices were closed in observance of Good Friday. No issue was published March 29.

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