Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study.

Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study.

Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said.

Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD).

Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said.

Sotio Biotech AS’s SOT-106 has been granted orphan drug designation by the FDA for the treatment of osteosarcoma. SOT-106 is a next-generation antibody-drug conjugate targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumor-associated stroma.

Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing.

Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing.

Pilatus Bio Inc. is working to address a major under-addressed target of current checkpoint inhibitors: metabolic stress within solid tumors. “Traditional immunotherapies release immune ‘brakes,’ but they do not address the underlying metabolic stress in tumors,” Pilatus CEO and cofounder Raven Lin said. “That’s why more than 60% of solid tumor patients do not respond to treatment.”

With three rare pediatric disease priority review vouchers (RPD PRVs) awarded just since the end of March, the nearly year-and-a-half lapse in the program’s reauthorization seems to have had little short-term impact. The three new vouchers bring the total RPD PRVs granted so far this year to seven – one more than the agency issued all last year and down two from the nine given in 2024.