Kyowa Kirin, Inc.

The FDA approved Kura Oncology's once-daily pill Komzifti (ziftomenib) for treating relapsed/refractory acute myeloid leukemia with NPM1 mutations, affecting about 30% of AML cases.

Boehringer Ingelheim has acquired exclusive worldwide rights to a pre-clinical small molecule program from Kyowa Kirin targeting autoimmune diseases.

Japan's Ministry of Health, Labor and Welfare has granted Orphan Regenerative Medicine Product Designation to OTL-200, an investigational gene therapy for early-onset metachromatic leukodystrophy (MLD).

Kyowa Kirin shares gained 3.2% in the past week following FDA approval for a Parkinson's treatment, though the stock remains down 9.7% over the past month and 7% year-over-year.

Ultragenyx Pharmaceutical appointed Eric Olson as Chief Business Officer and Executive Vice President effective September 22, 2025, following Thomas Kassberg's planned retirement after 14 years with the company.

UK-based Charm Therapeutics secured $80 million in Series B funding to advance its next-generation menin inhibitor for acute myeloid leukemia into Phase I clinical trials starting Q1 2026.

Kura Oncology's lead asset ziftomenib has advanced to FDA Priority Review for relapsed/refractory NPM1-mutant acute myeloid leukemia, with a PDUFA date of November 30, 2025.

Kyowa Kirin will present results from the Phase 3 ROCKET IGNITE trial of rocatinlimab, an investigational anti-OX40 monoclonal antibody for moderate-to-severe atopic dermatitis, at the EADV 2025 Annual Meeting in Paris.

Orchard Therapeutics has completed enrollment in the HURCULES registrational trial for OTL-203, an investigational hematopoietic stem cell gene therapy for MPS-IH (Hurler syndrome), nearly one year ahead of schedule.

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.