Kyowa Kirin, Inc.

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.

The FDA has accepted Kura Oncology's new drug application for ziftomenib, an oral menin inhibitor for treating relapsed or refractory acute myeloid leukemia with NPM1 mutations.

• Lenmeldy (atidarsagene autotemcel), Orchard Therapeutics' hematopoietic stem cell gene therapy, demonstrated significant extension of life expectancy and preservation of motor and cognitive function in children with early-onset metachromatic leukodystrophy. • The New England Journal of Medicine publication details over 12 years of follow-up data with more than 250 patient-years of experience, showing durable efficacy in pre-symptomatic and early-symptomatic patients. • Orchard Therapeutics will present six studies at the upcoming American Society of Gene and Cell Therapy meeting, highlighting their expanding portfolio of hematopoietic stem cell gene therapies for rare neurometabolic diseases.

The global Adult T-cell Leukemia-Lymphoma (ATLL) market is projected to experience significant growth through 2035, driven by increasing HTLV-1 infections and advancements in immunotherapy technologies.

• Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) has become the first FDA-approved treatment for metachromatic leukodystrophy (MLD), a rare genetic disorder that causes rapid neurological deterioration in children. • At $4.25 million per one-time treatment, Lenmeldy now holds the title of the most expensive medicine in the United States, slightly higher than its European price of $3.7 million where it's marketed as Libmeldy. • Clinical trials showed remarkable results with all 37 treated children surviving to age six (compared to 58% in control groups), with 71% able to walk independently and 85% maintaining normal language and cognitive function.

The 35-year partnership between Kyowa Kirin and La Jolla Institute for Immunology has been renewed for three more years, representing one of the longest-standing industry-academic collaborations in drug discovery.

Preclinical data for Kura Oncology's next-generation farnesyl transferase inhibitor KO-2806 in combination with cabozantinib has been selected for oral presentation at the 2025 AACR Annual Meeting.

Amgen and Kyowa Kirin's Phase 3 IGNITE study of rocatinlimab met all primary and secondary endpoints, with 42.3% of patients in the higher dose group achieving EASI-75 compared to placebo.

Ziftomenib, a selective oral menin inhibitor developed by Kura Oncology and Kyowa Kirin, has successfully met primary endpoints in the Phase II Komet-001 registrational trial for acute myeloid leukemia.