Amchepry (raguneprocel) / Sumitomo Pharma, RACTHERA - LARVOL DELTA

Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II) (clinicaltrials.gov) - P1 | N=7 | Recruiting | Sponsor: University of California, San Diego | Trial primary completion date: Feb 2025 ➔ Dec 2027

Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease • Transplantation

iPS Cell-based Parkinson’s Drug Face Challenges Over High Cost, Unverified Efficacy After Receiving Temporary Approval (The Japan News) - "The decision to make an iPS-derived medicine for Parkinson’s disease available under public health insurance marks a major step forward for treatment. However, it remains necessary to verify its efficacy and whether it is worth its high price tag...The health ministry in March approved the manufacturing and marketing of Amchepry on a time-limited basis through March 2033...The company is also considering expanding the number of participating medical institutions in around 2030...The Central Social Insurance Medical Council, an advisory body to the health minister, approved Amchepry treatment to be set at around ¥55.3 million, a high cost compared to the majority of pharmaceuticals."

Reimbursement • Parkinson's Disease

Japan approves commercialization of 2 iPS-derived drugs in world’s first - "The Japanese government on Friday conditionally approved the commercialization of two regenerative medicine products prepared from induced pluripotent stem cells, marking the world's first. The two iPS-derived drugs are ReHeart, developed by Cuorips Inc. to treat patients with severe heart failure stemming from ischemic cardiomyopathy, and Amchepry, produced by Sumitomo Pharma Co. and Racthera Inc. to treat Parkinson's disease. Treatment using the drugs is expected to begin as early as this summer. ReHeart is expected to cost over 10 million yen ($63,500), and Amchepry will also be expensive."

Japan approval • Launch Japan • Pricing • Cardiomyopathy • Parkinson's Disease

Japan panel approves world’s first iPS cell-based therapies (Nikkei) - "The Ministry of Health, Labor and Welfare's committee on regenerative medicine products and biotechnologies approved heart muscle sheets derived from human iPS cells developed by Cuorips, a University of Osaka-born startup. The committee also approved Amchepry, a candidate treatment for Parkinson's disease developed by Japanese drugmaker Sumitomo Pharma....The trials observed symptom improvement in four of six Parkinson's patients, and in all eight heart failure patients."

Japan approval • Parkinson's Disease

A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects With Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=25 | Recruiting | Sponsor: Sumitomo Pharma America, Inc. | Phase classification: P1 ➔ P1/2 | Trial completion date: Mar 2029 ➔ Dec 2030 | Trial primary completion date: Mar 2029 ➔ Dec 2030

Phase classification • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Movement Disorders • Parkinson's Disease

Announcement of a Subsidiary’s Submission of the Application of Manufacturing and Marketing Authorization for an iPS Cell-Derived Product for Parkinson’s Disease in Japan (Sumitomo Chemical Press Release) - "Sumitomo Pharma Co., Ltd...and RACTHERA Co., Ltd...hereby announce that...they have submitted an application of manufacturing and marketing authorization for allogeneic iPS cell-derived dopaminergic neural progenitor cells (INN: 'raguneprocel', 'the Product') for indication of the improvement of motor functions during the off-time period of patients with advanced Parkinson’s disease. The applicant for the authorization is Sumitomo Pharma. This submission was based on the data from the investigator-initiated trial conducted by Kyoto University Hospital, and its results were published in the journal Nature in April 2025...The Product has been designated by the Ministry of Health, Labour and Welfare as a product for priority review under the SAKIGAKE Designation System."

Japan filing • Priority review • Parkinson's Disease

A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects with Parkinson's Disease (clinicaltrials.gov) - P1 | N=23 | Recruiting | Sponsor: Sumitomo Pharma America, Inc.

New P1 trial • Alzheimer's Disease • CNS Disorders • Movement Disorders • Parkinson's Disease

Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II) (clinicaltrials.gov) - P1 | N=7 | Recruiting | Sponsor: University of California, San Diego

New P1 trial • CNS Disorders • Movement Disorders • Parkinson's Disease • Transplantation