Announced details
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B). Clinical trial in patients under 6 years of age
The first subject received the first dose in this trial
MEDIPAL acquired the licensing rights to commercialise JR-446 overseas. The two companies entered into an overseas licensing agreement and a co-development and commercialisation agreement in Japan in September 2023. MEDIPAL holds exclusive rights, including the right to grant sublicences, worldwide outside Japan, while JCR leads the trial and development activities as the trial sponsor.
MEDIPAL HOLDINGS CORPORATION: Chuo-ku, Tokyo; President and Representative Director Watanabe Shuichi (渡辺秀一), JCR Pharmaceuticals Co., Ltd.: Ashiya, Hyogo; President and Representative Director Sonoda Hiroyuki (薗田啓之)
Orphan drug designation
US, European (EU) and Japanese authorities (individual agency names Not disclosed)
ClinicalTrials.gov registration number NCT07640984. Validity period: Not disclosed
In this announcement the company addresses none of Contract Value / Offering price / Impact on Business Performance.
Public information, including Ministry of Health, Labour and Welfare research group studies, and JCR's own research (basis for estimating the number of patients)
| Item | Details |
|---|---|
| Names of Products / Services Covered | JR-446, J-Brain Cargo® |
| Counterparty | MEDIPAL HOLDINGS CORPORATION, JCR Pharmaceuticals Co.,Ltd. |
| Counterparty overview | MEDIPAL HOLDINGS CORPORATION: Chuo-ku, Tokyo; President and Representative Director Watanabe Shuichi (渡辺秀一), JCR Pharmaceuticals Co., Ltd.: Ashiya, Hyogo; President and Representative Director Sonoda Hiroyuki (薗田啓之) |
| Relationship with the Company | MEDIPAL acquired the licensing rights to commercialise JR-446 overseas. The two companies entered into an overseas licensing agreement and a co-development and commercialisation agreement in Japan in September 2023. MEDIPAL holds exclusive rights, including the right to grant sublicences, worldwide outside Japan, while JCR leads the trial and development activities as the trial sponsor. |
| Contract Date | September 2023 (agreement executed). Reference disclosure: 28 September 2023 |
| Applicable area | Treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B). Clinical trial in patients under 6 years of age |
| Award/certification name | Orphan drug designation |
| Awarding / certifying body | US, European (EU) and Japanese authorities (individual agency names Not disclosed) |
| Registration number and expiry date | ClinicalTrials.gov registration number NCT07640984. Validity period: Not disclosed |
| Usage history | The first subject received the first dose in this trial |
| Source | Public information, including Ministry of Health, Labour and Welfare research group studies, and JCR's own research (basis for estimating the number of patients) |
Items this document does not state (7)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.Because patients with MPS IIIB and their families face a significant unmet medical need, and to deliver this drug as soon as possible.