PET aSyn — QST Specified Clinical Trial planning (Operational lead, Funding MJF, Schedule, IND, Goal, Protocol, Process, Contract, Freedom to operate, GLP-Tox)
QST Specified Clinical Trial — planning (PET aSyn vendor matrix continuation)
[20230809] CS to Check timeline to use a PET for SPAL-T-06 clinical studies, and Reach out to MJFF, to see if they would like to collaborate on SPAL-T-06 clinical studies. Paul Ito, Reach out to Yutoo to check required dataset for PET IND in US.
| Takeda | QST as Specified Clinical Trial | |
|---|---|---|
| Operational lead | ||
| Estimated cost | TBD | ~80M JPY (= US 559K JPY for actual drug manufacturing cost of SPAL-T-06 1Q SPAL-T-86 IFM 5 incidung tax) |
| Funding | MJF (full vs partial? TBD) The SPAL trials will keep SPAL-T-06 patent. If MJFF industry consortium participants would like to use SPAL-T-06, they have to have license agreement. | Ono, Takeda, and potentially QST. (not MJF) Takeda has announced the withdrawal from the SPAL-T-06 longitudinal study in Japan as QST and Ono Pharma before signing a contract because of no budget. |
| Schedule | All the data generated by the MJFF collaboration have to be shared with MJFF industry consortium participants (e.g. Merck, Biogen) and will be public information after an embargo period. → 20231113 One set to join. 8 months in total (2-3 months for IND and 4 months to finalize protocol). a joint research agreement 2025 fall: interim draft analysis results of data up to 12 months (at least about 10 cases) — summer of 2025 | |
| IND | NOT need IND filing because of Specified Clinical Research by QST | |
| Goal |
(1) Enrichment of baseline data by a larger number of subjects than in specific clinical studies of FIH; Comparison between MSA-P and MSA-C, Correlation with UMSARS, (correlation with other items, BMI, etc.) (2) Examination of changes over time (6 months, 12 months); Correlation between SUVR change rate and UMSARS change rate in key areas of interest, etc. [from Ono] | |
| Protocol |
-Target: Patients with MSA-P or MSA-C (~10-15 patients in total) -Entry criteria: <5 years of onset -PET imaging points: 3 time points (0m, 6m, 12m) live α-syn in CSF and/or plasma and NFL in plasma. Plasma sampling WITHOUT CSF should be feasible to proceed the longitudinal study in a timely manner. | |
| Protocol-BM | Ono agreed to the CSF sampling in US of planned | |
| Protocol-Tox | Ona has low priority of 1mo PET in an accumulation of tau in MSA seems to be important | |
| Protocol | Protocols in Japan and US should be identical as much as possible. Dr. Higuchi is planned join discussion with MJFF and lawyers about the study in US. | |
| Process |
Propose plan to MJFF consortium, make an agreement with MJFF and SPAL team, and transfer data for IND filing.
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| Contract | Agreement with QST, Eisai, Ono, MJFF, and Takeda to disclose confidential information to MJFF (Article 12, from 5 of the patent application agreement). If undisclosed information is shared with MJFF, the agreement is required → The agreement would be required to share non-GLP tox and dosimetry data for IND filing in US. | |
| Freedom to operate |
License Agreement 한송이라 어... 20230914 a-syn PET Pleockefelder "Freedom to operate" SPAL T-06.pdf Fushimi, Makoto The SPAL T-06 kickoff meeting with QST about MJFF industry consortium will be held on 30/31 Jan 2024. Attendees:
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| GLP-Tox |
cost: approx. 8 mil yen + GLP test substance manufacturing cost. 20230706 Keep discussion with QST and Ono about SPAL-T-06 GLP tox study and longitudinal study. | |
| Higuchi from QST: Join both discussion for US & Jap to have identical protocols as much as possible | ||