Table 2.
Clinical trials of drugs for neuronopathic MPS that utilize receptor-mediated transcytosis
| Disease | Compound | Clinical phase/status | Targeted receptor | Sponsor | Publication | Identifier |
|---|---|---|---|---|---|---|
| MPS I |
AGT-181 (valanafusp alpha) |
Phase I (completed) | Insulin receptor | ArmaGen | Giugliani et al. (2018b) | NCT02262338 |
| JR-171 | Phase I (recruiting) | Transferrin receptor | JCR Pharmaceuticals | Not available | NCT04227600 | |
| MPS II | AGT-182 | Phase I /II (completed) | Insulin receptor | ArmaGen | Not available | NCT03053089 |
| JR-141 (Pabinafusp alfa) | Phase III (completed) Approved in Japan | Transferrin receptor | JCR Pharmaceuticals | NCT03568175 | ||
| Phase III (Recruiting in US, EU and Brazil) | Not available | NCT04573023 | ||||
| DNL-310 | Phase I/II (recruiting) | Transferrin receptor | Denali Therapeutics | Not available | NCT04251026 |