HIE Starting with a PK study (one month long) in neonates to...

HIE

• Starting with a PK study (one month long) in neonates to open the IND, as already flagged by the company.
• Juvenile animal study data to support dosing in neonatal participants prior to submitting the IND application and initiating the clinical study. Neuren plans to run this in parallel with advancing preparation for the clinical study.
• Neuren still plans to commence the clinical study this year, as already flagged by the company.
• Neuren still plans to move to a pivotal trial following positive results in the PK study.
• As is standard, there will be another meeting with the FDA prior to the pivotal trial.
• An excellent outcome, aligning with what Neuren had said it hoped for.

Pitt Hopkins Syndrome

• Clinician and carer co-primary endpoints, as used for PMS and trofinetide in Rett syndrome pivotal trials.
• There will be a further meeting with the FDA this year to lock in the final trial design.
• Neuren still plans to commence the pivotal trial this year, as already flagged by the company.
• An excellent outcome, aligning with what Neuren had said it hoped for.

I’m very happy with the outcome of the two meetings. Together with the news this morning that the PRV voucher program has been reauthorized, this has been a good news day!