- Neuren Pharmaceuticals (NEU) submits an investigational new drug (IND) application to the US Food and Drug Administration (FDA)
- The application covers the investigation of NEU’s NNZ-2591 drug candidate as a potential treatment for Pitt Hopkins syndrome
- Meanwhile the company’s IND approval for the drug in Angelman syndrome has been placed on clinical hold following feedback from the FDA
- The Administration has concluded a 30-day review and requested amendments to the phase two trial protocol as a safety measure
- Shares are down 1.43 per cent at $2.07 each
Neuren Pharmaceuticals (NEU) has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA).
The application covers the investigation of NEU’s NNZ-2591 drug candidate as a potential treatment for Pitt Hopkins syndrome.
It follows earlier applications by the Melbourne-based company seeking IND approval of the drug to treat both Angelman and Phelan-McDermid syndromes.
Together, all three conditions are categorised as debilitating neurodevelopment disorders that emerge in childhood and currently have no approved medicines.
Accordingly, NEU has received feedback from the FDA following a 30-day review of its first IND application in Angelman syndrome.
As part of the feedback, the Administration has specified that additional clinical assessments be added to the phase two trial protocol to enhance safety monitoring in the first trial in patents.
As a result, Neuren said the IND had been placed on clinical hold until changes to the protocol are agreed with the FDA.
Formal notification of the hold will be issued in 30 days, after which the company said it would likely seek a meeting with the agency to negotiate the necessary amendments.
The company’s CEO Jon Pilcher said the delay was disappointing.
“We will work with the FDA to amend the protocol as quickly as possible, as well as confirming any necessary amendment to the protocols for the Phelan-McDermid and Pitt Hopkins phase two trials,” he said.
“We will also take this into account for our IND application and phase two protocol in Prader-Willi syndrome, which we plan to submit in the first half of 2022.”
Following the news, company shares were down 1.43 per cent at $2.07 each at 3:40 pm AEST.