ASX Health Stocks: Neuren Pharma to begin Phase 2 clinical trial of children with Pitt Hopkins syndrome

Neuren Pharma completes Phase 2 patient enrolment
Cyclopharm signed a deal with globally respected Duke University Hospital

Neuren’s Phase 2 Pitt Hopkins syndrome clinical trial

Market darling Neuren Pharmaceuticals (ASX:NEU) announced that enrolment of subjects into its Phase 2 clinical trial of NNZ-2591 in Pitt Hopkins syndrome has been completed.

Top-line results from the trial are expected to be available in Q2 2024.

The Phase 2 trial in children aged 3 to 17 years at five hospitals in the US will examine the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.

All subjects will receive NNZ-2591 as an oral liquid dose twice daily.

Pitt Hopkins syndrome (PTHS) is a neuro-developmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. The incidence of PTHS has been estimated at between 1 in 34,000 and 1 in 41,000 people. Some individuals with PTHS are diagnosed with autism.

Neuren is also conducting Phase 2 clinical trials of NNZ-2591 in children with three other neurodevelopmental disorders – Phelan McDermid syndrome, Angelman syndrome and Prader-Willi syndrome.

All four programs have been granted Orphan Drug designation by the US FDA, and are being developed under Investigational New Drug (IND) applications.

The company says that each syndrome is a seriously debilitating neurological disorder that emerges in early childhood, and has no or limited approved treatment options.

“We are grateful to all the people who have contributed to achieving this important milestone as we strive to develop a potential first therapy for Pitt Hopkins syndrome, including the Pitt Hopkins community and the trial site teams in the United States,” says Neuren CEO, Jon Pilcher.

“We now look forward to the remaining patients completing the trial, and to releasing the first results of treatment with NNZ-2591 in children with Pitt Hopkins syndrome.”

Cyclopharm signs deal with Duke University Hospital

Meanwhile, radiopharmaceutical specialist, Cyclopharm (ASX:CYC), announced that Duke University Hospital has signed the first commercial contract for the use of Cyclopharm’s Technegas product in the US.

This agreement follows the approval of Technegas by the US FDA in September.

Duke University Hospital is located in Durham, North Carolina, and is part of the larger Duke University Health System. It is a renowned academic medical centre known for its high-quality patient care, research and medical education.

Duke University Hospital is also consistently ranked among the top medical centres in the US, offering a wide range of specialties.

The hospital was one of 10 locations in the US that participated in Cyclopharm’s recent clinical trial that was submitted as part of the New Drug Application (NDA) for Technegas.

“I am thrilled that Duke University Hospital, a globally respected centre of healthcare excellence, innovation and a recent clinical trial site for Technegas, will be among the first to offer Technegas to their referring physicians and patients,” said Cyclopharm’s CEO, James McBrayer.

“The implementation of Technegas at Duke will be on full commercial terms as previously communicated to the market, and will be leveraging off Technegas’ wide indication for use approved by the US FDA.”