ASX Health Stocks: Neuren's treatment for cruel kids' disease on fast track

Neuren’s NNZ-2591 receives a Rare Pediatric Disease Designation from US FDA
This could lead to a Priority Review Voucher if drug is approved
Neuren is also preparing for a key FDA meeting, building on positive trial results

Neuren Pharmaceuticals (ASX:NEU) has received a Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its drug candidate, NNZ-2591.

This designation applies to Phelan-McDermid syndrome, a rare and severe genetic condition affecting children.

The designation is deemed significant because it offers Neuren the potential to receive a Priority Review Voucher (PRV). This voucher can be used to expedite the review process for a different product, or sold to another company.

The Rare Pediatric Disease PRV program is designed to encourage the development of treatments for serious rare diseases that affect children.

Neuren is now gearing up for an End of Phase 2 Meeting with the FDA, which is scheduled for the third quarter of 2024.

This meeting will be crucial for discussing the next steps in the development of NNZ-2591 for Phelan-McDermid syndrome.

Previously, Neuren reported promising results from a Phase 2 trial, which showed significant improvements as assessed by both clinicians and caregivers across various efficacy measures.

The announcement today came after Neuren’s Phase 2 trial for NNZ-2591 in another disease, Pitt Hopkins syndrome, demonstrated positive results per an information announced in May.

The company’s flagship drug however is DAYBUE (trofinetide), the world’s first treatment for Rett syndrome.

Rett syndrome is a rare genetic disorder that primarily affects girls and leads to severe cognitive and physical impairments.

Neuren is partnering with Nasdaq-listed Acadia Pharmaceuticals for the commercial development and marketing of DAYBUE.

As part of this collaboration, Neuren receives royalties from DAYBUE sales and is also eligible for milestone payments based on the drug’s sales performance from Acadia.

The Neuren stock price has doubled since October last year.

Memphasys gives key update

Meanwhile, Memphasys (ASX:MEM) has provided an update on its research and development activities.

Following the appointment of Klean Gene Pty Ltd in May to explore commercial opportunities for its animal applications, Memphasys has now prioritised its Oxidative Stress measurement system for potential commercialisation.

This decision comes after a thorough evaluation of its research technologies, guided by Research Director, Professor John Aitken and Klean Gene.

The Oxidative Stress measurement system is now considered a leading application due to its innovative approach and potential for significant market impact.

Oxidative stress, which results from an imbalance between harmful reactive oxygen species and protective antioxidants, is linked to various issues, including infertility and DNA damage in animals, and potentially in humans as well.

MEM’s system can quickly assess antioxidant levels, which is crucial for identifying animals in need of antioxidant supplementation and monitoring the effects of such interventions.

The system’s advantages include rapid point-of-care assessment, which can be applied across different sectors within the cattle industry, such as dairy and beef cattle. This could guide nutritional supplementation and enhance reproductive performance.

The unique features and wide range of applications give the system substantial commercial potential, as it addresses important needs in the industry.

Memphasys is now exploring industry partnerships and defining appropriate clinical on-farm partners for data and blood collection.

Once this process is complete, MEM will provide a detailed update on study progress, including partners, commencement date, and completion date.

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