Guess which ASX 200 healthcare stock is charging higher following FDA update

What is getting investors excited today? Let's find out.

| More on:
Shot of a young scientist using a digital tablet while working in a lab.

Image source: Getty Images

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

Neuren Pharmaceuticals Ltd (ASX: NEU) shares are having a strong start to the week.

In morning trade, the ASX 200 healthcare stock is up over 4% to $14.63.

Why is this ASX 200 healthcare stock charging higher?

The catalyst for this gain has been the release of an announcement this morning in relation to the US Food and Drug Administration (FDA).

According to the release, Neuren has reported positive outcomes from an End of Phase 2 Meeting with the US FDA to discuss proposals for the first ever pivotal clinical trial program in Phelan-McDermid syndrome (PMS).

Management notes that this is a big positive given that there are no medications approved for PMS, which has severe quality of life impacts on those living with it, as well as on parents and siblings.

At the FDA meeting, alignment was reached on key aspects of the NNZ-2591 Phase 3 program. This will see Neuren submit further information to confirm endpoints for the primary efficacy assessment.

Phase 3 trial

The ASX 200 healthcare stock notes that the single pivotal Phase 3 trial will be a randomised, double-blind, placebo-controlled trial of treatment for 13 weeks in children aged 3 to 12 years with PMS.

Participants may then continue into an open-label extension study continuing treatment until commercial launch.

There will be one active treatment group versus placebo, with a target dose equivalent to the dose that was tested in the Phase 2 trial.

Based on the safety data from the Phase 2 clinical trial, Neuren proposed a less burdensome safety monitoring plan for the Phase 3 and open label extension trials. This was considered reasonable by the FDA, subject to review of the final protocol.

Management highlights that as this study is the first ever pivotal clinical trial in PMS, there is no precedent for efficacy assessment.

Nevertheless, primary efficacy endpoints for the Phase 3 trial were considered in depth at the meeting, with a range of potential options discussed. The FDA has requested that Neuren submit further existing information before agreeing the final selection of the endpoints.

It is now compiling the further information on efficacy endpoints and updating the Phase 3 trial protocol. Each of these will be submitted to the FDA for review. In the meantime, Neuren is well advanced in the selection of service providers for the Phase 3 program and has commenced identification of potential trial sites.

The ASX 200 healthcare stock's CEO, Jon Pilcher, was pleased with how the meeting went. He commented:

We are pleased with the outcomes of a very collaborative meeting with the FDA and are eager to move forward in our mission to develop NNZ-2591 as a first approved treatment for Phelan-McDermid syndrome, which has an overwhelming unmet need.

Should you invest $1,000 in Neuren Pharmaceuticals Limited right now?

Before you buy Neuren Pharmaceuticals Limited shares, consider this:

Motley Fool investing expert Scott Phillips just revealed what he believes are the 5 best stocks for investors to buy right now... and Neuren Pharmaceuticals Limited wasn't one of them.

The online investing service he’s run for over a decade, Motley Fool Share Advisor, has provided thousands of paying members with stock picks that have doubled, tripled or even more.*

And right now, Scott thinks there are 5 stocks that may be better buys...

See The 5 Stocks *Returns as of 7 February 2025

Motley Fool contributor James Mickleboro has no position in any of the stocks mentioned. The Motley Fool Australia's parent company Motley Fool Holdings Inc. has no position in any of the stocks mentioned. The Motley Fool Australia has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. This article contains general investment advice only (under AFSL 400691). Authorised by Scott Phillips.

More on Healthcare Shares

A group of people in a corporate setting do a collective high five.
Healthcare Shares

Sigma share price up 9% on first day of trading post-merger with Chemist Warehouse

It's a post-merger party for Sigma Healthcare shareholders today.

Read more »

Cropped shot of an attractive young female scientist working on her computer in the laboratory.
Healthcare Shares

Up 135% in a year, why this ASX 200 healthcare stock is 'on track to become a truly great Australian success'

A leading fund manager expects more strong performance from this surging ASX 200 healthcare stock.

Read more »

Stethoscope with a piggy bank in the middle.
Healthcare Shares

With revenue up 9%, why does the NIB share price keep falling?

Will the ailing share price make a full recovery?

Read more »

Six smiling health workers pose for a selfie.
Healthcare Shares

Guess how much $5,000 invested in Sigma shares in December 2023 is worth now!

Sigma shares have soared over the past year alongside the merger with Chemist Warehouse.

Read more »

Woman with headphones on relaxing and looking at her phone happily.
Healthcare Shares

Everything you need to know about the latest CSL dividend

CSL's latest dividend should be turning investors' heads...

Read more »

thoughtful investor sitting at computer
Healthcare Shares

What are brokers saying about CSL shares after the selloff?

The biotech giant's shares hit a 52-week low yesterday. Is this a buying opportunity?

Read more »

A man looking at his laptop and thinking.
Earnings Results

CSL shares push higher despite 'mixed result with headline misses'

There were positives and negatives in the company's first half performance.

Read more »

Scientists working in the laboratory and examining results.
Earnings Results

CSL share price on watch amid $2.1b half year profit and dividend hike

Let's see how this biotech giant performed during the first half of FY 2025.

Read more »