Opening the first session of the 18th Bioshares Biotech Summit, Neuren Pharmaceuticals' CEO Jon Pilcher spoke of Neuren's success with trofinetide and NNZ-2591, and the commercial options on the table.
Pilcher devoted much of his presentation to discussing Neuren's options with its second drug candidate, NNZ-2591, which was the focus of the brief given.
Competitive landscape
Neuren has multiple opportunities to pursue with NNZ-2591, however the selection process starts with assessing the competitive landscape. With Rett syndrome, the power of getting to market first has been very obvious.
Neuren's key opportunities lie in Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS), where the global competitive landscape is entirely empty and NNZ-2591 can reach the market first.
In PMS, Neuren leads globally, with successful Phase II study results late last year. There are very few products in development for PMS, with the nearest having closed its Phase II trial several years ago. There are just over 1,700 patients identified in the US, although that figure could be more than 10 times higher.
Another key value driver is PTHS, for which Neuren completed a successful Phase II trial in mid-2024. The closest competitor – a research institute – is in Phase II but focusing only on gastrointestinal symptoms. There are virtually no other competitors.
This disease is rarer than PMS with around 500 patients identified in the US, but could be over 6,000. The number of patients identified in the last year in the US has grown by 30%.
Neuren is meeting with the FDA this quarter to discuss the Phase III pathway. Pilcher did not estimate what the Phase III program will look like but said there is a good precedent from Acadia's program in Rett syndrome with DAYBUE.
That involved a single 187 patient study for 12 weeks. Both primary endpoints needed to be positive. The trial was conducted in 21 sites in only the US. The trial took just two years to complete. This rolled into a 12 month open label safety study, where all participants were given DAYBUE, with continued access offered to patients after the study.
Of interest is that the Phase II results in PMS and PTHS were much better than with trofinetide in Rett syndrome (see table on previous page). This means the Phase III study could be considerably smaller.
More competitive is Angelman syndrome (AS), in which Ultragenyx and Ionis are planning to launch Phase III trials in FY2025, using RNA therapies.
Neuren's oral liquid NNZ-2591 addresses the symptoms of AS irrespective of the underlying mutation. Neuren revealed its successful Phase II AS top-line trial results for NNZ-2591 last week (see page 1).
Prader-Willi syndrome (PWS) is the most competitive of Neuren's indications, with one product NDA filed and two products in Phase III. All address hyperphagia and sleepiness, unlike the holistic treatment of NNZ-2591.
With respect to Fragile-X syndrome and Rett Syndrome, Acadia has the rights to develop NNZ-2591 for these indications. Pilcher said he would expect Acadia to develop the drug for these indications given the activity that has been seen with the compound so far.
Deal Potential Ahead
In 2018 Neuren licensed trofinetide to Acadia for US$10 million upfront with 10-15% royalties for the US market at the end of Phase II. Neuren had multiple options to license the rest-of-world rights earlier this year, signing with Acadia once again, but this time included US$100 million upfront and mid teen to low 20% royalties.
Pilcher drew a comparison to US biotech Reata Pharmaceuticals. Reata had one flagship, first-in-class orphan drug, targeting a neurology indication. It had just been approved in the US and was filed for approval in Europe. The patient population and pricing was similar to Rett syndrome and DAYBUE. Reata was acquired last year for US$7 billion by Biogen.
Pilcher said that there is a lot more interest from potential partners when you are closer to sales. However he indicated that the company will consider M&A or partnering at all stages ahead.
Another option is to license ex-US rights and sell direct into the US in the future, noting that Acadia launched DAYBUE with a team of just 50 people.
The immediate aim for Neuren however is to move into registration studies for NNZ-2591 for the treatment of PMS and PTHS.
Bioshares recommendation: Accumulate
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