- Neuren Pharmaceuticals (NEU) has posted a busy quarter of activity, and ended in a strong financial position even amid worldwide economic turmoil
- Advances were made across Neuren’s portfolio of novel therapies for childhood neurological disorders
- Trofinetide – a drug being trialled for Rett Syndrome – stands to gain a Priority Review Voucher upon marketing approval
- This voucher may be on-sold, and Neuren would take one-third of the sale price
- 2019 sales of the vouchers averaged about US$100 million each
- Neuren also announced compelling results in a Phase 1 trial for new drug NNZ-2591 in the treatment of Phelan-McDermid Syndrome
- The company also finished the quarter with a strong balance sheet, holding $13.4 million
- Neuren Pharmaceuticals is up 10.4 per cent to $1.75 per share
Neuren Pharmaceuticals (NEU) has posted a busy quarter of activity, and ended in a strong financial position even amid worldwide economic turmoil.
Neuro-active
Advances were made across Neuren’s portfolio of novel therapies for childhood neurological disorders.
Trofinetide
The biggest – and possibly most lucrative – progression was for the drug trofinetide, for potential use against Rett Syndrome.
Rett Syndrome is a neurodevelopmental disorder which almost exclusively affects females. Symptoms typically start around six to 18 months after birth and include decreased motor function and slowed physical development. It affects one in every 10,000 to 15,000 births worldwide.
Trofinetide has been granted U.S. Food & Drug Administration (FDA) Rare Paediatric Disease Designation, meaning Neuren and US partner ACADIA will be eligible to receive a Priority Review Voucher upon marketing approval. This voucher can be used to obtain FDA review of a New Drug Application for another product in an expedited period of 6 months.
The voucher may also be on-sold. Two voucher sales in 2019 fetched around US$100 million each. Neuren stands to earn one third of any sale under its agreement with ACADIA.
Neuren retains all rights to trofinetide outside the U.S., and has deferred any regional partnerships until Phase 3 trial results are returned. Neuren may use any data gained from this trial to shore up regulatory approvals and commercial partnerships outside the US, strengthening its bargaining hand.
NNZ-2591
Neuren also announced compelling results in a Phase 1 trial for new drug NNZ-2591 in the treatment of Phelan-McDermid Syndrome. The drug showed evidence of being dose-responsive, and the optimum dose was identified to continue Phase 2 studies.
Progress on both trofinetide and NNZ-2591 has now stalled due to challenges associated with COVID-19 and the strain on healthcare systems and resources. The programs are fully funded and ready to roll when activities are deemed reasonable to resume.
Finances
The company finished the quarter with a strong balance sheet and sufficient cash reserves to operate for around two years even amid trying global economic conditions. The company holds cash and cash equivalents of $13.4 million.
In an added bonus, Neuren gained around $1.1 million from its U.S. cash holdings with the weakening of the Australian dollar. This helped to substantially offset the company’s research and development expenditure, further firming up the bottom line.
With a couple of promising drug candidates in the pipeline and the potential for the highly lucrative sale of an FDA Priority Review Voucher, it seems Neuren is well placed to prosper. even during the worst pandemic and economic downturn in living memory.
Neuren Pharmaceuticals is up 10.4 per cent to $1.75 per share as at 2:32 pm AEST.