- Neuren Pharmaceuticals (NEU) releases its September quarterly report, highlighting the progress with its upcoming clinical trials
- The phase three LAVENDER trial in people with Rett syndrome is progressing and top-line results are expected before the end of the year
- Additionally, the company is waiting on FDA approval to undertake phase two trials of NNZ-2591 in people with Pitt Hopkins, Angelman and Phelan-McDermid syndromes
- Neuren ended the quarter with total available funding of $33.59 million, representing 8.9 quarters of use if spending levels remain the same
- Shares in Neuren were down 2.03 per cent on the market, trading for $1.81 at 12:30 pm AEDT
Neuren Pharmaceuticals (NEU) has released its September quarterly report, highlighting the progress with its upcoming clinical trials.
Rett syndrome phase three trial
In early August, Neuren’s US partner, Acadia Pharmaceuticals, completed enrolment for the phase three LAVENDER trial of trofinetide in Rett syndrome.
Rett syndrome is a brain disorder caused by a gene mutation which typically becomes apparent in female once they’ve reached six to 18 months of age.
Symptoms include problems with language, motor skills, breathing and development. Girls may also experience seizures, scoliosis, tremors and teeth grinding.
The 12 week trial is double-blind and placebo-controlled, and recruited roughly 180 females aged between five to 20 years.
A few weeks later, Acadia began the DAFFODIL study of trofinetide in girls aged between two to five years.
DAFFODIL is a 12-week, open-label, safety, tolerability and pharmacokinetics study of trofinetide.
Top-line results for the LAVENDER trial are due before the end of the year and positive results are expected to enable Neuren to engage with commercial partners in Europe and Asia.
NNZ-2591
In September, Neuren submitted three Investigational New Drug (IND) applications to the US Food and Drug Administration (FDA) to begin phase two trials in Phelan-McDermid, Angelman and Pitt Hopkins syndromes.
All three of these syndromes are neurological disorders, each characterised by impaired connections and signalling between brain cells.
Typical symptoms include problems with motor skills, language, behaviour and brain development.
Neuren is now waiting on confirmation of FDA requirements to begin phase two trials in all three syndromes.
The same month, the FDA granted Neuren Orphan Drug designation for NNZ-2591 in the treatment of Prader-Willi syndrome (PWS).
A phase two trial will begin mid next year and foundational work for phase three trials for all four syndromes will also begin.
Finances
For the three months ending September 30, Neuren burnt through $3.77 million with the majority going towards research and development.
The company ended the quarter with total available funding of $33.59 million, representing 8.9 quarters of use if spending levels stay the same.
Shares in Neuren were down 2.03 per cent on the market, trading for $1.81 at 12:30 pm AEDT.