Neuren's Angelman drug shows promise. Pic via Getty
Health & Biotech
Market darling Neuren Pharmaceuticals (ASX:NEU) has just reported promising results from its Phase 2 clinical trial of NNZ-2591 for Angelman syndrome, a rare genetic disorder that mainly affects the nervous system and severely affects development.
The trial involved children aged 3 to 17 and aimed to evaluate the drug’s safety and effectiveness.
The results showed that NNZ-2591 led to significant improvements in several key areas.
Children who received the drug showed better communication, behaviour, cognition, and motor skills compared to their baseline conditions.
This was measured using specialised tools designed for Angelman syndrome.
For instance, the Clinical Global Impression of Improvement (CGI-I) score, which assesses how much a child’s overall condition has improved, was statistically significant with a mean score of 3.0. This indicates that most children had noticeable improvements.
The Caregiver Overall Impression of Change (CIC) score, which reflects caregivers’ perceptions of changes, also showed meaningful improvements.
The drug was administered orally as a liquid twice daily, and it was well tolerated, with no serious adverse effects noted. This suggests that NNZ-2591 is both safe and effective for treating Angelman syndrome.
However, despite these positive results, Neuren’s stock dropped by 7% this morning.
There are a few possible reasons for this reaction. For example, investors might have expected even more dramatic results or clearer evidence of long-term benefits.
Avita Medical (ASX:AVH) has announced its Q2 financial results.
The company reported a strong commercial revenue of $15.1 million in the quarter, a 29% increase from the previous year, with gross profit margin of 86.2%.
During the quarter, the FDA approved the company’s RECELL GO treatment (for thermal burn wounds and “full-thickness skin defects” – per Avita’s website), and the first case of using the treatment was completed shortly after.
Avita also submitted a new application for the RECELL GO mini, which is designed for smaller wounds and will be reviewed by the FDA soon due to its Breakthrough Device status.
Avita anticipates FDA approval for RECELL GO mini by December 2024, and expects to publish results from its ongoing studies by the end of the year.
Looking ahead to Q3, Avita forecasts revenue between $19 and $20 million, and it expects full-year revenue to be between $68 and $70 million.
The company says it aims to break even and achieve profitability by the end of Q3 of 2025.
Meanwhile, IDT Australia (ASX:IDT) has landed a new contract with Sanofi Australia, valued between $2.5 million and $4 million.
This contract involves preclinical work, including formulating, developing, and manufacturing Sanofi’s messenger RNA (mRNA) technology for its clinical trials.
This follows an earlier Master Service Agreement (MSA) with Sanofi, announced on April 16, which allows Sanofi to select various services from IDT and set up additional projects.
Health & Biotech
Health & Biotech
Health & Biotech
