A new drug seeking to treat irritability associated with Autism Spectrum Disorder (ASD) could soon be making its way to clinical trials in healthy volunteers after Zelira Therapeutics (ASX: ZLD) concluded a positive round of discussions with the United States Food and Drug Administration (FDA) on a study design.

The proposed clinical trials are for Zelira’s HOPE autism drug with the FDA having provided a clear written response that provided essential clarity on multiple issues, particularly defining the indication for the treatment of irritability associated with ASD in patients with Phelan-McDermid Syndrome (PMS) and Smith-Magenis Syndrome (SMS).

The FDA provided guidance on the study design, aiming to evaluate the safety and pharmacokinetics of the proposed doses of ZEL-HOP1, ensuring a framework for further clinical development that would meet the regulator’s required data.

“The clarity provided by the FDA, especially regarding the indication and study design, is incredibly positive for our company and the HOPE SPV program,” said Dr. Oludare Odumosu, Managing Director of Zelira Therapeutics.

“This meeting represents a crucial step forward in our mission to develop effective treatments for autism-related irritability.”

The FDA meeting included detailed discussions on various aspects of the HOPE program, providing essential guidance for Zelira’s path forward if the Phase 1 trials are to progress to Phase 2 or beyond.

This positive development offers new light to Zelira’s journey to improve the quality of life for individuals affected by autism. The Company is now looking forward to receiving the official meeting notes from the FDA, which will guide the next steps in the IND submission process.

“We are eager to advance our HOPE program and are confident in the positive impact it will have on the lives of many,” added Dr Odumosu.

Zelira’s’ HOPE program is a dedicated initiative focused on developing treatments for irritability associated with ASD. The program’s advancement to the IND submission stage marks a significant step forward in the clinical development process.

Irritability is a common condition associated with ASD, characterised by severe temper tantrums, aggression, self-injury, and sudden mood changes. These behaviours stem from difficulties in communication, sensory sensitivities, and challenges in processing social interactions. For individuals with ASD, irritability can manifest as intense frustration and emotional outbursts, often triggered by minor changes in routine, sensory overload, or difficulty expressing needs and desires.

The impact of irritability on the quality of life for those with ASD is profound. It can hinder daily functioning, limit participation in social activities, and create barriers to education and employment opportunities. These challenges contribute to heightened anxiety and stress levels for the individual, exacerbating the cycle of irritability and emotional distress.

For families and caregivers, managing irritability in a loved one with ASD can be equally challenging. It often leads to emotional strain, disrupted family dynamics, and increased caregiving demands. The need for constant vigilance and intervention to prevent self-harm or harm to others can result in caregiver burnout and reduced overall well-being.

For the quarter ended 31 March 2024, Zelira reported net operating cash outflows of $1.2 million, primarily applied to its research and development initiatives. At the time, Zelira had $118k of cash on hand but this was recently propped up by Zelira’s Chairman, Osagie Imasogie, who issued the Company a USD $1.4 million unsecured loan to progress the HOPE SPV clinical trial as a sign of internal confidence behind the drug.