Sometimes known under the name of Lou Gehrig’s disease, ALS is a progressive, and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord. Credit: Shutterstock / Michael Vi

New Jersey-based biopharma, PTC Therapeutics said its Phase II trial of utreloxastat for patients with amyotrophic lateral sclerosis (ALS) has failed to meet its primary endpoints, forcing the trial to be axed.

The placebo-controlled global Phase II trial (NCT05349721) failed to meet its primary endpoint of slowing disease progression by lowering plasma neurofilament light chains (NfL), a crucial biomarker used to determine the impact of the condition on a patient. Although there was no statistical improvement, PTC noted some modest numerical benefit recorded in the primary endpoint. Additionally, there was no statistical improvement in any of the trial’s secondary endpoints.

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Utreloxastat was safe and well tolerated, but the lack of efficacy means that the company will no longer be progressing the product.

Sometimes known under the name of Lou Gehrig’s disease, ALS is a progressive, and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord.

Matthew Klein, CEO of PTC Therapeutics, said: “We wish to thank all of the patients, their families and physicians who participated in the CardinALS trial. We are of course disappointed that we were not able to demonstrate treatment efficacy and provide a potential therapy that could address the significant unmet medical need of ALS patients.”

Following the Phase II failure the company went on to announce the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million, granted by the US Food and Drug Administration earlier in the year alongside the approval of Kebilidi (eladocagene exuparvovec-tneq), a gene therapy designed for the treatment of children and adults with Aromatic L-amino acid decarboxylase (AADC) deficiency. Selling off PRVs is a tactic often used by small biotechs to source cash to keep the company operational.

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Market forecasts in GlobalData’s Pharmaceutical Intelligence Centre detail how by the end of 2024 the overall market for ALS treatments is estimated to sit at a value of $118m with that figure estimated to rise to $1.3bn by the end of 2028.

GlobalDatais the parent company of Clinical Trials Arena.

Elsewhere in the field of ALS trials, MaaT Pharma is eyeing up a Phase II study of its immune system modulator after its Phase Ib trial in patients with amyotrophic lateral sclerosis (ALS) met all of its primary endpoints. Meanwhile, WideTrial will participate in a National Institutes of Health-funded initiative to provide pre-approval access to the investigational ALS drug, ibudilas.