PTC Therapeutics Ends Utreloxastat Development After Rare Neurodegenerative Disease Trial Misses Goal, Analyst Maintains Outperform With Focus On Sepiapterin, Huntington's Programs

On Tuesday, PTC Therapeutics Inc PTCT revealed data from the global Phase 2 placebo-controlled CardinALS study of Utreloxastat in amyotrophic lateral sclerosis patients.

The study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.

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While modest numerical benefit was recorded on the primary endpoint, and a favorable clinical effect was correlated with lowering plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints.

Amyotrophic lateral sclerosis (ALS), sometimes referred to as motor neuron disease or Lou Gehrig’s disease, is a rare neurodegenerative disease that affects motor neurons in the brain and spinal cord.

Utreloxastat was demonstrated to be safe and well-tolerated in the CardinALS trial. However, further development is not planned due to the lack of efficacy and biomarker signal.

William Blair notes that while the CardinALS trial results were disappointing, the program was not a major factor in its investment thesis. As a result, ending the program does not affect the firm’s Outperform rating on the stock.

The analyst highlights the potential approval and launch of sepiapterin as a key growth driver over the next year, particularly in treating Phenylketonuria (PKU). William Blair believes the market is undervaluing this opportunity, with a PDUFA decision expected in July.

The analyst also points to growing investor interest in the company’s Huntington’s disease program. Given the strong commercial prospects of this small-molecule therapy, positive discussions with regulators about an accelerated approval pathway for PTC-518 could significantly boost the stock. A Type C meeting with the FDA is planned for the fourth quarter to discuss the regulatory pathway.

Additionally, the company aims to file a U.S. marketing application for vatiquinone in Friedreich’s ataxia (FA) by late 2024. William Blair expects approval based on the drug’s clinical data and the FDA’s recent neurology approvals, especially as no treatments exist for pediatric FA patients.

On Wednesday, PTC Therapeutics agreed to sell its Rare Pediatric Disease Priority Review Voucher for $150 million.

PTC was granted the voucher along with the FDA approval of Kebilidi (eladocagene exuparvovec-tneq) for children and adults with AADC deficiency.

Price Action: PTCT stock is down 1.98% at $45.10 during the premarket session at last check Wednesday.

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