PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients
PTC Therapeutics announced that its global Phase 2 placebo-controlled CardinALS study for utreloxastat in ALS patients failed to meet its primary and secondary efficacy endpoints. While the study showed a modest numerical benefit on the primary endpoint and correlation with lowering plasma neurofilament light chain (NfL), statistical significance was not achieved (p= 0.52). The drug was safe and well-tolerated, but due to lack of efficacy and biomarker signal, further development has been discontinued.
PTC Therapeutics ha annunciato che il suo studio globale di fase 2 controllato con placebo, CardinALS, per l'utreloxastat nei pazienti affetti da SLA non ha raggiunto i suoi obiettivi di efficacia primari e secondari. Sebbene lo studio abbia mostrato un modesto beneficio numerico sul risultato primario e una correlazione con la riduzione della catena leggera della neurofilamenta plasmica (NfL), non è stata raggiunta significatività statistica (p= 0.52). Il farmaco si è dimostrato sicuro e ben tollerato, ma a causa della mancanza di efficacia e del segnale biomarcatore, lo sviluppo ulteriore è stato interrotto.
PTC Therapeutics anunció que su estudio global de fase 2 controlado con placebo, CardinALS, para el utreloxastat en pacientes con ELA no cumplió con sus objetivos primarios y secundarios de eficacia. Aunque el estudio mostró un beneficio numérico modesto en el objetivo primario y una correlación con la reducción de la cadena ligera de neurofilamentos en plasma (NfL), no se alcanzó significación estadística (p= 0.52). El medicamento fue seguro y bien tolerado, pero debido a la falta de eficacia y señal de biomarcador, se ha interrumpido su desarrollo.
PTC Therapeutics는 ALS 환자를 위한 utreloxastat의 글로벌 2상 위약 대조 CardinALS 연구가 주요 및 보조 유효성 목표를 달성하지 못했다고 발표했습니다. 연구는 주요 목표에 대해 미미한 수치적 이점을 나타내고 혈장 신경섬유 경량 체인(NfL)의 감소와 상관관계가 있었지만, 통계적 유의성(p= 0.52)은 달성되지 않았습니다. 이 약물은 안전하고 잘 견딜 수 있었지만, 유효성과 바이오마커 신호의 부족으로 인해 추가 개발이 중단되었습니다.
PTC Therapeutics a annoncé que son étude mondiale de phase 2 contrôlée par placebo, CardinALS, pour l'utreloxastat chez les patients atteints de SLA n'a pas atteint ses objectifs d'efficacité primaires et secondaires. Bien que l'étude ait montré un modeste bénéfice numérique sur l'objectif principal et une corrélation avec la réduction de la chaîne légère de neurofilaments plasmatiques (NfL), la signification statistique n'a pas été atteinte (p= 0.52). Le médicament était sûr et bien toléré, mais en raison du manque d'efficacité et du signal biomarqueur, le développement a été interrompu.
PTC Therapeutics gab bekannt, dass die globale Phase-2-Studie CardinALS für Utreloxastat bei ALS-Patienten, die placebo-kontrolliert war, ihre primären und sekundären Wirksamkeitsendpunkte nicht erreicht hat. Während die Studie einen bescheidenen numerischen Vorteil beim primären Endpunkt zeigte und eine Korrelation mit der Senkung der plasmakompatiblen Neurofilament-Leichtkette (NfL) aufwies, wurde keine statistische Signifikanz erreicht (p= 0.52). Das Medikament war sicher und gut verträglich, aber aufgrund des Mangels an Wirksamkeit und Biomarkern wurde die weitere Entwicklung eingestellt.
- Drug demonstrated safety and good tolerability in trial patients
- Failed to meet primary endpoint of slowing disease progression
- Failed to achieve statistical significance on secondary efficacy endpoints
- Development program discontinued due to lack of efficacy
- Investment in trial development and resources resulted in unsuccessful outcome
Insights
The failure of PTC Therapeutics' CardinALS Phase 2 trial represents a significant setback in the ALS therapeutic landscape. The trial's inability to meet both primary and secondary endpoints, despite showing a modest numerical benefit and correlation with NfL biomarker reduction, highlights the challenging nature of developing effective ALS treatments. The p-value of 0.52 indicates the results were far from statistical significance, which typically requires p<0.05.
This outcome has immediate implications for PTC's pipeline and resource allocation, as the company has decided to discontinue further development of utreloxastat. The termination of this program will likely lead to cost savings but also removes a potential revenue stream. For ALS patients, this represents another disappointing outcome in the search for effective treatments in a disease area with significant unmet medical needs.
This negative trial outcome will likely impact PTC Therapeutics' market position and near-term prospects. With a market cap of
- Study failed to meet primary and secondary efficacy endpoints -
"We wish to thank all of the patients, their families and physicians who participated in the CardinALS trial," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "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."
Utreloxastat was demonstrated to be safe and well tolerated in the CardinALS trial. However, due to the lack of efficacy and biomarker signal, further development is not planned at this time.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis, sometimes referred to as ALS, motor neuron disease, or Lou Gehrig's disease, is a rare, progressive, and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord.1,2 ALS becomes life-threatening as people with this disease ultimately lose the ability to move, speak, eat, and breathe.1
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn and X.
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References:
[1] Kiernan MC, et al. Amyotrophic lateral sclerosis. The Lancet, 2011; 377(9769):942-955.
[2] Dhasmana S, et al. The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder, Life Sciences 2022;288:120156. https://doi.org/10.1016/j.lfs.2021.120156.
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SOURCE PTC Therapeutics, Inc.
FAQ
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