Amylyx Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on AMX0114 Phase 1 Clinical Trial for the Treatment of Amyotrophic Lateral Sclerosis
Amylyx Pharmaceuticals (NASDAQ: AMLX) announced that the FDA has lifted the clinical hold on its Phase 1 clinical trial of AMX0114, an antisense oligonucleotide targeting calpain-2 for ALS treatment. The company will proceed with opening U.S. sites for screening, enrollment, and dosing.
The Phase 1 LUMINA clinical trial is expected to begin in Canada in early 2025. This multicenter, randomized, placebo-controlled trial will evaluate AMX0114's safety and biological activity, involving approximately 48 ALS patients randomized 3:1 to receive either AMX0114 or placebo via intrathecal administration once every four weeks for up to 4 doses. The trial will assess ALS biomarkers, including changes in neurofilament light levels.
The company expects to obtain early cohort data from LUMINA in 2025.
Amylyx Pharmaceuticals (NASDAQ: AMLX) ha annunciato che la FDA ha revocato il fermo clinico sul suo studio clinico di Fase 1 relativo a AMX0114, un oligonucleotide antisenso mirato a calpain-2 per il trattamento della SLA. L'azienda procederà con l'apertura di siti negli Stati Uniti per screening, arruolamento e somministrazione.
Il trial clinico di Fase 1 LUMINA dovrebbe iniziare in Canada all'inizio del 2025. Questo studio multicentrico, randomizzato e controllato con placebo valuterà la sicurezza e l'attività biologica di AMX0114, coinvolgendo circa 48 pazienti con SLA randomizzati 3:1 per ricevere AMX0114 o placebo tramite somministrazione intratecale una volta ogni quattro settimane per un massimo di 4 somministrazioni. Lo studio valuterà i biomarcatori della SLA, inclusi i cambiamenti nei livelli della light neurofilament.
L'azienda prevede di ottenere dati precoci dal coorte di LUMINA nel 2025.
Amylyx Pharmaceuticals (NASDAQ: AMLX) anunció que la FDA ha levantado la suspensión clínica de su ensayo clínico de Fase 1 para AMX0114, un oligonucleótido antisentido dirigido a la calpaína-2 para el tratamiento de la ELA. La compañía procederá a abrir sitios en EE. UU. para la evaluación, el enrolamiento y la dosificación.
Se espera que el ensayo clínico de Fase 1 LUMINA comience en Canadá a principios de 2025. Este ensayo multicéntrico, aleatorizado y controlado con placebo evaluará la seguridad y la actividad biológica de AMX0114, involucrando aproximadamente 48 pacientes con ELA que serán aleatorizados en una proporción de 3:1 para recibir AMX0114 o placebo mediante administración intratecal una vez cada cuatro semanas hasta un máximo de 4 dosis. El ensayo evaluará biomarcadores de ELA, incluidos los cambios en los niveles de neurofilamento ligero.
La empresa espera obtener datos de cohortes tempranas de LUMINA en 2025.
아밀렉스 제약(Amylyx Pharmaceuticals) (NASDAQ: AMLX)이 FDA가 ALS 치료를 위한 칼파인-2를 표적으로 하는 AMX0114의 1상 임상 시험에 대한 임상 보류를 해제했다는 발표를 하였습니다. 이 회사는 스크리닝, 등록 및 투약을 위해 미국 내 사이트를 개설할 예정이다.
1상 LUMINA 임상 시험은 2025년 초 캐나다에서 시작될 예정입니다. 이 다기관, 무작위, 위약 대조 시험은 AMX0114의 안전성과 생물학적 활동을 평가할 것이며, 대략 48명의 ALS 환자가 AMX0114 또는 위약을 4주마다 1회, 총 4회의 경막내 투여를 받기 위해 3:1로 무작위 배정될 것입니다. 시험에서는 신경 필라멘트 경량 수준의 변화 등 ALS 바이오마커를 평가합니다.
회사는 2025년에 LUMINA의 조기 집단 데이터를 확보할 것으로 기대하고 있습니다.
Amylyx Pharmaceuticals (NASDAQ: AMLX) a annoncé que la FDA a levé la suspension clinique de son essai clinique de Phase 1 portant sur AMX0114, un oligonucléotide antisens ciblant la calpaïne-2 pour le traitement de la SLA. L'entreprise procédera à l'ouverture de sites aux États-Unis pour le dépistage, le recrutement et la dose.
L'essai clinique de Phase 1 LUMINA devrait commencer au Canada au début de 2025. Cet essai multicentrique, randomisé et contrôlé par placebo évaluera la sécurité et l'activité biologique d'AMX0114, impliquant environ 48 patients atteints de SLA randomisés dans un rapport de 3:1 pour recevoir soit AMX0114 soit un placebo par administration intrathécale une fois toutes les quatre semaines jusqu'à un maximum de 4 doses. L'essai évaluera les biomarqueurs de la SLA, y compris les changements des niveaux de neurofilaments légers.
L'entreprise s'attend à obtenir des données initiales de cohorte de LUMINA en 2025.
Amylyx Pharmaceuticals (NASDAQ: AMLX) gab bekannt, dass die FDA die klinische Sperre für seine Phase-1-Studie zu AMX0114 aufgehoben hat, einem Antisense-Oligonukleotid, das auf Calpain-2 zur Behandlung von ALS abzielt. Das Unternehmen wird mit der Eröffnung von US-Standorten für Screening, Rekrutierung und Dosierung fortfahren.
Die Phase-1-Studie LUMINA soll Anfang 2025 in Kanada beginnen. Diese multizentrische, randomisierte, placebokontrollierte Studie wird die Sicherheit und biologische Aktivität von AMX0114 bewerten und umfasst etwa 48 ALS-Patienten, die im Verhältnis 3:1 randomisiert entweder AMX0114 oder Placebo über intrathekale Verabreichung einmal alle vier Wochen für bis zu 4 Dosen erhalten. Die Studie wird ALS-Biomarker bewerten, einschließlich Veränderungen der Neurofilament-Leichtwerte.
Das Unternehmen rechnet damit, 2025 frühzeitige Kohortendaten von LUMINA zu erhalten.
- FDA lifted clinical hold on Phase 1 trial of AMX0114
- Trial expansion across North America
- Clear timeline for trial initiation in Canada (early 2025)
- Defined trial protocol with 48 participants
- Trial results not expected until 2025
- participant size (48 people)
Insights
The FDA's decision to lift the clinical hold on AMX0114's Phase 1 trial represents a critical regulatory milestone for Amylyx. The LUMINA trial design, involving 48 participants with a 3:1 randomization ratio, is statistically robust for an early-phase safety study. The focus on neurofilament light (NfL) as a biomarker is particularly strategic - NfL levels are increasingly recognized as a reliable indicator of neuronal damage and disease progression in ALS.
The mechanism targeting calpain-2 through antisense oligonucleotide technology is scientifically sound. Calpain-2's role in axonal degeneration and its interaction with key proteins like neurofilament, tau and TDP43 positions AMX0114 as a potentially differentiated therapeutic approach. The intrathecal administration route, while more invasive than oral delivery, ensures direct CNS exposure - important for ASO therapeutics.
For a company with a
The trial resumption significantly de-risks Amylyx's clinical development timeline. The multiple ascending dose design with four-week dosing intervals allows for careful safety monitoring while maintaining therapeutic exposure. The inclusion of both U.S. and Canadian sites enhances recruitment potential and geographical diversity of the patient population.
Key trial elements worth noting:
- 48-patient enrollment target is appropriate for Phase 1 safety assessment
- 4-dose maximum exposure provides adequate duration for initial safety and biomarker evaluation
- Intrathecal administration every 4 weeks balances patient burden with therapeutic needs
• Company will move forward with trial sites across
The announcement comes as Amylyx expects to initiate the Phase 1 LUMINA clinical trial in
LUMINA will also assess ALS biomarkers, including change from baseline in neurofilament light (NfL) levels. Approximately 48 people living with ALS will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks for a total of up to 4 doses.
“Based on several preclinical efficacy studies and what is known about the central role of calpain-2 in the process of axonal degeneration, we believe AMX0114 has the potential to be a treatment for ALS and other diseases,” said Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx. “Calpain-2 is a well-established target in a number of neurological diseases and a protease known to cleave many substrates including neurofilament, tau, and TDP43 proteins. In addition to being considered an essential protein in the process of axonal degeneration, calpain-2 has been repeatedly linked to neurofilament biology in published studies. We are dedicated to investigating therapies to potentially treat people living with ALS, and we are excited to work with study investigators and clinical trial sites across
Amylyx continues to anticipate early cohort data from LUMINA in 2025.
About AMX0114
AMX0114 is an investigational antisense oligonucleotide (ASO) targeting calpain-2 (CAPN2) for the potential treatment of ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was well-tolerated in in vivo preclinical safety studies.
About LUMINA
The Phase 1 LUMINA study (NCT06665165) is a multicenter, randomized, placebo-controlled, multiple ascending dose trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in 48 adults living with ALS. LUMINA will also assess change from baseline in calpain-2 levels, neurofilament light (NfL) levels, and other pharmacodynamic biomarkers of ALS.
About ALS
Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than
About Amylyx Pharmaceuticals
Amylyx is committed to the discovery and development of new treatment options for communities with high unmet needs, including people living with serious and fatal diseases. The Company has preclinical or clinical development programs underway in neurodegenerative, neuroendocrine, and endocrine diseases. Since its founding, Amylyx has been guided by science to address unanswered questions, keeping communities at the heart and center of all decisions. Amylyx is headquartered in
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’ expectations regarding: the potential for AMX0114 as a treatment for ALS and the planned initiation of a trial evaluating AMX0114 in ALS. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities; Amylyx’ ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx’ ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’ operations, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
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Source: Amylyx Pharmaceuticals, Inc.
FAQ
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