Amylyx Pharmaceuticals Receives Orphan Drug Designation From the European Commission for AMX0035 for the Treatment of Wolfram Syndrome
Amylyx Pharmaceuticals (NASDAQ: AMLX) has received Orphan Drug Designation from the European Commission for AMX0035 to treat Wolfram syndrome. This follows a similar designation by the FDA in 2020. Wolfram syndrome is a rare, progressive disease characterized by childhood-onset diabetes and neurodegeneration, with no approved treatments. Interim data from the Phase 2 HELIOS trial showed AMX0035 improved pancreatic function and glycemic control in 8 participants at Week 24. The drug was generally well-tolerated, with patients reporting disease improvement or stabilization. Topline data for all 12 participants is expected in fall 2024.
Amylyx Pharmaceuticals (NASDAQ: AMLX) ha ricevuto la Designazione di Farmaco Orfano dalla Commissione Europea per AMX0035 per il trattamento della sindrome di Wolfram. Questo segue una designazione simile da parte della FDA nel 2020. La sindrome di Wolfram è una malattia rara e progressiva caratterizzata da diabete insorto in età pediatrica e neurodegenerazione, senza trattamenti approvati. I dati interim del trial HELIOS di Fase 2 hanno mostrato che AMX0035 ha migliorato la funzione pancreatica e il controllo glicemico in 8 partecipanti alla Settimana 24. Il farmaco è stato generalmente ben tollerato, con i pazienti che hanno riportato un miglioramento o una stabilizzazione della malattia. I dati topline per tutti i 12 partecipanti sono attesi per autunno 2024.
Amylyx Pharmaceuticals (NASDAQ: AMLX) ha recibido la Designación de Medicamento Huérfano de la Comisión Europea para AMX0035 para tratar el síndrome de Wolfram. Esto sigue una designación similar por parte de la FDA en 2020. El síndrome de Wolfram es una enfermedad rara y progresiva caracterizada por diabetes de inicio infantil y neurodegeneración, sin tratamientos aprobados. Los datos interinos del ensayo HELIOS de Fase 2 mostraron que AMX0035 mejoró la función pancreática y el control glicémico en 8 participantes a la Semana 24. El medicamento fue generalmente bien tolerado, con pacientes que informaron mejora o estabilización de la enfermedad. Se esperan los datos topline para los 12 participantes en otoño de 2024.
Amylyx Pharmaceuticals (NASDAQ: AMLX)는 AMX0035에 대한 고아약 지정을 유럽연합(EU)으로부터 받았습니다. 이는 2020년 FDA로부터 받은 유사한 지정에 연이어 이루어진 것입니다. Wolfram 신드롬은 아동기 발병 당뇨병과 신경 퇴행으로 특징 지워지는 드문 진행성 질환으로, 승인된 치료법이 없습니다. 2상 HELIOS 시험의 중간 데이터는 24주차에 8명의 참가자에서 AMX0035가 췌장 기능과 당 조절을 개선했음을 보여주었습니다. 이 약물은 일반적으로 잘 견뎌졌으며, 환자들은 질병의 개선 또는 안정화가 있었다고 보고했습니다. 12명의 전 참가자에 대한 최종 데이터는 2024년 가을에 발표될 예정입니다.
Amylyx Pharmaceuticals (NASDAQ: AMLX) a reçu la désignation de médicament orphelin de la Commission européenne pour AMX0035 afin de traiter le syndrome de Wolfram. Cela fait suite à une désignation similaire de la FDA en 2020. Le syndrome de Wolfram est une maladie rare et progressive caractérisée par un diabète apparenté à l’enfance et une neurodégénérescence, sans traitements approuvés. Les données intermédiaires de l'essai de phase 2 HELIOS ont montré qu'AMX0035 améliorait la fonction pancréatique et le contrôle glycémique chez 8 participants à la semaine 24. Le médicament a été généralement bien toléré, les patients rapportant une amélioration ou une stabilisation de la maladie. Les données finales pour les 12 participants sont attendues pour l'automne 2024.
Amylyx Pharmaceuticals (NASDAQ: AMLX) hat von der Europäischen Kommission die Orphan Drug Designation für AMX0035 zur Behandlung des Wolfram-Syndroms erhalten. Dies folgt einer ähnlichen Bezeichnung der FDA im Jahr 2020. Das Wolfram-Syndrom ist eine seltene, fortschreitende Erkrankung, die durch Diabetes im Kindesalter und Neurodegeneration gekennzeichnet ist und für die es keine zugelassenen Behandlungen gibt. Interimdaten aus der Phase-2-Studie HELIOS zeigten, dass AMX0035 die Bauchspeicheldrüsenfunktion und die glykämische Kontrolle bei 8 Teilnehmern in Woche 24 verbesserte. Das Medikament wurde im Allgemeinen gut vertragen, und die Patienten berichteten von einer Verbesserung oder Stabilisierung der Erkrankung. Die Topline-Daten für alle 12 Teilnehmer werden im Herbst 2024 erwartet.
- Received Orphan Drug Designation from European Commission for AMX0035 in Wolfram syndrome
- Positive interim data from Phase 2 HELIOS trial showing improved pancreatic function and glycemic control
- All 8 participants in interim analysis met prespecified responder criteria for disease improvement or stabilization
- AMX0035 was generally well-tolerated in all participants
- None.
Insights
The European Commission's Orphan Drug Designation for AMX0035 in Wolfram syndrome is a significant development for Amylyx Pharmaceuticals. This designation, following the FDA's similar recognition in 2020, underscores the potential of AMX0035 in addressing a rare and severe condition with no current approved treatments.
The interim results from the Phase 2 HELIOS trial are particularly noteworthy:
- Improvement in pancreatic function and glycemic control, as evidenced by changes in C-peptide and HbA1c levels
- Disease stabilization or improvement reported by both patients and clinicians
- Potential visual improvements in some participants
These outcomes are remarkable given the progressive nature of Wolfram syndrome. The stabilization or improvement in multiple domains suggests that AMX0035 may be effectively targeting the underlying pathophysiology of the disease, particularly endoplasmic reticulum stress.
However, it's important to note that these results are based on an interim analysis of only eight participants. The upcoming topline data from all 12 participants will provide a more comprehensive understanding of the drug's efficacy and safety profile. If these positive trends continue, AMX0035 could potentially become the first approved treatment for Wolfram syndrome, addressing a significant unmet medical need.
The mechanism of action of AMX0035, targeting both ER stress and mitochondrial dysfunction, aligns well with the pathophysiology of Wolfram syndrome. This targeted approach, combined with the positive interim results, suggests a promising path forward for this therapy.
The Orphan Drug Designation from the European Commission for AMX0035 in Wolfram syndrome represents a significant milestone for Amylyx Pharmaceuticals. This designation, coupled with the FDA's previous recognition, positions AMX0035 favorably in both major pharmaceutical markets.
Key financial implications include:
- Market exclusivity: Orphan Drug Designation typically grants 10 years of market exclusivity in the EU upon approval, providing a substantial competitive advantage.
- Development incentives: The designation may offer financial benefits such as reduced regulatory fees and potential tax credits, lowering the overall development costs.
- Market potential: While Wolfram syndrome is rare, orphan drugs often command premium pricing, potentially leading to significant revenue streams if approved.
The positive interim results from the HELIOS trial further strengthen Amylyx's position. If the full dataset continues to show efficacy and safety, it could accelerate the path to market approval. This would be particularly impactful given the lack of approved treatments for Wolfram syndrome.
Investors should note that Amylyx is expanding AMX0035's potential beyond its current approval for ALS. This diversification strategy could mitigate risk and open up new revenue streams. However, it's important to remember that drug development, especially for rare diseases, carries inherent risks and uncertainties.
The anticipated fall 2024 topline data release will be a critical event for Amylyx. Positive results could significantly boost investor confidence and potentially lead to partnerships or increased institutional interest. Conversely, any setbacks could impact the company's valuation and future prospects.
- New designation follows the
- Topline data for all 12 participants from Phase 2 HELIOS trial studying impact of AMX0035 on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome anticipated fall 2024
Wolfram syndrome is a prototypical disease of endoplasmic reticulum (ER) stress that is rare, progressive, and monogenic and is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, deafness, diabetes insipidus, and neurodegeneration. There are currently no drugs approved for Wolfram syndrome, and many people with the disease die prematurely with severe neurological disabilities.
The FDA previously granted AMX0035 Orphan Drug Designation for the treatment of Wolfram syndrome in 2020. The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention, or diagnosis of rare, life-threatening, or chronically debilitating conditions where the product may represent a significant benefit over existing treatments.
Amylyx recently presented positive data from an interim analysis of its Phase 2 HELIOS study, including eight participants with Wolfram syndrome assessed at Week 24, which demonstrated that AMX0035 improved pancreatic function and glycemic control, as measured by C-peptide, HbA1c, and other markers of glucose metabolism. All eight participants met prespecified responder criteria showing either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change (PGIC) and the Clinical Reported Global Impression of Change (CGIC) scales. The majority of participants reported some improvement in vision. In Wolfram syndrome, progressive decline and worsening of outcomes would have been expected on all measures, so disease improvement or stabilization alone is clinically meaningful. AMX0035 was generally well tolerated in all participants. The Company anticipates reporting topline data from all 12 participants at Week 24 this fall.
“Wolfram syndrome is a disease where there are well-defined measurable biomarkers, rigorous supporting preclinical data, and clear rationale for our potential therapy based on its mechanism of action. Specifically, Wolfram syndrome is considered a prototypical endoplasmic reticulum (ER) stress disorder because of the clear link between WFS1 mutations and ER stress. AMX0035 is believed to target ER stress and mitochondrial dysfunction,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “The interim data from HELIOS showed stabilization or even improvement across key outcomes at Week 24. HELIOS follows strong preclinical research with data showing clear effects in cellular and animal models. We look forward to reporting topline data from all participants at Week 24 this fall. We aim to address an urgent unmet medical need, given there are no approved treatment options for Wolfram syndrome.”
About Wolfram Syndrome
Wolfram syndrome is rare, progressive, monogenic disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. The prognosis of Wolfram syndrome is poor, and many people with the disease die prematurely with severe neurological disabilities. Literature suggests approximately 3,000 people are living with Wolfram syndrome in
Wolfram syndrome is often characterized as a prototypical disease of endoplasmic reticulum (ER) stress. ER stress and mitochondrial dysfunction are believed to drive the underlying disease pathophysiology in Wolfram syndrome. Individuals with Wolfram syndrome generally have mutations in the WFS1 gene, which encodes wolframin, a protein spanning the membrane of the ER. Wolframin is thought to play a role in protein folding and aid in the maintenance of ER function by regulating calcium levels. Loss of wolframin function leads to ER stress and impaired mitochondrial dynamics.
About AMX0035
AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the
About the HELIOS Trial
The HELIOS trial (NCT05676034) is a 12-participant, open-label Phase 2 trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with Wolfram syndrome.
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: interactions with regulatory authorities; the ongoing evaluation of AMX0035 in Wolfram syndrome, including that early-stage results may not reflect later-stage results and the timing for expected topline data in the HELIOS study; and the potential for AMX0035 to help people living with Wolfram syndrome. Any forward-looking statements in this press release 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 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 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.
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FAQ
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