Zilganersen granted U.S. FDA Fast Track designation for people living with Alexander disease
Ionis Pharmaceuticals (Nasdaq: IONS) announced that the U.S. FDA has granted Fast Track designation to zilganersen for treating Alexander disease (AxD), an ultra-rare, progressive, and fatal neurological disorder. Zilganersen is the first investigational medicine in clinical development for adults and children with AxD. This designation aims to expedite the development and review of medicines addressing serious conditions with unmet medical needs.
The pivotal Phase 1-3 study of zilganersen has completed enrollment across 13 sites in eight countries. Topline data from this study is expected in the second half of 2025. Ionis designed zilganersen to target the underlying cause of AxD and potentially improve patient functioning. The company looks forward to working closely with the FDA to potentially bring forward the first approved AxD treatment.
Ionis Pharmaceuticals (Nasdaq: IONS) ha annunciato che la FDA degli Stati Uniti ha concesso la designazione Fast Track a zilganersen per il trattamento della malattia di Alexander (AxD), un disturbo neurologico ultra-raro, progressivo e fatale. Zilganersen è il primo farmaco in fase di sviluppo clinico per adulti e bambini con AxD. Questa designazione mira ad accelerare lo sviluppo e la revisione di farmaci che affrontano condizioni gravi con bisogni medici non soddisfatti.
Lo studio pivotale di Fase 1-3 di zilganersen ha completato l'arruolamento in 13 centri in otto paesi. Si prevede che i dati preliminari di questo studio siano disponibili nella seconda metà del 2025. Ionis ha progettato zilganersen per affrontare la causa sottostante dell'AxD e potenzialmente migliorare il funzionamento dei pazienti. L'azienda è ansiosa di collaborare strettamente con la FDA per poter eventualmente portare avanti il primo trattamento approvato per l'AxD.
Ionis Pharmaceuticals (Nasdaq: IONS) anunció que la FDA de EE. UU. ha otorgado la designación de Fast Track a zilganersen para el tratamiento de la enfermedad de Alexander (AxD), un trastorno neurológico ultra-raro, progresivo y mortal. Zilganersen es el primer medicamento en investigación en desarrollo clínico para adultos y niños con AxD. Esta designación busca acelerar el desarrollo y la revisión de medicamentos que aborden condiciones serias con necesidades médicas insatisfechas.
El estudio pivotal de Fase 1-3 de zilganersen ha completado la inscripción en 13 sitios en ocho países. Se espera que los datos preliminares de este estudio estén disponibles en la segunda mitad de 2025. Ionis diseñó zilganersen para atacar la causa subyacente de AxD y potencialmente mejorar el funcionamiento de los pacientes. La empresa espera trabajar en estrecha colaboración con la FDA para potencialmente llevar adelante el primer tratamiento aprobado para AxD.
아이오니스 제약(Ionis Pharmaceuticals, Nasdaq: IONS)은 미국 식품의약국(FDA)이 질간에르센(zilganersen)에 대해 패스트 트랙(Fast Track) 지정을 부여했다고 발표했습니다. 질간에르센은 알렉산더병(Alexander disease, AxD)을 치료하기 위한 것으로, 이는 초희귀이며 진행성이 있는 치명적인 신경학적 질환입니다. 질간에르센은 성인과 아동 모두를 위한 임상 개발 단계의 첫 번째 연구 의약품입니다. 이 지정은 의료적 필요가 충족되지 않은 심각한 질환을 다루는 의약품의 개발과 검토를 촉진하는 것을 목표로 합니다.
질간에르센에 대한 1-3상 주요 연구는 8개 국가의 13개 연구소에서 등록을 완료했습니다. 이 연구의 최종 데이터는 2025년 하반기에 발표될 것으로 기대됩니다. 아이오니스는 질간에르센을 AxD의 근본적인 원인을 타겟으로 설계하였으며, 환자의 기능 개선을 잠재적으로 목표로 하고 있습니다. 이 회사는 FDA와 긴밀히 협력하여 AxD에 대한 최초의 승인 치료제를 가능하게 하기 위해 노력할 것입니다.
Ionis Pharmaceuticals (Nasdaq: IONS) a annoncé que la FDA des États-Unis a accordé la designation Fast Track à zilganersen pour le traitement de la maladie d'Alexander (AxD), un trouble neurologique ultra-rare, progressif et fatal. Zilganersen est le premier médicament en développement clinique pour les adultes et les enfants atteints d'AxD. Cette désignation vise à accélérer le développement et l'examen des médicaments traitant des affections graves avec des besoins médicaux non satisfaits.
L'étude pivotale de Phase 1-3 de zilganersen a complété l'inscription dans 13 sites répartis dans huit pays. Les données préliminaires de cette étude devraient être disponibles dans la deuxième moitié de 2025. Ionis a conçu zilganersen pour cibler la cause sous-jacente de l'AxD et potentiellement améliorer le fonctionnement des patients. L'entreprise se réjouit de travailler en étroite collaboration avec la FDA pour éventuellement proposer le premier traitement approuvé pour l'AxD.
Ionis Pharmaceuticals (Nasdaq: IONS) gab bekannt, dass die US-amerikanische FDA die Fast Track-Bezeichnung für zilganersen zur Behandlung der Alexander-Krankheit (AxD) gewährt hat, einer ultra-seltenen, fortschreitenden und tödlichen neurologischen Erkrankung. Zilganersen ist das erste in klinischer Entwicklung befindliche Prüfmedikament für Erwachsene und Kinder mit AxD. Diese Bezeichnung zielt darauf ab, die Entwicklung und Überprüfung von Arzneimitteln zu beschleunigen, die ernsthafte Erkrankungen mit unerfülltem medizinischen Bedarf behandeln.
Die entscheidende Phase 1-3-Studie von zilganersen hat die Rekrutierung an 13 Standorten in acht Ländern abgeschlossen. Vorläufige Daten aus dieser Studie werden in der zweiten Hälfte des Jahres 2025 erwartet. Ionis hat zilganersen entwickelt, um die zugrunde liegende Ursache von AxD anzugehen und potenziell die Funktion der Patienten zu verbessern. Das Unternehmen freut sich darauf, eng mit der FDA zusammenzuarbeiten, um möglicherweise die erste genehmigte Behandlung für AxD voranzubringen.
- FDA granted Fast Track designation for zilganersen
- Zilganersen is the first investigational medicine in clinical development for Alexander disease
- Pivotal Phase 1-3 study completed enrollment
- Topline data expected in second half of 2025
- None.
Insights
The FDA's Fast Track designation for zilganersen is a significant development for Ionis Pharmaceuticals and patients with Alexander disease (AxD). This designation highlights the critical unmet need in treating this ultra-rare, fatal neurological disorder. Fast Track status could potentially accelerate the drug's path to market, which is important given the lack of approved treatments for AxD.
The pivotal Phase 1-3 study's completion of enrollment is a positive milestone. With topline data expected in H2 2025, investors should watch this closely as positive results could significantly impact Ionis' stock. Zilganersen's potential as a first-in-class treatment for AxD could open a new market for Ionis, albeit for an ultra-rare condition.
However, investors should note that the ultra-rare nature of AxD means the potential patient population is very small, which could limit the commercial potential. The success of zilganersen will depend on its efficacy, pricing strategy and Ionis' ability to navigate the challenges of marketing to a patient group.
Ionis' zilganersen represents a strategic move into the ultra-rare disease space, which can be highly lucrative despite small patient populations. The Fast Track designation not only expedites the regulatory process but also validates the drug's potential significance, potentially attracting investor attention and partnerships.
From a market perspective, orphan drugs for ultra-rare diseases often command premium pricing, which could translate to substantial revenue if zilganersen is approved. However, the development costs for such specialized therapies can be high relative to the potential patient pool.
Investors should consider Ionis' pipeline diversity and how zilganersen fits into their overall strategy. Success here could bolster Ionis' reputation in rare disease therapeutics, potentially opening doors for similar programs. The company's ability to leverage its antisense technology platform across various indications is a key strength to watch.
- Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease, an ultra-rare neurological condition
"With no approved treatments available for people living with AxD, receiving this Fast Track designation for zilganersen reflects the seriousness of this ultra-rare disease and the significant unmet need for treatment in this patient population," said Eugene Schneider, M.D., executive vice president and chief clinical development officer at Ionis. "Zilganersen was designed to address the underlying cause of disease and help improve the functioning of people living with AxD. We look forward to a data readout next year and working closely with the FDA to potentially bring forward the first approved AxD treatment."
The pivotal Phase 1-3 study of zilganersen in adults and children living with AxD completed enrollment earlier this year across 13 sites in eight countries. More information on the study (NCT04849741) is available at www.clinicaltrials.gov.
About Alexander Disease (AxD)
AxD is an ultra-rare, progressive and ultimately fatal type of leukodystrophy, which are a group of genetic disorders that affect the brain's white matter. AxD is estimated to occur in approximately one in one million to one in three million people worldwide and usually leads to death within 14 - 25 years after symptom onset. AxD can present throughout life as loss of independence and lack of ability to control muscles for swallowing, airway protection and purposeful movements, though the impact of AxD can vary depending on factors like age of onset. Diagnosing AxD is based on a combination of clinical presentation, brain magnetic resonance imaging (MRI) findings and genetic testing. AxD is caused by changes in the glial fibrillary acidic protein (GFAP) gene that lead to a build-up of abnormal proteins in cells, causing progressive damage to the nervous system. There are no medicines approved for people with AxD, and current treatments focus on managing their symptoms.
About Zilganersen (ION373)
Zilganersen is an investigational antisense oligonucleotide medicine being developed as a potential treatment for people with genetically confirmed Alexander disease (AxD). Zilganersen is designed to stop the excess glial fibrillary acidic protein (GFAP) production that accumulates because of disease-causing variants in the GFAP gene. In 2020, the U.S. Food and Drug Administration (FDA) granted zilganersen Orphan Drug designation and Rare Pediatric Disease designation. In addition, the European Medicines Agency (EMA) granted zilganersen Orphan Drug designation in 2019.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients.
To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, zilganersen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc.
Zilganersen is an investigational medicine that has not been approved for the treatment of any disease by regulatory authorities.
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. - IR@ionis.com - 760-603-2331
Ionis Pharmaceuticals Media Contact: Hayley Soffer - Media@ionis.com - 760-603-4679
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