Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy
Wave Life Sciences (Nasdaq: WVE) has received FDA Rare Pediatric Disease Designation for WVE-N531, a treatment for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. The company expects to deliver data from the potentially registrational FORWARD-53 trial, including dystrophin protein expression, in Q3 2024.
In a previous Part A study, WVE-N531 achieved 53% exon skipping and muscle tissue concentrations 20-30 times higher than those reported by technologies using muscle delivery conjugates. The treatment also demonstrated distribution to myogenic stem cells, a unique feature among exon skipping therapies. Preclinical data showed potential for cardiac and respiratory benefits.
Wave Life Sciences (Nasdaq: WVE) ha ricevuto la Designazione di Malattia Pediatrica Rara dalla FDA per WVE-N531, un trattamento per i pazienti affetti da distrofia muscolare di Duchenne (DMD) idonei per il bypass dell'esone 53. L'azienda prevede di presentare dati dal potenziale studio registrativo FORWARD-53, inclusa l'espressione della proteina distrofina, nel Q3 2024.
In uno studio precedente, la Parte A, WVE-N531 ha raggiunto un 53% di bypass dell'esone e concentrazioni di tessuto muscolare 20-30 volte superiori rispetto a quelle riportate da tecnologie che utilizzano coniugati per la somministrazione muscolare. Il trattamento ha anche dimostrato distribuzione alle cellule staminali miogeniche, una caratteristica unica tra le terapie di bypass degli esoni. I dati preclinici hanno mostrato potenzialità per benefici cardiaci e respiratori.
Wave Life Sciences (Nasdaq: WVE) ha recibido la Designación de Enfermedad Pediátrica Rara por parte de la FDA para WVE-N531, un tratamiento para pacientes con distrofia muscular de Duchenne (DMD) que son aptos para el salto del exón 53. La empresa espera presentar datos del potencial ensayo registral FORWARD-53, incluyendo la expresión de la proteína distrofina, en Q3 2024.
En un estudio previo de la Parte A, WVE-N531 logró un 53% de salto de exón y concentraciones de tejido muscular 20-30 veces superiores a las reportadas por tecnologías que utilizan conjugados de entrega muscular. El tratamiento también demostró distribución a células madre miogénicas, una característica única entre las terapias de salto de exón. Los datos preclínicos mostraron un potencial beneficio cardíaco y respiratorio.
웨이브 라이프 사이언스(Wave Life Sciences)(나스닥: WVE)는 듀센 근육 병증(DMD) 환자를 위한 WVE-N531의 FDA 희귀 소아 질환 지정을 받았습니다. 이 치료는 53번 엑손 우회를 위한 환자에게 적합합니다. 이 회사는 잠재적인 등록 시험인 FORWARD-53에서 단백질 디스트로핀 발현을 포함한 데이터를 2024년 3분기에 제공할 것으로 예상하고 있습니다.
이전의 A 부분 연구에서 WVE-N531은 53%의 엑손 우회와 근육 조직 농도가 20-30배 더 높은 것으로 나타났습니다. 이 치료는 엑손 우회 요법 중 독특한 특징인 근원 세포에 분포하는 것도 보여주었습니다. 전임상 데이터는 심장 및 호흡기 개선의 잠재력을 보여주었습니다.
Wave Life Sciences (Nasdaq: WVE) a reçu la désignation de maladie pédiatrique rare de la FDA pour WVE-N531, un traitement pour les patients atteints de dystrophie musculaire de Duchenne (DMD) éligibles pour le saut de l'exon 53. L'entreprise s'attend à fournir des données de l'essai potentiel à visée d'enregistrement FORWARD-53, y compris l'expression de la protéine dystrophine, au T3 2024.
Dans une étude antérieure, la Partie A, WVE-N531 a atteint un saut de 53% de l'exon et des concentrations de tissu musculaire 20 à 30 fois plus élevées que celles rapportées par des technologies utilisant des conjugats de livraison musculaire. Le traitement a également montré une distribution aux cellules souches myogéniques, une caractéristique unique parmi les thérapies de saut d'exon. Les données précliniques ont montré un potentiel bénéfice cardiaque et respiratoire.
Wave Life Sciences (Nasdaq: WVE) hat von der FDA die Auszeichnung für seltene pädiatrische Erkrankungen für WVE-N531 erhalten, eine Behandlung für Patienten mit Duchenne-Muskeldystrophie (DMD), die für das Überspringen des Exons 53 geeignet sind. Das Unternehmen erwartet, im Q3 2024 Daten aus der potenziell registrierbaren FORWARD-53-Studie, einschließlich der Expression des Dystrophin-Proteins, zu präsentieren.
In einer vorherigen Studie der Teil A erreichte WVE-N531 ein 53%iges Überspringen des Exons und Konzentrationen im Muskelgewebe, die 20-30 Mal höher waren als die von Technologien, die Konjugate zur Muskelabgabe verwenden. Die Behandlung zeigte auch eine Verteilung zu myogenen Stammzellen, ein einzigartiges Merkmal unter den Exon-Überspringungstherapien. Präklinische Daten zeigten Potenzial für kardiovaskuläre und respiratorische Vorteile.
- FDA granted Rare Pediatric Disease Designation for WVE-N531, potentially leading to a Priority Review Voucher
- WVE-N531 achieved industry-leading 53% mean exon skipping levels in Part A study
- Muscle tissue concentrations were 20-30 times higher than competing technologies
- Demonstrated distribution to myogenic stem cells, unique among exon skipping therapies
- Potential for cardiac and respiratory benefits based on preclinical data
- Data from the potentially registrational FORWARD-53 trial not yet available
- patient population (boys with DMD amenable to exon 53 skipping)
Wave Life Sciences' FDA Rare Pediatric Disease Designation for WVE-N531 is a significant milestone in the DMD treatment landscape. The industry-leading exon skipping levels of
The upcoming FORWARD-53 trial results in Q3 2024 are pivotal. Positive dystrophin expression data could position WVE-N531 as a best-in-class treatment, potentially expanding Wave's pipeline to cover
The FDA's Rare Pediatric Disease Designation for WVE-N531 underscores the persistent unmet needs in DMD treatment. WVE-N531's ability to achieve muscle tissue concentrations 20-30 times higher than current exon skipping technologies is remarkable. This could translate to more effective dystrophin production, potentially slowing disease progression.
The observed distribution to myogenic stem cells is a groundbreaking finding. If confirmed in larger studies, it could lead to more sustained therapeutic effects, addressing the challenge of muscle regeneration in DMD. The potential cardiac and respiratory benefits, suggested by preclinical data, could significantly improve patient outcomes, as heart and lung complications are major causes of morbidity in DMD.
The FDA Rare Pediatric Disease Designation for WVE-N531 is a positive development for Wave Life Sciences. If approved, the company would be eligible for a Priority Review Voucher, which can be sold for substantial sums (
Investors should closely monitor the Q3 2024 FORWARD-53 trial results. Positive data could catalyze a significant stock price movement and potentially attract partnership interest from larger pharmaceutical companies. However, the small trial size (11 participants) and the competitive landscape in DMD treatments present risks. The stock may experience volatility as the data readout approaches.
Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024
In previous Part A trial, WVE-N531 achieved industry-leading exon skipping of
CAMBRIDGE, Mass., Aug. 12, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to WVE-N531 for the treatment of boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. WVE-N531 is currently being evaluated in the potentially registrational FORWARD-53 clinical trial and Wave expects to deliver data, including dystrophin protein expression from muscle biopsies after 24 weeks of treatment, in the third quarter of 2024.
“This designation from FDA underscores that significant unmet needs remain in DMD, and it also supports Wave’s innovative and purposeful approach to drug development in the rare disease space,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Sciences. “With our WVE-N531 program, we are aiming to restore clinically meaningful levels of near full length, functional dystrophin protein. Positive data from FORWARD-53 would also unlock additional programs for other exons in our pipeline, with the goal of developing best-in-class medicines that address the underlying cause of the disease for up to
WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein. In the previously completed Part A study (three 10 mg/kg doses every other week), WVE-N531 achieved industry-leading mean exon skipping levels of
WVE-N531 is currently being investigated in FORWARD-53, a potentially registrational, open-label clinical trial in 11 boys with DMD. Endpoints include dystrophin expression after 24 and 48 weeks of treatment, as well as pharmacokinetic, safety and tolerability data.
Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases which primarily affect individuals less than 18 years of age and fewer than 200,000 people in the United States. If a New Drug Application for WVE-N531 is approved by the FDA, Wave would be eligible to receive a Priority Review Voucher.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn boys. Approximately
About Wave Life Sciences
Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s diversified pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity. Driven by the calling to “Reimagine Possible”, Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit www.wavelifesciences.com and follow Wave on X (formerly Twitter) and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the potential significance and implications of receiving the Rare Pediatric Disease Designation from the FDA; the potentially registrational nature of our FORWARD-53 trial for WVE-N531; our expectations that WVE-N531’s industry-leading exon skipping and muscle tissue concentrations has the potential to become the leading exon-skipping therapeutic in DMD; our expectations that high tissue concentrations and high exon skipping may result in high dystrophin restoration following a sufficient follow up period; our understanding of the importance of satellite cells for muscle regeneration; our expectation that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle; and our expectations of the anticipated therapeutic benefits of WVE-N531 for DMD over existing therapies. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release and actual results may differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.
Investor Contact:
Kate Rausch
+1 617-949-4827
krausch@wavelifesci.com
Media Contact:
Alicia Suter
+1 617-949-4817
asuter@wavelifesci.com
FAQ
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