WVE-006はAATDの原因であるSERPINA1遺伝子G→A変異転写産物を正して肝臓や肺が損なわれないようにします。
GSKはWave社の手による最初の臨床試験以降のWVE-006の開発や販売を担います。
同剤の初の臨床試験の投与は今年4Qに始まり、目当ての作用の検討結果は来年発表される見込みです。
WVE-006 is the first-ever RNA editing program to enter clinical development and is designed to restore production and circulation of functional, wild-type alpha-1 antitrypsin (AAT) protein and reduce levels of mutant Z-AAT protein, thereby addressing alpha-1 antitrypsin deficiency-related lung disease, liver disease or both
Wave expects to initiate dosing in the first-in-human clinical program in 4Q 2023 and deliver AAT protein restoration data from AATD patients treated with WVE-006 in 2024
Under its collaboration with GSK, Wave is eligible to receive substantial milestone payments for WVE-006 in 2023 and beyond
Wave plans to host a virtual “R&D Day” on September 28, 2023; topics to include the WVE-006 clinical program and how Wave is extending its leadership in RNA editing with additional programs
CAMBRIDGE, Mass., Sept. 05, 2023 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the submission of its first clinical trial application (CTA) for WVE-006 in alpha-1 antitrypsin deficiency (AATD). WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide (“AIMer”) and is designed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin (M-AAT) protein. The WVE-006 clinical program will be highlighted in Wave’s virtual “R&D Day” on September 28, 2023 at 10:00 a.m. ET, among other programs.
“With the submission of the first CTA for WVE-006, we have officially initiated clinical development of the industry’s first-ever RNA editing therapeutic candidate,” said Anne Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences. “We designed WVE-006 to correct the most common underlying genetic mutation that causes AATD, providing an innovative therapeutic option for individuals with lung disease, liver disease or both. Indeed, our preclinical data support this profile, with mouse models showing restored AAT protein well above 11 micromolar, as well as improvement in several markers of liver disease and inhibition of neutrophil elastase. As a GalNAc-RNA editing oligonucleotide, WVE-006 is reversible and re-dosable, with potential for infrequent subcutaneous dosing. WVE-006 is highly specific with no evidence of bystander editing and, by virtue of the mechanism of action, no permanent changes to the genome that occur with DNA-targeting approaches. For these reasons, we believe WVE-006 has potential to revolutionize how AATD is treated.”
The current clinical development plan for WVE-006 includes healthy volunteers as well as individuals with AATD who have the homozygous PiZZ mutation, and is designed to provide an efficient path to proof-of-mechanism as measured by restoration of M-AAT protein in serum. Wave expects to initiate dosing in healthy volunteers in the fourth quarter of 2023 and deliver proof-of-mechanism data in individuals with AATD in 2024.
“WVE-006 is on a path to potentially deliver the first-ever proof-of-mechanism clinical data for an RNA editing therapeutic. Positive data would be a pivotal milestone for people living with AATD and would unlock the potential of RNA editing more broadly. Additionally, WVE-006 is a foundational component of our collaboration with GSK and carries with it meaningful milestone and royalty payments, including near-term clinical milestones,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “RNA editing is a promising new therapeutic modality, capable of accessing untapped areas of disease biology. We are incredibly proud to be pioneers leading the way forward in RNA editing, and we plan to share more updates on AATD and other RNA editing programs during our R&D Day later this month.”
Earlier this year, Wave commenced its strategic collaboration with GSK to advance transformative RNA medicines using Wave’s multimodal RNA platform, including WVE-006. Wave received $170 million in upfront cash and equity and is also receiving research funding. Wave is eligible for up to $3.3 billion in potential milestone payments, including near-term preclinical and clinical milestones, as well as royalties, for WVE-006 and GSK’s eight collaboration programs. For WVE-006, Wave is eligible to receive up to $225 million in development and launch milestone payments, and up to $300 million in sales-related milestone payments, as well as double-digit tiered royalties as a percentage of net sales up to the high teens.
Wave expects that its cash and cash equivalents will be sufficient to fund operations into 2025. Wave does not include future milestones or other contingent payments in its cash runway.
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