This morning, Wave Life Sciences reported that they have received supportive initial feedback from FDA on WVE-003. They engaged the FDA following their positive SELECT-HD clinical results, and today, they announced that the FDA recognize the severity of HD and have indicated they are open to our plan to evaluate biomarkers, including caudate atrophy, as an endpoint to assess HD progression with the potential to predict clinical outcomes. FDA also granted WVE-003 Orphan Drug Designation last week.

Wave is now planning activities for an efficient global, potentially registrational Phase 2/3 study of WVE-003 and planning for an Investigational New Drug submission for this study in the second half of 2025.

Below is a part of the press release that focuses on WVE-003.

Read Complete Release

---PRESS RELEASE---

Wave Life Sciences Reports Third Quarter 2024 Financial Results and Provides Business Update

November 12, 2024 PDF Version Achieved first-ever RNA editing in humans in RestorAATion-2 trial of WVE-006 (GalNAc-AIMer) in alpha-1 antitrypsin deficiency and announced three wholly owned GalNAc-AIMer preclinical programs that offer first-in-class approaches to address unmet needs in cardiometabolic diseases

WVE-007 (INHBE GalNAc-siRNA) clinical trial on track for 1Q 2025; preclinical data demonstrates opportunities for monotherapy, for synergistic use with GLP-1s, and for maintenance to avoid rebound weight gain following cessation of GLP-1s

Received supportive initial feedback from FDA on WVE-003; FDA is engaged in discussing pathways to accelerated approval and open to Wave’s plan to evaluate biomarkers, including caudate atrophy, as an endpoint to evaluate HD progression; IND submission expected in 2H 2025

Delivered positive interim data from FORWARD-53 study of WVE-N531; expect feedback on a pathway to accelerated approval from regulators, as well as the complete 48-week FORWARD-53 data, in 1Q 2025

Cash and cash equivalents of $310.9 million as of September 30, 2024, plus additional ~$28 million net proceeds received on October 1 from full exercise of greenshoe option, with runway expected into 2027

Investor conference call and webcast at 8:30 a.m. ET today

CAMBRIDGE, Mass., Nov. 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 financial results for the third quarter ended September 30, 2024, and provided a business update.

“Since the start of the third quarter, we have delivered positive clinical updates across DMD and AATD, unlocked RNA editing for Wave, and advanced our novel, wholly owned pipeline focused on GalNAc-conjugated programs,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Our achievement of proof-of-mechanism for WVE-006 marked a historic breakthrough for the oligonucleotide field with the first-ever in-human evidence of RNA editing, providing a strong foundation to expand our wholly owned editing pipeline. We are also advancing WVE-007 for obesity and WVE-N531 for DMD towards key milestones in the first quarter. In HD, we are encouraged by a potential path forward to bring the first-ever therapeutic approach that preserves healthy wild-type protein to people living with HD, a devastating disease with limited treatment options. We continue to receive substantial interest on HD, including from potential strategic partners. With the continued demonstration of our novel chemistry in the clinic and cash runway expected into 2027, we are well capitalized and well positioned to deliver multiple value inflection points across our portfolio as we continue to build a leading RNA medicines company.”

HD (allele-selective silencing)

WVE-003 is a first-in-class, allele-selective oligonucleotide for the treatment of Huntington’s disease (HD). Results of the SELECT-HD clinical trial demonstrated the first-ever allele-selective reductions in CSF mutant huntingtin (mHTT) protein and preservation of healthy, wild-type huntingtin (wtHTT) protein with multiple doses of WVE-003, as well as a statistically significant correlation between mHTT reductions and slowing of caudate atrophy. By sparing wtHTT protein, which is critical to the health of the central nervous system, WVE-003 is uniquely positioned to address presymptomatic HD patients, as well as symptomatic patients.

• There are currently no disease modifying therapies for HD, which affects over 200,000 individuals across pre-symptomatic and symptomatic disease stages in the US and Europe. WVE-003 is expected to address approximately 40% of the HD population (potential $5 billion commercial opportunity), and up to 80% of patients with HD may be addressed in the future with other SNP-targeted candidates (potential $10 billion commercial opportunity).

• At Wave’s recent Research Day, Jeffrey Long, PhD, Professor of Psychiatry and Biostatistics at the University of Iowa, discussed the opportunity for caudate atrophy as a biomarker to expedite clinical development in HD. Dr. Long shared data which supports that the slowing of caudate atrophy predicts significant delays in the loss of function for people living with HD. The potential for changes in caudate volume to predict clinical outcomes in HD and its sensitivity to change early in the disease course makes it an optimal biomarker to enable smaller, more efficient clinical trials.

• In November 2024, Wave received supportive initial feedback from FDA, who recognize the severity of HD and are receptive to and engaged with Wave regarding a potential pathway to accelerated approval. FDA is open to Wave’s plan to evaluate biomarkers, including caudate atrophy, as an endpoint to assess HD progression with the potential to predict clinical outcome. Planning is underway for a global, potentially registrational Phase 2/3 study of WVE-003, including finalization of key aspects of design.

• In November 2024, the FDA granted Orphan Drug Designation to WVE-003.

• Expected upcoming milestone: Wave expects to submit an Investigational New Drug (“IND”) application for WVE-003 in the second half of 2025.