This section highlights FDA-related milestones and regulatory updates for drugs developed by WAVE Life Sciences (WVE).
Over the past two years, WAVE Life Sciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
WVE-003, WVE-006, WVE-007, and WVE-N531. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
WVE-003 - FDA Regulatory Timeline and Events
WVE-003 is a drug developed by WAVE Life Sciences for the following indication: Huntington's Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WVE-003
- Announced Date:
- June 25, 2024
- Indication:
- Huntington's Disease
Announcement
Wave Life Sciences Ltd. announced positive results from its Phase 1b/2a SELECT-HD clinical trial of WVE-003, which is being developed as a potential disease modifying therapeutic for Huntington's disease (HD).
AI Summary
Wave Life Sciences announced positive results from its Phase 1b/2a SELECT-HD trial for WVE-003, a first-in-class antisense oligonucleotide aimed at modifying Huntington’s disease. The trial showed that 30 mg multidose treatment led to a 46% reduction in mutant huntingtin protein (mHTT) in cerebrospinal fluid compared to placebo, while preserving the healthy wild-type huntingtin protein (wtHTT). This allele-selective silencing approach is significant because it targets the harmful mHTT without compromising essential neuronal functions provided by wtHTT.
Additionally, the study revealed a statistically significant correlation between the reduction of mHTT and slowed caudate atrophy, an imaging marker linked to better clinical outcomes. Based on these encouraging data, Wave plans to engage regulators to discuss a potential accelerated approval pathway for WVE-003, marking an important step forward in the fight against Huntington’s disease.
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WVE-006 - FDA Regulatory Timeline and Events
WVE-006 is a drug developed by WAVE Life Sciences for the following indication: For the treatment of alpha-1 antitrypsin deficiency (AATD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WVE-006
- Announced Date:
- June 25, 2024
- Indication:
- For the treatment of alpha-1 antitrypsin deficiency (AATD).
Announcement
Wave Life Sciences Ltd. RNA editing proof-of-mechanism data for WVE-006 in AATD in 2024,
AI Summary
Wave Life Sciences is on track to deliver RNA editing proof-of-mechanism data for its candidate WVE-006 in patients with Alpha-1 Antitrypsin Deficiency (AATD) in 2024. This data is expected to show that the company’s innovative RNA editing technology effectively targets and corrects specific RNA sequences, potentially offering a new therapeutic pathway for treating AATD.
The upcoming proof-of-mechanism data will further validate Wave’s advanced RNA medicines platform and its proprietary PN chemistry, reinforcing the potential of RNA-based therapies to address serious genetic disorders. As Wave continues to expand its pipeline with innovative candidates, the planned data release for WVE-006 highlights the company’s commitment to leveraging RNA editing to improve patient outcomes in AATD and other conditions.
Read Announcement- Drug:
- WVE-006
- Announced Date:
- April 30, 2024
- Indication:
- For the treatment of alpha-1 antitrypsin deficiency (AATD).
Announcement
Wave Life Sciences Ltd.announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of WVE-006, the company's first-in-class RNA editing oligonucleotide, which is being developed for the treatment of alpha-1 antitrypsin deficiency (AATD). WVE-006 is GalNAc-conjugated and subcutaneously administered; it does not use a lipid nanoparticle (LNP) delivery system.
AI Summary
Wave Life Sciences Ltd. has received approval for its first clinical trial application (CTA) for the RestorAATion-2 study of WVE-006, its first-in-class RNA editing oligonucleotide developed to treat alpha-1 antitrypsin deficiency (AATD). WVE-006 is designed to correct the disease-causing mutation by targeting the RNA, potentially restoring functional M-AAT protein. The drug is GalNAc-conjugated and administered subcutaneously without using a lipid nanoparticle (LNP) delivery system.
The RestorAATion-2 trial is a Phase 1b/2a open-label study that will assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of the drug in patients with the homozygous Pi*ZZ mutation. Early clinical data from dose-escalation studies in healthy volunteers have helped determine a starting dose expected to engage the target, with proof-of-mechanism results anticipated later this year.
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WVE-007 - FDA Regulatory Timeline and Events
WVE-007 is a drug developed by WAVE Life Sciences for the following indication: For obesity.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WVE-007
- Announced Date:
- June 20, 2025
- Indication:
- For obesity.
Announcement
Wave Life Sciences Ltd. announced the presentation of preclinical data supporting WVE-007, its GalNAc-siRNA designed to silence INHBE mRNA, an obesity target with strong evidence from human genetics.
AI Summary
Wave Life Sciences Ltd. announced today the presentation of preclinical data for its novel treatment candidate, WVE-007. This innovative GalNAc-siRNA is designed to silence INHBE mRNA, an obesity target supported by strong human genetics evidence. The preclinical studies demonstrated that a single dose of WVE-007 led to a potent and long-lasting reduction in Activin E, a protein linked to fat retention. In diet-induced obese mice, this reduction resulted in significant fat loss with preservation of muscle mass, along with a decrease in pro-inflammatory macrophages in visceral fat. These findings suggest that by lowering Activin E, WVE-007 might trigger healthy weight loss and reduce risks for conditions such as type 2 diabetes and coronary artery disease. The results highlight the potential of this therapy as a unique approach to obesity treatment.
Read Announcement- Drug:
- WVE-007
- Announced Date:
- February 6, 2025
- Estimated Event Date Range:
- October 1, 2025 - December 31, 2025
- Target Action Date:
- LATE 2025
- Indication:
- For obesity.
Announcement
Wave Life Sciences Ltd Proof-of-concept clinical data from INLIGHT are expected in 2025.
AI Summary
Wave Life Sciences Ltd has initiated the Phase 1 INLIGHT clinical trial to test its novel drug, WVE-007. This new GalNAc-siRNA targets the INHBE messenger RNA to promote healthy, sustained weight loss by encouraging fat burning while preserving muscle mass. The INLIGHT trial, which will enroll adults living with overweight or obesity, will assess the safety, tolerability, pharmacokinetics, and relevant biomarkers for target engagement. Wave’s unique approach could support once- or twice-yearly dosing, addressing the global obesity challenge for over a billion people. Most notably, Wave expects to deliver proof-of-concept clinical data from the INLIGHT trial in 2025, marking a key milestone for the company. This data will provide important insights into whether WVE-007 can offer a new, effective treatment for obesity by improving metabolic health and supporting sustainable weight loss.
Read Announcement- Drug:
- WVE-007
- Announced Date:
- February 6, 2025
- Indication:
- For obesity.
Announcement
Wave Life Sciences Ltd announced clinical trial application (CTA) approval and initiation of the Phase 1 INLIGHT clinical trial of WVE-007 in obesity.
AI Summary
Wave Life Sciences Ltd has received FDA approval for its clinical trial application and has initiated the Phase 1 INLIGHT clinical trial for its novel drug WVE-007 to treat obesity. WVE-007 is a long-acting GalNAc-conjugated siRNA that targets the INHBE gene, which plays a role in fat burning. By silencing INHBE, the drug aims to restart fat metabolism, supporting healthy weight loss while preserving muscle mass.
The Phase 1 trial is designed to enroll adults with overweight or obesity. It will assess the safety and tolerability of WVE-007, along with its pharmacokinetics, target engagement biomarkers, and impacts on body weight and metabolic health. Wave Life Sciences expects to deliver proof-of-concept clinical data in 2025, marking a potential new approach to obesity treatment with the possibility of once- or twice-yearly dosing.
Read Announcement- Drug:
- WVE-007
- Announced Date:
- January 13, 2025
- Indication:
- For obesity.
Announcement
Wave Life Sciences Ltd. announced key expected 2025 milestones across its clinical programs, growing pipeline, and leading RNA medicines platform ahead of the company's scheduled presentation at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Monday, January 13, 2025, at 9:45 a.m. PT / 12:45 p.m. ET.
AI Summary
Wave Life Sciences Ltd. announced key expected milestones for 2025 that will advance its clinical programs, expand its pipeline, and highlight its leading RNA medicines platform. The company will initiate dosing in its Phase 1 INLIGHT clinical trial with WVE-007, a novel GalNAc-siRNA treatment for obesity that targets the INHBE gene. This trial, expected to start in early 2025, is designed to demonstrate safety, tolerability, and biomarker effects related to healthy weight loss.
Wave plans to build on these achievements with further data from its diverse RNA editing and gene therapy programs, including ongoing efforts in areas such as DMD, HD, and AATD. The company will showcase these developments during its presentation at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on January 13, 2025, at 9:45 a.m. PT / 12:45 p.m. ET.
Read Announcement- Drug:
- WVE-007
- Announced Date:
- December 23, 2024
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 2025
- Indication:
- For obesity.
Announcement
Wave expects CTA approval and initiation of the first-in-human study of WVE-007 in the first quarter of 2025.
AI Summary
Wave Life Sciences, a clinical-stage biotechnology company, recently submitted its first clinical trial application for its investigational drug WVE-007, targeting obesity. The novel drug uses GalNAc-conjugated siRNA technology to silence the INHBE gene, which can help the body burn fat without losing muscle. Wave plans that once CTA approval is granted, the first-in-human study will begin in the first quarter of 2025.
This early clinical trial will evaluate safety, tolerability, and early signs of effectiveness in adults with overweight or obesity. The company hopes that WVE-007, potentially usable once or twice a year, will offer a sustainable solution for weight loss and cardiometabolic risk reduction by leveraging its innovative approach in RNA medicine.
Read Announcement- Drug:
- WVE-007
- Announced Date:
- December 23, 2024
- Indication:
- For obesity.
Announcement
Wave Life Sciences announced the submission of its first clinical trial application (CTA) for WVE-007 in obesity. WVE-007 is an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to silence Inhibin βE (INHBE) gene expression, which would induce fat burning (lipolysis) to decrease body weight without impacting muscle mass.
AI Summary
Wave Life Sciences has submitted its first clinical trial application (CTA) for WVE-007, a new treatment approach for obesity. This investigational therapy uses GalNAc-conjugated small interfering RNA (siRNA) to silence the Inhibin βE (INHBE) gene. By silencing INHBE, WVE-007 aims to trigger fat burning (lipolysis) to reduce body weight without affecting muscle mass, offering a novel method for achieving healthy and sustainable weight loss. The treatment is designed with the potential for once or twice-yearly dosing, making it a promising option for long-term obesity management and metabolic health improvement. Wave Life Sciences plans to begin the first-in-human study of WVE-007 in the first quarter of 2025, marking a significant step forward in the development of RNA medicines for obesity treatment.
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WVE-N531 - FDA Regulatory Timeline and Events
WVE-N531 is a drug developed by WAVE Life Sciences for the following indication: Duchenne Muscular Dystrophy GLN.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WVE-N531
- Announced Date:
- March 26, 2025
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave Life Sciences announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
AI Summary
Wave Life Sciences announced promising results from the Phase 2 FORWARD-53 trial of WVE-N531, an exon skipping oligonucleotide being developed for boys with Duchenne muscular dystrophy (DMD) who are eligible for exon 53 skipping. The study showed a significant 3.8-second improvement in Time-to-Rise compared to natural history data, marking the largest effect among approved dystrophin restoration therapies at 48 weeks. In addition to better muscle function, the trial reported improvements in muscle health, including a reduction in fibrosis driven by decreased inflammation and necrosis, and a shift in muscle tissue from a regenerative to a more mature state. Dystrophin levels remained stable, with most participants showing meaningful expression levels. The treatment was well tolerated with no serious adverse events, supporting further development toward a New Drug Application aimed at accelerating approval of WVE-N531.
Read Announcement- Drug:
- WVE-N531
- Announced Date:
- September 24, 2024
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave Life Sciences announced positive interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
AI Summary
Wave Life Sciences announced positive interim results from its ongoing Phase 2 FORWARD-53 study. The study is testing WVE-N531, an exon skipping oligonucleotide aimed at treating boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. Results after 24 weeks showed a mean muscle content-adjusted dystrophin level of 9.0% and an unadjusted level of 5.5%. The dystrophin produced comes from two isoforms, similar to those found in Becker muscular dystrophy, a milder form of the disease. In addition, the treatment demonstrated improvements in serum biomarkers for muscle health, along with clear evidence of myofiber regeneration. The long tissue half-life of 61 days supports a monthly dosing regimen, and preclinical data suggest even better outcomes in heart and diaphragm tissues. Wave expects further data from the full 48-week study in early 2025.
Read Announcement- Drug:
- WVE-N531
- Announced Date:
- September 24, 2024
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 2025
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave announced that it expects feedback on a pathway to accelerated approval from regulators, as well as the complete 48-week FORWARD-53 data, in 1Q 2025
AI Summary
Wave Life Sciences announced positive interim results from its Phase 2 FORWARD-53 trial evaluating WVE-N531 in boys with Duchenne muscular dystrophy amenable to exon 53 skipping. The data showed stable and consistent dystrophin production, with two isoforms that mirror those seen in milder Becker muscular dystrophy cases. The drug was found to be safe and well tolerated, with adverse events only being mild, supporting the feasibility of monthly dosing. Importantly, Wave expects to receive feedback from regulators on a pathway to accelerated approval in the first quarter of 2025, when the complete 48‐week FORWARD-53 data will be available. This regulatory interaction and final trial results will play a critical role in potentially advancing WVE-N531 as a therapeutic option for boys with DMD, addressing the urgent need for improved treatment strategies.
Read Announcement- Drug:
- WVE-N531
- Announced Date:
- August 12, 2024
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave Life Sciences Ltd 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.
AI Summary
Wave Life Sciences announced that the U.S. FDA has granted Rare Pediatric Disease Designation to its investigational drug WVE-N531, designed for treating boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. This designation highlights the significant unmet need for effective DMD therapies, as the disease leads to progressive muscle degeneration. Wave is evaluating WVE-N531 in the potentially registrational FORWARD-53 clinical trial, and promising dystrophin protein data from muscle biopsies is expected in the third quarter of 2024 after 24 weeks of treatment. The goal of WVE-N531 is to restore near full-length, functional dystrophin protein, which could improve muscle function in affected individuals. This recognition by the FDA underscores both the urgency for innovative solutions in DMD care and Wave’s commitment to addressing the underlying causes of the disease.
Read Announcement- Drug:
- WVE-N531
- Announced Date:
- August 12, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave expects to deliver data, including dystrophin protein expression from muscle biopsies after 24 weeks of treatment, in the third quarter of 2024.
AI Summary
Wave Life Sciences is advancing its FORWARD-53 trial, a potentially registrational study for boys with Duchenne muscular dystrophy (DMD) who are eligible for exon 53 skipping. The company expects to deliver important study data in the third quarter of 2024, including dystrophin protein expression analysis from muscle biopsies taken after 24 weeks of treatment. This data will help confirm whether WVE-N531, an exon-skipping oligonucleotide, can restore near full-length, functional dystrophin protein in patients, which is key for improving muscle function. The trial will provide additional insights into the drug’s safety, tolerability, and overall pharmacokinetic profile. Positive results could not only support this therapy for DMD but also pave the way for developing similar treatments for other exons, addressing a significant unmet need for up to 40% of boys with DMD.
Read Announcement- Drug:
- WVE-N531
- Announced Date:
- June 25, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Duchenne Muscular Dystrophy GLN
Announcement
Wave remains on track to deliver 6-month dystrophin data for WVE-N531 in DMD in 3Q 2024
AI Summary
Wave Life Sciences has announced positive clinical results from its Phase 1b/2a SELECT-HD trial for WVE-003, a candidate designed to selectively lower mutant huntingtin protein in Huntington’s disease while sparing the healthy version. The 46% reduction in mutant protein seen in CSF underscores the potential of Wave’s RNA medicines platform and innovative PN chemistry. Wave is now advancing its regulatory discussions based on these promising findings. Additionally, the company remains on track to deliver 6‐month dystrophin data for its Duchenne muscular dystrophy candidate, WVE-N531, in the third quarter of 2024. This upcoming data is seen as a significant milestone for expanding the application of RNA-based therapies. Wave’s continued progress in both its Huntington’s and DMD programs reinforces its commitment to transforming treatments for devastating neurological and genetic disorders.
Read Announcement
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