University of Oxford

Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs. natural history with largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks; additional functional benefits observed in other outcome measures including NSAA First-ever demonstration of substantial improvements in muscle health with exon skipping – statistically significant reduction in fibrosis driven by decreases in inflammation and necrosis, coupled with transition from regenerative to mature muscle; decreases in creatine kinase and circulating inflammatory biomarkers Dystrophin expression stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of boys above 5% average dystrophin; WVE-N531 remains safe and well-tolerated with no Serious Adverse Events Following recent feedback from FDA, Wave intends to file a New Drug Application in 2026 for accelerated approval, with data to support monthly dosing at launch Wave expects to file CTAs in 2026 for multiple DMD candidates for other exons, with preclinical data supporting a best-in-class exon skipping franchise Wave to host investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass., March 26, 2025 (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 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. The analysis was conducted after 48 weeks of treatment with 10 mg/kg WVE-N531 dosed every two weeks (Q2W). FORWARD-53 achieved all trial goals, demonstrating sustained and industry-leading exon skipping, muscle concentrations and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports monthly dosing. WVE-N531 continues to be safe and well-tolerated. Additionally, the data demonstrate substantial decreases in inflammation and necrosis, a statistically significant reversal of muscle fibrosis (28.6% reduction between week 24 to 48; p<0.01), and a transition from regeneration to maturation of muscle. A 50% decline (<0.001) in creatine kinase (CK), as well as decreases in IL-6 and MCP-1, were also observed. Time-to-Rise (TTR) data demonstrate a statistically significant and clinically meaningful 3.8-second improvement versus natural history (p<0.05), which is the largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks. Additional functional benefits were observed on the North Star Ambulatory Assessment (NSAA) versus natural history, and in hand grip strength versus baseline. “Despite progress in Duchenne, there remains a significant unmet need for therapeutics that meaningfully impact disease progression. These data demonstrate a promising continuum from dystrophin restoration, to regeneration and maturation of muscle tissue, to functional improvement,” said Pat Furlong, founder and president of Parent Project Muscular Dystrophy. “Paired with monthly administration and a continued favorable safety profile, WVE-N531 represents a significant step forward – not just for individuals amenable to exon 53 skipping, but also for the broader exon skipping field. PPMD looks forward to working with Wave as they expediently bring WVE-N531 and their broader exon skipping pipeline to the Duchenne community.” “As a clinician deeply involved in patient care and research in muscle diseases like Duchenne, I am encouraged by these data for WVE-N531 that show dystrophin restoration and several markers of improved muscle condition,” said Laurent Servais, MD, PhD, Professor of Paediatric Neuromuscular Disease at the University of Oxford and Principal Investigator in FORWARD-53. “To see a clinically meaningful and statistically significant difference on TTR versus natural history in a Phase 2 study is another encouraging finding. I am looking forward to the continued development of WVE-N531.” Wave also announced today that the company met with the U.S. Food and Drug Administration (FDA) on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial, where the Agency confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Based on the FDA feedback and the 48-week data, Wave intends to file a New Drug Application (NDA) in 2026 for accelerated approval of WVE-N531. The NDA filing will be based on all FORWARD-53 data, which will include additional data to support monthly dosing. Furthermore, Wave will continue to engage the Agency with the new 48-week data, including functional outcomes, and its planned global confirmatory trial of WVE-N531. “WVE-N531’s demonstrated ability to reach both myofibers and myogenic stem cells – the regenerative muscle cells – and sustain dystrophin restoration over time is impacting muscle health in ways never before seen in DMD,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. “With these transformational data and feedback from FDA in hand, we are now moving toward our first NDA filing, which puts us on the path toward becoming a commercial company.” Continued Dr. Bolno, “Most importantly, we are inspired by the opportunity ahead to deliver life-changing medicines to this community. We expect WVE-N531 to become the first-line treatment of choice for boys amenable to exon 53 skipping, including the 40-50% in the US who are not being treated with approved exon skippers due in part to the burden of weekly infusions coupled with limited efficacy. Additionally, we are accelerating our near-term clinical programs for other exons, which based on preclinical data, suggest a best-in-class exon skipping franchise. We are incredibly grateful to the trial participants and their families, as well as all our collaborators across the DMD community for their contributions to this program and all our DMD research over the past decade.” WVE-N531, as well as Wave’s programs for exons 52, 51, 45 and 44, leverage best-in-class oligonucleotide chemistry, including PN backbone chemistry and stereochemical control, enabling industry-leading muscle delivery and potency without requiring antibody or peptide conjugates. Preclinical data for Wave’s PN chemistry-containing exon skipping candidates have also demonstrated significantly higher dystrophin and drug concentrations in the heart and diaphragm versus skeletal muscle. Collectively, WVE-N531 plus Wave’s exon 52, 51, 45 and 44 programs would address ~40% of the DMD population and represent a >$2.4 billion total market opportunity in the United States alone. Wave expects to submit multiple clinical trial applications (CTAs) for other exon skipping programs in 2026. Detailed Results from FORWARD-53 Eleven boys amenable to exon 53 skipping (age 5-11; 10 ambulatory and 1 non-ambulatory) are enrolled in the ongoing open-label FORWARD-53 trial. Biopsy data are from eight of the 11 boys (all ambulatory and who had biopsies at 24 and 48 weeks) while safety and functional outcome assessments were available for all participants. All 11 boys have advanced to the extension portion of the study where they are now receiving monthly doses of WVE-N531. Results after 48 weeks of 10 mg/kg dosing every two weeks include: Safety and Tolerability WVE-N531 was safe and well-tolerated through 48 weeks. All treatment-related adverse events were mild to moderate in intensity. There were no Serious Adverse Events and no discontinuations due to any causes. Results from Muscle Biopsies Dystrophin and exon skipping: Dystrophin expression stabilized between 24 and 48 weeks of dosing with a mean of 7.8% [95% CI: 5.4-10.3%; 24-week dystrophin was 9.0% (95% CI: 6.5-11.5%) and 48-week dystrophin was 6.4% (95% CI: 3.8-9.0%); muscle content-adjusted dystrophin as measured by western blot]. The difference between the 24- and 48-week mean dystrophin measurements is within the established 30-35% inter-assay variability of the western blot, and consistency between these timepoints was confirmed with an orthogonal assay. 88% of boys (7/8) achieved greater than 5% average dystrophin between 24 and 48 weeks. Mean exon skipping reached steady state at 6 weeks and was consistent through 48 weeks of dosing with a mean of 54% (95% CI: 46-63%). Reversal of muscle damage: Participants showed significant and unprecedented improvements in multiple indicators of muscle health through 48 weeks, indicating a shift from dystrophic muscle towards healthy muscle. These data include: Decreases of the median muscle necrosis and inflammation scores from 2 to 1(representing mild damage) in muscle pathology. Decreases in markers of inflammation (MCP-1 and IL-6). A statistically significant decline in the amount of muscle fibrosis between 24 and 48 weeks (mean decrease of 28.6%, p<0.01). A 50% decrease (p<0.001) in serum creatine kinase (CK), which occurred on top of a stable corticosteroid regimen. Improved organization and uniformity of myofibers in muscle tissue. Decreases in number of stem cells and internalized nuclei, indicative of myofiber maturation between week 24 and 48. 88% of boys (7/8) achieved greater than 5% average dystrophin between 24 and 48 weeks. Mean exon skipping reached steady state at 6 weeks and was consistent through 48 weeks of dosing with a mean of 54% (95% CI: 46-63%). Decreases of the median muscle necrosis and inflammation scores from 2 to 1(representing mild damage) in muscle pathology. Decreases in markers of inflammation (MCP-1 and IL-6). A statistically significant decline in the amount of muscle fibrosis between 24 and 48 weeks (mean decrease of 28.6%, p<0.01). A 50% decrease (p<0.001) in serum creatine kinase (CK), which occurred on top of a stable corticosteroid regimen. Improved organization and uniformity of myofibers in muscle tissue. Decreases in number of stem cells and internalized nuclei, indicative of myofiber maturation between week 24 and 48. Functional Assessments Benefits were seen in multiple functional assessments among WVE-N531-treated boys (n=10), including Time-to-Rise (TTR) and North Star Ambulatory Assessment (NSAA), compared with a matched exon 53 natural history control group (n=18). TTR showed a statistically significant and clinically meaningful 3.8-second difference (p<0.05) favoring WVE-N531 compared with natural history. The minimal clinically important difference (MCID) for TTR was 1.4 seconds, based on the baseline functional characteristics of boys in the study. NSAA showed positive trends favoring WVE-N531 relative to natural history (1.2-point improvement; not significant). Positive trends were also observed among WVE-N531-treated boys (n=11) on grip strength versus baseline. TTR showed a statistically significant and clinically meaningful 3.8-second difference (p<0.05) favoring WVE-N531 compared with natural history. The minimal clinically important difference (MCID) for TTR was 1.4 seconds, based on the baseline functional characteristics of boys in the study. NSAA showed positive trends favoring WVE-N531 relative to natural history (1.2-point improvement; not significant). Additional Upcoming Milestones Across Wave’s Clinical RNA Medicines Pipeline Wave expects multiple additional milestones across its clinical pipeline of RNA medicines in 2025, including: WVE-006 (GalNAc-conjugated RNA editing oligonucleotide for alpha-1 antitrypsin deficiency): Wave expects to deliver 200 mg multi-dose data and 400 mg single-dose data from the Phase 1b/2a RestorAATion-2 trial in 2025. WVE-007 (GalNAc-conjugated siRNA for obesity, designed to silence INHBE mRNA): Wave expects to deliver clinical data from the Phase 1 INLIGHT trial in the second half of 2025, including safety, tolerability and biomarkers reflective of healthy weight loss. WVE-003 (allele-selective silencing for Huntington’s disease): Wave expects to submit an Investigational New Drug application in the second half of 2025 for its potentially registrational Phase 2/3 trial of WVE-003. Investor Conference Call and Webcast Wave will host an investor conference call today at 8:30 a.m. ET to review the FORWARD-53 data. A webcast of the conference call can be accessed by visiting “Investor Events” on the investor relations section of the Wave Life Sciences website: https://ir.wavelifesciences.com/events-publications/events. Analysts planning to participate during the Q&A portion of the live call can join the conference call at the audio-conferencing link here. Once registered, participants will receive the dial-in information. Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website. About WVE-N531 WVE-N531 is an exon skipping oligonucleotide being developed as a disease modifying treatment for boys with Duchenne muscular dystrophy amenable to exon 53 skipping. WVE-N531 was designed using Wave’s best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration. 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 8%-10% of boys with DMD have mutations amenable to treatment with an exon 53 skipping therapy. Exon skipping aims to address the underlying cause of DMD by promoting the production of dystrophin protein to stabilize or slow disease progression. 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 common 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 Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington’s disease, and obesity, as well as several preclinical programs utilizing the company’s broad RNA therapeutics toolkit. 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, our understanding of the anticipated therapeutic benefit of WVE-N531 as a therapy for DMD, and the broader exon skipping field; our understanding of the safety profile of WVE-N531; our plan and estimated timing to file an NDA for accelerated approval of WVE-N531, along with our expectations for commercialization; our expectations for WVE-N531 to become the first-line treatment of choice for boys amenable to exon 53 skipping; our plan to file CTA submissions for other exon skipping treatments and the potential for a best-in-class exon skipping franchise; addressable patient population estimates related to our therapeutic candidates; the potential commercial opportunities that our therapeutic candidates may address; anticipated milestones and anticipated benefits of our therapeutic candidates and pipeline compared to our competitors; and the potential benefits of PRISM, including our novel PN backbone chemistry modifications, and our stereopure oligonucleotides compared with stereorandom oligonucleotides. 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 DMD Community Contact: Chelley Casey +1 617-949-4830 ccasey@wavelifesci.com Source: Wave Life Sciences USA, Inc.

Visionary sector leaders Dan Menichella and Jo Brewer, PhD join as Non-Executive Directors Bringing strengths in strategy, immuno-oncology science execution from bench to approval, major partnerships and collaborations, and biotech scale up To support the acceleration of proof-of-concept cancer vaccine, ITOP1, into Phase I/IIa clinical development, to prevent the recurrence of oesophageal cancers OXFORD, England, March 20, 2025 /PRNewswire/ -- Infinitopes, a pioneering precision immuno-oncology company, today announced the appointments of Dan Menichella and Jo Brewer, PhD as Non-Executive Directors to its Board as the company prepares to transition into a clinical stage company with lead vaccine candidate, ITOP1, to treat patients with oesophageal cancer. Dan Menichella is a seasoned biotech executive, who brings extensive experience in vaccine development, strategic partnerships, and rapid corporate growth. As CEO and CBO of CureVac's U.S. subsidiary, he built out the Boston operation, delivered four deals in his first year – including a multi-product cancer vaccine collaboration with Eli Lilly worth up to $1.7 billion. He became CEO of CureVac AG in 2018. Before CureVac, Dan served as CBO at Bamboo Therapeutics, where he spearheaded the in-licensing of a Duchenne muscular dystrophy programme, unlocking the company's $43 million series A and subsequent $645 million buyout by Pfizer. He has significant fundraising experience and led many large transactions, including partnerships with Lilly, Biogen, and Takeda, and supported IPO's and successful exits. Dan's proven ability to forge high-value alliances and advance breakthrough therapeutics underscores his expertise in guiding life science ventures to success. Dan Menichella, incoming Non-Executive Director at Infinitopes, added: "I am honoured to join Infinitopes' Board of Directors at this exciting time for the company, as we begin the Phase I/IIa clinical trial in oesophageal cancer. Infinitopes is a highly innovative company with a strong commitment to cancer research. Its approach holds the promise to revolutionise cancer treatments for patients, and I am excited and committed to support Jonathan and the team as they embark on this groundbreaking path." Jo Brewer, PhD, is a recognised industry leader in cancer immunotherapy and cell therapies, with over 20 years of experience bringing cutting-edge treatments from discovery to the clinic. Currently serving as CSO at Adaptimmune, she has been instrumental in developing autologous and allogeneic T-cell therapies, leading to Tecelra®, the first engineered TCR-T cell therapy for solid tumours to reach the market. As a founding scientist at Adaptimmune, Jo played a critical role in the development of multiple immunotherapies, including lete-cel for sarcoma and uza-cel, now partnered with Galapagos. She has built and led multiple research teams at Adaptimmune and played a significant role in the upscaling from a small biotech to 500+ employees, driving innovation across discovery and translational science. Jo Brewer, PhD, incoming Non-Executive Director at Infinitopes, remarked: "Infinitopes, with its pioneering approach to precision cancer vaccines, driven by its innovative AI/ML-powered Immunomics™ platform, has the potential to revolutionise how we target and treat cancers. The precision and speed that its world-leading clinicians and scientists are advancing its novel immunotherapies is truly impressive, and I look forward to joining the Board at such a critical time of clinical development, to contribute my expertise to bringing these transformative treatments to patients." Jonathan Kwok, Chief Executive Officer & Co-Founder at Infinitopes, said : "I'm delighted to welcome Dan and Jo to our Board at a pivotal time, as we prepare to begin our first clinical trial for ITOP1, our proof-of-concept cancer vaccine candidate, initially targeting oesophageal cancers. Their leadership and deep expertise in immuno-oncology and biotech growth is invaluable as we advance our AI/ML-driven antigen discovery platform and push the boundaries of what is possible in cancer vaccine development. Their decision to join Infinitopes reflects the strength of our technologies, our ambitious mission, and the future potential impact for patients worldwide." In April 2024 Infinitopes announced its oversubscribed £12.8 million seed financing to advance its AI/ML Precision Immunomics™ bespoke antigen discovery platform. This proprietary technology enables the safe and highly accurate design of 'best-in-class' therapeutics for multiple cancer indications. Guided by an exceptional leadership team and distinguished Scientific Advisory Board (SAB) of world-class clinicians, scientists, and regulatory experts, Infinitopes is poised to initiate its vaccine candidate, ITOP1, in a highly anticipated double-blind, randomised, placebo-controlled Phase I/IIa clinical trial. This trial is aimed at treating patients with oesophageal cancer and is expected to begin at four UK NHS university cancer centres in the first half of 2025. About Infinitopes Infinitopes Ltd is a soon to be clinical stage, integrated cancer biotechnology company supported by Cancer Research UK (CRUK) and the University of Oxford. The Company combines two world leading platforms, in precision target discovery and in high efficiency, vector delivery systems, to develop immunologically durable vaccines against multiple solid tumour indications. The lead vaccine candidate is expected to begin Phase I/IIa trials in H1' 25. Infinitopes has gathered together in-house talent across antigen discovery, immunology, vaccinology, oncology, biomanufacturing, clinical trials and regulation, winning an 'Innovative Licensing and Access Pathway' (ILAP) innovation passport from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in 2022. Infinitopes has also won two prestigious, maximum size, nondilutive awards from Innovate UK, a Cancer Therapeutics Award (in 2022) and a Future Economy Investor Partnership (in 2023). Since incorporation, the Infinitopes team has raised nearly $20m from sector expert investors including Cancer Research Horizons, Cancer Research Institute, Kindred Capital, Manta Ray Ventures, Martlet, Meltwind, Octopus Ventures, Saras Capital, Wilbe and the Fundación CRIS Contra el Cáncer, funding its rapid growth from three academic co-founders to 28 full time equivalents. It is now the largest tenant of Oxford University's BioEscalator innovation accelerator. For more information, visit SOURCE Infinitopes WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Collaboration enhances efficiency and quality in liver recovery and availabilityOXFORD, United Kingdom and NEW YORK, March 17, 2025 (GLOBE NEWSWIRE) -- OrganOx Ltd., the pioneering company transforming organ transplantation and care with innovative technology, has announced a new partnership with ProCure On-Demand, a leading comprehensive organ recovery service provider in the U.S. The collaboration aims to significantly improve the recovery and assessment of livers for transplantation by combining OrganOx’s cutting-edge normothermic machine perfusion (NMP) technology with ProCure’s advanced surgical, perfusion, preservation, and logistics solutions. Through this partnership, OrganOx will support ProCure’s nationwide clinical teams by providing education and certification through its exclusive training program. This collaboration will offer transplant centers and Organ Procurement Organizations (OPOs) additional clinical services to aid in the recovery process, ultimately aiming to improve transplant outcomes for more patients. “Increasing the number of healthy, viable livers available for transplant is crucial as the global incidence of liver disease continues to rise,” said Craig Marshall, Chief Executive Officer of OrganOx. “Our partnership with ProCure On-Demand brings together a flexible range of high-quality services and resources to meet the unique needs of clinical teams and organizations, ensuring more successful transplants.” “We are excited to be partnering with an innovator like OrganOx,” commented Dr. Zachary Kon, ProCure’s CEO and Co-Founder. “We share many of the same foundational values including a commitment to saving lives by ensuring that every organ counts. Together, we are focused on making the liver transplant waitlist a thing of the past.” About OrganOxOrganOx is a commercial stage organ technology company, spun out of the University of Oxford in 2008, dedicated to developing technologies to improve outcomes for patients with acute or chronic organ failure. The OrganOx metra® is a normothermic machine perfusion (NMP) platform approved in the U.S., Europe, Canada, and Australia. It has been utilized in over 5,000 liver transplants to date to keep donor livers in a metabolically active state outside the body so that functional assessment of the organ can be performed prior to transplant, enabling an increased number of organs available for transplant. Learn more at www.organox.com. About ProCure On-DemandProCure On-Demand is transforming the organ transplant ecosystem and enabling more thoracic and abdominal organs to reach recipients in need. Founded as a Public Benefit Corporation, ProCure provides professional, high-quality organ recovery services worthy of the first step in the transplant process. ProCure’s flexible model offers a nationwide network of recovery surgeons and clinical professionals, advanced logistics and engagement with innovative technology solutions enabling more patients to receive the transplants they need. For more information, visit www.procureodx.com. ContactsOrganOx Investor Relations: Steve Deitsch Chief Financial Officer, OrganOx investorrelations@organox.com OrganOx Media:Emma YangHealth+Commerceemma@healthandcommerce.comProCure Media:Aaron CohenAaron Cohen PRaaroncohenPR@gmail.com301-633-6773