Aankondiging • May 21

Wave Life Sciences Ltd. Announces Positive Update on RestorAATion-2 Trial: WVE-006 (GalNAc-RNA Editing) Achieves MZ-Like Phenotype Across Both Biweekly and Monthly Dosing

Wave Life Sciences Ltd. announced updated data from the ongoing RestorAATion-2 trial of WVE-006, its investigational, GalNAc-conjugated, subcutaneously delivered RNA editing oligonucleotide (AIMer) for alpha-1 antitrypsin deficiency (AATD). These data further affirm WVE-006’s potential as a novel therapeutic addressing both lung and liver AATD by generating healthy, wild-type M-AAT, reducing harmful Z-AAT, and restoring the ability to dynamically produce functional AAT protein when needed. Individuals with PiZZ AATD cannot produce healthy, wild-type M-AAT, have unhealthy, mutant Z-AAT aggregates in liver, and cannot dynamically increase AAT, a protein responsible for protecting lungs against ongoing damage. Heterozygous PiMZ individuals have low risk of lung or liver disease and circulating M-AAT levels ranging from 57% to 71% of total (mean=64%), based on analysis of natural history study samples measured with the same assay used in RestorAATion-2. The only approved treatment for AATD is weekly intravenous plasma-derived augmentation therapy, which only addresses lung manifestations and may leave individuals living with AATD at risk if AAT protein levels fall too low during an acute phase response. There are no approved therapies for AATD liver disease. Updated RestorAATion-2 clinical data: RestorAATion-2 is an ongoing open-label Phase 1b/2a trial with three dose cohorts (each n=8), with single and multidose portions. In the multidose portions, individuals receive multiple doses of WVE-006 (200 mg biweekly, 400 mg monthly, 600 mg monthly), over 12 weeks, followed by 12 weeks of follow-up. As of the data cutoff, data is available for the 200 mg (single and multidose), 400 mg (single and multidose), and 600 mg (single dose) cohorts. Circulating M-AAT, Z-AAT, and total (M + Z) AAT protein in the serum were measured by LC-MS/MS assays and reported as mean maximums. Reductions in Z-AAT protein: In PiZZ individuals, Z-AAT aggregates in the liver, where it can result in progressive liver disease. Treatment with WVE-006 led to robust, dose-dependent reductions of circulating, mutant Z-AAT from baseline following a single dose of WVE-006, reaching 47.3% (200 mg), 49.7% (400 mg), and 59.1% (600 mg). There were further reductions in Z-AAT with multiple doses of WVE-006, reaching 70.5% in the 200 mg biweekly dose cohort (seven doses). Reduction of Z-AAT was similar when extending the dosing interval, reaching 67.7% in the 400 mg monthly dose cohort (four doses). Restoration of wild-type M-AAT protein: Individuals with PiZZ AATD cannot produce wild-type M-AAT, the protein responsible for protecting the lungs against ongoing damage. Treatment with WVE-006 led to robust, dose-dependent restoration of wild-type M-AAT protein (canonical M-AAT) as a percentage of total circulating AAT following single doses of WVE-006, reaching 44.4% (200 mg), 48.0% (400 mg), and 52.3% (600 mg). In each single dose cohort, total AAT reached therapeutically relevant levels (200 mg: 12.9 µM; 400 mg: 14.0 µM; 600 mg: 13.0 µM). In the 200 mg multidose cohort, total AAT was 11.9 µM and M-AAT levels reached 64.4% of total AAT, in line with heterozygous MZ individuals with low risk of disease. A similar robust response was also observed when extending the dose interval to monthly (400 mg multidose cohort), where M-AAT levels reached 58.7% of total AAT. Total AAT in the 400 mg multidose cohort reached 13.6 µM. Restoration of dynamic AAT protein: Notably, following treatment with WVE-006, three instances of dynamic and rapid production of AAT protein due to acute phase responses were observed across RestorAATion-2 as indicated by concurrent C-reactive protein (CRP) and AAT elevation. As previously reported, in the 200 mg single dose cohort, one individual produced a significant increase in total AAT (20.6 µM) following an acute phase response related to a kidney stone. In the 400 mg multidose cohort, there were two additional instances of significant increases in AAT (57.8% and 59.8% versus pre-event) following acute phase responses to mild upper respiratory infection (common cold). Additionally, across all available RestorAATion-2 data to date, CRP increases are strongly correlated with increases in AAT (r=0.73, p<0.001, n=19). Convenient dosing and strong safety: Data support monthly subcutaneous dosing, with editing sustained at least three months following the last dose in both the 200 mg and 400 mg multidose cohorts. WVE-006 continues to be well tolerated with a favorable safety profile to date. All adverse events (AEs) were mild to moderate in intensity, and there were no SAEs or clinically meaningful liver function test elevations. Data from the RestorAATion-2 clinical trial were also highlighted earlier today at the American Thoracic Society (ATS) International Conference in an oral presentation by Kenneth R. Chapman, MsC, MD, FRCPC, FACP, FERS, Department of Medicine, University of Toronto. Anticipated upcoming milestones for WVE-006: Wave expects to receive regulatory feedback on a potential accelerated approval pathway mid-2026. Wave expects to share data from the 600 mg (monthly) multidose cohort in the second half of 2026.

Aankondiging • Mar 28

Wave Life Sciences Ltd. Announces Positive Interim Phase 1 Data From INLIGHT

Wave Life Sciences Ltd. announced new data from the Phase 1 portion of its first-in-human INLIGHT trial evaluating WVE-007, an investigational INHBE GalNAc-siRNA (SpiNA design), in otherwise healthy individuals living with overweight or obesity. After six months of follow-up, a single 240 mg dose led to further improvements in body composition, including fat loss with muscle preservation, with clinically meaningful reductions in visceral fat and waist circumference in a population with less fat and lower BMI than those in Phase 2 and 3 obesity studies. At 6-month follow-up, a single 240 mg dose of WVE-007 (INHBE GalNAc-siRNA) continued to drive significant placebo-adjusted reductions in visceral fat (-14%). WVE-007 led to greater improvement in body composition (visceral fat-to-muscle ratio) at six months versus that seen with weekly GLP-1 in later-stage trial (BMI: ~37 kg/m2), even with participants in Phase 1 portion of INLIGHT having substantially lower BMI (~32 kg/m2) and lower levels of fat than those in Phase 2 and 3 obesity studies. Durable and dose-dependent suppression of serum Activin E sustained through at least seven months continues to support expectations for once or twice-yearly dosing. WVE-007 continues to be generally safe and well tolerated. In the 240 mg cohort, six-month data demonstrated further improvements in body composition following a single dose of WVE-007, with statistically significant reductions in visceral fat and clinically meaningful reductions in waist circumference, as well as reductions in body weight. There were 32 participants in the 240 mg cohort (3:1 treatment to placebo) that had an average BMI of 32 kg/m2 and less visceral and subcutaneous fat than those in Phase 2 or 3 obesity studies. Placebo-adjusted percent change from baseline* 240 mg 3-month follow-up 6-month follow-up DEXA Visceral fat mass -7.8% -14.3% Total fat mass -5.1% -5.3% Lean mass +2.2% +2.4% Clinical measurements Waist circumference -0.4% -3.3% Body weight -0.3% -0.9%. The results were analyzed with a pre-specified statistical analysis (MMRM) utilizing all available data across all timepoints and dose groups at the time of the current data analysis and utilizing a pooled placebo comparator. The improvement in body composition as measured by visceral fat-to-muscle ratio (VMR) observed with a single 240 mg dose of WVE-007 (-16.5% vs. baseline) in the Phase 1 INLIGHT trial was greater than that achieved with weekly 2.4 mg doses of semaglutide at six months (-12.2% vs. baseline) in the BELIEVE Phase 2 clinical trial, which enrolled a higher BMI population (average 37 kg/m²). VMR is an established measure of body composition integrating harmful visceral fat and beneficial lean mass into a single index. Lower VMR is associated with a decreased risk of MASH, type 2 diabetes, and cardiometabolic disorders. Consistent, durable, and dose-dependent serum Activin E reductions sustained through at least seven months continue to support WVE-007’s potential for once or twice-yearly dosing, with a mean maximum reduction of up to 88%. WVE-007 continues to be generally safe and well tolerated to date up to 600 mg. There were no treatment discontinuations, and no severe or serious treatment emergent adverse events (TEAEs). All TEAEs in the treatment groups were mild or moderate, and all treatment-related adverse events were mild. There were no clinically meaningful changes in clinical laboratory measurements, including lipid profiles or liver function tests. In the 400 mg cohort, three-month data demonstrated placebo-adjusted reductions in visceral fat (-5.0%), total fat (-0.7%), with lean mass preservation (-0.2%) from baseline following a single dose. Notably, the 400 mg cohort had a leaner baseline body composition, with lower BMI and more participants (10 out of 24 individuals) with healthy levels of visceral fat (=500 g). A post-hoc analysis of the three-month results demonstrated a more robust and statistically significant average reduction in visceral fat (-7.8%, p500g), similar to that observed in the 240 mg cohort. These results emphasize the impact of baseline body composition on therapeutic effect and support expectations that evaluating individuals with higher BMI and visceral fat at baseline will lead to greater improvements in body composition and weight loss. Wave is on track to initiate the Phase 2a multidose portion of INLIGHT evaluating WVE-007 as monotherapy in individuals with higher BMI (35-50 kg/m2) and comorbidities in the second quarter of 2026. This placebo-controlled (3:1) Phase 2a study will include multiple assessments over a 12-month period, including body weight, waist circumference, body composition (MRI and DEXA), liver fat (MRI-PDFF), HbA1c, lipid levels, CRP, and muscle function. The study is expected to demonstrate further body composition improvements, including greater fat loss with preserved muscle, and additional weight loss. The results will inform further development of WVE-007 in obesity, as well as MASH, type 2 diabetes, and cardiovascular disease. The first assessment in this portion of the trial is planned for three months after participants have received their first dose. Additional data from INLIGHT, including data from the 600 mg Phase 1 SAD cohort, are expected in 2026. Wave expects to initiate the Phase 2a multidose portion of INLIGHT in individuals with higher BMI and comorbidities in the second quarter of 2026 and initiate new clinical trials evaluating WVE-007 as an incretin add-on and as post-incretin maintenance in 2026. WVE-007 is an investigational GalNAc-siRNA that utilizes Wave’s best-in-class proprietary oligonucleotide chemistry and the company’s Stereopure interfering Nucleic Acid (SpiNA) next generation siRNA design. WVE-007 is designed to silence INHBE mRNA, an obesity target with strong evidence from human genetics. In preclinical models, INHBE GalNAc-siRNA led to adipocyte shrinkage, fewer pro-inflammatory macrophages, less fibrosis, and improved insulin sensitivity in visceral adipose tissue, supporting potential for metabolic improvement. As an add-on to semaglutide, Wave’s GalNAc-siRNA doubled weight loss in mice and prevented weight regain upon cessation of semaglutide. INLIGHT is an ongoing randomized, placebo-controlled (3:1) clinical trial that includes a Phase 1, single-ascending dose portion in otherwise healthy individuals living with overweight or obesity. This portion is designed to address safety, tolerability, pharmacokinetics, and Activin E target engagement. INLIGHT is currently ongoing at multiple trial sites, including in the US. A Phase 2a portion of INLIGHT evaluating multiple WVE-007 doses in individuals with high BMI and comorbidities is expected to initiate in the second quarter of 2026.

Rente dekkingsratio	n/a
Contant	US$544.59m
Aandelen	US$511.73m
Totaal verplichtingen	US$71.81m
Totaal activa	US$583.54m

Gegevens	Laatst bijgewerkt (UTC-tijd)
Bedrijfsanalyse	2026/07/15 22:57
Aandelenkoers aan het einde van de dag	2026/07/15 00:00
Inkomsten	2026/03/31
Jaarlijkse inkomsten	2025/12/31

Pakket	Gegevens	Tijdframe	Voorbeeld Amerikaanse bron ^*
Financiële gegevens bedrijf	10 jaar	Resultatenrekening Kasstroomoverzicht Balans	SEC-formulier 10-K SEC-formulier 10-Q
Consensus schattingen analisten	+3 jaar	Financiële prognoses Koersdoelen analisten	Onderzoeksrapporten van analisten Blue Matrix
Marktprijzen	30 jaar	Aandelenprijzen Dividenden, splitsingen en acties	ICE Marktgegevens SEC-formulier S-1
Eigendom	10 jaar	Top aandeelhouders Handel met voorkennis	SEC-formulier 4 SEC-formulier 13D
Beheer	10 jaar	Leiderschapsteam Raad van bestuur	SEC-formulier 10-K SEC-formulier DEF 14A
Belangrijkste ontwikkelingen	10 jaar	Bedrijfsaankondigingen	SEC-formulier 8-K

Analist	Instelling
Alec Stranahan	BofA Global Research
Madison Wynne El-Saadi	B. Riley Securities, Inc.
Whitney Ijem	Canaccord Genuity

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Financiële gezondheid criteriumcontroles 6/6

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