Naxos Disease

RIDGE is Largest Natural History Study Conducted with More Than 175 Participants Enrolled to Date with Arrhythmogenic Right Ventricular Cardiomyopathy Due to Mutations in the PKP2 Gene Patients Experience High Burden of Arrhythmias and Severe Disease Progression Despite Standard-of-Care Treatments Large Majority of PKP2-associated ARVC Patients Appear Eligible for TN-401 Gene Therapy Based on Low Rates of Preexisting Immunity to AAV9 Antibodies SAN DIEGO, April 24, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced interim data from its ongoing RIDGE (NCT06311708) natural history and seroprevalence study of adults with arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the Plakophilin 2 (PKP2) gene will be presented at the Heart Rhythm Society’s annual Heart Rhythm meeting taking place in San Diego, CA from April 24-27, 2025. RIDGE is the largest non-interventional natural history and seroprevalence study of adults with PKP2-associated ARVC to date and was designed to collect and assess participants’ clinical characteristics and medical history, as well as to test for preexisting neutralizing antibodies to AAV9. PKP2 gene mutations result in insufficient levels of critical proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. Tenaya is developing TN-401 gene therapy as a potential treatment for PKP2-associated ARVC (also known as arrhythmogenic cardiomyopathy, or ACM). TN-401 gene replacement therapy is designed to deliver a functional PKP2 gene into heart muscle cells using an adeno associated virus serotype 9 (AAV9) capsid. “Interim data from the RIDGE observational study bring into focus what we’ve long understood about PKP2-associated ARVC – the current treatment approaches such as ablation or prescribed medications are insufficient to address the high burden of arrhythmias or alter the progression of disease, placing patients at increased risk for life-threatening ventricular tachycardias,” said Hugh Calkins, M.D., Catherine Ellen Poindexter Professor of Cardiology and Director, Johns Hopkins Hospital ARVD Program and an investigator for the RIDGE study. “I am encouraged by the potential of disease-modifying treatment approaches, such as TN-401 gene therapy, which is intended to alter the course of disease by delivering a functional PKP2 gene to the heart.” The RIDGE study has enrolled more than 175 patients at approximately 20 clinical sites in the U.S., UK, France, Germany, Italy and Sweden. As of the February 2025 cut off, clinical data from 144 adults with PKP2-associated ARVC were analyzed. Key findings being shared at Heart Rhythm 2025 include: Adults with PKP2-associated ARVC experience a high burden of arrhythmias despite treatments with standard of care medications including beta blockers and anti-arrhythmic medications such as flecainide, as well as surgical interventions such as ablation and implantable cardioverter-defibrillator (ICD) placement. 83 percent (95 of 115) of participants with available Holter data had a premature ventricular contraction (PVC) count of 500 PVCs/day or greater, a threshold linked with increased risk of life-threatening ventricular arrhythmias. 49 percent (56 of 115) of participants for whom Holter monitoring data was available had a history of ventricular tachycardia Current treatments appeared to do little to halt or prevent progressive structural changes to the heart that occur as a result of PKP2 mutations. Study participants show evidence of progressive structural changes that occur as a result of PKP2 mutations, in which the instability and disintegration of cellular structures in the desmosome results in fibrofatty scar tissue 60 percent of patients with MRI data available showed disease progression, as indicated by measures of right and left ventricular function, and heart tissue health Electrocardiogram results affirmed that a majority of patients had T-wave inversions (an indicator of ventricular strain or myocardial injury). Both QRS prolongation, which can indicate a delay in electrical conduction, and terminal active duration (TAD) were not present in a majority of participants, potentially indicating that changes in fibrofatty replacement and conduction system develop more slowly than arrhythmia symptoms. 93 percent of patients show AAV9 neutralizing antibody titers ≤1:40 A large majority of participants in the RIDGE study meet key eligibility criteria for participation in Tenaya’s ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy 83 percent (95 of 115) of participants with available Holter data had a premature ventricular contraction (PVC) count of 500 PVCs/day or greater, a threshold linked with increased risk of life-threatening ventricular arrhythmias. 49 percent (56 of 115) of participants for whom Holter monitoring data was available had a history of ventricular tachycardia 60 percent of patients with MRI data available showed disease progression, as indicated by measures of right and left ventricular function, and heart tissue health Enrollment is also underway in Tenaya’s Phase 1b clinical trial, known as RIDGE™-1, to assess the safety, tolerability and activity of a one-time dose of TN-401. Tenaya plans to report safety and biopsy data from the first cohort of RIDGE-1 patients in the second half of 2025. “The RIDGE natural history study provides a rich source of data detailing the disease characteristics and burden for adults with ARVC. We extend our heartfelt gratitude to the patients and investigators involved in this important study. Their continued support has been instrumental in achieving the progress we've made thus far,” said Whit Tingley, M.D., Ph.D., Tenaya’s Chief Medical Officer. “As the largest collection of prospective and retrospective patient demographic and medical history data, RIDGE is expected to serve as a control arm to assess activity and efficacy of the interventional TN-401 study in determining meaningful clinical and surrogate endpoints for measuring gene therapy’s efficacy in the PKP2-associated ARVC indication, as well as serving as an important means of engaging with patients and clinical sites as we enroll our Phase 1b trial of TN-401.” The interim data from RIDGE will be presented at Heart Rhythm 2025 on April 26, 2025 by Dr. Calkins during the Basic Translational Science session in a poster titled “Determining Eligibility for RIDGE™-1, a Phase 1b Interventional Study to Evaluate Safety and Efficacy of TN-401 Gene Therapy in Adults with Plakophilin-2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC): Interim Results of an Observational Study to Assess Seroprevalence to Adeno-Associated Virus Serotype 9 (AAV9)”. About PKP2-Associated ARVCPlakophilin-2 (PKP2) mutations are the most common genetic cause of arrhythmogenic right ventricular cardiomyopathy (ARVC, also known as arrhythmogenic cardiomyopathy or ACM), estimated to represent approximately 40 percent of the overall ARVC population. The prevalence of PKP2-associated ARVC is estimated at more than 70,000 people in the U.S. alone. In PKP2-associated ARVC, mutations of the PKP2 gene results in insufficient expression of a protein needed for the proper functioning of the desmosomal complex that maintains physical connections and electrical signaling between heart muscle cells. As the desmosome structure degrades, cardiac muscle cells are replaced by fibrofatty tissue and electrical pulses in the heart become unstable, resulting in irregular heart rhythms. ARVC symptoms include arrhythmias, palpitations, lightheadedness, dizziness and fainting. It is typically diagnosed before age 40, and sudden cardiac arrest due to life-threatening ventricular arrhythmias is frequently the first manifestation of disease. Current treatments include anti-arrhythmic medications, implantable cardioverter-defibrillators (ICDs) and ablation procedures, which do not address the underlying genetic cause of disease. About TN-401 Gene Therapy and the RIDGE-1 Clinical TrialTN-401 is an investigational AAV9-based gene therapy being developed for the treatment of ARVC due to mutations in the PKP2 gene. AAV9 was selected as the vector for delivery of Tenaya’s PKP2 gene therapy based on its extensive clinical and commercial safety record and demonstrated ability to target heart muscle cells. In preclinical studies, Tenaya has shown that a single dose of TN-401 restored healthy levels of PKP2 protein, normalized heart rhythms, improved right and left ventricular size and function and extended survival. TN-401 has received Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration. Tenaya is conducting the RIDGE-1 Phase 1b clinical trial of TN-401 in patients with PKP2-associated ARVC. The RIDGE-1 Phase 1b clinical trial is a multi-center, open-label, dose escalation study being conducted in the U.S. and UK. RIDGE-1 will assess the safety, tolerability and preliminary clinical efficacy of a one-time intravenous infusion of TN-401. RIDGE-1 will seek to enroll up to fifteen adults who have been diagnosed with PKP2-associated ARVC, have an implantable cardioverter defibrillator (ICD) and are at increased risk for arrhythmias as determined by PVC count during screening. To learn more about gene therapy for ARVC and participation in the RIDGE-1 study, please visit ARVCstudies.com or ClinicalTrials.gov (NCT06228924). About Tenaya TherapeuticsTenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya employs a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of genetic medicines aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. Tenaya’s pipeline includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), TN-301, a small molecule HDAC6 inhibitor intended for heart failure with preserved ejection fraction (HFpEF), and multiple early-stage programs in preclinical development. For more information, visit www.tenayatherapeutics.com. Forward Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “potential,” “expected,” “plans, “will,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the clinical, therapeutic and commercial potential of, and expectations regarding TN-401; expectation’s regarding the RIDGE data in support of determining meaningful clinical and surrogate endpoints for measuring gene therapy’s efficacy in the PKP2-associated ARVC indication; the planned timing to report initial data from RIDGE-1 and RIDGE; and statements made by Tenaya’s Chief Medical Officer. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the potential failure of TN-401 to demonstrate safety and/or efficacy in clinical testing; actions and decisions of regulatory agencies; the timing and progress of RIDGE-1; availability of RIDGE-1 and RIDGE data at the referenced time; the potential for any RIDGE-1 clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenaya’s continuing compliance with applicable legal and regulatory requirements; Tenaya’s ability to raise any additional funding it will need to continue to pursue its product development plans; Tenaya’s reliance on third parties; Tenaya’s manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.Tenaya ContactsMichelle CorralVP, Corporate Communications and Investor RelationsIR@tenayathera.com InvestorsAnne-Marie FieldsPrecision AQ annemarie.fields@precisionaq.com MediaWendy RyanTen Bridge Communicationswendy@tenbridgecommunications.com

Acoramidis’s hazard ratio of .41 for time to ACM or first CVH versus placebo in the ATTRibute-CM study subgroup of ATTRv-CM patients achieved statistical significance in a pre-specified analysisThis profound treatment effect, due to the near-complete stabilization (≥90%) and binding of TTR, represents the greatest observed benefit to date for ATTRv-CM patients, an ATTR-CM population with poor prognosis In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRw-CM patients: In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placeboA 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA™ by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency PALO ALTO, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today presented results showing statistically significant improvements in clinical outcomes as compared to placebo for time to all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH) in both variant (ATTRv) and wild-type (ATTRwt) transthyretin amyloid cardiomyopathy (ATTR-CM) patients from a pre-specified subgroup analysis of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a poster presentation by Margot Davis, M.D. of Vancouver General Hospital, Canada. Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. “Variant ATTR-CM patients’ condition often presents at an earlier age and progresses more rapidly than patients with wild-type disease, which translates into a worse prognosis in many such patients. We know that pathogenic TTR variant tetramers are less stable than the wild-type tetramer and this property is directly responsible for the more aggressive disease trajectory for these patients. The findings from the ATTRibute-CM trial clearly demonstrate that the rapid and sustained increases in serum TTR levels upon initiation of acoramidis treatment in variant ATTR-CM patients were similar if not greater than those observed in wild-type ATTR-CM patients,” said Jonathan Fox, M.D., Ph.D., president and chief medical officer of BridgeBio Cardiorenal. “This provides evidence that acoramidis is the only disease-modifying therapy that provides near-complete stabilization of TTR, improving clinical outcomes in both variant and wild-type ATTR-CM patients to an extent that is independently statistically significant in both subgroups.” “Given the significant unmet need for patients with ATTRv-CM, we are highly encouraged by the magnitude of efficacy seen with acoramidis. A pre-specified analysis in the ATTRv-CM population has shown a statistically significant 59% hazard reduction for the composite of ACM and CVH at Month 30, demonstrating that acoramidis can make a profound impact on patients’ lives,” said Kevin Alexander, M.D. of Stanford University School of Medicine. Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature.1 The serum TTR level increase with acoramidis was accompanied by a significant reduction in the risk of ACM or first CVH versus placebo in both the ATTRv-CM (59.1% risk reduction, .41 hazard ratio) and ATTRwt-CM (31.2% risk reduction, .69 hazard ratio) subgroups. Acoramidis treatment also led to a greater proportional increase in serum TTR in ATTRv-CM patients and achieved similar absolute serum TTR levels in both ATTRv- and ATTRwt-CM patients, which is an in vivo reflection of acoramidis’ near-complete (≥90%) TTR stabilization. Additional acoramidis poster presentations and moderated posters at the ACC Annual Scientific Sessions & Expo included: Acoramidis Improves NYHA Class at Month 30 Versus Placebo in Patients with ATTR-CM: Results from the ATTRibute-CM Study shared by Kevin Alexander, M.D. of Stanford University School of Medicine In the ATTRibute-CM study, acoramidis treatment resulted in a greater proportion of patients whose New York Heart Association (NYHA) Class was stable or improved at Month 30 vs placebo, indicating better stabilization in their heart failure symptoms and functional status. The NYHA classification system categorizes heart failure patients into four classes based on a clinical assessment of their physical activity limitations and associated symptoms due to their condition In Participants Treated with Acoramidis, Addition of Concomitant Tafamidis Did Not Further Increase Serum TTR Levels shared by Mathew Maurer, M.D. of Columbia University Irving Medical Center The poster showed that in patients with ATTR-CM, treatment with acoramidis significantly increased serum TTR levels whether compared to placebo alone or in those who received placebo as well as tafamidis. Conversely, the addition of tafamidis to acoramidis did not demonstrate any further increase in serum TTR levels, reflecting the lack of any additional stabilization benefit of serum TTR from tafamidis in vivo, as previously demonstrated in vitro. The safety profile in this limited dataset of concomitant acoramidis and tafamidis was similar to the overall safety profile of acoramidis alone Primary Endpoint Efficacy Results in the ATTRibute-CM Study: Pre-specified Sensitivity Analyses Addressed Tafamidis Use shared by Daniel P. Judge, M.D. of Medical University of South Carolina Pre-specified analyses showed consistent results with the primary analysis, demonstrating that the concomitant use of tafamidis did not alter the statistical significance of the primary efficacy endpoint Acoramidis-mediated Early Increase in Serum Transthyretin Level Reduces Cardiovascular-related Hospitalizations and Mortality: Insights from the ATTRibute-CM Study shared by Nitasha Sarswat, M.D. of UChicago Medicine In this post-hoc analysis of ATTRibute-CM, incremental increases in serum TTR levels on Day 28, achieved with acoramidis, may independently predict greater reduction in risks of cardiovascular mortality and of first CVH in patients with ATTR-CM Robustness of Primary Endpoint Efficacy Results with Acoramidis in ATTR-CM in the ATTRibute-CM Study: Pre-specified NT-proBNP Sensitivity Analyses shared by Jan Griffin, M.D. of Medical University of South Carolina This poster shows that pre-specified sensitivity analyses using higher N-terminal pro-type natriuretic peptide (NT-proBNP) thresholds for declaring a difference in the changes in NT-proBNP levels between the treatment arms showed consistent efficacy favoring acoramidis in patients with ATTR-CM. This confirms the robustness of the acoramidis treatment effect regardless of NT-proBNP progression thresholds Geographic Healthcare Disparities and Diagnostic Trends Among Patients with Transthyretin Amyloid Cardiomyopathy shared by Joshua Mitchell, M.D. of Washington University School of Medicine in St. Louis Findings presented show that the diagnosed prevalence of amyloid has significantly increased since 2017 in the setting of available treatment, improved awareness and less invasive diagnostics. However, there remain geographic disparities and racial differences in ATTR-CM prevalence Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTRv-CM patients is planned for future medical meetings. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. About Attruby™ (acoramidis)Attruby is the first near-complete (≥90%) stabilizer of Transthyretin (TTR) approved in the U.S. for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Attruby was generally well-tolerated. The most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation. BridgeBio offers an extensive suite of programs to help patients access our medicines. About BridgeBioBridgeBio Pharma (BridgeBio; NASDAQ:BBIO) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook. BridgeBio Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “continue,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our clinical development program for acoramidis for patients with transthyretin amyloid cardiomyopathy and the statements regarding the potential clinical benefits or of potential benefits for ATTR-CM patients in the quotes of Dr. Alexander, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA and other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, our ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, the impacts of current macroeconomic and geopolitical events, including changing conditions from, hostilities in Ukraine, and in Israel and the Gaza Strip, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com (650)-789-8220

Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been approved by the Food and Drug Administration (FDA) to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a potentially fatal disease of the heart muscle. The RNAi therapeutic has been authorised to treat the cardiomyopathy of wild-type or hereditary transthyretin (TTR)-mediated amyloidosis in adults to reduce cardiovascular mortality, hospitalisations and urgent heart failure visits. Approximately 150,000 people in the US are affected by ATTR-CM, in which misfolded TTR protein builds up and causes irreversible cardiovascular damage over time. The majority of patients remain undiagnosed and untreated, Alnylam outlined, adding that a significant portion of those who are treated with commonly used therapies experience disease progression. Given quarterly via a subcutaneous injection, Alnylam’s Amvuttra works with the body’s natural system to rapidly knock down TTR and is already approved in the US to treat the polyneuropathy of hereditary TTR-mediated amyloidosis. The company’s chief executive officer, Yvonne Greenstreet, said: “The FDA approval of Amvuttra for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease.” The FDA’s latest decision on the therapy was based on positive results from the phase 3 HELIOS-B trial, in which Amvuttra demonstrated statistical significance compared to placebo on all ten pre-specified primary and secondary endpoints. In the overall study population, which included patients receiving Amvuttra as a monotherapy and those using the therapy alongside standard treatments, Alnylam’s drug reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular events by 28% during the double-blind treatment period of up to 36 months. In the monotherapy population, Amvuttra reduced the risk of ACM and recurrent cardiovascular events by 33% in the double-blind period and the risk of mortality by 35% at month 42. Mortality in the overall population was also reduced by 36% at month 42. HELIOS-B investigator Ronald Witteles, Stanford University School of Medicine and Stanford Amyloid Center, said: “The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by [Amvuttra’s] ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease.”