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Business Wire
11-08-2025
- Business Wire
Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present new data from its hypertension and transthyretin amyloidosis (ATTR) programs at the upcoming European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain, from August 29 - September 1, 2025. These presentations will highlight the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and reinforce Alnylam's commitment to advancing innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options. Highlights include data from Cohort A of the KARDIA-3 Phase 2 study evaluating the investigational RNAi therapeutic, zilebesiran, in patients with uncontrolled hypertension and high cardiovascular risk. This analysis will be presented as a late-breaking abstract in the Hot Line 4 session on August 30, 2025. Data from the Company's flagship TTR franchise will also be presented, including new analyses from the HELIOS-B Phase 3 study of AMVUTTRA ® (vutrisiran), which delivers rapid knockdown of transthyretin, in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). These presentations include results of 12-month follow-up from the ongoing open-label extension (OLE) period of HELIOS-B, which will provide further insights into the sustained longer-term benefits of treatment with AMVUTTRA, and an analysis examining the effect of AMVUTTRA on days lost to death and/or hospitalization (DLDH). ESC Congress Presentation Details Hypertension Abstracts KARDIA-3: Zilebesiran as add-on therapy in adults with hypertension and established cardiovascular disease or at high cardiovascular risk Hot Line 4 Session Presentation Time: Saturday, August 30, 2025, 4:30 – 4:45 pm (CEST), 10:30 – 10:45 am (EDT) Presenting Author: Neha Pagidipati, U.S. Room: Madrid, Main Auditorium Impact of zilebesiran, an investigational RNA interference therapeutic targeting hepatic angiotensinogen, on renin–angiotensin system biomarkers in patients with mild-to-moderate hypertension Moderated ePoster: Treatment of Hypertension: Randomized Controlled Trials Time: Saturday, August 30, 2025, 5:15 – 6:00 pm (CEST), 11:15 am – 12:00 pm (EDT) Presenting Author: Michael A Weber, U.S. Room: Station 4, Research Gateway ATTR Abstracts Vutrisiran reduces days lost to death and/or hospitalization versus placebo in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial Moderated ePoster: Multidisciplinary Care in Heart Failure Time: Sunday, August 31, 2025, 9:15 – 10:00 am (CEST), 3:15 – 4:00 am (EDT) Presenting Author: Mazen Hanna, U.S. Room: Station 3, Research Gateway HELIOS-B: 12-month results from the open-label extension period of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy Abstract Session: Therapeutic Advances in Amyloid Cardiomyopathy Time: Sunday, August 31, 2025, 11:15 – 11:25 am (CEST), 5:15 – 5:25 am (EDT) Presenting Author: Pablo Garcia-Pavia, Spain Room: Science Box 2 (Research Gateway) Investor Webcast Information Alnylam management will discuss the KARDIA-3 results in a live event which will be webcast on August 30, 2025, at 1:00 p.m. ET (7:00 pm CEST). The webcast will be available on the Investors section of the Company's website at An archived webcast will be available on the Alnylam website approximately two hours after the event. AMVUTTRA ® (vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION Indications In the EU, AMVUTTRA ® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, Europe, Brazil, Japan and UAE. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR). For US Media only: For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit For EU Media: For the full EU Summary of Product Characteristics, see About Zilebesiran Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of hypertension to reduce cardiovascular risk in high unmet need populations. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a role in blood pressure (BP) regulation. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein, and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent biannual subcutaneous dosing, increased selectivity and the potential to achieve continuous control of BP to impact cardiovascular risk. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide. 1-4 About Cardiovascular Disease and Hypertension Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. 5,6 Hypertension is the primary cause of and number one modifiable risk factor for CVD. 7 An estimated 1 in 3 adults worldwide have hypertension, and, despite wide availability of antihypertensives, up to 80% of all patients, and up to a third of treated patients, do not reach and maintain blood pressure (BP) targets. 8 Even when blood pressure appears well managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 9-15 These patients require novel approaches that not only reduce BP, but also lower overall cardiovascular risk. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. 16 Its discovery has been heralded as 'a major scientific breakthrough that happens once every decade or so,' and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. 17 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. 16 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its ' Alnylam P 5 x25 ' strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam's expectations regarding the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and genetic and other conditions; Alnylam's ability to advance innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options; the safety and efficacy of vutrisiran for the treatment of ATTR-CM and the safety and efficacy of zilebesiran for the treatment of hypertension; and Alnylam's ability to achieve its ' Alnylam P 5 x25 ' strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam's ability to successfully execute on its ' Alnylam P 5 x25 ' goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. 5 GBD 2021 Causes of Death Collaborators. Lancet. 2024;403:2100-2132. 6 Lindstrom M, DeCleene N, Dorsey H, et al. J Am Coll Cardiol. 2022;80:2372-2425. 7 Yusuf S, Joseph P, Rangarajan S, et al. Lancet. 2020;395:795-808. 8 NCD Risk Factor Collaboration (NCD-RisC). Lancet. 2021;398:957-980. 9 Ebinger JE, Driver M, Ouyang D, et al. eClinicalMedicine. 2022;48:101442. 10 Kario K. Prog Cardiovasc Dis. 2016;59:262-281. 11 Doumas M, Tsioufis C, Fletcher R, et al. J Am Heart Assoc. 2017;6:e006093. 12 Mezue K, Goyal A, Pressman GS, et al. J Clin Hypertens. 2018;20:1247-1252. 13 Rothwell PM, Howard SC, Dolan E, et al. Lancet. 2010;375:895-905. 14 Tatasciore A, Renda G, Zimarino M, et al. Hypertension. 2007;50:325-332. 15 Mokadem ME, Boshra H, El Hady YA, et al. J Hum Hypertens. 2019;34:641-647. 16 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498. 17 Zamore P. Cell. 2006;127(5):1083-1086.
Business Wire
29-07-2025
- Business Wire
Viz.ai Announces CMS Reimbursement Pathway for Hypertrophic Cardiomyopathy via AI-Enhanced Electrocardiogram
SAN FRANCISCO--(BUSINESS WIRE)-- the leader in AI-powered disease detection and intelligent care coordination, today announced that the Category III CPT codes for AI-enabled electrocardiogram (ECG) analysis accepted and established by the American Medical Association (AMA) CPT Editorial Panel establish a national reimbursement framework for AI algorithms that perform ECG analysis for cardiac pathology, including conditions like hypertrophic cardiomyopathy (HCM), for which Viz HCM was designed. The Centers for Medicare & Medicaid Services established a Medicare rate of $128.90, effective January 1, 2025, for CPT codes 0764T and 0765T which apply to 'electrocardiograph, computerized analysis with artificial intelligence, for detection of cardiac pathology, with physician or other qualified health care professional interpretation and report' related to concurrently performed ECG or previously performed ECG respectively. These codes facilitate national reimbursement for AI algorithms like Viz HCM, which analyzes 12-lead ECGs to identify patterns consistent with HCM, a condition that often goes undiagnosed until serious complications arise. 'This is a major milestone for AI in cardiovascular care,' said Jamie Stern, senior director of Care Pathways at 'Reimbursement for AI-powered ECG interpretation empowers clinicians to identify high-risk patients earlier, before symptoms progress or serious events occur—supporting more timely diagnosis, specialist referral, and treatment.' Hypertrophic cardiomyopathy is one of the most common inherited heart conditions and a leading cause of sudden cardiac death, particularly in younger adults. Yet the majority of individuals living with HCM are undiagnosed. Viz HCM uses deep learning to flag patients at risk based on ECG data captured across a healthcare system in routine clinical workflows, advancing early detection and closing critical gaps in care. A recent study, published in JACC: Clinical Electrophysiology, demonstrated that Viz HCM achieved a high degree of accuracy in detecting HCM 1. The AI-ECG successfully identified 574 HCM patients, and in 691 cases where HCM was not identified the AI-ECG assisted in identifying alternate clinically relevant diagnosis, highlighting Viz HCM's value for more effective disease detection. "Reimbursement is a meaningful step forward for the uptake of novel technologies,' said Joshua M. Lampert, MD, FACC, cardiac electrophysiologist and medical director of Machine Learning at Mount Sinai Fuster Heart Hospital. 'This development can provide institutions with a sustainable means to build and maintain the necessary infrastructure to provide safe and effective care for patients during an actively evolving healthcare modernization process." The CPT codes 0764T and 0765T were first published in the CPT 2023 code set, effective for use on or after January 1, 2023. However, CMS had not established a national payment for these codes until January 1, 2025. This development enables physicians and health systems to integrate reimbursable AI-based ECG interpretation into routine care delivery. Viz HCM is part of the One Platform, which connects disparate data and care teams in real time to accelerate diagnosis, streamline care coordination, and improve outcomes across a range of cardiovascular and neurological conditions. 1 Desai, M. Y., Rutkowski, K., Ospina, S., et al. (2025). Real‑world artificial intelligence–based electrocardiographic analysis to diagnose hypertrophic cardiomyopathy. JACC: Clinical Electrophysiology, 11(6). About is the pioneer in the use of AI algorithms and machine learning to increase the speed of diagnosis and care across 1,800 hospitals and health systems in the U.S. and Europe. The AI-powered One® is an intelligent care coordination solution that identifies more patients with a suspected disease, informs critical decisions at the point of care, and optimizes care pathways and helps improve outcomes. Backed by real-world clinical evidence, One delivers significant value to patients, providers, and pharmaceutical and medical device companies. For more information visit
Business Wire
21-07-2025
- Business Wire
PBI-Gordon Companies, Inc. Calls on Petspan to Cease and Desist False and Misleading Advertisement, Promotion and Sale of Compounded Rapamycin (Sirolimus) for HCM in Cats
SHAWNEE, Kan.--(BUSINESS WIRE)--PBI-Gordon Companies, Inc. the parent company of TriviumVet, which holds sole conditional new animal drug approval from the FDA for Felycin ® -CA1 (sirolimus delayed-release tablets) under its affiliated brand company Pegasus, through its outside counsel issued a cease-and-desist demand letter to Petspan for falsely promoting a compounded version of rapamycin (synonymous with sirolimus) for the treatment and management of clinical hypertrophic cardiomyopathy (HCM) in cats. Petspan's evaluation and prescribing practices do not comply with established veterinary practice principles and places the health and wellbeing of cats in jeopardy. Felycin-CA1 is the first and only drug to receive FDA conditional approval for management of ventricular hypertrophy with subclinical HCM in cats. It is also the only conditionally approved drug for which treatment claims are permitted based on studies that were conducted by or on behalf of the holder of the cNADA, TriviumVet. This conditional approval was announced in an FDA press release dated March 14, 2025. Pegasus Laboratories, Inc. ('Pegasus'), another wholly owned subsidiary of PBI-Gordon, launched Felycin-CA1 under the PRN Pharmacal brand at the American College of Veterinary Internal Medicine Forum held June 17-20, 2025, where veterinarians and other participants in attendance were made aware of the availability of Felycin-CA1 through standard distribution channels. PBI-Gordon asserts Petspan is engaging in a pervasive pattern of intentionally false and misleading communications aimed at both veterinarians and pet owners, including claiming product legitimacy from two clinical trials (RAPACAT and HALT-HCM trials) sponsored and conducted by TriviumVet, not Petspan. TriviumVet is currently conducting the HALT-HCM study which is necessary to produce the data required to advance Felycin-CA1 from a conditionally approved drug to a fully approved drug. Petspan's false and misleading public assertions imply that the safety and efficacy of TriviumVet's Felycin-CA1 are applicable to the Petspan compounded product. 'Petspan is illegally marketing an unapproved, unlicensed and potentially unsafe would-be copy of our product, and that has to be stopped,' said PBI-Gordon Companies, Inc. President & CEO Steve Clifford, 'We will take action to stop those who threaten patient safety and urgently call on regulators and law enforcement to do the same.' PBI-Gordon intends to pursue all necessary actions and legal remedies to ensure its product and intellectual property are protected against the false and misleading advertisement and sale of all unapproved versions of sirolimus for HCM, online or otherwise. Feline HCM is extremely common, affecting 1 in 7 of all pet cats. In animals 9 years of age and older, the prevalence rises to approximately 1 in 3 and is a leading cause of mortality in adult cats. The disease is characterized by abnormal thickening and impaired function of the ventricular wall of the heart. While some cats remain asymptomatic, around half go on to develop congestive heart failure or other severe complications. Subclinical HCM refers to cases that have ventricular wall thickening but have not yet developed clinical signs. 'Petspan's unlawful sale of its compounded drug – called Rapamycin – and its false and misleading promotion, poses a real danger to thousands of cats with this condition, and is a callous affront to their owners and veterinarians,' said Dr. Heather Davis DVM, PhD, DACVS-LA, Director of Clinical Affairs and Veterinary Services, Pegasus Laboratories, Inc. PBI-Gordon is also aware that Petspan advertises the diagnosis of HCM and prescription of its compounded 'rapamycin' through telemedicine visits with purported veterinarians. Current gold standard as established by veterinary boards, is to diagnose HCM in-person which involves a veterinarian physically performing a thorough evaluation to rule out other medical conditions that may present as HCM; clinical pathology sampling at a minimum is required in addition to physical examination to make such an assessment. Virtual telemedicine visits are insufficient to establish the relationship, diagnosis, and treatment protocol implementation for HCM in cats. A virtual treatment protocol that misdiagnoses HCM risks negative adverse events to the pet and harm to the future utility of Felycin-CA1 for HCM. More specifically, the letter demands that Petspan immediately cease and desist the following activities: Advertising compounded rapamycin (sirolimus) for the treatment of HCM in cats Relying on studies conducted by or on behalf of TriviumVet as the basis for the Petspan treatment protocol Posting misleading promotional and advertising materials on Petspan's website and social media, including references to studies conducted by or on behalf of TriviumVet Confusing the public by using a synonym for the drug (sirolimus) for which FDA approved the cNADA (i.e., 'rapamycin') About PBI-Gordon Companies PBI-Gordon Companies has been in business in the Kansas City metro area since 1947 and is 100 percent employee-owned. It is the parent company to four subsidiaries which develop, manufacture, and market leading products for Turf and Ornamental Industries (PBI-Gordon Corporation), Companion Animal Pharmaceuticals (Pegasus Laboratories & TriviumVet), and Companion Animal Nutritional Supplements and Grooming Supplies (PetAg). About Pegasus Laboratories Founded in 1986, Pegasus Laboratories is a pharmaceutical development and manufacturing organization focused on novel products to treat chronic conditions in cats, dogs, and horses. Pegasus provides full-service Contract Development and Manufacturing Organization (CDMO) focusing on the development and manufacturing with full turn-key technical services in a DEA-approved CGMP facility. Pegasus also provides a full portfolio of pharmaceuticals, nutritional therapeutics and supplements, parasite control and surgical and wound care under the PRN® and Sē·Qual™ brands. About TriviumVet TriviumVet is an Ireland-based research and development company. Working with some of the leading veterinarians and therapeutic specialists around the world to address the gaps in disease management by developing innovative new therapeutic solutions for some of the most serious and underserved conditions. Focused on finding effective treatments for unmet needs in cardiology, renal health, pain management, gastroenterology, wellness testing, and age-related diseases.
