Transthyretin cardiac amyloidosis

PALO ALTO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, announced today that outcomes data through 42 months from the ongoing long-term open-label extension of ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be shared in a featured science oral presentation at the American Heart Association (AHA) Scientific Sessions, taking place in Chicago, Illinois on November 16 – 18, 2024. In addition to the presentation, BridgeBio was selected to share three posters in moderated poster sessions on ATTR-CM. Featured Science Oral Presentation:Acoramidis Reduces All-Cause Mortality (ACM) and Cardiovascular-Related Hospitalization (CVH): Initial Outcomes from the ATTRibute-CM Open-Label Extension (OLE) StudySession Title: Featured Science: Amyloid, Hypertrophic, and Danon Cardiomyopathies: Targeted Therapies and Specific PopulationsPresenter: Daniel Judge, M.D. of Medical University of South CarolinaDate: Monday, November 18 at 9:45 am CT/10:45 am ET Moderated Digital Posters:Costs and Healthcare Resource Utilization in Transthyretin Amyloid Cardiomyopathy Exceeds That of Generalized Heart Failure Session Title: Socioeconomic Insights and Innovations in Heart FailurePresenter: Justin Grodin, M.D. of UT Southwestern Medical CenterDate: Sunday, November 17 at 10:00 am CT/11:00 am ET Evolving Baseline Risk in Patients with Transthyretin Amyloid Cardiomyopathy: A Systematic Literature Review of Clinical Trials Session Title: Cardiac Amyloidosis 2024: Advances in Prognostication and Management Presenter: Ahmad Masri, M.D. of Oregon Health & Science UniversityDate: Sunday, November 17 at 11:10 am CT/12:10 pm ET Acoramidis Improved Survival in Patients with Transthyretin Cardiac Amyloidosis Regardless of Prior Cardiovascular Hospitalization Session Title: Cardiac Amyloidosis 2024: Advances in Prognostication and Management Presenter: Kevin Alexander, M.D. of Stanford University School of MedicineDate: Sunday, November 17 at 11:20 am CT/12:20 pm ET The presentation and posters will be available following the sessions on the Presentations page within the Investors section of the BridgeBio website at https://investor.bridgebio.com. About BridgeBio Pharma, Inc.BridgeBio Pharma (BridgeBio) is a commercial-stage 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 Contact:Vikram Balicontact@bridgebio.com(650)-789-8220

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company will present results from the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy at the European Society of Cardiology (ESC) Congress 2024, taking place in London, UK, on August 30 - September 2, 2024. The Company recently announced positive topline results of the study which met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events, as well as all secondary endpoints, in both the overall population and in the monotherapy population, along with an acceptable safety profile and tolerability, consistent with its established profile. The Company will also host an Investor Webcast to discuss the HELIOS-B results on August 30, 2024. Vutrisiran is in development for the treatment of ATTR amyloidosis with cardiomyopathy. In addition, the Company will present findings from a subgroup analysis of the KARDIA-2 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension, as well as new data from post-hoc analyses of the APOLLO-B Phase 3 and APOLLO-OLE studies of patisiran in patients with ATTR amyloidosis with cardiomyopathy. Patisiran is not approved for the treatment of ATTR amyloidosis with cardiomyopathy. ESC Presentation Details Primary Results from HELIOS-B, a Phase 3 Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy London: Hot Line 1 Friday, August 30, 2024, 11:00 a.m. (BST), 6:00 a.m. (ET) Presenter: Marianna Fontana, M.D., Ph.D. Subgroup Results from KARDIA-2: Impact of Demographic and Baseline Disease Characteristics on Zilebesiran Response in Patients with Hypertension Uncontrolled by a Standard Oral Antihypertensive Science Box 4: Pharmacological Management in Hypertension Friday, August 30, 2024, 2:30 p.m. (BST), 9:30 a.m. (ET) Presenter: Manish Saxena, MBBS, MSc, FBHS Long-term Effects of Patisiran on Survival and Cardiac Parameters in Patients with Transthyretin-Mediated Cardiac Amyloidosis: Post-hoc Analyses of APOLLO-B and Cardiac Subpopulation of APOLLO-OLE Science Box 1: Advances in Amyloidosis Sunday, September 1, 2024, 5:36 p.m. (BST), 12:36 p.m. (ET) Presenter: Olivier Lairez, M.D. Investor Webcast Information Alnylam Management will discuss the HELIOS-B results via webcast on August 30, 2024, at 1:00 p.m. (BST), 8:00 a.m. (ET). A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events . An archived webcast will be available on the Company’s website approximately two hours after the event. About AMVUTTRA ® (vutrisiran) AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. In the UK, vutrisiran is specifically indicated for the treatment of hATTR in adult patients with stage 1 or stage 2 polyneuropathy. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. For US Media only : For more information about vutrisiran, including the full U.S. Prescribing Information , visit AMVUTTRA.com . For UK Media: For the UK Summary of Product Characteristics, see https://www.medicines.org.uk/emc/product/14060 . About ONPATTRO ® (patisiran) ONPATTRO ® (patisiran) is an RNAi therapeutic designed to silence TTR messenger RNA and reduce the production of TTR protein in the liver. Administered intravenously, patisiran is approved in forty countries for the treatment of the polyneuropathy of hereditary transthyretin-amyloidosis (hATTR-PN) in adults. For US Media only : For more information about patisiran, including the full U.S. Prescribing Information , visit ONPATTRO.com . For UK Media: For the UK Summary of Product Characteristics, see https://www.medicines.org.uk/emc/product/10368 . 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. About Zilebesiran Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations. AGT is the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a demonstrated role in blood pressure (BP) regulation and its inhibition has well-established anti-hypertensive effects. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein and ultimately, in the vasoconstrictor angiotensin II. Zilebesiran utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent subcutaneous dosing with increased selectivity and the potential to achieve tonic blood pressure control demonstrating consistent and durable blood pressure reduction throughout a 24-hour period, sustained up to six months after a single dose of zilebesiran. 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 Hypertension Uncontrolled hypertension is the chronic elevation of blood pressure (BP), defined by the 2017 ACC/AHA guidelines as ≥130 mmHg systolic blood pressure (SBP) and ≥80 mmHg diastolic blood pressure (DBP). More than one billion people worldwide live with hypertension. Approximately one in three adults are living with hypertension worldwide, with up to 80% of individuals remaining uncontrolled despite the availability of several classes of oral anti-hypertensive treatments. Despite the availability of anti-hypertensive medications, there remains a significant unmet medical need, especially given the poor rates of adherence to existing daily oral medications, resulting in inconsistent BP control and an increased risk for stroke, heart attack and premature death. In particular, there are a number of high unmet need settings where novel approaches to hypertension warrant additional development focus, including patients with poor medication adherence and in patients with high 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. 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. By harnessing the natural biological process of RNAi occurring in our cells, a 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. 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 planned presentation of detailed results from the HELIOS-B study as well as from other studies, the safety and efficacy of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy, and the potential for Alnylam to achieve its Alnylam P 5 x25 vision of becoming a leading biopharma company 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 ” strategy; Alnylam’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including vutrisiran; actions or advice of regulatory agencies and Alnylam’s ability to obtain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2023 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 its other SEC filings. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

Researchers found that men with a higher proportion of blood cells missing Y chromosomes have a higher mortality rate from a common cause of heart disease called transthyretin cardiac amyloidosis (ATTR-CA), informing future treatment for patients with ATTR-CA. Researchers at Boston Medical Center (BMC) and Boston University (BU) Chobanian & Avedisian School of Medicine, in collaboration with an international team of scientists, shared findings from a new study published in the American Heart Association journal, Circulation: Heart Failure that explores a common cause of heart disease in older men called transthyretin cardiac amyloidosis (ATTR-CA). The study examines the relationship between spontaneous loss of the Y chromosome (LOY), a condition in aging men where the Y chromosome is spontaneously deleted in blood cells, and ATTR-CA, a progressive disease that causes heart failure and death. The team found that men with a higher proportion of blood cells missing Y chromosomes have a higher ATTR-CA mortality rate, informing future treatment for patients with ATTR-CA. The study team included investigators from Columbia University, University of Virginia, and Osaka Metropolitan Hospital in Japan. LOY is the most common acquired genetic mutation in men, with more than half of men in their early 90s having lost the Y chromosome in some of their blood cells according to the National Cancer Institute. While LOY has been associated with heart failure survival rates in large population studies, it has never been examined in relation to ATTR-CA. The current study suggests that men with ATTR-CA who have LOY in greater than 21.6% of their blood cells were 2.6 times more likely to not survive this form of heart disease. "Our study suggests that spontaneous LOY in circulating white blood cells contributes both to the development of ATTR-CA in men and influences the severity of disease," said Frederick L. Ruberg, MD, Chief of Cardiovascular Medicine at BMC, Professor of Medicine at BU Chobanian & Avedisian School of Medicine, and lead researcher in this study. "Additionally, our study's findings indicate that elevated LOY may be an important reason why some patients do not respond to the ATTR-CA therapy that is typically effective." Current treatments for ATTR-CA work well for many patients, but roughly 30 percent of patients do not respond to treatment, leading to hospitalization and death. Findings from this study support elevated LOY as a potential barrier to treatment response. The findings could one day inform a clinician's choice in designing a treatment course for a patient with ATTR-CA and high level of LOY in hopes of a more favorable health outcome. Additionally, the findings could lead to the development of new treatments for those with heart disease, including ATTR-CA. "Our study team represents an international collaboration that sought to explore an association between a common blood disorder and ATTR-CA that has never been previously considered," said Ruberg. "We provide evidence that these two conditions may be related, supporting a new way of understanding how ATTR-CA progresses as well as how to develop new potential targets for treatment."