Senile 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

New data to be presented will include biomarkers of disease progression and functional capacity from the ongoing Phase 1 study of nex-z, an investigational in vivo CRISPR gene editing therapy for ATTR amyloidosis CAMBRIDGE, Mass., Oct. 01, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex-z, also known as NTLA-2001) will be presented in a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, taking place November 16 – 18 in Chicago, Illinois. Nex-z is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The presentation will include safety, reduction in serum TTR and biomarkers of disease progression, and functional capacity data in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). Presentation Details Title: Nexiguran ziclumeran (nex-z, also known as NTLA-2001), an investigational in vivo CRISPR-based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM): interim report of the Phase 1 study Session: Late-Breaking Science 1: Celebrating a Century of Cardiovascular Science: From Prevention to Treatment, to Cure Session Type: Late-Breaking Science Date and Time: Saturday, November 16, 2024, from 8:30-9:45 a.m. CST Presenter: Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist, University College London Centre for Amyloidosis, London, UK Location: Main Event I About nexiguran ziclumeran (nex-z, also known as NTLA-2001)Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Nex-z is the first investigational CRISPR therapy to be administered systemically to edit genes inside the human body. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. About Transthyretin (ATTR) AmyloidosisTransthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein. About Intellia TherapeuticsIntellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx. Forward-Looking Statements This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding: the safety, efficacy, success and advancement of its clinical program for nexiguran ziclumeran (“nex-z”, also known as “NTLA-2001”) for transthyretin (“ATTR”) amyloidosis pursuant to its clinical trial applications and investigational new drug submission, including its potential to become the first one-time treatment for ATTR amyloidosis; and the expected timing of data releases, including the oral presentation planned for November 16 at the 2024 American Heart Association (AHA) Scientific Sessions. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including nex-z; the risk that any one or more of Intellia’s product candidates, including nex-z, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies, such as the Phase 1 clinical study of nex-z, will not be predictive of future results in connection with future studies for the same product candidate or Intellia’s other product candidates; and risks related to Intellia’s reliance on collaborations, including that its collaboration with Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. Intellia Contacts: Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communicationsian.karp@intelliatx.com Lina LiSenior Director, Investor Relations and Corporate Communicationslina.li@intelliatx.com Media:Matt CrensonTen Bridge Communicationsmedia@intelliatx.com mcrenson@tenbridgecommunications.com This press release was published by a CLEAR® Verified individual.

iStock/ jauhari1 As it nears a crucial FDA action date for its transthyretin amyloid cardiomyopathy candidate, BridgeBio focuses on its late-stage pipeline. Bay area–based biotech BridgeBio, founded in 2015 to be a bridge between promising early-stage academic work and patients with genetic diseases, is now a growing commercial enterprise, with two products on the market and another expecting an FDA decision by the end of November. As such, the company is making strategic moves, spinning out some assets into two new companies, cutting an entire gene therapy program and focusing its remaining resources on its late-stage programs in transthyretin amyloid cardiomyopathy and achondroplasia, which analysts say are key to BridgeBio’s success. “We have the capability, by our tenth birthday, to have had six approved products for a billion-plus dollar market,” Neil Kumar, the company’s charismatic founder and CEO told BioSpace . “So that’s a very, very productive engine.” However, commercial success requires the company to make some adjustments, Kumar continued. “Our investors would like to see us focus our resources to show that we can commercialize,” Kumar said, with a specific focus on the company’s later-stage pipeline. “We’ve shown we can do R&D, but now we have to show we can commercialize and build a business against these products.” To satisfy these investors, BridgeBio has adopted a hub-and-spoke model. In May 2024, it spun out its KRAS-focused cancer portfolio into BridgeBio Oncology Therapeutics , which it endowed with $200 million in initial funding. And last month, BridgeBio debuted GondolaBio to further develop early-stage assets for tuberous sclerosis complex, erythropoietic protoporphyria and alpha-A1 antitrypsin deficiency. Then this week, the company announced that it is discontinuing development of its gene therapy candidate BBP-631 after it failed to show “transformational results” in a mid-stage study in congenital adrenal hyperplasia (CAH). “The data we generated did not suggest that our efforts would result in a meaningful step forward for patients as compared to what we think standard of care will be in the coming years,” Kumar said. “Therefore, we felt our efforts would be better applied to other areas of unmet need,” though the company is seeking a partnership to continue development of BBP-631 and other next-generation gene therapies for CAH. That leaves the company’s two approved products— Nulibry for molybdenum cofactor deficiency type A and the targeted cancer treatment Truseltiq (infigratinib), both approved in 2021—and four assets in late-stage development. First and foremost, BridgeBio is gearing up for a Nov. 29 FDA action date on its transthyretin amyloid cardiomyopathy (ATTR-CM) hopeful, acoramidis. At the same time, the company is continuing Phase III development on three more drug candidates —infigratinib for achondroplasia, BBP-418 for limb-girdle muscular dystrophy type 21/R9 and encaleret for autosomal dominant hypocalcemia type 1. Kostas Biliouris, director of biotech equity research at BMO Capital Markets, noted that the primary interest in BridgeBio is in acoramidis and infigratinib. “The other spinout companies are probably increasing efficiency” for these late-stage products because they effectively remove from BridgeBio’s portfolio “something that investors are not focused on,” he told BioSpace . The Path to Success If approved in November, acoramidis will be going up in the ATTR-CM market against Pfizer’s tafamidis, sold as Vyndaqel and Vyndamax, which Biliouris said is well-established in the market. Like tafamidis, acoramidis is a transthyretin stabilizer. Biliouris said that while acoramidis may be “incrementally better” than tafamidis, it needs to be dosed twice per day, compared to once per day for Pfizer’s drugs, which may limit uptake. In addition, Biliouris said, Pfizer has demonstrated that tafamidis can reduce the risk of mortality, whereas acoramidis did not show a statistically significant effect on this measure. In a survey of 30 physicians conducted by BMO Capital Markets, 50% of doctors said the efficacy is similar between acoramidis and tafamidis, while 40% believed acoramidis to be incrementally better, Biliouris shared. “That’s why it’s not an easy story.” If BridgeBio does secure FDA approval for acoramidis, the company will then need to beat sales expectations in 2025, Biliouris said, estimating these to be about $165 million in the U.S. “If BridgeBio can beat that number, it will be great,” he told BioSpace . “It will help the stock and the story. If not, it will be challenging.” Some of the drug’s success could also be tied to whether or not tafamidis goes generic in 2028 or 2035, Biliouris noted, adding that this won’t be known until 2028. Another piece of the puzzle, according to Biliouris, is infigratinib, which if approved, will go up against BioMarin’s Voxzogo in the achondroplasia space. BridgeBio is anticipating a readout for the drug in late 2025, according to Biliouris. This trial needs to meet its primary endpoint and show a good safety profile, he said. “Usually, this drug treats kids and safety is very sensitive here.” It also “needs to be at least as good, if not better, than its main competitor,” Voxzogo, Biliouris continued, and like acoramidis, will also need to beat sales expectations. Kumar, for one, is confident in BridgeBio’s future success, noting that its ultimate goal is “to serve as many patients as possible across as many indications that effectively allow us to do high probability drug discovery development. What some companies would do [in BridgeBio’s position] is they would push pause,” he said. “But I don’t believe in pushing pause.”