Stanford University School of Medicine

Experts and advocates to provide important strategic input and clinical expertise as ACELYRIN completes its Phase 2 trial of lonigutamab in TED and prepares to initiate Phase 3 program in first quarter of 2025 LOS ANGELES, Oct. 16, 2024 (GLOBE NEWSWIRE) -- ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma company focused on accelerating the development and delivery of transformative medicines in immunology, today announced the formation of a scientific and patient advisory board comprised of leading experts and advocates in thyroid eye disease (TED). This group of advisors will provide important strategic input, clinical expertise and patient perspectives as ACELYRIN prepares to advance lonigutamab, a subcutaneously delivered IgG1 monoclonal antibody targeting the anti-insulin-like growth factor 1 (IGF-1) receptor, into Phase 3 clinical development for TED in the first quarter of 2025. “Convening this esteemed group of clinical thought leaders and patient advocates is an important step forward for us as we prepare to advance lonigutamab into the pivotal Phase 3 program for TED,” said Shephard (Shep) Mpofu, M.D., Chief Medical Officer of ACELYRIN. “Each member brings distinct expertise spanning multiple facets of TED including disease pathology, clinical trial leadership and patient experience. We believe their collective insights will position us extremely well to execute on our goal of delivering the potentially best-in-class subcutaneous anti-IGF-1R treatment for TED patients.” Inaugural members of the ACELYRIN scientific and patient advisory board are listed below; full bios are available on the ACELYRIN website. Scientific Advisors George J. Kahaly, MD, PhD, Professor of Medicine and Endocrinology Metabolism and Chief Physician of the Endocrine Outpatient Clinic at the Johannes Gutenberg University Medical Center, Mainz, Germany;Andrea Lora Kossler, MD, FACS, Associate Professor of Ophthalmology at Stanford University School of Medicine and Director of the Oculofacial Plastic, Reconstructive Surgery and Orbital Oncology Service at the Byers Eye Institute at Stanford;Prem Subramanian, MD, PhD, Professor of Ophthalmology, Neurology, and Neurosurgery, University of Colorado School of Medicine; andShoaib Ugradar, MD, Department of Orbital and Oculoplastic Surgery, private practice, Beverly Hills, California. Patient Advisor Christine Gustafson, Executive Director and CEO of the TED Community Organization, the only global 501c3 nonprofit dedicated to Thyroid Eye Disease. About Thyroid Eye DiseaseThyroid Eye Disease (TED) is a vision-threatening autoimmune disease in which there is both inflammation and expansion of the tissues behind the eye, resulting in eye bulging, known as proptosis, and the subsequent inability to close the eyelids. Double vision, or diplopia, can occur, as well as the potential for compression of the optic nerve, which can lead to blindness. Thus, TED is a progressive, chronic inflammatory disease. More than 100,000 people in the United States are estimated to suffer from TED. About LonigutamabLonigutamab is a humanized IgG1 monoclonal antibody targeting the anti-insulin-like growth factor 1 (IGF-1) receptor and is delivered subcutaneously. Relative to standard of care, lonigutamab binds to a distinct epitope, which results in internalization of the receptor within minutes, and in preclinical binding and functional laboratory assays, it has been shown to be 75-fold more potent. The characteristics of lonigutamab that enable subcutaneous delivery also enable the potential for longer-term, convenient dosing, which can potentially improve depth and durability of clinical response. About ACELYRINACELYRIN, INC. (Nasdaq: SLRN) is focused on providing patients life-changing new treatment options by identifying, acquiring, and accelerating the development and commercialization of transformative medicines. ACELYRIN’s lead program, lonigutamab, is a subcutaneously delivered monoclonal antibody targeting IGF-1R being investigated for the treatment of thyroid eye disease. For more information about ACELYRIN, visit us at www.acelyrin.com or follow us on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements, including, but not limited to, statements regarding ACELYRIN’s ability to address unmet needs of patients, business plans and potential future benefits of our pipeline. These forward-looking statements are based on ACELYRIN’s current plans, objectives and projections, and are inherently subject to risks and uncertainties that may cause ACELYRIN’s actual results to materially differ from those anticipated in such forward-looking statements. Such risks and uncertainties include, without limitation, those associated with the successful completion of development and regulatory activities with respect to ACELYRIN’s product candidates, the risk that future results could differ materially and adversely from early clinical data, and other risks and uncertainties affecting ACELYRIN including those described from time to time under the caption “Risk Factors” and elsewhere in ACELYRIN’s current and future reports filed with the Securities and Exchange Commission. Forward-looking statements contained in this press release are made as of this date, and ACELYRIN undertakes no duty to update such information except as required under applicable law.

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

MONDAY, Sept. 30, 2024 -- In a clinical practice guideline issued by the American Academy of Pediatrics and published online Sept. 30 in Pediatrics , recommendations are presented for opioid prescribing for acute pain management in children and adolescents in outpatient settings. Scott E. Hadland, M.D., M.P.H., from Harvard Medical School in Boston, and colleagues recommend that acute pain should be treated using a multimodal approach, including appropriate use of nonpharmacologic therapies, nonopioid medication, and opioid medication, when needed. For children and adolescents with acute pain, opioids should not be prescribed as monotherapy. Clinicians should prescribe immediate-release opioid formulations, start with the lowest age- and weight-appropriate doses, and provide an initial supply of five or fewer days when using opioids for acute pain management, unless the pain is related to trauma or surgery with an expected duration of more than five days. Codeine or tramadol should not be prescribed for children younger than 12 years; for adolescents age 12 to 18 years with obesity, obstructive sleep apnea, or severe lung disease; for treating postsurgical pain after tonsillectomy or adenoidectomy in patients younger than 18 years; or for any breastfeeding patient. Caution should be exercised when prescribing opioids for acute pain in children or adolescents who are taking sedating medications. Naloxone should be provided when opioids are prescribed, and patients and families should be counseled on the signs of opioid overdose and how to respond. "For a patient with mild-to-moderate pain, doctors should always start nonopioid medications and treatment," coauthor Rita Agarwal, M.D., from the Stanford University School of Medicine in California, said in a statement. Two authors disclosed ties to the pharmaceutical industry. Clinical Practice Guideline Technical Report Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.