Bone Diseases, Developmental

2024 Harrington Prize for Innovation in Medicine Recognizes Pioneering Work in Immunology CLEVELAND, March 19, 2024 /PRNewswire/ -- The eleventh annual Harrington Prize for Innovation in Medicine has been awarded to Arlene H. Sharpe, MD, PhD, Kolokotrones University Professor and Chair of the Department of Immunology at Harvard Medical School. The award recognizes her breakthrough discoveries in immune regulation, which have established foundational principles in immunology and led to new cancer therapies that act by boosting the immune response to cancer. The Harrington Prize for Innovation in Medicine, established in 2014 by the Harrington Discovery Institute at University Hospitals and the American Society for Clinical Investigation (ASCI), honors physician-scientists who have moved science forward with achievements notable for innovation, creativity and potential for clinical application. Dr. Sharpe's research in immunology has led to significant advances in medical treatments. Her work helped identify key pathways that restrain the activity of T lymphocytes (immune cells) to fight cancer. Her work defined the PD-1 pathway and its immunoinhibitory functions as well as the inhibitory functions of CTLA-4 to restrain the activity of the immune system. Dr. Sharpe's research thus laid the foundation for the development of 'immune checkpoint inhibitors', drugs that target PD-1 and related pathways. These therapies, including pembrolizumab and nivolumab, have been approved by the FDA for treating numerous types of cancer. They work by preventing inhibition of T cell function, thereby unleashing the body's immune system to fight cancer cells more effectively. This approach has become a cornerstone in cancer treatment strategies. "Dr. Sharpe's body of work has shaped our understanding of the role of the T cell in regulating the immune response. Her innovative scientific discoveries are among the best examples of bench-to-bedside work in the past three decades," said Benjamin D. Humphreys, MD, PhD, Joseph Friedman Professor of Renal Diseases in Medicine and Chief, Division of Nephrology, Washington University in St. Louis and 2023-2024 President of the ASCI. "Immune checkpoint inhibitors are drugs that allow for dramatic remission, and even cures, of cancers that had previously been considered untreatable. Dr. Sharpe's foundational insights have thus been harnessed to transform cancer therapy. Her work provides a beautiful example of physician impact through creative science," said Jonathan S. Stamler, MD, President, Harrington Discovery Institute, Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine and of Biochemistry at University Hospitals and Case Western Reserve University. A committee composed of members of the ASCI Council and the Harrington Discovery Institute Scientific Advisory Board reviewed nominations from leading academic medical centers globally before selecting the 2024 Harrington Prize recipient. In addition to sharing the Prize's $20,000 honorarium, Dr. Sharpe will deliver the Harrington Prize Lecture at the 2024 AAP/ASCI/APSA Joint Meeting on April 5, will be a featured speaker at the 2024 Harrington Scientific Symposium May 22-23, and will publish an essay in the Journal of Clinical Investigation. The Harrington Prize has recognized outstanding and diverse innovations in medicine: 2014: Harry Dietz, MD, Johns Hopkins University, for his contributions to the understanding of the biology and treatment of Marfan syndrome, a disorder leading to deadly aneurysms in children and adults. 2015: Douglas R. Lowy, MD, The National Cancer Institute, in recognition of his discoveries that led to the development of the Human Papillomavirus vaccine to prevent cervical cancer. 2016: Jeffrey M. Friedman, MD, PhD, The Rockefeller University, for his discovery of leptin, which controls feeding behavior and is used to treat related clinical disorders. 2017: Jointly awarded to Daniel J. Drucker, MD, Mount Sinai Hospital, Canada, Joel F. Habener, MD, Massachusetts General Hospital, and Jens J. Holst, MD, DMSc, University of Copenhagen, Denmark, for their discovery of incretin hormones and for the translation of these findings into transformative therapies for major metabolic diseases such as diabetes. 2018: Helen H. Hobbs, MD, UT Southwestern Medical Center, for the discovery of the link between a gene mutation (PCSK9) and lower levels of LDL, which has improved the treatment of high cholesterol. 2019: Carl H. June, MD, University of Pennsylvania, for advancing the clinical application of CAR T therapy for cancer treatment, and for his sustained contributions to the field of cellular immunology. 2020: Stuart H. Orkin, MD, Harvard University, for breakthrough discoveries on red blood cells that offer new treatments for patients with sickle cell disease and beta-thalassemia, which are among the most common genetic disorders. 2021: Warren J. Leonard, MD, and John J. O'Shea, MD, NIH, for their respective contributions to the field of immunology, from fundamental discovery to therapeutic impact. 2022: James E. Crowe Jr., MD, and Michel C. Nussenzweig, MD, PhD, for their groundbreaking work, which has elucidated fundamental principles of the human immune response and enabled the use of human antibodies to treat COVID-19. 2023: Jean Bennett, MD, PhD, and Albert M. Maguire, MD, for their groundbreaking translational research to restore sight in inherited genetic diseases. SOURCE Harrington Discovery Institute

Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement. In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

Poster presentations to highlight new quantitative and serologic diagnostic methods for Celiac Disease detection and determination of intestinal mucosal health New tools may serve as companion diagnostics for identifying seroactive patients and for monitoring the efficacy of Latiglutenase, the Company’s Celiac Disease therapeutic program BOCA RATON, Fla., March 18, 2024 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc., (NASDAQ: FWBI), (“First Wave BioPharma” or the “Company”), a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that two abstracts have been accepted for presentation at the 2024 Digestive Disease Week Conference (DDW 2024) taking place May 18-21, 2024, in Washington, D.C. The first presentation, titled, “A Composite Mucosal Scale for Celiac Disease Encompassing both Morphology and Inflammation,” will highlight VCIEL, a new quantitative composite scale, combining the histologic scales of villus height to crypt depth ratio (Vh:Cd) and density of intraepithelial lymphocytes (IEL), designed to enhance the accuracy and precision for measuring small intestinal mucosal health of celiac disease (CeD) patients with particular benefit for histologic endpoint measurements in clinical trials. The second presentation, titled, “Dynamics of Serologic Change to Gluten in Celiac Disease Patients,” will report research involving the largest database of CeD clinical results and will explore key issues related to the diagnostics of CeD, such as the effects of resuming a gluten-containing diet on seropositivity and the correlation between two different tTG-IgA assays. “The abstracts accepted for presentation at DDW 2024 showcase significant advancements in the field of gastroenterology, particularly in the precision diagnosis and management of celiac disease through the innovative VCIEL scale that our team has developed,” stated James Sapirstein, Chairman and CEO of First Wave BioPharma. “The invitation to present our findings on the VCIEL scale for measuring mucosal health of the small intestine as well as our research on the impact of gluten reintroduction as a diagnostic intervention for serologic measurements at one of the most influential meetings on gastrointestinal disease validates the potential for our work to advance better understanding and treatment of celiac disease,” said Jack Syage, PhD, President and Chief Operating Officer of First Wave. Details on the presentations are as follows: Presentation Title: A Composite Mucosal Scale for Celiac Disease Encompassing both Morphology and Inflammation Presenters: Jack Syage, PhD. President & Chief Operating Officer, First Wave BioPharma Jennifer Sealy-Voyksner, PhD., Vice President R&D, First Wave BioPharma Session Title: Celiac Disease and Gluten Related Disorders Date and Time: May 19, 2024, from 12:30 PM to 1:30 PM EDT Presentation Title: Dynamics of Serologic Change to Gluten in Celiac Disease Patients Presenters: Jack Syage, PhD. President & Chief Operating Officer, First Wave BioPharma Jennifer Sealy-Voyksner, PhD., Vice President R&D, First Wave BioPharma Session Title: Celiac Disease and Gluten Related Disorders Date and Time: May 19, 2024, from 12:30 PM to 1:30 PM EDT About Celiac Disease Celiac disease is a chronic, hereditary autoimmune and inflammatory disease triggered by gluten consumption. Celiac disease is characterized by damage to the lining of the small intestine, causing malabsorption, gastrointestinal dysfunction and debilitating symptoms. Over the course of a lifetime, untreated or poorly managed celiac disease is often associated with deteriorating general health, multiple serious intestinal and extra-intestinal medical complications, and increased morbidity and mortality. Celiac disease is a global disease and affects approximately 1% of the population worldwide and is increasing in prevalence with improved diagnostic tools and improved awareness. About First Wave BioPharma, Inc. First Wave BioPharma is a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The Company is currently advancing a therapeutic development pipeline with multiple late-stage clinical programs built around three proprietary technologies – latiglutenase, a Phase 3-ready, potentially first-in-class, targeted, oral biotherapeutic for celiac disease; capeserod, a selective 5-HT4 receptor partial agonist being developed for gastroparesis; and adrulipase, a recombinant lipase enzyme designed to enable the digestion of fats and other nutrients in cystic fibrosis and chronic pancreatitis patients with exocrine pancreatic insufficiency. First Wave BioPharma is headquartered in Boca Raton, Florida. For more information visit . Forward-Looking Statements This press release may contain certain statements relating to future results which are forward-looking statements. It is possible that the Company’s actual results and financial condition may differ, possibly materially, from the anticipated results and financial condition indicated in these forward-looking statements, depending on factors including whether any financing or licensing transaction may be completed, completed with different terms, in an untimely manner, or not at all; whether the Company will be able to realize the expected benefits of its acquisition of ImmunogenX; the Company’s ability to integrate the assets and contemplated commercial operations acquired from ImmunogenX into the Company’s business; whether results obtained in preclinical and nonclinical studies and clinical trials will be indicative of results obtained in future clinical trials; whether preliminary or interim results from a clinical trial will be indicative of the final results of the trial; whether the Company will be able to maintain compliance with Nasdaq’s continued listing criteria and the effect of a delisting from Nasdaq on the market for the Company’s securities; the size of the potential markets for the Company’s drug candidates and its ability to service those markets; the effects of the First Wave Bio, Inc. acquisition, the related settlement and their effect on the Company’s business, operating results and financial prospects; and the Company’s current and future capital requirements and its ability to raise additional funds to satisfy its capital needs. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, under the heading “Risk Factors,” as well as the Company’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements included in this press release are made only as of the date of this press release, and we do not undertake any obligation to publicly update or correct any forward-looking statements to reflect events or circumstances that subsequently occur or of which we hereafter become aware. For more information: First Wave BioPharma, Inc. 777 Yamato Road, Suite 502 Boca Raton, FL 33431 Phone: (561) 589-7020 info@firstwavebio.com Media contact: Tiberend Strategic Advisors, Inc. David Schemelia (609) 468-9325 dschemelia@tiberend.com