Fairbank Disease

Once issued, the patent is expected to extend protection of RHB-204 to 2041 RHB-204 is currently undergoing a Phase 3 study in the U.S. as the first stand-alone standard of care first-line drug candidate for Non-tuberculosis Mycobacteria (NTM) disease caused by MAC infection RHB-204 has received U.S. FDA Fast Track, Orphan and QIDP priority designations as well as EMA Orphan Drug designation, providing eligibility for 12 years market exclusivity in the U.S. and 10 years exclusivity in the EU The Company is advancing discussions with several prospective partners for RHB-204 across multiple territories TEL AVIV, Israel and RALEIGH, N.C., Jan. 26, 2023 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced that the U.S. Patent and Trademark Office (USPTO) has issued a Notice of Allowance for the granting of a patent covering RHB-204's1 oral fixed-dose combination, methods for treating pulmonary Mycobacterium avium Complex (MAC) disease, and kits comprising a supply of fixed-dose combination products for treating pulmonary MAC disease. Once issued, the patent is expected to protect RHB-204 through 2041. "Non-tuberculosis mycobacterial disease (NTM) is particularly threatening for vulnerable people with underlying lung disease or weakened immune systems, and the incidence is increasing worldwide, with up to 180,000 people in the U.S. thought to be living with NTM disease2. It is notoriously difficult to treat, requiring multiple antibiotics to be taken over a lengthy period, and with no approved first-line stand-alone therapies in the U.S., EU, and Japan, there is an urgent need for new treatment innovation," said Danielle T. Abramson, Ph.D., RedHill's SVP Global Head of Intellectual Property. "This USPTO Notice of Allowance for RHB-204 recognizes the innovation behind RHB-204's specific fixed-dose combination therapy for the treatment of NTM disease and is expected to extend protection for this innovation to 2041." A U.S. Phase 3 study is ongoing to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by MAC infection (NCT04616924). The study protocol provides for 6 months co-primary endpoint of sputum culture conversion (SCC) and clinical outcome (patient-reported outcomes - PRO) in a randomized placebo-controlled design, followed by open label active treatment with RHB-204 for 12 months from conversion. The Company is advancing discussions with several prospective partners for RHB-204 across multiple territories. About Pulmonary Nontuberculous Mycobacteria (NTM) Infections Pulmonary nontuberculous mycobacteria (NTM) disease is a chronic, serious, and debilitating lung disease that is becoming a significant public health concern with prevalence rising approximately 8% per year worldwide2. It is estimated that up to 180,000 people in the U.S. may be living with NTM disease, with women, the elderly, and those with underlying lung conditions or weakened immune systems at particular risk. It is caused by ubiquitous environmental bacteria, found in soil as well as natural and engineered water systems. The most common symptoms of NTM disease include loss of lung function, persistent cough, fever, weight loss, chest pain, and blood in sputum3, contributing to a reduction in quality of life. NTM infections can lead to recurring cases of bronchitis and pneumonia and can, in some cases, lead to respiratory failure4. Although rare, the incidence and prevalence of pulmonary NTM disease are increasing in many areas of the world5. There were an estimated 110,000 pulmonary NTM disease patients in the U.S. in 20176 and an estimated 28,000 in the EU. Pulmonary manifestations account for 80-90% of all NTM-associated diseases7, and approximately 80% of pulmonary NTM infections are caused by Mycobacterium avium complex (MAC)8. Treatment of NTM infection can be difficult with guidelines recommending that MAC infection be treated with a multi-drug regimen for treatment, with at least three antimicrobials, for at least 12 months9. There is no FDA- or EMA-approved first-line standard-of-care combination therapy. Many patients fail current therapy, and more than half will have either recurring disease or a new infection after completing treatment10. Thus, new treatment options for NTM are needed. About RHB-204 RHB-204 is a Phase 3-stage proprietary, fixed-dose oral capsule containing a combination of clarithromycin, rifabutin and clofazimine, developed for the treatment of pulmonary NTM disease caused by MAC. RHB-204 was granted FDA Fast Track and Orphan Drug Designation, in addition to QIDP Designation under the Generating Antibiotic Incentives Now Act (GAIN Act), extending U.S. post-approval U.S. market exclusivity to a potential total of 12 years. RHB-204 has additionally been granted EU Orphan Designation, providing eligibility for 10 years EU post-approval market exclusivity. RedHill is seeking protection of RHB-204, and its use in treating pulmonary MAC disease, on a global scale, providing protection until 2041. About RedHill Biopharma RedHill Biopharma Ltd. (Nasdaq: RDHL) is a specialty biopharmaceutical company primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik® for opioid-induced constipation in adults11, Talicia® for the treatment of Helicobacter pylori (H. pylori) infection in adults12, and Aemcolo® for the treatment of travelers' diarrhea in adults13. RedHill's key clinical late-stage development programs include: (i) RHB-204, with an ongoing Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) disease; (ii) opaganib (ABC294640), a first -in -class oral broad-acting, host-directed SK2 selective inhibitor targeting multiple indications, with potential for pandemic preparedness, with a Phase 2/3 program for hospitalized COVID-19, a Phase 2 program in oncology and a radiation protection program ongoing; (iii) RHB-107 ( upamostat), an oral broad-acting, host-directed, serine protease inhibitor with potential for pandemic preparedness and is in a Phase 3-stage study as treatment for non-hospitalized symptomatic COVID-19, and targeting multiple other cancer and inflammatory gastrointestinal diseases; (iv) RHB-104, with positive results from a first Phase 3 study for Crohn's disease; and (v) RHB-102 , with positive results from a Phase 3 study for acute gastroenteritis and gastritis and positive results from a Phase 2 study for IBS-D. More information about the Company is available at twitter.com/RedHillBio. This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words "intends," "may," "will," "plans," "expects," "anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes," "potential" or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company's control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, the risk that the Company will not initiate the Phase 3 clinical study in all or part of the sites in the U.S. or will be delayed; the risk that the U.S. Phase 3 clinical study evaluating RHB-204 will not be successful or, if successful, will not suffice for regulatory marketing approval without the need for additional clinical and/or other studies; as well as risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company's research, manufacturing, pre-clinical studies, clinical trials, and other therapeutic candidate development efforts, and the timing of the commercial launch of its commercial products and ones it may acquire or develop in the future; (ii) the Company's ability to advance its therapeutic candidates into clinical trials or to successfully complete its pre-clinical studies or clinical trials or the development of a commercial companion diagnostic for the detection of MAP; (iii) the extent and number and type of additional studies that the Company may be required to conduct and the Company's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company's therapeutic candidates and Talicia®; (v) the Company's ability to successfully commercialize and promote Talicia®, Movantik® and Aemcolo®; (vi) the Company's ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company's therapeutic candidates and the results obtained with its therapeutic candidates in research, pre-clinical studies or clinical trials; (ix) the implementation of the Company's business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xii) estimates of the Company's expenses, future revenues, capital requirements and needs for additional financing; (xiii) the effect of patients suffering adverse experiences using investigative drugs under the Company's Expanded Access Program; and (xiv) competition from other companies and technologies within the Company's industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on March 17, 2022. All forward-looking statements included in this press release are made only as of the date of this press release. The Company assumes no obligation to update any written or oral forward-looking statement, whether as a result of new information, future events or otherwise unless required by law. Category: R&D 1 RHB-204 is an investigational new drug, not available for commercial distribution. 2 Strollo SE, et al. The Burden of Pulmonary Nontuberculous Mycobacterial Disease in the United States. Ann Am Thorac Soc. 2015 Oct;12(10):1458-64. 3 Kim RD, et al. Pulmonary Nontuberculous Mycobacterial Disease. Prospective Study of a Distinct Preexisting Syndrome Am J Respir Crit Care Med. 2008; 178(10):1066–74. 4 The American Lung Association, 2020. 5 Winthrop KL, et al. Incidence and Prevalence of Nontuberculous Mycobacterial Lung Disease in a Large U.S. Managed Care Health Plan, 2008-2015. Ann Am Thorac Soc. 2020 Feb;17(2):178-185. 6 Foster|Rosenblatt, 2017 7 Griffith DE, et al. An official ATS/IDSA statement: diagnosis, treatment, and prevention of nontuberculous mycobacterial diseases Am J Respir Crit Care Med. 2007;175(4):367-416. 8 Prevots DR et al. Nontuberculous mycobacterial lung disease prevalence at four integrated health care delivery systems. Am J Respir Crit Care Med 2010; 182:970-76; Winthrop KL, et al. Pulmonary nontuberculous mycobacterial disease prevalence and clinical features: an emerging public health disease. Am J Respir Crit Care Med 2010; 182: 977-82 9 Daley CL, et al. Treatment of Nontuberculous Mycobacterial Pulmonary Disease: An Official ATS/ERS/ESCMID/IDSA Clinical Practice Guideline: Executive Summary. Clinical Infectious Diseases. Ciaa241, . 10 Henkle E, et al. Patient-Centered Research Priorities for Pulmonary Nontuberculous Mycobacteria (NTM) Infection. An NTM Research Consortium Workshop Report Annals of the American Thoracic Society 2016; S379-84. 11 Movantik® (naloxegol) is indicated for opioid-induced constipation (OIC). Full prescribing information see: 12 Talicia® (omeprazole magnesium, amoxicillin and rifabutin) is indicated for the treatment of H. pylori infection in adults. For full prescribing information see: . 13 Aemcolo® (rifamycin) is indicated for the treatment of travelers' diarrhea caused by noninvasive strains of Escherichia coli in adults. For full prescribing information see: . Logo - SOURCE RedHill Biopharma Ltd.

Session to have special translational research emphasis on PRPH2 and inherited macular dystrophies. COLUMBIA, Md., Jan. 25, 2023 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, in partnership with the Nixon Visions Foundation and the Shiley Eye Institute, at the University of California, San Diego, will host the free in-person PRPH2 and Associated Retinal Diseases Workshop on March 29–31, 2023, at the Hilton La Jolla Torrey Pines in San Diego, CA. Registration information can be found at . The PRPH2 and Associated Retinal Diseases Workshop is designed to bring together leading experts from academia and industry, to discuss openly, and in detail, what is known about PRPH2 disease pathology, disease models, clinical characteristics, and therapeutic approaches. In addition, affected individuals and their families will attend to share their experiences with PRPH2 disease and to hear the latest developments for care, treatment, and strategies for therapies. The workshop will also serve as a launching point for the Nixon Visions Foundation sponsored funding initiative that will provide multiple annual grants to scientific teams addressing high-impact gaps in the field. Claire M. Gelfman, PhD, chief scientific officer, Foundation Fighting Blindness, along with Radha Ayyagari, PhD, Shiley Eye Institute, University of California, San Diego (UCSD), and Shyamanga Borooah, MD, PhD, Shiley Eye Institute, University of California, San Diego (UCSD), will serve as workshop co-chairs. In addition, representatives from academia and industry who will address the research landscape and emerging therapies include: Steve Daiger, PhD – Director, Laboratory for Molecular Diagnosis of Inherited Eye Diseases, Professor, Human Genetics Center, School of Public Health and Ruiz Department of Ophthalmology and Visual Science, UTHealth Muna Naash, PhD – John S Dunn Endowed Professor of Biomedical Engineering, University of Houston Vadim Arshavsky, PhD – Helena Rubinstein Foundation Distinguished Professor of Ophthalmology, Duke University Francois Paquet-Durand, PhD – Professor, Institute for Ophthalmic Research, Tubingen University Peter Quinn, PhD – Principal Investigator and Associate Research Scientist, Columbia University Fred K. Chen, MBBS, PhD – Associate Professor, Lions Eye Institute, Australia Rachel Heath Jeffery, MChD, MPH – Resident, Lions Eye Institute, Australia Deepak Lamba, MBBS, PhD – Associate Professor of Ophthalmology, University of California, San Francisco Shannon Conley, PhD – Assistant Professor of Cell Biology, The University of Oklahoma Health Sciences University Kari Branham, MS, CGC – Assistant Professor, Ophthalmology and Visual Sciences, Genetic Counselor, Inherited Retinal Dystrophy Clinic, Kellogg Eye Center, University of Michigan Rob Collin, PhD – Principal Investigator, Department of Human Genetics, Radboud University Medical Center David Birch, PhD – Scientific Director, Retina Foundation of the Southwest Daniel Chung, DO, MA – Chief Medical Officer, Sparing Vision Eric Daniels MD, MBA – Chief Development Officer, Kiora Pharmaceuticals Ash Jayagopal, PhD – Chief Science Officer, Opus Genetics "We are privileged to be partnering with the Nixon Visions Foundation and the Shiley Eye Institute, University of California, San Diego, to bring awareness, build a community across PRPH2-related retinal disease and accelerate related research on biological and therapeutic approaches," said Claire M. Gelfman, chief scientific officer of the Foundation Fighting Blindness. "Our hope is that through this workshop we will facilitate knowledge sharing to illuminate the current landscape and to identify key roadblocks impeding translation to therapies." To register for the PRPH2 and Associated Retinal Diseases Workshop and get the latest updates on the event, visit . About the Foundation Fighting Blindness Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. About the Nixon Visions Foundation Nixon Visions Foundation is dedicated to the support of organizations and individuals who are striving to reach their potential. Based in San Diego, the foundation provides scholarships and other funding for education, training, social welfare, public information, and research. Its newest initiative is to provide meaningful support for scientific research leading to critical treatments and, ultimately, cures for rare inherited retinal degenerative diseases through partnerships with the Foundation Fighting Blindness and UC San Diego Health's Shiley Eye Institute, each targeting rare gene mutations such as those of the PRPH2 gene. Visit . About the Shiley Eye Institute The Shiley Eye Institute is the only academic institution in the San Diego area with comprehensive programs for the clinical care of patients with eye disorders, cutting edge research on surgical techniques and treatments of eye diseases, education in the field of ophthalmology and innovative outreach to the community. To learn more about the Shiley Eye Institute, visit . Media Contact: Chris Adams Vice President, Marketing & Communications [email protected] 410-423-0585 SOURCE Foundation Fighting Blindness

A variety of healthy eating patterns are linked to reduced risk of premature death, according to a new study. They found that participants who scored high on adherence to at least one of four healthy eating patterns were less likely to die during the study period from any cause and less likely to die from cardiovascular disease, cancer, or respiratory disease, compared with people with lower scores. A variety of healthy eating patterns are linked to reduced risk of premature death, according to a new study led by Harvard T.H. Chan School of Public Health researchers. They found that participants who scored high on adherence to at least one of four healthy eating patterns were less likely to die during the study period from any cause and less likely to die from cardiovascular disease, cancer, or respiratory disease, compared with people with lower scores. The findings are consistent with the current Dietary Guidelines for America, which recommend multiple healthy eating patterns. "The Dietary Guidelines for Americans are intended to provide science-based dietary advice that promotes good health and reduces major chronic diseases. Thus, it is critical to examine the associations between DGAs-recommended dietary patterns and long-term health outcomes, especially mortality," said corresponding author Frank Hu, Fredrick J. Stare Professor of Nutrition and Epidemiology and chair of the Department of Nutrition. The study will be published online January 9, 2023, in JAMA Internal Medicine. Few studies have evaluated whether greater adherence to the DGAs-recommended dietary patterns is associated with long-term risk of total and cause-specific mortality. The researchers used health data collected over 36 years from 75,230 women participating in the Nurses' Health Study and 44,085 men in the Health Professionals Follow-up Study. All participants were free of cardiovascular disease or cancer at the beginning of the study and completed dietary questionnaires every four years. Their information was scored based on each of the four dietary pattern indexes (Healthy Eating Index 2015, Alternate Mediterranean Diet, Healthful Plant-based Diet Index, and Alternate Healthy Eating Index). All share key components including whole grains, fruits, vegetables, nuts, and legumes, although other components differ across different eating patterns. A higher score on at least one of the indexes was associated with lower risk of premature death from all causes, and from cardiovascular disease, cancer, and respiratory disease. Higher scores on the AMED and the AHEI were associated with lower risk of death from neurogenerative disease. The results were consistent for non-Hispanic white, non-Hispanic Black, and Hispanic people. The current DGAs (2015-2020) recommend multiple healthy eating patterns that can be adapted toindividual food traditions and preferences. An updated version of the Guidelines is released every five years by the U.S. Departments of Health and Human Services (HHS) and Agriculture (USDA). "It is important to evaluate adherence to DGAs-recommended eating patterns and health outcomes, including mortality, so that timely updates can be made," said Hu. "Our findings will be valuable for the 2025-2030 Dietary Guidelines Advisory Committee, which is being formed to evaluate current evidence surrounding different eating patterns and health outcomes." Other Harvard Chan School co-authors of the study included Zhilei Shan, Fenglei Wang, Yanping Li, Megu Baden, Shilpa Bhupathiraju, Dong Wang, Qi Sun, Eric Rimm, Lu Qi, Fred Tabung, Edward Giovannucci, Walter Willett, JoAnn Manson, and Qibin Qi. Funding for the study came from grant R01HL060712 from the NHLBI. The Nurses' Health Studies and Health Professional Follow-up Studies are supported by the grants UM1 CA186107, P01 CA87969, R01 CA49449, R01 HL034594, R01 HL088521, U01 CA176726, R01 CA67262, U01 CA167552, R01 HL35464, and U01 H145386 from the NIH. Q. Qi is supported by grants K01HL129892 and R01 HL140976 from the NHLBI and grant R01 DK119268 and R01 DK120870 from the National Institute of Diabetes and Digestive and Kidney Diseases. F. Wang is supported by postdoctoral fellowship grant 897161 from the American Heart Association.