AQP1

PRINCETON, N.J., Oct. 25, 2024 /PRNewswire/ -- RiboX Therapeutics Ltd. (RiboX), a pioneering biopharmaceutical company, focused on discovering and developing fully engineered circular RNA therapeutics, announced that the US Food and Drug Administration (FDA) has cleared its IND application for the Phase I/IIa Study of RXRG001 (SPRINX-1 Study) on October 25th, 2024. RXRG001 is the first-ever circular RNA therapy to receive FDA IND clearance. This important advancement marks a significant milestone as circular RNA drugs advance into the clinical development phase. The first-in-human SPRINX-1 study is designed to evaluate the safety and efficacy of RXRG001 in patients with radiation-induced-xerostomia (RIX) (dry mouth) and hyposalivation (low saliva secretion). Xerostomia and hyposalivation are common side effects of radiation therapy in head and neck cancer (HNC) patients. The incidence of HNC is increasing and has become the sixth most common malignant tumor worldwide. Radiation therapy, a main treatment for HNC, often results in damage to salivary glands and a significant decrease in saliva secretion, frequently leading to dry mouth, difficulties in chewing and swallowing, impaired oral health, and diminished quality of life. It has been two decades since the last xerostomia treatment was approved by the FDA. Patients reported that current treatments are ineffective, temporary, and costly. There is a significant unmet medical need to develop a new treatment for xerostomia and hyposalivation. RXRG001 is a proprietary product developed using innovative circular RNA technology. In recent years, circular RNA has emerged as the next generation mRNA therapy due to its high protein expression efficiency, low immunogenicity, and manufacture stability. RXRG001 consists of the circular RNA coding human aquaporin 1 (hAQP1, a water channel protein of cell membrane), encapsulated in lipid nanoparticles (LNPs). RXRG001 increases saliva production by restoring water permeability via overexpression of hAQP1, therefore it alleviates dry mouth symptoms. Non-clinical studies demonstrated a favorable risk and benefit profile of RXRG001 in animal models. For instance, a single administration of RXRG001 led to a significant increased salivary flow which was sustained for about four weeks. "RIX is a devastating life-long health issue for patients with HNC. Although radiation therapy improves patients' survival, its damage to the salivary glands leads to an impaired quality of life of many cancer survivors," said Dr. Yizhen Xu, Chief Medical Officer of RiboX. "RXRG001 may offer a potential effective and sustained therapeutic option for RIX patients. We are excited to conduct clinical trials to further evaluate its efficacy and safety in patients." "The FDA's clearance of the IND application for RXRG001 is an acknowledgment of our innovative capabilities, robust technology, and competitiveness in the field of circular RNA therapeutics." Dr. Weiyi Zhang, Chief Executive Officer of RiboX, stated "RiboX will advance the clinical development of RXRG001, continue leveraging the unique advantages of circular RNA technology in therapeutic applications, and introduce more pioneering treatments to patients around the world." About RiboX Therapeutics RiboX is a globally operated biotech, dedicated to discovering and developing fully engineered circular RNA therapeutics, cofounded by Drs. Ling-Ling Chen, Dan Peer and Weiyi Zhang in 2021. The company has operations in Princeton, NJ, USA, Rehovot, Israel and Shanghai, China. Circular RNA therapy is a novel modality, which is considered as the next generation RNA therapy. Compared to mRNA, circular RNA has a covalently closed single-stranded RNA structure that confers superior stability, lowers immunogenicity, and tunable protein expression, subsequently overcoming the current limitations of mRNA therapeutics and realizing the full potential of RNA medicines. RiboX has developed proprietary plug-and-play circular RNA drug design and engineering, combined with novel LNPs platforms and robust manufacturing capabilities. These innovative technologies provide support for the clinical development of circular RNA therapeutics. RiboX's pipeline spans various therapeutic areas, including radiation-induced xerostomia, monogenic diseases, autoimmune diseases, and vaccines. RiboX is committed to pioneering innovative research in next-generation RNA therapies, with a vision to improve the quality of life for patients worldwide. For further information, please visit: Media contact: [email protected] SOURCE RiboX Therapeutics Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Company's Receptor Co-Agonists Demonstrate Potent Body Weight Reductions, Decreased Food Intake and Improved Metabolic Pro Healthy Rats and Diet-Induced Obese Mice SAN DIEGO, June 24, 2024 /PRNewswire/ -- Viking Therapeutics Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced the presentation of preclinical data from a series of internally developed dual agonists of the amylin and calcitonin receptors at the 84th Scientific Sessions of the American Diabetes Association. The presentation highlights the effects of treatment on body weight, food intake and metabolic pro healthy rats and diet-induced obese (DIO) mice as compared to control cohorts treated with vehicle or the dual amylin and calcitonin receptor agonist cagrilintide. The studies were summarized in a poster presentation at the annual scientific conference of the American Diabetes Association, being held June 21-24, 2024, in Orlando, Florida. The study results demonstrate that Viking's series of dual amylin and calcitonin receptor agonists (DACRAs) reduced food intake in lean rats in the period from 0 – 72 hours following a single subcutaneous dosing. At 72 hours following a single subcutaneous dose, Viking's novel compounds resulted in up to 8% body weight reductions compared to vehicle-treated animals. In a DIO mouse model, treatment with Viking's series of co-agonists for 24 days resulted in body weight reductions that were comparable to those achieved in cagrilintide-treated animals. Additionally, improvements in key metabolic markers, including blood glucose levels, were observed in DIO mice treated with the company's compounds for the 24-day time period. Highlights from poster 2024-LB-5842: Novel Amylin and Calcitonin Receptor Co-Agonists Reduce Food Intake and Body Weight in Rodents. Viking DACRAs demonstrated EC50 values ranging from low nM to micromolar on the human amylin 3 receptor and a similar range of potencies on the human calcitonin receptor. Treatment with single doses of Viking DACRAs resulted in up to 8% mean reductions in body weight in lean rats after 72 hours. Treatment of DIO mice for 24 days with Viking DACRA compounds demonstrated up to 10% weight loss from baseline (p<0.05 vs. baseline). Viking DACRA compounds demonstrated up to 24% reductions in blood glucose in DIO mice after 24 days (p<0.05 vs. baseline and cagrilintide control). The results of these and other preclinical studies provide the rationale for Viking's continued advancement of its internal dual amylin and calcitonin receptor agonist development program. "The amylin receptor plays an important role in food intake and metabolic control, making it an attractive target for therapeutic intervention in obesity," said Brian Lian, Ph.D., chief executive officer of Viking. "These data demonstrate the potent activity of a series of novel, internally developed, amylin and calcitonin dual agonists and represent an exciting expansion of our pipeline in obesity and metabolic diseases. This program provides Viking with additional opportunities to develop novel, differentiated therapies for obesity with potentially best-in-class pro " About Amylin and Dual Amylin/Calcitonin Agonists Amylin has important effects on glucose management, glucagon production, gastric emptying time and satiety. Amylin analogs have been shown to significantly reduce body weight and dosage of insulin. Dual amylin and calcitonin receptor agonists are the most potent amylin receptor agonists. Cagrilintide is a dual amylin/calcitonin receptor agonist that is currently in clinical development. About Viking Therapeutics, Inc. Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel first-in-class or best-in-class therapies for the treatment of metabolic and endocrine disorders, with three compounds currently in clinical trials. Viking's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking's clinical programs include VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders. The compound successfully achieved both the primary and secondary endpoints in a recently completed Phase 2b study for the treatment of biopsy-confirmed non-alcoholic steatohepatitis (NASH) and fibrosis. In a Phase 2a trial for the treatment of non-alcoholic fatty liver disease (NAFLD) and elevated LDL-C, patients who received VK2809 demonstrated statistically significant reductions in LDL-C and liver fat content compared with patients who received placebo. Viking is also developing VK2735, a novel dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors for the potential treatment of various metabolic disorders. Data from a Phase 1 and a Phase 2 trial evaluating VK2735 (dosed subcutaneously) for metabolic disorders demonstrated an encouraging safety and tolerability pro well as positive signs of clinical benefit. The company is also evaluating an oral formulation of VK2735 in a Phase 1 trial. In the rare disease space, Viking is developing VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the potential treatment of X-linked adrenoleukodystrophy (X-ALD). VK0214 is currently being evaluated in a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD. For more information about Viking Therapeutics, please visit . Forward-Looking Statements This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including statements about Viking's expectations regarding its clinical and preclinical development programs, anticipated timing for reporting clinical data and cash resources. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials, including those for VK2735, VK0214, VK2809, the company's incretin receptor agonists, and its amylin and calcitonin receptor agonists; risks that prior clinical and preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent Quarterly Reports on Form 10-Q, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law. Company Codes: NASDAQ-SMALL:VKTX

ROCKVILLE, Md. and SUZHOU, China, Jan. 9, 2023 /PRNewswire/ -- Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, metabolic, autoimmune, ophthalmology and other major diseases, today announced that the first participant has been successfully dosed in a multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trial (DREAMS-1) of mazdutide (Innovent R&D Code: IBI362), a glucagon-like peptide 1 receptor (GLP-1R) and glucagon receptor (GCGR) dual agonist, in Chinese patients with type 2 diabetes. This is a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical study to evaluate the efficacy and safety of mazdutide in Chinese patients with type 2 diabetes inadequately controlled by diet and exercise alone (ClinicalTrials.gov, NCT05628311). The study plans to enroll approximately 300 subjects, randomized in a 1:1:1 ratio, to receive either mazdutide 4.0 mg, mazdutide 6.0 mg or placebo. The study treatment period will be 48 weeks in total, including a 24-week double-blind treatment period and a 24-week extension treatment period. The primary endpoint will be the change from baseline to week 24 in glycated hemoglobin (HbA1c) levels. In a phase 2 clinical study of mazdutide in Chinese patients with type 2 diabetes（ClinicalTrials.gov, NCT04965506）, mazdutide was well tolerated and the overall safety profile was similar to other GLP-1 class drugs. The mean change in HbA1c from baseline to week 20 was up to -1.67% with mazdutide (0.03% with placebo and -1.35% with dulaglutide). The mean change in body weight from baseline to week 20 was up to -7.11% with mazdutide (-1.38% with placebo and -2.69% with dulaglutide). In the study, mazdutide also offered multiple metabolic benefits to subjects, including reduced blood pressure, blood lipids and liver enzyme levels. The DREAMS-1 study will further evaluate the long-term efficacy and safety of mazdutide in Chinese patients with type 2 diabetes inadequately controlled by diet and exercise alone. Professor Dalong Zhu, the principal investigator of the study, Nanjing Drum Tower Hospital, stated, "Diabetes has become a global health problem. At present, there are many kinds of medicines for the treatment of diabetes, and GLP-1 receptor agonists have been widely used in recent years because of their good efficacy and safety, especially the function of cardiovascular protection. The latest research and development direction of GLP-1 receptor agonists is multi-target. Mazdutide, as a representative of the next generation of GLP-1 multi-target receptor agonists, not only has a significant hypoglycemic and weight-loss effect but also reduces blood pressure, blood lipids and liver enzymes in the phase 2 study, suggesting that mazdutide may be able to effectively control blood glucose while also offering multiple metabolic benefits to patients with type 2 diabetes. In addition, both the once-weekly dosing regimen and innovative autoinjector device can significantly improve the quality of life and compliance of patients. I look forward to mazdutide delivering excellent efficacy in the phase 3 study and will potentially bring a new treatment option to more patients with type 2 diabetes." Professor Jiajun Zhao, the principal investigator of the study, Shandong Provincial Hospital, stated, "With the economic development of China, the prevalence of diabetes and other metabolic diseases, such as obesity, gout, hyperlipidemia, etc. has gradually increased in China, especially the patients with diabetes and multiple diseases tend to be exposed to significantly increased cardiovascular risk factors, which leads to the increased burden of disease as well as the socio-economic burden for patients. Thus, treatments that can significantly reduce blood glucose, body weight, and multiple cardiovascular risk factors are urgently needed. Mazdutide, as a new GLP-1R/GCGR dual agonist worldwide, has a novel mechanism, in addition to the GLP-1 receptor, its additional agonistic glucagon receptor may bring benefits such as increasing basal metabolic rate, improving blood lipid profile, reducing liver fat, and enhancing appetite suppression. In the phase 2 clinical study, mazdutide have been shown to have significant glucose-lowering and weight-loss efficacy in Chinese patients with type 2 diabetes, and may offer multiple benefits to patients, including a favorable safety profile. I am confident that mazdutide will be successful in phase 3 clinical study and look forward to more clinical value of mazdutide to be unlocked." Dr. Lei Qian, Vice President of Clinical Development at Innovent, stated, "Mazdutide is a dual GLP-1R/GCGR agonist with both hypoglycemic and weight-loss efficacy, reflecting the best-in-class potential among GLP-1 receptor agonists and co-agonists. The results of the phase 2 study in Chinese subjects with type 2 diabetes demonstrated the efficacy and safety of mazdutide, showing significant clinical value and laying a solid foundation for the phase 3 program. We hope to bring mazdutide to patients as soon as possible, providing a new, efficacious, safe and patient-friendly treatment option for patients with type 2 diabetes." About Diabetes The prevalence of diabetes among adults in China is 11.6%, of which type 2 diabetes accounts for about 90% of the total number of diabetic patients, and the number of patients is still increasing. Poor glycemic control will lead to irreversible microvascular and macrovascular complications such as decreased visual acuity, blindness, renal insufficiency, peripheral neuropathy, myocardial infarction, stroke, amputation, etc. As a latent disease with serious complications and high incidence, diabetes mellitus has seriously threatened human health. Currently, there are many treatment options for diabetes, and in addition to effective glycemic control, the development of new hypoglycemic drugs may also provide additional benefits for diabetic patients in terms of weight loss and cardiovascular risk reduction. About Mazdutide Innovent entered into a licensing agreement with Eli Lilly and Company (Lilly) for the development and potential commercialization of OXM3 (also known as mazdutide), a GLP-1R and GCGR dual agonist, in China. In parallel, Lilly is developing OXM3 outside China. Mazdutide is a long-acting synthetic peptide related to mammalian oxyntomodulin (OXM) with a fatty acid side chain attached thereto. Mazdutide exhibits a prolonged duration of action allowing for once-weekly administration. Mazdutide is thought to exert its biological effects by activating the GLP-1 receptor and glucagon receptor in human beings, which improves glucose tolerance and induces weight loss, mimicking the effects of endogenous oxyntomodulin. In addition to the effects of GLP-1 receptor agonists on promoting insulin secretion, lowering blood glucose and reducing body weight, mazdutide may also increase energy expenditure and improve hepatic fat metabolism through the activation of glucagon receptor. About Innovent Inspired by the spirit of "Start with Integrity, Succeed through Action," Innovent's mission is to develop, manufacture and commercialize high-quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high-quality innovative medicines for the treatment of cancer, autoimmune disease, metabolic disorder and other major diseases. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801.HK. Since its inception, Innovent has developed a fully integrated multi-functional platform which includes R&D, CMC (Chemistry, Manufacturing, and Controls), clinical development and commercialization capabilities. Leveraging the platform, the company has built a robust pipeline of 36 valuable assets in the fields of cancer, metabolic disorder, autoimmune disease and other major therapeutic areas, with 8 approved products on the market. These include: TYVYT® (sintilimab injection), BYVASDA® (bevacizumab biosimilar injection), SULINNO® (adalimumab biosimilar injection), HALPRYZA® (rituximab biosimilar injection) , Pemazyre® (pemigatinib oral inhibitor), olverembatinib (BCR-ABL TKI) , Cyramza® (ramucirumab) and Retsevmo® (selpercatinib). An additional 3 assets are under NMPA NDA review, 5 assets are in Phase 3 or pivotal clinical trials, and 20 more molecules are in clinical studies. Innovent has built an international team with advanced talent in high-end biological drug development and commercialization, including many global experts. The company has also entered into strategic collaborations with Eli Lilly and Company, Sanofi, Adimab, Incyte, MD Anderson Cancer Center and other international partners. Innovent strives to work with many collaborators to help advance China's biopharmaceutical industry, improve drug availability and enhance the quality of the patients' lives. For more information, please visit: . and . Note: TYVYT® (sintilimab injection) is not an approved product in the United States. BYVASDA® (bevacizumab biosimilar injection), SULINNO®, and HALPRYZA® (rituximab biosimilar injection) are not approved products in the United States. TYVYT® (sintilimab injection, Innovent) BYVASDA® (bevacizumab biosimilar injection, Innovent) HALPRYZA® (rituximab biosimilar injection, Innovent) SULINNO® (adalimumab biosimilar injection, Innovent) Pemazyre® (pemigatinib oral inhibitor, Incyte Corporation). Pemazyre® was discovered by Incyte Corporation and licensed to Innovent for development and commercialization in Mainland China, Hong Kong, Macau and Taiwan. CYRAMZA® (ramucirumab, Eli Lilly). Cyramza® was discovered by Eli Lilly and licensed to Innovent for commercialization in Mainland China. Retsevmo® (selpercatinib, Eli Lilly). Retsevmo® was discovered by Eli Lilly and licensed to Innovent for commercialization in Mainland China. Disclaimer: 1. This indication is still under clinical study, which hasn't been approved in China. 2. Innovent does not recommend any off-label usage. Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent, are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialise or turn out to be incorrect. SOURCE Innovent Biologics