TRPM8

CRANBURY, N.J., Feb. 10, 2025 /PRNewswire/ -- IVIEW Therapeutics Inc., a clinical-stage biotechnology company dedicated to advancing innovative treatments for ocular diseases, today announced positive topline data from its Phase 1/2 clinical trial evaluating 2 concentrations of IVW-1001, a novel TRPM8 agonist delivered via an ophthalmic eyelid wipe, for the treatment of dry eye disease. The trial enrolled 150 patients in 10 sites in the United States (NCT06400459). Given the novel compound and novel delivery method, the purpose of the trial was to assess tolerability/safety, determine appropriate dose, and identify potential efficacy endpoints for future trials. Safety Results Confirm Favorable Tolerability Profile IVW-1001 was well-tolerated across all treatment groups, with the majority of adverse events (AEs) being mild or moderate in severity. Ocular irritation was the most commonly reported AE, observed slightly more frequently in the IVW-1001 treatment groups (3%) compared to vehicle (0%). Importantly, no serious adverse events (SAEs) related to the investigational product (IP) were reported, and no patients discontinued the study due to IP-related AEs. These findings reinforce the safety of IVW-1001 and its suitability for further clinical development. Preliminary Efficacy Results Identify Potential Endpoints For Future Trials The trial achieved statistically significant and clinically meaningful improvements in key signs and symptoms of dry eye patients at the final timepoint (Week 4), including reductions in corneal fluorescein staining (total and inferior zones) and significant reductions in symptom scores, including Eye Dryness Score and SANDE (frequency and severity). There were also positive trends in both basal (anesthetized) and reflex (unanesthetized) tear production. Moreover, the data demonstrated a dose-response relationship, with the higher dose (0.2%) consistently showing greater efficacy across multiple endpoints. Key Data Highlights at Week 4: Total Corneal Fluorescein Staining (Zones 1-5): IVW-1001 0.2% reduced staining (-1.7 vs. -1.3 for vehicle, p=0.0097) Eye Dryness Score: IVW-1001 0.2% demonstrated a significant reduction (-22.8 vs. -18.3 for vehicle, p=0.0315) SANDE (Symptom Frequency): IVW-1001 0.2% significantly reduced symptom frequency (-14.4 vs. -9.0 for vehicle, p=0.0017) Advancing Toward Larger Pivotal Trials "We are thrilled to have reached this key milestone in our clinical development program for IVW-1001," said Houman Hemmati, MD, PhD, Board Director, Chief Medical Advisor at IVIEW Therapeutics Inc. "The data from this Phase 1/2 trial underscore the potential of IVW-1001 to address both the signs and symptoms of dry eye disease. These results provide a strong foundation for advancing into pivotal trials with confidence, further exploring IVW-1001's efficacy and safety in a larger patient population." A Step Forward in Innovative Ophthalmic Treatments Bo Liang, PhD, MBA, Co-founder, Chairman, and CEO of IVIEW Therapeutics Inc., commented, "Both the statistically significant and clinically meaningful improvements observed across both signs and symptoms reaffirm the potential of our TRPM8 agonist program to address the unmet needs of patients with dry eye disease. IVW-1001's novel mechanism of action and unique delivery via an ophthalmic eyelid wipe offer a differentiated approach to treatment. We are committed to advancing this program rapidly to bring a new, effective therapeutic option to patients worldwide." About IVIEW Therapeutics Inc. IVIEW Therapeutics Inc. is a clinical-stage biotechnology company focusing on innovative ophthalmic therapeutics. Driven by the pursuit of cutting-edge science, we aim to develop differentiated products that fulfill unmet medical needs. We invest in novel mechanisms of action and advanced drug delivery technology platforms to bring forward assets with potentially superior target product profiles. Our innovative portfolio of small molecules and gene therapy assets covers dry eye, myopia, conjunctivitis, glaucoma, and presbyopia. Located in Cranbury, New Jersey, IVIEW operates a state-of-the-art 11,045 sq. ft. laboratory and office space in the Princeton area. For additional information, please visit our website at . For further inquiries or media contacts, please reach out to our team at [email protected]. SOURCE IVIEW Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CRANBURY, N.J., Sept. 17, 2024 /PRNewswire/ -- IVIEW Therapeutics Inc., a clinical-stage biotechnology company dedicated to advancing innovative treatments for ocular diseases, today announced the completion of patient recruitment for its Phase 1/2 clinical trial evaluating IVW-1001, a novel TRPM8 agonist delivered via ophthalmic eyelid wipe, for the treatment of signs and symptoms of dry eye disease. The company anticipates reporting topline data in the first quarter of 2025. Advancing Toward Promising Results "We are thrilled to have reached this significant milestone in our clinical development program for IVW-1001," said Houman Hemmati, MD, PhD, Chief Medical Advisor at IVIEW Therapeutics Inc. "Completing patient enrollment brings us one step closer to potentially offering a new therapeutic option for individuals suffering from dry eye disease. We are eagerly awaiting the trial results and remain optimistic about the potential impact on patient care." A Step Forward in Innovative Ophthalmic Treatments Bo Liang, PhD, MBA, Co-founder, Chairman, and CEO of IVIEW Therapeutics Inc., commented, "The completion of patient recruitment ahead of schedule is a testament to the dedication of our clinical team and the interest from the ophthalmic community. IVW-1001's novel mechanism of TRPM8 activation and its innovative delivery through an ophthalmic eyelid wipe could offer a differentiated treatment for dry eye disease worldwide. We look forward to sharing our findings as soon as they are available." About IVIEW Therapeutics Inc. IVIEW Therapeutics Inc. is a clinical-stage biotechnology company focusing on innovative ophthalmic therapeutics. Driven by the pursuit of cutting-edge science, we aim to develop differentiated products that fulfill unmet medical needs. We invest in novel mechanisms of action and advanced drug delivery technology platforms to bring forward assets with potentially superior target product profiles. Our innovative portfolio of small molecules and gene therapy assets covers dry eye, myopia, conjunctivitis, glaucoma, and presbyopia. Located in Cranbury, New Jersey, IVIEW operates a state-of-the-art 11,045 sq. ft. laboratory and office space in the Princeton area. For additional information, please visit our website at . SOURCE IVIEW Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

MANCHESTER, England & MOUNTAIN VIEW, Calif.--( BUSINESS WIRE )--Axalbion, a clinical-stage biopharmaceutical company focused on developing novel medicines to treat cough, announced that the first patient has been dosed in the second part of a Phase 2 proof-of-concept, randomized, placebo-controlled clinical study in chronic cough with AX-8, its lead compound. AX-8 is a potent and selective oral agonist of the transient receptor potential melastatin 8 (TRPM8) ion channel. The company has raised additional financing from existing and new investors to support the trial execution and Phase 3 planning. “We are pleased to announce that Axalbion has launched the second part of its Phase 2 clinical trial with AX-8 in chronic cough, a disease with significant unmet needs,” said Michael Kitt, MD, chief executive officer of Axalbion. “In the first part of this study, AX-8 showed promising results with a robust and sustained reduction in cough frequency, particularly in patients experiencing the greatest throat discomfort, a distressing symptom observed in most chronic cough patients.” Data were presented in an oral session at the American Thoracic Society (ATS) 2023 International Conference. Based on the initial positive findings, the company initiated Part 2 of the study of AX-8 in patients with chronic cough with moderate-to-severe throat discomfort (i.e., those with a throat discomfort visual analog scale score ≥50mm out of 100mm), who are the most likely to benefit from treatment with AX-8. The Phase 2 trial is a randomized, double-blind, placebo-controlled, crossover study evaluating the efficacy and safety of AX-8 in approximately 50 patients with refractory or unexplained chronic cough (RCC/UCC). Patients will receive 40 mg of the drug or matching placebo thrice daily (as opposed to twice daily in Part 1) approximately four hours apart for two weeks, followed by a seven-day washout period, before crossing over to the opposite treatment for two weeks. AX-8 is formulated as an orally disintegrating tablet placed on the tongue. Cough frequency will be assessed with an objective cough recording device. The primary efficacy endpoint will be the placebo-adjusted change in cough frequency compared to baseline. The study will take place at multiple centers in the U.K. and is expected to be completed by the second half of 2025. For more information, please visit clinicaltrials.gov: NCT04866563 . About TRPM8 and AX-8 TRPM8 is expressed in many of the sensory fibers innervating the upper airways. Axalbion believes that activating TRPM8-expressing fibers with AX-8, a potent TRPM8 agonist, will normalize the upper airway sensitivity in chronic cough patients, decreasing coughing and irritation of the throat. David Julius and Ardem Patapoutian were awarded the 2021 Nobel Prize in Physiology or Medicine for their discoveries of thermal and mechanical transducers, including the discovery of TRPM8. Since its cloning in 2002, extensive research has identified TRPM8 as a potential target to relieve symptoms or cure several diseases. Animal models have shown that activation of TRPM8-expressing fibers in the skin inhibits sensory hypersensitivity in peripheral neuropathy and inflammation, as well as itching. About Chronic Cough Chronic cough is defined as a cough lasting for more than eight weeks, often occurring in bouts. More than 80% of patients in tertiary cough clinics have throat irritation which triggers cough bouts, defining a population with oropharyngeal hypersensitivity potentially responding to a topical locally acting drug such as AX-8. After potential triggers such as gastro-intestinal reflux disease (GERD) and asthma have been excluded or treated, cough still remains a significant and common problem for many patients. Chronic cough has a substantial impact on quality of life, for example, around half of female patients having urinary stress incontinence. There are no approved drugs for the treatment of chronic cough in the U.S., making it a significant unmet need affecting approximately 26 million people in the U.S. alone, with approximately three million having RCC/UCC lasting for more than a year and approximately six million having RCC/UCC lasting more than eight weeks and less than one year. About Axalbion Axalbion is a privately held, clinical-stage biotechnology company developing novel medicines for the treatment of chronic cough. The company, founded in December 2016, is based in Manchester, United Kingdom, a region known for its centers of excellence in respiratory research. For further information, please visit www.axalbion.com .