Proof-of-Concept Study with AX-8 Designed to Demonstrate Efficacy in Patients Suffering from Chronic Cough with Moderate-to-Severe Throat Discomfort
MANCHESTER, UK & MOUNTAIN VIEW, CA, USA I August 27, 2024 I 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.
SOURCE: Axalbion
Post Views: 2,052
Proof-of-Concept Study with AX-8 Designed to Demonstrate Efficacy in Patients Suffering from Chronic Cough with Moderate-to-Severe Throat Discomfort
MANCHESTER, UK & MOUNTAIN VIEW, CA, USA I August 27, 2024 I 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.
SOURCE: Axalbion
Post Views: 2,052
