Dosing Begins in Phase 1 Trial of ETD002, Potential CF Inhalation Therapy

Dosing Begins in Phase 1 Trial of ETD002, Potential CF Inhalation Therapy
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Enterprise Therapeutics announced that dosing has begun in a Phase 1 clinical trial testing ETD002, a potential inhaled treatment for cystic fibrosis (CF).

The study (NCT04488705) will assess the safety and tolerability of EDT002 in ascending single and multiple doses in healthy people as compared with a matching placebo. It is due to conclude in December.

“We are excited to have begun the clinical stage of development for ETD002,” John Ford, PhD, CEO of Enterprise Therapeutics, said in a press release. The therapy “has the potential to significantly increase the quality of life for people living with CF, for many of whom existing therapies are not effective.”

ETD002 is designed to boost — or potentiate — the activity of TMEM16A, a chloride channel found on the surfaces of cells in airway tissues, where it helps to regulate the amount of salt and fluids.

ETD002 enhanced the activity of TMEM16A in preclinical studies, thereby increasing fluid flow into the airways, as well as promoting mucus thinning and clearance, the company reports. Excessive mucus buildup in the airways is one of the main causes of breathing difficulty in CF patients, and raises the risk of infection.

CFTR modulation is another available and approved approach to treat CF. These therapies largely aim to correct mutations found in the CFTR (cystic fibrosis transmembrane conductance regulator) gene, which is the defective gene causing CF. Because of their specificity to certain mutations however, they are not always available or effective for all CF patients.

TMEM16A potentiation works regardless of a patient’s CFTR mutational status, making this approach applicable to all  with CF, and potentially to patients with other lung diseases.

“Although CFTR modulators have successfully demonstrated improved clinical outcomes in those genetically suited to these therapies, we are hopeful that TMEM16A potentiation via ETD002 will provide clinical benefit to the many people with CF who do not share these CFTR mutations,” David Morris, MD, Enterprise’s chief medical officer, said.

ETD002 is expected to work both as a single therapy and in combination with other therapies, including those that repair the mutated CFTR.

“We look forward to generating our first data in human volunteers over the next few months and are grateful to the subjects and investigators who are helping us to advance this novel treatment for individuals with CF,” Morris concluded.

The trial, which seeks to enroll about 88 adult participants, is currently recruiting in the U.K. More information can be found here. The trial is supported by a Therapeutics Development Award from the Cystic Fibrosis Foundation.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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