Zymedi and NHLBI collaborate to advance ZMA001 in PAH

Partners plan to conduct Phase 1 clinical trial to test treatment's safety

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by Mary Chapman |

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Zymedi is collaborating with the National Heart, Lung, and Blood Institute (NHLBI) to advance the development of its first-in-class pulmonary arterial hypertension (PAH) treatment candidate ZMA001.

The South Korea-based company and the NHLBI, part of the National Institutes of Health, have signed a clinical Cooperative Research and Development Agreement (CRADA), a formal agreement which facilitates research and development collaboration between at least one federal lab and at least one nonfederal entity.

Under the agreement, Zymedi plans to work with investigators on the Intramural NIH PAH Translational Research Team to conduct a clinical trial to test the safety of ZMA001. The first-in-human Phase 1 trial will enroll healthy volunteers.

“We are excited to collaborate with the NIH to advance the development of ZMA001 for PAH,” Sunghoon Kim, PhD, Zymedi founder and CEO, said in a press release. “The NHLBI and the NIH Clinical Center are world-renowned institutions with a wealth of clinical expertise and resources, and we believe that this collaboration has the potential to bring meaningful benefit to patients in need.”

Pulmonary arterial hypertension, caused by a narrowing of the pulmonary arteries, is a type of pulmonary hypertension, a chronic and progressive disorder associated with high blood pressure in pulmonary vessels.

Currently approved treatments are designed to reduce blood pressure to help manage symptoms, which can include shortness of breath, excessive fatigue, weakness, chest pain, fainting, and ultimately heart failure, but there is an urgent need for therapies that can address the root cause of PAH and extend survival.

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ZMA001 expected to reduce inflammation, tissue scarring in PAH patients

ZMA001 is a human monoclonal antibody designed to control or inhibit inflammation in pulmonary blood vessels by inhibiting lysyl-tRNA synthetase-dependent (KARS1), a protein required for the infiltration of certain immune cells.

By binding to this target, the compound is expected to reduce inflammation and tissue scarring in PAH patients, without inducing low blood pressure, with often occurs with other treatments that use vasodialators.

Preclinical studies in animal models of PAH have shown that ZMA001 significantly improves a number of outcomes, including heart health, immune cell infiltration to the lung, and tissue scarring (fibrosis). The treatment also extended survival and showed promise when combined with sildenafil (sold as Revatio and Liqrev), a standard vasodilator used in PAH.

“Discovery and development of new and more effective therapies for PAH remains an unmet need,” said Jason Elinoff, MD, the upcoming trial’s principal investigator and co-director of the NHLBI Translational Pulmonary Arterial Hypertension Program. “Therefore, ZMA001, a novel monoclonal antibody targeting pathways thought to play a key role in PAH pathogenesis, may contribute to meeting this important goal.”

The research partnership will initially focus on establishing the experimental therapy’s safety in patients, Elinoff said. If that is successful, future clinical research will explore whether the experimental therapy can halt or reverse PAH progression.

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