Articles from Pretzel Therapeutics

Pretzel Therapeutics, a leader in harnessing cellular energetics to develop novel treatments for a range of conditions spanning neurological, muscle, metabolic and rare diseases, today announced that new preclinical data supporting mitochondrial RNA polymerase (POLRMT) as a novel target for the treatment of metabolic disease will be presented at ObesityWeek 2025 in Atlanta, Georgia. The POLRMT program, the lead initiative in Pretzel’s energetics modulation franchise, represents a novel liver-targeted mechanism for the treatment of metabolic conditions, including obesity, and is currently in preclinical development.

Pretzel Therapeutics, a leader in harnessing cellular energetics to develop novel treatments for a range of conditions spanning neurological, muscle, metabolic and rare diseases, today announced the appointment of Ashish Dugar, Ph.D., MBA as Chief Development Officer (CDO). Dr. Dugar joins Pretzel with nearly 25 years of industry experience across clinical development, medical affairs and commercial leadership roles, most recently at Dyne Therapeutics where he oversaw global medical affairs for rare neuromuscular diseases.

Pretzel Therapeutics, a leader in harnessing cellular energetics to develop novel treatments for a range of conditions spanning neurological, muscular atrophies, metabolic and rare disease, today announced the publication of new research findings in Nature elucidating a novel disease-modulating mechanism of action for the treatment of mitochondrial DNA (mtDNA) depletion syndromes. The article, “Small Molecules Restore Mutant Mitochondrial DNA Polymerase Activity” (Valenzuela et al., Nature, 10.1010.1038/s41586-025-08856-9) provides critical insights into a unique bioenergetics restoration pathway, reinforcing the therapeutic potential of Pretzel’s development pipeline and supporting continued advancement of its lead therapeutic, PX578, which recently entered Phase 1 clinical development.

Pretzel Therapeutics, a leader in harnessing cellular energetics to develop novel treatments for a range of conditions spanning neurological, muscular atrophies, metabolic and rare disease, today announced that it has initiated recruitment in a Phase 1 clinical study of PX578, the lead therapeutic in its bioenergetics restoration franchise. The randomized, double-blind, placebo-controlled study is designed to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of PX578 in healthy adult participants, with a potential clinical trial in MDDS patients planned for 2026.

Pretzel Therapeutics, a biotechnology company harnessing the intricacies of mitochondrial biology to develop groundbreaking therapies, launched today with a $72.5 million Series A financing to pioneer novel therapies to modulate mitochondrial function. The financing was led by ARCH Venture Partners and Mubadala Capital with participating investors HealthCap, Cambridge Innovation Capital, Cambridge Enterprise, Angelini Ventures, GV, Invus, Eir Ventures, GU Ventures, and Karolinska Institutet Holding.