PX578 / Pretzel Therapeutics - LARVOL DELTA

A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part A & B) (ANZCTR) - P1 | N=96 | Completed | Sponsor: Pretzel Therapeutics Inc. | Recruiting ➔ Completed

Trial completion • Metabolic Disorders

Pretzel Therapeutics Receives FDA Fast Track Designation for PX578 for the Treatment of POLG-mediated Primary Mitochondrial Disorders (Businesswire) - "Phase 2 (POLARIS) study initiation planned for late 2026...FDA Fast Track designation reflects the significant unmet medical need in POLG disease and supports ongoing engagement with FDA as Pretzel advances PX578 toward Phase 2 study initiation."

Fast track • New P2 trial • Genetic Disorders • Rare Diseases

A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part C) (ANZCTR) - P1 | N=12 | Completed | Sponsor: Pretzel Therapeutics Inc. | Not yet recruiting ➔ Completed | N=24 ➔ 12

Enrollment change • Trial completion • Metabolic Disorders

A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part A & B) (ANZCTR) - P1 | N=112 | Recruiting | Sponsor: Pretzel Therapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Metabolic Disorders

Pretzel Therapeutics Initiates Phase 1 Clinical Study Evaluating PX578, Lead Therapeutic in its Bioenergetics Restoration Franchise (Businesswire) - "Pretzel Therapeutics...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."

New trial • Trial status • Genetic Disorders • Rare Diseases