GT20029 is the first topical PROTAC compound in the world that has completed a phase 1 clinical trial in both China and the United States

Kintor Pharmaceutical Limited’s GT20029 was safe and well tolerated and had good pharmacokinetic characteristics in healthy people and people with androgenetic alopecia (AGA) or acne, according to a phase 1 clinical trial.

GT20029 is the first topical PROTAC compound in the world that has completed a phase 1 clinical trial in both China and the United States.

This is a randomized, double-blind, placebo-controlled, parallel group, dose escalation phase 1 clinical study to evaluate the safety, tolerability, and pharmacokinetics of GT20029 following topical single ascending dose administration (SAD) in healthy subjects and multiple ascending dose administration (MAD) in subjects with AGA or acne.

The results showed that GT20029 was safe and well tolerated at all dose levels in all cohorts. No treatment-emergent adverse events (TEAEs) related to GT20029 in the SAD stage were reported. The most common TEAEs in the MAD stage were mild and included dryness, itching, burning, and pain at the application site. No serious adverse events were reported. No severe (grade ≥ 3) TEAEs and no subject withdrawal or death caused by TEAEs was reported.

In the SAD stage, study participants had no systemic exposure at all dose levels, and all sample concentrations were below the lower limit of quantification (LLOQ, 0.003 ng/mL). In the MAD stage, after 14 days of continuous administration in subjects with AGA or acne, the systemic exposure was very limited, and the mean maximum observed concentration (Cmax) of all dose levels fluctuated near the LLOQ, with the highest not exceeding 0.015 ng/mL.

In preclinical studies, by degrading androgen receptor (AR) protein, GT20029 could block the shrinkage and miniaturization of hair follicles that was caused by the activation of the AR signaling pathway. As the result, it prevented the hair from thinning, softening, and falling out. GT20029 could also effectively inhibit sebaceous gland development and sebum secretion. With limited skin penetration, GT20029 could avoid high systemic exposure and achieve a better safety profile. The repeated pharmacodynamics studies in a dihydrotestosterone-induced mouse model showed that GT20029 significantly promoted hair growth, with statistical difference. The study of testosterone propionate-induced skin hamster flank organ acne model showed that GT20029 significantly inhibited enlargement of the flank organ, with statistical difference.

"We will accelerate the initiation of a phase 2 clinical trial of GT20029, maintain our leading position in the development of topical PROTAC drug candidate globally,” says Dr. Youzhi Tong, founder, chairman, and CEO of Kintor Pharma, in a news release.  “We believe that GT20029 would strengthen our market position in the dermatology area, together with our novel drug KX-826, to provide a diversified portfolio of therapies to patients with AGA or acne. "

About GT20029

GT20029 is a topical AR degrader developed by Kintor Pharma's PROTAC platform. The China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA) cleared GT20029's clinical trial applications for treating AGA and acne in April 2021 and July 2021, respectively. In August 2022, Kintor Pharma had completed the enrollment and dosing of subjects for its China phase 1 clinical trial. In November 2022, Kintor Pharma announced the top-line results for its China phase 1 clinical trial. In October 2022, Kintor Pharma announced completion of subject enrollment and dosing in its US phase 1 clinical trial.