Fei Chen, Peter Hillemanns, Hongjie Ruan, Xinyan Chen, Yue Wang, Zoltán Novàk, Youzhong Zhang, Zhixue You, Bing Wei, Weiguo Lü, Jing Yu, Xiujie Sheng, Linn Woelber, Vladimír Dvořák, Weihua Song, You‐Lin Qiao, Joanna Zhang, Eileen He, Jinghe Lang, Lan Zhu
BACKGROUND: Ablative/excisional surgery is the recommended therapy for cervical high-grade squamous intraepithelial lesions (HSILs) but is associated with cervical damage and elevated risk of complications upon subsequent pregnancies. METHODS: ) versus placebo in adult women with cervical HSILs (randomization ratio: 2:1). Upon completion of 6-month treatment, patients in the APL-1702 group entered into a 6-month extension phase with observation only. The primary efficacy endpoint was treatment response, as defined by normal histology or low-grade squamous intraepithelial lesion (LSIL) histology plus human papillomavirus (HPV) clearance, at 6 months in a modified intent-to-treat (mITT) population. FINDINGS: A total of 402 women were enrolled. The 6-month response rate was 41.1% (104/253) in the APL-1702 group versus 21.7% (28/129) in the placebo group (p < 0.001). The HPV16/18 clearance rates were 31.4% (49/156) and 15.4% (12/78) in the APL-1702 and placebo groups, respectively (p = 0.011). The rates of treatment-emergent adverse events were 56.8% (151/266) and 56.0% (75/134) in the APL-1702 and placebo groups, respectively. At the end of the 6-month extension period, 54.9% (56/102) of the patients who responded at the end of the 6-month treatment period maintained a response. CONCLUSIONS: APL-1702 significantly increased the 6-month response rate in patients with cervical HSILs versus placebo control, with a favorable safety profile. FUNDING: This trial was sponsored by Asieris Pharmaceuticals (Shanghai, China).