A Randomized, Placebo-Controlled, Multi-Center, Phase 2 Clinical Trial to Evaluate the Efficacy and Safety of GV1001 in Patients with Benign Prostatic Hyperplasia (BPH).

To evaluate the efficacy and safety of three dosing schemes of GV1001 in patients with BPH.

Eligible patients were male and ≥50 years old, with an International Prostate Symptom Score (IPSS) ≥13, peak urine flow 5-15 mL/sec, residual urine volume ≤200 mL, and prostate volume ≥30 cc. After a 4 week run-in period, patients were randomly assigned to one of three treatment schedules (Group 1: GV1001 0.4 mg, 2 week interval; Group 2: GV1001 0.56 mg, 2 week interval; Group 3: GV1001 0.56 mg, 4 week interval) or placebo (Group 4). The eligible patients were administered GV1001 or placebo, for a total of 7 intradermal injections that were administered at 2-week intervals at Weeks 0, 2, 4, 6, 8, 10, and 12. Treatment continued for 12 weeks, and efficacy was evaluated at weeks 4, 8, 12, 13, and 16. Safety was evaluated throughout the 16 week period. The primary efficacy variable was change from baseline in total IPSS. Secondary endpoints were changes in maximum urinary flow rate (Qmax), residual urine volume, prostate volume, International Index of Erectile Function (IIEF), plasma testosterone, dihydrotestosterone (DHT), and prostate-specific antigen (PSA).

A total of 161 patients were included (Group 1, n = 41; Groups 2-4, n = 40). Most patients (88.8%) received all planned doses of study treatment. At week 13, a statistically significant difference in the mean change in IPSS from baseline was seen in GV1001 treatment Groups 1 and 2 versus the control group for the full analysis population (-3.5 [control] vs -7.2 and -6.8 in Groups 1 and 2, respectively; both p < 0.05). Statistically significant differences in changes from baseline were also observed at weeks 8, 12, 13, and 16 in treatment Groups 1 and 2 versus control in the per protocol population. A statistically significant reduction in prostate gland volume was seen at week 16 versus control in all treatment groups (0.8 [control] vs -4.6, -2.5, and -4.2 in Groups 1-3, respectively; all p < 0.05). No statistically significant differences were seen in other secondary outcome measures. AE reporting was similar across all four groups. No treatment-emergent AEs were considered to be related to study drug.

Study results indicate that GV1001 was effective and well tolerated, and may provide potential beneficial effects in patients with BPH. Compared with medical therapies that require daily dosing, the convenient dosing regimen of GV1001 may provide greater patient adherence. Further investigation of these observations will require large-scale clinical evaluation. This article is protected by copyright. All rights reserved.

BJU international. 2018 Apr 06 [Epub ahead of print]

Kyong Tae Moon, Tag Keun Yoo, Se Yun Kwon, Ji Yong Ha, Seok Soo Byun, Jang Hwan Kim, Jae Il Chung, Tae Hyoung Kim, Hong Sang Moon, Sang Jae Kim, Kyung Seop Lee

Department of Urology, Eulji General Hospital, Eulji University School of Medicine, Seoul, Korea., Department of Urology, Dongguk University College of Medicine, Gyeongju, Korea., Department of Urology, Keimyung University School of Medicine, Daegu, Republic of Korea., Department of Urology, Seoul National University College of Medicine, Seoul National University Bundang Hospital, Seongnam, Korea., Departments of Urology, Yonsei University College of Medicine, Seoul, Korea., Department of Urology, Busan Paik Hospital, Inje University, Busan, Korea., Department of Urology, College of Medicine, ChungAng University, Seoul, Korea., Department of Urology, Hanyang University College of Medicine, Seoul, Korea., Teloid Inc., 920 Westholme Ave, Los Angeles(City), CA, 90024, U.S.A.