Fexapotide triflutate: results of long-term safety and efficacy trials of a novel injectable therapy for symptomatic prostate enlargement

These studies were undertaken to determine if fexapotide triflutate 2.5 mg transrectal injectable (FT) has significant long-term (LT) safety and efficacy for the treatment of benign prostatic hyperplasia (BPH).

Two placebo controlled double-blind randomized parallel group trials with 995 BPH patients at 72 sites treated 3:2 FT:placebo, with open-label FT crossover (CO) re-injection in 2 trials n = 344 and long-term follow-up (LF) 2-6.75 years (mean 3.58 years, median 3.67 years; FT re-injection CO mean 4.27 years, median 4.42 years) were evaluated. 12 months post-treatment patients elected no further treatment, approved oral medications, FT, or interventional treatment. Primary endpoint variable was change in Symptom Score (IPSS) at 12 months and at LF. CO primary co-endpoints were 3-year incidence of (1) surgery for BPH in FT treated CO patients versus patients crossed over to oral BPH medications and (2) surgery or acute urinary retention in FT-treated CO placebo patients versus placebo patients crossed over to oral BPH medications. 28 CO secondary endpoints assessed surgical and symptomatic outcomes in FT reinjected patients versus conventional BPH medication CO and control subgroups at 2 and 3 years.

FT injection had no significant safety differences from placebo. LF IPSS change from baseline was higher in FT treated patients compared to placebo (median FT group improvement - 5.2 versus placebo - 3.0, p < 0.0001). LF incidence of AUR (1.08% p = 0.0058) and prostate cancer (PCa) (1.1% p = 0.0116) were both reduced in FT treated patients. LF incidence of intervention for BPH was reduced in the FT group versus oral BPH medications (8.08% versus 27.85% at 3 years, p < 0.0001). LF incidence of intervention or AUR in placebo CO group with FT versus placebo CO group with oral medications was reduced (6.07% versus 33.3% at 3 years, p < 0.0001). 28/28 secondary efficacy endpoints were reached in LF CO re-injection studies.

FT 2.5 mg is a safe and effective transrectal injectable for LT treatment of BPH. FT treated patients also had reduced need for BPH intervention, and reduced incidence of PCa and AUR.

World journal of urology. 2018 Jan 29 [Epub ahead of print]

Neal Shore, Ronald Tutrone, Mitchell Efros, Mohamed Bidair, Barton Wachs, Susan Kalota, Sheldon Freedman, James Bailen, Richard Levin, Stephen Richardson, Jed Kaminetsky, Jeffrey Snyder, Barry Shepard, Kenneth Goldberg, Alan Hay, Steven Gange, Ivan Grunberger

Carolina Urologic Research Center, Myrtle Beach, SC, USA. ., Chesapeake Urology Research Associates, Baltimore, MD, USA., Accumed Research, Garden City, NY, USA., San Diego Clinical Trials, San Diego, CA, USA., Atlantic Urology Medical Group, Long Beach, CA, USA., Urological Associates of Southern Arizona, Tucson, AZ, USA., Freedman Urology, Las Vegas, NV, USA., First Urology, Louisville, KY, USA., Chesapeake Urology Research Associates, Towson, MD, USA., Jean Brown Research, Salt Lake City, UT, USA., University Urology, New York, NY, USA., Genitourinary Surgical Consultants, Denver, CO, USA., Urological Surgeons of Long Island, Garden City, NY, USA., U T Southwestern Dept of Urology, Lewisville, TX, USA., Willamette Urology, Salem, OR, USA., Summit Urology Group, Salt Lake City, UT, USA., Brooklyn Urology, Brooklyn, NY, USA.

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