Urovant Sciences® Presents Interim Data from Phase 2a Study of Investigational Novel Gene Therapy, URO-902, Supporting Safety, Tolerability, and Efficacy Endpoints at 2022 American Urological Association Meeting

  • Interim 12-week analysis from an ongoing Phase 2a trial of an investigational novel gene therapy product (plasmid human cDNA encoding maxi-K channel) showed clinically relevant improvement in the common symptoms of overactive bladder (OAB) compared to placebo. (Plenary Presentation PLLBA-03)
  • In this prespecified 12-week analysis of a Phase 2a trial of women with OAB and urge urinary incontinence (UUI) who have failed oral therapies, a single dose of URO-902 24 mg or 48 mg was associated with clinically relevant improvement in efficacy and was well tolerated.
  • The most common adverse event (24 mg URO-902/48 mg URO-902/placebo) was urinary tract infection (0%/15.4%/3.8%, respectively).
Reno, Nevada (UroToday.com) -- Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., announced that positive interim results from a Phase 2a trial of its investigational novel gene therapy, URO-902, were presented Friday at the 2022 annual meeting of the American Urological Association (AUA2022). The meeting is being held in New Orleans, from May 13-16, 2022.


Presented as a late-breaker during the plenary session, the interim results support that in women with OAB, not well managed by oral therapies, a single dose of URO-902 was safe and well tolerated. This was a prespecified, 12-week interim analysis of a 48-week multicenter, randomized, double-blind, placebo-controlled, dose-escalation study (NCT04211831). URO-902 was administered via direct intradetrusor injections via cystoscopy under local anesthesia.

“These positive findings indicate that URO-902 has the potential to be a new therapeutic option for overactive bladder patients who have failed oral pharmacologic therapy,” said presenting author Kenneth Peters, MD, Principal Investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Women’s Urology and Pelvic Health Center; and Professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Mich.
Of the 80 female patients who were randomized, 68 completed week 12 of the study and 74 were included in the intent-to-treat population. The mean age was 64.7 years.

At week 12, both URO-902 24 mg and 48 mg were associated with clinically relevant improvement compared with placebo in mean daily micturition (urination), urgency episodes, UUI episodes, OAB questionnaire symptom bother score, and proportion of patient global impression of change responders. Treatment-emergent adverse events occurred in 45.5% of patients receiving URO-902 24 mg, 46.2% receiving 48 mg, and 50.0% receiving placebo. The most commonly occurring adverse event was urinary tract infection (0% in individuals receiving the 24 mg dose of URO-902; 15.4% in those receiving the 48 mg dose; and 3.8% in those receiving placebo). One patient in the 48 mg arm of the study had asymptomatic elevated post-void residual urine volume at week 2; this resolved spontaneously and did not require catheterization.

“We are encouraged by these promising interim safety and efficacy findings for URO-902,” said Sef Kurstjens, MD, PhD, Executive Vice President and Chief Medical Officer of Urovant Sciences. “This is in line with our ongoing efforts to develop innovative and effective treatments for patients in need.”

Source: "Urovant Sciences® Presents Interim Data From Phase 2A Study Of Investigational Novel Gene Therapy, URO-902, Supporting Safety, Tolerability, And Efficacy Endpoints At 2022 American Urological Association Meeting | Urovant Sciences". 2022. Urovant Sciences
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