Targacept Announces Initiation of Phase 2b Study of TC-5214 in Overactive Bladder
WINSTON-SALEM, N.C.--May 29, 2013-- Targacept, Inc. (NAS: TRGT) , a clinical-stage biopharmaceutical company developing novel NNR Therapeutics ™, today announced the initiation of a Phase 2b clinical study of TC-5214 as a treatment for overactive bladder (OAB). TC-5214 acts potently on alpha3beta4 and other neuronal nicotinic receptors (NNRs) located in or around the bladder that are believed to play a key role in bladder contraction and signaling of the urge to urinate. Because orally administered TC-5214 is eliminated more than 90% unchanged through the bladder, Targacept believes the compound offers potential for a low dose to be beneficial while minimizing systemic side effects characteristic of current therapies and enhancing patient compliance.
"Overactive bladder has a devastating effect on quality of life for millions of individuals, and currently available medications have significant drawbacks," said Eric Rovner, M.D., Professor of Urology, Medical University of South Carolina, College of Medicine, and an investigator in Targacept's study. "Based on the novel mechanism and favorable tolerability profile associated with TC-5214, I am encouraged by the potential for this clinical study to support what could be a truly novel therapy for patients who suffer from OAB."
TC-5214 was associated with exaggerated bladder effects in preclinical studies, including increased urinary retention and beneficial changes in bladder contraction, bladder capacity and frequency of urination. In addition, TC-5214 already has a substantial safety database resulting from prior clinical studies in approximately 2,400 subjects in a different indication.
"With a strong scientific rationale, supportive findings, measurable clinical endpoints and a well-defined regulatory path, we view overactive bladder as a promising indication for development of this advanced compound," said Dr. Stephen A. Hill, Targacept's President and Chief Executive Officer. "As NNR science continues to evolve, we believe the potential for TC-5214 in OAB serves as a compelling opportunity to expand our pipeline beyond central nervous system diseases in support of our goal to bring important new therapies to patients."
About the Phase 2b Study in Patients with OAB
The Phase 2b study is a double blind, placebo controlled, randomized, parallel group trial planned to be conducted at sites in the United States. The study's co-primary endpoints are change in micturition frequency per 24 hours and change in urinary incontinence episodes per 24 hours, in each case from baseline to 12 weeks. The study, which is designed to enroll approximately 750 patients, includes a 3- to 5-week screening period followed by a 12-week treatment period during which patients receive either one of three doses of TC-5214 (0.5mg, 1mg or 2mg) or placebo twice daily, randomized in a ratio of 2:1:1:1 (placebo, low dose, mid dose, high dose).
About Overactive Bladder
Overactive bladder is a disorder that causes a sudden and frequent urge to urinate that may be difficult to suppress and may lead to incontinence and nocturia. OAB poses a significant reduction in quality of life due to a decreased ability to socialize and participate in normal life activities, sleep disturbances and decreased emotional well-being. It is estimated that approximately 1 in 6 adults in the United States suffer from OAB1.
Targacept is developing a diverse pipeline of innovative NNR Therapeutics™ for difficult-to-treat diseases and disorders of the nervous system. NNR Therapeutics selectively modulate the activity of specific neuronal nicotinic receptors, unique proteins that regulate vital biological functions that are impaired in various disease states. Targacept's clinical pipeline includes multiple Phase 2 product candidates, all representing first-in-class opportunities. Targacept leverages its scientific leadership and diverse pipeline to attract significant collaborations with global pharmaceutical companies. For more information, please visit www.targacept.com.
Building Health, Restoring Independence®
This press release includes "forward-looking statements" made under the provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding without limitation: the location of clinical sites or the number of subjects for the ongoing clinical trial of TC-5214 in OAB; the medical benefits or tolerability of, or patient compliance with, a low dose of TC-5214; the competitive position of TC-5214 or the commercial opportunity in OAB; or Targacept's plans, expectations or future operations, financial position, revenues, costs or expenses. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various important factors, including without limitation risks and uncertainties relating to: the conduct and results of the ongoing clinical trial of TC-5214 in OAB and any future clinical trials and non-clinical studies and assessments of TC-5214; whether findings from nonclinical studies and assessments of TC-5214 and clinical trials of TC-5214 in a different indication will be predictive of a positive outcome in the ongoing clinical trial of TC-5214 in OAB; whether TC-5214 will be eligible for treatment in the United States as a new chemical entity with a five-year statutory exclusivity period, either because Targacept submits a new drug application for TC-5214 prior to October 1, 2017 or because the applicable statutory provision is reauthorized by the U.S. Congress; Targacept's ability to protect its intellectual property with respect to TC-5214; and the timing and success of submission, acceptance and approval of regulatory filings with respect to TC-5214. Risks and uncertainties that Targacept faces are described in greater detail under the heading "Risk Factors" in Targacept's most recent Annual Report on Form 10-K and in other filings that it makes with the Securities and Exchange Commission. As a result of the risks and uncertainties, the results or events indicated by the forward-looking statements may not occur. Targacept cautions you not to place undue reliance on any forward-looking statement.
In addition, any forward-looking statement in this press release represents Targacept's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Targacept disclaims any obligation to update any forward-looking statement, except as required by applicable law.
NNR Therapeutics™ and Building Health, Restoring Independence® are trademarks or service marks of Targacept, Inc. Any other service marks, trademarks and trade names appearing in this press release are the properties of their respective owners.
1Stewart WF, Van Rooyen JB, Cundiff GW, Abrams P, Herzog AR, Corey R, et al. Prevalence and burden of overactive bladder in the United States. World J Urol. May 2003; 20(6):327-36.