Emerging from the Company’s development efforts is BTL-hs, a Phase 2 clinical stage program. Disorders relating to muscle weakness targeted by this agent include urinary incontinence: stress, urge, and mixed. The initial target market for this platform will be stress urinary incontinence (UI).
Market Impact: The markets being targeted for treatment with BTL-hs have high levels of unmet need and a large accessible patient population. Nearly 63 million adults suffer from UI in the U.S. alone, with approximately 17 million seeking clinical treatment. Despite this high prevalence, due to the significant unmet need associated with existing therapies only about 3 million adults in the U.S. receive pharmacological treatment annually. Given the need for a safer, more effective therapeutic agent in this large patient population, peak year product revenues are projected to be $300 million worldwide in urge/mixed UI, with upside potential of $700 million worldwide in stress incontinence. The current worldwide prescription market for UI and over-active bladder is $3 billion.
Safety: In two formal clinical trials to date, no serious adverse events were reported.
Efficacy: A pilot clinical study showed a 70 percent improvement in incontinence symptoms in 8 of 9 women with mixed and stress urinary incontinence. Patients were treated for four weeks with sublingual drops of the test product or placebo. Results showed all test parameters to be in favor of the treatment population, with the most significant (p=0.039) reflecting a decrease in the use of pads for leakage.
Mechanism of Action: In vitro skeletal muscle studies have shown significant increases in force generation after exposure to BTL-hs compared to untreated controls. The proposed mechanism of action of BTL-hs has been corroborated using in vitro microarrays indicating that the regulation of various genes in the G-protein coupled receptor signaling pathway are linked to improved smooth muscle contraction. Results from a 2012 preclinical animal rat study of BTL-hs at the Cleveland Clinic Urology Center point toward a potential breakthrough in treating stress incontinence as well as illuminating a likely mechanism of action.
Intellectual Property: U.S. and foreign patents have been issued.
There are currently no approved pharmacological agents for stress UI in the U.S., and treatments for urge/mixed UI leave significant room for a product of improved efficacy and tolerability. The Company’s evaluation of BTL-hs for muscular dystrophy represents another application of this formulation for muscle-related disorders.