$INSY In-Licenses Novel Mast Cell Therapy for Chronic Pelvic Pain

PHOENIX, AZ–(Marketwired – Feb 24, 2015) – Insys Therapeutics, Inc. (NASDAQ: INSY) has entered into an exclusive sub-license agreement with Gold Coast Therapeutics to develop a unique combination of Cromolyn Sodium and Cetirizine as a new chemical entity for the treatment of chronic prostatitis/chronic pelvic pain syndrome, or CP/CPPS.

Data from preclinical studies conducted at Northwestern University implicate mast cells as mediators of chronic pelvic pain associated with the prostate, and demonstrate that mast cell markers are elevated in men with CP/CPPS.

“Urologic chronic pelvic pain syndromes are devastating conditions that afflict as much as 10% of the population. In men, chronic pelvic pain is most often in the form of CP/CPPS, which has a major impact on quality of life with significant economic and healthcare burdens. Unfortunately, we have no effective therapies that we can offer our patients, so there is a tremendous unmet need for new and effective drugs to treat CP/CPPS,” said Anthony Schaeffer, MD, Chair, Department of Urology, and the Herman L. Kretschmer Professor of Urology at Northwestern University Feinberg School of Medicine. “We have recently pursued a mechanistic approach to develop novel CP/CPPS therapies by specifically targeting the underlying cells and pain signals that likely contribute to CP/CPPS pain.”

Design is underway for preclinical studies and clinical trials studies to be conducted at Northwestern University/Northwestern Medicine with Dr. Schaeffer assisted by David Klumpp, PhD, and Praveen Thumbikat, DVM, PhD.

“We have demonstrated a central role for mast cells and histamine as mediators of pelvic pain in clinically-relevant murine models. Genetic approaches established the mechanistic relevance, and pharmacologic experiments then confirmed the feasibility of targeting mast cells and histamine for therapies,” said Dr. Klumpp.

Addressing the relevance to potential CP/CPPS therapies, Dr. Thumbikat said, “Our peer reviewed preclinical data demonstrate impressive efficacy in reducing chronic pelvic pain using a co-formulation of Cromolyn Sodium and Cetirizine that shows unique synergism. The drug combination was developed based on mechanistic studies into the pathogenesis of CP/CPPS in humans and comparative animal models.”

“Based on promising preclinical data, we believe mast cell therapy holds great promise for the treatment of chronic pelvic pain, which dovetails with our core focus on the prevention and/or treatment of pain,” said Michael L. Babich, President and Chief Executive Officer of Insys. “This unique non-narcotic chemical entity complements our portfolio of in-market products and developmental-stage pharmaceutical cannabinoids and sublingual sprays. We look forward to further investigating the potential of Cromolyn Sodium in combination with Cetirizine in treating chronic pelvic pain.”

Northwestern University developed intellectual property around the usage of agents targeting mast cells as potential novel therapies for pelvic pain, which Gold Coast Therapeutics exclusively licensed from Northwestern University and has now exclusively sub-licensed to Insys.

The preclinical studies result from the strongly collaborative environment for urologic research at Northwestern Medicine, where clinicians and scientists with longstanding National Institutes of Health, or NIH, funding and research focus on pelvic pain, both in the clinic and in the laboratory. Drs. Klumpp and Schaeffer are co-directors of the Northwestern Integrated Pelvic Health Program within NIH’s flagship research initiative in pelvic pain, the Multi-Disciplinary Approaches to Chronic Pelvic Pain Research Network, or MAPP.

About Chronic Prostatitis/Chronic Pelvic Pain Syndrome
Chronic prostatitis/chronic pelvic pain syndrome, or CP/CPPS, is a debilitating condition diagnosed in the presence of chronic pelvic pain and lower urinary tract symptoms.¹ As prevalent as multiple sclerosis, CP/CPPS is the most common,² yet most poorly understood “prostatitis syndrome”. With a quality of life similar to that following a myocardial infarction, angina, or Crohn’s disease,³ CP/CPPS is truly a devastating disease. In the U.S., it is estimated that chronic prostatitis affects 1.8% of men⁴ and approximately 2 million men suffer from chronic prostatitis. Combined, these conditions account for 8% of urologist office visits and 15% of primary care physician office visits each year, with estimated annual charges of $9,184 per person.⁵

About Insys Therapeutics, Inc.
Insys Therapeutics is a specialty pharmaceutical company that develops and commercializes innovative drugs and novel drug delivery systems of therapeutic molecules that improve the quality of life of patients. Using our proprietary sublingual spray technology and our capability to develop pharmaceutical cannabinoids, Insys addresses the clinical shortcomings of existing commercial products. Insys currently markets two products, Subsys^®, which is sublingual Fentanyl spray for breakthrough cancer pain, and a generic version of Dronabinol (THC) capsules. Our lead product candidate is Dronabinol Oral Solution, a proprietary orally administered liquid formulation of dronabinol. Insys is also developing a pipeline of sublingual sprays, as well as pharmaceutical cannabidiol.

Subsys^® is a registered trademark of Insys Therapeutics, Inc.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the promise for treatment of chronic pelvic pain with mast cell therapy and the potential of Cromolyn Sodium in combination with Cetirizine in treating chronic pelvic pain, and the development of Dronabinol Oral Solution and Insys’ pipeline of sublingual sprays and pharmaceutical cannabinoids. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors, many of which are beyond our control. For a description of these risks facing the company, please see the risk factors described in our filings with the United States Securities and Exchange Commission, including those factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2013 and any subsequent updates that may occur in our Quarterly Reports on Form 10-Q. Forward-looking statements speak only as of the date of this press release and we undertake no obligation to update or revise these statements, except as may be required by law.

¹ Schaeffer AJ, Datta NS, et al. Overview summary statement. Diagnosis and management of chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) Urology. 2002; 60:1-4.

² Collins MM, Stafford RS, et al. How common is prostatitis? A national survey of physician visits. J Urol. 1998;159:1224-8.

³ McNaughton Collins M, Pontari MA, et al. Quality of life is impaired in men with chronic prostatitis: the Chronic Prostatitis Collaborative Research Network. J Gen Intern Med.2001;16:656-62.

⁴ RAND Interstitial Cystitis Epidemiology (RICE) study in 2013.

⁵Urol Dis Amer, 2009.