OREANDA-NEWS. Aquinox Pharmaceuticals, Inc. (“Aquinox”) (NASDAQ:AQXP), a clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology, today provided a corporate update and reported financial results for the first quarter ending March 31, 2016.

“We have now submitted our first planned Phase 3 clinical trial protocol to the FDA and continue to anticipate initiating enrollment in the third quarter of this year,” said David Main, President & CEO of Aquinox. “Our facilities in San Bruno, CA are now fully operational and we continue to add key personnel for our development and planned commercial activities. We are proceeding on budget, confident that our cash will carry us through topline data from our first planned Phase 3 trial.” 

Business Highlights

Submission of our First Phase 3 Clinical Trial Protocol to the FDA. Aquinox has submitted to the FDA its protocol for the planned Phase 3 trial with AQX-1125 for the treatment of pain and urinary symptoms in patients suffering from interstitial cystitis/bladder pain syndrome (IC/BPS).

Meeting and Recommendations from the European Medicines Agency (EMA). Aquinox met with the EMA in March 2016 to discuss plans for development of AQX-1125 in the treatment of patients with IC/BPS and has since received feedback from the EMA including recommendations regarding proposed Phase 3 trial design. The recommendations received from the EMA are generally consistent with minutes received from the FDA following our meeting in December 2015. Similar to the U.S., we are preparing to submit our protocol and supporting documents with a number of health authorities in Europe to conduct our first Phase 3 clinical trial.

Presentation of LEADERSHIP 201 Data at EAU and AUA. Results from the LEADERSHIP 201 trial were recently presented at the European Association of Urology Conference in Munich on March 11-15, 2016 and in moderated poster sessions at the American Association of Urology meeting held in San Diego, CA from May 6-10, 2016.

LEADERSHIP 201 Results Accepted for Publication in the Journal of Urology. The results from the LEADERSHIP 201 trial have been accepted for publication in an upcoming issue of the peer-reviewed Journal of Urology. Lead authored by Dr. Curtis Nickel, the publication is entitled “A Phase II Study of Efficacy and Safety of a Novel, Oral SHIP1 Activator, AQX-1125, in Subjects with Moderate to Severe Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)” and is now available for viewing online.

New Facilities in San Bruno, CA and Expansion of Vancouver, BC Offices. To accommodate continued growth and increasing operational, regulatory and commercial development activities, Aquinox has opened new offices in San Bruno, CA and will be expanding its existing offices in Vancouver, BC.

Summary of Financial Results

Cash Position. Cash, cash equivalents, short-term and long-term investments totaled $107.3 million as of March 31, 2016, compared to $112.9 million as of December 31, 2015. This decrease was primarily driven by the ongoing advancement of AQX-1125 towards initiation of our first Phase 3 clinical trial. Aquinox expects its cash, cash equivalents, short-term and long-term investments to be sufficient to complete the first planned Phase 3 clinical trial in IC/BPS with AQX-1125 as well as supportive activities related to manufacturing, toxicology and additional clinical development.

R&D Expenses. Research and development expenses for the first quarter of 2016 increased to $4.9 million from $4.6 million in the first quarter of 2015. This increase was primarily driven by increased expenditures on clinical activities as we progress towards the initiation of our Phase 3 clinical trial of AQX-1125 in IC/BPS.

G&A Expenses. General and administrative expenses for the first quarter of 2016 increased to $2.0 million from $1.5 million in the first quarter 2015. This increase was primarily driven by higher personnel related costs and the establishment of an office in San Bruno, CA

Net Loss. Net loss for the first quarter of 2016 was $6.7 million compared to a net loss of $6.4 million for the first quarter of 2015. This increase was primarily driven by increased operating expenditures offset by a reduction in the foreign exchange losses.

About Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)

IC/BPS is a chronic inflammatory bladder disease characterized by pelvic pain and increased urinary urgency and/or frequency. For many sufferers, these symptoms are severe and adversely affect all major aspects of their lives, including overall physical and emotional health, employment, social and intimate relationships, and leisure activities. While the cause of the disease remains largely unknown, erosion of the bladder lining is thought to be a significant contributor. IC/BPS is estimated to affect between 5 and 12 million people in the United States. Most IC/BPS patients continue to suffer this debilitating condition, despite treatment with existing therapies. Most current therapies and those in development are focused solely on symptomatic relief of IC/BPS. Aquinox believes new and innovative therapies that target the underlying disease in order to reduce the chronic pain and urinary symptoms are needed.

About the LEADERSHIP 201 Trial

The LEADERSHIP 201 trial was a multicenter, randomized, double-blind, placebo-controlled, Phase 2 clinical trial investigating the ability of 200 mg oral, once daily AQX-1125 to reduce pain in female patients with IC/BPS. The primary endpoint was to measure the difference in the change from baseline in the average daily bladder pain score based on an 11-point numeric rating scale (NRS) at six weeks recorded by electronic diary. Results demonstrated a positive trend in the primary endpoint and statistically significant changes on secondary endpoints including both pain and urinary symptoms. A total of 69 subjects were enrolled. 

About AQX-1125

AQX-1125, Aquinox's lead drug candidate, is a small molecule activator of SHIP1, which is a regulating component of the PI3K cellular signalling pathway. By increasing SHIP1 activity, AQX-1125 accelerates a natural mechanism that has evolved to maintain homeostasis of the immune system and reduce immune cell activation and migration to sites of inflammation. AQX-1125 has demonstrated preliminary safety and favorable drug properties for once daily oral administration in multiple preclinical studies and seven completed clinical trials. Aquinox completed a successful Phase 2 clinical trial with AQX-1125 for the treatment of IC/BPS in 2015 and anticipates initiation of a Phase 3 trial with AQX-1125 in IC/BPS in the third quarter of 2016.

About Aquinox Pharmaceuticals, Inc.

Aquinox Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology. Aquinox's lead drug candidate, AQX-1125, is a small molecule activator of SHIP1 suitable for oral, once daily dosing. With a successful Phase 2 clinical trial completed in 2015, Aquinox plans to initiate a Phase 3 trial in 2016 with AQX-1125 for treatment of IC/BPS. Aquinox has a broad intellectual property portfolio and pipeline of preclinical drug candidates that activate SHIP1.

AQUINOX PHARMACEUTICALS, INC.
 
Condensed consolidated balance sheets
(Unaudited)
(In thousands of U.S. dollars)
 
  MARCH 31,
2016		   DECEMBER 31,
2015
       
Assets      
Cash, cash equivalents, short-term and long-term investments $   107,338     $   112,940  
Other current assets   772       314  
Other long-term assets   104       89  
Total assets $   108,214     $   113,343  
       
Liabilities      
Current liabilities $   5,723     $   4,792  
Non-current liabilities   106       131  
Total liabilities $   5,829     $   4,923  
Stockholders’ equity     102,385         108,420  
Total liabilities and stockholders' equity $     108,214     $     113,343  
AQUINOX PHARMACEUTICALS, INC.  
   
Condensed consolidated statements of operations  
(Unaudited)  
(In thousands of U.S. dollars, except per share and share amounts)  
   
  THREE MONTHS ENDED
MARCH 31,		  
    2016       2015    
Operating expenses        
Research and development $   4,881     $   4,567    
General and administrative   1,953       1,472    
Total operating expenses   6,834       6,039    
Other income (expenses)   123       (373 )  
Net loss before income taxes   (6,711 )     (6,412 )  
Net loss $ (6,711 )   $ (6,412 )  
                 
Net loss per common stock - basic and diluted $    (0.39 )   $     (0.60 )  
Basic and diluted weighted average common stock outstanding   17,211,986       10,711,967