Aquinox Pharmaceuticals (NASDAQ: AQXP) stock surges 1000% on positive Phase II results for AQX-1125 in patients with bladder pain syndrome, or interstitial cystitis (BPS/IC). The stock hit a 52-week high of $55.75 on Monday morning, which was 3,000% above Thursday’s lows.
Interestingly, the stock plunged 67.48% on July 09, just one month ago, when the company announced AQX-1125 failed to demonstrate efficacy in chronic obstructive pulmonary disease (COPD).
Aquinox’s lead product candidate, AQX-1125, is a SHIP1 activator. SHIP1 functions as a negative regulator of immune cells. SHIP1 activators have an anti-inflammatory effect. AQX-1125 is the only SHIP1 activator currently in clinical trials. Aquinox is investigating AQX-1125 in three Phase II trials, known as FLAGSHIP trial in COPD, LEADERSHIP trial in BPS/IC, and KINSHIP trial in atopic dermatitis.
As mentioned before, the FLAGSHIP trial has failed. The company has no further development plans for COPD. The ongoing KINSHIP trial is expected to read out 2015Q4. Let’s turn our attention to bladder pain. Actually, AQX-1125 missed the primary endpoint in the LEADERSHIP trial. AQX-1125 demonstrated a positive trend in reduction of pain compared to placebo, but the difference was not statistically significant (p=0.061).
The primary endpoint was based on an 11-point numeric rating scale (NRS). AQX-1125 demonstrated an average 2.4-point reduction in pain score, while placebo led to a mean 1.3-point reduction. Investors didn’t buy the disappointed top line data. The miss sent Aquinox shares down 7.74% on June 25.
On August 07, the company announced that the LEADERSHIP trial met multiple secondary endpoints. The additional positive results suggest that a longer or larger trial may have achieved the statistical goal. A loss of treatment effect was observed during an additional four-week follow up period, which validates the efficacy of AQX-1125.
BPS/IC is a chronic bladder disease characterized by pelvic pain and increased urinary frequency. The disease currently affects 5-12 million people in the U.S. There is no known cure for BPS/IC. The only oral therapy, Elmiron, was first approved in 1996. However, in two Phase IV trials, Elmiron showed no benefit over placebo.
Elmiron is priced at $625 per month, and generated annual sales of $279 million in 2014. The drug requires taking three times per day, while AQX-1125 is a once-daily therapy. Aquinox believes the opportunity for AQX-1125 could exceed $1 billion in the U.S. alone. Investors should be more cautious. IC/BPS is still very challenging to diagnose and treat due to a lack of specific biomarker and definitive diagnostic test.
Allergan (NYSE: AGN) acquired worldwide rights to TARIS Biomedical’s lead program, LiRIS, in August 2014. The company paid $67.5 million upfront and agreed to pay $520 million milestones. The program is now in Phase II trials (NCT02411110, NCT02395042).
By the way, Aquinox is a spin-off from the University of British Columbia. The company is backed by leading VCs including Ventures West, Baker Brothers, Pfizer Venture and J&J. Baker Brothers purchased 548,534 shares on July 09 and additional 2.5 million shares on August 07.