Mast Therapeutics (MSTX) Reports Vepoloxamer Phase 3 in Sickle Cell Missed Primary Endpoint

September 20, 2016 5:02 PM EDT

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Mast Therapeutics, Inc. (NYSE: MSTX) reported top-line results from EPIC, a Phase 3 clinical study of its investigational new drug vepoloxamer (also known as MST-188) for the treatment of individuals with sickle cell disease experiencing vaso-occlusive crisis (VOC). The study did not meet its primary efficacy endpoint of demonstrating a statistically significant reduction in the mean duration of VOC (82 hours in the vepoloxamer group compared to 78 hours in the placebo group in the intent-to-treat population (p=0.09)). There were no statistically significant differences between treatment groups in the intent-to-treat population across the two secondary efficacy endpoints, rate of re-hospitalization for VOC and the occurrence of acute chest syndrome. Consistent with previously conducted studies, vepoloxamer was generally well tolerated with no statistically significant differences in treatment-related serious adverse events in the vepoloxamer group compared to the placebo group. No deaths occurred on the study.

"We are exceedingly disappointed with these top-line results. While clearly not the outcome we wanted, we believe the insights and data from the largest placebo-controlled clinical trial ever completed in sickle cell disease will substantially advance the understanding of vaso-occlusive crisis and the still maturing clinical science necessary to support the development of new therapeutics for this debilitating disease," stated Brian M. Culley, the Company's Chief Executive Officer. "We wish to reiterate our sincere appreciation for all of the patients, caregivers, and others who aided us in conducting this informative study."

"These analyses are limited to just top-line data, so in the coming weeks the Company intends to review the full data set from EPIC. In addition, we plan to perform an interim analysis of the ongoing heart failure trial of vepoloxamer. However, based on the data we've seen to date, we expect we will terminate all clinical development of vepoloxamer. Consequently, while we evaluate our options, we intend to significantly and immediately reduce our operating expenses and continue our efforts with AIR001, our lead asset in heart failure with preserved ejection fraction, which currently is the subject of a 100-patient phase 2 study expected to complete enrollment by the end of 2017," continued Mr. Culley.

Investor Conference Call The Company will hold a conference call tomorrow, September 21, 2016, at 8:00 a.m. ET / 5:00 a.m. PT to discuss the Phase 3 results. Interested parties may access the conference call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from outside the U.S. and should request the Mast Therapeutics, Inc. Call. A live webcast of the conference call will be available online from the Investors section of Mast's website at http://www.masttherapeutics.com/investors/events/. Replays of the webcast will be available on the Company's website for 30 days and a telephone replay will be available through September 28, 2016 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada, and (412) 317-0088 from elsewhere outside the U.S. and entering replay access code 10093117.



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