September 26, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN DIEGO – Following the failure of its sickle cell drug vepoloxamer, California-based Mast Therapeutics announced this morning that it was pulling the plug on the program and instead focusing on its other experimental drug, AIR001, for the treatment of heart failure with preserved ejection fraction (HFpEF).
Vepoloxamer did not meet its primary efficacy endpoint of demonstrating a statistically significant reduction in the mean duration of vaso-occlusive crisis (VOC). Mast said 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.” Vaso-occlusions can occur everywhere blood flows and lead to an accumulating disease burden in every organ system with the ultimate loss of vital organ function and significantly reduced lifespan. Vaso-occlusive crisis is characterized by intense and debilitating pain, which can last for days and even weeks.Mast said it is in the process of analyzing additional data from the Phase III clinical study of vepoloxamer in sickle cell disease and a Phase II study in heart failure to “assess opportunities to create value from this asset.”
One vepoloxamer study will continue, the company said. The study is exploring the use of vepoloxamer combined with tissue plasminogen activator in experimental models of embolic stroke. That study is being supported through a Small Business Innovation Research grant from the National Institute Of Neurological Disorders And Stroke of the National Institute of Health.
Although the company did not specifically say it was terminating employees associated with the sickle cell drug program, in a statement, Brian Culley, Mast’s chief executive officer, said the decision to terminate the program “likely will have a significant impact on our talented team members.”
The failure of the drug was predicted by Martin Shkreli, the former head of Turing Pharmaceuticals who faces seven counts of securities fraud. A former hedge fund manager, Shkreli made his prediction that the drug would fail on Benzinga’s Premarket Prep.
“This is a company that’s probably, I would say, worthless, that’s gonna drop 90 percent or more in the next few weeks,” Shkreli said according to the Benzinga write-up.
After the company announced vepoloxamer failed to meet endpoints, the stock cratered more than 80 percent. This morning shares of Mast are trading at 13 cents, up two cents from Friday’s close. Before the crash, shares of Mast were trading at 59 cents.
Mast is hoping its heart failure drug will be the rock upon which it can build a solid business foundation. Mast said it will continue to support three ongoing Phase II clinical studies of AIR001 through 2017. As it focuses on AIR001, Mast said it is looking for potential partnership opportunities for its assets. AIR001 is a sodium nitrite solution for intermittent inhalation via nebulization. Earlier this year, the Company reported positive top-line results from a blinded and randomized Phase IIa clinical study of AIR001 in HFpEF patients.
Mast said it currently has cash, cash equivalents and investment securities of $30.3 million as of Aug. 31—however that was before the failure of vepoloxamer. Following anticipated cost savings from the termination of its sickle cell drug, Mast anticipates operating expenses of $9 to $10 million for 2017.
“This anticipated level of spend reflects an approximate 70 percent reduction from estimated operating expenses for 2016 of approximately $32 to $34 million, excluding share-based compensation expense, the company said in a statement.
