Mast Therapeutics will announce Phase III results for their Orphan Sickle Cell Disease Drug MST-188 in Q2-2016.
We strongly believe that MST-188 Phase III will be a success.
SCD patients have been waiting for a new treatment the past 17 years. FDA might want to bring a new drug on the markets to sustain the patient lives.
Vepoloxamer compound might work as well for the heart failure. This is the major secondary catalyst for the share price.
Mast Therapeutics (mstx) has a market cap of little over $50m. That is a very low valuation for a company that is shortly going to announce their Phase III results for the Sickle Cell Disease. Their drug vepoloxamer (MST-188) holds an Orphan status in the United States and Europe.
In our opinion the investor community has completely missed the story of MST-188 and the information that is available around the EPIC trials.
EPIC Trial: A High Chance For A Regulatory Approval
The fact that FDA allowed EPIC-E launch is a very positive sign. It signals that there are no major drug safety concerns in the air. Now also the study placebo participants will be receiving vepoloxamer during their subsequent crises in an unblinded manner.
The eventual MST-188 regulatory approval is very likely because:
A…..MST-188 is the only drug for SCD in an advanced development phase. The competition is about 2 years behind.
B…..This orphan drug would be the first proper treatment for the SCD patients in 17 years.
C…..We have not seen any alarming secondary indications. The recent approved launch of EPIC-E is giving even more confidence of the drug safety profile.
D…...The duration of vaso-occlusive crises, hospitalisation time and intake of analgesics were all reduced with the prior sponsor's purified poloxamer 188 drug.
Using Past Purified Poloxamer 188 Data for EPIC Design
Vepoloxamer is a purified version of poloxamer 188 that did undergo Phase III study several years ago with an enrollment of 73%. We discuss here some facts that
"Heart failure with preserved ejection fraction is a major public health problem that has no proven effective treatment, yet currently afflicts 2 to 3 million Americans," stated Dr. Borlaug. "One factor that complicates treatment is that the hemodynamic perturbations causing morbidity such as high filling pressures and low cardiac output are typically present only intermittently—being absent at rest but observed during stress such as exercise. As such, an ideal therapy would become more effective during stress, without untoward effects on resting cardiovascular function. The results observed with AIR001 in this study support our hypothesis that acute administration of nebulized inhaled sodium nitrite unloads the heart during exercise without excessive reduction in resting pressures or arterial blood pressure."
"These results are an important step in validating our second asset and establishing the potential clinical utility of AIR001 in HFpEF," stated Brian M. Culley, Chief Executive Officer of Mast Therapeutics. "Mayo Clinic is a well-known leader in the characterization and treatment of heart failure and we thank Dr. Borlaug for working with us and leading this study," continued Mr. Culley. "We look forward to advancing AIR001 in this area of high unmet medical need for which there is no FDA-approved therapy available. We also anticipate reporting interim data from a second investigator-sponsored Phase 2a study of AIR001 in HFpEF patients around the middle of this year