MSTX CEO blog
Dear Friends,
The second quarter of 2014 was a busy time for Mast. Most importantly, we focused on enrolling patients in our pivotal phase 3 study of MST-188 in sickle cell disease (SCD) and advanced many other initiatives to support MST-188 in sickle cell disease.
For example, we reported data from an ex vivo study further supporting MST-188 for SCD and announced the initiation of a sub-study within our phase 3 EPIC study to measure the effects of MST-188 on tissue oxygenation. I’m also pleased to share that the EPIC study’s Drug Safety and Monitoring Board (DSMB) held its first scheduled data review meeting and, based on data from the first cohort of subjects enrolled in EPIC, it recommended that the study proceed without any changes to the protocol. From progress like this, we continue to believe Mast is well-positioned to develop MST-188 as the first drug approved to treat an ongoing vaso-occlusive crisis in patients with sickle cell disease.
Many people are unaware that MST-188 is the most clinically-advanced new drug in development for SCD. But as such, we have the honor of leading a growing and important field, which unfortunately has historically suffered from too little awareness or investment. Remarkably, there has been only one FDA-approved drug for SCD and that approval came a whopping 16 years ago. Surely, patients deserve better. And clearly, the industry can do better. So, in addition to seeking approval for MST-188 in SCD, we support SCD community programs such as the Sickle Cell Disease Foundation of California’s Inspiring Dreams Walk-A-Thon and Camp Crescent Moon and the Foundation for Sickle Cell Research’s 8th Annual Sickle Cell Disease Research and Educational Symposium. Additionally, a free mobile app we developed for patients, VOICE Crisis Alert™, now has over 900 downloads.
We also created and again will host the 3rd Annual Sickle Cell Disease Therapeutics Conference in New York City on September 15th. This event serves as a forum to raise awareness for SCD among the investment community. This year, conference participants will hear presentations from members of the U.S. Food and Drug Administration, the Centers for Disease Control and the Sickle Cell Disease Association of America. In addition, clinical-stage companies (both public and private), leading physicians, patients, and healthcare analysts will discuss key issues and emerging trends in SCD.
With regard to our other pipeline programs, we’ve made significant progress with the development of MST-188 in acute limb ischemia (ALI), an indication for which we were granted orphan drug status by the FDA late last year. We initiated our phase 2 study of MST-188 in combination with rt-PA earlier this year. And, as planned, we submitted our application for orphan drug designation of MST-188 for the treatment of ALI in the European Union during the second quarter.
Furthermore, we recently initiated a nonclinical study of MST-188 in an experimental model of thrombotic stroke to evaluate MST-188’s potential to improve the therapeutic effect of rt-PA and expand the window in which it is effective. If MST-188 increases the therapeutic benefits of rt-PA in this nonclinical model and our phase 2 study in ALI demonstrates that MST-188 in combination with rt-PA accelerates time-to-reperfusion, we believe there will be interest from potential partners to develop MST-188 in stroke, a major market opportunity.
For the phase 2 asset we obtained via the acquisition of Aires Pharmaceuticals earlier this year, we recently locked the database for 20 patients who completed treatment in Aires’ phase 2 study in patients with pulmonary arterial hypertension. We anticipate announcing data from this trial in the third quarter of 2014. This data, along with ongoing discussions with experts in pulmonary hypertension and heart failure, will help us determine the optimal development strategy for AIR001, which we also expect to announce in Q3.
Finally, we announced last month the appointment of Howard C. Dittrich, M.D., F.A.C.C., to our Board of Directors. Dr. Dittrich, a cardiologist by training, has more than 20 years of experience in cardiac therapeutic research and clinical development. His medical training and extensive clinical development and regulatory affairs experience, particularly with cardiovascular agents, is highly relevant to our development programs and brings a wealth of development experience to our team.
Our 24 employees are proud of the progress we’ve made, and as always, I look forward to keeping you informed here and through our social media outlets, including Twitter (@MastThera) and Facebook.
