All of our efforts over the past several years combined to set the stage for what proved to be an eventful and rewarding 2016. It was a year of significant progress on all fronts – clinical, regulatory and business development – that culminated in two firsts for the company: the receipt of Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and a global strategic partnership for Pracinostat.

Our core strength is in oncology drug development and we aim to leverage our resources to continue to advance new treatments for patients. Daniel Gold, Ph.D., President & Chief Executive Officer

With the Breakthrough Therapy Designation, the FDA recognizes that our preliminary clinical data demonstrate that the combination of Pracinostat and azacitidine may result in a substantial improvement in the lives of certain patients with acute myeloid leukemia (AML) over available therapy. Effective new therapies for AML patients unfit to receive intensive chemotherapy is a clear unmet medical need and we are excited that the FDA sees the promise of Pracinostat to address this need.

Our exclusive licensing, development and commercialization agreement with Helsinn Healthcare SA, worth up to $464 million, represented a turning point for our Company. We are proud to have a strong commercial partner in Helsinn now on board. With this partnership in place, we are well positioned to move forward with a fully funded Phase III study in AML, evaluate an optimized dosing regimen in myelodysplastic syndrome (MDS) and retain attractive economics on the future commercial success of Pracinostat.

In the process, we have significantly strengthened our financial position, enabling us to exploit our core strength in oncology drug development. We aim to leverage these resources in the coming year to continue to advance new treatment options for patients.

One of our most anticipated milestones in the coming year will be data from the Phase Ib dose-escalation study of our PI3K delta inhibitor, ME-401, in patients with recurrent chronic lymphocytic leukemia or follicular non-Hodgkin’s lymphoma. PI3K delta is a class of drugs that has shown promise in the treatment of B-cell malignancies, but with certain toxicities. We believe this provides an opportunity for a new oral drug that can produce therapeutic responses at a safe, effective dose.

Results from our first-in-human study of ME-401 in healthy volunteers suggest that it has the potential for an improved therapeutic window compared to the approved PI3K delta idelalisib (marketed as Zydelig®). The goal of the Phase Ib study is to demonstrate this therapeutic window with repeated dosing in cancer patients. Interim data is expected by the middle of 2017.

We also look forward to seeing data from an investigator-sponsored study of our mitochondrial inhibitor drug candidate, ME-344, in combination with the VEGF inhibitor bevacizumab (marketed as Avastin®) in patients with HER2-negative breast cancer. Data from the study, which is being conducted in collaboration with the Spanish National Cancer Research Centre in Madrid, are expected by the end of 2017.

Before I close, I would like to take this opportunity to thank our employees, our clinical investigators and our shareholders for their continued support. A number of you have been steadfast supporters as we have learned firsthand about the characteristics of Pracinostat and its performance in defined patient populations. I would also like to thank Helsinn for bringing a shared passion and commitment to the Pracinostat program. We are excited to work together to bring this promising drug to patients in need.

Sincerely,

Daniel P. Gold, Ph.D.

Daniel P. Gold, Ph.D.