2018 Annual Report. Next
I’m proud, and very pleased, to report that MEI achieved significant progress over the course of the 2018 fiscal year across all four of our clinical programs, as well as with operational and financial initiatives. We start the new year in our strongest position ever:
- All four of our oncology drug candidates have yielded data supporting ongoing clinical development,
- By calendar year-end 2018, two of these candidates will be in global studies that could support marketing authorization; and
- Our fiscal year started with nearly $103 million in cash, cash equivalents and short-term investments in our accounts.
Our success over the past year makes us well positioned to take the next steps, continue to optimally build value to advance our candidates towards marketing approval, and to prepare for commercialization, either independently or via partnership.
A large part of our success has been achieved by the focused and efficient deployment of resources to create a return on our investments, and a straight forward, purposeful strategy to build value. Specifically, our portfolio is built around drug candidates with validated targets and is designed to develop medicines that are: (1) differentiated, (2) address unmet medical needs and (3) deliver improved benefit to patients either as standalone treatments or in combination with other medicines.
This approach has guided the identification and development of the four clinical-stage drug candidates in our pipeline, each of which utilizes a different mechanism of action. Achievements from each of these programs are notable:
Last August, the first patient was dosed in the Phase 3 registration trial evaluating patients with acute myeloid leukemia who are unfit to receive intensive chemotherapy. In January we received Orphan Drug Designation from the European Medicines Agency, adding to the Breakthrough Therapy Designation granted by the FDA in 2016. In May we jointly announced with our partner Helsinn that an interim analysis of our Phase 2 study in patients with MDS achieved a predefined discontinuation threshold. Based on that successful planned interim analysis the study expanded open-label enrollment to 60 patients. We look forward to updating you on the further developments of this study later in the year.
ME-401, our PI3K inhibitor, continues to exceed our high expectations. Results from our Phase 1b study in relapsed/refractory follicular lymphoma and chronic lymphocytic leukemia were presented at the 2018 ASCO meeting and demonstrated that ME-401 administered as a single-agent achieved a high overall response rate of 90% among all 30 evaluable patients and 86% response rate in the 21 patients with follicular lymphoma – this compares to response rates starting at slightly more than 40% and reaching into the high 50% range for approved therapies in a similar patient population. Armed with these data and informed by FDA discussions, we have designed a Phase 2 clinical trial to support accelerated approval of a marketing application with the FDA. The study will be a global Phase 2 study investigating the efficacy, safety, and tolerability of ME-401 in patients with follicular lymphoma after failure of at least two prior systemic therapies. The study will evaluate two different ME-401 single agent dosing regimens: a daily continuous schedule and an intermittent schedule.
ME-401 represents an opportunity to develop a best-in-class PI3K delta inhibitor, including potential to develop ME-401 in other lines of therapy and additional potential to utilize ME-401 in combination with other modalities to obtain further benefits with applications across additional indications.
To optimize the value MEI can realize from the ME-401 opportunity, we see independent commercialization of ME-401 in the US as an attractive option. We are therefore taking the next step to achieve that value and have begun making an investment in the planning and implementation of early-stage commercialization activities. In parallel, we will consider potential partnering opportunities for ME-401 in markets outside the U.S.
Voruciclib is an orally available CDK inhibitor differentiated by potent inhibition of CDK9 in addition to CDK6, 4 and 1. CDK9 is a valuable target because it regulates the myeloid leukemia cell differentiation protein, or MCL1, a member of the family of anti-apoptotic proteins which, when elevated, may prevent cells from undergoing cell death. This is important because there’s a growing acknowledgement in the medical community that MCL1 upregulation is a resistance mechanism to the BCL-2 inhibitor venetoclax, an FDA approved medicine indicated for the treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma.
We have initiated a Phase 1b dose ranging study evaluating voruciclib in patients with B-cell malignancies. After obtaining initial dosing information from this study, we plan to evaluate voruciclib in combination with venetoclax to confirm synergies observed in the preclinical data and to assess opportunities for combination treatment across multiple indications with BCL-2 inhibitors.
ME-344 is our mitochondrial inhibitor -- a novel drug candidate that we and our investigators continue to be excited by its prospects. A randomized clinical proof of concept study is being conducted in Madrid evaluating ME-344 in combination with Avastin in women recently diagnosed with HER2 negative breast cancer. Interim data was announced earlier this year suggesting that inhibition of tumor proliferation with Avastin plus ME-344 may be greater than that observed with Avastin alone. Final results are expected in the first half of 2019.
As to our corporate and operational achievements, most notable was our $75 million private placement which included investments from multiple leading healthcare investment firms. We were also very happy to add industry veteran Fred Driscoll to our Board of Directors, and to promote David Urso to Chief Operating Officer.
In summary, we are very encouraged with our progress over the last year; we have a diverse clinical-stage pipeline, the prospect of having two candidates in global studies to support marketing authorization by year-end, and a very healthy balance sheet. We are in a strong position to continue to take the next steps to build value for our shareholders as we work diligently to advance our clinical candidates towards regulatory approval to provide new benefits to cancer patients.
I’d like to thank the many patients involved in our trials, our investigators in our programs, and of course our employees for their relentless efforts to advance these innovative new drug candidates towards becoming approved medicines that can provide new benefits to patients with cancer. I’d also like to thank our shareholders for their tremendous support. I look forward to reporting on progress in the years to come.