RE: Shareholder update NEW21 Jan 2024 19:27
Dear Shareholders,
As we embark on a new year, I am pleased to provide this summary to you. In it you will find highlights
from 2023 and our plans for Q1, 2024.
Clinical Trial Update
The phase 1 trial being conducted at the Medical College of Wisconsin (MCW) continues to warrant our
full attention. Patients are enrolling at the 2000 mg dose level and are experiencing no significant side
effects. The goal of phase 1 is still unmet in that the maximum tolerated dose (MTD) has yet to be
determined. The clinical team is reviewing all data and discussing what, if any, criteria could be used in
lieu of MTD to define the recommended phase 2 dose (RP2D). This would then result in phase 1 being
closed and allow the preparation for a phase 2 trial to hit full stride.
While the phase 1 trial maintains momentum, significant regulatory milestones have been achieved that
formally validate the promising potential of GaM (as well as remind us of the lack of viable treatment
alternatives for brain tumor patients). In late December, the FDA designated the development of oral GaM
for the “treatment of adult patients with relapsed or refractory glioblastoma, IDH-wildtype” as Fast Track.
The Fast Track Designation (FTD) process facilitates the development of new drugs that treat serious
conditions and fill unmet clinical needs. Going forward, we intend to fully leverage the increased
communication and collaboration with the US FDA afforded by this process.
Along with the two Orphan Drug Designations (ODD) received last year, these regulatory
accomplishments have boosted the enthusiasm of our internal team and that of the leadership at the
Medical College of Wisconsin (MCW). Motivated by these accomplishments, we are launching an
Expanded Access Program (EAP). The benefits of an EAP are multi-faceted, and I will touch upon a few
significant ones here. First, the agent itself is very stable and does not require special storage or
handling. Thus, an oral agent that can be taken at home is an ideal candidate for executing an EAP.
Second, the EAP would make the agent accessible to patients throughout the U.S. and allow patients to
obtain GaM who otherwise could not participate, due to proximity to MCW or the inclusion/exclusion
criteria of the phase 1, etc. Based on the data obtained from phase 1, albeit limited, we believe the agent
has potential benefit for these patients. Third, additional data from the EAP would be obtained faster than
the traditional phased trial pathway and would be considered “real-world-data” (RWD). RWD is
increasingly valued as it is a truer representation of the normal population of patients. RWD can also
identify critical information, such as biomarkers, that can significantly influence the size, scope, and cost
of subsequent phases. This data could then be used in a New Drug Application (NDA) to the FDA. In fact,
there have been agents approved by the FDA that relied solely on EAP data, and even more agen