Clinical Data
The primary endpoint of the study was to determine the dosage regimen to be used in a proposed Phase II study of the
safety and efficacy of MTX110 in patients with DIPG. Preliminary high‐level data from the UCSF study supports a dose of
between 60μM and 90μM of MTX110, depending upon patient tolerance over the course of 12 infusions in Phase II.In total, seven patients were recruited into the UCSF study. Patients were newly diagnosed with DIPG and received focal
external beam radiation therapy four to 14 weeks before commencement of MTX110 treatment. Eligibility required a
pontine location of the tumour with diffuse involvement of at least two thirds of the pons and no evidence of metastatic
disease. Patients were not excluded by total tumour volume. MTX110 was administered directly into the tumour via a
micro‐catheter using convection enhanced delivery ("CED") with gadolinium‐enhanced intra‐operative MRI to guide and
track drug distribution to the tumour. Patients could receive up to 12 cycles of treatment every four to eight weeks. The
dose was escalated between and within patients as tolerated initially by increasing the infusion volume at a concentration
of 30μM MTX110 and then with higher drug concentrations of 60μM and 90μM as the sixth and seventh dose increments,
respectively.At the interim cut‐off date (30 September 2020), median overall survival based on Kaplan Meier analysis was 26.06 months
(CI 11.3 ‐ 26.06 months) and overall survival at 12 months (OS12) was 71.4% (five of seven patients alive). Three patients
remain alive and continue to be monitored. Survival was not an endpoint of the UCSF study nor was the study powered for
statistical significance and therefore no conclusions as to the impact of MTX110 on overall survival rates can be drawn from
these data.