(Sharecast News) - Biotechnology company Midatech Pharma announced "encouraging" headline results from a phase 1 study at the University of California, San Francisco (UCSF) in patients with diffuse intrinsic pontine glioma (DIPG) on Monday.
The AIM-traded firm said the primary endpoint of the study was to determine the dosage regimen to be used in a proposed phase 2 study of the safety and efficacy of 'MTX110' in patients with DIPG.
Preliminary high-level data from the UCSF study supported a dose of between 60μM and 90μM of MTX110, depending on patient tolerance over the course of 12 infusions in phase 2.
It said that 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 the start 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, the board said.
It explained that MTX110 was administered directly into the tumour via a micro-catheter, using convection enhanced delivery (CED), with gadolinium-enhanced intraoperative 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 on 30 September, median overall survival based on Kaplan Meier analysis was 26.06 months, and overall survival at 12 months was 71.4%.
Midatech said three patients remained alive, and were still being monitored.
Survival was not an endpoint of the UCSF study, nor was the study powered for statistical significance, and thus no conclusions as to the impact of MTX110 on overall survival rates could be drawn from the data, the board cautioned.
It said the proposed phase 2 trial is expected to evaluate overall survival at 12 months as the primary endpoint, in 19 evaluable patients.
The planned design was single-arm and statistically-powered for comparisons with defined historical survival data.
MTX110 was expected to be delivered using an alternative CED catheter system, which would enable regular drug infusions directly into the tumour, without the need for repeated surgery.
"DIPG is a devastating pediatric brain cancer with limited treatment options and very poor outcomes," said executive vice-president of research and development Steve Damment.
"The overall survival data from this Phase I study are encouraging, although further study of MTX110 in DIPG is required to establish whether it can make a difference to these patients and their families."
At 1111 BST, shares in Midatech Pharma were up 24.58% at 41.11p.
11 Apr 2016 12:20