The study is a combined Phase I and II study in approximately 40 patients to be conducted at specialist centres at the University of California, San Francisco and the Memorial Sloan Kettering Cancer Center in New York.
The Phase I safety component of the study is expected to read out in the second half of 2019 and the Phase II efficacy component is expected to read out in 2020. Following this, and pending positive study results, the Company may attempt to secure expedited regulatory approval for MTX110.
DIPG is a rare, fatal childhood brainstem tumour with overall median survival of approximately nine months, despite decades of clinical trial research focussed on potential treatments.
On the basis of generally accepted prevalence statistics, there are up to 300 cases each year in the US and Europe, and up to 1,000 cases globally.
MTX110 takes the known active HDACi panobinostat (which is available in oral form only), and solubilises it into liquid form using Midatech's nano-inclusion (NI) technology. This increases available routes of administration for panobinostat and is essential because the drug does not cross the blood-brain barrier effectively when given orally.
MTX110 can therefore be delivered directly to a patient's tumour via a catheter system (Convection Enhanced Delivery, or "CED"), infused into and around the tumour under pressure, thereby bypassing the blood-brain barrier.
This technique allows for high drug concentrations to be delivered to the tumour while simultaneously minimising systemic toxicity and other side effects.
Panobinostat has demonstrated high potency against DIPG tumour cells in in vitro and in vivo models, and in a key study it was the most effective of 83 anticancer agents tested in several patient DIPG cell lines.
To date, MTX110 has been used to treat a number of patients on a compassionate basis and the drug has been generally well tolerated in these instances.
The Company has plans for additional studies of MTX110 in the US and Europe.
Midatech CEO-designate Craig Cook said: "We are pleased to commence this study for MTX110, one of our three lead assets and a potentially pioneering treatment for DIPG. It is a testament to the strength and dedication of our team that, having only begun research work on this programme in 2015, this complex and novel potential therapy is now entering the clinic less than 30 months later.
“We are excited by MTX110's potential to make a difference to the children who suffer from DIPG, and for whom the only existing therapeutic options are palliative. We look forward to continuing to progress our R&D pipeline, addressing unmet needs in significant markets such as this and creating value for our stakeholders."
Source: Company Press Release.