Midatech liver cancer drug candidate gets Orphan Drug Designation

Specialty pharmaceutical company Midatech announced on Wednesday that the European Medicines Agency has granted Orphan Drug Designation for its advanced liver cancer drug candidate, MTD119.

The AIM-traded firm described MTD119, previously designated MTR104, as a treatment for hepatocellular carcinoma (HCC) - a rare, aggressive cancer which affects the liver.

It said there were very few extant treatments for HCC, particularly for patients with an advanced form of the disease, and fewer than 10% of patients with HCC currently survived beyond five years of diagnosis.

MTD119 was a combination of Midatech's gold nanoparticle (GNP) technology with the potent anti-tubulin chemotherapeutic maytansine, the company’s board said, in which the GNP was designed to improve the biodistribution and targeted delivery of the maytansine agent to the tumour site.

The Orphan Drug Designation programme provided orphan status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders which affect fewer than five in 10,000 people in the European Union.

Sponsors who obtained ODD benefited from development assistance, and were eligible for 10 years of market exclusivity once the medicine was on the market.

Midatech said a recent pre-clinical study comparing the efficacy of MTD119 with sorafenib, the current standard of care, showed a greater response rate and suggested the potential for an improved efficacy profile for MTD119.

The global HCC market was estimated to be worth around $1bn by 2024 however, due to the limited efficacy and high toxicity of existing treatments, the board said it believed there was scope to significantly expand that market.

Midatech said the programme was currently being prepared for clinical trials, with MTD119 having commenced formal investigational new drug (IND) application enabling toxicology studies, with completion of the pilot studies expected in the first half of 2018.

That would be followed by assessment and full toxicology with an expected IND submission to the US Food and Drug Administration for first in human studies sometime in the second half of 2018 or the first half of 2019.

“We are pleased that the EMA has granted MTD119 Orphan Drug Designation to treat patients with liver cancer,” said Midatech chief executive Dr Jim Phillips.

“Not only does this represent an important regulatory milestone for Midatech, but the decision means Midatech will receive financial and developmental support, as well as marketing exclusivity following approval of the drug.

“Our focus now is on advancing the development of MTD119 as fast as possible while also continuing to work on our exciting pipeline of oncology and immunotherapy assets, which contains several potentially significant and value-driving inflection points in 2018.”