Orphan Designation Granted by EMA to MTX110 Development for the Treatment of Glioma
Midatech Pharma PLC (AIM: MTPH.L; Nasdaq: MTP), an R&D biotechnology company focused on improving the bio-delivery and biodistribution of medicines , is pleased to announce that following the submission of an application to the European Medicines Agency (“EMA”), its development programme of MTX110 for the treatment of glioma has been granted Orphan Medicinal Product designation by the agency.
Midatech has been developing MTX110 for the treatment of recurrent glioblastoma (“rGBM”) in adult patients and diffuse intrinsic pontine glioma (“DIPG”) and medulloblastoma in paediatric patients.
Orphan designation is granted by the EMA to medicines that meet pre-specified criteria, including treatment of a life-threatening condition and prevalence of no more than 5 in 10,000 in the EU. The designation is intended to offer a range of incentives that facilitate development of the medicine, such as protocol development assistance, reduction in fees and market exclusivity upon successful approval of the drug.
Commenting, Dmitry Zamoryakhin, CSO of Midatech, said: “Both rGBM and DIPG are devastating and incurable cancers marked by short survival rates and universal recurrence. Receiving Orphan designation for MTX110 is an important milestone for the development of the drug, as it demonstrates the need for novel and effective treatment options for these fatal diseases and highlights a potential benefit that the development of MTX110 might bring to patients.”
MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD), that enables convection-enhanced delivery (CED) at potentially chemotherapeutic doses directly to the site of the tumour. Panobinostat is a hydroxamic acid and acts as a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor). The currently available oral formulation of panobinostat lactate (Farydak®) is not suitable for treatment of brain cancers owing to poor blood-brain barrier penetration and inadequate brain drug concentrations. Based on favourable translational science data, MTX110 is being evaluated clinically as a treatment for DIPG (NCT03566199, NCT04264143) and recurrent medulloblastoma (NCT04315064), and preclinically for treatment of glioblastoma (SNO 2020 Abstract TMOD-27). MTX110 is delivered directly into and around the patient’s tumour via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumour to very high drug concentrations while simultaneously minimising systemic drug levels and the potential for 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 promising of 83 anticancer agents tested in 14 patient-derived DIPG cell lines (Grasso et al, 2015. Nature Medicine 21(6), 555-559).
For more information, please contact:
Midatech Pharma PLC
Dmitry Zamoryakhin, CSO
Tel: +44 (0)29 20480 180
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