Clinical Trials

A brighter future

Our mission is to advance medicines that will provide a brighter future for patients with serious conditions that are not fully served by existing treatments. Our ability to evaluate the safety and efficacy of new therapies is a fundamental part of the development process. We rely on volunteers to enroll in our studies and are very grateful for their participation.
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Serenta Clinical Trial for patients suffering with Familial Adenomatous Polyposis (FAP)

The Serenta trial is a Phase 3 clinical trial for people diagnosed with familial adenomatous polyposis (FAP). The purpose of this trial is to test an experimental treatment, eRapa, to evaluate its ability to improve clinical outcomes in FAP patients vs placebo (inactive substance).

eRapa (encapsulated rapamycin) Named Patient Programme

The eRapa Named Patient Programme has been established to provide access to eRapa, an investigational oral formulation of rapamycin, for certain patients diagnosed with Familial Adenomatous Polyposis (FAP).
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Expanded Access Policy

Publications

High dose MTX110 (soluble panobinostat) safely administered into the fourth ventricle in a non-human primate model

PNOC015: An Open Label Single Arm Study of MTX110 Delivered by Convection-enhanced Delivery (CED) in Patients with Diffuse Intrinsic Pontine Glioma (DIPG) Previously Treated with External Beam Radiation Therapy

A Phase I study examining the feasibility of intermittent convection-enhanced delivery (CED) of MTX110 for the treatment of children with newly diagnosed diffuse midline gliomas

Efficacy of soluble panobinostat (MTX110) in preclinical models of adult glioblastoma

Singleton et al: The distribution, clearance, and brainstem toxicity of panobinostat administered by convection-enhanced delivery

Printing particles: A high-throughput technique for the production of uniform, bioresorbable polymer microparticles and encapsulation of therapeutic peptides