Clinical phase I trial of MTH1 inhibitors for cancer treatment
| Reference number | |
| Coordinator | Karolinska Institutet - Institutionen för medicinsk biokemi och biofysik |
| Funding from Vinnova | SEK 5 000 000 |
| Project duration | November 2014 - January 2018 |
| Status | Completed |
Purpose and goal
The overall aim is to develop and commercialize a novel anticancer treatment. We have identified and developed inhibitors against the enzyme MTH1 and developed a clinical candidate, Karonudib. Karonudib was shown to be well tolerated, oral available, and was approved by Medical Product Agency and Regional Ethical Committe to be tested in clinical phase 1 study with the aim to investigate tolerance and safety and determine recommended phase 2 dose in patients with advanced solid malignancies. IPR is secured. The subgoals of the project have been fulfilled.
Expected results and effects
Preclinical studies required by regulatory authorities have been performed, reported and scrutinized by Medical Products Agency and Regional Ethical Committe before approval and initiation of clinical phase 1 study using Karonudib. In discussions with Big Pharma , we have obtained information on Pharma´s requirements on clinical candidates. This information has been taken into consideration when developing the project. Next step is to perform a proof of concept Phase II study, and re-establish the contact with Big Pharma to continue the clinical development all the way to market.
Planned approach and implementation
Big Pharma was contacted early on in the project to obtain their view on the science, project plan, clinical candidate requirements. We secured IPR and established a business strategy. In parallel, the preclinical development was performed including e.g. pharmacology, safety assessment, formulation, clinical protocol design. Most of the studies were performed according to the original plan with some few adjustments and amendments.