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Assessing the feasibility of MT-011, a first-in-class drug to treat glaucoma and other neurodegenerative diseases via a breakthrough mechanism-of-action

Periodic Reporting for period 1 - SA-VOIR (Assessing the feasibility of MT-011, a first-in-class drug to treat glaucoma and other neurodegenerative diseases via a breakthrough mechanism-of-action)

Reporting period: 2019-12-01 to 2020-05-31

MT-act is working to develop novel small molecule drugs for neuroprotection. Despite enormous medical progress in diagnosing and slowing some neurodegenerative diseases, protecting neurons from further damage is still a large unmet need. MT-act targets a novel pathway for neuronal preservation, which has been reported to be dysregulated in several prominent neurodegenerative diseases, including glaucoma.
MT-act’s work is important for society as it is directly addresses the burden of glaucoma. Glaucoma is the world's leading cause of irreversible blindness, affecting 20 million people in Europe and the US. Known as the Alzheimer’s of the eye, glaucoma is a neurodegenerative disorder that causes deterioration in peripheral vision. Affected patients report ‘seeing through a black tunnel’. Treatments to halt vision loss are available however, they are only applicable for the ‘high tension glaucoma’ subtype. For those diagnosed with ‘normal tension', which comprises over 30% of glaucoma patients, no treatments are available, and vision loss is imminent.
In this feasibility study, the overall objective was to perform competitor, market and preclinical/clinical development on MT-act’s lead candidate, and to update their business plan with results from the study.
The work performed as part of the feasibility study follows that outlined in the Implementation Section of the SA-VOIR proposal.
TASK1: Define commercial value of MT-043
Main result: Based on the outcomes of desk research and KOL interviews, we identified a high potential commercial value for MT-043, specifically in normal-tension glaucoma (NTG). Considering existing competition and predicted barriers to market, we also propose an additional development scenario in an orphan indication, which typically accelerates market access and revenues from an exit deal.
TASK 2: Define preclinical drug development plan for MT-043
Main result: Based on existing and newly established contacts with major companies in the ophthalmology space, we developed a plan, including internal costs and identified service providers, for the preclinical development of MT-043. We additionally sketch a foreseeable clinical trial design based on existing comparable trials considering limitations and constrains within the field.
TASK 3: In this task, sections of our business plan were updated.
Main result: The indications and development plans were expanded with descriptions of the commercial, technical and clinical justifications for selecting specific target indications.
In this study we had revealed further insight into the market and therefore the feasibility of our drug assets.
MAIN IMPACT: The main impact of the study will be de-risked and expedited drug development pathways for our drug asset. This feasibility study has allowed us to select the main target indications on which to focus, as to allow for the most clinically impactful, fastest and most profitable strategy towards commercialization.
SOCIETAL IMPACT: The outline of a sound drug development pathway will allow us to accelerate development of our assets towards clinical trials. This in turn reduces the time to which our drugs will have ultimate impact on the patients they are designed to treat.
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