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

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Glaucoma is the world's leading cause of irreverisble 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. New drugs that inhibit VASH, a newly elucidated enzyme that is hyperactivated in many neurogenerative diseases, have potential to address this need. We at MT-act are a biotechnological start-up who have identified a family of VASH-inhibiting small molecule drugs. Using an in-house proprietary biochemical assay followed by lead optimization, we have identified MT-011, which inhibits VASH with nanomolar affinity, as our lead candidate. Aside from glaucoma, VASH dysregulation is also implicated in other prominent neurodegenerative diseases such as Alzheimer’s and Parkinson’s. Our assets therefore have an inherently broad applicability. We aim to develop MT-011 up to the stage of Phase 1 clinical trials, upon which we aim to commercialize the drug via a licensing deal with a pharmaceutical partner. We will then receive upfront and milestone payments with a estimated total deal size of €40M. We are currently in discussion with world-leading pharmaceutical companies Pfizer and Santen Pharmaceuticals, who have already indicated interest in our drugs. In SA-VOIR, we aim to strengthen the business case for MT-011 and to outline a detailed technical plan for its continued development. These documents will not only guide us in developing MT-011 towards Phase 1 trials in an effective and de-risked manner, it will also form critical components of a business plan that we will use to attract further investment.