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Late Clinical Development of KH176: an innovative orphan drug to reach mitochondrial disease patients & market

Description du projet

Un nouveau médicament pour les maladies mitochondriales

Le syndrome MELAS (Mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes) est un trouble qui affecte principalement les systèmes nerveux et musculaire. Les mitochondries mutantes ne parviennent pas à produire l’énergie requise, ce qui affecte l’homéostasie et la survie des cellules. Il n’existe actuellement que des soins de soutien et aucun traitement approuvé pour stopper la progression du MELAS, qui provoque une déficience neurologique. Le projet KHON2bTREAT, financé par l’UE, a pour objectif de répondre à ce besoin médical grâce au nouveau médicament, le sonlicromanol, qui est évalué dans un essai clinique de phase II. Le projet portera le test du sonlicromanol à une étude de phase III, suivie de sa commercialisation et de son approbation pour d’autres maladies mitochondriales.

Objectif

Mitochondrial diseases are chronic progressive disorders for which no cure exists, affecting approximately 250,000 patients worldwide. KH176 is a proprietary small molecule designed by the Dutch SME Khondrion BV to treat a large group of rare mitochondrial and related diseases. With KH176, a new redox-modulator, Khondrion aims to stall disease progression and revert clinical complaints in mitochondrial diseases; it corrects cellular consequences of mitochondrial dysfunction and has broad market potential.
Khondrion’s management has >25 years experience in mitochondrial medicine, patient care and drug development, and is supported by highly experienced entrepreneurs. Since 2012, Khondrion completed preclinical, Phase 1&2a clinical, EMA&FDA orphan drug designation providing market exclusivity, and has a strong patent portfolio. Khondrion operates in a small but highly lucrative niche market. The addressable market for KH176 is 50,000-250,000 mitochondrial disease patients in Europe&US. Base-case cumulative revenue on KH176 is expected €35M by 2024, a return of >10 times this grant. Worldwide orphan drug sales are to increase at 11% CAGR to $209b in 2022; orphan drugs are projected to account for 21.4% of worldwide prescription sales by 2022 (excl. generics) from 6% in 2000. Worldwide, Khondrion is one of few SMEs devoted to develop a drug for mitochondrial diseases.
Khondrion follows fast, risk-reduced development for KH176; first market approval in adult patients (MELAS/MIDD), entering expedited authorization for other mitochondrial diseases. As spin-out, Khondrion aims licensing KH176 for major indications (preclinical data on KH176 in genetic forms of Parkinson’s completed). Financing of this KHON2bTREAT project by the EIC SME instrument Phase 2 will enable Khondrion to perform the dose-finding clinical trial (Phase 2b) on KH176 in its patient population, reaching essential milestones for investor/co-development financing of the Phase 3 trial for market approval.

Appel à propositions

H2020-EIC-SMEInst-2018-2020

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Sous appel

H2020-SMEInst-2018-2020-2

Régime de financement

SME-2 - SME instrument phase 2

Coordinateur

KHONDRION BV
Contribution nette de l'UE
€ 2 336 267,50
Adresse
VAN HEEMSTRAWEG 49 E
6641 AA Beuningen
Pays-Bas

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PME

L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention.

Oui
Région
Oost-Nederland Gelderland Arnhem/Nijmegen
Type d’activité
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Liens
Coût total
€ 3 337 525,00