Innovating Works

KHON2bTREAT

Financiado
Late Clinical Development of KH176 an innovative orphan drug to reach mitochond...
Late Clinical Development of KH176 an innovative orphan drug to reach mitochondrial disease patients market 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 g... 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. ver más
30/06/2022
KHONDRION
3M€
Duración del proyecto: 45 meses Fecha Inicio: 2018-09-26
Fecha Fin: 2022-06-30

Línea de financiación: concedida

El organismo H2020 notifico la concesión del proyecto el día 2022-06-30
Línea de financiación objetivo El proyecto se financió a través de la siguiente ayuda:
EIC-SMEInst-2018-2020: SME instrument
Cerrada hace 6 años
Presupuesto El presupuesto total del proyecto asciende a 3M€
Líder del proyecto
KHONDRION BV No se ha especificado una descripción o un objeto social para esta compañía.
Perfil tecnológico TRL 4-5