Descrizione del progetto
Un nuovo farmaco per trattare le malattie mitocondriali
La sindrome MELAS, che comprende miopatia mitocondriale, encefalopatia, acidosi lattica ed episodi simili a ictus, è un disturbo che colpisce principalmente l’apparato nervoso e muscolare. A causa di questa sindrome, i mitocondri mutanti non riescono a produrre l’energia necessaria, condizionando quindi l’omeostasi e la sopravvivenza cellulare. Attualmente, sono disponibili solo cure di sostegno e nessun trattamento approvato per arrestare la progressione di questa sindrome che provoca un deterioramento neurologico. Il progetto KHON2bTREAT, finanziato dall’UE, intende affrontare questa esigenza medica insoddisfatta attraverso il nuovo farmaco sonlicromanol, che è in fase di valutazione in uno studio clinico di fase II. Il progetto procederà con uno studio di fase III a cui farà seguito la commercializzazione e l’autorizzazione del sonlicromanol per altre malattie mitocondriali.
Obiettivo
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.
Campo scientifico
Parole chiave
Programma(i)
Argomento(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-SMEInst-2018-2020-2
Meccanismo di finanziamento
SME-2 - SME instrument phase 2Coordinatore
6641 AA Beuningen
Paesi Bassi
L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione.