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

Project description

A new drug for mitochondrial diseases

Mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes (MELAS) syndrome is a disorder that primarily affects the nervous and muscular systems. Mutant mitochondria fail to produce the necessary energy, affecting cell homeostasis and survival. Currently, there is only supportive care available and no approved treatments to halt MELAS progression, which leads to neurological impairment. The EU-funded KHON2bTREAT project aims to address this unmet medical need through the novel drug sonlicromanol, which is being evaluated in a Phase II clinical trial. The project will bring sonlicromanol testing to a Phase III study followed by commercialisation and authorisation for other mitochondrial diseases.


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.

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Net EU contribution
€ 2 336 267,50
6641 AA Beuningen

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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Oost-Nederland Gelderland Arnhem/Nijmegen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Total cost
€ 3 337 525,00