Opendata, web and dolomites


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

Total Cost €


EC-Contrib. €






 KHON2bTREAT project word cloud

Explore the words cloud of the KHON2bTREAT project. It provides you a very rough idea of what is the project "KHON2bTREAT" about.

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Project "KHON2bTREAT" data sheet

The following table provides information about the project.


Organization address
postcode: 6641 AA

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Netherlands [NL]
 Project website
 Total cost 3˙337˙525 €
 EC max contribution 2˙336˙267 € (70%)
 Programme 1. H2020-EU.3. (PRIORITY 'Societal challenges)
2. H2020-EU.2.3. (INDUSTRIAL LEADERSHIP - Innovation In SMEs)
3. H2020-EU.2.1. (INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies)
 Code Call H2020-SMEInst-2018-2020-2
 Funding Scheme SME-2
 Starting year 2018
 Duration (year-month-day) from 2018-10-01   to  2020-12-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    KHONDRION BV NL (BEUNINGEN) coordinator 2˙336˙267.00


 Project objective

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.


List of deliverables.
Dissemination material Websites, patent fillings, videos etc. 2020-01-14 14:59:05
Press releases and information papers on KHON2bTREAT to relevant stakeholders Websites, patent fillings, videos etc. 2020-01-14 14:49:17

Take a look to the deliverables list in detail:  detailed list of KHON2bTREAT deliverables.

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The information about "KHON2BTREAT" are provided by the European Opendata Portal: CORDIS opendata.

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