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KHON2bTREAT SIGNED

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

Total Cost €

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EC-Contrib. €

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Partnership

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Outcomes and
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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.

entrepreneurs excl niche expedited kh176 drug preclinical dose exclusivity patent fda forms complaints instrument ema investor drugs group disorders 209b fast midd cagr cure modulator molecule 2a entering financing designed portfolio trial worldwide rare times generics authorization cellular proprietary progressive bv reaching revenue care exists khon2btreat clinical prescription mitochondrial projected patient disease 2024 base perform broad co sme 2022 designation smes diseases 2b revert risk 2000 sales progression treat stall operates follows 25 lucrative licensing dysfunction medicine patients 250 parkinson genetic addressable khondrion indications 35m melas eic corrects return 4 dutch first gt cumulative redox population 2012 11 6 devoted approximately chronic market approval small few orphan grant adult data spin milestones 21

Project "KHON2bTREAT" data sheet

The following table provides information about the project.

Coordinator	KHONDRION BV Organization address address: VAN HEEMSTRAWEG 49 E city: BEUNINGEN postcode: 6641 AA website: http://www.khondrion.nl 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	http://www.khondrion.com/about-us/
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

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	KHONDRION BV KHONDRION BV Organization address address: VAN HEEMSTRAWEG 49 E city: BEUNINGEN postcode: 6641 AA website: http://www.khondrion.nl contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (BEUNINGEN)	coordinator	2˙336˙267.00

Map

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.

Deliverables

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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