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KHON2bTREAT SIGNED

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

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EC-Contrib. €

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Partnership

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 Outcomes and results

 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.

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

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 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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