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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 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.

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

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.
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 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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