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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.

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

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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