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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.

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

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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