Das Projekt beinhaltet die Entwicklung von personalisierten Therapien für Betroffene mit seltenen NME, bei denen bisher Behandlungsmöglichkeiten fehlen. Zu den Patient:innen gehören auch Kinder und junge Erwachsene, die bislang keine Aussicht auf Heilung oder zumindest auf Linderung der Erkrankung hatten. Für sie sollen neue therapeutische Ansätze entwickelt werden, die im Sinne eines “Bench-to-Bedside”-Prinzips (aus dem Labor an das Krankenbett) direkt in individuelle Heilversuche überführt werden können. Dabei kommen sogenannte Omics-Technologien sowie Bioinformatik inkl. Künstliche Intelligenz (KI) zum Einsatz, um therapeutisch relevante Krankheitsmechanismen zu identifizieren und in die klinische Anwendung zu überführen.

Ausgangspunkt für diesen Ansatz sind die Patient:innenauswahl, die Analyse von  Hautbiopsien und die nachfolgende Phänotypisierung (Klassifizierung des Erscheinungsbildes) der gewonnenen Hautzellen (genauer: Fibroblasten). Diese Phänotypisierung, die von Wissenschaftler:innen in Düsseldorf, Bochum und Essen durchgeführt wird, wird zum Verständnis der zugrunde liegenden Eigenschaften dieser Zellen führen. Da Hautzellen dieselben genetischen Informationen wie Muskelzellen tragen, lassen sich ihre krankheitsverursachenden Veränderungen mit Omics-

Technologien untersuchen. Omics bezeichnet die gesamtheitliche Charakterisierung unter anderem aller Gene, Proteine oder Metabolite (Stoffwechselprodukte). Omics-Daten liefern einen wichtigen Ansatzpunkt in der personalisierten Medizin, da sie Aufschluss über individuelle Krankheitsvorgänge und mögliche Therapieansätze geben. Die Firma Singleron wird die zugrunde liegenden Genaktivitäten (sogenannte Transkriptomanalysen) durchführen, während ISAS-Forschende die entsprechenden Proteine und Stoffwechselprodukte auf Basis speziell entwickelter Massenspektrometrie-Verfahren analysieren werden.

 Suche nach bereits bekannten und unbekannten Arzneimittelwirkstoffen

Die gewonnenen Omics-Daten werden Bioinformatiker:innen am ISAS anschließend mithilfe von KI auswerten und mit Datenbanken zu vorhandenen Arzneimittelwirkstoffen abgleichen. Das Ziel dieser Analysen ist es, geeignete, bereits bekannte Wirkstoffe zu finden, mit denen sich die im ersten Schritt identifizierten fehlregulierten zellulären Prozesse bei NME adressieren lassen – und die damit für die Behandlung der Patient:innen infrage kommen können. Außerdem wird das Lead Discovery Center nach weiteren potenziell wirksamen Substanzen suchen. Sowohl die neuen, als auch bereits bekannten Wirkstoffe werden anschließend im Labor an den gewonnenen Fibroblasten getestet. In einem weiteren Schritt werden die Wissenschaftler:innen die Relevanz der fehlregulierten zellulären Prozesse in Nerven- und Muskelzellen prüfen. Erfolgversprechende Substanzen sollen danach in den Kliniken direkt für eine personalisierte Behandlung in individuellen Heilversuchen in den Patient:innen eingesetzt werden.

Insgesamt hat das Konsortium aus Kliniker:innen, Grundlagenwissenschaftler:innen und Bioinformatiker:innen bei diesem Forschungsprojekt sowohl den medizinischen, als auch den gesellschaftlichen Nutzen im Fokus. Eine verbesserte Lebensqualität der NME-Patient:innen durch neue personalisierte Therapien wirkt sich nicht nur positiv auf die einzelnen Betroffenen, sondern auch auf ihre Familien und das gesamte gesellschaftliche Umfeld aus.

Übersicht der Projektpartner

Unter der Leitung von Prof. Dr. Tobias Ruck von der Universitätsmedizin Düsseldorf vereint das Konsortium führende medizinische Forschungsinstitutionen und Industrieunternehmen. Dieser synergistische neuartige Ansatz ermöglicht innovative Therapiestrategien zur schnelleren Diagnostik und Behandlung von NME. Zentral für diese Ziele ist eine intensive Zusammenarbeit der Projektpartner:innen:

• Universitätsklinikum Düsseldorf, Klinik für Neurologie
• Universitätsmedizin Essen, Abteilung für Neuropädiatrie
• BG Universitätsklinikum Bergmannsheil Bochum
• Leibniz-Institut für Analytische Wissenschaften – ISAS e.V.
• Lead Discovery Center GmbH (LDC), Dortmund
• Singleron Biotechnologies GmbH, Köln

Weitere Informationen: b2b-rare.de

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Über die Klinik für Neurologie des Universitätsklinikums Düsseldorf

In der Klinik für Neurologie des Universitätsklinikums Düsseldorf (Klinikleitung: Prof. Dr. med. Dr. rer. nat. Sven Meuth; Stellvertretung: Prof. Dr. med. Tobias Ruck) wird das gesamte Spektrum neurologischer Erkrankungen bei Erwachsenen ab 18 Jahren diagnostiziert und behandelt. Insgesamt werden in der Klinik für Neurologie jährlich ca. 15000 Patient:innen stationär und ambulant behandelt (ca. 2000-2500 Patient:innen davon mit neuromuskulären Krankheitsbildern). Die Klinik ist Teil des Neuromuskulären Zentrums Nordrhein. Zudem ist die Klinik für Neurologie ein zertifiziertes Myasthenie-Zentrum. In der Klinik wird das Konzept einer engen Vernetzung von Patient:innenversorgung, klinischer Forschung und Grundlagenwissenschaft verfolgt. Das Team möchte, dadurch die Ursachen neurologischer Erkrankungen besser verstehen und neue Therapiemöglichkeiten entwickeln. Die intensive Einbindung in internationale Studien gibt die Möglichkeit, Patient:innen   Zugang zu neuesten innovativen Therapien zu verschaffen.

Weitere Informationen: www.uniklinik-duesseldorf.de

Über die Neurologische Klinik und Poliklinik des BG-Universitätsklinikums Bergmannsheil

Die Neurologische Klinik und Poliklinik des BG-Universitätsklinikums Bergmannsheil (Interimsleitung Prof. Dr. med. Matthias Vorgerd) befasst sich neben der klinischen Versorgung sämtlicher neurologischer Erkrankungen von Patient:innen ab einem Alter von 18 Jahren mit einem breiten Spektrum klinischer und grundlagenwissenschaftlicher Forschung. Die Klinik ist Teil des Muskelzentrums Ruhrgebiet und von der Deutschen Gesellschaft für Muskelkranke e.V. (DGM) zertifiziertes Neuromuskuläres Zentrum. Sie ist an verschiedenen nationalen und internationalen Studien beteiligt. Zudem verfügt die Klinik über ein eigenes neuromuskuläres Labor, wo diagnostische und wissenschaftliche Untersuchungen am entnommenen Skelettmuskel, Nerven und Haut durchgeführt werden.  

Ein besonderer Schwerpunkt liegt auf der Erforschung von neuromuskulären Erkrankungen (NME), die sich vor allem auf die Untersuchung von Muskel-, Nerven- und Hautbiopsien stützt. Die Forschungsaktivitäten auf dem Gebiet der NME werden durch das Heimer Institut für Muskelforschung gefördert, welches in die Organisationstruktur der Neurologischen Klinik eingeordnet ist. Diese enge Vernetzung von klinischer Arbeit, spezialisierter Labordiagnostik und grundlagenwissenschaftlicher Forschung soll zur Entwicklung neuer Behandlungsmöglichkeiten beitragen.

Über die Abteilung für Neuropädiatrie der Universitätsmedizin Essen AöR

Die Abteilung für Neuropädiatrie beschäftigt sich neben der routinediagnostischen Analyse der Muskulatur von Patient:innen auch mit der Erforschung der molekularen Ursachen der jeweiligen Krankheitsbilder. Dazu zählen die Identifikation neuer Gene, sowie die Analyse der biochemischen Ursachen neurodegenerativer und muskulärer Erkrankungen. Die Forschungsaktivitäten des neuromuskulären Labors werden hierbei durch Herrn PD Dr. rer. Nat. Andreas Roos (Adjunct Professor der University of Ottawa) und Frau Prof. Dr. med. Ulrike Schara-Schmidt geleitet. Forschungsinteressen des Muskellabors liegen auf der Identifikation von Pathomechanismen und Biomarkern für neuromuskuläre Erkrankungen mit einem ausgesprochenen Fokus auf die Nutzung von Biomaterial. Dabei steht auch die Nutzung von Biomaterialien, welche minimal-invasive gewonnen werden können, im Fokus.

Im Rahmen von translationalen Prozessen ist das neuromuskuläre Labor Bestandteil zahlreicher nationaler und internationaler Studien (clinicaltrials.gov) sowie an übergreifenden nationalen und internationalen Studien zur Genotyp-Phänotyp Korrelationen beteiligt.

Über das Leibniz-Institut für Analytische Wissenschaften – ISAS e.V. –

Das Leibniz-Institut für Analytische Wissenschaften – ISAS – e.V. entwickelt leistungsfähige Analyseverfahren für die Gesundheitsforschung. Mit seinen Innovationen trägt es dazu bei, die Prävention, Frühdiagnose und Therapie von Erkrankungen zu verbessern. Ziel des Instituts ist es, die personalisierte Therapie voranzutreiben. Dafür kombiniert das ISAS das Wissen aus Chemie, Biologie, Medizin, Pharmakologie, Physik und Informatik. Das Institut kooperiert mit nationalen und internationalen Partnern aus der Wissenschaft und Industrie.

Weitere Informationen: www.isas.de

Über die Singleron Biotechnologies GmbH

Singleron Biotechnologies fördert die Präzisionsmedizin und die menschliche Gesundheit durch wegweisende Lösungen zur Analyse von Einzelzell-Multi-Omics. Das aktuelle Produktportfolio umfasst Hochdurchsatzgeräte für die automatisierte Verarbeitung von Einzelzellen und Gewebedissoziation, Reagenzien, Bioinformatik-Software sowie eine umfassende Wissensdatenbank für Einzelzellen.

Gegründet im Jahr 2018, ist Singleron weltweit tätig und verfügt über Büros, Labore und Produktionsstätten in Deutschland, Singapur, China und den USA. Die Produkte des Unternehmens werden in über 3000 Laboren in Krankenhäusern, Forschungsinstituten und Pharmaunternehmen in mehr als 20 Ländern eingesetzt.

Weitere Informationen: www.singleron.bio. Folgen Sie uns auf LinkedIn für aktuelle Updates.

Über die Lead Discovery Center GmbH

Die Lead Discovery Center GmbH wurde 2008 von der Technologietransfer-Organisation Max-Planck-Innovation gegründet, um das Potenzial exzellenter Grundlagenforschung für die Entwicklung neuer, dringend benötigter Medikamente besser zu nutzen. LDC nimmt vielversprechende Projekte aus der akademischen Forschung auf und entwickelt sie typischerweise weiter bis zu pharmazeutischen Leitstrukturen (Proof-of-Concept in Modellsystemen) oder bis zu präklinischen Kandidaten. In enger Zusammenarbeit mit führenden Partnern aus der akademischen Forschung und Industrie entwickelt LDC ein umfangreiches Portfolio an Projekten im Bereich niedermolekularer Wirkstoffe sowie therapeutische Antikörper mit außergewöhnlich hohem medizinischem und kommerziellem Potenzial.

LDC unterhält eine enge Partnerschaft mit der Max-Planck-Gesellschaft und dem KHAN Technology Transfer Funds I + II (KHAN-I, -II). Es hat weltweit zahlreiche Kooperationen mit verschiedenen Organisationen geschlossen, u.a. AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Qurient, invIOs, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea AS und den Helmholtz-Zentren, für Infektionsforschung (HZI) und dem Deutschen Krebsforschungszentrum (DKFZ). Das LDC arbeitet außerdem mit führenden akademischen Wirkstoffforschungszentren und Investoren zusammen, um Firmengründungen zu unterstützen.

Weitere Informationen: www.lead-discovery.de

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Scope of Collaboration

Antibody-based inhibitors have gained  relevance in the field of drug discovery due to their exceptional affinity, selectivity and physicochemical properties. Within this collaboration, Icosagen will generate, identify, and discover potent antibodies against innovative and therapeutically relevant targets, supplying them to LDC for further development.

Utilizing its proprietary technology, Icosagen will produce the proteins of interest and enhance their display on the surface of virus-like particles, which serve as vehicles for the production of antibodies with affinity for the targets. Subsequently, an extensive library will be established to facilitate the selection of high-affinity monoclonal antibodies, which will undergo further analysis at LDC through a streamlined process of high-throughput screening. This partnership highlights Icosagen’s capability to scale its proprietary platforms and synergize effectively with LDC’s workflow.

“Partnering with LDC will further help expand the opportunities for antibody therapeutics, especially in the challenging field of GPCR-targeted treatments” said professor Mart Ustav, CEO of Icosagen. “This will not only highlight our technological expertise but also perfectly aligns with our mission to transform scientific discoveries into life-changing treatments.”

“We are excited to partner up with Icosagen, a competent and highly experienced player in the field of antibody research” remarked Bert Klebl, CEO and CSO of LDC. “This project will greatly benefit from Icosagen’s expertise and state-of-the-art capabilities as we pursue our shared goal: the development of novel therapeutics that can make a meaningful difference in healthcare.”

Future Prospects

LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential. The partnership between Icosagen and LDC holds promise in advancing targeted therapeutics, with the potential to revolutionize treatment approaches and improve outcomes for patients worldwide. The successful development of these monoclonal antibodies is anticipated to significantly advance the understanding and treatment of diseases.

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About Icosagen

Icosagen is a renowned biotech company with over 20 years of experience in pioneering antibody research and production. With a strong commitment to innovation, Icosagen has developed a range of proprietary platforms for the efficient discovery, development and manufacturing of high-quality antibodies, serving the global research community and pharmaceutical industry.

Further information available at: www.icosagen.com

About LDC

Lead Discovery Center GmbH (LDC) was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential.

LDC sustains a long-term partnership with the Max Planck Society and its institutes as well as with KHAN-I, and has formed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi Sankyo, Qurient, InvIOS, Novo Nordisk, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea Lead Discovery AS, HLB Pharma, the Helmholtz Center for Infection Research, e.g. In addition, LDC also works with leading translational drug discovery centers and with various investors to provide its assets for company creation.

Further information available at: www.lead-discovery.de

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Contact:

Lead Discovery Center GmbH
Otto-Hahn-Straße 15
44227 Dortmund, Germany
pr@lead-discovery.de
www.lead-discovery.de

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Tromsø, Norway, and Dortmund, Germany – April 25th, 2024. KinSea Lead Discovery AS, a biopharmaceutical start-up pioneering the use of marine bioactives for the treatment of human diseases, announces the successful first closing of its seed financing round. It includes an equity investment from KHAN Technology Transfer Fund I GmbH & Co KG (KHAN-I), an early-stage life sciences venture fund based in Germany, and welcomes the new investor Berners AS, a North Norwegian investment company. A year ago, KinSea had already secured a convertible loan from KHAN-I, which was recently converted into shares.

The financing enables the company to further develop its lead program, a FLT3 kinase inhibitor based on unique chemistry from marine sources, towards preclinical and clinical testing. Data from in vivo proof-of-concept studies suggest superior properties over existing FLT3 inhibitors, including potential broad activity against known drug-induced and drug-resistant FLT3 mutations, improved selectivity, and outstanding in vivo potency. The program originates from the successful collaboration of the founding partners, Arctic University of Norway (UiT), University of Bergen (UiB), Norinnova, and Lead Discovery Center GmbH (LDC).

‘We are grateful for the continued confidence and support from KHAN-I, and delighted to welcome Berners AS on board’, says Jeanette Hammer Andersen, CEO of KinSea. ‘This first closing validates the transformative potential of our approach. We are very committed to take our FLT3 inhibitors through the next stages of drug discovery and development in order to provide entirely new treatment options for AML patients that are safer and more effective’.

KinSea also plans to gradually expand its drug discovery pipeline and establish a diversified portfolio of high-potential drug candidates based on novel chemical scaffolds from the Arctic Ocean for the treatment of cancer and other diseases.

‘We are excited to reaffirm our commitment to KinSea and its groundbreaking work in the field of marine-derived therapeutics. The team has made significant progress over the last year, and we are convinced that their unique approach and capable team will continue to drive the maturation and expansion of their pipeline, and eventually make a meaningful impact on patients’ lives, in particular with regard to urgently needed, improved therapies for AML patients’, comments Bert Klebl, Managing Director of KHAN-I.

Mats Sæverud, CEO of Berners AS, adds: ‘In KinSea, we found an ambitious startup company that wants to make an important impact by employing natural products from the Arctic Ocean. The KinSea team has bold visions, scalable solutions, excellent expertise and skills, and fits well with Berners´ ambitions.’

KinSea has already started discussions with further investors for a second and final closing.

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Contact
Lead Discovery Center GmbH
Otto-Hahn-Straße 15
44227 Dortmund
Germany
Phone: +49 231 97 42 70 00
Mail: pr@lead-discovery.de

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About KHAN-I

KHAN Technology Transfer Fund I GmbH & Co KG (KHAN-I) is an early-stage life sciences venture fund with €70 million under management. Our mission is to create value through cooperative drug development partnerships with academic innovators in Europe. KHAN-I focuses on first-in-class therapies for attractive markets with a high unmet medical need. The fund is managed by Khanu Management GmbH, an experienced team of professionals with proven track records in early-stage drug development and academic spin-offs as well as pharma licensing and partnering. KHAN-I received an investment from the European Investment Fund (EIF) with the support of InnovFin Equity, and with the financial backing of the European Union under Horizon 2020 Financial Instruments and the European Fund for Strategic Investments (“EFSI”) under the Investment Plan for Europe. KHANI is also supported by Austria Wirtschaftsservice GmbH (AWS with funds provided by the Austrian

Federal Ministry for Digital and Economic Affairs and the Austrian Foundation for Research, Technology, and Development), Max Planck Foundation, and Thyssen’sche Handelsgesellschaft mbH. In addition, KHAN-I sustains a preferred partnership with the Max-Planck Society (Max-Planck Gesellschaft e.V.).

Further information at www.khanu.de

About Berners AS

Berners AS is a newly established, privately owned investment company, based in Tromsø, Norway. Through investments and active ownership, Berners will contribute to the development of profitable and sustainable businesses, especially within the seafood and marine sector. We aim to be a supporter of the development of brands and competence clusters in Northern Norway. Our investment strategy is based on our knowledge and love for the coast and the sea, and the opportunities that exist there. Berners is owned by Triko AS (80%) and Larren Invest AS (20%).

About Norinnova

Norinnova is one of Northern Norway’s most competent and experienced agencies for research commercialization. Norinnova connects researchers, start-up environments, companies and commercial actors to develop and utilize the region’s innovation power. For more than 30 years, Norinnova has worked closely with researchers and leading research communities in Northern Norway to harness the power of innovation in this region. This collaboration has contributed to the creation of brand-new businesses and has reinforced existing companies through new products and services. Norinnova secures rights, helps provide funding, investigates market potential, finds relevant partners, and contributes so that the scientists can get their product or service to the market.

Further information available at: www.norinnova.no

About LDC

Lead Discovery Center GmbH (LDC) was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential.

LDC sustains a long-term partnership with the Max Planck Society and its institutes as well as with KHAN-I, and has formed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi Sankyo, Qurient, InvIOS, Novo Nordisk, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea Lead Discovery AS, HLB Pharma, the Helmholtz Center for Infection Research, e.g. In addition, LDC also works with leading translational drug discovery centers and with various investors to provide its assets for company creation.

Further information available at: www.lead-discovery.de

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LDC and the University of Duisburg-Essen receive EUR 905,000 for the development of new therapies for age-related blindness

Dortmund, December 13, 2018 – The Lead Discovery Center GmbH (LDC) and the University of Duisburg-Essen (UDE) have received EUR 905,000 in funding from the state of North Rhine-Westphalia as part of its lead market competition, LifeSciences.NRW. Together with the LDC’s own financial contribution, this gives the partners a total of EUR 1.3 million with which to develop new drugs for the treatment of age-related macular degeneration (AMD).

 
AMD is the most common form of blindness. In Germany alone, approximately 6.9 million people are affected, 480,000 of them at an advanced stage of the disease. There is no satisfactory therapy to date.
New research has shown that AMD patients frequently have a high level of a particular enzyme, HTRA1-protease. This leads to an increased breakdown of proteins, resulting in damage to the macula, which is crucial for sight. The joint development project builds on research by Prof. Dr Ehrmann and his team at the UDE that suggests targeted inhibition of HTRA1 is a promising new approach to preventing the progression of the disease.

 
In an initial collaborative project, also supported by the state of North Rhine-Westphalia, the LDC and the UDE have already identified a series of chemical substances that specifically inhibit HTRA1. In the current follow-on project, the partners will be optimizing and validating these substances. Their goal is to develop a well-characterized lead structure that meets all the requirements of a therapeutic agent and demonstrates efficacy in an in-vivo model system.

 
To this end, the LDC and Prof. Dr Ehrmann and his team will be working closely with two further research groups at the UDE. Prof. Kaiser and his colleagues will contribute their extensive knowledge of protease inhibitors, and Prof. Dr Sanchez Garcia’s group provides longstanding experience in the computation and stimulation of biomolecules and biochemical processes. The LDC is coordinating the project, and contributes the competencies required for professional drug development, above all in the areas of medicinal chemistry and pharmacology. “This cooperation is a fantastic chance to develop results from basic research to benefit patients,” says Prof. Ehrmann. “In view of the aging world-wide population, we urgently need better drugs for the treatment of AMD.”

 
“We are delighted to be working together with this terrific and proven team,” adds Dr Bert Klebl, CEO of the LDC. “The project will profit enormously from their competence and experience. Together we can reach a stage of development that will be highly attractive to industry partners, enabling subsequent transfer to clinical development.”

 
The joint development project will run for 3 years und is supported by the European Regional Development Fund.

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Alliance for medicinal chemistry optimization and development of novel checkpoint blockade approach

Vienna, Austria, 19 September 2018: APEIRON Biologics AG, an Austrian company focused on cancer immunotherapy, today announced a collaboration agreement with the Lead Discovery Center GmbH (LDC), a translational research organization established in 2008 by Max Planck Innovation, to develop novel immune checkpoint modulators. The two companies will perform drug discovery activities to optimize and develop novel small molecules addressing immune checkpoints, important gatekeepers in cancer immunity. All molecules deriving from this collaboration are proprietary to APEIRON and will strengthen APEIRON’s development pipeline.

Peter Llewellyn-Davies, Chief Executive Officer of APEIRON Biologics, said: “We are delighted to announce this alliance with LDC. APEIRON has an excellent and broad pipeline being pursued to develop therapies both aiming at targeting tumor cells and stimulating the immune system to fight cancer. With its track record in pharmaceutical drug discovery and long‑standing small molecule expertise, the LDC is the ideal partner for us to identify new drug candidates and to further accelerate the development of new immune checkpoint modulators. I am confident our combined R&D forces will drive our technology in the field of immune-oncology forward and strengthen our product candidate portfolio in small molecules.”

The LDC will use its expertise in small molecule drug discovery to design and optimize new substances, which can activate immune cells and drive their anti-tumor activity. The medicinal chemistry efforts are based on a new substance class, which was identified by phenotypic screening, discovered in APEIRON’s cooperation with Evotec AG, and has shown to activate peripheral blood mononuclear cells (PBMCs). The intellectual property generated under the terms of the agreement solely belongs to APEIRON. The LDC will receive research funding during the collaboration period and is eligible to an upfront payment, milestone payments and royalties. Further financial details of the collaboration agreement were not disclosed.

The work will be carried out at both APEIRON’s R&D facilities in Vienna, Austria, and in the LDC laboratories in Dortmund, Germany. APEIRON will be responsible for further preclinical and clinical development of the product candidates, as well as subsequent marketing and commercialization.

About the LDC

The Lead Discovery Center (LDC) was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. The LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads that reach initial proof-of-concept in animals. In close collaboration with high-profile partners from academia and industry, the LDC is building a strong and growing portfolio of small molecule leads with exceptional medical and commercial potential. The LDC sustains a strong partnership with the Max Planck Society and has performed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi-Sankyo, Qurient, Johnson&Johnson Innovation, Sotio, Gruenenthal and Roche, as well as leading translational drug discovery centres around the globe. Further information is available at www.lead-discovery.de.

About APEIRON Biologics AG

Apeiron is a privately-held commercial-stage biopharmaceutical company based in Vienna, Austria, focused on the discovery, development and commercialization of novel cancer immunotherapies. The company is leveraging its innovative therapeutic targets based on tumor-specific targeted approaches and the stimulation of the immune system via novel and proprietary unique mechanisms of action (checkpoint blockade) to eradicate cancer by engaging the human body’s natural defense mechanisms. For additional information please visit www.apeiron-biologics.com and follow us on Twitter @apeironbio.

For further information please contact:

APEIRON Biologics AG

Peter Llewellyn-Davies, CEO

Email: investors@apeiron-biologics.com

www.apeiron-biologics.com

Public Relations LDC

Lead Discovery Center GmbH

T: +49 231 9742 7000

Email: pr@lead-discovery.de

FORWARD LOOKING STATEMENTS

Information set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of APEIRON as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

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Aachen, Dortmund and Göttingen, Germany, June 21, 2018 – Grünenthal, Lead Discovery Center GmbH (LDC), Max-Planck Innovation and Max Planck Institute of Experimental Medicine (MPI-EM) have entered into a research collaboration to develop novel therapies for patients suffering from Charcot-Marie-Tooth 1A (CMT1A), an inherited neurological disorder.

The collaboration combines the disease proficiency of MPI-EM, the drug discovery expertise of LDC and Grünenthal’s competency in drug discovery and development as well as pain management. The project’s scientific foundation was laid at MPI-EM by Michael Sereda, Klaus-Armin Nave and Moritz Rossner.

Within the framework of this research collaboration, LDC and MPI-EM intend to establish a novel screening platform to identify small molecule modulators to generate innovative drug candidates. These research efforts are equally funded by Grünenthal and Max Planck Society. Grünenthal will assume responsibility for the development of any drug candidates derived from this research collaboration from the identification of preclinical candidate onwards.

“It’s our aspiration to provide patients with disease modifying treatments. We therefore team up with academia and foster research collaborations leveraging basic research,” said Gabriel Baertschi, CEO Grünenthal. “There is no curative treatment available for patients affected by CMT1A. This neurological disorder places a burden on patients and we’re looking forward to developing potential treatment options with our partners.”

“We are enthusiastic to collaborate with Grünenthal and our MPI-EM colleagues on this early-stage drug discovery project”, said Bert Klebl, Managing Director and CSO at LDC. “As a leading specialist in pain research and pain management, Grünenthal’s synergistic capabilities will help advance this joint project to hopefully make a difference in the lives of CMT1A patients”.

About Charcot-Marie-Tooth
Charcot-Marie-Tooth is one of the most common inherited neurological disorders, affecting approximately 1 in 2.500 people. It affects both motor and sensory nerves and symptoms include weakness of the foot and lower leg muscles, foot deformities, pain, and numbness.

CMT1A is the most common form of CMT and results from a duplication of the gene on chromosome 17 that carries the instructions for producing the peripheral myelin protein 22 (PMP22). PMP22 is a critical component of the myelin sheath that isolates human peripheral nerve. Overexpression of this gene causes the structure and function of the myelin sheath to be abnormal, resulting in a slower conduction of nerve impulses. Currently, there is no curative therapeutic treatment available.

About Grünenthal
Grünenthal is an entrepreneurial, science-based pharmaceutical company specialized in pain, gout and inflammation. Our ambition is to deliver four to five new products to patients in diseases with high unmet medical need by 2022 and become a €2 bn company. We are a fully integrated research & development company with a long track record of bringing innovative pain treatments and state-of-the-art technologies to patients. By sustainably investing in our R&D above the industrial average, we are strongly committed to innovation.

Grünenthal is an independent, family-owned company headquartered in Aachen, Germany. We are present in 32 countries with affiliates in Europe, Latin America and the US. Our products are sold in more than 100 countries and approx. 5,200 employees are working for Grünenthal worldwide. In 2017, Grünenthal achieved revenues of
approx. €1.3 bn.

More information: www.grunenthal.com
Follow us on LinkedIn “Grunenthal Group”.

For further information, please contact:

Štěpán Kráčala, Head Global Communications
Tel.: +49 241 569-1335,
Stepan.Kracala@grunenthal.com
Grünenthal GmbH, 52099 Aachen, Germany

Public Relations LDC
Tel.: +49 231 9742 7000
pr@lead-discovery.de
Lead Discovery Center GmbH, 44227 Dortmund, Germany

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Innovative network of eight partners for North Rhine-Westphalia

Dortmund, 01.05.2018 – Drug Discovery Hub Dortmund (DDHD) is the name of the new initiative at the Zentrum für integrierte Wirkstoffforschung (ZIW) of the TU Dortmund University (TU), which got started on 1 April 2018. Scientists from eight institutions combine their manifold expertise to develop an infrastructure for drug discovery in North Rhine-Westphalia. In addition to the TU Dortmund, the Max-Planck-Institute for Molecular Physiology (MPI), the Leibniz Research Centre for Working Environment and Human Factors (IfADo), the Leibniz-Institut für Analytische Wissenschaften – ISAS – e.V., the Taros GmbH & Co. KG, the PROvendis GmbH, the BioMedizinZentrum Dortmund (BMZ), and the Lead Discovery Center GmbH (LDC) are participating.

The project has a total volume of 11 million euros over a 3-year-period. It is funded in the scope of the Forschungsinfrastrukturen NRW Program (NRW research infrastructures) of the state of North Rhine-Westphalia with funds from the European Fund for Regional Development (EFRE). The DDHD is to bridge the critical innovation gap between basic academic research and industrial application. Since the process of drug discovery is extremely complex, it requires an interdisciplinary cooperation of many different scientific specialist fields. Thus, the eight partners form a unique and well-proven network at the Dortmund site.

The DDHD acts as an incubator for drug discovery projects throughout North Rhine-Westphalia. In cooperation with the patent marketing organisation PROvendis, results of excellent basic drug discovery are to be translated into economic projects. Innovative findings made in basic academic research are to be transformed into lead substances compatible with industrial expectations, which specifically means that compounds are multiparametric optimised so that they fully comply with the high pharmaceutical standards. The molecular lead compounds will be available for further in depth scientific studies. In case of success, the projects are then transferred to the pharmaceutical industry and developed to marketability. Alternatively, individual projects may be transferred to spin-offs for further development. The source for such drug discovery projects are the universities and academic institutions of North Rhine-Westphalia.

“We are very pleased about the funding which enables the building of a unique infrastructure and the implementation of innovative projects“, states Prof. Daniel Rauh, DDHD Coordinator and Professor for Chemical Biology and Medicinal Chemistry at the TU Dortmund University. “In its capacity as an integrative initiative, the DDHD represents the scientific competence area of “Biomedicine and Drug Discovery” of the Dortmund Science Masterplan. It plays an important role in the strengthening of Dortmund as a biotechnology location and as an innovation center in North Rhine-Westphalia. Together with complementing projects, such as „Medicinal Chemistry in Dortmund“, funded by the Federal Ministry for Education and Research, the DDHD emphasises the national importance of Dortmund as a location for early-stage drug discovery.”

”The DDHD initiative will further strengthen the translational activities in Dortmund as well as the pioneering role of the location in early-stage drug discovery“, adds Dr. Bert Klebl, Chief Scientific Officer and Managing Director of the LDC. “The funding we received means to us a confirmation and support of the interdisciplinary cooperation between the different partners from academia and industry in Dortmund and in North Rhine-Westphalia.”

Contact Consortium:
Prof. Dr. Daniel Rauh
Chemical Biology and Medicinal Chemistry
E: daniel.rauh@tu-dortmund.de
T: +49 231-755 7080

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Dortmund/Munich, 23 April 2018 – The Lead Discovery Center GmbH (LDC) has established a branch – LDC Biologics – in the Munich Biocluster. While the team in Dortmund focuses on chemical compounds (small molecules), in Munich the emphasis will be on the development of therapeutic antibodies. LDC Biologics will produce, optimise and humanise innovative antibodies according to industrial standards in close cooperation with partners from academia and therefore connecting academic research and industrial drug discovery. The Munich team, under the management of Dr. Johannes Bange, can resort to more than 15 years of experience in the pre-clinical and clinical development of therapeutic antibodies.

With this branch office, LDC is also expanding its long-standing expertise in the pharmaceutical drug discovery with partners from academia and industry to include the development of therapeutic antibodies. This allows for the use of technologies and methods complying with the highest quality standards and industrial best practices at a very early project stage and creates a sustainable transfer solution for excellent innovative ideas from academia and the industry.

“With the expansion of LDC activities to include biological agents we are responding to the wish of our industry partners to offer a solution not only for chemical compounds, but also for biological ones. In doing this, we will continue with the established and proven LDC translation model of close cooperation with our partners from academia in respect of biological agents. The “LDC Biologics” department is ideally located in the Munich Biotechcluster, the centre of research of biological agents in Germany“, states Dr. Bert Klebl, Managing Director and Chief Scientific Officer of the LDC.

In addition, the entire drug discovery value chain at the Munich site is supported by LDC´s well-proven global pharmaceutical and investor network through licencing and/or a spin-off of new companies.
The new site of the antibody group is in direct proximity to the Martinsried Biotech Campus, which connects academic basic research and applied development.

“We are very pleased that LDC has located its biologics division in the Greater Munich Area. Our location provides the best scientists and an excellent LifeScience ecosystem, which has been established for years, for the successful translation from academia to medical use”, adds Prof. Horst Domdey, Managing Director of the BioM Biotech Cluster Development GmbH.
There is also a close interconnection with the newly founded ISAR Bioscience GmbH which will also cooperate closely with LDC in the future. LDC Biologics has already entered into active partnerships in the field of antibody development with local research facilities, such as Helmholtz Zentrum München – German Research Center for Environmental Health. One project of the LDC Biologics portfolio is funded by the Bavarian Ministry of Economy.

Contact

Dr. Johannes Bange
E.: Bange@lead-discovery.de

Public Relations LDC
E.: pr@lead-discovery.de
T.: +49 231 9742 7000

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