Wings4innovation and KHAN-I are accelerating drug development with expertise and significant financial resources. Both play a crucial role in translating research findings into new medicines by providing both expertise and substantial financial support. Compared to the other two projects previously highlighted by LISAvienna, this project sees wings4innovation and KHAN-I taking a particularly active operational role. Peter Nussbaumer, Managing Director of wings4innovation and Khanu Management GmbH (General Partner of KHAN-I), oversees the project, leveraging KHAN-I’s extensive know-how and network, as well as the expertise of Lead Discovery Center GmbH (LDC), a professional incubator working closely with KHAN-I. Peter Nussbaumer’s extensive knowledge is built on a PhD in organic chemistry from TU Wien, years of experience as Executive Director Medicinal Chemistry at Novartis, and his leadership at LDC. He regularly discusses experimental data with Margot Ernst and Marko Mihovilovic to determine the next steps in the research process. Margot Ernst, a chemist with a deep interest in medical applications, has been with the Center for Brain Research at the Medical University of Vienna since 2001. She contributes her expertise in GABA-A receptors, as well as on biological and pharmacological aspects of the project. GABA-A receptors are large, complex biomolecules located in the cell membranes of neurons, particularly in the brain and spinal cord. They interact with the neurotransmitter GABA (gamma-aminobutyric acid), enabling signal transmission to target cells. The novel chemical lead structure at the heart of this project originates from Marko Mihovilovic, a chemist specializing in bioorganic synthesis, drug development, and medicinal chemistry. As Dean of the Faculty of Technical Chemistry at TU Wien, he actively drives research in these fields across various applications. The proposed lead structure is currently being optimized for further development.
FWF Doctoral Program as a starting point
Marko Mihovilovic on the origins of the project idea: “The project’s history highlights why wings4innovation and KHAN-I are so crucial for the funding landscape. It stems from fundamental research rooted in an FWF-funded doctoral program. Over an extended period, substances were developed and investigated for potential neuropsychiatric applications, leading to the discovery of a highly promising chemical compound that acts on GABA-A receptors. We refined this lead structure and synthesized a highly potent compound with an exciting activity profile.” The MolTag doctoral program, focused on the development and study of molecular drug targets, was a joint initiative of the University of Vienna, the Medical University of Vienna, TU Wien, and IST Austria, supported by the FWF (Austrian Science Fund) from 2011 to 2023. More than 70 PhD students were trained across 10 research groups. Margot Ernst adds: “The FWF funding allowed us not only to conduct interdisciplinary research and teaching but also to establish a high-caliber Scientific Advisory Board. We know that we are not just training students for academia but also preparing them for careers in the pharmaceutical industry. That’s why it was important for us to bring in experts directly from drug development through this advisory board. Peter Nussbaumer was a member of our Scientific Advisory Board, and ultimately, this project emerged from that collaboration. During one of our discussions within the doctoral program, in which Marko Mihovilovic and I were involved, Peter Nussbaumer suggested exploring whether findings on GABA-A receptors could be a good fit for wings4innovation. He was looking for an entirely new, patentable chemotype, a hypothesis on the mechanism of action, and ideas addressing medical needs. Once we had all these elements, we were able to apply for funding.” Marko Mihovilovic adds: “It was ideal to have wings4innovation available as a funding instrument to assess the translational potential of our new chemical lead structure. The data we generate in this project should enable us to secure an industry partner. With some luck, we might convince a company to advance this new substance class into the clinical pipeline and eventually bring it to market.”
Aiming for a more favorable drug profile
Margot Ernst on the project’s goal: “This project focuses on the development and testing of small molecules designed to selectively target a specific group of GABA-A receptors. Our aim is to make certain neuropsychiatric disorders more treatable by developing a therapeutic option with fewer side effects. The goal is a better drug profile – one that alleviates symptoms without causing severe side effects like those associated with benzodiazepines, which are commonly used for anxiety relief, sedation, sleep induction, and epilepsy treatment. Benzodiazepines, however, carry a high risk of dependence and withdrawal syndromes, which have become a major concern, particularly in the context of the opioid and benzodiazepine crisis in the United States.” Every year, over 100,000 people in the U.S. die – at least in part – because of uncritical, widespread prescription of addictive medications. Despite these risks, benzodiazepines remain the best treatment option for some indications, also in Europe. Benzodiazepines replaced barbiturates in the 1960s, as barbiturate overdoses were even more dangerous and often fatal. However, alternative benzodiazepine replacements have been sought for decades. Z-drugs, for instance, have been shown to have sedative effects, but they are less effective in reducing anxiety and muscle tension and come with their own set of side effects. Ernst explains: “To achieve our goal, we are working on compounds that are active at certain GABA-A receptors while showing no activity at others. Our hypothesis is that this approach will reduce side effects, even though addiction mechanisms are not yet fully understood at the molecular level. It remains unknown or unpublished which of the estimated 100 different members of the GABA-A receptor family (co-called subtypes) are most critical in substance dependence and how exactly the brain changes in response to addiction. Beyond receptor targeting, our compounds must also meet numerous additional criteria to be viable as pharmaceuticals – for example, they must not break down too quickly in the body and must be able to cross the blood-brain barrier. Through step-by-step structural modifications, we are systematically investigating structure-activity relationships to determine the limits of what can be altered in the structure to maintain the desired effect, and what cannot. With these hypotheses in mind, we are working toward a molecule that will hopefully be suitable for clinical use.” Such candidate substances must ultimately prove their effectiveness in preclinical and clinical trials.
A different approach to research
The pharmaceutical industry requires a solid foundation for the costly and time-consuming development of new drugs. Marko Mihovilovic explains: “In the pharma sector, we’re talking about investments worth several billion euros before a drug reaches the market. Naturally, this means we need to meet validation criteria that aren’t typically considered in academic research. However, wings4innovation and KHAN-I understand these requirements. Their role is to generate high-quality, value-adding data that serve as a solid basis for much larger investments needed to bring a product to completion. With these additional industry-grade data, academic inventions become more credible and move one step closer to real-world application. KHAN-I’s funding model differs fundamentally from all other grants currently available in the Austrian innovation ecosystem. In our case, most of the operational research is conducted externally, coordinated by wings4innovation/KHAN-I. The academic partners – after providing the initial, research results driving the project – focus primarily on the intellectual guidance of the project. This makes the project unique in several ways: No direct funding goes to the academic institutions – KHAN-I directly pays the contracted service providers. The goal is to prepare research findings for industrial application, which requires a different mindset and approach compared to academia. And instead of focusing on scientific publications, we concentrate on securing intellectual property through patents. This shift in thinking is fascinating to me, and it’s one reason why I enjoy working at TU Wien, where technology transfer is highly valued. Our goal is to ensure real-world application, to make sure industry partners recognize our findings as relevant and valuable and worth further development. The great advantage of this project is that by the time we submit it to wings4innovation, the core research work has already been done. We already have an invention, an innovation, and the collaboration with wings4innovation/KHAN-I focuses on guiding the next steps, mainly on an intellectual level. We discuss which experiments are needed, what the results imply, and how to move forward. Peter Nussbaumer understands the process of drug development inside out and has built a strong network, which we use to drive the project forward with the necessary experiments.”
Peter Nussbaumer adds: “This project is significant because it brings together two universities, integrating expertise in chemistry, biology, and pharmacology. I firmly believe this is the future – chemistry alone is not enough, nor is biology or pharmacology on their own. But through collaboration, the perspective changes entirely. With wings4innovation and KHAN-I, we are building a bridge to commercialization. We understand what the industry needs and incorporate that into our work. However, our approach is not about lecturing our partners – we engage in open, collaborative discussions about expectations and requirements. Right now, we are waiting for further results, and the ongoing exchange of ideas is invaluable. Everyone involved contributes insights, and this collaborative approach greatly benefits the project. We focus on our strengths and bring them together to push this innovation forward.”
Wings4innovation and KHAN-I bring in the best partners
"Our goal is to achieve the best possible results in collaboration with our academic partners. To do this, we allocate resources where they make the most sense in terms of content, quality, and cost. We have a broad network of service providers and directly cover external service costs from the project budget, so our academic partners don’t have to worry about administrative tasks," explains Peter Nussbaumer. In addition to the Lead Discovery Center (LDC) in Dortmund, which brings extensive expertise in drug design, the project involves a German provider for chemical synthesis and Vienna-based ChanPharm for biological testing. Margot Ernst describes the process: "ChanPharm receives the compounds, which are then tested on different GABA-A receptor subtypes and other key parameters. In our project meetings, we discuss which subtypes to analyze and how to interpret the results. These insights guide our decisions on what to develop, synthesize, and test next. Step by step, we move closer to our goals with each iteration."
Overcoming the biggest challenges
Peter Nussbaumer notes, "There were setbacks and delays along the way, especially due to the pandemic – at times, TU Wien was completely shut down. However, since our project incurs costs only when we commission external partners for experiments, rather than funding an in-house team from the project budget, we were able to extend the timeline without financial strain. The biggest challenge so far has been ensuring that our compounds can reach brain tissue, which is essential for the primary indications. This was a major hurdle, but through hypothesis-driven development steps, we’ve now managed to overcome it. The next step is advancing profiling and optimization in preparation for further studies."
Margot Ernst adds, "We now have a much better understanding of how derivatives of the compound initially introduced by Marko Mihovilovic need to be structured to cross into the brain. Achieving both the desired activity at GABA-A receptors and the necessary brain penetration turned out to be far more difficult than we initially expected. Despite these challenges, we are very satisfied with our progress. The project is still in an early phase when considering the full drug development cycle. During the hit-to-lead phase, the compound must be optimized to the point where it can be reliably used in pharmacological models. This requires iterative, collaborative, and critical evaluation of experimental data. The strong trust we have built over many years enables us to work in an open and highly productive setting."
Maximum flexibility with a clear exit-clause
The great flexibility of wings4innovation and KHAN-I, alongside their expertise and extensive network, are key strengths of this innovative support and financing tool. The decision on the level of involvement is made on a project-specific basis and is guided by the needs for professional, efficient project management. Unlike other financing vehicles, wings4innovation and KHAN-I allow even the therapeutic indication to be changed, should experimental data suggest such a shift. Peter Nussbaumer explains this extraordinary flexibility: "We seek the best application for excellent scientific results. We start with a specific therapeutic goal in a primary indication for the project. But we have the freedom to change that completely and adjust the project plan flexibly. These decisions are always based on the data generated. We continuously decide, based on the data, whether to continue as planned or adjust our approach. However, our approach is much more application-oriented than that of academic research projects, which aim not at commercializing research results but at generating new knowledge. Our shared goal as a consortium is definitely to make money from the project." This is only possible if enough exciting, robust data are generated and milestones are reached that can attract the interest of industry partners or are sufficient for building a spin-off. The agreement that the partners made at the start of the project outlines what share of any potential revenues each institution will receive. If a critical milestone is not reached and, based on the data, does not appear achievable in the future, the project is set to be terminated. Should the exit-clause be triggered, the confidentiality obligation will end, no patents will be filed, and the academic partners will receive all protocols and data produced with KHAN-I funding for publishing the results. Margot Ernst adds: "Scientifically, this is important, especially when something as promising as our new approach is at stake. If it ultimately doesn’t work, it is crucial that no other researchers test the same hypothesis and waste more money. The data from the project itself are extremely high quality and worthy of publication." As long as the project progresses well and moves toward drug development, patents will be filed first, and publication will follow later.
To be continued?
"Through wings4innovation and KHAN-I, it is possible to identify the potential for the commercialization of inventions and innovations that might otherwise go unnoticed. As researchers, we focus on our area of expertise in science. The detailed knowledge about the products needed on the market, and how they should be designed, comes from our partners," says Mihovilovic. He emphasizes: "This funding vehicle offers so many advantages, not only for academic institutions but also for Austria's economic landscape. It is crucial to ensure that academic institutions continue to have access to a targeted, specialized commercialization option in the future. The pharmaceutical development sector requires very specific expertise, and we have that expertise at hand here." This expertise is highly valued in Germany at hotspots such as Göttingen, Munich, Heidelberg, or the Ruhr area, and also provides universities and university hospitals outside of the Max Planck Society with access to the know-how of LDC, an extensive network surrounding drug development, and the financial resources of KHAN-I.
Thanks to wings4innovation, projects from Austria have received the necessary attention. Without this national point of contact for scouting and coordination with academic partners, researchers interested in funding their projects through KHAN-I would be competing with all the others trying to secure it. Mihovilovic adds: "Wings4innovation and KHAN-I are clear success stories. Despite COVID-19, we have managed to advance a number of projects here that would not have otherwise emerged or received support. I would be very disappointed if political decision-makers decided that Austria would not be involved in the launch of KHAN-II and that wings4innovation would be terminated. We would lose an innovative and novel instrument that has given exciting impulses to the research landscape in recent years and competently driven the commercialization of knowledge." The first closing for KHAN-II, amounting to 51 million euros, was recently completed – without Austrian involvement at this stage. A further closing is scheduled for later this year to reach the target volume of 100 million euros, providing Austria with an important opportunity to contribute and support the progress of drug development at local research institutions and start-ups.
About TU Wien
The TU Wien is Austria's largest research and educational institution in the field of technology and natural sciences. More than 4,000 scientists are researching "technology for people" in five main research areas at eight faculties. The content of the studies offered is derived from the excellent research. More than 26,000 students in 62 degree programs benefit from this. As a driver of innovation ranked among Austria’s top 10 regarding patents filed, TU Wien strengthens the business location, facilitates cooperation and contributes to the prosperity of society.
Additional information: https://www.tuwien.at/
About the Medical University of Vienna
Medical University of Vienna (MedUni Vienna) is one of the longest-established medical education and research facilities in Europe. With almost 8,600 students, it is currently the largest medical training centre in the German-speaking countries. With more than 6,500 employees, 30 departments and two clinical institutes, 12 medical theory centres and numerous highly specialized laboratories, it is one of Europe's leading research establishments in the biomedical sector.
Additional information: https://www.meduniwien.ac.at/
About KHAN-I and KHAN-II
The KHAN Technology Transfer Fund I GmbH & Co KG (KHAN-I) is an early-stage life sciences venture fund with 70 million euros in assets under management. Our mission is to create value through cooperative partnerships with academic innovators in Europe. KHAN-I focuses on first-class therapies for attractive markets with high unmet medical needs. The fund is managed by Khanu Management GmbH, an experienced team of professionals with a proven track record in early-stage drug development, academic spin-offs, as well as in the licensing of drugs and partnerships. Investors in KHAN-I include i) the European Investment Fund (EIF) with support from InnovFin Equity and financial backing from the European Union under the Horizon 2020 financial instruments and the European Fund for Strategic Investments (EFSI) as part of the Investment Plan for Europe, ii) Austria Wirtschaftsservice GmbH (aws) with funds from the Austrian Federal Ministry of Labour and Economy and the Austrian National Foundation for Research, Technology and Development, iii) the Max-Planck-Förderstiftung, and iv) the Thyssen'sche Handelsgesellschaft mbH. Additionally, KHAN-I maintains a preferred partnership with the Max Planck Society e.V.
At the beginning of February 2025, Khanu announced the first closing in fundraising for KHAN Technology Transfer Fund II GmbH & Co KG (KHAN-II), the follower fund of KHAN-I. As limited partners of KHAN-II, the European Investment Fund (EIF, Luxembourg), Akros Pharma Inc. (USA), the Max Planck Foundation (Germany), the Thyssen’sche Handelsgesellschaft (Germany), and KHAN-II Vermögensverwaltung committed in a first closing to invest a total of EUR 51 million for early-stage drug discovery investments. Khanu was established as general partner. An additional closing is planned this year to target a total of EUR 100 million. Assets will be sourced predominantly from academia in Germany (such as Max Planck Institutes, among others) and Europe, focusing on innovative therapeutic opportunities in indications with high unmet medical need.
Additional information: https://khanu.de/
About wings4innovation
In Austria, KHAN-I subsidiary wings4innovation GmbH (w4i) has been set up as a national contact point for scouting and project management. 20 research institutions from across Austria that conduct research in the life sciences have signed a framework agreement as cooperation partners with w4i and KHAN-I. At w4i, experienced experts contribute their know-how about the pharmaceutical market and their network to early-stage projects. w4i scouts and evaluates project ideas and, following approval by the w4i Advisory Board, proposes suitable projects to KHAN-I for funding. In funded projects, w4i coordinates the work packages on behalf of KHAN-I and in collaboration with the academic project team. As part of scouting, w4i also offers academic partners general advice on translational options of their results and hypotheses and on topics relating to industrial drug research and development.
Additional information: https://w4i.org/
In a position paper by the Austrian University Conference, Austria’s universities clearly advocate the continuation of w4i and KHAN, as this model closes a crucial gap in the Austrian science and funding landscape and creates great added value for Austria. The investment period of KHAN-I ended in 2024.
Contacts for inquires
Media requests:
- TU Wien: Florian Aigner | florian.aigner(at)tuwien.ac.at
- MedUni Wien: Johannes Angerer | johannes.angerer(at)meduniwien.ac.at
- Wings4innovation und KHAN-I: Anne-Kathrin Klebl | pr(at)lead-discovery.de
Technology transfer issues:
- TU Wien: Peter Karg | peter.karg(at)tuwien.ac.at
- MedUni Vienna: Claudia Ernst-Ballaun | claudia.ernst-ballaun(at)meduniwien.ac.at
Project specific questions:
- TU Wien: Marko Mihovilovic | marko.mihovilovic(at)tuwien.ac.at
- MedUni Vienna: Margot Ernst | margot.ernst(at)meduniwien.ac.at
- wings4innovation GmbH: Peter Nussbaumer | nussbaumer(at)w4i.org