Prous Institute for Biomedical Research: driving biomedical research in fragile X syndrome and other areas of the central nervous system

Prous-Institute
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Biomedical research has never before been bestowed with such a promising future. The combination of science and technology has expanded the horizon of therapeutic innovation and become a great ray of hope for patients.

Perhaps because of this, accelerating breakthroughs in relation to health is the primary goal of Prous Institute for Biomedical Research. Not only does this institution promote its own developments, but it is also linked with and invests in innovative scientific initiatives in which the protection of the Intellectual Property plays a key role.

In this interview, Dr. Josep Prous, Jr., Vice-president of Research and Development, tells us about how the knowledge that has been accumulated in over more than 50 years of family tradition has turned into an excellent driving force behind research in Biomedicine.

From Prous Science to Prous Institute for Biomedical Research

 

The surname Prous has a long-standing relationship with science. For those who are not familiar with it, what is the Prous Institute?

Prous Institute for Biomedical Research is a family-owned group with over 50 years of experience in the field of biomedical research and clinical practice. Our headquarters are located in Barcelona and we came to existence as a spin-off of Prous Science, a world leader in the creation of databases, publications and information systems intended for the international scientific and medical communities (with brands such as Integrity, Drugs of the Future or Timely Topics in Medicine).

Prous Science was acquired by Thomson Scientific in 2007, and it then became part of one of the largest professional information groups on a global level with brands as famous as Derwent or ISI.

How did Prous Institute start?

The beginning of the project came about under the guidance of the company founder, Dr. Josep R. Prous Snr., for the purpose of discovering new medicinal products by means of developing expert computational systems which allowed theoretical knowledge to first be acquired that could then be experimentally validated.

We understand that the project has served as a basis and assistance for the development of major inventions. Could you tell us about some of them?

The applied research programmes derived from these methodologies have led to new experimental drugs in different areas such as cancer, central nervous system diseases or metabolic diseases with multiple international patents. It should be mentioned that said expert systems have been implemented in a number of international regulatory agencies and pharmaceutical companies.

These are undoubtedly very important contributions. Do you work in conjunction with other institutions or organisations to develop your projects?

The different initiatives of Prous Institute have been invigorated in conjunction with internationally prominent entities in the health field and the participation in European research projects and consortiums in the field of human health.

In this context, the company grants the award called Prous Institute – Overton and Meyer Award for New Technologies in Drug Discovery, given by the European Federation of Medicinal Chemistry for stimulating biomedical research by means of the use of new technologies.

Finally, it should be highlighted that Prous Institute holds shares in and positions on the administrative boards of companies in the biomedical sector that are leaders in their respective specialities and combine scientific excellence with the intensive application of disruptive technologies.

How does Prous Institute contribute to the growth and development of these projects?

A unique and extremely important aspect comes from what we could call corporate empathy. It allows us to contribute our experience to specific situations which founders and their teams have faced and we have internalised throughout our historical evolution as a group. The creation of a company and value is a path that is full of challenges, and the sooner these challenges are identified and resolved, the greater the competitive edge the organisations we participate in will have.

Furthermore, access to an important source of contacts in public research centres, in the pharmaceutical industry, and in regulatory agencies allows us to build and speed up the future plans of our investees.

In summary, Prous Institute contributes to invigorating these projects by providing resources and knowledge resulting from the over 50 years of experience we have in the fields of health and biomedical research.

Seven disruptive projects in biomedical research

Let’s get into the details of those projects. Your portfolio today consists of seven organisations. How do you choose them?

The Prous group biomedical sector investment strategy corresponds to specific founding values. These are based on the identification of opportunities where synergies between scientific excellence and advanced technologies may lead to a relevant impact on human health.

While the organisations in which we invest vary significantly in terms of their final applications, they all combine the will to respond to unmet needs in the sector, the leadership capacity of their founders, a strong technological innovation component, and the capacity for growth and internationalisation.

In looking over that portfolio, what can you tell us about Accure Therapeutics?

Accure Therapeutics, which is the result of the recent merger between Bionure and iProteos, is a biotechnological company in the field of the central nervous system, with a portfolio of investigational drugs in four different areas: optical neuritis, multiple sclerosis, Parkinson’s disease and epilepsy.

The company has recently completed a funding round that will allow it to move forward toward proof of concept in patients in their different R&D programmes, including the BN-201 molecule which is about to start phase II trials in the area of optical neuritis.

And what about Bioinfogate?

Bioinfogate is a technological company that provides information technology- and data science-based solutions to problems derived from medical and pharmaceutical research.

At present, the company has successfully launched on the market its first product, OFF-X™, which allows professionals in the pharmaceutical industry, regulatory agents and academic researchers to determine the safety profile of drugs and their therapeutic targets from the discovery phase up until their use in clinical practice.

Some of its clients include global pharmaceutical companies, academic institutions and government agencies such as the US Food and Drug Administration (FDA).

What can you tell us about Chemotargets?

Chemotargets was founded as a spin-off of the Systems Pharmacology Research Group of the Medical Research Institute (Instituto de Investigaciones Médicas, IMIM) of Hospital del Mar.

Chemotargets has developed the CLARITYplatform, which is a set of unique artificial intelligence-based tools for identifying, optimising, and guaranteeing efficacy and safety profiles of new candidate drugs. The platform is widely used in companies in the international pharmaceutical sector and by medicinal product regulatory agencies, among which the US Food and Drug Administration (FDA) stands out.

The company is currently completing a funding round so that it may extend its activities to the discovery and development of new drugs in collaboration with prominent international research centres.

Artificial intelligence is also the basis of QUIBIM, correct?

That’s right. QUIBIM, which is a spin-off of Hospital de la Fe of Valencia, is a medical technology company specialising in artificial intelligence and imaging technologies applied to the development of biomarkers in the field of radiology.

The company has an AI algorithm and software platform, QUIBIM Precision, which extracts and quantifies specific disease biomarkers from medical images with high precision. This platform is used worldwide in hospitals, pharmaceutical companies and R&D centres.

The company has recently completed a funding round led by prominent international venture capital funds.

Virtual Bodyworks is also included in your portfolio. What does this company do?

Virtual Bodyworks is a spin-off of the EVENT Lab of the University of Barcelona and IDIBAPS, and is committed to improving health by means of the unique integration of computer engineering, neuroscience and psychology. The company specialises in immersive virtual reality focused on medical and psychological rehabilitation

Its various solutions are used in multiple clinical scenarios in collaboration with prestigious hospital centres and companies in the health sector.

Could you summarise the field in which MedBioinformatics Solutions is conducting its research?

MedBioinformatics Solutions, which is a spin-off of IMIM and UPF, develops software and associated consultancy services based on the DisGeNET platform. This computer platform consolidates scientific information as relevant as relationships between genes and diseases, and it has become a reference in the area of international biomedical research.

Finally, we are going to learn about the Connecta Therapeutics project in further detail. Could you give us an advance on what it consists of?

Connecta Therapeutics arose as a spin-off of Prous Institute for Biomedical Research. It is a biotechnological company that develops new treatments for unresolved medical needs of the central nervous system.

At present, the company is developing a first-in-class drug for the treatment of the fragile X syndrome, CTH120, derived from the intensive application of in-silico technologies in drug discovery.

The company has recently completed a funding round that will allow it to reach proof-of-concept in patients for said compound.

Connecta Therapeutics and fragile X syndrome

(From left to right) David Prous, Jordi Fàbrega and Josep Prous, founders of Connecta Therapeutics

How did this spin off come about?

Prous Institute for Biomedical Research applied its computational artificial intelligence tools for more than three years to evaluate over 30 molecular and cell targets related to neuroplasticity.

As a result of this research programme, a neuroplasticity modulator with polypharmacological action was discovered, yielding excellent results in in vivo preclinical models conducted at the Genomic Regulation Centre (Centro de Regulación Genómica) in Barcelona. In the in vivo tests, pro-cognitive effects, improved sociability and behaviour in mouse models, as well as normalisation of dendritic spines in different experimental models of disease were observed.

These very promising results led to the creation of the spin-off Connecta Therapeutics, for the purpose of expanding the development of the molecule to the proof-of-concept stage in patients in different pathologies and extending the product pipeline in the central nervous system field.

The company has recently managed to close a funding round that has allowed it to reach these milestones.

The first drug being developed by Connecta Therapeutics, CTH120, is a treatment for patients with fragile X syndrome (FXS). What does this disease consist of?

Fragile X syndrome, also known as fragile X chromosome syndrome or Martin-Bell syndrome is a genetic disease causing a significant cognitive impairment and a number of behavioural manifestations due to an X chromosome disorder.

It is the first hereditary cause of mental disability and the second cause associated with genetic factors after Down syndrome.

Today, it is diagnosed by means of DNA blood tests, but there is no specific treatment targeting the cause of the disease, only approaches which help to palliate the symptoms.

From the physiopathological viewpoint, like in other conditions of the central nervous system, a common network of proteins involved in synaptic and structural plasticity seems to be compromised. These changes in the synthesis of synaptic proteins cause changes in the morphogenesis of the neurons/dendritic spines, which are the basis of a number of pathologies.

Estimates tell us that this disease affects 1 out of every 4,000 men and 1 out of every 8,000 women. What impact will this drug have on patients’ lives?

Right now there is no definitive treatment for fragile X syndrome (FXS). The only help the patient gets is through education (for example, with speech and behavioural therapies and physical exercise) to develop the maximum potential of patients affected with the disorder and by means of using drugs that can treat associated symptoms.

It should be pointed out that in patients with FXS, neuroplasticity, that is, the property of the brain to recover and reorganise from a molecular and cellular viewpoint, is affected.

In the case of humans, what implications does a neuroplasticity disorder have?

Neuroplasticity is key to development and learning, and any neuroplasticity disorder would cause in the patients a reduced intellectual capacity (ranging from mild to severe mental impairment) and cause social and behavioural issues (attention deficit, hyperactivity, autism, etc.).

In this sense, the design of our drug is aimed at modulating neuroplasticity, as has been demonstrated in preclinical experiments, and therefore it is a therapeutic approach based on the physiopathological basis of FXS.

Finally, it should also be added that although the first indication targeted by compound CTH120 is fragile X syndrome, it could be widely applied in a number of neuroplasticity-related conditions, such as Rett syndrome, autism spectrum disorders and Down syndrome, among others.

1.7 million Euros were recently acquired in a funding round led by Inveready and the CDTI. What will that influx of capital allow you to do?

The funding round led by Inveready and the CDTI is aimed at obtaining proof-of-concept in humans for the CTH120 molecule. In this sense, this influx of capital will be complemented with the search for non-dilutive public financing.

The funding will allow the first clinical trial to be conducted in healthy volunteers administering the compound by oral route, with a dose scaling to validate its safety and to obtain initial information about the therapeutic dose to be applied to the future phase II trial, according to the following milestones:

  • Complete the study of the mechanism of action of candidate drug CTH120
  • Validate its effective dose
  • Synthesis and scaling of the compound under in GLP and GMP conditions
  • Conduct regulatory toxicology and ADME studies
  • Conduct the first clinical trial on safety in humans
  • Complete the phase IIa study in adult FXS patients

A research consortium has been defined for this purpose, in which Connecta will participate in collaboration with prestigious public research centres, including the Medical Research Institute (Instituto de Investigaciones Médicas, IMIM) of Hospital del Mar, the Genomic Regulation Centre (Centro de Regulación Genómica, CRG) or the University of California Riverside.

Industrial Property Protection: one of the key mainstays

How important do you think industrial property protection was in this funding round?

For any biotech company it is essential to have a large patent portfolio with solid coverage considering that the business model is based on the licensing or sale of the available patents. Therefore, in a funding round, IP aspects represent one of the four essential mainstays along with the business prospects, scientific innovation, and the capacity of the management team.

Where have you protected the CTH120 molecule up until now?

In the case of Connecta Therapeutics, the CTH120 molecule under development is protected by a family of patents granted in the United States, Japan, Europe and Canada, among other territories. It should be mentioned that it is a product patent, which means it has a much broader coverage. Furthermore, it should also be pointed out that the disease for which it is being studied at present, fragile X syndrome, is considered a rare disease, and this will allow the patent protection to be supplemented with exclusive protection periods on the market in the United States and Europe for 7 and 10 years, respectively.

Furthermore, all the results of the project which have the potential to be protected will follow the industrial property policy set by the company and will be incorporated in the patents portfolio.

From the outset of the project, ABG has provided all the experience and diligence of its team in defining the industrial and intellectual property strategy, the actions to be taken in different territories, and the decision-making for optimising the valuation of our innovations.

Enric Carbonell

Our IP attorney highlights:

“It has been an extremely interesting challenge to obtain product protection in over 20 countries for this development which shows tremendous potential for helping in the treatment of fragile X syndrome and other conditions presenting with cognitive learning, or memory deficits.”

Enric Carbonell

ABG IP
ABG is a leading European IP firm. ABG’s more than 60 professionals combine experience, outstanding technical knowledge and deep legal expertise to be at the forefront of IP law.
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