- Initiation of Phase II clinical trial of OATD-01 (KITE) for the treatment of pulmonary sarcoidosis following approval from the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA).
- World’s first administration of a chitotriosidase 1 inhibitor (or placebo) to a patient with active pulmonary sarcoidosis as part of the Phase II OATD-01 clinical trial (proof-of-concept in human) at a hospital in the UK.
- First success in mRNA platform development – inhibited translation of pathogenic proteins in a cellular assay (in vitro proof-of-concept) – achieving an important discovery milestone in the development of a breakthrough small-molecule drug platform targeting mRNA.
- Further advances in the Phase I clinical trial of OATD-02, the first-in-class dual arginase inhibitor being developed for the treatment of cancer, systematic dose escalation and progression to a fourth cohort of patients with solid tumours, to determine the maximum tolerated dose and recommended dose for the next phase of trials.
- Further development of the most advanced project in the preclinical phase – the USP7 inhibitor programme for use in anti-cancer therapies using artificial intelligence tools and models.
Warsaw, April 3, 2024. – Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage and leverages its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function to treat a range of incurable diseases, has published its 2023 annual report. The report is available at: https://molecure.com/pl/informacje-dla-inwestorow/
“2023 was an exceptional year for Molecure as we made significant progress in the development of both clinical and preclinical programmes, particularly in the area of small-molecule drugs targeting mRNA. We initiated clinical trials of our drug candidate, which was discovered and brought into clinical trials for cancer patients by Molecure. We have made significant progress with OATD-02, a dual arginase inhibitor, and will soon begin administering the drug (at a dose of 20 mg) to a fourth cohort of patients with solid tumors. We are also seeing an increase in biomarkers suggesting a pharmacodynamic effect in the absence of significant side effects.
In 2023, our focus was also on preparing for a Phase II clinical trial for OATD 01, Molecure’s flagship clinical programme. We obtained FDA and then MHRA approval to initiate a Phase II clinical trial of OATD-01 in patients with pulmonary sarcoidosis. It is noteworthy that we went through the whole procedure very smoothly, which demonstrates the high quality of our research and documentation. We are very pleased to report that the first patient has started dosing in a clinical trial at a clinical site in the UK. OATD-01 is a first-in-class chitinase inhibitor developed by Molecure scientists for the treatment of sarcoidosis, which will be administered to placebo-controlled patients. We are shortly awaiting Phase II clinical trial approvals for OATD-01 from regulatory authorities in Denmark, France, Greece, Germany and Norway.
Last year was also a time to continue Molecure’s transformation into a cutting-edge company, applying the latest generative and predictive artificial intelligence techniques to our early drug discovery programmes, including a platform of small-molecule drugs interacting directly with mRNA targets. We achieved our first major success with Proof-of-Concept in vitro, confirming the inhibition of protein translation with compounds targeting the mRNA encoding the protein. We also adopted an approach to identifying the most promising preclinical projects, aiming to maintain a balanced portfolio of projects with strong clinical and transactional potential.
The last two years have been difficult for biotech companies due to the destabilisation of the geopolitical environment, inflation and interest rates, which slowed down partnership discussions and capital raising. Despite these difficulties, thanks to investors’ faith in the potential of our programmes, we successfully completed a capital round and raised approximately PLN 50 million in growth funding from investors through a share issue.
Our clinical development is incurring increasing costs, but we have our own funding and awarded grants that will allow us to continue with both clinical projects and those with the best earlier stage of development until at least early 2025. In parallel, we are applying for new grants to support projects and IT and computational methods, which will allow us to accelerate the development of programmes.
We are confident that pursuing our mission will open up new treatment opportunities for patients and provide a significant return on investment for shareholders. We thank you for your trust to date and encourage you to continue working with us.” – said Marcin Szumowski, CEO of Molecure S.A.
Presentation for investors
The Company’s presentation to investors will take place on 10th April 2024, at 2 pm (CEST) in an online meeting format, under the link: https://livingmedia.com.pl/live/molecure/2023-en
Selected key developments in 2023 and up to the date of publication of the financial report
- Strategy Update
The Company’s main strategic objectives in the area of R&D and business development are:- Continued intensive clinical development of two key projects: completion of a Phase II study for OATD-01 in sarcoidosis and completion of a Phase I clinical trial for OATD-02 in oncology patients, with possible expansion to additional indications and combination therapies
- Further development of early preclinical stage projects, including the identification of 1-2 advanced lead compounds (candidates for preclinical development) and the introduction of a further programme to the clinical trial stage
- Accelerate the development of a breakthrough platform of small-molecule drugs targeting mRNA, including the achievement of in vitro PoC and the selection of lead molecules
- Increasing the efficiency of drug discovery processes (by reducing time and cost and lowering the risk of failure) through investment in machine learning and generative artificial intelligence (GenAI) technology
- Conclusion of at least 1 high-value partnership agreement for at least one clinical-stage project, as well as the establishment of a number of commercial collaborations, including profit-sharing, for earlier-stage programmes
Investment expenditures, among others, for the implementation of the objectives set out in the Strategy in the period from 2024 to the end of 2025 have been set at approximately PLN 150 million.
- Successful completion of a public offering (SPO)
- Molecure has successfully raised gross proceeds of approximately PLN 50m from its public share offering from existing and new shareholders
- The funds raised from the SPO and expected grants will be used to fund the Company’s growth plans and continue to build a balanced portfolio of breakthrough therapies including first-in-class drugs, including the completion of a Phase II study of OATD-01 in sarcoidosis and the completion of a Phase I clinical trial of OATD-02 in oncology patients, with the potential to expand into additional indications and combination therapies
- Phase II clinical trial approvals for OATD-01 and initiation of the trial
- Molecure has received approval from the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to conduct a Phase II clinical trial for OATD-01 in the US and UK. The proof-of-concept Phase II study is the first to administer the drug to patients with pulmonary sarcoidosis. The world’s first patient administration of chitotriosidase inhibitor 1 (CHIT1) or placebo took place in March this year at the Royal Infirmary in Edinburgh. As part of the trial, patients will take a fixed dose of 25 mg OATD-01 or placebo daily in tablet form for 12 weeks
- Molecure has reapplied for permission to conduct a Phase II clinical trial of OATD-01 in EU countries: Denmark, France, Greece, Germany and Norway. In the previous coordinated assessment procedure, a refusal decision was issued by the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, and the effect of the refusal was that, despite receiving approval from the ethics committees in Greece, Germany, France, Denmark, Poland and Norway, it was not possible to conduct the clinical trial in the requested EU countries, as Poland was reported in the application as the rapporteur country. In the re-proceeding, Molecure proposed Denmark as a rapporteur country in the application assessment process.
- Phase I clinical trial OATD-02
- Achieving significant progress in the clinical development of OATD-02, an oral, potent and selective first-in-class arginase (ARG1 and ARG2) inhibitor, in cancer therapies
- March 2023 saw the administration of OATD-02 to the first oncology patient in a Phase I clinical trial to evaluate the safety, tolerability and preliminary efficacy of OATD-02 in patients with advanced and/or metastatic solid tumors
- Continued systematic dose escalation and planned imminent commencement of administration in a fourth cohort of patients with solid tumors, to determine the maximum tolerated dose and recommended dose for the next phase of the study
- Development of the mRNA platform
- Molecure confirms the efficacy of small molecules in inhibiting the translation of pathogenic proteins achieving a key milestone in the development of a breakthrough platform of small-molecule drugs targeting mRNAs
- In vitro proof-of-concept (PoC) assay confirms inhibition of protein translation with compounds targeting the mRNA encoding the protein
- The PoC stage for the first molecule developed in the mRNA platform provides evidence of the effectiveness of Molecure’s strategy to identify mRNA-binding compounds with therapeutic potential
- The success achieved in the development of the mRNA platform increases the likelihood of signing a collaboration agreement with partners working in this area
- Receipt of funding for a project to develop small-molecule drugs directly interacting with mRNA under the SMART pathway of the FENG programme (European Funds for a Modern Economy). The amount of funding is approximately PLN 32.5 million with a total project budget of approximately PLN 51.5 million. The project will be implemented between 2023 and 2028
- Business Development
- In 2023, Molecure representatives held a total of 107 meetings with biotech industry representatives in Europe and the US (Basel, London, Boston, Munich), mainly with potential partners, investors and global pharmaceutical companies. OATD-01 (37 meetings), OATD-02 (25 meetings) and USP7 (23 meetings) attracted the most interest. As a result of the meetings held, the Company signed 11 non-disclosure agreements (CDAs) with interested companies. This year, representatives of the Company were present at the BIO-Europe conference in Barcelona, which took place from 18-20 March
Key organisational changes in 2023 relevant to the growth and advancement of research programme
- Appointment of Dr Samson Fung – Chief Medical Officer to the Company’s Board of Directors to support the clinical development of OATD-01 and OATD-02
- Dr Zbigniew Zasłona has been promoted to Chief Scientific Officer from his previous role as VP Research Biology. Dr Zasłona remains a member of the Board of Directors of Molecure
Summary of financial data in 2023
- Operating income of PLN 1.3 million, up from PLN 1.6 million in 2022.
- Operating expenses were PLN 23.4 million, up PLN 4.8 million from 2022, due to the progressive advancement of the Company’s programmes and pipeline expansion. Increase mainly due to an increase in salaries and wages, increasing early-stage research costs and third-party service costs
- Net loss in 2023 amounted to PLN 18.3 million compared to a net loss of PLN 15.3 million in the same period in 2022
- As at 31 December 2023, Molecure had cash and cash equivalents of nearly PLN 64 million.
As at the date of publication of the Annual Report (29 March 2024), the Company has approximately PLN 54 million at its disposal – the Company has a stable capital position necessary to continue to finance the Company’s dynamic growth. In addition, contracted grant funding for the coming years amounts to PLN 32.5 million. The Company plans to obtain further grants for projects within the framework of the pipeline developed so far.
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About Molecure S.A.
Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, using its own unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, could provide therapies for many incurable diseases.
Molecure has generated a diverse portfolio of seven distinct programmes with the support of leading academic research institutions around the world, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw (MIBMiK).
The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter Phase II clinical trials. The Phase II study in sarcoidosis patients started in Q4 2023 in the US and the UK and will also continue in the EU and Norway, after gaining the appropriate regulatory approval.
The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, whose Phase I clinical trial has begun with first patient administration in Q1 2023.
Molecure’s headquarters and laboratories are located in Warsaw and Lodz. The company is listed on the Warsaw Stock Exchange (ticker: MOC).
Detailed information can be found on: https://molecure.com/pl/
LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA
