In H1 2024, Molecure focused on the development of two key molecules: OATD-01 and OATD-02, as well as expanding research activities based on, among other things, advanced artificial intelligence technologies
In May, Molecure received approval to initiate a Phase II clinical trial (KITE) for clinical candidate OATD-01 for the treatment of pulmonary sarcoidosis in the European Union and Norway. Earlier, Molecure received approval from the U.S. FDA and the U.K. MHRA
In the OATD-01 clinical trial, the Company currently has 13 sites activated in the US, the European Union, the UK and Norway. More will be activated by mid-November this year.
Progress in the Phase I clinical trial of OATD-02, the first-in-class dual arginase inhibitor being developed for the treatment of cancer. Following analysis of clinical and pharmacokinetic data and Safety Review Committee (SRC) recommendations, recruitment continues with the addition of 3 additional patients in the fourth cohort at the 20 mg dose level, with the goal of expanding the amount and scope of data to inform decisions on further dose escalation
Molecure has confirmed in in vitro tests the ability of the second molecule already being developed within the mRNA platform to effectively bind to the presumed mRNA fragment and block the translation process of the pathogenic protein. Reaching the PoC (in vitro proof-of-concept) stage for yet another molecule being developed in the mRNA platform is evidence of the effectiveness of Molecure’s strategy to identify mRNA-binding compounds with therapeutic potential.
In July, Molecure and Avicenna Biosciences entered into a strategic research collaboration agreement to optimize USP7’s small-molecule drug discovery and development processes. The collaboration will combine Molecure’s extensive drug discovery and USP7 biology expertise with Avicenna’s machine learning-based medicinal chemistry platform to identify new drug candidates with superior pharmaceutical properties.
In August, Dr Piotr Iwanowski, M.D., was appointed Chief Medical Officer (CMO) and Member of the Management Board of Molecure S.A., he will assume the above functions on September 30th this year.
Warsaw, 27 September 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 wide range of incurable diseases, has released its report for the first half of 2024. The report is available at: https://molecure.com/pl/informacje-dla-inwestorow/
Marcin Szumowski, CEO Molecure S.A.
„In the past six months, our main focus has been on the continued clinical development of OATD-01 and OATD-02. We continued to recruit to clinical trial of OATD-01, a first-in-class chitotriosidase 1 (CHIT1) inhibitor, in patients with pulmonary sarcoidosis on both continents. A positive decision by regulatory authorities from European countries, following earlier approval in the US and UK, has given us the “green light” to conduct Phase II trials of OATD-01 also in Denmark, France, Greece, Germany and Norway. We still hope that we will be able to involve Polish centers and Polish patients in this study. We are planning the first readouts of intermediate signals of therapeutic efficacy and safety in patients with sarcoidosis at the first half of 2025. However, due to the double-blinding requirement of the study, the publication of the final results will occur after the completion of the study and is scheduled for 2026. Our attention in the OATD-01 program is increasingly focused on the area of metabolic diseases, particularly MASH (metabolic dysfunction-associated steatohepatitis). We have very good preclinical results and there are a growing number of scientific publications from genetic studies and based on samples from MASH patients highlighting the key role of CHIT1 in disease escalation. The MASH market is projected to grow at more than 40% CAGR annually to a size of more than $25 billion in 2032. Molecure aims to play a significant role in treatment in MASH in partnership with a major company in the area of progressive metabolic liver disease. Scientific data and market projections support our assumptions about the therapeutic potential of blocking chitinases in multi-organ diseases.
In the Phase I clinical trial of OATD-02, an innovative arginase inhibitor, we are continuing to recruit and add 3 more patients to this cohort after analyzing data from the fourth cohort at the 20 mg daily dose level and the Safety Review Committee’s recommendation. As recommended by the SRC, we want to collect more extensive pharmacokinetics and pharmacodynamics data at this stage in order to decide whether to further increase the dose. We are confident that this next phase will bring us significantly closer to bringing breakthrough therapies to market that can change the lives of patients around the world.
We see great potential for a platform of small-molecule drug discovery that modulates mRNA function. In December 2023, we obtained positive in vitro results for the first of our molecules, and just a few months later we confirmed translation inhibition for another molecule targeting a different mRNA target. At the BIO International Convention in San Diego in early June, we had many meetings with representatives of Big Pharma companies interested in collaborating on the mRNA platform. In small molecule technology targeting mRNA, we are at the European and even global forefront, so we assume that establishing a commercially attractive collaboration with a large industry partner is likely within the next year.
Also noteworthy is the establishment of a strategic collaboration with Avicenna Biosciences to support the discovery and development of new small-molecule drugs targeting ubiquitin-specific protease 7 (USP7), a deubiquitinating enzyme that promotes oncogenesis and whose high expression is observed in a number of cancers. By combining Molecure’s expertise on this attractive, in vivo validated target with Avicenna’s machine learning-driven medicinal chemistry platform, we intend to generate better drug candidates with optimal pharmacological profiles for further preclinical and clinical development.”
Webinar with Molecure’s Management Board
A meeting will be held on October 1, 2024 at 2:00 pm to discuss the Company’s operational and financial situation for 1H 2024, including the presentation of progress in ongoing research programs, as well as development plans for the coming quarters. The meeting will be held in online format, at the link:
Original path – Polish:https://livingmedia.com.pl/live/molecure/1H2024-pl
English: https://livingmedia.com.pl/live/molecure/1H2024-en
Business Development activities
In the first half of 2024, representatives of the Company participated in two important industry conferences: BIO Europe Spring in Barcelona, where they held 38 meetings with biotech and pharmaceutical industry representatives, mainly potential partners, investors and global pharmaceutical companies. The key topics of discussion were OATD-01 (more than 50% of the meetings), OATD-02 and the RNA platform, BIO International Convention in San Diego, where they held a total of a record 42 meetings, of which as many as 28 were related to OATD-01, demonstrating the growing and increasingly concrete interest in the results of preclinical and clinical research in lung diseases and also in metabolic diseases such as MASH.
In addition, representatives of the Company participated in conferences: “Immunometabolism in Health and Disease” organized by Gordon Research Conferences at the American Bryant University in Smithfield, congress The European Congress of Life Sciences EUROBIOTECH in Krakow, Poland, International Conference on Medicinal Chemistry in Bordeaux, France, PHASE2024 in Warsaw, Americas Association of Sarcoidosis and Other Granulomatous Disorders (AASOG) in Baltimore, USA, IPF Summit in Boston, USA, and many others..
The company will participate in more industry conferences this year, including but not limited to major global events i.e. Bio Europe in Stockholm, as well as WASOG in India.
Summary of financial data in 1H 2024*
Operating income of PLN 0.4 million, up from PLN 1.8 million in 1H 2023.
Operating expenses amounted to PLN 15.6 million, compared to PLN 17.2 million in 1Q 2023. 1H 2024 saw a decrease of PLN 1.6 million in operating expenses compared to 1H 2023. The main factor for the change was a decrease of PLN 0.5 million in program expenses, which, in accordance with the new accounting policy, are no longer capitalized under uncompleted development work. In addition, costs of legal and consulting services decreased by about PLN 0.9 million in 2024 compared to 2023.
Net loss amounted to PLN 14.6 million compared to PLN 12.9 million in the same period in 2023.
As of June 30, 2024, Molecure had cash of more than PLN 38 million, and the end of September this year. PLN 27 million. In addition, contracted grant funding for the coming years amounts to PLN 32.5 million. The company plans to raise further project grants as part of its expanding pipeline of clinical and preclinical projects.
* Comparative figures for the first half of 2024 have been restated for comparability due to a change in accounting policy.
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About Molecure S.A.
Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its own unique expertise in medicinal chemistry and biology to explore 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 programs 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, Poland (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, and MASH, which is in phase II clinical trials.
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).
For more detailed information, please visit: https://molecure.com/pl/
LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA
