Molecure has reduced the number of active projects from seven to five, focusing resources on the most promising assets;
Key areas for further development and value building for Molecure are: OATD-01 as a potential breakthrough therapy for the treatment of sarcoidosis and MASH, and OATD-02 in trials in patients with solid tumors. Development of projects in the discovery phase powered by machine learning and generative artificial intelligence (GenAI) methods includes a small-molecule drug discovery platform that modulates mRNA translational function and the USP7 program;
Molecure’s strategic goal is to conclude a partnership agreement in the OATD-01 program, enabling it to initiate a Phase II clinical trial in its next indication, MASH (ang. metabolic dysfunction-associated steatohepatitis), with its partner as soon as possible;
Molecure’s OATD-02 clinical trial is nearing the determination of a maximum tolerable dose (MTD) or potentially therapeutic dose (Ph2RD), which should increase interest from potential oncology partners;
In active programs at an earlier stage of development, in early 2025 Molecure plans to identify a candidate for preclinical development in the USP7 project (a deubiquitinase inhibitor in immuno-oncology), followed by intensified partnering discussions in this program, and to identify lead compounds for two mRNA targets for which the Company has previously obtained proof-of-concept in vitro and to sign a collaboration or partnership agreement in the mRNA platform;
Molecure’s expenditures to complete both clinical trials and continue development of the most promising early-stage projects between Q4 2024 and the end of 2025 are estimated at about PLN 95 million;
Molecure intends to use four possible sources of financing: transaction proceeds such as up-front payment(s) on one or more partnering agreements, grants and subsidies, and equity issuances with possible use of debt instruments.
Warsaw, 15 October 2024 – Molecure S.A. (“Molecure”, WSE ticker: MOC), a clinical-stage biotechnology company that 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 translational function to treat incurable diseases, has updated its portfolio of research programs, focusing on its most promising clinical projects: OATD-01 and OATD-02, and the preclinical USP7 project and mRNA platform.
Reducing the number of projects that are in the early stages is intended to manage resources more efficiently and focus on clinical programs that have the greatest commercial potential. At the same time, Molecure is continuing promising preclinical projects such as USP7 and early-stage projects in its mRNA platform, in which the Company uses artificial intelligence (AI) to virtually screen and optimize drugs targeting mRNA.
„We have decided to update our research pipeline, focusing resources and efforts on the development and commercialization of projects that will bring the greatest increase in the Company’s value, contributing to our mission. We are getting closer to reaching a milestone in the development of OATD-01 in Phase II clinical trials, while aiming to determine the maximum tolerable drug dose (MTD) and/or recommended Phase II dose (Ph2RD) for OATD-02. Both molecules are being administered to pulmonary sarcoidosis patients and oncology patients in Phase II and Phase I clinical trials, respectively. Progress in both trials should reinforce interest from strategic partners in the coming year.
We see tremendous potential for value especially in the OATD-01 project, which, thanks to its unique and pioneering mechanism of action – blocking chitinase activity – has the potential to become a therapy in the area of unmet medical needs and diseases that affect a growing proportion of the population. The huge body of compelling preclinical data we have accumulated – ours and those of our partners – as well as literature data confirm that elevated chitinase expression leads to excessive macrophage activity, which in turn is responsible for the induction of many serious diseases, including sarcoidosis and MASH. Molecure is a clear leader and pioneer in the development of chitinase inhibitors, and we are the only company in the world to bring such a compound into clinical trials, currently at the proof of concept stage in humans, being tested in patients with active pulmonary sarcoidosis. We want to see preliminary confirmation of the efficacy of our compound through data from an interim analysis, which we plan for Q3 2025.
MASH disease, or steatohepatitis associated with metabolic disorders, in particular, is an extremely attractive therapeutic and commercial area. Over the past decade or so alone, the incidence rate of the disease has increased several to a dozen times, and the global market is projected to be worth more than $25 billion in 2032. Positive data confirming efficacy in part of the mechanisms responsible for the disease processes of MASH and the approval this year of the first drug in this indication, developed by Madrigal, have contributed to a more than 4-fold increase in the market value of this company and resulted in a significant increase in interest in MASH among big pharma and large biotech companies. We are determined to capitalize on the potential of our molecule and our strong data for development in precisely this indication, so we want to attract a partner who will support our research from the financial side, as well as further strengthen our expertise in the area of metabolic diseases. We are confident that success in Phase II clinical trials in one, and even more so in both indications (MASH and sarcoidosis) will lead to a multiplication of the value of our flagship molecule, and thus the Molecure” – says Marcin Szumowski, CEO, co-founder and shareholder of Molecure S.A.
Molecure is also advancing its most promising preclinical programs. In the USP7 project, among other collaborations with Avicenna Biosciences, the Company has overcome challenges in optimizing drug-like parameters for lead compounds (drug-like properties), and is developing a revised formulation of the drug. As a result of this work, a candidate is planned for official preclinical development in early 2025, followed by intensified partnering talks. It is worth recalling that in 2023, Exelixis acquired an exclusive global license from Insilico Medicine for the development and commercialization of the USP1 inhibitor (identified through its AI platform), a molecular target in the mechanisms of synthetic lethality. The up-front payment in this transaction was $80 million.
In the mRNA program, on the other hand, the Company is well on its way to selecting lead compounds for two mRNA targets for which it has reached proof-of-concept stage in vitro over the past 12 months, confirming the ability of small molecules to bind to mRNA fragments and efficacy in inhibiting translation of pathogenic proteins. The Company’s priorities in this area, having already achieved initial validation of the methodology’s efficacy and generation of functional in vitro molecules, are to identify lead compounds and enter into commercial service or partnering agreements.
Molecure’s ability to commercialize early-stage projects has been demonstrated through a deal with U.S. biotechnology company Ocean Biomedical, Inc. (NASDAQ: OCEA) in the YKL-40 program. On October 15, 2024, the Company entered into an exclusive license agreement with its U.S. partner covering the sale of rights to molecules from the YKL-40 program, including the lead compound OAT-3912, previously developed by Molecure at the early discovery stage, and a second patent for a molecular screening assay, allowing Molecure to continue its research program. The revenue potential for Molecure assuming the achievement of milestones in program development by Ocean Biomedical and the launch of the drug, according to the “bio-dollar value” formula, was set at approximately $32 million.
Recognizing the greatest commercial potential in clinical programs, Molecure has reduced the number of active programs in the discovery phase, closing the least promising program conducted under a know-how licensing agreement with the University of Michigan and Michigan State University (the Company plans to terminate this agreement, which will be done at no cost to the Company). In addition, development activities in the USP21 project have been temporarily suspended.
Molecure’s outlays for completing both clinical trials and continuing the development of the most promising early-stage projects in the period from Q4 2024 to the end of 2025 will amount to about PLN 95 million, including:
Clinical phase programs – approx. PLN 40 million
Discovery and preclinical development programs (including AI tools) – approx. PLN 16 million
mRNA platform (including AI tools) – approx. PLN 23 million
General and administrative expenses, including business development and IR – approx. PLN 16 million.
In parallel with the reduction of the Molecure pipeline, there was also a reduction in the workforce in the area of the three early programs, which translated into savings of more than PLN 3 million by the end of September this year, while by the end of 2025 the total planned savings from this will amount to about PLN 13 million.
Taking into account Molecure’s cash holdings (PLN 28.6 million as of the end of September this year) in order to complete the two clinical trials and continue the development of the most promising early-stage projects, the Company estimates a funding requirement of about PLN 70 million. To raise funds, Molecure intends to use four possible sources of financing: transaction proceeds such as up-front payment(s) from one or more partnering agreements, grants and subsidies, and issuance of equity and/or debt instruments.
Summary of financial data in 1-3Q 2024.*
Operating income of PLN 1.1 million (including a grant from the NIH and income from the Innovative Workers Credit), compared to PLN 3.3 million for 1-3Q 2023.
Operating expenses amounted to PLN 22.8 million, compared to PLN 25.2 million for 1-3Q 2023.
Operating expenses for 9Q 2024 were lower y-o-y by PLN 2.4 million, driven by lower expenses for programs in early stages of development (by PLN 1.3 million), as well as lower expenses for consulting services, including legal services (by PLN 1.0 million).
Net loss amounted to PLN 20.8 million, compared to PLN 18.4 million for the same period in 2023.
As of September 30, 2024, Molecure had cash and cash equivalents of PLN 28.6 million. In addition, contracted grant funding for future years is more than PLN 32 million. The company plans to attract further grants for projects within its developing pipeline of clinical and preclinical programs.
* Comparative figures for Q1-Q3 2023 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, Trinity College Dublin (TCD) 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, 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).
Detailed information can be found on: https://molecure.com/pl/
LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA
