Strategic update on the progress and development of Molecure's key clinical projects in 2024

Molecure achieved key milestones in the development of its innovative therapies in 2024 and plans to accelerate research and development in its most advanced clinical programs: OATD-01 and OATD-02. A summary of the most important events of 2024:

• Initiation of Phase II clinical trial (KITE) for OATD-01 in the treatment of pulmonary sarcoidosis at centers in the USA and Europe. Randomization and administration of OATD-01 or placebo to several patients with pulmonary sarcoidosis have been completed.
• Efforts to initiate clinical development in MASH (Metabolism Associated Steatohepatitis) driven by strong preclinical results, independent research and projected growth of the MASH market to over $25 billion by 2032. Clinical development of OATD-01 for MASH treatment will be carried out in partnership.
• Progress in the first-in-human study of OATD-02, a key oncology program in Phase I, where no dose-limiting toxicity (DLT) has been observed in patients who have received the drug (during dose escalation). Positive recommendation from the Safety Review Committee (SRC) to continue recruitment and to include additional patients at the 20 mg daily dose level. In early 2025, after another positive recommendation from the SRC, Molecure decided to modify the first-in-human study protocol and submitted a request to the relevant authorities and ethics committees to move to higher doses than initially planned for new patients.
• Progress in the mRNA-targeted drug platform – two therapeutic targets validated (proof-of-concept in vitro), with a plan to select lead compounds. Molecure plans to enter into a collaboration or partnering agreement for these programs.
• Collaboration with Avicenna Biosciences in the USP7 program (deubiquitinase inhibitor in immuno-oncology) to optimize lead compounds using gen-AI tools. Molecure plans to select a preclinical candidate and accelerate partnering discussions to advance the program.
• Success of the stock issuance – raising approximately PLN 30 million (approximately $7.72 million as of March 27, 2025) and securing funding for the development of key projects until the first half of 2026.
• New roles on the Management Board – Dr. Piotr Iwanowski assumed the position of Chief Medical Officer and Member of the Management Board.
• Optimization of the organizational structure and reduction of discovery-phase programs from five to three, enabling the concentration of resources on the most promising clinical assets.

Warsaw, March 28, 2025 – Molecure S.A. (“Molecure”, GPW ticker: MOC), a biotechnology company discovering and developing drugs to the clinical stage, utilizing unique worldwide expertise in medicinal chemistry and biology to search for and develop first-in-class small molecule drugs that directly modulate protein activity and mRNA functions for the treatment of many incurable diseases, published its annual report for 2024. The report is available at: https://molecure.com/pl/informacje-dla-inwestorow/

Comment by Marcin Szumowski, CEO of Molecure S.A.

“The past year was a period of intense work, strategic decisions, and key progress in our clinical projects. We are consistently pursuing our vision of creating innovative therapies that address unmet medical needs for patients worldwide.

One of the major achievements in 2024 was the initiation and progress of the international KITE clinical trial for OATD-01, which includes patients with pulmonary sarcoidosis. Several patients have already been randomized in the KITE trial, and the recruitment pace is accelerating. We are currently recruiting at five centers in the USA, with additional sites opening soon in Europe. To speed up recruitment, we have strengthened our cooperation with the Foundation for Sarcoidosis Research and are conducting educational activities aimed at doctors and patients.

OATD-01 was also a key focus of partnering discussions. In 2024, we had several dozen meetings with potential partners, and we are now in advanced discussions with a number of them.

The KITE trial not only evaluates the efficacy and safety of OATD-01 but also provides crucial data on the CHIT1 protein – a biological target whose role may be significant in the pathogenesis of other diseases, including MASH (Metabolism Associated Steatohepatitis). According to forecasts, the MASH market is expected to exceed $25 billion by 2032, making this area extremely attractive for potential therapeutic applications of OATD-01.

At the same time, we are advancing our second key clinical candidate – OATD-02. Currently, dose escalation continues to determine the pharmacologically active dose (PAD) and maximum tolerated dose (MTD) of the drug. The primary goal of this phase of the trial is to assess the drug's safety, pharmacokinetics, and pharmacodynamics. So far, no dose-limiting toxicity has been observed in any of the 14 patients who received the drug, allowing for the continuation of the trial and dose escalation.

Financially, 2024 was a positive year for Molecure. Despite the challenging market situation, we raised about PLN 30 million from a stock issuance (approximately $7.72 million as of March 27, 2025). This investor support will allow us to execute the next stages of our trials, including accelerating recruitment for the KITE trial and conducting an interim analysis in the second half of 2025. We also prioritized our pipeline and optimized the organizational structure, leading to measurable savings and an extended cash runway.

We believe that 2025 will bring crucial milestones in our research programs that will strengthen Molecure’s position. Our priority is to enter into a partnering agreement for OATD-01, which will allow for further research in MASH and strengthen our financial position.”

Webinar with the Management Board of Molecure

On April 2, 2025, at 1:00 PM, a meeting will be held in Polish (with translation to English) to present the progress of the ongoing research programs, especially clinical trials, as well as development plans for the coming quarters and an overview of the operational and financial situation of the Company for 2024. The meeting will be held online, at the following link: https://livingmedia.com.pl/live/molecure/2024-en 

Development of Molecure's Preclinical Programs

In the USP7 project, in collaboration with Avicenna Biosciences, lead compound optimization continues, leveraging advanced computational methods and AI driven approaches.

New classes of compounds with more favorable pharmacological properties have also been identified. Currently, non-GLP toxicology studies are underway, and positive results will allow the official preclinical development of a selected molecule for oncology applications. Identifying a candidate for preclinical development will intensify partnering discussions.

In the mRNA program, Molecure is on track to select lead compounds for two mRNA targets, having reached the in vitro proof-of-concept stage, confirming the ability of small molecules to bind to mRNA fragments and inhibit the translation of pathogenic proteins. Plans for 2025 include selecting two lead compounds and further developing expertise in modern biophysical techniques, as well as entering into service collaboration or partnering agreements.

Business Development Activities

• • In 2024, representatives of the Company participated in three major industry conferences:
    
    • In March, at BIO Europe Spring in Barcelona, management held 38 meetings with representatives from the biotechnology and pharmaceutical industries, mainly with potential partners, investors, and global pharmaceutical companies. The key discussion topics were around OATD-01), OATD-02, and the RNA platform;
    • In June, at the BIO International Convention in San Diego, management held a record 42 meetings, with 28 focusing on OATD-01, indicating growing and more specific interest in the preclinical and clinical research results for lung diseases as well as metabolic diseases such as MASH. There was also interest in the RNA platform (6 meetings) and the OATD-02 program (8 meetings). Some of the meetings at BIO International Convention in San Diego concerned more than one program, reflecting a positive reception and interest in the Company's entire pipeline.
    • In November, BIO Europe in Stockholm, where the Company held 25 meetings with potential partners, primarily regarding OATD-01.
  • In March 2025, Company representatives attended the BIO-Europe Spring conference in Milan (March 17-19), where they held 30 meetings, including 22 focused on OATD-01, 4 on OATD-02, and 4 on other programs – USP7 and mRNA. Most of the meetings were with mid-sized pharmaceutical companies and large biotech companies. Some meetings were a continuation of previous interactions at BIO conferences.
  • Additionally, in the past year,  Company representatives participated in scientific and medical conferences such as "Immunometabolism in Health and Disease" at Bryant University in Smithfield, the European Congress of Life Sciences EUROBIOTECH in Krakow, the International Conference on Medicinal Chemistry in France, PHASE2024 in Warsaw, the European Respiratory Society (ERS) Congress in Vienna, the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) in India, the IPF Summit in the USA, and many others.
  • In 2025, Company representatives will attend several more industry conferences, including major global events such as Bio Europe (fall edition), BIO International, as well as WASOG, ERS, and the European Association for the Study of the Liver.

Summary of Financial Data for 2024 (Comparative 2023 data was restated)

• • Operating revenues amounted to PLN 2.9 million, compared to PLN 3.7 million in 2023.
  • Operating costs amounted to PLN 35.2 million (including PLN 4.0 million in costs related to the incentive program) compared to PLN 35.8 million in 2023. The slight reduction in operating costs was due to lower external service costs (including consulting services) and employee benefits.
  • The net loss amounted to PLN 31.3 million, compared to PLN 28.4 million in 2023.
  • Cash position: PLN 18 million as of December 31, 2024. Additionally, in Q4 2024, the Company raised funds from a stock issuance amounting to approximately PLN 30 million (capital increase registration took place in December 2024, and the funds were received by the Company in early January 2025)
  • The Company has secured financing from grants for the upcoming years amounting to approximately PLN 30 million and plans to secure additional funding for projects within its expanding pipeline of clinical and preclinical programs.

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About Molecure S.A.

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, utilizing its unique expertise in medicinal chemistry and biology to search for and develop first-in-class small molecule drugs that can directly modulate unexplored protein and RNA targets for the treatment of many incurable diseases.

Molecure has created a diversified portfolio of seven distinct programs with the support of leading academic institutions worldwide, 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 Cellular Biology in Warsaw (MIBMiK).

The most advanced drug candidate developed by Molecure is OATD-01, the first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, as well as MASH, which is in Phase II clinical trials.

The second drug candidate is OATD-02, an oral, selective, first-in-class dual arginase (ARG1 and ARG2) inhibitor for the treatment of cancer, whose Phase I clinical trial began with the first patient dose in Q1 2023.

Molecure’s headquarters and laboratories are located in Warsaw and Łódź. 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   

For further information, please contact:

Molecure S.A. (PR & IR)
Marta Borkowska                                            
Email: m.borkowska@molecure.com

+(48) 728 728 143