Kategoria: News

• The Independent Data Monitoring Committee (IDMC) recommended continuation of the global Phase II PoC study in patients with active pulmonary sarcoidosis without any changes to the current study protocol.
• The positive recommendation was issued following the IDMC’s review of the complete safety and efficacy dataset specified for the interim analysis in 30 patients and confirms that these results warrant continuation of the study with no modifications.
• Based on unblinded data, the IDMC conducted a futility analysis of safety and efficacy to determine whether the results obtained to date in 30 patients support continuation of the trial in its current form and indicate sufficient probability of achieving the primary endpoint, based on differences in PET/CT imaging, as well as some of the selected secondary endpoints.
• A preliminary biomarker analysis conducted by Molecure using blinded data showed inflammatory biomarker trends consistent with the expected biological response and the mechanism of action of OATD-01.
• The Company is currently in the process of randomizing additional 20 patients in preparation for the second and final interim analysis in the study, which will be based on data from a total of 50 patients and involve statistical re-evaluation of the required number of patients in the study
• The Committee’s positive recommendation should support further acceleration of patient recruitment and boost ongoing business development discussions concerning a potential partnering for accelerated development of OATD-01 towards Phase III and market.

 

Warsaw, 24 July 2026 – Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company discovering and developing drug candidates through the clinical stage and leveraging globally unique medicinal chemistry and biology capabilities to identify and develop first-in-class small-molecule therapies that directly modulate protein activity for the treatment of multiple currently incurable diseases, has received a positive recommendation from the Independent Data Monitoring Committee (IDMC) to continue the Phase II KITE clinical trial without changes to the protocol. The KITE trial is evaluating the efficacy and safety of the Company’s lead clinical asset, OATD-01, a first-in-class CHIT1 inhibitor, in patients with active pulmonary sarcoidosis.

The IDMC reviewed data collected following completion of the 12-week treatment period by the first 30 patients receiving either OATD-01 or placebo. The Committee recommended that the trial should continue without changes to the protocol.

“The IDMC recommendation is highly reassuring at this stage. The Committee recommended that KITE continue without any changes to the protocol, which suggests that, based on the statistical analysis of unblinded data, the study has a good chance of achieving its primary efficacy endpoint and meets the required safety criteria. We are extremely proud that a molecule discovered and developed entirely by the Molecure team has potential to become an effective treatment for pulmonary sarcoidosis among other indications. This recommendation confirms that the OATD-01 is progressing in line with our ambitious objectives. From a business perspective, we consider the IDMC recommendation, along with the trends observed in the analyzed (blinded) biomarkers and a favorable safety profile of OATD-01 to date, strengthens our position in ongoing partnering discussions” said Dr Marcin Szumowski, Chief Executive Officer of Molecure S.A. “The strong interest we received during the BIO International Convention in San Diego in June confirms the attractiveness of OATD-01 in inflammatory and fibrotic diseases. We held well over 30 meetings concerning OATD-01, including numerous returning partners under CDA that were particularly interested in the results of the first interim analysis. The positive IDMC recommendation serves as a catalyst for these discussions, and we believe it brings us significantly closer to entering a strategic partnership” added Marcin Szumowski.

 

Biomarker analysis is an important component of the relatively early stage of the trial. The Company analyzed blinded data from 23 patients, focusing on inflammatory biomarkers. The trends observed, considered together with the IDMC recommendation, suggest a demonstration of OATD-01 anti-inflammatory activity in line with multiple preclinical data. However, because the study remains blinded, no definitive conclusions can be drawn at this stage, and the observations cannot yet be attributed to the investigational drug or interpreted as confirmation of its efficacy.

“I am very gratified to hear about the positive IDMC recommendation and the progress in the KITE study,”  said Daniel Culver, one of the world leading lung sarcoidosis experts from Cleveland Clinic, one of the largest reference centers for pulmonary sarcoidosis. “I was also highly impressed with the biomarker data. These data are consistent with the possibility that there are between-group differences related to the study drug for biomarkers of sarcoidosis inflammation. It is exciting to see that a study for a novel mechanism of action and innovative trial design is making good progress  The clinicians and the patients are all  looking forward to seeing unblinded results when it is completed.” adds dr. Culver

We have entered an extremely exciting stage of the study, as it is the first time we have been able to analyze data from patients. Despite the limitations of the double-blind nature of the study the changes observed in selected inflammatory biomarkers are highly encouraging. For some of the parameters analyzed, we are seeing a biological response consistent with the proposed mechanism of action of OATD-01. Together with the IDMC recommendation to continue the trial without changes, these findings reinforce our confidence in the asset’s potential for further development. We also believe that this positive information will support further patient recruitment” said Dariusz Stencel, MD, PhD, Chief Medical Officer of Molecure S.A.

 

KITE study is an international, multicenter, double-blind, placebo-controlled Phase II clinical trial being conducted at nearly 30 sites in the United States, the European Union, Norway and the United Kingdom. Its objective is to evaluate the efficacy, pharmacokinetics and safety of a fixed 25 mg dose of OATD-01 administered over 12 weeks.

OATD-01 is a first-in-class CHIT1 inhibitor with therapeutic potential in diseases associated with significant unmet medical need, including interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, as well as metabolic dysfunction-associated steatohepatitis (MASH).

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About Molecure S.A.

Molecure S.A. is a biotechnology company discovering and developing drug candidates through the clinical stage. The Company leverages its proprietary and unique capabilities in medicinal chemistry and biology to identify and develop first-in-class small-molecule therapies that bind directly to previously unexplored protein targets and may offer new treatment options for multiple currently incurable diseases.

With the support of leading academic and research 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 Cell Biology in Warsaw (IIMCB), Molecure has built a diversified portfolio of distinct development programmes.

Molecure’s most advanced drug candidate is OATD-01, a first-in-class CHIT1 inhibitor being developed for the treatment of interstitial lung diseases, including sarcoidosis and idiopathic pulmonary fibrosis, as well as metabolic dysfunction-associated steatohepatitis (MASH). OATD-01 is currently in Phase II clinical development.

The Company’s second drug candidate is OATD-02, an oral, selective, first-in-class dual arginase inhibitor targeting ARG1 and ARG2 for the treatment of cancer. Its Phase I clinical trial commenced with the first patient dosed in the first quarter of 2023.

Molecure’s headquarters and laboratories are located in Warsaw and Łódź. The Company is listed on the Warsaw Stock Exchange under the ticker MOC.

Further information is available at: molecure.com

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA

 

• Molecure is focusing its financial and operational resources on the most promising clinical programs: OATD-01 and OATD-02, as well as on the development of a service using a drug discovery and development tool, co-financed under the FENG program
• The Company has decided to discontinue the development of the RNA platform and to suspend all research activities related to this project
• The decision to discontinue the program results from an analysis of the RNA platform development outcomes, which indicated that the assumed project objectives could not be achieved, particularly within the research methodologies and budgetary assumptions set out in the grant agreement and consequently that satisfactory commercialization of the RNA platform at this stage of development is not feasible
• Molecure will submit a final payment application to the Polish Agency for Enterprise Development (PARP) under the RNA project grant agreement (signed in December 2023). Based on its best knowledge, the Company believes it will not be required to return the funds already received from the PARP grant

Warsaw, 30 January 2026 r. – Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company discovering and developing drugs up to the clinical stage, leveraging unique global expertise in medicinal chemistry and biology to identify and develop first-in-class small-molecule drugs that directly modulate protein activity in the treatment of numerous incurable diseases, has decided to close the RNA project and discontinue further research activities in this area.

The decision to close the RNA program was preceded by a detailed analysis of the scientific results, which demonstrated the lack of feasibility for further cost-effective and scalable development of the project within the methodological and budgetary assumptions defined in the grant rules.

Molecure will promptly submit a final payment application to PARP and conduct a formal settlement of the grant. Based on its best knowledge, the Company believes it will not be required to return the received and utilized funds, amounting to approximately PLN 30.73 million. At the same time, the Company will not utilize or receive (other than the final payment) the remaining portion of the awarded grant, amounting to approximately PLN 22.35 million.

Molecure’s available financial and operational resources will be redirected toward the development of clinical programs and the implementation of an AI-based program supporting the discovery and development of new drugs.

„We have decided to focus our activities on projects that are most critical for building Molecure’s value, namely the OATD-01 and OATD-02 programs and the development of an AI-powered drug discovery platform. At the same time, following an analysis of the results achieved to date within the RNA platform, we concluded that, given the analytical methods stipulated in the grant agreement and the adopted budgetary assumptions, the project did not offer realistic opportunities for further development toward repeatable, scalable, and economically efficient solutions with commercial potential within a reasonably near time horizon, which ultimately determined our decision to discontinue it. These conclusions were also supported by discussions with potential partners, for whom the project was still at too early a stage, meaning that the timeframe for executing a transaction and generating more tangible revenues would, in our assessment, extend beyond the next few years. Initial achievements in the RNA project, including the attainment of in vitro proof-of-concept for two molecules, demonstrating effective binding to the intended mRNA fragment and inhibition of pathological protein translation, were very promising. However, particularly within the original research plan and project budget envisaged in the grant, we encountered difficulties in progressing to the next development stage and validating the assumptions at a larger scale using reliable and reproducible research tools. The decision to close the RNA program represents another step toward optimizing the use of financial resources at Molecure and maintaining strong capital discipline.

Focusing on the OATD-01 and OATD-02 clinical programs and on developing AI competencies offers the greatest potential for building long-term value for our Shareholders. We are pleased with the accelerating pace of patient recruitment in the first-in-class OATD-01 program (the world’s first chitinase inhibitor), particularly in recent weeks, which indicates that achieving the planned interim analysis results for a group of 30 patients in 1H 2026 is highly realistic. Our second clinical study – OATD-02, in which oncology patients receive our dual arginase inhibitor is also progressing in line with the clinical protocol and is approaching final results” – said Marcin Szumowski, President of the Management Board of Molecure S.A.

Molecure has commenced the implementation of the project titled “Development of a new small-molecule compound discovery service with therapeutic potential using artificial intelligence models” in a consortium with the Institute of Organic Chemistry of the Polish Academy of Sciences. The project received funding under the SMART Path. The grant agreement with the National Centre for Research and Development (NCBR) was signed in December 2025. The total project budget amounts to PLN 50.78 million, with total funding of PLN 35.72 million, of which PLN 27.03 million is allocated to Molecure. The project is planned for implementation between 2025 and 2029 and aims to significantly shorten timelines and reduce costs associated with drug discovery and development. Molecure expects that the developed service will also be offered to entities in the biopharmaceutical sector to support their drug development processes addressing unmet medical needs.

 

***

About Molecure S.A.

Molecure S.A. is a biotechnology company discovering and developing drugs up to the clinical stage, leveraging its proprietary and unique expertise in medicinal chemistry and biology to identify and develop first-in-class small-molecule drugs that, through direct binding to previously unexplored protein targets, may offer therapies for many incurable diseases.

Molecure has generated a diversified portfolio of independent programs with the support of leading academic research 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 Cell Biology in Warsaw (IIMCB).

Molecure’s most advanced drug candidate is OATD-01, a first-in-class CHIT1 inhibitor intended for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, as well as MASH, currently 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 commenced with first patient dosing 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 information, please visit: molecure.com

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA

Molecure S.A. is pleased to invite you to a meeting dedicated to the presentation of the progress of the ongoing research programmes, in particular clinical trials, as well as the development plans for the next quarters and the discussion of the Company’s operational and financial situation for 2024.

The meeting will be attended by:

– Dr Marcin Szumowski – Chief Executive Officer, President of the Management Board

– Dr Zbigniew Zasłona – Chief Scientific Officer, Member of the Management Board

– Dr Piotr Iwanowski – Chief Medical Officer, Member of the Management Board

– Sławomir Broniarek – Chief Financial Officer

_____

date: April 2, 2025 (Wednesday)

time: 13:00 | 1:00 pm (CET)

 

Link to the broadcast in English : https://livingmedia.com.pl/live/molecure/2024-en

Link to the original track: https://livingmedia.com.pl/live/molecure/2024-pl

 

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Important technical information:

to ensure the smooth running of the event, we recommend using:

– up-to-date browser version with javascript enabled

– open internet ports: 1935, 80, 443, 53

– an internet connection with a minimum actual bandwidth of 4Mbps

 

Please report technical problems to: wsparcie@livingmedia.pl

Warsaw, Poland and Durham, North Carolina, USA, July 31, 2024 – Molecure S.A. (‘Molecure’, SWE Ticker: MOC) and Avicenna Biosciences, Inc. announced today that they have entered into a strategic research collaboration to facilitate the discovery and development of novel small molecule drugs targeting ubiquitin-specific protease 7 (USP7), a deubiquitinase relevant in a number of tumors.  The collaboration will combine the extensive expertise of Molecure related to drug discovery and biology of USP7 with Avicenna’s machine learning-driven medicinal chemistry platform to identify novel drug candidates with superior pharmaceutical properties.

 

USP7 is an attractive target in cancer which regulates the stability of crucial proteins involved in pathways relevant for tumor suppression and immune response. Under the terms of the agreement, Avicenna will design algorithms to identify new chemical entities which inhibit USP7, and meet pre-specified pharmacological criteria. Molecure will be responsible for synthesizing and evaluating these new chemical entities and selecting one or more for further development. Molecure retains the right to develop and commercialize novel drug candidates resulting from the collaboration.

Avicenna, under the risk-sharing model, will receive an upfront fee and research funding, which will be contingent upon the generated compounds meeting the established success criteria. Additionally, if Molecure exercises the option to license the generated molecules, Avicenna will be eligible for license fees and will retain a specified share in the future revenues derived from drug candidates developed by Molecure.

 

Marcin Szumowski, Molecure’s CEO said: “By combining Molecure’s in-depth knowledge of this attractive, validated in vivo target with Avicenna’s ML-driven medicinal chemistry platform,  we intend to generate superior drug candidates with optimal pharmacological profiles for further preclinical and clinical development.”

Chris Meldrum, President & CEO of Avicenna, stated: “We have been very impressed with the Molecure team, their deep expertise in targeting USP7, and their efforts to deliver new treatment options to patients with difficult-to-treat cancers. Avicenna welcomes the challenge to transform chemical potential to biological and clinical reality for this important target.”

 

***

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 search for and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, may provide therapies for many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programs with support from 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, idiopathic pulmonary fibrosis and MASH. 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 at: https://molecure.com/pl/ 

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA

 

 

About Avicenna Biosciences

Avicenna was founded in 2020 by Drs. Thomas Kaiser and Pieter Burger out of the Liotta Research Group at Emory University.  The company is on a mission to solve the intractable drug design challenges that previously stopped drug candidates in their tracks.  Its machine learning-driven medicinal chemistry platform makes lead-to-drug optimization faster, cheaper and more successful – transforming sub-optimal clinical candidates into life-saving drugs.  Avicenna is backed by DCVC Bio. The company’s peer-reviewed research has been published in outlets such as Journal of Chemical Information and Modeling.  For more information, visit www.avicenna-bio.com and follow the company on LinkedIn.

 

Avicenna Media Contact

Kerry Walker

kerry@walkercomms.com

• OATD-01 is a first-in-class chitotriosidase 1 (CHIT1) inhibitor with the potential to modify the course of disease in sarcoidosis and other interstitial lung diseases.
• Obtaining regulatory approvals in Denmark, France, Greece, Germany, and Norway enables the conduct of the Phase II clinical trial of OATD-01 in these countries.
• First administration of OATD-01 in the European Union and Norway is planned for the third quarter of this year.
• Previously, Molecure received clinical trial approvals from the US FDA and the UK MHRA, with first patients dosed in March 2024 in the UK.
• First-ever administration of a chitotriosidase 1 (CHIT1) inhibitor is a significant milestone in the clinical development of Molecure’s leading program.

 

Warsaw, 21st May 2024 – Molecure S.A. (‘Molecure’, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage, leveraging 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 for the treatment of multiple incurable diseases, announces today that it has received national regulatory approvals from Denmark, France, Greece, Germany and Norway to conduct a Phase II clinical trial for OATD-01, a first-in-class chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential in pulmonary sarcoidosis.

 

Marcin Szumowski, Molecure’s CEO said: “An important milestone has been reached in the Phase II clinical trial of OATD-01 with the first patient administration in March this year at the Royal Infirmary in Edinburgh. We are pleased to receive further regulatory approvals from selected EU countries and Norway to conduct this breakthrough clinical study. The first dose is expected to be administered to patients in the selected countries in the third quarter of this year. The Phase II clinical trials to be conducted in both the USA and Europe, the two most commercially important markets, will enable us to collect the necessary data that, if positive, will confirm clinical proof of concept in a pulmonary sarcoidosis patient population that currently has few treatment options with limited efficacy in disease modification. OATD-01 has demonstrated remarkable potential in preclinical studies, suggesting it could redefine the standard of care for pulmonary sarcoidosis. The results of these studies will be crucial for further development and commercialization of OATD-01 in this and other potential indications such as NASH/MASH, IPF or inflammatory bowel disease (IBD). We look forward to presenting the unblinded results of this study in late 2025.”

 

The Phase II clinical trial for OATD-01 is a randomized, double-blinded, placebo-controlled, multicenter study to evaluate the safety and efficacy of OATD-01 in approximately 100 patients with active pulmonary sarcoidosis, including patients previously receiving other therapies with no clinical improvement and patients previously untreated.

Due to the requirement for double blinding in the study, the final unblinded results publication will occur after its completion and is scheduled for the end of 2025. To measure efficacy of OATD-01 in the study, an innovative primary endpoint has been agreed upon with the regulatory authorities, which is the response to 12-week administration of OATD-01, measured by the degree of granulomatous inflammation reduction in the lung parenchyma, assessed by PET/CT imaging. Following the completion of full dosing along with a monitoring period involving approximately 50 patients, an interim analysis (intermediate checkpoint) is planned to evaluate the statistical results by an independent committee and make decisions regarding the study’s continuation in terms of patient numbers in early Q1 of 2025.

The study will involve approximately 20-30 centers in the USA, European Union, Norway, and the United Kingdom. The renowned Contract Research Organization (CRO) responsible for organizing and conducting the comprehensive study is Simbec Orion.

 

About OATD-01

OATD-01 is an oral, once-daily, first-in-class, and highly selective CHIT1 inhibitor for potential use in the treatment of sarcoidosis. The CHIT1 enzyme is a promising molecular target due to its role in transforming local anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. Blocking CHIT1 activity by OATD-01 has resulted in documented anti-inflammatory and anti-fibrotic effects.

The OATD-01 molecule has shown strong anti-inflammatory and anti-fibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with unmet medical needs, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH), recently relabeled as Metabolic Dysfunction-Associated Steatohepatitis (MASH).

Molecure has obtained orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis and has received approval to initiate a Phase II clinical trial for the treatment of pulmonary sarcoidosis in the US, UK, selected countries of the European Union, and Norway.

 

About sarcoidosis

Sarcoidosis is a multi-organ disease of unknown etiology characterized by the formation of granulomatous structures in various organs, primarily in the lungs and lymphatic system.

It is a globally occurring disease affecting both men and women with an estimated incidence of 5-50 cases per 100,000 population, with 70% of patients being between 25-45 years old.

The most serious and common complication of sarcoidosis is pulmonary fibrosis, usually associated with significant impairment of lung function. Pulmonary fibrosis is the cause of most sarcoidosis-related deaths in Western countries.

 

About Molecure S.A.

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, leveraging its 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, can provide therapy for many incurable diseases.

Molecure has generated 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, 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.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancers, with Phase I clinical trials commencing with the first patient administration in the first quarter of 2023.

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

 

• In March this year, the Edinburgh (UK) clinic saw the first patient dosing of the drug or placebo in the Phase II clinical trial of OATD-01 (the KITE trial), a first-in-class chitinase inhibitor for potential use in inflammatory and fibrotic diseases 
• Two patients are currently dosed in the UK, and more are in the pipeline (screening) for inclusion in the Phase II clinical trial of OATD-01 for the treatment of pulmonary sarcoidosis 
• In the USA, the process of enrolling additional sites to conduct the OATD-01 clinical trial is at an advanced stage, with a total of three sites active there, which have started the recruitment process  
• In April, an application was submitted to the US NIH (National Health Institute) for US$2.2 million for funding for the US portion of the OATD-01 clinical trial and a grant of US$0.3 million was awarded by the NIH for the development of inhibitors of key signalling pathways responsible for the fibrosis process – a programme licensed from University of Michigan  
• Progress in the Phase I clinical trial of OATD-02, the first-in-class dual arginase inhibitor being developed for the treatment of cancer – commencement of drug administration to the 4th cohort of patients (20 mg dose). The aim of the study is to determine the maximum tolerated dose and the recommended dose for the next phase of trials 
• Consistent support of preclinical programme development processes with artificial intelligence (AI) tools, including for the designation of an official preclinical development candidate in the USP7 immuno-oncology programme for use in anti-cancer therapies 
• As at 31 March 2024, the Company had cash resources of PLN 52.4m  

Warsaw, May 17, 2024 – Molecure S.A. (‘Molecure’, WSE ticker: MOC), a biotechnology company discovering and developing drugs to the clinical stage 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 function to treat a range of incurable diseases, has released its Q1 2024 report. The report is available at: https://molecure.com/pl/informacje-dla-inwestorow/ 

 

Marcin Szumowski’s Comment, CEO of Molecure S.A. 

„In the first quarter, we focused on advancing the development of the OATD-01 and OATD-02 clinical programs, which are the most important elements of our pipeline, building Molecure’s value from a partnering perspective. OATD-01 is a drug candidate for the potential treatment of pulmonary sarcoidosis, a globally prevalent disease for which there are currently no effective therapies. In the second phase of the OATD-01 study, we plan to demonstrate that blocking chitinases in patients has a therapeutic effect and inhibits inflammatory processes. We have already commenced Phase II trials in the United Kingdom and are ready to enroll patients in several centers in the USA. A positive decision from regulatory authorities in European countries, which we anticipate, will also give us the „green light” to conduct Phase II OATD-01 trials in Denmark, France, Greece, Germany, and Norway. We plan to have the first readings of therapeutic efficacy signals in patients with sarcoidosis at the beginning of 2025. However, due to the requirement for a double-blind study, the final results will be published upon its completion and are expected at the earliest by the end of 2025. 

In our second clinical program, currently in Phase I, OATD-02, we are gathering data and gaining more knowledge from successive cohorts of patients. In April of this year, we began administering a 20mg dose of OATD-02 to the first patient and are now preparing to administer it to the second patient in the 4th cohort. We expect to have analyzed data from the fourth cohort by September of this year, and based on the recommendation of the Safety Review Committee, we will decide on the next dose level. The primary goal of our Phase I oncology study is to determine the safety profile of OATD-02, which is essential for determining the dose for the next phase. So far, we see that the data indicate a safe profile for the drug at the doses used and no significant adverse effects. As we obtain data from subsequent cohorts, we will also gain more indirect information about the antitumor activity of our compound. 

In the longer term, a key element of our pipeline is our small molecule drug discovery platform targeting mRNA. The first milestones we have achieved in this area validate the effectiveness of our methodology, generating the first molecules that successfully and selectively bind to and modify RNA function. This represents a true breakthrough and enhances Molecure’s development potential and long-term value. The breakthrough nature of this technology is evidenced by the fact that major partnering transactions are being concluded at the development stage we are currently entering. We are experiencing significant interest in our mRNA platform at leading global industry conferences and are actively building a service offering that meets the expectations of potential partners.” 

  

Business Development Activities 

• In the first quarter of 2024, company representatives participated in industry conferences, including BIO Europe Spring in Barcelona, where they held 38 meetings with representatives from the biotechnology and pharmaceutical industries, primarily potential partners, investors, and global pharmaceutical companies. The key discussion topics were OATD-01 (over 50% of the meetings), OATD-02, and the RNA platform. 
• The company will participate in further industry conferences this year, including major global events such as Bio International in San Diego and Bio Europe in Stockholm, as well as WASOG, AASOG, likely ESMO, and the RNA Assay Development & Screening Summit. 
  

 

Scientific publications 

• In March 2024, a scientific publication authored by the Molecure team (Zbigniew Zasłona, Katarzyna Drzewiecka) on the role of CHIT1 in cellular metabolism, titled „Metabolism-driven glycosylation represents therapeutic opportunities in interstitial lung diseases,” was published. Targeting enzymes that regulate protein glycosylation processes (including CHIT1) can effectively block immunometabolic changes leading to inflammation and fibrosis. This peer-reviewed review article was published in the prestigious journal Frontiers in Immunology.
  
  The publication is available at this link: https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2024.1328781/full 

The Molecure team is working on another (mechanistic) publication in collaboration with Professor Luke O’Neill, a world expert in innate immunity and inflammatory diseases, who joined the Molecure Scientific Advisory Board in 2023. 

 

Summary of financial data in Q1 2024 

• Operating revenues amounted to 0.03 million PLN, compared to 1.75 million PLN in Q1 2023*. 
• Operating expenses were 7.29 million PLN, compared to 8.65 million PLN in Q1 2023*. In Q1 2024, there was a decrease in operating expenses by 1.36 million PLN compared to the first quarter of 2023. The main factor for this change was a 1.02 million PLN reduction in expenditures on programs that, according to the new accounting policy, can no longer be capitalized under unfinished development work. Additionally, costs for external services not directly related to projects decreased by approximately 0.57 million PLN in 2024 compared to 2023. 
• Net loss amounted to 6.84 million PLN, compared to 5.89 million PLN in the same period of 2023*. 
• As of March 31, 2023, Molecure had cash resources of over 52 million PLN. Additionally, contracted grant funding for the coming years amounts to 32.5 million PLN. The company plans to secure further grants for projects within its developing pipeline of clinical and preclinical projects. 

*Data for the first quarter of 2023 has been restated for comparability due to changes in accounting policy. 

 

*** 

About Molecure S.A. 

Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its unique expertise in medicinal chemistry and biology to identify and develop first-in-class small molecule drugs. These drugs target previously unexplored protein and RNA targets, offering potential therapies for many incurable diseases. 

Molecure has generated a diverse 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, 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, currently 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 cancer treatment, with its Phase I clinical trial having begun with the first patient dosing in the first quarter of 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 websites: https://molecure.com/ 

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA       

• 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, March 29, 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 SOP 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 programmes  

-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.

 

***

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 2 clinical trials. The Phase 2 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

We are pleased to invite you to a meeting focused on a comprehensive review of the Company’s operational and financial performance in FY 2023, also including presentations on the progress of our research programs and forthcoming development plans.

The meeting will be conducted mainly in Polish. Simultaneous English translation will be provided. The anticipated duration of the meeting is approximately 90 minutes.

Presenting team:

Dr. Marcin Szumowski – Chief Executive Officer, President of the Board

Dr. Samson Fung – Chief Medical Officer, Member of the Board

Dr. Zbigniew Zasłona – Chief Scientific Officer, Member of the Board

Sławomir Broniarek – Chief Financial Officer, Member of the Board

_____

date: 10 April 2024 (Wednesday)

time: 2:00 p.m. (CEST) Warsaw

 

Registration link (with the option to ask questions in writing): https://livingmedia.com.pl/live/molecure/2023-en 

_____

Technical requirements

To participate in the video broadcast you need:

· Current version of browser with javascript enabled

· Open Internet ports: 1935, 80, 443, 53

· An internet connection with a minimum actual bandwidth of 4Mbps

Please report technical problems to: support@livingmedia.pl or k.tadeusiak@molecure.com

– OATD-01 is an innovative, first-in-class chitinase inhibitor for the treatment of sarcoidosis among other diseases where chronic inflammation leads to tissue remodeling and fibrosis

– In the phase II clinical trial (KITE study), patients will take a daily oral dose of 25 mg OATD-01 or placebo (in tablet form) for 12 weeks

– The double-blind, randomised, placebo-controlled study is designed to determine the clinical efficacy, pharmacokinetics, pharmacodynamics and safety of OATD-01

– The Phase II study will be conducted in the UK, US, and the European Union including Norway, and will involve approximately 100 patients

– OATD-01 is a first-in-class chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential in sarcoidosis and other interstitial lung diseases

Warsaw, March 22, 2024. – Molecure S.A. („Molecure”, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage and uses 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 translation to treat a range of incurable diseases, has started the clinical trial where OATD-01 is being administered to patients with active pulmonary sarcoidosis as part of a Phase II clinical trial (proof-of-concept in human). The world’s first administration of the chitotriosidase 1 (CHIT1) inhibitor (or placebo) to patient took place at the Royal Infirmary in Edinburgh. As part of the trial, patients will take a daily fixed dose of 25 mg OATD-01 or placebo tablets for 12 weeks. Patient safety will be monitored regularly through laboratory tests, neurological examinations and ECG and spirometry.

The Phase II clinical trial for OATD-01 is designed as a randomised, double-blind, placebo-controlled, multi-center study to evaluate the safety and efficacy of the oral CHIT1 inhibitor (OATD-01) in approximately 100 patients with active pulmonary sarcoidosis, including patients both previously receiving other therapies and previously untreated. The study will involve approximately 20-30 centres in the US, the European Union, Norway and the UK. The renowned CRO (Contract Research Organisation) Simbec Orion is responsible for the organization and comprehensive conduct of the study.

„The first administration to a patient with pulmonary sarcoidosis is the realization of our mission focused on helping and transforming the lives of patients with incurable diseases through the development of therapies and the opportunity to offer them new treatment options. Launched in the UK, the Phase II trial involving patients with active disease, the 'proof-of-concept in human’ phase, is a significant milestone in the clinical development of our lead program. It is a landmark moment also because it is the first time that a chitotriosidase 1 inhibitor has been offered to patients. Molecure is a pioneer and global leader in the development of therapies based on the inhibition of chitinase activity. The 'PoC in human’ study is key to validating this therapeutic approach, as there is a whole spectrum of diseases whose development is associated with similar molecular mechanisms. We will conduct the study in the United States, the United Kingdom, Norway and European Union countries, after obtaining approvals from the relevant EU regulators. The results of this study will be strategically important for further value creation and commercialization of OATD-01, and we look forward to the results of the study in 2025″ – said Dr Marcin Szumowski, Chief Executive Officer and Chairman of the Management Board of Molecure S.A.

Due to the double-blinding requirement of the study, publication of the final unblinded results will follow completion of the study and is anticipated by the end of 2025. For the efficacy trial, an innovative primary endpoint has been agreed with the FDA, namely the response to 12-week administration of OATD-01 as measured by the degree of reduction in granulomatous inflammation in the lung parenchyma, as assessed by PET/CT imaging. After approximately 50 patients have completed their participation in the study, a sub-analysis (intermediate checkpoint) will be scheduled to statistically evaluate the results by an independent committee and decide how to proceed with the study in terms of the number of patients.

„We are very proud of the first patient administration in the KITE study. In a broad spectrum of preclinical studies, we have confirmed the great potential of OATD-01 to become the new standard of care for pulmonary sarcoidosis. In phase 1 clinical trials involving 129 healthy volunteers, we in turn confirmed the good safety profile of OATD-01 and the compound’s ability to effectively block chitinases. In a phase 2 study, we aim to demonstrate that blocking chitinases in patients has a therapeutic effect and inhibits inflammatory processes. The primary endpoint of the study is the arrest or reversal of disease progression and lung damage as assessed by the granulomatous inflammation reduction score. The therapy also aims to alleviate the symptoms of the disease understood as an improvement in lung function, as measured by a change in the so-called Forced Vital Capacity (FVC), as well as an improvement in quality of life and a shift away from corticosteroids’’ – said Dr Samson Fung, Chief Medical Officer and Board Member of Molecure S.A.

 

About OATD-01

OATD-01 is an orally administered once-daily, first-in-class and highly selective CHIT1 inhibitor for potential use in the treatment of sarcoidosis. The CHIT1 enzyme represents a promising molecular target due to its role in converting local anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. Blocking CHIT1 activity by OATD-01 resulted in documented anti-inflammatory and anti-fibrotic effects.

The OATD-01 molecule has demonstrated potent anti-inflammatory and anti-fibrotic effects in various disease models and has high therapeutic potential in a variety of inflammatory and fibrotic diseases representing an unmet medical need, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH).

Molecure has obtained orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis.

About sarcoidosis

Sarcoidosis is a multi-organ disease of unknown etiology, characterized by the formation of granulomatous structures in various organs, mainly the lungs and lymphatic system.

Sarcoidosis is a globally prevalent disease, affecting both men and women with an incidence estimated at 5-50 cases per 100,000 population, with 70% of patients being between 25-45 years of age.

The most serious and common complication of sarcoidosis is pulmonary fibrosis. It is usually associated with significant impairment of lung function. Pulmonary fibrosis accounts for the majority of sarcoidosis-related deaths in Western countries.

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 search for and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, may provide therapies for many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programmes with support from 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, which is ready to enter Phase 2 clinical trials. The phase 2 trial 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 started 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 at: https://molecure.com/pl/

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA