Category: News

Warsaw, 1 August 2024 r. – 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, announces the appointment of Piotr Iwanowski, M.D., Ph.D., as Chief Medical Officer (CMO) and Board Member of Molecure S.A., effective as of 30 September 2024.

 

„We are delighted that Piotr is joining the Molecure team. He is a high-level professional with many years of experience in the management, design, medical monitoring, oversight, and reporting of international clinical trials, with multiple interactions with regulators, including EMA and FDA. Piotr has been involved in the planning and execution of dozens of international clinical trials on all continents, both early and late stage. Several drugs from clinical trials in which Piotr had a significant impact at the operational level have already reached the market’’ says Marcin Szumowski, CEO of Molecure. ,,Piotr has already been co-operating with Molecure as a consultant in our clinical programs, scientific advise and other regulatory procedures. His expert knowledge and familiarity with Molecure’s research programs are key assets and will allow him to quickly establish himself in the effective management of Molecure’s clinical team, crucial in accelerating their progress and building Molecure’s value” – adds Marcin Szumowski.

 

„I am honored to have the opportunity to join such an innovative Polish biotechnology company. Having worked for more than 20 years in international teams involved in the clinical development of drugs and medical devices, I am delighted that I can now apply my experience in a domestic biotech company. I assume that my knowledge of clinical trials, both early and late phases, will be crucial to the development of the company’s promising portfolio. I am enthusiastic about the upcoming collaboration with the talented Molecure team. Together, we will accelerate innovations under development to deliver new, effective and safe therapies for patients in Poland and around the world” – says Dr Piotr Iwanowski.

 

Piotr Iwanowski is a doctor of medicine and an expert with extensive international experience in the clinical development of medicinal products and medical devices, which he acquired in various roles including senior medical positions. His responsibilities have included management of the clinical teams, interactions with principal investigators at clinical sites involved in ongoing clinical trials, study design, shaping the clinical development strategy, medical and regulatory supervision of clinical trial progress, scientific procedures with regulators, scientific publications, as well as support of business development processes from the clinical end.

Previously, Piotr served as associate vice president of clinical research in Europe at Wockhardt Bio and associate director of clinical operations in CEE and Turkey at Bristol-Myers Squibb. He also has several years of experience as a manager, monitor and auditor of clinical trials at Servier Poland. Since 2017, he has been a consultant physician at the Centre for Rare Diseases at the Children’s Memorial Health Intitute in Warsaw.

Piotr Iwanowski graduated in medicine and obtained his thesis in medical sciences at the Medical University of Białystok and specialized in clinical genetics. He completed postgraduate studies in medical law, bioethics and sociology of medicine at the University of Warsaw. He is fluent in Polish, English, French, German and Russian.

He has authored numerous scientific publications in the field of clinical drug development, including a co-authored publication with the Molecure team on arginase inhibitors.

He plays an active role in various scientific and educational organizations, including as a lecturer and faculty member for public grant application evaluation. He has also held positions in the Polish Association for Good Clinical Practice, including vice-president and board member.

 

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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 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 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 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 at: https://molecure.com/pl/

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

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.”

 

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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 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

– In December 2023, Molecure confirmed the inhibition of protein translation with compounds targeting the mRNA in an in vitro proof-of-concept (PoC) using a cell-based assay 

– This month, Molecure confirmed inhibition of the protein translation function for another mRNA target. Another small molecule discovered and synthesized by Molecure showed efficacy in an in vitro assay, expanding the number of selective binders in its mRNA discovery platform. 

– The achievement of the PoC stage for another molecule and a second target in the mRNA platform provides evidence of the effectiveness of Molecure’s strategy to identify mRNA-binding compounds with therapeutic potential 

– Strong biopharmaceutical industry interest in Molecure’s mRNA targeting technology increases the  commercial potential of Molecure’s mRNA discovery platform and the likelihood of signing a collaboration or partnership agreement. 

 

Warsaw, 12th June 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 search for and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA translation for the treatment of multiple incurable diseases, has confirmed in vitro the ability to bind to another mRNA target and demonstrated efficacy of a second molecule in inhibiting the translation mechanism of a pathological protein using compounds that target the mRNA encoding that protein. This is another success in the development of the mRNA discovery platform and one of the Company’s strategic goals for 2023-2025. 

 

„The discovery of another class of molecules binding to a second mRNA biological target, confirmed in in vitro assays, is the result of our team’s tremendous commitment and innovative approach to finding new molecules that modulate the translational function of mRNA. In December 2023, we obtained positive in vitro results for the first class of our molecules, and just a few months later we confirmed translational inhibition for another molecule targeting a different mRNA target. This attests to the broad scientific competence of our team and the high quality of our drug design processes, which we are improving through the use of advanced AI tools and computational methods. In the mRNA-targeting small molecule technology, we are in the European and even global vanguard and see great commercial potential for our mRNA discovery platform.  At the June BIO International Convention in San Diego, we had many meetings with representatives of companies interested in collaborating on discovering and developing small molecules targeting mRNA. Most of them were BigPharma representatives. We see many large partnership deals in this space signed at very early stages of drug development. In line with our strategy, this should allow us to intensify our partnering discussions and increase the likelihood of establishing a commercially attractive collaboration with a large industry partner. We are very pleased with multiple discussions we had during the recent BIO in the US with potential partners regarding our research program pipeline” – said dr Zbigniew Zasłona, CSO Molecure S.A. 

 

Molecure in the area of the mRNA platform is considering several commercialisation models, including a service model based on generating revenue from collaborating on the development of mRNA targets selected by the partner, which, through the use of a platform supported by AI tools, the Company can validate and then design and optimise molecules that interact with these targets. Another model is the classic licensing to a partner of Molecure’s newly discovered molecules that directly model mRNA function. Molecure is one of the few biotech companies in the world that develops small-molecule drugs that directly interact with mRNA targets.  

 

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.  

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    

• 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. 

 

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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 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

• Molecure has resubmitted an application for approval to conduct a Phase II clinical trial for OATD-01 in Denmark, France, Greece, Germany and Norway. The company has proposed Denmark as the rapporteur Member State for the application evaluation process. 
• In the previous coordinated evaluation procedure, the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products refused the request, and the consequence of this refusal was that, despite the approval of the ethics committees in Greece, Germany, France, Denmark, Poland and Norway, it was not possible to conduct the clinical trial in the requested European Union countries because Poland was reported in the request as the rapporteur Member State. 
• Following approvals from the US Food and Drug Administration (FDA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the relevant ethics committees in those countries to conduct a Phase II clinical trial for OATD-01, Molecure is progressing smoothly in preparation for the start of the clinical trial and is finalising the contracting of sites in the US and UK. 

Warsaw, 9th February 2024. – The company Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical development stage and leverages its globally unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function for the treatment of multiple incurable diseases, has submitted a new Phase II clinical trial application for OATD-01 in patients with active pulmonary sarcoidosis in the countries of the European Union and Norway, as announced, and Denmark has been proposed as the rapporteur Member State for the application evaluation process.  

In the previous proceedings, Poland was the rapporteur Member State, in accordance with the EMA’s recommendations and the Company’s proposal, but due to the strict limits imposed by Polish law on the dose of ionising radiation a patient can receive in a clinical trial, Poland ultimately refused to allow the trial to proceed, despite the Company providing documentation indicating that the radiation level in diagnostic imaging could be reduced below the limit indicated in the Ordinance of the Minister of Health of 11 January 2023 on the conditions for the safe use of ionising radiation for all types of medical exposure (Journal of Laws 2023, item 195), and indication of the specific authorised technology that makes this possible. 

„As announced, we have resubmitted an updated application for approval to conduct the clinical trial in the European Union countries and Norway. We have proposed Denmark as a rapporteur Member State in the new application, not least because the prevalence of this disease in Scandinavian countries, including Denmark, is one of the highest in relation to other European countries. We hope that our proposal will proceed smoothly, not least because the relevant authorities in the individual countries have already taken note of our proposal in the first procedure and the relevant ethics committees in all countries have agreed to the study. Following the approval of our application, we expect the first administration in the European Union and Norway to take place at the end of the second and beginning of the third quarter of this year. We were very keen to enable patients in Poland suffering from sarcoidosis to take part in the trial and thereby give them access to an innovative therapy with significant potential. We regret that we ultimately had to abandon the clinical trial in Polish centres due to the difficulty of performing the full range of medical procedures foreseen in the protocol, including PET/CT imaging. This diagnostic method is crucial for assessing the efficacy of the investigational drug and we chose it after consultation with a broad international group of advisors and key opinion leaders in pulmonology and nuclear medicine. It is worth noting that the first administration of OATD-01 will soon take place in both the US and the UK, where we have already received approvals from the relevant regulators and are completing the contracting of the last study sites. Poland, being the only EU country with such restrictive limits on diagnostic imaging that cannot be justified scientifically, effectively excludes many patients with non-oncological diseases from access to potentially groundbreaking experimental therapies that save their lives and health. ‘We would be happy to return to Poland if the current limits are changed or the Polish regulator agrees to use certified technology that reduces this irradiation.” – comments Marcin Szumowski, CEO of Molecure S.A. 

 

As designed, the Phase II clinical trial for OATD-01 is a multicentre, double-blinded, randomised, placebo-controlled study to assess safety and efficacy in approximately 90 patients with active pulmonary sarcoidosis. Due to the double-blinding requirement of the study, publication of the final unblinded results will follow its completion and is anticipated in the second half of 2025. 

The study has an established innovative primary efficacy endpoint. This is the level to which OATD-01 will reduce granulomatous inflammation in the pulmonary parenchyma after 12 weeks of administration, as assessed by PET/CT imaging. 

OATD-01 has shown disease-modifying potential in preclinical studies. Molecure believes this molecule could become a new medical standard for the treatment of pulmonary sarcoidosis. 

 

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 activity in various disease models and has high therapeutic potential in a variety of inflammatory and fibrotic diseases, the treatment of which represents an unmet medical need; such diseases include sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH)). 

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

 

About sarcoidosis 

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

Sarcoidosis is a globally prevalent disease, affecting both men and women with an estimated incidence of 5-50 cases per 100,000 people, with 70% of patients being between 25 and 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 development 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 currently entering Phase II clinical trials. The Phase II trial in sarcoidosis patients, a proof-of-concept study, will begin in Q1 2024 in the US and UK, and will also continue in the EU and Norway following approval from European regulators. 

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) being developed for use in cancer treatment, with a Phase I clinical trial commencing 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