First patient in the UK is dosed in the OATD-01 Phase 2 KITE study in pulmonary sarcoidosis

– 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 submitted a new application for approval to conduct a Phase II clinical trial for clinical candidate OATD-01 in the countries of the European Union and Norway

  • 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      

 

Molecure invites to the R&D Day – an online scientific and educational meeting on February 6, 2024

Warsaw, Poland – 29th January 2024 – Molecure S.A. (WSE ticker: MOC) a biotechnology company discovering and developing drugs to the clinical stage, utilizes world-class expertise in medicinal chemistry and biology to search for and develop first-in-class small molecule drugs that directly modulate the activity of proteins and mRNA functions in the therapy of various incurable diseases, presents the agenda for the Molecure R&D Day online meeting and introduces profiles of external experts (Key Opinion Leaders). The invited experts will discuss topics related to methods used to study RNA structure and interaction with small-molecule ligands during the meeting.

 

The Molecure R&D Day online is scheduled for February 6, 2024 (Tuesday), 13:00-15:30 CET.

 

Link to registration (option to submit written questions):

in English: https://livingmedia.com.pl/live/molecure/RD-Day-en

in Polish: https://livingmedia.com.pl/live/molecure/RD-Day-pl

Presentation: R&D Day 6.02.2024

The meeting will be conducted primarily in English, as well as in Polish (simultaneous translation to Polish will be provided), with the option to ask questions in both Polish and English.

 

The Molecure managers will discuss the progress of clinical programs and provide an overview of the deubiquitinase platform and mRNA platform. Additionally, invited external experts (Key Opinion Leaders) will present selected aspects related to methods used for studying RNA structure and interaction with small-molecule ligands

 

Agenda:

13:00-15:00

  • Development plans in 2024
  • OATD-01 i OATD-02 programs – 2024 a year of clinical studies
  • DUBs program – deubiquitinase as a significant group of therapeutic targets in oncology
  • Future of mRNA platform
  • “MoleCuring” – treatment of diseases through the utilization of mRNA targeting mechanisms – methods for the identification of compounds – Joanna Sztuba-Solińska, PhD
  • Study of RNA structure using methods such as DyRNA Thermometry and cryo-electron microscopy (cryo-EM) – Jakub Nowak, PhD

15:00-15:30

  • Q&A session

 

 

External experts (Key Opinion Leaders):

 

  • Joanna Sztuba-Solińska, PhD

An eminent specialist in the field of RNA recombination mechanisms, for many years she has been conducting research on the structure and function of coding and non-coding RNA. She has been affiliated with various institutions, including the National Institutes of Health (National Cancer Institute, Frederick, USA), where she investigated the relationship between the structure and function of viral RNA. Her work also involved identifying secondary and tertiary interactions of RNA motifs that modulate viral infections. Currently, she serves as the Principal Scientist at Pfizer in the Vaccines Research and Development Department.

  • Jakub Nowak, PhD

A specialist in biotechnology and molecular biology, with extensive scientific experience gained at renowned institutions such as Jagiellonian University, the University of Chicago, and the University of Edinburgh. He has worked as an application scientist, specializing in developing and supporting biophysical applications for biomolecular interactions and stability at NanoTemper Technologies. In his current research at the Małopolska Centre of Biotechnology at Jagiellonian University and within the Max Planck Research Group under the ERC project, he combines knowledge from the field of biophysics with his RNA expertise to develop innovative approaches to characterize RNA stability using state-of-the-art analytical systems.

 

 

Molecure S.A. Presenters:

 

  • Marcin Szumowski, PhD – Chief Executive Officer, President of the Board
  • Zbigniew Zasłona, PhD – Chief Scientific Officer, Board Member
  • Samson Fung, PhD – Chief Medical Officer, Board Member

 

 

About Molecure

 

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, which uses its own unique competences in the field of 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 be the therapy of many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic scientific 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 use in the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter phase II clinical trials. The phase II study in patients with sarcoidosis will start in 1Q 2024 in the United States and the United Kingdom, and upon obtaining approval from regulatory authorities, it will also be continued in the European Union and Norway.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for use in cancer treatment, whose phase I clinical trial began with its first administration to a patient 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

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

For further information, please contact:

Media and individual investors:

Michał Wierzchowski, cc group

tel. +48 531 613 067

e-mail: michal.wierzchowski@ccgroup.pl

Institutional investors and sell-side analysts:

Katarzyna Mucha, cc group

tel. +48 697 613 712

e-mail: katarzyna.mucha@ccgroup.pl

Molecure’s success in development of the mRNA discovery platform

Molecure confirms that small molecules designed to target mRNA inhibit translation of the encoded protein relevant in cancer

Proof-of-Concept has been demonstrated in a cell assay

Reaching the PoC stage for the first mRNA target provides evidence of the effectiveness of Molecure’s strategy in identifying mRNA-binding compounds with therapeutic potential

The success in advancing the mRNA platform increases the likelihood of signing a collaboration agreement with partners operating in this field

 

Warsaw, December 12, 2023 – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and mRNA targets to treat multiple incurable diseases, has confirmed in vitro that small molecule binding to the selected mRNA fragment inhibit the translation of the protein encoded by that mRNA. Confirmation of the mechanism of stopping the pathological proteins translation in a dose dependent manner, represents a significant milestone in the development of the mRNA platform and has been one of the strategic goals of the Company during 2023-2025.

‘The initial stage of scientific research in the mRNA platform involved identifying the structure of the mRNA region with high potential for binding small molecules (druggable regions). After confirming the functionality of this region, we conducted modeling of its tertiary structure, a necessary step to proceed to virtual screening. The binding of the best compounds to the mRNA fragment was confirmed using biophysical methods. The identified molecules, directly interacting with the mRNA fragment, demonstrated inhibition of the protein translation encoded by the given mRNA in a dose dependent manner. These results mark our anticipated Proof-of-Concept (PoC) for the first mRNA target in the platform. This big success achieved by our scientists validated of our ambitious approach for platform development. The next stage will involve evaluating the possibilities of continued preclinical and potentially clinical development of lead molecules optimized in this program. The company is also expanding its in-house expertise in identifying new mRNA regions that may serve as attractive therapeutic targets’ says Dr. Zbigniew Zasłona, Chief Scientific Officer, and Member of the Board at Molecure S.A.

 

‘We are proud to have achieved the in vitro Proof-of-Concept (PoC) for the first hit molecules developed in the mRNA platform. As the result of combining creativity, innovative approach, and the determination of our team in discovery of new molecules, leveraging strong in-house expertise in drug design using advanced digital methods. It confirms that we are among the global leaders in the field of mRNA-targeting small molecule drugs, a breakthrough technology with the potential to change the paradigm of treating many diseases, where the protein structure itself prevents direct interaction with small molecules (so-called undruggable targets). The development of the mRNA discovery platform, alongside our two clinical programs, is one of Molecure’s strategic priorities. Achieving the in vitro proof-of-concept this year, as outlined in our 2023-2025 strategy, is a significant milestone that we have reached and confirmed as planned. This will enable us to intensify partnering discussions and increase the likelihood of establishing commercially attractive collaborations with industry partners’ says Dr. Marcin Szumowski, CEO, and President of the Board at Molecure S.A.

The business model of the Company in the mRNA platform involves a hybrid approach. Molecure aims to develop its own proprietary projects targeting internally selected mRNA structures as well as provide services to external companies in the biopharmaceutical sector. These services involve validating mRNA fragments chosen by the client as therapeutic targets. This approach enables partnering and the generation of revenue as early as the optimization stage of active binders, i.e., after achieving in vitro Proof-of-Concept (PoC) for the relevant mRNA target.

Molecure is one of the few biotechnology companies globally developing small molecule drugs that directly interact with mRNA targets.

 

About Molecure S.A.

Molecure is a clinical stage biotechnology company that uses its world leading medicinal chemistry and biology capabilities to discover and develop first-in-class small molecule drug candidates that directly modulate underexplored protein targets and the function of RNA to treat multiple incurable diseases.

Molecure has generated a diverse pipeline of seven distinct programs with the support of leading academic life science institutions globally, 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 (IIMCB).

Molecure’s most advanced in-house drug candidate is OATD-01, a first-in-class dual chitinase inhibitor for the treatment of interstitial lung diseases, such as sarcoidosis and idiopathic pulmonary fibrosis, that is Phase II ready. A Phase II trial in patients with sarcoidosis is expected to start in 4Q 2023.

OATD-02 is the second candidate drug, an oral, selective, first-in-class, dual inhibitor of arginase (ARG1 and ARG2), intended for the treatment of cancers. The Phase I clinical trial commenced with the first patient dosing in 1Q 2023.

The headquarters and laboratories of Molecure are located in Warsaw and Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

Further details can be found on the following pages: https://molecure.com/pl/

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

Molecure Announces Third Quarter 2023 Financial Results and Pipeline Highlights

  • U.S. Food and Drug Administration (FDA) approval to conduct a Phase II clinical trial for novel chitinase inhibitor OATD-01 in pulmonary sarcoidosis with first patient expected to be dosed in fourth quarter of 2023
  • Continued enrollment in the Phase I clinical trial of OATD-02, an oral, first in class dual arginase inhibitor for the treatment of cancer with initial data expected early 2024
  • Successful Secondary Public Offering (SPO) raising gross proceeds of approximately PLN50 million (USD12 million) to fund the company through significant development milestones and value inflection points

 

Warsaw, Poland – 31 October 2023 – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases announces third quarter results for the period ended 30 September 2023. The full report in Polish can be found here  https://molecure.com/pl/informacje-dla-inwestorow/

 

Marcin Szumowski, CEO and President of the Management Board of Molecure said: “We have made substantial financial and operational progress during 2023 including the completion of a significantly oversubscribed secondary public offering. This will enable the company to deliver meaningful additional data which will be important in fulfilling the longer-term potential of our pipeline.

We have also achieved key milestones in clinical development with the dosing of the first cancer patient in our Phase I study with OATD-02 with anticipated initial results in the next few months. Additionally, we expect to dose the first pulmonary sarcoidosis patient in the Phase II study of OATD-01 before the end of the year.

Molecure is entering a very exciting stage in its development with a number of near-term value-creating milestones set to be achieved in our pipeline and we look forward to providing meaningful updates on our progress over the next year. “

Commercial & Operational Highlights in the third quarter

Successful Public Offering

  • Molecure successfully raised, through a Secondary Public Offering, gross proceeds of approximately PLN50m (USD12m) from existing and new shareholders,
  • Proceeds and expected grant awards will be used to fund and build Molecure’s first in class sustainable pipeline of breakthrough therapies through significant value inflection points including completion of the Phase II study for OATD-01 in sarcoidosis and completion of the Phase I clinical trial for OATD-02 in oncology patients, with the possibility of expansion into additional indications and combination therapies.

US FDA Clearance to conduct Phase II clinical trial – OATD-01

  • Molecure received US FDA Investigational New Drug (IND) approval for OATD-01 which will allow the company to conduct Phase II clinical trials in the US. The planned Phase II proof-of-concept study will be the first to treat patients suffering from pulmonary sarcoidosis and is expected to start in the fourth quarter 2023.
  • Molecure has submitted applications to the EMA and UK MHRA to initiate a Phase II clinical trial in the European Union and Norway and the UK respectively.
  • Molecure signed an agreement with Simbec-Orion, a leading global Clinical Research Organization which will conduct the clinical trial on behalf of Molecure. The Phase II trial will be conducted in the US and several European Union Countries and enroll 90+ patients with active pulmonary sarcoidosis.

 Nine Months Financial Highlights

  • Operating income of PLN1.3 million, in line with the same period in 2022.
  • Operating expenses totaled PLN16.3 million, an increase of PLN4.3 million vs the same period last year. This was mainly due to higher research costs as the company’s pipeline advances, higher salaries and costs of external services.
  • Net loss for the first nine months of the year totaled PLN11.5 million vs net loss of PLN9.4 million in the first nine months of the year in 2022.
  • As of September 30, 2023, Molecure had cash of nearly PLN85 million (US$20 million).
  • US$/PLN exchange rate 4.2 as of 30 September 2023.

 

 

 

 

ENDS

For further information, please contact:

Molecure S.A. (PR & IR)

Marta Borkowska                                  

Email: m.borkowska@molecure.com

+(48) 728 728 143

 

MEDiSTRAVA Consulting (Financial PR)                           

Frazer Hall, Sandi Greenwood

molecure@medistrava.com

+44 (0)203 928 6900

 

About Molecure

 

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, which uses its own unique competences in the field of 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 be the therapy of many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic scientific 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 use in the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter phase II clinical trials. The start of a phase II trial in sarcoidosis patients is expected in Q4 2023.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for use in cancer treatment, whose phase I clinical trial began with its first administration to a patient 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

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

Molecure Announces First Half 2023 Results – Significant Financial and Operating Momentum

  • Molecure strategy update for 2023-2025 based on the development of a diversified portfolio of innovative research projects with the potential for breakthrough therapies for incurable diseases
  • Completed successful Secondary Public Offering (SPO) of gross proceeds of approximately PLN 50 million (USD12million)
  • Proceeds will fund the company through significant development milestones and value inflection points
  • First patient dosed in Phase I trial with OATD-02, a novel, first in class dual arginase inhibitor for the treatment of cancer
  • U.S. Food and Drug Administration (FDA) approval to conduct a Phase II clinical trial for OATD-01, a first-in-class inhibitor of chitotriosidase 1 (CHIT1)
  • Applications also submitted to European Medicine Agency and UK MHRA to conduct Phase II clinical testing of OATD-01

 

Warsaw, Poland – 29 September 2023 – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases announces first half results for the period ended 30 June 2023. The full report in Polish can be found here.

 

Marcin Szumowski, CEO and President of the Management Board of Molecure said “Many events of this year were crucial for further development of Molecure. In June, we announced the updated strategy for 2023-2025, the implementation of which will enable us to continue building the company’s value. In a very difficult market for biotech, attracting investor interest leading to high oversubscription in our secondary public offering was clearly a great accomplishment in our quest to build a clinical stage biotechnology company able to change the fate of patients. This year we have made excellent progress developing our first in class product portfolio and have achieved key milestones throughout this year including dosing the first cancer patient in our Phase 1 study with OATD-02, and receiving FDA approval to proceed with clinical trials in the US for OATD-01.

Bringing two of our most advanced programs to patients opened a new and exciting chapter for Molecure. I would like to thank our shareholders for their trust and the entire Molecure team for their dedication and unwavering commitment to improving the lives of patients. We eagerly await the initial read outs from our clinical trials and look forward to achieving further milestones in the development of our balanced pipeline, which we hope will ultimately lead to commercial success through strategic partnership agreements.”

Investor Presentation

The Company’s first half results presentation for investors will be held on October 3, 2023 at 2:00 PM (CET) in an online meeting. Link https://livingmedia.com.pl/live/molecure/2Q2023-en

The meeting will be conducted mainly in Polish and partly in English with simultaneous translation. It is expected to last approximately 90 minutes.

Commercial & Operational Highlights in H1 and post-period

  • Significant progress made on OATD-02, an oral, potent and selective first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer
  • First patient dosed in March 2023 in Phase I clinical trial to assess safety, tolerability and preliminary efficacy of OATD-02 in patients with advanced and/or metastatic solid tumors
    • lnitial clinical data expected at the end of 2023
  • Publication in Molecular Cancer Therapeutics, a journal of the American Association of Cancer Research, entitled “Arginase 1/2 inhibitor OATD-02: from discovery to first-in-man setup in cancer immunotherapy”. Link here
  • In June, the publication of an update to the Strategy for the years 2023-2025. The Company’s primary strategic objectives in the areas of R&D and business development are:

– Continuation of the intensive clinical development of two key projects: completion of the Phase II study for OATD-01 in sarcoidosis and completion of the Phase I clinical trial for OATD-02 involving oncology patients, with the potential for expanding into additional indications and combination therapies,

– Further advancement of early-stage preclinical projects, including the identification of 1-2 lead compounds (candidates for preclinical development) and the initiation of another program into the clinical trial phase,

– Acceleration of the development of a groundbreaking small molecule drug platform targeting mRNA, including achieving in vitro Proof of Concept (PoC) and selecting lead molecules.

– Enhancement of the drug discovery processes efficiency (by reducing time and costs and mitigating the risk of failure) through investments in machine learning technology and generative artificial intelligence (GenAI),

– Execution of at least 1 high value partnering agreement for at least one project in the clinical phase, as well as the establishment of a series of commercial collaborations, including profit-sharing arrangements, for programs in earlier stages of development,

The total investment expenditure to achieve the goals outlined in the Strategy for the period from mid-2023 to the end of 2025 has been estimated at approximately PLN 250 m.

  • In July, Molecure successfully raised, through a Secondary Public Offering, gross proceeds of approximately PLN50m (USD12m) from existing shareholders,

– These proceeds and expected grant awards will be used to fund and build Molecure’s first in class sustainable pipeline of breakthrough therapies through significant value inflection points including completion of the Phase II study for OATD-01 in sarcoidosis and completion of the Phase I clinical trial for OATD-02 in oncology patients, with the possibility of expansion into additional indications and combination therapies.

 

  • We continue to make excellent progress with OATD-01:

– In July Molecure, received US FDA Investigational New Drug (IND) approval for OATD-01 which will allow the company to conduct Phase II clinical trials in the US. The planned Phase II proof-of-concept study will be the first to treat patients suffering from pulmonary sarcoidosis and is expected to start in the fourth quarter 2023.

– Molecure has also submitted applications to the EMA and UK MHRA to initiate a Phase II clinical trial in the European Union and Norway and the UK respectively.

Molecure has signed an agreement with Simbec-Orion, a leading global Clinical Research Organisation which will conduct the clinical trial on behalf of Molecure. The Phase II trial will be conducted in the US and several European Union Countries and enroll approximately 90 patients with active pulmonary sarcoidosis.

Key organizational changes to drive the Company through its next phase of growth and clinical development

  • Samson Fung, Chief Medical Officer appointed to the Company’s Management Board to support the clinical development of OATD-01 and OATD-02
  • Zbigniew Zasłona, promoted to Chief Scientific Officer from his former position as VP Research Biology. Dr. Zasłona, remains on Molecure’s Management Board

 

First Half Financial Highlights

  • Operating income of PLN1.0 million, in line with 1H 2022.
  • Operating expenses totaled PLN10.9 million, an increase of PLN2.2 million. This was mainly due to increasing research costs as the company’s pipeline advances, higher salaries and costs of external services.
  • Net loss for the first six months of the year totaled PLN7.4 million vs net loss of PLN7.1 million in 1H 2022.
  • As of June 30, 2023, Molecure had cash of nearly PLN 50million (US$11.5 million).
  • As of the publication date of the 1H 2023 report (September 29), the Company’s cash position amounted to approximately 85 million PLN, taking into account the funds raised from the recently finalized public offering (US$19.5 million).
  • US$/PLN exchange rate 4.35 as of 30 June 2023.

 

ENDS

For further information, please contact:

Molecure S.A. (PR & IR)

Marta Borkowska                                  

Email: m.borkowska@molecure.com

+(48) 728 728 143

 

MEDiSTRAVA Consulting (Financial PR)                           

Frazer Hall, Sandi Greenwood

molecure@medistrava.com

+44 (0)203 928 6900

 

About Molecure

 

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, which uses its own unique competences in the field of 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 be the therapy of many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic scientific 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 use in the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter phase II clinical trials. The start of a phase 2 trial in sarcoidosis patients is expected in early Q4 2023.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for use in cancer treatment, whose phase I clinical trial began with its first administration to a patient 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  https://molecure.com

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

Invitation to investor meeting 3rd October 2023, 2:00 pm (CET)

We are pleased to invite you to a meeting focused on a comprehensive review of the Company’s operational and financial performance during H1 2023, also including presentations on the progress of our research programs and forthcoming development plans.

 

The meeting will be conducted mainly in Polish and partly in English. Simultaneous interpretation 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: 3 October 2023 (Tuesday)
time: 2:00 p.m. (CEST) Warsaw

Registration link (with the option to ask questions in writing): https://livingmedia.com.pl/live/molecure/2Q2023-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

Molecure submits application to the European Medicines Agency (EMA) to initiate a phase II clinical trial in the European Union and Norway to investigate OATD-01 for the treatment of pulmonary sarcoidosis

  • OATD-01 is a first-in-class inhibitor of chitotriosidase 1 (CHIT1) with disease-modifying potential in sarcoidosis and other interstitial lung diseases
  • EMA approval will enable clinical trials to be conducted in centers in the European Union, including several sites in Poland, and in Norway
  • In July 2023, Molecure received approval from the U.S. Food and Drug Administration (FDA) to conduct a phase II clinical trial for OATD-01 in the U.S., expected to start in the fourth quarter 2023
  • In September, the Central Institutional Review Board approved the Company’s application, which enables the qualification of sites conducting clinical trials in the USA

Warsaw, September 8, 2023 – Molecure S.A. (“Molecure”, GPW ticker: MOC), clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases, has applied to the European Medicines Agency (EMA) for permission to initiate a phase II  study  for OATD-01, a first-in-class and potentially disease-modifying chitotriosidase 1 (CHIT1) inhibitor. EMA approval will enable Molecure to start a phase II clinical trial in the European Union and in Norway to evaluate the safety and efficacy of OATD-01 for the treatment of pulmonary sarcoidosis.

“This is an important step for Molecure, bringing us closer to the start of a phase II clinical trial for OATD-01 in Europe. The European Union – after the USA – is the largest regulated market where we intend to conduct a clinical trial for our innovative CHIT1 inhibitor. said Marcin Szumowski, President of the Management Board of Molecure S.A.

“Administration of the drug to the first patient with pulmonary sarcoidosis is scheduled for the fourth quarter 2023. We will start the study in the USA at the same time, having received FDA approval in July, and where we have the approval of the American bioethics committee to qualify medical centers where the study will be carried out.

“The clinical development of our leading program is entering a very important clinical proof-of-concept stage and the protocol of this study has gained wide approval among eminent specialists in the field of lung diseases. We expect the completion of the US study together with the final report in 2025. I am convinced that OATD-01, which has been shown to modify the course of the disease in preclinical studies, has the potential to become the new standard of care for pulmonary sarcoidosis. This is another step forward in the implementation of our mission – to improve the health and quality of life of patients struggling with incurable diseases. We believe that our drug, if trials are successful, could change the fate of patients with pulmonary sarcoidosis” – added Mr. Szumowski.

The phase II clinical trial for OATD-01 is designed as a multicenter, randomized, double-blind, placebo-controlled trial to evaluate safety and efficacy in approximately 90 patients with active pulmonary sarcoidosis.

For the efficacy study, an innovative primary endpoint was established with the FDA, i.e. response to 12-week administration measured by the degree of reduction of granulomatous inflammation in the lung parenchyma, assessed by PET/CT imaging. In the middle of the study, i.e. for about 50 patients, an intermediate checkpoint is planned in order to assess it statistically and decide on the further course of the study in terms of the number of patients – this should take place 2024. The disclosure of the final trial results is anticipated in 2025.

 

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 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 shown strong anti-inflammatory and anti-fibrotic effects in various disease models and has high therapeutic potential in a variety of inflammatory and fibrotic diseases that represent an unmet medical need, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH).

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

About sarcoidosis

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

Sarcoidosis is a globally occurring disease, affecting both men and women with an estimated incidence of 5-50 cases per 100,000 inhabitants, with 70% of patients between the ages of 25-45.

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

 

For further information, please contact:

Molecure S.A. (PR & IR)                                      

Marta Borkowska

Email: m.borkowska@molecure.com

(+48) 728 728 143

 

MEDiSTRAVA Consulting

Frazer Hall, Sandi Greenwood

+44 (0)203 928 6900

Email: molecure@medistrava.com

 

About Molecure S.A.

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, which uses its own unique competences in the field of 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 be the therapy of many incurable diseases.

Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic scientific 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 use in the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter phase II clinical trials. The start of a phase 2 trial in sarcoidosis patients is expected in early Q4 2023.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for use in cancer treatment, whose phase I clinical trial began with its first administration to a patient 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).

Detailed information can be found at: https://molecure.com/pl/

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

The U.S. Food and Drug Administration Gives the Green Light to Molecure’s Flagship Program – Phase II Clinical Testing of OATD-01 is Ready for Launch

  • Molecure can proceed with clinical trials in the U.S.A – Phase II proof-of-concept study will be the first to treat patients suffering from pulmonary sarcoidosis with OATD-01
  • In the coming weeks Molecure will also seek the European Medicine Agency’s approval to conduct phase II clinical testing of OATD-01 in the European Union
  • OATD-01 is the first-ever chitotriosidase 1 (CHIT1) inhibitor with disease modifying potential in sarcoidosis and other interstitial lung diseases

22 July 2023, Warsaw – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases, announces that, has received of its Investigational New Drug (IND) Application from the U.S. Food and Drug Administration (FDA). This IND will allow the company to conduct phase II clinical testing of OATD-01, the first-ever chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential. The first pulmonary sarcoidosis patients in this Phase II study are scheduled to begin receiving treatment in Q4 2O23.

“We are extremely excited to share this great news – not only for us, but for every patient looking for a better and more effective way of treating sarcoidosis. This marks the beginning of a new chapter in the development of our flagship project, as we enter human proof-of-concept and begin treating pulmonary sarcoidosis patients with OATD-01. As we have confirmed in a range of preclinical studies, OATD-01 has the ability to modulate macrophage activity meaning it has the potential to treat various inflammatory and fibrotic diseases which develop based on a similar molecular mechanism. In the coming weeks we are also planning to file for approval of phase II clinical trials with the European Medicines Agency (EMA). That would make it possible for us to begin testing OATD-01 in the European Union – including Poland. We expect to conclude these Phase II clinical studies in mid-2025 with the publication of a report containing analyzing the headline data.” – says Marcin Szumowski, CEO of Molecure S.A.

In recent  months we’ve put a lot of effort into raising the profile of our clinical research plans with OATD-01 internationally – a process designed to build relationships with renowned clinical experts who specialize in lung diseases (including sarcoidosis), as well as foundations and other organizations which build communities to support patients suffering from a range of difficult diseases that have a significant and negative impact on the quality of their lives. The Molecure team has also participated in numerous seminars and conferences such as the World Association for Sarcoidosis and Other Granulomatous Disorders (WASOG) 2023 – the largest international meeting place focused on the future of sarcoidosis and other granulomatous disorders. At WASOG we presented the disease modifying potential of OATD-01 to global industry opinion leaders and we are confident that that this will help us get more U.S. and EU research faculties involved in our trials, resulting in faster patient recruitment.” – adds Marcin Szumowski.

OATD-01 has displayed disease modifying abilities in preclinical trials and has the potential to become the new standard of care for treating pulmonary sarcoidosis.

The phase II clinical trial of OATD-01 is expected to be a multi-center, randomized, double-blind, placebo-controlled study assessing the drug’s safety and effectiveness in treating approximately 90 pulmonary sarcoidosis patients. As result of the double-blind requirement the study’s final unblinded results will be published after its conclusion which is scheduled for the first half of 2025. The study has an innovative primary efficacy end point – the level to which OATD-01  is able to reduce granulomatous inflammation in the pulmonary parenchyma over a 12-week period based on PET/CT scan results. This endpoint was agreed with the FDA following a pre-IND meeting.

About OATD-01

OATD-01, is an oral, once-daily, first-in-class highly selective CHIT1 inhibitor for the treatment of sarcoidosis.  CHIT1 is a promising molecular target through its role in transforming resident, anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types.  The inhibition of CHIT1 by OATD‑01 has been shown to reduce inflammation & fibrosis.

OATD-01 has demonstrated potent anti-inflammatory and antifibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with high unmet medical needs such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and NASH.

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

About Sarcoidosis

Sarcoidosis is a systemic disease of unknown cause that is characterized by the formation of immune granulomas in various organs, mainly the lungs and the lymphatic system. Sarcoidosis is a global disease, affecting both men and women with a prevalence of about 5–50 in 100,000 with 70% of patients aged between 25 and 45 years.

The most severe and frequent complication of sarcoidosis is the occurrence of pulmonary fibrosis. This is usually associated with significant impairment of pulmonary function. Pulmonary fibrosis results in the majority of deaths related to sarcoidosis in western countries.

About Molecure

Molecure is a clinical stage biotechnology company that uses its world leading medicinal chemistry and biology capabilities to discover and develop first-in-class small molecule drug candidates that directly modulate the function of underexplored protein and RNA targets to treat multiple incurable diseases.

Molecure has generated a diverse pipeline of seven distinct programs with the support of leading academic life science institutions.

Molecure’s most advanced in-house drug candidate is OATD-01, a first in class dual chitinase inhibitor for the treatment of interstitial lung diseases, such as sarcoidosis and idiopathic pulmonary fibrosis, that is Phase II ready. A Phase II trial in patients with sarcoidosis is expected to start in 2023.

Our second proprietary candidate is OATD-02, an oral, potent and selective first in class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, which advanced to Phase I clinical development in Q1 2023.

Molecure’s headquarters and laboratories are located in Warsaw, Poland with an additional laboratory facility in Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

For more information, please visit molecure.com/en/

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