Author: Katarzyna Tadeusiak-Wądołowska

• 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

– 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

Warsaw, Poland – 29^th 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

• 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 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

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

• 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 agreement with Simbec-Orion includes organizing and fully executing a proof-of-concept Phase II clinical trial in patients with pulmonary sarcoidosis.
• The Phase II proof-of-concept study of OATD-01 will be conducted as multi-center and international clinical trial in the United States and the European Union and is expected to start in Q4 2023.
• OATD-01 is a first-in-class chitotriosidase 1 (CHIT1) inhibitor that modulates macrophage function and has broad therapeutic potential for the treatment of inflammatory and fibrotic diseases.
• Molecure has filed an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) for a Phase II study in the United States.
• Molecure has received Orphan Drug Designation (ODD) from the FDA for OATD-01 in sarcoidosis and idiopathic pulmonary fibrosis.

 

Warsaw, 3rd July 2023 – Molecure S.A. (“Molecure”: WSE: MOC) 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 unexplored protein and RNA targets to treat multiple incurable diseases, announced that it has entered into an agreement with Orion Sante Sarl (Simbec-Orion), based in France, to organize and conduct a comprehensive Phase II clinical trial for OATD-01.

The OATD-01 Phase II trial will be conducted in the US and several European Union countries. Phase II is planned as a double-blind, placebo-controlled, clinical trial with a fixed dose of compound OATD-01 administered once daily for 12 weeks. The study is designed to evaluate the clinical efficacy, pharmacokinetics, pharmacodynamics, and safety of OATD-01 in patients with active pulmonary sarcoidosis. The value of the agreement with Simbec-Orion is approximately €10.1 million (PLN 45 million – according to the average exchange rate of the National Bank of Poland published on 3rd July 2023, i.e., PLN 4.43).

“The signing of an agreement with Simbec-Orion, an experienced international CRO, marks another important step in recent days towards the initiation of a Phase II trial of OATD-01 in sarcoidosis patients. We recently announced the submission of an IND application to the US Food and Drug Administration (FDA) for this trial, which was preceded by a meeting with the regulator to discuss the dossier and the design of the clinical trial, including its innovative endpoints. We have provided the FDA with more than 20,000 pages of documentation from preclinical studies conducted to date and from the completed Phase I study in 2020. In the near future, we will submit a similar clinical trial application to the EMA under the European Centralised Procedure. We expect to enroll the first patient in the trial in early Q4 2023. The trial will enroll approximately 90 patients with active pulmonary sarcoidosis in the US and Europe, including Poland. This phase of the trial, known as “proof-of-concept in humans”, is critical to the validation of the chitinase platform as it is the first time a chitinase inhibitor has been administered to patients worldwide. Positive results from this phase could pave the way for many other indications with significant unmet medical needs worldwide, such as other interstitial lung diseases or non-alcoholic steatohepatitis (NASH), where similar molecular mechanisms lead to disease development,” – said Marcin Szumowski, CEO of Molecure S.A.

“Sarcoidosis is a rare but highly debilitating disease that significantly reduces the quality of life and, in some cases, leads to death of patients. It remains an unmet clinical need, and the needs of patients are often overlooked. We are, therefore, committed to initiating this trial as soon as possible as we believe that OATD-01 can play an important role in helping patients, not only by alleviating their symptoms but also by improving their quality of life and potentially halting the progression of the disease. Our innovative compound OATD-01, by blocking chitotriosidase 1, is the first-in-class potential drug using this mechanism to modify macrophage function. Our planned study includes an innovative primary efficacy endpoint, consulted with the FDA in a pre-IND meeting. This is the response to 12-week administration of OATD-01, verified by PET/CT imaging, reduction in size and number of granulomas in patients’ lungs. We prepared the study design, including the recruitment criteria and its endpoints, in collaboration with key opinion leaders in fibrotic and pulmonary diseases. We hope that with our most advanced molecule, we will soon be able to bring help to patients with sarcoidosis, fulfilling our mission,” – said Dr. Samson Fung, MD, Chief Medical Officer, and Board Member of Molecure S.A.

Simbec-Orion was selected following a competitive tender organized by Molecure.

 

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/

About Simbec-Orion

Simbec-Orion is an experienced, full-service Contract Research Organisation (CRO), with offices across the UK, Europe, and the United States. Established for over 45 years, providing clinical trial expertise to a wide range of small to mid-sized biotech and pharmaceutical partners across Europe, North America and beyond.

Across the organisation, scientific teams leverage both a wide therapeutic experience in clinical pharmacology, as well as more specialist expertise in Phase I-IV oncology and rare disease. Simbec-Orion’s adaptable, highly experienced teams continuously take a quality-first approach, whilst maintaining the short communication lines and Senior Leadership Team oversight only achievable with a lean management structure. Learn more at www.simbecorion.com

• OATD-01 is a first in class chitotriosidase 1 (CHIT1) inhibitor with disease modifying potential in sarcoidosis and other interstitial lung diseases
• Clearance of the IND application will pave the way for Molecure to initiate the study in the United States becoming only the second Polish biotech company from Poland ever to do so

Warsaw, Poland 22 June 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 that it has filed an Investigational New Drug (IND) application with the U.S. FDA for OATD-01, a first in class and potentially disease modifying Chitotriosidase (CHIT1) inhibitor. Clearance of the IND would allow Molecure to begin an international Phase 2 proof of concept study to evaluate OATD-01 for the treatment of pulmonary sarcoidosis.

“Today’s IND filing is another landmark milestone for Molecure and its lead product OATD-01” said Molecure CEO Marcin Szumowski. “There are currently no approved therapies for sarcoidosis, and while steroids are often used, they have serious side effects and deliver only short-term benefits with little evidence of extended therapeutic efficacy. As a result, there is a significant unmet need for better treatments that can prevent disease progression. We believe that OATD-01, which has demonstrated efficacy in several pre-clinical models, could alter the fate of sarcoidosis patients by becoming the new standard of care for sarcoidosis. Clearance of our IND would allow Molecure to become only the second, Polish biotech company in history to initiate in the United States a multicenter Phase 2 study for an innovative drug, a major accomplishment for everyone involved in this programme.”

Samson Fung, Molecure CMO and Member of the Management Board added, “This IND application brings us a step closer to a new treatment option for underserved sarcoidosis patients. We are excited about our plans to advance OATD-01 into a Phase 2 trial as we believe this highly promising new agent could play a key role in the treatment of this rare disease, improving symptoms as well as quality of life and potentially stopping disease progression. We anticipate beginning our Phase 2 trial in patients in the US and EU in the fourth quarter of this year.

This Phase 2 trial is expected to be a global, multi-center, randomized, double blind and placebo-controlled study to evaluate the safety and efficacy of OATD-01 in approximately 90 patients with active pulmonary sarcoidosis. The results of this double-blinded study will be available once the study is complete, expected in H1 2025. The innovative primary endpoint, already discussed and cleared by the FDA during the pre-IND meeting procedure is the response to a 12-week treatment with OATD-01 measured by the reduction of granulomatous inflammation in pulmonary parenchyma evaluated by PET/CT imaging.”

 

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

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.

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.

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, Eleanor Perkin

molecure@medistrava.com

+44 (0)203 928 6900

 

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/

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