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 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 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

Molecure has signed an agreement to conduct a Phase II clinical trial for its lead molecule OATD-01, taking another important step in the development of this program.

  • 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

Molecure Files an Investigational New Drug (IND) application for lead clinical candidate OATD-01 with U.S. FDA ahead of a planned Phase 2 pulmonary sarcoidosis study

  • 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

Molecure Announces Full Year Financial 2022 Results – A Year of Significant Progress

  • Entering 2023 with strong momentum and a robust pipeline of proprietary, first-in-class compounds
  • First patient dosed in Phase I trial with OATD-02, a novel, first in class dual arginase inhibitor for the treatment of cancer
  • Lead proprietary candidate, OATD-01, a novel chitinase inhibitor in sarcoidosis, expected to advance into a Phase II study with first patient dosed in second half of 2023
  • Chief Medical Officer Samson Fung, MD, appointed to the Molecure Management Board to lead global clinical development, translational science and regulatory strategies
  • Zbigniew Zasłona, Director of Biology, promoted to Chief Scientific Officer

 

 Warsaw, Poland – 30 March 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, announces full year results for the period ended 31 December 2022. The full report in Polish can be found here  https://molecure.com/pl/informacje-dla-inwestorow/

“Molecure has made significant progress over 2022, becoming a clinical stage biotechnology company preparing our two most advanced assets to start multi-center phase I and phase II studies respectively” said Marcin Szumowski, CEO and President of the Management Board of Molecure. “The decision to change our name to Molecure reflects our mission, vision and confidence in the clinical and market potential of our pipeline which we believe has the potential to make an important difference to patients with cancer and interstitial lung diseases. In recent weeks, we achieved an important milestone dosing the first cancer patient in a Phase I study with OATD-02, the first and only dual arginase inhibitor. This novel small molecule has been designed to treat a broad range of solid tumors as well as leukemia, particularly in combination with other anti-cancer therapeutics, such as immune checkpoint inhibitors.

OATD-01, a novel chitotriosidase 1 (CHIT1) inhibitor with disease modifying potential in patients with pulmonary sarcoidosis, is nearing start of Phase II, with the first patient expected to be dosed in the second half of 2023. Positive results of this clinical proof-of-concept study in Sarcoidosis may open doors to treatment of other Interstitial Lung Diseases (ILDs), including idiopathic pulmonary fibrosis (IPF) as well as nonalcoholic steatohepatitis (NASH) which represent significantly larger global patient populations. I am looking forward to data from this study which we hope will confirm the key role of CHIT1 inhibition as a new treatment pathway for diseases where chronic inflammation leads to tissue remodeling and fibrosis.

We believe that a successful outcome to this study may mark an important value inflection milestone that will further enhance Molecure’s profile with both potential pharma partners and investors.”

Investor Presentation

The Company’s full year presentation to investors will be held on April 5, 2023 at 2:00 PM (CET) in an online meeting format, available at the link: https://us06web.zoom.us/webinar/register/WN_dKmDwNAUTdiu72mZ0lVitw

The meeting will be conducted in Polish and English with simultaneous translation. It is expected to last approximately 90 minutes. Selection of the meeting language will be available after joining the event.

Commercial & Operational Highlights

  • In 2022, Molecure announced a new name and brand identiy. This change reflects the Company’s “DNA” as well as its growth and maturity, with ongoing commitment to discover and develop novel small molecule therapies for millions of people suffering from serious and incurable diseases worldwide.

 

  •  Molecure regained all rights to its novel, chitinase inhibitor OATD-01
  • Data generated to date demonstrate an attractive pharmacological profile for OATD-01 and scientific advice received from EMA and FDA is highly supportive of progressing OATD-01 into Phase II clinical development. The first patient in the planned Phase II trial is expected to be dosed in the second half of 2023
  • Multiple discussions with leading pulmonologists & KOLs in interstitial lung diseases have taken place throughout 2022 to help shape the planned clinical development program of this novel drug candidate
  • OATD-01 could be the first drug targeting sarcoidosis that is disease modifying and moreover it has potential in several other respiratory indications including IPF as well as nonalcoholic steatohepatitis (NASH),which addressing much larger patient populations

 

  • The President of the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products granted permission to conduct the first clinical trial of OATD-02 in November 2022
  • Following the CTA approval, Molecure has recently initiated a Phase I clinical trial with the first patient dosed in March 2023. The study will assess safety, tolerability and preliminary efficacy of OATD-02 in patients with advanced and/or metastatic solid tumors
  • OATD-02 is an oral, potent and selective first-in-class, dual arginase inhibitor (ARG1 and ARG2) with the potential to treat a broad range of cancers in combination with other anti-cancer agents
  • A Publication* entitled “OATD-02 Validates the Benefits of Pharmacological Inhibition of Arginase 1 and 2 in Cancer” was published in August 2022
    • This paper covered the first study validating the potential benefits of pharmacological inhibition of ARG2 in cancer and demonstrates that OATD-02 is a potent dual ARG1/ARG2 inhibitor with intracellular activity (necessary for targeting ARG2) and exhibiting immunomodulatory and direct antitumor efficacy in pre-clinical models

* Link to publication: https://www.mdpi.com/2072-6694/14/16/3967

 

  • Significant progress in deubiquitinase inhibitors programs
  • USP7 inhibitor program is progressing to the advanced lead optimization stage
    • The lead molecule OATD–4828 has shown encouraging dose-dependent inhibition of tumor growth in preclinical models of various cancer types
  • A promising selective and potent compound has recently been discovered with high affinity for USP21, a novel and biologically validated therapeutic target for cancer treatment

 

  • Further development and expansion of the mRNA targeting small molecule discovery platform
  • Continued development of this novel platform, with a broad range of analytical techniques being applied to confirm the secondary structure of 6 most promising target regions
  • Expansion of both cellular and molecular screening capabilities
  • Ongoing collaborations with leading international RNA centers to expand and leverage the company’s expertise and alternative approach to identify compounds interacting with selected mRNA regions
  • A profit-sharing based service offering is being developed to provide potential industry partners interested in discovery and development of small molecules that interact with mRNA an option to pursue targets and any NCE leads discovered under the collaboration in exchange for revenue sharing with Molecure in the form of development milestones & royalties

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

  • Appointment of Samson Fung, M.D., as Chief Medical Officer to lead global clinical development, translational science and regulatory strategies. Dr Fung will lead the advancement of 0ATD-01 and OATD-02 into Phase II and Phase I clinical trials respectively
  • Zbigniew Zasłona, former VP research biology promoted to Chief Scientific Officer
  • Supervisory board expanded to include Dr. Paul van der Horst, Dr. Nancy Van Osselaer and Paweł Trawkowski as its new members

 

Important Post-period Highlights

  • 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”

Arginase 1/2 inhibitor OATD-02: from discovery to first-in-man setup in cancer immunotherapy | Molecular Cancer Therapeutics | American Association for Cancer Research (aacrjournals.org)

 

  • Samson Fung, Chief Medical Officer appointed to the Company’s Management Board to support the successful 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

 

  • Partnering events and investor conferences
  • Post period end, CEO, Marcin Szumowski and business development director, Nicolas Beuzen attended partnering conference Bio-Europe in Basel Switzerland in March. The event brought together thousands of outstanding biopharma companies enabling meaningful partner discussions
  • The company will also attend and present pipeline progress at the LSX Congress in London, 3-4th The LSX Congress is one of the largest healthcare conferences and partnering events held in Europe.

Full Year Financial Highlights

  • Operating income of PLN1.64 million, a slight increase from 2021 due to grants received during the year
  • Operating expenses totaled PLN18.63 million, an increase of PLN3.41 million. This was mainly due to higher wages as the company’s pipeline advances together with increasing costs of external services
  • Net loss for the year ended 31 December 2022 totaled PLN15.26 million vs net loss of PLN13.64 million in 2021 due to higher costs
  • As of December 31, 2022, Molecure had cash of PLN66 million (US$15 million)
  • Current cash balances are expected to fund the company’s operating expenses and capital expenditure requirements for at least another 12 months.
  • US$/PLN exchange rate 4.38 as of 31 December 2022

 

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, David Dible, 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 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 inhibitor of CHIT1 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 the second half of 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 has advanced to Phase I clinical development in March 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  https://molecure.com

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

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