Kategoria: News

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

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

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

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

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

 

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

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

 

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

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

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

 

About Molecure S.A.

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

Molecure has generated a diverse pipeline of seven distinct programs with the support of leading academic life science institutions globally, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw (IIMCB).

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

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

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

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

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

• 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

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

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

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

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

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

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

About OATD-01

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

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

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

About Sarcoidosis

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

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

About Molecure

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

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

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

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

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

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

• Closing of equity offering of 2,776,000 series H shares by private placement, offered within the authorised capital, at an issue price of PLN 18 per share
• Series H shares were subscribed for by 35 investors, new and existing institutional investors participated in the placement
• Investor interest was very high and the offer was oversubscribed
• The Company will leverage the recently completed capital raise to co-finance the implementation of Molecure’s strategic plans for 2023-2025

Warsaw, 18 July 2023. – Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company discovering and developing drugs to the clinical stage, which leverages its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function to treat a wide range of incurable diseases, announced that it has successfully closed the SPO and entered into subscription agreements for all 2,776,000 H shares offered by way of private placement, within the authorized capital. The issue price was set at PLN 18, bringing the gross value of the offering to approximately USD 12 milion (PLN 50 million).

“We have successfully closed the H-share offering, which garnered significant interest from investors, including those who previously were not part of our shareholder base. We would like to thank all investors for participating in the offering and for the trust they have placed in Molecure. During the discussions with investors throughout the offering, we encountered a very good reception to our Strategy and a positive evaluation of the Company’s growth potential, which translated into investors’ interest several times higher than the number of shares offered. Considering the current market conditions, we view the execution of the share issue as a remarkable achievement. The proceeds from the offering will be utilized to finance the advancement of Molecure’s research programmes, aimed at developing effective therapies to address numerous incurable diseases afflicting patients” – says Marcin Szumowski, CEO and shareholder of Molecure S.A.

The proceeds raised through the offering will co-finance the implementation of the Company’s strategic plans for 2023-2025, including, in particular, significant progress in the clinical development of two flagship programmes, namely OATD-01 (a first-in-class chitotriosidase 1 inhibitor, CHIT1) and OATD-02 (a first-in-class dual arginase inhibitor). Additionally, efforts will be intensified in a portfolio of early-stage programmes, including breakthrough small-molecule drug technology that modulates mRNA translation and therapeutics targeting previously unexplored protein targets. These initiatives are bolstered by advanced machine learning and generative artificial intelligence (GenAI) methods. Molecure estimates that the capital expenditure associated with the strategy from mid-2023 to the end of 2025 will amount to approximately PLN 250 million.

Molecure is the exclusive owner – as the only biotech company in Poland – two first-in-class compounds at the clinical trial stage and is consistently building the value of these programmes, aiming to secure at least one high-value partnership agreement between 2023 and 2025, which could translate into tangible benefits for Shareholders.

“The primary business objective of our strategy is to transform the intellectual value we have created into commercial success. We anticipate concluding a partnering agreement for at least one of our clinical assets before 2025, while also establishing commercial collaborations for programmes currently at an earlier stage of development. We are pleased that investors participating in the share offering have recognised our progress thus far and our Strategy for future growth. The funds raised from the share issue, combined with other sources of funding, will provide vital support for the successful and on-track progress of our programme development and further strengthen our negotiating position with partners” – adds Marcin Szumowski.

LEGAL DISCLAIMER

This material is for information purposes only. It is published by the Company solely for the purpose of providing relevant information regarding the terms and conditions of the offer of H Shares. This material is in no way intended to promote, directly or indirectly, the offer, subscription or purchase of H Shares, nor does it constitute any advertisement or promotional material prepared or published by the Company for the purpose of promoting the H Shares, their subscription, purchase or offer or for the purpose of encouraging investors, directly or indirectly, to purchase or subscribe for H Shares. The Company has not published to date and does not intend to publish after the date of this current report any material for the purpose of promoting the Series H Shares, their subscription or purchase.

This material does not identify or suggest, and is not intended to identify or suggest, any risks [direct or indirect] that may be associated with an investment in the H Shares. Any investment decision to subscribe for or purchase H Shares pursuant to an offer, subscription or sale of such shares must be made solely on the basis of publicly available information.

This material does not constitute an invitation to underwrite, subscribe for or otherwise acquire or dispose of any securities in any jurisdiction. This material does not constitute a recommendation regarding an investor’s decision to offer, subscribe for or purchase H Shares. Each investor or potential investor should conduct its own investigation, analysis and evaluation of the business and data described in this material and publicly available information. The price and value of securities can go up as well as down. Past performance is not a guide to future performance.

For further information, please contact:

media and retail investors:
Michał Wierzchowski, cc group
mob. +48 531 613 067
e-mail: michal.wierzchowski@ccgroup.pl

institutional investors and sell-side analysts:
Katarzyna Mucha, cc group
mob. +48 697 613 712
e-mail: katarzyna.mucha@ccgroup.pl

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 early Q4 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 https://molecure.com/

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