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
• Dr. 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. 

“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 could 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 the 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. Link here.

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 identity. 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 addresses a much larger patient population

• 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 here. 

• Significant progress in deubiquitinase inhibitor 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”. Link here.

• 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,  3-4^th 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