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Moderna Builds on Clinical Validation of Systemic Delivery with Two Additional Development Candidates in New Autoimmune Therapeutic Area

BusinessWire

mRNA-6231 encodes a long-acting selective IL-2 to preferentially expand regulatory T cells that suppress immune activity in autoimmune diseases

mRNA-6981 encodes PD-L1 to treat autoimmune disease, initially to be developed in autoimmune hepatitis

Company to expand pipeline of innovative vaccines in the near term based on clinical success of infectious disease vaccine portfolio to date

CAMBRIDGE, Mass–(BUSINESS WIRE)–Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that it has entered a new autoimmune therapeutic area. Building on the clinical validation of systemic delivery of mRNA provided by data from its antibody against the chikungunya virus (mRNA-1944) program, this new therapeutic area will include autoimmune and inflammatory diseases. The Company also announced that it will expand its pipeline of innovative vaccines in the near term, following six positive Phase 1 clinical trial readouts from its infectious disease portfolio and the initiation of a Phase 2 study for its CMV vaccine (mRNA-1647).

Moderna’s pipeline is organized into six modalities based on similar mRNA technologies, delivery technologies and manufacturing processes. The Company’s approach is to leverage early programs within a modality to generate clinical data and insights that reduce the technology risk of subsequent programs and accelerate the expansion of the pipeline in that modality. Today’s announcement reflects the Company’s belief that recent positive Phase 1 data from its infectious disease vaccine portfolio, including its complex CMV vaccine, and chikungunya antibody program have de-risked its prophylactic vaccines and systemic therapeutics modalities. As a result, Moderna intends to bring new development candidates forward within these two areas.

“2019 was an inflection point for Moderna with significant clinical advances resulting from our investments in science and manufacturing capabilities. The positive Phase 1 results from our CMV vaccine and chikungunya antibody programs validate our approach and help clinically de-risk the delivery technologies for our prophylactic vaccines and systemic therapeutics modalities. Based on these learnings, we are excited to enter a new therapeutic area in autoimmune disease and announce two new development candidates,” said Stéphane Bancel, Moderna’s chief executive officer. “We are entering 2020 with clear priorities, a strong cash balance and a talented team of employees focused on achieving our mission. With our CMV vaccine, we are preparing for our first pivotal Phase 3 study and we look forward to announcing additional new development candidates in our two clinically de-risked modalities, prophylactic vaccines and systemic therapeutics.”

Mr. Bancel will present an update on the Company and its pipeline of mRNA development programs on Monday, January 13, 2020 at 4:30 p.m. PT at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco. The presentation will be followed by a question and answer session. A live webcast of both the presentation and question and answer session will be available under “Events & Presentations” in the investors section of Moderna’s website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for 30 days following the presentation.

Moderna currently has 21 mRNA development candidates in its portfolio with 13 in clinical studies. Across Moderna’s pipeline, more than 1,500 participants have been enrolled in clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline.

About Moderna’s New Autoimmune Therapeutic Area

Autoimmune diseases are characterized by immune activation in response to antigens normally present in the body, reflecting a loss of tolerance. Within this therapeutic area, the Company is developing two potential medicines, mRNA-6231 and mRNA-6981, designed to engage peripheral tolerance pathways to dampen autoimmune activation and help restore immune homeostasis, thereby reducing autoimmune pathology.

mRNA-6231 is an mRNA encoding for a long-acting IL-2 mutein designed to preferentially activate and expand the regulatory T cell population, dampening the immune response.

mRNA-6981 is an mRNA encoding for PD-L1 and is designed to augment cell surface levels of PD-L1 on myeloid cells, providing co-inhibitory signals to self-reactive lymphocytes.

As an initial step to addressing a range of autoimmune indications, the Company plans to conduct a Phase 1 study of mRNA-6231 in healthy adult volunteers and intends to pursue proof-of-concept with mRNA-6981 in a Phase 1 study in type 1 autoimmune hepatitis (AIH), a condition that involves liver inflammation and can lead to cirrhosis and liver failure. The Phase 1 study of mRNA-6231 will be the first clinical demonstration of subcutaneous administration of this delivery technology.

Both of these new autoimmune development candidates share the same delivery technology as mRNA-1944, the antibody against chikungunya, reducing technology risk. The autoimmune therapeutic area is Moderna’s fifth therapeutic area, in addition to infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases.

Program Updates by Modality:

Prophylactic vaccines: Moderna is developing vaccines against viral diseases where there is unmet medical need – including complex vaccines with multiple antigens for common diseases, as well as vaccines against threats to global public health.

Serious infections transmitted from mother to baby

Serious respiratory infections

Global public health programs

Cancer Vaccines: These programs focus on stimulating a patient’s immune system with antigens derived from tumor-specific mutations to enable the immune system to elicit a more effective anti-tumor response.

Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.

Localized Regenerative Therapeutics: Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues.

Systemic Secreted & Cell Surface Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are either secreted or expressed on the cell surface.

Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Information about each development candidate in Moderna’s pipeline, including those discussed in this press release, can be found on the investor relations page of its website: https://investors.modernatx.com/.

Corporate Objectives

Moderna continues to execute on its corporate objectives for 2019-2020, which include:

  1. Generate human proof-of-concept data for multiple medicines
  2. Execute on current development pipeline
  3. Create new development candidates in existing modalities
  4. Invent new modalities

Corporate Updates

Key 2020 Investor and Analyst Event Dates

About Moderna

Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. mRNA medicines are designed to direct the body’s cells to produce intracellular, membrane or secreted proteins that have a therapeutic or preventive benefit with the potential to address a broad spectrum of diseases. Moderna’s platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, providing the Company the capability to pursue in parallel a robust pipeline of new development candidates. Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, independently and with strategic collaborators.

Headquartered in Cambridge, Mass., Moderna currently has strategic alliances for development programs with AstraZeneca, Plc. (NASDAQ: AZN) and Merck, Inc. (NASDAQ: MRK), as well as the Defense Advanced Research Projects Agency (NASDAQ: DARPA), an agency of the U.S. Department of Defense and the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS). Moderna has been named a top biopharmaceutical employer by Science for the past five years. To learn more, visit www.modernatx.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but limited to, statements concerning: the success of the Company’s new autoimmune and inflammatory diseases therapeutic area; the timing and success of the Company’s planned Phase 1 studies of mRNA-6231 and mRNA-6981; the timing of expansion of its pipeline of innovative vaccines; the timing of the initiation of the Phase 2 study for mRNA-1647; the adequacy of the Company’s cash, cash equivalents and investments; the Company’s ability to retain its senior management and employees; the Company’s ability to maintain current and enter into new collaborations with partners; the Company’s ability to obtain funding to support the future clinical development of mRNA-1440 and mRNA-1388; the Company’s ability to fund its influenza H7N9 vaccine program through approval; the timing and expected outcome of Moderna’s clinical studies of its other development candidates; the Company’s ability to advance its preclinical-stage development candidates into clinical development; the Company’s ability to execute on its corporate objectives; and the Company’s ability to receive additional grant funding. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the success of the Company’s new autoimmune and inflammatory diseases therapeutic area; the clinical success of subcutaneous administration of its delivery technology; the ability to build upon the clinical validation of the systemic delivery of mRNA provided by mRNA-1944; the Company’s belief that recent positive Phase 1 data from its infectious disease vaccine portfolio and chikungunya antibody program have de-risked its prophylactic vaccines and systemic therapeutics modalities; the timing and success of Moderna’s clinical studies of its development candidates; the ability of the Company to advance a rare disease program into clinical testing; and those risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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1Biomedical Advanced Research and Development Authority (BARDA), Defense Advanced Research Projects Agency (DARPA) and The Bill and Melinda Gates Foundation (BMGF). Additional funding is subject to agreement on scope of additional projects.

Contacts

Media:
Colleen Hussey

Senior Manager, Corporate Communications

203-470-5620

Colleen.Hussey@modernatx.com

Dan Budwick

1AB

973-271-6085

Dan@1abmedia.com

Investors:
Lavina Talukdar

Head of Investor Relations

617-209-5834

Lavina.Talukdar@modernatx.com

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