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mRNA Therapeutics™ for cardiometabolic diseases and cancer


“Together with Moderna, we are pushing the boundaries of science in the pioneering field of messenger RNA Therapeutics™. Where current drug discovery technologies can target only a fraction of the disease-relevant proteins in the human genome, we have the potential to create revolutionary new medicines to treat patients with serious cardiometabolic diseases and cancer.”
— Pascal Soriot, Chief Executive Officer of AstraZeneca


Moderna and AstraZeneca entered into a five-year strategic option agreement in 2013 to discover, develop and commercialize mRNA Therapeutics for the treatment of serious cardiovascular, metabolic and renal diseases, as well as cancer.

Under the terms of the agreement, Moderna received an upfront cash payment and is entitled to additional payments for the achievement of three technical milestones. AstraZeneca has the option to select up to 40 drug products for clinical development, and Moderna will be entitled to development and commercial milestone payments as well as royalties on drug sales ranging from high single digits to low double digits for each product.

AstraZeneca will lead the preclinical, clinical development and commercialization of therapeutics resulting from the agreement, and Moderna will be responsible for designing and manufacturing the messenger RNA against selected targets.

In July 2016, Moderna and AstraZeneca announced that a Clinical Trial Application (CTA) has been filed in Europe for a Phase 1 study of AZD8601, an investigational mRNA-based therapy that encodes for vascular endothelial growth factor-A (VEGF-A). This represents the first program from the collaboration to progress towards clinical trials.

Immuno-oncology mRNA Therapeutics

In 2016, Moderna announced a new collaboration with AstraZeneca, along with its global biologics research and development arm, MedImmune, to discover, co-develop and co-commercialize mRNA therapeutic candidates for the treatment of a range of cancers.

Under the terms of the new agreement, AstraZeneca and Moderna will collaborate on two specific immuno-oncology programs. Moderna will fund and be responsible for discovery and preclinical development of product candidates, with the aim of delivering one Investigational New Drug (IND) application-ready molecule for each of the two programs. AstraZeneca will be responsible for early clinical development, led by MedImmune, and Moderna and AstraZeneca will share the costs of late-stage clinical development.

The two companies will co-commercialize resulting products in the US under a 50:50 profit sharing arrangement. AstraZeneca will lead ex-US commercialization efforts, with Moderna receiving tiered royalties up to substantial double digits on ex-US sales.


mRNA-based vaccines and passive immunity treatments against viral diseases


"By combining Merck’s strength in vaccine and antiviral therapeutic development with Moderna’s mRNA Therapeutics technology, we are well positioned to develop differentiated candidates with the potential to provide meaningful benefit to patients. We look forward to working with the scientific and technical teams at Moderna."
— Roger M. Perlmutter, M.D., Ph.D., President of Merck Research Laboratories

 


In 2015, Moderna announced a license and collaboration agreement with Merck, known as MSD outside the United States and Canada, for the discovery and development of mRNA-based vaccines and passive immunity treatments against viral diseases.

The three-year research collaboration (with the possibility of a one-year extension) is focused on the development of new mRNA-based treatments and vaccines against four undisclosed viruses. Under the terms of the agreement, Merck made an upfront cash payment to Moderna for the ability to utilize the granted licenses to commercialize five product candidates, and also made an equity investment in the company.

Moderna will be eligible for undisclosed per-product development and commercial milestones, as well as tiered royalties on commercial sales. Merck will lead the discovery, development and commercialization, while Moderna will be responsible for the design and synthesis of the mRNA product candidates against selected targets.

In January 2016, Moderna and Merck announced that Merck licensed a new vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing infectious disease collaboration between the two companies.

mRNA-based Personalized Cancer Vaccines


"While the area of personalized cancer vaccine research has faced challenges in the past, there have been many recent advances, and we believe that working with Moderna to combine an immuno-oncology approach, using KEYTRUDA, with mRNA-based personalized cancer vaccines may have the potential to transform the treatment of cancer.”
— Roger M. Perlmutter, M.D., Ph.D., President of Merck Research Laboratories


Merck and Moderna announced a second strategic collaboration in June 2016 to develop and commercialize novel mRNA-based personalized cancer vaccines. The companies will develop personalized cancer vaccines that utilize Moderna’s mRNA vaccine technology to encode a patient’s specific neoantigens, unique mutations present in that specific patient’s tumor.

Under the terms of the agreement, Merck will make an upfront cash payment to Moderna, which Moderna will use to lead all research and development efforts through proof of concept. The development program will entail multiple studies in several types of cancer and include the evaluation of mRNA-based personalized cancer vaccines in combination with Merck’s KEYTRUDA. Moderna will also utilize the upfront payment to fund a portion of the build-out of a GMP manufacturing facility in suburban Boston for the purpose of personalized cancer vaccine manufacturing.

Following human proof of concept studies, Merck has the right to elect to make an additional undisclosed payment to Moderna. If exercised, the two companies will then equally share cost and profits under a worldwide collaboration for the development of personalized cancer vaccines. Moderna will have the right to elect to co-promote the personalized cancer vaccines in the U.S. The agreement entails exclusivity around combinations with KEYTRUDA. Moderna and Merck will each have the ability to combine mRNA-based personalized cancer vaccines with other (non-PD-1) agents.


mRNA Therapeutics for the treatment of cystic fibrosis


“The collaboration with Moderna underscores our commitment to invest in and pioneer multiple innovative approaches to expand and enhance the future treatment of CF. Moderna’s messenger RNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF.”
— David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer


In July 2016, Moderna and Vertex announced that the companies entered into an exclusive research collaboration and licensing agreement aimed at the discovery and development of mRNA Therapeutics for the treatment of cystic fibrosis (CF). The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery.

Under the terms of the collaboration, Moderna will lead discovery efforts. Vertex will lead all preclinical, development and commercialization activities associated with the advancement of mRNA Therapeutics that result from the collaboration and will fund all expenses related to the collaboration.

Vertex will make an upfront cash payment to Moderna and will also make an investment in Moderna in the form of a convertible note that will convert to equity. The investment will provide Vertex with an ownership stake in Moderna. Vertex will also pay Moderna future development and regulatory milestones, as well as tiered royalty payments on future sales.