Moderna Therapeutics has raised an additional $500 million in a financing round the company says will help it fund further R&D into its pipeline of messenger RNA (mRNA) treatment candidates.

Moderna said the financing will help it continue to develop its pipeline of 19 candidates—10 of them in clinical phases—by initiating several Phase II trials, as well as corporate priorities for 2018/2019 that include:

  • Advancing its development and delivery platform by investing in mRNA science and technology
  • Building out its manufacturing infrastructure and digital backbone (though the company’s announcement of the financing offered no details)
  • Focusing on drug discovery for rare disease and prophylactic vaccines

Moderna announced its first rare-disease candidate in September with mRNA-3704, designed to treat methylmalonic acidemia (MMA). At the J.P. Morgan 36th Annual Helthcare Conference last month, Moderna unveiled a new rare-disease candidate, mRNA-3927, designed to direct liver expression of a deficient enzyme in patients with propionic acidemia.

“This past year we have demonstrated an ability to scale our platform and introduce new development candidates across multiple disease modalities, including promising opportunities in rare disease, immuno-oncology, and cardiovascular and infectious diseases,” Moderna CEO Stéphane Bancel said in a statement.

“We continue to execute across the organization and against our goal of rapidly building a pipeline of mRNA medicines, which we have demonstrated by moving 10 candidates into the clinic over the past 24 months.”

Advancing to the Clinic

According to Moderna’s website, three of those candidates advanced into the clinic in the last two months of 2017.

In November, Moderna began a Phase I study of mRNA-1647, a prophylactic vaccine designed to treat cytomegalovirus (CMV). mRNA-1647 combines six mRNAs encoding for six viral proteins, including five proteins that comprise the CMV gH Pentamer complex (gH, gL, UL128, UL130, and UL131A), as well as another CMV antigen, the herpesvirus glycoprotein (gB) protein.

Also in November, Moderna launched the Phase I KEYNOTE-603 study of mRNA-4157, an mRNA-based personalized cancer vaccine being developed with Merck & Co. through a $100-million-plus mRNA partnership launched in January 2015. The vaccine is designed to encode for peptides containing neoantigens and other unique mutations present in each patient’s specific tumor. When injected in the body, the mRNA will direct cells to produce and express these neoantigens, according to the company.

Moderna says mRNA-4157 could potentially improve clinical outcomes associated with checkpoint inhibitor therapies—including Merck’s marketed anti-programmed death 1 (PD-1) cancer immunotherapy, Keytruda® (pembrolizumab). KEYNOTE-603 is expected to enroll up to 90 patients and will assess the safety, tolerability, and immunogenicity of mRNA-4157 alone in subjects with resected solid tumors and in combination with Keytruda.

In December, Moderna launched a Phase I study of mRNA-1653, a multivalent vaccine designed to immunize against human metapneumovirus (HMPV) and parainfluenza virus 3 (PIV3). mRNA-1653 combines mRNA encoding for a viral antigenic protein associated with HMPV and mRNA encoding for a viral antigenic protein associated with PIV3 into a single vaccine.

Moderna expects its first mid-stage pipeline candidate to be AZD-8601, a localized mRNA therapeutic encoding for vascular endothelial growth factor, VEGF-A, being developed with AstraZeneca. At the J.P. Morgan conference, Moderna announced plans for a Phase IIa study of AZD-8601, to be led by AstraZeneca, adding that design and target indication for the study would be detailed in coming weeks. Moderna and AstraZeneca are co-developing mRNA therapeutics under an up-to-$420-million-plus collaboration launched in 2013.

$1.4B in Cash Raised

Moderna raised the financing from a global group of new investors that included EDBI, the dedicated corporate investment arm of the Singapore Economic Development Board; a wholly-owned subsidiary of the Abu Dhabi Investment Authority; BB Biotech; Julius Baer; and Sequoia Capital China.

They joined existing investors that included Fidelity Management & Research Company, the investment advisor to Fidelity's family of mutual funds; Pictet; Viking Global Investors; ArrowMark Partners; and Alexandria Venture Investments.

“We are delighted by the strong support from new U.S. and global investors in our business, science, and vision for the future, and the continued and expanded support from existing investors,” Bancel added.

Moderna’s latest financing isn’t the first time the mRNA treatment developer has pulled off a nine-figure financing. The company completed the largest private financing of 2016 by raising $474 million, also with pipeline development and enhanced manufacturing capabilities in mind.

With the latest financing, Moderna said its balance sheet has swelled to nearly $1.4 billion in cash, plus access to additional $250 million in grants. Potentially half of that could come from the Biomedical Advanced Research and Development Authority (BARDA), which awarded the company up to $125 million in September 2016 toward developing a Zika virus vaccine—three years after the company won up to $25 million from the Defense Advanced Research Projects Agency (DARPA) toward preclinical work for the Zika mRNA vaccine.

In January 2016, the Bill & Melinda Gates Foundation awarded Moderna an initial $20 million grant to develop a new affordable combination of mRNA-based antibody therapeutics geared toward preventing HIV infection, with the potential to receive a total of $100 million from the foundation.

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