Moderna Therapeutics has become as well known for its valuation as it has for the secrecy surrounding its messenger RNA (mRNA) technology since its launch in 2010. Paul Jeng, PhD, Infectious Diseases Analyst, GlobalData, analyzes the growing stature of the young biotech as it punches above its weight. The company has enjoyed a flourishing channel of funding, raising over $1.6 billion in equity and $1 billion in partnerships in recent years, capped by a recent $125 million Series H round from Merck, despite remaining privately held and remaining relatively secretive about data.

With a price tag of staggering $7.5 billion, Moderna is now one of the biotech startup companies with the highest valuation. STAT news reported in September that the Cambridge-based developer may soon target an IPO by the end of 2018 as its biggest step out of the shadows yet. Will Moderna be able to justify its high valuation as an intriguing but unproven biotech focused on developing typically low-margin infectious disease vaccines?

Moderna Embracing Transparency Moderna was founded on the research of Harvard scientists Derrick Rossi and Tim Springer. Following the appointment of CEO Stéphane Bancel to lead the business, the company experienced rapid expansion. Moderna announced a $240 million upfront payment as part of a multiyear collaboration with AstraZeneca on cardiovascular diseases and cancer in 2013. After that, partnerships and funding continued to flow, including collaborations with Merck, Vertex, and the Defense Advanced Research Projects Agency (DARPA) of the United States.

However, it proved difficult to become the first company to bring an mRNA medicine to market. Delivery and side effects of engineered mRNA have been major challenges for many developers, including Moderna, despite the well-understood mechanism by which RNA transcripts in cells produce proteins. STAT news reported at the beginning of 2017 that safety concerns had caused Moderna to delay indefinitely the treatment for the rare disease Crigler Najjar Syndrome Drugs Development Market.

Moderna began to embrace transparency in 2017 by publishing eight papers in leading clinical and translational research journals as a response to setbacks and a growing cynicism among scientists. In addition, the company announced nine vaccine programs for diseases ranging from influenza to human metapneumovirus (HMPV), consolidating its focus on infectious pathogens.

Because mRNA vaccines typically only require a single dose and are well tolerated, GlobalData is of the opinion that Moderna is well-positioned to transform the infectious disease market. This is because moderna vaccines are well-tolerated. In addition, Moderna has the chance to use a customized mRNA vaccine platform to quickly combat emerging infectious diseases worldwide. For Chikungunya virus (mRNA-1388) and Zika virus (mRNA-1325), the company currently has two Phase I products in the pipeline.

mRNA-1647, a cytomegalovirus (CMV) preventative vaccine, is a notable Moderna product. mRNA-1647 began Phase I human testing in late 2017 after strong neutralizing antibody responses were observed in rodents and non-human primates in preclinical studies. Following the Phase III failure of Vical and Astellas' DNA vaccine ASP0113, GlobalData surveyed key opinion leaders (KOLs) who were of the opinion that the CMV vaccine market is ripe for new players. Moderna recently appointed Dr. Wellington Sun, the former director of the Division of Vaccines and Related Product Applications at the FDA, as its head of Vaccine Strategy and Regulatory Affairs to improve its chances.

A dozen other candidates from oncology, rare diseases, and cardiology make up the remainder of Moderna's pipeline. The vascular endothelial growth factor-A (VEGF-A) mRNA-8601, developed with AstraZeneca for the treatment of cardiometabolic diseases, is the company's lead product. The results of mRNA-8601's early clinical trials were encouraging, and in February 2018, the drug became Moderna's first candidate for Phase II testing. In order to move into more lucrative fields like immuno-oncology, Moderna hopes to have at least one of its ten products in clinical development. However, Moderna's initial bet on infectious diseases, particularly those with high unmet needs, will likely prove to be a wise strategy in the face of growing pressure from rival clinical-stage mRNA companies like Curevac.