It hasn’t been proven yet that synthetic messenger RNA molecules (mRNA) can be effective medicines. But their promise as a source for new vaccines, primarily for cancer, has enticed multiple pharmaceutical companies. The latest evidence comes from Sanofi, which just cut its second mRNA vaccine deal to date, this time with Translate Bio.
Sanofi Pasteur, the vaccines division of French pharma giant Sanofi, will pay Translate—a Lexington, MA, company formerly known as RaNA Therapeutics—$45 million up front for rights to experimental vaccines for up to five infectious diseases. Translate Bio, which is currently gearing up for an IPO, could see up to $805 million in downstream payments and royalties if Sanofi opts into all the programs, and they all get to market. The deal has an initial three-year term.
Translate Bio is developing synthetic mRNA drugs, meant to coax the body into producing proteins that might be dysfunctional or missing in disease. If mRNA drugs work, the potential could be enormous, unlocking an entirely new way to treat disease. But the method is largely unproven in humans. Translate Bio is one of several companies working on it, along with CureVac, BioNTech, and Moderna Therapeutics.
There has been no shortage of hype surrounding mRNA medicines, and that’s in part because of the fundraising success of Moderna. While privately held and without much human clinical data, Moderna has secured more than $1.6 billion in venture financings, another $1 billion in partnerships, and has a valuation north of $7 billion. Moderna has had some stops and starts in terms of its clinical strategy, but the company has chosen to put the bulk of its initial work into experimental vaccines for cancer and infections like Zika, chikungunya virus, cytomegalovirus.
To date, Moderna and its rivals’ efforts to develop mRNA cancer vaccines have drawn significant investments from multiple pharmaceutical companies—including Sanofi. Merck, for instance, recently put $125 million into Moderna on top of two other deals it cut with the company in 2015 and 2016. Sanofi paid BioNTech $60 million in 2015 to co-develop mRNA cancer vaccines (BioNTech is aligned with Genentech as well.) CureVac has alliances with Boehringer Ingelheim and Eli Lilly. The thinking goes that these vaccines can help people respond to immunotherapies known as checkpoint inhibitors, which still only work in a fraction of patients despite all the progress they’ve made in a variety of cancers.
Deals that use mRNA technology to develop vaccines for infectious diseases have been less common, but Sanofi has now bought in. Sanofi is aiming for what Sanofi Pasteur senior VP of R&D John Shiver referred to as “next-generation vaccines.” mRNA vaccines could be made more cheaply and improve the “breadth of immune response for infectious disease vaccines,” says Shiver. Sanofi and Translate Bio didn’t say which infections they would focus on first.
The infectious disease work isn’t Translate Bio’s main focus. The company’s lead drug candidate, MRT5005, is for cystic fibrosis and began Phase 1/2 clinical testing last month. Its second drug, MRT5201, targets a rare liver disease and should enter the clinic early next year. Translate got these programs through a deal with Shire (NASDAQ: SHPG) at the end of 2016, making Shire Translate’s largest stockholder. The company filed for an IPO on June 1.