Moderna Therapeutics, a superlatively well-funded private biotech, has made the leap from preclinical bluster to first-in-human trials, testing out its inaugural candidate and planning to move 5 more into the clinic this year.
The company's core technology is focused on using messenger RNA, or mRNA, to spur the production of human proteins within patient cells, creating what Moderna describes as an in vivo factory for targeted therapies. The promise of such an approach has helped the company raise more than $1.2 billion in venture and partnering cash since its inception in 2011, and now the Cambridge, MA, biotech is making the leap into clinical development.
First up is mRNA 1440, which entered a Phase I trial in Europe last year, Moderna said. The treatment, which targets an undisclosed infectious disease, has so far been administered to 12 healthy volunteers, and the company is enrolling two more patient cohorts this week, Moderna said. The company has also filed with the FDA to begin clinical studies on a second infectious disease treatment, mRNA 1851, with plans to start recruitment this quarter.
Moderna CEO Stéphane Bancel gave the public its first glimpse of his company's pipeline during a standing-room-only presentation at the annual J.P. Morgan Healthcare Conference in San Francisco on Monday. Since coming out of stealth mode in 2013, Moderna has been an object of fascination in biotech circles, raising loads of cash, signing big-name partnerships--with AstraZeneca ($AZN), Merck ($MRK) and Alexion ($ALXN)--but never starting a clinical trial, seemingly content to amass a self-described 80-asset stable of early-stage mRNA projects.
Until now. With the seal broken on its clinical ambitions, Moderna expects to have 6 total candidates in human trials by the end of 2016. Behind its two lead therapies, Moderna is pressing toward the clinic with MRK 1777, a Merck-partnered vaccine, and mRNA 1388, a DARPA-backed infectious disease treatment. In rare diseases, the company is working with Alexion on ALXN 1540, a therapy for the rare Crigler-Najjar syndrome. And in the cardiovascular field, Moderna has teamed up with AstraZeneca on AZD 8601, an mRNA treatment designed to repair heart damage.
To execute on its broad ambitions, the company is planning to expand its current workforce of roughly 320 people to about 500 by year's end, Bancel said. Moderna said it closed 2015 with about $802 million in cash.
The company is planning to invest $350 million this year in four subsidiaries established over the past few years, each focused on oncology, infectious diseases, rare diseases or cancer vaccines. From there, the company will earmark $100 million each year to build out its pipeline and R&D infrastructure, Bancel said, eyeing a 20-year horizon with hopes of churning out dozens of mRNA products.
And Moderna is already looking forward to its second generation of clinical candidates, pointing to follow-on deals with AstraZeneca and Merck that have put in motion immuno-oncology and infectious disease projects on track for Phase I trials in 2017.
"As you know, most deals in biotech and pharma don't drive any value," Bancel said at J.P. Morgan, pausing for audience laughter. "They don't. They don't drive any value."
The fact that AstraZeneca and Merck came back to the well so quickly, he said, is a sign that partnering with Moderna breaks the industry mold.
"More than ever, the future of this company is unbelievable," he said.
- read the statement