Moderna, nine months on from raising a record sum on Wall Street, offered up Thursday the latest evidence it could achieve the lofty goals it's pitched to investors.
Hosting an R&D day in New York City, executives of the nine-year-old biotech presented results from two Phase 1 studies that indicate progress in the company's development of messenger RNA-based medicines.
In one, Moderna showed for the first time it can spur in vivo production of secreted antibodies using a drug containing the nucleic acid code upon which cells rely to translate genetic instructions. It's the most difficult technical feat Moderna's attempted in its step-wise strategy for proving its mRNA technology.
The concept is also core to Moderna's promise and multi-billion dollar valuation, which has come under pressure since the company raised $600 million in a public offering last December. Rather than use manufactured proteins to treat disease, Moderna set out to co-opt the body's own protein machinery by delivering mRNA into cells.
In the other study presented Thursday, interim data for a cytomegalovirus (CMV) vaccine has Moderna confident enough to advance the experimental candidate into Phase 2 and begin planning for a pivotal Phase 3 trial.
"We've said from the get-go that we're exploring things on two axes: One is increasing biological and medical complexity, and the other is pushing the technology into new domains," said Tal Zaks, Moderna's chief medical officer since 2015, in an interview.
"What you see is us widening our canvas on both axes."
Up until recently, much of Moderna's disclosed work has involved prophylactic vaccines — vitally important for public health but not always seen by investors as commercially compelling. This year, though, the biotech has unveiled initial data for programs in other areas, including for a cancer vaccine that's partnered with Merck & Co. and a regenerative therapy now being advanced by AstraZeneca.
Thursday's results for what Moderna calls mRNA-1944 — aimed at chikungunya virus — could take the company's capabilities one step further.
"We can make antigens of viruses, we can make human protein, and now we've shown we can make antibodies [with mRNA]," said first time CEO Stéphane Bancel, whose ambitions are not modest.
"Any protein under the sun we can make."
mRNA-1944 comprises two mRNA sequences that encode for an antibody known to have activity against chikungunya. Moderna's aim is for the drug to confer passive immunity to infection by the virus, for which there is no approved vaccine.
Across three dose levels given to 16 healthy patients, treatment with mRNA-1944 led liver cells to produce the desired antibody. For those given the mid- and high-dose, antibody levels were sufficiently high to be protective against chikungunya for an estimated 16 weeks, Moderna said.
While that's not long enough for individuals living in areas where the virus is endemic, mRNA-1944 could offer protection for travelers. Moderna also sees potential for the drug to be paired with a vaccine, which normally take months to become effective compared to the few hours for secreted antibodies.
In a note of caution, however, Moderna is still sifting through the safety data on mRNA-1944, which triggered infusion-related side effects in three of the four participants given the highest dose. One patient experienced tachycardia, or rapid heart beat, that was judged to be Grade 3.
Such reactions were expected, Zaks said, but Moderna is considering using steroid pre-medication as a precaution moving forward and hasn't yet dosed the last two planned participants at the highest dose.
For all of Moderna's promises — the company describes its work as "building a new class of medicines" — its choice of pursuing vaccines before other areas disappointed some.
Bancel and Zaks said the decision to work first on vaccines was part of Moderna's management of the risk inherent in developing a new kind of therapy. And Bancel disputes the idea pursuing vaccines constrains Moderna's market opportunity.
"There are still many viruses like CMV, for which no vaccine is on the market," said Bancel. "We think these are very big pieces for patients and of value for investors."
CMV infections are the leading infectious cause of birth defects in the U.S., according to Moderna, with the virus transmitted from mother to child.
Phase 1 data for mRNA-1647, which combines six mRNA sequences encoding for viral antigens, showed treatment of seronegative study participants resulted in increased neutralizing antibodies to above levels associated with CMV infection.
Moderna will soon start a Phase 2 of mRNA-1647 in about 250 patients to confirm a dose for Phase 3, which would likely enroll thousands of patients to prove the drug can prevent primary CMV infections.
If successful, Bancel suggested mRNA-1647 could eventually be a billion-dollar product for Moderna, alluding to Pfizer's Prevnar 13, Merck's Gardasil and GlaxoSmithKline's Shingrix.
Those products, of course, are also among the most lucrative products on the market. A closer comparison might be the rubella vaccine, given to children as part of a standard vaccine course.
Whether investors agree will test Moderna's valuation, which has ebbed from above $7 billion to around $5 billion since the biotech debuted on public markets.