‘This is not how you do science’: Cambridge biotech Moderna’s potential COVID-19 vaccine stirs hope — and criticism

Robert S. Langer, the prolific inventor and biomedical engineering professor at MIT who cofounded Moderna, said the results of the early stage clinical trial were impressive.Barry Chin/Globe Staff

Eight people. That’s all it took to move the market on Monday.

The eight were study participants who received a potential COVID-19 vaccine and produced antibodies that killed the coronavirus in a laboratory dish.

Within hours of issuing a press release about this finding, Moderna, the Cambridge-based maker of the experimental vaccine, saw its stock price jump by 20 percent. By day’s end, Moderna was credited with helping drive a 4 percent rise in the stock market.

Never mind that it’s not known whether the dozens of other people in the study will have the same results. Or that these are partial findings from a small, early-stage study. Or that Moderna did not publish any data to back up its press release.

To some degree, the company is riding high on the value of another much-needed commodity: hope.

Moderna is one of eight companies worldwide testing COVID-19 vaccines in humans, but the first to report any results, the first hint of a way out of the devastating pandemic.

And the company is getting a good price for the hope it inspires. A small biotech that went public only in 2018, Moderna is now valued at $29 billion.

So far, Moderna has not brought a single drug to market — not an unusual situation in the biotech world. The company has more than 20 drugs in the pipeline, including potential treatments for rare diseases and cancer, and nine possible vaccines against various viruses.

Moderna officials and others who cheer the early findings say the hope is well-founded. But critics point to the scarcity of data provided and the many unanswered questions.

"This is not how you do science. It’s really impossible to make sense of their statements,” said Dr. Peter J. Hotez, co-director of the Texas Children’s Hospital Center for Vaccine Development, which is also working on a COVID-19 vaccine.

For example, Moderna said that the eight patients developed neutralizing antibodies — capable of stopping the virus from replicating — at levels comparable to those in people who have recovered from the infection. But that’s not necessarily good news, Hotez said.

A recent paper from Rockefeller University "shows that convalescent patients have a very low level of neutralizing antibodies, so that would not be your desired target. You’d want to do much better than that. But they did not provide a number,” he said.

Questions such as these may have even reversed Monday’s stock market gains. A story by Helen Branswell in STAT, which quoted other experts who found Moderna’s data insufficient, was blamed for driving down Moderna’s stock by 10 percent by the end of trading on Tuesday — and the stock market fell with it.

Robert S. Langer, the prolific inventor and biomedical engineering professor at MIT who cofounded Moderna, said the results of Moderna’s early stage clinical trial, while small, were impressive. The vaccine produced antibodies in all 45 healthy volunteers who participated in the study. In the eight whose blood was tested in the laboratory, the antibodies stopped the virus from replicating, the company said.

“To top it off, there really weren’t any bad side effects,” said Langer, who sits on Moderna’s board of directors and scientific advisory board. “I felt as a scientist it was extremely encouraging.”

Moderna was founded in 2010 to take advantage of the therapeutic potential of messenger RNA, or mRNA, a single-stranded molecule that enters cells and provides the blueprint for making the proteins that cells need to function.

Moderna’s technology reprograms mRNA strands to instruct cells to create other proteins, substances that treat or prevent diseases. This essentially turns a person’s cells into little drug factories. And the company doesn’t have to start from scratch with each different drug, it just reprograms the mRNA.

The coronavirus pandemic provided an opportunity for Moderna to deploy its technology in a big way, potentially producing a vaccine that could end the crisis. It won $500 million in federal aid.

An mRNA vaccine can be produced much faster than older methods that require growing the vaccine in cells. Moderna was first out of the gate with human trials starting in March. And it was the first to report, on Monday, some early results.

The company put out its press release at 7:30 a.m., two hours before the stock market opened, and received quick coverage from most major media outlets.

Even before Monday’s announcement, an investor told STAT’s Adam Feuerstein, “I think Moderna’s valuation is insane, but the world wants their coronavirus vaccine to work.”

On Tuesday, Feuerstein, a longtime biotech reporter known for his influence and skepticism, said that investors were telling him that Moderna stocks are being purchased by “investors who don’t specialize in the biotech or pharma sector” and are thus more susceptible to the media-fueled optimism.

Langer, the Moderna cofounder, dismissed such sniping as sour grapes. Plenty of biopharmaceutical companies, from Genentech to Amgen, were criticized as being hyped and overvalued but later proved successful, he said.

As an example, he mentioned the small New Jersey-based biotech, Pharmasett, that developed a new generation of hepatitis C drugs. That company was widely considered overvalued when Gilead bought it for $11 billion in 2011, but the acquisition led to blockbuster drugs that cure hepatitis C and is now viewed as a steal for Gilead.

Alan Carr, an analyst at Needham & Co., sides with the optimists. His enthusiasm for Moderna goes beyond COVID-19 — he believes its technology has potential for so many diseases that it will make the company an important player in biotech and medicine long into the future.

“There’s some sort of revenue opportunity likely for Moderna around this vaccine,” he said. “But the other aspect of it is more validation of the platform technology. It gives Moderna a chance to grow up even faster."

Carr was unconcerned about the lack of published data, saying that it’s normal to announce results with a press release and that the full data set will be published at some point this year.

He was impressed to learn that all 45 participants made antibodies to COVID-19, and that the higher the dose of the vaccine, the more they produced. And he called it “a pretty good sign” when the company tested the blood of eight participants in the laboratory, and all eight had antibodies that stopped virus replication.

Carr said he believes "it’s likely that they will have a vaccine that will be helpful for COVID-19.”

Still, many steps and potential pitfalls lie ahead.

Monday’s press release reported partial results of a Phase 1 study, which measures side effects and immune response. The company apparently still needs to do laboratory tests on the blood of the remaining 37 participants.

Meanwhile, Moderna already has approval to start a Phase 2 study, which will examine the drug’s effects in hundreds of people. And it expects in July to advance to Phase 3, which will test the drugs in thousands of people.

Other drug companies are moving forward with their vaccine candidates. The hope is that there won’t be a single “winner" in this race, but rather several successful vaccines, enough to reach the world’s billions.