The next two months are likely to bring a flurry of news as many drugmakers begin reporting earnings for the first quarter, beginning with Johnson & Johnson on Tuesday.
Investors will once again try to read the tea leaves of commentary from Biogen executives about their experimental Alzheimer's drug aducanumab, now under review at the Food and Drug Administration with a decision expected by June 7. For Vertex and Sarepta, meanwhile, questions on two key drugs are likely, as fresh data are slated to arrive within weeks.
But two of the most anticipated developments will come from AstraZeneca, which should soon ask the FDA for emergency clearance of its coronavirus vaccine, and J&J, which awaits U.S. regulators' verdict on whether vaccinations with its shot can safely resume.
Between now and June figures to be a crucial period for biotech, pharma and the pandemic response. Here's what to watch:
A restart on vaccinations with J&J's shot?
Millions of doses of Johnson & Johnson's coronavirus vaccine have been in limbo since April 13, when U.S. health agencies recommended a pause in immunizations due to concerns over a rare but serious side effect.
At the time, regulators and the company had identified an unusual combination of clotting near the brain and low levels of platelets, the sticky cells that help blood coagulate, in six younger adult women given the vaccine. All six were hospitalized and one died.
The symptoms were akin to what had been observed in hundreds of individuals vaccinated in Europe and the U.K. with AstraZeneca's shot, which uses similar delivery technology as J&J's. (On Tuesday, the European Medicines Agency confirmed a "possible link" between the vaccine and the condition, as it had previously with AstraZeneca's shot.)
While the risk appeared very low, advisers to the Centers for Disease Control and Prevention who met on April 14 to discuss the apparent side effect decided they didn't yet have enough information to issue appropriate guidance on safe use.
They'll meet again on Friday and are expected to recommend resuming vaccinations with some restrictions, perhaps by age. “I would be very surprised," said Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, on CBS Sunday, "if we don't have a resumption in some form by Friday."
A decision could inject those millions of doses back into use, potentially helping accelerate the U.S. immunization effort further. Restoring public faith in the shot, however, could prove a challenge.
The U.S. status of AstraZeneca's coronavirus vaccine
On March 25, AstraZeneca appeared on the cusp of finally bringing its coronavirus vaccine to the FDA for a review. The vaccine proved protective in a U.S.-based Phase 3 trial and AstraZeneca — after a variety of communication missteps — said it would file for an emergency use authorization “in the coming weeks.” One top executive, on CNBC, predicted the application would be submitted by mid-April.
Since then, however, the situation has gotten far more complicated. An unusual public spat with a trial monitoring board led the drugmaker to revise its trial results slightly, raising questions about the company's handling of the data. Foreign regulators, meanwhile, established a possible link between the vaccine and a rare, yet serious side effect involving abnormal blood clots and low platelet counts, leading multiple European countries to either suspend or restrict its use. (This side effect was similar to the six cases reported with s J&J's shot.)
It's now been a month since AstraZeneca disclosed its results, and the company has yet to file its application with the FDA. Moderna, Pfizer, and J&J, by comparison, sought FDA clearance within two weeks of reporting data.
In the meantime, millions of doses pre-ordered by the U.S. government are sitting in storage.
The FDA's long-awaited decision on Biogen's Alzheimer's drug
The world is weeks away from learning the fate of a closely watched Alzheimer's disease drug called aducanumab.
Rather than target symptoms of the disease — as all other approved Alzheimer's medications do — aducanumab is meant to combat its underlying symptoms. One large clinical trial of patients with early Alzheimer's found those who were given a high dose of aducanumab over enough time experienced slower cognitive decline than their peers who got placebo.
But those results are controversial, as are the ways in which aducanumab's developer, Biogen, analyzed them. Another, near-identical trial showed no benefit to the drug at all. Many doctors and researchers are therefore uncertain what, if any, benefit the drug has on patients' daily lives.
What happens next is anyone's guess. The Food and Drug Administration is reviewing aducanumab, with an approval verdict expected by June 7. While the agency is under immense pressure from patients and advocacy groups to bring the treatment to market, its own statisticians and advisers have been resolutely against approving the drug.
Whatever decision the FDA makes, it is likely to have wide-ranging effects on patient care, Biogen's future and on the agency's reputation.
A big moment for Vertex's future
Vertex grew to be one of the world's largest biotechnology companies through its work in cystic fibrosis. Its four marketed drugs are able to treat around 90% of people with the rare genetic disease, and now bring in annual sales of more than $6 billion.
Though this success has helped lift Vertex's share price and market value — as of mid-April, the company was worth $57 billion — investors are eager to see what its next big play will be.
One of the company's more closely watched programs goes after a genetic disorder in which patients experience lung and liver damage because their bodies don't make enough of a certain protein, called alpha-1 antitrypsin or AAT. Vertex's first attempt at treating AAT deficiency was with a drug known as VX-814, but safety issues led the company to stop developing it late last year.
Now, all eyes are on a second drug that Vertex claims is structurally different from VX-814.
This other drug, VX-864, is being tested in a mid-stage study of about 40 participants, with data expected within weeks. Vertex hopes the study will provide the necessary evidence to show VX-864 works as intended. But even if it doesn't, the company has a third AAT drug that could begin human testing sometime this year.
While Vertex continues to express confidence in its AAT research, others aren't so convinced. After last year's setback with VX-814, Geoffrey Porges, an analyst at SVB Leerink, told clients he expected Wall Street to "flush all value" tied to AAT deficiency out of Vertex forecasts — in part because the company has a "not encouraging" track record of keeping programs going after the lead drug fails.
The next update for Sarepta's Duchenne gene therapy
In a year filled with regulatory delays and underwhelming clinical results, the Phase 2 data for Sarepta Therapeutics's gene therapy for Duchenne muscular dystrophy stands out.
Prior to January, Sarepta was the clear leader in a competitive race to prove out a gene therapy for the rare, progressive neuromuscular disease. The biotech had accrued results showing its treatment, called SRP-9001, appeared to boost production of a key protein the young boys who received it lack. Executives suggested Sarepta might be nearing a regulatory submission if the gene therapy could improve motor function in a placebo-controlled Phase 2 trial as well.
That didn't happen, however. The gene therapy missed its main study goal, cutting Sarepta's shares in half and enabling rival programs from Pfizer and Solid Biosciences to gain ground. But executives were adamant the failure was a fluke due to an unlucky randomization process, and claimed future results would prove that out.
Those results, from a "crossover" portion of the Phase 2 trial, likely won't come until later this year. But in the meantime, Sarepta could help its cause by showing that a commercial grade form of its gene therapy — the version it plans to test in a Phase 3 study and eventually market — is comparable to what it's been using in clinical testing. Data from the first 11 patients treated with the commercial version of the treatment are expected this quarter.