Updated: The micro-cap that tapped a mask-skeptic congressman for their Covid DSMB is applying for an EUA. Their evidence? 21 patients
Publishers note: After this report was published, NeuroRx CEO Jonathan Javitt emailed Endpoints with more information on the DSMB, which we added to the story. He got in touch again, and suggested the story wasn’t true. We stand by our story. And I’ve decided to publish Javitt’s email response to this story in its entirety. It appears inline next to each point of contention.
—Arsalan Arif, publisher
NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.
(NOT TRUE. Jason Mast has no idea what data the FDA is looking at. It’s far more than 21 patients — Jonathan Javitt)
Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.
Both EUAs came amid persistent public lobbying from President Trump and one, hydroxychloroquine, was later rescinded. Its authorization had been based only on lab data and a couple small and much-critiqued studies.
Susan Ellenberg, a former FDA reviewer and a biostatistician at the University of Pennsylvania Perelman School of Medicine, said an EUA could be warranted on such limited data, but only if the evidence was overwhelming. She offered the hypothetical of a Phase I study that got multiple complete responses in a hard-to-treat metastatic cancer.
“If they were all severely ill and on ventilators and the treatment resulted in their immediate recovery, testing negative for the virus and rapid discharge from hospital, then maybe,” Ellenberg, who had not reviewed the results, said in an email. “When you achieve something really dramatic in a small number of patients with a fatal disease, FDA is pretty flexible.”
Did NeuroRx meet that threshold, though? Of the 21 patients who received the drug, known as aviptadil, under an expanded protocol, 19 survived and seven were discharged. Seven, though, were still in immediate care and 5 were in the ICU. Of five patients on ECMO, three were decannulated — breathing tube removed — and two were discharged.
“The activity of the synthetic Vasoactive Intestinal Peptide (aviptadil) appears promising,” University of Minnesota virologist David Boulware said in an email. “Their reported results are very good but not earth shattering. Without a randomized control group, what the true actual effect is unknown. If highly effective, then a small randomized clinical trial should be able to quickly confirm these initial results.”
NeuroRx, which had been developing a drug for depression before pivoting when the pandemic hit, launched such a trial in March. At the beginning of August, CEO Jonathan Javitt touted “overnight recovery” in a couple of patients who took the drug outside the Phase II study, under an FDA-authorized expanded access protocol. He said at the time the DSMB had cleared them back in July and promised more data would soon come, with another review scheduled for Aug. 24, after they enrolled their 81st patient.
“The reality is, within a month, we’re going to have a lot more data,” he told Endpoints News at the time. “We’re going to have data from the RCT.”
Those results have yet to materialize, though, and last week, Politico raised questions about the DSMB’s composition. Javitt, they reported, had picked mask-skeptic Republican congressman Andy Harris and two other acquaintances to run the board, raising concerns from some ethicists who questioned how independent they could be and how qualified they were to assess whether a trial should continue. Javitt and Harris defended the decision, pointing to Harris’ medical experience.
In an email after publication, Javitt said the DSMB also included biostatistician Phillip Lavin
(TRUE, Phil has been the statistician on more new drug approvals than any statistician in US history — Jonathan Javitt)
… and had never worked directly with Al Sommer, one of the other members of the DSMB Politico reported,
(I never said that. It’s readily verifiable that Sommer and I were co-authors on a number of scientific papers in the 1980s and 90s. I did tell Politico about Sommer’s credentials as a Lasker Awardee and member of the National Academy of Sciences, which of course did not interest them because their objective was to smear Harris. — Jonathan Javitt)
… and had never met Rita Colwell, the other member, before.
(TRUE — Jonathan Javitt)
Colwell is an aquatic biologist who has not previously served on DSMBs,
(Not True. Dr. Colwell says she has served before — Jonathan Javitt)
but Javitt said he “wanted a non-medical member of the committee who could understand the data and still represent the lay public’s point.”
(TRUE, and the FDA guidance says to include members of the public — Jonathan Javitt)
He said the FDA had requested they delay the August 24 meeting after the DSMB recommended a change in endpoint. The agency wanted 21 more patients and have just enrolled patient number 100. “Likely the DSMB will meet in the next four weeks,” he said.
In a press release about the EUA submission, Javitt said they were applying for the EUA because they have discovered many hospitals do not allow patients access to experimental drugs. They say it would only cover patients who have exhausted other treatment options and would have been eligible for the EAP.
“Tragically, we have found that many patients who might benefit from the Expanded Access protocol are hospitalized in centers that do not allow patients access to investigational drugs,” he said. “Our staff is working around the clock to address requests for RLF-100™ under the appropriate mechanism. All too often, patients of color and those of lower socioeconomic status are denied access.”
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