NeuroRx, the tiny biotech that came under fire last week after Politicoreported 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.

Such an application would test the agencys 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Both EUAs came amid persistent public lobbying from President Trump and one, hydroxychloroquine, was later rescinded.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 smalland 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, were going to have a lot more data, he told Endpoints News at the time. Were going to have data from the RCT.

Those results have yet to materialize, though, and last week,Politicoraised questions about the DSMBs 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 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.

Javitt has not responded to multiple inquiries about the status of the trial, the results of the interim analysis and when they expect to have data.

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The micro-cap that tapped a mask-skeptic congressman for their Covid DSMB is applying for an EUA. Their evidence? 21 patients - Endpoints News