In a press re­lease is­sued in ear­ly June an­nounc­ing a BLA ac­knowl­edg­ment let­ter from the FDA, Cy­to­Dyn CEO Nad­er Pourhas­san said he is hope­ful about get­ting a PDU­FA date for its lead drug, leron­limab, on Ju­ly 10.

In­stead, they re­ceived a refuse-to-file let­ter to­day.

The com­pa­ny said the agency is just look­ing for “cer­tain in­for­ma­tion need­ed to com­plete a sub­stan­tive re­view.” No clin­i­cal tri­als are nec­es­sary; all that’s need­ed is ad­di­tion­al analy­sis and a meet­ing with the FDA.

The spurn of reg­u­la­tors marked one of the surest episodes in a sub­mis­sion cam­paign shroud­ed in un­cer­tain­ty. Back in late April, Cy­to­Dyn had told share­hold­ers that it’s sub­mit­ted a com­plete BLA for leron­limab as a com­bi­na­tion ther­a­py with an an­ti­retro­vi­ral reg­i­men for HIV pa­tients who are high­ly treat­ment-ex­pe­ri­enced. It then added on May 7 that it need­ed to send in clin­i­cal datasets with­in days be­fore the FDA would con­sid­er the fil­ing; the press re­lease about the ac­knowl­edg­ment let­ter didn’t come out un­til June 8.

The chaot­ic com­mu­ni­ca­tion was cit­ed promi­nent­ly in a short re­port by Cit­ron Re­search, call­ing for the SEC to “im­me­di­ate­ly halt this stock pro­mo­tion scheme.”

Pourhas­san hit back by call­ing the re­port fraud­u­lent and tout­ing the $87.5 mil­lion in mile­stones he ne­go­ti­at­ed in a li­cens­ing deal with Vy­era Phar­ma, the lat­est rein­car­na­tion of Mar­tin Shkre­li-found­ed Tur­ing Phar­ma.

Vy­era holds rights to mar­ket and dis­trib­ute leron­limab in the US for HIV — if it gets ap­proved. RTF let­ters can take months, if not years, to re­solve, even for ex­pe­ri­enced drug­mak­ers. Cy­to­Dyn has nev­er brought a new com­pound to mar­ket.

A CCR5 an­tag­o­nist, leron­limab sup­pos­ed­ly works by in­hibit­ing vi­ral en­try and pro­tect­ing healthy T cells from vi­ral in­fec­tion.

Aside from HIV, Cy­to­Dyn has the drug for NASH and CCR5+ metasta­t­ic triple neg­a­tive breast can­cer. More re­cent­ly, Covid-19 was spot­light­ed as a dis­ease po­ten­tial­ly ad­dressed by the an­ti­body.

Cy­to­Dyn tasked Bruce Pat­ter­son, the chief of its di­ag­nos­tic part­ner In­cellDx, with ex­plain­ing how:

“Leron­limab has both the po­ten­tial to en­hance the cel­lu­lar im­mune re­sponse by sup­press­ing Treg cells that, in turn, in­hib­it the an­ti-vi­ral T-cell re­spons­es and the po­ten­tial to re­po­lar­ize macrophage ac­tiv­i­ty,” Pat­ter­son said in Jan­u­ary, when Cy­to­Dyn be­gan an eval­u­a­tion. “Lung (alve­o­lar) macrophages in coro­n­avirus in­fec­tions have been im­pli­cat­ed as a con­tribut­ing fac­tor to sig­nif­i­cant mor­bid­i­ty and mor­tal­i­ty of the in­fec­tious dis­ease. Leron­limab could po­ten­tial­ly syn­er­gize with oth­er retro­vi­ral ther­a­pies that cur­rent­ly be­ing used for the po­ten­tial treat­ment of 2019-nCoV.”

Phase II da­ta from a tri­al in­volv­ing mild to mod­er­ate pa­tients are ex­pect­ed lat­er this month, as are re­sults from a safe­ty re­view for a Phase III study in the se­vere/crit­i­cal pop­u­la­tion.

Ju­ly is shap­ing up to be an im­por­tant month, Pourhas­san promised, and the RTF is noth­ing to wor­ry about.

“We are 100% com­mit­ted and con­fi­dent we can pro­vide the nec­es­sary in­for­ma­tion to the FDA as soon as pos­si­ble,” he said in a state­ment. “No ad­di­tion­al tri­als will be re­quired and all the in­for­ma­tion the FDA has re­quest­ed is ob­tain­able.”

In­vestors are less con­fi­dent. Shares $CY­DY fell 21.99% to $3.69.

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Republican members of Congress want FDA Commissioner Robert Califf and his agency to provide more information on the research FDA has funded that manipulates certain viruses like the SARS-CoV-2 virus — after the FDA confirmed that certain studies it funded involved viral manipulation, but in standard ways that are common in molecular biology.

The three-page letter, signed Monday by House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Subcommittee Chairs Brett Guthrie (R-KY) and Morgan Griffith (R-VA), kicked things off by saying that the committee was looking into FDA’s oversight of “FDA-funded research that may pose significant biosafety or biosecurity risks.”

The FDA has scheduled or planned for three advisory committee meetings in May: one for an over-the-counter birth control pill, one for a nasal spray version of the EpiPen, and another for  Intarcia Therapeutics, which has all but completely folded already as a company.

Perrigo will bring its daily contraceptive, Opill, to the joint Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive and Urologic Drugs Advisory Committee on May 9 and 10 for its over-the-counter birth control application.

Republican leaders of the House Committee on Energy & Commerce sent a five-page letter yesterday announcing an investigation into the ongoing drug shortages that have rankled the US during the pandemic and the FDA’s response to it.

The letter, signed by Chair Cathy McMorris Rodgers (R-WA), explains how shortages have become more common over the past decade, while pointing to a report from the National Academies of Science, Engineering and Medicine finding that drug shortages have been “on the rise” over the past several decades and are lasting longer, with new drug shortages in the US seeing a 30% increase from 2021 to 2022.