Nader Pourhassan, CytoDyn CEO (Jeff Kravitz/FilmMagic for CytoDyn’s Pro, Getty Images)

Cy­to­Dyn shares slammed as BLA fil­ing for leron­limab in HIV hits a wall

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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

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.

Rep. Cathy McMorris Rodgers (R-WA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House com­mit­tee chair re­quests in­fo from FDA on fund­ing for tri­als in­volv­ing 'vi­ral ma­nip­u­la­tion'

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.”

FDA plans up­com­ing ad­comms for OTC con­tra­cep­tive, ep­i­neph­rine nasal spray, Intar­ci­a's last shot

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.

Robert Califf, FDA commissioner (Photo by Drew Angerer/Getty Images)

House com­mit­tee to in­ves­ti­gate FDA's re­sponse to on­go­ing drug short­ages

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.

Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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Kevin Lee, Bicycle Therapeutics CEO

No­var­tis rides with Bi­cy­cle for new pact on tar­get­ed ra­dio­ther­a­pies

Novartis has inked a three-year deal with Bicycle Therapeutics to develop new targeted radiotherapies for cancer.

Novartis will pay Bicycle $50 million upfront, with downstream milestones adding up to a potential $1.7 billion. In exchange, Bicycle will use its virus-based platform to discover new bicyclic peptides, which it calls bicycles, that would be used for radiotherapies. Those bicycles would act as a homing beacon for radioactive isotopes, delivering them to cancer cells to kill the cells while limiting radiation to healthy tissue.

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