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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

(Photo courtesy Pfizer)

FDA's vac­cine ad­comm votes al­most unan­i­mous­ly in fa­vor of Pfiz­er's Covid-19 vac­cine for younger chil­dren

The FDA’s Vaccines and Related Biological Products Advisory Committee on Tuesday voted 17-0, with one panelist abstaining, that the benefits of the Pfizer-BioNTech Covid-19 vaccine outweigh the risks for children between the ages of five and 12.

The vote will likely trigger a process that could allow the shots to begin rolling out as early as next week.

The vaccine, which is one-third of the adult Pfizer dose, proved to be about 90% effective in a placebo-controlled trial in which about 1,500 kids in this age range received the vaccine, and only about 12% of those receiving the vaccine had any adverse event. All serious adverse events in the trial were unrelated to the vaccine.

Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

UP­DAT­ED: As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.