FDA re­jects Covid-19 drug over un­cer­tain­ties around small tri­al

Fol­low­ing the ad­vice of a pan­el of out­side ex­perts, the FDA has turned down a po­ten­tial Covid-19 drug that on­ly had da­ta from a small tri­al.

Mi­a­mi, FL-based Veru had re­pur­posed a mi­cro­tubule dis­rup­tor called sabiz­ab­u­lin, which it had been test­ing in can­cer, to treat hos­pi­tal­ized adult pa­tients with mod­er­ate to se­vere Covid-19 who are at high risk for ARDS, or acute res­pi­ra­to­ry dis­tress syn­drome.

CEO Mitchell Stein­er sug­gest­ed that the FDA re­ject­ed the drug “be­cause of the pos­si­bil­i­ty of un­known in­flu­ences, or un­cer­tain­ties that may have af­fect­ed the study.” The com­pa­ny not­ed that the agency of­fered com­ments for a pro­posed con­fir­ma­to­ry Phase III.

Shares $VERU fell more than 30% in pre-mar­ket trad­ing to $2.55.

To sup­port its EUA, Veru sub­mit­ted da­ta from 204 pa­tients hos­pi­tal­ized with Covid. It stopped the tri­al ear­ly be­cause of clin­i­cal ben­e­fit.

In brief­ing doc­u­ments pre­pared ahead of an ad­vi­so­ry com­mit­tee, the FDA had spelled out its con­cerns with the piv­otal da­ta that Veru sub­mit­ted. It ac­knowl­edged that sabiz­ab­u­lin hit the mark on mor­tal­i­ty in the small tri­al, but not­ed un­cer­tain­ties or im­bal­ances that, while not prob­lem­at­ic in­di­vid­u­al­ly, “raise ques­tions about the re­sults.”

FDA’s Pul­monary-Al­ler­gy Drugs Ad­vi­so­ry Com­mit­tee gen­er­al­ly of­fered sup­port, with some qualms, on the sur­vival da­ta. Yet when it came to rec­om­mend­ing an OK, the ad­comm mem­bers large­ly ar­gued that there weren’t enough safe­ty and ef­fi­ca­cy da­ta, un­der­stand­ing of the drug was still lim­it­ed, and that the demon­strat­ed ben­e­fits don’t out­weigh the risks. Vot­ing 8 to 5 against its au­tho­riza­tion, many asked for a new tri­al.

Treat­ment op­tions for Covid-19 re­main lim­it­ed, with many of the new ther­a­pies — from an­ti­bod­ies to an­tivi­rals — in mild-to-mod­er­ate set­tings. For hos­pi­tal­ized pa­tients, the FDA has cleared sev­er­al an­ti-in­flam­ma­to­ry drugs, in­clud­ing Actem­ra, Olu­mi­ant and Kineret.

Veru said it re­mains com­mit­ted to de­vel­op­ing sabiz­ab­u­lin for the high-risk pa­tient pop­u­la­tion, and added that the drug is un­der reg­u­la­to­ry re­view in oth­er coun­tries.

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.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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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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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Blue­bird to miss Q1 dead­line for lo­vo-cel fil­ing, shares sliced

Bluebird bio had expected to submit a BLA for its sickle cell disease gene therapy lovotibeglogene autotemcel, or lovo-cel, by Friday. Now, that deadline is getting punted for at least a couple of weeks.

CEO Andrew Obenshain detailed the update during the company’s full-year earnings call Wednesday morning after the gene therapy specialist reported the development in its Q4 and 2022 report.

“We will likely miss the Q1 2023 submission goal,” Obenshain said, noting the application is fully written and ready to go. But bluebird is still waiting on FDA feedback for the company’s CMC module submitted in early March.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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