Mitchell Steiner, Veru CEO

FDA ten­ta­tive­ly hints at EUA, with a tri­al re­quire­ment, for Veru's new Covid drug ahead of ad­comm

While ac­knowl­edg­ing that Veru’s po­ten­tial drug for Covid-19 hos­pi­tal­iza­tions hit the mark on mor­tal­i­ty in a small tri­al, and may have enough for an EUA with a con­fir­ma­to­ry tri­al add-on, the FDA al­so point­ed to un­cer­tain­ties in the da­ta ahead of an ad­comm meet­ing Wednes­day.

The FDA’s Pul­monary-Al­ler­gy Drugs Ad­vi­so­ry Com­mit­tee will dis­cuss the re­pur­posed drug, known as sabiz­ab­u­lin, which was orig­i­nal­ly tar­get­ed at cas­tra­tion-re­sis­tant prostate can­cer and now may be au­tho­rized for hos­pi­tal­ized adults with mod­er­ate to se­vere Covid-19 who are at high risk for acute res­pi­ra­to­ry dis­tress syn­drome.

Ac­cord­ing to brief­ing doc­u­ments re­leased ahead of the meet­ing on Mon­day morn­ing, the agency sound­ed a cau­tious­ly pos­i­tive but un­cer­tain tone for the mi­cro­tubule in­hibitor, as FDA says the “mech­a­nism of ac­tion in COVID-19 is un­cer­tain.”

De­spite some ques­tion marks, the pan­dem­ic has shown so far that the bar for an EUA is sig­nif­i­cant­ly low­er than for a full ap­proval, and there have been nu­mer­ous prod­ucts (drugs and tests) that have now re­ceived EUAs dur­ing this pan­dem­ic and since been pulled from the mar­ket.

Even still, FDA said it is “con­sid­er­ing re­quir­ing a tri­al to be con­duct­ed as a con­di­tion of au­tho­riza­tion (in line with the above statu­to­ry pro­vi­sion) if au­tho­riza­tion is grant­ed.”

That sig­nal sent Veru’s share price up more than 50% at one point on Mon­day.

While re­quest­ing that the pan­el “dis­cuss rel­e­vant de­sign as­pects of this tri­al to be con­duct­ed as a con­di­tion of au­tho­riza­tion,” the FDA ex­plains how its re­view of the da­ta shows that “none of these un­cer­tain­ties or im­bal­ances alone in­val­i­date the mor­tal­i­ty ben­e­fit ob­served” in the spon­sor’s key study, “but all of these is­sues to­geth­er in a small tri­al which is more vul­ner­a­ble to im­bal­ances raise ques­tions about the re­sults.”

Un­cer­tain­ties raised by FDA’s brief­ing doc­u­ments in­clude: “High place­bo group mor­tal­i­ty rate; Po­ten­tial for un­blind­ing events with en­ter­al tube ad­min­is­tra­tion; Base­line im­bal­ances in stan­dard of care ther­a­pies; Dif­fer­ences in hos­pi­tal­iza­tion du­ra­tion pri­or to tri­al en­roll­ment; Un­cer­tain ef­fects of goals of care de­ci­sions on all-cause mor­tal­i­ty; Neg­a­tive stud­ies with oth­er mi­cro­tubule dis­rup­tors in COVID-19; Un­cer­tain­ty in iden­ti­fi­ca­tion of a clin­i­cal­ly rel­e­vant pa­tient pop­u­la­tion.”

The FDA is ask­ing the pan­el to con­sid­er these un­cer­tain­ties and how they af­fect the in­ter­pretabil­i­ty of the mor­tal­i­ty da­ta, mak­ing clear that even when us­ing an ob­jec­tive end­point like mor­tal­i­ty, “ob­served re­sults can be sub­ject to bi­as­es in a small tri­al of short du­ra­tion in crit­i­cal­ly ill pa­tients.”

The one vot­ing ques­tion on Wednes­day for the pan­el will ad­dress whether “the known and po­ten­tial ben­e­fits of VERU-111, when used for the treat­ment of adult pa­tients hos­pi­tal­ized with COVID-19 at high risk of ARDS, out­weigh the known and po­ten­tial risks of VERU-111.” The FDA does not have to fol­low the ad­vice of its ad­vi­so­ry com­mit­tees, but it of­ten does.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Days af­ter re­port­ing PhI­II fail­ure, GSK pulls BC­MA drug from US mar­ket — but it's not giv­ing up en­tire­ly yet

GSK is pulling its BCMA-targeting drug from the US market, ending a short, two-year run for a high-profile product that, among other things, was hailed for marking the pharma giant’s return to oncology.

The company is initiating the process for withdrawal at the request of the FDA, which in turn was based on the negative readout of a confirmatory Phase III trial earlier this month. In that trial, GSK’s Blenrep failed to extend progression-free survival over standard of care for patients with multiple myeloma who have received at least two prior lines of therapy.

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