Covid-19 roundup: Fau­ci, Collins in­ter­vene to stop FDA plas­ma au­tho­riza­tion, cit­ing weak da­ta; Roche, Re­gen­eron try to take an­ti­bod­ies glob­al

The FDA has placed on hold an emer­gency au­tho­riza­tion for the use of plas­ma as a Covid-19 treat­ment fol­low­ing the in­ter­ven­tion of top fed­er­al health of­fi­cials who claimed the avail­able da­ta were too weak, ac­cord­ing to a re­port in the New York Times.

NI­AID clin­i­cal di­rec­tor H. Clif­ford Lane con­firmed the hold to the Times, adding plas­ma may still be au­tho­rized some­time in the fu­ture.

Much has been made about the prospect of uti­liz­ing do­nat­ed plas­ma from re­cov­ered Covid-19 pa­tients to treat those cur­rent­ly in­fect­ed with the dis­ease. The the­o­ry goes that an­ti­bod­ies in the plas­ma can help re­duce the time pa­tients spend in hos­pi­tals as well as mor­tal­i­ty rates if the treat­ment is ad­min­is­tered quick­ly.

The use of plas­ma to treat dis­ease has been around since the ear­ly 20th cen­tu­ry and doc­tors used sim­i­lar ther­a­pies dur­ing the in­fluen­za pan­dem­ic of 1918. One of the biggest stud­ies in­to adapt­ing the tech­nique for Covid-19 came from the Mayo Clin­ic last week, which showed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in mor­tal­i­ty rates among the most se­vere cas­es. In­di­vid­u­als re­ceiv­ing plas­ma treat­ment with­in three days of di­ag­no­sis had a sev­en-day death rate of 8.7% while those who got plas­ma four days or lat­er had an 11.9% rate.

How­ev­er, that study was lim­it­ed by the lack of a place­bo group, a draw­back the Mayo Clin­ic it­self ad­dressed at the time. Sev­er­al top health of­fi­cials, such as Lane, An­tho­ny Fau­ci and Fran­cis Collins, urged the FDA to hold off on plas­ma au­tho­riza­tion based on the da­ta from that study, the Times wrote.

Wednes­day’s hold fol­lows a cadre of for­mer FDA com­mis­sion­ers ac­knowl­edg­ing the po­ten­tial ben­e­fits of plas­ma treat­ments in an op-ed to the Wash­ing­ton Post two weeks ago. They cau­tioned, though, that more re­search in­to the ther­a­py’s safe­ty and ef­fi­ca­cy is need­ed. — Max Gel­man

UP­DAT­ED: Roche part­ners with Re­gen­eron on a Covid-19 an­ti­body cock­tail, adding glob­al man­u­fac­tur­ing/mar­ket­ing mus­cle

It’s one of those un­like­ly al­liances you’d on­ly see in a pan­dem­ic: Roche is sign­ing on to be a man­u­fac­tur­ing and dis­tri­b­u­tion part­ner for Re­gen­eron’s tai­lor-made Covid-19 an­ti­body cock­tail.

There’s no up­front or mile­stones, but the com­pa­nies say they’ve al­ready be­gun the tech trans­fer process for Roche, with an agree­ment in place since late Ju­ly. The Swiss phar­ma gi­ant will be tasked with dis­tri­b­u­tion — plus all the clin­i­cal and reg­u­la­to­ry work need­ed — out­side of the US, while the Tar­ry­town, NY-based biotech fo­cused on the home turf.

By team­ing up with Roche, Re­gen­eron ex­pects to in­crease sup­ply of REGN-COV2 by at least three and a half times. Roche will hold the rights for at least sev­en years af­ter the first com­mer­cial sales in the Eu­ro­pean Union, as­sum­ing all goes smooth­ly.

In Ge­of­frey Porges’ view, the col­lab­o­ra­tion adds cred­i­bil­i­ty to a pro­gram that still doesn’t have clin­i­cal val­i­da­tion and could have been re­strict­ed by man­u­fac­tur­ing ca­pac­i­ty, crowd­ed out by US or­ders.

“While the Roche col­lab­o­ra­tion will re­duce REGN’s prof­its from REGN-COV2, it should sub­stan­tial­ly re­duce the de­vel­op­ment cost, which would have bur­dened Re­gen­eron for the next few years (est. half-bil­lion dol­lar cost for REGN-COV2 in 2020E based on Re­gen­eron’s Q2 guid­ance re­vi­sion),” he wrote.

While Re­gen­eron CEO Len Schleifer has pre­vi­ous­ly pre­dict­ed it could have emer­gency dos­es ready by the end of sum­mer, clin­i­cal tri­als ap­pear to be tak­ing longer than ex­pect­ed as in­ves­ti­ga­tors strug­gle to en­roll pa­tients in over­whelmed hos­pi­tals. In the most re­cent an­a­lyst call, Schleifer said they’re now hop­ing to gen­er­ate ini­tial da­ta by ear­ly Sep­tem­ber.

Porges es­ti­mates the bench­mark for ef­fi­ca­cy in the treat­ment set­ting to be 50% to 60% re­duc­tion in dis­ease mor­tal­i­ty, and that for pro­phy­lax­is to be 60% or greater re­duc­tion in clin­i­cal­ly sig­ni­cant, symp­to­matic Covid-19 dis­ease — both like­ly achiev­able judg­ing from pri­mate da­ta.

A spokesper­son told End­points News that the ini­tial da­ta in Sep­tem­ber will in­clude vi­rol­o­gy and bio­mark­ers.

In ad­di­tion to hav­ing Roche joint­ly fund the on­go­ing Phase III pre­ven­tion study, a Phase I mul­ti-dose safe­ty tri­al and any new glob­al stud­ies, Re­gen­eron is al­so keep­ing its hand on a sig­nif­i­cant por­tion of the prof­its.

From the 8-K:

Any world­wide gross prof­its from the An­ti­body Prod­uct will be ag­gre­gat­ed and shared based on a pre-spec­i­fied for­mu­la, which is es­ti­mat­ed to re­sult in Re­gen­eron re­ceiv­ing ap­prox­i­mate­ly 50% – 60% of the world­wide gross prof­its, de­pend­ing on the amount of man­u­fac­tured An­ti­body Prod­uct de­liv­ered by each par­ty.

Bill An­der­son

Bill An­der­son, the CEO of Roche Phar­ma, high­light­ed the po­ten­tial for REGN-COV2 both as a treat­ment and pro­tec­tion for peo­ple ex­posed to the coro­n­avirus, mak­ing it a “crit­i­cal line of de­fense.” De­signed specif­i­cal­ly to com­bat SARS-CoV-2, the ther­a­py com­pris­es two dif­fer­ent virus-neu­tral­iz­ing an­ti­bod­ies that bind to the re­cep­tor bind­ing do­main of the virus’ spike pro­tein.

“We’re com­mit­ting our man­u­fac­tur­ing ex­per­tise and ca­pac­i­ty, and our glob­al dis­tri­b­u­tion net­work to bring Re­gen­eron’s po­ten­tial an­ti­body com­bi­na­tion to as many peo­ple around the world as we pos­si­bly can,” he said.

That trans­lates to at least 100,000 liters of “an­nu­al­ized biore­ac­tor ca­pac­i­ty on a full-time cam­paign ba­sis” to man­u­fac­ture the prod­uct, ac­cord­ing to an SEC fil­ing from Re­gen­eron. On its part, Re­gen­eron will ded­i­cate 40,000 liters of its in-house ca­pac­i­ty to the Covid-19 pro­gram.

His­toric ri­vals in eye dis­eases and in­flam­ma­to­ry con­di­tions, Roche and Re­gen­eron (part­nered with Sanofi) took their re­spec­tive IL-6 an­ti­bod­ies to tri­als in hopes of damp­en­ing the dam­ag­ing im­mune re­sponse that plague pa­tients with se­vere Covid-19 — on­ly to be slapped by fail­ures. That left Roche with lit­tle to of­fer in the ther­a­peu­tic fight against the coro­n­avirus, even as its di­ag­nos­tics unit con­tin­ues to sell much-need­ed anti­gen and an­ti­body tests.

Schleifer sees the deal as val­i­da­tion for his team’s record speed and tire­less work as the phar­ma part­ner pro­vides “im­por­tant scale” to the en­deav­or.

He has struck a $450 mil­lion deal with Op­er­a­tion Warp Speed to sup­ply up to 1.6 mil­lion dos­es in the US, both for ther­a­peu­tic and pro­phy­lac­tic use. Back in March he not­ed the com­pa­ny could be­gin mak­ing 200,000 clin­i­cal dos­es a month be­gin­ning in Au­gust, and it’s been look­ing to max­i­mize ca­pac­i­ty on com­mer­cial scale ma­te­r­i­al. — Am­ber Tong

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.

President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

Covid-19 roundup: J&J boost­er shot da­ta show promise; CD­C's ACIP meet­ing this week to dis­cuss Pfiz­er boost­ers

J&J revealed a summary of new Covid-19 vaccine data today, including new results showing booster shots may help with protection.

A Phase III study (ENSEMBLE 2) looked at booster shots at two different points in time: a second shot 56 days after the first shot, or a second shot six months after the first. The eight-week shot showed increased protection against symptomatic Covid-19, with the following levels of protection:

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.