No­var­tis, BeiGene's PD-(L)1 play takes home an­oth­er win in late-stage da­ta, adding some heft to its mar­ket po­ten­tial

Con­tin­u­ing its quest to shake up the PD-1 mar­ket, BeiGene re­vealed more da­ta Fri­day for its check­point in­hibitor tislelizum­ab, this time in re­cur­rent or metasta­t­ic na­sopha­ryn­geal can­cer.

Re­veal­ing in­ter­im topline re­sults from a Phase III study, BeiGene said the drug met its pri­ma­ry end­point of pro­gres­sion-free sur­vival in the first-line set­ting, com­bined with chemother­a­py. The drug topped chemother­a­py alone at a sta­tis­ti­cal­ly sig­nif­i­cant rate, BeiGene said, po­ten­tial­ly set­ting up a Chi­nese ap­proval in the in­di­ca­tion.

Yong Ben

“This is our fifth pos­i­tive Phase III read­out for tislelizum­ab, which we are de­vel­op­ing broad­ly as a po­ten­tial­ly dif­fer­en­ti­at­ed an­ti-PD-1 an­ti­body,” CMO of I/O Yong Ben said in a state­ment.

First ap­proved in Chi­na in late 2019, tislelizum­ab has steadi­ly been chalk­ing up more ap­provals af­ter be­ing first con­di­tion­al­ly OK’ed for third-line re­lapsed/re­frac­to­ry Hodgkin’s lym­phoma. The drug has since gar­nered full ap­proval for first-line ad­vanced squa­mous NSCLC, as well as con­di­tion­al ap­provals in blad­der can­cers with high PD-L1 ex­pres­sion.

The lat­est Phase III ef­fort comes from a tri­al of 263 pa­tients in Asia, who were ran­dom­ized 1:1 in­to the drug or place­bo plus chemo arms. BeiGene did not re­lease any fig­ures or p-val­ues re­gard­ing the re­sults.

Fri­day’s re­sults are part of BeiGene’s con­tin­u­ing ef­fort to ex­pand the drug’s use in Chi­na, and NPC could prove to be a big one. Near­ly half of all glob­al NPC cas­es oc­cur in the coun­try, with high­er rates in south­ern Chi­na. Rough­ly 60,000 Chi­nese in­di­vid­u­als were di­ag­nosed with the dis­ease in 2018, BeiGene said.

But BeiGene is al­so ap­par­ent­ly ready to soon take the drug over­seas. Ear­li­er this year, No­var­tis plunked down $650 mil­lion in up­front cash to part­ner with the biotech, promis­ing up to $1.55 bil­lion in fu­ture mile­stones. For that price, No­var­tis won com­mer­cial and co-de­vel­op­ment rights to most ma­jor mar­kets out­side of Chi­na.

They haven’t sub­mit­ted any reg­u­la­to­ry fil­ings just yet, but the duo is chas­ing more than a dozen po­ten­tial in­di­ca­tions. Among the group are NSCLC, he­pa­to­cel­lu­lar car­ci­no­ma, esophageal squa­mous cell car­ci­no­ma, gas­tric can­cer and NPC.

The drug is al­ready see­ing big up­take in Chi­na as well, record­ing $48.9 mil­lion sales in the first quar­ter — a fig­ure up near­ly 140% from the first quar­ter of 2020. Es­ti­mates have on­ly grown for the drug, with Leerink an­a­lyst An­drew Berens last year rais­ing peak sales ex­pec­ta­tions to $768 mil­lion, up from $461 mil­lion pre­vi­ous­ly.

BeiGene’s NPC da­ta come from a swath of po­ten­tial reg­is­tra­tional tri­als: The biotech said it has launched or com­plet­ed 17 dif­fer­ent stud­ies for the check­point in­hibitor.

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.

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.

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.

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.

Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

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 117,800+ biopharma pros reading Endpoints daily — and it's free.

Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

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 117,800+ biopharma pros reading Endpoints daily — and it's free.

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.