Mer­ck KGaA, Pfiz­er's Baven­cio set to seal blad­der can­cer ap­proval af­ter con­fir­ma­to­ry tri­al scores

Baven­cio could use the win.

More than two years af­ter its mak­ers — Ger­many’s Mer­ck KGaA and Pfiz­er — se­cured FDA ap­proval for a form of blad­der can­cer, the com­pa­nies un­veiled that the con­fir­ma­to­ry study test­ing the PD-L1 in­hibitor had per­formed as ex­pect­ed, help­ing pa­tients live longer as a main­te­nance treat­ment ver­sus stan­dard-of-care.

The drug was grant­ed ac­cel­er­at­ed ap­proval on the ba­sis of tu­mor re­sponse and du­ra­tion of re­sponse in May 2017 in pa­tients with lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma (UC) whose dis­ease has pro­gressed dur­ing or af­ter plat­inum-con­tain­ing chemother­a­py. On Mon­day, the com­pa­nies said that da­ta from the con­fir­ma­to­ry JAVELIN Blad­der 100 tri­al showed pa­tients treat­ed with Baven­cio lived sig­nif­i­cant­ly longer than those who re­ceived stan­dard care.

A sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in over­all sur­vival was demon­strat­ed in the Baven­cio arm in each of the co-pri­ma­ry pop­u­la­tions: all ran­dom­ized pa­tients and pa­tients with PD-L1–pos­i­tive tu­mors, the com­pa­ny added, with­out dis­clos­ing the num­bers. The da­ta will be hand­ed over to the FDA.

Baven­cio was late to the check­point in­hibitor game. Mer­ck’s mar­ket-lead­ing Keytru­da and Bris­tol-My­ers’ pi­o­neer­ing Op­di­vo were the first two PD-1/L1 drugs to make the cut, and have since scored var­i­ous ap­provals across im­muno-on­col­o­gy and now reap in bil­lions in sales. Re­gen­eron, Roche and Chi­na-based drug de­vel­op­ers al­so have their own check­points — PARP in­hibitors are al­so the mix.

Last Sep­tem­ber, Cowen an­a­lysts shaved their 2024 pro­jec­tions for Baven­cio by $170 mil­lion to $480 mil­lion, cit­ing flat growth and rel­a­tive­ly few cat­a­lysts ahead. In com­par­i­son, Op­di­vo is ex­pect­ed to gen­er­ate a meaty $12.4 bil­lion that year, and king Keytru­da a hefty $20.6 bil­lion, the an­a­lysts es­ti­mat­ed.

Baven­cio, for which Pfiz­er paid $850 mil­lion up­front in 2014 to part­ner with Mer­ck KGaA on, has had some se­ri­ous set­backs in its quest to make a mark. The part­ners were forced to ad­mit de­feat in ovar­i­an can­cer in March last year af­ter three straight tri­al fail­ures. Months lat­er in No­vem­ber, the drug al­so fiz­zled as a first-line main­te­nance ther­a­py for gas­tric can­cer pa­tients, years af­ter fal­ter­ing in a gas­tric can­cer study to im­prove over­all sur­vival. In 2018, Baven­cio al­so botched a key study in the lu­cra­tive field of lung can­cer.

The com­pa­nies, how­ev­er, did have some luck in kid­ney can­cer. Baven­cio was grant­ed ap­proval in May 2019 in com­bi­na­tion with Pfiz­er’s ty­ro­sine ki­nase in­hibitor In­ly­ta for use as a front­line ther­a­py for re­nal cell car­ci­no­ma, af­ter late-stage da­ta showed the com­bi­na­tion demon­strat­ed a sig­nif­i­cant pro­gres­sion-free sur­vival ben­e­fit. It is al­so ap­proved for use in Merkel cell car­ci­no­ma.

UC ac­counts for about 90% of all blad­der can­cer cas­es. When blad­der can­cer is metasta­t­ic, the five-year sur­vival rate is 5%, ac­cord­ing to Amer­i­can Can­cer So­ci­ety es­ti­mates.

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.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? 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.

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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.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.