Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfiz­er and Mer­ck KGaA have de­tailed the Phase III da­ta that in­spired FDA reg­u­la­tors to des­ig­nate Baven­cio a “break­through” for first-line ad­vanced blad­der can­cer and of­fered an ear­ly glance at how the PD-L1 can help pa­tients with a rare gy­ne­co­log­i­cal can­cer — carv­ing out nich­es in the check­point space for it­self af­ter be­ing shut out of nu­mer­ous oth­ers.

In JAVELIN Blad­der 100, Baven­cio led to a 31% re­duc­tion in risk of death com­pared to stan­dard care alone. It al­so ex­tend­ed me­di­an sur­vival by more than sev­en months — a his­toric feat in this set­ting, ac­cord­ing to in­ves­ti­ga­tors at Queen Mary Uni­ver­si­ty of Lon­don.

All 700 pa­tients in the tri­al com­plet­ed chemother­a­py be­fore get­ting ran­dom­ized to ei­ther main­te­nance ther­a­py with Baven­cio or reg­u­lar check­ing. Those on the drug arm saw mOS of 21.4 months, com­pared with 14.3 months on the com­para­tor group.

Around one in 10 pa­tients stopped the drug due to treat­ment prob­lems, though the com­pa­nies said side ef­fects were in line with ex­pec­ta­tions.

“This high­lights the po­ten­tial ben­e­fits of a main­te­nance ap­proach with avelum­ab in pa­tients to pro­long their lives fol­low­ing chemother­a­py,” Thomas Powles, a study lead and pro­fes­sor of gen­i­touri­nary on­col­o­gy at Queen Mary, said in a state­ment.

The find­ings could be “prac­tice-chang­ing,” he added, es­pe­cial­ly con­sid­er­ing these pa­tients have can­cer that has spread be­yond the blad­der.

The drug was al­ready sanc­tioned, un­der ac­cel­er­at­ed ap­proval, for use in pa­tients fol­low­ing dis­ease pro­gres­sion — an OK ce­ment­ed by tu­mor re­sponse and du­ra­tion of re­sponse back in 2017. Pfiz­er and Mer­ck KGaA have sub­mit­ted an ap­pli­ca­tion for a full OK.

With As­traZeneca’s PD-L1 Imfinzi fail­ing to prove it­self in a piv­otal study (de­spite hav­ing an ac­cel­er­at­ed ap­proval), they have few ri­vals to wor­ry about.

That’s like­ly the same mind­set that the two phar­ma gi­ants are tak­ing in­to ges­ta­tion­al tro­phoblas­tic tu­mors, a kind of ab­nor­mal growth of cells in­side a woman’s uterus. It’s a small Phase II study that en­rolled on­ly 15 pa­tients, but re­searchers were en­cour­aged by how Baven­cio kept the dis­ease from re­turn­ing in eight of them. One pa­tient even went on to have a healthy preg­nan­cy.

The pa­tients en­rolled in­to the Phase II are re­sis­tant to mono­chemother­a­py and must en­dure tox­ic chemo reg­i­mens. The main ef­fi­ca­cy mea­sure was the nor­mal­iza­tion of a hor­mone known as hu­man chori­on­ic go­nadotropin.

No oth­er im­munother­a­py has been test­ed in GTT pa­tients be­fore, sci­en­tists wrote in an ab­stract, but PD-L1 is ex­pressed in all sub­types of this can­cer.

A group of re­searchers in South Ko­rea have al­so planned a sim­i­lar study with Mer­ck’s Keytru­da, ac­cord­ing to clin­i­cal­tri­als.gov.

Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

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Jeff Albers, Blueprint CEO

Di­ag­nos­tic champ Roche buys its way in­to the RET ti­tle fight with Eli Lil­ly, pay­ing $775M in cash to Blue­print

When Roche spelled out its original $1 billion deal — $45 million of that upfront — with Blueprint to discover targeted therapies against immunokinases, the biotech partner’s RET program was still preclinical. Four years later, pralsetinib is on the cusp of potential approval and the Swiss pharma giant is putting in much more to get in on the commercial game.

Roche gains rights to co-develop and co-commercialize the drug, with sole marketing responsibility for places outside the US and China (where CStone has staked its claim).

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President Trump (left) and NIAID chief Anthony Fauci in the White House press room, April 22, 2020 (Michael Reynolds/Sipa via AP Images)

White House tries to dis­cred­it An­tho­ny Fau­ci — could he be on his way out?

For two months in late winter and early spring, Anthony Fauci and President Trump stood in uneasy co-existence at White House briefings — an unlikely truce between an infectious disease official who had helped combat AIDS and Ebola and a president who repeatedly denied the danger of a virus that would go on to kill 100,000 Americans, repeatedly rejected masks and certain social distancing efforts, and promoted a drug with little scientific basis.

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FDA re­jects Ver­ri­ca’s skin warts pitch, a month af­ter flag­ging ap­pli­ca­tion ‘de­fi­cien­cies’

Two years ago, Verrica Pharma landed a $75 million IPO on the promise they could develop the first ever-FDA approved drug for molluscum contagiosum, an infection that causes warts. Now that won’t happen, at least not this summer.

The FDA today rejected Verrica’s application to have their lead topical drug VP-102 approved for molluscum contagiosum. The news likely came as little surprise to Verrica or its investors; the biotech disclosed in late June that the FDA sent a letter saying “deficiencies” in their application precluded a discussion around labeling or post-marketing commitments.

Andrew Allen, Gritstone Oncology CEO

A neoanti­gen pi­o­neer says its tech is work­ing great. So what wrecked the share price?

Gritstone Oncology was one of the original neoantigen upstarts, raising cash and planning to disrupt the immuno-oncology field with a bold new approach to fighting cancer with a new brand of vaccines.

On Monday, the crew in charge ran out a full display of what they’ve been seeing in a Phase I study. And everything seems to be working perfectly with one big exception: It didn’t significantly shrink tumors, let alone eradicate them.

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Stéphane Bancel, Moderna CEO (Andrew Harnik/AP Images)

A top an­a­lyst turns the spot­light on Mod­er­na, fu­el­ing a fast-and-fu­ri­ous Street race over the fu­ture of mR­NA

Bioregnum Opinion Column by John Carroll

Four months ago, one of the favorite talking points on the biopharma social media wave length was whether Moderna shares $MRNA were priced right or were wildly inflated.

After all, said the naysayers, the company had never actually pushed a treatment to an approval. Did messenger RNA really work, coding cells to make a drug or a vaccine? And how about all that chatter about how ‘secretive’ they are, or were?

Now, as CEO Stéphane Bancel and the top execs push the company to the forefront of a frantic race to develop the first vaccine to fight against the reignited wildfire spread of Covid-19, all those questions have been magnified — along with the stock price.

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En­gi­neer­ing an on/off switch for CAR-T out of yeast and Juras­sic Park

Almost as soon as CAR-T emerged in the mid-2010s as a near-cure for some cancers, so did a question: How do you give this without risking killing patients?

At the time, James Patterson was wrapping an MD-PhD at a yeast lab at London’s Francis Crick Institute. Yeast may seem an unlikely place to find a fix for cancer therapy, but reading through other researchers’ solutions to CAR-T’s toxicity, Patterson wondered if a method long used by yeast biologists called auxotrophy might be useful. You genetically modify cells to make them dependent on a particular nutrient. Then you can make them live or die — proliferate or deplete — by giving or taking away that nutrient.

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Janet Woodcock, CDER chief (AP Images)

More Warp Speed con­tracts com­ing, vac­cine pro­duc­tion to be­gin in 4-6 weeks — of­fi­cials

Operation Warp Speed has already handed out 4 of what they once said would be 3-5 major contracts to develop Covid-19 vaccines, but administration officials indicated Monday that more would be on their way.

“The slate is not closed,”  a senior HHS official said on a call with reporters. “We’ve invested in four … but the slate is not closed.”

At the same time, the official indicated that Warp Speed would continue to focus on three technologies: mRNA, viral vectors and protein subunits. That leaves the door open for a wide range of platforms, notably including both of Merck’s vaccine candidates — one of which has already received BARDA funding — and one of Sanofi’s candidates. It appears to preclude, though, the potential for Inovio and Vaxart, among certain other small developers that have hyped their ties to the Trump administration, to be included.

Stéphane Bancel, Moderna CEO (Andrew Harnik/AP Images)

Covid-19 roundup: Philip Mor­ris-backed Med­ica­go be­gins dos­ing to­bac­co-based vac­cine; Af­ter re­ports of de­lays, gov­ern­ment dis­putes, Mod­er­na sets PhI­II launch

One of the dark horse candidates in the race to develop a new vaccine to fight Covid-19 has begun a Phase I trial of a new jab developed with tobacco leaves.

Philip Morris-backed Medicago, which has deals in place to use an adjuvant from GSK and another from Dynavax in their vaccine, dosed their first patients in Phase I on Monday. And a Phase II/III trial is slated to begin in October.

That’s around the same time that the leaders in this race plan to start seeking emergency use approvals for the first round of vaccines.

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