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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.