Roche shares surge as a dou­ble-shot win in I/O and he­mo­phil­ia rais­es block­buster ex­pec­ta­tions

Roche has scored a win with one of the key im­muno/on­col­o­gy tri­als of the year, post­ing sig­nif­i­cant top-line gains for pro­gres­sion-free sur­vival and risk of death for a seg­ment of lung can­cer pa­tients get­ting first-line treat­ment with a triple com­bo of its check­point ther­a­py Tecen­triq, Avastin and chemo. And it swept a dou­ble head­er ear­ly this morn­ing with their re­port of an­oth­er suc­cess­ful late-stage study for its new­ly ap­proved he­mo­phil­ia A drug Hem­li­bra, fur­ther set­ting the stage for a painful show­down with mar­ket leader Shire.

The back-to-back suc­cess­es drove up Roche’s shares $RHH­BY by more than 6% this morn­ing, adding $12 bil­lion in mar­ket cap as in­vestors ral­lied to ev­i­dence that the phar­ma gi­ant could suc­cess­ful­ly put the patents to a trio of fran­chise drugs in the rear view mir­ror while join­ing Mer­ck in the front ranks of lung can­cer con­tenders. Mer­ck shares dipped 2%.

First, Tecen­triq.

San­dra Horn­ing

Some an­a­lysts had al­ready raised doubts that Roche could field a triple with Avastin and chemo in the all-im­por­tant mar­ket for first-line treat­ment of peo­ple with ad­vanced non-squa­mous non-small cell lung can­cer. Avastin could look too tox­ic and Mer­ck’s Keytru­da/chemo com­bo looked like the right ap­proach for now.

But IM­pow­er 150 suc­ceed­ed where As­traZeneca’s com­bi­na­tion of Imfinzi (dur­val­um­ab) and the CT­LA 4 im­munother­a­py treme­li­mum­ab had flunked out — at least in the first shot at PFS. Roche’s top-line re­port notes that it still has to cap­ture a ma­ture snap­shot of over­all sur­vival da­ta in the first half of next year.

In the mean­time the lead­ers in I/O, in­clud­ing Mer­ck $MRK, now face a new ri­val in one of the biggest can­cer mar­kets they’re scrap­ping over in a fren­zied race for mar­ket su­prema­cy. That will like­ly have se­ri­ous con­se­quences for Mer­ck, which worked hard to wres­tle the lead from Bris­tol-My­ers Squibb $BMY — an­oth­er play­er still in con­tention. And As­traZeneca $AZN has made lung can­cer a top pri­or­i­ty for its own I/O fran­chise.

The win by Roche al­so bol­sters the ar­gu­ment in fa­vor of adding chemo to the drug reg­i­men, with an on­go­ing de­bate over the ad­van­tages and dis­ad­van­tages of in­clud­ing chemo or an­oth­er im­munother­a­py.

“Many ex­perts in the field will be re­lieved be­cause there has been un­cer­tain­ty … I think this will re­al­ly en­cour­age many of us to use this com­bi­na­tion up­front,” on­col­o­gist Ste­fan Zim­mer­mann told Reuters.

Roche chief med­ical of­fi­cer San­dra Horn­ing says that the com­pa­ny is now test­ing the triple in a range of can­cer types. And the full da­ta set on the triple will ar­rive at a con­fer­ence in De­cem­ber.

Now Hem­li­bra.

John­ny Mahlangu

Roche just won an ac­cel­er­at­ed ap­proval of this drug for he­mo­phil­ia A on Fri­day, with da­ta from HAVEN 1 and 2. In HAVEN 3, Roche re­peat­ed its ear­li­er suc­cess and added a beat, demon­strat­ing su­pe­ri­or­i­ty to fac­tor VI­II pro­phy­lax­is. And it did it with­out trig­ger­ing any new cas­es of throm­bot­ic events that dogged its ear­li­er tri­als, forc­ing reg­u­la­tors to add a black box warn­ing on the la­bel.

“It is well es­tab­lished that pro­phy­lax­is is the pre­ferred ap­proach for treat­ment of he­mo­phil­ia A, but this can re­quire fre­quent in­tra­venous in­fu­sions, and some pa­tients on pro­phy­lax­is can still ex­pe­ri­ence bleeds, while oth­ers pre­fer on-de­mand treat­ment,” said John­ny Mahlangu, who’s on the fac­ul­ty of health sci­ences at the Uni­ver­si­ty of the Wit­wa­ter­srand and NHLS in Jo­han­nes­burg, South Africa. “Giv­en its po­ten­tial to be dosed through sub­cu­ta­neous in­jec­tion on­ly once week­ly or every oth­er week, Hem­li­bra may pro­vide a fur­ther ef­fec­tive pro­phy­lac­tic treat­ment op­tion for more peo­ple with he­mo­phil­ia A and help al­le­vi­ate some of the ad­min­is­tra­tion bur­den as­so­ci­at­ed with cur­rent treat­ment.”

The suc­cess­es weighed on he­mo­phil­ia play­er Shire, which is fac­ing a more pow­er­ful con­tender for its mar­ket share in he­mo­phil­ia, and As­traZeneca. Both ri­vals saw their shares slip against the Roche spike this morn­ing.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMedO

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.

Covid-19 roundup: CDC de­bat­ing who should get first avail­able vac­cines; EU in Gilead talks af­ter US gob­bled first remde­sivir dos­es

The federal government has now spent billions of dollars accelerating the development of a Covid-19 vaccine, and yet they’ve remained hush-hush on who, precisely, would actually get inoculated once the first doses are approved and available. Internally, though, they have been debating it.

The CDC and an advisory committee of outside health experts have been working since April to devise a ranking system that would determine who receives a vaccine and when, The New York Times reported. The question of who is first in line for inoculation is important because no matter how many doses developers can make or how quickly they can make them, doses will still come out in batches; 300 million inoculations will not appear overnight.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Francis Collins, NIH director (Sean Zanni/Patrick McMullan via Getty Images)

NIH kicks off Covid-19 vac­cine, mon­o­clon­al an­ti­body re­search net­work

The NIH today announced the kickoff of a national clinical trials network to test vaccines and other treatments that could prevent infection with SARS-CoV-2, the virus that causes infection with Covid-19.

The network has been established by the National Institute of Allergy and Infectious Diseases (NIAID) through the merger of four previously existing clinical trials networks that focused on HIV/AIDS.

FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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