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.

Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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John McHutchison in 2012. Getty Images

The $1.1M good­bye: Gilead CSO John McHutchi­son is out as Daniel O’Day shakes up the se­nior team

Just a little more than a year after John McHutchison grabbed a promotion to become CSO at Gilead in the wake of Norbert Bischofberger’s exit, he’s out amid a shakeup of the senior team that is also triggering the departure of two other top execs.

Gilead stated that McHutchison “has decided to step down” from the job as of August 2nd. And their SEC filing notes that he’ll be getting a $1.1 million check to settle up on his contract.

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Thomas Gajewski, David Steinberg. (CRI, Pyxis)

Bay­er, Long­wood back star re­searcher's deep dive in­to the tu­mor mi­croen­vi­ron­ment for new I/O tar­gets

From PD-1 targeting to the RAS pathway to the STING complex, Thomas Gajewski has spent the past two decades of his career decoding the various ways the immune system can be unleashed to defend against cancer. So when the University of Chicago professor comes around to putting all his findings into a new platform for finding new targets, VCs and pharma groups alike pay attention.

“He’s been studying T cells for 20 years, plus he’s one of the world’s leaders if not the world leader in the space,” David Steinberg, partner at Longwood Fund, said. “Furthermore, let me add he did a lot of the foundational research and also some of the seminal clinical trials in the existing set of I/O agents. He understands the space really well, he understands the current strengths, and I think he understood really well what was missing, so he knew where to look.”

Kamala Harris speaking yesterday at the Des Moines Register Iowa Presidential Candidate Forum [via Getty]

Who’s the tough­est on drug prices? A game of po­lit­i­cal one-up­man­ship is dri­ving the pol­i­cy de­bate in Wash­ing­ton

Earlier this week we got a look at Senator Kamala Harris’ position on drug prices. She’s proposing that HHS take an average price from single-payer systems like the UK, Germany and Canada — which leverage market access for lower prices — and use that to set the US price. Anything drug companies collect above that would be taxed at a rate of 100%.

And the rhetoric is scathing:
While families struggle to make it to the end of the month, pharmaceutical companies are turning record profits. They’re spending nearly as much on advertising as R&D. They’re manipulating their market power to hike prices on lifesaving generic drugs. They’re making twice the profit of the average industry in America and still increased drug prices by 10.5% over the past six months alone. Meanwhile, they are charging dramatically higher prices to American consumers.
That’s an escalation on Joe Biden’s plan, which includes drug importation from those cheaper markets as well as allowing Medicare to negotiate prices — something that virtually all Dems agree on now.

SJ Lee [File photo]

Go­ing in­side cells, Sung Joo Lee has sketched some big goals for his small — but glob­al — team of drug hunters

For a small biotech based in South Korea with a research arm in Cambridge, MA, Orum Therapeutics has sketched out some big goals aimed at developing antibodies for intracellular targets. And now they have a new $30 million round to push the work forward, aiming at a slate of currently undruggable quests.

Orum has been working on a platform tech out of Ajou University that relies on endocytosis to smuggle antibodies and their cargo inside a cell. They’ve published work in Nature that illustrates its preclinical potential in RAS mutations, and KRAS is on their list of targets. 

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Astel­las buys in­to Fre­quen­cy's re­gen­er­a­tive med strat­e­gy with a $625M al­liance on hear­ing loss

The executive team at Frequency Therapeutics never oversold the results of their maiden Phase I/II study for a new drug to rectify hearing loss. It was, they said back in April, primarily about safety and tolerability, where their drug FX-322 performed as they had hoped. 

That early glimpse of efficacy everyone searches for in their first try on humans? 

(I)mprovements in hearing function, including audiometry and word scores, were observed in multiple FX-322 treated patients.

We don’t know exactly what that means. But whatever the details, Astellas found enough in the data to jump in with a sizable collaboration deal.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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