Am­gen’s star PhI­II os­teo­poro­sis drug ro­mo slammed by sur­pris­ing car­dio risk, a big plus for ri­val Ra­dius drug

Am­gen’s ro­mosozum­ab, one of its top late-stage drug prospects aimed at os­teo­poro­sis, is in some se­ri­ous trou­ble.

Late Sun­day the big biotech $AMGN and its part­ner UCB an­nounced what at first glance would ap­pear to be hap­py news: the late-stage tri­al com­par­ing ro­mosozum­ab to Fos­amax hit its pri­ma­ry and key sec­ondary end­points. But as you wind through the re­lease, some big is­sues start to rise up that raise se­ri­ous ques­tions about its fu­ture — if it has one. And that could make a big dif­fer­ence for its new­ly ap­proved ri­val at Ra­dius Health $RDUS.

Big event one: There was a no­table car­dio risk im­bal­ance be­tween ro­mo and Fos­amax; 2.5% for ro­mo and 1.9% for Fos­amax.

Big event two: The FDA wants to eval­u­ate the new set of head-to-head da­ta in siz­ing up an ap­proval. And that means no de­ci­sion is ex­pect­ed this sum­mer, as had been as­sumed ear­li­er.

Am­gen shares dropped 2.3% in pre-mar­ket trad­ing, while UCB plunged 14.5%. Ra­dius shares jumped about 15% on the news.

Umer Raf­fat at Ever­core ISI spot­ted the sig­nif­i­cance quick­ly. He not­ed:

Am­gen’s Ph 3 up­date on bone drug just now is clear­ly neg­a­tive, and very sur­pris­ing.  At this point, we are tak­ing out all ro­mo sales from the mod­el.  Con­sen­sus has ~$800M peak for this drug – if you take it out (and bake in the part­ner­ship split with UCB), EPS re­vi­sion of ~$0.32/share.  We sus­pect stock like­ly down ~3-4% on this.

That all sound­ed sus­pi­cious­ly like odanacat­ib, Mer­ck’s os­teo­poro­sis drug which the phar­ma gi­ant de­cid­ed to shelve last fall af­ter wit­ness­ing an in­crease risk of stroke for the drug. And Ge­of­frey Porges at Leerink cal­cu­lat­ed the odds of Am­gen scrap­ping the drug.

We be­lieve that the prod­uct now has on­ly a 50/50 prob­a­bil­i­ty of com­ing to mar­ket at all, and these odds are sub­ject to the re­sults of dis­cus­sions with spe­cial­ists and in­ves­ti­ga­tors, as well as full dis­clo­sure of the na­ture and sever­i­ty of the car­dio­vas­cu­lar events ob­served in both stud­ies.

Just a few weeks ago Ra­dius won their first ever drug ap­proval for abaloparatide, now be­ing lined up for the os­teo­poro­sis mar­ket as Tym­los. An­a­lysts like Ge­of­frey Porges had thought Ra­dius may try to go af­ter a unique mar­ket with a com­mer­cial strat­e­gy tak­ing in­to ac­count Eli Lil­ly’s ag­ing For­teo with the ri­val ro­mosozum­ab from Am­gen and UCB be­ing steered in­to a Ju­ly 19 PDU­FA date.

But that’s at the very least sig­nif­i­cant­ly de­layed — Ra­dius CEO Bob Ward said it nev­er was a fac­tor — as far as ro­mo is con­cerned.

Last fall an­a­lysts were talk­ing more about a straight shot at a quick ap­proval for ro­mo this sum­mer — with some sig­nif­i­cant caveats. Ro­mo — which tar­gets the scle­rostin pro­tein — fol­lowed by Am­gen’s Pro­lia (deno­sum­ab) clear­ly vault­ed the bar in Phase III for re­duc­ing ver­te­bral frac­tures, with a hefty 75% risk re­duc­tion com­pared to a place­bo plus deno­sum­ab. In­ves­ti­ga­tors al­so were able to show a bet­ter safe­ty pro­file in its pre­sen­ta­tion at the an­nu­al con­fab of the Amer­i­can So­ci­ety for Bone Min­er­al Re­search. And there was an in­crease in bone min­er­al den­si­ty among the drug arm in the study, which re­cruit­ed 7,180 high-prism post­menopausal women.

But the drug missed a key sec­ondary end­point. The drug did not sig­nif­i­cant­ly im­prove pa­tients’ risk of non-ver­te­bral frac­tures, leav­ing Ra­dius Health — which has post­ed an 86% risk re­duc­tion in ver­te­bral frac­tures — with a pos­si­ble dis­tinct ad­van­tage on that score.

Am­gen and its part­ners at UCB didn’t ig­nore the sit­u­a­tion to­day, af­ter high­light­ing the pos­i­tive.

Iris Loew-Friedrich

“We are im­pressed with the sta­tis­ti­cal­ly sig­nif­i­cant su­pe­ri­or frac­ture risk re­duc­tion of EVENI­TY over al­en­dronate, a cur­rent stan­dard of care in os­teo­poro­sis. When we think that pa­tients who have had a frac­ture are high­ly like­ly to suf­fer an­oth­er one, the im­por­tance of post-frac­ture care can­not be em­pha­sized enough,” said Iris Loew-Friedrich, UCB’s chief med­ical of­fi­cer. “We are work­ing on un­der­stand­ing the ob­served car­dio­vas­cu­lar safe­ty sig­nal and will con­tin­ue to dis­cuss these re­sults with glob­al reg­u­la­tors and ex­perts in the field.”

This is a bad twist for Am­gen. The bio­phar­ma play­er has had a se­ries of is­sues, in­clud­ing a big back­lash by pay­ers tar­get­ing its PC­SK9 drug. Watch­ing an­oth­er late-stage drug go un­der a cloud like this won’t help Am­gen’s rep with an­a­lysts.

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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