Ron De­Pin­ho rais­es seed cash for MD An­der­son col­league's work on a 'Holy Grail mol­e­cule' in can­cer, in­flam­ma­tion

Ron De­Pin­ho has blazed a wide trail in can­cer re­search. As found­ing di­rec­tor of the Belfer In­sti­tute for Ap­plied Can­cer Sci­ence at Dana-Far­ber and for­mer pres­i­dent of MD An­der­son Can­cer Cen­ter in Hous­ton — where he de­part­ed af­ter a rocky tenure which he now fierce­ly de­fends — De­Pin­ho spear­head­ed trans­la­tion­al pro­grams and man­aged, among oth­er things, to build a clin­i­cal tri­al en­gine that proved piv­otal in a num­ber of FDA ap­provals.

That’s why when he learned about his col­league David Tweardy’s work on STAT3 — a pro­tein tied to a wide range of dis­eases — the se­r­i­al en­tre­pre­neur went all in to co-found Tvar­di Ther­a­peu­tics.

“It’s been one of these Holy Grail mol­e­cules that folks have tried to de­vel­op in­hibitors for,” he tells me.

David Tweardy

Us­ing a screen he de­signed 10 years ago, Tweardy, head of in­ter­nal med­i­cine at MD An­der­son, had found a com­pound that pre­vents STAT3 from be­com­ing phos­pho­ry­lat­ed by the re­cep­tors it typ­i­cal­ly in­ter­acts with, mean­ing it can­not then go on to turn on genes that are im­por­tant in can­cer, fi­bro­sis and in­flam­ma­tion. Giv­en that hy­per­ac­ti­vat­ed STAT3 is present in more than 50% of can­cers — of­ten turn­ing up im­muno­sup­pres­sion in the tu­mor mi­croen­vi­ron­ment — he the­o­rized that a po­tent in­hibitor here that doesn’t trig­ger safe­ty is­sues could have a big im­pact.

That seems to be the case through­out the pre­clin­i­cal test­ing done in his lab, which led Tweardy and De­Pin­ho to the Phase I that Tvar­di is cur­rent­ly con­duct­ing. Now, with a fresh $9 mil­lion in Se­ries A fund­ing, the biotech ex­pects to wrap up dose and safe­ty stud­ies in 3 to 4 can­cer types for its lead oral drug, ex­plore an IV for­mu­la­tion for use in non-can­cer in­di­ca­tions, and hus­tle a sec­ond pro­gram to the door of the clin­ic.

Some­where in that time­line are tri­als in­volv­ing check­point in­hibitors, as Tvar­di be­lieves TTI-101 could be used in com­bo or as a fol­low-up to PD-1 agents.

And they hope to do all that by the end of 2019, with the help of a per­ma­nent CEO they are still search­ing for.

It’s not a ton of mon­ey in to­day’s mega-round stud­ded world, but De­Pin­ho says stay­ing flex­i­ble and cap­i­tal ef­fi­cient would be a key strat­e­gy for the com­pa­ny’s ear­ly days — a strat­e­gy that al­so in­volves seek­ing non-di­lu­tive grant fund­ing. Most of the in­vestors in this round will re­main un­named, but he vol­un­teered Mon­i­ca Beam at Alexan­dria as an ex­am­ple of the “very so­phis­ti­cat­ed but pri­vate in­vestors” he chose to work with.

“We don’t have a large bu­reau­crat­ic or­ga­ni­za­tion, so we’re able to make de­ci­sions and be very flex­i­ble which is crit­i­cal­ly im­por­tant here, be­cause STAT3 has so many dif­fer­ent op­por­tu­ni­ties […] that one needs to have a cer­tain lev­el of fo­cus and pri­or­i­ti­za­tion,” he says.

At around a dozen em­ploy­ees, the small com­pa­ny is rem­i­nis­cent of Karyopharm, an­oth­er on­col­o­gy com­pa­ny De­Pin­ho helped start be­fore join­ing MD An­der­son — where he is still fac­ul­ty af­ter step­ping down from a six-year tenure as pres­i­dent. Dur­ing that time, the cen­ter ex­pe­ri­enced re­port­ed op­er­at­ing loss­es of more than $460 mil­lion over 16 months and hun­dreds of staffers were laid off.

De­Pin­ho, how­ev­er, sees it dif­fer­ent­ly.

“It was an ex­tra­or­di­nar­i­ly suc­cess­ful fi­nan­cial sit­u­a­tion,” he tells me, cit­ing “$3.7 bil­lion in mar­gin, the health­i­est fi­nan­cial bal­ance sheet in the Unit­ed States for an aca­d­e­m­ic med­ical cen­ter, record phil­an­thropy and ranked No. 1 in cor­po­rate al­liance rev­enues and IP rev­enues.”

He’s now moved on to new en­deav­ors, in­clud­ing Tvar­di. Once the Phase I da­ta are in, De­Pin­ho ex­pects to ramp up busi­ness de­vel­op­ment talks. He’s al­ready think­ing about the next round, which “would like­ly be our last one en route to ei­ther an IPO or oth­er event.”


Im­age: Ron De­Pin­ho. TVAR­DI

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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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).

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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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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