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

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA.