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

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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(Credit: Shutterstock)

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Alan Wise (L) and Peter Trill (Duke Street Bio)

They sold their last biotech to Mer­ck. Now they're back with a PARP out­fit named af­ter a Lon­don street

In 2016, Peter Trill and Alan Wise sold IOmet Pharma (an I/O outfit as the name suggests) to Merck for $400 million.

Now, some six years later, the duo has returned with another cancer biotech, Duke Street Bio, that emerged from stealth Tuesday. Duke Street Bio, named for the street where it’s located in London, is making its public debut as a next-gen PARP player, hoping to break into a field that already has a number of Big Pharma competitors.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”