Bil­lion­aire-backed Park­er In­sti­tute tack­les the Holy Grail of can­cer R&D — with a vir­tu­al un­known

Up un­til about 6 months ago, Fred Rams­dell, the vice pres­i­dent for re­search at the Park­er In­sti­tute for Can­cer Im­munother­a­py, had nev­er heard of Tes­sa Ther­a­peu­tics. But he and some of the top can­cer re­searchers as­so­ci­at­ed with the Park­er In­sti­tute’s far flung net­work of in­ves­ti­ga­tors have been mak­ing up for lost time.

Fred Rams­dell

The Park­er In­sti­tute — fund­ed by tech mogul Sean Park­er — is an­nounc­ing Mon­day that it is mak­ing a leap in­to next-gen adop­tive cell ther­a­pies for can­cer, join­ing with Sin­ga­pore-based Tes­sa. Their new biotech al­ly is now en­gaged in a Phase III tri­al that hopes to open a new chap­ter in the field, look­ing to share the spot­light with the lead­ers which have dom­i­nat­ed the im­munother­a­py are­na so far. And the part­ners have some big plans for the fu­ture.

“Ob­vi­ous­ly there’s a lot of press and clin­i­cal da­ta and ex­cite­ment around CAR-T,” says Rams­dell, the vice pres­i­dent for re­search at the Park­er In­sti­tute. “That’s great and won­der­ful. What Tes­sa is do­ing is a lot dif­fer­ent.”

In­stead of ex­tract­ing T cells and reengi­neer­ing them to hunt down and kill can­cer cells, a CAR-T ap­proach that re­quires ag­gres­sive man­age­ment of some se­vere re­ac­tions but with some re­mark­able re­sults for liq­uid can­cers, Tes­sa takes blood and ex­pands and adapts virus-spe­cif­ic T cells to tar­get vi­ral­ly-as­so­ci­at­ed tu­mors like cer­vi­cal and head and neck can­cer, swarm­ing the cells.

“As much as its tech­no­log­i­cal as­pect, it’s al­so a philo­soph­i­cal align­ment,” Rams­dell adds about the al­liance. “They have a very good sci­en­tif­ic un­der­pin­ning. They know what they’re do­ing; very well round­ed….You nev­er know, but it looks re­al­ly quite good.”

In this new col­lab­o­ra­tion, star im­munother­a­py spe­cial­ists like Phil Green­berg at the Fred Hutch and Crys­tal Mack­all of Stan­ford are be­ing brought in to work with Tes­sa’s re­search team, head­ed by Chief Sci­en­tif­ic Of­fi­cer John Con­nol­ly and CMO Han Chong Toh, the deputy di­rec­tor of the Na­tion­al Can­cer Cen­tre Sin­ga­pore and one of the top can­cer in­ves­ti­ga­tors in the re­gion. Oth­er in­ves­ti­ga­tors in the Park­er net­work can al­so pitch ideas on ad­vanc­ing the work, pos­si­bly even get­ting fund­ing for small proof-of-con­cept stud­ies to test their no­tions.

“In any col­lab­o­ra­tion,” says Rams­dell, “we can be the hub of that wheel and bring the dif­fer­ent ap­proach­es to­geth­er.”

That fits in per­fect­ly with Tes­sa’s strat­e­gy, Con­nol­ly tells me. The vet­er­an Bay­lor Col­lege of Med­i­cine in­ves­ti­ga­tor ran in­to Han Chong Toh — who had been at the Cen­ter for Cell and Gene Ther­a­py at Bay­lor Col­lege of Med­i­cine work­ing un­der found­ing di­rec­tor Mal­colm Bren­ner — af­ter he moved to Sin­ga­pore in 2010 to set up a lab.

John Con­nol­ly

Tes­sa’s Phase III will read out in 2018, says Con­nol­ly, giv­ing them a piv­otal chance to demon­strate how they can trans­form B cells with the Ep­stein-Barr virus, mov­ing be­yond ran­dom ac­ti­va­tion of T cells with chimeric anti­gen re­cep­tors to a po­ten­tial­ly much more tar­get­ed cell ther­a­py de­signed to con­tin­u­al­ly hunt down and kill spe­cif­ic can­cer cells. The HPV pro­gram can do the same for head and neck and oth­er can­cers. And when you start to mar­ry this ap­proach with oth­er im­munother­a­pies, you can start to vi­su­al­ize Tes­sa’s goal: elim­i­nat­ing sol­id tu­mors, the Holy Grail of cell ther­a­py.

Con­nol­ly and Han Chong Toh are di­rect­ing the late-stage pro­gram, sanc­tioned with the FDA’s fast track and or­phan drug des­ig­na­tions, which is be­ing con­duct­ed in 5 coun­tries with 29 sites.

Rams­dell was at­tract­ed by an ap­proach that should avoid off­site tox­i­c­i­ty. And with the nat­ur­al sig­nal­ing in­volved, he al­so be­lieves you’re more like­ly to cre­ate mem­o­ry cells that can keep up the at­tack — promis­ing a durable re­sponse.

“Tes­sa (with a base of IP in-li­censed from Bay­lor Col­lege of Med­i­cine) adds their own se­cret sauce of cy­tokines that ac­ti­vate in a par­tic­u­lar way for good killing and dura­bil­i­ty,” notes Rams­dell, help­ing avoid the risk of burn­ing the cells out, which hap­pens when you mul­ti­ply their num­bers.

The ear­ly re­sults “showed beau­ti­ful sur­vival da­ta,” says Con­nol­ly, which helped at­tract a line­up of fam­i­ly of­fices and high net worth in­vestors to back a biotech that now has a staff of about 130.

Tes­sa turned up at AS­CO in ear­ly June with Phase II da­ta on 35 pa­tients with rare cas­es of Stage 4c na­sopha­ryn­geal car­ci­no­ma, or NPC, as­so­ci­at­ed with Ep­stein-Barr virus. Their con­clu­sion:

The 2- and 3-year over­all sur­vival rates were 62.9% and 37.1% re­spec­tive­ly, which rep­re­sent the best re­port­ed sur­vival out­come for first-line treat­ment of ad­vanced NPC when com­pared to his­tor­i­cal clin­i­cal tri­als.

Tes­sa added that it has bio­mark­ers to iden­ti­fy which pa­tients are like­ly to ben­e­fit, the kind of strat­e­gy that the FDA prefers.

‘We’re very in­ter­est­ed in mov­ing CAR-T to next-gen as well, putting pay­loads in­to CAR-T, etcetera, etcetera,” says Rams­dell. “The par­al­lel here is that as you move in­to sol­id tu­mors, Tes­sa is more ad­vanced tech­ni­cal­ly, con­cep­tu­al­ly and philo­soph­i­cal­ly.”

Just a few months ago Tes­sa launched a col­lab­o­ra­tion with Rochester, MN-based Vyr­i­ad, which has clin­i­cal-stage on­colyt­ic virus­es that in­fect and de­stroy can­cer cells, but al­so use anti­gens to flag an im­mune sys­tem at­tack (one of a group of next-gen on­colyt­ics com­pa­nies look­ing to do T-Vec bet­ter. Tes­sa sees it as a match of syn­er­gis­tic tech­nolo­gies for fight­ing can­cer.

In March Tes­sa al­so bought out an­oth­er Sin­ga­pore start­up, Eu­chloe, which has been work­ing on a slate of an­ti­bod­ies, in­clud­ing PD-1, as well as chimeric anti­gen re­cep­tor tech­nolo­gies for next-gen CARs.

Tes­sa may just be still in the ear­ly stages of its de­but on the glob­al biotech scene. But with Asian biotechs tak­ing a more cen­tral role in drug de­vel­op­ment, it could al­ready be on the verge of do­ing some­thing big.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.