CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Fi­nal­ly freed from the re­straints of a par­tial FDA clin­i­cal hold on its lead HER2-pos­i­tive sol­id tu­mor can­di­date, Pieris Phar­ma­ceu­ti­cals is now rac­ing to­ward Phase II.

The FDA slapped a par­tial hold on Pieris’ PRS-343 back in Ju­ly, re­strict­ing the biotech from en­rolling new pa­tients in a Phase I tri­al. While Pieris was al­lowed to con­tin­ue dos­ing pa­tients who were al­ready en­rolled, the agency re­quest­ed they con­duct an ad­di­tion­al “in-use and com­pat­i­bil­i­ty study” be­fore re­cruit­ing any more.

PRS-343 con­sists of a 4-1BB-tar­get­ing An­ti­calin pro­tein and a HER2-tar­get­ing an­ti­body. It’s a frozen for­mu­la­tion that’s re­moved from a freez­er and pre­pared for IV in­fu­sion at the tri­al site, CEO Stephen Yo­der said.

“There was a re­quest from FDA to test the mol­e­cule, the drug prod­uct, un­der var­i­ous con­di­tions in­clud­ing stress con­di­tions to show that it has a very ro­bust sta­bil­i­ty pro­file, giv­en the vari­abil­i­ty that can hap­pen in the dif­fer­ent clin­i­cal sites,” Yo­der told End­points. 

At the time, the biotech had en­rolled pa­tients in the two high­est-dose co­horts — 8 mg/kg and 18 mg/kg — and in com­bi­na­tion stud­ies with Tecen­triq un­der a drug sup­ply agree­ment with Roche.

At vir­tu­al ES­MO 2020, Pieris said the drug showed a “durable clin­i­cal ben­e­fit in the ac­tive dose co­horts, in­clud­ing a con­firmed com­plete re­sponse, in heav­i­ly pre-treat­ed pa­tients across mul­ti­ple HER2-pos­i­tive tu­mor types” in Phase I monother­a­py and Tecen­triq com­bi­na­tion stud­ies.

As of a Ju­ly 27 cut­off date, re­searchers not­ed a con­firmed com­plete re­sponse at the 18mg/kg dose lev­el and three par­tial re­spons­es in the 8 mg/kg lev­el (out of 33 evalu­able pa­tients), ac­cord­ing to the ES­MO da­ta. And in the ate­zolizum­ab com­bi­na­tion tri­al, four out of 29 evalu­able pa­tients achieved a con­firmed par­tial re­sponse at ac­tive dose lev­els.

“We’re sat­is­fied that we have da­ta for our rec­om­mend­ed Phase II dose and we’re now re­al­ly fo­cused on mo­bi­liz­ing for Phase II ini­ti­a­tion,” Yo­der said on Wednes­day, adding that the study is ex­pect­ed to launch this year.

Pieris $PIRS has worked for years on en­gi­neer­ing pro­teins that are lighter and more ver­sa­tile than an­ti­bod­ies, so that they can work where an­ti­bod­ies find their en­try barred. On Wednes­day, their stock was up 1.5%, pric­ing in at $2.71 per share.

“What sets our ap­proach apart from even oth­er im­muno-on­col­o­gy agents such as the check­point in­hibitors is that we are ag­o­niz­ing po­tent­ly in the tu­mor mi­croen­vi­ron­ment tu­mor-spe­cif­ic T cells by tar­get­ing 4-1BB,” Yo­der said. “What that does is it im­proves the meta­bol­ic fit­ness of T cells, and it caus­es al­so their pro­lif­er­a­tion.”

“You don’t just re­ac­ti­vate T cells, but you ex­pand them, and you can po­ten­tial­ly give them a lot more longevi­ty and more dura­bil­i­ty,” he added.

De­spite the par­tial clin­i­cal hold, Eli Lil­ly struck a col­lab­o­ra­tion deal with Pieris in Au­gust to fur­ther ad­vance PRS-343 stud­ies in com­bi­na­tion with its Cyra­mza and chemother­a­py pa­cli­tax­el for the sec­ond-line treat­ment of pa­tients with HER2-pos­i­tive gas­tric can­cer. Lil­ly agreed to sup­ply the ad­di­tion­al com­pounds and col­lab­o­rate on da­ta while Pieris pre­pared to ini­ti­ate Phase II.

That isn’t Pieris’ first Big Phar­ma pact. Back in 2017, As­traZeneca com­mit­ted up to $57.5 mil­lion to seed an R&D pact with Pieris, which was charged with tak­ing PRS-060 — an An­ti­calin against in­ter­leukin-4 re­cep­tor al­pha — in­to a Phase I asth­ma tri­al. And be­fore that, it inked a deal to work with Servi­er for PRS-332 and four more im­muno-on­col­o­gy pro­grams.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

For­mal­i­ties com­plete, the FDA pro­vides a snap emer­gency ap­proval for J&J's Covid-19 vac­cine as the sup­ply chain ramps up

After providing for a brief mime of regulatory judiciousness, the FDA stamped their approval on J&J’s Covid-19 vaccine Saturday, ushering in the 3rd jab aimed at ending the pandemic quickly — at least in the US.

The EUA was widely flagged in the nation’s capitol, following the unanimous vote favoring the vaccine by agency experts on Friday as well as an in-house FDA review that left little to the imagination when it came to the predicted outcome.

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Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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The fu­ture of mR­NA, J&J's vac­cine ad­comm, Mer­ck­'s $1.85B au­toim­mune bet and more

Welcome to the third installment of 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.

If this report was helpful in recapping it all for you, please do share it with your colleagues.

Get ready for FDA’s third Covid-19 vaccine

On the heels of a ringing endorsement from FDA reviewers earlier in the week, J&J‘s single-dose vaccine — which proved 66% effective at preventing symptomatic Covid-19, and 85% effective at stopping severe disease 28 days after administration — the advisory committee convened by the agency voted unanimously to recommend its emergency use authorization. It was “a relatively easy call,” according to one of the committee members — although that doesn’t mean they didn’t have questions. Jason Mast has the highlights from the discussion, including new information from the company, on this live blog.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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FDA re­view­ers en­dorse J&J sin­gle-dose shot, lay­ing path for 3rd US vac­cine

J&J’s single-dose vaccine may not have produced quite the stellar numbers of the mRNA shots, but it still won a ringing endorsement from FDA reviewers, who argued in briefing documents that it could provide robust protection against the still-raging virus.

The FDA confirmed that across just under 40,000 volunteers, the vaccine proved 66% effective at preventing symptomatic Covid-19, including 72% effective in the United States. Although that’s short of the 95% figures put up by Moderna and Pfizer-BioNTech, the shot was still 85% effective at stopping severe disease 28 days after administration. There were seven deaths in the placebo group — zero in the vaccine group.

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With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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