Nek­tar takes the spot­light at SITC tout­ing some promis­ing ear­ly da­ta for hard-to-treat can­cers

WASH­ING­TON, DC — One of Nek­tar Ther­a­peu­tics’ top can­cer drugs gained the spot­light at the So­ci­ety for Im­munother­a­py of Can­cer meet­ing over the week­end with the first cut of some promis­ing re­sponse rates in a range of tu­mor types. And the pub­lic biotech $NK­TR be­lieves the re­sults point them to a short path at the FDA that could lead to the drug’s first, fast ap­proval.

Mary Tagli­a­fer­ri

The drug is NK­TR-214, an IL-2 im­muno-on­col­o­gy ther­a­py — built from the ground up by the biotech — which is de­signed to bind to the CD122 re­cep­tor on the sur­face of CD-8 and CD-4 pos­i­tive im­mune cells to whip up an at­tack on var­i­ous can­cers. Nek­tar struck a 50/50 deal with Bris­tol-My­ers Squibb $BMY to use their drug in com­bi­na­tion with Op­di­vo (nivolum­ab), match­ing a ther­a­py aimed at dri­ving an im­mune re­sponse with a pop­u­lar check­point block­buster that helps take the brakes off the as­sault.

At SITC, the com­pa­ny pro­duced a med­ley of da­ta points, in­clud­ing:

  • In treat­ment-naïve first-line pa­tients with stage IV melanoma, re­searchers tracked re­spons­es in 7 of 11 pa­tients (63%), with 2 com­plete re­spons­es — no vis­i­ble signs of the dis­ease — and 5 par­tial re­spons­es. Not all of these num­bers are pre­cise. It’s im­por­tant to note though that one of the CRs and one of the PRs were un­con­firmed — em­pha­siz­ing just how ear­ly these re­sults are.
  • Among 13 kid­ney can­cer pa­tients with one or more base­line scans, re­spons­es were ob­served in 6 (46%), with 1 com­plete re­sponse and 5 par­tials.
  • For a small group of 4 pa­tients with ad­vanced PD-L1/neg­a­tive non-small cell lung can­cer, the in­ves­ti­ga­tors tracked a re­sponse in 3 (75%), with 1 com­plete and 2 par­tials.

“Sin­gle-agent nivolum­ab is known for all these in­di­ca­tions, with a 34% re­sponse for melanoma,” Mary Tagli­a­fer­ri, se­nior vice pres­i­dent, clin­i­cal at Nek­tar, tells me. “We have a 64% re­duc­tion.”

That’s what in­vestors liked to hear. Nek­tar’s stock quick­ly jumped 23% in pre-mar­ket trad­ing on Mon­day.

Jonathan Za­levsky

“Our re­sponse rate is just over 50% even in PD-L1 neg­a­tive pa­tients,” says Jonathan Za­levsky, Nek­tar’s se­nior vice pres­i­dent of bi­ol­o­gy and pre­clin­i­cal de­vel­op­ment. “And what we like about those met­rics, it gives us a nice con­fir­ma­tion of the in­crease we’re see­ing in our com­bi­na­tion.”

This is an ear­ly snap­shot of this drug, to be sure, but Nek­tar’s team be­lieves that the safe­ty and ef­fi­ca­cy pro­files they’re see­ing in the first snap­shot of da­ta un­der­scores the biotech’s chances of go­ing af­ter an ac­cel­er­at­ed ap­proval for cer­tain pa­tient groups that face par­tic­u­lar­ly poor prospects.

“There is an op­por­tu­ni­ty for Nek­tar to pur­sue an ac­cel­er­at­ed path­way in those pa­tient pop­u­la­tions where the re­sponse rate is very, very low,” says Tagli­a­fer­ri.

They wouldn’t be the first to suc­ceed at that, ei­ther. The FDA has proven ea­ger to get new ther­a­pies to pa­tients with poor prospects, and Nek­tar has filed for “break­through” sta­tus to help en­list reg­u­la­tors’ sup­port for their plans.

The Phase II has now been ex­pand­ed from 250 to 330 pa­tients with 13 co­horts.

So what’s an ap­proval worth here?

Cowen an­a­lyst Chris Shibu­tani is one of the most op­ti­mistic an­a­lysts cov­er­ing the drug, es­ti­mat­ing its 2023 rev­enue at $950 mil­lion — but every­thing has to go right for it to get close to the block­buster mark.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it has the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s latest cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Bank­rupt an­tibi­otics mak­er Ar­a­digm turns to old part­ner/in­vestor for fi­nal $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.

DB­V's peanut pre­ven­tion patch ap­proach­es key stage of ap­proval process

Almost a year and a half after DBV Technologies pulled its peanut allergy immunotherapy patch from FDA review, the biotech will get their day in court. The FDA has scheduled an advisory committee hearing for May 15.

In the two-horse race to develop the first immunotherapy for peanut allergy, DBV had the early lead, filing an NDA for their patch in 2018. But on December 20 of that year, the company withdrew their application after, they said, meeting with regulators and determining they had not submitted “sufficient detail regarding data on manufacturing procedures and quality controls.” Aimmune filed their BLA 3 days later and won approval as the first immunotherapy for peanuts this month.

Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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An­to­nio Gual­ber­to starts post-Ku­ra ca­reer at Ei­sai sub­sidiary H3; eF­FEC­TOR co-founder Siegfried Re­ich jumps to Turn­ing Point

→ Days after Kura Oncology announced the departure of co-founder Antonio Gualberto, we finally know where he wound up. Eisai subsidiary H3 Biomedicine has recruited him as CMO to finding the right patients to its four clinical-stage small molecule assets hitting genomic drivers of cancer.

“Challenges of these and many other precision medicine approaches are on one hand technical — a need for robust and precise diagnostics — and on the other hand derived by the challenge to alter standard clinical practice in settings where patient screening, e.g. by tumor DNA sequencing, is not standard practice,” he wrote to Endpoints News on his way back to Boston from Eisai’s Tokyo offices. “Only compelling clinical activity can drive clinicians and pathologists to modify standard clinical practice.”

Gen­fit de­lays high-stakes PhI­II NASH read­out; Bris­tol-My­ers grabs first Op­di­vo OK in esophageal can­cer

→ Genfit is delaying a highly anticipated Phase III readout of its NASH drug elafibranor. It now expects to report topline interim results some time in the second quarter, after incorporating new “FDA insights” to be delivered by the end of March. The French biotech insists that the delay — which is technically a delay in unblinding — isn’t related to efficacy and safety concerns or any business matters. The stakes are high: The RESOLVE-IT trial was set up to be a crucial test of the PPAR approach in tackling the fatty liver disease and support a historic accelerated approval.