Paul Hastings (Nkarta)

Nkar­ta signs its name to an am­bi­tious fu­ture with man­u­fac­tur­ing hub for not one but mul­ti­ple mar­ket­ed drugs

Rid­ing sci­ence out of Dario Cam­pana’s famed lab in Sin­ga­pore, West Coast biotech Nkar­ta has been a high-pro­file en­try in­to the race for the in­dus­try’s first nat­ur­al killer cell-based ther­a­py. The biotech, miles away from a po­ten­tial ap­proval, is dou­bling down on its own am­bi­tion and set­ting up a man­u­fac­tur­ing fa­cil­i­ty of the fu­ture far in ad­vance.

Nkar­ta has signed a lease on an 88,000-square-foot fa­cil­i­ty in South San Fran­cis­co that will act as the biotech’s new head­quar­ters as well as the home of its com­mer­cial man­u­fac­tur­ing op­er­a­tions, the com­pa­ny said Wednes­day.

Nkar­ta, which is de­vel­op­ing off-the-shelf NK cell im­munother­a­pies for can­cer, cur­rent­ly churns sup­ply out of a small, 2,700-square-foot clin­i­cal man­u­fac­tur­ing fa­cil­i­ty that was com­plet­ed ear­li­er this year at its ex­ist­ing site in South San Fran­cis­co. But as the biotech en­rolls a Phase I clin­i­cal test for NK cell can­di­date NKX101, which tar­gets NKG2d lig­ands on tu­mor cells, and ap­proach­es the clin­ic with an­oth­er, Nkar­ta re­al­ized its hum­ble sur­round­ings didn’t match its am­bi­tions as a mul­tidrug phar­ma.

Now, the biotech’s new site, which is ex­pect­ed to go on­line in late 2023, will be the in­cu­ba­tor for Nkar­ta’s goal of bring­ing a slate of drugs over the fin­ish line in what would be a sig­nif­i­cant achieve­ment giv­en there are no ap­proved NK cell ther­a­pies.

All of Nkar­ta’s can­di­dates, which are en­gi­neered to ex­press a chimeric anti­gen re­cep­tor (CAR) and a mem­brane-bound IL-15 cy­tokine to spur the NK cells’ growth and ac­tiv­i­ty, use cells de­rived from healthy donors and can po­ten­tial­ly be pro­duced at a frac­tion of the cost at com­mer­cial scale of oth­er cell ther­a­pies, the com­pa­ny said. Nkar­ta at­trib­uted the cost gap to its pro­pri­etary man­u­fac­tur­ing plat­form, which can the­o­ret­i­cal­ly pro­duce hun­dreds of in­di­vid­ual dos­es off of a sin­gle run, as well as op­ti­mized freez­er stor­age for off-the-shelf use.

“Our goal is to en­sure that cost-ef­fec­tive, com­mer­cial-scale pro­duc­tion of cell ther­a­pies can be made avail­able wide­ly and rapid­ly to the can­cer pa­tients who need them, and we ex­pect this new fa­cil­i­ty will en­able us to do just that,” CEO Paul Hast­ings said in a state­ment.

In ad­di­tion to NKX101, which is cur­rent­ly en­rolling a Phase I tri­al against AML, Nkar­ta is prepar­ing the way for NKX019, which tar­gets CD19, to hit the clin­ic lat­er this year. Mean­while, the com­pa­ny thinks its newest fa­cil­i­ty could pro­vide enough el­bow room to ad­vance the pipeline in­to ex­cit­ing new ar­eas, par­tic­u­lar­ly a CRISPR/Cas9 ap­pli­ca­tion for its NK cell tech, the com­pa­ny said in a state­ment.

Samarth Kulka­rni

The path there has al­ready been paved af­ter Nkar­ta signed a dis­cov­ery deal in May worth an undis­closed amount of cash up­front to part­ner with CRISPR Ther­a­peu­tics, the biotech us­ing tech pi­o­neered in part by gene edit­ing maven Em­manuelle Char­p­en­tier. The col­lab­o­ra­tion will look to co-de­vel­op and com­mer­cial­ize two CAR-NK ther­a­pies, in ad­di­tion to an NK+T pro­gram. The duo will split all R&D costs — and any world­wide prof­its — 50/50, Hast­ings pre­vi­ous­ly told End­points News.

There’s some over­lap in terms of what both biotechs are try­ing to do. In Oc­to­ber, CRISPR an­nounced two of four pa­tients giv­en a spe­cif­ic dose of its CAR-T can­di­date for CD19+ B cell ma­lig­nan­cies achieved com­plete re­spons­es (the re­sults, how­ev­er, were marred by the death of a pa­tient in a high­er dose co­hort).

“While we are bull­ish about our al­lo­gene­ic CAR-T ap­proach, we be­lieve that NK cells can be an im­por­tant part of fu­ture on­col­o­gy ther­a­pies,” CRISPR CEO Samarth Kulka­rni said in an email.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Am­gen adds new NC plant to the list as part of $1B man­u­fac­tur­ing ex­pan­sion plans state­side

What can $1 billion buy? If you’re Amgen, it’s good for two manufacturing facilities in the US.

The California-based drug giant will invest close to $550 million in a drug substance plant in Holly Springs, NC, adding itself to an ever-growing list of biotech companies that have decided to call North Carolina home, and marking its second drug manufacturing announcement in a little more than a month.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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