CEO David Main (Notch)

'In­dus­tri­al­iz­ing the pro­duc­tion of cell­s': An­oth­er iP­SC play­er joins the quest for off-the-shelf cell ther­a­pies

Sci­en­tists have been af­ter the “Holy Grail” of cell ther­a­py — an off-the-shelf prod­uct — for years. Notch Ther­a­peu­tics is now join­ing the quest with a tech plat­form it says can in­dus­tri­al­ize the pro­duc­tion of cells. And on Wednes­day, it un­veiled a $85 mil­lion Se­ries A to get go­ing.

Notch was found­ed in 2018 by Juan Car­los Zúñi­ga-Pflück­er and Pe­ter Zand­stra, but “re­al­ly kicked off” in late 2019 when it signed a deal with Al­lo­gene to re­search al­lo­gene­ic cell ther­a­py can­di­dates, CEO David Main said. The nascent biotech got $10 mil­lion up­front, and stands to earn up to an­oth­er $294.2 mil­lion in mile­stones.

Main, who came on board in 2020, plans on us­ing Notch’s Se­ries A funds to grow two things: the staff and the pipeline. Over the next two years, Main ex­pects the 35-per­son com­pa­ny to ex­pand to about 100 em­ploy­ees, and file its first IND.

What sets Notch apart from oth­ers work­ing on al­lo­gene­ic cell ther­a­pies is what it calls its “En­gi­neered Thymic Niche” plat­form, Main said. Rather than take blood and ex­tract the im­mune cells you want to give to a pa­tient, Notch cre­ates the im­mune cells from pluripo­tent stem cells. Pluripo­tent es­sen­tial­ly means the cell can be dif­fer­en­ti­at­ed in­to any cell you want, Main said.

To do so, they’ve cre­at­ed an ar­ti­fi­cial thy­mus, which is a small or­gan near the ster­num where T cells ma­ture. A cell go­ing through the thy­mus is ex­posed to a num­ber of sig­nals that turn it in­to an im­mune cell, Main ex­plained.

The idea of us­ing in­duced pluripo­tent stem cells, or iP­SCs, to grow cells in a lab in­stead of ex­tract­ing them from donors isn’t new. John­son & John­son inked an up to $3.1 bil­lion deal with Fate Ther­a­peu­tics back in April to work on iP­SC-de­rived CAR-T and CAR-NK can­di­dates. And Ver­sant Ven­tures’ Cen­tu­ry Ther­a­peu­tics nabbed a meaty $250 mil­lion round in 2019 to ad­vance iP­SC prod­ucts for can­cer.

“Peo­ple have known in sci­ence for many, many decades, how to dif­fer­en­ti­ate stem cells and T cells, but they haven’t been able to do it out­side of the test tube in a re­search li­brary. Or if they tried to com­mer­cial­ize it that way they’re hav­ing to have thou­sands of petri dish­es to make enough cells for ther­a­py,” Main said.

With the so-called En­gi­neered Thymic Niche plat­form, Notch says it can dif­fer­en­ti­ate stem cells in a “high­ly con­trolled fash­ion,” us­ing large scale batch re­ac­tors like the ones used to grow up an­ti­bod­ies or oth­er pro­teins.

“We can be mak­ing, you know, bil­lions of cells at a time,” Main said. “So re­al­ly in­dus­tri­al­iz­ing the pro­duc­tion of cells,” he added lat­er.

Notch — named af­ter the sig­nal that must be trig­gered to dif­fer­en­ti­ate a stem cell in­to a T cell — is on the verge of hit­ting the first pre­clin­i­cal mile­stone in the Al­lo­gene deal, ac­cord­ing to Main, though he de­clined to pro­vide more de­tail on that pro­gram.

It’s al­so work­ing on es­tab­lish­ing it­self in Seat­tle, in ad­di­tion to its cur­rent Toron­to and Van­cou­ver bases. The pur­pose? To build a de­vel­op­ment team around new­ly hired se­nior vice pres­i­dent of pre­clin­i­cal trans­la­tion­al sci­ences Chris Bond, who Notch poached from Kite.

Al­lo­gene chipped in­to the Se­ries A, in ad­di­tion to Lu­mi­ra Ven­tures, and CCRM En­ter­pris­es Hold­ings, EcoR1 Cap­i­tal, Cas­din Cap­i­tal, Sam­sara Cap­i­tal, Am­pli­tude Ven­tures and an undis­closed in­vest­ment firm. An­oth­er undis­closed fund led the round.

Main called al­lo­gene­ic cell ther­a­pies the “Holy Grail” of the field. By the time a pa­tient and their doc­tor de­cide a cell ther­a­py is the best ap­proach, it of­ten takes weeks, or some­times months, to get the cells, pu­ri­fy them, grow them up, stim­u­late them and get them back to the pa­tients, Main said. Dur­ing that time, a pa­tient’s dis­ease can progress. Not to men­tion that it’s cost­ly and la­bor in­ten­sive.

“What we think we’re go­ing to be at is, we’ll be able to make the cells, have them cry­op­re­served sit­ting in a cen­tral dis­pen­sary some­where, and as soon as they’re or­dered, with­in hours they can be de­liv­ered and ad­min­is­tered to the pa­tient,” Main said.

He added lat­er, “We think we bring that fi­nal piece … to re­al­ly un­lock the po­ten­tial of cell ther­a­py to be­ing broad­ly ap­plic­a­ble and be­ing more drug-like.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

David Coman, Science 37

Amid vir­tu­al tri­al craze, Sci­ence 37 earns uni­corn sta­tus and a trip to Nas­daq on the back of SPAC deal

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As the Covid-19 pandemic made conventional trials impossible for some drugmakers, more and more companies moved to decentralize their clinical studies, accelerating business for tech developers like Science 37. Leveraging that boost, the company is on the verge of a SPAC merger, landing unicorn status and its very own stock ticker.