Jaime Shamonki, Generate CMO

Al­ready sit­ting on a ma­jor stem cell bank, Gen­er­ate looks to grow its re­gen­er­a­tive med­i­cines busi­ness

Gen­er­ate Life Sci­ences is the largest pri­vate new­born stem cell biorepos­i­to­ry, and with the an­nounce­ment of its own in-house man­u­fac­tur­ing fa­cil­i­ty, the com­pa­ny is look­ing to grow its re­gen­er­a­tive med­i­cine pipeline.

Gen­er­ate has es­tab­lished a fa­cil­i­ty in La Jol­la, CA, to pro­vide end-to-end man­u­fac­tur­ing for new­born stem cell bi­o­log­ics. The site will al­low Gen­er­ate to evolve as it adds a per­son­al­ized ther­a­peu­tics de­vel­op­er to its cur­rent busi­ness mod­el. The new site has been de­signed to har­ness mes­enchy­mal stem cells from the tis­sue of the um­bil­i­cal cord.

“With this fa­cil­i­ty, we will have the in-house ca­pa­bil­i­ty to take these stem cells through the com­plete process of col­lec­tion, cryo­genic stor­age and ul­ti­mate­ly bi­o­log­ics man­u­fac­tur­ing,” CMO Jaime Sha­mon­ki said in a press re­lease. “This will al­low us to pow­er our stud­ies in a much more ef­fi­cient and stream­lined man­ner and help us bet­ter serve the fam­i­lies who have stored over 1.2 mil­lion stem cell units with us.”

Sha­mon­ki and her team have the chance to do some­thing spe­cial: They’re work­ing to find a treat­ment for “long Covid,” or the lin­ger­ing side ef­fects from the dis­ease.

But while com­ing up with a long-term so­lu­tion to a prob­lem that’s sure to im­pact peo­ple around the world for years to come is im­por­tant, there’s an added ben­e­fit to the project. The chief med­ical of­fi­cer for Gen­er­ate Life Sci­ences gets to learn how the cells re­pro­gram the im­mune sys­tem, and di­rect those find­ings to­ward dis­cov­er­ing cures for au­toim­mune dis­eases, Ly­me dis­ease and chron­ic fa­tigue syn­drome.

Ear­li­er in June, Gen­er­ate ac­quired Cell Care in­ter­na­tion­al cord blood bank­ing group, with in­cludes Cell Care in Aus­tralia, and In­scep­tion Lifebank and Cells for Life in Cana­da. The move helps Gen­er­ate be­come more glob­al with its reach, and pro­vides ac­cess to fam­i­lies in Cana­da and Aus­tralia.

The com­pa­ny’s reach now stretch­es across three coun­tries, in­clud­ing the US.

Stem cells from um­bil­i­cal cords of­fer an­ti-in­flam­ma­to­ry and tis­sue repar­a­tive prop­er­ties. Gen­er­ate is in the mid­dle of stud­ies right now that are look­ing at tar­get­ing au­toim­mune dis­ease, neu­ro­log­i­cal dis­eases and lung dis­eases. Af­ter back­ing out of the Covid-19 vac­cine field due to over­crowd­ing, Gen­er­ate es­tab­lished a tri­al for pa­tients with res­pi­ra­to­ry com­pli­ca­tions stem­ming from the virus us­ing a ther­a­py de­rived from new­born stem cells. It’s al­so look­ing at clin­i­cal tri­als for a treat­ment for Type 1 di­a­betes.

“We are see­ing this rapid change in our sci­en­tif­ic ori­en­ta­tion of how they can be used, so pre­vi­ous­ly it was trans­plants but sort of low-in­ci­dence con­di­tions for how things could be treat­ed this way, like thank god child­hood leukemia is not all that com­mon,” Sha­mon­ki said. “But now we’re look­ing at re­gen­er­a­tive med­i­cine in­di­ca­tions.”

The com­pa­ny is try­ing to in­vest in both clin­i­cal tri­als with aca­d­e­m­ic part­ners and their own man­u­fac­tur­ing fa­cil­i­ties, to en­able the donor fam­i­lies to eas­i­ly ac­cess their own units, if need­ed for ther­a­pies in the fu­ture. With Gen­er­ate’s ac­qui­si­tion of Cell Care in­ter­na­tion­al, it will al­low for ex­pand­ed ac­cess to do­nat­ed tis­sue and cord blood from a larg­er pool, as well as uti­lize its sci­en­tif­ic and reg­u­la­to­ry knowl­edge spe­cif­ic to each coun­try.

“It’s re­al­ly all about de­liv­er­ing val­ue to the fam­i­lies who’ve banked new­born stem cells with us, as op­posed to try­ing to get 10 bi­o­log­ics to see what we can get through Phase III and what sticks,” Sha­mon­ki said.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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