Bob Nelsen (Getty Images)

Bob Nelsen ex­its board and pres­i­dent steps down at Uni­ty as fall­out from the an­ti-ag­ing biotech's PhII miss con­tin­ues

Uni­ty Biotech­nol­o­gy is still reel­ing rough­ly four months af­ter ax­ing its lead pro­gram, as it saw sig­nif­i­cant change at the ex­ec­u­tive lev­el Mon­day.

The com­pa­ny an­nounced that ARCH Ven­ture Part­ners’ Bob Nelsen, one of Uni­ty’s co-founders back in 2011, has left the board. In ad­di­tion, pres­i­dent and co-founder Ned David is step­ping down at the end of the year, and an­oth­er board mem­ber, David Lacey, is al­so leav­ing the biotech.

With the de­par­tures, Uni­ty is ap­point­ing Gilmore O’Neill to the board to chair the com­pa­ny’s sci­ence com­mit­tee.

“We are in­cred­i­bly grate­ful to Ned, Bob and Dave for their ser­vice,” chair­man Kei­th Leonard said in a state­ment. “Ned’s ear­ly re­search in­to fun­da­men­tal path­ways of ag­ing bi­ol­o­gy form the foun­da­tion of UNI­TY. Bob played an in­stru­men­tal role in cre­at­ing and shap­ing a com­pa­ny tar­get­ing dis­eases of ag­ing, and David’s drug dis­cov­ery in­sights have shaped our pipeline.”

Mon­day’s moves were not the first de­par­tures since Uni­ty’s lead pro­gram flopped a ma­jor Phase II in os­teoarthri­tis of the knee in Au­gust. That whiff set this re­struc­tur­ing in mo­tion, and Nelsen had long been one of the head­lin­ers in a celebri­ty group of in­vestors that al­so in­clud­ed Jeff Be­zos and Pe­ter Thiel. Uni­ty had raised more than $200 mil­lion over the last decade, hop­ing its big-name part­ners could vault it to the fore­front of the buzzy an­ti-ag­ing field.

The com­pa­ny pre­vi­ous­ly said in Sep­tem­ber it was slash­ing its work­force by 30% in or­der to con­tin­ue its re­fo­cus­ing to­ward oph­thal­mol­o­gy and neu­rol­o­gy, putting the San Fran­cis­co-based biotech on track to em­ploy 75 full-timers by the end of 2020.

Au­gust’s tri­al miss it­self in­volved a 183-per­son study for what was then Uni­ty’s on­ly clin­i­cal pro­gram, UBX0101. Pa­tients in the treat­ment arms showed vir­tu­al­ly no dif­fer­ence com­pared to those tak­ing place­bo, and the can­di­date didn’t even reg­is­ter a dose-de­pen­dent re­sponse.

On­ly pa­tients who re­ceived the low­est dose nu­mer­i­cal­ly out­per­formed the con­trol, but the p-val­ue clocked in at an abysmal p=0.52.

Uni­ty’s big the­o­ry re­volves around what are known as senes­cent cells — non-di­vid­ing cells that clut­ter an ag­ing body. Uni­ty says the buildup in such cells is as­so­ci­at­ed with the re­lease of dozens of pro­teins that pro­mote in­flam­ma­tion, and re­mov­ing them, with­out al­ter­ing the bal­ance of cells the body needs, could pro­mote a longer, health­i­er life.

With the os­teoarthri­tis pro­gram scrapped, Uni­ty is now fo­cus­ing on a dif­fer­ent group of senes­cent cells for eye dis­eases. The next ex­per­i­men­tal drug is UBX1325, which is de­signed to tar­get Bcl-xL. And if all goes ac­cord­ing to plan, Uni­ty ex­pects to dose the first pa­tients in a Phase I study for the can­di­date lat­er this year.

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.

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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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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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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‘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.

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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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

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.

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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