Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Pas­sage Bio — Jim Wil­son’s self-de­scribed “lega­cy com­pa­ny” — has wooed a sea­soned biotech ex­ec­u­tive to steer the clin­i­cal en­try of its first three gene ther­a­py pro­grams.

Bruce Gold­smith

Bruce Gold­smith jumps to the helm of Pas­sage af­ter a brief CEO stint at Civet­ta, a can­cer-fo­cused start­up he helped launch while a ven­ture part­ner at Deer­field. He takes over from Or­biMed part­ner and in­ter­im chief Stephen Squin­to, who will now lead the R&D team.

He joins as the biotech preps IND fil­ings for three lead pro­grams in rare, mono­genic dis­eases of the cen­tral ner­vous sys­tem in 2020 — the lyso­so­mal stor­age dis­or­ders GM1 gan­gliasi­do­sis and Krabbe dis­ease, as well as fron­totem­po­ral de­men­tia.

“Bruce is ide­al­ly suit­ed to lead Pas­sage Bio as chief ex­ec­u­tive of­fi­cer giv­en his strong neu­ro­science back­ground cou­pled with his ro­bust health­care and biotech­nol­o­gy in­dus­try ex­pe­ri­ence,” board chair­man Tachi Ya­ma­da said in a state­ment.

Pas­sage launched last Feb­ru­ary with $115 mil­lion from a mar­quee group of Se­ries A in­vestors in­clud­ing Fra­zier (where Ya­ma­da is a part­ner), Or­biMed, Ver­sant Ven­tures, New Leaf Ven­ture Part­ners, Vi­vo Cap­i­tal and Lil­ly Asia Ven­tures. With an of­fice just a 10-minute walk away from Wil­son’s lab at the Uni­ver­si­ty of Penn­syl­va­nia, the com­pa­ny was de­signed to ap­ply the gene ther­a­py pi­o­neer’s 35-year ex­pe­ri­ence in­to “cross-cor­rec­tion­al ther­a­pies” for CNS.

Stephen Squin­to

Ac­cord­ing to what he calls the Jim Wil­son 90/10 rule, Squin­to pre­vi­ous­ly told End­points News, AAV vec­tors can cov­er and trans­duce 90% of mo­tor neu­ron cells but on­ly 10% to 15% of oth­er brain cells — mak­ing it dif­fi­cult to go af­ter in­di­ca­tions where broad trans­duc­tion is need­ed. But it can still prove use­ful in dis­or­ders that re­sult from mu­ta­tions in en­zymes that can be tak­en up by neigh­bor­ing cells once se­cret­ed nor­mal­ly.

A close pact with Penn’s Gene Ther­a­py Pro­gram and Or­phan Dis­ease Cen­ter gave Pas­sage rights to five pro­grams right out of the gate, with op­tions to li­cense sev­en more.

“It’s a very ag­gres­sive clin­i­cal de­vel­op­ment strat­e­gy across a mul­ti­tude of pro­grams,” Squin­to said as he closed a $110 mil­lion Se­ries B in Sep­tem­ber. “We’re not gonna re­ly on any one pro­gram to dri­ve the val­ue of Pas­sage, we’re gonna re­ly on what is a very very full pipeline of op­por­tu­ni­ties.”

Gold­smith will now lead a team of about 25 to build on pre­clin­i­cal and IND-en­abling da­ta from Wil­son’s lab — a com­pa­ny grow­ing ex­er­cise he honed as COO of Lyc­era. There, he was al­so cred­it­ed for a num­ber of busi­ness de­vel­op­ment ini­tia­tives.

The tran­si­tion in­to the clin­ic would al­so mean mov­ing pro­duc­tion from ear­ly fa­cil­i­ties at Penn to Paragon’s GMP sites, and even­tu­al­ly to a cus­tomized suite slat­ed for com­ple­tion in the third quar­ter of this year.

Squin­to, a rare dis­ease ex­pert who de­vot­ed much of his ca­reer to Alex­ion, will con­tin­ue to help over­see all of that as a board di­rec­tor.

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.

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

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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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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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‘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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