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.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Look­ing to move past an R&D fi­as­co, Ipsen poach­es their new CEO from Sanofi

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew — who’s been leading one of the most advanced efforts to develop vaccines for Covid-19 — is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.