Jim Wilson, Tachi Yamada, Stephen Squinto

How much does it cost to boot­strap a gene ther­a­py start­up? Not too much if you're Jim Wil­son, per Pas­sage Bio's $125M IPO fil­ing

Days ago when Deer­field part­ner Bruce Gold­smith jumped to helm Pas­sage Bio, he cit­ed a few rea­sons why it’s an “ex­treme­ly ex­cit­ing time” to be join­ing the gene ther­a­py play­er: IND fil­ings for three lead pro­grams, a da­ta-rich 2021 and an ex­pand­ing pipeline of ex­per­i­men­tal drugs for rare, mono­genic CNS dis­eases.

Bruce Gold­smith

It turns out there’s one more rea­son he held back: The Philadel­phia-based start­up was prep­ping an IPO.

Pas­sage Bio has pen­ciled in $125 mil­lion for its pub­lic de­but on the Nas­daq, with the ma­jor­i­ty of the pro­ceeds go­ing to the three AAV-based prod­uct can­di­dates slat­ed to en­ter the clin­ic in 2020.

Here’s the break­down on the tech be­hind each of them — of­fer­ing a glimpse of the ar­se­nal of de­liv­ery tools co-founder Jim Wil­son has as­sem­bled over the decades:

  1. PBGM01 uti­lizes a next-gen­er­a­tion AAVhu68 cap­sid to de­liv­er to the brain and pe­riph­er­al tis­sues a func­tion­al GLB1 gene en­cod­ing lyso­so­mal be­ta-galac­tosi­dase, or b-gal, for in­fan­tile GM1 gan­gliosi­do­sis
  2. PBFT02 uti­lizes an AAV1 cap­sid to de­liv­er to the brain a func­tion­al GRN gene en­cod­ing pro­gran­ulin, or PGRN, for FTD caused by pro­gran­ulin de­fi­cien­cy, or FTD-GRN
  3. PBKR03 uti­lizes a next-gen­er­a­tion AAVhu68 cap­sid to de­liv­er to the brain and pe­riph­er­al tis­sues a func­tion­al gene en­cod­ing the hy­drolyt­ic en­zyme galac­to­syl­ce­rami­dase, or GALC, for in­fan­tile Krabbe dis­ease

There are some com­peti­tors work­ing on each of the dis­eases they are tack­ling, in­clud­ing Ax­o­vant and Lyso­gene for GM1 as well as Alec­tor and Pre­vail for FTD, but Pas­sage Bio was con­fi­dent about be­ing an in­te­grat­ed play­er ca­pa­ble of tack­ling mul­ti­ple “cross-cor­rec­tion­al ther­a­pies” in the CNS.

Wil­son, a well-re­spect­ed pi­o­neer in the gene ther­a­py field, had in­tend­ed for this to be his lega­cy com­pa­ny, Or­biMed part­ner Stephen Squin­to pre­vi­ous­ly told End­points News. Thanks to Wil­son’s con­nec­tion at the Uni­ver­si­ty of Penn­syl­va­nia, Pas­sage se­cured a li­cens­ing deal with the uni­ver­si­ty for an up­front of just $2.5 mil­lion in cash and 3,720,000 shares in stock (then val­ued at $0.9 mil­lion) — plus a promise to fund cer­tain pre­clin­i­cal ac­tiv­i­ties, the S-1 re­vealed.

The pact cov­ered rights to 12 ther­a­pies in to­tal, and should Pas­sage ex­er­cise those op­tions by the 2022 ex­piry date, up­front fees would be some­where be­tween $0.8 mil­lion to $1 mil­lion. They have al­ready made the call on six; Penn is el­i­gi­ble for up to $16.5 mil­lion in de­vel­op­ment mile­stones and $55 mil­lion in sales mile­stones for each.

That, plus the $10 mil­lion up­front Pas­sage paid Catal­ent’s Paragon sub­sidiary for man­u­fac­tur­ing work, amount­ed to the bulk of the biotech’s ex­pense. While it’s bagged two hefty megarounds, Pas­sage has on­ly burned through $58.7 mil­lion so far.

The syn­di­cate stayed ba­si­cal­ly the same through­out Se­ries A and B, fea­tur­ing Fra­zier Life Sci­ences (13.9%, rep­re­sent­ed by Wil­son’s long­time men­tor Tachi Ya­ma­da on the board), Or­biMed (19.6%), Ver­sant Ven­tures (14.8%) Lil­ly Asia Ven­tures (7.6%), New Leaf Ven­tures (7.0%), Vi­vo Cap­i­tal (7.0%) and Ac­cess In­dus­tries’ AI Pas­sage (6.5%). Wil­son re­tained 7.0% for him­self.

With his rare dis­ease ex­pe­ri­ence gleaned from years at Alex­ion, Squin­to ini­tial­ly took up the CEO role be­fore pass­ing the reins to Gold­smith. His com­pen­sa­tion pack­age for the past year to­taled $2.2 mil­lion com­pen­sa­tion, and he con­tin­ues to be part of the team as in­ter­im head of R&D.

Chief tech­ni­cal of­fi­cer Alex Fo­topou­los and chief med­ical of­fi­cer Gary Ro­mano re­ceived $2.25 mil­lion and $2.02 mil­lion, re­spec­tive­ly.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

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David Meline, who forged his rep during a 6-year run at Amgen, slipped out the exit right after his Q2 “retirement” party in California — presumably virtual — and started the next chapter of his career at a biotech company betting big on revolutionizing the vaccine R&D space.

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