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.

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

As the Covid-19 pandemic made conventional trials impossible for some drugmakers, more and more companies moved to decentralize their clinical studies, accelerating business for tech developers like Science 37. Leveraging that boost, the company is on the verge of a SPAC merger, landing unicorn status and its very own stock ticker.