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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

Overnight for­tunes are be­ing made in biotech these days — and it's both en­cour­ag­ing and more than a lit­tle bit scary

Just to complete the last leg of a running story I’ve been tracking for a few weeks, Olema $OLMA has come through its IPO from the Thursday night pricing at $19 a share with a market cap just north of $2 billion.

That leaves newly-named CEO Sean Bohen holding a batch of 1,110,896 shares with a strike price of $4.82. As of Tuesday morning, the stock is now trading at $53.40, giving him a portfolio value of $53.4 million. Not bad for someone who was hired in September.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.