Plot­ting clin­i­cal en­try, James Wilson's gene ther­a­py start­up brings in bil­lion­aire for $110M Se­ries B

James Wil­son

The gene ther­a­py biotech that James Wil­son helped found to take some rare dis­ease pro­grams out of Penn all the way to an ap­proval has scored $110 mil­lion in its lat­est fi­nanc­ing.

Pas­sage Bio is get­ting a boost just sev­en months af­ter launch­ing with an­oth­er megaround. Ac­cess Biotech­nol­o­gy — the ther­a­peu­tic-fo­cused in­vest­ment arm of bil­lion­aire Len Blavat­nik’s con­glom­er­ate — is lead­ing the Se­ries B, which al­so in­clud­ed all the mar­quee in­vestors who pro­vid­ed the ini­tial $115 mil­lion: Or­biMed, Fra­zier Health­care Part­ners, Ver­sant Ven­tures, Lily Asia Ven­tures, New Leaf Ven­ture Part­ners and Vi­vo Cap­i­tal. New back­ers in­clude Box­er Cap­i­tal of Tavi­s­tock Group, High­line Cap­i­tal Man­age­ment, Lo­gos Cap­i­tal and Sphera Funds Man­age­ment.

Stephen Squin­to

The com­pa­ny is on track to ini­ti­ate clin­i­cal tri­als for its two lead pro­grams in the first half of 2020 as planned. True to its stat­ed mis­sion of tack­ling mono­genic CNS dis­ease, the ther­a­pies would treat GM1 gan­gliosi­do­sis, a neu­ron-de­stroy­ing dis­or­der most com­mon in in­fants, and fron­totem­po­ral de­men­tia, which over­whelm­ing­ly af­fects the el­der­ly, re­spec­tive­ly.

A third pro­gram in Krabbe dis­ease — in­tend­ed to fix a ge­net­ic de­fect caus­ing a short­age of galac­to­syl­ce­rami­dase, an im­por­tant en­zyme for mak­ing myelin — is ex­pect­ed to fol­low lat­er in the year.

Keep­ing the stream of pro­grams flow­ing, Pas­sage Bio dis­closed that it’s li­censed two ad­di­tion­al in­di­ca­tions from the Uni­ver­si­ty of Penn­syl­va­nia while keep­ing an op­tion to grab sev­en more.

Tachi Ya­ma­da

Once Penn’s Gene Ther­a­py Pro­gram — where Wil­son is di­rec­tor — com­pletes the IND-en­abling pre­clin­i­cal work with the help of the Or­phan Dis­ease Cen­ter, the ba­ton is passed to Pas­sage Bio for all clin­i­cal de­vel­op­ment, reg­u­la­to­ry strat­e­gy and even­tu­al com­mer­cial­iza­tion.

“As we con­tin­ue to work to­ward ad­vanc­ing our three lead pro­grams in­to the clin­ic over the com­ing year, we are fo­cused on our mis­sion to serve pa­tients by of­fer­ing best-in-class, life-trans­form­ing ther­a­pies,” said Stephen Squin­to, the Alex­ion co-founder and Or­biMed part­ner who is run­ning the shop as in­ter­im CEO, in a state­ment.

Liam Rat­cliffe, the new­ly mint­ed head of Ac­cess Biotech­nol­o­gy, is join­ing the board chaired by Tachi Ya­ma­da.

Ya­ma­da, a VC at Fra­zier for­mer­ly of Glax­o­SmithK­line and Take­da, was an old men­tor of Wil­son’s who of­fered cru­cial sup­port in the im­me­di­ate af­ter­math of a clin­i­cal tri­al that led to the death of a teenag­er and sent the gene ther­a­py field in­to a harsh win­ter.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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