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.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.