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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

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First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.