Am­i­cus blue­prints growth plans for Philly-based gene ther­a­py group; Cash-strapped Com­pu­gen re­struc­tures, cuts 35 staffers

→ As Roche puts down its foot in Philadel­phia as the base for its bud­ding gene ther­a­py op­er­a­tions built around new sub­sidiary Spark Ther­a­peu­tics, Am­i­cus $FOLD is blue­print­ing its own gene ther­a­py group in the city.

Jeff Castel­li

Am­i­cus first made its for­ay in­to gene ther­a­py via the ac­qui­si­tion of Ce­lenex, which came with 10 pro­grams in neu­ro­log­ic lyso­so­mal stor­age dis­or­ders from Bat­ten dis­ease to Tay Sachs. Short­ly there­after, the rare dis­ease spe­cial­ist teamed up with James Wil­son at the Uni­ver­si­ty of Penn­syl­va­nia to ad­vance gene ther­a­py treat­ments for Pompe dis­ease, Fab­ry dis­ease, CD­KL5 de­fi­cien­cy and one oth­er undis­closed rare meta­bol­ic dis­or­der.

When com­plet­ed lat­er this year, its new gene ther­a­py cen­ter — which will al­so be its new glob­al R&D hub — will take up 75,000 square feet in uCi­ty Square, a short walk away from Wil­son’s lab. Jeff Castel­li, chief port­fo­lio of­fi­cer and now head of gene ther­a­py, will even­tu­al­ly lead a team of 200 at the fa­cil­i­ty along­side CSO Hung Do.

→ An­oth­er drug de­vel­op­er — Anaveon — is work­ing on an im­proved ver­sion of an IL-2 sans the tox­i­c­i­ty that has stymied the use of the orig­i­nal, Pro­leukin. The Swiss biotech, found­ed in late 2017 and spun out of the Uni­ver­si­ty of Zurich, has won the back­ing of Syn­cona and No­var­tis in a CHF 35 mil­lion (rough­ly $35 mil­lion) Se­ries A round, and has big am­bi­tions for its drug, aim­ing for broad use in on­col­o­gy: as a cell ther­a­py, vac­cine, check­point in­hibitor and in com­bi­na­tion with ra­dio­ther­a­py. Syn­cona — a UK-based in­vest­ment firm that counts The Well­come Trust (al­so a founder) and Can­cer Re­search UK as its in­vestors — has al­so tak­en 47% stake in Anaveon.

→ Strapped for cash to com­plete an ex­pand­ed Phase I im­muno-on­col­o­gy pro­gram, Com­pu­gen is cut­ting in­to its 100-per­son work­force and con­sol­i­dat­ing all op­er­a­tions in its Is­rael lo­ca­tion. Around 35 em­ploy­ees are be­ing laid off, most­ly in R&D and pre­sum­ably US-based. The de­ci­sion, which is ex­pect­ed to ex­tend the com­pa­ny’s cash run­way through mid-2020 by sav­ing up to $10 mil­lion per year, is a re­sult of a strate­gic re­view fol­low­ing two dis­cov­ery part­ner­ships with Bris­tol-My­ers Squibb and As­traZeneca, says CEO Anat Co­hen-Dayag.

Pur­due Phar­ma sub­sidiary Im­bri­um Ther­a­peu­tics has en­list­ed Tetra­Ge­net­ics in a quest to dis­cov­er and de­vel­op non-opi­oid, ion-chan­nel an­ti­body ther­a­pies for chron­ic pain. Fea­tur­ing a $25 mil­lion up­front and biobucks up to $248 mil­lion, the deal comes as Pur­due is en­gulfed in a po­lit­i­cal storm for its role in the opi­oid epi­dem­ic.

→ An an­ti-ag­ing start­up called Sam­sara Ther­a­peu­tics — fo­cused on screen­ing for small mol­e­cules that ex­tend healthy lifes­pan across species — has se­cured undis­closed seed fund­ing from the ag­ing-fo­cused VC Apol­lo Ven­tures. The up­start, which has a part­ner­ship with Evotec, has de­buted with a pa­per in Na­ture, char­ac­ter­iz­ing the life-ex­tend­ing ef­fects of a nat­ur­al mol­e­cule de­rived from a Japan­ese herb called ashita­ba con­sumed on the is­land of Ok­i­nawa, which hosts the great­est num­ber of su­per­cente­nar­i­ans. It is al­so the first time Apol­lo has not just pro­vid­ed seed fund­ing to one of its port­fo­lio com­pa­nies, but is al­so help­ing build the com­pa­ny by pro­vid­ing the full sci­en­tif­ic team.

Mallinck­rodt has inked a re­search col­lab­o­ra­tion with Ger­many’s Tran­sim­mune to un­cov­er the mech­a­nism of ac­tion and po­ten­tial ap­pli­ca­tions of pho­to­phere­sis, the method of treat­ing blood with ul­tra­vi­o­let light that un­der­lies Mallinck­rodt’s Ther­akos plat­form. With Tran­sim­mune’s ex­per­tise in im­munother­a­py, the part­ners are hop­ing to find new ev­i­dence that pho­to­phere­sis can work in graft-ver­sus-host dis­ease or­gan trans­plant re­jec­tion and au­toim­mune dis­eases oth­er than cu­ta­neous T- cell lym­phoma, for which the treat­ment is al­ready ap­proved.

→ Hav­ing failed to win over in­vestors with its spin on some mid-stage can­cer vac­cine da­ta and seen its stock ham­mered in the months since, Sel­l­as is now plead­ing for help. The re­view of strate­gic al­ter­na­tives, as the com­pa­ny calls it, cov­ers every­thing from a sale, re­verse merg­er, fi­nanc­ing to fund­ed part­ner­ship. Mean­while, Sel­l­as still has a Phase III planned for gal­in­pepimut-S, which it’s al­so test­ing in an ear­ly tri­al in com­bi­na­tion with Mer­ck’s Keytru­da


With con­tri­bu­tion by Na­tal­ie Grover.

Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.

“Macrophages are interesting because we were all educated probably 20 years ago that they are the big eaters in the immune system, but they’re really the orchestrators of the immune system,” CEO Christine Bunt said.

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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CSL ac­cus­es ri­val Pharm­ing of par­tic­i­pat­ing in a scheme to rip off IP on HAE while re­cruit­ing se­nior R&D staffer

Pharming has landed in the middle of a legal donnybrook after recruiting a senior executive from a rival R&D team at CSL. The Australian pharma giant slapped Pharming with a lawsuit alleging that the Dutch biotech’s new employee, Joseph Chiao, looted a large cache of proprietary documents as he hit the exit. And they want it all back.
Federal Judge Juan Sanchez in the Eastern District Pennsylvania court issued an injunction on Tuesday prohibiting Chiao from doing any work on HAE or primary immune deficiency in his new job and demanding that he return any material from CSL that he may have in his possession. And he wants Pharming to tell its employees not to ask for any information on the forbidden topics.
For its part, Pharming fired off an indignant response this morning denying any involvement in extracting any kind of IP from CSL, adding that it’s cooperating in the internal probe that CSL has underway.

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Eli Lil­ly’s first PhI­II show­down for their $1.6B can­cer drug just flopped — what now?

When Eli Lilly plunked down $1.6 billion in cash to acquire Armo Biosciences a little more than a year ago, the stars seemed aligned in its favor. The jewel in the crown they were buying was pegilodecakin, which had cleared the proof-of-concept stage and was already in a Phase III trial for pancreatic cancer.

And that study just failed.

Lilly reported this morning that their cancer drug flopped on overall survival when added to FOLFOX (folinic acid, 5-FU, oxaliplatin), compared to FOLFOX alone among patients suffering from advanced pancreatic cancer.

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