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.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

And it’s impressive.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.