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.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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