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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.