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.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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News brief­ing: Com­pa­ny de­vel­op­ing 'mag­ic mush­room' treat­ment hits Nas­daq; Spring­Works en­ters spon­sored re­search agree­ment with Fred Hutch

Compass Pathways seeks to raise $127.5 million in an upsized IPO, by offering 7,500,000 shares priced at $17 apiece. The London-based company had initially filed for a $100 million IPO, according to its F-1 filing.

The company is researching psilocybin therapy, which combines psilocybin — a psychoactive ingredient found in some species of “magic mushrooms” — with psychological support to treat mental illnesses such as depression, anxiety and addiction. It plans on using IPO money to fund research and clinical development of its COMP360 psilocybin therapy.

#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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Stronger to­geth­er? Boehringer and Mi­rati team to put first KRAS-KRAS com­bo in the clin­ic

Researchers are still waiting to see how much any of the vaunted KRAS drugs now in the clinic can, after decades of preclinical research and some early human studies, help patients. But while they do, two of the leading developers will look to see whether a KRAS-KRAS combo might pose a better shot than any KRAS alone.

Boehringer Ingelheim and Mirati have signed a collaboration to combine Mirati’s closely-watched lead KRAS inhibitor, MRTX849, in a clinical trial with the pan-KRAS blocker that Boehringer has quietly developed with high expectations behind their flashier contenders.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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Christian Itin, Autolus CEO (Autolus)

#ES­MO20: Au­to­lus pro­vides glimpse of next-gen­er­a­tion CAR-T pro­gram, show­ing ear­ly pos­i­tive safe­ty da­ta

CAR-T therapies were hailed as a breakthrough when Novartis received the first FDA approval for Kymriah back in 2017. Though highly effective at treating certain types of blood cancers, CAR-Ts are also associated with severe and potentially deadly side effects, including lethal instances of cytokine release syndrome.

With this in mind, Autolus Therapeutics is looking to take a crack at a safer CAR-T and presented Phase II cohort data for its AUTO3 program at virtual ESMO 2020. The data showed that, among the 35 patients in the cohort being treated for r/r diffuse large B cell lymphoma, there were no instances of Grade 3 or higher CRS. Eight individuals saw Grade 1 inflammation while another four patients reached Grade 2.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

UP­DAT­ED: A mi­cro-cap with a po­ten­tial­ly promis­ing coro­n­avirus drug en­lists mask-skep­tic con­gress­man for DSMB

A small biotech that has talked up a potentially promising but unproven treatment for Covid-19 enlisted an unusual member for its study’s Data and Safety Monitoring Board: a sitting Republican congressman with close ties to the CEO and a history of mask skepticism.

NeuroRx, an Israeli biotech testing a lung inflammation drug in Covid-19 patients, tapped Maryland Rep. Andy Harris for the DSMB, Politico reported. Harris is an anesthesiologist but not a biostatistician, and he has questioned the CDC about a “cult of masks” in the US. Harris has known NeuroRx CEO Jonathan Javitt since the two worked at Johns Hopkins together over 20 years ago.

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