EC ex­pands ap­proval of Am­gen's Blin­cy­to; FDA gives uniQure green sig­nal to be­gin Hunt­ing­ton's study in hu­mans, Cy­to­ki­net­ics feed­back on SMA tri­al

→ The first ther­a­py to win ap­proval for can­cer pa­tients in re­mis­sion with min­i­mal resid­ual dis­ease in Eu­rope is Am­gen‘s $AMGN BiTE im­munother­a­py, Blin­cy­to. The drug’s EC ap­proval has been ex­pand­ed to in­clude cer­tain pa­tients with acute lym­phoblas­tic leukemia in first or sec­ond com­plete re­mis­sion with min­i­mal resid­ual dis­ease (MRD) – the pres­ence of can­cer cells that re­main de­tectable de­spite re­mis­sion – greater than or equal to 0.1%. The EC ap­proval is based on da­ta from the Phase II BLAST study, which demon­strat­ed the drug led to no de­tectable MRD, in 78 per­cent of pa­tients with­in one treat­ment cy­cle. The EC nod fol­lows a sim­i­lar FDA ap­proval last March.

→ De­spite stretched re­sources at the FDA, the agency has been busy. On Tues­day it cleared an ap­pli­ca­tion by gene ther­a­py de­vel­op­er uniQure $QURE to test its ex­per­i­men­tal drug, AMT-130, in Hunt­ing­ton’s dis­ease pa­tients. This puts the Dutch com­pa­ny on track to dose pa­tients  in a Phase I/II study in the sec­ond half of 2019. “We be­lieve fu­ture re­sults from AMT-130 will serve as im­por­tant proof of con­cept for uniQure’s CNS pro­grams, and more specif­i­cal­ly, the miQURE tech­nol­o­gy. For now, the ma­jor­i­ty of our fo­cus re­mains on the on­go­ing Phase 3 tri­al for AMT-061, for he­mo­phil­ia B,” Can­tor Fitzger­ald’s El­e­mer Piros wrote in a note.

Sep­a­rate­ly, the agency al­so pro­vid­ed Cy­to­ki­net­ics $CYTK feed­back that the six minute walk test is an ac­cept­able pri­ma­ry ef­fi­ca­cy end­point for a po­ten­tial reg­is­tra­tion pro­gram for the com­pa­ny’s ex­per­i­men­tal SMA drug, relde­sem­tiv, in pa­tients who have main­tained am­bu­la­to­ry func­tion. “Next up is the de­ter­mi­na­tion of next de­vel­op­ment steps in con­junc­tion with glob­al part­ner Astel­las, which is now al­so to in­clude a Phase 1 in healthy vol­un­teers (slat­ed to be­gin in 1Q19) to test the ef­fect of high­er dos­es on plas­ma con­cen­tra­tions,” HC Wain­wright’s Joseph Pant­gi­nis wrote in a note.

Pe­ter Thiel has joined a slate of Amer­i­can and Chi­nese in­vestors for Im­munochi­na’s $20.4 mil­lion Se­ries C as the com­pa­ny ex­tends its CAR-T am­bi­tions be­yond Chi­na. The fi­nanc­ing is ex­pect­ed to fund an ex­pan­sion of man­u­fac­tur­ing ca­pa­bil­i­ties as well as tri­als for its lead prod­uct for B-cell acute lym­phoblas­tic leukemia and non-Hodgkin’s lym­phoma. Shougang Fund, Qingzhe Cap­i­tal, Chi­na Re­sources In­no­v­a­tive Eq­ui­ty In­vest­ment Fund and Sher­pa Ven­ture Cap­i­tal are al­so in for the ride.

→ An in­ter­im snap­shot of ear­ly-stage da­ta eval­u­at­ing its non-opi­oid painkiller, resinifer­a­tox­in, is com­pelling enough to start plan­ning piv­otal tri­als this year, ac­cord­ing to Sor­ren­to Ther­a­peu­tics $SRNE. Da­ta on 30 pa­tients with pain due to os­teoarthri­tis in the knee were ex­am­ined from the on­go­ing study, and the re­sults sug­gest­ed pain re­duc­tion was ob­served “as ear­ly as the day fol­low­ing drug ad­min­is­tra­tion and sus­tained over time”, and pa­tients will be mon­i­tored for 12 months fol­low­ing drug ad­min­is­tra­tion, af­ter which a dose will be se­lect­ed for fur­ther de­vel­op­ment.

→ As it con­tin­ues a so­lo — and still low-pro­file — ef­fort to prove its cell ther­a­py can serve as a uni­ver­sal bone mar­row donor source, Gami­da Cell $GM­DA will get a hand from the ser­vice provider Be The Match Bio­Ther­a­pies in both ex­e­cut­ing late-stage de­vel­op­ment pro­grams and reach­ing pa­tients if the treat­ment be­comes avail­able. The col­lab­o­ra­tion gives Is­rael-based Gami­da Cell ac­cess to Be The Match Bio­Ther­a­pies’ donor reg­istry as well as case man­age­ment, lo­gis­tics tools and re­la­tion­ships with cord blood banks around the world.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Bomb squad called to As­traZeneca vac­cine plant; Lu­men nabs CARB-X award for low-cost an­tidiar­rheal

A plant located in Wrexham, Wales that is packing the Oxford/AstraZeneca Covid-19 vaccine into vials was surrounded by a bomb squad after officials called police to report a suspicious package.

The alert caused a partial evacuation of the plant, the BBC was among those to report Wednesday. The owner of the plant, British drugmaker Wockhardt UK, said it was cooperating with local authorities and that there were no reports of any injuries.

Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

As new Covid-19 task force gets un­der­way, threat looms of vac­cine, mon­o­clon­al an­ti­body-re­sis­tant vari­ants

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Florian Brand (L) and Srinivas Rao (ATAI)

Psy­che­del­ic biotech ATAI hopes to ex­pand port­fo­lio through re­search part­ner­ship with Mass Gen­er­al

Psychedelics have made a comeback for mental health research, with companies like startup biotech ATAI Life Sciences raising millions and earning the backing of prominent investors like Peter Thiel, but there’s a hole at the heart of the resurgence: Researchers still don’t fully understand how they work.

A new partnership between ATAI and world-renowned Mass General Hospital hopes to change that.