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.

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.

Jim Mellon [via YouTube]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million Series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017. Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer $PFE and Amarin $AMRN.

AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.