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.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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SQZ, Ery­tech kick off $57M cell ther­a­py part­ner­ship; Jean-Paul Kress lands new CEO gig at Mor­phoSys

→ In a mar­riage of two tech­nolo­gies meant to make cell ther­a­pies more pow­er­ful, SQZ Biotech is team­ing up with France’s Ery­tech Phar­ma for a col­lab­o­ra­tion, with $57 mil­lion re­served for the first project and $50 mil­lion for each sub­se­quent ap­proval (prod­uct or in­di­ca­tion). Hav­ing ac­cess to Ery­tech’s method of fash­ion­ing ther­a­peu­tics from red blood cells, the Cam­bridge, MA-based com­pa­ny said, will am­pli­fy SQZ’s cell en­gi­neer­ing ca­pa­bil­i­ties and al­low them to de­vleop a new class of im­munomod­u­la­to­ry ther­a­pies. Its own tech — so far ap­plied in can­cer but al­so has po­ten­tial in di­a­betes — tem­po­rary dis­rupts the cell mem­brane by squeez­ing the cell, thus cre­at­ing a brief win­dow for tar­get ma­te­ri­als such as anti­gens to en­ter.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three ma­jor buy­outs an­nounced: Take­da/Shire; Bris­tol-My­ers/Cel­gene and now Ab­b­Vie/Al­ler­gan. And with this lat­est deal it’s in­creas­ing­ly clear that the sharp fall from grace suf­fered by high-pro­file play­ers which have seen their share prices blast­ed has cre­at­ed an open­ing for the growth play­ers in big phar­ma to up their game — in sharp con­trast to the pop­u­lar bolt-on deals that have been dri­ving the growth strat­e­gy at No­var­tis, Mer­ck, Roche and oth­ers.

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UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Tasly Bio­phar­ma pitch­es long-await­ed IPO — will it trig­ger an­oth­er $1B gold rush on HKEX?

In the run up to the Hong Kong stock ex­change’s an­tic­i­pat­ed rule change — open­ing the door for Chi­nese pre-rev­enue biotechs to go pub­lic clos­er to home — more than a year ago, Tasly Bio­phar­ma was one of the big play­ers whose ru­mored in­ter­est helped stoke en­thu­si­asm for the new list­ing venue. The com­pa­ny has since kept the drum­roll rum­bling in the back­ground, rais­ing a pre-IPO round and con­vinc­ing part­ner Trans­gene to swap own­er­ship in a joint ven­ture for eq­ui­ty. Now the oth­er shoe has fi­nal­ly dropped as ex­ecs out­line plans for a pipeline dom­i­nat­ed by car­dio­vas­cu­lar drugs.

With 4 more biotech IPOs due to wrap up Q2, how is the class of 2019 far­ing?

With 22 biotech IPOs on the books and four more set to price in the last week of June, in­vest­ment ad­vis­er Re­nais­sance Cap­i­tal has tak­en the pulse of the re­cent rush.

By the IPO ex­perts’ count, 25 out of 32 health­care of­fer­ings this year have been from biotechs — dif­fer­ing slight­ly from Brad Lon­car’s tal­ly — and the over­all pic­ture is one of un­der­per­for­mance. While they av­er­aged a first-day re­turn of 9.0%, col­lec­tive­ly they have trad­ed down to a 5.9% re­turn. Turn­ing Point $TP­TX and Cor­texyme $CRTX emerged on top at the half-year mark, ris­ing 135% and 109% re­spec­tive­ly.

Eye­ing a $500M peak sales pot, Almi­rall dou­bles down on le­brik­izum­ab as Der­mi­ra lines up PhI­II

With eyes on what it be­lieves is a $500 mil­lion peak rev­enue op­por­tu­ni­ty in Eu­rope, Barcelona-based Almi­rall has stepped up with $50 mil­lion in cash to take up the op­tion on Der­mi­ra’s IL-13 an­ti-in­flam­ma­to­ry drug le­brik­izum­ab just ahead of the start of Phase III. And there’s an­oth­er $30 mil­lion due as the late-stage pro­gram gets geared up.

That shouldn’t be long from now, as Der­mi­ra ex­pects to be­gin the late-stage tri­al work for atopic der­mati­tis be­fore the end of this year as it fol­lows a trail that ex­ecs in­sist leads to block­buster re­turns. Along the way, they’ll need to take on the 600-pound go­ril­la in atopic der­mati­tis: the IL-13/IL-4 drug Dupix­ent, from Re­gen­eron and Sanofi. Ri­vals al­so in­clude Leo Phar­ma, in its piv­otal with tralok­izum­ab, and Anap­tys­Bio in the hunt with a mid-stage pro­gram for etokimab, pre­vi­ous­ly re­ferred to as ANB020.