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.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.

As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.