Glob­al Blood Ther­a­peu­tics buoyed by good news from FDA; Cigna to help cov­er pricey ther­a­pies

→ Glob­al Blood Ther­a­peu­tics $GBT has much to cheer about. Not on­ly has the FDA grant­ed its sick­le cell dis­ease (SCD) drug pri­or­i­ty re­view, the agency is not hold­ing an ad­vi­so­ry com­mit­tee to de­lib­er­ate on the drug. Shares of the San Fran­cis­co-based biotech jumped more than 14% to $54.25 be­fore the bell on Thurs­day. The US reg­u­la­tor is ex­pect­ed to de­cide on the once-dai­ly pill vox­elo­tor — which is de­signed to work by in­creas­ing he­mo­glo­bin’s affin­i­ty for oxy­gen — by Feb­ru­ary 26, 2020.

The field for SCD ther­a­pies is get­ting crowd­ed. Months ago, No­var­tis$NVS SCD drug, crizan­l­izum­ab, se­cured pri­or­i­ty re­view for its abil­i­ty to pre­vent de­bil­i­tat­ing­ly painful va­so-oc­clu­sive cri­sis in pa­tients. Oth­er drug­mak­ers, in­clud­ing blue­bird bio $BLUE, Imara, and part­ners CRISPR Ther­a­peu­tics $CR­SP and Ver­tex $VRTX, are al­so work­ing on their own ther­a­pies. Pfiz­er $PFE and Gly­coMimet­ics $GLYC are test­ing their SCD drug, riv­ipansel, in an on­go­ing Phase III study. In 2017, Em­maus Life Sci­ences se­cured SCD ap­proval from the FDA for En­dari — a phar­ma­ceu­ti­cal ver­sion of L-glu­t­a­mine, al­though the EU reg­u­la­tors did not back the drug.

→ Health in­sur­er Cigna $CI, which late last year merged with Ex­press Scripts, has found a way to cov­er pricey gene ther­a­pies. As part of a new of­fer­ing, it will now cov­er Lux­tur­na, ap­proved for rare cas­es of ge­net­ic blind­ness, from Spark Ther­a­peu­tics — a com­pa­ny which is in the long process of be­ing ac­quired by Roche — as well as the world’s most ex­pen­sive gene ther­a­py, No­var­tis$NVS Zol­gens­ma, for an in­her­it­ed mus­cle-wast­ing con­di­tion called spinal mus­cu­lar at­ro­phy. Con­sumers who are cov­ered un­der the new pro­gram will have no out-of-pock­et pay­ments re­lat­ed to the cost of the med­ica­tions, the com­pa­ny said on Thurs­day.

→ Days af­ter un­veil­ing plans to make a $60 mil­lion Nas­daq de­but, Mon­tre­al-based Bel­lus Health has up­sized its IPO, rais­ing $70 mil­lion by of­fer­ing about 9.9 mil­lion shares at $7.10/share. Bel­lus has po­si­tioned it­self as David to Mer­ck‘s Go­liath in the field of chron­ic cough by de­vel­op­ing drugs to block the P2X3 re­cep­tor, which is un­der­stood to play a role in hy­per­sen­si­tiv­i­ty dis­or­ders. In its IPO fil­ing, Bel­lus un­der­scored that Mer­ck’s late-stage drug, gefapix­ant, trig­gered taste loss or al­ter­ation in over 70% of pa­tients who re­ceived 50 mg of the drug in its lat­est tri­al — ver­sus 5% of pa­tients in Bel­lus’s study. In Ju­ly, Bel­lus be­gan its Phase II tri­al for chron­ic cough, which af­fects 26 mil­lion Amer­i­cans.

→ Af­ter their top­i­cal treat­ment for alope­cia area­ta, li­censed from Rigel Phar­ma, failed a mid-stage study back in June — tum­bling their stock by 32.58% — Aclaris Ther­a­peu­tics is lay­ing off 86 em­loy­ees and look­ing to part­ner out some pro­grams to help right the ship. The new­ly com­plet­ed strate­gic re­view calls for a trim­ming of Aclaris’ sales­force, of­fload­ing the re­spon­si­bil­i­ty of com­mer­cial­iz­ing cer­tain prod­ucts to an­oth­er com­pa­ny. The pro­grams in­clude Rho­fade (oxymeta­zo­line hy­drochlo­ride) cream, 1%; their drug can­di­date A-101 45% top­i­cal so­lu­tion (a po­ten­tial treat­ment for ver­ru­ca vul­garis or com­mon warts); and the drug can­di­dates ATI-501 (oral) and ATI-502 (top­i­cal), which are in­ves­ti­ga­tion­al Janus ki­nase (JAK) 1/3 in­hibitors for the po­ten­tial treat­ment of alope­cia.

Aclaris will con­tin­ue the de­vel­op­ment of ATI-450 (in­ves­ti­ga­tion­al oral MK2 in­hibitor), cur­rent­ly in a Phase I clin­i­cal tri­al for rheuma­toid arthri­tis, and for ATI-1777. The ter­mi­na­tions are es­ti­mat­ed to cost be­tween $3.0 mil­lion and $3.5 mil­lion over the next 6 months.

Schrödinger an­nounced the ex­pan­sion of its mul­ti-year, mul­ti-tar­get drug dis­cov­ery col­lab­o­ra­tion with Take­da, which comes a few months af­ter the com­pa­ny re­ceived an ad­di­tion­al $110 mil­lion back­ing in its first ven­ture round. The com­pa­ny says that the “ex­pan­sion in­creas­es the po­ten­tial num­ber of drug dis­cov­ery pro­grams that can be ini­ti­at­ed un­der the col­lab­o­ra­tion and in­creas­es the cu­mu­la­tive val­ue of po­ten­tial fu­ture mile­stone pay­ments that Schrödinger is el­i­gi­ble to re­ceive per pro­gram.”

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.