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.”

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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