GSK sub­mits its first ap­pli­ca­tion for dapro­du­s­tat in Japan; Two drug­mak­ers part with $15M to avoid opi­oid tri­al

→ Just as Hal Bar­ron planned, GSK $GSK has sub­mit­ted its first reg­u­la­to­ry ap­pli­ca­tion for its oral hy­pox­ia-in­ducible fac­tor pro­lyl hy­drox­y­lase in­hibitor (HIF-PHI), dapro­du­s­tat, in Japan for pa­tients with re­nal ane­mia due to chron­ic kid­ney dis­ease (CKD). The ap­pli­ca­tion is based on pos­i­tive Phase III da­ta con­duct­ed in Japan on ane­mic pa­tients across the spec­trum of CKD from stages 3-5 — which in­clud­ed pa­tients on and not on dial­y­sis, re­gard­less of pri­or ane­mia treat­ment with ery­thro­poiesis-stim­u­lat­ing agents (ESAs). If ap­proved, dapro­du­s­tat will be ex­clu­sive­ly dis­trib­uted in Japan by Ky­owa Kirin Co.

→ Two drug­mak­ers — En­do $ENDP and Al­ler­gan $AGN — have agreed to part with $15 mil­lion al­to­geth­er to avoid go­ing to tri­al in Oc­to­ber in a land­mark case by two Ohio coun­ties, who have ac­cused a raft of drug man­u­fac­tur­ers and dis­trib­u­tors of stok­ing the opi­oid cri­sis, Reuters re­port­ed on Tues­day. Oth­er drug firms em­broiled in the mul­ti­dis­trict lit­i­ga­tion in­clude Pur­due, Te­va and J&J as well as drug dis­trib­u­tors such as McKesson $MCK, Car­di­nal Health $CAH and Amerisource­Ber­gen $ABC.

“(T)he En­do set­tle­ment in two OH coun­ties would ex­trap­o­late to a po­ten­tial na­tion­al set­tle­ment of $1.8B for En­do, <50% of our pub­lished es­ti­mate,” SVB Leerink an­a­lysts wrote in a note. “The En­do set­tle­ment pro­vides a pos­i­tive readthrough for My­lan and Am­neal in­di­cat­ing that the set­tle­ment num­bers for them could end up be­ing <50% of our pub­lished es­ti­mates of $0.8B and $1.2B, re­spec­tive­ly. For Te­va and Mallinck­rodt, the readthrough is less clear and varies de­pend­ing on whether we use sales mix or vol­ume mix as our ba­sis…a key piece of the puz­zle in ad­di­tion to the city/coun­ty law­suits is the state AG law­suits which re­main a wild card, and we un­der­stand that tri­als for these cas­es will not be held un­til 2020.”

Cere­cor‘s $CERC oral for­mu­la­tion of D-man­nose — a nat­u­ral­ly oc­cur­ring mono­sac­cha­ride — called CERC-802 has been grant­ed fast track sta­tus by the FDA to treat Man­nose-Phos­phate Iso­merase De­fi­cien­cy (MPI-CDG). CDG’s are rare, in­her­it­ed, meta­bol­ic dis­or­ders with high in­fant mor­bid­i­ty and mor­tal­i­ty and no FDA-ap­proved treat­ments. The over­all es­ti­mat­ed oc­cur­rence of MPI-CDG glob­al­ly is less than 50 cas­es, al­though re­searchers sus­pect it is un­der-di­ag­nosed.

Tetra Bio-Phar­ma — a com­pa­ny fo­cused on de­vel­op­ing cannabi­noid-based ther­a­peu­tics — is join­ing forces with Thorne Re­search and Onegevi­ty to form a joint ven­ture, CB2 Ther­a­peu­tics. CB2 will fo­cus on de­vel­op­ing com­pounds for chron­ic in­flam­ma­to­ry con­di­tions with high un­met needs that aren’t ad­e­quate­ly served with cur­rent ther­a­pies. The joint ven­ture is tar­get­ing an ini­tial raise of $10,000,000 to start its Phase II clin­i­cal tri­als for ir­ri­ta­ble bow­el syn­drome (IBS) and the com­mer­cial­iza­tion of its di­etary sup­ple­ments for in­ter­sti­tial cys­ti­tis.

Ca­dent Ther­a­peu­tics — cur­rent­ly in col­lab­o­ra­tion with No­var­tis $NVS to de­vel­op a neg­a­tive al­losteric mod­u­la­tor, now in Phase II for the treat­ment of treat­ment-re­sis­tant de­pres­sion — wel­comed John McBride on board as CFO. McBride jumps over from the same role at Syn­tim­mune, where he helped with the com­pa­ny’s ac­qui­si­tion to Alex­ion $ALXN. Pri­or to his time at Syn­tim­mune, McBride served as the COO and CFO at Tokai Phar­ma­ceu­ti­cals and had stints at Glouces­ter Phar­ma­ceu­ti­cals, Phar­ma­cia, Cy­toTher­a­peu­tics and Phytera. In ad­di­tion, the com­pa­ny an­nounced the ap­point­ment of sev­er­al oth­er hires, in­clud­ing Min­nie Mild­woff, Vice Pres­i­dent, Reg­u­la­to­ry Af­fairs and Qual­i­ty As­sur­ance; Robert Gaffey, Vice Pres­i­dent, Fi­nance and Ad­min­is­tra­tion; Nicole Lu­osey, Vice Pres­i­dent, Strate­gic and Pro­gram Op­er­a­tions and Noah Good­man, Ex­ec­u­tive Di­rec­tor, Busi­ness De­vel­op­ment.

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

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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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 acquisitions, 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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'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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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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