Guardant blood test shows promise in pre­dict­ing im­munother­a­py re­sponse; As­traZeneca's di­a­betes drug wins EU la­bel ex­pan­sion

→ The field of liq­uid biop­sy is bur­geon­ing, and each play­er (there are plen­ty) is dogged­ly pur­su­ing con­sis­ten­cy and ac­cu­ra­cy — false pos­i­tives in­duce un­nec­es­sary anx­i­ety, and are cost­ly. Late on Sun­day, Guardant Health said its as­say, Guardant360, showed in a study it can ac­cu­rate­ly de­tect mi­crosatel­lite in­sta­bil­i­ty (MSI), a key bio­mark­er used to pre­dict re­sponse to im­munother­a­py re­gard­less of tu­mor type. MSI is a change that oc­curs in the DNA of cer­tain cells (such as tu­mor cells) in which the num­ber of re­peats of mi­crosatel­lites (short, re­peat­ed se­quences of DNA) is dif­fer­ent than the num­ber of re­peats that was in the DNA when it was in­her­it­ed.

Re­searchers com­pared the re­sults of 1,145 Guardant360 sam­ples to MSI sta­tus de­ter­mined us­ing stan­dard-of-care tis­sue test­ing; the Guardant360 re­sults were aligned with stan­dard test­ing in 98.4% of cas­es. In a co­hort of 16 high MSI gas­tric can­cer pa­tients treat­ed with check­point in­hibitors, out­comes pre­dict­ed by the Guardant test were con­sis­tent with re­spons­es for pa­tients iden­ti­fied by tis­sue test­ing.”Un­for­tu­nate­ly, less than half of all ad­vanced colon can­cer pa­tients are test­ed for this im­por­tant bio­mark­er. And across all sol­id tu­mors, we sus­pect the test­ing rate is far low­er, in part due to chal­lenges of work­ing with tis­sue sam­ples,” said Jeeyun Lee, as­so­ciate pro­fes­sor, di­vi­sion of hema­tol­ogy/on­col­o­gy, Sam­sung Med­ical Cen­ter in a state­ment.”By in­cor­po­rat­ing these re­sults in­to a sim­ple blood test, we should be able to in­crease the num­ber of pa­tients who get this in­for­ma­tion and ben­e­fit from ap­pro­pri­ate im­munother­a­py.”

As­traZeneca‘s $AZN SGLT2 di­a­betes drug, da­pagliflozin, has won a la­bel up­grade cour­tesy of the EMA. The drug, sold as Forx­i­ga, will car­ry a la­bel in­clud­ing pos­i­tive car­dio­vas­cu­lar (CV) out­comes and re­nal da­ta from a late-stage tri­al in­volv­ing adults with type 2 di­a­betes — a ma­jor­i­ty of whom had no ex­ist­ing car­dio­vas­cu­lar dis­ease. “For pa­tients with type-2 di­a­betes, heart fail­ure is one of the ear­li­est car­dio­vas­cu­lar com­pli­ca­tions be­fore heart at­tack or stroke. In the Phase III DE­CLARE-TI­MI 58 tri­al,Forx­i­ga re­duced the com­pos­ite end­point of hos­pi­tal­i­sa­tion for heart fail­ure or car­dio­vas­cu­lar death in pa­tients with type-2 di­a­betes,” said Mene Pan­ga­los, ex­ec­u­tive VP of Bio­Phar­ma­ceu­ti­cals R&D, in a state­ment.

→ UK-based drug de­vel­op­er Verona Phar­ma $VR­NA has scored in a mid-stage study test­ing its dry pow­der in­haler for­mu­la­tion of en­sifen­trine in COPD pa­tients. The pos­i­tive study fol­lows a failed Phase II tri­al test­ing the neb­u­liz­er for­mu­la­tion of the drug. Verona has tout­ed the drug as the first po­ten­tial ther­a­py for res­pi­ra­to­ry dis­eases that acts as both a bron­chodila­tor and an­ti-in­flam­ma­to­ry agent in a soli­tary com­pound.

Squarex‘s lead drug, SQX770, has cleared a mid-stage tri­al test­ing the top­i­cal im­munomod­u­la­tor for the longterm pre­ven­tion of re­cur­rent cold sores (her­pes labi­alis) and oth­er in­fec­tions caused by her­pes sim­plex virus­es. Da­ta from the 139-pa­tient study showed a sin­gle top­i­cal dose of SQX770 ap­plied to the up­per arm sig­nif­i­cant­ly ex­tend­ed time to next out­break and re­duced both the fre­quen­cy and sever­i­ty of out­breaks. “Our find­ings con­firm and val­i­date the re­sults from two pre­vi­ous stud­ies in­clud­ing a pre­vi­ous place­bo con­trolled clin­i­cal tri­al and there­fore, pave the road for the ini­ti­a­tion of a Phase 3 tri­al,” said Squarex chief Jack Tal­ley. “With no cur­rent pre­ven­ta­tive treat­ment op­tions on the mar­ket for the mil­lions of pa­tients who suf­fer from re­cur­rent HSV-1 out­breaks, we are ex­cit­ed to con­tin­ue the clin­i­cal de­vel­op­ment and hope­ful­ly be­com­ing the first and on­ly choice for com­bat­ing her­pes labi­alis – cold sores or oral her­pes.”

→ As 3-V Bio­sciences ex­e­cutes a strate­gic piv­ot to the in­creas­ing­ly crowd­ed NASH field, the San Ma­teo biotech is al­so chang­ing its name to Sagimet (a com­bi­na­tion of Sagit­ta and me­tab­o­lism) its new fo­cus on the liv­er. To be sure, it will con­tin­ue its re­search ef­forts in can­cer with its fat­ty acid syn­thase in­hibitor, but NASH will be­come much more top of mind now. As­cle­tis, the Chi­nese play­er best known for its he­pati­tis drugs, is en­dors­ing this strat­e­gy by lead­ing the sec­ond tranche of a $25 mil­lion Se­ries E. Qian­hai Ark, Klein­er Perkins and New En­ter­prise As­so­ci­ates (NEA) al­so par­tic­i­pat­ed.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.