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 fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

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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Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

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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