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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.