Ver­tex shut­ters a PhI­II cys­tic fi­bro­sis com­bo study, shares slip

Shares of Ver­tex slid Mon­day af­ter­noon af­ter the com­pa­ny said it will scrap one of its late-stage stud­ies for a com­bi­na­tion ther­a­py for cys­tic fi­bro­sis af­ter it failed to pass muster in a fu­til­i­ty analy­sis.

Ver­tex $VRTX is halt­ing its study of VX-661 and Ka­ly­de­co (iva­caftor) in peo­ple with one copy of the F508del mu­ta­tion and one copy of a mu­ta­tion that re­sults in min­i­mal CFTR pro­tein func­tion (F508del het/min). The study stum­bled in its first phase, with­out an im­prove­ment in lung func­tion.

Shares of Ver­tex slipped 3.4% on the news.

That het/min tar­get rep­re­sent­ed the largest mar­ket op­por­tu­ni­ty for Ver­tex with VX-661, ac­cord­ing to a note from Leerink’s Ge­of­frey Porges this morn­ing. But Porges al­so called it the least like­ly of the four tri­als to suc­ceed. Even so, the re­main­ing late-stage stud­ies in the VX-661 stud­ies of­fer a sub­stan­tial mar­ket op­por­tu­ni­ty. Notes Porges:

Our analy­sis and con­ver­sa­tions with MEDA­Corp spe­cial­ists sug­gest that Vx-661/Ka­ly­de­co could gen­er­ate an im­prove­ment in ppFEV1 in the range of 4-5% in the ho­mozy­gous delF508 pa­tients, com­pared to Orkam­bi’s 2-3%, with greater ef­fects on hos­pi­tal­iza­tions and ex­ac­er­ba­tions as well; Vx-661 should be eas­i­er to take (less drug-drug in­ter­ac­tions, less tol­er­a­bil­i­ty and meta­bol­ic ef­fects) and have more ef­fect than Orkam­bi; this suc­cess should trans­late in­to in­cre­men­tal rev­enue po­ten­tial that we es­ti­mate to be in the range of ~$485mm, as­sum­ing com­pa­ra­ble pric­ing to Orkam­bi in all mar­kets.

Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz

“While we rec­og­nize that peo­ple with CF with min­i­mal func­tion mu­ta­tions have a form of the dis­ease that is par­tic­u­lar­ly dif­fi­cult to treat, we be­lieved it was im­por­tant to eval­u­ate whether a dual com­bi­na­tion of VX-661 and iva­caftor could pro­vide some ben­e­fit to these pa­tients giv­en they do not to­day have a med­i­cine to treat the cause of their dis­ease,” said Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz. “These re­sults sug­gest that a triple com­bi­na­tion reg­i­men may pro­vide this group of peo­ple with CF the best chance at ob­tain­ing a mean­ing­ful ben­e­fit and we look for­ward to be­gin­ning the first study of a next-gen­er­a­tion cor­rec­tor to­geth­er with VX-661 and iva­caftor in this group of pa­tients lat­er this year, pend­ing da­ta from our on­go­ing Phase 1 stud­ies in healthy vol­un­teers.”

VX-661 is Ver­tex’s sec­ond-gen cor­rec­tor, a suc­ces­sor to the first-gen cor­rec­tor lumacaftor, in­clud­ed in the drug com­bi­na­tion for Orkam­bi. Two third-gen cor­rec­tors, VX-152 and VX-440, have been in ear­ly-stage stud­ies as Ver­tex plumbs new ap­proach­es that can treat CF bet­ter than Orkam­bi, which has suf­fered a high dropout rate. The treat­ment tar­gets the un­der­ly­ing ge­net­ic cause of the dis­ease.

The VX-661 pro­gram in­cludes four Phase III stud­ies in mul­ti­ple dif­fer­ent groups of peo­ple with CF who have at least one copy of the F508del mu­ta­tion.

Ver­tex has a $25.4 bil­lion mar­ket cap.

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.