Ver­tex shares shoot up, adding $8B in mar­ket cap af­ter cys­tic fi­bro­sis triples 'knocked it out of the park'

Ver­tex CMO Jef­frey Chodake­witz

Ver­tex came out of the chute af­ter the mar­ket closed on Tues­day boast­ing about the ef­fi­ca­cy da­ta their re­searchers are see­ing in a range of three Phase I and II stud­ies for its new triple com­bos for cys­tic fi­bro­sis. The da­ta are still ear­ly but clear­ly bode well for Ver­tex, which is work­ing hard to cast a far wider net among cys­tic fi­bro­sis pa­tients who have achieved mod­est but sig­nif­i­cant gains with their lead drug Ka­ly­de­co, which has won a sharp kick­back from pay­ers over its hefty price.

One pos­si­ble fly in the oint­ment to­day: Two pa­tients ex­pe­ri­enced a spike in liv­er en­zymes, though that re­solved af­ter they stopped ther­a­py. And that didn’t slow a mar­ket ral­ly in af­ter-hours trad­ing, with shares $VRTX soar­ing 26% on the care­ful­ly pack­aged news, adding $8 bil­lion to its mar­ket cap.

For a biotech that start­ed Tues­day with a mar­ket cap of $32 bil­lion, that’s a clean hit.

The fo­cus to­day is on VX-440, VX-152 and VX-659.

For triple com­bi­na­tion reg­i­mens us­ing VX-152 or VX-440 there was a mean ab­solute im­prove­ment in FEV1 — a key mea­sure of lung ca­pac­i­ty — from base­line of 9.7% and 12% from two Phase II stud­ies.

Bri­an Sko­r­ney, Baird an­a­lyst

For the triple us­ing VX-659, teza­caftor and iva­caftor in peo­ple with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, the triple demon­strat­ed a 9.6% im­prove­ment in FEV1 in a Phase I study.

An­a­lysts of a va­ri­ety of stripes were clear­ly wowed by the da­ta. The clap­ping got start­ed quick­ly the minute the com­pa­ny’s re­lease hit the wires. Ver­tex has at­tract­ed plen­ty of buy­out talk with its suc­cess in CF, and the fo­cus now is on bil­lions of dol­lars in po­ten­tial new rev­enue – pro­vid­ed the da­ta con­tin­ue to break its way with­out any spoil­ers from ri­vals in the field.

Ver­tex “knocked it out of the park,” not­ed Baird’s Bri­an Sko­r­ney ear­ly Wednes­day. “Based on the re­port­ed re­sults, up­wards of 70% of CF pa­tients on a Ver­tex reg­i­men will like­ly see a ben­e­fit on par with what Ka­ly­de­co pro­vides to those with gat­ing mu­ta­tions.”

Ge­of­frey Porges, Leerink

Ge­of­frey Porges at Leerink, who’s urged Gilead to buy Ver­tex, put it this way:

The re­sults ex­ceed­ed our ex­pec­ta­tions for these tri­als, with all three com­bi­na­tions test­ed in the het min pop­u­la­tion ex­ceed­ing our ex­pec­ta­tions for the tri­al, and the sin­gle com­bi­na­tion stud­ied in delF508 ho­mozy­gous pa­tients al­so ex­ceed­ing our ex­pec­ta­tions. Ver­tex is study­ing four dif­fer­ent “sec­ond mech­a­nism” cor­rec­tors, added to the back­bone of its teza­caftor-iva­caftor com­bi­na­tion. Ver­tex al­so re­port­ed Cys­tic Fi­bro­sis Ques­tion­naire-Re­vised (CFQ-R) score im­prove­ments of 18.3 and 20.7 points for VX-440 200mg and 600mg, re­spec­tive­ly, com­pared to place­bo of 2.2 points. This score cal­cu­lates the qual­i­ty of life im­prove­ment on drug, and the gold-stan­dard CF treat­ment Ka­ly­de­co re­sult­ed in a low­er 8.1-8.6 point in­crease, while Orkam­bi led to a much low­er 1.5-2.9 point in­crease.

Michael Yee at Jef­feries not­ed:

VRTX showed all three dif­fer­ent triple pill com­bos worked in het-min CF pa­tients – ex­pand­ing the mar­ket and sug­gest­ing peak sales for VRTX go­ing from $2-4B to­wards $4-6B and set to be pre­mier growth sto­ry in large cap biotech now.

Ver­tex has been work­ing on four of what it de­scribes as next-gen cor­rec­tors, look­ing for the best to move ahead first. To­day Ver­tex said that it has a VX-445 Phase II study un­der­way, with a VX-659 Phase II set to be­gin in ear­ly Au­gust. VX-445 and VX-659 will be eval­u­at­ed in triple com­bi­na­tion with teza­caftor and iva­caftor in peo­ple with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion and will be eval­u­at­ed in peo­ple with two copies of the F508del mu­ta­tion who are al­ready re­ceiv­ing teza­caftor and iva­caftor. Da­ta from both Phase II stud­ies are ex­pect­ed in ear­ly 2018.

“These safe­ty and ef­fi­ca­cy da­ta are clear and com­pelling, in­di­cat­ing sig­nif­i­cant po­ten­tial ben­e­fit for peo­ple with CF from each of these three dif­fer­ent triple com­bi­na­tion reg­i­mens,” said Jef­frey Chodake­witz, ex­ec­u­tive vice pres­i­dent and CMO at Ver­tex.  “We will be col­lect­ing and eval­u­at­ing ad­di­tion­al da­ta from these and oth­er stud­ies and will make a de­ci­sion on which reg­i­men(s) to take for­ward in­to piv­otal pro­gram(s), which we ex­pect to be­gin in the first half of 2018.”

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.