Zo­genix rock­ets up as PhI­II study of­fers im­pres­sive da­ta on Dravet syn­drome, vault­ing over a ri­val

Zo­genix of­fered in­vestors ex­act­ly the kind of gourmet da­ta they were look­ing for this morn­ing with a clean sweep of promis­ing da­ta for its late-stage drug ZX008, de­signed to tack­le a se­vere form of child­hood epilep­sy. And the news pow­ered a big spike in its share price $ZGNX as Wall Street con­tin­ues to show­er re­wards on clear suc­cess­es.

Stephen Farr

The first of two Phase III stud­ies for this drug, re­searchers say they tracked a clear dose-de­pen­dent re­sponse for this treat­ment, with a dra­mat­ic drop in the rate of seizures ex­pe­ri­enced by pa­tients with Dravet syn­drome.

The key score: At the high end, the dose cut the month­ly con­vul­sive seizure fre­quen­cy by 72.4% in the 0.8 mg/kg/day pa­tients drug arm com­pared to a 17.4% drop in place­bo pa­tients.

The drug al­so scored a clean sweep on the sec­ondary end­points.

Zo­genix’s stock price ini­tial­ly soared more than 200% on the news, a sign of the eu­pho­ria that now greats a big win in biotech. But the zeal cooled a lit­tle and by mid-morn­ing the biotech had to set­tle for a 123% jump in share price.

Leerink’s Paul Mat­teis was among the first an­a­lysts to of­fer a con­grat­u­la­to­ry note this morn­ing:

Not on­ly did the clin­i­cal tri­al meet its pri­ma­ry end­point with a high de­gree of sta­tis­ti­cal sig­nif­i­cance – the mag­ni­tude of clin­i­cal ben­e­fit was bet­ter than we ex­pect­ed, and in the ball­park of pri­or com­pas­sion­ate use stud­ies that con­veyed an un­prece­dent­ed lev­el of ben­e­fit.

The biotech is well on its way to com­plet­ing en­roll­ment for Study 2, which is ex­pect­ed to read out in H1 2018, with a new drug ap­pli­ca­tion to fol­low in the sec­ond half — pro­vid­ed all goes ac­cord­ing to plan.

Zo­genix’s suc­cess came at the ex­pense of GW Phar­ma­ceu­ti­cals $GW­PH, which had less im­pres­sive – though pos­i­tive — late stage da­ta of its own last year. GW said that Epid­i­olex — a liq­uid ther­a­py us­ing pu­ri­fied cannaba­di­ol — trig­gered a mean re­duc­tion in con­vul­sive seizures of 39% among treat­ment-re­sis­tant pa­tients with Dravet syn­drome, com­pared to on­ly 13% among the place­bo group.

GW’s stock plunged 15% this morn­ing.

This is the last big stock cat­a­lyst ex­pect­ed in Q3, which has seen a se­ries of dra­mat­ic ups and downs in the biotech are­na.

“We are ex­treme­ly pleased with the top-line ef­fi­ca­cy and safe­ty re­sults from Study 1 which re­in­force the po­ten­tial of ZX008 to be an im­por­tant new treat­ment for seizure con­trol in chil­dren with Dravet syn­drome.  We look for­ward to pre­sent­ing fur­ther da­ta in fu­ture pub­li­ca­tions and at med­ical con­fer­ences,” said CEO Stephen Farr in a state­ment.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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