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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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