Zo­genix wins in­side track at FDA with child­hood epilep­sy ‘break­through’

Zo­genix has won the FDA’s break­through ther­a­py des­ig­na­tion fol­low­ing some im­pres­sive Phase III da­ta an­nounced last fall, gain­ing an in­side track at the agency for its close­ly watched Dravet syn­drome drug ZX008.

Gail Far­fel

A bit of a Wall Street dar­ling at the mo­ment, Zo­genix $ZGNX has watched its stock soar over 175% since it re­leased da­ta on the first of two Phase III stud­ies for this drug. The com­pa­ny re­port­ed ZX008, de­signed to tack­le a se­vere form of child­hood epilep­sy, trig­gered a dra­mat­ic drop in the rate of seizures ex­pe­ri­enced by pa­tients with Dravet syn­drome. At the high end, the dose cut the month­ly con­vul­sive seizure fre­quen­cy by 72.4% com­pared to a 17.4% drop in place­bo pa­tients.

“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,” wrote Leerink’s Paul Mat­teis in an in­vestor’s note at the time of the an­nounce­ment.

This new FDA des­ig­na­tion will speed de­vel­op­ment and re­view of the drug, like­ly set­ting Zo­genix up to com­pete with epilep­sy con­tender GW Phar­ma­ceu­ti­cals $GW­PH. GW re­cent­ly re­port­ed its drug Epid­i­olex showed no­table ef­fect against Dravet syn­drome, in which it trig­gered a 39% re­duc­tion in con­vul­sive seizures com­pared to a 13% place­bo group in late-stage tri­als.

“We are very pleased that the FDA has grant­ed break­through ther­a­py des­ig­na­tion based on the ef­fi­ca­cy and safe­ty re­sults from Study 1 re­port­ed in fall of 2017,” said Gail Far­fel, chief de­vel­op­ment of­fi­cer at Zo­genix, in a state­ment. “We look for­ward to work­ing close­ly with the FDA as we con­clude our Phase 3 clin­i­cal pro­gram in Dravet syn­drome, a rare and cat­a­stroph­ic form of child­hood epilep­sy.”

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Mer­ck makes a triple play on Covid-19: buy­ing out a vac­cine biotech, part­ner­ing on an­oth­er pro­gram and adding an an­tivi­ral to the mix

Merck is making a triple play in a sudden leap into the R&D campaign against Covid-19.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

The deal with IAVI covers recombinant vesicular stomatitis virus (rVSV) technology that is the basis for Merck’s successful Ebola Zaire virus vaccine. That’s going into the clinic later this year.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Andrew Hopkins, Exscientia founder and CEO (Exscientia)

Af­ter years of part­ner­ships, AI biotech Ex­sci­en­tia lands first ma­jor fi­nanc­ing round at $60M

After years racking up partnerships with biotechs and Big Pharma, the AI drug developer Exscientia has landed its first large financing round.

The UK-based company raised $60 million in a Series C round led by Novo Holdings — more than double the $26 million it garnered in a Series B 18 months ago. The round will help further the company’s expansion into the US and further what it calls, borrowing a term from the software world, its “full-stack capabilities,” i.e. its ability to develop drugs from the earliest stage to the market.

Covid-19 roundup: Janet Wood­cock steps aside — for now — as FDA drug czar; WHO hits the brakes on hy­droxy study af­ter lat­est safe­ty alarm

The biopharma industry will soon get a look at what the FDA will look like once CDER’s powerful chief Janet Woodcock retires from her post.

Long considered one of the most influential regulators in the agency, if not its single most powerful official when it counts, Woodcock is being detached to devote herself full-time to the White House’s special project to fast-forward new drugs and vaccines for the pandemic. The move comes a week after some quick reshuffling as Woodcock and CBER chief Peter Marks joined Operation Warp Speed. Initially they opted to recuse themselves from any FDA decisions on pandemic treatments and vaccines, after consumer advocates criticized the move as a clear conflict of interest in how the agency exercises oversight on new approvals.

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Piv­otal myas­the­nia gravis da­ta from ar­genx au­gur well for FcRn in­hibitors in de­vel­op­ment

Leading the pack of biotechs vying for a piece of the generalized myasthenia gravis (gMG) market with an FcRn inhibitor, argenx on Tuesday unveiled keenly anticipated positive late-stage data on its lead asset, bringing it one step closer to regulatory approval.

Despite steroids, immunosuppressants, acetylcholinesterase inhibitors, and Alexion’s Soliris, patients with the rare, chronic neuromuscular disorder (more than 100,000 in the United States and Europe) don’t necessarily benefit from these existing options, leaving room for the crop of FcRn inhibitors in development.

Af­ter de­cou­pling from Re­gen­eron, Sanofi says it’s time to sell the $13B stake picked up in the mar­riage

With Regeneron shares going for a peak price — after doubling from last fall — Sanofi is putting a $13 billion stake in their longtime partner on the auction block. And Regeneron is taking $5 billion of that action for themselves.

Sanofi — which has been decoupling from Regeneron for more than a year now — bought in big in early 2013, back when Regeneron’s stock was going for around $165 a share. Small investors flocked to the deal, buzzing about an imminent takeover. The buyout chatter wound down long ago.

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Janet Woodcock, director of the Center for Drug Evaluation and Research (AP Images)

Covid-19 roundup: Hit with new con­flict ac­cu­sa­tions, Janet Wood­cock steps out of the agen­cy's Covid-19 chain of com­mand

Two weeks ago, FDA drug chieftain Janet Woodcock was assuring a top Wall Street analyst that any vaccine approved for combating Covid-19 would have to meet high agency standards on safety and efficacy before it’s approved. But over the weekend, after she and Peter Marks took top positions with the public-private operation meant to speed a new vaccine to lightning-fast approvals — they both recused themselves from the review process after an advocacy group argued their roles close to the White House could pose a conflict of interest.

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FDA ap­proves the first gener­ic for Amar­in's Vas­cepa — but is a fish oil price war im­mi­nent?

Late last year, enthusiasm for Amarin’s fish-oil pill Vascepa burgeoned when the FDA signed off on expanding the cholesterol fighter’s label to include the drug’s beneficial impact on cardiovascular risk, but months later the exuberance for the blockbuster-to-be took a big hit when a judge invalidated key patents protecting Vascepa.

Despite Amarin’s $AMRN pledge to appeal — a process that could take months — the ruling opened the door for generic competition. Hikma Pharmaceuticals, one of three challengers in the Nevada suit, on Friday said that its generic copy of pure EPA, the omega-3 fatty acid that constitutes Vascepa, has been approved by the FDA.

Eric Edwards, Phlow president and CEO (PR Newswire)

BAR­DA of­fers a tiny start­up up to $812M to cre­ate a US-based drug man­u­fac­tur­er — and the CEO comes with a price goug­ing con­tro­ver­sy on his ré­sumé

BARDA has tapped a largely unknown startup to ramp up production of a list of drugs that may be at risk of running short in the US. And the deal, which comes with up to $812 million in federal funds, was inked by a CEO who found himself in the middle of an ugly price gouging controversy a few years ago.

The feds’ new partner — called Phlow — won a 4-year “base” contract of $354 million, with another $458 million that’s on the table in potential options to sustain the outfit. That would make it one of the largest awards in BARDA’s history.

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