AveX­is of­fers com­pelling rea­sons for a fast FDA OK for gene ther­a­py to treat spinal mus­cu­lar at­ro­phy in in­fants

For most in­fants suf­fer­ing from spinal mus­cu­lar at­ro­phy type 1, the chances are quite high that they could die or re­quire con­stant res­pi­ra­to­ry as­sis­tance ear­ly on to al­low them to keep breath­ing, with no mo­tor func­tion abil­i­ty for sim­ple things like sit­ting unas­sist­ed. Most die be­fore the age of 4.

But in a small Phase I study of 15 pa­tients treat­ed with AveX­is’ $AVXS gene ther­a­py for SMA, all of the pa­tients were able to reach 20 months with­out the need for con­stant res­pi­ra­to­ry sup­port, or dy­ing. And many achieved im­prove­ments in mo­tor symp­toms, speak­ing to the po­ten­tial — though still un­proven — of this as a once-and-done ther­a­py for a rare but dire ail­ment.

The New Eng­land Jour­nal of Med­i­cine pub­lished those com­plete Phase I da­ta points, out to­day, as the biotech pur­sues a piv­otal study while ex­plor­ing the pos­si­bil­i­ty of an ac­cel­er­at­ed FDA ap­proval that could — pos­si­bly — pro­vide a short­cut to com­mer­cial work.

Sean Nolan

The news to­day is re­mark­able for sev­er­al rea­sons. It puts AveX­is in the front ranks of gene ther­a­py com­pa­nies as Spark ap­pears poised to gain a pi­o­neer­ing US ap­proval for its lead gene ther­a­py for an eye dis­ease. And it al­so pos­es a di­rect threat to Bio­gen’s new fran­chise for Spin­raza, priced at $750,000 for the first year — one of the top 10 most ex­pen­sive ther­a­pies in the world — and half that rate for each year af­ter.

“We do have a meet­ing sched­uled late in the fourth quar­ter to meet with the FDA,” says Chica­go-based AveX­is CEO Sean Nolan. “We’ve said we would do every­thing we could to move along the fil­ing and hope­ful­ly the ap­proval. We do see there are a cou­ple of po­ten­tial sce­nar­ios that could lead to a more ac­cel­er­at­ed ap­proval.”

AveX­is an­nounced a month ago that it was launch­ing its piv­otal study of the ther­a­py.

SMA is trig­gered by a ge­net­ic de­fect that de­rails a pro­tein need­ed for mo­tor neu­rons. AveX­is’ ther­a­py is de­signed to pen­e­trate the blood brain bar­ri­er and in­tro­duce cor­rect­ed copies of that gene to fix the prob­lem in SMA1, an in­fan­tile form of the dis­ease that be­comes ap­par­ent in the first 6 months, mak­ing it a par­tic­u­lar­ly good tar­get for gene ther­a­py work.

Bri­an Kas­par

“These kids al­ways go down,” says AveX­is CSO Bri­an Kas­par, who took a leave from his post at the gene ther­a­py cen­ter at Na­tion­wide Chil­dren’s Hos­pi­tal in Colum­bus to spear­head the clin­i­cal work. “They nev­er gain mile­stones, no mo­tor func­tion mile­stones. They won’t sit unas­sist­ed; they’ll nev­er achieve the abil­i­ty to roll over or stand and walk.”

Giv­en its tar­get, and the less­er ef­fect that Spin­raza has demon­strat­ed in the clin­ic, reg­u­la­tors are like­ly to feel con­sid­er­able pres­sure to open the door ear­ly on this ther­a­py, par­tic­u­lar­ly giv­en the pre­ferred treat­ment that Sarep­ta gained for Ex­ondys51 with­out any ac­tu­al da­ta the it helped boys suf­fer­ing from Duchenne MD.

New FDA com­mis­sion­er Scott Got­tlieb has vowed to do what’s nec­es­sary to make cut­ting edge ther­a­pies avail­able as soon as they’ve cleared a bar on ef­fi­ca­cy and safe­ty. And he now has a test case for just where that line has been drawn.

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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A preda­tor's world? Top an­a­lyst sees the 'haves' and the 'haven't­s' di­verge as biotech bub­bles form — and col­lapse

Josh Schimmer

We’ve all seen the deluge of cash that’s been pouring into biotech from every angle: VCs, IPOs and follow-ons have generated billions in capital for new and emerging drug developers with ready access to some powerful new tech. But Evercore ISI’s Josh Schimmer is asking where we’re headed from here.

His answer is neither apocalyptic nor universally blissful, but if he’s right — and this is a discussion we’re hearing much, much more about at a time of growing economic and industry uncertainty — we may well be at a crossroads that could affect valuations, M&A and the entire global industry that has formed over the past 5 years.

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US mulls tar­iffs on Swiss drug ex­ports, weigh­ing on No­var­tis and Roche –  re­port

The leading Swiss newspaper has reported that the US is considering placing tariffs on pharmaceuticals from Switzerland. Roche and Novartis stock each fell 1% after the news broke.

Neue Zürcher Zeitung reported that US Trade Representative Robert Lighthizer told pharmaceutical representatives the Trump administration was considering the move. Tariffs do not appear to be in the immediate offing, but they would potentially affect Swiss giants Novartis and Roche along with other companies that manufacture in Switzerland, including Merck KGaA and US biotech Biogen, which is currently constructing a new facility in the country.

Neil Kumar, Endpoints

Bridge­Bio drops bid to re­claim Ei­dos af­ter di­rec­tors spurn 3 of­fers

A couple of months ago a newly public BridgeBio turned some heads by disclosing that it had made a bid for subsidiary Eidos Therapeutics in hopes of gobbling up the 34% stake that it doesn’t already own. Two offers later, the parties are calling it off.

A special committee of independent directors at the smaller biotech led by RA Capital’s Rajeev Shah and ex-Portola CEO William Lis first rejected the parent company’s initial offer — which would swap 1.3 BridgeBio shares for each Eidos share — on September 12. In the latest announcement, BridgeBio revealed that it eventually raised the offer to 1.5 shares and made $110 million available for all-cash or mixed consideration options, but Eidos still wasn’t interested.

Mark Foley, Revance

HR vi­o­la­tion push­es Re­vance co-founder out, vault­ing for­mer Zel­tiq chief to the helm

Months after Revance amended the terms of its Botox biosimilar collaboration with Mylan, the Newark, California-based drug developer disclosed its co-founder Dan Browne is stepping down, in what appears to be mysterious circumstances.

The company — which is also developing a rival to Allergan’s formidable Botox franchise — on Monday said Browne is departing “due to misjudgment in handling an employee matter,” that has also culminated in his resignation from Revance’s board of directors.

In-house FDA re­view flags a sus­pi­cious im­bal­ance in deaths as Sh­iono­gi hunts an OK for an­tibi­ot­ic

Shionogi has some big questions to answer if they plan to win an FDA panel’s backing for their new antibiotic.

While investigators have provided positive efficacy data for their new product to treat cases of complex urinary tract infections, an FDA review has flagged an imbalance of deaths between the antibiotic and a control arm. And they want the agency’s outside advisers to take a good hard look at that when they meet on Wednesday.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.