Thumbs Up/Thumbs Down: The FDA backs an­oth­er R&D scheme, Kite shows its speed while Eli Lil­ly and As­tra stum­ble again

 

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

An­oth­er price-goug­ing alert for the FDA

Let’s face it. Bio­phar­ma has a bad rep. New drugs come along with jaw-drop­ping prices and the pub­lic feels like it’s get­ting fleeced. The prob­lem, though, has noth­ing to do with tru­ly in­no­v­a­tive new drugs or their price. The US can well af­ford the high cost of in­no­va­tion – with all its ben­e­fits. It’s be­ing tak­en to the clean­ers for the ever ris­ing price of old meds that needs to be the fo­cus. This week’s sto­ry on Marathon’s move to gain an ap­proval for an an­cient steroid called de­flaza­cort sums it all up. Nev­er ap­proved in the U.S., Marathon test­ed it as a mus­cle strength­en­er for Duchenne mus­cu­lar dy­s­tro­phy. And they have scored a slew of ad­van­tages at the FDA, in­clud­ing its se­lec­tion as a rare pe­di­atric ther­a­py. You can be sure that Marathon plans to charge much more than the buck-a-pill price charged by on­line phar­ma­cies in Cana­da. The agency should re­serve its fa­vors for re­al­ly great new drugs. And the in­dus­try needs to learn how to shape an ar­gu­ment that can ac­tu­al­ly win the pub­lic over.

Kite Phar­ma takes the CAR-T lead.

A week can make a huge dif­fer­ence in biotech. Or six days. That’s how long Juno’s JCAR015 was on hold, and it end­ed up push­ing back its launch plans from 2017 to 2018. Kite, rac­ing ahead on man­u­fac­tur­ing op­er­a­tions, says it could be ready to go lat­er this year, af­ter an­nounc­ing an ag­gres­sive plan for a 30-day re­view of the first 50 pa­tients en­rolled in its piv­otal tri­al. Rush­ing ahead can in­crease the risk of a bad stum­ble, but Kite ap­pears con­fi­dent that it can quick­ly cap­i­tal­ize on its break­through rep and score a pi­o­neer­ing ap­proval well be­fore its ri­vals show up. That’s a bold move.

Eli Lil­ly’s lat­est pipeline stum­ble

The phar­ma gi­ant has sur­vived its trek through a leg­endary R&D drought, com­ing up with some im­por­tant late-stage ef­forts that de­serve a re­assess­ment of Lil­ly’s prospects. But when its CDK 4/6 drug abe­maci­clib failed to hit pay­dirt at the in­ter­im analy­sis, this drug lost a con­sid­er­able amount of sheen. Pfiz­er got to the mar­ket first with an ear­ly win for Ibrance. No­var­tis looks to be right on track af­ter end­ing its ri­val piv­otal study ear­ly. And now Lil­ly won’t be able to file be­fore next year, un­able to de­clare a sim­i­lar win. For long­time ob­servers, it’s an­oth­er sign of the slows at a com­pa­ny that needs bad­ly to move fast.

Here’s a thumbs-up to a clear mes­sage mak­er.

Al­ler­gan’s Brent Saun­ders went right back to singing the same tune about deals once the merg­er with Pfiz­er fell through. He’s go­ing to stay fo­cused on key ar­eas, snap up pro­grams prefer­ably af­ter Phase II and (with­out hit­ting this too hard) stay­ing away from dis­cov­ery work and most of the nit­ty-grit­ty in Phase I. He likes to buy things, rather than rent-to-own, and he’s think­ing high deal vol­ume, not one big, knock­out M&A punch. Those ru­mors about buy­ing Bio­gen? Not go­ing to hap­pen. Oh, and he has lots of cash for the right kinds of deals. Got it? OK then.

As­traZeneca now 0 and 2 on selume­tinib

The UK com­pa­ny’s stock has en­joyed a re­nais­sance re­cent­ly af­ter some ru­mors about a pos­si­ble No­var­tis takeover be­gan to cir­cu­late. But this com­pa­ny has a his­to­ry of mis­steps in the pipeline that con­tin­ues to dog its R&D rep. This week, its can­cer drug selume­tinib raised its head for the sec­ond time as a Phase III los­er. And that, once again, raised Pas­cal So­ri­ot’s pre­dic­tion that this drug is a block­buster in the mak­ing. So­ri­ot has been wrong about a lot of things in the pipeline, with the ex­cep­tion of Tagris­so. Chances are that this drug will ei­ther be cut loose or sold for scrap, like bro­dalum­ab and lesin­u­rad.

The End­points 100

For­give us our unashamed self-pro­mo­tions. We’re halfway through the re­cruit­ment process for our new, on­go­ing quar­ter­ly con­fi­dence sur­vey of the biotech in­dus­try, bring­ing to­geth­er 100 top in­dus­try ex­ecs to track hir­ing trends, ac­cess to cap­i­tal, the reg­u­la­to­ry en­vi­ron­ment and more. Cov­er­ing this in­dus­try can’t re­ly sole­ly on re­port­ing the news as it hap­pens. We aim to keep our thumb on the pulse.

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.