Thumbs Up. Thumbs Down: On rook­ie mis­takes, a class ac­tion suit and get­ting past fail­ure

 

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

Por­to­la makes a rook­ie mis­take, and gets ham­mered by a CRL

If any com­pa­ny looked to be sit­ting in the right place at the right time with a new drug ap­pli­ca­tion, it was Por­to­la. The FDA had al­ready giv­en the com­pa­ny a break­through des­ig­na­tion, of­fer­ing ready ac­cess that com­pa­ny ex­ecs say they took ad­van­tage of reg­u­lar­ly. But when it came down to the PDU­FA date, the biotech was forced to an­nounce that the agency had re­ject­ed the ap­pli­ca­tion. Why? In a call with an­a­lysts, Por­to­la’s crew sound­ed like they had fun­da­men­tal­ly mis­judged the FDA’s at­ti­tude on man­u­fac­tur­ing and da­ta is­sues. Points they thought could have been clar­i­fied af­ter an ap­proval couldn’t wait. And now it will spend some time an­swer­ing the FDA’s CRL rather than mar­ket­ing its an­ti-an­ti­co­ag­u­lant. This was a sim­ple rook­ie mis­take, but we’ve seen re­peat­ed re­jec­tions this year based on CMC is­sues. Oth­er de­vel­op­ers should take very care­ful note of it.

Some­times, a law­suit may be just what the doc­tor or­dered

Class ac­tion law­suits in the wake of a biotech set­back are a dime a dozen. They clus­ter like flies on an open wound, re­gard­less of what caused the wound. So why did I stop and write up a re­cent bit of lit­i­ga­tion about Juno?  Most­ly be­cause there are still some very im­por­tant unan­swered ques­tions about how the FDA han­dled the deaths of pa­tients from cere­bral ede­ma in Juno’s JCAR015 study. The first death went un­re­port­ed, though Juno ex­ecs talked it over with the agency. More deaths fol­lowed. And when the agency did drop its clin­i­cal hold, it on­ly last­ed for a few days. Juno’s ready ex­pla­na­tion, that adding flu­dara­bine to the reg­i­men for prep­ping pa­tients, was seem­ing­ly swal­lowed whole. The FDA, of course, re­fused to ex­plain their po­si­tion on this. But this is some­thing that needs to be care­ful­ly ex­am­ined and ex­plained. By all means, let Juno pro­ceed with its work. But leav­ing this one in the gray zone is ir­re­spon­si­ble. Maybe a law­suit will help clar­i­fy mat­ters.

Let’s set a high­er bar on bio­phar­ma takeover sto­ries

There was a time when the busi­ness press prid­ed it­self on a good takeover scoop. Se­cret bids. Board­room squab­bles. It made for great read­ing. Now, any old ru­mor will qual­i­fy for a sto­ry: An ex­ec­u­tive at com­pa­ny A asked an ex­ec­u­tive at com­pa­ny B about a pos­si­ble tie up? A bit of wa­ter cool­er chat­ter dur­ing a break? It’s all fod­der for the beast now, with ex­pres­sions of in­ter­est and ca­su­al over­tures, or just sim­ple ru­mors, trig­ger­ing sto­ries and fol­low-up sto­ries. Reuters and Bloomberg have good cause to hus­tle, with both look­ing to get the juicy stuff up on ter­mi­nals first. But there should be some kind of stan­dard on what qual­i­fies for a sto­ry. This is out of con­trol.

Some­times peo­ple fail in biotech. Get over it.

Con­grats to An­drew Hirsch for land­ing a new job as CFO at Agios. Hirsch was left to han­dle the un­wel­come task of putting Bind Ther­a­peu­tics in the ground af­ter it was forced in­to Chap­ter 11. The lead drug proved to be a dis­ap­point­ment. Debt had ac­cu­mu­lat­ed. And this is no time to look for in­vestors to bail you out of a tight spot. Biotech fail­ure is a re­al threat to every­one in this busi­ness, re­gard­less of the size of a com­pa­ny or its lev­el of ex­per­tise. Hirsch dealt with it and moved on. And that’s eas­i­er to do in the US than, say, the UK, where no one ever for­gets. Plus one for the US mod­el. These ex­pe­ri­enced biotech vets have made Boston and San Fran­cis­co in­to thriv­ing glob­al hubs. They’re need­ed to get new gen­er­a­tions of star­tups to the fin­ish line. Let the dead bury the dead. It’s time to move on.

Where are the deals?

Not to be a churl­ish ob­serv­er, but I have to ad­mit I’m more than a lit­tle dis­ap­point­ed by the slow pace of deal­mak­ing we’ve seen this year. There are some big out­fits that have been holed up for far too long. Big com­pa­nies re­ly on the biotech food chain to keep their pipelines in­ter­est­ing. And af­ter the big drop in biotech stocks, this is the time to move. I’m bet­ting that we get hit with a burst of deals in the fall. There’s plen­ty of cash be­hind that dam of de­sire. It’s time to put it in cir­cu­la­tion.

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.