Puma's fresh shin­er, what the biotech IPO surge tells us, and the trou­ble with can­cer R&D

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

Puma gets an­oth­er shin­er as BioTwit­ter chews on an­oth­er morsel of in­for­ma­tion

One thing you don’t want to see in the fi­nal few days lead­ing up to an FDA pan­el re­view of your big drug is trou­ble on the reg­u­la­to­ry team. This, af­ter all, is where the rub­ber hits the road for reg­u­la­to­ry as they steer the ap­pli­ca­tion to­ward a group of peo­ple at the FDA and their cho­sen ad­vis­ers who will of­fer their ex­pert opin­ions on what you’ve been hop­ing to sell for bad­ly need­ed rev­enue.

Point­ed ques­tions will be raised. Point­ed an­swers need to be sup­plied. And the reg­u­la­to­ry head is sup­posed to be your guide for what’s ahead, help­ing shore up your ar­gu­ment in the com­pa­ny’s fa­vor. They may not do a lot of talk­ing in pub­lic, or qual­i­fy as a top biotech ex­ec, but strat­e­gy and prep from your reg­u­la­to­ry li­ai­son is key to a good pan­el pre­sen­ta­tion and de­fense.

So when Puma Biotech­nol­o­gy $PBYI briefly not­ed on Thurs­day that their reg­u­la­to­ry head — Se­nior VP Robert Char­nas — had de­cid­ed to leave the com­pa­ny “for health rea­sons” in 11 days, 9 days ahead of their pan­el re­view, BioTwit­ter swung in­to ac­tion and be­gan to of­fer a va­ri­ety of con­spir­a­cy the­o­ries to fill the in­for­ma­tion gap.

Did he, per­haps, know that Puma had al­ready been tor­pe­doed in the FDA’s drug re­view?

It sound­ed bad, and Puma’s stock, which was trad­ing at $200 a share a year ago, took an­oth­er hit, cost­ing it about 10% of its share price. And this af­ter the stock closed at $36.55.

It didn’t help that the rea­son cit­ed in their 8-K for Char­nas’ de­par­ture may have been a lit­tle af­ter-the-fact. Cred­it Su­isse says they talked to CEO Alan Auer­bach, and he told an­a­lysts that there was some fric­tion be­tween the reg­u­la­to­ry chief and the team at Puma. The CEO sug­gest­ed a month and a half ago that Char­nas start con­sid­er­ing what he’d like to do next with his ca­reer, and the health rea­son was cit­ed in the reg­u­la­to­ry chief’s res­ig­na­tion last Fri­day. That all hap­pened be­fore he could have seen the FDA’s docs.

So case closed?

Not a chance. There are oth­er an­a­lysts notes and more con­jec­ture. The de­bate over Puma’s chances at the FDA — with a drug that has been both praised and pum­meled for ef­fi­ca­cy as well as some se­vere side ef­fects — is just reach­ing red-hot tem­per­a­tures as a sure-fire cat­a­lyst ap­proach­es. So it’s a nat­ur­al for Twit­ter, where small in­vestors flock for tips and cues. Mon­ey will be made and lost in a war of words where mo­ti­va­tion and iden­ti­ty are the first ca­su­al­ties in the fog of blog war.

It’s not ide­al, but it is the new re­al­i­ty. For biotech CEOs look­ing to go pub­lic, that’s some­thing you’ll want to keep front of mind.

Biotech IPOs flare again, and the bright light spot­lights a new com­pa­ny-build­ing strat­e­gy

Speak­ing of biotech stocks.

Bio­haven got its pipeline the new fash­ioned way. It found a promis­ing ther­a­py on the shelf at Bris­tol-My­ers Squibb, bagged it for a small amount and put it at the top of the pipeline. Hes­to presto and you have a com­pa­ny ready to go pub­lic, Ax­o­vant style.

That’s a lit­tle flip, to be sure, but not too ex­treme. Any­way, what makes it im­por­tant is that Bio­haven pulled off an up­sized $168 mil­lion IPO over the range at $17 a share this week. That’s not some­thing you see very of­ten in biotech cir­cles, where the biotech boom of 2014 is now some­thing of a dis­tant echo. Add that with an up­sized IPO for Uro­Gen and a sol­id score by Ovid, and we have the mak­ings of a biotech IPO resur­gence — at least for a brief pe­ri­od.

I’m not a stock pick­er and there are plen­ty of peo­ple who are bet­ter at sort­ing out these trends than I am, but a mar­ket that’s ready to em­brace biotech again — even as in­sid­ers shore up the price and prob­lems abound with the ul­tra risk in­volved in drug de­vel­op­ment — would prove help­ful to keep­ing the food chain tuned up.

What you shouldn’t over­look in this is the role big­ger play­ers in bio­phar­ma have in set­ting up this next wave of biotech com­pa­nies. And not just at Bio­haven. Ovid got its lead in a cre­ative 50/50 deal with Take­da, which is wide open to new ideas and re­la­tion­ships. In a re­lat­ed move, Ma­gen­ta, which brought its ven­ture tal­ly to $98.5 mil­lion this week, gained a lead drug from their con­tacts at No­var­tis.

So as the big or­ga­ni­za­tions re­or­ga­nize and reeval­u­ate, new op­por­tu­ni­ties are be­ing made for new com­pa­nies.

These IPO trends can turn on a dime. So let’s say con­grats to the win­ners and keep an eye on this. It’s been a good week. Pick­ing up R&D as­sets like this may tur­bo charge a com­pa­ny’s growth. But not all of these re­dis­cov­ered di­a­monds in the rough will sparkle in Phase III.

Can­cer is the king of all pipeline strate­gies — so be care­ful

Con­grats to As­traZeneca for their ap­proval of dur­val­um­ab this week. Yes, it was ex­pect­ed. Yes, they were fifth to the par­ty with the third OK for blad­der can­cer. No, that’s not go­ing to win any prizes in R&D work, par­tic­u­lar­ly for a phar­ma gi­ant that faces mul­ti­ple pipeline is­sues at a time it’s still strug­gling to mount a turn­around.

As­traZeneca, though, has scored some im­por­tant wins in can­cer un­der CEO Pas­cal So­ri­ot and this tro­phy can be added to the block­buster show­case.

As­traZeneca CEO Pas­cal So­ri­ot

But there are some big­ger trends at work here that de­serve some at­ten­tion.

It’s clear from the lat­est fore­cast from Quin­tiles­IMS that can­cer is go­ing to be the big fo­cus in bio­phar­ma R&D over the next five years. As­traZeneca went af­ter check­points be­cause the bi­ol­o­gy is well un­der­stood and you can make some mon­ey here. But their late ar­rival al­so un­der­scores just how ex­tra­or­di­nar­i­ly com­pet­i­tive this field can be­come al­most overnight. Mon­ey is be­ing poured in­to a whole pipeline of PD-1 and PD-L1 check­points. In the near fu­ture you’ll see ap­provals tar­get­ing par­tic­u­lar coun­tries, like Chi­na. Com­pa­nies with a can­cer vac­cine or a next-gen CT­LA-4, re­lat­ed check­points, etc., will want their own check­point. Like In­cyte.

And why not? We know how to make these ther­a­pies.

The FDA, which has helped ush­er in a rev­o­lu­tion in can­cer R&D by en­cour­ag­ing ac­cel­er­at­ed ap­pli­ca­tions, will have lit­tle prob­lem re­view­ing and ap­prov­ing these new meds.

Pay­ers aren’t blind to this. Com­pe­ti­tion will force prices down with for­mu­la­ries as the weapon of choice. Biotech’s role here is to look be­yond where drugs are now and on to the new cock­tails of the fu­ture, like Cy­tomX is do­ing. And out of all the churn­ing, there will be a few win­ners and many losers.

This is not work for the weak of heart; win­ning isn’t al­ways go­ing to be enough to be suc­cess­ful.

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.

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.

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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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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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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Re­searchers de­fine ex­act­ly what they saw in the first pos­i­tive remde­sivir study for Covid-19. But what's that worth to Gilead?

Remdesivir can work in fighting Covid-19, particularly for patients with less severe cases, but this is just a first step in the journey to finding combos that can do the job much better.

That’s the bottom line from Gilead’s randomized study published in the New England Journal of Medicine. Analysts were quick to draw conclusions about how the big biotech could turn this into a profitable advantage — with widespread expectation of considerable pricing restraint on Gilead’s part. Anyone looking for a new mountain of cash to count as the world grapples with the pandemic is likely to come away disappointed.

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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