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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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