Pre­emp­tive sham­ing as­sault on No­var­tis’ CAR-T pi­o­neer un­der­scores a dis­con­nect on R&D costs

It’s no se­cret that tax­pay­ers fund a lot of the ba­sic sci­ence work done in the US. But should that ear­ly re­search sup­port for new drugs trans­late in­to low­er prices?

A group called Pa­tients for Af­ford­able Drugs thinks so, launch­ing a pre­emp­tive strike against No­var­tis’ CAR-T drug CTL019— now up for an ap­proval — de­mand­ing fair pric­ing in light of the $200 mil­lion-plus that was used to back the trans­la­tion­al work on CAR-T in gen­er­al. There’s no break­down on what slice of that helped No­var­tis.

David Mitchell, Pa­tients for Af­ford­able Drugs

“I urge you in the strongest pos­si­ble terms to price your CAR-T drug fair­ly in light of the fact that U.S. tax­pay­ers in­vest­ed hun­dreds of mil­lions of dol­lars to de­vel­op CAR-T be­fore your com­pa­ny be­came se­ri­ous­ly in­volved,” David Mitchell, pres­i­dent of Pa­tients For Af­ford­able Drugs, wrote in the let­ter to No­var­tis chief Joe Jimenez.

That com­ment de­serves some added con­text.

No­var­tis spent close to $9 bil­lion on R&D last year, about 18% of rev­enue. The phar­ma gi­ant spent more than $9 bil­lion on R&D the year be­fore that, and it will do the same in 2017. While it will like­ly pub­licly han­dle this gam­bit on pric­ing with its usu­al wood­en (and very cor­po­rate) lack of feel­ing, it has no rea­son to apol­o­gize for what it charges for its CAR-T.

At one point the com­pa­ny had a large, 400-em­ploy­ee group de­vot­ed to cell and gene ther­a­py and a blank-check ap­proach to CAR-T from Jimenez that in­clud­ed huge sup­port for the Uni­ver­si­ty of Penn­syl­va­nia. That strat­e­gy was scrapped, but No­var­tis paid dear­ly to stay a pi­o­neer in this per­son­al­ized med­i­cine field, as Kite and Juno took their own in­de­pen­dent ap­proach in dri­ving de­vel­op­ment.

No­var­tis CEO Joe Jimenez

The first CAR-Ts won’t be cheap by any stan­dard. Rev­o­lu­tions in drug sci­ence don’t come cheap. But while No­var­tis and Kite and Juno and the oth­ers owe a debt to fed­er­al­ly spon­sored R&D in the field, the ini­tial amounts pro­vid­ed by the feds are on­ly a small part of the to­tal.

The pub­lic doesn’t gen­er­al­ly rec­og­nize that. But they should if we want to dis­cuss drug pric­ing se­ri­ous­ly. There are plen­ty of big is­sues sur­round­ing pric­ing and rein­ing in costs that de­serve re­al de­bate. But this is the wrong ap­proach on R&D.

The NIH is be­com­ing more di­rect­ly in­volved in drug de­vel­op­ment than ever be­fore. That’s ev­i­dent in two oth­er sto­ries in to­day’s edi­tion, from Thomas Wynn’s de­ci­sion to move from NIH to Pfiz­er and the new work on TCR en­gi­neer­ing re­lat­ed to Kite. That move needs to be fos­tered, if we’re se­ri­ous about get­ting new drugs to pa­tients.

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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