Scott Got­tlieb will push to pub­lish CRLs; Marathon's demise of­fers the in­dus­try a cau­tion­ary tale

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


Scott Got­tlieb did the right thing of­fer­ing to pub­lish those CRLs

Dur­ing Scott Got­tlieb’s last round of writ­ten an­swers to the Sen­ate com­mit­tee re­view­ing his nom­i­na­tion as FDA com­mis­sion­er, I was pleased to see him vol­un­tar­i­ly pro­pose that the agency start pub­lish­ing com­plete re­sponse let­ters. Got­tlieb has done that be­fore, but so far we haven’t seen a recom­mit af­ter his nom­i­na­tion.

Why is this so im­por­tant?

We know that there have been a num­ber of in­stances when drug com­pa­nies re­ly on the agency’s si­lence to put a PR spin on their CRLs. Or they just don’t say any­thing at all, which is just as com­mon.

Frankly, af­ter com­pa­nies have had a chance to talk up their prospects — of­ten for years — for drugs on­ly to see them turned back at the fin­ish line with­out any hon­est ac­count­ing for the re­jec­tion is un­ac­cept­able. Physi­cians, pa­tients, in­vestors, tri­al sub­jects (and, yes, jour­nal­ists) have a right to find out what trig­gers a reg­u­la­to­ry boo­by trap.

No one forced Got­tlieb’s arm on this. There was no spe­cif­ic query on CRLs. He pro­posed it, with a caveat on redact­ing pro­pri­etary in­fo, which is the right thing to do — one rea­son why we’ve been sup­port­ive of his nom­i­na­tion. It’s al­so a prac­ti­cal way to dis­tance him­self from ac­cu­sa­tions from some De­moc­rats his past busi­ness ties leave him hope­less­ly con­flict­ed.

The com­mit­tee vote send­ing the nom­i­na­tion to the Sen­ate floor will hope­ful­ly be act­ed on in the next cou­ple of weeks. The soon­er the bet­ter. It’s time to get start­ed on the next chap­ter on the FDA and bio­phar­ma.


As PTC preps a con­tro­ver­sial launch of de­flaza­cort, Marathon is wind­ing down

In a Sun­day news scoop, we re­vealed that Marathon Phar­ma­ceu­ti­cals is be­ing wound down now that PTC has ac­quired US rights to its steroid de­flaza­cort.

These days, get­ting held up as an ex­am­ple of ram­pant price goug­ing can cre­ate an en­vi­ron­ment that is too tox­ic to deal with.

The next step in this process will be to see how much of the tox­i­c­i­ty is ab­sorbed by PTC, which bought the drug and has the un­en­vi­able task now to try and come up with an ac­cept­able price af­ter Marathon failed spec­tac­u­lar­ly at $89,000 a year. For par­ents who had been buy­ing it from the UK’s Mas­ters at a lit­tle more than $1,000, that was a shock. How PTC hopes to make cash now is hard to un­der­stand.

You can be sure that PTC had a firm price in mind when they bought this drug. But it ap­pears we’ll have to wait in­to ear­ly May to get a look at the stick­er.  We’ll be wait­ing.

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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