A fa­ther's plea: The FDA needs to make sure that Juno's lethal dis­as­ter is­n't re­peat­ed

The FDA nev­er has pub­licly ex­plained just why it de­cid­ed to lift a clin­i­cal hold on Juno’s lead CAR-T drug JCAR015 just days af­ter the stun­ning news that the tri­al was be­ing halt­ed af­ter three pa­tients tak­ing the cell ther­a­py died of a cere­bral ede­ma. But then, it nev­er does, cit­ing se­cre­cy rules that fre­quent­ly keep reg­u­la­tors silent.

Now the fa­ther of one of those vic­tims has joined in a pe­ti­tion from the Cen­ter for Re­spon­si­ble Sci­ence seek­ing some re­al trans­paren­cy at the FDA on the way it han­dles pa­tient deaths — with ma­jor im­pli­ca­tions for the peo­ple who par­tic­i­pate in clin­i­cal tri­als.

Al­most a year af­ter Michael Vokhgelt’s 24-year-old son Max died in Juno’s ROCK­ET study, and five months af­ter telling STAT that “he died for greed,” Vokhgelt is still look­ing for an­swers. Max didn’t die from leukemia, says his fa­ther. He was killed by the drug, which has since been scrapped by Juno.

Not on­ly did Juno not an­nounce Max Vokhgelt’s death, a week lat­er it put out a re­lease cit­ing the “en­cour­ag­ing” and “im­pres­sive” re­sults it was see­ing.

In the fa­ther’s words:

It wasn’t un­til two more tri­al vol­un­teers died and FDA is­sued a clin­i­cal hold that on Ju­ly 7, 2016, Juno an­nounced the deaths. Juno blamed the deaths on flu­dara­bine, a chemother­a­py pre­con­di­tion­ing treat­ment in con­junc­tion with the CAR-T ther­a­py. FDA ac­cept­ed Juno’s ex­pla­na­tion and al­lowed Juno to re­sume the tri­al with­out Flu­dara­bine. I was dev­as­tat­ed when I learned that two more tri­al par­tic­i­pants died in No­vem­ber from cere­bral ede­ma. How can this hap­pen? I don’t even know the ra­tio­nale be­hind FDA’s de­ci­sion to lift the clin­i­cal hold af­ter my son and two oth­ers died, be­cause that is con­sid­ered “pro­pri­etary”.

I have read the Cit­i­zen Pe­ti­tion from the Cen­ter for Re­spon­si­ble Sci­ence and sup­port the re­quest­ed reg­u­la­to­ry amend­ments to al­low for the use of the pre­clin­i­cal test that is best to pre­dict what will hap­pen to clin­i­cal tri­al par­tic­i­pants. If tra­di­tion­al tests don’t al­ways pre­dict dead­ly tox­i­c­i­ties, drug spon­sors must be al­lowed to use more pre­dic­tive tests that bet­ter pre­dict what hap­pens in hu­mans.

It is FDA’s re­spon­si­bil­i­ty to pro­tect hu­man health and pro­tect the pub­lic from dan­ger­ous drugs. To achieve that man­date, all avail­able tools to pre­dict safe­ty must be used. I don’t want an­oth­er fam­i­ly to go through what my fam­i­ly went through.

In a let­ter sent last April, and first re­port­ed by RAPS’ Zachary Bren­nan, the Cen­ter for Re­spon­si­ble Sci­ence not­ed 19 treat­ment-re­lat­ed deaths in clin­i­cal tri­als from Ju­ly 2016 through April 2017.  Three of those were in a study of a ri­val CAR-T by Kite, which re­cent­ly was hit with the death of its first pa­tient from a case of cere­bral ede­ma.

The CRS wants things to change be­fore more pa­tients die.

“We ar­gue that every­thing that can be done to pro­tect clin­i­cal tri­al vol­un­teers must be done. Rather than re­sume a tri­al with­out know­ing the ac­tu­al cause of the dead­ly tox­i­c­i­ty, spon­sors should have made the drug avail­able so that it could be test­ed in a hu­man-rel­e­vant plat­form.”

The FDA says it’s still un­der re­view. Let’s hope they do bet­ter by tri­al vol­un­teers.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.

Bristol Myers Squibb (Alamy)

CVS re­sumes cov­er­age of block­buster blood thin­ner af­ter price drop fol­lows Jan­u­ary ex­clu­sion

Following some backlash from the American College of Cardiology and patients, Bristol Myers Squibb and Pfizer lowered the price of their blockbuster blood thinner Eliquis, thus ensuring that CVS Caremark would cover the drug after 6 months of it being off the major PBM’s formulary.

“Because we secured lower net costs for patients from negotiations with the drug manufacturer, Eliquis will be added back to our template formularies for the commercial segment effective July 1, 2022, and patient choices will be expanded,” CVS Health said in an emailed statement. “Anti-coagulant therapies are among the non-specialty products where we are seeing the fastest cost increases from drug manufacturers and we will continue to push back on unwarranted price increases.”

#Can­nes­Lions2022: Con­sumer health ex­ecs call on agen­cies to in­volve pa­tients in cre­ative process

CANNES — When Tamara Rogers joined GSK back in 2018, “science was king and R&D were the gods.” Now the global chief marketing officer of consumer healthcare wants to make room for another supreme being: the consumer.

As health and wellness becomes more relevant to consumers amid the pandemic, four health-focused executives called on marketers to involve patients in their creative process in a panel discussion at the Cannes Lions advertising creativity festival.

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