Bris­tol My­ers dusts off an old Cel­gene chemother­a­py for use in chil­dren with a rare blood can­cer

Bris­tol My­ers Squibb is bring­ing new life to a chemother­a­py drug from the old Cel­gene pipeline as it touts an­oth­er ap­proval in a rare form of blood can­cer af­fect­ing young chil­dren.

The FDA on Fri­day ap­proved Vi­daza (chem­i­cal­ly known as azac­i­ti­dine) for pe­di­atric pa­tients 1 month and old­er with new­ly di­ag­nosed ju­ve­nile myelomono­cyt­ic leukemia (JMML). The new in­di­ca­tion marks the first ap­proval for Vi­daza in more than a decade, though it’s com­mon­ly used to treat acute myeloid leukemia and myelodys­plas­tic syn­dromes (MDS).

JMML oc­curs when white blood cells called mono­cytes and mye­lo­cytes don’t ma­ture prop­er­ly, caus­ing bleed­ing and bruis­ing, joint pain (of­ten mis­tak­en for “grow­ing pains”), ane­mia and dif­fi­cul­ty breath­ing, among oth­er symp­toms. Most pa­tients are di­ag­nosed when they’re younger than 4 years old, ac­cord­ing to St. Jude Chil­dren’s Re­search Hos­pi­tal, though it’s ex­treme­ly rare — on­ly 1 or 2 cas­es out of a mil­lion chil­dren are re­port­ed each year.

BMS eval­u­at­ed Vi­daza in an open-la­bel study with 18 JMML pa­tients pri­or to hematopoi­et­ic stem cell trans­plan­ta­tion (HSCT). The pa­tients re­ceived the in­tra­venous drug dai­ly on days 1 through 7 of a 28-day cy­cle, which was re­peat­ed a min­i­mum of three times and a max­i­mum of six times.

Half of the chil­dren saw a con­firmed re­sponse to the drug, three of whom achieved clin­i­cal com­plete re­mis­sion and six of whom achieved par­tial re­mis­sion. The me­di­an time to re­sponse was 1.2 months, ac­cord­ing to the FDA. Near­ly all of the pa­tients (94%) re­ceived HSCT, the me­di­an time to which was 4.6 months.

The most com­mon side ef­fects were fever, rash, up­per res­pi­ra­to­ry tract in­fec­tion, and ane­mia.

Cel­gene ac­quired Vi­daza in its $2.9 bil­lion buy­out of Pharmion back in 2007. At its peak, the drug brought Cel­gene $600 mil­lion per year, be­fore it end­ed up in BMS’ pipeline in 2019. A year lat­er, BMS snagged a win for an oral ver­sion of the drug — known as Onureg — in adults with acute myeloid leukemia.

While that re­mains Onureg’s on­ly oth­er ap­proved in­di­ca­tion, the drug is in on­go­ing stud­ies for an­gioim­munoblas­tic T-cell lym­phoma and low­er-risk MDS, ac­cord­ing to BMS’ web­site.

Gilead ran in­to trou­ble ear­li­er this year when the FDA slammed the brakes on en­roll­ment in tri­als that com­bined the CD47 drug ma­grolimab with Vi­daza. The com­pa­ny said reg­u­la­tors flagged an “ap­par­ent im­bal­ance” in the sus­pect­ed un­ex­pect­ed se­ri­ous ad­verse re­ac­tions be­tween study arms.

A month lat­er, Gilead re­vealed that the agency had ex­pand­ed the hold to its dif­fuse large B-cell lym­phoma and mul­ti­ple myelo­ma stud­ies.

The FDA lift­ed its par­tial hold on stud­ies for ma­grolimab and azac­i­ti­dine in April, and Gilead said it’s on track for an in­ter­im read­out in 2023.

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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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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Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.

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.

Ankit Mahadevia, Spero CEO

Spero’s UTI can­di­date gets the CRL ham­mer as the com­pa­ny falls in­to pen­ny stock sta­tus

Spero Therapeutics has been struggling in the past few years, dealing with FDA holds and staff reductions amidst a rough biotech market, and the latest news from the Massachusetts-based company confirms what it anticipated in May: a CRL.

The company was slapped with the no-go for its NDA, the biotech disclosed Monday. The company was seeking approval for tebipenem HBr oral tablets, intended for the treatment of adult patients with complicated urinary tract infection, or cUTI, including pyelonephritis. The FDA had set a PDUFA date of June 27.

Bob Nelsen (Lyell)

As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In a statement, ARCH managing director and co-founder Bob Nelsen appeared to brush off concerns about the broader market troubles, alluding to the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels.

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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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(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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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.”