Servi­er ex­pands Tib­so­vo la­bel with first com­bi­na­tion OK in a rare form of AML

Just over a year af­ter Servi­er shelled out near­ly $2 bil­lion to pick up Agios’ on­col­o­gy port­fo­lio — in­clud­ing the acute myeloid leukemia drug Tib­so­vo — the French phar­ma has scored an ap­proval in com­bi­na­tion with a com­mon­ly used chemother­a­py.

Servi­er se­cured an ap­proval on Thurs­day for Tib­so­vo to treat cer­tain new­ly di­ag­nosed IDH1-mu­tat­ed AML pa­tients along­side azac­i­ti­dine, which is sold by Bris­tol My­ers Squibb un­der the brand name Vi­daza.

While AML is one of the most com­mon types of leukemia in adults, IDH1 mu­ta­tions are much rar­er, oc­cur­ring in on­ly 6 to 10% of all AML cas­es. Reg­u­la­tors cleared the Tib­so­vo com­bi­na­tion for new­ly di­ag­nosed pa­tients who are 75 years or old­er, or who have co­mor­bidi­ties that pre­clude the use of in­ten­sive in­duc­tion chemother­a­py.

Su­san Pandya

“Peo­ple liv­ing with acute myeloid leukemia, es­pe­cial­ly those who are new­ly di­ag­nosed and are not el­i­gi­ble for in­ten­sive chemother­a­py, have had few treat­ment op­tions,” Su­san Pandya, Servi­er’s VP of clin­i­cal de­vel­op­ment and head of can­cer me­tab­o­lism glob­al de­vel­op­ment in on­col­o­gy and im­muno-on­col­o­gy, said in a news re­lease.

Servi­er is al­so en­gag­ing with oth­er reg­u­la­to­ry au­thor­i­ties world­wide, she added, call­ing Thurs­day’s ap­proval a “ma­jor ad­vance­ment” in the US.

Tib­so­vo was first ap­proved as a monother­a­py back in 2018 to treat re­lapsed or re­frac­to­ry AML pa­tients with the IDH1 mu­ta­tion. The fol­low­ing year, it scooped up an­oth­er OK for new­ly di­ag­nosed IHD1 AML pa­tients who aren’t el­i­gi­ble for in­ten­sive chemother­a­py.

In part, be­cause so few pa­tients have AML with IDH1 mu­ta­tion, Agios strug­gled to turn Tib­so­vo in­to a com­mer­cial suc­cess, col­lect­ing on­ly about $30 mil­lion per quar­ter be­fore its sale to Servi­er.

In 2021 — months af­ter Servi­er swiped the on­col­o­gy busi­ness in a $1.8 bil­lion deal — Tib­so­vo won an­oth­er ap­proval in pre­vi­ous­ly treat­ed pa­tients with IDH1-mu­tat­ed cholan­gio­car­ci­no­ma.

Reg­u­la­tors based their Thurs­day de­ci­sion on da­ta from the Phase III AG­ILE study, in which pa­tients on the com­bo treat­ment saw sig­nif­i­cant im­prove­ments in event-free sur­vival (p=0.0038) and over­all sur­vival (p=0.001), ac­cord­ing to Servi­er. Pa­tients on Tib­so­vo and azac­i­ti­dine saw a three-fold im­prove­ment in me­di­an OS (24 months), com­pared to pa­tients who took on­ly azac­i­ti­dine (7.9 months), Servi­er re­port­ed.

The most com­mon side ef­fects were nau­sea, vom­it­ing, pro­longed elec­tro­car­dio­gram QT, in­som­nia, dif­fer­en­ti­a­tion syn­drome, leuko­cy­to­sis, hematoma, hy­per­ten­sion, arthral­gia, dys­p­nea, and headache.

This marks Vi­daza’s sec­ond ap­proval this week, af­ter not see­ing any la­bel ad­di­tions for more than a decade. On Mon­day, the FDA ap­proved the chemother­a­py for pe­di­atric pa­tients 1 month and old­er with new­ly di­ag­nosed ju­ve­nile myelomono­cyt­ic leukemia (JMML).

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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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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Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.