In the wake of deep R&D cuts, Bay­er flags safe­ty con­cerns for a top PhI­II drug

Less than a week af­ter Bay­er an­nounced plans to carve down its in­ter­nal R&D group, lay­ing off 900 R&D staffers, the Ger­man com­pa­ny has tipped the mar­ket that one of its cru­cial late-stage de­vel­op­ment ef­forts has run in­to se­ri­ous trou­ble.

Jo­erg Moeller

R&D chief Jo­erg Moeller, who gath­ered the reins in re­search af­ter the de­par­ture of An­dreas Busch in late 2017, told an in­vest­ment group in Lon­don Mon­day that the com­pa­ny was halt­ing its de­vel­op­ment work on vi­laprisan, a uter­ine fi­broid drug that has been billed as a top block­buster prospect at the com­pa­ny.

“For vi­laprisan we have just some days ago put clin­i­cal de­vel­op­ment of our on­go­ing tri­als on hold,” he not­ed, ac­cord­ing to a re­port in Reuters. “That is due to very re­cent safe­ty find­ings in long-term tox­i­col­o­gy stud­ies … We have there­fore de­cid­ed as a pre­cau­tion­ary mea­sure to stop en­roll­ment in­to our on­go­ing pro­gram and eval­u­ate the da­ta.”

I queried Bay­er about the is­sue, and got this back mid-morn­ing:

We re­cent­ly ob­served safe­ty sig­nals in pre-clin­i­cal tox­i­co­log­i­cal stud­ies in ro­dents in­ves­ti­gat­ing long term safe­ty of vi­laprisan – a drug can­di­date we de­vel­op for the treat­ment of uter­ine fi­broids and en­dometrio­sis.

Al­though these are pre­lim­i­nary pre-clin­i­cal find­ings that have not been ob­served in oth­er vi­laprisan stud­ies we de­cid­ed as a pre­cau­tion­ary mea­sure to pause re­cruit­ment in the on­go­ing clin­i­cal stud­ies with vi­laprisan un­til a thor­ough analy­sis of the find­ings has been achieved.

No new pa­tients will be en­rolled in the pro­gram. Pa­tients cur­rent­ly on treat­ment will con­tin­ue their on­go­ing treat­ment course ac­cord­ing to study pro­to­col un­til fur­ther no­tice from Bay­er.

Bay­er went on to note that they had alert­ed the prop­er au­thor­i­ties and will mon­i­tor pa­tients for safe­ty is­sues.

This isn’t the first uter­ine fi­broid ef­fort to raise alarms.

The set­back oc­curs as Al­ler­gan is still deal­ing with the FDA’s re­jec­tion of its uter­ine fi­broid drug Es­mya af­ter Eu­ro­pean reg­u­la­tors cit­ed it for trig­ger­ing a rare liv­er dis­ease. The EMA ear­li­er this year put re­stric­tions on the use of the drug to pro­tect women on the ther­a­py.

Ab­b­Vie, mean­while, has been rolling out Orilis­sa af­ter that drug was first ap­proved for en­dometrio­sis last Ju­ly. The ther­a­py has passed muster for uter­ine fi­broids in Phase III as well, rais­ing hopes that Ab­b­Vie can gain sig­nif­i­cant rev­enue from this drug as they ex­pand the mar­ket.

Bay­er, mean­while, is tran­si­tion­ing its R&D op­er­a­tions as it works out a glob­al re­struc­tur­ing, with plans to cut back on what it spends on in­ter­nal re­search in fa­vor of more al­liances with out­side bio­phar­ma com­pa­nies. A cou­ple of years ago, though, they in­clud­ed vi­laprisan among a group of drugs they tout­ed as like­ly block­busters able to earn more than $6 bil­lion in peak sales.

What hap­pened with the rest?

Their PI3k can­cer drug Aliqopa was first ap­proved a year ago, go­ing in­to a third-line set­ting against heavy com­pe­ti­tion. They have a high-risk heart drug called veri­ciguat, ane­tum­ab rav­tan­sine failed a mid-stage study for mesothe­lioma and no longer fea­tures on the late-stage end of the pipeline re­port and finerenone is in Phase III for kid­ney dis­ease, along­side daro­lu­tamide for can­cer. Daro­lu­tamide hit its goal in Phase III for non-metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer a cou­ple of months ago, though they held back all the da­ta for a lat­er con­fer­ence.

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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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.

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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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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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.

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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