FDA pos­es some tough ques­tions for GSK’s pitch to gain an ap­proval for Nu­cala in COPD

Glax­o­SmithK­line faces a steep, up­hill climb on Wednes­day in its quest to gain an ap­proval for Nu­cala (mepolizum­ab) as a ther­a­py for COPD.

Al­ready ap­proved in asth­ma, top reg­u­la­tors at the FDA laid bare some big prob­lems with the phar­ma gi­ant’s ap­pli­ca­tion on COPD. And they plan to chal­lenge a pan­el of out­side ex­perts on the com­pa­ny’s as­ser­tions that this IL-5 drug is ready for much wider use.

The key is­sues at the FDA, out­lined in their in­ter­nal re­view:

Hal Bar­ron

For one, GSK’s drug $GSK failed one late-stage test while scor­ing a suc­cess in the oth­er, ac­cord­ing to the FDA memo pre­sent­ed by Sal­ly Sey­mour and Banu Kari­mi-Shah at the Di­vi­sion of Pul­monary, Al­ler­gy, and Rheuma­tol­ogy Prod­ucts. With on­ly two Phase III stud­ies in this in­di­ca­tion, that leaves reg­u­la­tors con­sid­er­ing an ap­proval with­out sup­port da­ta from two well-con­trolled stud­ies — the agency gold stan­dard.

There was no dose re­sponse they could find in the two dosage arms.

The FDA added:

Some key sec­ondary end­points, such as time to first mod­er­ate-to-se­vere COPD ex­ac­er­ba­tion showed nu­mer­i­cal trends fa­vor­ing mepolizum­ab. How­ev­er, there were no con­sis­tent and clin­i­cal­ly mean­ing­ful dif­fer­ences in se­vere COPD ex­ac­er­ba­tion rate, FEV1, or SGRQ be­tween mepolizum­ab 100 mg and place­bo.

GSK’s tri­al de­signs spurred ques­tions, with no base­line col­lec­tion of asth­ma his­to­ry — which could have in­flu­enced the out­come of the study. How will GSK prop­er­ly de­fine the pa­tient pop­u­la­tion for this drug?

And then there’s safe­ty:

As de­scribed in the Re­view of Safe­ty, analy­ses by both the Ap­pli­cant and the Agency re­vealed con­sis­tent im­bal­ances in car­dio­vas­cu­lar throm­bot­ic events to­wards mepolizum­ab de­spite dif­fer­ent analy­sis meth­ods. Ad­di­tion­al se­ri­ous and non-se­ri­ous ad­verse event im­bal­ances are not­ed in the Re­view of Safe­ty sec­tion of the brief­ing doc­u­ment.

It’s a bad week for GSK to be chal­lenged. New R&D chief Hal Bar­ron is ex­pect­ed to lay out his strat­e­gy on Wednes­day, while the pan­el meets to con­sid­er if the re­search group at GSK han­dled the COPD pro­gram prop­er­ly. Af­ter years of de­vel­op­ment set­backs, out­side of HIV and vac­cines, it’s these kinds of crit­i­cal dis­cus­sions that the phar­ma gi­ant has to grap­ple with once and for all.

This ap­pli­ca­tion al­so presents a chal­lenge for the FDA. At a time some crit­ics are warn­ing that the FDA has cut stan­dards to a bare min­i­mum, will they let an­oth­er drug with ques­tion­able da­ta out on the mar­ket?

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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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.

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.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.