What’s in a (drug) name? FDA to study how they af­fect ef­fi­ca­cy per­cep­tions

When it comes to pro­pri­etary drug names over­stat­ing their ef­fi­ca­cy, taint­ed sex­u­al en­hance­ment prod­ucts dis­guised as sup­ple­ments may take the cake (see: “2 Own The Knight” or “XXXPlo­sion Ul­tra”).

For the le­git­i­mate phar­ma­ceu­ti­cal world, the FDA wants to take a clos­er look at how cer­tain brand names af­fect con­sumer and health care providers’ per­cep­tions about ef­fi­ca­cy and the med­ical con­di­tions for which they’re in­di­cat­ed.

The agency is prepar­ing to con­duct what it says is the first wide­spread study in­to pro­pri­etary pre­scrip­tion drug names. The plan is to look at the per­cep­tions of 500 con­sumers and 500 health care providers on cer­tain fic­tion­al names for two med­ical con­di­tions — high cho­les­terol and gas­troe­sophageal re­flux dis­ease.

The study will com­pare five tar­get names that may just sug­gest a med­ical con­di­tion or vary in terms of how the name por­trays a drug’s ef­fi­ca­cy, with one name that ex­plic­it­ly sug­gests strong ef­fi­ca­cy (Cures­Flux) and one that is more neu­tral (Zer­pex­in).

Par­tic­i­pants will an­swer ques­tions about the names, be­fore and af­ter they have been told what each drug’s in­di­ca­tion is. “Tar­get names will vary such that some ef­fi­ca­cy im­pli­ca­tions are more ap­par­ent than oth­ers, and some will more clear­ly im­ply the med­ical con­di­tion for which a drug is in­di­cat­ed than oth­ers,” FDA said.

Al­ready the agency has signed off on such pro­pri­etary names that sug­gest the med­ical con­di­tion (e.g. As­traZeneca’s HER2-pos­i­tive breast can­cer treat­ment En­her­tu) or may not seem as ran­dom as oth­ers (e.g. Pro­tein Sci­ences’ flu vac­cine Flublok or Gilead’s CAR-T ther­a­py Yescar­ta).

The agency on Mon­day of­fered more de­tail and re­spond­ed to com­ments on how its study, first pre­viewed in Jan­u­ary 2020, will be con­duct­ed and why it’s look­ing in­to this is­sue.

“The pur­pose of the cur­rent study is to de­ter­mine whether a pro­pri­etary name it­self could play a role in in­flu­enc­ing con­sumer and HCP [health care provider] per­cep­tions of drug risks or ben­e­fits by sug­gest­ing the med­ical con­di­tion for which the drug is in­di­cat­ed or by sug­gest­ing an over­state­ment of the ef­fi­ca­cy of the drug,” FDA said.

The agency fur­ther clar­i­fied that the pur­pose of in­clud­ing this “ex­treme” name, Cures­Flux, is not to have da­ta on names that won’t be used in the re­al world, but to have some­thing against which to com­pare the tar­get names, which are sim­i­lar to the kind of names that would be sub­mit­ted to FDA for ap­proval.

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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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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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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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.

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.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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