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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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In­cyte hit by CRL on ex­tend­ed-re­lease JAK tablets, mud­dy­ing plans for Jakafi fran­chise ex­pan­sion

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

Sergio Traversa, Relmada Therapeutics CEO

Rel­ma­da makes 'crit­i­cal changes' to PhI­II tri­al to try and save de­pres­sion drug

Relmada Therapeutics is making changes to its Phase III study of its lead drug for major depressive disorder, in an attempt to avoid problems with a prior trial that showed little difference between the drug and a placebo.

That failure in October wiped 80% from Relmada’s stock price, and was followed by another negative readout a few months later. In both cases, the company said that there had been trial sites that were associated with what it called surprising placebo effects that skewed the results compared with the drug, REL-1017.

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Paul Song, NKGen Biotech CEO

NK cell ther­a­py-fo­cused biotech eyes SPAC deal

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

Peter Hecht, Cyclerion Therapeutics CEO

Hard pressed for cash, Cy­cle­ri­on looks for help fund­ing rare dis­ease drug

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.