Phar­ma com­pa­nies out­per­form in­sti­tu­tions in Eu­ro­pean clin­i­cal tri­al re­port­ing amid prob­lem­at­ic over­all trends — Ox­ford study

Half of all tri­als sup­posed to be re­port­ed in a Eu­ro­pean data­base are not, de­spite clear le­gal re­quire­ments, re­searchers have found in the first such study of com­pli­ance, which al­so notes that phar­ma com­pa­nies do bet­ter in this re­gard than aca­d­e­m­ic in­sti­tu­tions, char­i­ties and hos­pi­tals.

Ben Goldacre

Comb­ing through 7274 tri­als on the EU Clin­i­cal Tri­als Reg­is­ter (EU­C­TR) that have passed the due date for post­ing da­ta, a team out of Ox­ford’s Ev­i­dence-Based Med­i­cine Data­L­ab not­ed that on­ly 49.5% re­port­ed their re­sults — mean­ing half of the time, tri­als spon­sors have breached a 2012 Eu­ro­pean Com­mis­sion guide­line that man­dates dis­clo­sure with­in 12 months of study com­ple­tion.

The team — led by the high-pro­file Ox­ford re­search Ben Goldacre — has launched an in­ter­ac­tive on­line data­base that they plan to up­date month­ly in ad­di­tion to post­ing a pa­per in The BMJ. The old­est tri­al tracked dates back to 2004.

In gen­er­al, com­mer­cial spon­sors are much more like­ly to post re­sults; 68% of their tri­als are re­port­ed, com­pared to 11% for a non-com­mer­cial spon­sor. Scale al­so mat­tered, as the big­ger spon­sors tied to a large num­ber of tri­als col­lec­tive­ly re­port­ed more of their tri­als (78%) than their small­er coun­ter­parts (18%).

Fur­ther­more, all 11 en­ti­ties record­ing a 100% re­port­ing rate are phar­ma com­pa­nies: Boehringer In­gel­heim, Chiesi Far­ma­ceu­ti­ci, Almi­rall, Gilead, Ot­su­ka, CSL Behring, Al­con, Ver­tex, Genen­tech, Dai­ichi Sankyo and Leo Phar­ma. Even the worst per­form­ing phar­ma— Pierre Fab­re — re­port­ed 55% of the tri­als it’s sup­posed to.

No­var­tis and Glax­o­SmithK­line, the on­ly two spon­sors with more than 1,000 tri­als on the EU­C­TR, have re­port­ed rates of 94.7% and 92.1% re­spec­tive­ly.

“Al­though poor re­port­ing rates in some sec­tors is a source of con­cern, the ex­treme­ly high rate of com­pli­ance among com­mer­cial spon­sors con­duct­ing a large num­ber of tri­als is pos­i­tive: it shows that, with an un­am­bigu­ous re­quire­ment for all tri­als to re­port re­sults, near per­fect com­pli­ance can prac­ti­cal­ly be de­liv­ered,” the re­searchers wrote.

There’s a catch, though.

The re­searchers didn’t tech­ni­cal­ly count all the tri­als that were due to re­port. That’s be­cause omis­sions and in­con­sis­ten­cies in tri­al com­ple­tion dates made it im­pos­si­ble to de­ter­mine whether a spon­sor was com­pli­ant in 3392 cas­es — which makes up 29.4% of all tri­als marked “com­plet­ed” or “ter­mi­nat­ed.” And these prob­lems show up even in tri­als con­duct­ed by the 100% com­pli­ant spon­sors.

Source: The BMJ

Click on the im­age to see the full-sized ver­sion

It’s just as much of a prob­lem as non-re­port­ing to reg­u­la­tors — and the EMA can do a bet­ter job mon­i­tor­ing it while spon­sors al­so clean up their act.

“We can’t make in­formed choic­es if the re­sults of clin­i­cal tri­als are with­held from doc­tors, re­searchers, and pa­tients,” the re­searchers wrote on their data­base, adding in the pa­per: “We hope that the ac­ces­si­ble and time­ly in­for­ma­tion on the com­pli­ance sta­tus of each in­di­vid­ual tri­al and spon­sor pro­vid­ed by our EU.tri­al­strack­ will help to im­prove re­port­ing rates”.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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