Eu­rope preps for the launch of its new clin­i­cal tri­als data­base, with a push to in­crease tri­al par­tic­i­pa­tion

At the end of this month, the Eu­ro­pean Med­i­cines Agency will roll out its new, long-await­ed Clin­i­cal Tri­als In­for­ma­tion Sys­tem, a one-stop-shop for all clin­i­cal tri­al doc­u­men­ta­tion in the EU that will al­so sup­port the spon­sors and reg­u­la­tors through­out the tri­als.

Once it’s live, CTIS will of­fer a sin­gle clin­i­cal tri­al ap­pli­ca­tion dossier, cov­er­ing clin­i­cal tri­al ap­pli­ca­tions sub­mit­ted to EU/EEA mem­ber states (and to Na­tion­al Com­pe­tent Au­thor­i­ties and ethics com­mit­tees) and reg­is­tra­tion of the tri­al, all in one in­te­grat­ed sub­mis­sion.

The launch is the cul­mi­na­tion of the adop­tion of the Clin­i­cal Tri­al Reg­u­la­tion, which en­tered in­to force back in 2014. But its ap­pli­ca­tion was de­pen­dent on the full func­tion­al­i­ty of CTIS, fol­low­ing an in­de­pen­dent au­dit that was com­plet­ed last April.

Lin­da Bowen, glob­al head of reg­u­la­to­ry pol­i­cy and in­tel­li­gence at Seagen, told End­points News that the CTIS is vol­un­tary for the time be­ing and most com­pa­nies will like­ly start with one or two sub­mis­sions in 2022.

“EMA has done a great job in pro­vid­ing train­ing, es­pe­cial­ly for the mas­ter train­ers. We will just have to see how it re­al­ly works. My gut feel­ing is that spon­sors will start slow­ly,” she said.

CTIS will al­so of­fer the gen­er­al pub­lic and re­searchers a search­able clin­i­cal tri­al data­base, with re­sults avail­able both as a tech­ni­cal sum­ma­ry and in lay lan­guage, ac­cord­ing to the EMA.

The launch of the new CTIS, which the EMA clar­i­fies is not meant to re­place a clin­i­cal tri­al man­age­ment sys­tem for spon­sors, comes as about 40% of tri­als in Eu­rope are spon­sored by acad­e­mia. Those are of­ten small and on­ly con­duct­ed in a sin­gle coun­try. The oth­er 60% of tri­als are spon­sored by the phar­ma in­dus­try.

The EMA not­ed “a re­cent shift to more aca­d­e­m­ic tri­als be­ing done dur­ing the COVID-19 pan­dem­ic,” adding that both acad­e­mia and phar­ma spon­sors “need greater sup­port and en­able­ment for Eu­rope to flour­ish as a glob­al fo­cus for clin­i­cal re­search, both in the de­vel­op­ment of new med­i­cines and in the re­fine­ment of use of ex­ist­ing med­i­cines.”

As part of this push for more tri­als and tri­al par­tic­i­pa­tion in Eu­rope, the reg­u­la­tors this week es­tab­lished a new ini­tia­tive, known as the Ac­cel­er­at­ing Clin­i­cal Tri­als in the EU (ACT EU), which lists among its ob­jec­tives the need to sup­port more large-scale multi­na­tion­al tri­als “with broad­er ge­o­graph­i­cal scope,” as well as “re­duc­ing ad­min­is­tra­tive bur­den and in­creas­ing ef­fi­cien­cy.”

Among its plans, ACT EU wants to de­vel­op key per­for­mance in­di­ca­tors and a dash­board to track the per­for­mance of the Eu­ro­pean clin­i­cal tri­als en­vi­ron­ment.

ACT EU, which is run by the EMA, the Heads of Med­i­cines Agen­cies and the Eu­ro­pean Com­mis­sion, al­so plans to de­vel­op and pub­lish key method­olo­gies guid­ance, in­clud­ing on the im­pact of AI and ma­chine learn­ing on clin­i­cal tri­als, com­plex tri­als, de­cen­tral­ized tri­als and more, as part of its work to “strength­en links be­tween in­no­va­tion and sci­en­tif­ic ad­vice fo­ra.”

And by lever­ag­ing the var­i­ous aca­d­e­m­ic, non-prof­it, Eu­ro­pean and in­ter­na­tion­al ini­tia­tives around tri­als, they’re al­so look­ing to im­prove the way re­search is fund­ed with ev­i­dence-based de­ci­sion mak­ing.

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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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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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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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

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.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

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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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.