Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of Jan­u­ary, the Eu­ro­pean Med­i­cines Agency of­fi­cial­ly launched its new clin­i­cal tri­als in­fo sys­tem (CTIS), al­though the mi­gra­tion to the new plat­form has on­ly re­al­ly just be­gun, and spon­sors have un­til the end of Jan­u­ary 2023 be­fore all ini­tial tri­al ap­pli­ca­tions must be sub­mit­ted through CTIS.

Over­all, 56 clin­i­cal tri­al ap­pli­ca­tions have been sub­mit­ted in CTIS dur­ing the first 3 months since the launch of the sys­tem on Jan. 31, ac­cord­ing to new da­ta post­ed by the EMA. By com­par­i­son, about 4,000 new tri­als are au­tho­rized each year across Eu­rope.

As far as the num­ber of tri­als for which a de­ci­sion has been is­sued in CTIS by the EU mem­ber states is con­cerned, 4 non-com­mer­cial apps have re­ceived a de­ci­sion so far, and 0 com­mer­cial apps, al­though the vast ma­jor­i­ty of apps sub­mit­ted are still un­der re­view.

“Cur­rent­ly one multi­na­tion­al clin­i­cal tri­al has a de­ci­sion (au­tho­rised) in CTIS with 14 Mem­ber States Con­cerned,” the re­port notes. “An av­er­age will be pro­vid­ed in fu­ture re­ports when more than one multi­na­tion­al tri­al has a de­ci­sion record­ed.”

By Jan. 31, 2025, all on­go­ing tri­als ap­proved un­der the cur­rent EU tri­als di­rec­tive will be gov­erned by the new tri­als reg­u­la­tion, and have to be tran­si­tioned to the new CTIS.

Mean­while, to help tri­al spon­sors ful­fill their trans­paren­cy re­quire­ments un­der the new reg­u­la­tion, EMA said it is prepar­ing guid­ance on the pro­tec­tion of per­son­al da­ta and com­mer­cial­ly con­fi­den­tial in­for­ma­tion in CTIS.

And lat­er this sum­mer, EMA said it will host a CTIS-fo­cused event on lessons learned in the first six months from the CTIS launch.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonucleotides, a wide variety of synthetically modified RNA or RNA/DNA hybrids that bind to a target RNA sequence to alter RNA and/or protein expression, have been winning approvals in recent years (e.g. Novartis’ cholesterol drug Leqvio), the regulatory agency is offering new draft guidance for those looking to follow a similar path.

The non-binding guidance, titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry” deals with pharmacokinetic, pharmacodynamic, and safety assessments required as part of oligonucleotide therapeutics R&D.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.