In quest to elim­i­nate trop­i­cal dis­eases, Mer­ck KGaA will sub­mit an­ti-worm­ing drug for ap­proval in young chil­dren

As Mer­ck KGaA makes moves to treat the ne­glect­ed trop­i­cal dis­ease schis­to­so­mi­a­sis, it has an­nounced that its treat­ment arpraz­i­quan­tel has yield­ed pos­i­tive Phase III tri­al re­sults in chil­dren be­tween the ages of 3 months and 6 years old, and the com­pa­ny will now seek reg­u­la­to­ry ap­proval.

A pe­di­atric ver­sion of the stan­dard drug praz­i­quan­tel, arpraz­i­quan­tel is an an­ti-worm med­ica­tion that pre­vents new­ly hatched, par­a­sitic worms from grow­ing or mul­ti­ply­ing in­side a pa­tient. The tri­al could of­fer a cure for mil­lions.

In tri­als in Cote d’Ivoire and Kenya, more than 90% of par­tic­i­pants had no more par­a­site eggs in their stool or urine af­ter up to three weeks of treat­ment, Mer­ck KGaA said. The com­pa­ny will ap­ply for a sci­en­tif­ic opin­ion from EMA through the EU’s high-pri­or­i­ty med­i­cine pro­ce­dure, in­tend­ed for use out­side of Eu­rope. Mer­ck KGaA has got­ten sup­port from the Glob­al Health In­no­v­a­tive Tech­nol­o­gy Fund, Far­man­guin­hos in Brazil and Uni­ver­sal Cor­po­ra­tion in Kenya to ex­tend lo­cal pro­duc­tion ca­pac­i­ties.

Pe­ter Guenter

“With this mile­stone, we con­tin­ue our com­mit­ment to elim­i­nat­ing schis­to­so­mi­a­sis and en­sur­ing all peo­ple af­fect­ed by this ne­glect­ed trop­i­cal dis­ease have ac­cess to a life-sav­ing ther­a­py. To­geth­er with our con­sor­tium part­ners, we are stead­fast in our vi­sion to bring new hope to the world’s most vul­ner­a­ble pop­u­la­tions,” Mer­ck KGaA’s health­care CEO Pe­ter Guenter said in a press re­lease.

The com­pa­ny is ad­dress­ing key re­quire­ments of the WHO’s 2021-2030 roadmap for ne­glect­ed trop­i­cal dis­eases through its Schis­to­so­mi­a­sis Elim­i­na­tion Pro­gram.

The pri­ma­ry end­point of the tri­al was de­fined as no par­a­site eggs in stool 17 to 21 days af­ter treat­ment, or in urine 35 to 40 days af­ter treat­ment. No new po­ten­tial risks or safe­ty con­cerns were iden­ti­fied, the com­pa­ny said in a press re­lease.

Arpraz­i­quan­tel is de­liv­ered through an oral tablet. There are an es­ti­mat­ed 50 mil­lion preschool-aged chil­dren with schis­to­so­mi­a­sis.

Kio Yam­abe, the act­ing CEO of the GHIT Fund, said in a state­ment:

Hav­ing part­nered with the Pe­di­atric Praz­i­quan­tel Con­sor­tium since 2013, we be­lieve that in­ter­na­tion­al col­lab­o­ra­tions like this are key to ad­dress­ing the bur­den of ma­jor in­fec­tious dis­eases in the de­vel­op­ing world. The suc­cess­ful joint de­vel­op­ment of praz­i­quan­tel by Con­sor­tium part­ners, Astel­las, Mer­ck and Far­man­guin­hos em­bod­ies our un­wa­ver­ing com­mit­ment to dri­ve Japan­ese in­no­va­tion and tech­nol­o­gy through glob­al part­ner­ships.

The dis­ease is al­so known as bil­harzia. Fa­mous­ly, Is­raeli pro­fes­sion­al cy­clist Chris Froome has bat­tled the dis­ease since 2010, and test­ed pos­i­tive for an­oth­er flare-up ear­li­er this year. Doc­tors told him that the worm could have been dor­mant for near­ly a decade be­fore flar­ing up again. Froome learned of the flare-up af­ter hav­ing di­ges­tive is­sues af­ter the Tour de France.

The drug works by caus­ing se­vere spasms and paral­y­sis of the worms’ mus­cles. Of­ten, those are passed in the pa­tient’s stool, but some­times, they are com­plete­ly de­stroyed in the bow­els, ac­cord­ing to the Mayo Clin­ic.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

What's fair? New ICER re­port shows pay­ers gen­er­al­ly en­sur­ing fair ac­cess to drugs

The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.

While noting the lack of transparency hindered the report’s results, ICER said that the “great majority” of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines “fair access.”

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

GSK 'Target the Future' Ad Campaign

Glax­o­SmithK­line's friend­ly ‘shark tank’ crowd­sources ideas for mul­ti­ple myelo­ma, kicks off big­ger push

GlaxoSmithKline is inviting everyone to its friendly shark tank. Its “Think Tank” challenge launching today aims to gather the best pitches for ideas in multiple myeloma with a final pitch-off “Shark Tank” TV show-style finish next year.

The innovation contest kicks off GSK’s bigger “Target the Future” unbranded campaign to advance innovation and awareness in multiple myeloma.

“We have a good sense of where the unmet need lies and what tools may be welcomed by this community, and we’ll continue to do that as part of this program, but the ‘Think Tank’ kickoff is to bring new ideas to the table — things that come from a more grassroots perspective than a large pharma perspective,” Christine Roth, senior VP and global head of oncology at GSK, said.

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Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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