Bio­gen ex­tends its set­back streak as Japan­ese reg­u­la­tors push back against their con­tro­ver­sial Alzheimer's drug

An­oth­er bad week for Bio­gen and its Tokyo-based part­ners at Ei­sai was ex­tend­ed on Wednes­day with news that a pan­el re­view of their con­tro­ver­sial Alzheimer’s drug ad­u­canum­ab earned a push­back from the health min­istry in Japan.

Ac­cord­ing to overnight news re­ports, their pan­el con­clud­ed that in­con­sis­tent Phase III da­ta and lack of clin­i­cal sig­nif­i­cance in re­duc­ing amy­loid plaque in pa­tients made it dif­fi­cult to de­ter­mine if the ther­a­py worked, but of­fered to re­view it again once the 2 part­ners lined up more da­ta.

That lat­est push­back comes just days af­ter Bio­gen and Ei­sai were forced to dig a de­fen­sive perime­ter around the for­mal Eu­ro­pean reg­u­la­to­ry de­ci­sion against an ap­proval and right on the heels of Bio­gen’s de­ci­sion to slash the US price and be­gin a com­pa­ny re­or­ga­ni­za­tion as sales stag­nate in the face of deeply em­bed­ded re­sis­tance to the drug — which of­fered one of the most con­tro­ver­sial ap­provals in FDA his­to­ry.

Bio­gen says it’s not giv­ing up on Japan. In a state­ment sent to End­points News, a spokesper­son not­ed:

The com­pa­nies will con­tin­ue to ac­tive­ly en­gage with the Phar­ma­ceu­ti­cals and Med­ical De­vices Agency (PM­DA) in Japan to agree on ad­di­tion­al da­ta re­quire­ments. Bio­gen and Ei­sai re­main com­mit­ted to bring­ing ad­u­canum­ab to pa­tients in Japan ex­pe­di­tious­ly.

Japan was seen as a pos­si­ble op­por­tu­ni­ty to stop some of the bleed­ing over the dis­as­trous roll­out, with Ei­sai deeply en­trenched in the coun­try and look­ing to mar­ket what it and its part­ners in­sist is the first dis­ease-mod­i­fy­ing drug for Alzheimer’s. Ei­sai spear­head­ed the reg­u­la­to­ry ef­fort in Japan and stood to earn the li­on’s share of the mon­ey from the big Asian mar­ket.

Ex­perts, though, have shak­en their heads in uni­son over a mud­dle of con­flict­ing da­ta, per­plexed that the FDA would of­fer an ac­cel­er­at­ed ap­proval based on the sus­pect the­o­ry that amy­loid re­duc­tion would like­ly help pa­tients fight back against the steady drain of their mem­o­ries.

Those ex­perts show no sign of go­ing away, as we al­so saw this week with a line­up of re­searchers in the field who signed a pe­ti­tion de­mand­ing the FDA pro­vide an ac­cel­er­at­ed with­draw­al of the drug from the US mar­ket.

As of to­day, the FDA re­mains the sole pre­mier drug agency in the world that thinks the drug should be on the mar­ket, based on what we know so far.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Fu­ji­film to build $188M man­u­fac­tur­ing plant in North Car­oli­na’s re­search tri­an­gle

As the Japanese conglomerate Fujifilm continues to invest heavily in its CDMO arm, one of its manufacturing divisions is teeing up a major investment.

Fujifilm Irvine Scientific announced on Tuesday that parent Fujifilm is making a $188 million investment to build a cell culture media manufacturing site in the Research Triangle Park in North Carolina. The new site will mark Fujifilm Irvine’s fifth manufacturing site globally and its second in the US.