Prothena nets more cash from No­vo Nordisk; Khon­dri­on fails to hit end­point

A year af­ter No­vo Nordisk paid $100 mil­lion up­front to grab Prothena’s an­ti­body and wider pro­gram in AT­TR amy­loi­do­sis, the deal has hit a mile­stone, earn­ing more cash for the biotech.

Prothena an­nounced on Tues­day that it has earned a $40 mil­lion mile­stone pay­ment from the Dan­ish phar­ma. The pay­ment is re­lat­ed to its can­di­date NNC6019, for­mer­ly known as PRX004, a Prothena spokesper­son told End­points News in an email. The com­pa­ny hit the clin­i­cal mile­stone due to the “ini­ti­a­tion of the phase II study,” the spokesper­son said. The can­di­date is meant to treat pa­tients with AT­TR car­diomy­opa­thy.

The agree­ment with No­vo Nordisk could net Prothena up to $1.2 bil­lion up­on the achieve­ment of sev­er­al de­vel­op­ment and sales mile­stones. No­vo Nordisk, for its part, will gain full world­wide rights to the in­tel­lec­tu­al prop­er­ty and oth­er rights of the AT­TR amy­loi­do­sis busi­ness.

Khon­dri­on’s as­set fails to hit the pri­ma­ry end­point

Dutch biotech Khon­dri­on has dropped the re­sults for its Phase IIb study in 27 pa­tients for its as­set son­li­cro­manol, which is be­ing in­ves­ti­gat­ed for use in adults with MELAS spec­trum dis­or­ders. The drug missed its pri­ma­ry mark.

Pa­tients who re­ceived both the 50mg and 100mg dos­es for 28 days did not achieve a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in the “at­ten­tion do­main score” of cog­ni­tive func­tion­ing, com­pared to those who re­ceived a place­bo.

Yet Khon­dri­on is tout­ing the post hoc analy­ses of the study show­ing “pos­i­tive trends” in oth­er da­ta points re­lat­ed to cog­ni­tion and mood-re­lat­ed sec­ondary end­points. The da­ta showed a p-val­ue for Beck De­pres­sion In­ven­to­ry at p=0.01 and the Cog­ni­tive Fail­ure Ques­tion­naire hav­ing a val­ue of p=0.007. The biotech stat­ed that the 100mg dose is more ef­fec­tive as well.

Khon­dri­on CEO Jan Smeitink said in a state­ment:

“While son­li­cro­manol’s ef­fect on the pri­ma­ry end­point with­in a 28-day pe­ri­od was in­suf­fi­cient in the Phase IIb tri­al, we be­lieve the broad­er re­sults from the Phase IIb pro­gramme, now up to 52 weeks, pro­vide un­equiv­o­cal ev­i­dence of the pos­i­tive clin­i­cal im­pact and im­por­tant pa­tient ben­e­fit that son­li­cro­manol can pro­vide. We are par­tic­u­lar­ly en­cour­aged by the pos­i­tive sig­nals on over­all dis­ease sever­i­ty, mood, cog­ni­tion, fa­tigue, bal­ance and pain, all be­long­ing to the most bur­den­some symp­toms pa­tients ex­pe­ri­ence in their dai­ly lives, as men­tioned in UMDF’s Voice of the Pa­tient Re­port 2019.

Smeitink added in his state­ment that he will be dis­cussing plans with US and Eu­ro­pean reg­u­la­tors as the com­pa­ny will push for­ward with a Phase III tri­al, which is pre­dict­ed to start lat­er next year.

FDA’s pri­or­i­ty re­view for Gen­mab’s lym­phoma as­set ac­cept­ed

Gen­mab is mak­ing head­way for its lym­phoma as­set with the FDA.

Ac­cord­ing to the com­pa­ny, the FDA has ac­cept­ed the pri­or­i­ty re­view for a BLA for sub­cu­ta­neous ep­cori­ta­m­ab, a bis­pe­cif­ic an­ti­body meant to treat pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma af­ter two or more lines of “sys­temic ther­a­py.”

The FDA has set a PDU­FA date for May 21, 2023.

The move has al­so trig­gered an $80 mil­lion mile­stone pay­ment from Ab­b­Vie, with whom Gen­mab is col­lab­o­rat­ing on the work on ep­cori­ta­m­ab as part of a wider on­col­o­gy pact. Both com­pa­nies share com­mer­cial re­spon­si­bil­i­ties in Japan and the US. The as­set is be­ing in­ves­ti­gat­ed as a monother­a­py and in com­bi­na­tion with sev­er­al dif­fer­ent ther­a­pies.

Nona Bio­sciences and Drag­on­fly Ther­a­peu­tics en­ter drug dis­cov­ery deal

Nona Bio­sciences and Drag­on­fly Ther­a­peu­tics are team­ing up.

The deal is based on Nona’s “ful­ly hu­man heavy chain on­ly” an­ti­body (HCAb) trans­genic mice plat­form, which aims to dis­cov­er and de­vel­op those an­ti­bod­ies.

Nona will pro­vide Drag­on­fly with ac­cess to its HCAb tech and an­ti­body gen­er­a­tion ser­vices for tar­gets that are des­ig­nat­ed by Drag­on­fly. The fi­nan­cial terms of the deal were not dis­closed.

This comes af­ter Nona roped in a li­cense and col­lab­o­ra­tion agree­ment with Mod­er­na for “nu­cle­ic acid-based im­munother­a­pies” for cer­tain on­col­o­gy tar­gets.

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!

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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.

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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.

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Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; 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.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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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.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

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.”

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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.