News brief­ing: Re­gen­eron/Sanofi post pos­i­tive PhI­II Dupix­ent da­ta in pe­di­atric asth­ma; Vineti scores $33M Se­ries C ex­ten­sion

Re­gen­eron and Sanofi re­vealed pos­i­tive Phase III da­ta for their block­buster Dupix­ent on Tues­day, paving a po­ten­tial path for­ward in pe­di­atric asth­ma.

Dupix­ent, eval­u­at­ed in chil­dren be­tween ages 6 and 11, met its pri­ma­ry end­point of low­er­ing the rate of se­vere asth­ma at­tacks in two pa­tient pop­u­la­tions by 65% and 59% per year com­pared to place­bo. The drug al­so showed im­proved lung func­tion by 10.15 and 10.53 per­cent­age points over base­line, com­pared to 4.83 and 5.32 per­cent­age points for place­bo.

The tri­al en­rolled 408 chil­dren with mod­er­ate-to-se­vere asth­ma who did not see im­prove­ments up­on the stan­dard of care, in this case in­haled cor­ti­cos­teroids. Dur­ing the 52-week treat­ment pe­ri­od, pa­tients re­ceived in­jec­tions of Dupix­ent or place­bo every two weeks, ei­ther at the 100 mg or 200 mg dosage lev­el.

Dupix­ent is al­ready ap­proved to treat asth­ma in pa­tients old­er than 12 as well as a few oth­er in­di­ca­tions — atopic der­mati­tis and chron­ic rhi­nos­i­nusi­tis with nasal polyps.

With Tues­day’s re­sults, Re­gen­eron and Sanofi plan to file for an ap­proval in the pe­di­atric asth­ma field some­time in the first quar­ter of 2021. Dupix­ent is the duo’s biggest sell­er, net­ting more than $1.9 bil­lion in glob­al sales through the first half of 2020. — Max Gel­man

Vineti adds $33M to Se­ries C and Eli Cas­din to its board

Vineti has some new cash — and a new high-pro­file board mem­ber.

Amy DuRoss

The San Fran­cis­co-based com­pa­ny an­nounced a $33 mil­lion ex­ten­sion from its Feb­ru­ary Se­ries C round and that Eli Cas­din, founder of Cas­din Cap­i­tal, has joined its board of di­rec­tors. Vineti plans to use the funds to help boost de­vel­op­ment for its per­son­al­ized late-stage can­cer treat­ments, in­clud­ing CAR-T ther­a­py.

Tues­day’s ex­ten­sion was led by Car­di­nal Health with par­tic­i­pa­tion from Marc Be­nioff and ex­ist­ing Vineti in­vestors, in­clud­ing Canaan, Thresh­old Ven­tures, Sec­tion 32, Cas­din Cap­i­tal, No­var­tis Phar­ma AG, McKesson Ven­tures, and Life­Force Cap­i­tal. Over­all, Vineti has raised more than $115 mil­lion to­tal.

“2020 has dri­ven one key point home — the con­tin­ued suc­cess of ad­vanced ther­a­pies re­lies on stan­dard­ized, de­pend­able in­fra­struc­ture,” Vineti CEO Amy DuRoss said in a state­ment. “Vineti was built for [the] pur­pose to serve per­son­al­ized med­i­cine.” — Max Gel­man

Abi­vax grabs a €15M life­line lead­ing to ul­cer­a­tive col­i­tis da­ta

French biotech Abi­vax has ob­tained a $17.6 mil­lion (€15 mil­lion) loan from Kre­os Cap­i­tal to keep it afloat un­til a key read­out in the sec­ond quar­ter of 2021.

Hart­mut Ehrlich

“We con­tin­ue to fo­cus on the ABX464 Phase 2b ul­cer­a­tive col­i­tis tri­al, with top-line re­sults ex­pect­ed in Q2 2021, while the ABX464 Phase 2a study in rheuma­toid arthri­tis is al­so pro­gress­ing well,” CEO Hart­mut Ehrlich said in a state­ment. “Fur­ther­more, the fi­nanc­ing will be used to ad­vance the projects that are cru­cial for the fu­ture de­vel­op­ment of the Com­pa­ny, such as the prepa­ra­tion of the clin­i­cal Phase 3 in UC and the ini­ti­a­tion of a piv­otal Phase 2b/3 study in Crohn’s dis­ease.”

A small mol­e­cule de­signed to mod­u­late RNA splic­ing, ABX464 is al­so be­ing test­ed in a Phase IIb/Phase III tri­al for Covid-19.

Kre­os’ debt fund­ing is di­vid­ed in­to two tranch­es. The first €10 mil­lion is to be ful­ly drawn im­me­di­ate­ly, while the oth­er €5 mil­lion will be col­lect­ed be­fore No­vem­ber 1. Abi­vax, which is cur­rent­ly fo­cused on chron­ic in­flam­ma­to­ry dis­eases, said it will be look­ing for oth­er fi­nanc­ing op­tions. — Am­ber Tong

Twist and Neo­gene to work to­geth­er on CAR-T and TCR can­di­dates 

Twist Bio­science and Neo­gene Ther­a­peu­tics are join­ing forces to work on CAR-T and TCR ther­a­pies for can­cer pa­tients.

Emi­ly Lep­roust

Un­der the strate­gic part­ner­ship, Twist will cre­ate a TCR li­brary a self-pro­claimed “li­brary of li­braries” to aid Neo­gene’s search for en­gi­neered TCRs against tar­gets in can­cer. Twist will al­so use the li­brary to dis­cov­er an­ti­bod­ies for fu­ture Neo­gene CAR-T can­di­dates.

The com­pa­nies kept the fi­nan­cial terms of the deal un­der wraps, but said that Twist will get tech­nol­o­gy ac­cess fees, and mile­stones and roy­al­ties for any an­ti­bod­ies or TCRs that come from the part­ner­ship. Neo­gene gets ex­clu­sive rights to the syn­thet­ic TCR li­brary for tar­get­ing vi­ral and neo-anti­gens in on­col­o­gy.

“Putting our plat­forms to­geth­er, we be­lieve we will be able to ex­pe­dite the iden­ti­fi­ca­tion and ge­net­ic en­gi­neer­ing of TCR genes to cre­ate per­son­al­ized T cell ther­a­pies for can­cer, bring­ing new hope to ad­dress the cur­rent lim­i­ta­tions of treat­ments avail­able to­day,” Twist CEO and co-founder Emi­ly Lep­roust said in a state­ment. — Nicole De­Feud­is 

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sergio Traversa, Relmada Therapeutics CEO

Rel­ma­da makes 'crit­i­cal changes' to PhI­II tri­al to try and save de­pres­sion drug

Relmada Therapeutics is making changes to its Phase III study of its lead drug for major depressive disorder, in an attempt to avoid problems with a prior trial that showed little difference between the drug and a placebo.

That failure in October wiped 80% from Relmada’s stock price, and was followed by another negative readout a few months later. In both cases, the company said that there had been trial sites that were associated with what it called surprising placebo effects that skewed the results compared with the drug, REL-1017.

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Paul Song, NKGen Biotech CEO

NK cell ther­a­py-fo­cused biotech eyes SPAC deal

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

In­cyte hit by CRL on ex­tend­ed-re­lease JAK tablets, mud­dy­ing plans for Jakafi fran­chise ex­pan­sion

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Peter Hecht, Cyclerion Therapeutics CEO

Hard pressed for cash, Cy­cle­ri­on looks for help fund­ing rare dis­ease drug

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.

Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”