FDA ac­cepts pri­or­i­ty re­view of CSL-uniQure he­mo­phil­ia B gene ther­a­py; Fo­s­un Phar­ma nets an­oth­er deal

The first po­ten­tial gene ther­a­py for he­mo­phil­ia B has got­ten one step clos­er to reach­ing pa­tients as the FDA has ac­cept­ed CSL Behring’s BLA for its uniQure-part­nered one-time treat­ment. The ther­a­py, dubbed etranaco­gene deza­parvovec, was ac­cept­ed un­der “ac­cel­er­at­ed as­sess­ment” by the Eu­ro­pean Med­i­cines Agency in March.

A pri­or­i­ty re­view will be con­duct­ed for the gene ther­a­py in­tend­ed for pa­tients with the hered­i­tary bleed­ing dis­or­der, CSL said Tues­day morn­ing. The Penn­syl­va­nia biotech did not in­di­cate the reg­u­la­tor’s de­ci­sion date, but a pri­or­i­ty re­view gen­er­al­ly means the treat­ment’s fate will be hand­ed down with­in six months rather than 10.

Reg­u­la­tors will base their de­ci­sion on a Phase III study that found the treat­ment to be non-in­fe­ri­or to base­line Fac­tor IX pro­phy­lac­tic ther­a­py on an­nu­al­ized bleed­ing rate (ABR) af­ter 18 months. The com­pa­nies said the treat­ment was al­so sta­tis­ti­cal­ly su­pe­ri­or at re­duc­ing ABR at the 18-month mark in the HOPE-B study.

It’s wel­come news for pa­tients and the com­pa­nies af­ter a five-month hold last year be­cause of a safe­ty is­sue that found a pa­tient in the study had de­vel­oped liv­er can­cer. UniQure con­clud­ed its ther­a­py was “high­ly un­like­ly” to have led to the can­cer.

SVB Se­cu­ri­ties an­a­lysts said they ex­pect an EMA de­ci­sion in the third quar­ter and an FDA one the fol­low­ing quar­ter. The an­a­lysts pegged the gene ther­a­py as hav­ing a “com­pelling ap­proval po­ten­tial that could reignite in­ter­est in the gene ther­a­py space broad­ly,” not­ing an­oth­er key de­ci­sion will be of Bio­Marin’s he­mo­phil­ia A gene ther­a­py, which is un­der re­view in Eu­rope and slat­ed for a June re­sub­mis­sion in the US.

Mean­while, Cowen an­a­lysts not­ed that in their sur­vey of es­ti­mat­ed he­mo­phil­ia B gene ther­a­py up­take, physi­cians said 13% of pa­tients would use such a treat­ment three years post-launch, where­as pay­ors pre­dict­ed 31% would do so.

An­a­lysts have pegged glob­al peak sales at $1.2 bil­lion, with uniQure stand­ing to earn $485 mil­lion in peak roy­al­ties af­ter sell­ing the ex­clu­sive glob­al rights to CSL in a $2 bil­lion pact. — Kyle LaHu­cik

Fo­s­un Phar­ma nets deal with Ver­Im­mune for li­cens­ing and op­tions agree­ment  

Fo­s­un Phar­ma is look­ing to keep churn­ing out deals as they are net­ting a new deal in the can­cer space.

Fo­s­un Phar­ma USA, the US-based sub­sidiary of Shang­hai-based Fo­s­un Phar­ma­ceu­ti­cal, has inked a deal with Wash­ing­ton DC-based biotech Ver­Im­mune Inc, for an ex­clu­sive li­cense and op­tion agree­ment ef­fec­tive May 18, 2022.

Ver­Im­mune is de­vel­op­ing a nov­el ther­a­peu­tic Virus-In­spired Par­ti­cle (ViP) modal­i­ty that redi­rects the body’s pre-ex­ist­ing im­mu­ni­ty against vi­ral pathogens to at­tack can­cer.

Un­der the terms of the agree­ment, Ver­Im­mune has grant­ed Fo­s­un Phar­ma an ex­clu­sive li­cense to Ver­Im­mune’s lead an­ti-tu­mor im­mune redi­rec­tion, or AIR, can­di­date, called VERI-101, with an op­tion to li­cense cer­tain po­ten­tial fu­ture AIR-ViP prod­ucts aris­ing from the de­vel­op­ment of Ver­Im­mune’s VERI-101 pipeline. This will in­clude li­cens­ing for the main­land Chi­nese, Hong Kong, Macau and Tai­wan. Fo­s­un Phar­ma will be re­spon­si­ble for all clin­i­cal de­vel­op­ment and com­mer­cial­iza­tion in these ter­ri­to­ries.

Ver­Im­mune is el­i­gi­ble to re­ceive up to $125M in to­tal up­front and mile­stone pay­ments plus tiered roy­al­ties up to the low dou­ble dig­its on net sales of VERI-101 in spec­i­fied ter­ri­to­ries. Sub­ject to Fo­s­un Phar­ma’s ex­er­cise of its op­tion to se­lect fu­ture AIR-ViP prod­uct can­di­dates, Ver­Im­mune is el­i­gi­ble to re­ceive ad­di­tion­al mile­stone pay­ments and roy­al­ties on net sales for each ad­di­tion­al prod­uct can­di­date.

“Can­cer is a glob­al un­met med­ical need and thus we are very ex­cit­ed to be work­ing with the Fo­s­un Phar­ma com­pa­nies to bring our nov­el lead AIR-ViP im­munother­a­peu­tic prod­ucts to Chi­na. Fo­s­un Phar­ma has both the ex­per­tise and ac­cess to reach these fast-grow­ing mar­kets and we hope to ben­e­fit more Chi­nese pa­tients and the re­gion through our co­op­er­a­tion” said Joshua Wang, Ver­Im­mune founder and CEO.

This is not the on­ly deal Fo­s­un has inked this year as they seized a deal with In­sil­i­co, who re­ceived $13 mil­lion up­front from Fo­s­un for an R&D col­lab­o­ra­tion project, which in­cludes co-de­vel­op­ment of In­sil­i­co’s QPCTL pro­gram. — Tyler Patchen

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Bob Nelsen (Lyell)

As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In a statement, ARCH managing director and co-founder Bob Nelsen appeared to brush off concerns about the broader market troubles, alluding to the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels.

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Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Hank Safferstein, Generian CEO

Astel­las sub­sidiary to part­ner with Pitts­burgh up­start in search for 'un­drug­gable' pro­teins

As Astellas continues its drive to build out its gene therapy portfolio and capabilities, a subsidiary of the Japanese pharma company has entered into a collaboration with a little-known Pittsburgh biotech.

Astellas-owned Mitobridge and Generian Pharmaceuticals announced on Wednesday that they will work together in a new deal for “undruggable” protein targets. Generian will net an undisclosed upfront payment and could get up to $180 million in milestones, should anything from its platform prove successful, as well as single-digit royalties on global net sales.

Adam Simpson, Icosavax CEO

Reel­ing from Covid flop, Icosavax says its RSV can­di­date passed ear­ly test. But in­vestors need some more con­vinc­ing

Three months separated from a disappointing readout of its Covid-19 vaccine, Icosavax is back with what it calls positive topline data for a different VLP vaccine candidate — although investors aren’t impressed.

IVX-121, a vaccine candidate for respiratory syncytial virus (RSV), appeared to generate “robust” immune responses among both young and older adults, as measured by neutralizing antibodies, and appeared generally well-tolerated, Icosavax reported.

Shehnaaz Suliman, ReCode Therapeutics CEO (Photo by Jennifer Leahy)

Pfiz­er, Sanofi-backed LNP out­fit goes back to the well and draws $120M for its trek to the clin­ic

A preclinical biotech touting a five-lipid drug delivery platform is looking to break out of its preclinical mold, and it just secured a sizable raise to do just that.

ReCode Therapeutics reported Wednesday morning that Leaps by Bayer and Matrix Capital Management affiliate AyurMaya co-led a Series B extension round, adding $120 million to the biotech’s previous Series B haul of $80 million. The biotech has been backed by several players in Big Pharma, notably Pfizer and Sanofi from its original Series B close last fall. And in this extension — featuring all new investors, CEO Shehnaaz Suliman tells Endpoints News — Amgen’s VC arm jumped on board.

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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