Astel­las' hot flash­es drug will get speedy re­view at FDA; US opts out of Val­ne­va vac­cine

The FDA will de­cide on Astel­las’ menopausal symp­tom drug by Feb. 22 of next year, as the Japan­ese phar­ma dis­closed it had paid about $97 mil­lion to get a pri­or­i­ty re­view vouch­er to speed up the re­view.

Astel­las said the agency has ac­cept­ed the phar­ma’s ap­pli­ca­tion for fe­zo­line­tant for the treat­ment of mod­er­ate to se­vere va­so­mo­tor symp­toms (VMS) as­so­ci­at­ed with menopause. VMS in­cludes hot flash­es and/or night sweats. The com­pa­ny said as many as 80% of women in the US ex­pe­ri­ence those symp­toms dur­ing or af­ter the menopausal tran­si­tion.

The reg­u­la­tor will re­view da­ta from three Phase III clin­i­cal tri­als of the oral, non­hor­mon­al drug, which was test­ed in more than 2,800 women across the US, Cana­da and Eu­rope in the late-stage BRIGHT SKY stud­ies.

The drug went 4-for-4 on pri­ma­ry end­points in the two ef­fi­ca­cy stud­ies, which ob­served the once-dai­ly drug at 30 and 45 mg dos­es. Astel­las is propos­ing a 45 mg dose of the drug, which the phar­ma ac­quired through an $860 mil­lion buy­out of Bel­gian biotech Oge­da in 2017.

But the drug flopped in a late-stage tri­al in Chi­na, Ko­rea and Tai­wan ear­li­er this year.

US opts out of Japan­ese en­cephali­tis vac­cine con­tract

The US De­part­ment of De­fense will not opt in­to a sec­ond-year con­tract for Val­ne­va’s Japan­ese en­cephali­tis vac­cine, mar­ket­ed as Ix­i­aro.

Val­ne­va said the DoD’s ex­ist­ing sup­ply lev­els meet de­mand be­cause of the im­pact of the Covid-19 pan­dem­ic. The French biotech said the US agency is in­ter­est­ed in ne­go­ti­at­ing a new sup­ply con­tract next year, “once in­ven­to­ry re­turns to stan­dard lev­els.”

De­liv­er­ies un­der the first op­tion year will con­tin­ue through the fourth quar­ter. The sec­ond op­tion year had a min­i­mum val­ue of about $36 mil­lion for 250,000 dos­es, the com­pa­ny said.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Pfiz­er and Sang­amo re-open PhI­II of he­mo­phil­ia A gene ther­a­py; AEON head­ed to PhI­II

A year after voluntarily pausing the Phase III study of their hemophilia A gene therapy program, and four months after FDA lifted the clinical hold, Pfizer and Sangamo Therapeutics say they will resume dosing next month.

The move pushes back the original data readout by two years. At the time of the first patient dosing in October 2020, a Pfizer spokesperson had told Endpoints News that a pivotal readout would come in early 2022. Following the hurdles caused by the clinical hold, Pfizer said Friday morning the new anticipated timeline is the first half of 2024.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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