Philip Krause

For­mer FDA vac­cines deputy Krause heads down un­der as Mesoblast tries again on near­ly en­tire pipeline

Philip Krause will take his 11 years of in­sight from the up­per ech­e­lons of the FDA — es­pe­cial­ly dur­ing pan­dem­ic vac­cine nods — and ap­ply it to an Aus­tralian biotech that wants a sec­ond chance at se­cur­ing ap­proval in the US, par­tic­u­lar­ly as it looks to bounce back from a nixed No­var­tis deal.

The for­mer sec­ond-in-com­mand for vac­cines lead­er­ship at the FDA de­part­ed un­der murky cir­cum­stances last fall when he and Mar­i­on Gru­ber, di­rec­tor of the reg­u­la­tor’s Of­fice of Vac­cines Re­search & Re­view, re­port­ed­ly were miffed by the fed­er­al ad­min­is­tra­tion’s side­step­ping of the FDA on mat­ters of Covid-19 boost­er shots.

Now, Krause will pro­vide reg­u­la­to­ry lessons to Mesoblast as a board mem­ber. The move comes at a crit­i­cal junc­ture for the Aussie drug de­vel­op­er as it looks to win over the FDA for its cell ther­a­py to treat chil­dren with steroid-re­frac­to­ry acute graft ver­sus host dis­ease (SR-aGVHD). The FDA re­ject­ed the drug, remestem­cel-L, on the grounds that a sin­gle-arm, open-la­bel study was not enough in an Oc­to­ber 2020 de­ci­sion.

The agency want­ed a ran­dom­ized tri­al, but Mesoblast ap­plied af­ter a sin­gle-arm study showed sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit on the pri­ma­ry goal against a his­tor­i­cal con­trol rate in a phase III study. Reg­u­la­tors con­vened an ad­vi­so­ry com­mit­tee to dis­cuss the ap­pli­ca­tion, and the study de­sign was dif­fi­cult for some mem­bers to en­dorse since par­ents and pe­di­a­tri­cians would be re­luc­tant to place chil­dren in a place­bo arm.

Since then, the biotech has said it’s been in a “well-es­tab­lished process with FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search.” The biotech is tee­ing up for a re­sub­mis­sion of the ther­a­py.

“Mesoblast has now gen­er­at­ed sub­stan­tial new da­ta that it be­lieves es­tab­lish the rel­e­vance of the pro­posed po­ten­cy as­say mea­sur­ing remestem­cel-L’s in vit­ro an­ti-in­flam­ma­to­ry and im­munomod­u­la­to­ry ac­tiv­i­ty to the in vi­vo clin­i­cal ef­fect of the prod­uct in the Phase 3 tri­al in chil­dren with SR-aGVHD, in­clud­ing sur­vival and bio­mark­ers of in vi­vo ac­tiv­i­ty,” the com­pa­ny said last month in its lat­est quar­ter­ly re­port.

As a new­ly mint­ed board mem­ber, Krause did not mince words on the cen­tral role he thinks he can play in Mesoblast’s path to mar­ket.

“I be­lieve I can make a sub­stan­tial con­tri­bu­tion at this very im­por­tant time in the com­pa­ny’s tran­si­tion to­wards com­mer­cial­iza­tion,” the 30-year FDA vet­er­an said in a state­ment.

Krause, who was in­volved in vac­cines de­lib­er­a­tions dur­ing the height of the pan­dem­ic, joins Mesoblast as it al­so looks to re­bound from a failed $1.2 bil­lion deal with No­var­tis in which the biotech test­ed the aGVHD drug in ven­ti­la­tor-de­pen­dent Covid-19 pa­tients with acute res­pi­ra­to­ry dis­tress syn­drome.

The No­vem­ber 2020 deal flunked with­in weeks as the da­ta safe­ty mon­i­tor­ing board rec­om­mend­ed they no longer en­roll new pa­tients and wrap the tri­al ear­ly be­cause it was un­like­ly to meet a 30-day mor­tal­i­ty re­duc­tion goal. Twelve months lat­er, No­var­tis ditched its col­lab­o­ra­tor.

But, as it ap­pears to go at Mesoblast, there’s a sec­ond try at every­thing. Last month, the biotech chalked up the con­tin­u­ing need for new Covid-19 ther­a­pies due to new vari­ants and still high num­bers of pa­tients on ven­ti­la­tors.

Af­ter talks with the FDA, Mesoblast will con­duct an­oth­er tri­al in this pa­tient pop­u­la­tion — peo­ple with mod­er­ate or se­vere ARDS due to the pan­dem­ic dis­ease — and that could pave the way for an emer­gency use nod, a route that Krause has fa­mil­iar­i­ty with around the Covid-19 jabs.

Al­so on the dock­et is a new Phase III tri­al in the US for rexleme­stro­cel-L in pa­tients with chron­ic low back pain. That study may sup­port ap­proval sub­mis­sions in the US and EU, Mesoblast said last month. Don’t fret, Mesoblast al­so wants to give the cell ther­a­py an­oth­er shot in chron­ic heart fail­ure af­ter a Phase III flop in De­cem­ber 2020.

For Krause, this isn’t the first biotech he’s linked arms with since leav­ing FDA HQ. He be­came a strate­gic ad­vi­sor at Ad­ju­vance Tech­nolo­gies in Jan­u­ary, a lit­tle-known Lin­coln, NE biotech work­ing on whoop­ing cough and shin­gles vac­cines.

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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Wendy Lund, WPP chief client officer for health and wellness

WPP taps Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund for new health and well­ness client role

Wendy Lund is going home – to WPP, that is. Lund is leaving the Merck women’s health spinoff Organon where she is chief communications officer for a newly created role as WPP chief client officer for health and wellness. Before Organon, Lund led GCI Group, a WPP healthcare communications agency, as CEO for 11 years.

Lund joins WPP’s group of global client leaders who act as a single point of contact or entry for clients with WPP brands and businesses inside the holding company. and in this case, for the WPP health and wellness business.

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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.

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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