No­var­tis bags Mesoblast's stem cell ther­a­py for ARDS, in­clud­ing in Covid-19, in a deal worth up to $1.2B+

No­var­tis has li­censed a new stem cell ther­a­py from Mesoblast, just weeks af­ter the FDA re­ject­ed the Aus­tralian biotech’s pitch for an ap­proval on a sep­a­rate in­di­ca­tion.

The Swiss phar­ma an­nounced Thurs­day af­ter­noon it is part­ner­ing with Mesoblast $MESO to de­vel­op remestem­cel-L for the treat­ment of acute res­pi­ra­to­ry dis­tress syn­drome, in­clud­ing ARDS re­lat­ed to Covid-19. As part of the deal, No­var­tis will shell out $25 mil­lion in up­front cash and take a $25 mil­lion stake in the biotech, while of­fer­ing up to $1.255 bil­lion in po­ten­tial mile­stone pay­ments.

Mesoblast in­vestors em­braced the news, send­ing shares up 11% on the Aus­tralian stock ex­change Fri­day. The com­pa­ny’s stock was al­so up rough­ly 17% on the Nas­daq be­fore Fri­day’s open­ing bell.

The mile­stone pay­ments are split as such, per Mesoblast: $505 mil­lion will be avail­able pre-com­mer­cial­iza­tion, with an ad­di­tion­al $750 mil­lion set aside for hit­ting cer­tain sales tar­gets and dou­ble-dig­it roy­al­ties.

Remestem­cel-L, or Ry­on­cil, acts as an an­ti-in­flam­ma­to­ry and con­sists of cul­ture-ex­pand­ed mes­enchy­mal stem cells de­rived from a bone mar­row donor. Cur­rent­ly, the drug is be­ing eval­u­at­ed in a Phase III study for Covid-19-re­lat­ed ARDS with 300 pa­tients, and the first cut of da­ta is ex­pect­ed in ear­ly 2021.

Should that out­come prove suc­cess­ful, No­var­tis will launch a Phase III in non-Covid ARDS af­ter the deal clos­es. The com­pa­nies high­light­ed No­var­tis’ abil­i­ty to rapid­ly scale up cell-based ther­a­pies from the clin­ic to the com­mer­cial phase as a mo­ti­va­tor for the col­lab­o­ra­tion.

The drug had been ex­am­ined in a small com­pas­sion­ate use pro­gram for Covid-19 ARDS back in March, which in­clud­ed 12 pa­tients re­quir­ing ven­ti­la­tors. Remestem­cel-L treat­ment demon­strat­ed an 83% sur­vival rate in that pro­gram and was the ba­sis for the on­go­ing Phase III.

Thurs­day’s deal comes less than two months af­ter the FDA is­sued a CRL for remestem­cel-L in Mesoblast’s pe­di­atric acute graft-ver­sus-host dis­ease pro­gram. The re­jec­tion, which de­nied the com­pa­ny an ac­cel­er­at­ed ap­proval, came af­ter an ODAC ad­comm in Au­gust vot­ed 9 to 1 in fa­vor of ap­proval as pan­el mem­bers strug­gled to en­vi­sion what a piv­otal tri­al might look like.

Dur­ing both the ad­comm and in their CRL, reg­u­la­tors took is­sue with Mesoblast’s study de­sign giv­en that the com­pa­ny sub­mit­ted its ap­pli­ca­tion on the ba­sis of one, sin­gle-arm and open-la­bel tri­al. In the study, Remestem­cel-L demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit in its pri­ma­ry end­point against the his­tor­i­cal con­trol rate.

But be­cause many par­ents and pe­di­a­tri­cians are re­luc­tant to risk putting chil­dren in­to the place­bo arm of a ran­dom­ized study, Mesoblast ar­gued that key opin­ion lead­ers said an ad­di­tion­al study was not fea­si­ble. The ve­to came de­spite the FDA ap­prov­ing a sim­i­lar drug — In­cyte and No­var­tis’ Jakafi — based on one sin­gle-arm tri­al, some­thing for which ODAC mem­bers chas­tised the FDA.

Ear­li­er this week, Mesoblast met with the agency for its Type A meet­ing, and the com­pa­ny re­port­ed in its third quar­ter earn­ings that it does not ex­pect the FDA to re­verse its de­ci­sion for ac­cel­er­at­ed ap­proval. Mesoblast is still wait­ing to re­ceive fi­nal meet­ing min­utes to know whether that’s in­deed the case. The CRL set back po­ten­tial ap­proval in GvHD from 2021 to 2024, per an­a­lysts.

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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Luke Miels, GSK chief commercial officer

Lend­ing a hand to a biotech in trou­ble, GSK drops $75M cash to add late-stage an­tibi­ot­ic to port­fo­lio

GSK likes to take pride in being one of the few Big Pharma players still active in antibiotics R&D. And that means keeping tabs on what the field has to offer.

In a move to beef up the late-stage pipeline, GSK is licensing a late-stage antibiotic candidate from Spero Therapeutics. In doing so, it’s coming to the rescue of a struggling biotech that’s crumbled in the wake of an FDA rejection and raised doubts about its ability to carry on.

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David Chang, Allogene CEO (Jeff Rumans)

Servi­er cuts off col­lab­o­ra­tion agree­ment with Al­lo­gene on CD19 prod­ucts, send­ing shares sput­ter­ing

Allogene Therapeutics said in an SEC filing today that French partner Servier has cut off its involvement in a partnership developing therapies directed against CD19, including the most advanced candidates in Allogene’s pipeline.

Shares of Allogene $ALLO, an outfit run by Kite vets Arie Belldegrun and research chief David Chang, fell by almost 10% on Wednesday, even as the San Francisco-based company said that Servier’s discontinuation “does not otherwise affect our current exclusive license for the development and commercialization of CD19 Products in the United States.”

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

Lina Khan, FTC chair (Graeme Sloan/Sipa USA/Sipa via AP Images)

FTC chair Lina Khan to Sen­ate: Big Phar­ma M&A is still a pri­or­i­ty tar­get

As the Federal Trade Commission has already sought new ideas for analyzing pharma mergers, FTC chair Lina Khan reiterated Tuesday to a Senate subcommittee that reviewing Big Pharma mergers is a priority.

While comparing this merger analysis in the pharma space to the study of public utilities in the 1930s “that exposed rampant financial fraud,” Khan said in prepared testimony that the commission is going to target unlawful conduct.

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