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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Roche and Genen­tech re­searchers plot $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Roche and Genentech have committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Researchers from both Roche and its big South San Francisco hub — mixing teams from gRED and pRED this time — will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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J&J ad­comm live blog: Com­mit­tee votes 22-0 to rec­om­mend an FDA OK for the J&J vac­cine, set­ting up 3rd US Covid-19 jab

The US could have a third authorized Covid-19 vaccine within hours.

The FDA’s advisory committee voted unanimously — 22-0 — to recommend the agency issue an emergency use authorization for J&J’s vaccine. If they follow the precedent of the Pfizer and Moderna vaccine,  the FDA will likely authorize the vaccine by Saturday, immediately adding a few million doses to the US supply and adding a 100 million by June. An authorization would give the world its first single-dose vaccine, a major weapon in the effort to vaccinate the world and bring the virus to heel, particularly in rural and developing areas.