With sparse R&D prospects, In­cyte bets $900M on Mor­phoSys' CAR-T ri­val taf­a­sita­m­ab

Hav­ing suf­fered a string of pipeline set­backs in re­cent years, In­cyte is rein­vig­o­rat­ing its R&D prospects with a pact to de­vel­op and mar­ket Mor­phoSys’ an­ti-CD19 an­ti­body taf­a­sita­m­ab, a drug be­ing primed as an al­lur­ing al­ter­na­tive to the ex­ist­ing CAR-T ther­a­pies Kym­ri­ah and Yescar­ta in pa­tients with a com­mon, treat­ment-re­sis­tant form of non-Hodgkin’s lym­phoma.

Un­der the deal, Mor­phoSys and In­cyte will co-com­mer­cial­ize taf­a­sita­m­ab in the Unit­ed States, while In­cyte has ex­clu­sive com­mer­cial­iza­tion rights out­side the re­gion. For these rights, In­cyte is giv­ing Mor­phoSys an up­front pay­ment of $750 mil­lion as well as mak­ing an eq­ui­ty in­vest­ment worth $150 mil­lion in the com­pa­ny. The Ger­man drug­mak­er is al­so el­i­gi­ble to re­ceive mile­stone pay­ments of up to $1.1 bil­lion, in ad­di­tion to roy­al­ties.

Mor­phoSys ex­pects the FDA to make its de­ci­sion on taf­a­sita­m­ab, or MOR208, for use in pa­tients with re­lapsed or re­frac­to­ry dif­fuse large B-cell lym­phoma (DL­B­CL) by mid-2020.

The ap­pli­ca­tion to mar­ket the drug was based on da­ta that showed taf­a­sita­m­ab, in com­bi­na­tion with lenalido­mide, in­duced me­di­an pro­gres­sion-free sur­vival of 12.1 months, with a me­di­an du­ra­tion of re­sponse at 21.7 months. The over­all re­sponse rate among 80 pa­tients was 60%, with a for­mi­da­ble 43% com­plete re­sponse rate.

It will com­pete with No­var­tis’ Kym­ri­ah — which se­cured ap­proval for DL­B­CL in 2018 based on an over­all re­sponse rate of 50%, and a com­plete re­sponse rate of 32% in 68 evalu­able pa­tients — al­though its adop­tion has been plagued by man­u­fac­tur­ing prob­lems, mak­ing way for Gilead’s Yescar­ta to deep­er pen­e­trate the CAR-T mar­ket. Taf­a­sita­m­ab — which has been be­stowed with the FDA’s break­through ther­a­py sta­tus — is a gar­den va­ri­ety an­ti­body un­like the two CAR-T ther­a­pies, which re­quire an elab­o­rate per­son­al­ized man­u­fac­tur­ing process (cells are iso­lat­ed from the pa­tient, ma­nip­u­lat­ed in the lab by adding chimeric anti­gen re­cep­tors to di­rect T cells to snuff out can­cer cells and then re-in­fused in­to the pa­tient).

The two part­ners are al­so plan­ning to co-de­vel­op taf­a­sita­m­ab in oth­er DL­B­CL in­di­ca­tions, as well as fol­lic­u­lar lym­phoma (FL), mar­gin­al zone lym­phoma (MZL) and chron­ic lym­pho­cyt­ic leukemia (CLL).

In 2019, Mor­phoSys saw some top man­age­ment changes and some re­or­ga­ni­za­tion as the com­pa­ny pre­pared for taf­a­sita­m­ab’s loom­ing ap­proval. When the com­pa­ny’s 17-year vet­er­an Markus En­zel­berg­er de­part­ed from his post as chief sci­en­tif­ic of­fi­cer, the com­pa­ny’s re­search arm was merged in­to the de­vel­op­ment di­vi­sion un­der chief Malte Pe­ters. CEO Si­mon Mo­roney al­so un­veiled plans he would step aside — he was re­placed by Jean-Paul Kress, the for­mer CEO of Boston’s Syn­tim­mune, which was swal­lowed by Alex­ion.

The Mor­phoSys deal is key for In­cyte — ear­li­er this month the drug­mak­er’s ex­per­i­men­tal itac­i­tinib failed a piv­otal study, dubbed GRAV­I­TAS-301, in first-line acute graft vs host dis­ease.

“While itac­i­tinib did not rep­re­sent a ma­jor fun­da­men­tal dri­ver of val­ue in our mod­el, ~ $5/share, we be­lieve the fail­ure of GRAV­I­TAS-301, which fol­lows three pri­or high pro­file pipeline dis­ap­point­ments in four years (Jakafi in sol­id tu­mors, epaca­do­stat, and Olu­mi­ant), may lead some in­vestors to ques­tion the com­pa­ny’s abil­i­ty to con­sis­tent­ly gen­er­ate val­ue from R&D in­vest­ment,” SVB Leerink An­drew Berens wrote in a note in ear­ly Jan­u­ary.

In­cyte for years has leaned on its flag­ship JAK in­hibitor Jakafi, which se­cured about $1.2 bil­lion in sales in the first three quar­ters of 2019.

“We be­lieve this is a sol­id deal, as the tafa pro­file ap­pears to be the best-in-class CD19/CD20 for the treat­ment of R/R dif­fuse large B-cell lym­phoma (DL­B­CL), giv­en the to­tal­i­ty da­ta of ef­fi­ca­cy, safe­ty, and treat­ment con­ve­nience. While the deal could so­lid­i­fy IN­CY’s pipeline and fur­ther di­ver­si­fy rev­enue, we be­lieve the deal may dis­ap­point some in­vestors who are look­ing for a trans­for­ma­tive deal to strate­gi­cal­ly ex­tend the Jakafi in­tel­lec­tu­al prop­er­ty run­way be­yond 2027,” Berens wrote in a note on Mon­day.

“Giv­en the SVB Leerink’s es­ti­mate of taf­a­sita­m­ab’s world­wide peak sales of ~$1bn, we al­so think deal might not be suf­fi­cient to po­ten­tial­ly re­place Jakafi rev­enue for the long term. Al­so, due to the fi­nan­cial terms, it is un­clear to us when the com­pa­ny would be ca­pa­ble of do­ing a more trans­for­ma­tive deal.”

So­cial im­age: In­cyte, AP Im­ages

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Here's how Bris­tol My­er­s' CEO Gio­van­ni Caforio com­plet­ed a $13B buy­out: He moved fast, upped the bid quick­ly and de­mand­ed every­one to keep up

Bristol Myers Squibb CEO Giovanni Caforio does not waste time. He also likes everyone around him to keep up.

Anyone reading over the insider account filed with the SEC of the back-and-forth over his $13 billion buyout of MyoKardia $MYOK could reach only one conclusion: The CEO who had willingly crafted a $74 billion Celgene acquisition had found something else he liked — and he was willing to pay a nice premium to get it.

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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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