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

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

Endpoints News

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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