Lit­tle Leo Phar­ma en­ters the prize-fight ring with pos­i­tive PhI­II atopic der­mati­tis da­ta. Now they just have to beat Dupix­ent

A day af­ter new Sanofi CEO Paul Hud­son staked his rep­u­ta­tion and the fu­ture of the phar­ma gi­ant on mak­ing Dupix­ent a mega­suc­cess sto­ry, lit­tle Leo Phar­ma is throw­ing down the gaunt­let on atopic der­mati­tis.

Three years ago Leo paid As­traZeneca $115 mil­lion to buy up rights to use tralok­inum­ab against atopic der­mati­tis — with $1 bil­lion more on the ta­ble in mile­stones — the Dan­ish com­pa­ny says their drug has swept up pos­i­tive re­sults for all pri­ma­ry and sec­ondary end­points in three Phase III tri­als. Now they plan to start the fi­nal push for reg­u­la­to­ry ap­provals so they can chal­lenge the heavy­weight cham­pi­ons in this slugfest.

To put it mild­ly, it’s not go­ing to be easy — un­der the best of cir­cum­stances.

At the time As­traZeneca did the deal with Leo, they were busy find­ing as­sets to auc­tion off as Pas­cal So­ri­ot looked to soft­en the ug­ly fi­nan­cial blows they were tak­ing as rev­enue slid steadi­ly down­ward. As­traZeneca — which over the last year has en­joyed a long-await­ed turn­around — kept their big late-stage ef­fort for tralok­inum­ab in asth­ma, con­vinced that the IL-13 drug could be a game-chang­er. In­stead, it failed in 3 straight late-stage tri­als and So­ri­ot end­ed up punt­ing it out of the pipeline.

Leo, though, nev­er veered away. To­day’s state­ment on­ly her­alds the top line re­sults, with none of the da­ta de­tails that will be cru­cial if they in­tend to be com­pet­i­tive in an in­tense­ly com­pet­i­tive field.

Ever­core ISI’s Umer Raf­fat finds the whole tralo saga in­trigu­ing, not­ing that Leo has ev­i­dent­ly been sit­ting on the re­sults for about a year. That’s not a good sign. And it left him with sev­er­al big ques­tions:

  • Why would a com­pa­ny put out first press re­lease on Ph 3s over a year af­ter they were com­plet­ed?  Not clear
  • Why didn’t Leo put out ac­tu­al ef­fi­ca­cy da­ta in to­day’s up­date?  Not clear
  • My best guess: tralo da­ta are not com­pet­i­tive.  But I can’t know for sure.

In a fol­lowup to this sto­ry, Leo’s com­mu­ni­ca­tions team got in touch to say that Raf­fat was wrong about the year-long de­lay, not­ing that the tri­als were sched­uled to wrap up in a se­ries, start­ing last Au­gust. I re­spond­ed by ask­ing when the first Phase III da­ta were ac­tu­al­ly de­liv­ered. This morn­ing, they replied — with­out an­swer­ing the ques­tion.

Be­fore is­su­ing any com­mu­ni­ca­tions on the de­vel­op­ment progress of tralok­inum­ab, it was nec­es­sary to eval­u­ate all pri­ma­ry and sec­ondary out­come mea­sures from all three piv­otal Phase 3 clin­i­cal tri­als, in ad­di­tion to ad­verse events. The full da­ta set for the blind­ed 52-week tri­als plus ad­di­tion­al weeks for safe­ty fol­low-up was not avail­able for analy­sis un­til No­vem­ber 2019.

Stan­dard op­er­at­ing pro­ce­dure in the in­dus­try is to re­port the da­ta out by tri­al as it is avail­able. The spokesper­son for the com­pa­ny, though, said Leo didn’t re­lease any hard da­ta to avoid dam­ag­ing their chances of get­ting the da­ta pub­lished in a peer-re­viewed jour­nal.

Raf­fat al­so points out there’s an­oth­er late-stage IL-13 in the AD mix: Der­mi­ra’s $DERM le­brik­izum­ab, which Roche out-li­censed af­ter they had their own failed ef­fort in asth­ma, with one suc­cess­ful Phase III and one flop.

Then there are the JAK drugs like Xel­janz, which is ap­proved for atopic der­mati­tis with a black box safe­ty warn­ing. Eli Lil­ly has been bull­ish about Olu­mi­ant, amid skep­ti­cism. Safe­ty is a con­cern with all the JAKs, which is one rea­son why the Dupix­ent group is so bull­ish about their prospects. Ab­b­Vie has Rin­voq in late-stage stud­ies, And Gilead hopes to get their break with fil­go­tinib.

More are com­ing in.

Kim Kjøller Leo

Atopic der­mati­tis is an aw­ful af­flic­tion, spurring le­sions that pro­voke a pow­er­ful urge to itch, while leav­ing a pub­lic stig­ma on ap­pear­ance. And it’s been a block­buster tar­get for a va­ri­ety of play­ers, aside from Sanofi and Re­gen­eron. Just a few days ago J&J paid $750 mil­lion for a drug called bermekimab, with plans to run it through their own piv­otal for atopic der­mati­tis and more.

In their re­lease, Leo seems to be de­ter­mined to find open­ings in the mar­ket for those who aren’t hap­py with the re­sults they’re get­ting now.

“De­spite re­cent treat­ment ad­vances, we con­sis­tent­ly hear from health­care pro­fes­sion­als around the world that ad­di­tion­al treat­ment op­tions are need­ed to ad­dress the dif­fer­ent signs and symp­toms for each pa­tient,” not­ed Kim Kjøller, the head of R&D at Leo.

Mar­ket­ing ap­pli­ca­tions are head­ed to the reg­u­la­tors ear­ly next year.

So­cial im­age: Leo Phar­ma

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

Gallia Levy, Spark CMO (Roche)

Spark Ther­a­peu­tics nabs new CMO from Genen­tech, fill­ing a ma­jor post-merg­er de­par­ture

Spark Therapeutics is getting a new CMO from their new owners.

The gene therapy company-turned-subsidiary has named Gallia Levy, who had been running rare blood disorders — including clinical development for their blockbuster-potential hemophilia antibody Hemlibra for Roche’s big biotech sub Genentech — to run medical affairs.

The appointment is a fitting one. Roche spent $4.8 billion to acquire Spark last year in large part to get their hands on their hemophilia gene therapy, SPK-8011, and expand the toe-hold Hemlibra gave them in an crowded hemophilia space.  It’s also a somewhat ironic appointment: The FTC held up the Spark acquistions for nearly a year, reportedly over concerns about the anti-trust implications of Roche owning both a top chronic treatment in Hemlibra and a top one-time treatment in Spark’s gene therapy.

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