Sarep­ta plays nice with the FDA, sets out plans for sec­ond ac­cel­er­at­ed Duchenne pitch by year's end

At­tempt­ing to mend a rather tense re­la­tion­ship with reg­u­la­tors, Sarep­ta has wrapped up talks with the FDA on how it should move for­ward with its new Duchenne mus­cu­lar dy­s­tro­phy drug: golodirsen. With notes from the FDA in hand, Sarep­ta $SRPT says it plans to seek rapid ap­proval, with an NDA filed by the end of this year.

El­lis Unger

The Cam­bridge, MA-based com­pa­ny stirred some con­tro­ver­sy at the agency back in 2016, when the FDA ap­proved Sarep­ta’s first DMD drug Ex­ondys 51. Some reg­u­la­tors were skep­ti­cal of the drug’s ben­e­fit to pa­tients, in­clud­ing El­lis Unger, di­rec­tor of the Of­fice of Drug Eval­u­a­tions. At the time, Unger warned that Ex­ondys 51 was “es­sen­tial­ly a sci­en­tif­i­cal­ly el­e­gant place­bo.” He was over­ruled, how­ev­er, and the drug came to mar­ket lat­er that year. It’s now a de­cent rev­enue dri­ver for the com­pa­ny, bring­ing in $57 mil­lion in Q4 sales last year.

Now, Sarep­ta wants a new ap­proval for a sub­set of DMD pa­tients. The ex­per­i­men­tal drug golodirsen is de­signed to treat pa­tients whose con­di­tion is caused by an er­ror in the DNA se­quence called ex­on 53. Da­ta on the drug shows it in­creas­es dy­s­trophin, the pro­tein miss­ing in Duchenne, to 1.2% of what nor­mal pa­tients would pro­duce. That’s a big im­prove­ment from the ob­served ben­e­fit for pa­tients on Ex­ondys 51, which makes 0.28% of the nor­mal amount of dy­s­trophin.

Doug In­gram

Sarep­ta wants ac­cel­er­at­ed ap­proval of the drug, and the FDA ap­pears to be open to it — as long as the drug­mak­er lat­er pro­duces da­ta that cor­re­lates the bio­mark­er with re­al clin­i­cal out­comes in pa­tients. The com­pa­ny be­lieves its ex­ist­ing Phase III tri­al, Essence, will do just fine to sat­is­fy the FDA’s re­quire­ment, as it’s de­signed to mea­sure dystophin pro­duc­tion and im­prove­ments in clin­i­cal out­comes by com­par­ing golodirsen to a place­bo.

“Ob­vi­ous­ly, whether golodirsen will ob­tain ac­cel­er­at­ed ap­proval is a re­view de­ci­sion that will come af­ter the sub­mis­sion and re­view of our NDA,” said Sarep­ta’s CEO Doug In­gram in a state­ment. “But we great­ly ap­pre­ci­ate the will­ing­ness of the Neu­rol­o­gy Di­vi­sion to en­gage and pro­vide clear di­rec­tion to us on the steps nec­es­sary to sup­port an NDA sub­mis­sion for ac­cel­er­at­ed ap­proval.”

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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