Once tout­ed as Lyri­ca ri­val for di­a­bet­ic nerve pain, Ko­re­an biotech's plas­mid prod­uct im­plodes in PhI­II

The Ko­re­an biotech in­dus­try’s bad year just got worse as one of its high­ly tout­ed mem­bers ad­mit­ted an em­bar­rass­ing late-stage flop.

He­lixmith — known as Vi­roMed just a few months ago — had much go­ing for it head­ing in­to the 507-pa­tient Phase III: The FDA had grant­ed VM202 or­phan drug and fast track des­ig­na­tions, and it even picked up an RMAT badge dur­ing the study. Some­times clas­si­fied as a gene ther­a­py, VM202 is a plas­mid prod­uct that en­codes for the hu­man he­pa­to­cyte growth fac­tor (HGF). Due to its dual neu­rotroph­ic and an­gio­genic prop­er­ties, the drug was be­lieved to al­le­vi­ate pain caused by di­a­bet­ic neu­ropa­thy.

The re­sults from the place­bo-con­trolled tri­al though did not bear that hy­poth­e­sis out. The pri­ma­ry end­point around the 3-month change in av­er­age pain score was not sta­tis­ti­cal­ly mean­ing­ful, the com­pa­ny re­port­ed.

Ex­ecs at­tempt­ed to sal­vage some in­vestor sym­pa­thy by dis­clos­ing that some pa­tients in the place­bo group was found to car­ry VM202 (al­so dubbed En­gen­sis) DNA, while oth­ers in the treat­ment arm had sus­pi­cious­ly low con­cen­tra­tions of the plas­mid. He­lixmith claims that re­mov­ing those sub­jects from the analy­sis gave dif­fer­ent re­sults, but de­tails won’t be avail­able un­til an in­ves­ti­ga­tion in­to those phar­ma­co­ki­net­ic re­sults — led by Vi­cal ex­ec Leonard Fish — is wrapped up.

The com­pa­ny plans to share more with the FDA in De­cem­ber, but post hoc analy­sis is un­like­ly to sway reg­u­la­tors un­til He­lixmith can pro­vide some sol­id da­ta from the two oth­er Phase III tri­als they are plan­ning.

Those stud­ies will start in the next six months and be com­plet­ed in ear­ly 2020, it said in a state­ment.

He­lixmith saw its shares drop 4.4% on the news, mak­ing a small dent in its $3 bil­lion mar­ket cap.

Ear­ly in VM202’s de­vel­op­ment, the biotech had her­ald­ed its po­ten­tial to beat Pfiz­er’s Lyri­ca, a block­buster treat­ment for di­a­bet­ic nerve pain.

“Ex­ist­ing drugs are most­ly pain killers with re­port­ed side-ef­fects such as headache, in­som­nia and de­pres­sion ex­pe­ri­enced if tak­en re­peat­ed­ly, not to men­tion the has­sle of tak­ing them twice a day for years, even for decades,” R&D di­rec­tor Jong-Mook Kim boast­ed at the time. “On the con­trary, VM202-DPN showed to have ex­cel­lent ther­a­peu­tic ef­fects even though it was tak­en at much less­er fre­quen­cy – on­ly two in­jec­tions at 2-week in­ter­vals.”

The sore dis­ap­point­ment to­day echoes a larg­er trend for Ko­re­an biotechs that have built uni­corn val­ues on bold promis­es, on­ly to tum­ble down on bad da­ta. Sil­la­Jen’s re­cent fail­ure with its on­colyt­ic virus, which shaved off $800 mil­lion in mar­ket cap, un­der­scored just how much could be at stake. And that fol­lowed some long­stand­ing trou­ble that’s been brew­ing at big­ger play­ers like Han­mi and Sam­sung Bi­o­Log­ics.

He­lixmith’s ex­per­i­men­tal drug is al­so be­ing test­ed for amy­otroph­ic lat­er­al scle­ro­sis, coro­nary artery dis­ease and foot ul­cers as­so­ci­at­ed with di­a­betes.

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.

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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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.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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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.