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.

Leonard Fish

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.

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.