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.

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).

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.

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.