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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

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Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

John Leonard, Intellia CEO

In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

The first in vivo CRISPR/Cas9 gene editing program has some new durability data showing sustained reduction of a toxic protein in ATTR amyloidosis at all four dose levels in a small 15-patient study.

Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”