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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

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.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

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.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

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.

Mi­rati's KRAS drug looks like the ear­ly fa­vorite in colon can­cer with new da­ta, putting the pres­sure square on Am­gen

With Amgen already providing proof-of-concept for KRAS inhibitors with its sotorasib, Mirati Therapeutics is piecing together a follow-up effort in lung cancer with data it thinks are superior. But in colon cancer, where solo sotorasib has turned in a dud, Mirati may now have a strong case for superiority.

Mirati’s adagrasib, dosed solo or in combination with chemotherapy cetuximab, showed numerically higher response rates than sotorasib solo and as part of a combination study in a similar patient population also revealed this week at #ESMO21. Mirati’s data were presented as part of a cohort update from the Phase II KRYSTAL-1 study testing adagrasib in a range of solid tumors harboring the KRAS-G12C mutation.

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Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

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.