Ver­sar­tis shares ham­mered by a PhI­II flop as growth hor­mone flunks key test

Two years af­ter Ver­sar­tis $VSAR got out from un­der a clin­i­cal hold on so­mavaratan (VRS-317), the biotech says its big Phase III study proved to be a bad flop.

Jay Shep­ard

The long-act­ing form of re­com­bi­nant hu­man growth hor­mone failed to meet the pri­ma­ry end­point for non-in­fe­ri­or­i­ty in a com­par­i­son against dai­ly Genotropin, with the stan­dard of care ac­tu­al­ly beat­ing out the ex­per­i­men­tal ri­val. Height ve­loc­i­ty for so­mavaratan twice month­ly was 9.44 cm, ver­sus 10.70 cm for those re­ceiv­ing Genotropin.

This was a big cat­a­lyst for the Men­lo Park, CA-based com­pa­ny, which will now have to ac­count for the set­back with in­vestors.

The biotech was tagged with a clin­i­cal hold by the FDA back in the sum­mer of 2015 as reg­u­la­tors sought out ad­di­tion­al in­fo on the drug, de­lay­ing the ar­rival of the piv­otal da­ta by 6 months.

Ver­sar­tis bagged $126 mil­lion from their 2014 IPO large­ly by boast­ing about the po­ten­tial of this pro­gram to dis­rupt a multi­bil­lion-dol­lar busi­ness dom­i­nat­ed by dai­ly in­jec­tions. And with prac­ti­cal­ly all of its eggs in this one bas­ket, the dam­age was ex­ten­sive.

The biotech’s shares were evis­cer­at­ed, drop­ping more than 80% on the news in af­ter-mar­ket trad­ing.

“We are very sur­prised and dis­ap­point­ed to learn the out­come of the VE­LOC­I­TY tri­al. So­mavaratan showed height ve­loc­i­ty in the range we had hoped, but it was not suf­fi­cient to demon­strate non-in­fe­ri­or­i­ty in this tri­al,” stat­ed Jay Shep­ard, pres­i­dent and CEO of Ver­sar­tis. “We have done an ini­tial analy­sis of the top-line da­ta and are con­tin­u­ing to thor­ough­ly re­view the re­sults to gain greater in­sight in­to the tri­al out­come. We plan to pro­vide a cor­po­rate up­date lat­er this year. I would like to thank the in­ves­ti­ga­tors, pe­di­atric GHD pa­tients and fam­i­lies that par­tic­i­pat­ed in the VE­LOC­I­TY tri­al.”


BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.