No­var­tis makes its case on why RTH258 can grab a block­buster piece of Re­gen­eron's AMD mar­ket

Vas­ant Narasimhan, No­var­tis’ CEO-des­ig­nate Bloomberg

Soon-to-be No­var­tis $NVS CEO Vas Narasimhan has peeled back the cov­er on more de­tailed Phase III da­ta for their block­buster Eylea ri­val RTH258 (brolu­cizum­ab), ex­plain­ing why he be­lieves the phar­ma gi­ant has a new drug for wet age-re­lat­ed mac­u­lar de­gen­er­a­tion that has proven to be con­sid­er­ably bet­ter than Re­gen­eron’s $5 bil­lion fran­chise ther­a­py Eylea. But he has one oth­er hur­dle to clear be­fore mak­ing a slight­ly paused ar­rival at the FDA.

In a pre­view of da­ta be­ing re­leased at the Amer­i­can Acad­e­my of Oph­thal­mol­o­gy con­fer­ence in New Or­leans, the phar­ma gi­ant’s de­vel­op­ment chief once again hit on the 52% to 57% suc­cess rate for their two big Phase II­Is. That hand­i­ly cleared the 40% to 50% bar need­ed to qual­i­fy for an im­pres­sive suc­cess set by some an­a­lysts cov­er­ing the com­pa­ny.

No­var­tis is bet­ting that mov­ing pa­tients from once every 8 weeks for Re­gen­eron’s flag­ship ther­a­py to a 12-week reg­i­men will win over a big slice of the mar­ket. But they’re back­ing up the ap­peal to pa­tients with a pitch to pay­ers and physi­cians, of­fer­ing ev­i­dence of the drug’s abil­i­ty to re­duce the flu­id that builds up be­hind the reti­na as the dis­ease pro­gress­es, which dam­ages the eye and leads to blind­ness.

“Pay­ers wants to see flu­id is­sues re­solved to make sure the dis­ease is con­trolled,” not­ed Narasimhan in a con­fer­ence call with re­porters. The drug, he ex­plains, is a nov­el an­ti­body frag­ment that is built in a way that al­lows an in­jec­tion to squirt a high con­cen­tra­tion of ther­a­py in­to the eye. Bet­ter pen­e­tra­tion is a key part of its ar­gu­ment that this drug can be more ef­fec­tive at longterm care than Eylea as well as the No­var­tis/Roche drug Lu­cen­tis.

At week 16 the two RTH258 arms had 33% and 35% few­er pa­tients with dam­ag­ing reti­nal flu­id, and at 48 weeks one arm had 41% few­er pa­tients in the dan­ger zone, com­pared to the com­peti­tor. Ac­tive dis­ease was ob­served in 23.5% of brolu­cizum­ab 6 mg pa­tients ver­sus 33.5% of afliber­cept pa­tients in HAWK, and in 21.9% of brolu­cizum­ab pa­tients ver­sus 31.4% of afliber­cept pa­tients in HAR­RI­ER.

The drug, says Narasimhan, “was con­sis­tent­ly su­pe­ri­or to Eylea.” And there’s a lot at stake, with Re­gen­eron tak­ing in $953 mil­lion in US Eylea sales in Q3.

But there is one po­ten­tial hitch. No­var­tis ob­tained the drug through the ac­qui­si­tion of a small com­pa­ny, through which they in­her­it­ed one man­u­fac­tur­ing process. No­var­tis sub­se­quent­ly de­vel­oped its own process and now reg­u­la­tors want the phar­ma team to run some pa­tients through a small test to see if the new process holds up equal­ly well.

As a re­sult, Narasimhan says the com­pa­ny plans to file at the end of next year, rather than hus­tling the drug to the agency now, as most man­u­fac­tur­ers would pre­fer. That’s to­ward the end of the H2 2018 sched­ule that No­var­tis has told in­vestors, putting back any com­mer­cial show­down with Re­gen­eron un­til lat­er in 2019, af­ter Narasimhan moves in­to his new job as­sur­ing in­vestors that the com­pa­ny knows how to de­vel­op big new prod­ucts.

That will buy Re­gen­eron some more time to line up a pos­si­ble com­pet­i­tive re­sponse. They’re work­ing on nes­vacum­ab (Ang2)/Eylea Phase II stud­ies in DME and wet AMD that an­a­lysts are watch­ing close­ly. And a spokesper­son notes that Re­gen­eron is al­so push­ing ahead with its own quar­ter­ly dos­ing reg­i­men of Eylea.

Re­gen­eron is one of the most ag­gres­sive biotechs in the in­dus­try. They won’t sim­ply stand aside as No­var­tis ze­roes in on their big fran­chise.

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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Covid-19 roundup: Mod­er­na dos­es first pa­tients in PhII vac­cine tri­al; Eli Lil­ly touts clin­i­cal en­try of Ab­Cellera-dis­cov­ered an­ti­body

With an eye on kick-starting Phase III for its mRNA vaccine in July, Moderna said it’s dosed the first participants of each age cohort enrolled in the Phase II study.

Moderna is responsible for the whole trial, which is designed to recruit 600 healthy volunteers — 300 between the ages of 18 and 55, and another 300 older than 55. Two doses (50 μg and 100 μg) will be tested alongside placebo, and each participant receives two shots.

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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.

Jean-Jacques Bienaime, 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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Ver­sant-backed, Bris­tol My­ers-stamped Re­pare Ther­a­peu­tics guns for $100M IPO

With a Bristol Myers Squibb endorsement in tow, Versant-backed cancer drug developer Repare Therapeutics has set its sights on a Nasdaq debut.

On Friday, the Montreal-based company with operations in Cambridge, Massachusetts that is yet to enter the clinic, unveiled plans for a $100 million IPO, banking on its “synthetic lethality” platform.

The basic idea is to target the genetic basis of tumors, a common idea across precision oncology medicines. But instead of targeting the perpetrator mutation directly, the compound is designed to go after the other gene in the gene pair. The rationale is based on the decades-old genetic principle that indicates two mutations are lethal only when combined together.

Lynn Seely, Myovant CEO

My­ovant’s re­l­u­golix wins a piv­otal prostate can­cer show­down with an old stan­dard — com­ing down to the wire on ap­provals

Myovant $MYOV has rounded the final turn in its development race to get relugolix down to the regulatory wire at the FDA. And the biotech joined the virtual crowd at ASCO with the kind of data needed to keep the investor crowd’s attention.

Much of the attention on the drug has been focused on uterine fibroids, where AbbVie just scored a regulatory win for their rival drug Oriahnn (elagolix) as the biotech posted results in prostate cancer at the ASCO meeting.

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In a sting­ing set­back, Pfiz­er’s can­cer block­buster Ibrance flops in key ad­ju­vant set­ting

One of Pfizer’s top, long-running R&D catalysts has gone up in smoke, and it took an $11 billion bite of their market cap in the process.

The monitoring committee determined that Pfizer’s adjuvant study using Ibrance combined with standard endocrine therapy in an adjuvant setting for early-stage breast cancer has officially failed to make the cut. The combo failed to beat the standard alone, tripping over the futility analysis. And the Pfizer team will now wrap the study early after pumping up hopes that their blockbuster cancer therapy could find billions more by proving its efficacy for disease-free survival in a major area — something AstraZeneca just accomplished with Tagrisso to great fanfare.

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