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.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA.