Months af­ter Re­gen­eron se­cured FDA ap­proval for 12-week Eylea reg­i­men, No­var­tis buys speedy US re­view for its ri­val

Vas Narasimhan

No­var­tis’ brolu­cizum­ab could do to Re­gen­eron’s Eylea what the flag­ship eye treat­ment did to Roche’s Lu­cen­tis, some ex­perts have pre­dict­ed. Vas Narasimhan has shout­ed from the rooftops the promise of brolu­cizum­ab even be­fore his el­e­va­tion to No­var­tis chief. On Mon­day, No­var­tis came one step clos­er to launch­ing its chal­lenge to Re­gen­eron’s for­mi­da­ble block­buster, se­cur­ing a speedy re­view for brolu­cizum­ab for use in wet age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD), set­ting it up for ap­proval by the end of this year.

Wet AMD is the lead­ing cause of se­vere vi­sion loss and le­gal blind­ness in peo­ple over the age of 65 in North Amer­i­ca, Eu­rope, Aus­tralia and Asia, im­pact­ing an es­ti­mat­ed 20 to 25 mil­lion peo­ple glob­al­ly.

Last Au­gust, the FDA al­lowed Re­gen­eron $REGN to start mar­ket­ing a 12-week reg­i­men for Eylea for wet AMD pa­tients, ver­sus the orig­i­nal bi-month­ly reg­i­men. Brolu­cizum­ab is al­so a 12-week reg­i­men, al­though No­var­tis $NVS has ar­gued piv­otal tri­al da­ta sug­gests the drug was “con­sis­tent­ly su­pe­ri­or” to its Re­gen­eron ri­val, which gen­er­at­ed more than $4 bil­lion in net US sales last year. 

No­var­tis se­cured the quick re­view af­ter sub­mit­ting a pri­or­i­ty re­view vouch­er. The mar­ket­ing ap­pli­ca­tion was large­ly based on da­ta from two late-stage stud­ies: the HAWK and HAR­RI­ER tri­als, in which the drug met the main goal by show­ing it was as ef­fec­tive as Eylea in im­prov­ing vi­su­al acu­ity af­ter two years of treat­ment. Key sec­ondary end­points as­sess­ments al­so showed sig­nif­i­cant­ly few­er brolu­cizum­ab pa­tients with dis­ease ac­tiv­i­ty and reti­nal flu­id, ver­sus Eylea, No­var­tis said.  

“Giv­en up­com­ing com­pe­ti­tion from brolu­cizum­ab, we sense that (Re­gen­eron) man­age­ment is open to pro­vid­ing dis­counts or re­bates if nec­es­sary to main­tain Eylea’s com­pe­ti­tion in case brolu­cizum­ab pro­vides at­trac­tive fi­nan­cial terms,” Cowen’s Yaron Wer­ber wrote in a note last month.

Yaron Wer­ber

“With Roche’s faricimab com­ing in 2022/23 and Eylea biosim­i­lars in Eu­rope in 2025, the fran­chise is go­ing to be fac­ing pres­sure,” he added in a note pub­lished in Feb­ru­ary.

Mean­while, Al­ler­gan’s $AGN abic­i­par is al­so in the mix for pa­tients with wet AMD, hav­ing demon­strat­ed non-in­fe­ri­or­i­ty to Lu­cen­tis giv­en every 8 or 12 weeks in a pair of Phase III tri­als. But the rate of in­traoc­u­lar in­flam­ma­tion ob­served abic­i­par-treat­ed pa­tients in the MAPLE study were sig­nif­i­cant­ly high­er than the rate ob­served in Lu­cen­tis and Eylea, which could lim­it its suc­cess as a re­al play­er, SVB Leerink an­a­lysts wrote in a note ear­li­er in April.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.