Am­gen scores pos­i­tive PhI­II car­dio out­comes for its PC­SK9 fran­chise drug Repatha

Sean Harp­er, Ex­ec­u­tive Vice Pres­i­dent, Re­search and De­vel­op­ment

Am­gen got an­oth­er round of good news for its PC­SK9 fran­chise drug Repatha. Hot on the heels of a ma­jor patent fight vic­to­ry over Sanofi and Re­gen­eron, the big phar­ma com­pa­ny says in­ves­ti­ga­tors gar­nered pos­i­tive top-line car­dio out­comes da­ta from their crit­i­cal FOURI­ER tri­al.

There’s no da­ta avail­able yet, but the com­pa­ny re­ports that Repatha proved to sig­nif­i­cant­ly re­duce the risk of car­dio events in pa­tients with clin­i­cal­ly ev­i­dent ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease. The drug al­so hit on an end­point for cog­ni­tive func­tion.

Am­gen shares surged 3.4% in af­ter-mar­ket trad­ing.

Am­gen has had high hopes for Repatha, but sales have lagged well be­hind ini­tial ex­pec­ta­tions — Q3 2016 sales were on­ly $40 mil­lion — as pay­ers proved re­luc­tant to cov­er the drug with­out some clear ev­i­dence of a car­dio ben­e­fit. The ques­tion now is whether Am­gen can trans­late its da­ta, what­ev­er they are, in­to greater cov­er­age.

The lev­el of ben­e­fits that Am­gen was look­ing to achieve in Fouri­er, though “are rel­a­tive­ly mod­est, and thus, im­pact on sales may not be ‘trans­for­ma­tion­al,’” Baird’s Bri­an Sko­r­ney re­cent­ly not­ed. An­a­lysts will get a look at the de­tails in March, dur­ing the Amer­i­can Col­lege of Car­di­ol­o­gy an­nu­al meet­ing.

Not­ed Leerink’s Ge­of­frey Porges:

In­vestors will con­tin­ue to pon­der the im­pact of mod­est ben­e­fit (ie 15-20% risk re­duc­tion), com­pared to what con­sen­sus con­sid­ers a more sig­nif­i­cant ben­e­fit (20-25%) re­duc­tion, and the pos­si­bil­i­ty that the com­pa­ny achieved a ma­te­ri­al­ly bet­ter than con­sen­sus out­comes ben­e­fit (>25% risk re­duc­tion).

Am­gen re­cruit­ed 27,500 pa­tients for this study, an in­di­ca­tion of what they’re will­ing to in­vest to make this drug a block­buster. The pri­ma­ry end­point was the time to car­dio­vas­cu­lar death, my­ocar­dial in­farc­tion, stroke, hos­pi­tal­iza­tion for un­sta­ble angi­na, or coro­nary revas­cu­lar­iza­tion. The key sec­ondary end­point was the time to car­dio­vas­cu­lar death, my­ocar­dial in­farc­tion or stroke.

Just a few weeks ago Am­gen scored a stun­ning court­room win over Sanofi and Re­gen­eron when a fed­er­al judge banned sales of the ri­val PC­SK9 drug Pralu­ent. The rul­ing, now be­ing ap­pealed, said that Am­gen would suf­fer “ir­repara­ble harm” if the com­pe­ti­tion was al­lowed to con­tin­ue af­ter the two com­pa­nies vi­o­lat­ed the patents used to pro­tect Repatha.

Sanofi and Re­gen­eron have their own out­comes study un­der­way, with da­ta due to­ward the end of the year.

“In the GLAGOV study, we demon­strat­ed that Repatha has an ef­fect on ath­er­o­scle­ro­sis, the un­der­ly­ing cause of car­dio­vas­cu­lar dis­ease. These FOURI­ER re­sults show un­equiv­o­cal­ly the con­nec­tion be­tween low­er­ing LDL cho­les­terol with Repatha and car­dio­vas­cu­lar risk re­duc­tion, even in a pop­u­la­tion al­ready treat­ed with op­ti­mized statin ther­a­py,” said Sean E. Harp­er, M.D., ex­ec­u­tive vice pres­i­dent of Re­search and De­vel­op­ment at Am­gen. “Car­dio­vas­cu­lar dis­ease re­mains the num­ber one health bur­den in the world, and we look for­ward to shar­ing these out­comes da­ta with the sci­en­tif­ic com­mu­ni­ty at the ACC 66th An­nu­al Sci­en­tif­ic Ses­sion.”

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.

Pelosi drug pric­ing bill promis­es sav­ings, but could gag R&D — CBO analy­sis

The Democrats’ drug pricing bill — unveiled by Speaker Nancy Pelosi last month — could save Medicare spending by $345 billion over a seven year period, a new analysis suggests. But the venomous climate of impeachment proceedings and the intensifying discord between the Democrat-controlled House and Republican-majority Senate portends the bill will unlikely ever become law.

Technically, both sides of the aisle agree drug prices in the United States need some lowering. The Democrats’ bill, H.R.3 – Lower Drug Costs Now Act of 2019, is engineered to empower the HHS to negotiate prices for the 125 most expensive prescription drugs without at least two competitors — the Trump administration has already backed such a measure for the Veterans Association. Under the bill, prices for this category of medicines are not intended to exceed 120% of the average price in certain other countries (Australia, Canada, France, Germany and the United Kingdom), akin to a proposal floated by Trump earlier this year, which suggested prices be pegged against what other nations were paying as part of an “international pricing index”.

First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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