Re­gen­eron makes a block­buster case for its PhI­II drug pipeline

While Bio­gen is strug­gling to get its pipeline to per­form, Re­gen­eron ex­ecs put on a demon­stra­tion to­day on how a biotech com­pa­ny with a sec­ond-tier R&D bud­get can per­form bet­ter on the late-stage front than much big­ger com­pa­nies spend­ing much more on re­search. And it led off with bull­ish re­marks on a pair of would-be block­busters, in­clud­ing their top Phase III drug dupilum­ab.

Dur­ing their sec­ond quar­ter re­view, the ex­ec­u­tive team not­ed:

— Sar­ilum­ab is up for an Oc­to­ber 30 PDU­FA date at the FDA, with a re­cent Eu­ro­pean fil­ing at the EMA to fol­low up on. Peak sales es­ti­mates top $1 bil­lion for this drug, which beat megablock­buster Hu­mi­ra in a head-to-head study on rheuma­toid arthri­tis. This will like­ly be the sec­ond drug part­nered with Sanofi to hit the mar­ket – the first was the PC­SK9 drug Pralu­ent, which has had a slow up­take since its in­tro­duc­tion.

— It’s dupilum­ab, though, that is get­ting the full red car­pet treat­ment at Re­gen­eron. The IL-4/IL-13 block­ing drug – al­so part­nered with Sanofi – is ex­pect­ed to get ap­proved in the first half of 2017. Most an­a­lysts note that with stel­lar Phase III da­ta from a slate of atopic der­mati­tis stud­ies, along with the FDA’s break­through drug des­ig­na­tion to help speed things along, that ap­proval is like­ly to land ear­ly. Peak sales es­ti­mates tend to hov­er around $2.5 bil­lion a year, mak­ing it a con­tender to be­come a top 2017 drug. Some op­ti­mistic sell-siders have pushed their peak es­ti­mates to $4 bil­lion or $5 bil­lion.

R&D chief George Yan­copou­los

“Dupilum­ab is re­al­ly a fran­chise un­to it­self,” says R&D chief George Yan­copou­los in the Q2 call with an­a­lysts to­day. The pos­i­tive da­ta in atopic der­mati­tis has spurred their be­lief that the drug has “huge” po­ten­tial. There’s al­ready sig­nif­i­cant pent­up de­mand from pa­tients who ei­ther don’t re­spond to cur­rent­ly avail­able reme­dies or can’t tol­er­ate them, says Re­gen­eron’s ex­ec­u­tive team. There’s al­so no oth­er com­pe­ti­tion in the late-stage pipeline to con­tend with.

“This re­al­ly has a chance to pro­vide some­thing to pa­tients that don’t have any oth­er al­ter­na­tives at this point,” says Yan­copou­los. And the com­pa­ny is push­ing ahead in asth­ma and oth­er in­di­ca­tions, look­ing to push the bound­aries on its fran­chise po­ten­tial.

— Then there’s REGN 2810, a PD-1 check­point drug al­so part­nered with Sanofi which is al­ready in a pos­si­ble piv­otal study. This is part of an­oth­er am­bi­tious col­lab­o­ra­tion deal with Sanofi, which is look­ing to fi­nal­ly get things go­ing in the can­cer are­na.

Re­gen­eron al­so has a brand new al­liance of its own with Adicet and its CEO, Aya Jakobovits, who earned a big en­dorse­ment from Yan­copou­los.

“We re­al­ly be­lieve in peo­ple and we be­lieve in syn­er­gies,” says the R&D chief, tout­ing a match-up on Adicet’s off-the-shelf ap­proach to de­vel­op­ing cell ther­a­pies. “We have had a long­stand­ing in­ter­est to work with Aya Jakobovits. We have enor­mous re­spect for her and her ca­pa­bil­i­ties.”

Be­yond that, though, Yan­capou­los wasn’t say­ing much about the tech­nol­o­gy and what dis­tin­guish­es it from ri­vals in the field.

— And fi­nal­ly there’s fas­inum­ab (REGN475), an an­ti-NGF pain drug for os­teoarthri­tis, which is up against some com­pe­ti­tion af­ter Pfiz­er and Eli Lil­ly got things go­ing again af­ter a long pause to as­sess safe­ty is­sues. Pa­tients had a bad habit of blow­ing out their joints af­ter tak­ing the drug, and de­vel­op­ers had to be care­ful to ex­clude pa­tients at risk.

Put it all to­geth­er, and Re­gen­eron’s rel­a­tive­ly mod­est $1.5 bil­lion R&D bud­get – aug­ment­ed by Sanofi – is out­per­form­ing most of its peers, and many much larg­er com­pa­nies that have yet to nail down a suc­cess­ful R&D strat­e­gy.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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