Med­i­cines Co, Al­ny­lam gen­er­ate pos­i­tive buzz with ev­i­dence of a durable, 6-month LDL re­sponse to PC­SK9 ri­val

Clive Mean­well, CEO

The Med­i­cines Com­pa­ny $MD­CO and its part­ner Al­ny­lam $AL­NY post­ed an im­pres­sive set of 6-month re­sults for the first batch of pa­tients to reach the an­nu­al halfway mark on their new and per­haps much eas­i­er PC­SK9 drug reg­i­men. A sin­gle 300 mg in­jec­tion of their RNAi drug — now dubbed in­clisir­an — reg­is­tered a mean 43% re­duc­tion in bad LDL cho­les­terol af­ter 180 days, trig­ger­ing some ex­cit­ed buzz among the an­a­lysts fol­low­ing this pro­gram.

The Med­i­cines Com­pa­ny has at­tract­ed some con­sid­er­able en­thu­si­asm for a cho­les­terol drug that might on­ly be need­ed to be dosed two or three times a year—high­light­ing a drug that could well slap down ap­proved ther­a­pies from Am­gen and Sanofi/Re­gen­eron. So when they turned up with a late-break­er at the Amer­i­can Heart As­so­ci­a­tion Sci­en­tif­ic Ses­sions 2016 in New Or­leans, an­a­lysts were ready to clap at the first sol­id sign of dura­bil­i­ty from the first 189 sub­jects in the 497-pa­tient study.

Two 300 mg in­jec­tions of the drug trig­gered a 57% mean drop in LDL on day 120 and 52% by day 180.

“To­geth­er these da­ta sup­port fea­si­bil­i­ty for tri-an­nu­al and po­ten­tial­ly bi-an­nu­al dos­ing,” wrote Gena Wang, an an­a­lyst at Jef­feries.

Umer Raf­fat from Ever­core ISI not­ed:

Ph 2 PC­Sk9 da­ta pre­sent­ed at AHA just now looks very con­sis­tent and durable through day 180 (pts get­ting sin­gle in­jec­tion had -43% LDL re­duc­tion at day 180).

I am get­ting sev­er­al ques­tions on 1 death in the 500 mg arm:  it was in a pa­tient with ex­ten­sive CAD his­to­ry and mul­ti­ple CV events in the past (note that ex­clu­sion cri­te­ria on­ly se­lect­ed out pts with MACE event with­in last 6 months).  Al­so, 1 case of ALT>3ULN oc­curred in a pa­tient es­ca­lat­ing dose of statin.”

Both Am­gen as well as the team from Sanofi/Re­gen­eron have been forced to lay out dis­ap­point­ing launch­es for their PC­SK9 of­fer­ings, both tapped as like­ly block­busters in the mak­ing. The cost, as well as ques­tions of the un­der­ly­ing health ben­e­fits, have con­spired to lim­it their ini­tial use.

The dream sce­nario from Clive Mean­well, the CEO:

“We will fo­cus our re­sources on in­clisir­an for ag­gres­sive Phase 3 de­vel­op­ment to en­sure that this promis­ing agent is in­ves­ti­gat­ed thor­ough­ly and rapid­ly in Phase III, sub­mit­ted to world­wide reg­u­la­to­ry agen­cies and, if ap­proved, made avail­able to mil­lions of at-risk, of­ten non-ad­her­ent, pa­tients world­wide who con­tin­ue to grap­ple with the re­al­i­ties and risks of high LDL-C.”

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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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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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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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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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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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.