Mile­stone Phar­ma preps a leap in­to PhI­II car­dio study with $55M round backed by No­vo

A lit­tle more than two months af­ter scor­ing the proof-of-con­cept da­ta that he was look­ing for, Joe Olive­to is steer­ing Mile­stone Phar­ma­ceu­ti­cals in­to a Phase III ef­fi­ca­cy study that will put their one and on­ly drug to a piv­otal test. And he has a $55 mil­lion round led by No­vo to pay for it.

Joe Olive­to

Mile­stone, based out of Que­bec with a satel­lite of­fice in North Car­oli­na, where the CEO lives, has kept the crew small and fo­cused. The drug is etri­pamil, which is de­signed to treat a tricky, hard-to-track dis­ease called parox­ys­mal supraven­tric­u­lar tachy­car­dia, or PSVT.

PSVT is an er­rat­ic ar­rhyth­mia; a rac­ing heart beat that can be mis­di­ag­nosed as a pan­ic at­tack.

“It feels like you’re hav­ing a heart at­tack,” says the CEO, “then goes away” — of­ten by the time you get to the ER, leav­ing a mys­ti­fied group of doc­tors and nurs­es puz­zling it out.

Olive­to, though, es­ti­mates that about 1.5 to 2 mil­lion peo­ple have it in the US, though he freely ad­mits that is a rough ball­park fig­ure. Hos­pi­tal records can on­ly tell you so much about this.

Cur­rent­ly, one of the ways that this con­di­tion is treat­ed is through catheter ab­la­tion, where you use heat or cold to kill heart tis­sue. IV adeno­sine is used in the hos­pi­tal. At Mile­stone, they’re work­ing on a spray for­mu­la­tion of a short-act­ing cal­ci­um chan­nel block­er that can be ad­min­is­tered at home through a res­cue de­vice, much the way asth­mat­ics con­trol a sud­den at­tack.

Olive­to and the 10-mem­ber crew at Mile­stone took four dos­es in­to a Phase II study and came away with some dose-de­pen­dent re­spons­es on the top three.

Based on his talks with the FDA, Mile­stone reck­ons it can lim­it the Phase III ef­fi­ca­cy study to about 300 pa­tients, plan­ning to get start­ed late this year or ear­ly next and wrap it around mid-2019. At that point the com­pa­ny will need to look for more cash if it wants to go so­lo in­to the sep­a­rate safe­ty study that the FDA will re­quire for a car­dio ther­a­py like this.

Small team, sin­gle as­set biotech work­ing in the daunt­ing field of heart ther­a­pies sounds like a pro­file for a buy­out, if the cards fall the right way. Olive­to, who head­ed Chelsea Ther­a­peu­tics when the com­pa­ny was sold to Lund­beck in a $658 mil­lion deal, knows a bit about that top­ic. But he’s keep­ing his op­tions open, think­ing about new ven­ture cash or an IPO or … a sale.

Time will tell. In the mean­time, he al­so plans to start lay­ing the ground­work for mar­ket­ing.

Fol­low­ing No­vo Hold­ings’ lead is For­bion Cap­i­tal Part­ners and funds man­aged by Tekla Cap­i­tal Man­age­ment, with par­tic­i­pa­tion from Mile­stone’s ex­ist­ing in­vestors: Do­main As­so­ci­ates, Fonds de sol­i­dar­ité FTQ, BDC Cap­i­tal, Pap­pas Cap­i­tal, and GO Cap­i­tal.

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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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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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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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

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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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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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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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Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

We now know the full, early-stage story behind the drug that inspired Gilead CEO Dan O’Day’s recent $5 billion acquisition of Forty Seven.

Following up on their ASCO abstract from a couple of weeks ago, the team at Forty Seven is making their return appearance this week holding clearly promising early-stage data on their lead drug magrolimab as they ponder whether they should roll on a quest to obtain an accelerated approval.