The new se­r­i­al en­tre­pre­neur in the biotech are­na launch­es an­oth­er up­start look­ing to drug RNA

Bill Haney

Click on the im­age to see the full-sized ver­sion

Af­ter a life­time of en­tre­pre­neur­ship, Bill Haney has dis­cov­ered how much fun it can be to set up new biotechs.

A year af­ter launch­ing the can­cer start­up Drag­on­fly — al­ly­ing him­self with MIT’s Tyler Jacks and David Raulet out of Berke­ley — he’s now back with his sec­ond up­start called Sky­hawk Ther­a­peu­tics, which is jump­ing in­to the fast-grow­ing new com­pa­ny seg­ment fo­cused on drug­ging RNA with small mol­e­cules.

“Sky­hawk tech­nol­o­gy and in­sight of­fers an en­tire­ly nov­el way to treat a very broad class of dis­eases by en­abling them to tar­get mu­ta­tions in ex­on-splic­ing, (tar­get­ing) loss of func­tion dis­eases,” Haney tells me.

Their first project, which could be in the clin­ic in 2019, ze­roes in on what Haney de­scribes as a pre­vi­ous­ly un­drug­gable onco­gene.

Haney — who is al­so a doc­u­men­tary film­mak­er and chair­man of the green home builder Blu Homes — is tak­ing a leaf from the same play­book he used to launch Drag­on­fly, an­oth­er Boston-area launch that has been work­ing on new tech­nol­o­gy to leapfrog where check­point ther­a­pies are right now, link­ing on­to nat­ur­al killer cells and drag­ging them to a can­cer cell to tack­le a wide range of can­cers.

Like Drag­on­fly, Haney is once again work­ing with a tight-knit group of fam­i­ly of­fice in­vestors like his old friend Tim Dis­ney, who’s come back in on this lat­est ven­ture as well. The Duke of Bed­ford joined the back­ers club, along with Alexan­dria Ven­ture In­vest­ments and oth­er undis­closed pri­vate in­vestors.

They’ve put up $8 mil­lion in seed cash to get things rolling at Sky­hawk, which has a staff of about 25 and is on its way to its first IND and the clin­ic in about a year.

Like his co-in­vestors, Haney works with peo­ple who are rec­og­nized ex­perts in their field, and whom he trusts im­plic­it­ly to do the right thing.

Kath­leen Mc­Carthy

He first met co-founder Kath­leen Mc­Carthy back when she was a col­lege stu­dent work­ing with AIDS pa­tients in a de­vel­op­ing coun­try. And he’s not in the least bit re­luc­tant to praise her as a “strong mind­ed, fe­ro­cious” young sci­en­tist out to do some­thing com­plete­ly new.

Says Haney: “I have a lot of con­fi­dence in Kath­leen. I would help her in any cir­cum­stance.”

That’s the key to back­ing any­one in biotech, he adds: Con­fi­dence and trust. Once you get that out of the way, he adds, every­one can fo­cus on the sci­ence.

This is the fourth biotech to come out of stealth mode with plans to drug RNA. Michael Gilman got the par­ty start­ed last Feb­ru­ary when he ush­ered Ar­rakis out af­ter more than a year of qui­et­ly set­ting up the plat­form. Then at the be­gin­ning of this month Ex­pan­sion Ther­a­peu­tics had its com­ing out par­ty, dis­play­ing a plat­form with R&D roots in the lab of Scripps’ Matthew Dis­ney (not re­lat­ed to Tim), who’s had a long­time in­ter­est ex­plor­ing the field. Ri­bometrix is al­so in­volved.

Sky­hawk’s work is in­spired by an ex­pe­ri­enced group led by Mc­Carthy, who worked at Roche on the SMA drug RG7916 — now in piv­otal tri­als — with a stint at the Spinal Mus­cu­lar At­ro­phy Foun­da­tion, where she had worked on a small mol­e­cule ther­a­peu­tic tar­get­ing mR­NA-pro­tein in­ter­ac­tions for SMA.

When I talked to Haney Wednes­day morn­ing, he was in Cal­i­for­nia af­ter spend­ing time with Jim Al­li­son in Texas, shoot­ing a doc­u­men­tary on the leg­endary sci­en­tist and the I/O rev­o­lu­tion he helped spark.

With two biotechs, ad­di­tion­al ven­tures and a doc­u­men­tary se­ries on can­cer planned, you could say Haney is stay­ing busy. Does he have enough time to squeeze in a third start-up?

He won’t rule it out.

“It’s a maybe,” he says.


Im­age: Bill Haney. Drag­on­fly Ther­a­peu­tics

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Chris Garabedian. Xontogeny

Per­cep­tive teams up with Chris Garabe­di­an to open up a new, $210M biotech fund fo­cused on A rounds

Perceptive Advisors is one of those prolific biotech investor groups which has traditionally enjoyed zeroing in on clinical-stage investments and crossover rounds, a group that prefers more established drug development players with near-term payoff potential.

But now they’re partnering with Xontogeny chief and longtime biotech entrepreneur Chris Garabedian on a $210 million fund — with money contributed by institutional investors and family funds — to go into the launch space with their first early-stage VC fund. Dubbed the Perceptive Xontogeny Venture Fund, LP, or just PXV Fund, they plan to favor upstarts that Garabedian is fostering in his incubator. But they’ll also plan to reach outside that inner circle for more A rounds to back, with plans to dominate initial funding with $10 million to $20 million per newborn biotech.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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US biosim­i­lar launch­es about to turn a cor­ner

The US biosimilar industry has lingered in the shadow of the European market since the US pathway for approvals was initiated in 2009.

Ten years later (or less than five years since the first FDA approval of a biosimilar), and just 42% (11 out of 26) of FDA-approved biosimilars have launched. But in the next three months (see chart below), a clutch of new biosimilars will hit the market, including new ones in oncology, hinting at a wave of uptake.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.