Re­genxbio bags one of its gene ther­a­py vec­tor part­ners, buy­ing out a strug­gling Di­men­sion

Gene ther­a­py play­er Re­genxbio $RGNX has struck a deal to buy the strug­gling Di­men­sion Ther­a­peu­tics $DMTX af­ter a set­back in the clin­ic ear­li­er this year bru­tal­ly ham­mered the biotech’s mar­ket cap down to on­ly $30 mil­lion.

Re­genxbio has agreed to buy out Di­men­sion for $3.41 a share, all in stock and close to three times yes­ter­day’s $1.20 close. The deal val­ues the com­pa­ny at about $86 mil­lion, just a frac­tion of what it was worth two years ago when Di­men­sion priced its IPO shares at $13. In re­turn, Re­genxbio gets a slate of new pro­grams for its own pipeline which it is al­ready in­ti­mate­ly fa­mil­iar with, as they use its de­liv­ery vec­tor un­der one of a line­up of li­cens­ing deals the com­pa­ny set up as gene ther­a­py be­gan to take off a few years ago.

Di­men­sion shares shot up 158% on Fri­day as in­vestors caught up with the buy­out price.

Di­men­sion got start­ed as one of those new-wave gene ther­a­py de­vel­op­ers, helmed by An­nal­isa Jenk­ins af­ter the Bris­tol-My­ers vet left the lead R&D role at Mer­ck KGaA. But ear­li­er in the year Di­men­sion’s lead drug looked weak in fight­ing he­mo­phil­ia B, while ri­vals were surg­ing for­ward, crush­ing its share price and leav­ing the small de­vel­op­er look­ing to find a sur­vival plan.

Ken Mills

That lead ther­a­py — DTX-101 — is out of the pic­ture for Re­genxbio. It’s pluck­ing two ear­ly-stage gene ther­a­py drugs out of the Di­men­sion pipeline in the deal.  DTX301 is de­signed to treat or­nithine tran­scar­bamy­lase (OTC), us­ing a vec­tor to de­liv­er the OTC gene to the liv­er. And the pre­clin­i­cal DTX401 de­liv­ers a copy of the glu­cose-6-phos­phatase (G6Pase) gene to liv­er cells for glyco­gen stor­age dis­ease type Ia.

Re­genxbio al­so gets DTX201 for the treat­ment of he­mo­phil­ia A, part­nered with Bay­er. And a set of three more pre­clin­i­cal phenylke­tonuria, Wil­son dis­ease and cit­rulline­mia type I.

Re­genxbio has been close­ly al­lied with James Wil­son, the gene ther­a­py pi­o­neer who pro­vid­ed much of the IP they use in their de­liv­ery vec­tor.

“This ac­qui­si­tion con­firms Re­genxbio’s lead­er­ship in the field of AAV gene ther­a­py and ex­pands our pipeline in meta­bol­ic dis­eases us­ing NAV Tech­nol­o­gy with a clin­i­cal as­set and sev­er­al pre­clin­i­cal as­sets. Re­genxbio has the re­sources and ex­per­tise to be suc­cess­ful in ad­vanc­ing a port­fo­lio of gene ther­a­pies for in­her­it­ed meta­bol­ic dis­eases tar­get­ing the liv­er,” said Re­genxbio CEO Ken­neth Mills in a pre­pared state­ment. “We be­lieve that DTX301 and DTX401 are prod­uct can­di­dates that ad­dress dis­eases with high un­met need and will be­come an im­por­tant part of a strong in­ter­nal pipeline at Re­genxbio that has the po­ten­tial to achieve mul­ti­ple mile­stones through the end of 2018, start­ing with our in­ter­im up­dates an­tic­i­pat­ed for the end of this year on RGX-314 for wet AMD and RGX-501 for HoFH. The ac­qui­si­tion of Di­men­sion is an­oth­er mean­ing­ful step in build­ing a ro­bust clin­i­cal pipeline of gene ther­a­py prod­uct can­di­dates with the goal of im­prov­ing treat­ment op­tions for pa­tients and fam­i­lies in many dis­eases.”

Im­age: Shut­ter­stock

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.