With tech from Spin­raza­'s mak­er, French start­up Dy­nacure gets $55M to tack­le neu­ro­mus­cu­lar dis­ease

Drug dis­cov­ery pow­er­house Io­n­is gave Bio­gen one of its biggest wins in re­cent times with the de­liv­ery of Spin­raza, the spinal mus­cu­lar at­ro­phy drug ex­pect­ed to de­liv­er $750 mil­lion in sales this year. Since its Spin­raza days, Io­n­is has churned out promis­ing pro­gram af­ter promis­ing pro­gram, ce­ment­ing its rep­u­ta­tion for de­liv­er­ing clin­i­cal as­sets to its part­ners.

Now, a col­lab­o­ra­tion be­tween Io­n­is and a re­search in­sti­tute in France has laid the foun­da­tion for a start­up that’s tack­ling an­oth­er neu­ro­mus­cu­lar dis­or­der. This time, they’re shoot­ing to de­vel­op a drug for cen­tronu­clear my­opa­thy, a con­di­tion char­ac­ter­ized by mus­cle wast­ing and mus­cle weak­ness. And a group of in­vestors led by An­dera Part­ners (for­mer­ly Ed­mond de Roth­schild In­vest­ment Part­ners) has ponied up $55 mil­lion to sup­port the re­search.

The com­pa­ny is called Dy­nacure, and it was found­ed back in 2016 as a spin­out of the French re­search cen­ter called the In­sti­tute of Ge­net­ics and of Mol­e­c­u­lar and Cel­lu­lar Bi­ol­o­gy. Both the in­sti­tute and Dy­nacure are lo­cat­ed in Stras­bourg, a city in North­east­ern France.

Stéphane van Rooi­jen

Stéphane van Rooi­jen, Dy­nacure’s CEO, tells me the re­search group had teamed up ear­ly with Io­n­is to pair their an­ti­sense find­ings with the Carls­bad, CA com­pa­ny’s ex­per­tise in the field.

“The (in­sti­tute) has deep ex­per­tise in the bi­ol­o­gy around our tar­get, dy­namin 2, and had de­vel­oped a mouse an­ti­sense mod­el,” van Rooi­jen said. “Un­der an R&D agree­ment with Io­n­is, a mouse an­ti­sense com­pound was de­vel­oped, and the an­i­mal mod­el showed spec­tac­u­lar re­sults with dis­ease re­ver­sion.”

From there, the duo launched Dy­nacure, with Io­n­is start­ing out with a 15% stake. Van Rooi­jen said the com­pa­ny still has a mi­nor­i­ty stake but de­clined to share the ex­act per­cent­age. The start­up’s lead pro­gram is coined Dyn101 — an an­ti­sense oligonu­cleotide ther­a­py — and they’re tak­ing on cen­tronu­clear my­opa­thy first, with plans to en­ter hu­man tri­als in the first half of 2019.

“(Cen­tronu­clear my­opa­thy) is a de­bil­i­tat­ing, pro­gres­sive dis­ease that large­ly af­fects chil­dren and young adults, and qual­i­ty-of-life can be se­vere­ly im­pact­ed due to the loss of au­ton­o­my, and breath­ing and swal­low­ing dif­fi­cul­ties,” van Rooi­jen said. “Dis­ease man­age­ment and ther­a­py help peo­ple suf­fer­ing from CNM, but there are no phar­ma­co­log­i­cal agents that mod­i­fy the course of the dis­ease. Our an­i­mal da­ta sug­gests that Dyn101 may be able to halt dis­ease pro­gres­sion or po­ten­tial­ly re­verse it in mouse mod­els.”

Join­ing An­dera on the fi­nan­cial bet are in­vestors Pon­tif­ax, Bpifrance, Kur­ma Part­ners and Id­In­vest Part­ners.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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