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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: Searching the Pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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No­var­tis chips in $10M for IPO-bound part­ner Pli­ant; Tenax shares soar on heart drug da­ta

Novartis is coming in with $10 million to help support the looming IPO of a partner. Pliant Therapeutics posted a new filing with the SEC showing that Novartis is buying the shares at $15, the mid-point of the range. It’s adding several million shares to the offering, bringing the total to around $135 million. Biotech companies have been enjoying quite a run on virtual Wall Street, with investors boosting new offerings to some big hauls.

Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”