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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Af­ter sell­ing to Genen­tech, the old Je­cure team is back at an RNA-fo­cused start­up — and more en­thu­si­as­tic than ever

When Genentech swooped in to buy NASH-focused Jecure Therapeutics back in 2018, a handful of the startup’s executives weren’t quite ready to disperse.

It had been just three years since Jecure launched with a preclinical portfolio of NLRP3 inhibitors — and the takeover came sooner than anyone, including CEO Jeff Stafford, had expected. So he got talking with James Veal and Gretchen Bain, two serial entrepreneurs in charge of Jecure’s R&D.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.

Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.