Dy­nacure se­cures $55M as their Io­n­is-de­vel­oped drug moves in­to clin­ic

A month af­ter dos­ing their first ever pa­tient with their Io­n­is-li­censed tech, Dy­nacure has an­nounced a new round of fund­ing to ad­vance the tri­al.

The French biotech an­nounced €50 mil­lion ($55 mil­lion) in Se­ries C fund­ing, led by Per­cep­tive Ad­vi­sors. Al­though list­ed as a Se­ries C, it is the sec­ond tranche of cash the com­pa­ny has ever an­nounced, af­ter their ini­tial $55 mil­lion raise in 2018. The cash will float Dy­nacure through its Phase I/II tri­al in a class of neu­ro­mus­cu­lar dis­eases known as my­otubu­lar and cen­tronu­clear my­opathies.

Stéphane van Rooi­jen

Dy­nacure was found­ed in 2016 as part of a part­ner­ship be­tween Io­n­is and a French re­search cen­ter called the In­sti­tute of Ge­net­ics and Mol­e­c­u­lar and Cel­lu­lar Bi­ol­o­gy. Io­n­is, af­ter two decades de­vel­op­ing its an­ti­sense tech­nol­o­gy, was on the verge of its block­buster ap­proval in Spin­raza, a treat­ment for the rare pro­gres­sive neu­ro­mus­cu­lar dis­or­der spinal mus­cu­lar at­ro­phy. The new com­pa­ny promised to use an­ti­sense to tack­le an­oth­er neu­ro­mus­cu­lar con­di­tion: cen­tronu­clear my­opa­thy, a group of dis­or­ders marked by mus­cle wast­ing and weak­ness.

The lead com­pound they de­vel­oped us­es an­ti­sense to tar­get Dy­namin-2, a gene im­pli­cat­ed in some forms of the dis­ease. As cells send RNA mes­sages to build the pro­tein, the an­ti­sense binds to the RNA, block­ing des­ig­nat­ed base pairs and ei­ther chang­ing the pro­tein that is ul­ti­mate­ly pro­duced or sig­nif­i­cant­ly low­er­ing the amount of pro­tein pro­duced. A Phase I/II tri­al be­gan last month on pa­tients over 16 with one of two forms of the ge­net­ic dis­or­der, ei­ther X-linked or au­to­so­mal dom­i­nant. A third — known as au­to­so­mal re­ces­sive — is me­di­at­ed by a dif­fer­ent gene.

The com­pa­ny said they are hop­ing to ex­pand the tri­al to younger pa­tients and have oth­er dis­ease pro­grams in the works. Their ap­proach is based on mouse mod­els that sug­gest a 50% re­duc­tion in dy­namin pro­tein can ef­fec­tive­ly mute the dis­ease.

“Our an­i­mal da­ta sug­gests that Dyn101 may be able to halt dis­ease pro­gres­sion or po­ten­tial­ly re­verse it,” CEO Stéphane van Rooi­jen told End­points News in 2018.

The tri­al now un­der­way is tiny — 18 pro­ject­ed pa­tients — but it will soon give the com­pa­ny a glimpse at an an­swer.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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UP­DAT­ED: Three biotechs price hefty IPOs just be­fore the week­end, while a fourth and a SPAC seek spots on Wall Street

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

A handful of biotechs are hitting Wall Street just before the start of the weekend, with three companies — Caribou Biosciences, Sophia Genetics and Absci — all pricing big raises Wednesday and Thursday. Gamma delta T cell-focused IN8bio relaunched its IPO campaign months after postponing it last November, seeking a slightly lower raise. And another SPAC has filed for a public debut.

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Victor Perlroth, Kodiak Sciences CEO

Ko­di­ak turns down $125M pay­ment from Bak­er Bros. deal, slash­es roy­al­ty cap by 55%

Following a massive public raise last November, Kodiak Sciences has re-worked a royalty sale agreement with an old partner — and declined new funds in the process.

Kodiak is turning down a planned $125 million payment from Baker Bros. Advisors, according to an SEC filing, cutting short an agreement that saw the biotech hand over a 4.5% stream of royalty sales on its experimental anti-VEGF therapy KSI-301 for retinal vascular diseases. In conjunction with the move, Kodiak is shrinking the royalty cap from just over $1 billion to $450 million.

EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Laurent Fischer, Adverum CEO

Ad­verum faces murky fu­ture af­ter re­view turns up deep­er safe­ty is­sues for gene ther­a­py

Three months after revealing that a patient lost significant vision in one eye after receiving its experimental gene therapy, Adverum announced it found the safety issues were more widespread: Five of 12 patients who received a high dose of the therapy saw “similar clinically-relevant events.”

Three required surgery on their treated eye. And all 12 are being recommended “aggressive immunomodulatory treatments” to prevent further injury.

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