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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Spanish Prime Minister Pédro Sanchez and European Commission President Ursula von der Leyen (AP Photo/Geert Vanden Wijngaert)

EU to launch vac­cine de­vel­op­ment and man­u­fac­tur­ing part­ner­ship with Latin Amer­i­can and Caribbean coun­tries

While European companies, including BioNTech, are focused on increasing vaccine access to African countries by setting up vaccine manufacturing facilities, the European Union is looking westward to Latin America and the Caribbean.

Speaking at a press conference with Spanish Prime Minister Pédro Sanchez, EU Commission president Ursula von der Leyen said that the EU is launching a new initiative for vaccines and medicines manufacturing in Latin America, to get drugs to Latin America and the Caribbean faster.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.