Doug Ingram, Sarepta CEO

Sarep­ta li­cens­es next-gen AAV tech from Broad In­sti­tute in deal cov­er­ing five in­di­ca­tions — in­clud­ing Duchenne

Sarep­ta’s re­search agree­ment with the Broad In­sti­tute is tak­ing the next step Mon­day.

The biotech will li­cense a new group of ade­no-as­so­ci­at­ed virus­es from the in­sti­tute for an undis­closed up­front pay­ment and mile­stone promis­es, the pair an­nounced Mon­day morn­ing. Un­der the agree­ment, Sarep­ta will have the rights to five neu­ro­mus­cu­lar and car­diac in­di­ca­tions, in­clud­ing Duchenne mus­cu­lar dy­s­tro­phy, where it’s al­ready well-versed.

Ac­cord­ing to Sarep­ta, the deal cov­ers the MyoAAV pro­gram that aims to de­liv­er more ef­fi­cient gene ther­a­pies us­ing mod­i­fied cap­sids. End­points News has al­so reached out to the Broad In­sti­tute and will up­date this sto­ry ac­cord­ing­ly.

The group of virus­es, per Sarep­ta:

  • De­liv­ered 25-50 times greater gene ex­pres­sion in mul­ti­ple skele­tal mus­cles and 10-15 times greater gene ex­pres­sion in car­diac mus­cle;
  • Demon­strat­ed re­duced de­liv­ery to the liv­er by 50 per­cent and showed low­er ac­cu­mu­la­tion in the liv­er;
  • Can be used at up to a log low­er dose than tra­di­tion­al AAV vec­tors, due to in­creased ef­fi­cien­cy.
Louise Rodi­no-Kla­pac

Full re­search for MyoAAV in a Duchenne mouse mod­el was pub­lished in Cell in 2021, the pair added. Sarep­ta CEO Doug In­gram said in a state­ment that the virus­es could “sub­stan­tial­ly re­duce vi­ral load,” while CSO Louise Rodi­no-Kla­pac tout­ed the plat­form’s “broad ap­plic­a­bil­i­ty.”

That Sarep­ta says it’s plan­ning to con­tin­ue pur­su­ing next-gen­er­a­tion Duchenne ther­a­pies sug­gests the biotech has no in­ten­tion of rest­ing on its lau­rels. Ex­ecs shep­herd­ed the first Duchenne gene ther­a­py across the FDA fin­ish line back in 2016 and have since won two more ap­provals. They’re seek­ing a fourth lat­er this year.

But those green lights have not come with­out con­tro­ver­sy. Al­most five years be­fore the Bio­gen brouha­ha with Aduhelm, Sarep­ta won an ac­cel­er­at­ed OK de­spite out­side ex­perts rec­om­mend­ing against ap­proval due to a lack of da­ta clar­i­ty. Though the FDA is not bound by law to fol­low its pan­el’s ad­vice, they typ­i­cal­ly do so in about four out of every five cas­es.

Sarep­ta’s next two ap­provals al­so fol­lowed the ac­cel­er­at­ed path, and the up­com­ing ap­pli­ca­tion will al­so seek a quick OK. A key part of this path­way in­volves a study to con­firm a ther­a­py’s ben­e­fit in or­der to con­vert the ac­cel­er­at­ed ap­proval to a full en­dorse­ment, but Sarep­ta has still not com­plet­ed any of the three con­fir­ma­to­ry tri­als.

An­a­lysts ex­pect an­oth­er ad­comm for Sarep­ta’s newest pitch, and the pan­el is like­ly to dis­cuss heart in­flam­ma­tion as­so­ci­at­ed with the com­pa­ny’s lat­est tri­al. Though the ef­fi­ca­cy da­ta proved large­ly pos­i­tive, a new oc­cur­rence of my­ocardi­tis in a sep­a­rate co­hort of old­er pa­tients (and clas­si­fied as a se­ri­ous ad­verse event) cloud­ed the read­out.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Luke Miels, GSK chief commercial officer

Lend­ing a hand to a biotech in trou­ble, GSK drops $75M cash to add late-stage an­tibi­ot­ic to port­fo­lio

GSK likes to take pride in being one of the few Big Pharma players still active in antibiotics R&D. And that means keeping tabs on what the field has to offer.

In a move to beef up the late-stage pipeline, GSK is licensing a late-stage antibiotic candidate from Spero Therapeutics. In doing so, it’s coming to the rescue of a struggling biotech that’s crumbled in the wake of an FDA rejection and raised doubts about its ability to carry on.

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David Chang, Allogene CEO (Jeff Rumans)

Servi­er cuts off col­lab­o­ra­tion agree­ment with Al­lo­gene on CD19 prod­ucts, send­ing shares sput­ter­ing

Allogene Therapeutics said in an SEC filing today that French partner Servier has cut off its involvement in a partnership developing therapies directed against CD19, including the most advanced candidates in Allogene’s pipeline.

Shares of Allogene $ALLO, an outfit run by Kite vets Arie Belldegrun and research chief David Chang, fell by almost 10% on Wednesday, even as the San Francisco-based company said that Servier’s discontinuation “does not otherwise affect our current exclusive license for the development and commercialization of CD19 Products in the United States.”

Lina Khan, FTC chair (Graeme Sloan/Sipa USA/Sipa via AP Images)

FTC chair Lina Khan to Sen­ate: Big Phar­ma M&A is still a pri­or­i­ty tar­get

As the Federal Trade Commission has already sought new ideas for analyzing pharma mergers, FTC chair Lina Khan reiterated Tuesday to a Senate subcommittee that reviewing Big Pharma mergers is a priority.

While comparing this merger analysis in the pharma space to the study of public utilities in the 1930s “that exposed rampant financial fraud,” Khan said in prepared testimony that the commission is going to target unlawful conduct.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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