Or­chard dis­creet­ly ax­es li­cense for 'bub­ble boy' gene ther­a­py, de­spite re­cent pos­i­tive long-term da­ta up­date

Less than a month af­ter pub­lish­ing pos­i­tive long-term da­ta for an ex­per­i­men­tal gene ther­a­py, Or­chard Ther­a­peu­tics has qui­et­ly ter­mi­nat­ed its li­cens­ing agree­ment over the pro­gram.

In an 8-K fil­ing to the SEC last Fri­day, Or­chard re­port­ed that it is end­ing the li­cense re­lat­ed to its OTL-101 can­di­date, in de­vel­op­ment to treat adeno­sine deam­i­nase se­vere com­bined im­mun­od­e­fi­cien­cy or ADA-SCID, known col­lo­qui­al­ly as “bub­ble boy syn­drome.” It’s a rare ge­net­ic dis­ease where pa­tients have al­most no im­mune sys­tem what­so­ev­er.

The move comes af­ter Or­chard pub­lished long-term re­sults in the New Eng­land Jour­nal of Med­i­cine show­ing all 50 pa­tients who re­ceived the treat­ment were still alive af­ter at least two years. Event-free sur­vival was al­so high, with 97% of US pa­tients and 95% of UK par­tic­i­pants not need­ing to re­turn to pri­or ther­a­pies.

Or­chard had been de­vel­op­ing the ther­a­py af­ter sign­ing the agree­ment with UCLA and Uni­ver­si­ty Col­lege Lon­don back in Feb­ru­ary 2016. The biotech has an­oth­er ther­a­py for ADA-SCID known as Strimvelis, ap­proved in Eu­rope in 2016, but halt­ed dos­ing af­ter a pa­tient treat­ed un­der com­pas­sion­ate use be­gan see­ing treat­ment for leukemia.

The idea be­hind OTL-101 is to use a lentivirus with hematopoi­et­ic stem cells, ad­min­is­tered ex vi­vo, to in­sert a func­tion­al copy of the ADA gene in­to pa­tients’ cells. Or­chard had been look­ing for an ap­proval for a while, hav­ing pre­vi­ous­ly won break­through ther­a­py des­ig­na­tion for OTL-101 from the FDA.

An FDA green­light had elud­ed Or­chard with the Covid-19 pan­dem­ic forc­ing them to push back plans to sub­mit a rolling ap­proval ap­pli­ca­tion. But last week’s SEC re­port may have been in the works for at least a year.

Back in May 2020, Or­chard an­nounced a new op­er­a­tional strat­e­gy in­volv­ing lay­ing off 25% of its staff and re­struc­tur­ing its pipeline to fo­cus less on rare dis­eases. Part of that re­struc­tur­ing, Or­chard said at the time, was to re­duce in­vest­ment in OTL-101 and an­oth­er pro­gram — steer­ing re­sources in­stead to­ward four oth­er can­di­dates clas­si­fied as “top near-term pri­or­i­ties.”

The pro­grams in­volved are be­ing set up to treat: metachro­mat­ic leukody­s­tro­phy (OTL-200), Wiskott-Aldrich syn­drome (OTL-103), Mu­copolysac­cha­ri­do­sis type I (OTL-203) and Mu­copolysac­cha­ri­do­sis type II­IA, al­so known as San­fil­ip­po syn­drome type A (OTL-201).

Or­chard went pub­lic in 2018, rid­ing a $200 mil­lion IPO af­ter two oth­er nine-fig­ure rounds with a $110 mil­lion Se­ries B and $150 mil­lion Se­ries B. The biotech worked with Glax­o­SmithK­line, too, ac­quir­ing the Big Phar­ma’s rare dis­ease unit — in­clud­ing Strimvelis — in ex­change for rough­ly 20% eq­ui­ty, al­so in 2018. The lack of an FDA-ap­proved prod­uct at this stage, how­ev­er, has left an­a­lysts with ques­tions.

So­cial im­age: Bob­by Gas­par, Or­chard Ther­a­peu­tics CEO

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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