GSK aban­dons Ion­is' FDA-ready in­ot­ersen as new CEO sweeps out rare dis­ease ef­forts

Just three months af­ter Io­n­is spelled out pos­i­tive Phase III ef­fi­ca­cy da­ta on its top prospect in­ot­ersen, Glax­o­SmithK­line is sweep­ing their al­liance on the drug out the back door, along with rights to an­oth­er treat­ment they have rights to.

Em­ma Walm­s­ley, GSK CEO

It’s a stun­ning 11th hour re­ver­sal for Io­n­is $IONS, which says it now plans to push for an FDA ap­proval of in­ot­ersen on its own this fall as it thinks through whether it will com­mer­cial­ize or co-com­mer­cial­ize in the US on its own.

Ac­cord­ing to Io­n­is, GSK ex­ecs de­cid­ed to aban­don in­ot­ersen, de­signed to treat rare cas­es of TTR amy­loi­do­sis (AT­TR), with an ini­tial fo­cus on polyneu­ropa­thy due to hered­i­tary TTR amy­loi­do­sis (hAT­TR-PN). That drug is a close­ly-watched ri­val to Al­ny­lam’s lead drug patisir­an. Al­so out: IO­N­IS-FB-LRx, now at the thresh­old of Phase II for dry age-re­lat­ed mac­u­lar de­gen­er­a­tion, as GSK turns its back on rare dis­eases as part of its top-to-bot­tom R&D re­vamp un­der new CEO Em­ma Walm­s­ley.

“Our goals for in­ot­ersen are to max­i­mize its com­mer­cial suc­cess and op­ti­mize our com­mer­cial par­tic­i­pa­tion. To achieve these goals, we are ac­tive­ly con­sid­er­ing form­ing a com­mer­cial sub­sidiary to com­mer­cial­ize or co-com­mer­cial­ize in­ot­ersen in North Amer­i­ca, as well as oth­er op­tions. Our re­cent ex­pe­ri­ence build­ing a com­mer­cial sub­sidiary has pre­pared us for this op­por­tu­ni­ty. We have sub­stan­tial in­ter­est from po­ten­tial part­ners and are in dis­cus­sions with sev­er­al par­ties. We be­lieve that, to­geth­er with the right com­mer­cial part­ner, we can max­i­mize the com­mer­cial suc­cess of the drug world­wide,” said Sarah Boyce, chief busi­ness of­fi­cer of Io­n­is Phar­ma­ceu­ti­cals.

Sarah Boyce, Io­n­is CBO

Fil­ing for ap­proval and get­ting it are two dif­fer­ent things, though. Io­n­is’ late-stage ef­fort has al­so been flagged for se­ri­ous safe­ty con­cerns which could al­so ham­per its quest for an OK, as well as pos­si­bly sign­ing up a new com­mer­cial­iza­tion part­ner. Paul Mat­teis at Leerink swift­ly not­ed:

Nor­mal­ly in such a sit­u­a­tion like this in­vestors might wor­ry that there’s some not-yet seen-el­e­ment of the full da­ta that may be prob­lem­at­ic or dis­con­cert­ing. To this point — giv­en the well doc­u­ment­ed safe­ty is­sues as­so­ci­at­ed with In­ot­ersen (i.e., throm­bo­cy­tope­nia), GSK’s de­ci­sion may in­crease in­vestor scruti­ny on TTR­rx’s safe­ty pro­file as it is de­pict­ed in ad­di­tion­al full da­ta pre­sen­ta­tions this Fall (Amer­i­can Neu­ro­log­i­cal As­so­ci­a­tion, Oct. 15-17, oth­ers). Our mod­el cur­rent­ly as­sumes a 75%/25% AL­NY (MP) ver­sus IONS split, and while we are en­cour­aged that IONS’s plans for a YE17 MAA fil­ing are still on track, we are a bit con­cerned that GSK’s de­ci­sion may al­so put IONS be­hind the curve with re­spect to launch pre­pared­ness.

Stan­ley Crooke, Io­n­is CEO

Two oth­er al­liances will con­tin­ue. GSK is cur­rent­ly in Phase II with IO­N­IS-HB­VRx and IO­N­IS-HBV-LRx.

CEO Stan­ley Crooke not­ed that Io­n­is is now “ac­cel­er­at­ing the ex­pan­sion of our TTR pro­gram for pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis and the de­vel­op­ment of our LI­CA fol­low-on drug. Our ex­pe­ri­ence in the com­plet­ed Phase 3 NEU­RO-TTR study pro­vides im­por­tant in­for­ma­tion to aid in de­sign of a study in pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis. We have al­ready iden­ti­fied a more po­tent and con­ve­nient LI­CA fol­low-on and we ex­pect de­vel­op­ment of the LI­CA drug to al­so pro­ceed rapid­ly.”

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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In a win for Re­gen­eron, No­var­tis' sy­ringe for AMD drug de­clared 'un­patentable'

Regeneron has won a patent case against Swiss pharma giant Novartis over the delivery system for its eye drug Eylea.

The US Patent Trial and Appeal Board ruled that Novartis’ pre-filled syringe for injecting its eye medication Lucentis was “unpatentable” and handed the victory to Regeneron and its AMD drug Eylea.

In the initial complaint in 2020, Novartis alleged to the US International Trade Commission that certain pre-filled syringes for the intravitreal injection, and ultimately Regeneron’s delivery system for Eylea, were infringing on Novartis’ patent. Regeneron filed a petition to review Novartis’ claims in 2021.

'Tis the sea­son: GSK ad­dress­es win­ter virus surges with celebri­ty and in­flu­encer vac­cine aware­ness cam­paigns

GSK is rounding up the usual suspects this winter — flu, respiratory syncytial and even shingles viruses — for multiple marketing efforts all aimed at encouraging vaccinations.

Mom influencers take center stage in its “Flu is a Family Affair” campaign to reach family decision-makers or “chief health officers.” GSK is asking them in the digital campaign to take care of themselves, and take the family along, when they go to the pharmacy or doctor’s office for a flu vaccine.

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Bris­tol My­ers claims win with CAR-T ther­a­py Breyanzi in leukemia

Bristol Myers Squibb is looking to expand Breyanzi into more indications — and the pharma’s newest data readout makes progress on that front.

The Big Pharma put out word Thursday that the CAR-T cell therapy met the primary endpoint of complete response rate compared to historical control in a subset of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) that were refractory to a BTK inhibitor and pretreated with a BCL-2 inhibitor.

FDA takes next step in Tor­rent Phar­ma­ceu­ti­cal­s' trou­bled In­dia plant saga, is­sues OAI

The FDA has handed Torrent Pharmaceuticals an official action indicated (OAI) status for a previously inspected manufacturing facility in India.

Torrent Pharma sent a letter to the National Stock Exchange of India earlier this week with word that the manufacturer has received a “communication from the FDA determining the inspection classification as ‘Official Action Indicated’ (OAI)” for one of its sites. An OAI classification from the FDA comes after the agency has completed an inspection and determines if the facility complies with the applicable laws and regulations. Being given an OAI classification means that regulatory or administrative actions will be recommended to Torrent. However, the details on the recommended actions have not been given.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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