GSK's asth­ma bi­o­log­ic Nu­cala is one step clos­er to ap­proval in key chron­ic rhi­nos­i­nusi­tis pop­u­la­tion

Months af­ter GSK’s Nu­cala cleared in a piv­otal rare blood dis­or­der study, the asth­ma bi­o­log­ic has scored in a late-stage tri­al in chron­ic rhi­nos­i­nusi­tis pa­tients with nasal polyps.

The British drug­mak­er on Fri­day dis­closed da­ta from the SYNAPSE study, which test­ed Nu­cala (al­so known as mepolizum­ab) against a place­bo on top of stan­dard-of-care in more than 400 pa­tients, all of whom had a his­to­ry of pre­vi­ous surgery (ap­prox­i­mate­ly one in three had ≥3 surg­eries) and re­quired surgery due to se­vere symp­toms and big­ger polyps.

Nu­cala met both the main goals demon­strat­ing sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in both the size of nasal polyps (p<0.001) at week 52 and in nasal ob­struc­tion (p<0.001) dur­ing weeks 49 to 52. A key sec­ondary end­point, timed to first ac­tu­al nasal surgery up to week 52, was al­so sta­tis­ti­cal­ly sig­nif­i­cant — show­ing a re­duc­tion of 57% (p=0.003) ver­sus place­bo, GSK said.

“These re­sults show mepolizum­ab rep­re­sents a promis­ing al­ter­na­tive for pa­tients who are caught in a cy­cle of surg­eries. Each surgery car­ries risks and can dis­able the pa­tient for sev­er­al weeks,” Claus Bachert, pro­fes­sor and Chief of Clin­ics ENT De­part­ment at Ghent Uni­ver­si­ty said in a state­ment.

GSK said it plans to sub­mit ap­pli­ca­tions to mar­ket the drug in the pa­tient pop­u­la­tion lat­er this year.

Chron­ic rhi­nos­i­nusi­tis (CRS), char­ac­ter­ized by in­flam­ma­tion of the nose and si­nus cav­i­ties, is a com­mon con­di­tion, af­fect­ing al­most 12% of the glob­al adult pop­u­la­tion. About a fifth of pa­tients with CRS have nasal polyps, which are be­nign growths in the nasal cav­i­ties, ac­cord­ing to the Amer­i­can Acad­e­my of Al­ler­gy, Asth­ma & Im­munol­o­gy.

First ap­proved in 2015 for se­vere eosinophilic asth­ma, Nu­cala tar­gets IL-5. The drug is be­lieved to work by pre­vent­ing IL-5 from bind­ing to its re­cep­tor on the sur­face of eosinophils di­min­ish­ing blood eosinophils, but not erad­i­cat­ing them al­to­geth­er, ac­cord­ing to GSK.

Last year, the FDA spurned GSK’s ap­pli­ca­tion to ex­pand the drug’s use in pa­tients with COPD, af­ter it failed one late-stage test but cleared an­oth­er. Af­ter an ex­pert pan­el vot­ed 16 to 3 against ap­proval, the re­jec­tion was wide­ly an­tic­i­pat­ed. In No­vem­ber, the mon­o­clon­al an­ti­body helped pa­tients with hy­per­e­osinophilic syn­drome (HES) — a group of life-threat­en­ing in­flam­ma­to­ry dis­or­ders char­ac­ter­ized by a per­sis­tent over­pro­duc­tion of a type of white blood cell called eosinophils — in a 108-pa­tient late-stage study.

Last year, Re­gen­eron and Sanofi se­cured the first ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps with their flag­ship IL-4 and IL-13 bi­o­log­ic Dupix­ent. No­var­tis’ best­seller Xo­lair — which is en­gi­neered to im­munoglob­u­lin E — is ex­pect­ed to win ap­proval in the pa­tient pop­u­la­tion this year.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntington’s disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a “big opportunity” in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

Glax­o­SmithK­line's vac­cines group aims for a first as it kicks off PhI­II RSV stud­ies

One of GlaxoSmithKline’s big projects at its global vaccine R&D center in Rockville, MD is set to enter Phase III after passing early-stage tests with flying colors.

Eyeing the wide-open respiratory syncytial virus (RSV) space, GSK is pushing two different vaccine candidates: GSK3888550A is designed to confer protection to infants via maternal immunization, while GSK3844766A is meant for the elderly.

Pur­due Phar­ma signs guilty plea, preps $8B+ set­tle­ment on Oxy con­tro­ver­sy — re­port; Flag­ship brings in a comms chief

Purdue Pharma may soon be signing off on a guilty plea and an $8 billion-plus settlement to wrap up its controversial role distributing OxyContin.

The AP has the breaking story this morning.

Purdue filed for bankruptcy last year, along with Insys and followed by Mallinckrodt, as it navigated its way through a blizzard of litigation surrounding Oxy, which triggered an epidemic of abuse around the country.