Levi Garraway, Roche CMO

Roche snags an FDA ap­proval for an ad­vanced melanoma drug cock­tail — but it's up against some fierce com­pe­ti­tion

Roche’s PD-L1 in­hibitor Tecen­triq snagged an FDA ap­proval to treat cer­tain ad­vanced melanoma pa­tients in a three-drug cock­tail, pro­vid­ing an­oth­er op­tion for pa­tients with a typ­i­cal­ly poor prog­no­sis.

In a Phase III IM­spire150 study, Tecen­triq com­bined with Cotel­lic (co­bime­tinib) and Zelb­o­raf (ve­mu­rafenib) sig­nif­i­cant­ly beat a place­bo mix in ex­tend­ing pro­gres­sion-free sur­vival in BRAF V600 mu­ta­tion-pos­i­tive ad­vanced melanoma pa­tients. Par­tic­i­pants on Tecen­triq lived a me­di­an 15.1 months with­out wors­en­ing dis­ease, com­pared to 10.6 months in pa­tients on Cotel­lic, Zelb­o­raf, and the place­bo.

Those are all per­fect­ly good num­bers, but they’re like­ly not good enough to move the nee­dle in this mar­ket niche, where Roche will butt heads with the dom­i­nant ri­vals who re­shaped treat­ment stan­dards in the field.

Mer­ck’s Keytru­da and Bris­tol My­ers Squibb’s Op­di­vo — both of which got DA green lights in 2014 — pose tough com­pe­ti­tion. When re­searchers test­ed the ef­fec­tive­ness of Cotel­lic and Tecen­triq alone in treat­ment-naive pa­tients, they found the cock­tail didn’t mea­sure up to Keytru­da’s stan­dard of care. And a five-year check-in on a Bris­tol My­ers Squibb clin­i­cal tri­al showed pa­tients who were giv­en both Op­di­vo and Yer­voy had a 52% sur­vival rate, as op­posed to 44% for just Op­di­vo, or 26% for just Yer­voy.

Stifel an­a­lyst Stephen Wil­ley has said the Tecen­triq triple-drug cock­tail is more of a “check the box” than “ex­pand the mar­ket” move.

“When re­ceiv­ing a can­cer im­munother­a­py com­bined with tar­get­ed ther­a­pies, pa­tients with BRAF V600 mu­ta­tion-pos­i­tive ad­vanced melanoma were able to live for more than 15 months with­out their dis­ease wors­en­ing,” Levi Gar­raway, Roche CMO and head of glob­al prod­uct de­vel­op­ment, said in a pre­pared state­ment. “To­day’s FDA ap­proval of this Tecen­triq com­bi­na­tion rep­re­sents an im­por­tant step for­ward for many pa­tients liv­ing with ad­vanced melanoma.”

Tecen­triq is cur­rent­ly pre­scribed to treat non-small cell lung can­cer. Last year, it be­came the first im­munother­a­py ap­proved for triple-neg­a­tive breast can­cer pa­tients who are metasta­t­ic and PD-L1 pos­i­tive. In cur­rent and fu­ture Phase III tri­als, Genen­tech will test Tecen­triq’s use alone and in cock­tails for var­i­ous lung, gen­i­touri­nary, skin, breast, gas­troin­testi­nal, gy­ne­co­log­i­cal and head and neck can­cers.

Cotel­lic, an Ex­elix­is drug, is be­ing co-de­vel­oped with Roche.  The Swiss phar­ma al­so has a clin­i­cal col­lab­o­ra­tion agree­ment to de­vel­op Zelb­o­raf, which was a Plexxikon drug be­fore the com­pa­ny was ac­quired by Dai­ichi Sankyo.

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.

Lit­tle Zosano takes an­oth­er beat­ing as the FDA slaps down their ap­pli­ca­tion for a mi­graine patch

Zosano $ZSAN has officially come up short in its bid to develop a migraine patch.

The FDA rejected the company’s application to repurpose the triptan zolmitriptan in a new delivery system as Qtrypta, Zosano said Wednesday morning, issuing a CRL for the microneedle patch. Regulators cited inconsistent exposure levels across multiple clinical trials as the main reason for the thumbs down.

Investors did not take too kindly to the news, with Zosano shares plunking down around 25%. The company is requesting a Type A meeting to “provide clarity on the next steps for the program,” CEO Steven Lo said in a statement.

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.