News brief­ing: A sweep of CHMP OKs for Al­ny­lam, Kite, No­var­tis and oth­ers; Sanofi's big can­cer play gets OK from UK watch­dog

At this month’s meet­ing, the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use is­sued rec­om­men­da­tions of ap­proval for 10 drugs. They in­clude:

  • Oxlu­mo (lumasir­an), Al­ny­lam’s RNAi can­di­date that snagged pri­or­i­ty re­view from the FDA back in May. In June, the biotech re­leased piv­otal da­ta which showed that 21 of 25 pa­tients with PH1 (pri­ma­ry hy­per­ox­aluria type 1), a rare liv­er con­di­tion, saw their ox­alate lev­els fall to near the nor­mal range af­ter be­ing treat­ed with lumasir­an, and just over half had num­bers in­side the nor­mal range. That was com­pared to 0% in the place­bo group, ac­cord­ing to Al­ny­lam.
  • Kite’s Tecar­tus (KTE-X19), a CAR-T ther­a­py ap­proved by the FDA in Ju­ly to treat adult pa­tients with re­lapsed or re­frac­to­ry man­tle cell lym­phoma, a rare form of NHL. Ken Takeshi­ta, head of glob­al de­vel­op­ment at Gilead sub­sidiary Kite Ther­a­peu­tics, boast­ed that the drug’s da­ta were bet­ter than the com­pa­ny’s CAR-T drug Yescar­ta, which was ap­proved in 2017. A com­pa­ny spokesper­son said in Ju­ly that the drug will be sold for $373,000.
  • No­var­tis’ Leqvio (in­clisir­an), the LDL-C drug that was for­mer­ly a Med­i­cines Com­pa­ny can­di­date. An Au­gust read-out from two Phase III tri­als showed con­sis­ten­cy in ef­fi­ca­cy and safe­ty among pa­tients with both hy­per­lipi­demia and ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease de­spite statin ther­a­py, ac­cord­ing to the com­pa­ny. Around 88% of pa­tients hit 50% re­duc­tion of LDL-C in at least one point dur­ing the 17-month study. Af­ter the 17-month mark, al­most two-thirds of pa­tients (66.4%) had a 50% re­duc­tion com­pared to 2.5% per­cent in the place­bo group.
  • Or­chard Ther­a­peu­tics’ Lib­meldy for metachro­mat­ic leukody­s­tro­phy
  • Janssen’s Rekam­bys for hu­man im­mun­od­e­fi­cien­cy virus type 1 (HIV-1)
  • Vi­iV Health­care’s Vo­cabria for HIV-1
  • Zo­genix’s Fin­tepla for the treat­ment of seizures as­so­ci­at­ed with Dravet syn­drome
  • Aim­mune’s Pal­forzia for de­sen­si­tis­ing chil­dren and ado­les­cents to peanut al­ler­gy
  • As­traZeneca’s Trix­eo Aeros­phere for the main­te­nance treat­ment of chron­ic ob­struc­tive pul­monary dis­ease
  • My­lan’s lenalido­mide for the treat­ment of mul­ti­ple myelo­ma and fol­lic­u­lar lym­phoma

Nicole De­Feud­is 

Sanofi’s big can­cer play gets the OK from UK price watch­dog

Sanofi won FDA and EMA ap­proval for their first home­grown can­cer drug in a decade ear­li­er this year. To­day, they se­cured an­oth­er vic­to­ry as a UK drug pric­ing watch­dog cleared the ther­a­py for use in the UK.

The mul­ti­ple myelo­ma drug nabbed the rec­om­men­da­tion af­ter show­ing a pro­gres­sion-free sur­vival of 11.5 months in piv­otal tri­als, com­pared to 6.5 months for the stan­dard-of-care. Be­cause full da­ta on sur­vival ben­e­fit are not yet avail­able, the coun­cil, known as NICE, did not rec­om­mend it for “rou­tine” NHS cov­er­age. How­ev­er, it rec­om­mend­ed the drug for in­clu­sion in the Can­cer Drugs Fund, a fund set up to help cov­er costs of can­cer drugs that are not cost ef­fec­tive.

Sanofi is tak­ing on J&J and their mul­ti­ple myelo­ma block­buster Darza­lex as they mar­ket their first big on­col­o­gy win in years.

Ja­son Mast

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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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.

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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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 million-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.

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.

Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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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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