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

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.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Covid-19 roundup: WSJ of­fers in­side look at Ox­ford-As­traZeneca deal, in­clud­ing fi­nan­cial terms; Lil­ly plant un­der scruti­ny again

Oxford scientists developing a Covid-19 vaccine are working with British drugmaker AstraZeneca, and together the pair have become one of the frontrunners in the race to end the pandemic. But a new Wall Street Journal report out Wednesday offered a behind-the-scenes look at how that deal came together in the wake of a scholar-led revolt over a potential collaboration with Merck, and included previously unreported financial terms of the AstraZeneca deal.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.

Peter Kolchinsky and Raj Shah (file photo)

Pe­ter Kolchin­sky and Raj Shah's RA Cap­i­tal has $461M more to play with, af­ter 'rapid' in­vest­ment in the last 15 months

Just over 15 months after launching its first venture fund, RA Capital Management is ready for more. And this time the firm is bringing an even bigger load of cash to the table.

Announcing the close of its Nexus II fund on Wednesday, RA said it raised $461 million for investments in private companies across the biotech industry. The first venture fund, which raised $300 million, has churned through roughly 80% of its capital already, a pace that managing partner Raj Shah called unusually quick.

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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Philipp Spycher, Araris CEO

Promis­ing bet­ter link­er tech to ADC field, Araris has 'very, very am­bi­tious' plans for the clin­ic

A couple months after raising CHF 2.5 million ($2.76 million) in initial seed funding, one-year-old Araris Biotech is topping off the round with another CHF 12.7 million ($14 million).

The Paul Scherrer Institute and ETH Zurich spinout now has CHF 15.2 million to work with, and CEO Philipp Spycher has big plans. He hopes to bring one of the company’s antibody-drug conjugates (ADC) to the clinic by late 2022 or early 2023. “It’s very, very ambitious, but we are very optimistic that we actually can make it,” he said.

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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