Up­dat­ed: Cel­gene, Agios win a land­mark FDA OK for new AML drug Id­hi­fa

David Schenkein, Agios CEO

Cel­gene and Agios didn’t have to wait un­til the PDU­FA date at the end of Au­gust to get a speedy FDA de­ci­sion on enasi­denib (AG-221), their new ther­a­py for acute myeloid leukemia. The FDA cut to the chase and ap­proved it to­day.

Cel­gene $CELG re­worked its deal with Agios $AGIO last year, but kept its hands on its rights to AG-221 in pa­tients with ad­vanced hema­to­log­ic ma­lig­nan­cies with an IDH2 mu­ta­tion. In turn, Agios stayed ra­zor fo­cused on grab­bing the ear­li­est pos­si­ble ap­proval, hus­tling Phase I/II da­ta straight to reg­u­la­tors for an ac­cel­er­at­ed re­view.

The drug will now be sold as Id­hi­fa, con­cen­trat­ing on about 9% to 13% of the AML mar­ket.

“The use of Id­hi­fa was as­so­ci­at­ed with a com­plete re­mis­sion in some pa­tients and a re­duc­tion in the need for both red cell and platelet trans­fu­sions,” not­ed the FDA’s Richard Paz­dur in herald­ing the OK to­day. The drug will be used with the Re­al­Time IDH2 As­say, which is used to de­tect spe­cif­ic mu­ta­tions in the IDH2 gene in pa­tients with AML.

It won’t come cheap. From Cel­gene:

The ap­proval of ID­HI­FA rep­re­sents a new, tar­get­ed ther­a­py for a sub­type of AML with an ex­treme­ly rare ge­net­ic mu­ta­tion. The 8-19% of AML pa­tients that have an IDH2 mu­ta­tion rep­re­sent about 1,200 to 1,500 in­di­vid­u­als in the U.S. These pa­tients are liv­ing with dis­ease that has a poor prog­no­sis and no stan­dard of care and no oth­er cur­rent­ly ap­proved op­tions un­til to­day. Pair­ing this ther­a­py with an ap­proved com­pan­ion di­ag­nos­tic al­so sig­nif­i­cant­ly im­proves iden­ti­fi­ca­tion of el­i­gi­ble pa­tients who could ben­e­fit from this new­ly ap­proved treat­ment.

The month­ly whole­sale ac­qui­si­tion cost of ID­HI­FA is $24,872. In the piv­otal study, the me­di­an time on ther­a­py for pa­tients was 4.3 months

Cel­gene first al­lied it­self with Agios back in 2010, part­ner­ing on its work in can­cer me­tab­o­lism. That deal led Cel­gene to grab world­wide rights to AG-221 in 2014. Cel­gene hand­ed back rights to AG-120, an IDH1 in­hibitor, but al­so an­ted up an­oth­er $200 mil­lion to grab op­tions on a slate of ex­per­i­men­tal drugs in the biotech’s pipeline.

The re­la­tion­ship paid off nice­ly for Cel­gene. The jour­ney from first-in-hu­man test­ing to a reg­u­la­to­ry fil­ing took just three years, an in­cred­i­bly fast speed in R&D. And Agios plans to do that again with oth­er drugs in its pipeline. Leerink’s says it’s a big step for both com­pa­nies, which will be fol­lowed close­ly dur­ing the mar­ket launch.

This is the first drug to emerge from Cel­gene’s col­lab­o­ra­tion with Agios, and from Agios’ re­search plat­form in can­cer me­tab­o­lism, and al­so the first drug to be ap­proved from Cel­gene’s net­worked re­search arrange­ments. It pro­vides im­por­tant val­i­da­tion for both plat­form and strat­e­gy, and in­vestors will close­ly mon­i­tor the drug’s com­mer­cial per­for­mance to as­sess how well these ad­vances trans­late in­to com­mer­cial suc­cess. Cel­gene is the pri­ma­ry com­mer­cial­iz­ing par­ty in the US, al­though Agios has opt­ed in to co-com­mer­cial­ize the prod­uct in the US. Cel­gene has sug­gest­ed that they will add ap­prox­i­mate­ly 30 head­count to sup­port the prod­uct’s com­mer­cial­iza­tion on the US. Cel­gene es­ti­mates that there is a tar­get­ed pa­tient pop­u­la­tion of 1,200-1,500 pa­tients in the US and ex­pects the du­ra­tion of treat­ment to ap­prox­i­mate the 4 months ob­served in the phase I/II tri­al.

Leerink’s Michael Schmidt al­so took a step back and sized up the im­pli­ca­tions for the biotech, now that it owns the IDH1 ther­a­py. It all adds up to a siz­able op­por­tu­ni­ty, he notes:

In our view, FDA-ap­proval of Id­hi­fa al­so re­duces reg­u­la­to­ry risk for ivosi­denib in AML, the lat­ter be­ing pro­pri­etary to AGIO and hence rep­re­sent­ing a more sig­nif­i­cant val­ue dri­ver for the com­pa­ny. AGIO’s IDH1 & 2 in­hibitors ad­dress a ~$270M and ~$370M glob­al mar­ket op­por­tu­ni­ty in re­lapsed/re­frac­to­ry AML, re­spec­tive­ly, based on our up­dat­ed es­ti­mates; po­ten­tial front-line use in com­bi­na­tion with ap­proved or in­ves­ti­ga­tion­al agents re­flects a sig­nif­i­cant­ly larg­er $1Bn+ com­mer­cial op­por­tu­ni­ty based on our re­cent due dili­gence should on­go­ing and planned front-line clin­i­cal tri­als suc­ceed.

CEO David Schenkein is fond of re­call­ing Agios start­ed out with lit­tle more than a blank sheet of pa­per. Since then, the pages have been filled with high-pro­file part­ner­ships, an IPO for its po­ten­tial cut­ting-edge work and now its first new drug ap­proval. It’s a wa­ter­shed mo­ment for this biotech.

 

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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Philipp Spycher

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.

Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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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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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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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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