Agios scores its sec­ond new drug ap­proval, ivosi­denib heads to the AML mar­ket

Agios has hit an­oth­er big goal in its decade-long track record. The FDA to­day sig­naled the ac­cel­er­at­ed ap­proval of ivosi­denib (AG-120) for ad­vanced acute myeloid leukemia.

David Schenkein

This marks the sec­ond drug ap­proval for Agios, a biotech com­pa­ny found­ed by David Schenkein, which he’s fond of say­ing got start­ed with a “blank sheet of pa­per.”

That sheet is get­ting quite crowd­ed now.

The new drug will be mar­ket­ed by Agios as Tib­so­vo, and the com­pa­ny is charg­ing $26,115 for a one-month sup­ply. Agios shares $AGIO were down just slight­ly — this was no great sur­prise — but the stock is up 56% 0f where it was a year ago.

Billed as “the first tar­get­ed treat­ment for R/R AML pa­tients with an IDH1 mu­ta­tion,” Agios sub­mit­ted it back in Feb­ru­ary with noth­ing but ear­ly-stage da­ta that had been on dis­play at ASH. 

The OK comes close to a year af­ter the biotech’s first ap­proval for Id­hi­fa, an IDH2 drug for an­oth­er slice of the AML mar­ket be­ing han­dled by its part­ners at Cel­gene. And just like Id­hi­fa, the FDA hur­ried this new ap­proval along with a pri­or­i­ty re­view.

To­geth­er, the two drugs cov­er 20% of the AML mar­ket.

This new drug was test­ed in an open la­bel study, with no con­trol arm. In­ves­ti­ga­tor found:

•CR+CRh rate of 32.8% (57 of 174 pa­tients) (95% CI: 25.8, 40.3).

•The CR rate was 24.7% (43 of 174 pa­tients) (95% CI 18.5, 31.8) and the CRh rate was 8% (14 of 174 pa­tients) (95% CI 4.5, 13.1).

•Me­di­an du­ra­tion of CR+CRh was 8.2 months (95% CI: range 5.6, 12 months).

•For pa­tients who achieved a CR or CRh, the me­di­an time to best re­sponse of CR or CRh was 2.0 months (range, 0.9 to 5.6 months).

•Among the 110 pa­tients who were de­pen­dent on red blood cell (RBC) and/or platelet trans­fu­sions at base­line, 41 (37.3%) be­came in­de­pen­dent of RBC and platelet trans­fu­sions dur­ing any 56-day post-base­line pe­ri­od.

“The FDA ap­proval of Tib­so­vo – our first whol­ly owned drug and the sec­ond ap­proved med­i­cine from our re­search plat­form in less than a year – is an in­cred­i­bly ex­cit­ing mile­stone for our com­pa­ny and, im­por­tant­ly, for the ap­prox­i­mate­ly 6-10% of AML pa­tients with an IDH1 mu­ta­tion who have been wait­ing for new treat­ment op­tions that work rad­i­cal­ly dif­fer­ent than con­ven­tion­al chemother­a­py,” said Schenkein.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (via YouTube)

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

CEO Grace Colón (InCarda)

Look­ing to re­pur­pose an old drug to treat ir­reg­u­lar heart­beats, In­Car­da rais­es $30M in first Se­ries C close

A little less than two years after completing its $42 million Series B round, InCarda has returned to the venture well.

The San Francisco-based biotech announced the first portion of its Series C on Wednesday, pulling in $30 million in new funding. Most of the money will give enough runway for InCarda’s InRhythm program, an inhaled therapeutic aiming to treat sudden episodes of irregular heartbeats, through its Phase II trials and prepare it for Phase III.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: FDA has fi­nal­ly post­ed dis­cus­sion items for to­mor­row's ad­comm; As­traZeneca could soon re­sume US vac­cine tri­als

It may have come a day late, but the FDA has finally posted the discussion items that its outside experts will review during tomorrow’s adcomm on the new wave of Covid-19 vaccines now in the clinic.

There are no specific vaccines or data to discuss. Instead, the agency wants feedback on its overall approach. And they’re willing to go late into the evening to get it.

Here’s the rundown:

1. Please discuss FDA’s approach to safety and effectiveness data as outlined in the respective guidance documents.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,100+ biopharma pros reading Endpoints daily — and it's free.

Un­fazed by PhII miss, Roche ush­ers Prothena's Parkin­son's drug in­to late-stage tri­al — a $60M move

Prothena’s prasinezumab may not have met the primary endpoint in Phase II, but its partners at Roche are seeing enough to move it into a late-stage trial for Parkinson’s disease.

The Phase IIb will build on the Phase II PASADENA study, adding a subgroup of early Parkinson’s patients on stable levodopa therapy to the population.

It’s a significant milestone for a $600 million deal that dates back to 2013, as dosing of the first patient — expected next year — will trigger a $60 million milestone payment to Prothena.