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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

UP­DAT­ED: EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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