Jackie Fouse, Agios CEO

Af­ter sell­ing off Tib­so­vo and en­tire can­cer pipeline, Agios nets first ap­proval for new 'an­chor pro­duc­t'

Agios’ long-term plan is start­ing to come in­to fo­cus, as the biotech won its first FDA ap­proval since piv­ot­ing away from on­col­o­gy Thurs­day.

The FDA ap­proved Agios’ mi­tapi­vat in a rare blood dis­or­der known as pyru­vate ki­nase de­fi­cien­cy, which man­i­fests as red blood cells be­ing de­stroyed faster than they can be made. Agios won the OK in adult pa­tients and tout­ed mi­tapi­vat, to be brand­ed as Pyrukynd, as the con­di­tion’s first ap­proved dis­ease-mod­i­fy­ing ther­a­py.

In a Fri­day morn­ing con­fer­ence call, Agios an­nounced the av­er­age an­nu­al price (WAC) of the drug would be $334,880 and won’t be raised for five years.

Thurs­day’s ap­proval marks a first step to­ward Agios mak­ing good on a promise that mi­tapi­vat will be­come the biotech’s “an­chor prod­uct,” as CEO Jack­ie Fouse told End­points News in De­cem­ber 2020. It fol­lows Agios’ de­ci­sion to sell off its en­tire on­col­o­gy port­fo­lio, in­clud­ing the pi­o­neer­ing drug Tib­so­vo, to Servi­er for $1.8 bil­lion cash up­front.

Soon af­ter the deal closed, Agios be­gan im­me­di­ate­ly buy­ing up its out­stand­ing shares, re­pur­chas­ing a near­ly $350 mil­lion stake from Bris­tol My­ers Squibb last April — good for about 10%.

The piv­ot to mi­tapi­vat had been a risky propo­si­tion in the eyes of some an­a­lysts, as the PKR ac­ti­va­tor ap­peared to be less im­pres­sive than oth­er com­peti­tors in the space. No­tably, the drug’s ASH da­ta from 2020 un­der­whelmed in sick­le cell dis­ease, a view reaf­firmed in 2021, with a ri­val from For­ma seem­ing­ly prov­ing more ef­fi­ca­cious (in­sert your fa­vorite cross-tri­al com­par­i­son caveat here).

Sick­le cell is like­ly to be the biggest sell­ing in­di­ca­tion for each of these can­di­dates, though SVB Leerink not­ed Agios’ peak sales could hit $1.7 bil­lion com­bined in SCD, pyru­vate ki­nase de­fi­cien­cy and tha­lassemia. The two com­pa­nies are tak­ing dif­fer­ent paths to the clin­ic in SCD, with For­ma shoot­ing for ac­cel­er­at­ed ap­proval and Agios aim­ing at reg­u­lar ap­proval, ac­cord­ing to a re­port from Eval­u­ate Van­tage.

Ques­tions sur­round­ing the piv­ot could re­main un­til the rest of the SCD da­ta play out, and Agios is cur­rent­ly study­ing mi­tapi­vat in a Phase II/III study for the con­di­tion. There are al­so two Phase III tri­als be­ing con­duct­ed in tha­lassemia pa­tients who are and are not reg­u­lar­ly trans­fused, and two more Phase III stud­ies in chil­dren with PK de­fi­cien­cy are ex­pect­ed to launch in mid-2022.

The ap­proval was based on two Phase III stud­ies look­ing at PK de­fi­cien­cy in adults who reg­u­lar­ly re­ceive blood trans­fu­sions and those who do not. Six­teen of 40 non-reg­u­lar­ly trans­fused pa­tients achieved a he­mo­glo­bin re­sponse, com­pared to ze­ro in the place­bo arm.

In ad­di­tion, in those un­der­go­ing fre­quent trans­fu­sions, nine of 27 pa­tients achieved at least a 33% re­duc­tion in trans­fu­sion bur­den dur­ing the 24-week treat­ment pe­ri­od. Fig­ures were com­pared to per­son­al his­tor­i­cal da­ta rather than place­bo.

Due to a sourc­ing er­ror, a pre­vi­ous ver­sion of this ar­ti­cle in­cor­rect­ly stat­ed the num­ber of pa­tients en­rolled and the num­ber of re­spon­ders in Agios’ PK de­fi­cien­cy stud­ies. In non-reg­u­lar­ly trans­fused pa­tients, 16 of 40 pa­tients saw a re­sponse, while in reg­u­lar­ly trans­fused pa­tients, nine of 27 pa­tients achieved at least a 33% re­duc­tion dur­ing the treat­ment pe­ri­od, not six of 16 and three of nine, re­spec­tive­ly. 

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

CMS spent more than $18B in four years on ac­cel­er­at­ed ap­provals with in­com­plete con­fir­ma­to­ry tri­als, in­spec­tor gen­er­al finds

The battle over whether and how to reform the FDA’s accelerated approval pathway is heating up again, just as the Senate punted any talks until the lame duck session just before the end of the year.

On Thursday, HHS’ inspector general released a new report reiterating concerns, also noted recently by the FDA’s Oncology Center of Excellence, about delayed or slowed confirmatory trials that are necessary to prove that the accelerated approvals were worth their salt in the first place.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA (Sipa via AP Images)

Sen­ate pass­es bill to re­work an­i­mal test­ing re­quire­ments for drug de­vel­op­ers

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would alter a federal mandate for animal testing on new drugs, but stops short of removing animal testing entirely.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.

Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.