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. 

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Sen. Bill Cassidy (R-LA) (J. Scott Applewhite/AP Images)

Sen­ate un­veils its ver­sion of ac­cel­er­at­ed ap­proval re­forms as bi­par­ti­san duo calls on FDA and PTO to work to­geth­er

The Senate is joining its House counterparts and advancing accelerated approval pathway reforms to the FDA user fee legislation that must be signed by President Joe Biden before the end of September or else the FDA will have to start laying off its staff.

While Sen. Richard Burr (R-NC) warned yesterday that the user fee deals could be delayed by the infant formula crisis, the newly introduced bill on Friday shows how the Senate is aligning with its House counterparts on similar accelerated approval reforms.

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Co-CEOs Chintu and Chirag Patel (Amneal)

Look out, Neu­las­ta: A 5th biosim­i­lar is com­ing

As Neulasta sales slip, Amgen has yet another biosimilar to look out for: Amneal Pharmaceuticals and Kashiv Biosciences’ Flynetra.

Flynetra became the fifth approved biosimilar to Neulasta on Friday, snagging a win in neutropenia, a condition common among chemotherapy patients where neutrophils, a type of white blood cell that fights infection, are too low.

As of last summer, the list price of Neulasta was more than $6,400 per dose. It’s designed to be taken in a single dose per chemotherapy cycle. Amneal declined to reveal how much it intends to charge for Flynetra in an email to Endpoints News. 

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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