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. 

Roger Perl­mut­ter lines up deals, fresh fund­ing at Eikon; Sec­ond RSV vac­cine ap­proved; Sev­er­al biotechs flash­ing red; 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.

As you come back to our website this weekend for ASCO news, don’t forget to check out our updated event lineup at BIO, which will cover everything from the current state of VC investing in biotech to top pharma R&D chiefs discussing how to make pipeline decisions.

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Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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Grail’s blood test charts path for di­ag­nos­ing pa­tients sus­pect­ed of hav­ing can­cer in large study: #AS­CO23

Grail’s vision is simple but bold. The blood testing company has long held that people are often diagnosed with cancer too late. If seemingly healthy people were screened for early signs of the disease before symptoms appear, they may be able to get more effective treatments that nip cancer in the bud.

That premise is the basis of Grail’s commercial blood test, Galleri, which searches for the genetic fingerprints of cancer in the blood. The test, launched in 2021, reaped $55 million in sales last year, but now the company is setting its sights on a new market: patients suspected of having cancer due to symptoms such as abdominal pain, rectal bleeding or unexplained weight loss. Rather than administering expensive scans or conducting invasive biopsies right away, Grail hopes doctors will consider a simple blood test.

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GSK pro­motes rou­tine im­mu­niza­tions for adults amid post-pan­dem­ic vac­cine back­slide

GSK launched a new initiative on Thursday and committed up to $1 million in grant funding to improve adult routine vaccination rates.

While the pandemic spotlight was trained on the race for novel Covid-19 vaccines, other routine vaccination rates plummeted, raising concerns that missed doses may put children and even some adults at risk of preventable diseases such as measles or shingles. The World Health Organization last year reported the largest drop in childhood vaccinations in roughly three decades.

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Tammie Denyse speaks up about Black women and breast cancer inequity in Gilead's first TikTok campaign. (Gilead Sciences)

Gilead joins Tik­Tok with on­col­o­gy aware­ness cam­paign fea­tur­ing di­verse group of can­cer ad­vo­cates

Gilead Sciences is taking over the opening page on TikTok for the next two weeks. A Gilead-sponsored video, featuring cancer advocates talking about equity and other issues, will show up as the landing page, called the “For You” page, for millions of TikTok watchers.

The cancer awareness campaign will begin on Monday and run for two weeks, a Gilead spokesperson told Endpoints News. The TikTok ad debut is timed around the ASCO medical conference, but the work is aimed more broadly at healthcare professionals, as well as people touched by cancer and people interested in advancing Black and general health equity.

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Stephen MacMillan, Hologic CEO (Photo by Riccardo Savi/Getty Images for Concordia Summit)

Il­lu­mi­na names Ho­log­ic CEO as new board mem­ber and chair

Illumina’s board appointed two new members, including Hologic CEO Stephen MacMillan as the non-executive chair, a move that followed a proxy fight that saw shareholders oust the company’s board chair.

The DNA sequencing company also appointed Scott Ullem, the CFO of Edwards Lifesciences, to the board, according to a company statement.

Illumina’s plans to add two new board members came as Carl Icahn waged a board proxy campaign culminating with shareholders electing his candidate, Andrew Teno, over board chair John Thompson. Illumina CEO Francis deSouza survived a threat to his board seat by securing more than twice the shareholder votes than his challenger. Another Illumina candidate, Robert Epstein, was also elected and remained on the board.

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Novartis headquarters in Basel, Switzerland (Kyle LaHucik for Endpoints News)

No­var­tis’ Kisqali pre­vents breast can­cer from com­ing back for longer — but can it best Eli Lil­ly’s Verzenio? #AS­CO23

CHICAGO — Novartis’ CDK4/6 inhibitor Kisqali helped early breast cancer patients stay cancer-free for longer after surgery, according to interim study results presented at ASCO.

In a Phase III study, Kisqali was added on top of endocrine therapy — the current standard treatment for early breast cancer patients. Kisqali reduced the risk of disease relapse by 25% compared with endocrine therapy alone in patients with Stage II or III HR-positive, HER2-negative breast cancer.

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Catal­ent makes ad­di­tion­al lay­offs at In­di­ana fa­cil­i­ty

Contract manufacturer Catalent is making more staff cuts at one of its locations in the US amid dramatic corrective actions it’s been taking over the past few months.

In an email to Endpoints News, a Catalent spokesperson confirmed the company is making “a number of personnel changes” at a manufacturing facility in Bloomington, IN. While a specific number was not given to Endpoints, several local media outlets, including Indiana Public Media and the Bloomington Herald-Times, have put the number of layoffs at 150. No postings have been made to the Indiana Department of Workforce Development’s WARN notice.

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