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. 

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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