HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

Leg­is­la­tors call HHS to ac­tion on Xtan­di march-in rights

More than two dozen law­mak­ers are putting re­newed pres­sure on the fed­er­al gov­ern­ment to use its march-in rights to low­er the price of prostate can­cer drug Xtan­di.

The group — led by Sens. Eliz­a­beth War­ren (D-MA) and An­gus King (I-ME), and Rep. Lloyd Doggett (D-TX) — penned a let­ter to HHS Sec­re­tary Xavier Be­cer­ra on Tues­day re­quest­ing a “long over­due” pub­lic hear­ing on a pe­ti­tion to low­er Xtan­di’s price filed by pa­tients more than a year ago. Ear­li­er this month, the pe­ti­tion­ers got word that the NIH is still “cur­rent­ly co­or­di­nat­ing with HHS to re­view and as­sess the in­for­ma­tion,” ac­cord­ing to the let­ter.

“The In­fla­tion Re­duc­tion Act (IRA) has been signed in­to law and will pro­vide crit­i­cal re­lief for Medicare ben­e­fi­cia­ries, but there are ad­di­tion­al ac­tions – in­clud­ing grant­i­ng march-in rights for Xtan­di – that the ad­min­is­tra­tion can take to fol­low through on its com­mit­ment to re­duce drug prices for all Amer­i­cans,” the let­ter reads.

Xtan­di was first ap­proved back in 2012 for late-stage cas­tra­tion-re­sis­tant prostate can­cer, and has since been award­ed new in­di­ca­tions in non-metasta­t­ic cas­tra­tion-re­sis­tant and metasta­t­ic cas­tra­tion-sen­si­tive pa­tients. The av­er­age whole­sale price is more than $189,000 per year, the pe­ti­tion­ers wrote to Be­cer­ra in No­vem­ber.

The pe­ti­tion­ers ar­gue that the fed­er­al gov­ern­ment should ex­er­cise its rights un­der the Bayh-Dole Act to grant patent li­cens­es for gener­ic man­u­fac­tur­ers to pro­duce cheap­er ver­sions of the drug, which was de­vel­oped at UCLA us­ing gov­ern­ment fund­ing.

Robert Sachs, one of the prostate can­cer pa­tients who sub­mit­ted the Xtan­di march-in pe­ti­tion to NIH, came to be in­volved af­ter the fall of 2020 when his prostate can­cer had metas­ta­sized. He was pre­scribed Xtan­di, and said his co­pays at the time would amount to about $10,000 per year.

“We’re hap­py if they des­ig­nate this for a hear­ing. A pub­lic hear­ing with an in­de­pen­dent de­ci­sion mak­er, with an op­por­tu­ni­ty for pub­lic par­tic­i­pa­tion and let the facts dri­ve the de­ci­sion,” Sachs, who’s now in re­mis­sion, tells End­points News.

Leg­is­la­tors on Tues­day re­quest­ed that HHS an­swer sev­er­al ques­tions by the end of the month, in­clud­ing whether HHS has pro­ce­dures in place that gov­ern how of­fi­cials re­spond to march-in pe­ti­tions, and whether the agency has set an in­ter­nal dead­line to re­spond to the Xtan­di pe­ti­tion. They al­so asked the agency to name the HHS of­fi­cials in­volved in the re­view process.

“You have the pow­er to take on the mo­nop­oly abus­es of the phar­ma­ceu­ti­cal in­dus­try and the re­spon­si­bil­i­ty to en­sure Amer­i­cans have af­ford­able ac­cess to the med­i­cines they need,” the let­ter states.

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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EMA rec­om­mends re­vok­ing au­tho­riza­tion of No­var­tis' sick­le cell drug

The European Medicines Agency’s committee for medicinal products for human use (CHMP) on Friday recommended revoking the marketing authorization for Novartis’ treatment for a painful complication related to sickle cell, after a recent study did not confirm its clinical benefit.

CHMP’s review looked at results of the STAND study, finding that Adakveo (crizanlizumab) did not reduce the number of painful crises leading to a healthcare visit, and patients treated with Adakveo had slightly more painful crises on average, with a subsequent healthcare visit, over the first year of treatment, compared with those on placebo.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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