Argenx touts first-in-class win for a rare disease that causes life-threatening muscle weakness

Tim Van Hauwermeiren, argenx CEO

If you look on ar­genx’s web­site to­day, you’ll see a burst of con­fet­ti. That’s be­cause — in ad­di­tion to get­ting a new type of ther­a­py past the FDA for pa­tients with a chron­ic neu­ro­mus­cu­lar dis­ease called myas­the­nia gravis — the com­pa­ny is cel­e­brat­ing its first drug ap­proval.

Reg­u­la­tors on Fri­day gave the thumbs up to ar­genx’s ef­gar­tigi­mod, now mar­ket­ed as Vyv­gart, for pa­tients with gen­er­al­ized myas­the­nia gravis (gMG) who test pos­i­tive for the an­ti-acetyl­choline re­cep­tor (AChR) an­ti­body. CEO Tim Van Hauw­er­meiren said in a state­ment that the ap­proval trig­gers the ‘start of a new era for ar­genx.’

The com­pa­ny’s stock $ARGX was up more than 8% in pre-mar­ket trad­ing Mon­day.

MG oc­curs when im­munoglob­u­lin G (IgG) an­ti­bod­ies dis­rupt com­mu­ni­ca­tion be­tween nerves and mus­cles, caus­ing po­ten­tial­ly life-threat­en­ing mus­cle weak­ness. Most pa­tients progress to gMG with­in 18 months, where mus­cles through­out the body may be af­fect­ed, caus­ing dif­fi­cul­ties with fa­cial ex­pres­sion, speech, swal­low­ing and breath­ing.

De­spite steroids, im­muno­sup­pres­sants, acetyl­cholinesterase in­hibitors and Alex­ion’s Soliris, some pa­tients don’t nec­es­sar­i­ly ben­e­fit from ex­ist­ing op­tions. So a slate of play­ers be­gan work­ing on a new class of med­i­cines called neona­tal Fc re­cep­tor (FcRn) in­hibitors.

FcRn re­cy­cles IgG an­ti­bod­ies by shut­tling them away from lyso­so­mal degra­da­tion, there­by pre­serv­ing path­o­gen­ic an­ti­body lev­els in IgG-me­di­at­ed dis­eases like MG. FcRn in­hibitors, there­fore, are de­signed to de­crease to­tal IgG to treat pa­tients.

It’s been a long jour­ney for the new class, with Im­muno­vant hit­ting a safe­ty snag ear­li­er this year and Alex­ion drop­ping out of the race al­to­geth­er af­ter ear­ly-stage da­ta failed to im­press. Ar­genx crossed the fin­ish line first, af­ter show­ing that about 68% of pa­tients re­spond­ed to Vyv­gart dur­ing the first cy­cle of treat­ment, com­pared to about 30% of place­bo pa­tients, based on a mea­sure that as­sess­es the im­pact of MG on dai­ly func­tion (p<0.0001).

The drug was al­so well-tol­er­at­ed, ac­cord­ing to ar­genx — which is im­por­tant, af­ter Im­muno­vant found pa­tients on a high dose of their FcRn in­hibitor saw their ‘bad cho­les­terol’ rise 60% af­ter 12 weeks. At the time, ar­genx, put out a state­ment that it had seen no cho­les­terol in­creas­es across 117 pa­tients.

Im­muno­vant vol­un­tar­i­ly paused dos­ing in two stud­ies back in Feb­ru­ary, then an­nounced plans to re­sume in June af­ter a pro­gram-wide eval­u­a­tion sug­gest­ed the in­creas­es were dose-de­pen­dent and re­versible up­on dis­con­tin­u­a­tion of the drug.

Alex­ion aban­doned its own FcRn in­hibitor back in 2020, and an­a­lysts spec­u­lat­ed that the move may have been due to a neg­a­tive safe­ty sig­nal.

The FDA, how­ev­er, did note that as Vyv­gart caus­es a re­duc­tion in IgG lev­els, the risk of in­fec­tions may in­crease. The most com­mon side ef­fects were res­pi­ra­to­ry tract in­fec­tions, headache, and uri­nary tract in­fec­tions, ac­cord­ing to reg­u­la­tors. And hy­per­sen­si­tiv­i­ty re­ac­tions such as eye­lid swelling, short­ness of breath, and rash have been re­port­ed in some pa­tients.

‘Peo­ple liv­ing with gMG have been in need of new treat­ment op­tions that are tar­get­ed to the un­der­ly­ing patho­gen­e­sis of the dis­ease and sup­port­ed by clin­i­cal da­ta,’ prin­ci­pal in­ves­ti­ga­tor James Howard said in a state­ment. ‘This ther­a­py has the po­ten­tial to re­duce the dis­ease bur­den of gMG and trans­form the way we treat this dis­ease.’

CALQUENCE is a registered trademark of the AstraZeneca group of companies.

At the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition, blood cancer researchers from around the world gathered virtually to discuss the progress that has been made in the field of hematology. Over the past decade, that progress has been tremendous. We’ve seen not only breakthrough approaches to care, but also significant improvement upon existing novel treatments and exploring combinations within those medicines.1 These advances have transformed expectations of what a blood cancer diagnosis now means for patients. While we’ve come a long way, I believe the most exciting scientific discovery is yet to come, and that future advances will truly transform patient care.

Michel Vounatsos (Credit: World Economic Forum/Valeriano Di Domenico)

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Right as the new Omicron variant is poised to increase rapidly across the US, the federal government has effectively run out of the only monoclonal antibody treatment that works against it, and at least one major hospital system is now halting all mAb infusions.

Late last month, the federal government paused shipments of GlaxoSmithKline and Vir’s mAb treatment sotrovimab in order to conserve supplies of the only treatment that might work against the Omicron variant. Last week, however, HHS told Endpoints News that the move to hold back sotrovimab was unrelated to Omicron, and due to a surplus of Eli Lilly mAbs, which aren’t effective against Omicron.

John Oyler, BeiGene CEO (Endpoints News, PharmCube)

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Stéphane Bancel, Moderna CEO (Andrew Harnik/AP Images)

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Richard Pazdur (via AACR)

There’s no denying that Merck’s Keytruda set a high bar for checkpoint inhibitors in development everywhere. But when it comes to the often redundant development of PD(L)-1 antibodies worldwide, FDA’s top cancer doctors Rick Pazdur and Julia Beaver are calling for more industry coordination.

‘Efforts to corral this enthusiasm should focus on increased international partnerships between sponsors of approved checkpoint inhibitors and those developing novel agents to be used with anti–PD-1 and anti–PD-L1 antibodies rather than developing ‘me too’ drugs,’ Beaver and Pazdur wrote Wednesday in the New England Journal of Medicine.

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The FDA on Wednesday not only approved the first generic versions of the decades-old diabetes insipidus treatment vasopressin, but also simultaneously offered a particularly damning rebuke of a citizen petition attempting to block the generic, while promising to pass along the matter to the Federal Trade Commission.

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Robert Califf (Graeme Sloan/Sipa via AP Images)

As Rob Califf likely makes his return as FDA commissioner next month, his confirmation hearing yesterday offered a peek into some of the larger obstacles he’s going to face in the coming months and years.

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