May 24 is go­ing to go down as a big day in No­var­tis’ 2019 high­light reel.

Not on­ly did the FDA stamp an OK on the prospec­tive gene ther­a­py block­buster Zol­gens­ma, with a record-set­ting $2.1 mil­lion price tag, reg­u­la­tors al­so green-light­ed alpelis­ib, one of their oth­er top block­buster can­di­dates of the year.

Vas Narasimhan Linkedin

These are 2 of the slate of 4 block­busters the phar­ma gi­ant $NVS high­light­ed for a com­mer­cial roll­out this year. And it’s the third big ap­proval this year, be­hind the MS drug Mayzent, mak­ing 2019 a wa­ter­shed year for the com­pa­ny and new CEO Vas Narasimhan.

Alpelis­ib, ini­tial­ly code-named BYL719, will now hit the mar­ket as Piqray. It will be sold for HR+/HER2- ad­vanced breast can­cer with a PIK3CA mu­ta­tion — the first such tar­get­ed ther­a­py OK’d by the agency.

The da­ta cleared all the hur­dles need­ed for the FDA. Piqray plus ful­ves­trant near­ly dou­bled me­di­an pro­gres­sion-free sur­vival com­pared to ful­ves­trant alone in HR+/HER2- ad­vanced breast can­cer pa­tients with a PIK3CA mu­ta­tion (me­di­an PFS 11.0 months vs 5.7 months; HR=0.65). The ORR for the com­bo was 35.7% vs 16.2% for ful­ves­trant alone, p=0.0002.

It won’t come cheap. The drug will be sold at a whole­sale ac­qui­si­tion price of $15,500 a month, or right around $200,000 a year.

That’s a win­ner. It’s al­so a source of pride for No­var­tis R&D. The multi­na­tion­al bought Zol­gens­ma a year ago when it was well on its way, but they brought this drug all the way up from their dis­cov­ery group at NI­BR, mak­ing it a home­grown suc­cess sto­ry. And the FDA ex­e­cut­ed the ap­proval as the first un­der its re­al-time re­view pro­gram, which lets reg­u­la­tors start con­sid­er­ing an ap­proval be­fore the for­mal sub­mis­sion of the ap­pli­ca­tion.

More amaz­ing news. Thrilled to share this mile­stone ap­proval of our new tar­get­ed ther­a­py. More than ten years in the mak­ing, this pro­gram was sup­port­ed by count­less in­ter­nal NI­BR cham­pi­ons. Con­grats to all who car­ried this ther­a­py through to pa­tients. https://t.co/fb­Jt­DRqITx

— Jay Brad­ner, M.D. (@jay­brad­ner) May 24, 2019

“To­day’s ap­proval is ex­pect­ed to change the way we prac­tice med­i­cine in ad­vanced breast can­cer. For the first time, physi­cians can test for PIK3CA bio­mark­ers and de­vel­op a treat­ment plan based on the ge­nom­ic pro­file of a pa­tient’s can­cer,” said Fab­rice An­dré, a pro­fes­sor in the de­part­ment of med­ical on­col­o­gy at In­sti­tut Gus­tave Roussy in Ville­juif, France, and the lead in­ves­ti­ga­tor in the study.

No­var­tis is fresh off mak­ing some bold promis­es to an­a­lysts about the 25-plus prospec­tive drugs it has in the pipeline. That gives the still new CEO Narasimhan a sol­id foun­da­tion to build his com­pa­ny on new drug cam­paigns, with the in-house de­vel­op­ment work sup­ple­ment­ed by a slate of planned bolt-on deals.

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.