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.

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Fresh off the high of its Nas­daq IPO de­but, and the low of com­par­isons to Cymabay — whose NASH drug re­cent­ly stum­bled — Gen­fit on Mon­day un­veiled an up to $228 mil­lion deal with transpa­cif­ic biotech Terns Phar­ma­ceu­ti­cals to de­vel­op its flag­ship ex­per­i­men­tal liv­er drug — elafi­bra­nor — in Greater Chi­na.

The deal comes weeks af­ter Gen­fit $GN­FT is­sued a fiery de­fense of its dual PPAR ag­o­nist elafi­bra­nor, when com­peti­tor Cymabay’s PPARδ ag­o­nist, se­ladel­par, fiz­zled in a snap­shot of da­ta from an on­go­ing mid-stage tri­al. The main goal at the end of 12 weeks was for se­ladel­par to in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er fat con­tent, but da­ta showed that pa­tients on the place­bo ac­tu­al­ly per­formed bet­ter.