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.

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.