In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

The p-val­ue was an aw­ful 0.17.

Re­searchers blamed a high place­bo ef­fect, teas­ing out the pos­i­tive as­pects of an im­proved nu­mer­i­cal out­come for sec­on­daries on the 6-minute walk test and a need for whole lung lavage — a pro­ce­dure used to clean the lungs of pa­tients. Those sec­on­daries al­so failed the sta­tis­ti­cal analy­sis.

Bruce Trap­nell Cincin­nati Chil­dren’s

But that didn’t stop Savara from sug­gest­ing that a suc­cess­ful score on a pa­tient-re­port­ed qual­i­ty of life as­sess­ment/out­comes test could help set the stage for an ap­proval. 

“Tak­en to­geth­er, the trends in re­duc­tion of A-aDO2, as well as the im­prove­ment in the dif­fu­sion ca­pac­i­ty, are very con­sis­tent with re­duc­tion of the sur­fac­tant bur­den that caus­es the clin­i­cal symp­toms of aPAP,” said Bruce Trap­nell, the lead in­ves­ti­ga­tor. “Most im­por­tant­ly, the im­pres­sive im­prove­ment in qual­i­ty of life, as mea­sured by the SGRQ, sug­gest that Mol­gradex not on­ly im­proved ob­jec­tive mea­sures of oxy­gena­tion, but al­so had a clin­i­cal­ly mean­ing­ful ther­a­peu­tic ef­fect. I be­lieve these da­ta demon­strate that Mol­gradex can be­come an im­por­tant phar­ma­co­log­i­cal treat­ment op­tion for pa­tients with aPAP.”

In­vestors weren’t buy­ing it, though. The stock cratered on the num­bers, plung­ing more than 70% af­ter the bell on Wednes­day.

Im­age: Shut­ter­stock

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.