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

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.