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

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.

When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.

The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.