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

In a leap to the gene therapy space, Daiichi Sankyo has dropped $200 million to access Ultragenyx’s manufacturing technology, providing the rare disease biotech with plenty of cash and a stock boost amid a general cash crunch.

For $125 million in cash and a $75 million equity investment, Daiichi Sankyo has bought a non-exclusive license to the IP around two platforms with which it plans to develop AAV-based gene therapy products. The Japanese pharma is purchasing the stock $RARE at $60 per share, more than a third above its current price of $44.43.

Gilead’s bid to overturn a jury verdict that ordered it to pay Bristol Myers Squibb about $752 million for CAR-T patents owned by its subsidiary Juno Therapeutics has ended in vain.

The ruling leaves Gilead vulnerable to an even bigger $1.5 billion payment that Bristol is now demanding — adding fuel to the fiery criticism some analysts are already heaping on its $11.9 billion Kite buyout.

In a 30-page document unsealed on Monday, Judge James Otero of the district court in Los Angeles struck down several different arguments for a new decision. Here are Morgan Stanley analysts’ takeaways:
The court, in particular, denied Kite’s contentions (1) that Juno’s patent is invalid, (2) the damages award was unreasonable, and (3) that a new trial should take place. The court also denied Kite’s argument that its infringement was not willful.
Gilead is likely to appeal to the federal circuit, they noted, but the odds are not in their favor as the same standard for evidence will be applied in that court. Appeals typically take 16 months.

Scientists began with making recombinant versions of naturally-occurring human proteins, then graduated to monoclonal antibodies. Now, rather than replicating moieties within the body, researchers are modifying these molecules to have precise biology in a functional manner.

This technology, referred to as bispecific antibodies, is already being employed to fight cancer. In early 2018, Pandion Therapeutics was born to reverse-engineer the science into the realm of autoimmune and inflammatory disorders.

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).