Ver­tex's new dilem­ma: With more pos­i­tive PhI­II da­ta on hand, which of their promis­ing com­bos should go to the FDA, EMA?

Ver­tex $VRTX has a pleas­ant dilem­ma on its hands.

The big biotech re­port­ed that its Phase III tri­al for a cock­tail used to treat cys­tic fi­bro­sis has come in with pos­i­tive re­sults for VX-445, teza­caftor and iva­caftor.

In fact, they’re al­most iden­ti­cal to the Phase III re­sults they got for VX-659 com­bined with teza­caftor and iva­caftor. And that means the ju­ry on which triple will head to reg­u­la­tors on both sides of the At­lantic is still out.

Now the ex­ec­u­tives at Ver­tex say they’ll wait for 24-week da­ta due on both late-stage com­bi­na­tions in Q2, then de­cide which will get pitched to the FDA and EMA. Bot­tom line:

Da­ta from a pre-spec­i­fied in­ter­im analy­sis of the Phase III study in peo­ple with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion showed a mean ab­solute im­prove­ment in ppFEV1 of 13.8 per­cent­age points from base­line at week 4 of treat­ment com­pared to place­bo (p<0.0001). In the Phase 3 study in peo­ple with two F508del mu­ta­tions, the ad­di­tion of VX-445 in pa­tients al­ready re­ceiv­ing teza­caftor and iva­caftor re­sult­ed in a mean ab­solute im­prove­ment in ppFEV1 of 10.0 per­cent­age points from base­line at week 4 of treat­ment com­pared to the con­trol group in whom place­bo was added to teza­caftor and iva­caftor (p<0.0001).

VX-659 was cred­it­ed with a 14% place­bo-ad­just­ed im­prove­ment from base­line in FEV1 for pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, with a clear score on the p val­ue (p<0.0001). In a sep­a­rate Phase III pa­tients ho­mozy­gous for the F508del mu­ta­tion al­ready tak­ing Symdeko saw an av­er­age 10% im­prove­ment with VX-659 added on.

Cue the en­thu­si­as­tic re­sponse from an­a­lysts. Not­ed SVB Leerink’s Ge­of­frey Porges:

This triple da­ta is, if any­thing, even bet­ter than the pre­vi­ous­ly re­leased VX-659 triple re­sults, and in our view ef­fec­tive­ly guar­an­tees the launch, ap­proval, com­mer­cial­iza­tion and adop­tion of one of these com­bi­na­tions, with re­sult­ing mul­ti-bil­lion dol­lar sales to Ver­tex (re­gard­less of the UK’s in­tran­si­gence). ​

Resh­ma Ke­wal­ra­mani

“Both the VX-659 and VX-445 triple com­bi­na­tion reg­i­mens showed high­ly con­sis­tent and sig­nif­i­cant im­prove­ments in lung func­tion across our Phase III pro­grams, un­der­scor­ing the im­por­tant clin­i­cal ben­e­fit that a triple com­bi­na­tion reg­i­men may pro­vide to pa­tients with two F508del mu­ta­tions and to those with one F508del and one min­i­mal func­tion mu­ta­tion,” said Ver­tex CMO Resh­ma Ke­wal­ra­mani. “We look for­ward to sub­mit­ting glob­al reg­u­la­to­ry ap­pli­ca­tions for one of these triple com­bi­na­tion reg­i­mens for both pa­tient pop­u­la­tions lat­er this year.”

Ver­tex has ac­quired quite a cheer­ing sec­tion among an­a­lysts cov­er­ing the mar­ket, with many see­ing the com­pa­ny step­ping up with a triple that can sig­nif­i­cant­ly broad­en their mar­ket share.

Once ap­proved — if the op­ti­mists are cor­rect — Ver­tex will al­so throw their com­bo in­to the bat­tle over ac­cess in Eu­rope. Sev­er­al sin­gle pay­er sys­tems, most no­tably the UK, have bucked at the price of their best­seller Orkam­bi. But CEO Jeff Lei­den has waged a PR war against his pric­ing foes, adamant­ly seek­ing more than what NICE and oth­ers are will­ing to pay. 

That show­down will come to a head to­mor­row, as Ver­tex ex­ecs square off with pub­lic health of­fi­cials in a high pro­file, head-to-head bat­tle over the price. The triple can on­ly add more heat to the al­ready fetid con­fronta­tion that has now lin­gered for 3 years, with pa­tients caught in the mid­dle.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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The majority of these roles are from our field organization and, though formally associated with our South San Francisco headquarters, are actually located throughout the United States. These position eliminations are not related to the COVID-19 pandemic, the clinical trials for which Actemra is being studied in COVID-19 pneumonia, or any specific therapeutic area.

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