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.

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