The ver­dict is in from Ver­tex’s clin­i­cal team.

The biotech was pre­sent­ed with a pleas­ant dilem­ma in March when a cock­tail for cys­tic fi­bro­sis mix­ing VX-​445, teza­caftor and iva­caftor passed Phase III with fly­ing col­ors, match­ing the re­sults they got for VX-659 with the oth­er two drugs, al­so known as Symdeko and Ka­ly­de­co. That left ob­servers hang­ing as to which com­bo will head to reg­u­la­tors.

Ver­tex ul­ti­mate­ly de­cid­ed to go with VX-445 — af­ter re­view­ing some more da­ta it’s dis­clos­ing to­day.

Resh­ma Ke­wal­ra­mani Ver­tex

At week 24 of treat­ment, pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion on the drug main­tained ab­solute im­prove­ment in per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume in one sec­ond of 14.3% (p<0.0001). For a key sec­ondary end­point, the same group of pa­tients saw a 63% re­duc­tion in the an­nu­al­ized rate of pul­monary ex­ac­er­ba­tions com­pared to place­bo, with the same stel­lar p-val­ue.

As pre­vi­ous­ly re­port­ed, pa­tients on the VX-445 triple had their ppFEV1 im­prove by an av­er­age of 13.8% at week 4. In a sep­a­rate tri­al for VX-​659, the num­ber was 14%.

“Peo­ple with CF who have one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion are the largest re­main­ing group of CF pa­tients with­out a treat­ment op­tion for the un­der­ly­ing cause of their dis­ease,” CMO Resh­ma Ke­wal­ra­mani said in a state­ment.

The biotech fur­ther not­ed that the de­ci­sion “was based on a de­tailed as­sess­ment of mul­ti­ple fac­tors, in­clud­ing fa­vor­able pro­files for safe­ty, tol­er­a­bil­i­ty and drug-drug in­ter­ac­tions, the abil­i­ty for co-ad­min­is­tra­tion with hor­mon­al con­tra­cep­tives, and the lack of pho­to­sen­si­tiv­i­ty.”

Ver­tex will now take these da­ta, along­side an­oth­er 4-week Phase III study CF pa­tients with two F508del mu­ta­tions, to the FDA in Q3 and then the EMA in Q4.

Cred­it Su­isse an­a­lysts are ful­ly on board with the plan, say­ing it sig­nals a “clear path for­ward for the triple com­bo, with longer-term da­ta sup­port­ing con­tin­ued ef­fi­ca­cy. We ex­pect ap­proval in 1H20, al­low­ing the co. to treat 90% of CF pts WW. OUS re­im­burse­ment ques­tions re­main.”

Im­age: Ver­tex Phar­ma­ceu­ti­cals

AbbVie picked up some bonus points earlier this year as one of its pipeline adds from the $63 billion Allergan buyout hit its top-line marks. And now the researchers have produced the detailed data on the case they are making with regulators, with an eye on a major new market and a hoped-for approval before New Year’s.

AGN-190584 is aiming to be the first easy-on eyedrop for presbyopia, a common ailment for large numbers of people who find it harder and harder to read things like a watch or cell phone close up. Anyone who’s held a book out at arm’s length in order to read it will be very familiar with the condition, if not the exact diagnosis.

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.