A new re­port try­ing to set­tle a decades-old de­bate about Alzheimer’s dis­ease has reached a dra­mat­ic con­clu­sion: An­ti­body drugs that tar­get sticky amy­loid be­ta pro­teins in the brain sim­ply don’t work.

The meta-analy­sis, which com­piles da­ta from more than 20,000 pa­tients across 17 clin­i­cal tri­als, de­ter­mined that treat­ment with amy­loid an­ti­bod­ies re­sult­ed in “lit­tle to no dif­fer­ence” in cog­ni­tive func­tion, de­men­tia sever­i­ty and func­tion­al abil­i­ty. The au­thors con­clud­ed that fu­ture Alzheimer’s drugs “should fo­cus on oth­er mech­a­nisms of ac­tion.”

But the study is filled with lim­i­ta­tions sure to send many neu­ro­sci­en­tists who have de­vot­ed their lives to these drugs in­to a tizzy. It over­looks the strongest ar­gu­ments about why so many old­er at­tempts have failed; it ig­nores re­cent tech­no­log­i­cal break­throughs in de­liv­er­ing these kinds of drugs in­to the brain more ef­fi­cient­ly; and it skirts over big­ger and longer clin­i­cal tri­als that are still on­go­ing but promise to pro­vide the most con­clu­sive ev­i­dence on amy­loid drugs yet.

As the prices paid for pri­or­i­ty re­view vouch­ers (PRVs) have crept up over the past year — the last two have sold for at least $200 mil­lion each — com­pa­nies now have to make dif­fi­cult de­ci­sions on whether us­ing the vouch­ers to speed up their own ap­provals makes fi­nan­cial sense.

For Mod­er­na, the FDA re­cent­ly dis­closed, and the com­pa­ny con­firmed, that it re­deemed two of its PRVs to ex­pe­dite its own RSV vac­cine ap­provals in old­er and then younger adults in 2024 and 2025 — in­clud­ing via its ma­te­r­i­al threat med­ical coun­ter­mea­sure PRV that it ac­quired af­ter win­ning ap­proval for its Covid vac­cine in 2022.

But those months of shaved FDA re­view times haven't trans­lat­ed in­to ear­ly ad­di­tion­al sales so far. Mod­er­na not­ed just $33 mil­lion in RSV sales in an SEC fil­ing in Feb­ru­ary and cit­ed its fierce com­pe­ti­tion with Pfiz­er and GSK, which en­tered the US mar­ket pri­or to Mod­er­na.

Roche said it will run a new Phase 3 tri­al for the con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py Ele­v­idys in an­oth­er at­tempt to win ap­proval for the ther­a­py in Eu­rope and oth­er re­gions.

The new study comes af­ter the Eu­ro­pean Med­i­cines Agency last Ju­ly rec­om­mend­ed against Ele­v­idys’ ap­proval for chil­dren aged 3 to 7 with Duchenne mus­cu­lar dy­s­tro­phy, say­ing that Ele­v­idys’ piv­otal tri­al hadn’t shown an ef­fect on pa­tients’ move­ment at one year. The Swiss phar­ma holds ex-US rights to the gene ther­a­py de­vel­oped by Sarep­ta Ther­a­peu­tics.

The new study has some key dif­fer­ences from the orig­i­nal Phase 3 tri­al called EM­BARK, which was con­duct­ed by Sarep­ta. The new pri­ma­ry end­point will be a mea­sure around the time it takes pa­tients to rise from the floor af­ter 72 weeks, which is a dif­fer­ent pri­ma­ry end­point and longer fol­low-up com­pared to EM­BARK.

💰 MeiraGTx buys back eye gene ther­a­py from J&J for just $25M: The gene ther­a­py, known as bo­ta-vec, failed a Phase 3 study for X-linked re­tini­tis pig­men­tosa (XL­RP) last year. But physi­cians and pa­tient ad­vo­cates be­lieved the da­ta showed the gene ther­a­py still had promise, and urged J&J to at least ap­ply to reg­u­la­tors for ap­proval. How­ev­er, J&J did not do so. The big phar­ma had bought a suite of ther­a­pies in­clud­ing bo­ta-vec from MeiraGTx for $100 mil­lion up­front in 2019. Now, MeiraGTx will be tak­ing bo­ta-vec back for $25 mil­lion. The Lon­don and New York-based biotech “in­tends to im­me­di­ate­ly pur­sue glob­al reg­u­la­to­ry fil­ings for ap­proval of bo­ta-vec,” it said Thurs­day morn­ing. MeiraGTx al­so an­nounced a $100 mil­lion pub­lic of­fer­ing that’s in part meant to fund the launch of bo­ta-vec. — Lei Lei Wu