AveX­is shares soar af­ter it out­lines FDA’s sup­port for a loom­ing piv­otal study on SMA

AveX­is CEO Sean Nolan

Shares of AveX­is $AVXS ripped high­er af­ter the mar­ket close on Tues­day af­ter the gene ther­a­py biotech out­lined the FDA’s ac­cep­tance of a small, sin­gle arm piv­otal study for rare cas­es of spinal mus­cu­lar at­ro­phy Type 1 (SMA).

Ac­cord­ing to the Chica­go-based biotech, reg­u­la­tors signed off on a 20-pa­tient study that would use a his­tor­i­cal com­par­i­son for eval­u­at­ing ef­fi­ca­cy. And that lays a straight path to a piv­otal study now slat­ed to be­gin in the first half of a loom­ing 2017.

AveX­is shares shot up 25% on the tri­al plans.

Here’s the sum­ma­ry:

At the Type B meet­ing and in the meet­ing min­utes, the FDA ac­knowl­edged the com­pa­ny’s ra­tio­nale for a sin­gle-arm piv­otal study and pro­vid­ed a num­ber of con­struc­tive sug­ges­tions to help op­ti­mize such a tri­al de­sign. The FDA al­so in­di­cat­ed its pref­er­ence for a de­sign with co-pri­ma­ry end­points con­sist­ing of a mea­sure of de­vel­op­men­tal mile­stone achieve­ment (such as sit­ting unas­sist­ed) along with a clin­i­cal­ly mean­ing­ful mea­sure of sur­vival (such as time to an “event” as de­scribed above). Based on FDA’s sug­ges­tions as well as oth­er ex­pert in­put, AveX­is con­tin­ues to eval­u­ate a num­ber of the de­tails of the tri­al de­sign. More spe­cif­ic in­for­ma­tion will be made avail­able at the time the study is ini­ti­at­ed, which is ex­pect­ed in the first half of 2017.

“We be­lieve the Type B meet­ing had a pos­i­tive tone, with FDA of­fer­ing a num­ber of con­struc­tive sug­ges­tions which we be­lieve will bet­ter en­able im­ple­men­ta­tion of a piv­otal study de­sign that is most ap­pro­pri­ate for the pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease,” said AveX­is CEO Sean Nolan in a state­ment. “With the feed­back need­ed from the FDA to move for­ward with our piv­otal tri­al, we plan to pro­ceed as ex­pe­di­tious­ly as pos­si­ble to be­gin the study in the first half of 2017.”

Not on­ly that, the FDA — which ap­pears to be quite ac­com­mo­dat­ing in AveX­is’s sum­ma­ry — is al­so will­ing to con­sid­er an ap­proval on the Phase I study. At least one an­a­lyst says these de­vel­op­ments could pose a prob­lem for Bio­gen and Io­n­is, which are part­nered on nusin­ersen for SMA. Notes Jef­feries’ Bri­an Abra­hams:

While ear­ly da­ta for AVXS’s AVXS-101 has looked promis­ing in SMA, it is hard to com­pare be­tween treat­ments and BI­IB re­tains a first mover ad­van­tage that could po­ten­tial­ly lim­it up­take of ‘101. Ad­di­tion­al­ly, nusin­ersen is al­so be­ing ex­plored in stud­ies of Type 2/3 SMA, which rep­re­sent ad­di­tion­al sig­nif­i­cant mar­ket op­por­tu­ni­ties, and the pos­si­bil­i­ty of broad ini­tial ap­proval across all sub­types re­mains; in con­trast, AVXS is just be­gin­ning to ex­plore such ad­di­tion­al SMA pop­u­la­tions. Sys­temic ad­min­is­tra­tion of AVXS-101 could, how­ev­er, of­fer greater con­ve­nience, at least in Type 1 pts. We would not rule out the pos­si­bil­i­ty the ther­a­pies could be used in the same pts, though pric­ing could be an is­sue. We cur­rent­ly mod­el a 2017 nusin­ersen launch with prob­a­bil­i­ty-ad­just­ed rev­enues po­ten­tial­ly reach­ing $1.5B by 2025.

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