Tiny phar­ma hits a new low with FDA re­fus­ing to re­view com­pa­ny's re­sponse to its CRL

Just be­fore the long week­end, tiny Zosano Phar­ma re­vealed an­oth­er blow to its ap­pli­ca­tion for ap­proval for a mi­graine patch.

The FDA said the com­pa­ny’s re­sub­mis­sion for the patch didn’t ad­e­quate­ly re­spond to the de­fi­cien­cies iden­ti­fied in the FDA’s re­jec­tion let­ter, and that the agency will not re­view this re-sub­mit­ted ap­pli­ca­tion un­til a new com­plete re­sponse is sub­mit­ted.

Fre­mont, CA-based Zosano sent its re­sponse to the FDA last month, try­ing to res­ur­rect the gener­ic trip­tan zolmitrip­tan in a new de­liv­ery sys­tem known as Qtryp­ta. The agency in Oc­to­ber 2020 re­ject­ed the patch over in­con­sis­tent ex­po­sure lev­els across mul­ti­ple clin­i­cal tri­als, as well as oth­er is­sues.

The FDA found that Zosano hadn’t ad­e­quate­ly shown its prod­uct gave pa­tients sim­i­lar ex­po­sure to the mi­graine drug as an ex­ist­ing nasal spary, as the com­pa­ny ex­plained Fri­day in its fil­ing:

Among oth­er things, the FDA’s re­sponse let­ter stat­ed that the Com­pa­ny’s strat­e­gy for es­tab­lish­ing a phar­ma­co­ki­net­ic (‘PK’) bridge to Zomig Nasal Spray 5 mg (‘Zomig NS’) by re­ly­ing pri­mar­i­ly on da­ta from the Com­pa­ny’s re­cent­ly com­plet­ed Phase 1 PK study (‘Study CP-2021-001’) was not ac­cept­able, due in part to dif­fer­ences be­tween the de­sign of Study CP-2021-001, which com­pared the PK of M207 to two se­quen­tial dos­es of 5 mg Zomig NS, and the cri­te­ria for re-dos­ing set forth in the la­bel­ing in­struc­tions for 5 mg Zomig NS.

Zosano not­ed that FDA’s re­sponse let­ter de­scribed al­ter­na­tive meth­ods to es­tab­lish equiv­a­lence to 5 mg Zomig NS, in­clud­ing: “(i) by demon­strat­ing bioe­quiv­a­lence to Zomig NS us­ing stan­dard cri­te­ria for all PK ex­po­sure met­rics, in­clud­ing through a com­bi­na­tion of rel­e­vant PK da­ta and mod­el­ing or sim­u­la­tion pro­ce­dures; or (ii) by con­duct­ing a rel­a­tive bioavail­abil­i­ty study in healthy vol­un­teer sub­jects.”

But the com­pa­ny, whose stock price fell by about 37% ear­ly Tues­day, said it may have to reeval­u­ate whether it can con­tin­ue to pur­sue the patch.

“The Com­pa­ny is eval­u­at­ing next steps in re­la­tion to the FDA’s re­sponse let­ter as part of its fi­nan­cial and strate­gic plan­ning; how­ev­er, there can be no as­sur­ances that the Com­pa­ny’s re­sources will en­able the Com­pa­ny to con­tin­ue to pur­sue FDA ap­proval of M207,” the SEC fil­ing said.

Zosano’s ap­pli­ca­tion for Qtryp­ta was based on a Phase II/III tri­al from 2017 that met both co-pri­ma­ry end­points, with 41.5% of pa­tients treat­ed with the 3.8 mg dose of Qtryp­ta achiev­ing pain free­dom at 2 hours, and 68.3% re­port­ing free­dom from their most both­er­some symp­tom al­so at 2 hours.

In a long-term safe­ty study, Zosano said 95% of side ef­fects were mild, and more than 80% re­solved with­in 48 hours. Less than 2% of pa­tients re­port­ed trip­tan-like neu­ro­log­i­cal side ef­fects typ­i­cal­ly found in the class, such as dizzi­ness and pares­the­sia. But in the CRL, FDA cit­ed high plas­ma con­cen­tra­tion lev­els of zolmitrip­tan in five pa­tients.

Zosano has had a rocky his­to­ry af­ter rais­ing $50 mil­lion for an IPO at the be­gin­ning of 2015. Soon af­ter de­but­ing, the com­pa­ny’s $11 share price was shred­ded when its two big part­ners — Eli Lil­ly and No­vo Nordisk — pulled out of their col­lab­o­ra­tions.

“As pre­vi­ous­ly dis­closed, the Com­pa­ny has re­tained Sier­ra­Con­stel­la­tion Part­ners, LLC as an in­de­pen­dent fi­nan­cial ad­vi­sor to as­sist in ex­plor­ing fi­nan­cial and strate­gic al­ter­na­tives to max­i­mize val­ue, which may in­clude, but not be lim­it­ed to, as­set or eq­ui­ty sales, joint ven­ture and part­ner­ship op­por­tu­ni­ties, and re­struc­tur­ing, amend­ment or re­fi­nanc­ing of ex­ist­ing li­a­bil­i­ties, and is eval­u­at­ing var­i­ous al­ter­na­tives to im­prove its liq­uid­i­ty,” Zosano added.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

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