More pos­i­tive PhI­II da­ta set stage for Scynex­is' vagi­nal yeast in­fec­tion drug ap­proval

Months af­ter un­veil­ing da­ta that showed its lead ex­per­i­men­tal com­pound, ibrex­a­fungerp, helped pa­tients with vagi­nal yeast in­fec­tions, an­ti­fun­gal drug de­vel­op­er Scynex­is is one step clos­er to the fin­ish line with a sec­ond pos­i­tive piv­otal tri­al.

The com­pa­ny — which burst in­to the zeit­geist as one of the few com­pa­nies test­ing a Can­di­da au­ris ther­a­py (the same drug ibrex­a­fungerp) in hu­man tri­als — is gear­ing up to sub­mit a mar­ket­ing ap­pli­ca­tion in the sec­ond half of this year, for what it claims is the first and on­ly oral, non-azole treat­ment for vagi­nal yeast in­fec­tions, a con­di­tion that af­fects three out of four women across their life­times but has lim­it­ed treat­ment op­tions.

Mar­co Tagli­et­ti Scynex­is

Scynex­is chief Mar­co Tagli­et­ti said in an in­ter­view he does not fore­see any is­sue in fil­ing the mar­ket­ing ap­pli­ca­tion, and since the ap­proval is ex­pect­ed to come by mid-2021, the FDA will like­ly be work­ing nor­mal­ly.

Giv­en the ma­jor­i­ty of women who get these in­fec­tions are in the child­bear­ing age of be­tween 20 to 40, a vir­tu­al launch is some­thing the com­pa­ny has in any case been think­ing about, he added.

It has been decades since a fresh fam­i­ly of an­ti­fun­gals has come on to the mar­ket — at the mo­ment there are three class­es of an­ti­fun­gals in clin­i­cal use, in­clud­ing azoles and echinocan­dins. The most pop­u­lar an­ti­fun­gals in use to­day were in­tro­duced in the 1980s and this class is in­creas­ing­ly los­ing its po­ten­cy, as fun­gal pathogens mu­tate.

Ibrex­a­fungerp be­longs to a new class of an­ti­fun­gals called glu­can syn­thase in­hibitors, and has shown to be ef­fec­tive against a broad range of fun­gal in­fec­tions, in­clud­ing re­sis­tant strains, ac­cord­ing to Tagli­et­ti.

In the Unit­ed States, there is one oral an­ti­fun­gal, flu­cona­zole, which has been used for over two decades for fun­gal in­fec­tions like vagi­nal yeast in­fec­tions and the co­pay for pa­tients is typ­i­cal­ly in the range of $15 to $20, Tagli­et­ti said.

Since Scynex­is’ treat­ment (if ap­proved) would be treat­ing pa­tients who do not re­spond to flu­cona­zole, the drug will be priced at a pre­mi­um — a list price of $300 to $400, which would work out to a co­pay of $25, to $30, he added.

The lat­est tri­al da­ta em­anate from the 366-pa­tient VAN­ISH-306 study, which test­ed the drug against a place­bo. Pa­tients en­rolled had ex­pe­ri­enced an acute episode of vul­vo­vagi­nal can­didi­a­sis (VVC), with signs and symp­toms score of four or greater on a scale of ze­ro to 18.

The drug in­duced a ‘clin­i­cal cure’ (de­fined as com­plete res­o­lu­tion — or score of 0 — at the test-of-cure vis­it on day 10) rate of 63.3 %, mark­ing a sta­tis­ti­cal­ly sig­nif­i­cant per­for­mance over the place­bo arm, Scynex­is said.

The sec­ondary end­point of my­co­log­i­cal erad­i­ca­tion was al­so met. Place­bo da­ta were not dis­closed. On the safe­ty side, se­vere ad­verse events were rare, and no se­vere side-ef­fects were at­trib­uted to the drug.

The da­ta re­in­forced the re­sults of the VAN­ISH-303 tri­al, which read­out in No­vem­ber.

Source: Scynex­is, 2020

Click on the im­age to see the full-sized ver­sion

The drug is al­so be­ing eval­u­at­ed for use in the CAN­DLE study in pa­tients with re­cur­rent VVC — the re­sults are ex­pect­ed in 2021. This study, since it con­cerns pa­tients who do not have any treat­ment op­tions, is ex­pect­ed to con­tin­ue en­rolling as planned and be com­plet­ed by the end of the year, Tagli­et­ti said.

There are rough­ly 14 mil­lion VVC cas­es treat­ed with oral flu­cona­zole an­nu­al­ly in the US and at least 15% of pa­tients ex­pe­ri­ence treat­ment fail­ure, Need­ham an­a­lyst Alan Carr wrote in a note.

“A 25% peak pen­e­tra­tion of the 15% fail­ure sub­group in 2030 gen­er­ates sales of around $450M ($500 net rev/ course w/ 5% an­nu­al price in­creas­es; ap­prox­i­mate­ly 585,000 pa­tients on ibrex­a­fungerp in 2030)”

Mean­while, ri­val drug de­vel­op­er My­covia is con­duct­ing a pair of late-stage tri­als test­ing its long-act­ing azole VT-1161 in re­cur­rent VCC pa­tients — da­ta from both stud­ies are ex­pect­ed in the sec­ond half of 2020.

Tri­als test­ing the ibrex­a­fungerp’s use in in­va­sive as­pergillo­sis and re­frac­to­ry in­va­sive fun­gal in­fec­tions are al­so con­tin­u­ing undis­turbed, Tagli­et­ti said. “These are hos­pi­tal­ized pa­tients with se­vere in­fec­tions, in­va­sive in­fec­tions, that are life-threat­en­ing. There­fore Covid-19 or no Covid-19 — these pa­tients, they need some­thing.”

At Scynex­is, the biggest change since the ex­plo­sion of Covid-19 has been that the ma­jor­i­ty of the com­pa­ny’s work­force has been work­ing from home, al­though em­ploy­ees did have the op­tion to work from home for 2-3 days a week pre­vi­ous­ly.

“I can tell you that I don’t like to work re­mote­ly … there is no sep­a­ra­tion be­tween work and pri­vate life, there is no dif­fer­ence be­tween the week­end and work weeks, and every­thing be­comes Ground­hog Day. And hon­est­ly, the re­frig­er­a­tor is too close,” Tagli­et­ti quipped. “I read some­where that the 19 of Covid-19 are the num­ber of pounds that peo­ple will gain on av­er­age, by work­ing from home.”

When End­points News in­ter­viewed Tagli­et­ti this time last year, he re­layed his ex­pe­ri­ence that an­ti­fun­gals were not a pri­or­i­ty in Wash­ing­ton and that there were not ad­e­quate in­cen­tives in place to en­cour­age de­vel­op­ment ef­forts.

Since then, the CDC has put Can­di­da au­ris in its list of ur­gent threats, and the Covid-19 out­break, for bet­ter or for worse, has put the spot­light on how dev­as­tat­ing in­fec­tious dis­eases can be.

At the mo­ment, or­ga­ni­za­tions like NIH or BAR­DA or FDA in Wash­ing­ton are all square­ly fo­cused on Covid-19, and right­ly so —  it is a “pow­er­ful re­minder” that an­ti-in­fec­tives need to be con­tin­u­al­ly de­vel­oped, Tagli­et­ti said.

“And I do be­lieve that there will be a re­newed aware­ness and re­newed ap­pre­ci­a­tion of what an­ti-in­fec­tive com­pa­nies are do­ing,” he said. “There will be more sup­port from the pub­lic, pol­i­cy law­mak­ers, in­vestors and al­so Big Phar­ma.”

So­cial: Mar­co Tagli­et­ti via YouTube

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?

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Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

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The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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Clay Siegall, Morphimmune CEO

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Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

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FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

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