Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

Sep­tem­ber 23, 2020 – Hong Kong, Bei­jing, Shang­hai (Chi­na) and Mataró, Barcelona (Spain)  

• Mi­no­ryx will re­ceive an up­front and mile­stone pay­ments of up to $78 mil­lion, as well as dou­ble dig­it roy­al­ties on an­nu­al net sales 

• Sper­o­genix will re­ceive ex­clu­sive rights to de­vel­op and com­mer­cial­ize lerigli­ta­zone for the treat­ment of X-linked adrenoleukody­s­tro­phy (X-ALD), a rare life-threat­en­ing neu­ro­log­i­cal con­di­tion

Sper­o­genix Ther­a­peu­tics, a plat­form com­pa­ny ded­i­cat­ed to de­vel­op­ing and com­mer­cial­iz­ing rare dis­ease ther­a­peu­tics in Chi­na, and Mi­no­ryx Ther­a­peu­tics, a com­pa­ny that spe­cial­izes in the de­vel­op­ment of in­no­v­a­tive treat­ments for or­phan Cen­tral Ner­vous Sys­tem (CNS) dis­eases, to­day an­nounce that they have en­tered in­to an ex­clu­sive li­cense agree­ment for the de­vel­op­ment and com­mer­cial­iza­tion of lerigli­ta­zone, Mi­no­ryx’s brain pen­e­trat­ing dis­ease-mod­i­fy­ing PPAR-y ag­o­nist.

Un­der the terms of the agree­ment, Sper­o­genix will re­ceive ex­clu­sive rights to de­vel­op and com­mer­cial­ize lerigli­ta­zone in main­land Chi­na, Hong Kong spe­cial ad­min­is­tra­tive re­gion (SAR) and Macau SAR for the treat­ment of X-linked adrenoleukody­s­tro­phy (X-ALD) a life-threat­en­ing or­phan neu­ro­log­i­cal con­di­tion. Mi­no­ryx will re­ceive an ini­tial up­front pay­ment and pre-de­fined reg­u­la­to­ry and com­mer­cial mile­stone pay­ments of up to $78 mil­lion , as well as dou­ble-dig­it roy­al­ties on an­nu­al net sales.

Lerigli­ta­zone is a nov­el bioavail­able and se­lec­tive PPAR-γ ag­o­nist, with the po­ten­tial to be­come the world’s first treat­ment for X-ALD. It has re­ceived Or­phan Drug Des­ig­na­tion for X-ALD in both the U.S. and Eu­rope, and Fast Track Des­ig­na­tion as well as Rare Pe­di­atric Dis­ease Des­ig­na­tion from the U.S. FDA. A reg­is­tra­tion en­abling tri­al in adult X-ALD pa­tients with adreno­myeloneu­ropa­thy (AMN) is cur­rent­ly on­go­ing in the EU and in the U.S. (AD­VANCE tri­al). It is a glob­al mul­ti-cen­tric, dou­ble-blind, place­bo-con­trolled Phase 2/3 study with da­ta ex­pect­ed by the end of 2020. In ad­di­tion, lerigli­ta­zone is be­ing eval­u­at­ed in a reg­is­tra­tion en­abling open-la­bel Phase 2 study in pe­di­atric pa­tients with cere­bral X-ALD (cALD) in Eu­rope, with topline re­sults an­tic­i­pat­ed by mid-2021.

“This ex­clu­sive li­cense agree­ment en­tered for lerigli­ta­zone fur­ther strength­ens Sper­o­genix’s pipeline in neu­ro­log­i­cal rare dis­eases,” said Mr. Alan (Zhiyu) Yan, co-founder, chair­man and CEO of Sper­o­genix. “It demon­strates our strong ded­i­ca­tion and long-term com­mit­ment to ad­dress the huge un­met med­ical needs in the rare dis­ease field in Chi­na. We look for­ward to col­lab­o­rat­ing with all stake­hold­ers to bring this ther­a­py to Chi­nese pa­tients as ear­ly as pos­si­ble.”

“We are very pleased to en­ter in­to this ex­clu­sive li­cense agree­ment with Sper­o­genix based on its deep in­sights in rare dis­eases in Chi­na and its unique ca­pa­bil­i­ties, which make us high­ly con­fi­dent in our part­ner­ship,” said Dr. Marc Mar­tinell, co-founder and CEO of Mi­no­ryx. “This is a ma­jor mile­stone that un­der­scores the po­ten­tial of lerigli­ta­zone to ad­dress an im­por­tant un­met med­ical need.”

About Sper­o­genix Ther­a­peu­tics 

Sper­o­genix Ther­a­peu­tics is a plat­form com­pa­ny ded­i­cat­ed to de­vel­op­ing and com­mer­cial­iz­ing rare dis­ease ther­a­peu­tics in Chi­na with fo­cus on mid-to-late clin­i­cal stage and com­mer­cial stage prod­ucts. With pri­or­i­tized ther­a­peu­tic ar­eas, such as Pul­monary Vas­cu­lar Dis­or­ders, Neu­ro­log­i­cal Dis­or­ders, In­her­it­ed Meta­bol­ic Dis­eases, and Non-on­col­o­gy Hema­tol­ogy Dis­or­ders, Sper­o­genix is ded­i­cat­ed to es­tab­lish­ing an in­no­v­a­tive com­mer­cial mod­el tai­lored to the Chi­na rare dis­ease field, in or­der to pro­vide af­ford­able and re­li­able prod­ucts and ser­vices to Chi­nese physi­cians and pa­tients. Sper­o­genix was found­ed in 2019 and is backed by bio­phar­ma in­dus­try blue chip in­vestors in­clud­ing Lil­ly Asia Ven­tures (LAV) and Morn­ing­side Ven­ture in the A-round fi­nanc­ing.

www.sper­o­genix.com

About Mi­no­ryx Ther­a­peu­tics

Mi­no­ryx is a clin­i­cal stage biotech com­pa­ny fo­cus­ing on the de­vel­op­ment of nov­el ther­a­pies for or­phan CNS dis­eases with high un­met med­ical needs. The com­pa­ny’s lead pro­gram, lerigli­ta­zone (MIN-102), a nov­el, se­lec­tive PPARγ ag­o­nist, is cur­rent­ly be­ing eval­u­at­ed in X-ALD and Friedre­ich’s Atax­ia. The com­pa­ny is backed by a syn­di­cate of ex­pe­ri­enced in­vestors, which in­cludes Caixa Cap­i­tal Risc, Roche Ven­ture Fund, Ys­ios Cap­i­tal, Kur­ma Part­ners, Fund+, Chiesi Ven­tures, S.R.I.W, Id­in­vest, SF­PI-FPIM, HealthE­quity and Sam­brin­vest, and has sup­port from a net­work of oth­er or­ga­ni­za­tions. Mi­no­ryx was found­ed in 2011, has op­er­a­tions in Spain and Bel­gium and has so far raised more than €50M.

www.mi­no­ryx.com

About lerigli­ta­zone

Lerigli­ta­zone (MIN-102) is a nov­el bioavail­able and se­lec­tive PPAR-γ ag­o­nist with a po­ten­tial best-in-class pro­file in­di­cat­ed for CNS dis­eases. It has a demon­strat­ed suf­fi­cient brain pen­e­tra­tion and fa­vor­able safe­ty pro­file. It showed ro­bust pre­clin­i­cal proof-of-con­cept in an­i­mal mod­els of mul­ti­ple dis­eases by mod­u­lat­ing path­ways lead­ing to mi­to­chon­dr­i­al dys­func­tion, ox­ida­tive stress, neu­roin­flam­ma­tion, de­myeli­na­tion and ax­on­al de­gen­er­a­tion. Lerigli­ta­zone has suc­cess­ful­ly com­plet­ed a phase 1 clin­i­cal tri­al show­ing good safe­ty, tol­er­a­bil­i­ty and CNS en­gage­ment of PPAR-γ re­cep­tors at lev­els equiv­a­lent to those re­quired for ef­fi­ca­cy in pre­clin­i­cal mod­els. Lerigli­ta­zone has the po­ten­tial to treat sev­er­al CNS dis­or­ders, in­clud­ing or­phan dis­eases, and is cur­rent­ly be­ing eval­u­at­ed in a reg­is­tra­tion en­abling Phase 2/3 study in AMN, a reg­is­tra­tion en­abling Phase 2 in cALD and in a Phase 2 in Friedre­ich’s Atax­ia.

About X-ALD

X-ALD (X-linked adrenoleukody­s­tro­phy) is an or­phan neu­rode­gen­er­a­tive dis­ease. AMN and cALD are the two most com­mon phe­no­types of X-ALD, which ac­count for 45% and 35% re­spec­tive­ly. The glob­al in­ci­dence of X-ALD is ap­prox­i­mate­ly 6.2/100,000 live births. X-ALD is in­clud­ed in the first Chi­na Rare Dis­ease Cat­a­logue pub­lished in 2018.

The age of on­set of cALD pa­tients is typ­i­cal­ly 4-8 years old. Un­treat­ed pa­tients progress quick­ly, as se­vere neu­ro­log­i­cal func­tion im­pair­ment ap­pears 6-24 months af­ter dis­ease on­set, lead­ing to ear­ly death in 2-4 years.

MN is the most preva­lent form and is char­ac­ter­ized by pro­gres­sive spas­tic para­pare­sis, sen­so­ry dys­func­tion and in­con­ti­nence. This form pro­gress­es chron­i­cal­ly with on­set of symp­toms typ­i­cal­ly in adult­hood and poor prog­no­sis.

There is cur­rent­ly no ther­a­peu­tic treat­ment avail­able for X-ALD. Sev­er­al ob­ser­va­tion­al stud­ies have demon­strat­ed that hematopoi­et­ic stem cell trans­plan­ta­tion (HSCT) may im­prove the five-year over­all sur­vival for cALD pa­tients. How­ev­er, there is no ev­i­dence that HSCT im­proves clin­i­cal out­comes of pa­tients with AMN.

For fur­ther in­for­ma­tion, please con­tact:
For Mi­no­ryx： juli­ette@ala.com
For Sper­ro­genix: lei.xi­ao@sper­o­genix.com