Io­n­is is fo­cused on treat­ing the un­treat­able with med­ical break­throughs for pa­tients

By cre­at­ing a bet­ter way for drug dis­cov­ery and de­vel­op­ment, Io­n­is is now re­al­iz­ing the full po­ten­tial of its an­ti­sense tech­nol­o­gy.

With two treat­ments ap­proved in the last two years and at least four more head­ed in­to late-stage clin­i­cal tri­als by the end of 2019, Io­n­is Phar­ma­ceu­ti­cal’s nov­el ther­a­pies are treat­ing the un­treat­able and bring­ing hope to sick peo­ple around the world. The com­pa­ny is fo­cused on us­ing its an­ti­sense tech­nol­o­gy to pro­duce break­through ther­a­pies to treat a range of dis­eases that have not be­fore been ef­fec­tive­ly tack­led.

Be­fore Io­n­is cre­at­ed SPIN­RAZA®*, and it be­came the stan­dard of care in the treat­ment of spinal mus­cu­lar at­ro­phy (SMA), chil­dren born with the con­di­tion lived with pro­gres­sive de­gen­er­a­tion and had a dras­ti­cal­ly short­ened life ex­pectan­cy. To­day, ba­bies born with the dis­ease, who are treat­ed pre-symp­to­mati­cal­ly, are de­vel­op­ing sim­i­lar­ly to healthy ba­bies. Io­n­is went on to de­vel­op TEGSE­DI™, the world’s first RNA-tar­get­ing sub­cu­ta­neous in­jec­tion to treat polyneu­ropa­thy caused by hered­i­tary transthyretin-me­di­at­ed amy­loi­do­sis (hAT­TR) in adults. A de­gen­er­a­tive and fa­tal dis­ease that dras­ti­cal­ly im­pacts peo­ple’s abil­i­ty to car­ry out their ba­sic ac­tiv­i­ties of dai­ly liv­ing, hAT­TR has se­vere­ly lim­it­ed the lives of those liv­ing with the dis­ease. TEGSE­DI sig­nif­i­cant­ly re­duces lev­els of the dis­ease-caus­ing pro­tein, of­fer­ing ef­fec­tive treat­ment with a sim­ple in­jec­tion that gives pa­tients the free­dom to treat where and when they choose.

This is ex­act­ly what Stan­ley T. Crooke, M.D., Ph.D., Io­n­is Founder, Chair­man and CEO, en­vi­sioned when he launched the com­pa­ny in 1989. Dr. Crooke knew that with the right sci­ence, the right peo­ple, and an un­wa­ver­ing com­mit­ment to a bet­ter way of dis­cov­er­ing and de­vel­op­ing drugs, Io­n­is could de­liv­er tru­ly in­no­v­a­tive treat­ments to pa­tients where no oth­ers have proven ef­fec­tive or, in some cas­es, ever ex­ist­ed.

“We wake up every day think­ing about the sick peo­ple who de­pend on us. There is a tremen­dous sense of grat­i­fi­ca­tion that comes from know­ing the time spent in the lab can change lives in such a sig­nif­i­cant way. And this is on­ly the be­gin­ning. With po­ten­tial­ly 10 pro­grams en­ter­ing piv­otal tri­als by 2020, the pace of in­no­va­tion at Io­n­is is on­ly in­creas­ing,” said Dr. Crooke.

Io­n­is Lead­ing in In­no­v­a­tive RNA-Tar­get­ed Ther­a­peu­tics

This pas­sion is dri­ving un­prece­dent­ed pro­duc­tiv­i­ty in the Io­n­is pipeline that in­cludes 45 po­ten­tial first-in-class or best-in-class ther­a­pies for a broad range of dis­eases. All of this is be­ing ac­com­plished by a team of sci­en­tists who are com­mit­ted to ful­ly re­al­iz­ing the po­ten­tial of an­ti­sense tech­nol­o­gy and de­liv­er­ing trans­for­ma­tive med­i­cines to those who need them to­day and long in­to the fu­ture.

“From the rarest of dis­eases like Pri­on and Amy­otroph­ic Lat­er­al Scle­ro­sis (ALS) to those that im­pact mil­lions of peo­ple like car­dio­vas­cu­lar dis­ease, we nev­er turn down a treat­ment chal­lenge. If the need is great and we be­lieve our tech­nol­o­gy is the way for­ward, we will say yes and work to find new ther­a­pies,” said Dr. Crooke.

As the leader in the dis­cov­ery and de­vel­op­ment of RNA-tar­get­ed ther­a­peu­tics, Io­n­is has evolved its nov­el an­ti­sense tech­nol­o­gy. An­ti­sense med­i­cines in­ter­act pre­cise­ly with spe­cif­ic se­quences of RNA that dis­rupt the dis­ease process and change its course. The next wave of Io­n­is-de­vel­oped treat­ments will con­tin­ue to ad­dress in­tractable rare dis­eases, as well as dis­eases im­pact­ing mil­lions of peo­ple.

In No­vem­ber, Io­n­is and its com­mer­cial af­fil­i­ate Akcea an­nounced Phase 2 da­ta eval­u­at­ing AKCEA-APO(a)-LRx. The tri­al was the largest study ever con­duct­ed in pa­tients with car­dio­vas­cu­lar dis­ease (CVD) and el­e­vat­ed lipopro­tein(a), al­so known as Lp(a), high lev­els of which triple the risk of hav­ing a heart at­tack or stroke at an ear­ly age. Re­sults from the study showed that AKCEA-APO(a)-LRx sig­nif­i­cant­ly low­ered lev­els of Lp(a) in pa­tients with pre-ex­ist­ing car­dio­vas­cu­lar dis­ease.

“Be­cause Lp(a) can­not be eas­i­ly tar­get­ed with small mol­e­cules or an­ti­bod­ies, in­hi­bi­tion of apolipopro­tein(a) is a great ex­am­ple of how an­ti­sense tech­nol­o­gy can have an im­pact where no oth­er ther­a­peu­tic ap­proach­es have proven ef­fec­tive. A drug that re­duces Lp(a) could be a par­a­digm shift for the car­dio­vas­cu­lar com­mu­ni­ty and the 8 to 10 mil­lion treat­able pa­tients es­ti­mat­ed to be liv­ing with car­dio­vas­cu­lar dis­ease and el­e­vat­ed lev­els of Lp(a),” said Brett Mo­nia, Ph.D., Io­n­is’ chief op­er­at­ing of­fi­cer.

Al­so head­ing in­to a piv­otal Phase 3 tri­al in 2019 is IO­N­IS-HTTRx (RG6042), the first po­ten­tial­ly dis­ease-mod­i­fy­ing ther­a­py to be dis­cov­ered and de­vel­oped to treat Hunt­ing­ton’s Dis­ease. Hunt­ing­ton’s Dis­ease is a fa­tal, in­her­it­ed brain dis­or­der that re­sults in the pro­gres­sive loss of both men­tal fac­ul­ties and phys­i­cal con­trol. Cur­rent­ly avail­able treat­ments on­ly mask symp­toms but do not slow the un­der­ly­ing pro­gres­sion of the dis­ease. Pa­tients treat­ed with IO­N­IS-HTTRx saw re­duced lev­els of the dis­ease-caus­ing hunt­ingtin pro­tein and for the first time in his­to­ry, Hunt­ing­ton’s Dis­ease pa­tients have new hope for a drug that treats the un­der­ly­ing cause of the dis­ease.

“We’re on the precipice of find­ing ef­fec­tive ther­a­pies in so many ar­eas. We are con­stant­ly work­ing to broad­en the util­i­ty of an­ti­sense tech­nol­o­gy and ful­ly cap­i­tal­ize on the breadth of its ther­a­peu­tic po­ten­tial to bring hope to pa­tients and val­ue to share­hold­ers. This cou­pled with our in­no­va­tion-cen­tered busi­ness has cre­at­ed a high­ly ef­fi­cient, high­ly pro­duc­tive drug dis­cov­ery and de­vel­op­ment en­gine that will con­tin­ue to bring trans­for­ma­tive ther­a­pies to the world for years to come,” said Dr. Crooke.

For more in­for­ma­tion, vis­it Ion­is­phar­

*SPIN­RAZA® is mar­ket­ed by Bio­gen.


Ionis Pharmaceuticals