A New Fron­tier: The In­ner Ear

What hap­pens when a suc­cess­ful biotech ven­ture cap­i­tal­ist is un­ex­pect­ed­ly di­ag­nosed with a chron­ic, life-dis­rupt­ing ver­ti­go dis­or­der? In­no­va­tion in neu­ro­tol­ogy.

That ven­ture cap­i­tal­ist was Jay Lichter, Ph.D., and af­ter learn­ing there was no FDA-ap­proved drug treat­ment for his con­di­tion, Ménière’s dis­ease, he de­cid­ed to cre­ate a com­pa­ny to bring drug de­vel­op­ment to neu­ro­tol­ogy. Oton­o­my was found­ed in 2008 and is ded­i­cat­ed to find­ing new drug treat­ments for the huge­ly un­der­served com­mu­ni­ty liv­ing with bal­ance and hear­ing dis­or­ders. Help­ing pa­tients like Jay has been the dri­ving force be­hind Oton­o­my, a com­pa­ny head­ing in­to a trans­for­ma­tive 2020 with three clin­i­cal tri­al read­outs: Phase 3 in Ménière’s dis­ease, Phase 2 in tin­ni­tus, and Phase 1/2 in hear­ing loss. These cat­a­lysts, to­geth­er with oth­ers in the field, high­light the emerg­ing op­por­tu­ni­ty in neu­ro­tol­ogy.

Oton­o­my is lead­ing the way in neu­ro­tol­ogy

Neu­ro­tol­ogy, or the treat­ment of in­ner ear neu­ro­log­i­cal dis­or­ders, is a large and un­tapped mar­ket for drug de­vel­op­ers: one in eight in­di­vid­u­als in the U.S. have mod­er­ate-to-se­vere hear­ing loss, tin­ni­tus or ver­ti­go dis­or­ders such as Ménière’s dis­ease.¹ With no FDA-ap­proved drug treat­ments avail­able for these con­di­tions, the bur­den on pa­tients—in­clud­ing so­cial anx­i­ety, low­er qual­i­ty of life, re­duced work pro­duc­tiv­i­ty, and high­er rates of de­pres­sion—can be sig­nif­i­cant.^{2, 3, 4}

A ma­jor rea­son for the lack of ther­a­pies is the chal­lenge of de­liv­er­ing drugs to the hear­ing and bal­ance or­gans, which are pro­tect­ed with­in the in­ner ear. Us­ing patent­ed for­mu­la­tion tech­nol­o­gy, Oton­o­my pi­o­neered the abil­i­ty to achieve sus­tained ot­ic drug lev­els via a sin­gle lo­cal ad­min­is­tra­tion called an in­tratym­pan­ic in­jec­tion. This is a sim­ple pro­ce­dure rou­tine­ly per­formed by an ENT in the of­fice and it holds the po­ten­tial to rev­o­lu­tion­ize treat­ment for neu­ro­tol­ogy in the same way that in­trav­it­re­al in­jec­tions trans­formed treat­ment in oph­thal­mol­o­gy.

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“As a physi­cian who dai­ly sees the sig­nif­i­cant im­pact that hear­ing and bal­ance dis­or­ders can have on pa­tients and their fam­i­lies, I am ex­cit­ed by the prospect of new drug treat­ments for these con­di­tions,” says An­tho­ny Mikulec, MD, MBA, Chief of Otol­ogy and Neu­ro­tol­ogy and Pro­fes­sor at St. Louis Uni­ver­si­ty Med­ical Cen­ter. “By over­com­ing the chal­lenge of de­liv­er­ing drugs to the in­ner ear, Oton­o­my is lead­ing the way in neu­ro­tol­ogy drug de­vel­op­ment and spurring in­ter­est in the field that now in­cludes a breadth of clin­i­cal pro­grams hold­ing great promise for pa­tients.”

While most peo­ple are prob­a­bly fa­mil­iar with hear­ing loss due to ag­ing, there is low aware­ness of oth­er types of neu­ro­to­log­i­cal dis­or­ders—de­spite the large num­bers of peo­ple af­fect­ed by symp­toms that can be life-al­ter­ing.

Clin­i­cal pro­gram: Ménière’s dis­ease

Take for in­stance Ménière’s dis­ease, an acute ver­ti­go dis­or­der ac­com­pa­nied by hear­ing loss and tin­ni­tus that af­fects more than 850,000 peo­ple in the U.S. As ex­pe­ri­enced by Oton­o­my’s founder, the ver­ti­go at­tacks as­so­ci­at­ed with Ménière’s dis­ease oc­cur with­out warn­ing, last from min­utes to hours and can re­cur for months. When the world is spin­ning, pa­tients can’t func­tion ei­ther at home or at work, and there are no FDA-ap­proved drug treat­ments to help re­duce the fre­quen­cy or sever­i­ty of at­tacks. Oton­o­my has com­plet­ed one suc­cess­ful Phase 3 tri­al for OTIVIDEX™, a sus­tained-ex­po­sure steroid for­mu­la­tion, and re­sults of an on­go­ing Phase 3 tri­al are ex­pect­ed in the third quar­ter of 2020.

Clin­i­cal pro­gram: Tin­ni­tus

Tin­ni­tus (ring­ing in the ear) is an­oth­er neu­ro­to­log­i­cal con­di­tion that can sig­nif­i­cant­ly af­fect a per­son’s phys­i­cal and emo­tion­al well-be­ing. Thir­ty mil­lion peo­ple in the U.S. ex­pe­ri­ence tin­ni­tus, and for 8 mil­lion the ring­ing is se­vere enough that it im­pacts their abil­i­ty to sleep, re­lax and con­cen­trate at work. In these cas­es, anx­i­ety and de­pres­sion are com­mon. Un­for­tu­nate­ly for pa­tients, there are no FDA-ap­proved tin­ni­tus drug treat­ments avail­able. To ad­dress this need, Oton­o­my is de­vel­op­ing OTO-313, a sus­tained-ex­po­sure for­mu­la­tion of the po­tent and se­lec­tive NM­DA re­cep­tor an­tag­o­nist gacy­cli­dine. Re­sults from a Phase 2 tri­al of OTO-313 are ex­pect­ed in the sec­ond quar­ter of 2020.

Clin­i­cal pro­gram: Hear­ing Loss

Af­fect­ing near­ly half a bil­lion peo­ple world­wide,⁵ hear­ing loss is the most com­mon dis­or­der in neu­ro­tol­ogy and is on­ly ex­pect­ed to grow as our pop­u­la­tion ages and our world be­comes nois­i­er. Of­ten, the first sign is dif­fi­cul­ty un­der­stand­ing a con­ver­sa­tion in a noisy en­vi­ron­ment. Re­searchers call this “speech-in-noise” hear­ing dif­fi­cul­ty and have demon­strat­ed that it is due to dam­aged neu­ronal con­nec­tions in the ear. Oton­o­my is eval­u­at­ing OTO-413, a sus­tained-ex­po­sure for­mu­la­tion of the nerve growth fac­tor BD­NF, as a po­ten­tial treat­ment for speech-in-noise hear­ing loss. Re­sults from a Phase 1/2 tri­al of OTO-413 are ex­pect­ed in the sec­ond half of this year.

“Neu­ro­to­log­i­cal dis­eases af­fect mil­lions of peo­ple of all ages, and they all de­serve a fu­ture less en­cum­bered by the dai­ly bur­den of hear­ing and bal­ance dis­or­ders,” says David A. We­ber, Ph.D., Pres­i­dent and CEO of Oton­o­my. “Our three clin­i­cal mile­stones in 2020 pro­vide a trans­for­ma­tion­al op­por­tu­ni­ty for Oton­o­my and will, we hope, serve as an im­por­tant in­flec­tion point for the emer­gence of this ex­cit­ing new field.”

To fol­low the lat­est de­vel­op­ments on the neu­ro­tol­ogy field and Oton­o­my, vis­it www.oton­o­my.com.

Ac­tu­al re­sults may dif­fer from those in­di­cat­ed from the for­ward-look­ing state­ments here­in as re­sult of risks and un­cer­tain­ties as de­scribed in the risk fac­tors in Oton­o­my’s fil­ings with the SEC.

¹ ClearView Health­care Part­ners analy­sis.
² Ruther­ford BR, Brew­ster K, Gol­ub JS, et al. Sen­sa­tion and psy­chi­a­try: link­ing age-re­lat­ed hear­ing loss to late-life de­pres­sion and cog­ni­tive de­cline. Am J Psy­chi­a­try. 2018;175(3):215-224.
³ Amer­i­can Tin­ni­tus As­so­ci­a­tion. Im­pact of tin­ni­tus. https://www.ata.org/un­der­stand­ing-facts/im­pact-tin­ni­tus. Ac­cessed 6 June 2019.
⁴ Tyrell et al. The Cost of Ménière’s Dis­ease: A Nov­el Mul­ti­source Ap­proach. Ear & Hear­ing. 2016;37(3):e202-209.
⁵ World Health Or­ga­ni­za­tion. Glob­al es­ti­mates on preva­lence of hear­ing loss. [Pre­sen­ta­tion] 2018. https://www.who.int/pbd/deaf­ness/es­ti­mates/en. Ac­cessed 5 June 2019.