In a re­cent fire­side chat held at End­points News’ JPM26 event, Bob­by Aza­mi­an, MD, PhD, CEO and Chair­man of Tar­sus Phar­ma­ceu­ti­cals, joined End­points News’ Ar­salan Arif to dis­cuss how Tar­sus achieved its re­cent com­mer­cial suc­cess with a strat­e­gy of “cat­e­go­ry cre­ation,” and how it plans to ex­pand its eye care foot­print based on this premise.

One of Tar­sus’ core strengths is iden­ti­fy­ing large, un­der­served pa­tient pop­u­la­tions rou­tine­ly seen by physi­cians but large­ly over­looked by the phar­ma­ceu­ti­cal in­dus­try. Aza­mi­an shared how nu­anced ex­per­tise, ear­ly val­i­da­tion work and a touch of serendip­i­ty un­veiled the unique op­por­tu­ni­ty to tack­le the high­ly preva­lent prob­lem of De­mod­ex ble­phar­i­tis (eye­lid mites). The abil­i­ty to iden­ti­fy pa­tients at scale, run ro­bust clin­i­cal tri­als and pri­ori­tise clin­i­cal ed­u­ca­tion has helped the team es­tab­lish a new stan­dard of care with its first ap­proved prod­uct, which is al­so on a tra­jec­to­ry to hit block­buster sales.

Aza­mi­an out­lined how that same ap­proach is guid­ing Tar­sus’ next phase of growth. He em­pha­sized the role of ear­ly physi­cian and pa­tient ed­u­ca­tion in build­ing aware­ness for un­der­diag­nosed con­di­tions and sup­port­ing sus­tained adop­tion over time. Look­ing ahead, Tar­sus is ad­vanc­ing ad­di­tion­al pro­grams in oc­u­lar rosacea and Ly­me dis­ease pre­ven­tion, both in Phase 2 clin­i­cal tri­als. Eye care is the com­pa­ny’s core fo­cus, while pipeline di­ver­si­fi­ca­tion re­mains a strate­gic pri­or­i­ty, with po­ten­tial glob­al ex­pan­sion in Eu­rope and Japan in the com­ing years. The long-term vi­sion is cen­tered on scal­ing the pipeline with a num­ber of po­ten­tial block­busters and be­com­ing a lead­ing eye care com­pa­ny known for the cre­ation of new dis­ease cat­e­gories.

Key Take­aways:

• Tar­sus’ cat­e­go­ry cre­ation ap­proach seeks to serve high­ly preva­lent dis­eases over­looked by the phar­ma in­dus­try.
• The com­pa­ny’s first ap­proved prod­uct is on track to hit block­buster sales, with peak po­ten­tial still un­fold­ing.
• Phase 2 pipeline pro­grams and glob­al ex­pan­sion plans are shap­ing Tar­sus’ next chap­ter.

• Bob­by Aza­mi­an, MD, PhD, CEO and Chair­man, Tar­sus Phar­ma­ceu­ti­cals
• Ar­salan Arif, CEO and Co-Founder, End­points News

Arif: Here with Bob­by Aza­mi­an, CEO and Chair­man of Tar­sus Phar­ma­ceu­ti­cals. So Bob­by, re­al­ly im­pres­sive over here. He's a physi­cian sci­en­tist, Har­vard trained, Rhodes schol­ar. And then he left the clin­ic to start biotech com­pa­nies and to go af­ter dis­eases that no one else is pay­ing at­ten­tion to. So Tar­sus is worth bil­lions now, over 400 em­ploy­ees, and XDEMVY, their flag­ship drug, is on a block­buster tra­jec­to­ry. And they did it all by con­vinc­ing the world that eye­lid mites are a re­al prob­lem worth solv­ing. So wel­come to the End­points stage, Bob­by.

Aza­mi­an: Thank you, Ar­salan.

Arif: So of all the dis­eases that you could have tack­led, you could have gone af­ter can­cer, you could have gone af­ter Alzheimer's, you could have gone af­ter heart dis­ease, you went af­ter eye­lid mites. So walk me through that de­ci­sion.

Aza­mi­an: Did it and nev­er looked back. It's made all the dif­fer­ence. Yeah, so thanks for the kind words. I was an in­ternist, and I had a pas­sion for try­ing to solve some of those big chron­ic dis­eases. And what my team and I re­al­ized along the way is if you go af­ter a big dis­ease that no­body has ever gone af­ter, that can make all the dif­fer­ence. And we call that cat­e­go­ry cre­ation. And that's re­al­ly per­me­at­ed every­thing we've done from day one at Tar­sus.

And in a way, we've kind of flipped the biotech mod­el. We've said, "Let's take the deep­est risk on the dis­ease. We're go­ing to have to prove it." Ul­ti­mate­ly, we've done that by com­mer­cial­iz­ing XDEMVY, and we con­tin­ue to do that. But we've tried to take less risk at some of the oth­er ma­jor mile­stones along the way. Get a mol­e­cule that's re­al­ly tar­get­ed to the root cause of the dis­ease. Make sure that you're es­tab­lish­ing a great prod­uct pro­file through­out your stud­ies. Ul­ti­mate­ly, re­al­ly tai­lor the so­lu­tion for the doc­tor and the pa­tient and make that work com­mer­cial­ly.

So love all those clin­i­cal prob­lems you men­tioned. We have a lot more prob­lems to tack­le at Tar­sus. We can talk about how cat­e­go­ry cre­ation is scaled, but eye­lid mites have made all the dif­fer­ence for us.

Arif: So the mol­e­cule, it was al­ready proven in pet flea col­lars, right? So was there a mo­ment where you looked at that and said, "Yes, this mol­e­cule is go­ing to work in hu­mans"?

Aza­mi­an: There was, yeah. I mean, there's been sev­er­al mo­ments of serendip­i­ty along the way. In terms of the mol­e­cule, when our team in the ear­ly days was try­ing to find the best an­tipar­a­sitic that we could put in an oc­u­lar prod­uct, an eye drop, and treat the root cause of mites, we had a mo­ment that we chuck­le about where we were look­ing at all the great an­tipar­a­sitics and we found that an­i­mals have mites too, they have mange. And so we said, "Let's go study this in mangy dogs and cats. Let's try to get some proof of con­cept."

And as we went to our friend­ly vet­eri­nary der­ma­tol­o­gist, ac­tu­al­ly my co-founder's sis­ter, she said, "I would, but I don't have mangy dogs and cats any­more be­cause of this new class of chem­istry called the isox­a­zo­lines." And that's when our ears perked up. We said, "What is that? How do you spell it?" And we were very for­tu­nate to li­cense loti­lan­er from Elan­co and bring it to all of hu­man med­i­cine, start­ing with De­mod­ex ble­phar­i­tis, but as we'll talk about, cre­at­ing oth­er cat­e­gories, hope­ful­ly with oc­u­lar rosacea and Ly­me treat­ments as well.

Arif: Okay. So Bob­by, you've said, and I've seen you say this in a cou­ple dif­fer­ent places, "If there's one thing that Tar­sus is about, it's about cre­at­ing a new cat­e­go­ry of med­i­cines." So what's the dif­fer­ence be­tween find­ing white space and chas­ing some­thing that no­body wants?

Aza­mi­an: Well, I would ar­gue that you al­ways want to chase some­thing that some­body wants. So when we talk about cat­e­go­ry cre­ation, it is chas­ing those white spaces that peo­ple have been try­ing to solve and that pa­tients and doc­tors want a so­lu­tion for, but maybe the phar­ma in­dus­try has not want­ed to go af­ter for var­i­ous rea­sons. So that's how I would frame it.

The sec­ond piv­otal tri­al of Com­pass Path­ways’ psilo­cy­bin for­mu­la­tion for treat­ment-re­sis­tant de­pres­sion has suc­ceed­ed, the com­pa­ny said Tues­day, a re­sult that could help the drug gain ap­proval next year.

The first ap­proval tri­al of the pill, which is known as COMP360, hit its pri­ma­ry end­point in June, al­though by a mar­gin slim­mer than Com­pass had hoped. At that point the com­pa­ny near­ly halved in val­ue.

This new study has re­versed Com­pass’ for­tunes to some ex­tent: Its shares CMPS rose more than 35% in mid­morn­ing trad­ing.

The study, called COMP006, had an un­usu­al de­sign. Un­like the first piv­otal tri­al, which com­pared COMP360 with place­bo, the sec­ond pit­ted dif­fer­ent dos­es against each oth­er. Rough­ly half the pa­tients in the tri­al took two 25 mg dos­es of the drug three weeks apart. A quar­ter of the tri­al’s sub­jects re­ceived two dos­es of 10 mg, and an­oth­er quar­ter two dos­es of 1 mg.

The re­jec­tion of Disc Med­i­cine’s rare dis­ease treat­ment marks the first neg­a­tive out­come of the FDA's con­tro­ver­sial vouch­er pro­gram and sug­gests drugs that are se­lect­ed won't be guar­an­teed an ap­proval.

Dis­c's bitop­er­tin was an ear­ly test case of the Com­mis­sion­er's Na­tion­al Pri­or­i­ty Vouch­er (CN­PV) pro­gram, a pi­lot that aims to sig­nif­i­cant­ly short­en the typ­i­cal 10- to 12-month re­view times. The FDA said in a com­plete re­sponse let­ter from Fri­day that it had ques­tions about the bio­mark­er in­tend­ed to be used for ac­cel­er­at­ed ap­proval.

An­a­lysts viewed the re­jec­tion as a sur­prise, par­tic­u­lar­ly since Disc was one of the first com­pa­nies to get the vouch­er.

Plus, news about Cyra­no, Sanofi and Teva's du­vak­i­tug, Roche’s Gazy­va, Eli Lil­ly's Retev­mo, John­son & John­son’s Ry­bre­vant, Newron Phar­ma­ceu­ti­cals, and Gelmedix: