Breaking the Vicious Circle of Dry Eye DiseaseRx Dry Eye ; aqueous deficient . 3Q2019: FPFV 2: nd....
Transcript of Breaking the Vicious Circle of Dry Eye DiseaseRx Dry Eye ; aqueous deficient . 3Q2019: FPFV 2: nd....
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Breaking the Vicious Circle of Dry Eye DiseaseBernhard Guenther, Founder & President Novaliq Inc.
OIS@SECO | Feb 21th 2019, New Orleans
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Challenge water-based drug deliveryOnly 1-10% of a water-based topical eye medication reachestarget tissue
First and only water-free technology – EyeSol®Technology is clinically validated, safe and well accepted
EyeSol® unlocks the full potential of APIs for drug treatmentsPipeline addresses all key indications in Ophthalmology
NOV03 and CyclASol® have potential to redefine current DED treatmentsBoth programs well positioned to generate two NDA submissions by 2021
Novaliq is Transforming Ocular Therapeutics
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EyeSol® Technology – First and Only Water-free Drug Delivery Technology
1) ARVO 2018, Poster: Ocular and Systemic Distribution of 14C-Perfluorohexyloctane following Topical Ocular Administration to Rabbits2) Agarwal et al.; Semifluorinated alkane based systems for enhanced corneal penetration of poorly soluble drugs; IntJPharm 2018; 538:119-129
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Pathogenesis-driven Dry Eye Disease Clinical PhenotypesOcular Surface Damage and Tear Film Instability impair Visual Function
aqueous deficient
evaporative
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• Aqueous deficient DED− Early onset of action − Sustained efficacy on ocular surface healing− High responder rates− Impact on visual symptoms
• Critical Mode-of-Action in DED − Immunomodulator & anti-inflammatory
• Outstanding tolerability & safety profile
• Evaporative DED associated with MGD− Impressive effect on a variety of symptoms− Early onset of action − Treats signs and symptoms of DED
• Unique Mode-of-Action− Stabilizes lipid layer for hours− Penetrates meibomian glands
• Outstanding tolerability & safety profile
Two Late-Stage Drug Candidates in Dry Eye – Breaking the Vicious Circle of DED
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Recent Randomized Double Masked Controlled US Trials
ESSENCE: 328 patients pivotal US trial Predominantly aqueous deficient DED
• Low Schirmer: ~5 mm• Significant corneal damage
• Central cornea involved• Symptomatic, OSDI: ~45
SEECASE: 336 patients phase 2 US trial Predominantly evaporative DED associated with Meibomian Gland Dysfunction (MGD)
• Low TBUT: ~ 3 sec• MGD involvement
• Normal Schirmer: ~15 mm• Highly symptomatic, OSDI: ~55 • Mild to moderate corneal damage
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NOV03 – Clinically Significant Improvements in Signs and Symptoms of DED
SEECASE trial met its prespecified primary sign endpoints at 8 weeksHighly clinically relevant improvements in various symptoms over active control
Severely symptomatic population characterized by MGD and low TFBUT
tCFS scale ranges from 0 to 15; tCFS graph relates to „study eyes“; error bars show standard error of the mean (SEM); VAS scales ranges from 0 to 100, p-values relate to two sided testing
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CyclASol® 0.1% – Clinical Significant Improvement in Sign and Symptoms
Significant symptom improvements at primary endpoint
Onset of effect as early as 2 weeks and sustained over trial duration
tCFS scale ranges from 0 to 15; tCFS graph relates to „study eyes“; error bars show standard error of the mean (SEM); VAS scales ranges from 0 to 100, p-values relate to two sided testing
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Highly significant and clinical meaningful benefit in majority of patients
High responder rates on cornea and conjunctiva
CyclASol® 0.1% – Majority of Patients Benefit from Treatment
all graphs relate to „study eyes“, scales are cCFS: 0-3, tCFS: 0-15, conjunctival: 0-10, p-values relate to Fisher‘s exact test
p=0.004 p=0.03 p=0.03
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Dry Eye Disease Impacts Visual FunctionImprovement in Corneal Fluorescein Staining is associated with improvements in visual function
ESSENCE data show response in tCFS resulted in a clinically relevant improvement in reading speed (>10 words per minute)
*p=0.049
p=0.296
***p=0.0009
tCFS response was defined as ≥ 3 scores (NEI scale) improvement from baseline
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CyclASol® 0.1% – Fastest Onset of Action with Excellent Tolerability
Instillation site irritation (burning) in % of patients (meta-analysis)0 5 10 15 20 25 30
2%
2.5%
Onset of action defined as primary sign improvement statistically superior to vehicle(in months)
0 1 2 5 7643
CYS-0020.05% (N=51)
0.1% (N=51)
ESSENCE (CYS-003)0.1% (N=162)
2%
CYS-002ESSENCE (CYS-003)
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Novaliq DED Rx Portfolio Has Ability to Redefine DED Therapy
SEECASE (NOV03) and ESSENCE (CyclASol® 0.1%) provide novelties in DED therapy in two distinct patient groups
1) Outcomes for both studies are clinically relevant
2) Both products have the potential to treat more patients for better treatment outcomes:
• NOV03 for treating evaporative DED associated with Meibomian Gland Dysfunction
• CyclASol® is intended for treating predominantly aqueous deficient DED
3) Both products show an unprecedented risk - benefit profile
Novaliq intends to accelerate both development programs for NDA submission in 2021
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Novaliq’s Clinical Pipeline
Product/Program Indication Disc. Pre-clinic Phase I Phase II Phase III Reg. Market Near-term milestonesCyclASol® (cyclosporin)
Rx Dry Eye aqueous deficient
3Q2019: FPFV 2nd Phase 3NDA submission 2021
NOV03(perfluorohexyloctane)
Rx Dry Eye evaporative, MGD*
4Q2019: FPFV final Phase 3NDA submission 2021
NOV07(cannabinoid)
Rx Dry Eyeocular pain Prepare for clinical phase
Dry Eye Disease Product Family
Glaucoma & Retina Development Programs
Product/Program Indication Disc. Pre-clinic Phase I Phase II Phase III Reg. Market Near-term milestones
NOV04 Rx Glaucoma(topical)
License-out and develop a glaucoma asset in the USA
NOV05(Tacrolimus)
Non infectious anterior Uveitis (topical) Ready for pre-IND meeting
NOV06(sustained release)
Rx Retinal Diseases (Back of the Eye, IVI) Sustained release of IVI
„ESSENCE“ US trial, 328 patients
Successful pre-IND, Dec 2017
„SEECASE“ US trial, 336 patients
* MGD = Meibomian Gland Dysfunction
Successful proof-of-principle studies in dogs completed
Safety of in vitreous and protein stability/activity demonstrated
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Your Contacts
Bernhard Günther, Founder & President Novaliq [email protected]
Novaliq Inc., Harvard SquareOne Mifflin Place, Suite 400Cambridge, MA 02138, USAwww.novaliq.com