Breaking the Vicious Circle of Dry Eye Disease - Bernhard Guenther, Founder & President Novaliq Inc.
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Breaking the Vicious Circle of Dry Eye Disease Bernhard Guenther, Founder & President Novaliq Inc. OIS@SECO | Feb 21th 2019, New Orleans © 2019 Novaliq 1
Novaliq is Transforming Ocular Therapeutics
Challenge water-based drug delivery
Only 1-10% of a water-based topical eye medication reaches
target 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 treatments
Pipeline addresses all key indications in Ophthalmology
NOV03 and CyclASol® have potential to redefine current
DED treatments
Both programs well positioned to generate two NDA
submissions by 2021
© 2019 Novaliq 2
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
Rabbits
2) Agarwal et al.; Semifluorinated
alkane based systems for enhanced
corneal penetration of poorly soluble
drugs; IntJPharm 2018; 538:119-129
© 2019 Novaliq 3
Pathogenesis-driven Dry Eye Disease Clinical Phenotypes
Ocular Surface Damage and Tear Film Instability impair Visual Function
evaporative
aqueous deficient
© 2019 Novaliq 4
Two Late-Stage Drug Candidates in Dry Eye – Breaking the Vicious Circle of DED
• Evaporative DED associated with MGD • Aqueous deficient DED
− Early onset of action
− Impressive effect on a variety of symptoms
− Sustained efficacy on ocular surface healing
− Early onset of action
− High responder rates
− Treats signs and symptoms of DED
− Impact on visual symptoms
• Unique Mode-of-Action
− Stabilizes lipid layer for hours • Critical Mode-of-Action in DED
− Penetrates meibomian glands − Immunomodulator & anti-inflammatory
• Outstanding tolerability & safety profile • Outstanding tolerability & safety profile
© 2019 Novaliq 5
Recent Randomized Double Masked Controlled US Trials
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
ESSENCE: 328 patients pivotal US trial
Predominantly aqueous deficient DED
• Low Schirmer: ~5 mm
• Significant corneal damage
• Central cornea involved
• Symptomatic, OSDI: ~45
© 2019 Novaliq 6
NOV03 – Clinically Significant Improvements in Signs and Symptoms of DED
Severely symptomatic population characterized by MGD and low TFBUT
SEECASE trial met its prespecified primary sign endpoints at 8 weeks
Highly clinically relevant improvements in various symptoms over active control
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
© 2019 Novaliq 7CyclASol® 0.1% – Clinical Significant Improvement in Sign and Symptoms
Onset of effect as early as 2 weeks and sustained over trial duration
Significant symptom improvements at primary endpoint
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
© 2019 Novaliq 8CyclASol® 0.1% – Majority of Patients Benefit from Treatment
p=0.004 p=0.03
p=0.03
High responder rates on cornea and conjunctiva
Highly significant and clinical meaningful benefit in majority of patients
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
© 2019 Novaliq 9Dry Eye Disease Impacts Visual Function
Improvement in Corneal Fluorescein Staining is associated with improvements in visual function
***
p=0.0009
*
p=0.049
p=0.296
tCFS response was defined as ≥ 3 scores (NEI scale) improvement from baseline
ESSENCE data show response in tCFS resulted in a clinically relevant
improvement in reading speed (>10 words per minute)
© 2019 Novaliq 10CyclASol® 0.1% – Fastest Onset of Action with Excellent Tolerability
CYS-002
ESSENCE (CYS-003)
0 1 2 3 4 5 6 7
Onset of action defined as primary sign improvement statistically superior to vehicle
(in months)
CYS-002
0.05% (N=51) 2%
0.1% (N=51) 2%
ESSENCE (CYS-003)
0.1% (N=162) 2.5%
0 5 10 15 20 25 30
Instillation site irritation (burning) in % of patients (meta-analysis)
© 2019 Novaliq 11Novaliq 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
© 2019 Novaliq 12Novaliq’s Clinical Pipeline
Dry Eye Disease Product Family
Product/Program Indication Disc. Pre-clinic Phase I Phase II Phase III Reg. Market Near-term milestones
CyclASol® Rx Dry Eye „ESSENCE“ US trial, 3Q2019: FPFV 2nd Phase 3
(cyclosporin) aqueous deficient 328 patients NDA submission 2021
NOV03 Rx Dry Eye „SEECASE“ US trial, 4Q2019: FPFV final Phase 3
(perfluorohexyloctane) evaporative, MGD* 336 patients NDA submission 2021
NOV07 Rx Dry Eye
Successful pre-IND, Dec 2017 Prepare for clinical phase
(cannabinoid) ocular pain
Glaucoma & Retina Development Programs
Product/Program Indication Disc. Pre-clinic Phase I Phase II Phase III Reg. Market Near-term milestones
Rx Glaucoma Successful proof-of-principle License-out and develop a
NOV04
(topical) studies in dogs completed glaucoma asset in the USA
NOV05 Non infectious anterior Uveitis
Ready for pre-IND meeting
(Tacrolimus) (topical)
NOV06 Rx Retinal Diseases Safety of in vitreous and protein
stability/activity demonstrated
Sustained release of IVI
(sustained release) (Back of the Eye, IVI)
* MGD = Meibomian Gland Dysfunction
© 2019 Novaliq 13Your Contacts
Bernhard Günther, Founder & President Novaliq Inc.
+1-617-580-1721
bguenther@novaliq.com
Novaliq Inc., Harvard Square
One Mifflin Place, Suite 400
Cambridge, MA 02138, USA
www.novaliq.com
© 2019 Novaliq 14You can also read
