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Breaking the Vicious Circle of Dry Eye Disease Christian Roesky PhD, Chief Executive Officer and Managing Director OIS@AAO | Oct 25 th 2018, Chicago
© 2018 Novaliq, confidential 1
Breaking the Vicious Circle of Dry Eye DiseaseChristian Roesky PhD, Chief Executive Officer and Managing Director
OIS@AAO | Oct 25th 2018, Chicago
© 2018 Novaliq, confidential 2
Novaliq is Transforming Ocular Therapeutics
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
CyclASol® and NOV03 have potential to redefine current DED treatmentsBoth programs well positioned to generate two NDA submissions by 2021
© 2018 Novaliq, confidential 3
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. Semifluorinatedalkane based systems for enhanced corneal penetration of poorly soluble drugs
© 2018 Novaliq, confidential 4
• Critical Mode-of-Action in DED - Immunomodulator & anti-inflammatory
• Aqueous deficient DED- Early onset of action - Sustained efficacy on ocular surface healing- High responder rates- Impact on visual symptoms
• Outstanding tolerability & safety profile
• New Mode-of-Action- Stabilizes lipid layer for hours- Penetrates meibomian glands
• Evaporative DED associated with MGD- Impressive effect on a variety of symptoms- Early onset of action - Treats signs and symptoms of DED
• Outstanding tolerability & safety profile
Two Late-Stage Drug Candidates in Dry Eye - Breaking the Vicious Circle of DED
© 2018 Novaliq, confidential 5
Vicious Circle of DED - Targeting Key Drivers Impacting Visual Function
aqueous deficient
evaporative
© 2018 Novaliq, confidential 6
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
© 2018 Novaliq, confidential 7
NOV03 - Clinically Significant Improvements in Signs and Symptoms of DED
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 one sided testing
SEECASE trial met its prespecified primary sign endpoints at 8 weeksHighly clinically relevant improvements in various symptoms over active control
Baseline values for VAS Severity of Dryness:NOV03 QID 68.6; NOV03 BID 70.0; Saline QID/BID 66.8
Severely symptomatic population characterized by MGD and low TFBUT
*
** ***
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p=0.009
***
**
p=0.0003
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***
**
**
**
*
Baseline values for tCFS:NOV03 QID 7.0; NOV03 BID 6.7; Saline QID/BID 6.7
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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
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****
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p=0.0002
p=0.002
p=0.01
p=0.04
p=0.03
p=0.03
p=0.02
Baseline values for tCFS: CyclASol® 0.1% 11.5; Vehicle 11.5Baseline values for VAS Severity of Dryness: CyclASol® 0.1% 68.5; Vehicle 69.9
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
© 2018 Novaliq, confidential 9
CyclASol® 0.1% - Majority of Patients Benefit from Treatment
High responder rates on cornea and conjunctiva
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
Highly significant and clinical meaningful benefit in majority of patients
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Instillation site irritation (burning) in % of patients (meta-analysis)0 5 10 15 20 25 30
CyclASol® 0.1% - Fastest Onset of Action with Excellent Tolerability
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’s Clinical Pipeline
Product/Program Indication Disc.
Pre-clinic
Phase I
Phase II Phase III Reg. Market Near-term milestones
CyclASol® (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 Eye
ocular pain1Q2019: prepare for clinical phase
Dry Eye Disease Product Family
Glaucoma & Retina Development ProgramsProduct/Program Indication Disc.
Pre-clinic
PhaseI
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)
n.i. anterior Uveitis
(topical)4Q2018: ready for pre-IND meeting
NOV06(sustained release)
Rx Retinal Diseases
(Back of the Eye, IVI)1Q2019 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
© 2018 Novaliq, confidential 12
ESSENCE (CyclASol® 0.1%) and SEECASE (NOV03) provide novelties in DED therapy in two distinguished patient groups
1) Outcomes for both studies are clinically relevant
2) Both products have the potential to treat more patients for better treatment outcomes:
• CyclASol® is intended for treating predominantly aqueous deficient DED
• NOV03 for treating evaporative DED associated with Meibomian Gland Dysfunction
3) Both products show an unprecedented risk - benefit profile
Novaliq intends to accelerate both development programs for NDA submission in 2021
Novaliq DED Rx Portfolio Has Ability to Redefine DED Therapy
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Your Contacts
Novaliq GmbH
Im Neuenheimer Feld 515
69120 Heidelberg
Germany
www.novaliq.com
Dr. Christian Roesky, CEO
+49 6221-50259-220
