1. No category

Penetrating mucosal barriers to maximize
ocular therapeutic outcome
Charles McDermott
Interim President and CBO
Ophthalmology Innovation Summit – April 2015
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 1
Kala Pharmaceuticals: Phase 3 Stage Company
MPP Platform Technology: Successful Clinical Translation
• Advancing mucus-penetrating particle (MPP) technology based
ophthalmic product candidates into late stage clinical development
• Translation from research stage technology to positive phase 2 and 3
clinical trials in less than 2 years.
Kala Eyecare Focus: Front, Middle, and Back of the Eye
• Topical formulations target multiple ophthalmic indications affecting the
front, middle, and back of the eye
• KPI-121 hit all primary endpoints in first phase 3 trial in cataract surgery
and hit the primary sign endpoint in initial phase 2 dry eye trial.
• Novel RTKis formulated in MPP technology for retinal disease progressing
toward IND stage development
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 2
Kala’s Eye Care Pipeline
Discovery
Post-surgical
Inflammation
Dry Eye
Meibomian Gland
Disease
AMD/DME/
RVO/Orphan
Glaucoma
Preclinical
Phase I
Phase II
Phase III
NDA
KPI-121 Ophthalmic MPP Suspension
1st Phase 3 trial complete
KPI-121 Ophthalmic MPP Suspension
Phase 2/3 trial complete
KPI-121 Ophthalmic MPP Suspension
Phase 2 trial ongoing
RTKi* MPP (topical))
Exploratory
KPI-121 = Kala’s Loteprednol Etabonate Mucus Penetrating Nano-Particle Formulation
RTKi = Kala’s Novel Receptor Tyrosine
in MPP Formulations
C O N F I DKinase
E N T I Inhibitors
AL
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 3
KPI-121: Phase 3 Post-Surgical Results
KPI-121: statistically significant improvements for both primary endpoints
Primary Endpoints: Inflammation and Pain
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 4
KPI-121: Phase 2 Dry Eye Sign Results
KPI-121: statistically significant improvements in the primary sign endpoint –
bulbar conjunctival hyperemia
Primary Sign Endpoint: Conjunctival Hyperemia
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 5
KPI-121: Phase 2 Dry Eye Symptoms Results
KPI-121: improvements in both severity and frequency of symptoms
Primary Endpoint: Discomfort Severity
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 6
Secondary Endpoint: Discomfort Frequency
Kala Topical RTKis to Treat BOE Diseases
• Novel compounds with potent VEGFR2 inhibition and good selectivity
• Demonstrated topical delivery to back of the eye in large animals
• Demonstrated efficacy in VEGF driven rabbit leakage model
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 7
Kala Driving Late Stage KPI-121 Clinical Development
Clinical Results: Successful Translation of MPP into Patients
• KPI-121 positive clinical results validates MPP technology applicability in
ophthalmology
Kala’s Next Steps:
• Advance development of KPI-121 in dry eye and post operative
inflammation.
• Readout of KPI-121 phase 2 trial in MGD.
• Advance RTKi program to clinical development candidate for VEGF driven
indications.
• Continue discussions with prospective partners regarding KPI-121 and
MPP technology applications in new areas.
Additional Applications: Women’s Health, GI, and Respiratory
• In-vivo data across many classes of compounds show promise in
therapeutic areas beyond ophthalmology.
KALA PHARMACEUTICALS | Ophthalmology Innovation Summit| April 2015 | Page 8