TY - JOUR
T1 - A phase II randomized trial to evaluate the long-term (12-week) efficacy and safety of OC-01 (varenicline solution) nasal spray for dry eye disease
T2 - The MYSTIC study
AU - Quiroz-Mercado, Hugo
AU - Hernandez-Quintela, Everardo
AU - Chiu, Kuei Hsun
AU - Henry, Eugenia
AU - Nau, Jeffrey A.
N1 - Funding Information:
H.Q.-M. and E.H.-Q. have no conflicts of interest to declare. K.H.C. and E.H. are employees of Firma Clinical Research, which was funded by Oyster Point Pharma, Inc. for this work. J.A.N. is an employee of and shareholder in Oyster Point Pharma, Inc.
Funding Information:
This study was sponsored by Oyster Point Pharma, Inc. , manufacturer/licensee of OC-01 (varenicline solution) nasal spray.
Funding Information:
The authors would like to thank all study participants. Medical writing assistance was provided by Fiona Nitsche, PhD, CMPP, of Envision Pharma Group, and was funded by Oyster Point Pharma, Inc. Envision Pharma Group's services complied with international guidelines for Good Publication Practice (GPP3).
Publisher Copyright:
© 2021
PY - 2022/4
Y1 - 2022/4
N2 - Purpose: Dry eye disease is characterized by loss of tear film stability. OC-01 (varenicline solution) is a small-molecule nicotinic acetylcholine receptor agonist administered as a nasal spray that stimulates tear production. Methods: In MYSTIC (NCT03873246) patients aged ≥22 years with dry eye disease were randomized 1:1:1 to OC-01 0.03 mg, OC-01 0.06 mg, or vehicle (n = 41 per group), administered twice daily via intranasal spray, for 12 weeks (84 days). Primary efficacy endpoint was mean change from baseline in anesthetized Schirmer's test score (STS) in study eye at day (D) 84. Results: Patients receiving OC-01 0.03 and 0.06 mg had statistically significantly increased tear production at D84 versus vehicle; least squares mean changes from baseline in STS were 10.8 mm and 11.0 mm for OC-01 0.03 and 0.06 mg, respectively. A trend toward a higher proportion of patients experiencing ≥10-mm improvement in STS from baseline was observed with OC-01 0.03 mg (36.6%; p > 0.05), and was significant for OC-01 0.06 mg (48.8%; p = 0.024), versus vehicle (24.4%). Non-ocular treatment-emergent adverse events (TEAEs) were reported by 21 patients; the most common was sneezing (OC-01 0.03 mg, 2 [4.9%]; OC-01 0.06 mg, 3 [7.3%]), with similar frequencies between treatment groups. No severe or serious TEAEs were reported. Conclusions: OC-01 (varenicline solution) nasal spray improved tear production in patients with dry eye disease over a long-term (12-week) period, and represents a receptor neuro-activator with a nasal route of administration that spares the ocular surface to stimulate tear production.
AB - Purpose: Dry eye disease is characterized by loss of tear film stability. OC-01 (varenicline solution) is a small-molecule nicotinic acetylcholine receptor agonist administered as a nasal spray that stimulates tear production. Methods: In MYSTIC (NCT03873246) patients aged ≥22 years with dry eye disease were randomized 1:1:1 to OC-01 0.03 mg, OC-01 0.06 mg, or vehicle (n = 41 per group), administered twice daily via intranasal spray, for 12 weeks (84 days). Primary efficacy endpoint was mean change from baseline in anesthetized Schirmer's test score (STS) in study eye at day (D) 84. Results: Patients receiving OC-01 0.03 and 0.06 mg had statistically significantly increased tear production at D84 versus vehicle; least squares mean changes from baseline in STS were 10.8 mm and 11.0 mm for OC-01 0.03 and 0.06 mg, respectively. A trend toward a higher proportion of patients experiencing ≥10-mm improvement in STS from baseline was observed with OC-01 0.03 mg (36.6%; p > 0.05), and was significant for OC-01 0.06 mg (48.8%; p = 0.024), versus vehicle (24.4%). Non-ocular treatment-emergent adverse events (TEAEs) were reported by 21 patients; the most common was sneezing (OC-01 0.03 mg, 2 [4.9%]; OC-01 0.06 mg, 3 [7.3%]), with similar frequencies between treatment groups. No severe or serious TEAEs were reported. Conclusions: OC-01 (varenicline solution) nasal spray improved tear production in patients with dry eye disease over a long-term (12-week) period, and represents a receptor neuro-activator with a nasal route of administration that spares the ocular surface to stimulate tear production.
KW - Dry eye disease
KW - Nasal spray
KW - Nicotinic acetylcholine receptor
KW - Pharmacologic neuro-activator
KW - Phase 2 clinical trial
KW - Varenicline
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UR - http://www.scopus.com/inward/citedby.url?scp=85121580582&partnerID=8YFLogxK
U2 - 10.1016/j.jtos.2021.12.007
DO - 10.1016/j.jtos.2021.12.007
M3 - Article
C2 - 34920097
AN - SCOPUS:85121580582
SN - 1542-0124
VL - 24
SP - 15
EP - 21
JO - Ocular Surface
JF - Ocular Surface
ER -