Item 8.01. Other Events.
OTX-TKI Clinical Trial Update
On May 14, 2020, Ocular Therapeutix, Inc. (the “Company”)
disclosed the receipt of interim data regarding its ongoing Phase 1 clinical trial of OTX-TKI, a hydrogel implant incorporating
axitinib, a small molecule tyrosine kinase inhibitor, delivered by intravitreal injection for the potential treatment of wet age-related
macular degeneration (AMD) and other retinal diseases. The Phase 1 clinical trial is a multi-center, open-label, dose-escalation
study in Australia designed to evaluate the safety, biological activity, durability and tolerability of OTX-TKI. At the time of
the prior disclosure, two cohorts of six subjects each had been enrolled, a lower dose cohort of 200 µg and a higher dose
cohort of 400 µg. The Company also amended the trial protocol to provide for a third cohort of subjects to receive a higher
dose of 600 µg.
The Company previously disclosed that, as of May 13, 2020, the
first two patients in the second (400 µg) cohort had shown a clinically meaningful reduction in intraretinal and/or subretinal
fluid out to six months with a single implant. The Company has now received further information from the clinical trial site in
Australia where these two patients are being treated. Specifically, the clinical trial site has reported to the Company that one
of these two patients showing a clinically meaningful reduction in intraretinal and/or subretinal fluid was treated with anti-VEGF
medication at a site visit at month 4.5 (in mid-March 2020) and at month 6 (in early May 2020). The patient who was treated with
anti-VEGF medication at the visits at month 4.5 and month 6 exhibited a clinically meaningful reduction in intraretinal fluid out
to 4.5 months with a single OTX-TKI implant before treatment with anti-VEGF medication. However, because of the use of anti-VEGF
medication beginning with the 4.5 month visit, the Company is not able to appropriately evaluate durability of treatment with OTX-TKI
in this patient beyond 4.5 months.
The clinical trial protocol and/or guidelines for completing
electronic case report forms for the trial provide that (1) anti-VEGF medication may be administered at the investigator’s
discretion based on consideration of predefined, specified criteria relating to a loss of visual acuity or increase in retinal
fluid, (2) the clinical trial site should notify a medical monitor within three days of treatment with anti-VEGF medication, (3)
administration of anti-VEGF medication should be entered into the clinical trial database in a timely manner and (4) a follow-up
visit should occur within seven to ten days after administration of anti-VEGF medication to determine the effect of treatment.
However, based on information made available to the Company, none of these elements of the clinical trial protocol or electronic
case report form guidelines were followed at the clinical trial site with respect to treatment of this particular patient with
anti-VEGF medication after insertion of the OTX-TKI implant. In particular, the patient was treated with anti-VEGF medication despite
not meeting the predefined criteria relating to loss of visual acuity or increase in retinal fluid and the administration of anti-VEGF
medication at the month 4.5 visit and at the month 6 visit was not entered into the clinical trial database until after a subsequent
visit at month 7.5 in mid-June 2020. As a result, this information was not available to or known by the Company in connection with
its prior disclosures. It is the Company’s understanding that at the time of and following the 4.5 month visit in mid-March
2020, the clinical and research operations of the trial site were being adversely impacted by the effects of the COVID-19 pandemic.
This Phase 1 trial is continuing, and the Company intends to
present more complete data for the first two cohorts when available at a future medical conference. As previously planned, the
third 600 µg cohort of the trial was initiated, with the first patient in this cohort being enrolled. The Company also plans
to file an application by the end of 2020 to initiate clinical development of OTX-TKI in the United States for patients with wet
age-related macular degeneration, diabetic macular edema (DME) and retinal vein occlusion (RVO). Based on trial data currently
available to the Company, OTX-TKI continues to be generally well tolerated and has been observed to have a favorable safety profile,
with no ocular serious adverse events to date.
Cautionary Note on Forward Looking Statements
Any statements in this Current Report on Form 8-K about future
expectations, plans, and prospects for the Company, including the commercialization of DEXTENZA®, ReSure®
Sealant, or any of the Company’s product candidates; the commercial launch of, and effectiveness of reimbursement
codes for, DEXTENZA; the development and regulatory status of the Company’s product candidates, such as the Company’s
development of and prospects for approvability of DEXTENZA for additional indications including allergic conjunctivitis, OTX-CSI
for the treatment of dry eye disease, OTX-TIC for the treatment of primary open-angle glaucoma and ocular hypertension, OTX-TKI
for the treatment of retinal diseases including wet AMD, and OTX-IVT as an extended-delivery formulation of the VEGF trap aflibercept
for the treatment of retinal diseases including wet AMD; the ongoing development of the Company’s extended-delivery hydrogel
depot technology; the potential utility of any of the Company’s product candidates; the potential benefits and future operation
of the collaboration with Regeneron Pharmaceuticals, including any potential future payments thereunder; the expected impact of
the COVID-19 pandemic on the Company and its operations; the sufficiency of the Company’s cash resources and other statements
containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend”,
“goal,” “may”, “might,” “plan,” “predict,” “project,”
“target,” “potential,” “will,” “would,” “could,” “should,”
“continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities
Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as
a result of various important factors. Such forward-looking statements involve substantial risks and uncertainties that could
cause the Company’s clinical development programs, future results, performance or achievements to differ significantly from
those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the timing and
costs involved in commercializing DEXTENZA, ReSure Sealant or any product candidate that receives regulatory approval, including
the conduct of post-approval studies, the ability to retain regulatory approval of DEXTENZA, ReSure Sealant or any product candidate
that receives regulatory approval, the ability to maintain reimbursement codes for DEXTENZA, the initiation, timing and conduct
of clinical trials, availability of data from clinical trials and expectations for regulatory submissions and approvals, the Company’s
scientific approach and general development progress, the availability or commercial potential of the Company’s product
candidates, the Company’s ability to generate its projected net product revenue on the timeline expected, if at all, the
sufficiency of cash resources, the Company’s existing indebtedness, the ability of the Company’s creditors to accelerate
the maturity of such indebtedness upon the occurrence of certain events of default, the outcome of the Company’s ongoing
legal proceedings, the severity and duration of the COVID-19 pandemic including its effect on the Company’s and relevant
regulatory authorities’ operations, the need for additional financing or other actions and other factors discussed in the
“Risk Factors” section contained in the Company’s quarterly and annual reports on file with the Securities and
Exchange Commission. In addition, the forward-looking statements included in this Current Report on Form 8-K represent the Company’s
views as of the date of this report. The Company anticipates that subsequent events and developments will cause the Company’s
views to change. However, while the Company may elect to update these forward-looking statements at some point in the future,
the Company specifically disclaims any obligation to do so except as required by law. These forward-looking statements should
not be relied upon as representing the Company’s views as of any date subsequent to the date of this Current Report on Form
8-K.