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0001372299
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2026-08-06
2026-08-06
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
August 6, 2026
OCUGEN, INC.
(Exact name of registrant as specified in its charter)
| Delaware |
|
001-36751 |
|
04-3522315 |
(State or other jurisdiction
of incorporation) |
|
(Commission File
Number) |
|
(IRS Employer
Identification No.) |
|
11 Great Valley Parkway
Malvern, Pennsylvania |
|
19355 |
| (Address of principal executive offices) |
|
(Zip Code) |
Registrant’s telephone number, including
area code: (484) 328-4701
N/A
(Former name or former address, if changed since
last report.)
Check the appropriate box below if the Form 8-K filing is intended
to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction
A.2. below):
| ¨ |
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| |
|
| ¨ |
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| |
|
| ¨ |
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| |
|
| ¨ |
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class |
|
Trading
Symbol(s) |
|
Name of each exchange
on which registered |
| Common Stock, par value $0.01 per share |
|
OCGN |
|
The Nasdaq Stock Market LLC
(The Nasdaq Capital Market) |
Indicate by check mark whether the registrant is an emerging growth
company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange
Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ¨
If
an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying
with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and Financial Condition.
On August 6, 2026, Ocugen, Inc (the
“Company”) issued a press release announcing certain financial results for the quarter ended June 30, 2026. The Company
has scheduled a conference call and webcast for 8:30 a.m. Eastern Time on August 6, 2026, to discuss these financial results
and business updates. The Company will use presentation materials in connection with the conference call and webcast, which presentation
materials will be posted on the Company's website at www.ocugen.com. Copies of the press release and presentation materials are furnished
herewith as Exhibit 99.1 and Exhibit 99.2, respectively, to this Current Report on Form 8-K (this “Report”)
and incorporated herein by reference.
The information disclosed under Item 2.02 of this
Report, including Exhibit 99.1 and Exhibit 99.2, is being furnished and shall not be deemed “filed” for purposes
of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities
of that section, and shall not be deemed to be incorporated by reference in any Company filing under the Securities Act of 1933, as amended
(the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit Number |
|
Description |
| |
|
|
| 99.1 |
|
Earnings Press Release of Ocugen, Inc., dated August 6, 2026. |
| 99.2 |
|
Earnings Release Presentation of Ocugen, Inc., issued August 6, 2026. |
| 104 |
|
Cover Page Interactive Data File (embedded within the Inline XBRL document). |
SIGNATURE
Pursuant to the requirements
of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto
duly authorized.
| |
|
OCUGEN, INC. |
| |
|
|
| Date: August 6, 2026 |
By: |
/s/ Shankar Musunuri |
| |
|
Name: Shankar Musunuri |
| |
|
Title: Chairman, Chief Executive Officer, & Co-Founder |
Exhibit 99.1
Ocugen Provides Business Update with Second
Quarter 2026 Financial Results
Conference Call and Webcast Today at 8:30 a.m. ET
| · | Received
U.S. Food and Drug Administration (FDA) clearance to initiate OCU410 Phase 3 trial for geographic
atrophy (GA), secondary to dry age-related macular degeneration (AMD); on track to initiate
Phase 3 this quarter |
| · | Granted
Regenerative Medicine Advanced Therapy (RMAT) designation by FDA for OCU410, enabling eligibility
for priority review and accelerated approval |
| · | Signed
a binding term sheet with Roots Pharmaceutical, and its strategic partner Al-Dhow International
Holding, to negotiate an exclusive license for OCU400 in retinitis pigmentosa (RP) across
the Middle East and North Africa (MENA) region |
| · | Successfully
completed OCU400 Process Performance Qualification (PPQ) batches, supporting Biologics License
Application (BLA) and commercial launch supplies |
| · | Closed
$130.0 million convertible senior notes financing, extending cash runway into 2028 |
| · | Remain
on track to announce top-line results for two late-stage clinical programs, OCU400 for RP
and OCU410ST for Stargardt disease in 1Q 2027 and 2Q 2027, respectively |
| · | Strengthened
leadership team with the appointments of Mohamed Genead, M.D., M.Sc., as Chief Medical Officer
and Chris Clark as Head of Corporate Communications |
MALVERN, Pa., August 6, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc.
(“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness
diseases, today reported second quarter 2026 financial results along with a general business update.
“The second quarter of 2026 marked a pivotal inflection point
for Ocugen. We closed $130 million convertible senior notes financing, extending our cash runway into 2028, and signed a binding term
sheet for exclusive license of OCU400 in retinitis pigmentosa across the Middle East and North Africa region,” said Dr. Shankar
Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “Ocugen is a leading gene therapy company, focused on vision
loss diseases with significant unmet medical needs, with multiple catalysts through 2028 across three distinct retinal disease indications.
As we advance toward key data milestones in the first half of 2027, we remain focused on creating long-term value for our patients and
shareholders.”
Unlike traditional gene therapies that correct a single mutation,
Ocugen's modifier gene therapy platform targets master regulatory genes that control multiple gene networks of biological pathways, supporting
a gene-agnostic approach applicable across a broad range of genetic mutations, as well as diseases with complex pathways such as dry
AMD. This allows the Company to address large, underserved patient populations rather than narrow single-gene subsets. This platform
underpins each of Ocugen's three late-stage programs, which advanced meaningfully during the quarter.
Clinical Program Updates
OCU410 (GA)
| · | Received
FDA clearance for the Phase 3 registrational trial (ArMaDa3) for GA secondary to dry age-related
macular degeneration, anchored by positive 12-month Phase 2 ArMaDa data (statistically significant
31% reduction in GA lesion growth versus control [patient population: lesion size ≥2.5
mm2 and ≤17.5 mm2], p<0.05, at the optimal dose planned for Phase
3) |
| · | Planned
combined global Phase 3 trial of approximately 237 subjects powered at 95% for primary end
point on track to initiate this quarter, with BLA and MAA filings targeted for 2028 |
| · | Granted
Regenerative Medicine Advanced Therapy RMAT designation by FDA, enabling eligibility for
priority review and accelerated approval |
| · | GA
affects approximately 2 to 3 million people in the U.S. and Europe |
OCU410ST (Stargardt disease)
| · | Completed
enrollment and dosing of 63 subjects ahead of schedule, in less than nine months, in the
pivotal Phase 2/3 GARDian3 trial evaluating OCU410ST in patients with all mutations of Stargardt
disease |
| · | Topline
results anticipated in the second quarter of 2027, with a BLA submission to follow mid-2027 |
| · | Holds
Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Medicinal Product
Designation and Advanced Therapy Medicinal Product classification from the EMA |
| · | Stargardt
disease affects approximately 100,000 patients across the U.S. and Europe, with no approved
therapies globally |
OCU400 (RP)
| · | Completed
enrollment in liMeliGhT (N=140), the first and largest genetic medicine registrational trial
for broad RP patients, spanning more than 30 genetic mutations |
| · | FDA
feedback confirmed that the path to rolling BLA submission remains tied to topline data expected
in the first quarter of 2027, and the company is advancing preparation accordingly, including
successfully completing Process Performance Qualification (PPQ) batches |
| · | Approximately
300,000 people in the U.S. and Europe are living with RP |
Corporate Updates
| · | Closed
the offering of $130.0 million aggregate principal amount of 6.75% Convertible Senior Notes
due 2034, including the full exercise of the $15.0 million over-allotment option, for net
proceeds of approximately $112.5 million |
| o | Approximately $32.7 million of net proceeds was used to fully retire
the Avenue Capital loan, eliminating 12.25% interest-rate debt from the Company’s capital
structure |
| o | The offering extends Ocugen’s cash runway into 2028 |
| · | Signed
a binding term sheet with Roots Pharmaceutical to negotiate an exclusive OCU400 license in
the MENA region, with up to $255 million in sales milestones and a 22% royalty on net sales,
as well as moderate upfront payment to Ocugen |
| · | Ocugen
appointed two new members to its leadership team |
| o | Mohamed Genead, M.D., M.Sc., was named Chief Medical Officer on June 11,
2026. Dr. Genead is an ophthalmologist and retina specialist with more than 20 years
of experience in ophthalmology and gene therapy, having previously served as Co-Founder and
Chief Executive Officer of Aviceda Therapeutics and in senior leadership roles at GenSight
Biologics, Biogen, and Allergan |
| o | Chris Clark was named Vice President, Corporate Communications on
July 16, 2026. He has more than 20 years of communications and investor relations experience
in the pharmaceutical and biopharmaceutical industries, having held leadership positions
at Bausch + Lomb, Idorsia Pharmaceuticals, and Pfizer, as well as communications and
investor relations roles at Novo Nordisk, Bristol Myers Squibb, Endo Pharmaceuticals, and
Johnson & Johnson |
Second Quarter 2026 Financial Results
| · | The
Company’s cash, cash equivalents, and restricted cash totaled $100.4 million as of
June 30, 2026, compared to $32.2 million as of March 31, 2026. |
| · | The
Company had 339.0 million shares of common stock outstanding as of June 30, 2026. |
| · | Total
operating expenses for the three months ended June 30, 2026, were $17.9 million and
included research and development expenses of $10.7 million and general and administrative
expenses of $7.2 million, compared to total operating expenses for the three months ended
June 30, 2025, of $15.2 million that included research and development expenses of $8.4
million and general and administrative expenses of $6.8 million. |
| · | Ocugen
reported a $0.07 net loss per common share for the three months ended June 30, 2026,
compared to a $0.05 net loss per common share for the three months ended June 30, 2025. |
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET
today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which
will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.
Attendees are invited to participate on the call using the following
details:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646)
307-1963 for international callers
Conference ID: 2222566
Webcast: Available on the events section of the Ocugen investor site
A replay of the call and archived webcast will be available on the
Ocugen investor site.
About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology company developing
gene therapies for blindness diseases. The Company’s breakthrough modifier gene therapy platform has the potential to address significant
unmet medical needs across large patient populations through a gene-agnostic approach. Unlike traditional gene therapies and gene-editing
technologies that target a single gene mutation, Ocugen’s modifier gene therapies are designed to address the underlying disease
biology by restoring balance across multiple gene networks. The Company is currently advancing programs for inherited retinal diseases
and other causes of blindness that affect millions worldwide, including retinitis pigmentosa, Stargardt disease, and geographic atrophy,
an advanced form of dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on LinkedIn and
X.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives
for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates,
including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements
of current ongoing preclinical and clinical trials, including the timing of enrollment and data readouts, the ability to initiate new
clinical programs, Ocugen’s financial condition and expected cash runway into 2028, statements regarding qualitative assessments
of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development
timelines, statements regarding potential market size and commercial possibilities of Ocugen’s product candidates, which are subject
to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,”
“proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,”
“intends,” “may,” “could,” “might,” “will,” “should,” or other
words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject
to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current
expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative
of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through
further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing may not be predictive of the
results or success of later clinical trials; and that clinical trial data are subject to differing interpretations and assessments, including
by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the
Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in
the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only
as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained
in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contacts:
Investors:
Candice Masse
astr partners
candice.masse@astrpartners.com
Media:
Chris Clark
chris.clark@ocugen.com
(Tables to follow)
OCUGEN, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
(Unaudited)
| |
|
June 30, 2026 |
|
|
Dec 31, 2025 |
|
| Assets |
|
|
|
|
|
|
|
|
| Current assets |
|
|
|
|
|
|
|
|
| Cash |
|
$ |
100,051 |
|
|
$ |
18,571 |
|
| Prepaid expenses and other current assets |
|
|
6,670 |
|
|
|
5,769 |
|
| Total current assets |
|
|
106,721 |
|
|
|
24,340 |
|
| Property and equipment, net |
|
|
13,347 |
|
|
|
14,392 |
|
| Restricted cash |
|
|
320 |
|
|
|
316 |
|
| Other assets |
|
|
3,818 |
|
|
|
4,468 |
|
| Total assets |
|
$ |
124,206 |
|
|
$ |
43,516 |
|
| Liabilities and stockholders' equity |
|
|
|
|
|
|
|
|
| Current liabilities |
|
|
|
|
|
|
|
|
| Accounts payable |
|
$ |
5,140 |
|
|
$ |
6,202 |
|
| Accrued expenses and other current liabilities |
|
|
11,014 |
|
|
|
14,733 |
|
| Operating lease obligations |
|
|
839 |
|
|
|
858 |
|
| Convertible notes |
|
|
82,359 |
|
|
|
- |
|
| Derivative liability |
|
|
33,708 |
|
|
|
- |
|
| Current portion of long-term debt |
|
|
20 |
|
|
|
1,250 |
|
| Total current liabilities |
|
|
133,080 |
|
|
|
23,043 |
|
| Non-current liabilities |
|
|
|
|
|
|
|
|
| Operating lease obligations, less current portion |
|
|
3,039 |
|
|
|
3,494 |
|
| Long term debt, net |
|
|
1,729 |
|
|
|
27,542 |
|
| Other non-current liabilities |
|
|
2,914 |
|
|
|
1,603 |
|
| Total non-current liabilities |
|
|
7,682 |
|
|
|
32,639 |
|
| Total liabilities |
|
$ |
140,762 |
|
|
$ |
55,682 |
|
| |
|
|
|
|
|
|
|
|
| Stockholders' equity |
|
|
|
|
|
|
|
|
| Common stock |
|
|
3,390 |
|
|
|
3,125 |
|
| Treasury stock |
|
|
(48 |
) |
|
|
(48 |
) |
| Additional paid-in capital |
|
|
432,010 |
|
|
|
392,763 |
|
| Accumulated other comprehensive income |
|
|
213 |
|
|
|
61 |
|
| Accumulated deficit |
|
|
(452,121 |
) |
|
|
(408,067 |
) |
| Total stockholders' equity |
|
|
(16,556 |
) |
|
|
(12,166 |
) |
| Total liabilities and stockholders' equity |
|
$ |
124,206 |
|
|
$ |
43,516 |
|
OCUGEN, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
AND COMPREHENSIVE LOSS
(in thousands, except share and per share amounts)
(Unaudited)
| | |
Three months ended June 30, | | |
Six months ended June 30, | |
| | |
2026 | | |
2025 | | |
2026 | | |
2025 | |
| Collaborative arrangement revenue | |
$ | 1,488 | | |
$ | 1,373 | | |
$ | 3,022 | | |
$ | 2,854 | |
| Total revenue | |
| 1,488 | | |
| 1,373 | | |
| 3,022 | | |
| 2,854 | |
| Operating expenses | |
| | | |
| | | |
| | | |
| | |
| Research and development | |
| 10,690 | | |
| 8,402 | | |
| 21,945 | | |
| 17,932 | |
| General and administrative | |
| 7,242 | | |
| 6,766 | | |
| 15,359 | | |
| 13,218 | |
| Total operating expenses | |
$ | 17,932 | | |
$ | 15,168 | | |
$ | 37,304 | | |
$ | 31,150 | |
| Loss from operations | |
| (16,444 | ) | |
| (13,795 | ) | |
| (34,282 | ) | |
| (28,296 | ) |
| Interest income | |
| 395 | | |
| 227 | | |
| 526 | | |
| 571 | |
| Interest expense | |
| (4,476 | ) | |
| (1,285 | ) | |
| (5,796 | ) | |
| (2,543 | ) |
| Loss on extinguishment of debt | |
| (2,383 | ) | |
| - | | |
| (2,383 | ) | |
| - | |
| Change in fair value of derivative liability | |
| (1,891 | ) | |
| - | | |
| (1,891 | ) | |
| - | |
| Other (expense) income, net | |
| (78 | ) | |
| 114 | | |
| (228 | ) | |
| 179 | |
| Total other (expense) income | |
$ | (8,433 | ) | |
$ | (944 | ) | |
$ | (9,772 | ) | |
$ | (1,793 | ) |
| Net loss | |
$ | (24,877 | ) | |
$ | (14,739 | ) | |
$ | (44,054 | ) | |
$ | (30,089 | ) |
| Other comprehensive income (loss) | |
| | | |
| | | |
| | | |
| | |
| Foreign currency translation adjustment | |
| 49 | | |
| (28 | ) | |
| 152 | | |
| (36 | ) |
| Comprehensive loss | |
$ | (24,828 | ) | |
$ | (14,767 | ) | |
$ | (43,902 | ) | |
$ | (30,125 | ) |
| | |
| | | |
| | | |
| | | |
| | |
| Net loss attributable to common shareholders — basic and diluted | |
| (24,877 | ) | |
| (14,739 | ) | |
| (44,054 | ) | |
| (30,089 | ) |
| Weighted shares used in calculating net loss per common share — basic and diluted | |
| 338,679,856 | | |
| 292,067,192 | | |
| 333,142,618 | | |
| 292,032,072 | |
| Net loss per share attributable to common shareholders — basic and diluted | |
$ | (0.07 | ) | |
$ | (0.05 | ) | |
$ | (0.13 | ) | |
$ | (0.10 | ) |
Exhibit 99.2
| 
| Courageous Innovation
Dedicated to Bringing Game-Changing Gene
Therapies to Market and Working Even Harder to
Provide Access to Patients Globally |
| 
| 2
Pipeline
A quarter of execution across three programs
Ocugen – Second Quarter Business Update
OCU410ST
Stargardt Disease
100K U.S.+EU patients ·
No approved therapy
ODD · RPDD · OMPD · ATMP
Current Stage
Phase 2/3 GARDian3 —
Enrollment complete
Ph1 Ph2 Ph3 BLA Approved
Next Catalysts
Interim outcome analysis:
3Q 2026
Topline data: 2Q 2027
BLA target: Mid-2027
BLA 2027
OCU400
Retinitis Pigmentosa
300K U.S.+EU patients ·
298K untreated
RMAT · ODD · ATMP · OMPD
Current Stage
Phase 3 — Enrollment Complete
Ph1 Ph2 Ph3 BLA Approved
Next Catalysts
Topline data: 1Q 2027
BLA target: 2Q 2027
BLA 2027
OCU410
Geographic Atrophy
2–3M U.S.+EU patients ·
No gene therapy approved
RMAT · ATMP (EMA)
Current Stage
Phase 2 ARMADA Complete
Ph1 Ph2 Ph3 BLA Approved
Next Catalysts
Phase 3 initiation: 3Q 2026
BLA target: 2028
BLA 2028 |
| 
| 3
Current GA therapy targets the lesion; none target the vision —
OCU410 was designed to do both
31%
Lesion growth reduction vs. control (p<0.05)
OCU410 Phase 2 ARMADA (12 months)
Pivotal P3 population:
(lesion size ≥2.5 mm2 and ≤17.5 mm2
)
27%
EZ preservation
(correlates to visual function)
Pivotal P3 population:
(lesion size ≥2.5 mm2 and ≤17.5 mm2
)
~15–22%
Lesion reduction shown
by Izervay (12 months) + Syfovre (24 months)
no functional benefit
Lesion reduction — OCU410 vs. approved therapies (12/24 months)
OCU410 31%
Syfovre (Apellis/Biogen) 22%
Izervay (Astellas) 15%
* Approved options require monthly, or every other month, intravitreal injections with no
meaningful functional improvement. OCU410 = single subretinal injection.
Why OCU410 can win in GA
Functional wins, not just structural
Patients with GA want to see better. OCU410 data shows EZ preservation —
that's what drives adoption and reimbursement.
One injection vs. monthly burden
Syfovre and Izervay require monthly, or every other month, intravitreal
injections indefinitely. A single subretinal injection is a fundamentally
different value proposition.
First-mover in gene therapy for GA
No gene therapy is approved for GA. Phase 3 start in Q3 2026 gives OCU410
a clean path to first-in-class before any competitor arrives.
OCU410 is outperforming the standard-of-care with a single injection vs. monthly chronic dosing
References: Apellis OAKS/DERBY (Heier et al, Lancet, Product Insert), Khanani A, Patel S, Staurenghi G et al. Efficacy and safety of
avacincaptad pegol in patients with geographic atrophy (GATHER2): 12-month results from a randomised,
double-masked, phase 3 trial The Lancet, 2023; 402, 1449-1458
OCU410 · Geographic Atrophy
Ocugen – Second Quarter 2026 Business Update |
| 
| Proposed Endpoints
Primary • Rate of change (slope) of square root-transformed GA lesion area
(√mm²/year) from baseline to Month 12, treated eyes vs control eyes as
assessed by FAF
Secondary • Proportion of subjects with LLVA ≥15 Letter Loss from Baseline to M12
as assessed by ETDRS visual charts at two consecutive visits.
• Rate of change of EZ area loss through Month 12, by SD-OCT
Phase 3 Trial Design
Study Design
Population • Patients ≥55 years of age or older with clinical diagnosis of GA
secondary to dry AMD
Key Eligibility
Criteria
• BCVA ≥25 letters but ≤80 letters (ETDRS chart)
• Total GA area ≥2.5 and ≤17.5 mm2 ; foveal or non-foveal GA
with uni-or multifocal lesion
• If GA is multifocal, at least one lesion must be ≥1.25mm2
• Presence of CNV in fellow eye is non exclusionary
OCU410 Global Phase 3 (ArMaDa 3) Trial Design for GA
OCU410 · Geographic Atrophy
Ocugen – Second Quarter 2026 Business Update
Received FDA alignment on the Phase 3 study design; in discussions with EMA |
| 
| 5
First-in-Class Gene Therapy for Stargardt Disease
OCU410ST · Stargardt Disease
100K
U.S. + EU patients
No approved therapy
globally
Q3 2026
GARDian3 enrollment
interim outcome
Q2 2027
topline data expected
EMA accepted single
U.S. trial for MAA
Stargardt is the most common inherited macular dystrophy — caused by mutations in ABCA4
(1,200+ mutations), causing progressive central vision loss typically beginning in childhood.
What sets OCU410ST apart
Modifier gene approach covers all 1,200+ ABCA4 mutations with a single
therapy. Traditional approaches can't address this genetic complexity.
Phase 1 GARDian1 published
Results published in EYE journal supports favorable safety and efficacy
profile as GARDian3 pivotal confirmatory trial advances.
Pivotal confirmatory Phase 2/3 trial. 51 subjects, 2:1 randomized.
Primary endpoint: change in atrophic lesion size. One-year data
used for BLA filing.
ODD + RPDD (FDA) · OMPD + ATMP (EMA). Rare Pediatric Disease
Designation adds priority review voucher optionality at approval.
Trial: GARDian3
Designations
Ocugen – Second Quarter 2026 Business Update |
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| 6
The largest orphan gene therapy trial ever run, targeting
approval across all RP mutations
OCU400 · Retinitis Pigmentosa
140
patients enrolled in
Phase 3 liMeliGhT trial
(largest orphan gene therapy Ph3)
100+
RP-causing genes
addressed by a single
subretinal injection
Q1 2027
topline data expected
BLA submission to follow
POPULATION
Early- to late-stage RP, including pediatrics aged 3+
PRIMARY ENDPOINT
12-month change in visual function via LDNA Lux Level
RANDOMIZATION
2:1 — treatment vs. untreated control
BLA PATHWAY
BLA targeted for 2Q 2027 · Single U.S. trial accepted by EMA for MAA
Trial Design:
Enrolling patients across all mutations — early and late stage, pediatric and adult — means a positive read delivers the broadest possible label. No competitor
has attempted this breadth in a single trial.
G L O B A L V A L I D A T I O N S I G N A L
Kwangdong OCU400 deal — $180M+ projected 10-yr Korean sales + 25% royalties
Roots Pharmaceutical binding term sheet signed – up to $255 million + 22% royalties
Ocugen – Second Quarter 2026 Business Update |
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| 7
Three indications. 3+ million patients. Multibillion market.
Market Opportunity
OCU410 — Geographic Atrophy
2–3M
patients · U.S. + EU
2.5M+
untreated today
$300-600k
pricing assumption (market potential)1
Current treatment landscape unable to
support visual gain
OCU410ST — Stargardt Disease
100K
patients · U.S. + EU
100K
untreated today
$1-2M
No current market approved therapies for
patients
Ocugen – Second Quarter 2026 Business Update
per patient per patient
pricing assumption (market potential)
OCU400 — Retinitis Pigmentosa
300K
patients · U.S. + EU
298K
untreated today
$1-2M
OCU400 is gene-agnostic, treats every
mutation
pricing assumption (market potential)2
per patient
1 ClearView Healthcare Partners analysis of 2037 worldwide sales
2 RETINA vol. 46,4 (2026) |
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| OCU400 US Commercial Roadmap
Payer engagement and CMS
Center of Excellence
Patient Registry
Distribution
Sales & Marketing
• Increased engagement
• Setup 25+ COEs
• Enhance disease awareness
• Frictionless supply
• 1Q2026 TLR inflection point
Commercial |
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| 9
Milestones
Anticipated Milestones – Three BLAs by 2028
Retinitis
Pigmentosa
Stargardt
Disease
Geographic
Atrophy
OCU400
OCU410ST
OCU410
Interim outcome
analysis1
Phase 3
topline data
Initiate Phase 3
2026 2027
1Q 2Q 3Q 4Q
Phase 3
topline data
BLA
Submission
Approval/
Launch
Approval/
Launch
BLA
Submission
1H 2H
2028
Phase 3
enrollment completion
1Re-estimation to minimize clinical risk (Outcome -Impact / no-impact to BLA timeline)
1Q 2Q 3Q 4Q
BLA
Submission
Ocugen – Second Quarter 2026 Business Update |
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| Financial Update
Statement of Operations
Three months ended June 30,
2026 2025
Research and development expense $10.7 $8.4
General and administrative expense $7.2 $6.8
Interest (expense) income, net $(4.1) $(1.1)
Other (expense) income, net $(4.4) $0.1
Net loss $(24.9) $(14.7)
Balance Sheet Data June 30, 2026 Dec 31, 2025 (Audited)
Cash, cash equivalents, and restricted cash $100.4 $18.9
Debt $1.7 $28.8
Convertible Note $116.1 _
Shares outstanding 339,044,893 312,379,972
10
Except as otherwise noted, all amounts are unaudited; in millions, except share amounts
Certain amounts may not add due to rounding
Ocugen – Second Quarter 2026 Business Update |
| 
| Financial Update
Statement of Operations
Six months ended June 30,
2026 2025
Research and development expense $21.9 $17.9
General and administrative expense $15.4 $13.2
Interest (expense) income, net $(5.3) $(2.0)
Other (expense) income, net $(4.5) $0.2
Net loss $(44.1) $(30.1)
Balance Sheet Data June 30, 2026 Dec 31, 2025 (Audited)
Cash, cash equivalents, and restricted cash $100.4 $18.9
Debt $1.7 $28.8
Convertible Note $116.1 _
Shares outstanding 339,044,893 312,379,972
11
Except as otherwise noted, all amounts are unaudited; in millions, except share amounts
Certain amounts may not add due to rounding
Ocugen – Second Quarter 2026 Business Update |