Ocugen Provides Business Update with Third Quarter 2025 Financial Results

Rhea-AI Summary

Ocugen (NASDAQ: OCGN) provided a business update and reported third quarter 2025 results. The company closed a $20M registered direct offering in August and would receive an additional $30M if warrants are fully exercised, and reported $32.9M cash on hand as of September 30, 2025.

Clinical progress includes 50% enrollment in the OCU410ST Phase 2/3 GARDian3 trial with a path toward a 1H 2027 BLA filing and EMA acceptability of a single U.S. trial for MAA submission. OCU400 Phase 3 enrollment is nearing completion with planned BLA/MAA filings in 2026. Ocugen executed an exclusive license for OCU400 in South Korea with upfront/milestone payments and sales milestones projected to reach $180M in the first 10 years and a 25% royalty.

Positive

• Exclusive South Korea license with Kwangdong including up to $7.5M upfront/development milestones
• Sales milestones projected to reach $180M+ in first 10 years with 25% royalty
• Closed $20M registered direct offering and potential $30M additional proceeds if warrants exercised
• 50% enrollment completed in OCU410ST Phase 2/3, supporting a 1H 2027 BLA timeline
• OCU400 Phase 3 enrollment nearing completion with planned 2026 BLA/MAA submissions

Negative

• Cash decreased to $32.9M as of September 30, 2025 from $58.8M at year-end 2024
• Operating expenses rose to $19.4M in Q3 2025 from $14.4M in Q3 2024
• Current cash runway expected only through Q2 2026 absent additional financings

Insights

Senior biotech analyst

Progress on pivotal trials and a licensing deal are meaningful, but cash runway and near-term financing needs limit the immediate positive impact.

Ocugen shows clear operational momentum: the Phase 3 OCU400 program is nearing enrollment with an intended BLA/MAA submission in 2026, and the Phase 2/3 OCU410ST trial is 50% enrolled with regulatory acceptability from the EMA for a single U.S. trial. The company also executed an exclusive South Korea licensing agreement for OCU400 that includes upfront and milestone payments, sales milestones and a 25% royalty, which creates a defined regional commercialization path and potential near‑term non‑dilutive value.

Financially the update is mixed. The company closed a registered direct offering raising ~$20 million and reported 32.9 million in cash as of September 30, 2025, which the company states supports operations into 2Q 2026. The warrants could provide an additional 30 million if fully exercised, but that proceeds contingent on market action. Operating expenses increased year‑over‑year, reflecting higher R&D and G&A spend, which tightens the near‑term funding picture.

Watch for three concrete near‑term milestones: Phase 2 full data for OCU410 in 1Q 2026, interim 50% patient data for OCU410ST mid‑2026, and Phase 3 top‑line data for OCU400 in 4Q 2026. These readouts, plus any exercise of warrants or additional financing, will materially change the company’s funding outlook and the value of the licensing economics for South Korea within a 6–18 month horizon.

Conference Call and Webcast Today at 8:30 a.m. ET

• Phase 2/3 OCU410ST GARDian3 pivotal confirmatory trial is progressing toward 1H 2027 Biologics License Application (BLA) filing with 50% enrollment completed to date
• • European Medicines Agency (EMA) provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application (MAA)
    
• Executed licensing agreement with Kwangdong Pharmaceutical for exclusive rights in South Korea to OCU400
  • Sales milestones of $1.5 million for every $15 million of sales in South Korea, projected to reach $180 million or more in first 10 years of commercialization and royalties equaling 25% of net sales
    
• Closed $20 million registered direct offering of common stock and accompanying premium warrants
  • The Company will receive $30 million of additional gross proceeds if the warrants are exercised in full
    

MALVERN, Pa., Nov. 05, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported third quarter 2025 financial results along with a general business update.

“With two late-stage modifier gene therapies on track to meet 2026 and 2027 BLA/MAA filings, it’s remarkable to look back and recognize we only began dosing the first patient in the Phase 1/2 OCU400 clinical trial in 2022,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “The OCU410ST Phase 2/3 GARDian3 pivotal confirmatory trial is following close behind the OCU400 Phase 3 liMeLiGhT clinical trial, and with 50% enrollment completed to date, we believe recruitment will be completed in the first quarter of 2026. This progress not only reinforces our commitment to file three BLAs in the next three years, but it also brings us closer to addressing the incredible unmet medical needs that exist for patients facing vision loss.”

In September, Ocugen announced its exclusive licensing agreement with Kwangdong Pharmaceutical Co., Ltd. (Kwangdong) for the rights to OCU400 in South Korea. Under the agreement, the Company will receive up to $7.5 million in upfront and development milestone payments, plus sales milestones of $1.5 million for every $15 million of sales in South Korea, projected to reach $180 million or more in the first 10 years of commercialization. The Company will also earn a 25% royalty on net sales generated by Kwangdong and will be responsible for manufacturing and supplying OCU400. A regional approach preserves Ocugen’s rights to larger geographies to maximize total patient reach while also generating a potential return for shareholders.

Enrollment in the OCU400 Phase 3 liMeliGhT clinical trial is nearing completion, and the program remains on track for BLA and MAA submissions in 2026. This is the only known broad retinitis pigmentosa (RP) gene-agnostic trial to address multiple genetic mutations and multiple disease pathways with a single therapeutic approach. There are approximately 300,000 people in the U.S. and Europe combined living with RP, which affects greater than 100 genes. Ocugen’s gene-agnostic approach has the potential to treat multiple gene mutations associated with RP with a one-time subretinal injection.

The Phase 2/3 GARDian3 pivotal confirmatory trial for OCU410ST for Stargardt disease is well underway and in August the Company announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA provided acceptability of a single U.S.-based trial for submission of an MAA. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined, and approximately 1 million globally. Currently, there is no FDA-approved treatment available for Stargardt disease.

Also in August, Ocugen closed a registered direct offering pursuant to a securities purchase agreement with Janus Henderson Investors for the purchase and sale of 20,000,000 shares of common stock and warrants to purchase up to an aggregate of 20,000,000 shares of common stock at a purchase price of $1.00 per share and accompanying warrant at a premium exercise price of $1.50 per share. The gross proceeds to the Company were approximately $20 million, which Ocugen anticipates will extend the Company’s cash runway into the second quarter of 2026. The Company will receive $30 million of additional gross proceeds if the warrants are exercised in full extending runway into 2027.

“We will continue to pursue financing opportunities along with strategic business development to fund the Company into commercialization,” said Dr. Musunuri. “We have engaged with potential funding and business partners during various investor and global conferences. I look forward to additional substantive conversations between now and the end of the year.”

Upcoming inflection points for Ocugen’s novel modifier gene therapy platform include OCU410 (Geographic Atrophy) Phase 2 full data release expected in the first quarter of 2026, OCU410ST (Stargardt disease) interim data on 50% of patients at eight months of treatment expected mid-year 2026, and OCU400 (RP) Phase 3 top line data expected in the fourth quarter of 2026. The Company looks forward to providing the market and key stakeholders with near-term catalysts supporting Ocugen’s strong path forward.

Modifier Gene Therapy Platform—a Novel First-in-Class Platform

• OCU400 – Enrollment in the Phase 3 liMeliGhT clinical trial is nearing completion. The Company secured an exclusive licensing agreement with Kwangdong for rights to OCU400 in South Korea and will continue to pursue regional partnerships. Intend to initiate BLA rolling submission in the first half of 2026 and release Phase 3 top-line data in the fourth quarter of 2026.
  
• OCU410ST – Pivotal confirmatory Phase 2/3 trial is ahead of schedule. CHMP of the EMA provided acceptability of a single U.S.-based trial for submission of an MAA. Intend to release interim data (50% of patients at 8 months of treatment) mid-year 2026.
• OCU410 – Intend to release full data from the Phase 2 clinical trial in the first quarter of 2026 and begin Phase 3 in 2026.
  

Ophthalmic Biologic Product

• OCU200 – Intend to complete enrollment in the Phase 1 clinical trial in 4Q 2025.
  

Third Quarter 2025 Financial Results

• With the recent $20 million financing in the third quarter, we expect our current cash position provides sufficient runway to operate through 2Q 2026.
• The Company’s cash, cash equivalents and restricted cash totaled $32.9 million as of September 30, 2025, compared to $58.8 million as of December 31, 2024.
• Total operating expenses for the three months ended September 30, 2025 were $19.4 million and included research and development expenses of $11.2 million and general and administrative expenses of $8.2 million. This compares to total operating expenses for the three months ended September 30, 2024 of $14.4 million that included research and development expenses of $8.1 million and general and administrative expenses of $6.3 million.
  

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 or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 3029428
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.

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, the ability to initiate new clinical programs, Ocugen’s financial condition and expected cash runway into the second quarter of 2026, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, and Ocugen’s projections under its license agreement with Kwangdong Pharmaceutical Co., Ltd., 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 of may not be predictive of the results or success of later clinical trials; and that 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.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

OCUGEN, INC. CONSOLIDATED BALANCE SHEETS (in thousands) (Unaudited)
	September 30, 2025			December 31, 2024
Assets
Current assets
Cash	$	32,565		$	58,514
Prepaid expenses and other current assets		5,074			3,168
Total current assets		37,639			61,682
Property and equipment, net		14,946			16,554
Restricted cash		314			307
Other assets		4,697			3,899
Total assets	$	57,596		$	82,442
Liabilities and stockholders' equity
Current liabilities
Accounts payable	$	4,574		$	4,243
Accrued expenses and other current liabilities		14,932			15,500
Operating lease obligations		855			519
Current portion of long term debt		-			1,326
Total current liabilities		20,361			21,588
Non-current liabilities
Operating lease obligations, less current portion		3,709			3,313
Long term debt, net		28,400			27,345
Other non-current liabilities		1,593			564
Total non-current liabilities		33,702			31,222
Total liabilities		54,063			52,810
Total stockholders' equity		3,533			29,632
Total liabilities and stockholders' equity	$	57,596		$	82,442

OCUGEN, INC. CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) (Unaudited)
	Three months ended September 30,								Nine months ended September 30,
		2025				2024				2025				2024
Collaborative arrangement revenue	$	1,752			$	1,136			$	4,606			$	3,291
Total revenue		1,752				1,136				4,606				3,291
Operating expenses
Research and development		11,149				8,108				29,081				23,836
General and administrative		8,228				6,280				21,446				20,372
Total operating expenses		19,377				14,388				50,527				44,208
Loss from operations		(17,625	)			(13,252	)			(45,921	)			(40,917	)
Other income (expense), net		(2,426	)			282				(4,219	)			743
Net loss	$	(20,051	)		$	(12,970	)		$	(50,140	)		$	(40,174	)

FAQ

How much cash did Ocugen (OCGN) report on September 30, 2025?

Ocugen reported $32.9M in cash, cash equivalents and restricted cash as of September 30, 2025.

What financing did Ocugen (OCGN) complete in 3Q 2025 and what additional proceeds are possible?

Ocugen closed a $20M registered direct offering and would receive an additional $30M if warrants are exercised in full.

What are the key clinical milestones and timelines for Ocugen (OCGN) gene therapies?

OCU400 Phase 3 top-line data expected Q4 2026 with BLA/MAA submissions in 2026; OCU410ST interim 50% data expected mid-2026; OCU410 Phase 2 full data expected Q1 2026.

What did the EMA say about Ocugen's OCU410ST Stargardt trial for OCGN?

The CHMP provided acceptability of using a single U.S.-based trial for an MAA submission for OCU410ST.

What are the commercial terms of Ocugen's (OCGN) South Korea license for OCU400?

The license includes up to $7.5M in upfront/development milestones, sales milestones of $1.5M per $15M sales, projected $180M+ first-10-year sales, and a 25% royalty.

How long is Ocugen's (OCGN) current cash runway expected to last?

With the recent financing, Ocugen expects its cash runway to extend into Q2 2026, with potential extension into 2027 if warrants are exercised.