OCU400 Update Shows Improvement In Three Vision Measures

2Q23 Reported With New Clinical Trials To Start Before YE23

Moving Past Covaxin, Focusing On Gene Therapy and Pipeline Products

Positive Preliminary Data Shows Proof-Of-Concept For OCU400

Robert LeBoyer

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Ocugen, Inc.

Summary.&nbsp;Ocugen provided an update to its Phase 1/2 clinical trial data from OCU400 in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). These are rare eye diseases in which genetic mutations cause loss of vision and progress to blindness. In addition to demonstrating continued safety, the data reported three measures of vision that showed OCU400 could improve vision or stabilize disease progression in 8 out of 12 patients. We see these data as clinically meaningful for OCU400 in these indications and as proof-of-concept for Ocugenâ€™s method of targeting a â€œmaster control geneâ€ that has downstream effects on other genes.
We See Proof-of-Concept For The &#8220;Master Control Gene&#8221; Approach.&nbsp;RP is a group of genetic disorders with over 100 possible genetic mutations that may be present and contributing to loss of vision. This makes the symptoms of disease highly variable and difficult to target with a single therapy. Ocugen has been developing OCU400 to target the RHO and NR2E3 genes that control pathways and regulate other genes that act later in those pathways. The data&nbsp;show that OCU400 can affect several gene pathways in eye, stabilizing the disease and/or improving three measures of vision.
In April 2023, Ocugen presented preliminary data in the 7 patients from the first two cohorts with assessments at 6 or 9 months after treatment. The data update now includes 12 patients from three cohorts treated for at least 6, 9, or 12 months.&nbsp;There were no tolerability or safety concerns reported at the longer timeframe or the highest dose.&nbsp;
Figure 1.&nbsp; Cohorts and Dosing In The OCU400 Data Reports.&nbsp; In April 2023, Ocugen reported data from the first 7 patients. The date update now includes 12 out of the 18 patients enrolled.&nbsp; An additional 3 adult patients with the LCA CEP290 gene mutation are enrolling, and 3 pediatric patients with RHO, NR2E3, or CEP290 are enrolling.
<img decoding="async" src="https://www.noblelinx.com/x/research/images/reports/ocu400entry.jpg" /> 
 
Source: Ocugen, Inc.&#8212;&#8212;&#8212;- page break &#8212;&#8212;&#8212;-
The data show that 10 out of 12 patients (82%) had stabilization or improvement in all three measures of efficacy. As shown in Figure 2, 1 patient had improvement in 2 measures and 3 had improvement in 1 measure. 
 
Figure 2.&nbsp; Venn Diagram Showing Improvement In More Than One Endpoint.&nbsp;&nbsp;Out of the 12 patients in the trial, 8 showed improvement in all three areas. We believe this shows the impact OCU on multiple pathways and supports Ocugen&#8217;s &#8220;Master Control Gene&#8221; approach and gene agnostic approach.
<img decoding="async" src="https://www.noblelinx.com/x/research/images/reports/venn400.jpg" style="width: 467px;height: 243px" /> 
 
Source:&nbsp; Ocugen, Inc.
 
Data Is Consistent With Improvements Seen Earlier.&nbsp;The previous data presented in April 2023 was from the first 7 patients to reach 6 or 9 months after treatment. The update included 12 patients with treatment from 6 months, 9 months, or 12 months. The outcome measures presented were BCVA (best corrected visual acuity), LLVA (low luminescence visual acuity) and MLMT (the multi luminance mobility test, a measure of the ability to detect levels of light. These are clinically meaningful measures that showed consistent effects.
Figure 3.&nbsp; Data Showed Improvements In All Three Measures.&nbsp;The patients showed improvements in these three measures indicating stable disease or improvements in vision.&nbsp;
<img decoding="async" src="https://www.noblelinx.com/x/research/images/reports/threemeasures3.jpg" style="width: 544px;height: 230px" /> 
 
Source: Ocugen, Inc.
BCVA (best corrected visual acuity). This measures the ability of a patient to read a standard eye chart, measured by the gain or loss of letters that the patient can read correctly. Out of the 12 subjects in the trial, 10 (83%) showed stabilization or improvements from baseline in their treated eyes. Improvements of 4 letters were seen in 5 out of 12 patients (42%) with 4 out of 12 (33%) showing 7 letter improvements.&nbsp; In the RHO mutation subjects, 4 out of 7 (57%) had a 4 letter improvement and 3 out of 7 (43%) had a 7 letter improvement from baseline.
LLVA (low luminescence visual acuity). This is an early clinical marker of change in central retinal function. Patients may maintain visual acuity until late in the disease process, making LLVA an indicator early changes and a measure of the patient&#8217;s level of everyday visual function. Out of 12 patients, LLVA scores in 10 patients (83%) showed stabilization or improvement from baseline. Improvements of 5 letters (or 1 line) were seen in 5 out of 12 (42%) patients and 3 out of 7 (25%) had increases of 10 letters (2 lines). Out of the 7 RHO mutation patients, 3 (43%) showed a 5 letter improvement in LLVA, with 2 out of 7 (29%) showing increases of 10 letters (2 lines).
MLMT (multi luminance mobility test) Was Improved or Stable. The MLMT measures the patient&#8217;s ability to see objects in levels of light measured in Lux levels (a quantity of light per square meter). Out of the 12 patients, 9 (75%) had stable or improvement from baseline in their MLMT scores. An improvement of 1 lux luminance level was seen in 4 out of the 12 patients (33%) with 17% improving by 3 lux levels.&nbsp; Stabilization or increases in the MLMT scores from baseline was seen in 6 out of 7 patients (86%), with 3 out of 7 (29%) improving by 3 lux levels.
 Ocugen Plans To Move Forward To Phase 3.&nbsp;The updated data are consistent and strengthen the conclusion the OCU400 is able to control gene pathways in RP and LCA. Although the trial was not powered to show statistical significance, we believe the trial size for these Orphan indications will be sufficient for a Phase 3 trial. The company plans to discuss the data with the FDA to design a Phase 3 trial in the coming months, prepare the protocols, then begin the trial in late 2024.
NeoCart Continues On Schedule. Construction on the cGMP facility to manufacture the cellular therapy NeoCart is expected to be completed by YE2023. We expect pilot manufacturing lots and plant certification in 1H24, allowing for the start of the Phase 3 clinical trial in 2H24. The lead product, NeoCart, is for repair of knee cartilage.&nbsp; 
 
Inhaled Vaccine Development Moves Toward INDs.&nbsp;Although the COVID-19 pandemic has been declared over, the emergence of new viral variants continues to cause concern. Ocugen is proceeding with the development of its inhaled mucosal vaccines that would stimulate immunity in the nasal mucosa where viruses enter the body. Inducing nasal immunity is intended to stop the infection before it spreads as well as the transmission of virus to the surrounding environment. 
The OCU500/510/520 products in the platform include a bivalent COVID-19, a seasonal influenza vaccine, and a quadravalent vaccine combining flu and COVID-19 in a single vaccine. Preclinical work for OCU500, OCU510, and OCU520 continues to prepare for INDs in 4Q23/1Q24. Ocugen has submitted proposals for government grants and plans to wait for responses to its applications before starting clinical trials.
Conclusion.&nbsp;Ocugen&#8217;s gene therapy program has met another development milestone on time and with strong data. In 2Q23, Ocugen filed IND applications for OCU410ST in Stargardt disease and OCU410 in Geographic Atrophy (GA) in dry age-related macular degeneration (dry AMD), with Phase 1/2 trials expected before year-end. We are reiterating our Outperform rating and price target of $5 per share.&#8212;&#8212;&#8212;- page break &#8212;&#8212;&#8212;-
Figure 4. Ocugen&#8217;s Product Development Pipeline.
<img decoding="async" src="https://www.noblelinx.com/x/research/images/reports/pipeline923.jpg" style="width: 530px;height: 306px" /> 
 
Source:&nbsp; Ocugen, Inc.

Ocugen, Inc. (OCGN)

OCU400 Update Shows Improvement In Three Vision Measures

Fundamental Analysis:

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OCU400 Update Shows Improvement In Three Vision Measures (OCGN)

2Q23 Reported With New Clinical Trials To Start Before YE23 (OCGN)

Moving Past Covaxin, Focusing On Gene Therapy and Pipeline Products (OCGN)

Positive Preliminary Data Shows Proof-Of-Concept For OCU400 (OCGN)

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