Biotechnology firm Ocugen (NASDAQ:OCGN) — which specializes in developing gene therapies to address blindness diseases — saw its shares move higher on an encouraging clinical development. An advisory committee responsible for overseeing safety matters approved Ocugen to use a medium dosage of its retinal therapeutic. Subsequently, OCGN stock moved higher on the positive implications.
According to the company’s press release, Ocugen received a green light from the Data and Safety Monitoring Board (DSMB) for the Phase 1/2 clinical trial of OCU410, an orphan nuclear receptor that can potentially limit damage to the retina due to dry age-related macular disease (AMD). Specifically, the DSMB stated that Ocugen can proceed with the medium dose of OCU410.
Per the release, three subjects suffering from geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date. An additional three subjects will be dosed with the medium dose. In addition, researchers will select three patients to undergo a high-dosage regimen.
According to Dr. Peter Chang, the DSMB Chair for the OCU410 clinical trial, the committee observed no serious adverse effects (SAEs) related to the target therapeutic to date. In turn, Ocugen’s team expressed satisfaction with the approval, emphasizing OCU410’s safety and tolerability profile.
OCGN Stock Rises on Positive Care Implications
According to Ocugen, currently approved therapeutics to treat GA suffer from significant limitations. Fundamentally, they require multiple injections per year and only target one pathway contributing to GA. In contrast, OCU410 regulates multiple pathways involved with the disease. These include lipid metabolism, inflammation, oxidative stress and membrane attack complex. Thus, the possibility of a superior solution boosted OCGN stock.
Moreover, Ocugen features a wide addressable market as far as rare diseases are concerned. GA is an advanced form of AMD, which can have a devastating impact on vision. More than five million people worldwide suffer from GA, including nearly one million in the U.S.
Across the world, over 200 million individuals are affected by AMD. Approximately 20 million people in the U.S. suffer from the condition. Given the critical need, it’s not surprising that the recorded safety of OCU410 lifted OCGN stock.
Why It Matters
Undeniably, OCGN stock represents an extremely risky investment. While it has more than doubled over the past 52 weeks, it’s down almost 73% in the trailing five years. However, since May of last year, three analysts have rated shares a buy. Interestingly, the lowest price target of $4 still implies about a 121% profit from the time of writing.
On the date of publication, Josh Enomoto did not have (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.
