Aldeyra Therapeutics (ALDX), a clinical-stage biotech company, is rallying today (+65%) on some positive clinical data. Since the name is not well known, we wanted to provide some color. Aldeyra Therapeutics focuses on developing products that treat diseases thought to be related to endogenous aldehydes, a naturally occurring class of pro-inflammatory and toxic molecules.
Aldeyra's lead product candidate, ADX-102, is an aldehyde trap in development as topical eye drops for the treatment of ocular inflammation. ADX-102 has now been tested in over 250 patients in Phase 2 clinical trials in dry eye disease, allergic conjunctivitis, and noninfectious anterior uveitis. A dermatologic form of ADX-102 is in late-stage clinical development for the treatment of ichthyosis due to Sjogren-Larsson Syndrome, an inborn error of aldehyde metabolism. ADX-102 has not yet been approved for sale in the US or elsewhere.
Turning to today's news, Aldeyra announced positive results from a Phase 2a clinical trial of topical ocular ADX-102 in patients with dry eye disease, a persistently challenging condition for many people worldwide. The trial investigated three formulations of ADX-102 (0.1% ophthalmic solution, 0.5% ophthalmic solution, and 0.5% lipid formulation) in 51 dry eye disease patients (17 per arm) treated for 28 days. The results from the pooled data demonstrated statistically significant improvement from baseline.
Improvements in dry eye disease signs and symptoms were evident within one week of therapy. A modest dose-response was observed, and activity increased over the duration of therapy, supporting evidence of the effect of drug. Levels of malondialdehyde, a pro-inflammatory aldehyde mediator sequestered by ADX-102, were significantly reduced in the tears of patients.
The primary objective of the trial was to select a formulation and dose range for a Phase 2b clinical trial. ADX-102 has now been nominated for advancement. There were no safety concerns observed for any of the formulations of ADX-102, and no serious adverse events were reported.
The company notes that these data represent the fourth set of positive Phase 2 results with ADX-102 in ocular inflammation. The breadth of activity across noninfectious anterior uveitis, allergic conjunctivitis, and now dry eye disease confirms the potential of ADX-102 as an important and differentiated therapy in ophthalmology. Aldyera is particularly excited about the potential of ADX-102 in the dry eye disease population, which is generally perceived to be inadequately treated but accounted for approximately $1.8 billion in prescription sales in the US in 2016.
Just a bit more about dry eye disease. It's a common inflammatory disease estimated to affect approximately 20 mln people in the US, and is characterized by insufficient moisture and lubrication in the anterior surface of the eye, leading to dryness, inflammation, pain, discomfort, irritation, and in severe cases, decreased vision.
In sum, investors are excited about today's news. The stock has been trending lower over the past year. Just about a year ago, the stock was above $8 and it closed at $4.20 yesterday. So this was some much needed good news. It's important because existing therapy for dry eye disease is generally regarded as inadequate.
In patients with dry eye disease, pro-inflammatory aldehyde mediators may contribute to ocular inflammation. By diminishing aldehyde levels, Aldeyra's topical ocular aldehyde trap platform represents a novel and differentiated approach for the treatment of dry eye disease. Looking ahead, the company expects to initiate a Phase 2b clinical trial in dry eye disease in 1H18.