Tiny Aldeyra Therapeutics says it has seen “statistically and clinically significant improvements” in a midstage trial for its experimental eye med ADX-102, although not all data points were positive.
The $67 million market cap biotech said the phase 2a test, which was primarily focused on finding the right dosage for the med (out of 0.1% ophthalmic solution, 0.5% ophthalmic solution, and 0.5% lipid formulation), looked at 51 dry eye disease patients (17 per arm) treated over four weeks.
Aldeyra says that results from the pooled data showed “significant improvement” from baseline in Symptom Assessment in Dry Eye (SANDE) Score (p=0.003), Ocular Discomfort Score (p=0.00002), Overall Four-Symptom Score (p=0.0004), Schirmer (tear volume) Test (p=0.008), tear osmolarity (p=0.003), and Lissamine Green ocular surface staining score (p=0.002).
The biotech also said that levels of malondialdehyde, a pro-inflammatory aldehyde mediator sequestered by ADX-102, were “significantly reduced in the tears of patients (p=0.009), supporting the differentiated mechanism of action relative to other therapies in dry eye disease.”
But there are issues. The company says that, after sifting through the data, it had picked the 0.1% dose of ADX-102 to work on in the future. But when looking into the data from this part of the small test, the outcome was a mixed bag.
On the SANDE Score, statistical significance was missed (p=0.09), as so it was for tear osmolarity (p=0.06); and it was close to the edge on Overall Four-Symptom Score (p=0.048) and the Schirmer Test (p=0.04).
It did better on Ocular Discomfort Score (p=0.002), the dryness component of the Four-Symptom Score (p=0.01), and tear aldehyde levels (p=0.007). The biotech said it is still planning to start a phase 2b in the first half of next year, based on these data.
There was also no comparator arm. But investors were happy on this, and its shares were up 35% in early trading, or $1.50.
Todd Brady, M.D., Ph.D., president and CEO of Aldeyra, said: “We are 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 United States in 2016.”
This comes three months after it posted a flop for the med in allergic conjunctivitis. The trial set out to assess whether Aldeyra’s experimental drug could elicit a one-point improvement over placebo on a scale of eye itching. For the study to hit its primary endpoint, the data had to link the treatment to such an improvement and a statistically-significant gain over the placebo.
The trial only met one of these requirements. While the trial showed higher, 0.5% dose of ADX-102 was statistically superior to the control, it failed to record a one-point or more improvement on the zero-to-four patient-reported rating scale. That resulted in the study failing to meet its primary endpoint.
It also decided to focus on the positives of the failed test, but its shares fell 19% on early trading on June 14 when it posted the data.
Aldeyra had $19 million in cash and $12 million in marketable securities at the end of March, since when it has moved to add up to $20 million to its bank balance through a stock sale agreement with Cantor Fitzgerald.
Aldeyra recently started a phase 3 trial of the drug in patients with non-infectious anterior-uveitis, and previously trialed a cream formulation of the aldehyde trap to treat skin disorders associated with Sjögren-Larsson syndrome.