Eloxx reports PhII cystic fibrosis fail as the biotech weighs its options
A rare disease biotech has fallen short in a clinical trial — and investors are none too happy about it.
Eloxx Pharmaceuticals reported Wednesday that its small molecule candidate ELX-02 combined with ivacaftor failed a small Phase II trial. The combination trial tested the two drugs in patients with Class 1 cystic fibrosis and at least one “nonsense mutation.” Ivacaftor is one-half of Vertex’s cystic fibrosis blockbuster drug Orkambi.
According to Eloxx, the combo failed to see statistical significance on certain efficacy endpoints, such as changes from baseline in sweat chloride concentration and perfect forced expiratory volume.
However, the biotech noted in an SEC filing:
Evidence of activity for ELX-02 was observed, as patients with higher baseline sweat chloride levels demonstrated increased responses as indicated by SCC (p=0.00013 at Day 35). Trial results were potentially confounded by high variability in sweat chloride and lung function measurement. The Company believes this variability could have been caused by very low drug exposures in the lung.
President and CEO Sumit Aggarwal tells Endpoints News that Eloxx will be working with the CF Foundation to determine the next steps in ELX-02’s development. The organization had financially backed Eloxx’s trial.
As for why the trial failed, the CEO said that efficacy is not the same as activity.
“We were measuring lung function improvement of functional response, not simply activity. So activity can exist but be drowned out by variability due to low drug exposure, patient characteristics, such that you don’t that doesn’t translate into efficacy in a statistically significant way when you have only 11 patients in a trial,” Aggarwal said.
On that note, Aggarwal added that the company has already started putting together a trial testing ELX-02 in eight patients with Alport syndrome, a genetic condition characterized by progressive loss of kidney function. The plan on that front is to have a readout in the first half of 2023.
“With our resources, they’re much better spent on Alport,” the chief executive noted.
The penny stock player $ELOX fell 39% in early morning trading Thursday, down to 23 cents a share. The biotech was subject to a delisting notice back in January, saying that it has until January 2, 2023, to get back into compliance with Nasdaq, essentially getting its stock price back over a dollar for the first time since November.
Aggarwal tells Endpoints that in light of the low stock price, the biotech has enough cash to get itself through Q4 of next year — and essentially all options, including a reverse split, are on the table.