Pharnext ditches Alzheimer's plans, saying more development would be too costly

Pharnext is walking away from its work developing a treatment for Alzheimer’s, saying additional development would be more money than the company is willing to spend. 

The French biotech was candid in its reasoning, claiming that while the asset, dubbed PXT864, had produced some encouraging data in its last trial seven years ago, it wasn't enough to lead to commercialization in the “short or medium term.” Prying out more data would require additional clinical development that the company isn’t willing to pay for at the moment. Instead, CEO Hugo Brugière said Pharnext is now on the search for a partner to take the clinical baton. 

With Alzheimer’s disease no longer on the docket for PXT864, the company is even more focused on seeing PXT3003 through the clinical finish. The phase 3 med is being developed to treat Charcot-Marie-Tooth disease type 1A (CMT1A) and topline data is slated for a fourth-quarter readout. CMT1A is an inherited genetic disorder that affects the peripheral nerves, resulting in muscle weakness and potentially sensory loss. The trial recently completed enrollment with almost 400 patients signed up across 52 sites in the U.S., Canada, Europe and Israel. 

The decision to cull work on Alzheimer’s disease comes more than seven years after the company wrapped up its phase 2 trial testing PXT864, recruiting 45 patients and testing three different dosing regiments. During the last 12 weeks of the study, investigators were authorized to tack on Eisai’s dementia treatment Aricept to the treatment plan. Pharnext has been coy about the data from that trial, but claims on its website that the therapy was well-tolerated and “may slow the progression of cognitive disability” in patients with mild disease.

For the time being, the biotech is relying on a handful of loans to keep it going across the phase 3 finish line, with a cash runway expected to last until the fourth quarter of 2024. 

As for PXT864, it may yet have a place in Pharnext’s pipeline but its future is unclear. The company says that it's trying to decide whether to also out-license it for another indication, amyotrophic lateral sclerosis, or develop it internally once it has completed the phase 3 trial for PTX3003.