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Pharnext blames ‘unexpected’ placebo results for lead neurological drug’s phase 3 failure

2023-12-11

临床3期临床2期

Pharnext hopes incoming data from a phase 3 trial run by its Chinese partner Tasly will offer some better news.

Pharnextn a year after Pharnext dropped its Alzheimer’s program over cost conTasly, the French biotech has seen its lead program fail a phase 3 trial in a rare neurological disease.

The company is developiPharnext03 in Charcot-Marie-Tooth disease type 1A, an inherited genetic disorder that affects the peripheral nerves, resulting in muscle neurological diseaseally sensory loss.

The 387-patient trial did PXT3003w thCharcot-Marie-Tooth disease type 1Actioninherited genetic disordersured by the Overall Neuropathy Limitation Scale (muscle weaknessany revealed in asensory lossease. Pharnext blamed an “unexpected” improvement in the disease among the placebo cohort, which it said “complicates the interpretation of the results.”

“It also confirms what the medical literature as a whole has been saying for many years, namely that ONLS is undoubtedly a rNeuropathydpoint over the long term, but not over such a short period of time as a clinical study,” the company claimed in the release.

Trying to see the bright side, the biotech said there had been “no deterioration” of the condition among the patients who received the drug. “This suggests that PXT3003 might stabilize the condition of patients, which is an important consideration for a disease where progression is generally inevitable,” it concluded.

PXT3003 is a combination of the approved meds baclofen, naltrexone and sorbitol. The oral drug is designed to downregulate the overexpression of the PMP22 proteiPXT3003der to improve neuronal signaling in dysfunctional peripheral nerves.

PXT3003t hopes incoming data from a phase 3 trbaclofenbynaltrexonese pasorbitolsly will offer some better news. In the meantime, the company “plans tPMP22 proteinalyzing the data, particularly in collaboration with potential partners for licensing or acquisition of PXT3003.”

Pharnexth the results we are sharing today are not exactly what we had hoped Taslythey are nonetheless very promising,” Pharnext’s Manager Hugo Brugière said in the release. “We are now going to make the most of all the data we have accumulated over the last 10 yearPXT3003uding our two phase 3 studies and our 6-year extension study, which tend to demonstrate a beneficial effect on patients.”

“I remind that, today, PXT3003 is the only ray of hope for all CMT1A patients, and that no other drug candidate is currePharnextan advanced stage,” Brugière added. “It seems to me, therefore, that efficacy results for a risk-free drug could support an application for regulatory approval and market authorization. This is for us the final stage, which we will move forward with the patient community."

Investors didn’t appearPXT3003 much cause for optimism, sendingCMT1Acompany’s share price plunging 68% on Monday morning. However, with the company’s stock already sitting at below one euro cent, the difference was negligible in practicable terms.

Pharnext’s stock crashed back in February, when the biotech walked away from its potential Alzheimer’s asset, dubbed PXT864, arguing that the encouraging data produced to date weren't enough to lead to commercialization in the “short or medium term.” Instead, Brugière said at the time that the company would search for a partner to take the clinical baton.

PharnextPharnext even more reliant on PXT3003. The company said in October that it had received potential internationPXT864ensing offers worth over 250 million euros for the drug, although it remains to be seen whether these as-yet-undisclosed deals will be impacted by today’s results.

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