Sunday, January 11

Combine Baclofen & Acamprosate and You Get A Promising Alzheimer's Treatment


Researchers know more about old drugs than new ones. That's why they put a lot of effort into "repurposing" existing drugs to cure Alzheimer's. One such drug is called PXT-864 from Pharnext. By combining two re-purposed drugs, they came up with a promising Alzheimer's therapy. Learn more about this drug and its Phase II clinical trial.

Pharnext SAS announced the publication of preclinical results in Nature's Scientific Reports for its lead pleodrug, PXT-864, in Alzheimer's disease. These data further validate its pleotherapy research and development approach based on a proprietary net work pharmacology platform that identifies synergic combinations of drugs already approved for other diseases.

In the publication titled, "Combining two repurposed drugs as a promising approach for Alzheimer's disease therapy" by Ilya Chumakov, et al., data show that i n two different rodent models, PXT-864, a combination of baclofen and acamprosate, demonstrated not simply a dditive but synergistic effect. Multiple induced ma rkers of Alzheimer's disease models were improved and / or normalized. Indeed, PXT-864 had synergistic and cons istent effects on the following:
  1. Significant alleviation of induced cognitive defici ts in the acquisition of long and short-term memory , novel objects recognition or learning and spatial w orking memory
  2. Protection of neurons from oxidative stress and death
  3. Preservation of the blood brain barrier that is als o damaged in patients with Alzheimer's disease
  4. Normalization in levels of pro-inflammatory factors associated with neuroinflammation
  5. Normalization of neuronal endogenous toxic factors levels and preservation of synaptic loss
Consistently with in vivo data, researchers also noted for PXT-864 a synergistic neuronal and vascular cellular protection in two in vitro models.

The paper can be found here:

Catherine Scart-Grès, M.D., chief medical officer of Pharnext , said, "The preclinical results obtained with PXT-864 open the possibility for a promising therapeutic approach for Alzheimer's disease. Patients suffering from Alzheimer's disease are marked by rapid and progressive deterioration of cognitive function leading to a loss of independence. Current treatment options for these patients are only moderately symptomatic and there is a great unmet need. The preliminary data with PXT-864 are promising results for our future work. A phase 2a is currently ongoing with first results expected in 2015."

Ilya Chumakov, Ph.D., D.Sc., co-founder of Pharnext and scientific advisory board chairman , said, "Treatment of Alzheimer's disease was a natural direction for our pleotherapy approach because there are so many signaling pathways associated with the disease. We designed PXT-864 using our network pharmacology technology to interfere with some of those key pathways. We look forward to continuing the development of PXT-864 further in upcoming clinical trials."

Daniel Cohen, M.D., Ph.D., chairman, chief executive officer and co-founder of Pharnext, said, "The positive data for PXT-864 published today validate our network pharmacology approach and pleotherapy platform of synergic repositioned drug combinations. We recently published positive clinical and preclinical data for our other lead pleodrug PXT-3003 in Charcot-Marie-Tooth disease Type 1A (CMT 1A). Altogether, these results comfort us in potential clinical efficacy for PXT-864 in Alzheimer's disease. We believe there is room for development of pleodrugs in many disease conditions.


Pharnext is an advanced clinical stage biopharmaceutical company developing new therapeutics that simultaneously target multiple key disease pathways for severe orphan and common neurological diseases. The proprietary research and development platform of Pharnext is based on network pharmacology. It allows the development of synergic combinations of repositioned drugs: pleodrugs. The company's two lead pleodrugs are PXT-3003 for the treatment of Charcot Marie Tooth type 1A (Phase 2 clinical trial completed) and PXT-8 64 for Alzheimer's disease (Phase 2 clinical trial ongoing ) and other neurologic indications (Parkinson's dis ease, amyotrophic lateral sclerosis). For further information, visit

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