Update on promising drug NU-9

Positive results in mouse studies

Whilst the focus is on MND (motor neuron disease) with this drug candidate currently, Dr Ozdinler has flagged this compound as having potential for treating HSP.

NU-9 lengthens axons of diseased neurons better than current FDA-approved drugs for MND

New drug’s effect is enhanced when given in combination with existing MND drugs

If the animal studies go well and the FDA approves, NU-9 could be in clinical trials in early 2023 

CHICAGO — New research on the experimental drug, NU-9 to treat MND (motor neuron disease) otherwise known as ALS (amyotrophic lateral sclerosis), shows it is more effective than existing FDA-approved drugs for the disease. 

More importantly, NU-9 has an enhanced effect when given in combination with those drugs, riluzole and edaravone.

The research, published recently in Scientific Reports, showed NU-9 lengthened the axons of diseased upper motor neurons in mice with MND model.

The axon is the segment of the upper motor neuron that connects the brain to the spinal cord and makes the corticospinal tract, which degenerates in people with MND and HSP.

“For a drug to be effective, it is important for that drug to improve axon outgrowth and axon health,” said co-lead study author Ozdinler. “This is very important for connecting the brain and the spinal cord and for revitalizing the motor neuron circuitry that degenerates in patients.”

NU-9 moving toward clinical trials

AKAVA Therapeutics is carrying out animal safety studies needed for the drug (now called AKV9 in the company) to receive FDA approval to become an Investigational New Drug. Those studies include determining dose level and toxic effects. 

“If everything goes well, we hope to start with healthy volunteers in a Phase 1 clinical trial early in 2023,” said co-lead study author Silverman.

“It is a long process – possibly 10 to 12 years – to discover and bring a new drug to the market,” Silverman said. “But this drug has us very excited and hopeful about its possibilities to improve the lives of ALS patients, who have been without hope for so long.“

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SOURCE:  Eureka Alert NEWS RELEASE 17-MAY-2022

New ALS ‘drug’ is more effective than existing ones

Northwestern University

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