A new drug for narcolepsy! FDA grants strong anti-adrenaline reuptake inhibitor AXS-12 breakthrough drug eligibility!

August 07, 2020 // -- Axsome Therapeutics is a clinical biopharmaceutical company focused on the development of innovative treatments for central nervous system (CNS) diseases.
recently announced that the U.S. Food and Drug Administration (FDA) has granted AXS-12 (reboxetine) a breakthrough drug (BTD) for the treatment of narcolepsy patients with narcolepsy.
narcolepsy (narcolepsy) is a serious, debilitating neurological disorder characterized by excessive daytime drowsiness (EDS) and sudden loss of muscle tension caused by strong emotions.
the disease is an incurable disease, and over time, the burden of the disease can have a profound impact on the patient's health.
AXS-12 (reboxetine) is a new, oral, highly selective, powerful epinephrine reuptake inhibitor that is about to enter Phase III clinical development for the treatment of onset sleeping sickness.
AXS-12 regulates epinephrine activity, promotes awakening, maintains muscle tension, and enhances cognitive ability.
, the FDA also granted AXS-12 orphan drug eligibility (ODD) for the treatment of onset sleeping sickness.
has extensive safety records in Europe and more than 40 countries where it has been approved for the treatment of depression.
the role of AXS-12 in onset sleeping sickness was supported by preclinical and preliminary clinical results showing that AXS-12 significantly reduced the onset of onset sleeping sickness in mice with lower vision secretion (appetite) deficiency.
an open-label pilot trial, AXS-12 significantly improved daytime drowsiness and reduced sudden fall in patients with narcolepsy.
BTD is a new drug review channel created by the FDA in 2012 to accelerate the development and review of new drugs for the treatment of severe or life-threatening diseases, and there is preliminary clinical evidence that the drug significantly improves one or more clinically significant endpoints compared to existing therapeutic drugs.
get BTD drugs and are developed with closer guidance, including from top FDA officials, to ensure that patients are offered new treatment options in the shortest possible time.
FDA granted AXS-12 BTD, based on positive results from Phase II CONCERT studies.
was a randomized, double-blind, placebo-controlled, cross-centered, multi-center U.S. trial.
the trial, 21 patients diagnosed with onset sleeping sickness with sudden collapse received 2 weeks of AXS-12 or placebo treatment, followed by cross-treatment after 1 week of downward titration and eluption.
results showed that AXS-12 reached the main endpoint: the average number of episodes per week (overall treatment effect) during the 2-week treatment period was significantly lower than the baseline (p 0.001) compared to placebo, and the average number of episodes per week at the end of the 2-week treatment period was significantly lower than the baseline (p.002).
addition, AXS-12 significantly improved EDS compared to placebos, measured by the Epworth Sleepiness Scale (ESS) and the frequency of accidental naps (p.003 and p.lt;0.001, respectively).
in addition, AXS-12 significantly improved cognitive function over a 2-week treatment period compared to placebo, based on the concentration of ability (NSAQ-AC) measurements from the Onset Sleep disease Symptom Assessment Questionnaire.
the trial, AXS-12 was well tolerated, with the most common adverse reactions being anxiety, constipation and insomnia. Dr. Herriot Tabuteau, Ceo of
Axsome, said, "The FDA's qualification of AXS-12 for the breakthrough drug for the treatment of sudden onset of sleeping sickness highlights the potential of the drug, if successfully developed, to bring meaningful benefits and substantial improvements to the patient population with this debilitating neurological disorder compared to current treatment options."
we are excited about the rapid clinical advance of AXS-12, which has previously been granted orphan drugs to treat onset sleeping sickness.
, Axsome has been granted three breakthrough drugs by the FDA, the other two being AXS-05 for severe depressive disorder and AXS-05 for Alzheimer's disease.
these qualifications reflect Axsome's commitment to developing potentially life-changing drugs, innovative clinical development methods, and the resulting differentiation and extensive late-stage central nervous system pipelines for patients with difficult-to-treat central nervous system diseases.
AXS-05 is made up of dextromethorphan and bupropion, using Axsome's metabolic inhibition technology.
AXS-05's right methafine group is a non-competitive N-methyl-D-winterine (NMDA) receptor antagonist, also known as glutamate receptor regulator, which is a new mechanism of action, which means that it functions differently from the current treatment of depression.
AXS-05's right methafine composition is also a sigma-1 receptor agitant, niacin acetylcholine receptor antagonist, serotonin, and de-adrenaline transporter inhibitors.
Amphetamine component of AXS-05 improves the biousability of right methadine, a de-adrenaline and dopamine reuptake inhibitor, and a niacin acetylcholine receptor antagonist.
AXS-05 holds more than 30 U.S. and international patents until 2034.
() Original source: Axsome Therapeutics Receives FDA Development Therapy Designation for AXS-12 for The Treatment of Narcolepsy.