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Phase 2 Trial Research

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Phase 2 Trial Research

Phase 2 Trial Research

In PHASE 2 – Ezogabine, Mexiletine, Gilenya, Rasagiline, AT-1501, Pimozide, BLZ945, RNS60, Inosine, Reldesemtiv, Zilucoplan, CNM-Au8, Nurtec, Colchicine, DNL747, Amx0035, Biotin, BIIB078

Ezogabine Completed Phase 2:

  • Activates the KCNQ family of channels in neuronal membranes in resting states and calms the excitability that cause seizures.
  • Results of phase 2 trial shows that it lessens abnormal motor neuron excitability, or responsiveness.

Ezogabine Completed Phase 2:

Gilenya (Tdi-132 Or Fingolimod) Phase 2 Completed:

  • Gilenya traps the immune cells in the lymph nodes and thus reduced unnecessary neuroinflammation.
  • Preclinical trials in mouse models of ALS demonstrated that Gilenya is able to significantly extend survival and improve symptoms.
  • A Phase 2a clinical trial (NCT01786174) investigating the safety and tolerability of Gilenya compared to a placebo in patient with ALS has been completed.

Gilenya (Tdi-132 Or Fingolimod) Phase 2 Completed:

At – 1501 (Anti-Cd40l) Started Phase 2a:

A protein called CD40L on immune cell surface triggers inflammation in the spinal cord.
  • CD40L pathway is overactive in ALS patients.
  • AT – 1501 is an antibody against CD40L and blocks neuroinflammation.
  • Phase 2a trial (NCT04322149) is now recruiting patients.

At – 1501 (Anti-Cd40l) Started Phase 2a:

PIMOZIDE:

  • Pimozide is a FDA approved neuroleptic drug used to treat psychosis, Tourette syndrome, and resistant tics.
  • Enhances communication in NMJ.
  • Phase I study showed that after only six weeks of treatment, patients were able to retain the control of the thenar muscles.

PIMOZIDE:

BLZ945:

  • Induces reduction of microglia.
  • Increased oligodendrocytes and astrogliogenesis.
  • Induces brain region – specific enhancement of neuronal remyelination.

BLZ945:

RNS60:

  • RNS60 is composed of saline and oxygen.
  • Acts on the immune and inflammatory mechanism.
  • Prevents cells damage or death.

RNS60:

INOSINE:

  • Rationale: increase urate levels by administering precursor inosine.
  • A small pilot study with 25 ALS patients over 12 weeks of follow up reported that inosine is safe.
  • Several markers of oxidative stress favourably changed from baseline levels.
  • An ongoing phase 2 trial is testing its efficacy (NCT03168711).

INOSINE:

RELDESEMTIV:

  • Reldesemtiv is a type of fast skeletal muscle troponin activator (FSTA).
  • Eases muscle contraction with minimal nerve stimulation to slow the muscle function decline.
  • Middle and fast ALS progression had a significant benefit from Reldesemtiv.
  • Patients were also 38% less likely to need durable medical equipment, including wheelchairs, non invasive ventilators, feeding tubes, and speech generating devices (FORTITUDE-ALS study NCT03160898).

RELDESEMTIV:

ZILUCOPLAN :

  • A small peptide that binds to key players of the complement cascade with high affinity and specificity (C5 and C5b).
  • Complement inhibition addresses tissue damage in generalized myasthenia gravis.

ZILUCOPLAN :

CLENBUTEROL:

  • It is a beta2 adrenergic agonist.
  • It has an anabolic effect on skeletal muscles.
  • It also stimulates the beta adrenoreceptors in the CNS leading to increase Neurotrophic growth factor (NGF).
  • It has shown neuroprotective effect on animal models.

CLENBUTEROL:

CNM - AU8 :

  • CNM-Au8 is being explored for ALS, PD and MS.
  • Suspension of Nanocrystalline gold.
  • generates cellular energy.
  • Remove the destructive by – products of cellular metabolism.
  • Protects motor neurons (preclinical studies).
  • 2 Phase – 2 trials currently underway.
  • Rescue – ALS (Australia), recruiting patients, and REPAIR – ALS (USA), not yet recruiting.

CNM - AU8 :

NURTEC (BHV-0223):

  • New formulation of Riluzole.
  • Sublingual treatment: It is placed under the tongue, where it will dissolve and be absorbed quickly.
  • More predictable treatment levels in the body.

NURTEC (BHV-0223):

COLCHICINE :

  • Enhances the expression of HSPB8 and of several autophagy players.
  • Blocks TDP – 43 accumulation in neurons.
  • Has an anti – inflammatory effect.

COLCHICINE :

DNL747:

  • Overactive RIPK1 protein is involved in excess inflammation and cell death in the brain.
  • DNL747 inhibits RIPK1 protein.

DNL747:

BIOTIN ENROLLING IN PHASE 2 :

  • Low biotin levels cause oligodendrocyte and axonal degeneration.
  • Treatment with Biotin prevents motor neuron degeneration by preserving oligodendrocytes.
  • Pilot study results show that Biotin is safe.

BIOTIN ENROLLING IN PHASE 2 :

H.P ACTHAR GEL IN:

  • Studies suspended due to increased incidence of pneumonia among ALS patients receiving H.P. Acthar Gel.

H.P ACTHAR GEL IN:

RASAGILINE COMPLETED PHASE 2:

  • Monoamine oxidase B inhibitor, mitochondrial stabilizer already approved for the treatment of Parkinson’s Disease.
Results: The average 12 months ALSFRS-R slope between rasagiline and the mixed placebo and historical control cohorts did not show any differences. Urine and blood biomarkers did not show any benefits. Rasagiline was well tolerated and no serious adverse events noted.

RASAGILINE COMPLETED PHASE 2:

NP001:

  • NP001 was an injected therapy that was being developed by Neuraltus to slow or stop the progression of ALS.
  • The molecule was designed to calm down immune cells of the CNS- microglia – and turn them into anti-inflammatory code
Study suspended due to failed trial results

NP001:

GENE THERAPY FOR C9ORF72 MUTATION: BIIB078 IN PHASE 2:

  • Lowers the accumulation of toxic RNA clumps.
  • Reduces the activity of this mutated gene.

GENE THERAPY FOR C9ORF72 MUTATION: BIIB078 IN PHASE 2:

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We are a dedicated group towards well being of people with MND and their families. We understand the difficult problems they have to face due to the disease. We are well aware of their special needs and challenges to be met. We support them in all possible ways.

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Clinic Address :

Plot No: 19, Sector 40, Near Seawoods Grand Central Railway Station, Off, Palm Beach Rd, Seawoods, Navi Mumbai, Maharashtra 400706

Registered Address :
402, Guruprabha Apartment, S. B. Road, Dadar West, Mumbai – 400028, Maharashtra, India
Facebook X-twitter Youtube

We are a dedicated group towards well being of people with MND and their families. We understand the difficult problems they have to face due to the disease. We are well aware of their special needs and challenges to be met. We support them in all possible ways.

Facebook X-twitter Instagram Youtube

Get in Touch

Donate Now

+91-9819741333

+91-8104546479

ashaekhope@gmail.com

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Quick Links

Home

About Us

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Terms & Conditions

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Facebook X-twitter Youtube

Addresses

Clinic Address :

Plot No: 19, Sector 40, Near Seawoods Grand Central Railway Station, Off, Palm Beach Rd, Seawoods, Navi Mumbai, Maharashtra 400706

Registered Address :
402, Guruprabha Apartment, S. B. Road, Dadar West, Mumbai – 400028, Maharashtra, India
Copyright ©2025 All rights reserved by Asha Ek Hope Foundation.
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