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KDDF-201512-10 Preclinical study of new chemical entity for orphan disease: infantile spasms(CNS Diseases, Chemical) [2019-06-24]

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Ongoing Project
Section Lead
Generation
Lead
Optimization
Preclinical Phase I Phase II Phase III

Development and Market Objectives

•To obtain US FDA Phase 1/2 study approval for infantile spasms (also known as West syndrome), a rare seizure disorder that occurs in young children, usually under one year of age.
•To increase the value of the compound by applying for the US FDA’s expedited review programs, which would result in faster review times and earlier marketing approval of our orphan drug for infantile spasms.

Unmet Medical Need & Target Patients

A. Target Patients
•2 to 24-month-old infants presenting with spasms (myoclonic jerks) of the body, and hypsarrhythmia or modified hypsarrhythmia on video EEG recording.

 

B. Unmet Medical Needs
•A significant portion of children with infantile spasms are refractory to the currently approved treatments, adenocorticotropic hormone and vigabatrin, and thus a more effective treatment option is needed at this time.
•As the currently approved treatments for infantile spasms have significant side effects, an effective treatment with a superior side-effect profile is needed.
•Approximately 60% ofchildren with infantile spasms develop other types of epilepsy, such as Lennox-Gastaut syndrome, as they age.
•Mental retardation occurs in 70-90% of children with infantile spasms. 
•Drugs with antiepileptogenic properties may be needed to prevent children with infantile spasms from progressing to Lennox-Gastaut syndrome, other types of epilepsy and also side effects including mental retardation.
 

Status

•Phase 1 study to assess safety, tolerability and pharmacokinetics in healthy volunteers has been completed under a US FDA IND and Health Canada CTA.
•Juvenile toxicity studies to support clinical Phase 1/2 study approval are ongoing.
•Preparations for a pre-IND meeting with the US FDA and for the submission of an orphan drug designation application are ongoing.

Intellectual Property

A. Composition of Matter Patent:
•Filed in 2010 (PCT/KR2011/004862: PHENYLCARBAMATE COMPOUND AND MUSCLE RELAXANT CONTAINING THE SAME)
•Received patent allowance in the US, Canada, Korea, Japan, and China.

 

B. Process Patent: 
•Filed in 2011 (PCT/KR2011/010105: PROCESS FOR PREPARATION OF PHENYL CARBAMATE DERIVATIVES)
•Received patent allowance in the US, Japan, Korea, and China.

 

C. Method of Use Patent:
•Epilepsy: Filed in 2011 (PCT/KR2012/011474: PHENYL CARBAMATE COMPOUNDS FOR USE IN PREVENTING OR TREATING EPILESY)
•Pain: Filed in 2011 (PCT/KR2012/011470: PHENYL CARBAMATE COMPOUNDS FOR USE IN ALLEVIATING OR TREATING PAIN AND NEUROPATHIC PAIN): Received patent allowance in the US.
•Stroke: Filed in 2011 (PCT/KR2012/011471: PHENYL CARBAMATE COMPOUNDS FOR USE IN PREVENTING OR TREATING STROKE): Received patent allowance in the US.
•11 other method of use patents are currently in patent prosecution.
 

Competitive Advantages

A. Efficacy
•Considerably superior efficacy compared to vigabatrin, an FDA approved drug, in a published symptomatic infantile spasms rat model.
•Potent anticonvulsant properties in a broad spectrum of epilepsy models.
•Potent efficacy in refractory epilepsy models, notably in models of benzodiazepine-resistant status epilepticus. 
•NIH/NINDS Anticonvulsant Screening Program has documented a comprehensive profile of the compound in a Red Book due to the excellent efficacy of the compound.

 

B. MoA
•Has a unique mechanism of action not presently associated with currently-used antiepileptic drugs.

Contact & Company Overview

  • Company Name :
    Bio-Pharm Solutions
  • Homepage :
  • Contact Person :
    Yongho Kwak
  • Contact :
    +82-031-888-9636

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