PIPELINE

Pipeline specialized for Musculoskeletal Diseases

Pipeline

이뮤노포지㈜는 근골격계 희귀질환 신약 후보물질을 발굴하며, 다수의 신약 파이프라인을 보유하고 있습니다.

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주요 파이프라인 현황

Identification Indication Market 기초연구
Opt
전임상
Tox
임상1상
P I
임상2상
P II
임상3상
P III
PF1801 DMD Global
BMD Global
Polymyositis Global
Inclusion Body Myositis Global
Senile Sarcopenia Global
PF1802 ALS Global
PF1803 Degenerative Arthritis Global
KF1601 CML Domestic
PF1801
  • Biological – A novel long acting GLP-1(glucagon-like peptide-1) receptor agonist
  • GLP-1 receptor activation may have a potential therapeutic application as a treatment for diseases that involve muscle atrophy and muscle wasting.
    (Ref: Hong et el., 2019)
  • Elastin-Like Polypeptide (ELP) half-life extension technology platform enables once weekly injection
  • Safety profile based on clinical data from phase 2b study in type 2 diabetes (20 weeks)
  • Drug re-positioning global development strategy :
    Targeting various indications involved in muscle atrophy including DMD, BMDand Polymyositis
  • Stage :
    IND submission for phase IIa in DMD

DMD (Duchenne Muscular Dystrophy)
BMD (Becker Muscular Dystrophy)

  • DMD and BMD are genetic disorders characterized by the progressive loss of muscle
Symptoms
  • Difficulty rising from a lying or sitting position
  • Large calf muscle, Walking on toes
  • BMD is a milder version of DMD
Causes
  • the mutated DMD gene fails to produce virtually any functional dystrophin
  • BMD genetic mutations make partially functional dystrophin
Standard Therapy
  • High dose glucocorticoids

Polymyositis (다발성 근염)

  • A rare, idiopathic, inflammatory myopathy
Symptoms
  • Symmetric proximal muscle weakness
  • In severe cases, causes dysphagia or dysphonia
  • Serious threat to the quality of daily life
Causes
  • Auto-immune responses in skeletal muscle
  • Lymphocyte infiltration in the muscle
Standard Therapy
  • High dose glucocorticoids
  • Glucocorticoid-induced toxicity

IBM (Inclusion body myositis, 봉입체 근염)

  • A complex disease with degenerative pathological mechanisms as well as autoimmune mediated by T lymphocytes
Symptoms
  • Progressive muscle weakness in distal finger flexor muscles and the quadriceps
  • Unlike other myositis, asymmetric and distal muscle weakness are likely to appear with slow disease progression
Causes
  • Inflammatory cells invade the muscle tissue and concentrate between the muscle fibers presenting multiple inclusion bodies that contain cellular material of dead tissue.
Standard Therapy
  • Immunosuppressant drugs
  • Corticosteroid doesn’t work for IBM.

Senile Sarcopenia (노인성 근감소증)

  • Senile sarcopenia is the loss of muscle mass due to the natural aging process
Symptoms
  • Loss of lean muscle mass and decreased muscle size
  • Reduced strength, functional decline and increased risk of falling
Causes
  • Senile sarcopenia is likely the result of multiple interacting factors such as changes in hormones, age-related muscle changes, nutrition, and lifestyle
Standard Therapy
  • Currently there are no medication to treat senile sarcopenia
PF1802
BBB penetration and bio-availability enhanced first-in-classALS therapeutic candidate
BBB penetration and bio-availability enhanced first-in-classALS therapeutic candidate
BBB penetration and bio-availability enhanced first-in-classALS therapeutic candidate
  • Indication
    : ALS (Amyotrophic lateral sclerosis)
    (also known as Lou Gehrig's disease Or motor neurone disease)
  • ALS
    : ALS is a devastating neurodegenerative disease that results in progressive loss of motoneurons, motor weakness and death within 3-5 years after disease onset.
  • Class
    : Biologics, Dual ligand Fc fusion protein
  • Global market size expectation : USD 800 million in 2026
  • Status : in vivo PK
  • Next plan : in vivo Efficacy test
PF1803
Indication: Degenerative arthritis
  • Class: First-in-class new biologic drug: dual function fusion protein
  • Most common degenerative joint disease with extreme pain from joint cartilage destruction or regression
  • Global market: $ 6 billion in 2019 → $ 9 billion in 2024 (CAGR: 8.1%)
  • Novel Disease-modifying osteoarthritis drug (DMOAD)
  • Status: Stable cell line development
KF1601
New Chemical Entity
  • KF1601 is a rationally designed BCR-ALB inhibitor
    with activity against the common resistance mutation T315I
Developmental Strategy
  • Ponatinib is the first licensed BCR-ABL inhibitor with activity against T315I, but has significant side effects, including cardiac and vascular events
  • KF1601 has been developing to overcome the side effects of ponatinib
Developmental Stage
  • KF1601 is planned to enter non-clinical development in