Pipeline specialized for Musculoskeletal Diseases


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

  • Home
  • >
  • Pipeline
  • >
  • Pipeline

주요 파이프라인 현황

Identification Indication Market 기초연구
PF1801 DMD Global
BMD Global
PM / DM Global
Inclusion Body Myositis Global
Senile Sarcopenia Global
PF1802 ALS Global
PF1803 Degenerative Arthritis Global
KF1601 CML Domestic
  • 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 al. 2019)
  • Elastin-Like Polypeptide(ELP) half-life extension technology platform enables weekly injection
  • Safety profile based on clinical data from phase IIb study in type 2 diabetes (20 weeks)
  • Drug re-positioning global development strategy :
    Targeting various indications that involve muscle atrophies including DMD, BMD and Polymyositis
  • Stage :
    IND approval for phase IIa in DMD & PM

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

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

Polymyositis (다발성근염) / Dermatomyositis (피부근염)

  • A rare, idiopathic, inflammatory myopathy
  • Symmetric proximal muscle weakness
  • In severe cases, causes dysphagia or dysphonia
  • Serious threat to the quality of daily life
  • 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
  • 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
  • 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
  • Loss of lean muscle mass and decreased muscle size
  • Reduced strength, functional decline and increased risk of falling
  • 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
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 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: Non-clinical study
Indication: Degenerative arthritis
  • Class: First-in-class new biologic drug: dual function fusion protein
  • Most common degenerative joint disease with extreme pain due to 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: Non-clinical study
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
  • Non-clinical study