Pipeline

Biosplice Therapeutics has designed an unprecedented compendium of new chemical entities which selectively bind to specific members of the CLK/DYRK family and feature drug-like properties amenable to local or systemic administration and CNS exposure.

Biosplice is leveraging the broad therapeutic potential of alternative splicing modulation in musculoskeletal diseases, immunology/inflammation, and oncology.

Biosplice has established clinical proof-of-concept for therapeutic modulation of alternative splicing with the development of lorecivivint, targeting osteoarthritic disease progression by inhibition of alternative splicing-dependent Wnt pathway protein production. Similarly, our clinical trials in oncology center on pan-CLK and DYRK inhibition for therapeutic modulation of alternative splicing.

Target Indication Stage
Pan-CLK/DYRK
(oral)
mCRPC, CRC, NSCLC
Cirtuvivint
(SM08502)

Phase 1

Select heme malignancies
Cirtuvivint
(SM08502)

IND Enabling

Additional solid tumors
Cirtuvivint
(SM08502)

IND Enabling

DYRK1A
(oral)
Select heme malignancies

Lead Optimization

Molecularly defined solid tumors

Lead Optimization

CLK2
(oral)
Indication-specific solid tumors

Lead Optimization

CLK3
(oral)
Indication specific solid tumors

Lead Optimization

Target Indication Stage
DYRK1A
(oral, brain penetrant)
Alzheimer’s disease

Lead Optimization

Traumatic brain injury

Discovery

Select tauopathies

Discovery

Target Indication Stage
Pan-CLK/DYRK
(intra-articular injection)
Osteoarthritis of the knee
Lorecivivint
(SM04690)

Phase 3

Osteoarthritis of the hip
Lorecivivint
(SM04690)

IND Enabling

* Phase 2

*
Osteoarthritis of the shoulder
Lorecivivint
(SM04690)

IND Enabling

* Phase 1

*
Degenerative disc disease
Lorecivivint
(SM04690)

IND Enabling

CLK: CDC2-like kinase

DYRK: Dual specificity tyrosine-phosphorylation-regulated kinase

*Likely accelerated program, based on knee OA program

Therapeutic
Area
Target Indication Discovery Lead
Optimization
IND
Enabling
Phase 1 Phase 2 Phase 3 NDA
Oncology Pan-CLK/DYRK
(oral)
mCRPC, CRC, NSCLC
Cirtuvivint (SM08502)
Select heme malignancies
Cirtuvivint (SM08502)
Additional solid tumors
Cirtuvivint (SM08502)
DYRK1A
(oral)
Select heme malignancies
Molecularly defined solid tumors
CLK2
(oral)
Indication-specific solid tumors
CLK3
(oral)
Indication-specific solid tumors
Neurology
DYRK1A
(oral, brain penetrant)
Alzheimer’s disease
Traumatic brain injury
Select tauopathies
Musculoskeletal Pan-CLK/DYRK
(intra-articular injection)
Osteoarthritis of the knee
Lorecivivint (SM04690)
Osteoarthritis of the hip
Lorecivivint (SM04690)
*
Osteoarthritis of the shoulder
Lorecivivint (SM04690)
*
Degenerative disc disease
Lorecivivint (SM04690)

CLK: CDC2-like kinase

DYRK: Dual specificity tyrosine-phosphorylation-regulated kinase

*Likely accelerated program, based on knee OA program