KeifeRx Pipeline

KeifeRx’s robust development pipeline leverages mechanisms of action inherent to TKIs which thus far have been underexplored. Our technologies are designed to treat neurodegenerative diseases and an array of inflammatory diseases where there are significant unmet clinical needs and limited therapeutic options, resulting in painful, life-diminishing, and discomforting consequences for patients.

Program
IND Enabling
Phase 1
Phase 2
Phase 3

KFRX01

Early Alzheimer's Disease (Nilotinib BE)
IND APPROVED

KFRX01 (Nilotinib BE) is an optimized re-formulation of nilotinib with improved bioavailability and solubility being developed for the treatment of early Alzheimer’s disease. KeifeRx has designed KFRX01 to be delivered as an oral capsule at lower doses (84mg and 112mg daily) compared to what is currently approved as a treatment for chronic myeloid leukemia (600mg- 1200mg daily).

Alzheimer’s disease is a progressive neurodegenerative disease that is characterized by impaired memory and thinking (dementia), and functional decline. With a rapidly accelerating worldwide prevalence, there is a growing societal pressure for curative treatment for Alzheimer’s patients. The burden of Alzheimer’s encompasses clinical burden to patients as well as economic burden to payers and patients and caregiver burden, adding to the urgent need for improved Alzheimer’s therapy. Alzheimer’s is the only top-ten leading cause of death with no known treatment to cure or even slow the progression of disease.

An IND for a 1,275 patient, multicenter, placebo-controlled, double-blind Phase 3 clinical trial to investigate the safety and efficacy of KFRX01 on the progression of dementia in early Alzheimer’s disease has been cleared by the FDA. The Phase 3 trial – NILEAD – is expected to initiate in late 2022.

KeifeRx is also conducting a clinical trial of nilotinib (Tasigna®) in Lewy body dementia (Phase 2) through Georgetown University.

LEWY BODY DEMENTIA (Nilotinib)
RECRUITING

KeifeRx is also conducting a clinical trial of nilotinib (Tasigna ® ) in Lewy body dementia (Phase 2) through Georgetown University.

LBD is the second most common type of progressive dementia after Alzheimer's disease. Because Lewy body dementia can closely resemble other more commonly known diseases like Alzheimer’s and Parkinson’s, it is currently widely under-diagnosed. LBD causes a progressive decline in mental abilities, including visual hallucinations and changes in alertness and attention. Other effects include Parkinson's disease-like signs and symptoms such as rigid muscles, slow movement and tremors.

KFRX02

Lewy Body Dementia (Bosutinib)
In preparation

KFRX02 is an optimized oral dose of bosutinib (Bosulif®). KeifeRx completed a Phase 2 clinical trial in Lewy Body Dementia (LBD), though Georgetown University, demonstrating that KFRX02 administered at 100mg daily is near at or the lowest effective dose in LBD.

LBD is the second most common type of progressive dementia after Alzheimer's disease. Because Lewy body dementia can closely resemble other more commonly known diseases like Alzheimer’s and Parkinson’s, it is currently widely under-diagnosed. LBD causes a progressive decline in mental abilities, including visual hallucinations and changes in alertness and attention. Other effects include Parkinson's disease-like signs and symptoms such as rigid muscles, slow movement and tremors.

KeifeRx plans to initiate a multicenter, Phase 2/3 placebo-controlled, double-blind study to investigate KFRX02 as a treatment for LBD in the second quarter of 2023. The Phase 2 portion of the study will be utilized to identify the optimal dose range for the Phase 3 trial, which will evaluate the safety and efficacy of KFRX02 in LBD.

KFRX03

Amyotrophic Lateral Sclerosis (ALS)

KFRX03 is an internally-developed novel TKI currently undergoing IND enabling studies for first- in-human (FIH) Phase 1 clinical trials. KFRX03 has demonstrated significant pre-clinical effects on a variety of central (autophagy and neuro-inflammation) and peripheral (mast cell immunity) pathologies. KeifeRx expects to complete IND-enabling work by Q4 2023. KeifeRx plans to initiate the FIH Phase 1 studies in Q1 2024 to determine a maximal tolerated dose in healthy volunteers. Following that, KeifeRx would advance KFRX03 to Phase 2 clinical trials in neurodegeneration, including ALS, and inflammatory diseases such as MCAS and Lyme disease.

Lyme Disease

KFRX03 is an internally-developed novel TKI currently undergoing IND enabling studies for first- in-human (FIH) Phase 1 clinical trials. KFRX03 has demonstrated significant pre-clinical effects on a variety of central (autophagy and neuro-inflammation) and peripheral (mast cell immunity) pathologies. KeifeRx expects to complete IND-enabling work by Q4 2023. KeifeRx plans to initiate the FIH Phase 1 studies in Q1 2024 to determine a maximal tolerated dose in healthy volunteers. Following that, KeifeRx would advance KFRX03 to Phase 2 clinical trials in neurodegeneration, including ALS, and inflammatory diseases such as MCAS and Lyme disease.

Mast Cell Activated Syndrome (MCAS)

KFRX03 is an internally-developed novel TKI currently undergoing IND enabling studies for first- in-human (FIH) Phase 1 clinical trials. KFRX03 has demonstrated significant pre-clinical effects on a variety of central (autophagy and neuro-inflammation) and peripheral (mast cell immunity) pathologies. KeifeRx expects to complete IND-enabling work by Q4 2023. KeifeRx plans to initiate the FIH Phase 1 studies in Q1 2024 to determine a maximal tolerated dose in healthy volunteers. Following that, KeifeRx would advance KFRX03 to Phase 2 clinical trials in neurodegeneration, including ALS, and inflammatory diseases such as MCAS and Lyme disease.

KFRX04

Mastocytosis/Urticaria

KFRX04 is an internally-developed novel TKI currently undergoing IND enabling studies for first- in-human (FIH) Phase 1 clinical trials. KFRX04 has demonstrated significant pre-clinical effects on a variety of central (autophagy and neuro-inflammation) and peripheral (mast cell immunity) pathologies. KeifeRx expects to complete all IND-enabling work by Q4 2023. KeifeRx plans to initiate the FIH Phase 1 studies in Q1 2024 to determine a maximal tolerated dose in healthy volunteers. Following that, KeifeRx would advance KFRX04 to Phase 2 clinical trials in neurodegeneration, including MCI, and inflammatory diseases, such as mastocytosis and urticaria.

Mild Cognitive Impairment

KFRX04 is an internally-developed novel TKI currently undergoing IND enabling studies for first- in-human (FIH) Phase 1 clinical trials. KFRX04 has demonstrated significant pre-clinical effects on a variety of central (autophagy and neuro-inflammation) and peripheral (mast cell immunity) pathologies. KeifeRx expects to complete all IND-enabling work by Q4 2023. KeifeRx plans to initiate the FIH Phase 1 studies in Q1 2024 to determine a maximal tolerated dose in healthy volunteers. Following that, KeifeRx would advance KFRX04 to Phase 2 clinical trials in neurodegeneration, including MCI, and inflammatory diseases, such as mastocytosis and urticaria.