ProgramMechanismDiscoveryPreclinicalPhase 1Phase 2Phase 3
CNS Portfolio
PIPE-307 Multiple Sclerosis
Myelin Restoration
M1R Antagonist

Myelin Restoration

 

Multiple Sclerosis is a progressive, inflammatory, and neurodegenerative disease of the central nervous system, or CNS. MS impacts approximately one million patients in the United States and over 2.8 million patients worldwide.

PIPE-307 is our proprietary, oral, selective antagonist of the muscarinic M1 receptor, or M1R, that drives the maturation of OPCs into myelin-producing oligodendrocytes. We are developing PIPE-307 to restore myelin as a potential treatment for MS.

In the second half of 2021 we completed a randomized, double-blind, placebo-controlled Phase 1 clinical trial of PIPE-307 in healthy volunteers, and we completed a Phase 1, single-center, open-label, adaptive-design study to investigate the occupancy of brain M1R after a single oral dose of PIPE-307 in healthy volunteers by positron emission tomography (PET) using the radioligand [11C] PIPE-307.

The company has received IND clearance from the FDA to initiate a Phase 1b/2a trial of PIPE-307 in relapsing-remitting MS patients.

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PIPE-791 Myelin Restoration and
Neuro-inflammation
LPA1R Antagonist

Myelin Restoration & Neuroinflammation

 

PIPE-791 is our most advanced preclinical program and focuses on antagonism of the LPA1 receptor.

LPA is a naturally occurring inflammatory lipid and its dysregulation has been implicated in inflammatory diseases, such as MS. Recent studies have shown that MS patients have elevated levels of LPA in serum and in the cerebral spinal fluid. Furthermore, LPA was observed to significantly increase in patients during disease flares compared to periods of remission. We believe LPA1 activation plays a role in limiting remyelination and driving neuroinflammation.

We recently nominated a development candidate and are moving into IND-enabling studies.

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Discovery Undisclosed

Axonal Repair

 

Axon loss and neurodegeneration constitute clinically debilitating consequences in demyelinating diseases such as MS and other neurodegenerative diseases. Novel discoveries of the underlying mechanisms of secondary axonal degeneration are helping to shape breakthrough therapies to treat this degenerative process.

Pipeline is currently pursuing its axonal repair program utilizing its clinical translational approach. We are currently in the target identification and validation stages for our axonal repair program.

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Neuro-Otology
PIPE-505 Sensorineural Hearing Loss
Synaptic Regeneration
Gamma Secretase Inhibitor

Synapse & OHC Regeneration

 

Hearing loss is the fourth leading cause of disability worldwide, impacting an estimated 466 million people globally. In the United States, hearing loss affects approximately 15 percent of adults. The rate of hearing loss increases with age and is considered to be the third most common health condition affecting older adults following heart disease and arthritis. The societal effects of hearing loss are profound and can directly impair human communication and affect an individual’s ability to participate in basic daily interactions. If left untreated over a period of five years, hearing loss is associated with an approximately 50 percent increase in dementia and depression.

PIPE-505 is our proprietary, locally-delivered, gamma secretase inhibitor that targets two key regenerative pathways in the cochlea, the Netrin/DCC pathway for synaptic regeneration and the Notch pathway for outer hair cell regeneration. These pathways are linked to the two main areas of deficient auditory function in sensorineural hearing loss (SNHL), intelligibility and audibility, respectively.

In the second half of 2021 we completed a randomized, double-blind, placebo-controlled, Phase1/2a clinical trial of PIPE-505 in patients with SNHL.

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DrugIndicationStage
CNS Portfolio
PIPE-307 Multiple Sclerosis
Myelin Restoration
M1R AntagonistPhase 1
PIPE-791 Myelin Restoration and
Neuro-inflammation
LPA1R AntagonistPreclinical
Discovery UndisclosedDiscovery
Neuro-Otology
PIPE-505 Sensorineural Hearing Loss
Synaptic Regeneration
Gamma Secretase InhibitorPhase 1

PIPE-307 Multiple Sclerosis
Myelin Restoration

Multiple Sclerosis is a progressive, inflammatory, and neurodegenerative disease of the central nervous system, or CNS. MS impacts approximately one million patients in the United States and over 2.8 million patients worldwide.

PIPE-307 is our proprietary, oral, selective antagonist of the muscarinic M1 receptor, or M1R, that drives the maturation of OPCs into myelin-producing oligodendrocytes. We are developing PIPE-307 to restore myelin as a potential treatment for MS.

In the second half of 2021 we completed a randomized, double-blind, placebo-controlled Phase 1 clinical trial of PIPE-307 in healthy volunteers, and we completed a Phase 1, single-center, open-label, adaptive-design study to investigate the occupancy of brain M1R after a single oral dose of PIPE-307 in healthy volunteers by positron emission tomography (PET) using the radioligand [11C] PIPE-307.

The company has received IND clearance from the FDA to initiate a Phase 1b/2a trial of PIPE-307 in relapsing-remitting MS patients.

PIPE-791 Myelin Restoration and
Neuro-inflammation

PIPE-791 is our most advanced preclinical program and focuses on antagonism of the LPA1 receptor.

LPA is a naturally occurring inflammatory lipid and its dysregulation has been implicated in inflammatory diseases, such as MS. Recent studies have shown that MS patients have elevated levels of LPA in serum and in the cerebral spinal fluid. Furthermore, LPA was observed to significantly increase in patients during disease flares compared to periods of remission. We believe LPA1 activation plays a role in limiting remyelination and driving neuroinflammation.

We recently nominated a development candidate and are moving into IND-enabling studies.

Discovery

Axon loss and neurodegeneration constitute clinically debilitating consequences in demyelinating diseases such as MS and other neurodegenerative diseases. Novel discoveries of the underlying mechanisms of secondary axonal degeneration are helping to shape breakthrough therapies to treat this degenerative process.

Pipeline is currently pursuing its axonal repair program utilizing its clinical translational approach. We are currently in the target identification and validation stages for our axonal repair program.

PIPE-505 Sensorineural Hearing Loss
Synaptic Regeneration

Hearing loss is the fourth leading cause of disability worldwide, impacting an estimated 466 million people globally. In the United States, hearing loss affects approximately 15 percent of adults. The rate of hearing loss increases with age and is considered to be the third most common health condition affecting older adults following heart disease and arthritis. The societal effects of hearing loss are profound and can directly impair human communication and affect an individual’s ability to participate in basic daily interactions. If left untreated over a period of five years, hearing loss is associated with an approximately 50 percent increase in dementia and depression.

PIPE-505 is our proprietary, locally-delivered, gamma secretase inhibitor that targets two key regenerative pathways in the cochlea, the Netrin/DCC pathway for synaptic regeneration and the Notch pathway for outer hair cell regeneration. These pathways are linked to the two main areas of deficient auditory function in sensorineural hearing loss (SNHL), intelligibility and audibility, respectively.

In the second half of 2021 we completed a randomized, double-blind, placebo-controlled, Phase1/2a clinical trial of PIPE-505 in patients with SNHL.