New treatments for patients affected by orphan diseases

Chord Therapeutics is a clinical stage pharmaceutical company developing drugs for patients with rare, life-threatening and severely disabling diseases. We are advancing our lead drug candidate CRD1 under orphan drug designation for the treatment of neuromyelitis optica spectrum disorders (NMOSD) and myasthenia gravis (MG).

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Dedicated to making a difference

At Chord Therapeutics, we are dedicated to changing the lives of patients affected by rare diseases that often have no or only limited treatment options.

We build our development programs on well-understood active substances with a history of clinical testing and use in human patients. We carefully select new indications based on a strong scientific rationale for a benefit of the mode of action of the drug in the selected disease. Taking a molecule with well studied safety and pharmacology forward to a new indication significantly reduces risk for patients and increases the likelihood for a successful development bringing new treatment options to patients and healthcare providers.

The active substance in our lead medicinal product candidate CRD1 is cladribine. The extensive previous experience with cladribine substantially reduces the risk of our developments. Our cladribine product and developments are completely distinct and independent from marketed drug products that contain cladribine as an active substance.

We are convinced that cladribine’s demonstrated safety and powerful, well-understood pharmacology can provide benefit in a variety of rare disorders and lead to de-risked treatment innovation.


Meet CRD1 / cladribine

The active substance in our lead medicinal product candidate CRD1 is cladribine. Cladribine’s mode of action and safety are well studied and understood. The safety and efficacy of cladribine has been studied in thousands of patients with autoimmune and oncologic diseases.

Cladribine reduces the number of B and T lymphocytes in the body. The reduction of lymphocytes, in particular of memory B cells, is long-term, which is considered to relate to cladribine’s efficacy. The long-term treatment effects are achieved with only a few cladribine administrations.

Cladribine has very limited activity against innate immune cells - another set of white blood cells - which remain available to fight infections. This is thought to avoid immune suppression in patients treated with cladribine. The effect of cladribine is mainly directed to lymphocytes. The relative selectivity for B and T cells is due to cladribine’s mode of action.

Cladribine seems particularly suitable for the treatment of diseases that are mediated by a pathological activity of B cells. A pronounced B cell-mediated pathology is the common theme in the rare disorders Chord Therapeutics selected for the development of CRD1.

The safety of cladribine is well studied and does not require extensive monitoring.


What is neuromyelitis optica spectrum disorder (NMOSD) ?

NMOSD is a rare, severe, chronic inflammatory disease of the central nervous system.

NMOSD presents in attacks. The symptoms are varied, depending on the areas of the central nervous system (CNS) that are affected.

NMOSD mainly presents as optic neuritis (ON) that affects eye function, and/or transverse myelitis (TM) that typically involves long segments of the spinal cord and affects limb, bladder & bowel function. Other symptoms of NMOSD include sharp pain or muscle twitches through the abdomen, back, or neck. Area postrema syndrome (APS) manifesting as intractable nausea, vomiting, or hiccups (INVH) is also often present.

NMOSD is an antibody mediated auto-immune disorder. This means the body directs an immune response against its own healthy cells and tissues. This immune attack causes the symptoms of NMOSD.

CRD1 is developed with the aim to block the auto-immune process and thus reduce relapses & change the course of NMOSD.


Latest updates from Chord Therapeutics

Dec 20, 2021

Merck Acquires Chord Therapeutics to Expand Neuroinflammatory Pipeline

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Oct 22, 2020

Spotlight on rare NMOSD: Chord launches with $16m Series A round

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Oct 15, 2020

Chord Therapeutics in the media

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