Pursuing what’s possible
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Ongoing clinical research and studies

Catalyst remains steadfast in our commitment to explore the full, innovative potential of our products, ensuring all products are safe for use.

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SUMMIT Study Investigator-Sponsored Research

SUMMIT Registry Study (DMD-001)

Following patients with DMD ages 2 years and older and currently on AGAMREE® (vamorolone) for 5 years to evaluate its long-term safety.

Investigator-Sponsored Research

At Catalyst Pharmaceuticals, we are committed to the advancement of medical and scientific knowledge that addresses the unmet needs of patients with rare, debilitating neuromuscular and neurological diseases.

Investigator-Sponsored Research (ISR)

ISR represents an invaluable type of medical research, providing an innovative method of discovery that can generate promising medical interventions. ISR comprises clinical studies that are initiated and managed by non-pharmaceutical company researchers, such as:

  • Individual investigators
  • Institutions
  • Collaborative study groups
  • Cooperative groups

Evaluating ISR submissions

Catalyst will only support legitimate, legal, and relevant study proposals. Catalyst carefully evaluates all ISR submissions, taking into account their scientific rigor, methodological considerations, patient safety, and their ability to generate meaningful data that can translate to clinical practice. All ISR submissions must align to Catalyst product development plans and the disease states that Catalyst is active in or may consider entering. Support by Catalyst may be in the form of funding, providing study drug, or a combination of both. All support must be disclosed.

Catalyst’s current areas of greatest strategic interest

AGAMREE® (vamorolone)-Related
  • Anti-inflammatory effects underlying rare diseases.
  • Diseases/pathogenic mechanisms relevant to NF-kB suppression in rare diseases
FIRDAPSE® (amifampridine)-Related
  • Effects of Firdapse in the neuromuscular junction with relevance to rare diseases

Cancer-associated LEMS

Cancer-associated LEMS is the most common-mediated neurologic paraneoplastic syndrome in SCLC. Symptoms of cancer-associated LEMS are caused by the production of anti-voltage-gated calcium channel (VGCC) antibodies that impair signal transmission at the neuromuscular junction.

Catalyst is also willing to discuss potential research studies in areas adjacent or related to the above. Submission of an ISR proposal or protocol does not imply or guarantee approval.

Do you have a clinical research proposal?
Send Request

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Catalyst offers financial support where it matters most through targeted sponsorships and charitable grants.

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Catalyst Background

“We truly understand what it takes to get products approved and successfully in the hands of the patient in the rare disease space.”  

—Preethi Sundaram, Business Development

Headshot of Catalyst employee, Preethi Sundaram