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Pyramid Biosciences Receives Orphan Drug Designation for PBI-200 From FDA

  • PBI-200 is being developed for the treatment of NRTK fusion-positive solid tumors, including primary and metastatic brain tumors
  • PBI-200 is presently being evaluated in a global Phase 1/2 multicenter clinical trial

WALTHAM, Mass.--(BUSINESS WIRE)--#NRTK--Pyramid Biosciences, Inc., a clinical-stage biotechnology company developing a portfolio of precision therapies targeting a range of serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for PBI-200, a next-generation, highly brain penetrant inhibitor of the neurotrophic tyrosine receptor kinase (NTRK) currently in clinical development for the treatment of NTRK fusion-positive solid tumors.

“Oncogenic NTRK gene fusions are found in a broad range of cancers,” said Pyramid Biosciences CEO Brian Lestini, MD, PhD. “Orphan designation for drugs in development such as PBI-200 are important to ensure continued innovation to address unmet needs for these patients, including primary and metastatic brain tumors.”

“We are extremely pleased that the orphan designation was granted, and we look forward to continued enrollment in our global Phase 1/2 clinical trial of PBI-200,” said Pyramid Biosciences co-founder and COO Jordan Leef.

The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations or rare disorders, that affect fewer than 200,000 people in the United States.

About PBI-200

PBI-200 is an oral, highly potent, and selective inhibitor of TRK kinase that is currently in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases. In some patients, the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. PBI-200 was discovered and developed by Pyramid Biosciences to overcome a wide range of on-target resistance mutations that have been described with first-generation TRK inhibitors. In addition, preclinical studies have shown that PBI-200 achieves high levels of penetration into brain, which translated into superior efficacy in intracranial xenograft models compared to other TRK inhibitors, as well as a favorable safety profile.

The global Phase 1/2 trial of PBI-200 (PBI-200-101) is a multicenter, open-label study in subjects with NTRK fusion-positive advanced or metastatic tumors including primary and metastatic CNS tumors. The trial consists of a dose-escalation phase, followed by a multicohort expansion at the recommended Phase 2 dose. Additional information can be found at: (NCT04901806).

About Pyramid Biosciences

Founded in 2015, Pyramid Biosciences is a clinical-stage biotechnology company dedicated to developing a portfolio of precision therapies targeting a range of serious diseases. Pyramid Biosciences is currently in clinical development of two highly selective tropomyosin receptor kinase (TRK) inhibitors. For more information, please visit:


Pyramid Biosciences Media Inquiries:
McDougall Communications on behalf of Pyramid Biosciences
Elizabeth Harness, +1-585-435-7379

Pyramid Biosciences Investor Inquiries:
Jordan Leef, Pyramid Biosciences
COO, CFO and Co-Founder, +1-781-530-4461

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Last Updated: 06-Jul-2022