AUM Biosciences Receives FDA Orphan Drug Designation for the Treatment of Solid Tumors With the NTRK Fusion Gene

AUM Biosciences recently announced the US FDA has granted Orphan Drug Designation (ODD) to AUM601, a highly selective, oral small molecule that inhibits not only pan-TRK (TRKA, TRKB, and TRKC), but also resistance mutations of TRKs, by blocking the activity of tyrosine kinases.

“We are thrilled to have received our first Orphan Drug Designation just months after presenting our results from the Phase 1 study of AUM601,” said Vishal Doshi, CEO of AUM Biosciences. “This decision from the FDA, in addition to our existing clinical evidence, further strengthens our ambition to bring a differentiated and improved treatment option for solid tumors with the NTRK Fusion Gene. We look forward to beginning the Phase 2 trial soon and providing additional insight into its indisputable advantage over current treatment options.”

Initial results from the Phase 1 study of AUM601 were presented as a poster presentation at the ESMO Targeted Anticancer Congress 2022, which was held virtually in March 2022. This study concluded that AUM601 was safe and well-tolerated at dose levels of 50 to 300 mg QD. Doses of 200 and 300 mg QD were defined as recommend Phase 2 dose (RP2D) based on PK profile and safety results. A multi-national phase 2 study will be conducted to assess the efficacy and safety in patients with NTRK fusions and mutations.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the US. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the US if the drug is ultimately approved for its designated indication.

The neurotrophin tyrosine receptor kinase (NTRK) family composed of three members, TRKA, TRKB, and TRKC. These Tropomyosin receptor kinase (TRK) proteins are single-pass transmembrane proteins, expressed in the nervous system and in many non-neuronal tissue types. It has been observed that the occurrence of NTRK gene fusion between NTRK1, 2, or 3 and various unrelated gene partners, represents the genomic alteration with oncogenic potential, which has been observed in a wide range of human tumor types in comparison to oncogenic mechanisms.

Firts-generation TRK inhibitors, including larotrectinib and entrectinib, have demonstrated clinical benefit in patients with solid malignancies harboring oncogenic NTRK fusions. Solvent front mutation, gatekeeper mutation, and glycine mutation of DFG at the beginning of the A-loop have been reported in clinical trials from larotrectinib- and entrectinib-refractory patients.

AUM601 is a highly selective, oral small molecule that inhibits not only pan-TRK (TRKA, TRKB, and TRKC), but also resistance mutations of TRKs, by blocking the activity of tyrosine kinases. AUM601 is differentiated from current treatment options as it has no neurotoxicity, is highly selective and potent against TRK (281 Kinases, and has superiority to Larotrectinib, Selitrectinib, Entrectinib, and Repotrectinib. It inhibits TRKA/B/C fusion, and solvent front, gatekeeper and xDFG mutations of TRKs and has robust activity in preclinical models at well-tolerated doses. AUM601 has 22x higher concentration of drug in tumors as compared to plasma as well as higher tumor exposure in vivo. It has superior PK compared to Larotrectinib, favorable AE profile (no DTLs), and has established dose dependency.

AUM Biosciences is a global clinical-stage biotech company, focused on advancing a clinical pipeline of precision oncology therapeutics designed to deploy multi-faceted inhibition strategies to reverse cancer resistance. The company’s management has an extensive track record of selecting distinctive early-stage assets, successfully exiting virtual biotech models, and has contributed significantly to the development of several currently marketed oncology treatments with annual peak sales up to $3 billion. The company completed a $27 million Series A funding round. For more information, visit www.aumbiosciences.com