OrphanDrug Designation for the non-radiolabeled version of anti-cancercompound TM601

TransMolecular, Inc., a biotechnology company focused on targetedtherapies for cancer, today announced that the FDA has granted OrphanDrug Designation for the non-radiolabeled version of its anti-cancercompound TM601, which is currently entering clinical trials for thetreatment of malignant glioma. The company had previously receivedOrphan designation for the radiolabeled version, 131I-TM601, whichrecently completed patient enrollment in a Phase 2 clinical trial inglioma and a Phase 1 trial in multiple tumor types. TM601 is a synthetic version of chlorotoxin, a naturally occurringpeptide derived from scorpion venom, which is highly specific intargeting both primary tumors and metastases. TM601 targets and bindsto receptors expressed on tumor cells but not on normal, healthycells. As TM601 binds primarily with the tumor cell receptor sites, italso delivers a targeted dose of radiation, killing the tumor cellwithout affecting nearby healthy cells. The data obtained frompreclinical and clinical data also suggest that native TM601 mayaffect a tumor's ability to grow and spread without added radiation,so the therapeutic potential as a non-radiolabeled peptide is alsobeing explored. The Company's robust development plan for TM601reflects its broad platform potential for multiple applications incancer. The FDA has granted it orphan drug status for patients withhigh-grade and malignant glioma, as well as a Fast Track designation.

"We are pleased to receive orphan drug status for TM601 inmalignant glioma," stated Michael Egan, President and Chief ExecutiveOfficer of TransMolecular. "The TM601 platform has performed very wellin recent Phase 1 and 2 clinical trials, showing specific tumortargeting in both primary and metastatic disease of multiple tumortypes. This adds to a strong clinical rationale supporting itstherapeutic promise, and we look forward to initiating Phase 1clinical trials with the non-radiolabeled form of this drug candidatein malignant glioma. This designation is part of TransMolecular'sstrategy to advance this program so that patients with thispoor-prognosis disease may eventually have a new treatment optionavailable to them." The FDA grants Orphan Drug Designation to promising products thataddress rare diseases affecting fewer than 200,000 Americans annually.If non-radiolabeled TM601 receives FDA approval for malignant glioma,this designation will entitle TransMolecular to exclusive marketingrights for the compound for the treatment of malignant glioma forseven years following the NDA approval. Orphan Drug Designationprovides financial and regulatory incentives for companies pursuingless common diseases. About TM601