On January 24, 2022 Lantern Pharma (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform the cost, pace, and timeline of oncology drug discovery and development reported that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation and Orphan Drug Designation for the company’s drug candidate LP-184 for the treatment of pediatric patients with ATRT (Press release, Lantern Pharma, JAN 24, 2022, View Source [SID1234606724]). LP-184 is being pursued as a potential new therapy across a range of genetically defined solid tumors, including pancreatic cancer, GBM (Glioblastoma Multiforme) and ATRT (Atypical Teratoid Rhabdoid Tumor).

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"Historical approaches to treating pediatric ATRT such as surgery, radiation, and chemotherapy have had largely unfavorable long term outcomes for children, and new options are urgently needed," said Kishor Bhatia, Ph.D., chief scientific officer of Lantern Pharma. "The gene SMARCB1 was included among several genes whose expression negatively correlated with LP-184 sensitivity in tumors. This in silico correlation was convincingly confirmed by in vitro and in vivo assessments of LP-184 in ATRT. The highest potency of LP-184 in-vivo has been seen in ATRT xenografts." Dr. Bhatia continued, "Based on both the in-silico and in-vivo observations, LP-184 has the potential to become a critical part of the armamentarium of approved treatment options specifically for these patients. Receiving Rare Pediatric Disease Designation from the FDA underscores the critical value of our growing focus on pediatric oncology indications at Lantern and represents another significant milestone for the LP-184 program."

The National Cancer Institute (NCI) classifies ATRT as Grade IV tumors, meaning they are malignant (cancerous), aggressive and fast-growing. ATRTs are very aggressive childhood malignancies of the central nervous system. The underlying genetic cause are inactivating bi-allelic mutations in SMARCB1 (also called INI1) or in SMARCA4. Nearly 90 percent of pediatric ATRTs are caused by changes in the gene known as SMARCB1.

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

LP-184 is being developed for multiple targeted oncology indications. Lantern Pharma intends to further advance LP-184 as a new, potent treatment option in genetically defined subsets of patient populations in areas of high unmet clinical need, including pancreatic cancers, GBM, and other cancers that are DNA damage repair deficient. The U.S. Food and Drug Administration (FDA) has previously granted LP-184 Orphan Drug Designation for the treatment of pancreatic cancer, and for the treatment of malignant glioma, including GBM.