On September 23, 2024 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer therapies using its proprietary RADR AI and machine learning ("ML") platform with multiple clinical-stage drug programs, reported that the company has been granted three rare pediatric disease designations (RPDD) by the FDA (Press release, Lantern Pharma, SEP 23, 2024, View Source [SID1234646824]). Lantern was granted these rare pediatric disease designations in: malignant rhabdoid tumors (MRT), rhabdomyosarcoma (RMS), and hepatoblastoma.
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"At Lantern, we’re harnessing AI and data-driven approaches to revolutionize cancer drug development, aiming to dramatically reduce costs, accelerate timelines, and enhance precision in bringing new therapies to patients," stated Panna Sharma, CEO and President of Lantern Pharma. "Our recent breakthrough in identifying three additional, high-potential indications for LP-184 in pediatric cancers exemplifies this progress. We believe that ‘AI for good’ should address both blockbuster opportunities as well as rare, often overlooked pediatric cases. The FDA’s Rare Pediatric Disease designation for these three potential programs is a testament to this commitment. We’re acutely aware that patients and their families are relying on innovators like us to speed up therapy development. These designations mark a crucial step forward in advancing our expanding portfolio of pediatric programs targeting these devastating and rare cancers. It reinforces our dedication to transforming hope into tangible solutions for those who need them most."
Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions primarily affecting children under 18, with fewer than 200,000 cases in the U.S. A key benefit of obtaining a RPDD is the potential to receive a priority review voucher following FDA approval of a product with RPDD if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. These vouchers, often called "golden tickets," can significantly expedite the review process for future NDAs or biologic license applications, reducing the standard review time from about ten months to six. Sponsors can either use these vouchers themselves or sell them to other companies. These vouchers, in the recent past, have commanded sales prices of approximately $100 million USD.
Lantern’s investigational drug candidate, LP-184, has shown preclinical activity in a wide range of solid tumors, garnering it multiple orphan and rare pediatric designations. LP-184 is currently in a multi-center Phase 1A clinical trial that is expected to enroll approximately 50 to 60 patients across a wide range of solid tumors. Based on the results and findings from this clinical trial and other collaborative studies, Lantern will plan and potentially develop future clinical trials for specific pediatric patients in ATRT, MRT, RMS and Hepatoblastoma.
About MRT – Malignant Rhabdoid Tumors
Malignant rhabdoid tumors are rare childhood cancers that typically affect the kidneys and soft tissues, sometimes occurring in the brain as atypical teratoid rhabdoid tumors (ATRT). The kidney and soft tissue variant, known as malignant rhabdoid tumor (MRT), is most common in infants and toddlers, with an average diagnosis age of 15 months. In the United States, only 35 to 50 new cases of MRT are diagnosed annually. These tumors can spread to other parts of the body. While the exact cause is unknown, research has linked a mutation in the SMARCB1 gene to nearly all rhabdoid tumors. This mutation can sometimes occur in a patient’s normal cells, increasing their risk of developing multiple tumors. Often, the first sign of MRT is an abdominal lump or mass, with some children experiencing urination difficulties or blood in the urine.
About RMS – Rhabdomyosarcoma
Rhabdomyosarcoma is a rare cancerous tumor that develops in the body’s soft tissues, which connect, support, and surround organs and other structures. It originates from rhabdomyoblast cells, which form early in embryonic development, making this cancer more prevalent in children than adults. The tumor commonly appears in the head, neck, bladder, vagina, arms, legs, and trunk, but can also occur in areas with minimal skeletal muscle, such as the prostate, middle ear, or bile duct system. Despite being the most common childhood soft-tissue sarcoma, rhabdomyosarcoma affects only about 250-300 children annually in the United States. There are two primary types of this cancer: embryonal rhabdomyosarcoma (ERMS), which is more common and typically affects children under six, and alveolar rhabdomyosarcoma (ARMS), which accounts for approximately 20 percent of cases and is more frequently found in older children.
About Hepatoblastoma
Hepatoblastomas are the most common primary malignant liver tumors in pediatric patients, typically occurring within the first two years of life. These tumors are classified into two histologic types: epithelial and mixed. While most hepatoblastomas are sporadic, about one-third of cases are associated with genetic conditions such as Beckwith-Weidemann syndrome, familial adenomatous polyposis (FAP), Edward syndrome (trisomy 18), nephroblastoma, and Down syndrome. Infants with low birth weight are at a higher risk of developing hepatoblastoma, and there is evidence linking the tumor to preeclampsia and parental tobacco smoking before and during pregnancy. The most common genetic mutation in hepatoblastoma involves the Wnt signaling pathway, leading to the accumulation of beta-catenin, particularly in sporadic cases. In more aggressive cases, activation of TERT (human telomerase reverse transcriptase) and MYC signaling has been observed. Hepatoblastoma is a rare tumor, accounting for approximately 1% of all pediatric tumors in North America and Europe. However, its incidence is slowly increasing globally, with a slight predominance in males.