LP-184 Earns 3 FDA Rare Pediatric Disease Designations in Solid Tumors

3D illustration of a tumor sample under a microscope: © catalin - stock.adobe.com

• The FDA has granted 3 rare pediatric disease designations (RPDD) to the investigational agent LP-184 in malignant rhabdoid tumors, rhabdomyosarcoma, and hepatoblastoma.
• LP-184 previously earned a RPDD in atypical teratoid rhabdoid tumors.
• Earning RPDD means a drug sponsor can be eligible for a priority review voucher under the current program.

LP-184 has earned 3 RPDDs in ultra-rare tumors including rhabdoid tumors, rhabdomyosarcoma, and hepatoblastoma.¹

The FDA defines rare pediatric diseases as serious or life-threatening conditions primarily affecting patients under the age of 18 with fewer than 200,000 incidences in the US. LP-184 was previously granted RPDD in atypical teratoid rhabdoid tumors, bringing the current RPDD count to 4.

With RPDD, Lantern Pharma, the sponsor, may qualify for a priority review voucher that can be redeemed to receive priority review for a different product if the initial agent is approved. The sponsor may also transfer or sell the voucher to another sponsor. However, the FDA is beginning to sunset this program starting September 30, 2024.²

“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,” said Panna Sharma, chief executive officer and president of Lantern Pharma, in a press release.¹

LP-184 is an investigational acylfulvene-derived agent that is activated by the oxidoreductase prostaglandin reductase 1 that alkylates at N3-adenine. This is not reported to be repaired by MGMT. Preclinical models showed that LP-184 was a promising agent in glioblastoma.³

A phase 1 dose-escalation study (NCT05933265) is investigating the agent in solid tumors and currently recruiting patients across 9 sites in the US.⁴ The study’s primary end point is incidence and severity of adverse events, and pharmacokinetics are being evaluated as a secondary end point.

LP-184 will be administered on days 1 and 8 of a 21-day cycle for at least 2 cycles. The planned dose escalation will consist of a minimum of 3 patient cohorts. After the maximum tolerated dose (MTD) is identified, more patients will be enrolled at 2 dose levels, including the MTD, to determine the recommended phase 2 dose.

REFERENCES:

1. Lantern Pharma announces three U.S. FDA rare pediatric disease designations granted to LP-184 in multiple ultra rare children’s cancers. News release. Lantern Pharma. September 23, 2024. Accessed September 24, 2024. https://tinyurl.com/5bxvfzxd

2. Rare pediatric disease designation and priority review voucher programs. FDA. Updated May 28, 2024. Accessed September 24, 2024. https://tinyurl.com/yj6dxma7

3. Lal B, Kulkarni A, McDermott J, et al. Preclinical efficacy of LP-184, a tumor site activated synthetic lethal therapeutic, in glioblastoma. Clin Cancer Res. 2023;29(20):4209-4218. doi:10.1158/1078-0432.CCR-23-0673

4. Study of LP-184 in patients with advanced solid tumors. ClinicalTrials.gov. Updated May 16, 2024. Accessed September 24, 2024. https://clinicaltrials.gov/study/NCT05933265