FDA Grants Orphan Drug Designation to PVSRIPO for Treatment of Advanced Melanoma

The novel viral immunotherapy PVSRIPO is designed to stimulate a patient’s innate and adaptive immune system to promote an antitumor response and establish long-term immunologic memory to help keep their cancer at bay.

The FDA has granted orphan drug designation to the novel viral immunotherapy PVSRIPO for the treatment of patients with advanced stage IIB through IV melanoma, according to Istari Oncology, the developer of the agent.1

PVSRIPO is designed to stimulate a patient’s innate and adaptive immune system to promote an anti-tumor response and establish long-term immunologic memory to help prevent the cancer’s return.

Istari is currently recruiting for the open-label, randomized, phase 2 LUMINOS-102 (NCT04577807) trial of PVSRIPO in patients with PD-1 refractory melanoma. The study plans to characterize the safety, tolerability, and initial efficacy of PVSRIPO intratumoral injection alone and in combination with a PD-1 inhibitor.

The company plans to dose the first patient this quarter.

“We are happy to kick off the new year with the announcement that our request for an orphan drug designation has been granted to PVSRIPO for the treatment of advanced melanoma,” Matt Stober, president and chief executive officer at Istari Oncology, said in a press release. “This is just one of many milestones to come in 2021 as we continue to drive the clinical development of PVSRIPO across multiple indications.”

LUMINOS-102 will follow the successful phase 1 monotherapy study (NCT03712358) of PVSRIPO in anti–-PD-1 refractory advanced melanoma, results of which were previously presented at the 2020 Society for Immunotherapy of Cancer (SITC) Annual Meeting.2

In this study, PVSRIPO injections were well tolerated with no serious adverse events (AEs) or dose-limiting toxicities reported. Moreover, all treatment-emergent AEs were grade 1 or 2, with grade 1 pruritus being the most common at 58%. All but 2 PVSRIPO-related, treatment-emergent AEs were localized to the injected or adjacent lesions (1 event each of grade 1 hot flash and grade 1 fatigue).

In spite of the limited number of PVSRIPO treatments relative to the overall lesion burden (67% patients >5 lesions), 4 of 12 patients (33%) achieved an objective response per independent radiologic review committee, including 4 of 6 (66%) who received the maximum administered dose of 3 injections. Pathologic complete response was also reported in 2 of 4 (50%) patients with in-transit disease.

Following treatment with PVSRIPO therapy, 10 of 12 patients (83%) again received immune checkpoint inhibitor (ICI)–-based therapy and 6 of 12 patients (50%) remained progression-free at the data cutoff.

“We are encouraged by the data from our phase 1 trial presented at last year’s Society for Immunotherapy of Cancer (SITC) 2020 Annual Meeting” Garrett Nichols, MD, MS, chief medical officer at Istari Oncology, said in the release. “We plan to build upon that success with LUMINOS-102. As those data and the data from our other solid tumor trials emerge, we will continue to work closely with the FDA toward the goal of bringing PVSRIPO to market.”


1. Istari Oncology announces FDA grants orphan drug designation for PVSRIPO for the treatment of advanced melanoma. News release. Durham, NC. Published January 10, 2021. Accessed January 19, 2021. https://istarioncology.com/press-release-fda-grants-orphan-drug-designation-for-pvsripo-for-the-treatment-of-advanced-melanoma/

2. Beasley G, Farrow N, Landa K, et al. A phase I trial of intratumoral PVSRIPO in patients with unresectable treatment refractory melanoma. J Immunotherapy of Cancer 2020;8(suppl 2):A185. Abstract 302. doi:10.1136/jitc-2020-SITC2020.0302

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