Rare Disease Report

FDA Grants Orphan Drug Designation to RP6530 for Treatment of CTCL

APRIL 09, 2018
Krista Rossi
This morning, Rhizen Pharmaceuticals SA announced that its product Tenalisib (RP6530) was granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of cutaneous T-cell lymphoma (CTCL).

Cutaneous T-cell lymphoma, one of the most common forms of T-cell lymphoma, is a general cancer subtype that affects the skin. Typically, more men than women are afflicted by CTLC and the disease most commonly presents during an individual’s fourth, fifth or sixth decade of life. While some patients only experience skin symptoms, it’s possible that others might experience rapidly progressing cancer that spreads to the lymph nodes and other internal organs.2

RP6530 is an orally active and highly selective dual PI3K delta/gamma inhibitor with efficient translations of activity through enzyme, cell, and whole blood-based studies. RP6530 plays a significant role in the modulation of the tumor microenvironment at clinically achievable concentrations. Additionally, it is able to inhibit the growth of immortalized cancerous cell lines and primary patient leukemic/lymphoma cells.1

Preclinical studies have shown RP6530 reprograms macrophages from an immunosuppressive M2-like phenotype (pro-tumor) to an inflammatory M1-like state (anti-tumor). This reaction can potentially enhance the activity of checkpoint inhibitors or help them overcome resistance to these drugs.

] Phase 1 and Phase 2 studies are not yet recruiting, and the anticipated start date is in April 2018. The last data collection is expected in August 2019. The trials are expected to enroll 57 adult participants, each of whom will receive Tenalisib in escalating doses orally twice a day (BID) and pembrolizumab as a fixed dose intravenously (IV).3

The primary outcomes are to assess the safety and tolerability of Tenalisib as well as to establish the maximum tolerated dose (MTD) for Tenalisib in combination with Pembrolizumab in patients with cHL Secondary outcomes measures will include the maximum observed plasma concentration; the overall response rate (ORR) with Tenalisib and Pembrolizumab combination; the duration of response (DoR) with Tenalisib and Pembrolizumab combination; the progression free survival (PFS) with Tenalisib and Pembrolizumab combination; the conversion rate with Tenalisib and Pembrolizumab combination; and the proportion of patients achieving complete response (CR) and partial response (PR) with Tenalisib and Pembrolizumab combination.

“We are pleased to receive US FDA orphan-drug designations for the active moiety of Tenalisib (RP6530) for the treatment of peripheral and cutaneous T-cell Lymphoma (PTCL and CTCL),” said Swaroop Vakkalanka, PhD, Founder and President of Rhizen Pharmaceuticals SA, “and we look forward to advancing the drug into further development for treatment of T-cell lymphoma.”

Tenalisib has also previously been granted both Fast Track and orphan drug designations of peripheral T-cell lymphoma (PTCL).

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  1. Rhizen Pharmaceuticals S.A. receives FDA orphan-drug designation for Tenalisib (RP6530) for treatment of cutaneous T-cell lymphoma (CTCL). GlobeNewswire. 9, Apr. 2018.
  2. About Lymphoma. Lymphoma.org.https://www.lymphoma.org/aboutlymphoma/nhl/ctcl/. Accessed April 9, 2018.
  3. Safety and Efficacy Study of Tenalisib (RP6530) in Combination With Pembrolizumab in Relapsed or Refractory cHL. National Institute of Health. 21, Mar. 2018.

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