Arcus’ pancreatic cancer drug quemliclustat receives FDA orphan status

HAYWARD, Calif. - Arcus Biosciences, Inc. (NYSE:RCUS), a biotechnology company with a strong balance sheet showing more cash than debt according to InvestingPro data, announced Thursday that its investigational CD73 inhibitor quemliclustat has received orphan drug designation from the U.S. Food and Drug Administration for pancreatic cancer treatment.

The designation provides incentives including tax credits for clinical trials, user fee exemptions, and potential seven-year market exclusivity upon approval. It is granted to treatments for diseases affecting fewer than 200,000 people in the United States.

Quemliclustat is currently being evaluated in the Phase 3 PRISM-1 study, which is testing the drug in combination with gemcitabine/nab-paclitaxel chemotherapy against chemotherapy alone for first-line treatment of metastatic pancreatic ductal adenocarcinoma. The company expects to complete enrollment of approximately 610 patients by the end of 2025.

"The orphan drug designation indicates the importance of developing new treatment options for rare diseases like pancreatic cancer, which has the highest mortality rate of all major cancers," said Richard Markus, Chief Medical Officer at Arcus Biosciences.

Earlier data from the Phase 1 ARC-8 study presented in January 2024 showed median overall survival of 15.7 months in patients treated with quemliclustat-based regimens, which the company reports exceeded historical benchmarks for chemotherapy alone.

Pancreatic cancer will affect approximately 67,440 Americans in 2025 according to the American Cancer Society. The disease has seen limited treatment advancements in over three decades, with chemotherapy remaining the standard of care. Metastatic pancreatic cancer has a five-year survival rate of only 3%.

Quemliclustat is being co-developed with Gilead Sciences and works by blocking the production of adenosine, which can suppress immune responses against tumors.

The company noted that quemliclustat is still investigational and has not received regulatory approval, with its safety and efficacy not yet established.

This article is based on information from a company press release.

In other recent news, Arcus Biosciences reported its Q1 2025 earnings, revealing a larger-than-expected loss with earnings per share at -1.14, missing the forecast of -1.02. Revenue also fell short, reaching $28 million against a projected $38.61 million. Despite the earnings miss, Arcus Biosciences maintains a strong cash position of $1 billion, which supports its ongoing research and development efforts. The company is focusing on strategic collaborations, notably with AstraZeneca, to advance its pipeline in the renal cell carcinoma (RCC) market.

Additionally, H.C. Wainwright reiterated a Buy rating for Arcus Biosciences, maintaining a price target of $24.00. This decision comes after the presentation of promising initial combination data for casdatifan and cabozantinib in treating clear cell renal cell carcinoma at the American Society of Clinical Oncology meeting. The trial results showed a confirmed overall response rate of 46%, with one complete response and ten partial responses among 24 evaluated patients. These developments highlight the potential of Arcus Biosciences’ treatment combinations, which demonstrated synergy in response rates without added toxicity.

Arcus Biosciences continues to face high research and development expenses, which are expected to peak in 2025. Nonetheless, the company remains committed to leveraging its substantial cash reserves and strategic partnerships to drive long-term growth. The company also maintains its full-year 2025 revenue guidance between $75 million and $90 million, focusing on advancing its product pipeline.

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