An innovative new drug that doubled survival for pancreatic cancer patients has emerged. It is Rasonque (ingredient name daraxonrasib), developed by Revolution Medicines.
Revolution Medicines said on the 26th (local time) that the U.S. Food and Drug Administration (FDA) approved Rasonque as a treatment for patients with metastatic pancreatic ductal adenocarcinoma who had received prior therapy. The FDA granted marketing authorization more than six months ahead of its original review deadline.
Rasonque is an oral therapy (a pill) that targets RAS proteins, which have been difficult to attack with drugs.
In a phase 3 clinical trial, it doubled overall survival (OS). At the American Society of Clinical Oncology (ASCO) in May, results showing progression-free survival (PFS) also doubled were presented, drawing a standing ovation from the audience at the time.
Pancreatic cancer is often detected late because early symptoms are not clear, and it frequently metastasizes to other organs, making it one of the most intractable cancers. According to the National Cancer Information Center, the 5-year relative survival rate (adjusted for non-cancer mortality) for pancreatic cancer in Korea is 17.0%, dropping to 2.4% in cases with distant metastasis. According to the National Cancer Institute (NCI), the 5-year relative survival rate for pancreatic cancer in the United States is in the 10% range, and around 3% when the cancer has spread to other organs.
The drug is drawing attention because it directly targets RAS proteins, which play an important role in pancreatic cancer.
RAS is a protein that regulates cell growth and division. But when RAS is abnormally activated in cancer cells, it sends signals that keep cells proliferating, promoting cancer growth and progression. In pancreatic cancer in particular, mutations in KRAS, part of the RAS family, are very common, making it a key therapeutic target.
The problem is that due to the structural characteristics of the protein, it has been difficult to block RAS function using conventional approaches.
The company applied a different drug design approach to solve this.
Rasonque works by inducing binding between the RAS protein and a protein called cyclophilin A, blocking RAS function. When RAS binds with cyclophilin A, RAS can no longer properly transmit the signals that promote cancer cell growth and division. Simply put, it grabs RAS, which sends growth signals to cancer cells, by tethering it to another protein so it cannot function.
The company said it will expand the scope of Rasonque while also accelerating development of follow-on RAS inhibitors. According to the company, another RAS inhibitor candidate, zoldonrasib, entered a phase 3 clinical trial for pancreatic cancer in June. It is also developing RAS inhibitor candidates targeting several other tumor types.
Allan Sandler, chief development officer (CDO) at Revolution Medicines, said at ASCO, "We are very confident in our approach," adding, "We are also confident that we are ahead of many other corporations at this point."
In the United States, a 30-day supply of Rasonque costs $39,800 (about 55 million won). Annualized, it amounts to $477,600 (about 660 million won). On the same day, the Wall Street Journal (WSJ) reported, citing a market research forecast, that if Rasonque proves effective in other cancers, its annual sales could exceed $20 billion. Rasonque is currently approved only in the United States.
Meanwhile, the company's share price on the Nasdaq rose from $79.02 on Jan. 2 to $215.44 on Aug. 26, surging about 173% this year.