Exelixis, Inc. (NASDAQ:EXEL) was an early mover in the biotechnology space this week, with the company jumping on the release of some news related to two of its lead development programs.
The first bit of news relates to an asset called cabozantinib. This one is an oncology drug and the company is trying to demonstrate that it can be an effective treatment for patients with advanced hepatocellular carcinoma (HCC), which is the most common type of primary liver cancer and occurs most often in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection.
In patients with an advanced form of the disease, treatment options are incredibly limited, with palliative care generally the only real possibility once surgical resection, chemotherapy and radiotherapy have all failed (or, if not failed, worked to a certain degree before reaching a stage of limited efficacy).
The drug is actually already approved in a couple of other oncology indications, including as a second line therapy for renal cell carcinoma (more on this shortly). It is a tyrosine kinase inhibitor, meaning it stops certain processes working that play a role in cell proliferation and replication – two features of cancer cells that make the disease so aggressive, and especially so in these sorts of kidney and liver-type cancers.
The news released on Monday this week specifically relates to a phase 3 trial of cabozantinib in the advanced HCC population and the data collected on the back of said trial. The randomized, global phase III CELESTIAL trial examined cabozantinib versus placebo in patients with advanced HCC who received up to 2 prior systemic cancer therapies for HCC and had adequate liver function, with total enrollment coming in at 773 patients.
As per the release, the trial met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant improvement in median OS compared to placebo in patients with advanced HCC.
Additionally, and as reported concurrent to the latest data, an independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis.
It wasn’t all about HCC this week, however.
Exelixis also has a supplemental New Drug Application with the FDA in the US related to the same drug, cabozantinib, but this time in a renal cell carcinoma indication. As mentioned above, it’s actually already approved as a treatment for patients with this type of the disease but the approval covers the drug as a second line therapy only, meaning patients have to have tried and failed another type of treatment before they are allowed to advance to cabozantinib therapy.
The supplemental New Drug Application that is with the agency right now is an attempt to expand the target population to include patients who have not yet received any form of treatment for the renal cell carcinoma, making the drug a first-line treatment in this indication, if approved.
As per the latest news, the FDA has now set a PDUFA date for the application and, not only that, but has decided to consider the application under what’s called priority review. A priority review designation brings with it a couple of benefits, but the primary is that the agency will look to come to a decision as to whether it wants to approve the drug in question or not within a six-month period. The standard review period for these sorts of applications is 10 months, meaning a priority review designation cuts the time the company must wait for a decision by 40%.
At this end of the biotechnology space, cash burn is a real risk factor, so the ability to have a chance at generating revenues from a fresh target population sooner rather than later is a major risk dissipation.
The PDUFA date that the FDA has set for cabozantinib in this first line renal cell carcinoma indication comes in on February 15, 2018, which actually only leaves four months until Exelixis gets its decision.
Both of these bits of news have translated to some strong upside momentum for the company, and Exelixis will open the session on Tuesday at a close to 20% premium to its pricing at the start of the week.