How often does Valius result in a prescribed therapeutic?

When we meet with families interested in Valius, the first question they often ask is how many of those ultimately did something differently directly as a result of Valius.

Since we've now worked with over 100 patients, we decided to quantify what we've seen thus far.

In about 30% of cases, we’re proud to have uncovered what we consider to be an actionable finding. Typically, this means that we discovered very high expression of a target of an approved therapy—or an investigational therapy with compelling earlyefficacy and safety data—which the patient’s care team didn’t know about beforehand. In the other 70% of cases, our workup has yielded more "incremental" information - perhaps confirming what was already known.

For the 30% of cases in which we've found something novel, about a third of them (e.g., 10% of the total), have subsequently gotten access to a therapy we highlighted.

Therapeutic access is always a challenge. Clinical trials have very strict inclusion and exclusion criteria. Many pharma companies are hesitant to do "expanded access", and it's a lot of paperwork for doctors and institutions.

That said, we believe that more aggressive prescribing of "biologically matched, but off-label" therapies is the way of the future, and we're committed to helping our patients now.

As we continue to scale to thousands of patients and beyond, the next step is to track response to therapy, not just access.

It's still early, but we're overjoyed that a handful of our patients have already registered a partial or complete response to a therapy suggested by our workup. Given the number of our patients who recently started new therapies, we’re hopeful this number will rise in the near future.

By focusing on patients with rare and aggressive cancers, we’ve deliberately chosen to work on some of the most challenging cases out there.

We’re immensely proud of our track record so far. Nontheless, we recognize that there’s so much more to be done, and we’re excited to do it. Along the way, we’ll continue to publish articles like this one, breaking down our results and tracking successes over time.