Vertex Pharmaceuticals (NASDAQ: VRTX) reported positive results in October from an early stage clinical study evaluating VX-880 in treating type 1 diabetes. The New York Times recently published an article about these results, suggesting that Vertex could even have a cure for type 1 diabetes.
In this Motley Fool Live video recorded on Dec. 1, 2021, Motley Fool contributors Keith Speights and Brian Orelli answer a viewer's question about Vertex's experimental diabetes therapy.
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Keith Speights: All right, Brian so in the last few minutes here, let's turn to catch up on some questions that we had from some folks on Monday.
One viewer asked earlier this week about that New York Times article that ran over the weekend about Vertex Pharmaceuticals. And the headline of the article was "A Cure for Type 1 Diabetes? For One Man, It Seems to Have Worked." And this article focused on positive results for one participant in Vertex's phase 1 study of this experimental type 1 diabetes cell therapy.
First of all, Brian, what do you think about Vertex's prospects with this program? Then second, could Vertex actually have a cure for Type 1 diabetes on the way as this New York Times article suggested?
Brian Orelli: Yeah. This is somewhat old news. We talked about it on the show when Vertex announced the results from that first patient in the study back in October.
The treatment -- what's called the VX-880 -- is a pancreatic islet cell replacement therapy derived from stem cells so islet cells regulate the insulin levels and then they're in response to blood sugar levels and they're not functioning in patients with Type 1 diabetes so the therapy is putting islet cells back into the patient.
There are a couple of caveats with calling this a cure. First, it was only 90 day data so the patient reached HbA1c level of 7.2, they had started at 8.6. Seven is usually a fairly good number to hit, that's HbA1c measures long-term blood sugar levels so seven is a good number to hit, so 7.2 is OK but it's not necessarily the best.
Then the patient was still taking some insulin but they reduced the insulin level required by 91 percent so that's pretty good. We'll have to see what happens beyond 90 days I think to really be able to call it a cure.
The second thing is in my graduate study advisor was fond of saying that one isn't a number. So we have no idea whether the therapy is 100 percent effective so is it going to work for everybody or is it just one in 100 effectiveness and Vertex just happened to get lucky with this one patient. We obviously need more data from more patients to know what the actual cure rate is.
And then third, VX-880 requires a patient to take immunosuppressive drugs so that they don't reject those islet cells. In the case of this patient, that's an OK trade-off because he had five severe potentially life-threatening episodes of low blood sugar level in the year prior to treatment at the risk of dying from low blood sugar levels, being at the risk of getting an infection, might kill you because you're on immunosuppressants is acceptable trade-off. But this isn't going to be a treatment for all patients with Type 1 diabetes because if they have their insulin regimen dialed in taking on the added risk of infection probably isn't worth it for those patients.
Vertex is developing an encapsulated islet cell program so those cell will be protected from the immune system and therefore, the patients wouldn't require immunosuppression. Vertex's planning on filing an IND, Investigational New Drug Application to start clinical trials for that program next year and so I think that one probably is really what investors should be watching out for. I am a shareholder in Vertex so I'm looking forward to, I'm looking at that clinical trial once it gets started.
Speights: I'm a Vertex shareholder as well, and I also look forward to that clinical study.
Brian Orelli, PhD owns Vertex Pharmaceuticals. Keith Speights owns Vertex Pharmaceuticals. The Motley Fool owns and recommends Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
