Blueprint Medicines has a potential blockbuster on its hands with Ayvakit, but it has to tackle the issues of COVID, patient identification and education to try and boost awareness of the diseases it targets.
It’s been a pivotal time for the company, going from a pure clinical-stage biotech player in 2019 to a commercial biopharma in early 2020 with a green light to treat certain patients with certain gastrointestinal stromal tumors (GIST), the first of its kind. The company then followed the approval up last summer with a new regulatory nod in a rare disorder and its subtypes.
This includes advanced systemic mastocytosis, which can dramatically cut life expectancy to six months to a few years, and aggressive systemic mastocytosis, a rare disorder that results in too many mast cells building up in your body, as well as systemic mastocytosis with an associated hematological neoplasm and mast cell leukemia.
Blueprint has not had everything go its way. It ditched a plan to explore the drug in other GIST indications after a phase 3 fail in fourth-line KIT-driven disease led to an FDA complete response letter.
But it’s also hoping for a positive readout later this year from the Pioneer study, focused on nonadvanced systemic mastocytosis, which could also lead to another new approval down the line, and access to more patients.
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And just this week it was all change at the top, when Blueprint announced a new CEO in the form of Kate Haviland, and also promoted Christy Rossi to chief operating officer (taking over Haviland’s former role), coming herself from being chief commercial officer.
Speaking to Fierce Pharma Marketing, Rossi said her focus remains firmly on the launch of Ayvakit, which could reach blockbuster status in the coming years. It made $17.3 million in the third-quarter of last year, the first full quarter since its approval in SM but is hoping to ramp that up in 2022.
Educate and test
The problem with a drug aimed at rare disease is not just about getting it into the hands of the right patients but actually finding those patients. To help combat this, the company recently set up a new blood testing deal with LabCorp for KIT D816V, a noninvasive test to find SM patients thus speeding up diagnosis and, Blueprint hopes, access to its drug. The company offers the test to eligible patients for free.
It’s early days as the test only rolled out at the end of 2021, but Rossi says “there has been a nice increase in testing for KIT D1816V” and while the pandemic impact is making it difficult to see the whole picture, she still expects to see an increase in diagnosis for SM as a result.
There’s around 32,000 patients with some form of SM in the U.S. (with the advanced form taking up around 10% of that number), but “only one-third are currently accurately diagnosed,” she explained, with the remaining two-thirds “either misdiagnosed or just not diagnosed at all.” Getting those two-thirds picked up will be crucial to boost sales for the med.
While GIST was its first approval, the second in SM is a major focus. “Systemic mastocytosis is the most significant opportunity to impact the most patients with Ayvakit,” Rossi explained. For this, it’s running a new educational campaign, Target SM, which hears from a group of doctors about their experience in working with SM patients. Rossi said that the hope is to expand this campaign in the future, should the Pioneer study work out and it gains that new indication.
“The idea [behind Target SM] is to simply help providers, using specialists, to answer the question: What is this disease? How do I understand it, how do I diagnose it? There’s not much branding around Ayvakit for this, we just want people to understand the disease itself.
“That’s important for advanced SM, and we’re going to continue to build on that as we head toward possibly treating nonadvanced SM. Here, disease education becomes even more critical because patients with that form of the disease, which is more chronic, tend to spend many years in what we call a ‘diagnostic odyssey’ where they know they have something, but they can’t get it diagnosed.”
Another pillar to marketing Ayvakit in its approved advanced SM indication is about getting its branding out to healthcare professionals and patients, which is being done in the normal way out in the field as well as through digital peer-to-peer programs to help educate on the therapy itself.
“But we really need to continue to educate,” Rossi said. There are SM patients in academic centers, where many have ended up in the past, but also many in the community that they still need to find. “This is a challenge as it’s rare and it’s complicated, so that’s our focus in 2022.”
