NEW YORK – With $110 million in new funding announced Thursday and growing data showing the impact of its RNA-based rheumatoid arthritis test on patient outcomes, Scipher Medicine is gearing up to make a case for broader adoption and reimbursement for the first of a planned menu of therapy selection tools for immune-mediated diseases.
The Boston-based company announced this morning that it had raised $110 million in a new financing round led by Cowen Healthcare Investments. The influx brings its total financing to $227 million to date and marks an inflection point for the firm as it reaches the one-year mark since launching PrismRA as a laboratory-developed test.
Scipher's approach to test development relies on a method that the company created to characterize protein interaction networks in order to identify putative predictive gene expression signatures. The first result from that process is the PrismRA test, which is intended to identify RA patients who are unlikely to respond to anti-tumor necrosis factor therapy and could be better served by one of the large number of other drugs on the market.
The firm published its first significant validation data for the assay in 2020, showing that the test could identify patients with a sub-adequate response to anti-TNF treatment with a positive predictive value of 89.7 percent and specificity of 86.8 percent.
Last December, investigators added to this data with a new, interim readout from Scipher's prospective observational study of the assay, published in Expert Review of Molecular Diagnostics. The new data, from a real-world testing cohort, confirmed that PrismRA identifies tumor necrosis factor inhibitor, or TNFi, non-responders and added new evidence that those patients with a molecular signature of non-response can achieve clinically meaningful responses to therapies with alternative mechanisms of action.
Sam Asgarian, Scipher's chief medical officer, said this week that because of the positive results the company has been seeing, it has been pushing hard on commercialization of the test.
Testing within the ongoing prospective observational study is fully sponsored by the company, but Scipher is also "out there, getting physician orders."
That said, he added, "It's still very early, so we're making sure that patients aren't billed, and that we really are working to understand with insurers how the coverage policies are shaping up and how we can help them shape them."
What that means in practice is that the company has what Asgarian said is "about 20 private insurers," with whom it has contracts to provide coverage for their members, both for rheumatoid arthritis diagnosis and therapeutic selections within their formulary.
According to Asgarian, Scipher is also making "great inroads with Medicare," hoping to have a local coverage determination sometime this year.
The newest data on PrismRA comes from an interim analysis of 377 patients tested within a network of more than 70 private and academic rheumatology practices participating in the firm's prospective observational study.
"As much as possible … we wanted it to be an all-comers type of study. So, the inclusion criteria was very broad [in terms of] … genders, geographies across the board. And then we would enroll them accordingly … until we got to the point right now where we had enough for an interim analysis," Asgarian said.
In the study, investigators analyzed PrismRA test results alongside documentation of patients' subsequent treatment and outcomes, reporting that about 74 percent of doctors in the cohort made a treatment decision based on PrismRA results. Among their patients, 40 percent had responses, meeting American College of Rheumatology 50 percent criteria by week 24, the study's primary endpoint.
About 45 percent were prescribed a TNFi, 22 percent a T cell co-stimulation inhibitor, 22 percent a JAK inhibitor, 8 percent an IL-6 R inhibitor, and 2 percent a B cell modulator.
Across the board, patients with a PrismRA result indicating TNFi non-response who received one of the non-TNFi therapies did significantly better than those who were treated with a TNFi, with a 35 percent response rate versus a 10 percent response rate, respectively.
"This indicates that predicted non-responders to TNFi therapies are not non-responders to other classes of RA targeted therapy," the study authors wrote.
For the 17 percent of doctors who didn't prescribe based on patients' PrismRA results, survey responses indicated that more than half did so because of insurance-related restrictions.
According to Asgarian, the survey responses portrayed instances where doctors would have used the test to inform treatment but were hobbled by either formulary constraints or certain authorization requirements requiring a patient try and fail TNFi treatment before moving on to another drug.
"The reason for that, for the most part, is because in this world that exists today, without PrismRA, trial and error is the status quo, so insurers have put on a lot of guardrails so that the physicians can adhere to that trial-and-error approach," Asgarian said.
"Our view is that once you have objective data like we get from our test, you can then be able to use that to help the insurers build much more dynamic models around accessibility and less constraints around what's within the formularies that physicians can choose from and when."
In other areas where personalized medicine and precision genetics walked this road, such a shift has often taken a long time, but Asgarian said Scipher's goal is to hopefully "short circuit that" through close work with private payors and CMS.
And although the company's observational data is looking positive, randomized controlled trials remain the gold standard for establishing clinical validity and utility.
"We are not averse to that," Asgarian said, adding that the company is already working to add a control group to its planned clinical utility study.
"We're working through how we can do that as fast as possible so that we're not spending years preventing … patients having access to this," he added, highlighting the possibility — in light of the US Food and Drug Administration's softening on the use of real-world data — of using EMRs to build out a control set.
"That's what we really aspire to do this year," he said.
Apart from supporting the firm in its efforts to gain reimbursement and adoption for PrismRA, the new financing will bolster ongoing development of additional tests for other disease areas.
Asgarian said Scipher is launching a study in ulcerative colitis this quarter, which would establish the backbone for an initial classifier. The hope would be to then rapidly expand to more broadly address gastrointestinal autoimmune disease by adapting the test to address other disorders like Crohn's disease.
The company has similar expansion goals for the current PrismRA, with researchers working to develop specific classifiers for tumor necrosis factor inhibitors, JAK inhibitors, and T-cell inhibitors that can be built into a more comprehensive therapy selection test.
Beyond that, Scipher is hoping to add additional tests on a yearly basis. Multiple sclerosis is a likely candidate for the next slot, "and then we'll go from there," Asgarian said.
"Seeing a lot of similarities in the pathology of these diseases … it gives us a lot of hope that we'll be able to be successful in designing, deploying, and then launching these products in a very validated way," he added.