The focus of preclinical research soon turned to non-Hodgkin’s lymphoma (NHL). And when MorphoSys received the latest interim results from one of the first clinical studies with tafasitamab, the message was clear: “We have a drug.” Some of the patients, as many as 30 %, had responded – and that was with tafasitamab as monotherapy.
In preclinical studies, we had already shown that the combination of the immunomodulator lenalidomide and tafasitamab showed promising efficacy which led to the decision to assess this combination in a clinical study.
The developers then set up a phase 2 clinical trial in DLBCL, called L-MIND, to investigate the efficacy of the tafasitamab-lenalidomide combination. It quickly became clear that it had the potential to be a promising combination. In 2017, based on the first interim results, the FDA granted breakthrough therapy designation, which is an important milestone for accelerated approval.
“This was enormously motivating for us,” says Mark Winderlich, Ph.D., Vice President and Head of Biostatistics & Data Management. However, there was one challenge: The L-MIND study, which was designed to be an exploratory study, was a single-arm study. To avoid losing any time, we pioneered a new approach in discussion with the FDA: To compare the data of the L-MIND combination study with data obtained with lenalidomide treatment alone, we generated a synthetic control arm from real-world data.
Collecting enough data from a prospectively designed retrospective study to assess the effectiveness of lenalidomide monotherapy compared to the tafasitamab-lenalidomide combination proved to be an exciting task: “We reached out to physicians in various countries and they reviewed hundreds of patient charts to identify patients treated with lenalidomide alone who also had comparable characteristics to those in L-MIND”.
Step-by-step, patient-by-patient, things progressed. And then, at the very end of 2019, on December 30, the application for approval, together with the necessary data, was submitted to the FDA.
While the team was focused on responding to the FDA’s inquiries along the way, the work continued to get ready for the launch. On January 13, 2020, MorphoSys and Incyte entered into a collaboration and license agreement to further develop tafasitamab and make it available to patients worldwide. Incyte is an attractive partner due not only to its experience with developing and commercializing hematology products, but also its vision, goals and scientific culture, which aligned with those of MorphoSys. The two companies are jointly responsible for the commercialization of Monjuvi which is the brand name within the U.S., whereas Incyte has the commercialization rights outside the U.S. This marked another step in preparing for the potential approval, which was granted by the FDA at the end of July 2020, one month ahead of the Prescription Drug User Fee Act (PDUFA) date. Monjuvi has since been available in the U.S. as the first and, to date, only second-line therapy in combination with lenalidomide for adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT).
By 2019, MorphoSys had the critical element it needed to launch into the DLBCL space – tafasitamab – a compound that could potentially fill a significant unmet need. And thousands of patients relapsing with DLBCL were waiting for effective treatment options. That was when MorphoSys made the decision to open MorphoSys US Inc. in Boston in preparation for the launch of the new blood cancer therapy. The goal for the team was clear. “We needed to get prepared to educate healthcare professionals about the new treatment option so the first patients could benefit from the treatment in a timely manner,” says Nuwan Kurukulasuriya, Ph.D., Senior Vice President and Global Head of Medical Affairs. MorphoSys US Inc. grew from a team of one in 2019 to a commercial and medical organization of 150 in 2020, sparking the transformation of MorphoSys into a fully integrated biopharma company.
Prior to the approval of Monjuvi, several objectives had to be met. The first was to understand the patient journey and educate healthcare professionals on the scientific rationale of targeting the CD19 antigen in DLBCL with a monoclonal antibody. Secondly, the team had to be comprehensively trained on the DLBCL clinical management and the Monjuvi profile. “Our goal all along was to ensure that this treatment regimen was getting to the right patients who could benefit from it,” says David Trexler, President of MorphoSys US Inc.
When the U.S. FDA granted the accelerated approval of Monjuvi, the MorphoSys’ field team was certified within days and began reaching out to physicians to introduce the new treatment option within the first week, knowing that every day counts for patients with this aggressive form of blood cancer. On July 31, 2020, MorphoSys and Incyte announced that the U.S. FDA had approved Monjuvi. Monjuvi was shipped to specialty distributors in the U.S. on August 5, the first customer order was received on August 7, and the first patient was dosed on August 13, less than two weeks after approval.