Days after going public, Viela Bio Inc. nailed down a deal with Mitsubishi Tanabe Pharma Corp. to develop and commercialize Viela's humanized anti-CD19 monoclonal antibody, inebilizumab, in nine Asia regions for the rare disease called neuromyelitis optica spectrum disorder (NMOSD), as well as other potential future indications.
A spokesperson for Viela said the Gaithersburg, Md.-based company was still in a quiet period and could not comment, but the firm said in a press release that it's collecting an up-front licensing fee of $30 million from Mitsubishi and could gain more if development and commercial milestones are met. Further payments could be based, in part, on sales revenue, Viela said.
Mitsubishi, of Osaka, Japan, will lead development and commercialization of inebilizumab in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, Philippines, Singapore and Taiwan. The FDA in late August accepted for review Viela's BLA for the compound in NMOSD, also known as Devic disease, a chronic disorder of the brain and spinal cord dominated by inflammation of the optic nerve and of the spinal cord. It can result in severe muscle weakness and paralysis, loss of vision, respiratory failure and neuropathic pain.
In September, The Lancet published results from Viela's pivotal study, the N-MOmentum trial, described as the largest global, placebo-controlled study in NMOSD, with 231 patients enrolled. The experiment met its primary endpoint and a majority of secondary endpoints. Results, presented at a plenary session of the annual meeting of the American Academy of Neurology meeting in May in Philadelphia, demonstrated significant reduction in risk of NMOSD attack and reduced disability scores as measured by expanded disability status scale, hospitalizations and new central nervous system magnetic resonance imaging lesions.
Spun out from London-based Astrazeneca plc's Medimmune unit, Viela this week closed its IPO of 9.08 million shares, a total that included 1.18 million shares sold from the exercise in full by the underwriters of their option to purchase more shares, at $19 each. Gross proceeds totaled about $172.6 million. Newly minted Viela stock (NASDAQ:VIE) closed Wednesday at $19.11, down $1.94. (See BioWorld, March 1, 2018.)
In 2017, the FDA gave its go-ahead to two CD19-directed CAR T-cell therapies: tisagenlecleucel (Kymriah, Novartis AG) for treating patients 25 and older with r/r B-cell precursor acute lymphocytic leukemia (ALL), and axicabtagene ciloleucel (Yescarta, Gilead Pharmaceuticals Inc.), for adults with r/r large B-cell lymphomas (LBCL) who have received two or more lines of systemic therapy. The label for tisagenlecleucel was broadened a year later to include adults with r/r LBCL who have received two or more lines of systemic therapy. But the first anti-CD19 therapy cleared by U.S. regulators came in late 2014, when blinatumomab (Blincyto, Amgen Inc.) was approved to treat adults with relapsed or refractory Philadelphia chromosome-negative precursor B-cell ALL.
CD19 remains a target of widespread interest. In May, Morphosys AG, of Martinsried, Germany, disclosed that its antibody in the space, tafasitamab (MOR-208), hit the primary endpoint of a pivotal phase II trial in patients with r/r diffuse large B-cell lymphoma (DLBCL). The L-Mind study was an open-label, single-arm trial that recruited 81 patients with r/r DLBCL who were deemed too elderly or frail to undergo high-dose chemotherapy and autologous stem cell transplantation. They were given tafasitamab in combination with Revlimid (lenalidomide), the blockbuster multiple myeloma drug from Summit, N.J.-based Celgene Corp. As a monotherapy, the latter drug has modest effects in DLBCL, but its immune activation mechanism appears to be synergistic with tafasitamab's B-cell-targeting mechanism. (See BioWorld, July 27, 2016, and May 17, 2019.)
The anti-CD19 approach doesn't always work, and research in the area proceeds apace. Findings from City of Hope labs published last month in Science Translational Medicine described how the first CAR T-cell therapy targeting the B-cell-activating factor receptor (BAFF-R) on cancerous cells eradicated CD19-targeted therapy-resistant human leukemia and lymphoma cells. Animal models with CD19 therapy-resistant human tumors (including Burkitt, mantel cell, and other non-Hodgkin lymphoma subtypes and acute lymphoblastic leukemia) received BAFF-R CAR T therapy. "Remarkable" tumor regression and prolonged survival were observed after treatment with those CAR T cells, City of Hope said. In models with human Burkitt lymphoma, BAFF-R CAR T therapy achieved complete tumor regression with 100% long-term survival. For another part of the study, the animal models had a mixed population of CD19-positive and negative human tumors. They received either CD19 CAR T-cell therapy or BAFF-R CAR T-cell therapy; the BAFF-R CAR T cells were able to eradicate both tumor populations, while treatment failed in those receiving CD19 CAR T cells. City of Hope has licensed the BAFF-R CAR T to privately held Pepromene Bio Inc., of Irvine, Calif., which has IND-enabling efforts underway.