Vaximm AG, a Swiss/German biotech company focused on developing oral T-cell immunotherapies, announced that the company's lead product candidate, VXM01, has received orphan designation from the European Commission and from the US Food and Drug Administration (FDA) for the treatment of glioma, a difficult-to-treat form of brain cancer. Orphan designation is designed to encourage the development of drugs to treat rare diseases and conditions and provides companies with certain incentives, including periods of market exclusivity upon approval, as well as user fee reductions or exemptions. The designation by the European Commission is for the treatment of glioma and by the US FDA for the treatment of malignant glioma.

"We are excited to have received orphan designation from both the European Commission and the US FDA, important achievements in the development of VXM01 to treat brain tumors, a challenging-to-treat cancer indication," said Matthias Schroff, Ph.D., Chief Executive Officer of Vaximm. "We have seen strong initial results in a Phase I trial of VXM01 in patients with recurrent glioblastoma. In May, we entered into a collaboration with Merck KGaA and Pfizer to evaluate, through Phase I/II clinical studies, the combination of VXM01 with avelumab to treat glioblastoma and colorectal cancer. We look forward to continuing the development of our novel immunotherapy to treat these devastating tumor types."

Phase I results in glioblastoma, a type of glioma, were presented at the American Society of Clinical Oncology (ASCO) 2017 Annual Meeting on June 5, 2017. The trial was designed to evaluate the safety and tolerability of, as well as clinical and immunogenic response to, VXM01 in patients with recurrent glioblastoma whose disease had progressed following treatment with at least radiochemotherapy including temozolomide, the standard of care. The reported data were from eight patients who were given a single dose of VXM01 on days 1, 3, 5 and 7 before planned surgery on day 35. Following surgery, patients could then receive a single administration every four weeks during the follow-up period. Median dosage was seven vaccinations.

Surgery was performed on seven of the treated patients. One patient experienced an objective and durable response, and three other patients had stable disease. Additionally, peripheral immune responses were observed, and five of seven patients had an increase in CD8+ T-cells in tumor tissue following re-operation compared to the primary tumor tissue. Four out of eight patients showed a specific T-cell response. In addition, in four patients a relevant increase in cerebral blood volume and apparent diffusion coefficient on post-vaccination MRI was observed. VXM01 was shown to be well tolerated.

The European Medicines Agency (EMA) examines the applications for orphan designation in the European Union and facilitates the development and authorization of medicines for rare diseases. The European Commission is responsible for granting orphan designation, which includes access to centralized marketing authorization in the European Union, ten years of protection from market competition with similar medicines in similar indications and fee reductions for regulatory activities.

In the US, orphan-drug designation qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing, waiver of user fees and potentially up to seven years of market exclusivity for the given indication, if approved.