News

Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced the full data set for its Phase II/III clinical trial of arimoclomol in NPC, a devastating rare genetic disorder. Treatment with arimoclomol adjunct to routine clinical care resulted in a 74% reduction in disease progression (p-value =0.0506) as measured by the primary endpoint, 5-domain NPC Clinical Severity Scale (NPC-CSS). In the predefined subgroup of patients of 4 years and older (44 out of 50 randomized patients in the trial), the treatment difference was statistically significant with a minimal disease progression at month 12 in the arimoclomol-treated group (p-value =0.0219). A highly statistically significant treatment difference was observed in another predefined subgroup analysis, requested by the European Medicines Agency (EMA), that compared arimoclomol to placebo control in patients receiving miglustat as a part of routine clinical care (p-value =0.0071).

Alligator Bioscience (Nasdaq Stockholm: ATORX), announces the launch of a novel concept in bispecific antibody formats, RUBY™. The RUBY concept gives Alligator competitive abilities to generate therapeutic antibodies that are both efficient and highly manufacturable in a shorter time period than what is currently possible.

“The bispecific format RUBY significantly strengthens our existing technology platform. We now have all technologies in-house to generate virtually any future bispecific therapeutic antibody, tailor-made for immuno-oncological applications,” said Per Norlén, CEO of Alligator. “Moreover, the RUBY format allows for superior binding and dramatically shortened development timelines which is a crucial competitive factor”.

VAXIMM AG, a Swiss/German biotech company focused on developing oral T-cell immunotherapies, today announced that the first patient has been dosed in a Phase I/II trial evaluating VXM01 oral immunotherapy in combination with avelumab*, a human anti-PD-L1 antibody, for the treatment of glioblastoma. The trial is part of a collaboration agreement with Merck KGaA, Darmstadt, Germany and Pfizer Inc.

This trial is a multicenter, open-label, Phase I/II trial (EudraCT #: 2017-003076-31) to evaluate the safety and efficacy of VXM01 in combination with avelumab in patients with recurrent glioblastoma. The trial is planned to enroll a total of 30 patients at approximately eight clinical sites in Europe. The primary objective of the study is to evaluate the safety and tolerability of VXM01 in combination with avelumab. Secondary objectives include progression-free survival and overall survival.

Anergis, a company developing proprietary ultra-fast Allergy Immunotherapy (AIT), announced today that the Company has successfully completed the mice proof-of-concept study of the second-generation COP allergy vaccines based on its research collaboration with Mymetics SA, a leader in the research and development of virosome-based vaccines. The Company plans to raise new financing to return to clinical trials in allergic patients as soon as possible.

The mice proof-of-concept immunogenicity study evaluated the effects of the Bet v 1 COPs (the birch pollen allergy proprietary peptides) using the five subcutaneous injection schedule used in clinical trials with Anergis’ first-generation product candidate AllerT. The development of AllerT (Bet v 1 COPs plus aluminum hydroxide) was discontinued by Anergis in 2017 following completion of a Phase 2 clinical trial showing evidence of sensitization to the peptides and a 7% reduction in seasonal allergy symptoms vs. placebo (p=0.0047).