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Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC).

The trial was a multi-center, prospective, double-blinded, placebo-controlled interventional study with a 12-month duration. In total, 50 patients were enrolled in the EU and US. The purpose of the trial was to assess the efficacy and safety of arimoclomol, compared to placebo, in the treatment of NPC, administered in addition to the patient’s standard-of-care. The primary endpoints, 5-domain NPC-CSS and Clinical Global Impression of Improvement (CGI-I), evaluated the treatment difference between the arimoclomol-treated and the placebo group after 12 months of treatment.

Overall, baseline characteristics were well-balanced across treatment arms. Arimoclomol was well-tolerated. The overall incidence of adverse events (AEs) was similar for arimoclomol (85.7%) and placebo (81.3%). Serious AEs occurred less frequently in the arimoclomol group (14.3%) compared to placebo (37.5%). The top-line data demonstrated a 74% reduction in progression on the primary endpoint, corroborated by consistent benefit across sub-populations. Placebo progression rates on the CGI-I were lower than expected impeding the ability to show a positive effect.

VAXIMM AG, a Swiss/German biotech company focused on developing oral T-cell immunotherapies, today announced the signing of a development and commercialization collaboration agreement with China Medical System Holdings Limited (CMS) (HKSE:0867).

VAXIMM will collaborate with CMS, which will have the full rights in China and other Asian countries (excluding Japan) to VAXIMM’s existing programs, enabling the development and commercialization of VAXIMM’s pipeline of oral cancer immunotherapy candidates in the region. The parties will coordinate and share data from their respective clinical studies. VAXIMM will also receive development and commercial milestones and royalties based on the achievement of sales milestones by CMS. In parallel with the agreement, CMS will make an equity investment in VAXIMM, as part of VAXIMM’s current Series B financing.

–  2-month treatment with AllerT COP allergy vaccine provides sustained clinical benefit for more than one pollen season
– Research program to develop next-generation COP allergy vaccines in progress
Anergis, a company developing proprietary ultra-fast Allergy Immunotherapy (AIT), announced today that the Company has initiated the development of the second generation of its ultra-fast COP allergy vaccines. Moreover, the Company reported the publication of key proof-of-concept data from a clinical phase IIb follow-up trial demonstrating sustained clinical efficacy of its birch pollen vaccine AllerT for more than one pollen season.

In 2016, results of a Phase IIb trial with the AllerT COP allergy vaccine published in the Journal of Allergy and Clinical Immunology [1] demonstrated the efficacy of AllerT during the natural birch pollen season immediately following a single 2-month treatment course. The new data published in this year’s August issue of the Journal of Allergy and Clinical Immunology [2] demonstrate that the efficacy of AllerT persisted during the following annual pollen season a year later without repeated treatment. Results were based on a follow-up including 196 of 240 patients that originally enrolled in the study.

Improvements in scores after the second season for both primary and secondary endpoints were over 20%, the threshold usually considered to be clinically relevant. Together with earlier evidence of immunological memory lasting for at least 4 years [3], these clinical data confirm the core concept of ultra-fast AIT, i.e. eliciting a persistent benefit by administering a single treatment course with a COP allergy vaccine.

For optimized clinical efficacy and tolerability, Anergis now has initiated a new preclinical research program to design the next generation of COP allergy vaccines. Second-generation COP allergy vaccines are designed to boost and at the same time to modulate the immunogenicity of COPs towards a more TH1 rather than TH2 immune response. As part of this program, Anergis has entered into a research collaboration with Mymetics Corporation (OTCQB:MYMX), a pioneer and leader in the research and development of virosome-based vaccines.

Research to identify the most suitable formulation has already started, with initial results expected in Q1/2019. Subsequently, Anergis aims to proceed to the clinical evaluation of its second-generation COP allergy vaccines as soon as possible.

Arcomas röntgensystem säljs i Nordamerika av Canons dotterbolag Virtual Imaging under produktnamnen RadPro Omnera 400A och 400T. Förutom genom Virtual Imagings egen organisation och distributörer kommer nu dessa produkter även kunna säljas av Canon Medical Systems USAs betydligt större organisation som förvärvades från Toshiba 2017. Detta  innebär en möjligt ökad marknadspenetration i Nordamerika för Arcomas produkter.

Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that the first patient has received the first dose in a Phase II clinical trial for Gaucher disease.

Anders Hinsby, Chief Executive Officer, said: “With the enrollment of the first patient in the Gaucher disease trial, we reach another important milestone in the development of our pipeline this year. Glucocerebrosidase deficiency leads to severe neurological manifestations in many patients with Gaucher disease. With this trial we move a step closer to a treatment option for these patients.”