Orphazyme News - LARVOL Sigma

|||||||||| felzartamab (MOR202) / Biogen
Felzartamab for IgA Nephropathy: Final Results of the IGNAZ Study (Room 6C, Convention Center) - Oct 12, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4653;
Felzartamab was generally well tolerated and led to sustained proteinuria reduction and reduced eGFR decline vs PBO, indicating potential disease modification in patients with IgAN. Investigation of felzartamab in patients at high risk for loss of kidney function is warranted.

|||||||||| Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
OR01-1. Ambulatory Anesthesia - Oral Presentations (Non-CME) (120A) - Jun 25, 2024 - Abstract #ASA2024ASA_87;

|||||||||| arimoclomol (OR-01) / Orphazyme, Zevra Therap
OR01. Neuroendocrinology: Gene Regulation and Function (151AB - BCEC) - May 5, 2024 - Abstract #ENDO2024ENDO_1935;

|||||||||| arimoclomol (OR-01) / Orphazyme, Zevra Therap
Journal: Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial. (Pubmed Central) - Sep 27, 2023
P2
Arimoclomol did not improve efficacy outcomes, relative to placebo, but had an acceptable safety profile in individuals with inclusion body myositis. This is one of the largest trials done in people with inclusion body myositis, providing data on disease progression that might be used for subsequent clinical trial design.

|||||||||| arimoclomol (OR-01) / Orphazyme, KemPharm, saracatinib (AZD0530) / AstraZeneca
Preclinical, Journal: Heat shock protein amplification improves cerebellar myelination in the Npc1 mouse model. (Pubmed Central) - Dec 2, 2022
This is one of the largest trials done in people with inclusion body myositis, providing data on disease progression that might be used for subsequent clinical trial design. These data increase our understanding of the mechanisms by which HSP70 regulates myelination and provide further support for the clinical development of HSP-amplifying therapies in the treatment of NPC.

|||||||||| arimoclomol (OR-01) / Orphazyme
Clinical, P2/3 data, Journal: Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomized, placebo-controlled, multinational phase 2/3 trial of a novel treatment. (Pubmed Central) - Feb 4, 2022
P2/3
These data increase our understanding of the mechanisms by which HSP70 regulates myelination and provide further support for the clinical development of HSP-amplifying therapies in the treatment of NPC. Arimoclomol provided a significant and clinically meaningful treatment effect in NPC and was well tolerated.

|||||||||| arimoclomol (OR-01) / Orphazyme
[VIRTUAL] A Randomized, Double-Blind, Placebo Controlled Study of Arimoclomol in Patients with Inclusion Body Myositis () - Oct 21, 2021 - Abstract #ANA2021ANA_463;
P2
Arimoclomol provided a significant and clinically meaningful treatment effect in NPC and was well tolerated. This trial did not demonstrate a benefit of arimoclomol in IBM with respect to its primary and second ary efficacy endpoints

|||||||||| arimoclomol (OR-01) / Orphazyme
Clinical, Journal: Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. (Pubmed Central) - Jun 22, 2021
P2/3
Trial registration NCT02612129. Registered 23 November 2015, https://clinicaltrials.gov/ct2/show/NCT02612129.

|||||||||| arimoclomol (OR-01) / Orphazyme
Journal: Symbiotic efficiency and genetic characterization of rhizobia and non rhizobial endophytes associated with cowpea grown in semi-arid tropics of Kenya. (Pubmed Central) - May 20, 2021
This study also reports for the first time the presence of an efficient native cowpea nodulating Beta-Rhizobia (Paraburkholderia phenoliruptrix BR3459a) in Africa...USDA 3456 (67.68 %). Our results form an important step in the development of efficient microbial inoculum and sustainable food production.

|||||||||| arimoclomol (OR-01) / Orphazyme
And #Arimoclomol too! @Orphazyme_AS (Twitter) - Mar 25, 2021

|||||||||| arimoclomol (BRX-220) / CytRx, arimoclomol (OR-01) / Orphazyme
CytRx Highlights Orphazyme’s Acceleration of Pre-Launch Activities for Arimoclomol https://t.co/nrBMvdGVu5 (Twitter) - Oct 27, 2020

||||||||||  Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Trial completion, Trial primary completion date:  Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (clinicaltrials.gov) -  Aug 14, 2017
P2/3, N=38, Completed,  Sponsor: University of Miami
Our results form an important step in the development of efficient microbial inoculum and sustainable food production. Active, not recruiting --> Completed | Trial primary completion date: Dec 2016 --> Dec 2014

||||||||||  Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Enrollment change:  Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (clinicaltrials.gov) -  Feb 19, 2015
P2/3, N=39, Active, not recruiting,  Sponsor: University of Miami
Active, not recruiting --> Completed | Trial primary completion date: Dec 2016 --> Dec 2014 N=80 --> 39

||||||||||  Miplyffa (arimoclomol) / Orphazyme, Zevra Therap
Enrollment closed:  Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (clinicaltrials.gov) -  Aug 7, 2014
P2/3, N=80, Active, not recruiting,  Sponsor: University of Miami
N=80 --> 39 Recruiting --> Active, not recruiting



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