ORPH - ORPH STOCK NEWS

CytRx Corporation (OTCQB: CYTR) addressed the recent negative trend vote by the European Medicines Agency (EMA) on Orphazyme A/S's arimoclomol for Niemann-Pick disease type C (NPC). Despite disappointment over this decision regarding a non-core asset, CytRx emphasized that its core assets remain unaffected. The company highlighted positive developments in the clinical trials of aldoxorubicin, its advanced drug conjugate, currently studied by ImmunityBio in late-stage trials for pancreatic cancer and other indications, with potential milestone revenues up to $343 million.

CytRx Corporation (OTCQB: CYTR) highlighted Orphazyme A/S (NASDAQ: ORPH) plans regarding the resubmission of its New Drug Application for arimoclomol targeting Niemann-Pick disease type C (NPC). Orphazyme intends to request a Type C Meeting with the FDA in Q2 2022 to align on resubmission plans for H2 2022. Additionally, a European Medicines Agency opinion is expected in Q1 2022 regarding the Marketing Authorisation Application for arimoclomol. CytRx, which licensed arimoclomol to Orphazyme, will continue to provide updates on this collaboration.

CytRx Corporation (OTCQB:CYTR) reported its Q3 2021 results, ending on September 30 with approximately $16.5 million in cash and a net loss of $1.7 million, an improvement from a $2.8 million loss in Q3 2020. General and administrative expenses decreased to $1.5 million from $2.2 million year-over-year. The company paid $165,000 as part of a $10 million securities purchase agreement. CytRx continues to explore strategic partnerships for clinical testing while supporting ImmunityBio's Phase 2 pancreatic cancer trial and Orphazyme's regulatory pursuits regarding arimoclomol.

Orphazyme has received encouraging feedback from the FDA following a Type A meeting regarding arimoclomol, a treatment for Niemann-Pick disease Type C (NPC). The FDA suggests that Orphazyme provide supplementary data and analyses to support its resubmission path after a previous Complete Response Letter indicated the need for more evidence regarding the drug’s efficacy. CytRx Corporation, which sold arimoclomol to Orphazyme, expressed optimism about the FDA’s invitation for further discussions and potential regulatory pathways.

CytRx Corporation highlights the positive results from the Phase 2/3 trial of arimoclomol, an investigational treatment for Niemann-Pick disease type C. The study showed a significant treatment effect with a 65% relative reduction in disease progression over 12 months. Notably, in patients aged 4 and above, arimoclomol demonstrated an 82% reduction. The treatment was well-tolerated, with fewer serious adverse events compared to placebo. Orphazyme is awaiting a regulatory decision in Europe by Q4 2021 and is assessing the path forward in the U.S.

CytRx Corporation (OTCQB: CYTR) reports on Orphazyme A/S (NASDAQ: ORPH) announcing that its phase 2/3 trial of arimoclomol for inclusion body myositis (IBM) failed to meet primary and secondary endpoints. The trial involved 150 patients at 12 North American and European sites. Patients were given either arimoclomol or a placebo over 20 months. No major safety concerns were found, but the results highlight the challenges in developing effective treatments for IBM. CytRx continues to monitor developments with Orphazyme regarding their agreement on arimoclomol.

CytRx Corporation (OTCQB: CYTR) updates on the FDA's review of Orphazyme A/S's NDA for arimoclomol, aimed at treating Niemann-Pick Disease Type C (NPC). The FDA extended the review period by three months, setting a new PDUFA target action date of June 17, 2021. Despite the extension, the NDA holds Priority Review status, which suggests potential for significant therapeutic advancement. Orphazyme has received several designations for arimoclomol and is committed to the drug's approval and subsequent commercialization in both the U.S. and Europe.