Phase II
Abeona Therapeutics’ shares popped 18.32% in pre-market trading after New York and Cleveland-based company announced magnetic resonance imaging (MRI) data from its Phase I/II Transpher A trial in San Filippo Syndrome Type A (MPS IIIA).
Updated trial data show patients treated with BioMarin Pharmaceutical’s severe hemophilia A gene therapy expressed substantially lower factor VIII levels after five years compared with one year, calling into question the drug’s long-term benefits.
It was another busy week for clinical trial updates. Here’s a look at some including Mesoblast Limited, CytoDyn, Magenta Therapeutics and more.
Shares of Adverum Biotechnologies plunged more than 20% in premarket trading after the company announced it was revising its clinical development plan for investigational gene therapy candidate ADVM-022 based on safety concerns in patients with diabetic macular edema (DME).
The company said it has decided to close the study’s open-label and maintenance portions given the inability to satisfy endpoint goals.
The study will also investigate the oral combination with PD-1 inhibitor pembrolizumab in EBV+ metastatic nasopharyngeal carcinoma (RM-NPC).
The drug is a nucleoside reverse transcriptase translocation inhibitor, with the results presented at the virtual 11th International AIDS Society Conference on HIV Science.
The firm would be moving forward to Phase III for the treatment of hypertrophic cardiomyopathy, which is expected to commence before the end of 2021.
It was a moderately busy mid-summer week for clinical trial news. Here’s a look.
The drug, a novel dopamine D3-receptor antagonist, is being evaluated in Phase IIb trials for people with Parkinson’s disease who experience levodopa-induced dyskinesia.
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