The FDA rejected leronlimab and requested additional analysis of existing data.
An FDA review of clinical data for belantamab mafodotin revealed safety concerns due to 71% of patients developing keratopathy, with 44% of patients experiencing severe symptoms. However, the FDA Oncologic Drugs Advisory Committee voted 12-0 to recommend approval with a REMS program to educate patients and physicians about the risk.
An FDA review of terlipressin questioned whether clinical data on hepatorenal syndrome type 1 reversal demonstrated decreased mortality, dialysis or intensive care days. However, the FDA Cardiovascular and Renal Drugs Advisory Committee voted 8-7 to recommend approval.
The FDA accepted the NDA for vericiguat for reducing the risk of cardiovascular death and heart failure hospitalization following a worsening heart failure event in patients with symptomatic chronic heart failure with reduced ejection fraction, in combination with other heart failure therapies. and set a PDUFA date of 1/20/2021.
The FDA accepted the NDA for the treatment of spasticity in multiple sclerosis patients and set a PDUFA date for 12/29/2020.
The FDA designated mosunetuzumab a Breakthrough Therapy for the treatment of relapsed or refractory follicular lymphoma who have received at least two prior systemic therapies.
Roche licensed pralsetinib from Blueprint Medicines for $675 million.
Announced Research Updates
Spark announced 2 to 3.3 year interim results from 12 patients enrolled in a Phase I/II trial, where SPK-8011 reduced the annual bleeding rate by 91% and infusions of FVIII by 96% in patients with Hemophilia A.
Aprea announced that in a 52 patient, Phase II trial, treatment with APR-246 and azacitidine resulted in an overall response rate of 76% in patients with TP53-mutant myelodysplastic syndrome or acute myeloid leukemia.
Lilly announced that in the 52-week, 1,465 patient, Phase III, OASIS-2 trial, 83.1 to 83.3% of patients treated with mirikizumab achieved a Static Physician's Global Assessment (sPGA) of 0 or 1 with at least a 2-point improvement from baseline compared to 68.5% with secukinumab in patients with moderate to severe plaque psoriasis. In the same trial, 81.4 to 82.4% of mirikizumab patients achieved a 90% or > improvement in Psoriasis Area and Severity Index (PASI 90) compared to 69.4% with secukinumab.
Amplyx announced that in a 20 patient, Phase II trial, 80% of patients treated with fosmanogepix achieved clearance of Candida from blood cultures with no additional antifungal treatment, and survival at the end of study treatment, in patients with candidemia.
Published Research Updates
In a 3-month, 8 patient, Phase III, open-label trial, treatment with setmelanotide resulted in a 5.5% decrease in weight in patients with Bardet-Biedl syndrome. Seven of the patients completed a 9-month extension and achieved a 16.3% weight loss at 12 total months.
In a 4-week, 152 patient, Phase II trial, patients treated with tradipitant had a 1.2 decrease in their patient reported nausea score compared to a 0.7 decrease with placebo in patients with idiopathic and diabetic gastroparesis. Patients who were experiencing nausea and vomiting at enrollment achieved a 1.4 point decrease.
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