Update for March 30, 2021

​Regulatory Update 
 
The FDA approved dasiglucagon (Zegalogue, Zealand Pharma), on 3/23/2021, for the treatment of severe hypoglycemia in people with diabetes aged 6 and older. Dasiglucagon will be available as an autoinjector and prefilled syringe.
 
The FDA approved idecabtagene vicleucel (Abecma, Bristol Myers Squibb and bluebird bio), on 3/27/2021, for the treatment of adult patients with multiple myeloma who have received at least four prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody). Idecabtagene vicleucel was approved with a black box warning for cytokine release syndrome, neurotoxic events, and prolonged cytopenias as well as hemophagocytic lymphohistiocytosis/macrophage activation syndrome.
 
The FDA’s Arthritis Advisory Committee and the Drug Safety and Risk Management Advisory Committee voted 19-1 against recommending approval for tanezumab, due to concerns over safety and the proposed drug safety strategy to prevent neuropathy and destructive joint disease.
 
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended ponesimod and estetrol/drospirenone for approval.
 
Announced Research Updates
 
Roche licensed tominersen from Ionis. Roche suspended the Phase I, GEN-PEAK trial in 2020 due to the occurrence of two intrathecal catheter related infections, but later restarted the trial. GEN-PEAK is evaluating tominersen’s pharmacokinetic and pharmacodynamic profile. Roche discontinued dosing in the Phase III GENERATION HD1 and GEN-EXTEND trials, in March 2021, at the recommendation of the independent data monitoring committee when an interim analysis questioned the drug’s potential benefit/risk profile. The trials are evaluating tominersen as a treatment for Huntington’s disease. The GEN-PEAK trial is continuing and Roche will continue to monitor safety and efficacy in patients from the GENERATION HD1 and GEN-EXTEND trials.
 
Novartis announced that in the 200 patient, open-label, Phase III, VISION trial, treatment with Lu-PSMA-617 improved overall survival and radiographic progression-free survival compared to standard of care in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer.
 
AZ announced that in a 109 patient, open-label, Phase II trial, treatment with cediranib plus olaparib did not improve progression-free survival compared to cediranib alone in patients with recurrent, metastatic or persistent endometrial cancer. 
 
BrainStorm announced that in a 28-week, 16 patient, open-label, Phase II trial, treatment with mesenchymal stem cells neurotrophic growth factors (MSC-NTF) resulted in 14% of patents having at least a 25% improvement in the timed 25-foot walk (T25FW) test and 13% had at least a 25% improvement in the 9-hole peg test (9-HPT) compared to none in a historical registry of patients with progressive multiple sclerosis.
 
LSK BioPharma announced that in the 152 patient, Phase II APPROVE trial, treatment with apatinib plus pegylated liposomal doxorubicin resulted in an objective response rate of 43.1% compared to 10.9% with pegylated liposomal doxorubicin alone in patients with platinum-resistant or refractory ovarian cancer.
 
Orphazyme announced that in a 20-week, 150 patient, Phase II/III trial, treatment with arimoclomol did not improve the body myositis functional rating scale compared to placebo in patients with inclusion body myositis.
 
UniQure announced that an investigation by an independent lab found that it was highly unlikely that etranacogene dezaparvovec caused hepatocellular carcinoma in a patient enrolled in the HOPE-B trial. The patient had several risk factors for developing liver cancer including hepatitis C, hepatitis B, non-alcoholic fatty liver disease and history of smoking.
 
Published Research Updates
 
In an 8-week, 301 patient, Phase III trial, treatment with viloxazine decreased the ADHD-RS-5 total score by 17.6 points with 200 mg and 17.5 points with 400 mg compared to an 11.7 point decrease with placebo in children 6 to 11 years of age with attention deficit hyperactivity disorder. 
 
In a four-week, 69 patient, Japanese, open-label, Phase II trial, treatment with a single subcutaneous dose of nemolizumab did not reduce pruritus compared to daily oral nalfurafine hydrochloride in hemodialysis patients with uremic pruritus. 
 
In the 16-week, 838 patient, Phase III, JADE COMPARE trial, an IGA score of clear or almost clear skin, with a 2 point or > improvement in the IGA score was achieved by 48.4% of patients treated with abrocitinib 200-mg, 36.6% with 100-mg abrocitinib group and 36.5% with dupilumab compared to 14.0% with placebo in patients with moderate to severe atopic dermatitis receiving topical therapy. A 75% or greater improvement in the Eczema Area and Severity Index (EASI-75) score was achieved by 70.3% of patients treated with abrocitinib 200-mg, 58.7% with 100-mg abrocitinib group and 58.1% with dupilumab compared to 27.1% with placebo
 
In a 24-month, 5,404 HIV-negative patient, Phase IIb/III trial, 138/2,404 patients who received five immunizations of the Sanofi Pasteur HIV vaccine with the GSK immunity-boosting adjuvant became infected with HIV compared to 133/2,700 patients that received placebo. The trial was terminated when an interim analysis determined a lack of efficacy compared to placebo.