An FDA review of Incyte’s retifanlimab questioned whether there was enough safety and efficacy data to support approval. The FDA’s Oncologic Drugs Advisory Committee agreed and voted 13-4 to recommend the FDA delay approval of retifanlimab until additional clinical evidence is available.
The FDA accepted the NDA for Alnylam’s vutrisiran for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis and set a PDUFA date for 4/14/2022.
EMA’s human medicines committee (CHMP) recommended approval of UCB’s bimekizumab to treat plaque psoriasis, FibroGen’s and AstraZeneca’s roxadustat to treat CKD anemia, and BioMarin’s vosoritide to treat achondroplasia.
Announced Research Updates
The FDA released documents related to the internal discussion on approval of aducanumab. The Office of Neuroscience supported accelerated approval, while the Office of Biostatistics did not. Senior FDA officials agreed with the Office of Neuroscience and approved the drug, based on the results of the higher dose cohort of the EMERGE trial and discounting the results from the ENGAGE trial. The FDA also based the decision on an analysis of six trials of Alzheimer’s drugs that reduce amyloid and demonstrated a relationship for plaque reduction and a beneficials clinical effect. That analysis has not been released and neither was the full statistical review.
UniQure announced that in the 52-week, 54 patient, Phase III, HOPE-B trial, a single dose of etranacogene dezaparvovec increased Factor IX activity from less than 2% at baseline to 39% at 26-weeks and 41.5% at 52-weeks in adult males with severe or moderate-severe Hemophilia B. The annualized rate of bleeding requiring treatment was reduced from 3.39 at baseline to 0.68 bleeding episodes. 96% of patients were able to discontinue Factor IX replacement therapy.
LSKB and Hengrui announced that in the 190 patient, open-label, Phase II, RESCUE trial, treatment with camrelizumab and apatinib resulted in overall survival of 20.1 months in previously untreated hepatocellular carcinoma patients and 21.8 months in patients receiving the combination as second-line treatment. The primary outcome of the RESCUE trial, objective response rate, was not reported.
Agios announced that in a 24-week, 20 patient, Phase II, open-label trial, 80% of patients treated with mitapivat lead to a 1 g/dL or > increase in hemoglobin in five patients with alpha thalassemia and 15 with beta thalassemia who were not transfusion-dependent.
Corbus announced that in the 28-week, 175 patient, Phase III, DETERMINE trial, adding lenabasum to standard treatment did not improve the Total Improvement Score compared to placebo in patients with active classic or amyopathic dermatomyositis.
Gilead announced that in the 120-week, 175-patient, Phase IIb, MYR204 trial, 24-weeks after the end of treatment, more patients treated with bulevirtide 2 mg plus pegylated interferon alpha 2a (PEG-IFN alpha), bulevirtide 10 mg plus PEG-IFN and bulevirtide 10 mg alone achieved at least a 2 log decrease or undetectable levels of hepatitis D (HDV) RNA and normalization of ALT levels compared to PEG-IFN alone in patients with chronic HDV. Gilead announced interim results after 24-weeks in the 48-week, 150 patient, Phase III, MYR301 trial, where 36.7% of patients treated with bulevirtide 2 mg and 28% of patients that received bulevirtide 10 mg achieved at least a 2 log decrease or undetectable levels of HDV RNA and normalization of ALT levels compared to 0% of untreated chronic HDV patients.
Fulcrum announced that in the 48-week, 80 patient, Phase IIb ReDUX4 trial, where treatment with losmapimod did not change DUX4-driven gene expression in affected skeletal muscle biopsies at 16 or 24 weeks compared to placebo in patients with facioscapulohumeral muscular dystrophy.
Madrigal announced interim results from 115 patients enrolled in the 52-week, 1,200 patient, Phase III, MAESTRO-NAFLD-1 trial, where treatment with resmetirom reduced the MRI-proton density fat fraction (MRI-PDFF) and fibrosis compared to placebo in patients with non-cirrhotic NASH.
Italfarmaco announced that in a 12-month, 51 patient, Phase II trial, treatment with givinostat did not decrease muscle fibrosis, but did decrease fat infiltration compared to placebo in patients with Becker Muscular Dystrophy. Givinostat did not demonstrate an improvement in functional status compared to placebo.
AstraZeneca and Sanofi announced that in the 150-day, 925 patient, Phase II/III, MEDLEY trial, nirsevimab was similar to palivizumab in the development of adverse events in healthy infants at high risk of respiratory syncytial virus entering their first RSV season.
Orphazyme announced interim data from an open-label extension of a 41 patent, Phase II/III Niemann-Pick disease trial, where 33 patents completed an additional 24-months of treatment. Patients that received arimoclomol for a total of 36-months had an increase in their NPC-CSS score of 3.5 points compared to an estimated 5.2 point increase in patients that received routine clinical care.
Published Research Updates
In a 70 patient, open-label Phase II trial, treatment with savolitinib for a mean 17.6 months resulted in an objective response rate of 42.9% in patients with locally advanced or metastatic METex14-positive pulmonary sarcomatoid carcinoma.
In the 831 patient, open-label, Phase III, VISION trial, patients treated with Lu-PSMA-617 plus standard of care achieved progression free survival of 8.7 months and overall survival of 15.3 months compared to 3.4 months and 11.3 months with standard of care alone in patients with advanced stage PSMA-positive metastatic castration-resistant prostate cancer previously treated an androgen inhibitor and taxane.
In a 263 patient, Phase III trial, treatment with camrelizumab plus gemcitabine and cisplatin improved progression-free survival to 9.7 months compared to 6.9 months with gemcitabine and cisplatin alone in Chinese patients with recurrent or metastatic nasopharyngeal carcinoma.
In the 40-week, 475 patient, Phase III SURPASS-5 trial, treatment with tirzepatide reduced HbA1C by 2.01% with 5 mg, 2.24% with 20 mg and 2.3% with 15 mg compared to a 1.86% decrease with semaglutide in patients with type 2 diabetes. Tirzepatide decreased body weight more than semaglutide by 1.9 kg with 5 mg, 3.6 kg with 10 mg and 5.5 kg with 15 mg.
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