The FDA rejected the BLA for Sanofi’s sutimlimab for the treatment of hemolysis in adult patients with cold agglutinin disease due to problems in a contracted manufacturing facility.
The FDA rejected the BLA for Alkermes’ ALKS 3831 a combination of olanzapine and samidorphan) to treat schizophrenia due to tablet coating problems during manufacturing.
The FDA has delayed approval of Bristol Myers Squibb’s lisocabtagene maraleucel due to travel restrictions delaying the inspection of the manufacturing plant. A new PDUFA date has not been announced.
Announced Research Updates
Pfizer announced that in the 52-week, 1,233 patient, Phase III, JADE REGIMEN trial, abrocitinib prevented an acute dermatitis flare (loss of at least 50% of the Eczema Area and Severity Index or EASI response seen at week 12 and an IGA score of two or higher) in 81.1% of patients treated with 200 mg and 57.4% of patients treated with 100 mg compared to 19.1% that received placebo in patients with moderate to severe atopic dermatitis.
OncoSec Medical announced interim data from 54 patients enrolled in the Phase IIb, open -label, PISCES/KEYNOTE-695 trial, where treatment with tavokinogene telseplasmid plus pembrolizumab resulted in a 30% overall response rate in patients with unresectable stage III or IV metastatic melanoma.
Amgen and AstraZeneca announced that in the 1,061 patient, 52-week, Phase III NAVIGATOR trial, treatment with tezepelumab reduced the annualized asthma exacerbation rate compared to placebo in patients with severe asthma who were receiving medium to high-dose inhaled corticosteroids plus at least one additional controller medication with or without oral corticosteroids.
Acceleron announced interim data from 10 patients enrolled in the 24-week, 25 patient, Phase II, open-label, SPECTRA trial, where treatment with sotatercept peak resulted in improvements in multiple hemodynamic measures as well as exercise tolerance and exercise capacity in patients with pulmonary arterial hypertension.
MeiraGTx, announced 12-month interim data from a 10 patient, open-label, dose-escalation, Phase I/II trial, where treatment with AAV- RPGR resulted in an improvement in mean retinal sensitivity and low light visual mobility in patients with X-linked retinitis pigmentosa (XLRP).
BrainStorm announced that in a 28-week, 189 patient, Phase III trial, treatment with MSC-NTF did not improve the number of patients that achieved at least a 1.25 point improvement per month in their Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score compared to placebo in patients with amyotrophic lateral sclerosis (ALS). In the trial, 34.7% of MSC-NTF patients achieved the ALSFRS-R improvement target compared to 27.7% with placebo. The study may have been underpowered due to more placebo patients achieving the ALSFRS-R improvement than anticipated.
CymaBay announced that in the 3-month, 110 patient, Phase III, ENHANCE trial, 78.2% of patients treated with seladelpar 10 mg achieved a composite of a decrease in alkaline phosphatase and total bilirubin compared to 12.5% on placebo in patients with primary biliary cholangitis and an inadequate response or intolerance to ursodeoxycholic acid. CymaBay is evaluating seladelpar as a treatment for primary biliary cholangitis in the 52-week, 180 patient, Phase III RESPONSE trial (NCT04620733).
BeyondSpring announced that in the 221 patient, Phase III PROTECTIVE 2 trial, grade 4 neutropenia was prevented in 31.5% of patients treated with plinabulin plus pegfilgrastim compared to 13.6% with pegfilgrastim alone in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide.
Published Research Updates
In a 26-week, 243 patient extension of the Phase III trial, 43.5% of patients that continued ravulizumab and 40.3% of patients that switched from eculizumab to ravulizumab achieved LDH normalization. Transfusion was avoided in 73.6% of patients that continued ravulizumab and 67.2% of patients that switched from eculizumab to ravulizumab.
In the 22-week, 8,256 patient, Phase III, GALACTIC-HF trial, 37% of patients treated with omecamtiv mecarbil experienced a composite endpoint of cardiovascular death or heart failure worsening compared to 39.1% with placebo in patients with symptomatic chronic heart failure and an ejection fraction of 35% or less.
In the 516 patient, Phase III, SANDPIPER trial, patients treated with taselisib plus fulvestrant achieved progression-free survival of 7.4 months compared to 5.4 months with fulvestrant alone in patients with estrogen receptor-positive, HER-2-negative, PIK3CA-mutant locally advanced or metastatic breast cancer.
In a 16-week, 272 patient, Phase II trial, the percentage points reduction in LDL over placebo with evinacumab was -56.0 with 450 mg weekly, -52.9 with 300 mg weekly, and -38.5 with 300 mg every two weeks in patients with refractory hypercholesterolemia.
In the 9-month, 1,222 patient, Phase III, SOLOIST-WHF trial, 51% of patients treated with sotagliflozin experienced the composite endpoint of death from cardiovascular causes or hospitalization/urgent visits for heart failure compared to 76.3% with placebo in patients with diabetes and recent worsening heart failure. In the 16-month, 10,584 patient, Phase III, SCORED trial, patients treated with sotagliflozin experienced 5.6 events per 100 patient-years of the composite endpoint of total number of deaths from cardiovascular causes, hospitalizations for heart failure, and urgent visits for heart failure compared to 7.5 events per 100 patient-years with placebo in patients with diabetes and recent worsening heart failure. Both the 22-month SOLOIST-WHF trial and the 33-month SCORED trial were terminated early due to the COVID-19 pandemic and discontinuation of funding from Lexicon and Sanofi.
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