The FDA granted GlycoMimetics’ rivipansel a Rare Pediatric Disease designation for the treatment of sickle cell disease.
The FDA’s Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Advisory Committee met in a joint session to review ALKS 3831, a combination of samidorphan and olanzapine. The committees voted 16-1 that the combination would reduce weight gain, 13-3 with one abstention that the drug was safe and 11-6 for labeling proposed by Alkermes that recommends a contraindication against the use of ALKS 3831 in patients who are opioid-dependent or chronically using opioids.
Announced Research Updates
Amgen announced that in the 126 patient, Phase II part of the CodeBreak 100 trial, treatment with sotorasib resulted in an ORR similar to that seen in the Phase I part of the study in patients with KRAS G12C-mutant advanced NSCLC, who had failed a median of two prior lines of anti-cancer therapies.
Santhera discontinued the Phase III SIDEROS trial after an interim analysis found no improvement with idebenone at 18 months in forced vital capacity % predicted (FVC%p) in patients with Duchenne muscular dystrophy. Santhera pulled the MAA for idebenone and discontinued development after failure of the SIDEROS trial.
J&J announced 9-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 central visual field progression rate in patients with X-linked retinitis pigmentosa (XLRP).
Peregrine announced that in a 36 patient, Phase II trial, treatment with bavituximab plus pembrolizumab resulted in an objective response rate of 19.4% in patients with advanced gastric or gastroesophageal junction cancer.
LSKB and Hengrui announced that in the 313 patient, Phase III, ACTIVE trial, treatment with apatinib plus gefitinib resulted in a progression-free survival of 13.7 months compared to 10.2 months with gefitinib alone in patients with non-small cell lung cancer.
Amgen, Cytokinetics and Servier announced that in the 20-week, 8,256 patient, Phase III, GALACTIC-HF trial, treatment with omecamtiv mecarbil reduced a composite endpoint of cardiovascular death or heart failure worsening compared to placebo in patients with heart failure with reduced ejection fraction (HFrEF).
Pfizer and Opko announced that in a 12-week, 44 patient, Phase III, open-label, crossover trial, once-weekly somatrogon injections improved the mean overall Life Interference total score compared to once-daily somatropin (8.63 vs 24.13 in pediatric patients with growth hormone deficiency.
Lilly announced that in a 40-week extension of the Phase II SERENITY trial, treatment with either intravenous or subcutaneous mirikizumab resulted in 59% of patients achieving a 50% reduction in their SES-CD score and 46% achieving PRO remission (stool frequency of 2.5 stools per day or less and abdominal pain no worse than baseline).
Published Research Updates
In the 6-week, 213 patient, Phase IIb, dose-ranging, CONDUCT trial, 21% of patients treated with two topical endoscopic administrations of the highest dose of cobitolimod (2 x 250 mg) achieved clinical remission compared to 7% with placebo in patients with refractory left-sided moderate to severe active ulcerative colitis. Three lower doses of cobitolimod (2 x 31 mg, 2 x 125 mg and 4 x 125 mg) did not achieve an improvement compared to placebo in the CONDUCT trial.
In a 127 patient, Phase II trial, treatment with icotinib plus radiotherapy resulted overall survival of 24 months compared to 16.3 months with radiotherapy alone in patients with esophageal squamous cell carcinoma.
In an 18-month, 96 patient, Phase II trial, 54% of patients treated with xevinapant plus cisplatin achieved locoregional control rate compared to 33% with placebo in patients with high-risk locally advanced squamous cell carcinoma of the head and neck. Debiopharm announced three-year results from the Phase II trial, where the overall survival rate with xevinapant was 66%, compared to 51% with placebo in the Phase II trial.
In the 48-week, 217 patient, Phase IIb, ENCORE-LF trial, emricasan did not improve all-cause mortality, reduce new decompensation events, improve the MELD-NA score or improve liver function compared to placebo in patients with decompensated NASH cirrhosis.
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