The FDA accepted the NDA for sotorasib for the treatment of patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer, following at least one prior systemic therapy. The PDUFA date for sotorasib’s NDA is 8/16/2021.
The FDA advised Brainstorm in February 2021 that current evidence was not strong enough to support a BLA for NurOwn.
Announced Research Updates
The FDA has placed a partial hold on an open-label extension of Cortexyme’s 54-week, 643 patient, Phase II/III GAIN trial. The trial is evaluating the effect of atuzaginstat on the MMSE score in 570 patients with mild to moderate Alzheimer's disease. The hold was ordered due to hepatic adverse events. No new patients will be enrolled in the GAIN trial and atuzaginstat will be discontinued in the trial extension. Current GAIN patients will complete atuzaginstat treatment with results expected by the end of 2021.
Lilly announced that in the 52-week, 1,444 patient, Phase III SURPASS-3 trial, treatment with tirzepatide reduced HbA1c by 1.93% to 2.37% and body weight by 7.5 Kg to 12.9 kg compared to an HbA1c decrease of 1.34% and 2.3 Kg gain with insulin degludec in insulin-naive patients with type 2 diabetes and inadequate glycemic control on stable doses of metformin with or without an SGLT-2 inhibitor. Lilly announced that in the 40-week, 475 patient, Phase III SURPASS-5 trial, treatment with tirzepatide reduced HbA1C by 2.23% to 2.59% and body weight by 6.2 Kg to 10.9 kg compared to an HbA1c decrease of 0.93% and 1.7 Kg gain with placebo in insulin-naive patients with type 2 diabetes.
Zymeworks announced that in a 280 patient, Phase II, open-label trial, treatment with zanidatamab alone resulted in an overall response rate (ORR) of 33% and 54% when used in combination with paclitaxel or capecitabine in patients with HER2-expressing gastroesophageal adenocarcinoma.
Immunic announced that in a 24-week, 30 patient, open-label, Phase II trial, only 18 patients were enrolled in the trial due to COVID19 restrictions. Of the 18 patients, only 11 completed treatment and had data available. Three of 11 patients treated with vidofludimus achieved a reduction of ALP of at least 25, while their AST increase at week 24 was no more than 33%, in patients with primary sclerosing cholangitis.
Dermavant announced interim results from 520 patients who completed either the PSOARING 1 or PSOARING 2, Phase III, tapinarof trials and were enrolled in the 40-week open-label, long-term extension, Phase III, PSOARING 3 trial. 57.3% of the study population achieved a PGA score of clear or almost clear in patients with plaque psoriasis, treated with tapinarof, who started the trial with a PGA score of two or more.
Published Research Updates
In a 165 Chinese patient, Phase II study anlotinib demonstrated an average PFS of 3.02 months compared to 1.41 months with placebo in patients with recurrent or metastatic esophageal squamous cell carcinoma.
In a 24-week, 695 patient, Phase IIb trial, treatment with lorecivivint 0.07 mg decreased the Pain Numeric Rating Scale (NRS) 0.7 points and 0.23 mg decreased the score by 0.82 points more than placebo in patients with knee osteoarthritis. Lorecivivint 0.23 mg also improved both the WOMAC pain (7.36 point decrease) and function (7.99 point decrease) scores. Lorecivivint 0.03 mg and 0.15 mg did not improve the Pain NRS or WOMAC scores compared to placebo.
In the 52-week, 331 patient, Phase III, ADVOCATE trial, 72.3% of patients treated with avacopan achieved remission (BVAS score of zero and no glucocorticoid treatment for anti-neutrophil cytoplasmic antibody-associated vasculitis for at least the preceding four weeks) at 26 weeks compared to 70.1% with corticosteroids in patients with antineutrophil cytoplasmic antibody–associated vasculitis. 65.7% of avacopan patients remained in remission at 52 weeks compared to 54.9% with glucocorticoids.
A pooled analysis of 925 patients that participated in the MUSE, TULIP-1 and TULIP-2 trials found the most common adverse events with anifrolumab compared to placebo were infections (69.7% vs 55.4%).
In a 24-week, 40 patient, open label, trial, treatment with tofogliflozin reduced the MRI-proton density fat fraction 4.12% compared to a 7.54% decrease with pioglitazone in Japanese patients with non-alcoholic fatty liver disease and type 2 diabetes.
In the 444 patient, Phase III, open-label, TAHOE trial, treatment with rovalpituzumab tesirine resulted in an overall survival of 6.3 months compared to 8.6 months with topotecan in patients with DLL3-high advanced or metastatic SCLC. AbbVie stopped enrollment in the TAHOE trial early, after an interim review by the independent data monitoring committee found shorter overall survival with rovalpituzumab tesirine as second line treatment.
Astellas announced data after 12 weeks from 1,028 patients enrolled in the Phase III SKYLIGHT 1 and SKYLIGHT 2 trials, where treatment with fezolinetant reduced the frequency and severity of vasomotor symptoms (VMS) compared to placebo in patients with moderate to severe VMS.
In a 52-week, 378 patient, Phase II trial, treatment with tilavonemab did not improve the Progressive Supranuclear Palsy Rating Scale score compared to placebo in patients with progressive supranuclear palsy. The trial was discontinued after a futility analysis failed to find a benefit with tilavonemab.
In the 456 patient, Phase II ICON6 trial, treatment and maintenance with cediranib improved progression-free survival compared to placebo in patients with relapsed platinum-sensitive ovarian cancer. The ICON6 trial was redesigned while the trial was going on, so overall survival was underpowered and did not show a benefit compared to placebo.
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