Pipeline News and Updates
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New Drugs Added to Knowledgebase
Last week we added 10 new drugs to the knowledgebase:
Regulatory Update The FDA rejected Levo Therapeutics’ intranasal carbetocin for the treatment of Prader-Willi syndrome and requested an additional Phase III trial. Pfizer announced the FDA rejected somatrogon, but did not provide a reason for the rejection. The FDA rejected Merck’s gefapixant for the treatment of chronic cough and requested additional information related to measurement of efficacy. The FDA delayed the PDUFA date by three months for Bluebird Bio’s betibeglogene autotemcel, to treat transfusion-dependent beta-thalassemia, to allow more time to review additional clinical information The FDA delayed the PDUFA date by three months for Bluebird Bio’s elivaldogene tavalentivec, to treat cerebral adrenoleukodystrophy, to allow more time to review additional clinical information The FDA recommended that GenSight conduct an additional trial for lenadogene nolparvovec due to the unexpected bilateral vision improvement seen in unilaterally treated Leber Hereditary Optic Neuropathy patients in the RESCUE, REVERSE and REFLECT trials. Announced Research Updates Results from two Phase III bimekizumab trials; the axial spondyloarthritis BE MOBILE 1 trial and the psoriatic arthritis BE COMPLETE trial Results from the HIMALAYA trial evaluating the addition of tremelimumab to a regimen to treat advanced hepatocellular carcinoma. 18-month data from the HELIOS-A trial evaluating vutrisiran to treat hATTR amyloidosis Published Research Updates Results from the Phase III, ECOSPOR III trial, evaluating SER-109 to prevent recurrent C. difficile infection. Results from the Phase III, GEMSTONE-302 trial, evaluating sugemalimab in the treatment of metastatic squamous and non-squamous non-small cell lung cancer. Results from a triple-negative breast cancer adavosertib trial. Results from an ovarian cancer evaluating cediranib Two Phase III daridorexant insomnia trials Four Phase III faricimab trials. Two evaluating faricimab as a treatment for neovascular age-related macular degeneration and two for diabetic macular edema. Check out our new lower subscription rates and don't miss out on these clinical updates. Access 53 recently approved and available drugs and 22 investigational drugs on the short list for approval.
Last week we added 12 new drugs to the knowledgebase:
The FDA approved abrocitinib (Cibinqo, Pfizer), on 1/14/2022, for the treatment of adults with refractory, moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with other systemic therapies. Like other JAK inhibitors, abrocitinib was approved with a boxed warning regarding the increased risk for serious infections, mortality, malignancy, major cardiovascular events and thrombosis. Announced Research Updates Sarepta announced data from 20 patients enrolled in Part 2 of a 48-week, 41 patient, Phase II trial, where treatment with delandistrogene moxeparvovec improved the NSAA score by two points compared to a 103 patient, weighted external control group (1.3 point increase vs 0.7 point decrease). We also added six new published research updates that subscribers can review
Regulatory Update
The FDA approved daridorexant (Quviviq, Idorsia), on 1/10/2022, for the treatment of insomnia in adults. The FDA accepted the NDA for tebipenem for the treatment of complicated urinary tract infections and set a PDUFA date for 6/27/2022. Announced Research Updates Biosight announced that in a 66 patients Phase IIb trial, treatment with aspacytarabine resulted in a 37% complete response rate in patients with acute myeloid leukemia who are not eligible for standard induction chemotherapy due to advanced age or comorbidities. BioMarin announced the annualized bleeding rate was maintained at 0.8 per year (0.9 during year one and 0.7 during year two) and Factor VIII infusions at 2.6 per year (1.5 during year one and 3.4 during year two) in 112 patients enrolled in the valoctocogene roxaparvovec GENEr8-1 trial that had been followed for 110 weeks. Published Research Updates In the 16-week, 215 patient, Phase III, TIMES 3 trial, adding imeglimin to insulin decreased HbA1c 0.6% at 16 weeks and 0.64% at 52 weeks compared to insulin alone in Japanese patients with type 2 diabetes. In the 714 patient, Phase II/III, RELATIVITY-047 trial, patients treated with relatimab plus nivolumab achieved progression-free survival of 10.1 months compared to 4.6 months with nivolumab alone in patients with previously untreated metastatic or unresectable melanoma. In an updated analysis of tirzepatide for the treatment of type 2 diabetes, ICER found that treatment with tirzepatide reduced HbA1c 2.5% and weight by 10.9 Kg when added to background therapy. Analyzing data from a direct comparison with semaglutide and a network meta-analysis with empagliflozin, tirzepatide has a greater decrease in HbA1c, weight loss, lipids, and blood pressure. Treatment with tirzepatide was associated with an increase in gastrointestinal adverse effects, severe adverse events, and discontinuation compared with semaglutide. Because only indirect data was available, a comparison of adverse events with tirzepatide was not possible. While semaglutide and empagliflozin have shown improvement in cardiovascular (CV) outcomes, the tirzepatide CV outcomes trial has not been completed. Due to the lack of the CV outcomes data, ICER found tirzepatide to provide comparable or better net health benefits to semaglutide and empagliflozin. ICER estimated an annual health-benefit price benchmark range of $5,500 to $5,700 for tirzepatide. Regulatory Update
The FDA approved inclisiran (Leqvio, Novartis), on 12/22/2021, as an adjunct to diet and maximally tolerated statin therapy for the treatment of atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) in patients who require additional lowering of LDL-C.
The FDA approved levoketoconazole (Recorlev, Xeris Biopharma), on 12/30/2021, for the treatment of endogenous hypercortisolemia in patients with Cushing’s syndrome for whom surgery is not an option or has not been curative. Levoketoconazole was approved with a Black Box Warning regarding hepatotoxicity and QT prolongation. The FDA placed a hold on HIV trials using Gilead’s injectable lenacapavir due to concerns regarding the compatibility of vials made of borosilicate glass with lenacapavir solution, which could potentially lead to the formation of sub-visible glass particles. The hold does not affect trials using the oral formulations of lenacapavir. The FDA accepted the NDA for Amylyx Pharmaceuticals’ AMX0035 for the treatment of amyotrophic lateral sclerosis(ALS) and set a PDUFA date for 6/29/2022. Lexicon submitted an NDA for sotagliflozin to reduce the risk of cardiovascular complications in heart failure patients with type 2 diabetes. The FDA granted Biogen and Eisai’s lecanemab a fast-track designation as a treatment of early Alzheimer's disease. Published Research Updates In a 155 patient, Phase II trial, treatment with balstilimab and zalifrelimab resulted in a 25.6% objective response rate (ORR) in patients with recurrent and/or metastatic cervical cancer, who had relapsed after prior platinum-based chemotherapy. In the 28-day, 439 patient, Phase III, GRAVITAS-301 trial, adding itacitinib to corticosteroids did not improve the overall response rate compared to corticosteroids alone in patients with treatment-naive acute graft-versus-host disease. In a 28-week, 189 patient, Phase III trial, treatment with MSC-NTF did not improve the number of patients that achieved a 1.25 point improvement or > per month in their Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score compared to placebo (33% vs 28%) in patients with amyotrophic lateral sclerosis (ALS). In the 16-week, 332 patient, Phase III, BE MOBILE 2 trial, more patients treated with bimekizumab achieved ASA40 compared to placebo in patients with Ankylosing Spondylitis who had an inadequate response to an NSAID or TNF-inhibitor. In the 92 patient, Phase III, REALITY trial, treatment with apatinib resulted in a PFS of 22.2 months compared to 4.5 months with placebo in patients with radioactive iodine-refractory differentiated thyroid cancer. In the 127 patient, Phase III TreeTopp trial, adding varlitinib to capecitabine did not improve progression-free survival or overall response rate compared to capecitabine alone in patients with biliary tract cancer. |
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