Regulatory Update
The FDA approved sacituzumab govitecan (Trodelvy, Immunomedics) on 4/22/2020 to treat resistant metastatic triple-negative breast cancer. Trodelvy has a boxed warning about the potential risks for severe diarrhea and neutropenia. Biogen has delayed plans to file a BLA for aducanumab (for the treatment of Alzheimer’s disease) from early 2020 to 3Q20. Announced Research Updates Scynexis announced that in the 188 patient, Phase III, VANISH-306 trial, treatment with ibrexafungerp resulted in clinical cure at day 10 in 68.3% of patients compared to an unknown percentage that received placebo in female patients with vulvovaginal candidiasis. Scynexis plans to submit an NDA for ibrexafungerp in second half of 2020. Cara announced that in the 12-week, 473 patient, Phase III, KALM-2 trial, 54% of patients treated with difelikefalin had a 3-point or > improvement in the weekly mean WI-NRS compared to 42% with placebo in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus. Cara plans to submit an NDA for difelikefalin by the end of 2020. Myovant announced that in the 24-week, 600 patient, Phase III, SPIRIT 2 trial, treatment with relugolix plus estradiol and norethindrone reduced dysmenorrhea (75.2% vs 30.4%) and non-menstrual pelvic pain (66.0% vs 42.6%) compared to placebo in patients with with pain associated with endometriosis. Myovant submitted an NDA for relugolix for the treatment of prostate cancer in April 2020 and plans to submit an NDA for use of the drug in the treatment of uterine fibroids in May 2020. GSK announced that interim data from 70 patients enrolled in the Phase III GARNET trial, suggested a 43% objective response rate with treatment with dostarlimab in patients with previously treated recurrent or advanced mismatch repair (MMR)-deficient endometrial cancer. Published Research Updates In a 12-week, 708 patient, Phase III trial, 18.4% of patients treated with clascoterone 1% cream achieved an Investigator's Global Assessment score of 0 (clear) or 1 (almost clear), and a 2-grade or greater improvement, compared to 9% with placebo in patients with moderate or severe facial acne. In a 12-week, 732 patient, Phase III trial, 20.3% of patients treated with clascoterone 1% cream achieved an Investigator's Global Assessment score of 0 (clear) or 1 (almost clear), and a 2-grade or greater improvement, compared to 6.5% with placebo in patients with moderate or severe facial acne. In the 18-month, 95 patient, Phase III, SAkuraStar trial, 70% of patients treated with satralizumab were relapse-free compared to 50% with placebo in patients with neuromyelitis optica spectrum disorder. In a 28-day, 29 patient, Phase I, open-label trial, 78.6% of patients treated with itacitinib 200 mg plus corticosteroids and 66.7% of patients receiving 300 mg plus corticosteroids achieved an overall response in patients who developed acute graft-versus-host disease following allogeneic hematopoietic cell transplantation. Regulatory Update
The FDA approved pemigatinib (Pemazyre, Incyte) on 4/17/2020 for the treatment of resistant advanced cholangiocarcinoma. The FDA approved tucatinib (Tukysa, Seattle Genetics) on 4/17/2020 the treatment of adults with advanced HER2-positive breast cancer who have received at least one prior line of therapy. WAC has been set at $18,500 per month; with a treatment duration of 6-months, a course of treatment will be $111,000 per patient. Due to restriction in social contact, a virtual launch for the drug is planned. The FDA accepted the BLA for Regeneron Pharmaceuticals’ REGN-EB3 on 4/16/2020 for the treatment of Ebola virus infections and set a PDUFA date of 10/25/2020. REGN-EB3 was designated as both an Orphan Drug and Breakthrough Therapy. The FDA granted Orphan Drug Designation to Ascendis Pharma’s lonapegsomatropin, as a treatment for growth hormone deficiency. The FDA has granted Fast Track designations to Hutchison China MediTech (Chi-Med or HCM)’s surufatinib for the treatment of both advanced and progressive pancreatic neuroendocrine tumors and extra-pancreatic tumors. Published Research Updates In a 4-week, 245 patient, Phase II trial, treatment with SEP-363856 decreased the PANSS total score by 17.2 points compared a 9.7 point decrease with placebo in patients hospitalized with an acute exacerbation of schizophrenia. In the 736 patient, Phase III, open-label, EAGLE trial, neither durvalumab monotherapy nor durvalumab plus tremelimumab improved overall survival compared to standard-of-care chemotherapy in patients with platinum-resistant head and neck squamous cell carcinoma. Regulatory Update
The FDA approved selumetinib (Koselugo, AstraZeneca) on 4/10/2020 for the treatment of pediatric patients with neurofibromatosis type 1. The FDA granted sacituzumab govitecan Fast Track status for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a checkpoint inhibitor and a platinum containing chemotherapy. Immunomedics stopped the Phase III, ASCENT trial early, due to a recommendation of the Data Safety Monitoring Committee, which found compelling evidence of efficacy as seen when sacituzumab govitecan improved progression-free survival in patients with metastatic triple-negative breast cancer. The FDA granted Fast Track status to balstilimab for the treatment of cervical cancer. The FDA designated sotatercept a Breakthrough Therapy for the treatment of pulmonary arterial hypertension. Alnylam completed a rolling NDA and submitted an MAA in April 2020 for lumasiran for the treatment of primary hyperoxaluria type 1. AZ is planning to file an NDA for anifrolumab for the treatment of systemic lupus erythematosus by the end of 2020. Published Research Updates In a 12-week, 303 patient, Phase IIb trial, Assessment in Spondyloarthritis International Society 40% (ASAS40) was achieved in 29.5% of patients treated with 16 mg of bimekizumab, 42.6% with 64 mg, 46.7% with 160 mg and 45.9% with 320 mg compared to 13.3% with placebo in patients with Ankylosing Spondylitis. In the 1,118, Phase III MYSTIC trial, tremelimumab plus durvalumab did not improve overall survival or progression free survival compared to a platinum-based chemotherapy in patients with metastatic non-small cell lung cancer. In the 24-week, 381 pateint, Phase III, DISCOVER-1 trial, ACR20 was achieved by 64% of patients treated with guselkumab every four weeks and 64% with treatment given every 8 weeks compared to 33% of placebo patients in patients with psoriatic arthritis. In the 24-week, 739 pateint, Phase III, DISCOVER-2 trial, ACR20 was achieved by 59% of patients treated with guselkumab every four weeks and 52% with treatment given every 8 weeks compared to 22% of placebo patients in biologic-naive patients with psoriatic arthritis. Regulatory Update
The FDA accepted the BLA for remestemcel-L for the treatment of children with steroid-refractory acute graft versus host disease and set a PDUFA date of 9/30/2020. Remestemcel-L is approved as Temcell in Japan. Blueprint completed a rolling NDA submission for pralsetinib for the treatment of RET fusion-positive non-small cell lung cancer. Rhythm completed a rolling submission for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. BMS and Celgene submitted a BLA for idecabtagene vicleucel for the treatment of refractory multiple myeloma. Announced Research Updates Verona announced that in a 40 patient, Phase II, open-label trial, treatment with ensifentrine pressurized metered-dose inhaler increased FEV1 47 mL to 391 mL with doses of 300 mcg to 6000 mcg dose, in patients with moderate to severe COPD. Published Research Updates In a 24-week, 849 patient, Phase III trial, patients treated with tanezumab 5 mg had a greater decrease in the WOMAC pain and physical function scores and the Patient’s Global Assessment of OA (PGA-OA) compared to placebo in patients with osteoarthritis. The 2.5 mg dose of tanezumab had a a greater decrease in the WOMAC pain and physical function scores, but not the PGA-OA compared to placebo. In the 60 patient, Phase II, open-label, ZUMA 2 trial, a single infusion of KTE-X19 resulted in an overall response rate of 93% in patients with relapsed or refractory mantle cell lymphoma. In a 24-hour, 151 patient, Phase III, Japanese-registered trial, 55% of patients treated with landiolol achieved a heart rate of 60-94 bpm compared to 33% in the placebo group in patients with sepsis-related tachyarrhythmia. |
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