Pipeline News and Updates
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Right to Try Update
The FDA has proposed requirements for the annual report for any investigational drug provided to eligible patients under the Right to Try Act. The FDA is accepting comments on the proposed rule until 9/22/2020. Regulatory Update The FDA approved brexucabtagene autoleucel (Tecartus, Gilead Sciences), on 7/24/2020, for the treatment of relapsed or refractory mantle cell lymphoma. Brexucabtagene autoleucel was approved with a boxed warning and REMS program due to the risks of CRS and neurologic toxicities. Brexucabtagene autoleucel is a one-time treatment and Gilead has priced the drug at $373,000, the same price as its other CAR-T cell therapy axicabtagene ciloleucel (Yescarta). The FDA approved abametapir (Xeglyze, Dr. Reddy's Laboratories), on 7/24/2020, for the treatment of head lice. The FDA designated MyoKardia’s mavacamten a Breakthrough Therapy for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy. The FDA accepted the NDA for Aurinia Pharmaceuticals’ voclosporin for the treatment of lupus nephritis and set a PDUFA date of 1/22/2021. CHMP (European new drug review committee) recommended approval of GlaxoSmithKline’s belantamab mafodotin for the treatment of relapsed/refractory multiple myeloma, filgotinib for the treatment of rheumatoid arthritis, and bupivacaine/meloxicam for the treatment of postoperative pain. Announced Research Updates Genfit is discontinuing development of elafibranor for NASH resolution without worsening fibrosis. The company will focus the drug’s development efforts on primary biliary cholangitis. Roche discontinued development of the investigational autism treatment, balovaptan, in July 2020. UCB announced that in the 16-week, 743 patient, Phase IIIB, BE RADIANT trial, treatment with bimekizumab was superior to secukinumab in moderate-to-severe chronic plaque psoriasis patients achieving PASI 100. Published Research Updates In the 237 patient, Phase III RECOVER trial, 20.3% of patients treated with eflapegrastim developed severe neutropenia compared to 23.5% with pegfilgrastim during the first cycle of docetaxel/cyclophosphamide treatment of breast cancer. Regulatory Update
The FDA rejected leronlimab and requested additional analysis of existing data. An FDA review of clinical data for belantamab mafodotin revealed safety concerns due to 71% of patients developing keratopathy, with 44% of patients experiencing severe symptoms. However, the FDA Oncologic Drugs Advisory Committee voted 12-0 to recommend approval with a REMS program to educate patients and physicians about the risk. An FDA review of terlipressin questioned whether clinical data on hepatorenal syndrome type 1 reversal demonstrated decreased mortality, dialysis or intensive care days. However, the FDA Cardiovascular and Renal Drugs Advisory Committee voted 8-7 to recommend approval. The FDA accepted the NDA for vericiguat for reducing the risk of cardiovascular death and heart failure hospitalization following a worsening heart failure event in patients with symptomatic chronic heart failure with reduced ejection fraction, in combination with other heart failure therapies. and set a PDUFA date of 1/20/2021. The FDA accepted the NDA for the treatment of spasticity in multiple sclerosis patients and set a PDUFA date for 12/29/2020. The FDA designated mosunetuzumab a Breakthrough Therapy for the treatment of relapsed or refractory follicular lymphoma who have received at least two prior systemic therapies. Roche licensed pralsetinib from Blueprint Medicines for $675 million. Announced Research Updates Spark announced 2 to 3.3 year interim results from 12 patients enrolled in a Phase I/II trial, where SPK-8011 reduced the annual bleeding rate by 91% and infusions of FVIII by 96% in patients with Hemophilia A. Aprea announced that in a 52 patient, Phase II trial, treatment with APR-246 and azacitidine resulted in an overall response rate of 76% in patients with TP53-mutant myelodysplastic syndrome or acute myeloid leukemia. Lilly announced that in the 52-week, 1,465 patient, Phase III, OASIS-2 trial, 83.1 to 83.3% of patients treated with mirikizumab achieved a Static Physician's Global Assessment (sPGA) of 0 or 1 with at least a 2-point improvement from baseline compared to 68.5% with secukinumab in patients with moderate to severe plaque psoriasis. In the same trial, 81.4 to 82.4% of mirikizumab patients achieved a 90% or > improvement in Psoriasis Area and Severity Index (PASI 90) compared to 69.4% with secukinumab. Amplyx announced that in a 20 patient, Phase II trial, 80% of patients treated with fosmanogepix achieved clearance of Candida from blood cultures with no additional antifungal treatment, and survival at the end of study treatment, in patients with candidemia. Published Research Updates In a 3-month, 8 patient, Phase III, open-label trial, treatment with setmelanotide resulted in a 5.5% decrease in weight in patients with Bardet-Biedl syndrome. Seven of the patients completed a 9-month extension and achieved a 16.3% weight loss at 12 total months. In a 4-week, 152 patient, Phase II trial, patients treated with tradipitant had a 1.2 decrease in their patient reported nausea score compared to a 0.7 decrease with placebo in patients with idiopathic and diabetic gastroparesis. Patients who were experiencing nausea and vomiting at enrollment achieved a 1.4 point decrease. Regulatory Update
The FDA approved the oral combination of decitabine and cedazuridine (Inqovi, Astex Pharmaceuticals), on 7/7/2020, for treatment of myelodysplastic syndrome or chronic myelomonocytic leukemia. The EU approved J&J’s Ad26.ZEBOV (Zabdeno) and MVA-BN-Filo (Mvabea), a two dose vaccine regimen for the prevention of Ebola virus infection. The FDA designated Concert Pharmaceuticals’ CTP-543 a Breakthrough Therapy for the treatment of moderate-to-severe alopecia areata. The FDA accepted the BLA for AstraZeneca/Leo Pharma’s tralokinumab for treatment of moderate-to-severe atopic dermatitis. Biogen filed a BLA for aducanumab for the treatment of Alzheimer’s Disease. ChemoCentryx submitted an NDA for avacopan for the treatment of patients with ANCA-associated vasculitis. Announced Research Updates Idorsia announced that in a 3-month, 924 patient, Phase III trial, treatment with daridorexant 25 mg improved sleep maintenance and total sleep time compared to placebo in patients with insomnia. The 25 mg dose did not improve sleep onset and daytime functioning compared to placebo and the 10 mg dose did not improve any of the four measures. Bayer announced that in the 48-month, 5,734 patient, Phase III, FIDELIO-DKD trial, patients treated with finerenone had a lower incidence of kidney failure and renal death compare to placebo in patients with chronic kidney disease and type 2 diabetes. Kidney failure was defined as a sustained decrease of estimated glomerular filtration rate (eGFR) greater than or equal to 40 percent from baseline over a period of at least four weeks. Atox Bio announced that in the 28-day, Phase III, ACCUTE trial, involving 290 patients with severe necrotizing soft tissue infection, treatment with reltecimod did not improve a composite of morbidity, no more than three debridements, no further amputations, and resolution of organ dysfunction compared to placebo (48.6% vs 39.9%) in a modified Intent-to-Treat responder analysis (primary endpoint), but demonstrated a benefit in a per protocol analysis (54.3% vs 40.3%) after 17 patients were excluded. Published Research Updates In a 15-month, 891 patient, Phase III trial, treatment with hydromethylthionine did not improve the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) or the Alzheimer's Disease Co-operative Study-Activities of Daily Living Inventory (ADCS-ADL) scores compared to placebo in patients with mild to moderate Alzheimer's disease. Most patients were receiving acetylcholinesterase inhibitors, memantine or both. A separate sub-group analysis of hydromethylthionine monotherapy suggested a benefit on cognitive scores. In a 16-week, 143 patient, Phase III trial, treatment with topical agents plus nemolizumab decreased the pruritus VAS score by 42.8% compared to a 21.4% decrease with only topical agents in Japanese patients with atopic dermatitis and moderate-to-severe pruritus and an inadequate response to topical agents. In a 12-month, 50 patient, open-label, Phase II trial, treatment with axalimogene resulted in overall survival of 38% in patients with persistent or recurrent metastatic carcinoma of the cervix. In a 95 patient, Phase II trial, treatment with margetuximab plus pembrolizumab resulted in an overall response rate of 18.48% in patients with metastatic HER2-positive gastroesophageal adenocarcinoma previously treated with trastuzumab and chemotherapy. In the 48-week, 374 patient, Phase II ARPEGGIO trial, treatment with laquinimod did not differ from placebo in the percentage brain volume change compared to placebo in primary progressive MS patients. Regulatory Update
The FDA approved triheptanoin (Dojolvi, Ultragenyx) on 6/30/2020 as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). Ultragenyx set WAC for triheptanoin at $4,875 per 500 ml vial, which indicates an average annual cost of $138,000. The FDA has approved remimazolam (Byfavo, Acacia) on 7/2/2020 for the induction and maintenance of procedural sedation in adults undergoing surgical procedures lasting 30 minutes or less. The FDA approved fostemsavir (Rukobia, ViiV Healthcare), on 7/3/2020, in combination with other antiretrovirals, for the of treatment heavily treated adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations. The FDA rejected Heron Therapeutics Bupivacaine, Meloxicam (HTX-011) for a second time and requested additional data regarding exposure of excipients in preclinical reproductive toxicology studies and a process change for the allowable level of an impurity during manufacturing. Mezzion submitted an NDA for udenafil to improve the physiology of patients 12 years of age and older with single ventricle heart disease (SVHD) who have undergone a Fontan operation. The FDA designated Nodic Nanovector Lutetium (177lu) lilotomab satetraxetan, a Fast Track therapy for treatment of adults with relapsed or refractory marginal zone lymphoma who received at least two prior systemic therapies. Announced Research Updates Iterum Therapeutics announced that in the 1,670 patient, Phase III SURE 1 trial, oral sulopenem/probenecid for five days did not meet non-inferiority for test of cure compared to oral ciprofloxacin for three days (66.8% vs 78.6%) in the treatment of uncomplicated urinary tract infections in patients infected with quinolone-susceptible bacteria. However, in patients infected with quinolone-resistant organisms, sulopenem/probenecid was superior to ciprofloxacin at the test of cure visit (62.6% vs 36%). Hua announced that in a 24-week, 766 patient, Phase III trial, dorzagliatin added to metformin lowered HbA1c 0.66% compared to metformin monotherapy (a decrease of 1.02% vs 0.36%) in Chinese patients with type 2 diabetes. Helsinn and MEI Pharma discontinued a Phase III trial evaluating pracinostat in combination with azacitidine in the treatment of acute myeloid leukemia patients who are unfit to receive standard intensive chemotherapy, when a futility analysis revealed little chance of treatment success. ObsEva announced that in the 24-week, 526 patient, Phase III PRIMROSE 2 trial, heavy menstrual bleeding was reduced to 80/ml or less with the reduction being at least 50% from baseline in 75.5% of patients treated with linzagolix 200 mg and hormone add-back therapy and 56.4% with linzagolix 100 mg monotherapy compared to placebo in patients with uterine fibroids and heavy menstrual bleeding. Published Research Updates An analysis of post-marketing data from Japan found that 12.6% of 6,451 patients that took tofogliflozin experienced an ADR, with the most commonly reported events being hypoglycemia, polyuria/pollakiuria, volume depletion-related disorders, urinary tract infections, genital infections and skin diseases. In a 52-week, 62 patient, Phase III trial, treatment with weekly somapacitan was similar to daily growth hormone in the reduction of visceral, subcutaneous and total adipose tissue in Japanese adults with growth hormone deficiency |
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