Pipeline News and Updates
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Regulatory Update
The FDA approved tezepelumab (Tezspire, Amgen & AstraZeneca), on 12/17/2021), as add-on maintenance treatment of severe asthma. The FDA approved efgartigimod, on 12/17/2021, for the treatment of generalized myasthenia gravis that is anti-acetylcholine receptor antibody positive. The FDA has placed a hold on patients under 18 years in trials evaluating lovotibeglogene autotemcel in the treatment of sickle cell disease, due to anemia in an adolescent patient that was treated 18 months ago. Enrollment of patients 18 and older is not affected. The FDA placed a hold on fordadistrogene movaparvovec trials for the treatment of Duchenne muscular dystrophy,after the death of patient in a Phase Ib trial. The FDA placed a hold on all islatravir HIV treatment trials due to the drop in T-cell counts. Ipsen licensed world-wide marketing and development rights from Genfit for elafibranor in the treatment for primary biliary cholangitis. Announced Research Updates Compass Pathways announced that in a 3-week, 19 patient, open-label trial, treatment with a single dose of psilocybin 25 mg decreased the MADRS score by 14.9 points in patients with treatment-resistant depression, who were receiving an SSRI. Sangamo announced that in the 11 patient, Phase I/II, ALTA trial, treatment with giroctocogene fitelparvovec results in an annualized bleeding rate of 0 after 1 year and 1.4 after 2 years with Factor VIII activity of 27.5% of normal in patients with severe hemophilia A. Lilly announced that in the 618 patient, BRUIN trial, treatment with pirtobrutinib resulted in an overall response rate of 51% in 134 MCL patients and 68% in 252 chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) patients. Actinium announced that in the 151 patient, Phase III, SIERRA trial, 100% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant compared to 17% that received salvage chemotherapy in patients with active, relapsed or refractory acute myeloid leukemia, eligible to receive a bone marrow transplant. Sanofi announced that in the eight-month, 120 patient, open-label, Phase III, ATLAS-A/B trial, the ABR for bleeding episodes requiring treatment was 0 for fitusiran compared to 21.8 for treatment with on-demand factor therapy in patients with hemophilia A or B with no inhibitors. 50.6% of fitusiran patients experienced no bleeding of any type compared to only 5% with on-demand factor therapy. Sanofi announced that in the eight-month, 57 patient, open-label, Phase III, ATLAS-INH trial, the ABR for bleeding episodes requiring treatment was 0 for fitusiran compared to 16.8 for treatment with on-demand bypassing agents in patients with hemophilia A or B with inhibitors to factor VIII or IX. 65.8% of fitusiran patients experienced no bleeding of any type compared to only 5.3% with on-demand bypassing agents. Kura announced that in a 65 patient, Phase II trial, treatment with tipifarnib resulted in a 56% overall response rate in patients with relapsed or refractory T-cell lymphoma. Galera announced that in the 455 patient, Phase III, ROMAN trial, 54% of patients treated with avasopasem experienced severe oral mucositis compared to 64% with placebo in patients with locally advanced head and neck cancer receiving seven weeks of radiotherapy plus cisplatin. Galera originally announced the results of the ROMAN trial were not significant, but after an error made by the contract research organization (CRO) was identified and corrected, the results achieved significance. Lilly announced that in the 12-week, 1,044 patient, Phase III, LUCENT-2 trial, more patients receiving mirikizumab maintained remission compared to placebo in patients with moderate-to-severe active UC, who had achieved remission with mirikizumab in the LUCENT-1 trial. Cidara announced that in the 30-day, 187 patient, Phase II, ReSTORE trial, treatment with once-weekly rezafungin was non-inferior to daily caspofungin for mortality at day 30 (23.7% vs 21.3%) and global cure at day 14 (59.1% vs 60.6%) in patients with candidemia and/or invasive candidiasis. Johnson & Johnson announced that in the 97 patient, open-label, Phase Ib/II, CARTITUDE-1 trial, treatment with ciltacabtagene autoleucel resulted in an overall response rate (ORR) of 98% with a progression-free rate of 61% and overall survival rate of 74% at 21.7 months in heavily pre-treated patients with refractory or relapsed multiple myeloma. Johnson & Johnson announced 14.3-month results from 20 patients enrolled in Cohort A of the CARTITUDE-2 trial, where treatment with ciltacabtagene autoleucel resulted in an ORR of 95% in heavily pre-treated patients with multiple myeloma. The ORR was 95% after 10.6 months in 91 multiple myeloma patients enrolled in Cohort B who were in early relapse after initial therapy. Novartis announced that in 2,119 patients enrolled in the 12-week, Phase III, PEARL 1 and 2 trials, treatment with ligelizumab decreased the Urticaria Activity Score (UAS) compared to placebo, but not omalizumab in patients with chronic spontaneous urticaria. Summit Therapeutics announced that in the 30-day, 759 patient, Phase III, Ri-CoDIFy trial, treatment with ridinilazole did not improve the Sustained Clinical Response rate compared to vancomycin in patients with Clostridium difficile infection. Ri-CoDIFy combined 355 patients from the Ri-CoDIFy 1 trial with 404 patients from the Ri-CoDIFy 2 trial. PhaseBio discontinued the Phase IIb, VIP trial evaluating pemziviptadil in the treatment of pulmonary arterial hypertension due to the impact of COVID-19 on drug manufacturing and trial enrollment. Aldeyra announced that in the 12-week, 422 patient, Phase III, TRANQUILITY trial, treatment with reproxalap did not decrease ocular redness compared to placebo in patients with dry eye disease Regulatory Update
Merck is pausing enrollment in the Phase III IMPOWER 22 and 24 trials, which are evaluating once-monthly islatravir for pre-exposure prophylaxis in high risk patients, due to a decrease in total lymphocyte and CD4+ T-cell counts. The EMA approved abrocitinib for the treatment of moderate-to-severe atopic dermatitis. The EMA has accepted the MAA for olipudase alfa or the treatment of Niemann-Pick disease type A and type B. Announced Research Updates GSK announced that in the 28-week, 614 patient, Phase III, ASCEND-NHQ trial, 77% of patients treated with daprodustat achieved at least a 1 g/dL increase in hemoglobin compared to 18% who received placebo in non-dialysis dependent CKD patients with anemia. Syndax announced that in a 354 patient, Phase III trial, adding entinostat to exemestane increased PFA to 6.32 months compared to 3.72 months with exemestane alone in Chinese patients with advanced HR+, HER2-, breast cancer. UniQure announced that at 18-months, FIX activity was 36.9% of normal with an ABR requiring treatment of 0.83, after treatment with etranacogene dezaparvovec, in Hemophilia B patients enrolled in the 12-month, Phase III, HOPE-B trial. Jiangsu HengRui announced that at two-years treatment with pyrotinib plus capecitabine resulted in a 66.6% overall survival compared to 58.8%% with lapatinib plus capecitabine in the PHOEBE trial Roche announced that in a 2.5 year follow-up of the Phase II, CITYSCAPE trial, the combination of tiragolumab plus atezolizumab maintained an improvement in ORR (38.8% vs 20.6%) and PFS (5.6 months vs 3.9 months). Moderna announced 29-day results from a 180 patient, Phase I trial, where immunization with mRNA-1010 elicited antibody titers that neutralized all H1N1 and H3N2 influenza strains tested. BeiGene announced that in the 263 patient, Chinese Phase III, RATIONALE 309 trial, patients treated with tislelizumab plus gemcitabine and cisplatin achieved progression free survival of 9.2 months compared to 7.4 months with gemcitabine and cisplatin alone in patients with recurrent or metastatic nasopharyngeal cancer. Vifor and Angion announced that in the 12-month, 253 patient, Phase II, GUARD trial, treatment with ANG-3777 did not reduce the increase in serum creatinine compared to placebo (8.4% vs 7.3%) in patients undergoing cardiac surgery involving cardiopulmonary bypass at risk for developing acute kidney injury. Roche announced that in a 62 patient, open-label, Phase I/Ib trial, treatment with mosunetuzumab induced a complete response in 60% and progression-free survival of 17.9 months in patients with relapsed or refractory follicular lymphoma. Roche announced that in a 262 patient, open-label, Phase I/Ib trial, treatment with mosunetuzumab plus polatuzumab induced an objective response rate (ORR) of 65% and a CR rate of 48.3% in patients with relapsed or refractory B-Cell Non-Hodgkin Lymphoma. UCB announced that in the 43-day, 200 patient, Phase III, MycarinG trial, treatment with rozanolixizumab resulted in an improvement in the MG-ADL score compared to placebo in patients with myasthenia gravis. Published Research Updates In the 52-week, 714 patient, Phase III, open-label, TIMES 2 trial, HbA1c was reduced 0.46% with imeglimin alone, 0.56% to 0.92% when combined with oral therapy, 0.12% with a GLP receptor agonist and 0.92% with a DPP-4 inhibitor in Japanese patients with type 2 diabetes. In the 53 patient, open label Phase I, EXPLORER trial, treatment with avapritinib resulted in an ORR of 75% in patients with advanced systemic mastocytosis. Interim data from 32 patients enrolled in the 103 patient, open label Phase II, PATHFINDER trial, suggested that treatment with avapritinib resulted in an ORR of 75% in patients with advanced systemic mastocytosis. In the 12-week, 510 patient, Phase III, PSOARING 1 trial, treatment with tapinarof 1% daily resulted in achievement of the Physician Assessment (PGA) score of clear or almost clear and at least a 2-point improvement in the PGA score in 35.4% of patients compared to 6% with placebo in patients with plaque psoriasis. In the 12-week, 515 patient, Phase III, PSOARING 2 trial, treatment with tapinarof 1% daily resulted in achievement of the PGA score of clear or almost clear and at least a 2-point improvement in the PGA score in 40.2% of patients compared to 6.3% with placebo in patients with plaque psoriasis. In a 26-week, 157 patient, open-label extension of the 4-week, Phase II trial, treatment with ulotaront decreased the PANSS total score by 22.6 points. In a 141 patient, open-label, Phase II trial, adding andecaliximab to nivolumab did not improve the objective response rate compared to nivolumab alone (10% vs 7%) in patients with pretreated metastatic gastric or gastroesophageal junction adenocarcinoma. Regulatory Update
The FDA rejected plinabulin for the prevention of chemotherapy-induced neutropenia and requested an additional clinical trial. The FDA delayed the PDUFA date for pacritinib to treat myelofibrosis with severe thrombocytopenia, by three months, to allow additional time to review newly submitted clinical data. The new PDUFA date is 2/28/2022. Announced Research Updates Vertex announced that in a 13-week, 13 patient, open-label, Phase II trial, treatment with VX-147 resulted in a 47.6% reduction in the urine protein to creatinine ratio (UPCR) in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS). ImmunoGen announced that in the 106 patient, open-label, Phase III, SORAYA trial, treatment with mirvetuximab soravtansine resulted in a 32.4% ORR in patients with platinum-resistant ovarian cancer whose tumors express high levels of folate receptor alpha. SAB Biotherapeutics announced that in a 28-day, 60 patient, Phase IIa trial, treatment with SAB-176 reduced the nasopharyngeal viral load and influenza signs and symptoms compared to placebo in patients challenged with a pandemic influenza virus strain (pH1N1). Kazia announced that in a 30 patient, Phase II trial, where treatment with paxalisib resulted in overall survival (OS) of 15.7 months and progression-free survival (PFS) of 8.4 month compared to 12.7 months OS and 5.3 months PFS with a historic group treated with temozolomide in patients with glioblastoma with an unmethylated O6-methylguaninemethyltransferase (MGMT) promotor following surgery and radiotherapy. Biohaven announced that in a 1,405 patient, Phase II/III trial, 24% of migraine patients treated with intranasal zavegepant were pain free at two hours compared to 15% with placebo. Patients were free of their most bothersome symptoms at two hours in 40% of zavegepant patients compared to 31% of placebo patients. Published Research Updates An FDA review of bardoxolone questioned whether the Phase III CARDINAL trial had study design flaws and therefore if it truly demonstrated an improvement in renal function. The FDA also expressed concerns regarding long-term safety of bardoxolone due to an increase in albuminuria and blood pressure. Regulatory Update
The FDA approved maribavir (Livtencity, Takeda), on 11/23/2021, to treat resistant, post-transplant, cytomegalovirus (CMV) infection/disease. The FDA approved pafolacianine (Cytalux, On Target Laboratories), on 11/29/2021, as an adjunct fluorescent imaging agent for intraoperative identification of malignant ovarian cancer lesions. The FDA extended the PDUFA date for Oleogel-S10 to allow additional time to review new data. The new PDUFA date is 2/28/2022 Zenas BioPharma acquired worldwide rights to develop and market obexelimab. Announced Research Updates Gilead and Merck announced they are temporarily pausing a 48-week, 75 patient, Phase II trial evaluating the once-weekly combination of islatravir and lenacapavir in maintenance of virologically suppressed HIV as Merck investigates the decrease in immune response in a trial evaluating MK-8507 and islatravir. Santhera and ReveraGen announced 48-week results from 114 patients that continued treatment in the VISION-DMD trial, where continued treatment with vamorolone or a change from prednisone to vamorolone maintained TTSTAND. Pfizer announced that in the 24-week, 286 patient, Phase IIB TRANSLATE-TIMI 70 trial, treatment with vupanorsenreduced non-HDL-C compared to placebo in patients with elevated non-HDL-C and triglycerides who were receiving a stable statin dose. |
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