Remdesivir – Update on Pricing and availability
Additional information became available on Monday, 29 June, 2020, so we have provided an update regarding pricing and availability. The FDA approved an emergency use authorization (EUA) for remdesivir to treat hospitalized patients with severe COVID-19. HHS signed an Agreement with Gilead for 500,000 treatment courses of remdesivir on 6/29/2020. This represents 100% of July production, 90% of August production and 90% of September production. The U.S. The Department of Health and Human Services will control distribution through AmerisourceBergen and hospitals will pay no more than WAC for the drug. The government will tell AmerisourceBergen how many vials can be shipped to a state. The department of health in each state will direct the wholesaler to deliver doses to the hospitals with the highest number of inpatient COVID-19 cases. The initial supply from the Federal Stockpile of 5 million doses were exhausted at the end of June 2020. HHS will stop managing distribution of remdesivir at the end of September, when supplies are expected to stabilize. Gilead has set AWP for remdesivir (Veklury) at $520 per vial or $3,120 for five days of treatment. This is the price for private insurance patients in the U.S. The U.S. government price for Medicaid and military hospitals is $390 per vial or $2,340 for a five-day course of treatment. This is also the price for developed countries. Pricing for remdesivir is more aligned with estimates from ICER that take into account the potential effect of dexamethasone. Gilead has entered into agreements with generic manufacturers to make the drug available at a substantially lower cost to developing countries, where healthcare resources, infrastructure and economics are lower. Regulatory Update The FDA rejected the BLA for abicipar pegol in June 2020 due to an unfavorable benefit-risk ratio in the treatment of neovascular (wet) age-related macular degeneration. CSL Behring licensed worldwide marketing rights for hemophilia B gene therapy etranacogene dezaparvovec from uniQure. Sarepta submitted an NDA for casimersen for the treatment of Duchenne muscular dystrophy with genetic mutations that are amenable to skipping exon 45 of the Duchenne gene. Ascendis submitted a BLA for lonapegsomatropin for the treatment for pediatric growth hormone deficiency. The FDA granted seviprotimut-L a Fast Track designation as an adjuvantive treatment of stage IIB/IIC melanoma patients, post-resection to improve recurrence-free survival. Announced Research Updates Orphazyme announced that in the 6-month, 37 patient, Phase II, dose-ranging, ORARIGAU-01 trial, treatment with arimoclomol 100mg, 200mg, or 400mg did not reduce serum chitotriosidase activity compared to placebo in patients with Gaucher disease. Acceleron announced that in the 24-week, 106 patient, Phase II, PULSAR trial, treatment with sotatercept reduced pulmonary vascular resistance by 20.5% with 0.3 mg/kg (n = 32) and 33.9% with 0.7 mg/kg (n=42) compared to a 2.1% reduction with placebo in patients with pulmonary arterial hypertension. Rhythm Pharmaceuticals announced 12-week interim results from 75 patients enrolled in a Phase II trial, where a weekly setmelanotide formulation achieved similar weight loss to a daily formulation in healthy obese patients. Published Research Updates In the 87 patient, Phase II MAJA trial, maintenance therapy with vinflunine plus best supportive care resulted in a mortality rate of 59.1% compared to 74.4% mortality with only best supportive care in parents with advanced urothelial carcinoma. In the 527 patient, Phase III FALUCA trial, treatment with fruquintinib did not improve overall survival compared to placebo (8.9 months vs 10.4 months) in patients with locally advanced/metastatic nonsquamous non-small-cell lung cancer. Regulatory Update
The FDA approved lurbinectedin (Zepzelca, Jazz Pharma/PharmaMar) on 6/16/2020 for the treatment of metastatic small cell lung cancer that has progressed during or after treatment with platinum-based chemotherapy. The FDA rejected Nabriva Therapeutics/Zavante Therapeutics’ injectable fosfomycin a second time in June 2020 due to the same manufacturing deficiencies found in 2019. The FDA has accepted the NDA for Myovant’s relugolix for the treatment of advanced prostate cancer, and set a PDUFA date of 12/20/2020. The FDA designated Insmed’s brensocatib as a Breakthrough Therapy for the treatment of non-cystic fibrosis bronchiectasis. TG Therapeutics submitted an NDA for umbralisib for the treatment of marginal zone lymphoma and follicular lymphoma. The FDA requested additional information on the efficacy analysis for Clovis Oncology’s rociletinib. The FDA has granted Ovid Therapeutics’ gaboxadol a Rare Pediatric Disease designation for the treatment of Angelman syndrome. The FDA has designated Chi-Med/Lilly’s fruquintinib Fast Track status for the treatment of patients with metastatic colorectal cancer. Announced Research Updates Brickell announced that in a 42-day, 281 patient, Phase III trial, 53.9% of patients treated with sofpironium improved their Hyperhidrosis Disease Severity Scale to two or less and had a 50% or more reduction in gravimetric sweat production compared to 36.4% that received placebo in Japanese patients with axillary hyperhidrosis. Global Data announced that a pooled analysis of six Phase II trials found that two months after midomafetamine treatment, 56% of patients did not meet PTSS diagnostic criteria and after 12 months, 67% did not qualify for a PTSD diagnosis. BeyondSpring announced interim data from 120 patients enrolled in the Phase III PROTECTIVE 2 trial, where treatment with plinabulin plus pegfilgrastim reduced grade 4 or severe neutropenia, compared to pegfilgrastim monotherapy, in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide. Hua Medicine announced that in a 24-week, 463 patient, Phase III SEED trial, treatment with dorzagliatin 0.75 mg reduced HbA1c 1.15% compared to a 0.58% decrease with placebo, in Chinese patients with type 2 diabetes. In a 28-week, open-label extension of the SEED trial, patients treated with dorzagliatin continued on the drug and maintained a 1.11% HbA1c reduction, while patients switched from placebo achieved a 1.27% reduction. Roche announced that in the 1,101 patient, Phase II, IPATential150 trial, ipatasertib, added to abiraterone and prednisone or prednisolone, improved radiographic progression-free survival compared to abiraterone plus prednisone/prednisolone in patients with metastatic castration-resistant prostate cancer (mCRPC) with PTEN loss, but not in all mCRPC patients regardless of PTEN status. Provention Bio announced extended follow-up data showing that the time to develop type 1 diabetes after treatment with teplizumab increased to five years compared to two years with placebo. Sanofi announced that in a 2.4 year, 34-patient follow-up to a Phase II trial, treatment with fitusiran lowered the annualized bleed rates to 0.84 in the non-inhibitor subgroup and to 0.44 in the inhibitor subgroup. Published Research Updates In the 12-week, 155 patient, Phase IIb ASTEROID 2 trial, 62.9% of patients treated with vilaprisan had a complete absence of bleeding or spotting compared to 55.4% with ulipristal and none with placebo in patients with uterine fibroids and heavy menstrual bleeding. In an 8-week, 9 patient, open-label pilot study, treatment with ganaxolone decreased the Montgomery-Asberg Depression Rating Scale [MADRS] score from 24.4 to 12.8, and 44% achieved remission (MADRS < 10) in postmenopausal women with persistent depression despite adequate antidepressant therapy. Regulatory Update
The FDA approved inebilizumab-cdon (Uplizna, Viela Bio) on 6/11/2020 for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. The FDA designated Insmed’s brensocatib a Breakthrough Therapy for the treatment of non-cystic fibrosis bronchiectasis. Leo Pharma announced the EU accepted the MAA for AstraZeneca/Leo Pharma’s tralokinumab as a treatment for moderate-to-severe atopic dermatitis. Announced Research Updates AbbVie announced that in the 12-week, 612 patient, Phase III, SELECT-CHOICE trial, patients treated with upadacitinib had a 2.52 reduction in their DAS28CRP score compared to a 2 point reduction with abatacept in patients with rheumatoid arthritis previously treated with a biologic. GSK announced interim data from 18 patients enrolled in the Phase I/II, open-label, DREAMM-6 trial, where treatment with belantamab mafodotin plus bortezomib and dexamethasone resulted in an overall response rate of 78% in patients with relapsed/refractory multiple myeloma. Pfizer announced that in a 12-week, 387 patient, Phase III, JADE MONO-TEEN trial, more patients treated with abrocitinib 200 mg achieved an IGA score of clear or almost clear skin and had a 2 point or > improvement in their IGA score compared to placebo in patients 12 to < 18 years of age with moderate to severe atopic dermatitis (AD) who were also on background topical therapy. Treatment with the 200 mg dose also resulted in more patients achieving at least a 75% or greater improvement in their Eczema Area and Severity Index (EASI) score compared to placebo. The 100 mg dose did not differ from placebo for IGA or EASI scores. Opthea announced that in a 12-week, 155 patient Phase IIa trial, treatment with OPT-302 plus aflibercept did not improve the number of patients that achieved a 5 letter or > gain in Best Corrected Visual Acuity compared to aflibercept monotherapy (52% vs 60%) in patients with persistent diabetic macula edema despite regular intravitreal administration of prior anti-VEGF-A monotherapy. Bluebird announced 6-month interim results for 16 patients enrolled in the Phase I/II, HGB-206 trial, where treatment with beta beglogene darolentivec resulted in total hemoglobin from 9.6 to 16.2 g/dL, HbAT87Q levels from 2.7 to 9.4 g/dL and a 99.5% mean reduction in annualized rate of vaso-occlusive crises and acute chest syndrome in patients with sickle cell disease. ADC announced that in the 145 patient, Phase II, open-label, LOTIS 2 trial, treatment with loncastuximab tesirine resulted in an ORR of 48.3% and a complete response rate (CRR) of 24.1% in patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL). ADC announced interim results from 18 patients enrolled in the Phase I/II, open-label, LOTIS 3 trial, where treatment with loncastuximab tesirine plus ibrutinib resulted in an ORR of 66.7% and a CRR of 33% in patients with relapsed or refractory DLBCL or mantle cell lymphoma (MCL). Published Research Updates In the 12-month, 81 patient, Phase II, open-label, L-MIND trial, treatment with tafasitamab plus lenalidomide resulted in an overall response rate of 60% in patients with diffuse large B cell lymphoma. In a 92 patient, Phase II, open-label trial, treatment with tafasitamab resulted in an overall response rate of 26% in diffuse large B cell lymphoma patients, 29% in follicular lymphoma patients and 27% in indolent non-Hodgkin's lymphoma patients. Regulatory Update
The FDA placed a hold on the AAVance trial, due to abnormalities in MRI imaging. Since the trial is fully enrolled and LYS-SAF302 is a single treatment, gene therapy for Sanfilippo syndrome, Lysogene stated the forecast completion in January 2022 is not expected to be delayed. The FDA designated MacroGenics’ margetuximab an Orphan Drug for the treatment of gastric and gastroesophageal junction cancer. Announced Research Updates Tenax announced that in the 6-week, 37 patient, Phase II, HELP trial, levosimendan did not reduce pulmonary capillary wedge pressure (PCWP) during exercise compared to placebo, but did reduce PCWP at rest in patients with pulmonary hypertension and heart failure with preserved ejection fraction. Gilead and Galapagos announced that in the 52-week, 1,759 patient, Phase III, FINCH 1 trial, the percentage of patients that achieved ACR20/50/70 with filgotinib 200 mg was 78%/62%/44%, filgotinib 100 mg was 76%/59%/38%, compared to adalimumab with 74%/59%/39% in patients with moderate-to-severe rheumatoid arthritis with an inadequate response to MTX. Gilead and Galapagos announced that in the 52-week, 975 patient, Phase III, FINCH 3 trial, the percentage of patients that achieved ACR20/50/70 with filgotinib 200 mg/MTX was 75%/62.3%/47.8%, filgotinib 100 mg/MTX was 73.4%/59.4%/40.1%, compared to filgotinib 200 mg monotherapy with 74.8%/61.4%/45.2%, and methotrexate monotherapy was 61.8%/48.3%/29.8% in patients with moderate-to-severe rheumatoid arthritis naive to methotrexate. Alnylam announced that in the 6-month, 39 patient, Phase III, ILLUMINATE-A trial, treatment with lumasiran decreased 24-hour urinary oxalate excretion by 53.5% compared to placebo in patients with primary hyperoxaluria type 1. Sarepta announced 1-year interim results from 3 patients enrolled in a Phase I/IIa, open-label trial, treatment with a low dose of SRP-9003 improved the North Star Assessment for Dysferlinopathy (NSAD), time to rise, four-stair climb, 100-m walk test and 10-meter walk test in patients with limb-girdle muscular dystrophy. In the same trial, 3 patients that received a high dose of SRP-9003 demonstrated a higher mean expression of transduced beta-SG in the muscle sarcolemma at 60-days compared to that seen with the lower dose. Published Research Updates In a 12-week, 391 patient, Phase III, JADE MONO-2 trial, an Investigator Global Assessment (IGA) response of clear [0] or almost clear [1], with improvement of 2 or more grades, was achieved by 38.1% of patients treated with abrocitinib 200 mg and 28.4% of those treated with 100 mg compared to 9.1% with placebo in patients with moderate to severe atopic dermatitis. A 75% improvement in the Eczema Area and Severity Index score (EASI-75) was achieved in 61% of patients treated with abrocitinib 200 mg and 44.5% of those treated with 100 mg compared to 10.4% with placebo. In a 24-week, 303 patient, Phase III trial, roxadustat, given orally three times per week, was non-inferior to darbepoetin, injected once per week, in maintaining hemoglobin between 10 to 12 g/dL (95.2% vs 91.3%) in Japanese hemodialysis patients. In a 27 patient, Phase II, open-label trial, treatment with camrelizumab added to gemcitabine, vinorelbine and pegylated liposomal doxorubicin followed by camrelizumab maintenance resulted in an overall response in 74% of patients with relapsed or refractory primary mediastinal B-cell lymphoma. Regulatory Update
Chi-Med plans to initiate a rolling NDA at the end of 2020 for surufatinib as a treatment of advanced neuroendocrine tumors. CHMP recommended that the EMA approve Ad26.ZEBOV and MVA-BN-Filo to prevent Ebola virus infection. The J&J Ebola vaccine is a two injection series. Zabdeno (Ad26.ZEBOV) is given first and followed two months later by Mvabea (MVA-BN-Filo). Announced Research Updates BioMarin announced that in a 4-year, 15 patient, Phase I/II, open-label extension trial, treatment with valoctocogene roxaparvovec resulted in a cumulative annualized bleeding rate of < 1, but Factor VIII levels declined while remaining in a range to provide hemostatic efficacy. Valoctocogene roxaparvovec is undergoing a priority review of its BLA due to being designated a Breakthrough Therapy. The drug has a PDUFA date of 8/21/2020. Immutep announced interim data from the 109-patient, Phase II, open-label TACTI-002 trial evaluating eftilagimod alpha plus pembrolizumab in the treatment of patients with non-small cell lung cancer (NSCLC) or head and neck squamous cell carcinoma (HNSCC). The data suggested an overall response rate (ORR) of 53% in 17 NSCLC patients and 39% in 18 patients with HNSCC. Iterum Therapeutics announced that in the 1,395 patient, Phase III SURE 2 trial, IV sulopenem followed by oral sulopenem/probenecid did not meet non-inferiority compared to IV ertapenem followed by oral ciprofloxacin/metronidazole or amoxicillin-clavulanate in the treatment of complicated urinary tract infections. Aveo announced that in the 38-month, 350 patient, Phase III, TIVO-3 trial, of patients treated with tivozanib, progression-free survival (PFS) was not significantly different from sorafenib (16.4 vs 19.2 months) in patients with highly refractory advanced or metastatic renal cell carcinoma that had failed at least two prior therapies, including a vascular EGFR TKI. (Note: results after 19-months have been published and suggested a PFS of 5.6 months with tivozanib compared to 3.9 months with sorafenib. Blueprint announced results from the 116 patient, Phase I/II, open label, ARROW trial. In the RET fusion–positive non-small cell lung cancer cohort, treatment with pralsetinib resulted in a 73% objective response rate (ORR) in treatment-naïve patients and 61% in patients that were previously treated with platinum-based chemotherapy. In the cohort of patients with RET-mutant medullary thyroid cancer, treatment-naïve patients had an ORR of 74% and previously treated patients had an ORR of 60%. The FDA accepted the NDA for pralsetinib for the treatment of RET fusion–positive non-small cell lung cancer and set a PDUFA date of 11/23/2020. Published Research Updates In the 1-year, 291 patient, Phase II, HER2CLIMB trial, treatment with tucatinib in combination with trastuzumab and capecitabine resulted in time to intracranial progression or death (CNS-PFS) of 9.9 months and overall survival of 18.1 months compared to 4.2 months CNS-PFS and 12 months OS with trastuzumab and capecitabine alone in patients with HER2-positive breast cancer with brain metastases. In the 48-week, 217 patient, Phase IIb BE ABLE 2 trial (a long-term follow-up to the Phase IIb BE ABLE 1 trial), 80 to 100% of bimekizumab responders maintained PASI90, 69-83% maintained PASI100 and 78-100% had an Investigator’s Global Assessment of 0/1. In the 406 patient, Phase III ADVANCE study, 16% of patients treated with eflapegrastim developed severe neutropenia compared to 24% with pegfilgrastim in patients with early-stage breast cancer. In the 55 patient, Phase II, open-label, INSIGHT trial, treatment with tepotinib did not improve progression free survival compared to standard platinum doublet chemotherapy (4.9 vs 4.4 months) in patients with epidermal growth factor receptor (EGFR)-mutant non-small-cell lung cancer (NSCLC) with MET overexpression (immunohistochemistry [IHC]2+ or IHC3+) or MET amplification having acquired resistance to EGFR inhibition. However the trial was terminated early due to poor recruitment, so all analyses are considered to be exploratory. Regulatory Update
Orphazyme filed an NDA for arimoclomol for the treatment of Niemann-Pick disease Type C. Aurinia submitted an NDA for voclosporin for the treatment of kidney inflammation. Protalix submitted a BLA for pegunigalsidase alfa for the treatment of adult patients with Fabry disease. In January 2016, the FDA placed a partial hold on a gemcabene Phase II trial pending completion of an animal carcinogenicity study. In May 2018, Gemphire submitted the data, which was determined as insufficient by the FDA. The partial hold was upheld in April 2020. Announced Research Updates Argenx announced that in the 26-week, 167 patient, Phase III, ADAPT trial, 67.7% of patients treated with efgartigimod achieved at least a 2-point improvement in their Myasthenia Gravis Activities of Daily Living (MG-ADL) score for four consecutive weeks compared to 29.7% with placebo in patients with acetylcholine receptor-antibody positive (AChR-Ab+) generalized myasthenia gravis on a stable drug regimen. Argenx plans to file a BLA for efgartigimod for the treatment of myasthenia gravis by the end of 2020. MD Serono announced that in a 24 week extension of a Phase II trial (48 weeks total treatment), the annualized relapse rate in patients treated with evobrutinib 75 mg twice daily at 48-weeks was 0.11 relapses/year compared to 0.37 relapses/year with placebo. At 108-weeks, the annualized relapse rate was 0.12 relapses/year. Abbvie announced that in the 509 patient, Phase III, BROCADE3 trial, veliparib added to carboplatin and paclitaxel improved PFS at 2-years to 27.5% and at 3-years to 17.5% compared to 15.3% and 8.6% with just carboplatin plus paclitaxel in 174 patients with hormone receptor (HR)-positive breast cancer who harbored a germline BRCA1/2 mutation. In 335 patients with triple-negative breast cancer who harbored a germline BRCA1/2 mutation, PFS at 2-years was 40.4% and at 3-years 35.3% with the triple therapy velaparib group compared to 25% and 13% with just carboplatin plus paclitaxel. GSK announced that in the 19-month (6-month trial, 13-month follow-up extension), 97 patient, Phase II, open-label, DREAMM-2 trial, treatment with belantamab mafodotin resulted in a 32% overall response rate and overall survival of 14.9 months in patients with relapsed/refractory multiple myeloma. Kura announced that in an 18 patient, Phase II trial, treatment with tipifarnib resulted in a 50% overall response rate and overall survival of 15.4 months compared to a historical 5-8 months in patients with HRAS mutant head and neck squamous cell carcinomas. BeiGene announced results from a 360 patient, open-label, Phase III, Chinese trial, where treatment with tislelizumab added to standard chemotherapy resulted in progression-free survival PFS) of 7.6 months and an objective response rate (ORR) of 73 to 75% compared to a PFS of 5.5 months and an ORR of 50% in patients treated with standard chemotherapy alone in patients with squamous non-small cell lung cancer. Minerva announced that in a 515 patient, Phase III trial, roluperidone did not reduce the PANSS Marder Negative Symptoms Factor Score or NSFS compared to placebo in patients with schizophrenia presenting with moderate to severe negative symptoms. Endocyte announced that in a 200 patient, Phase II trial, 66% of patients treated with Lu-PSMA-617 achieved a 50% or greater reduction in their PSA response rate (PSA50-RR) compared to 37% treated with cabazitaxel in patients with metastatic castration-resistant prostate cancer (mCRPC) who progressed after being treated with docetaxel. Medicenna announced that in a 125 patient, Phase IIb trial, treatment with MDNA55 resulted in overall survival (OS) of 11.6 months in patients with recurrent glioblastoma. When stratified by IL4R expression, patients with high IL4R had an OS of 15 months compared to 8.4 months in patients with low IL4R expression. TG Therapeutics announced that in a 117 patient, phase III GENUINE trial, ublituximab in combination with ibrutinib had an overall response rate of 93% compared to 78% with ibrutinib monotherapy in patients with relapsed/refractory chronic lymphocytic leukemia with confirmed presence of 17p deletion, 11q deletion, and/or TP53 mutation. Published Research Updates In a 25-week, 16 patient, Phase II, open-label trial, all 16 boys with DMD amenable to skipping exon 53 that received viltolarsen experienced a decrease in skipping exon 53 over baseline, which led to increased dystrophin production in muscles 5.7-5.9% of normal and had an improvement in physical function as measured by timed muscle function tests (time to stand from supine, time to run/walk, and 6-minute walk test) compared to matched controls. In the 16-week, 1,832 patient, Phase III, TANGO trial, treatment with tanezumab 10 mg reduced the daily average Low Back Pain Intensity score by 0.4 compared to placebo, but the 5 mg doses did not reach a significant difference in patients with chronic low back pain. In the 16-week, 59 patient, Phase II MAVERICK-HCM trial, treatment with mavacamten resulted in a 53% reduction in serum NT-proBNP and a 34% reduction in cardiac troponin I compared to a reduction of 1% and 4% with placebo in patients with symptomatic, non-obstructive hypertrophic cardiomyopathy. In a 43 patient, Phase II, open-label trial, treatment with anlotinib resulted in progression free survival of 14 months in patients with metastatic renal cell carcinoma that progressed after or were intolerant to sorafenib or sunitinib. In the 60 patient, Phase III, SAVOIR trial, savolitinib did not improve progression free survival compared to sunitinib (7 months vs 5.6 months) in patients with MET-driven, metastatic papillary renal cell carcinoma. In the 9-month, 99 patient, Phase II, open-label, VISION trial, treatment with tepotinib resulted in an objective response rate of 46% in patients with non-small cell lung cancer with metastatic NSCLC harboring MET exon 14 skipping alterations. In the 48-week, 930 patient, Phase III, HERO trial, 96.7% of patients treated with relugolix achieved sustained testosterone suppression to castrate levels (< 50 ng/dL) compared to 88.8% with leuprolide in men with advanced prostate cancer. |
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