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Regulatory Update
The FDA approved vericiguat (Verquvo, Merck, Bayer), on 1/20/2021, to reduce the risk of cardiovascular death and heart failure hospitalization following a hospitalization for heart failure or need for outpatient intravenous (IV) diuretics in adults with symptomatic chronic heart failure with reduced ejection fraction. The FDA approved voclosporin (Lupkynis, Aurinia), on 1/23/2021, for the treatment of lupus nephritis. The FDA approved cabotegravir/rilpivirine injection (Cabenuva, ViiV Healthcare), on 1/21/2021, for the treatment of HIV-1 infection as a replacement for a current antiretroviral regimen in those who are virologically suppressed on a stable antiretroviral regimen with no history of treatment failure and with no known or suspected resistance to either cabotegravir or rilpivirine. Cabotegravir/rilpivirine injection is given once a month. The FDA also approved cabotegravir tablets (Vocabria, ViiV Healthcare). The tablets are taken once a day in combination with oral rilpivirine for one month prior to initiating cabotegravir/rilpivirine long-acting injection to ensure the combination is well-tolerated. The FDA accepted the NDA for odevixibat for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis and set a PDUFA date for 7/20/2021. A site inspection by the FDA has delayed approval of GSK’s dostarlimab. GSK had expected the drug to be approved by the end of 2020, but travel restrictions due to COVID-19 has delayed an inspection of the manufacturing facility. A date has not been set for the inspection or a new PDUFA target. The FDA granted toripalimab Fast Track status as a first-line treatment of mucosal melanoma. Announced Research Updates LSKB announced that in a 30 patient, open-label, Phase II trial, treatment with camrelizumab plus apatinib resulted in a 43.3% overall response rate as second line therapy for Chinese patients with advanced esophageal squamous cell carcinoma. GSK announced data from 106 patients enrolled in cohort F of the open-label, Phase I, GARNET trial, where treatment with dostarlimab resulted in a 38.7% objective response rate in patients with deficient mismatch mutation repair (dMMR) or POLEmut non-endometrial solid tumors. Roche announced that in two 48-week, Phase III trials, which included the 671 patient TENAYA Trial and the 651 patient LUCERNE trial, the change in BCVA from treatment with faricimab, at dosing intervals from eight to 16-weeks, was non-inferior to aflibercept given every eight weeks in patients with neovascular age-related macular degeneration. Almost half (45%) of people in both trials were treated every 16-weeks with faricimab. Published Research Updates In a 3-month, 500 patient, Phase III trial, VM202 did not improve the 24-hour NRS pain score compared to placebo in patients with painful diabetic peripheral neuropathy. In an extension of the 500 patient, Phase III trial, 101 patients were followed for 12-months and VM202 decreased the 24-hour NRS pain score compared to placebo in patients with painful diabetic peripheral neuropathy. Greater pain reduction was found in patients not receiving gabapentin or pregabalin. In the 56 patient, open label Phase I, NAVIGATOR trial, treatment with avapritinib resulted in an 91% overall response rate (ORR) in patients with gastrointestinal stromal tumors and a PDGFRA D842V-mutations. In a 34-week, 112 patient open label extension of the OASIS trial, 84 patients received 2 mg of etrasimod during the extension study with 64% achieving a clinical response, 33% achieving clinical remission and 43% demonstrated an endoscopic improvement. Patients that achieved a clinical response, clinical remission, or endoscopic improvement at week 12 maintained the outcome to the end of treatment in 85%, 60%, or 69% of patients, respectively. In a 24-week, 33 patient, Japanese trial, patients treated with omarigliptin achieved a 0.2% decrease in HbA1c compared to a 0.4% increase with linagliptin in patients with type 2 diabetes mellitus. In a 138 patient, Phase IV trial, treatment with carbetocin decreased blood loss during surgery by 184 ml compared to placebo in women undergoing abdominal myomectomy. In a 138 patient, Phase III trial, treatment with ceftobiprole resulted in 95.7% of patients achieving early clinical response at day four and 90.4% achieving clinical cure compared to 93.2% and 97.7% with placebo in pediatric patients with pneumonia. In a 12-week, 45 patient, Phase II trial, treatment with elafibranor resulted in a 48.3% decrease in serum alkaline phosphatase with 80 mg and a 40.6% decrease with 120 mg compared to a 3% increase with placebo in patients with primary biliary cholangitis that had an inadequate response to ursodeoxycholic acid. In a 124 patient, Phase Il trial, treatment with adavosertib plus gemcitabine resulted in progression free survival of 4.6 months compared to 3 months with gemcitabine alone in patients with p53 tumor suppressor gene (TP53)-mutated ovarian cancer. In the 190 patient, Phase II, open-label, POLARIS-02 trial, treatment with toripalimab resulted in an objective response rate of 20.5% in Chinese patients with recurrent or metastatic nasopharyngeal carcinoma. Regulatory Update
The FDA accepted the NDA for finerenone for the treatment of chronic kidney disease in patients with type 2 diabetes, suggesting a PDUFA date of 7/12/2021. The FDA designated ligelizumab a Breakthrough Therapy for the treatment of chronic spontaneous urticaria in patients who have an inadequate response to H1-antihistamine treatment. The FDA granted padeliporfin a Fast Track designation for the treatment of low-grade and unifocal high-grade Upper Tract Urothelial Cancer. Announced Research Updates Mesoblast announced that in the 30-month, 537 patient, Phase III, DREAM-HF trial, treatment with a single dose of rexlemestrocel-L resulted in a 20.6% incidence of cardiac death, heart attack or stroke compared to 30% incidence with placebo in patients with chronic heart failure with reduced left ventricular ejection fraction, that were receiving optimal oral therapy. Published Research Updates In a 162 patient, Phase II, open-label trial, treatment with olmutinib resulted in an objective response rate of 46.3% in patients with T790M-positive non-small cell lung cancer (NSCLC) after treatment with an epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI). In the 96-week, 38 patient, Phase III, RESCUE trial, the eyes treated with lenadogen nolparvovec had an improvement in best corrected visual acuity of 10 letters on the ETDRS scale compared to a 9 letter gain in the eyes that received sham injection in patients with Leber Hereditary Optic Neuropathy. In the 12-week, 88 patient, dose-ranging, Phase II STARS trial, 66.7% of patients treated with gaboxadol reduced their Clinical Global Impressions-Improvement scale (CGI-I) score compared to 39.3% with placebo in patients with Angelman syndrome. A post-hoc analysis examined the CGI-I score to determine the number of patients that were very much improved or much improved and found that 32.1% of patients that received gaboxadol once daily reached this threshold compared to 7.1% of placebo patients. There was not a significant improvement compared to placebo with gaboxadol twice daily. In a 48-week, 75 patient, Phase II trial, patients treated with bimagrumab had a 20.5% decrease in total body fat mass compared to a 0.5% decrease with placebo in patients with obesity and type 2 diabetes. In the 52-week, 1,252 patient, Phase III, FINCH 3 trial, the percentage of patients that achieved ACR20 at 24 weeks was 81% with filgotinib 200 mg plus methotrexate and 80% with filgotinib 100 mg plus methotrexate compared to 71% with methotrexate alone in patients with moderate-to-severe rheumatoid arthritis naive to methotrexate. There was no difference between the number of patients that achieved AR20 with filgotinib 200 mg alone compared to methotrexate alone. Regulatory Update
The FDA designated tiragolumab in combination with atezolizumab, a Breakthrough Therapy for the treatment of metastatic non-small cell lung cancer with high PD-L1 expression with no EGFR or ALK genomic tumor aberrations. Announced Research Updates Sarepta announced interim data from 20 patients enrolled in a 48-week, 41 patient, Phase II trial, where SRP-9001 resulted in micro-dystrophin expression of 28.1% at 12-weeks. At 48-weeks, treatment with SRP-9001 improved the NSAA score by 1.7 points in the 20 treated patients, but the improvement was not statistically significant compared to a 0.9 point improvement in 21 placebo patients. The 41 patients enrolled in the study were between the ages of 4 to 7 at enrollment. Loncastuximab tesirine is available through an expanded access program (EAP) for relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Information on the EAP on be accessed at: https://adctherapeutics.com/expanded-access-program/ Loxo announced that in the 323 patient, Phase I/II, open-label, dose-ranging, BRUIN trial, treatment with LOXO-305 resulted in an overall response of 52% for mantle cell lymphoma (MCL), 68% for MCL and Waldenström macroglobulinemia, 75% for Richter transformation, 50% for follicular lymphoma, 24% for diffuse large B-cell lymphoma, and 22% for marginal zone lymphoma. Published Research Updates 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. Regulatory Update
The FDA approved ansuvimab (Ebanga, Ridgeback Biotherapeutics), on 12/21/2020, for the treatment for Zaire ebolavirus (Ebolavirus) infection in adults and children. The FDA approved vibegron (Gemtesa, Urovant Sciences), on 12/23/2020, for the treatment of overactive bladder with symptoms of urge urinary incontinence, urgency, and urinary frequency in adults. The FDA rejected arbaclofen due to inadequate justification for the statistical analysis of a primary endpoint (TNmAS-MAL scores at Day 84) in a pivotal trial. The FDA has recommended a new efficacy study be performed. In November 2020, Voyager paused the RESTORE-1 trial when the data safety and monitoring board (DSMB) requested time to analyze MRI imaging abnormalities. The FDA placed a hold on the RESTORE-1 trial in December 2020 due to these abnormalities. Chi-Med initiated a rolling NDA in December 2020 for surufatinib as a treatment of advanced neuroendocrine tumors and plans to complete the submission in the first half of 2021. The EMA approved ViiV Healthcare’s cabotegravir injection in combination with rilpivirine for the treatment of HIV-1 infection in adults who are virologically suppressed. Cara submitted an NDA for difelikefalin, for the treatment of moderate-to-severe pruritus in hemodialysis patients, in December 2020. The FDA designated Immunicum’s Ilixadencel an Orphan Drug for the treatment of hepatocellular carcinoma. Announced Research Updates Amgen and AstraZeneca announced that in the 48-week, 150 patient, Phase III SOURCE trial, treatment with tezepelumab did not reduce daily oral corticosteroids (OCS), without loss of asthma control, compared to placebo in patients with severe asthma who require continuous treatment with inhaled corticosteroids plus long-acting beta2-agonists, and chronic treatment with maintenance OCS. BioMarin announced one-year data from a 119 patient extension of a 121 patient, Phase III trial, where patients treated with vosoritide achieved an increase in height of 1.79 cm more than placebo in children with achondroplasia. ChemoCentryx and VFMCRP announced that in the 26-week, 88 patient, Phase II ACCOLADE trial, treatment with avacopan improved the C3G Histologic Index for Disease Activity by 2% compared to a 38% worsening with placebo in patients with C3 Glomerulopathy. Supernus announced that in a 6-week, 374 patient, Phase III trial, viloxazine decreased the ADHD Investigator Symptom Rating Scale (AISRS) score by 15.5 points compared to an 11.7 point decrease with placebo in adults (age 18 to 65) with attention deficit hyperactivity disorder. Aprea Therapeutics announced that in a 154 patient, Phase III trial, treatment with eprenetapopt plus azacitidine did not improve the complete remission rate compared to azacitidine alone in patients with TP53 mutant myelodysplastic syndromes. Published Research Updates In an 11.9-month interim analysis of a 412 patient, Phase III, open-label trial, carboplatin/pemetrexed/camrelizumab followed by pemetrexed/camrelizumab maintenance resulted in progression free survival (PFS) of 11.3 months compared to 8.3 months with carboplatin/pemetrexed followed by pemetrexed alone in Chinese patients with in non-small cell lung cancer. |
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