Update for March 2, 2021

Regulatory Update 
 
The FDA approved casimersen (Amondys 45, Sarepta), on 2/25/2021, for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 45 skipping. WAC to treat a 20 Kg patient is $300,000 per year.

The FDA approved melflufen (Pepaxto, Oncopeptides) in combination with dexamethasone, on 2/26/2021, as a fourth line treatment for relapsed or refractory multiple myeloma.
 
The FDA approved fosdenopterin (Nulibry, BridgeBio Pharma and Origin Bioscience) to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A.
 
The FDA accepted the NDA for gefapixant for the treatment of chronic cough and set a PDUFA date for 12/21/2021.
 
The FDA designated tipifarnib a Breakthrough Therapy for the treatment of recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency of 20% or > after disease progression on platinum-based chemotherapy.
 
The FDA designated Immunocore’s tebentafusp a Breakthrough Therapy for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma.
 
The European Medicines Agency’s (EMA) approved retifanlimab for the treatment of locally advanced or metastatic squamous cell anal carcinoma.
 
Announced Research Updates
 
The FDA has put a hold on trials for bluebird bio’s gene therapy beta beglogene darolentivec due to the development of acute myeloid leukemia and myelodysplastic syndrome in two trial patients.
 
Italfarmaco announced seven-year data from a long-term extension of a Phase II trial, where adding givinostat to corticosteroids resulted in a delay in loss of ambulation to 16 years compared to 13.4 years in a historical group that received corticosteroids only in patients with Duchenne Muscular Dystrophy. Respiratory function also declined at a slower rate.
 
AstraZeneca announced that in the 823 patent, Phase III, KESTREL trial, adding tremelimumab to durvalumab did not improve overall survival compare to cetuximab with cisplatin or carboplatin plus 5-fluorouracil in patients with recurrent or metastatic head and neck squamous cell carcinoma with tumors that expressed high levels of PD-L1.
 
Immunicum announced that in the 88 patient, Phase II, open-label, MERECA trial, treatment with ilixadencel plus sunitinib resulted in overall survival of 35.6 months compared to 25.3 months with sunitinib monotherapy in patients with renal cell carcinoma.
 
Amgen and AstraZeneca announced that in the 52-week, 1,061 patient, Phase III NAVIGATOR trial, treatment with tezepelumab reduced the annualized asthma exacerbation rate by 56% compared to placebo in patients with severe asthma who were receiving medium to high-dose inhaled corticosteroids plus at least one additional controller medication with or without oral corticosteroids. 
 
Published Research Updates
 
In the 128 patient, Phase II, dose-ranging, open-label, KarMMa trial, treatment with idecabtagene vicleucel resulted in an overall response rate of 73% in patients with resistant multiple myeloma.
 
In a five-week, 182 patient, Phase II trial, treatment with xanomeline and trospium decreased the total Positive and Negative Syndrome Scale (PANSS) score by 11.6 points compared to placebo in patients with schizophrenia who were experiencing acute psychosis.
 
In the 12-week, Phase I/II, Simplici-T1 trial, compared to placebo, TTP399 lowered HbA1c 0.7% in the 20 patient, Part 1 of the study and 0.21% in the 85 patient, Part 2 of the study in patients with type 1 diabetes receiving insulin.
 
In the 126 patient, Phase III GENUINE trial, ublituximab in combination with ibrutinib had an overall response rate of 83% compared to 65% with ibrutinib alone in patients with relapsed or refractory chronic lymphocytic leukemia with at least one f 17p deletion, 11q deletion, or TP53 mutation. 
 
In the 52-week, 594 patient, Phase III, ALPS trial, treatment with roxadustat improved hemoglobin by 1.692 g/dL compared to placebo in chronic kidney disease (CKD) patients with anemia. 
 
In a 52-week, 1,043 patient, open-label, Phase III, trial, roxadustat was non-inferior to epoetin alfa in changes in hemoglobin (2.57 vs 2.36 g/dL) and hemoglobin response (88.2% vs 84.4%) in CKD patients with anemia and new to dialysis.
 
In a 52-week, 387 Parkinson’s Disease patient extension of the Japanese COMFORT-PD trial, treatment with opicapone reduced OFF time in patients originally randomized to opicapone and those who originally received placebo but were changed to opicapone.
 
In a 144 patient, Phase IIb trial, treatment with TLPLDC vaccine did not improve disease-free survival in the intention-to-treat analysis, but an improvement in the vaccine group was found in the per protocol analysis (62.9% vs. 34.8%) in patients with resected stage III/IV melanoma.