Update for June 3, 2020

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.