Update for March 9, 2021

Regulatory Update 
 
The FDA approved the combination of serdexmethylphenidate plus methylphenidate (Azstarys, KemPharm), on 3/2/2021 for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years and older.
 
The FDA accepted the BLA for efgartigimod for the treatment of generalized myasthenia gravis and set a PDUFA date for 12/17/2021.
 
Strongbridge submitted an NDA for levoketoconazole, for the treatment of endogenous Cushing’s syndrome.
 
Reata submitted an NDA for bardoxolone methyl for the treatment of chronic kidney disease caused by Alport syndrome.
 
Shanghai Junshi Biosciences initiated a rolling BLA for toripalimab for the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma.
 
Announced Research Updates 
 
BrainStorm announced that in a 28-week, 189 patient, Phase III trial, treatment with MSC-NTF did not improve the number of patients that achieved at least a 1.25 point improvement per month in their Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score compared to placebo in patients with amyotrophic lateral sclerosis (ALS). In the trial 34.7% of MSC-NTF patients had a non-significant improvement in their ALSFRS-R score compared to 27.7% with placebo. The FDA calculated a slightly different non-significant difference between the two groups (32.6% vs 27.9%). The FDA also found a modest excess in mortality in patients treated with MSC-NTF.
 
Inovio announced that in the 36-week, 201 patient, Phase III REVEAL 1 trial, treatment with VGX-3100 did not result in a significant reduction in histopathological regression of high-grade squamous intraepithelial lesions (HSIL) compared to placebo (22.5 vs 11.1%) in patients with HPV-16/18-associated cervical HSIL. However, there was a significant difference in the 193 patient modified intention to treat group (23.7 vs 11.3%).
 
Lilly announced that in the 40-week, 1,879 patient, Phase III, SURPASS-2 trial, treatment with subcutaneous tirzepatide reduced HbA1c and weight by 2.09% and 7.8 Kg with 5 mg, 2.37% and 10.3 kg with 10 mg and 2.46% and 12.4 kg with 15 mg compared to a 1.86% and 6.2 kg reduction with subcutaneous semaglutide in patients with type 2 diabetes receiving metformin.
 
Galapagos announced interim results after 13 weeks from 240 patients enrolled in the Phase III MANTA and MANTA-RAy safety trials, where a similar number of men had a 50% or greater decline in sperm concentration with filgotinib (8.3%) or placebo (6.7%).
 
Pfizer announced that in a 12-week, 285 patient, Phase III, JADE TEEN trial, an IGA score of clear or almost clear skin with at least a 2 point improvement in the IGA score was achieved by 46.2% of patients treated with abrocitinib 200 mg and 41.6% with 100 mg compared to 24.5% with placebo in patients 12 to 17 years of age with moderate to severe atopic dermatitis (AD) who were also on background topical therapy. EASI-75 was achieved by 72% of patients who received abrocitinib 200 mg and 68.5% with 100 mg compared to 41.5% with placebo
 
Published Research Updates
 
In a 43 patient, phase I/II trial, patients treated with rovalpituzumab tesirine plus nivolumab with or without ipilimumab demonstrated an overall response rate of 30% in patients with previously treated extensive-stage small cell lung cancer.
 
In a 52-week, 323 Japanese patient, Phase III, trial, vadadustat was non-inferior to darbepoetin alfa in mean Hb at weeks 20 and 24 (10.61 g/dL vs 10.65 g/dL) in patients with hemodialysis-dependent CKD.
 
In the 15-day, 300 patient, Phase III CONFIRM trial, 32% of patients treated with terlipressin plus albumin achieved reversal of hepatorenal syndrome (HRS) compared to 17% with albumin alone in patients with Type 1 HRS (HRS-1). In the CONFIRM trial, HRS reversal was defined as two consecutive serum creatinine measurements of 1.5 mg per deciliter or less at least 2 hours apart and survival without renal-replacement therapy for at least 10 days after the completion of treatment.
 
A 32-month long-term extension, open-label Phase III trial, included 843 patients from three previous trials.  Treatment with peficitinib resulted in ACR20/50/70 being maintained in 78.%/63.3%/44.1% of Asian patients with rheumatoid arthritis. Adverse events were experienced in 94.4% of patients. Most ADR were mild with the most common were nasopharyngitis and herpes zoster.
 
In a 923-day long-term extension of the Phase II, diabetes prevention trial, the mean time to develop type 1 diabetes was 59.6 months in teplizumab patients compared to 27.1 months with placebo
 
Interim results from 60 patients enrolled in the 400 patient, Phase III OVAL trial, suggested that treatment with ofranergene obadenovec plus paclitaxel demonstrated a 58% CA-125 response in patients with platinum-resistant ovarian cancer.
 
In the 323 patient, Phase I/II, open-label, dose-ranging, BRUIN trial, 121 patients had efficacy data available for analysis. Treatment with pirtobrutinib resulted in an overall response of 62% in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma previously treated with a covalent BTK inhibitor.
 
In the 366 patient, Phase III, FORWARD I trial, treatment with mirvetuximab soravtansine did not improve progression-free survival compared to standard chemotherapy (paclitaxel, pegylated liposomal doxorubicin, or topotecan) in patients with folate receptor alpha-positive, platinum-resistant epithelial ovarian cancer, who had received up to three prior lines of therapy.
 
In a 51 patient, open-label, Phase II trial, treatment with camrelizumab plus decitabine resulted in progression-free survival of 20 to 21.6 months in patients with relapsed/refractory classical Hodgkin lymphoma who failed PD-1 inhibitors.