Update for December 22, 2020

Regulatory Update
 
The FDA approved tirbanibulin (Klisyri, Athenex), on 12/15/2020, for the treatment of actinic keratosis on the face or scalp. 
 
The FDA approved margetuximab (Margenza, MacroGenics), on 12/17/2020, to be given in combination with chemotherapy, for the treatment of metastatic HER2-positive breast cancer in patients who have received two or more prior anti-HER2 regimens, at least one of which was for metastatic disease. The drug was approved with a Boxed Warning regarding the potential for left ventricular dysfunction and embryo-fetal toxicity. MacroGenics anticipates availability of margetuximab in March of 2021.
 
The FDA approved relugolix (Orgovyx, Myovant Sciences) on 12/18/2020, for the treatment of advanced prostate cancer.
 
The FDA has delayed approval of Novartis’ inclisiran because travel restrictions have delayed the inspection of the manufacturing plant. A new PDUFA date has not been announced.
 
Amgen submitted a BLA for sotorasib for the treatment of metastatic non-small cell lung cancer with a KRAS G12C mutation.
 
Gilead and Galapagos are discontinuing development of filgotinib for the treatment of rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, and non-infectious uveitis in the U.S. Gilead will continue to develop filgotinib for the treatment of Crohn’s Disease, while Galapagos will develop the drug for ulcerative colitis. Gilead returned co-marketing and development rights for filgotinib in Europe to Galapagos. The changes were made due to concerns by the FDA on testicular toxicity with the highest dose of filgotinib.
 
The FDA placed a clinical hold on UniQure’s etranacogene dezaparvovec clinical trials after a preliminary diagnosis of hepatocellular carcinoma was made in one patient enrolled in the Phase III HOPE-B trial.
 
The EMA has granted Orphan status to Acceleron Pharma’s sotatercept for the treatment of patients with pulmonary arterial hypertension.
 
Announced Research Updates
 
Roche announced that in the 255 patient arm of the Phase II, IPATunity130 trial, treatment with ipatasertib added to paclitaxel did not improve progression free survival compared to paclitaxel alone (7.4 vs 6.1 months) in patients with locally advanced or metastatic triple-negative breast cancer with alterations to the PIK3CA, AKT1 and PTEN genes.
 
VTV announced that in the 6-month, 43 patient, Phase II, ELEVAGE trial, azeliragon did not improve cognition (ADAS-cog14) compared to placebo in patients with mild probable Alzheimer’s Disease.
 
AB Science announced that in a 24-week, 718 patient, Phase IIb/III trial, treatment with masitinib improved the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) compared to placebo in patients with mild to moderate Alzheimer’s disease who were receiving a cholinesterase inhibitor (donepezil, rivastigmine or galantamine) and/or memantine.
 
Daiichi Sankyo announced that in a 14-week, 274 patient, Phase III trial, treatment with mirogabalin reduced pain more than placebo in patients with central neuropathic pain after spinal cord injury.
 
Roche announced that in two 1-year, Phase III trials, which included the 951 patient RHINE Trial and the 940 patient YOSEMITE trial, the change in best-corrected visual acuity from treatment with faricimab, at dosing intervals from eight to 16-weeks, was non-inferior to aflibercept given every eight weeks in patients with diabetic macular edema.
 
Published Research Updates
 
In the 150 patient Phase II, ProCAID trial, adding capivasertib to docetaxel plus prednisone did not improve progression free survival in patients with metastatic castration-resistant prostate cancer.
 
In a 12-week, 156 patient, Phase II trial, treatment with praliciguat did not improve the urine albumin to creatinine ratio compared to placebo in patients with type 2 diabetes and diabetic nephropathy.
 
Daiichi Sankyo announced that in a 14-week, 274 patient, Phase III trial, treatment with mirogabalin reduced pain more than placebo in patients with central neuropathic pain after spinal cord injury.