Update for December 8, 2020

Regulatory Update
 
The FDA approved berotralstat (Orladeyo, BioCryst Pharmaceuticals), on 12/3/2020, for prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. BioCryst set WAC at $485,000 per year. Berotralstat is the first oral treatment approved for treatment of HAE.
 
The FDA accepted the NDA from Kadmon for belumosudil for the treatment of chronic graft-versus-host disease and set a PDUFA date for 5/30/2021.
 
The FDA accepted the NDA from BridgeBio Pharma for infigratinib to treat patients with cholangiocarcinoma, which suggests a PDUFA date in April or May 2021.
 
Iterum has submitted an NDA for oral sulopenem etzadroxil/probenecid for the treatment of uncomplicated urinary tract infections in patients with a quinolone non-susceptible pathogen.
 
TG Therapeutics initiated a rolling BLA submission for ublituximab plus umbralisib for the treatment of patients with chronic lymphocytic leukemia.
 
The FDA granted ilixadencel, a Fast Track Designation for the treatment of Gastrointestinal Stromal Tumors (GIST).
 
Agenus began a rolling BLA for balstilimab for the treatment of recurrent/metastatic cervical cancer in September 2020 and plans to complete the submission in the first half of 2021.
 
Announced Research Updates
 
Ovid announced that in the 12-week, 97 patient, Phase III NEPTUNE trial, gaboxadol did not improve the Clinical Global Impression-Improvement-Angelman syndrome (CGI-I-AS) score compared to placebo (0.7 points vs 0.8) in patients with Angelman syndrome.
 
Takeda announced that in the eight-week, 352 patient, Phase III, SOLSTICE trial, more patients treated with maribavir achieved confirmed CMV viremia clearance compared to investigator-assigned treatment (ganciclovir, valganciclovir, foscarnet, or cidofovir) in transplant patients with refractory/resistant CMV infection.
 
AB Science announced that in a 383 patient, Phase III trial, treatment with masitinib plus gemcitabine increased overall survival by 1.8 months compared to gemcitabine alone (13 vs 11.2 months) in patients with unresectable locally advanced or metastatic pancreatic cancer patients with pain at baseline or taking opioids.
 
J&J announced data after 12.4-months in the 97 patient, Phase Ib/II CARTITUDE-1 trial, where ciltacabtagene autoleucel demonstrated an overall response rate of 97% and complete response of 67% in patients with relapsed multiple myeloma.
 
Published Research Updates
 
Interim data from 96 patients enrolled in the 48-week, 420 patient, Phase III, TALENT trial, found that 80.4% of patients treated with weekly albuvirtide plus ritonavir-boosted lopinavir achieved a plasma viral load below 50 copies/mL compared to 66% of patients who received daily lopinavir-ritonavir, tenofovir and lamivudine alone in adults who  failed their first antiretroviral regimen and have HIV-1 RNA levels >= 1000 copies/mL.
 
In a 51 patient, Phase II, open-label trial, treatment with umbralisib resulted in progression-free survival of 23.5 months in chronic lymphocytic leukemia patients who are intolerant to prior BTK inhibitors or prior PI3K delta inhibitors.
 
In a 164 patient, Phase II trial, indoximod added to paclitaxel or docetaxel did not improve progression-free survival compared to taxanes alone in patients with ERBB2-negative metastatic breast cancer.
 
In the 40-month, 91 patient, Phase IIb, VITAL trial, treatment with gemogenovatucel-T resulted in recurrence-free survival of 11.5 months compared to 8.4 months with placebo in patients with stage III/IV high-grade serous, endometrioid, or clear cell ovarian cancer.
 
AstraZeneca announced that in the 805 patient, Phase III, open-label, CASPIAN trial, the addition of tremelimumab to durvalumab plus platinum–etoposide did not improve overall survival compared to durvalumab plus platinum–etoposide (10.4 vs 10.5 months), however, durvalumab plus platinum–etoposide did increase overall survival compared to platinum–etoposide (12.9 vs 10.5 months), in patients with extensive-stage small cell lung cancer.
 
In a 12-week, 61 patient, Phase II trial, median ESA usage decreased by 15,000, 15,000, and 33,000 IU/week with 2, 6, and 20 mg doses of ziltivekimab in patients on hemodialysis with inflammation and hyporesponsiveness to ESA therapy.
 
In a 24-week, 299 patient, Phase II trial, imeglimin lowered HbA1c compared to placebo by 0.52% with imeglimin 500 mg, 0.94% with 1000 mg and 1% with 1,500 mg in Japanese patients with type 2 diabetes.