Update for December 15, 2020

Regulatory Update
 
The FDA notified BMS of problems with sterilization procedures at the Washington plant that will manufacturer lisocabtagene maraleucel.
 
The FDA accepted the NDA for Scynexis's ibrexafungerp for the treatment of vulvovaginal candidiasis and set a PDUFA date for 6/1/2021.
 
The FDA accepted the NDA for Chimerix’s brincidofovir for the treatment of smallpox and set a PDUFA date for 4/7/2021.
 
The FDA accepted the NDA for reltecimod for the treatment of suspected organ dysfunction or failure in patients 12 years or older with necrotizing soft tissue infection, in conjunction with surgical debridement, antibiotic therapy, and supportive care. A PDUFA date has been set for 9/30/2021.
 
The FDA granted a Fast Track Designation to Immunicum’s Ilixadencel for the treatment of Gastrointestinal Stromal Tumors (GIST).
 
The FDA designated Amgen’s sotorasib a Breakthrough Therapy, for the treatment of locally advanced or metastatic non-small cell lung cancer with KRAS G12C mutation.
 
The FDA granted mavorixa a Fast Track designation for the treatment of WHIM syndrome.
 
The EMA has approved inclisiran for the treatment of hypercholesterolemia and mixed dyslipidemia.
 
Sanofi resumed fitusiran clinical trials in December 2020 with an adjusted dosing regimen designed to reduce adverse effects.
 
Announced Research Updates
 
ADC announced interim results from 18 patients enrolled in the Phase I/II, open-label, LOTIS 3 trial, where treatment with loncastuximab tesirine plus ibrutinib resulted in an ORR of 58.6% in patients with relapsed or refractory DLBCL and 83.3% in patients with mantle cell lymphoma (MCL). Patients with non-germinal center B-cell DLBCL had an ORR of 66.7% compared to 20% in germinal center B-cell DLBCL patients.
 
Oncopeptides announced interim data from 46 patients enrolled in the Phase II, open-label, ANCHOR trial, where patients treated with melflufen and dexamethasone plus daratumumab achieved an ORR of 73%, while patents treated with melflufen and dexamethasone plus bortezomib achieved a 62% ORR in patients with relapsed refractory multiple myeloma.
 
Gilead announced interim results from 64 patients enrolled in a Phase I, open-label trial, where treatment with magrolimab plus azacitidine resulted in a 63% objective response rate (ORR) in patients with myelodysplastic syndrome and a 63% ORR in patients with acute myeloid leukemia, who are ineligible for induction chemotherapy.
 
Lilly announced that in the 40-week, 478 patient, Phase III, SURPASS-1 trial, treatment with tirzepatide lowered HbA1c 1.87% with 5 mg, 1.89% with 10 mg and 2.07% with 15 mg compared to a 0.04% increase with placebo in patients with type 2 diabetes. Treatment with tirzepatide also lead to weight loss of 7 kg with 5 mg,7.8 kg with 10 mg and 9.5 kg with 15 mg compared to a 0.7 Kg loss with placebo. 
 
Biosight announced interim data from 31 patients enrolled in a Phase IIb trial, where treatment with aspacytarabine resulted in a 32% complete response rate in patients with acute myeloid leukemia who are not eligible for standard induction chemotherapy due to advanced age or comorbidities.
 
AlloVir announced that in the six-week, Phase II, CHARMS trial, patients treated with ALVR105 achieved a 93% clinical response (defined as complete response - viral load returning to normal range and resolution of clinical signs/symptoms or a partial response (50% or > decrease in viral load and/or 50% improvement of clinical signs/symptoms) in allo-hematopoietic stem cell transplantation patients with at least one treatment-refractory infection.
 
Roche announced interim results from 62 patients enrolled in a Phase I/Ib, open-label trial, where treatment with mosunetuzumab induced a complete response in 32/62 patients with relapsed or refractory follicular lymphoma.
 
Inovio announced that in a 6-month, 22 patient, Phase II trial, treatment with VGX-3100 resulted in resolution of HPV-16/18-associated precancerous anal lesions in 11/22 patients with high grade anal squamous intraepithelial lesions caused by HPV-16/18.
 
TG Therapeutics announced that in two 96-week Phase III trials involving 1,094 patients treatment with an intravenous infusion of ublituximab every six months resulted in a 60% annualized relapse rate (ARR) in ULTIMATE I and a 50% ARR in ULTIMATE II compared to daily oral teriflunomide in patients with relapsing forms of multiple sclerosis.
 
Apellis announced that in a 36-week, 77 patient, open-label extension of the Phase III PEGASUS trial, patients that continued pegcetacoplan maintained a hemoglobin increase of 2.7 g/dL and patients switched from eculizumab experienced a similar increase.
 
Published Research Updates
 
In a 24-week, 42 Japanese patient, Phase III, open label trial, treatment with vadadustat resulted in a mean Hb of 11.35 g/dL in patients with peritoneal dialysis-dependent CKD. 
 
In a 130-week, 69 peanut-allergy patient, Phase II trial, patients received 52-weeks of Viaskin Peanut 100 mcg, 250 mcg or placebo. After 52-weeks, all patients were switched to 250 mcg of Viaskin Peanut. At 130 weeks, desensitization was achieved in 1/20 placebo-250 mcg patients, 5/24 100 mcg-250 mcg patients and 9/25 participants who received 250 mcg for the full treatment duration.
 
In the 48-week, 1,045 patient, Phase III ATLAS-2M trial, 2% of patients that received IM injection of cabotegravir and rilpivirine every two months had an HIV-1 RNA of 50 copies/mL or > compared to 1% of patients that received the combination monthly, achieving non-inferiority for less frequent dosing.
 
In the 96-week, 37 patient, Phase III, REVERSE trial, lenadogen nolparvovec improved visual acuity from vision nadir by 15 letters in the treated eye, which was a non-significant difference compared to a 13 letter improvement in the eye that received a sham injection in patients with visual loss from Leber hereditary optic neuropathy
 
In a 26-week, 191 patient, open-label extension of a Phase III trial, patients that continued ravulizumab had lactate dehydrogenase (LDH) levels increased by 8.8% and four patients experienced breakthrough hemolysis compared to an LDH increase of 5.8% and one case of breakthrough hemolysis in patients switched from eculizumab to ravulizumab.
 
In the 157 patient, Phase II, open-label, HORIZON trial, treatment with melflufen plus dexamethasone resulted in an overall response rate of 29% in patients with multiple myeloma.