The FDA approved cefiderocol (Fetroja, Shionogi) on 11/15/2019 for the treatment of adult patients with complicated urinary tract infections (cUTI), including kidney infections caused by susceptible Gram-negative microorganisms, who have limited or no alternative treatment options. The package insert for cefiderocol will include a warning regarding higher all-cause mortality rate observed in cefiderocol-treated patients compared to those treated with other antibiotics in a trial in critically ill patients with multidrug-resistant Gram-negative bacterial infections.
The FDA approved crizanlizumab (Adakveo, Novartis) on 11/15/2019 to reduce the frequency of vaso-occlusive crisis in patients aged 16 years or older with sickle cell disease. WAC for crizanlizumab is $2,357 per vial. With an average dose of three to four vials per month, WAC would be $7,071, or $9,428, per month.
The FDA approved zanubrutinib (Brukinsa, BeiGene) on 11/13/2019 for the treatment of adults with mantle cell lymphoma who have received at least one prior therapy. WAC for a 30-day supply of zanubrutinib has been set at $12,935.
The FDA designated aztreonam/avibactam as a Qualified Infectious Disease Product (QIDP) and gave the combination Fast Track status for the treatment of complicated intra-abdominal infections (cIAI), complicated urinary tract infections (cUTI), and hospital-acquired bacterial pneumonia (HABP)/ventilator-associated bacterial pneumonia (VABP).
The EU approved Merck’s ebola vaccine.
The EMA’s CHMP recommended approval of osilodrostat for the treatment of Cushing syndrome.
Solid Biosciences’ SGT-001 had temporary holds on the Phase I/II IGNITE DMD trial in 2017 and 2018. The November 2017 hold was due to manufacturing problems and the January 2018 hold was due to a patientdeveloping thrombocytopenia and anemia. Both holds were resolved and the trial resumed in June 2018. The company added monitoring for complement activation and IV steroids to the protocol and made eculizumab available to treat complement activation if it developed. In November 2019, the FDA put a hold on the IGNITE DMD trial when a patient developed complement activation leading to thrombocytopenia, anemia, acute kidney injury, and cardio-pulmonary insufficiency. There were no cytokine-related nor coagulopathy-related abnormalities observed in the patient. The patient had received the highest dose of SGT-001, which is four times higher than the patient in the previous trial.
The FDA designated mavorixafor a Breakthrough Therapy for the treatment of WHIM syndrome.
The FDA granted a priority review to selumetinib to treat children with neurofibromatosis type 1 who've developed plexiform neurofibromas that cannot be treated with surgery.
CytoDyn has initiated an expanded access program for use of leronlimab in patients with triple-negative breast cancer who have exhausted all approved treatment options and do not qualify for a clinical trial. Details of the program can be found at: https://www.cytodyn.com/expanded-access
Announced Research Updates
Roche announced that in the 12-month, 180 patient, Phase III SUNFISH trial, treatment with risdiplam improved motor function as measured by the Motor Function Measure 32 (MFM32) score in patients with Type 2 or 3 spinal muscular atrophy.
Reata announced that in the 48-week, 157 patient, Phase III CARDINAL trial, treatment with bardoxolone resulted in an increase in the mean eGFR of 4.72 mL/min/1.73 sq m compared to a decrease of 4.78 mL/min/1.73 sq m with placebo in patients with Alport syndrome. After a 4-week wash-out period, the bardoxolone had a decline from baseline of 0.96 mL/min/1.73 sq m compared to a 6.11 mL/min/1.73 sq m decrease with placebo.
Akebia announced that in a 52-week, 304 Japanese patient, Phase III, open label trial, vadadustat was non-inferior to darbepoetin alfa in mean Hb (11.51 g/dL vs 11.58 g/dL) in patients with non-dialysis dependent CKD. In a 52-week, 323 Japanese patient, Phase III, trial, vadadustat was non-inferior to darbepoetin alfa in mean Hb (10.39 g/dL vs 10.62 g/dL) in patients with hemodialysis-dependent CKD.
AZ announced that in the 12-month, 373 patient, Phase III, TULIP 2 trial, 47.8% of patients treated with anifrolumab had a decrease in disease activity compared to 31.5% with placebo in patients with moderate-to-severe autoantibody-positive systemic lupus erythematosus.
UCB announced that in a 48-week, 265 patient, Phase IIb trial, 35.5%–64.0% of patients treated with bimekizumab achieved ASAS40 in patients with ankylosing spondylitis.
Mallinckrodt announced that in the 35-month, 300 patient, Phase III CONFIRM trial, 29.1% of patients treated with terlipressin achieved HRS-1 reversal (renal function improvement, avoidance of dialysis, short-term survival) compared to 15.8% with albumin monotherapy in patients with HRS-1.
Eiger announced interim data from 19 patients enrolled in the 24-week, 26 patient, Phase IIa, open-label, LIFT trial;18 /19 patients treated with the combination of lonafarnib, ritonavir and pegylated interferon lambda achieved a > 2 log IU/mL decline in HDV RNA with a median decline of 3.4 log IU/mL in patients with a chronic infection of the hepatitis delta virus.
Gilead and Galapagos announced that an extension of the Phase II DARWIN 1 & 2 trials monitored 739 patients for 156 weeks in the Phase II, DARWIN 3, open label trial, where AR20 was achieved by 89.7% of patients that received filgotinib monotherapy and 87.2% of patents that received filgotinib and MTX.
AnaptysBio announced that in a 20 patient, Phase IIa trial, 11/15 patients treated with etokimab were able to tolerate a 275 mg dose of peanut protein two weeks after a single injection compared to none (0/5) in the placebo group in patients with peanut allergy and a clinical history of anaphylaxis. At 45 days, 4/7 patients passed the peanut protein challenge compared to none with placebo.
BioMarin announced results from a 10 patient cohort enrolled in a 54 month Phase II, open label, achondroplasia trial, where treatment with vosoritide 15 µg/kg/day resulted in a mean increase of 9 cm compared to an age and gender matched natural history achondroplasia dataset (N=619).
ResTORbio announced that in the 16-week, 1,024 patient, Phase III, PROTECTOR 1 trial, treatment with dactolisib during winter cold and flu season did not decrease respiratory tract infections (RTI) compared to placebo (odds of experiencing an RTI 0.46 vs 0.44) in patients 65 or older, excluding current smokers and individuals with chronic obstructive pulmonary disease. Development of dactolisib for the prevention of respiratory tract infections in older patients was discontinued after the failure of the Phase III PROTECTOR 1 trial. Dactolisib remains in development with or without everolimus in the treatment of Parkinson’s disease with a Phase 1b/2a trial that is expected to be completed in mid-2020.
The Medicines Company announced that in the 18-month, 1,561 patient, Phase III, ORION-10 trial, the placebo adjusted reduction in LDL with inclisiran was 58% in patients with atherosclerotic cardiovascular disease or risk equivalents and elevated LDL despite maximum tolerated dose of statin with or without ezetimibe. In the 18-month, 1,617 patient, Phase III, ORION-11 trial, major adverse cardiovascular events (MACE) events were 47% lower with inclisiran than the placebo, but in the 18-month, 1,561 patient, Phase III, ORION-10 trial MACE events were slightly higher with inclisiran (4.1% vs 3.3%).
Published Research Updates
In the 12-week, 779 patient Phase III, CLEAR Wisdom trial, patients treated with bempedoic acid lowered LDL 15.1% compared to 2.4% with placebo in patients with atherosclerotic cardiovascular disease, heterozygous familial hypercholesterolemia, or both and LDL of 70 mg/dL or > despite maximally tolerated lipid-lowering therapy.
In the 12-week, 156 patient, Phase II, OASIS trial, treatment with etrasimod 2 mg improved the Mayo Clinic Score (a composite of rectal bleeding, stool frequency, and endoscopic findings) 1 point more than placebo, but the 1 mg dose was not significantly different from placbo in patients with moderate to severe ulcerative colitis.
In the 11.6-month, 307 patient Phase III, open label, ICARIA-MM trial, isatuximab added to pomalidomide and low-dose dexamethasone resulted in progression free survival of 11.5 months compared to 6.5 months with pomalidomide and dexamethasone alone in patients with relapsed/refractory multiple myeloma.
In a 31-month, 50 patient, Phase II trial, treatment with Lu-PSMA-617 resulted in a decrease in PSA values of at least 50% in 64% of patients and median overall survival was 13.3 months in men with metastatic castration-resistant prostate cancer.
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