Update for July 13, 2021

Regulatory Update
 
The FDA approved finerenone (Kerendia, Bayer), on 7/9/2021, to reduce the risk of kidney function decline, kidney failure, cardiovascular death, non-fatal heart attacks, and hospitalization for heart failure in adults with chronic kidney disease associated with type 2 diabetes. Bayer set WAC for finerenone at $570 per month.
 
The FDA accepted the BLA for AstraZenec’s and Amgen’s tezepelumab for the treatment of asthma.
 
The FDA accepted the NDA for Levo Therapeutics’ intranasal carbetocin for the treatment of hyperphagia and behavioral distress associated with Prader-Willi syndrome.
 
The FDA granted Fast Track status to Opthea’s OPT-302, in combination with anti-VEGF-A therapy, for the treatment of age-related macular degeneration.
 
Announced Research Updates
 
Abeona announced six-year results from a Phase I/IIa trial, where treatment with EB-101 resulted in wound healing of 50% or more in 69% (18/26) at 3-years, 93% (14/15) at 4 years, 80% (12/15) at 5 years, and 80% (4/5) at 6 years in patients with recessive dystrophic epidermolysis bullosa.
 
Sierra announced that in the 24-week, 432 patient, Phase III, SIMPLIFY-1 trial, transfusion independence was achieved by 67% of momelotinib patients compared to 49% with ruxolitinib. Sierra announced that in the 24-week, 156 patient, open-label, Phase III, SIMPLIFY-2 trial, transfusion independence was achieved by 43% of momelotinib patients compared to 21% of patients who received best available therapy (88% ruxolitinib) in thrombocytopenic patients with primary myelofibrosis, post-polycythemia vera or post-essential thrombocythemia myelofibrosis.
 
Published Research Updates
 
In a 7-month, 1,452 patient, open-label, Phase III, trial, 11.7% of patients treated with cytisinicline achieved carbon monoxide breath test confirmed abstinence compared to 13.3% in the varenicline group in Australian adult daily smokers. The results did not reach the non-inferiority margin of a 5% difference.
 
In the 12-week, 80 patient, Phase IIa, dose ranging, BALANCED trial, treatment with efruxifermin reduced hepatic fat fraction by 12.3% with 28 mg, 13.4% with 50 mg and 14.1% with 70 mg compared to a 0.3% decrease with placebo in patients with nonalcoholic steatohepatitis (NASH).
 
In the ANGEL-MS trial, a 48 week, 154 patient, extension of the CHANGE-MS trial, treatment with temelimab did not reduce gadolinium-enhanced T1-lesions compared to placebo at week 48 or 96.
 
In the 28-week, 116 patient, Phase III CASCADE trial, treatment with tezepelumab resulted in a nominal reduction in airway submucosal inflammatory cells in bronchoscopic biopsy samples compared to placebo (ratio of geometric least-squares means 0·15) in patients with uncontrolled, moderate-to-severe asthma.
 
Roche announced that in the 1,101 patient, Phase II, IPATential150 trial, ipatasertib, added to abiraterone and prednisolone, improved radiographic progression-free survival compared to abiraterone plus prednisolone alone (18.5 months vs 16.5 months) in 521 patients with metastatic castration-resistant prostate cancer (mCRPC) with PTEN loss. In the intent-to-treat population, there was no significant improvement with the addition of ipatasertib.