Update for August 27, 2020

Regulatory Update
 
The FDA approved ofatumumab (Kesimpta, Novartis) on 8/21/2020, for the treatment of adults with relapsing multiple sclerosis. Ofatumumab is administered monthly via an auto injector pen. Novartis has set WAC at $83,000 per year. Ofatumumab has the same mechanism of action as ocrelizumab (Ocrevus, Roche), which is administered by intravenous infusion every six months and has an annual WAC of $65,000. Ofatumumab (Arzerra, Novartis) was initially approved as an intravenous infusion in 2009 for the treatment of chronic lymphocytic leukemia.
 
The FDA rejected valoctocogene roxaparvovec and said that at least two years of safety and efficacy data from the ongoing Phase III trial would be needed to support the sustainability of the reduced/eliminated Annualized Bleeding Rate. Three-year data from a Phase I/II trial suggested that factor VIII levels seemed to fall off after 12 to 18 months. This could potentially require additional doses to sustain efficacy, so the FDA requested additional data in a larger population. The Phase III trial is expected to be concluded in November 2021, suggesting a 2020 resubmission of the valoctocogene roxaparvovec BLA.
 
The FDA rejected filgotinib due to concerns over toxicity with the 200 mg dose. The FDA requested data from the ongoing MANTA and MANTA-RAy trials, which are not expected to be completed until 2021.  That would potentially delay resubmission of filgotinib until mid-2021. The trials are evaluating whether filgotinib 200 mg affects sperm production. All approved JAK inhibitors (baricitinib, tofacitinib and upadacitinib) have a Black Box Warning regarding an increased risk for serious infections, lymphomas and thrombosis. It is expected that filgotinib would also have this warning.
 
The FDA accepted the NDA for casimersen for the treatment of Duchenne muscular dystrophy with genetic mutations that are amenable to skipping exon 45 of the Duchenne gene and set a PDUFA date for 2/25/2021.
 
BioMarin submitted an NDA for vosoritide as a treatment for children with achondroplasia.
 
Announced Research Updates
 
Immunicum announced that in the 88 patient, Phase II, open-label, MERECA trial, treatment with ilixadencel plus sunitinib resulted in a 43% overall survival rate compared to 33% with sunitinib alone in patients with renal cell carcinoma.
 
Novartis announced that in the 568 patient, Phase III, COMBI-i Trial, spartalizumab added to dabrafenib and trametinib did not improve progression-free survival compared to dabrafenib and trametinib alone in patients with unresectable or metastatic BRAF V600 mutation-positive cutaneous melanoma.
 
Onconova announced that in the 360 patient, Phase III INSPIRE trial, treatment with rigosertib plus best supportive care did not improve overall survival (OS) compared to physician’s choice chemotherapy (6.4 vs 6.3 months) in patients with high-risk myelodysplastic syndrome.
 
Published Research Updates
 
In a 24-week, 254 patient, Phase III trial, teneligliptan lowered HbA1C by 0.95% compared to a 0.14% decrease with placebo in Chinese patients with type 2 diabetes mellitus inadequately controlled with diet and exercise.
 
In a 6-month, 58 patient, Phase II trial, adding cediranib to docetaxel plus prednisone did not improve progression-free survival in patients with metastatic castrate-resistant prostate cancer.
 
In the 24-week, 65 patient, Phase III, ELIPSE HoFH trial, treatment with evinacumab resulted in a 47.1% reduction in LDL compared to a 1.9% increase with placebo in patients with homozygous familial hypercholesterolemia (HoFH) that remained uncontrolled despite aggressive therapy.
 
24-month interim data from 8 patients enrolled in a 36-month, Phase II, open-label trial, suggested that treatment with Oxalobacter formigenes decreased or normalized plasma oxalate levels in patients with PH type 1 and early end-stage renal disease.
 
In a 36 patient, Phase I, open-label trial, treatment with andecaliximab added to gemcitabine and nab-paclitaxel resulted in progression-free survival of 7.8 months and an objective response rate of 44.4% in patients with pancreatic adenocarcinoma.
 
In the 6-month, 538 patient, Phase III ENLIGHTEN-2 trial, patients treated with olanzapine plus samidorphan had a 4.21% weight gain compared to 6.59% gain with olanzapine monotherapy in patients with stable schizophrenia.