Update for November 24, 2020

Regulatory Update
 
The FDA approved lonafarnib (Zokinvy, Eiger BioPharmaceuticals) on 11/20/2020, to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older.
 
The FDA approved lumasiran (Oxlumo, Alnylam Pharmaceuticals) on 11/23/2020, for the treatment of primary hyperoxaluria type 1 (PH1). The drug is well tolerated with the most common adverse effects being injection site reaction and abdominal pain. The EMA also approved lumasiran for the treatment of PH1.
 
The FDA accepted the NDA for Apellis’ pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and set a PDUFA date for 5/14/2021.
 
The FDA designated ViiV’s cabotegravir a breakthrough therapy for the prevention of HIV infection.
 
The FDA granted Sanofi’s rilzabrutinib a Fast Track designation for the treatment of immune thrombocytopenia.
 
The FDA accepted the BLA for ADC Therapeutics’ loncastuximab tesirine for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and set a PDUFA date for 5/21/2021.
 
Novartis has licensed the rights to develop and market remestemcel-L for the treatment of acute respiratory distress syndrome (ARDS).
 
Amgen ended it collaboration with Cytokinetics for the development and commercialization of omecamtiv mecarbil.
 
Announced Research Updates
 
Eiger announced in the 24-week, 26 patient, Phase IIa, open-label, LIFT trial, 17 of 22 patients (77%) that completed 24 weeks of treatment 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 log IU/mL in patients with a chronic infection of the hepatitis D virus. At Week 48 (24 weeks after completing treatment), 5/22 patients (23%) had HDV RNA below detection and 11 of 20 patients (55%) demonstrated improvement in Histology Activity Index (HAI).
 
UniQure announced that in the 26-week, 54 patient, Phase HOPE-B trial, treatment with etranacogene dezaparvovec resulted in an increase in FIX activity from < 2% to 37.2% and 72% patients reporting no bleeding events in patients with hemophilia B.
 
Elevar Therapeutics announced that in a 30 patient, Phase I/II trial, treatment with apatinib plus nivolumab resulted in an overall response rate of 13.3% in patients with locally advanced unresectable or metastatic solid tumors.
 
Published Research Updates
 
In a 16-week, 558 patient, Phase II/III trial, fasinumab 9 mg every four weeks and every eight weeks reduced the daily low back pain intensity numeric rating score compared to placebo in patients with moderate-to-severe, chronic low back pain. The 6 mg dose was no better than placebo.
 
In the 24-month, 292 patient, Phase III, trial, patients treated with odanacatib increased lumbar bone mineral density by 5.6% more than placebo in men with osteoporosis. 
 
In the 76 patient Phase Ib/II ENCORE 601 trial, treatment with entinostat and pembrolizumab lead to an objective response rate (ORR) of 9.2%, which did not meet the prespecified ORR endpoint for positivity, in patients with non-small cell lung cancer
 
In a 183 patient, Phase I, open-label, dose-ranging trial, treatment with loncastuximab tesirine resulted in an ORR of 45.6% in patients with relapsed or refractory B-cell non-Hodgkin lymphoma.