Weekly Update for the Week Ending October 12, 2019

Regulatory Update
 
The FDA approved lasmiditan (Reyvow, Lilly) on 10/11/2019 for the treatment of acute migraines, with or without aura, in adult patients. Due to the occurrence of dizziness and sedation from CNS depression, patients are warned of potential impairment while taking lasmiditan and advised not to drive, operate machinery or take with alcohol or other CNS depressants for at least eight hours after taking the drug, even if they feel well enough to do so. The FDA announced that during clinical trials the most common ADR with lasmiditan were dizziness, fatigue, paresthesia and sedation.
 
The FDA approved brolucizumab (Beovu, Novartis) on 10/8/2019 for the treatment of wet age-related macular degeneration (AMD). Brolucizumab is administered by intravitreal Injection every three months. WAC was set at $1,850 per dose, the same as aflibercept (Eylea), which is given more frequently. After the loading dose, analysts estimate the annual brolucizumab WAC at $16,000 for both eyes compared to $24,000 for aflibercept and and $40,000 for ranibizumab (Lucentis).
 
The FDA approved afamelanotide (Scenesse, Clinuvel) on 10/8/2019 to treat phototoxic reactions in patients with erythropoietic protoporphyria.
 
The FDA accepted the BLA for Viaskin Peanut for the treatment of peanut-allergic children in October 2019 and set a PDUFA date of 8/5/2020.
 
Announced Research Updates
 
NGM announced that in a 24-week, 38 patient, Phase II trial, treatment with aldafermin resulted in a 7.9% decrease in liver fat content compared to 2% decrease with placebo in patients with biopsy-confirmed NASH with F2-F3 liver fibrosis.
 
Patients acted as their own controls in the REVERSE and RESCUE trials with one eye injected with lenadogen nolparvovec and the other eye receiving a sham injection. Both trials were done in patients with Leber Hereditary Optic Neuropathy (LHON) from the G11778A mutation in the mitochondrial ND4 gene. GenSight announced that in the 96-week, 37 patient, Phase III, REVERSE trial, lenadogen nolparvovec improved visual acuity from vision nadir by 15.4 letters in the treated eye, which was a non-significant difference compared to a 12.9 letter improvement in the eye that received a sham injection. GenSight announced that in the 96-week, 39 patient, Phase III, RESCUE trial, the eyes treated with lenadogen nolparvovec had an improvement in visual acuity of 24.9 letters in the ETDRS scale from vision nadir compared to a gain of 22.3 letters in the eyes that received sham injection. A study in monkeys determined that intravitreal injections of lenadogen nolparvovec into one eye will transfer the gene therapy to the other eye. This may explain the improvement of visual acuity in REVERSE and RESCUE, which would contradict the natural course of the disease in the untreated eye, where an improvement of 10 letters from Nadir is usually seen.
 
Helixmith announced that in a 12-month extension of the 500 patient, Phase III trial involving 101 patients, VM202 decreased the average pain score by 0.9 points compared to placebo in patients with painful diabetic peripheral neuropathy.
 
Redhill announced that in the 52-week, 30 patient, Phase III, open-label extension, MAP US2 trial, treatment with RHB-104 demonstrated a remission rate of 22.2% at week 52.
 
Published Research Updates
 
In the 2-year, 549 patient, Phase II FORWARD trial, patients treated with sprifermin 100 mcg every six months had an increase in femorotibial cartilage thickness of 0.05 mm and an increase of 0.04 mm when given every 12 months compared to placebo in patients with osteoarthritis. There was no improvement with either dosing regimen with 30 mcg of sprifermin compared to placebo. There were no improvements in WOMAC scores with any dose or frequency of sprifermin.
 
In a 12-week, 65 patient, Phase III trial, 57.6% of patients treated with relugolix had a maximum Numerical Rating Scale (NRS) score of 1 or less compared to 3.1% with placebo in patients with moderate-to-severe uterine fibroid-associated pain.