Weekly Update for the Week Ending September 21, 2019

Regulatory Update
 
The FDA approved daily oral semaglutide (Rybelsus, Novo Nordisk) on 9/20/2019 for the treatment of Type 2 Diabetes. Novo plans to have a commercial insurance savings card program when Rybelsus is launched to reduce out of pocket costs to as little as $10 a month. WAC for a month’s supply of oral semaglutide is $772 (30 tablets) which is similar to 4 weeks of injectable semaglutide (Ozempic) at $770. The WAC for a month’s supply of injectable liraglutide is $920. Oral semaglutide was approved with a Black Box warning regarding a potential increased risk ofthyroid c-cell tumors. The drug is contraindicated for patients with a past history or family history of medullary thyroid carcinoma or Multiple Endocrine Neoplasia syndrome type 2.
 
The FDA has accepted the BLA enfortumab vedotin for the treatment of locally advanced or metastatic urothelial cancer and set a PDUFA date of 3/15/2020.
 
The FDA has accepted the BLA for Merck’s Ebola vaccine for the prevention of infections with the Ebola virus and set a PDUFA date of 3/14/2020.
 
Announced Research Updates
 
Nanobiotix announced interim results from a Phase I/II trial, where 62.5% of hepatocellular carcinoma patients treated with NBTXR3 activated by stereotactic body radiation therapy (SBRT) have shown a complete response with the remainder demonstrating a partial response and 50% of patients with liver metastasis have shown a complete response with the remainder showing stable disease.
 
Sage Therapeutics announced that in a 45-day, 150 patient, Phase II, trial, a daily oral dose of zuranolone decreased the Hamilton Rating Scale for Depression (HAMD-17) at 15 days by 17.8 points and by 19.2 points at 45 days compared to a 13.6 points decrease with placebo at 15 days and a 14.2 point decrease at 45 days in women with severe post-partum depression.
 
MicuRx announced that in a 589 patient, Phase III trial, contezolid was non-inferior to linezolid (clinical cure 93% vs 93.4%) in Chinese patients with complicated skin and soft tissue infections. MicuRx is comparing contezolid to linezolid for the treatment of skin and skin structure infections in an ongoing U.S. Phase II trial.
 
Published Research Updates
 
In a 130-week, 120 patient, Phase II trial, patients were desensitized with a non-commercial peanut flour to tolerate 4000 mg of peanut protein. The patients were then assigned to no maintenance therapy, low dose peanut protein (300 mg) or placebo (oat flour) and retested. At 117 weeks the number of patients that passed a 4000 mg peanut protein challenge were 21/60 in the no therapy group, 19/35 in the low dose maintenance group and 1/25 in the placebo group. After 130 weeks the number of patients that passed the challenge test were 12/60 in the no therapy group, 15/35 in the low dose maintenance group and 1/25 in the placebo group. This suggested that discontinuation or a low dose maintenance after achieving a desensitization goal may result in patients losing their tolerance for the target peanut protein level.
 
In the  26-week, 731 patient, Phase III Pioneer-8 trial, treatment with oral semaglutide lowered HbA1c 0.5% with 3 mg, 0.9% with 7 mg and 1.2% more than placebo and lowered body weight 0.9 Kg with 3 mg, 2 kg with 7 mg and 3.3 kg with 14 mg more than placebo in patients with type 2 diabetics.
 
In a 38 patient, Phase III trial, treatment with galeterone did not improve radiographic progression-free survival compared with enzalutamide in patients with androgen receptor splice variant-7 (AR-V7) metastatic castration-resistant prostate cancer (mCRPC).
 
In an 8-week, 14 patient, Phase II trial, treatment with serlopitant did not improve the itch numeric rating scale compared to placebo in patients with moderate-severe epidermolysis bullosa related pruritus.