Update for the week ending August 3, 2019

Regulatory Update

The FDA approved darolutamide (Nubeqa, Bayer) on 7/31/2019 for the treatment of nonmetastatic castration-resistant prostate cancer. Darolutamide was approved three months before its PDUFA date.

The FDA approved pexidartinib (Turalio, Daiichi Sankyo) for the treatment of symptomatic tenosynovial giant cell tumor associated with severe morbidity or functional limitations and not responsive to improvement with surgery. Pexidartinib has Boxed Warning for the risk of serious and potentially fatal liver injury. The drugs is only available though a REMS program.

On 8/1/2019 Horizon Therapeutics initiated an FDA approved expanded access program for teprotumumab for up to 60 patients with active moderate to severe thyroid eye disease under ClinicalTrials.gov NCT number: NCT04040894. Physicians can request access to teprotumumab sending an e-mail to [email protected]. Horizon Therapeutics submitted a BLA for teprotumumab in July 2019.

The FDA accepted the NDA for dasotraline in July 2019 for the treatment of patients with moderate-to-severe binge eating disorder and set a PDUFA date in May 2020.

Ultragenyx has submitted an NDA for the use of triheptanoin in the treatment of long-chain fatty acid oxidation disorders.

The FDA designated elafibranor an orphan drug for the treatment of primary biliary cholangitis.

The FDA designated bempegaldesleukin in combination with nivolumab as a Breakthrough Therapy for initial treatment of unresectable or metastatic melanoma.

Announced Research Updates

Cidara announced that in 91 patients were enrolled in Part B of the STRIVE trial. Halfway through Part B of the trial, the dosing for rezafungin was changed to the dosing used in the Phase III trials, so only 15 rezafungin patients out of 46 received the higher dose. Treatment with rezafungin resulted in a higher clinical cure rate than caspofungin, but the small sample size and change in dosing limited the value and significance of the findings.

Sanofi announced that in the 26-week, Phase III SOTA-MET trial, treatment with sotagliflozin reduced HbA1c compared to placebo in type 2 diabetic patients receiving metformin. Sanofi announced that in the 26-week, Phase III SOTA-CKD3T trial, treatment with sotagliflozin reduced HbA1c compared to placebo in all patients with type 2 diabetes chronic kidney disease, but in a subgroup glomerular filtration rate of 30-<45 mL/min/1.73m2, there was no improvement with sotagliflozin over placebo. Sanofi announced that in the 26-week, Phase III SOTA-CKD4 trial, treatment with sotagliflozin did not improve HbA1c compared to placebo in type 2 diabetic patients with stage 4 chronic kidney disease. Sanofi ceased participation in the development of sotagliflozin after results from the 3 Phase III type 2 diabetes trials were announced.

Biogen announced that in the 5-week, 506 patient, Phase III, EVOLVE-MS-2 trial, where patients treated with diroximel fumarate reported fewer days with GI symptoms with a symptom intensity score ≥2 in patients with relapsing-remitting multiple sclerosis.

AB Science discontinued development of masitinib for melanoma after difficulties completing a Phase III trial, where the dacarbazine comparator arm was halted for ethical reasons at the request of regulatory agencies. Development of masitinib continues for the treatment of pancreatic cancer and prostate cancer.

Pfizer announced that in the 42-day, 345 patient, Phase III, RESET trial, treatment with rivipansel did not improve time to readiness-for-discharge compared to placebo in patients with sickle cell disease who were hospitalized for a vaso-occlusive crisis and required treatment with intravenous (IV) opioids

Published Research Updates

In a 12-week, 52 patient open label trial, switching patients to blonanserin decreased the Brief Psychiatric Rating Scale score by 13.7 points in schizophrenic patients unresponsive to other antipsychotic treatments.

In a 12-week, 52 patient open label trial, switching patients to blonanserin decreased the Brief Psychiatric Rating Scale score by 13.7 points in schizophrenic patients unresponsive to other antipsychotic treatments.

In a 49 patient, Phase Ia/Ib, dose ranging trial, 20% of patients treated with the combination of tislelizumab and pamiparib achieved an objective response in patients with advanced solid tumors.