New Drug Update for The Week Ending June 8

Regulatory Update

The FDA accepted the BLA and the EMA accepted the MAA for Celgene/Acceleron’s luspatercept in June 2019.

The FDA granted Gilead’s momelotinib Fast Track status in June 2019.

Bluebird Bio’s beta beglogene darolentivec was approved in Europe in June 2019.

Announced Research Updates

Sierra will evaluate momelotinib in the treatment of anemia in patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera (PV) Myelofibrosis, or Post Essential Thrombocythemia (ET) Myelofibrosis who were Previously Treated with JAK Inhibitor Therapy in the Phaser III MOMENTUM trial with plans to launch the trial in 4Q19.

Allegro announced that in a 28-week, 40 patient, Phase II trial, 48% of patients with intermediate dry AMD that received two doses of risuteganib (weeks 1 & 16) gained at least 8 letters of vision at week 12 compared to 7% that received placebo.

Published Research Updates

In a 6-month, 58 patient, Phase II trial, adding AstraZeneca’s cediranib to docetaxel plus prednisone did not improve progression-free survival rate compared to docetaxel plus prednisone alone in patients with in patients with metastatic castrate-resistant prostate cancer. In a 24-week, 48 patient, Phase II trial, cediranib reduced target marker lesion diameters by 8.3% compared to a 13.4% increase with placebo in patients with in patients with metastatic alveolar soft-part sarcoma.

A 21 patient Phase II trial was stop early after an interim futility analysis found no benefit for Roche’s taselisib in the treatment of patients with PI3K pathway-activated squamous non-small cell lung cancer.

In the 24-hour, Phase III APOLLO-2 trial, treatment with PCA oliceridine (Travena) resulted in effective analgesia in 61.0% that received 0.1 mg, 76.3% with 0.35mg, and 70.0% with 0.5 mg compared to 45.7% with placebo and 78.3% for morphine in patients with acute pain following abdominoplasty.  All doses of oliceridine were superior to placebo, but only the 0.35mg and 0.5mg doses were equianalgesic to morphine. 

In the 26-week, 711 patient, Phase III Pioneer-4 trial, daily oral semaglutide (Novo Nordisk) HbA1c was lowered by 1.2% compared to a 1.1% reduction with daily liraglutide and 0.2% with placebo in type 2 diabetic patients. In the 52-week, 504 patients, Phase III Pioneer-7 trial, 58%of patients treated with oral semaglutide achieved an HbA1c < 7% compared to 25% with sitagliptin in patients with inadequately controlled type 2 diabetes.

Ambit announced that in the 23.5-month, 367 patient, Phase III, QuANTUM-R trial, treatment with quizartinib resulted in an overall survival of 6.2 months compared to 4.7 months with salvage chemotherapy in patients with relapsed/refractory FLT3-ITD myeloid leukemia. 

In a 69-week, 104 patients, Phase II trial, subcutaneous crenezumab (Roche) lowered oligomeric forms of amyloid beta-protein by 48% compared to a 43% decrease with intravenous crenezumab and no chance with placebo in patients with Alzheimer's disease.
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In a 76 patient, Phase II trial, the mean time to develop type 1 diabetes was 48.4 months in patients that received a 14-day course of MacroGenics’ teplizumab compared to 24.4 months with placebo in patients at high-risk of developing type 1 diabetes. Diabetes was diagnosed in 19 (43%) of the teplizumab patients compared to 23 (72%) with placebo.