New Drug Update for The Week Ending June 15

Regulatory Update
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The FDA approved polatuzumab vedotin (Polivy, Genentech/Roche) on 6/10/2019 in combination with bendamustine and rituximab for the treatment of adults with resistant diffuse large B-cell lymphoma.

In June 2019, the FDA accepted the NDA and the EMA accepted the MAA for ozanimod. Celgene estimates an FDA decision on approval of ozanimod by 3/25/2020 and approval of the MAA in the first half of 2020.

After Novavax reported a failed Phase III trial in March, the FDA recommended an additional Phase III trial evaluating their respiratory syncytial virus vaccine’s (ResVax) ability to decrease RSV disease in infants born to mothers vaccinated during pregnancy.

The FDA's Psychopharmacologic Drugs Advisory Committee will review lumateperone on 7/31/2019. The drug is an atypical antipsychotic targeting acute and residual schizophrenia.

The EMA approved beta beglogene darolentivec in June 2019. Bluebird set a price of $1.77 million in Europe, which is paid as 315,000 euros per year, over 5 years. Payments are stopped or reduced if a patient requires an infusion after receiving a treatment. However, no European country has accepted the price yet. Bluebird forecasts approval in the US in 2020.

Announced Research Updates

Lilly announced that in a 52-week, 103 patient, Phase II trial, patients received placebo or mirikizumab for 16 weeks and then all patients received mirikizumab 300 mg every 8 weeks for 46 weeks if they had not reached PSASI-90. At the end of the 16-week period only 6.5% of patients had achieved complete scalp psoriasis clearance, but at the end of 46 weeks of maintenance treatment with mirikizumab, 80.4% had achieved scalp clearance in patients with scalp psoriasis.

Karyopharm announced interim results from three arms of the Phase Ib/II, STOMP, relapsed or refractory multiple myeloma trial. In one arm, selinexor, daratumumab and dexamethasone achieved an overall response rate in 73% of patients. In the second arm, selinexor, pomalidomide and dexamethasone achieved an overall response rate in 50% of patients. In the second arm, selinexor, carfilzomib and dexamethasone achieved an overall response rate in 78% of patients. 

Published Research Updates

Oral semaglutide

• In the 26-week, 703 patient, Phase III Pioneer-1 trial, oral semaglutide lowered HbA1c 0.6% with 0.3 mg, 0.9% with 7mg and 1.1% with 14mg more than placebo in patients with type 2 diabetes.
• In the 26-week, 324 patient, Phase III Pioneer-5 trial, patients treated with oral semaglutide lowered their HbA1c 1.0% and lost 3.4 kg compared to a HbA1c decrease of 0.2% and 0.9 kg loss with placebo in patients with type 2 diabetes and moderate renal impairment. 
• In the 16-week, 3,183 patient, Phase III, PIONEER-6 trial, semaglutide was non-inferior to placebo in the occurrence of a combined endpoint of cardiovascular death, non-fatal myocardial infarction or non-fatal stroke compared to placebo (3.8% occurrence vs 4.8%) when added to standard care in patients with type 2 diabetes. 

• Five of the semaglutide Pioneer trials have been published in the past two weeks, so we’ll provide a cheat sheet for all ten trials on Thursday.​
  

In a 12-week, 41 patient, Phase III, open label trial, patients treated with PBI-4050 monotherapyor PBI-4050 plus ninetedamib had no change in forced vital capacity (FVC), but PBI-4050 plus perfenidone caused a decline in FVC in patients with idiopathic pulmonary fibrosis. The combination of PBI-4050 and perfenidone resulted in reduced absorption and a shorter half-life of PBI-4050.

In a 12-week, 79 patient, Phase IIa Trial, patients that received bimekizumab plus certolizumab had a 1.41 decrease in their DAS28(CRP) compared to a 0.82 decrease with certolizumab monotherapy in patients with rheumatoid arthritis that remained uncontrolled after 8-weeks of certolizumab.

In the 262 patient, Phase III, open-label, SOLITAIRE-U trial, solithromycin did not reach non-inferiority compared to ceftriaxone/azithromycin (80% eradication vs 84%) in patients infected with gonorrhea with or without chlamydia infection.  

In a 69-week, 104 patient, Phase II trial, subcutaneous crenezumab lowered oligomeric forms of amyloid beta-protein by 48% compared to a 43% decrease with intravenous crenezumab and no change with placebo in patients with Alzheimer's disease.

In a 36-week, 14 patient, Phase I/II, open label trial, VTS-270 slowed decreases in cognition and adaptive behavior in patients with Niemann-Pick Type C1.

In the 24-week, 274 patient Phase III, GBT HOPE trial, 51% of patients treated with voxelotor 1,500 mg had at least a 1 g/dL increase in hemoglobin compared to 7% with placebo in patients with sickle cell disease. 

In a 62 patient, Phase II trial, intermittently dosed pemetrexed added to platinum chemotherapy resulted in an objective response rate of 35%, continuous administration of pemetrexed and platinum chemotherapy resulted in an ORR of 62% and platinum chemotherapy alone resulted in an ORR of 24% in patients with non-small cell lung cancer.

In a 179 patient, Chinese trial, treatment with icotinib plus pemetrexed and carboplatin resulted in a progression-free survival of 16 months compared to 10 months with icotinib monotherapy in patients with advanced lung adenocarcinoma with sensitive epidermal growth factor receptor (EGFR) mutations.

In a 50 patient, 14-day, Phase III trial, terlipressin reduced portal venous pressure compared to placebo in patients undergoing hepatobiliary surgery.