Prescribe Right Pharmaceutical Pipeline Update for September 11

Update on investigational drugs with recent PDUFA dates:

• In a 1,064 patient, Phase III, CAPSTONE-1 trial, a single dose of baloxavir marboxil (Genentech, Shionogi) reduced the time to alleviation of symptoms of influenza infection by 26.5 hours compared to placebo and was similar to 5 days of oseltamivir. Baloxavir marboxil also reduced viral load more rapidly than oseltamivir or placebo. The drug’s PDUFA date is December 24, 2018, but has no Priority Designations at present. 
  
• A phase II study showed a decrease in tumor volume and 4-5-month progression-free survival with talazoparib in resistant BRCA1/2 mutation breast cancer patients. Pfizer announced that in a 20 patient, Phase II trial of patients with BRCA+ breast cancer, patients received 6 months of talazoparib prior to surgery. Before surgery, 63% of patients had a residual cancer burden score of RCB0 or RCB1, which indicates the same positive expected outcome. In the 287 patient, Phase III, EMBRACA trial, patients treated with talazoparib had a PFS of 8.6 months compared to 5.6 months with standard chemotherapy and an ORR of 63% vs 27% in patients with advanced breast cancer who carry the BRCA1/2 mutations. PDUFA Date: December 15, 2018. No Priority Designations.

Ablynx’ Caplacizumab (Cablivi) is now approved in Europe. No PDUFA date, however, it has an Orphan Drug Priority Designation.

The FDA rejected Sunovion Pharmaceuticals’ dasotraline on 8/31/18 and requested additional data on efficacy and tolerability of dasotraline for the treatment of ADHD.

In a Phase III trial of 32 patients with heart failure and renal impairment, Orion Pharma’s levosimendan compared to dobutamine had similar increases in renal blood flow (22% and 26%, respectively), better glomerular filtration rate, and filtration fraction was not affected by levosimendan but decreased by 17% with dobutamine.

Biocryst announced topline results from the high dose cohort of the 95 patient, ZENITH-1 Phase II trial, where 64% of patients with Hereditary Angioedema, treated with BCX7353 had mild or no symptoms compared to 32% with placebo. BCX7353 reduced the need for rescue drugs by 32%. BCX7353 has a Fast Track Priority Designation but no PDUFA date.

China approved fruquintinib (Chi-Med, Lilly) in Sept 2018 for the treatment of metastatic colorectal cancer.

Bluebird Bio announced Interim results from the phase II/III Starbeam Study which demonstrated that 15 of 17 childhood cerebral adrenoleukodystrophy patients treated with Elivaldogene tavalentivec were free of major functional disabilities and no graft versus host disease almost 30 months after treatment. Twenty-nine patients had at least 4.2 months of follow-up and none had experienced grade 2 or higher acute graft-versus-host disease. The drug has a Breakthrough Therapy Priority Designation but no PDUFA date.

After failure in the CONCERTO and ARPEGGIO trials, Teva stopped development of laquinimod for multiple sclerosis. After the failure of a Phase II Huntington's disease trial, Teva ceased all development projects for laquinimod and returned all rights to Active Biotech.

Gilead and Galapagos announced that in the 12-week, 116 patient Phase II, TORTUGA trial, patients treated with filgotinib had a 1.5-point decrease in their Ankylosing Spondylitis Disease Activity Score (ASDAS) compared to a 0.6 decrease with placebo.

J&J has an ongoing Phase III trial testing esketamine for treatment-resistant depression and major depressive disorder with imminent risk of suicide. J&J filed an NDA for esketamine in September 2018 and plans to file an MAA before the end of 2018. 

Tezepelumab (AstraZeneca, Amgen) has been granted breakthrough status. No PDUFA date.

You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.