Four FDA actions plus five oncology developmental updates from the last week in February

Seven non-oncology development updates included below.

The FDA delayed a decision on NKTR-181 by three months to allow additional time to review preclinical data from two additional studies and set a new PDUFA date of 8/29/2019.

On 2/28/2019, the FDA Oncologic Drugs Advisory Committee recommend a delay in approval of selinexor-dexamethasone to treat triple class-refractory myeloma until after the results of the Phase III BOSTON trial are available in the first half of 2020.

The FDA accepted an NDA for diroximel fumarate on 2/25/2019 suggesting a PDUFA date of 12/25/2019.

The FDA designated AT-GAA as a Breakthrough Therapy in February 2019.

Twelve investigational drug developmental updates: 

1. Actinium announced interim data from the 47 patients in the 150 patient, Phase III, SIERRA trial, where 92% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant prior to day 100 compared to standard of care chemotherapy where 79% of patients failed to achieve a complete response in patients with active, relapsed or refractory acute myeloid leukemia.
2. Bayer has submitted an NDA for darolutamide for the treatment of nonmetastatic castration-resistant prostate cancer.
3. In a 59 patient, Phase II, open label trial, treatment with apatinib resulted in a progression-free survival of 3 months and overall survival of 7.93 months in patients with stage IV sarcoma who had failed chemotherapy. 
4. In a 138 patient, Phase II, open label trial, the addition of PEGPH20 to mFOLFIRINOX increased toxicity and decreased treatment duration compared to mFOLFIRINOX alone in patients with metastatic pancreatic cancer. 
5. ImmunoGen announced that in a 366 patient, Phase III trial, mirvetuximab soravtansine did not improve progression-free survival compared to standard chemotherapy in patients with platinum-resistant ovarian cancer that expressed medium to high levels of folate receptor alpha and had received up to three prior lines of therapy.
6. Santhera announced that in the 6-year, open label, SYROS trial, a long-term follow-up in 18 of 64 patients that completed the Phase III DELOS trial, where switching to or maintaining long-term treatment with idebenone reduced the rate of decline in forced vital capacity percent of predicted (FVC%p) by 50% in patients with Duchenne muscular dystrophy.
7. In a 12-week, 1,208 patient, Phase III, PERFECT 1 trial, more patients treated with trifarotene cream achieved an Investigator Global Assessment of IGA, clear/almost clear and ≥2 grade improvement than placebo (29.4% vs 19.5%), a reduction in facial lesion counts with inflammatory lesions (-19 vs -15.4) and non-inflammatory lesions ( -25 vs -17.9) in patients 9 years or older with moderate facial acne and moderate truncal acne. In a 12-week, 1,212 patient, Phase III, PERFECT 2 trial, more patients treated with trifarotene cream achieved an Investigator Global Assessment of IGA, clear/almost clear and ≥2 grade improvement than placebo (42.3% vs 25.7%), a reduction in facial lesion counts with inflammatory lesions (-24.2 vs -18.7) and non-inflammatory lesions ( -30.1 vs -21.6) in patients 9 years or older with moderate facial acne and moderate truncal acne. Mayne Pharma is evaluating trifarotene in the treatment of Lamellar Ichthyosis in a Phase II trial. Galderma has submitted an NDA for trifarotene for the treatment of facial and truncal acne.
8. Redhill plans to file an NDA for RHB-105 in the first half of 2019 with a potential launch by the end of the year if the drug is approved. 
9. Horizon announced that in the 24-week, 83 patient, Phase III, Optic trial, 82.9% of patients treated with teprotumumab had a 2 mm or more reduction in proptosis compared to 9.5% with placebo in patients with active thyroid eye disease. Horizon Pharma plans to file a BLA for teprotumumab in mid-2009. 
10. CHMP recommended approval of approval of sotagliflozin and risankizumab and conditional marketing authorization for volanesorsen.
11. In a 12-week, 43 patient, Phase II, open label trial, treatment with NGM282 reduced the NAFLD activity score by 1.9 points with 1 mg and 2.3 points with 3 mg in patients with biopsy‐confirmed nonalcoholic steatohepatitis. The fibrosis score decreased by 0.1 point with 1 mg and 0.5 points with 3 mg.
12. UCB announced that in a 60-week, long-term follow-up to the Phase IIb BE ABLE trial, 80 to 100% of bimekizumab responders maintained PASI90.