One FDA approval, two FDA actions and eleven research updates for the week ended 2/9/2019

The FDA approved caplacizumab (Cablivi by Sanofi) on 2/6/2019 for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The FDA's Oncologic Drugs Advisory Committee will review Karyopharm Therapeutics’ selinexor on 2/26/2019.

The FDA has granted Daiichi Sankyo/Plexxikon’s pexidartinib breakthrough therapy and orphan drug designations and set a PDUFA date of 8/3/2019.

1. Neurocrine Biosciences has entered a licensing deal with Voyager Therapeutics for the development and commercialization of VY-AADC.
2. In a 104 patient, Phase II trial, the addition of veliparib to temozolomide did not improve progression-free survival (PFS) or overall survival (OS) but did improve overall response rate at four months compared to temozolomide monotherapy in patients with small-cell lung cancer. PFS and OS were improved in patients with SLFN11-positive tumors.
3. In a 46 patient Phase I trial, 84% patients treated with ublituximab, umbralisib, and ibrutinib achieved an overall response in patients with advanced B-cell malignancies.
4. GenSight announced that in the 48-week, 39 patient, Phase III, RESCUE trial, the eyes treated with GS010 had a loss of visual acuity of 19 letters in the ETDRS scale compared to a loss of 20 letters in the eyes that received sham injection in patients with LHON who had suffered vision loss of less than 6 months. Vision usually decreases for 3-5 months in LHON before stabilizing. This was seen in the RESCUE trial. Improvement from the nadir to week 48 was 13 letters with GS010 and 11 letters with sham injection.
5. Eisai announced 6-month data from the 12-month, 949 patient, Phase III SUNRISE 2 trial, treatment with lemborexant reduced patient-reported sleep onset latency by 22 minutes with 5 mg and 28 minutes with 10 mg compared to 11 minutes with placebo in patients with insomnia disorder.
6. In a 65 patient, Phase III, open label trial, buparlisib did not demonstrate efficacy in treating PI3K-activated recurrent glioblastoma, likely due to incomplete blockade of the PI3K pathway.
7. In a 563 patient, Phase III trial, the addition of margetuximab increased progression-free survival by 24% compared to adding trastuzumab in previously treated HER-2 positive metastatic breast cancer patients who were receiving standard chemotherapy. 
8. In a 24-week, 257 patient, Phase II trial, treatment with alpelisib added to letrozole did not improve the objective response rate compared to monotherapy in patients with HR+ early breast cancer.
9. A four-year follow-up of two-Phase II trial involving 1,378 patients found the Sanofi dengue vaccine demonstrated long-term immunogenicity and had no safety concerns.
10. Treatment with guadecitabine in a 55 patient, Phase II trial, resulted in an overall response rate of 14.3% of patients with high-risk myelodysplastic syndrome and low blast count acute myeloid leukemia patients refractory or relapsing after azacitidine. Patients with no or few identified somatic mutations were more likely to respond.
11. In a 16-week, 205 patient, Phase II trial, 90% improvement in the Psoriasis Area and Severity Index was reached by 29.4%, 58.5% with 100mg and 66.7% with 300 mg compared to 0% with placebo in patients with moderate-to-severe plaque psoriasis.