Update for June 24, 2020

Regulatory Update

The FDA approved lurbinectedin (Zepzelca, Jazz Pharma/PharmaMar) on 6/16/2020 for the treatment of metastatic small cell lung cancer that has progressed during or after treatment with platinum-based chemotherapy.
 
The FDA rejected Nabriva Therapeutics/Zavante Therapeutics’ injectable fosfomycin a second time in June 2020 due to the same manufacturing deficiencies found in 2019.
 
The FDA has accepted the NDA for Myovant’s relugolix for the treatment of advanced prostate cancer, and set a PDUFA date of 12/20/2020.
 
The FDA designated Insmed’s brensocatib as a Breakthrough Therapy for the treatment of non-cystic fibrosis bronchiectasis.
 
TG Therapeutics submitted an NDA for umbralisib for the treatment of marginal zone lymphoma and follicular lymphoma.
 
The FDA requested additional information on the efficacy analysis for Clovis Oncology’s rociletinib.
 
The FDA has granted Ovid Therapeutics’ gaboxadol a Rare Pediatric Disease designation for the treatment of Angelman syndrome.
 
The FDA has designated Chi-Med/Lilly’s fruquintinib Fast Track status for the treatment of patients with metastatic colorectal cancer.
 
Announced Research Updates

​Brickell announced that in a 42-day, 281 patient, Phase III trial, 53.9% of patients treated with sofpironium improved their Hyperhidrosis Disease Severity Scale to two or less and had a 50% or more reduction in gravimetric sweat production compared to 36.4% that received placebo in Japanese patients with axillary hyperhidrosis.
 
Global Data announced that a pooled analysis of six Phase II trials found that two months after midomafetamine treatment, 56% of patients did not meet PTSS diagnostic criteria and after 12 months, 67% did not qualify for a PTSD diagnosis.
 
BeyondSpring announced interim data from 120 patients enrolled in the Phase III PROTECTIVE 2 trial, where treatment with plinabulin plus pegfilgrastim reduced grade 4 or severe neutropenia, compared to pegfilgrastim monotherapy, in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide.
 
Hua Medicine announced that in a 24-week, 463 patient, Phase III SEED trial, treatment with dorzagliatin 0.75 mg reduced HbA1c 1.15% compared to a 0.58% decrease with placebo, in Chinese patients with type 2 diabetes. In a 28-week, open-label extension of the SEED trial, patients treated with dorzagliatin continued on the drug and maintained a 1.11% HbA1c reduction, while patients switched from placebo achieved a 1.27% reduction.
 
Roche announced that in the 1,101 patient, Phase II, IPATential150 trial, ipatasertib, added to abiraterone and prednisone or prednisolone, improved radiographic progression-free survival compared to abiraterone plus prednisone/prednisolone in patients with metastatic castration-resistant prostate cancer (mCRPC) with PTEN loss, but not in all mCRPC patients regardless of PTEN status.
 
Provention Bio announced extended follow-up data showing that the time to develop type 1 diabetes after treatment with teplizumab increased to five years compared to two years with placebo.
 
Sanofi announced that in a 2.4 year, 34-patient follow-up to a Phase II trial, treatment with fitusiran lowered the annualized bleed rates to 0.84 in the non-inhibitor subgroup and to 0.44 in the inhibitor subgroup.
 
Published Research Updates
 
In the 12-week, 155 patient, Phase IIb ASTEROID 2 trial, 62.9% of patients treated with vilaprisan had a complete absence of bleeding or spotting compared to 55.4% with ulipristal and none with placebo in patients with uterine fibroids and heavy menstrual bleeding.
 
In an 8-week, 9 patient, open-label pilot study, treatment with ganaxolone decreased the Montgomery-Asberg Depression Rating Scale [MADRS] score from 24.4 to 12.8, and 44% achieved remission (MADRS < 10) in postmenopausal women with persistent depression despite adequate antidepressant therapy.