Weekly Update for the Week Ending December 21, 2019

Regulatory Update
 
The FDA approved trastuzumab deruxtecan (Enhertu, AstraZeneca/Daiichi Sankyo) on 12/19/19, for the treatment of unresectable or metastatic HER2-positive breast cancer that has received two or more prior anti-HER2-based regimens in the metastatic setting.
 
The FDA approved Merck’s Ebola virus vaccine (Ervebo) on 12/20/19, for the prevention of Ebola virus disease caused by the Zaire ebola virus in people at least 18 years old.
 
The FDA approved enfortumab vedotin (Padcev, Astellas/Seattle Genetics) on 12/18/19 for the treatment of locally advanced or metastatic urothelial cancer that has progressed on chemotherapy and immunotherapy.
 
The FDA rejected ViiV Healthcare/Janssen’s NDA for the combination of cabotegravir and rilpivirine for HIV treatment, due to manufacturing issues.
 
The FDA’s review of tazemetostat found a lack of efficacy compared to standard therapies and the potential for secondary malignancies that short trial durations may not have fully described. But the FDA’s Oncologic Drugs Advisory Committee voted 11-0 with two abstentions to recommend approval of tazemetostat for the treatment of metastatic or inoperable locally advanced epithelioid sarcoma, due to the low response rate with currently approved treatments.
 
Deciphera filed an NDA for ripretinib for the treatment of gastrointestinal stromal tumors.
 
The FDA designated Orphazyme’s arimoclomol a Fast Track drug for the treatment of sporadic Inclusion Body Myositis.
 
PharmaMar submitted an NDA for lurbinectedin for the treatment of small cell lung cancer. 
 
The FDA designated Cascadian Therapeutics’ tucatinib a Breakthrough Therapy when given in combination with trastuzumab and capecitabine, for the treatment of patients with locally advanced unresectable or metastatic HER2-positive breast cancer.
 
BMS filed a BLA for lisocabtagene maraleucel for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least two prior therapies.
 
MacroGenics filed a BLA for margetuximab for the treatment of patients with metastatic HER2-positive breast cancer in combination with chemotherapy.
 
Gilead submitted an NDA for filgotinib for the treatment of moderate-to-severe rheumatoid arthritis.
 
Announced Research Updates

1. Gilead announced that in the 48-week, 392 patient, Phase II ATLAS study, liver fibrosis was not improved by 1 stage or more without disease worsening compared to placebo in patients treated with selonsertib, cilofexor or firsocostat alone or in two-drug combinations in patients with advanced fibrosis (F3-F4) due to NASH.
   
2. BioMarin announced that in a 52-week, 121 patient, Phase III trial, treatment with vosoritide resulted in an increase of 1.6 cm/year in annualized growth velocity compared to placebo in children (ages 5 to 14) with achondroplasia.
   
3. Novartis announced that in two 52-week, Phase III trials (LUSTER 1 and LUSTER 2) that enrolled a total of 1,771 patients, treatment with fevipiprant did not reduce moderate -to-severe exacerbation compared to placebo in patients with inadequately controlled moderate-to-severe asthma. Novartis has stopped development of fevipiprant for the treatment of asthma.
   
4. Proxel announced that in the 52-week, 714 patient, Phase III, open-label, TIMES 2 trial, imeglimin reduced HbA1c 0.12 to 0.92 when added to non-insulin anti-diabetic drugs in Japanese patients with type 2 diabetes.
   
5. Lexicon announced that in the 26-week, 700 patient, Phase III SOTA-EMPA trial, treatment with sotagliflozin was superior to placebo and non-inferior to empagliflozin in reducing HbA1c in patients with type 2 diabetes and inadequate glycemic control on a DPP4 inhibitor with or without metformin.
   

Published Research Updates

1. In a 1,140 patient, Phase III trial, veliparib added to carboplatin and paclitaxel followed by veliparibmaintenance therapy improved progression-free survival (PFS) compared to carboplatin plus paclitaxel induction therapy alone (23.5 months vs 17.3 months) in patients with previously untreated stage III or IV high-grade serous ovarian carcinoma. In patients with a BRCA-mutation the difference in PFS was 34.7 months with veliparib compared to 22 months with placebo.
   
2. In a 73-patient, Phase I, open-label trial, treatment with belantamab mafodotin resulted in a 33% overall response rate in patients with multiple myeloma. In the 196-patient, Phase II, open-label, DREAMM-2 trial, treatment with belantamab mafodotin resulted in a 48% overall response rate in patients with multiple myeloma.
   
3. A 52-week, 701 patient, Phase III trial was terminated early, when treatment with mongersen did not improve the clinical remission rate compared to placebo at 12-weeks in patients with Crohn's disease.