Update for the week ending July 20, 2019

Regulatory Update
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• The FDA approved Merck’s combination of imipenem, cilastatin, and relebactam (Recarbrio) on 7/17/2019 for the treatment of complicated urinary tract infections and complicated intra-abdominal infections.
• The FDA accepted the NDA for opicapone on 7/10/2019, setting a PDUFA date of 4/26/2020. Neurocrine Biosciences has licensed opicapone from Bial for development and commercialization in the U.S. and Canada, as adjunct to levodopa therapy in patients with Parkinson's Disease experiencing end-of-dose motor fluctuations.
• FDA Accepts Allergan’s NDA for Bimatoprost SR: The FDA accepted Allergan’s AGN NDA looking for approval of bimatoprost sustained-release (bimatoprost SR) for treatment of open-angle glaucoma or ocular hypertension. Bimatoprost SR, an investigational implant, has been developed as the first sustained-release drop-free treatment option for lowering intraocular pressure in patients with open-angle glaucoma or ocular hypertension. The FDA is expected to give its decision in the first half of 2020.
• FDA Grants Priority Review to Novartis’ Sickle Cell Pain Crisis Candidate: The FDA granted priority review designation to Novartis’ biologics license application (BLA) for its pipeline candidate crizanlizumab (SEG101). Novartis is looking for approval of crizanlizumab to prevent pain crises (also called vaso-occlusive crises, or VOCs) in patients with sickle cell disease. With the FDA granting a priority review designation to the BLA, a decision is expected in the next six months.
• Astellas and Seattle Genetics submitted a BLA for enfortumab vedotin on 7/16/2019. Enfortumab vedotin is an antibody-drug conjugate, where the vedotin is attached to the antibody enfortumab that attaches to cancer cells expressing Nectin-4. Astellas and Seattle Genetics have an ongoing Phase III trial evaluating enfortumab vedotin in the treatment of locally advanced or metastatic urothelial cancer.
• J&J Files for Approval of Subcutaneous Darzalex: J&J filed an application with the FDA looking for approval of a subcutaneous formulation of its multiple myeloma drug, Darzalex. The BLA was based on data from the phase III COLUMBA study. Data from the study presented in June showed non-inferiority of the subcutaneous formulation, both as a monotherapy and as a combination medicine, compared to the currently approved formulation of intravenous administration.
• Motif Bio announced it has submitted a meeting request and package to the FDA related to iclaprim, the company's lead product candidate for the treatment of acute bacterial skin and skin structure infections (ABSSI). The company received a Complete Response Letter from the FDA in February, indicating that an additional clinical trial would be necessary to grant marketing approval. Motif Bio is now seeking a Type B meeting with the FDA to discuss study population and design.
• Blueprint submitted an NDA for avapritinib in June 2019. The EMA accepted the MAA for avapritinib for the treatment of PDGFR alpha D842V-mutant gastrointestinal stromal tumors in July 2019. 
  

Announced Research Updates

The bio-pharmaceutical company Orphazyme A/S has reportedly announced that it has finished enrollment in its phase 3 study assessing arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS) before schedule. The full analysis remains on track for the first half of 2021.

J&J is testing their 6-dose, multi-antigen HIV vaccine in 2,600 sexually active women aged between 18 and 35 years in Southern Africa in the Phase IIb Imbokodo trial (results expected in 2021) and in the Phase III Mosaico trial, involving 3,800 men who have sex with men and transgender people aged between 18 and 60 years in the U.S., South America and Europe. The two trials use vaccines composed of different antigens to reflect the predominant strains in the region.

Vanda announced that in the 126 patient, Phase II, Motion Sifnos trial, treatment with tradipitant decreased the percentage of participants vomiting (17.5% vs 39.7%) but did not improve the mean Motion Sickness Severity Scale (MSSS) worst score among participants on one of seven sailing voyages on the Pacific. Both a reduction in emesis and the MSSS was seen in rough seas, but no significant improvement was seen in calm conditions.

Published Research Updates

In a 52-week, 342 patient, Phase III, open-label trial, treatment with trifarotene cream achieved an IGA of clear/almost clear and ≥2 grade improvement in 65.1% of patients and a physician's global assessment in 66.9% of patients with moderate facial acne and moderate truncal acne.

In a 59 patient, Phase II trial, levosimendan did not improve sitting slow vital capacity in patients with amyotrophic lateral sclerosis (ALS).

In the 65-patient, Phase II trial, neither tremelimumab plus durvalumab nor durvalumab monotherapy appreciably improved the objective response rate (3% vs 0%) in patients with metastatic pancreatic ductal adenocarcinoma.

In a 32 patient, Phase III trial, 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 in patients with heart failure and renal impairment. In a 72-patient trial, levosimendan did not reduce the occurrence rate of acute kidney injury compared to milrinone in infants after open-heart surgery with cardiopulmonary bypass.

Glaxo’s Zejula Meets Main Goal in Late-Stage Study: Glaxo’s GSK phase III PRIMA study, evaluating Zejula (niraparib) for patients with ovarian cancer in first-line maintenance setting, met the primary endpoint. Data from the study showed that treatment with its PARP inhibitor, Zejula,` led to a statistically significant improvement in progression-free survival in women regardless of their biomarker status, thereby meeting the study’s primary endpoint. Zejula was added to Glaxo’s portfolio following the acquisition of Tesaro earlier this year.