Junlin Healthcare New Drug Bulletin Issue 9, 2020: Highlights and Pipeline Updates

This week, there were 9 new drug data entries, including 3 for oncology, 2 for metabolic diseases, 1 for antiviral, 1 for CNS, 1 for respiratory system, and 1 for ophthalmology.

Engage Therapeutics Announces That Its Orally Inhaled Therapy, Staccato Alprazolam, Met the Primary Endpoint in the Phase 2b StATES Clinical Trial for the Treatment of Patients with Epilepsy

Staccato Alprazolam is a compact, handheld, orally inhaled emergency treatment for seizures that combines the FDA-approved Staccato delivery technology with the marketed drug alprazolam.

The StATES trial is a randomized, double-blind, Phase 2b clinical study involving 116 patients.

The results showed that 50 out of 76 patients achieved the primary endpoint, defined as cessation of seizures within two minutes of treatment administration and no recurrence within two hours.

Boehringer Ingelheim Announces FDA Grants Fast Track Designation to Its SGLT2 Inhibitor Empagliflozin (Brand Name: Jardiance) to Reduce the Risk of Kidney Disease Progression and Cardiovascular Death in Adult Patients with Chronic Kidney Disease

By inhibitingThe transporter SGLT2 in the glomerulus; empagliflozin can reduce glucose reabsorption in the kidneys, causing it to be excreted in urine, thereby lowering bloodGlucose levels in

In the EMPA-KIDNEY trial, a total of 6,000 patients with chronic kidney disease were enrolled, regardless of whether they also had diabetes.

Previous results indicated that empagliflozin reduced the risk of onset and progression of kidney disease and cardiovascular disease by 39% in adult patients with type 2 diabetes; further results from the EMPA-KIDNEY trial are pending publication.

Data Source: BI

BMS Announces FDA Approval of Opdivo in Combination with Yervoy for the Treatment of HCC Patients Previously Treated with Sorafenib; Opdivo + Yervoy Is the First and Only Dual Immunotherapy Approved by the FDA for This Patient Population

Opdivo (nivolumab) and Yervoy (ipilimumab) are immune checkpoint inhibitors developed by Bristol Myers Squibb (BMS) that enhance T cell-mediated anti-tumor immune responses by inhibiting the PD-1 and CTLA-4 immune checkpoint proteins, respectively.

In the CheckMate-040 phase 1/2 clinical study, patients with hepatocellular carcinoma (HCC) who had previously received sorafenib treatment were followed up for at least 28 months.

33% (16/49; 95% CI: 20–48) of patients achieved a response; 8% (4/49) achieved a complete response (CR). The duration of response (DOR) ranged from 4.6 to 30.5+ months, with 88% lasting at least 6 months, 56% at least 12 months, and 31% at least 24 months.

Janssen Announces FDA Breakthrough Therapy Designation for Bispecific Therapy JNJ-6372 in Patients with Metastatic Non-Small Cell Lung Cancer (NSCLC) Harboring EGFR Exon 20 Insertion Mutations

JNJ-6372 is a bispecific antibody targeting EGFR and MET. It not only blocks the binding of ligands to EGFR and MET, promoting receptor degradation, but also triggers antibody-dependent cellular cytotoxicity.

In an open-label, multicenter Phase 1 clinical study, investigators will evaluate JNJ-6372 as monotherapy and in combination with lazertinib for the treatment of advanced non-small cell lung cancer (NSCLC) in adults; lazertinib is an innovative third-generation EGFR tyrosine kinase inhibitor (TKI).

Results to be disclosed later

ViiV Healthcare announced that its long-acting, innovative two-drug combination therapy for HIV met the primary endpoint in the Phase 3 ATLAS-2M clinical trial. This regimen consists of cabotegravir, developed by ViiV Healthcare, and rilpivirine, developed by Janssen.

Rilpivirine is an oral non-nucleoside reverse transcriptase inhibitor (NNRTI) already approved in the United States and the European Union, while cabotegravir is an integrase inhibitor (INI) under development.

In 1,045 patients infected with HIV-1, the antiviral activity and safety of a combination regimen consisting of long-acting cabotegravir and rilpivirine administered every 8 weeks were evaluated in comparison to administration every 4 weeks.

Trial results demonstrated that, in adults with HIV-1 infection, the dual-drug combination regimen administered every 8 weeks met the non-inferiority criterion for viral suppression compared with the every-4-week dosing regimen after 48 weeks of treatment.

(ILDs) Patients

Boehringer Ingelheim Announces U.S. FDA Approval of Ofev (nintedanib) for Patients with Chronic Progressive Fibrosing Interstitial Lung Diseases (ILDs), Marking the First FDA-Approved Therapy for Progressive Chronic Fibrosing ILDs

Ofev is a small-molecule tyrosine kinase inhibitor that simultaneously blocks three growth factor receptors, including vascular endothelial growth factor receptor, platelet-derived growth factor receptor, and fibroblast growth factor receptor, effectively inhibiting signaling pathways involved in the fibrotic process.

In the Phase III INBUILD clinical trial, investigators assessed the safety and tolerability of Ofev.

At Week 52, assessment of forced vital capacity (FVC) demonstrated that Ofev reduced the decline in lung function by 57%, thereby achieving the primary endpoint of the trial.

Kala Pharmaceuticals Announces That Eysuvis (KPI-121) Met Primary and Secondary Endpoints in the Phase 3 STRIDE 3 Clinical Trial for the Treatment of Patients with Dry Eye Disease (DED)

Eysuvis is a novel loteprednol etabonate nanoparticle formulation that leverages Kala Pharmaceuticals’ proprietary Mucus-Penetrating Particles (MPP) technology to target and enhance penetration into ocular tissues.

In the Phase 3 clinical trial, 901 patients with dry eye disease were enrolled.

Eysuvis Met Both Primary Efficacy Endpoints. After 15 Days of Treatment, Patients in the Intent-to-Treat Population and Those with Severe Ocular Discomfort Achieved Statistically Significant Improvements in Ocular Discomfort Severity (ODS).

(Cushing’s disease) patients

The U.S. FDA Announces Approval of Novartis’ Isturisa (osilodrostat) Oral Tablets for the Treatment of Adult Patients with Cushing’s Disease Who Are Unable to Undergo Pituitary Surgery or Who Have Persistent Disease Following Pituitary Surgery

Isturisa is the first FDA-approved therapy that directly addresses cortisol overproduction by blocking 11β-hydroxylase, thereby inhibiting cortisol synthesis.

In a study of 137 adult patients, the mean age was 41 years. The majority had persistent disease after pituitary surgery or were not candidates for surgery. Patients initially underwent 24 weeks of treatment, followed by an additional 8 weeks of therapy in a subset of patients.

After 24 weeks of treatment, cortisol levels decreased to within the normal range in nearly half of the patients. During the subsequent 8 weeks, 86% of patients receiving Isturisa maintenance therapy maintained cortisol levels within the normal range, compared with 30% in the placebo group.

Treatment Options for Cushing's Disease

Data source: Novartis

MorphoSys Announces U.S. FDA Acceptance of BLA for Tafasitamab Combination Therapy in Patients with Relapsed/Refractory DLBCL

MorphoSys Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Tafasitamab in Combination with Lenalidomide for the Treatment of Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

Tafasitamab is a humanized monoclonal antibody targeting the CD19 antigen.

In the Phase 2 L-MIND clinical trial, 80 patients with relapsed/refractory DLBCL who were ineligible for intensive chemotherapy or stem cell transplantation received combination therapy consisting of tafasitamab and lenalidomide.

Patients receiving treatment achieved an objective response rate (ORR) of 60% and a complete response rate (CR) of 43%. At a median follow-up of 17.3 months, the median progression-free survival (PFS) was 12.1 months.