Tracker

Additional US Patent allowed for Seelos’ Trehalose (SLS-005)

The allowed claims cover a method of using trehalose (SLS-005) to treat several neurodegenerative conditions including spinocerebellar ataxia (SCA), spinal and bulbar muscular atrophy (SBMA), dentatomral-pailidoluyssan atrophy (DRPLA), Pick’s disease, corticobasaldegeneration (CBD), progressive supranuclear palsy (PSP), and frontotemporal dementia. 

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

Precision Bio receives Fast Track Designation from FDA for PBCAR269A in r/rMM

PBCAR269A is being evaluated in a Phase 1/2a multi-center, non-randomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR269A in adults with relapsed/refractory multiple myeloma. The starting dose of PBCAR269A is 6 x 105 CAR T cells/kg body weight with subsequent cohorts treated with escalating doses to a maximum dose of 6 x 106 CAR T cells/kg body weight. The trial is being conducted at multiple U.S. sites with clinical trial material generated in-house at the Manufacturing Center for Advanced Therapeutics (MCAT) in Durham, North Carolina. 

Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then utilizes its unique ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured that is designed to prevent graft-versus-host disease. Precision optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naive and central memory CAR T cells throughout the manufacturing process and in the final product. 

TherapeuticsMD gets another patent for ANNOVERA female contraceptive

In addition, based on recent communications from the USPTO, a third ANNOVERA patent (U.S. Patent No. 10,780,047) is scheduled to be issued on 9/22/2020. These Orange Book eligible patents, which provide patent protection through 2039, cover ANNOVERA and its use for the labeled indication of preventing pregnancy in females of reproductive potential.

Before the issuance of the first Orange Book listable patent for ANNOVERA, the product was protected by FDA regulatory exclusivity through 2023 as a new chemical entity. These additional patents, which cover different aspects of ANNOVERA, provide independent layers of patent protection.

ANNOVERA is a critical component of the company’s drive towards EBITDA breakeven in 2021.

BeyondSpring’s plinabulin gets Breakthrough Therapy Designation from FDA in Chemotherapy-Induced Neutropenia

The Company expects to report the full PROTECTIVE-2 Phase 3 topline data in 4Q-2020 and file an NDA with the FDA by the end of 2020. The Company has already submitted an NDA for Plinabulin for the CIN indication to the NMPA on a rolling basis in Q1 2020. 

These results were further strengthened by the Company’s other CIN studies that have confirmed Plinabulin’s early onset action in Week 1 with protecting neutrophils in various cancer types and various chemotherapies, which is complementary to Week 2 neutrophil protection with G-CSFs.

FDA approves Blueprint’s pralsetinib in Metastatic RET Fusion-Positive NSCLC

GAVRETO is a once-daily oral RET-targeted therapy developed by Blueprint Medicines. It is designed to selectively and potently inhibit RET alterations that drive many cancer types, including approximately 1 to 2 percent of patients with NSCLC. Currently, RET is one of seven NSCLC biomarkers that can be targeted with an FDA-approved therapy.

GAVRETO was granted accelerated approval by the FDA, and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. In 87 patients previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 57 percent (95% CI: 46%, 68%) with a 5.7 percent complete response (CR) rate, and the median duration of response (DOR) was not estimable (95% CI: 15.2 months, not estimable). In 27 treatment-naïve patients who were ineligible for platinum-based chemotherapy per the study protocol, the ORR was 70 percent (95% CI: 50%, 86%) with an 11 percent CR rate, and the median DOR was 9.0 months (95% CI: 6.3 months, not estimable). GAVRETO has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk of impaired wound healing and risk of embryo-fetal toxicity.

GAVRETO is the second breakthrough therapy discovered by Blueprint Medicines that has received FDA approval in 2020, less than 10 years since the company started operations.

Blueprint Medicines and Genentech plan to make GAVRETO available in the U.S. within one week. GAVRETO will be available in a 100 mg dose strength, and the recommended starting dose is 400 mg once daily.