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Innovative Drug newsletter: bluebird bio Announces FDA Priority Review of the BLA for lovo-cel

Updated: Jul 11, 2023

R&D

1 Boston Pharmaceuticals Announces Positive Phase 2a Results from Monthly and Bi-weekly Dosing with Investigational BOS-580 NASH Program at EASL 2023

Boston Pharmaceuticals announced positive Phase 2a results for BOS-580, its investigational, proprietary, long-acting fibroblast growth factor 21 (FGF21) analog for the treatment of NASH. The study suggested promising results for monthly and bi-weekly doses of BOS-580, which achieved a statistically significant reduction in liver fat content, an exploratory endpoint in the trial of phenotypic NASH patients. Statistically significant reductions in additional exploratory endpoints, including biomarkers of liver injury and fibrosis, were also observed. In the trial, a low discontinuation rate due to treatment-emergent adverse events was observed. The most common adverse events in patients treated with BOS-580 were gastrointestinal in nature, mild to moderate, and transient.

2 bluebird bio Announces FDA Priority Review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events

bluebird bio announced that the FDA has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review. Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease ages 12 and older who have a history of vaso-occlusive events. It is specifically designed to treat the underlying cause of SCD through the addition of a functional gene that enables production of anti-sickling adult hemoglobin and is the most deeply studied gene therapy in development for this disease. The agency has set a PDUFA goal date of December 20, 2023.

3 Pieris Pharmaceuticals Announces AstraZeneca Discontinuation of Phase 2a Trial of Elarekibep (PRS-060/AZD1402) Due to New Non-Clinical Safety Findings From 13-week Toxicology Study

Pieris Pharmaceuticals announced that partner AstraZeneca communicated to Pieris its decision to discontinue and cease dosing in the ongoing clinical studies of elarekibep, an inhaled IL-4 receptor alpha inhibitor under development for the treatment of asthma. This decision was based on lung findings from a non-clinical 13-week GLP toxicology study with dry powder inhaler-formulated elarekibep, which are not a concern for the active clinical studies but do not support long-term use and progression to later-stage development. AstraZeneca's decision was made independent of any data from the Phase 2a study. Pieris will expedite a review of the implications of the data and AstraZeneca's decision on the program and will review its overall corporate priorities prior to sharing a further update.

4 PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington's Disease Patients

PTC Therapeutics shared interim data from the 12-week portion of the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease patients. The study demonstrated dose-dependent lowering of Huntingtin protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure in the cerebrospinal fluid was consistent with or higher than plasma unbound drug levels. PTC518 treatment was also demonstrated to be well tolerated, with no treatment-related serious adverse events, no reports of peripheral neuropathy or dose-limiting toxicities. Furthermore, there were no CSF neurofilament light chain protein treatment-related spikes, with an overall trend towards lowering of CSF NfL levels following 12 weeks of PTC518 treatment.

5 Teikoku Pharma granted FDA Fast Track Designation for TPU-006, a Novel 4-day Dexmedetomidine Transdermal System for Post Operative Pain Management

Teikoku Pharma announced that theFDA granted Fast Track Designation for TPU-006 in managing postoperative pain. TPU-006 is an investigational 4-day dexmedetomidine transdermal system, a novel drug delivery technology with a non-opioid active moiety for managing post-surgical pain. TPU-006's evaluation involves a randomized, double-blind Phase 2b clinical trial in patients undergoing abdominoplasty to examine this agent's efficacy and safety. The Company expects results from this study in the third quarter of 2023. These data will complement TPU-006's positive results from a randomized confirmatory Phase 2 study in bunionectomy.

6 UNION therapeutics announces positive topline results from the OSIRIS investigator-initiated proof-of-concept study of oral orismilast in patients with hidradenitis suppurativa

UNION therapeutics announces positive topline results from the OSIRIS investigator-initiated proof-of-concept study with oral orismilast in adult patients with HS. HS is a painful, scarring, inflammatory skin disease with few or limited treatment options. Study demonstrates clinically relevant improvements in patients with hidradenitis suppurativa (HS) receiving 16 weeks of treatment with oral orismilast. Orismilast is a high potency, next-generation PDE4 inhibitor with broad anti-inflammatory properties also in development for oral treatment of atopic dermatitis (AD) and psoriasis.

7 GSK shares positive data for AREXVY, its respiratory syncytial virus (RSV) older adult vaccine, indicating protection over two RSV seasons

GSK announced new data from the AReSVi-006 (Adult Respiratory Syncytial Virus) phase III trial evaluating the efficacy of a single dose of AREXVY (respiratory syncytial virus vaccine, adjuvanted) against lower respiratory tract disease (LRTD) caused by respiratory syncytial virus in adults aged 60 years and older over multiple RSV seasons and after annual revaccination. A similar pattern of vaccine efficacy over two seasons was also observed in adults with underlying comorbidities and in advancing age, reinforcing the impact the vaccine could have on those most at risk of the severe outcomes of RSV.

8 FDA Grants Full Approval for Blincyto (blinatumomab) to Treat Minimal Residual Disease-Positive B-Cell Precursor Acute Lymphoblastic Leukemia

Amgen announced that the FDA has approved the supplemental Biologics License Application (sBLA) for BLINCYTO® (blinatumomab) for the treatment of adults and pediatric patients with CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%, based on additional data from two Phase 3 studies that were submitted. The approval converts BLINCYTO's accelerated approval to a full approval.

9 Aldeyra Therapeutics Provides Regulatory Update on ADX-2191

Aldeyra announced receipt of a Complete Response Letter from the FDA for the 505(b)(2) NDA of ADX-2191 (methotrexate for injection, USP), an investigational drug candidate, for the treatment of primary vitreoretinal lymphoma (PVRL). Although no safety or manufacturing issues with ADX-2191 were identified, the FDA stated that there was a “lack of substantial evidence of effectiveness” due to “a lack of adequate and well-controlled investigations” in the literature-based NDA submission. Based on prior discussions with the FDA, Aldeyra did not conduct any clinical trials of ADX-2191 in PVRL.

10 uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

uniQure announced promising interim data, including up to 24 months of follow-up, from 26 patients enrolled in the ongoing U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease. Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in patients treated with the low-dose of AMT-130 and declining towards baseline at 12 months in patients treated with the high-dose of AMT-130 .

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