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Tuesday, August 4, 2020

Regenxbio announces positive one-year data on wet AMD gene therapy

REGENXBIO (NASDAQ:RGNX) announces positive one-year data from cohorts 4 & 5 of its Phase 1/2a clinical trial evaluating gene therapy RGX-314 in patients with wet age-related macular degeneration (wet AMD).
The treatment effect was stable with a mean +4 letter change in best corrected visual acuity (BCVA) and -2 letters from baseline. Mean changes in retinal thickness of -61 µm and -79 µm, respectively, were observed.
Anti-VEGF treatment burden dropped by 61% and 85%, respectively.
On the safety front, 18 serious adverse events were reported, one considered possibly related to RGX-314 (decrease in vision at month 11).
A pivotal study in wet AMD should launch by year-end.
Another Phase 2 assessing suprachoroidal delivery of RGX-314 in wet AMD patients will commence this quarter with interim data from the first cohort expected by year-end.
Management will host a conference call this morning at 8:30 am ET to discuss the results.


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FDA OKs Emergency Use of Impella Heart Pumps in Therapy to COVID-19 Patients August 4, 2020

The United States Food and Drug Administration (FDA) has issued an emergency use authorization (EUA) for left-sided Impella heart pumps to provide left ventricular unloading and support to COVID-19 patients who are undergoing ECMO treatment and develop pulmonary edema or myocarditis. Impella is manufactured by Abiomed (NASDAQ: ABMD).
COVID-19 causes widespread inflammation which can result in damage to the lungs and heart. This damage may cause severe left ventricular dysfunction manifesting as pulmonary edema and/or myocarditis. Impella combined with ECMO therapy (known as ECpella™) has become an important tool for physicians treating COVID-19 patients suffering from both heart and lung failure.
Impella is the world’s smallest heart pump. It first received FDA clearance in 2008 and FDA PMA approval as safe and effective in 2015. During the last 10 years, Impella has provided left ventricular unloading to approximately 10,000 ECMO patients in cardiogenic shock. The FDA’s EUA expands the use of Impella as an ECMO unloading therapy to include COVID-19 patients with pulmonary edema or myocarditis.
“The early clinical evidence continues to accrue in favor of including left ventricular unloading in many patients on ECMO,” said Christian Bermudez, MD, surgical director, lung transplantation and ECMO, and professor of surgery at the University of Pennsylvania. “The superior clinical outcomes from the combination of Impella and ECMO are likely the result of reducing the stress on the myocardium and work required of the left ventricle, also reducing secondary lung injury due to pulmonary congestion.”

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Tiziana to Expedite Anti-Interleukin-6-Receptor to Treat COVID-19 Patients

Recently signed agreements with four contract research organizations (“CROs”) to initiate GMP manufacturing, develop inhalation technology, conduct inhalation safety toxicity study in monkeys, and initiate a human clinical trial in COVID-19 patients.
Tiziana Life Sciences plc (Nasdaq: TLSA / AIM: TILS) (“Tiziana” or the “Company”), a biotechnology company focused on innovative therapeutics for oncology, inflammation and infectious diseases, today announced that it has signed agreements with four CROs to expedite clinical development of TZLS-501, a novel fully human anti-interleukin-6 receptor (anti-IL6R) monoclonal antibody (mAb) for the treatment of COVID-19 (SARS-CoV-2) patients. The Company intends to initiate the clinical study in Q1 2021 and will work with the following CROs:
  • FHI Clinical: A subsidiary of FHI 360, FHI Clinical is a multinational CRO specializing in clinical development of drugs for infectious diseases (https://www.fhiclinical.com). This company has conducted several recent trials in COVID-19 patients and has a large network of clinical sites throughout the US and abroad to expedite clinical trials with COVID-19 patients. In this trial, TZLS-501 will be delivered as an aerosol directly to the lungs using a hand-held nebulizer.
  • STC Biologics: STC Biologics, Inc. is a boutique Good Manufacturing Practice (GMP) CRO that provides full chemistry, manufacturing and control (CMC) services to enable its partners to advance their biologic products from discovery to commercial approval. GMP manufacturing of TZLS-501 is ongoing.
  • Sciarra Labs: Sciarra Laboratories, Inc., is an FDA-approved, current GMP manufacturer of drug solutions used in nebulizers, inhalers, metered dose inhalers (“MDI”) and nasal sprays. Tiziana worked with Sciarra Labs to establish clinical supply of nasal sprays of Foralumab used in the recently completed Phase 1. Sciarra Labs will be developing and testing a hand-held nebulizer and GMP manufacturing the anti-IL6R mAb solution to be used as clinical trial material for the clinical study in COVID-19 patients.
  • ITR Laboratories Canada: ITR has been in operation for the last 30 years and is known for its reputation in toxicology testing and other specialized testing services for biotechnology and pharmaceutical industries in North America, Europe and beyond. ITR is a Canadian Council on Animal Care (CCAC) and American Association for Accreditation of Laboratory Animal Care (AAALAC) CRO and is a fully compliant Good Laboratory Practices (GLP) CRO. ITR will be conducting safety and toxicity studies with TZLS-501 delivered by a nebulizer directly into the lungs of cynomolgus monkeys.
Tiziana holds a worldwide exclusive license for TZLS-501 (a.k.a NI-1201) from Bristol Myers Squibb™. TZLS-501 is a novel fully human mAb that binds to both the membrane-bound and soluble forms of IL-6R and rapidly depletes circulating levels of IL-6 in the blood (1). Excessive production of IL-6 is regarded as a key driver of cytokine release syndrome (CRS) and chronic inflammation in the lungs of patients with COVID-19 and acute respiratory illness such as Acute Respiratory Distress Syndrome (ARDS). Tiziana’s novel and proprietary approach to treatment is to deliver TZLS-501 directly to the lung via inhalation.

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DBV Technologies down big on FDA rejection of Viaskin Peanut application

DBV Technologies (NASDAQ:DBVT) slumps 30% premarket in reaction to its announcement that it received a Complete Response Letter (CRL) from the FDA regarding its BLA for investigational Viaskin Peanut (DBV712), a non-invasive, once-daily epicutaneous patch to treat peanut allergies in children ages 4 to 11 years.
The CRL cites that the FDA cannot approve the application in its present form. The Agency has identified concerns regarding the impact of patch-site adhesion on efficacy and indicated the need for patch modifications, and subsequently a new human factor study.
The Company was also notified that supplementary clinical data would be needed to support the modified patch and a request for additional Chemistry, Manufacturing and Controls data was made. The Agency did not raise any safety concerns related to Viaskin Peanut.

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Zimmer Biomet EPS beats by $0.74, beats on revenue

Zimmer Biomet (NYSE:ZBH): Q2 Non-GAAP EPS of $0.05 beats by $0.74; GAAP EPS of -$1.00 beats by $0.79.
Revenue of $1.23B (-38.2% Y/Y) beats by $307.84M.
Press Release

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Mallinckrodt EPS beats by $0.55, beats on revenue August 4, 2020

Mallinckrodt (NYSE:MNK): Q2 Non-GAAP EPS of $1.89 beats by $0.55; GAAP EPS of -$11.04 misses by $10.89.
Revenue of $700.9M (-14.9% Y/Y) beats by $70.71M.
Specialty brands revenue of $522.8M vs. consensus of $444.3M.
Shares +2.21% PM.
Press Release

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Amarin EPS beats by $0.11, misses on revenue

Amarin (NASDAQ:AMRN): Q2 Non-GAAP EPS of $0.04 beats by $0.11; GAAP EPS of $0.01 beats by $0.09.
Revenue of $135.32M (+34.3% Y/Y) misses by $14.16M.
Normalized prescriptions for VASCEPA increased by ~44% in Q2 compared to 47% in Q2 2019, based on data from Symphony Health and IQVIA, respectively.
Press Release

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