Stock Monitor: Oramed Pharma Post Earnings Reporting
LONDON, UK / ACCESSWIRE / January 18, 2018 / Active-Investors.com has just released a free research report on AveXis, Inc. (NASDAQ: AVXS). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=AVXS as the Company's latest news hit the wire. On January 16, 2018, the clinical-stage gene therapy company that provides treatments for patients suffering from rare and life-threatening neurological genetic diseases, shared details about the expanded clinical development program for AVXS-101, which is its initial gene therapy candidate for the treatment of spinal muscular atrophy (SMA). Register today and get access to over 1000 Free Research Reports by joining our site below:
Active-Investors.com is currently working on the research report for Oramed Pharmaceuticals Inc. (NASDAQ: ORMP), which also belongs to the Healthcare sector as the Company AveXis. Do not miss out and become a member today for free to access this upcoming report at:
Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, AveXis most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:
AveXis already has two ongoing studies for SMA, i.e. pivotal trial in SMA Type-1 (STR1VE) and Phase-1 trial in SMA Type-2 (STRONG). Apart from that, the Company intends to expand the study of AVXS-101 into additional SMA populations such as pre-symptomatic, older pediatric Type-2 and Type-3 SMA patients. AveXis also declared that the first patient has been dosed in the Phase-1 trial of AVXS-101 in SMA Type-2.
SMA, the Leading Cause of Infant Mortality
Spinal muscular atrophy is a severe neuromuscular disease, in which the loss of motor neurons leads to progressive muscle weakness and paralysis. It is caused by a genetic defect in the SMN1 gene, which codes SMN, i.e. the protein necessary for survival of motor neurons. At present, SMA is the leading cause of infant mortality - its incidence is nearly one in 10,000 live births.
Type-1 SMA is the most serious form of SMA, characterized by motor neuron loss and related muscle deterioration, which often leads to mortality or the need for permanent ventilation support before the age of two for more than 90% patients. On the other hand, SMA Type-2 generally occurs between six to 18 months of age. Majority of the SMA Type-2 patients cannot walk without support and some cannot even stand without support. This form of SMA results in mortality in over 30% of patients by the age of 25.
How AveXis Treats SMA?
AVXS-101 is AveXis' proprietary gene therapy candidate for one-time treatment of SMA Type-1 and -2. AVXS-101 focuses on the monogenic root cause of SMA and prevents further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. Besides, AVXS-101 is also designed to target motor neurons, which causes rapid onset of effect and crosses the blood brain barrier to allow targeting of both central and systemic features.
Overview of the Ongoing Clinical Trials of AVXS-101
- STR1VE, the Pivotal Trial of AVXS-101 in SMA Type-1: STR1VE is an ongoing, open-label, single-arm, single dose, multi-center trial, which is designed to assess the efficacy and safety of a one-time IV infusion of AVXS-101 in patients with SMA Type-1. For this trial, AveXis aims to enroll at least 15 patients with SMA Type-1 who are less than six months of age at the time of gene therapy, and who have one or two copies of the SMN2 backup gene, as determined by genetic testing and bi-allelic SMN1 gene deletion or point mutations. So far, three patients have been dosed for the trial.
- STRONG, Phase-1 Trial of AVXS-101 in SMA Type-2: STRONG is an on-going, open-label, dose-comparison, multi-center Phase-1 trial, which has been specifically designed to gauge the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two different age groups of patients with SMA Type-2. This Trial is based on the one-time intrathecal (IT) route of administration. For this trial, AveXis aims to enroll 27 infants and children who are symptomatic with a genetic diagnosis consistent with SMA, including the bi-allelic deletion of SMN1 and three copies of SMN2 without the SMN2 genetic modifier, who are able to sit but are not able to stand or walk. In this trial, only one person has been dosed up till now.
Overview of the Three Proposed Trials in SMA
- STR1VE EU, Pivotal Trial of AVXS-101 in SMA Type 1 in Europe: STR1VE EU has been specifically designed to assess the safety and efficacy of a one-time IV dose of AVXS-101. It is meant to evaluate achievement of motor milestones, patients' ability to sit unassisted, and an efficacy measure defined by the time from birth to an event, wherein event means death or a situation requiring at least 16 hours per day of ventilation support for breathing for greater than two weeks in the absence of an acute reversible illness, or preoperatively. For this trial, AveXis aims to enroll approximately 30 patients with SMA Type-1 who are less than six months of age at the time of gene therapy.
This trial is based on single-arm design, using natural history of the disease as a comparator. Besides, the Company has also incorporated scientific advice from the European Medicines Agency into the protocol design and expects to start the trial in the first half of 2018.
- SPRINT, for Pre-Symptomatic SMA Types 1, 2, 3: The multi-national trial SPRINT is meant to assess appropriate clinical endpoints, such as developmental milestones, survival, bulbar function, and safety, of a one-time IV infusion of AVXS-101. For this trial, AveXis aims to enroll around 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy. The Company intends to start this trial in the first half of 2018.
- REACH for Pediatric "All Comers" with SMA Types 1, 2, 3: The multi-national trial REACH is designed to evaluate a one-time IT dose of AVXS-101. For this trial, AveXis aims to enroll around 50 patients between six months and 18 years of age who do not qualify for other AVXS-101 trials at the time of gene therapy. The Company aims to commence this trial in end of 2018 or beginning of 2019.
Stock Performance Snapshot
January 17, 2018 - At Wednesday's closing bell, AveXis' stock slightly declined 0.13%, ending the trading session at $104.87.
Volume traded for the day: 2.91 million shares, which was above the 3-month average volume of 382.66 thousand shares.
Stock performance in the last month - up 5.99%; previous three-month period - up 2.80%; last six-month period - up 27.02%; past twelve-month period - up 99.71%.
After yesterday's close, AveXis' market cap was at $3.44 billion.
The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.9% at the end of the session.
Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles, and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.
A-I has not been compensated; directly or indirectly; for producing or publishing this document.
PRESS RELEASE PROCEDURES:
The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst [for further information on analyst credentials, please email firstname.lastname@example.org. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable-effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.
A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.
NOT AN OFFERING
This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.
For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:
Phone number: 73 29 92 6381
Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia
CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.