President Trump, the First Lady and COVID-19
Indeed, the complicated, nasty SARS-CoV-2 virus managed to infect President Trump and the First Lady when we were expecting a vaccine to soon be ready to protect the country and the world from COVID-19.
The president’s physicians did not treat the him with any of the investigational vaccines in late-phase trials; vaccines being developed by: Moderna (MRNA), Novavax (NVAX) and BioNTech (BNTX)/Pfizer (PFE) but, instead, decided to use an investigational monoclonal antibody, made by Regeneron (REGN), and Remdesivir, made by Gilead Sciences (GILD), which the FDA has granted emergency approval.
In addition to these treatments, the team of specialists at the Walter Reed Medical Center added Dexamethasone, Vitamin D, Vitamin C, Zinc and generic Pepcid.
What conclusions do we have after hearing this scenario?
All we can conclude is that Regeneron’s monoclonal antibody might soon be granted FDA emergency approval.
Because the top medical specialists in a top American hospital have chosen to use the Regeneron monoclonal antibody as the first treatment for the President of the United States. To make this decision they must have great confidence that the antibody is both safe and effective.
Nothing about the other drugs included in the treatment for the President would insinuate that his condition is more severe than what the team of doctors has been saying.
Some critics might take advantage to express their negative opinions of the vaccines, as they have not been used in treating President Trump.
The President stated, in a video posted on Twitter late yesterday, that he received great reports from the treating physicians. The President also took a short, last-minute motorcade ride to wave to his supporters who were standing around the hospital.
Regeneron is now trading at $605.08 UP $40.28.
As you might have observed, the stock has easily crossed the $600 barrier again, following unwarranted stock selloff by the bearish and the deceived.
Gilead is now trading at $63.60 UP $1.43, following a long-term suppression of the stock also by the bearish and the deceived.
Moderna is now trading at $71.95 UP $3.14.
Novavax is now trading at $110.17 UP $6.57.
BioNTech is now trading at $80.70 UP $7.00.
Regenxbio RGX-121 for Hunter Syndrome
So, what about the firms we selected that have nothing to do with COVID-19 and politics?
We start with Regenxbio (RGNX); the firm that managed to improve on the adeno-associated viral vectors that made it possible for the introduction of gene therapy into the worlds’ clinics.
The latest news from Regenxbio announced that the firm has expanded its RGX-121 program for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. The expansion is expected to enable additional insight into the neurodegenerative manifestations of the disease and evaluate the product RGX-121 in a broader patient population.
Regenxbio is a leading clinical-stage biotechnology company seeking to improve the lives of human beings through the curative potential of gene therapy. The Company’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. RegenxBio and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
The firm has 6 products in clinical trials and 2 products in research. Most have encouraging results.
From the Experts
Steve Pakola, M.D., Chief Medical Officer of Regenxbi,o said, “Today’s update reflects significant forward progress in our clinical program for the treatment of MPS II, as we expand the program to gain additional insight into the potential treatment effects of RGX-121 in more patients. Regenxbio is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients. In addition, we are announcing the initiation of an important prospective natural history study to provide critical data about the neurocognitive development of young pediatric patients with MPS II. We plan to share the data from this observational study with the patient community and look forward to working closely with researchers and advocates in order to enhance awareness of this study.”
Terri Klein, President and Chief Executive Officer of the National MPS Society said, “MPS II is a serious and debilitating lysosomal disease that affects 1 in 100,000 children, and available treatments are inadequate to treat the neurodegenerative manifestations of the disease. Initiating a natural history study will increase the understanding of neurocognitive effects and key biomarkers of severe MPS II, and is critical to advancing the development of new treatment options. We are grateful for Regenxbio’s dedication to MPS and commitment to share the learnings from this observational study with the community.”
Regenxbio RGX-121 Trials
An ongoing Phase I/II study is currently evaluating a single intracisternal administration of RGX-121 in severe MPS II patients under the age of five, in children. Six patients have been dosed across two dose levels. The study is evaluating the safety and tolerability of RGX-121, as well as the effects of RGX-121 on biomarkers of I2S enzyme activity, neurocognitive development, and other clinical measures.
As of September 16, 2020, RGX-121 is reported to be well-tolerated in all six patients with no drug-related serious adverse events (SAEs).
Regenxbio plans to immediately expand enrollment of patients in Cohort 2 based on support from MPS II treating physicians and the Independent Data Monitoring Committee. Up to six additional patients will be dosed with RGX-121 at the second dose level, 6.5×1010 genome copies per gram (GC/g) of brain mass. Regenxbio anticipates further updates from this trial by the end of 2020.
In addition, the firm announced that the U.S. FDA has cleared a new Investigational New Drug application (IND) and plans to initiate a second Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with severe MPS II ages 5-18 years old.
Up to six patients will be enrolled, and RGX-121 will be administered at a dose level of 6.5×1010 GC/g of brain mass will be delivered directly to the cerebrospinal fluid (CSF) through intracisternal or intracerebral oventricular injection.
The trial is designed to evaluate the following:
- The safety of a single administration of RGX-121.
- The effects of RGX-121 on biomarkers of I2S enzyme activity and
- The changes in cognitive function, adaptive behavior, daily function and quality of life.
A new observational natural history trial designed to provide detailed characterization of neurocognitive development and key biomarkers in patients with severe MPS II is expected to open for enrollment in the second half of 2020.
In severe forms of MPS II, early developmental milestones in a child may be met but delays become apparent by 18 to 24 months, with neurologic deficits and cognitive impairment appearing before the age of 6 years.
This trial is intended to prospectively document the changes in neurodevelopmental parameters of cognitive, behavioral and adaptive function over time in addition to biomarker activity in the CSF, serum and urine.
Up to 40 patients between the ages of 1 month and 8 years with a genetic diagnosis of severe MPS II will be enrolled in this trial and evaluated for up to two years.
As stated above, RGX-121 is to treat MPS II, also known as Hunter syndrome. It is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene which encodes the iduronate-2-sulfatase (I2S) enzyme to the central nervous system (CNS).
The delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.
The FDA has designated RGX-121 orphan drug and rare pediatric disease and granted it Fast Track designations
Mucopolysaccharidosis Type II (MPS II)
MPS II is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans, including heparan sulfate (HS) in tissues which ultimately results in cell, tissue and organ dysfunction.
In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II and prevent or stabilize cognitive decline remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate HS, which has been shown to correlate with neurocognitive manifestations of the disorder.
This stock has been suppressed during the pandemic, together with many, but not all gene therapy companies or other clinical-stage companies. The most critical reason has been due to the hospitals being flooded with COVID-19 infected patients leaving a very narrow space, or opportunity, for the companies to conduct their clinical trials.
Many firms have discovered their way toward finding spaces outside of hospitals in which to resume their clinical trials.
Other reasons for the decline in the gene therapy firms’ stocks as well as the decline of the marketed approved gene therapy sales’ revenues has been the high pricing of the approved gene therapy cures.
We believe that the asked prices are not as high as the complainers are stating when you consider the fact that curing disease with a one-time administrated cure is much less expensive than the lifetime of treatments that are currently being administered to patients with genetic disease.
RGNX closed today at $28.13, UP $1.61.