Regenxbio announces expansion of RGX-121 clinical program

Regenxbio Inc. (RGNX) announced the expansion of its Mucopolysaccharidosis Type II clinical program. The company stated that the expansion will allow it to gain additional understanding of the neurodegenerative aspects of the disease. The expansion will also let Regenxbio in a broader patients population.

The Phase I/II study seeks to examine a single intracisternal administration of RGX-121 in patients under the age of 5 years. All these patients have severe MPS II. The company has already dosed six patients across two dose levels. The main aim of the study is to assess the safety and tolerability of RGX-121 and to evaluate the impact of the drug candidate on biomarkers of I2S enzyme activity, neurocognitive development and other clinical measures.

Regenxbio intends to start expanded enrollment of patients in Cohort 2 immediately. The decision has been taken based on the basis of recommendations made by the Independent Data Monitoring Committee and MPS II treating physicians. Steve Pakola, M.D., Chief Medical Officer of Regenxbio said, 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.

As of September 16, 2020, RGX-121 was found to be well-tolerated in all six patients. No drug-related serious adverse events were reported. The company now plans to dose up to six additional patients with a second dose level of 6.5x1010 genome copies per gram (GC/g) of brain mass. Regenxbio expects to provide further updates from the study by the end of 2020.

Regenxbio also reported that the FDA has cleared its Investigational New Drug Application. The company plans to start the second Phase I/II trial of the drug candidate for treating pediatric patients with severe MPS II ages 5-18 years old. This multicenter, open-label trial will enroll up to six patients and the drug candidate will be administered directly to the cerebrospinal fluid through intracisternal or intracerebroventricular injection.

Regenxbio plans to open a new observational natural history trial for enrollment in the second half of 2020. This trial will study the neurocognitive development and key biomarkers in patients with severe MPS II. The trial will prospectively record the changes in neurodevelopmental issues of cognitive, behavioral and adaptive function over a period of time. It will also evaluate biomarker activity in serum, urine and the CSF. The trial will enroll up to 40 patients between the ages of 1 month and 8 years with genetic diagnosis of severe MPS II. The assessment will be carried out for up to two years.

RGX-121 is being developed for treating Mucopolysaccharidosis Type II. It aims to work by using the AAV9 vector for delivering the human iduronate-2-sulfatase gene which is responsible for encoding the iduronate-2-sulfatase (I2S) enzyme to the central nervous system. RGX-121 has been granted orphan drug product, rare pediatric disease and Fast Track designations by the FDA.

Investment Thesis: Regenxbio had a tumultuous past year and the stock has lost nearly 50 percent of its Pre-Covid valuation. While the latest news is encouraging, it may be a little too early to gauge the companys prospects ahead.

AbbVie Inc. (ABBV) announced that it has taken a voluntary decision so pull Fibristal from the Canadian market. The decision has been taken after the news of certain patients in Europe contracting rare cases of liver injury and requiring transplantation. AbbVie integrated Allergan's business to its core in May this year. The drug belongs to Allergan and the company decided that the recall is the best option in the prevailing circumstances.

Fibristal contains Ulipristal acetate as one of its main ingredients. Earlier this year, the European Medicines Agencys Pharmacovigilance Risk Committee (PRAC) had recommended the cancelation of the marketing authorization of products containing ulipristal acetate used for treating symptoms of uterine fibroids.

Fibristal in approved in Canada for treating moderate-to-severe symptoms of uterine fibroids in adult women of reproductive age. It is also approved as an intermittent treatment of moderate-to-severe signs and symptoms of uterine fibroids in adult women of reproductive age who are not eligible for surgery.

Health Canada is monitoring the effectiveness of the recall. It has advised the patients who take Fibristal to a) stop treatment and contact their doctor to discuss other treatment options; and b) immediately report to their doctor signs and symptoms of liver injury such as nausea, vomiting, stomach ache, severe tiredness, yellowing of the eyes or skin, or dark urine, which could occur after stopping treatment. Health Canada has also issued directives to healthcare professionals to stop prescribing or dispensing Fibristal and to carry out appropriate liver function monitoring.

Investment Thesis: AbbVie is a blue-chip stock with solid foundation. This negative news is not likely to have any material impact on the stock price as the company maintains its robust long-term outlook.

Ionis Pharmaceuticals Inc. (IONS) announced inking a multi-year collaboration with Genuity Science. The deal pertains to the discovery and development of innovative therapeutics across a range of up to 20 diseases. Ionis is looking to boost its presence in key segments with this collaboration and draw synergies.

Genuity Science focuses on high-quality, whole-genome sequence and deep phenotype data. Ionis plans to combine this approach with its proprietary antisense technology to expedite the drug development process. Brett P. Monia, Ph.D., chief executive officer of Ionis said, "The collaboration with Genuity Science gives us the potential to significantly increase our discovery opportunities and better understand the complexities of human diseases based on genetics."

Under the terms of the agreement, Genuity Science will be entitled to receive a combination of upfront payments and development milestones. It will also be entitled to gain product royalties. Genuity Science is a data sourcing, analytics and insights organization. It collaborates with global pharma companies to catalyze precision health services. It offers a wide range of services including high-quality sequencing and disease-specific data sourcing.

Ionis is mainly active in RNA-targeted drug discovery and development segment. The company has developed the first and only approved treatment for all patients, children and adults with spinal muscular atrophy. Its portfolio also contains the worlds first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. The company uses its antisense technology platform to boost its development pipeline. Its drug candidate Pelacarsen is in Phase 3 for cardio-renal indications. Some of its main development partners are Biogen, AstraZeneca and Novartis.

Investment Thesis: Ionis has a well-diversified development pipeline which is expected to keep its stock price buoyant. However, the price has tumbled quite a bit in the recent past, providing an exciting opportunity to build a portfolio with a long-term horizon.

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Link:
Regenxbio RGX-121 Clinical Program, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

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