Axovant Sciences Gene Therapy Showing Progress, Pipeline Review

Axovant Sciences Ltd. (NASDAQ: AXGT) ispart of the Roivant family of companies and is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases.

The company’s current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations.  


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Recent announcements/developments:

  • The company recently reported positive interim results from the first cohort at three months: SUNRISE-PD Phase 2 trial of AXOLenti-PD in Parkinson’s disease
  • The benefit was seen across all UPDRS OFF subscales
  • Based on initial DMC feedback, the first patient in cohort two expected to be dosed in Q2 2019, providing upcoming catalysts
  • On March 14th, Axovant Sciences announced the pricing of its underwritten public offering of 26,666,667 of its common shares at a price to the public of $1.50 per share. Gross proceeds to Axovant from the offering are expected to be approximately $40,000,000 before deducting underwriting discounts and commissions and estimated offering expenses. 

Multiple clinical milestones and data readouts expected in 2019

Key & unique differentiating factor of the company:

  • Robust, diversified pipeline in neurology and other areas of high unmet need
  • Leadership team with deep gene therapy experience from pioneering organizations
  • Integrated development capabilities and expertise from manufacturing to commercial launch
  • Encouraging clinical results with additional near-term data readouts in 2019
  • The company had $84.9 million in cash and cash equivalents as of December 31, 2018 

Analysts’ views: Research firms are positive about the progress of Axovant with most of them predicting a possible re-rating over the near to medium term. Per, Their average twelve-month price target is $6.00, suggesting that the stock has a potential upside of 322.54%. The high price target for AXGT is $7.00, and the low-price target for AXGT is $4.00. There are currently four buy ratings for the stock, resulting in a consensus rating of “Buy.”

Excerpts of the analyst rating history:

Robust Pipeline:

About the gene therapy: Gene therapy aims to address diseases at the most fundamental level: the genes that lead to the disease or its symptoms. There are several different approaches employed by gene therapy, including:

Each of Axovant’s gene therapies is specifically designed to address the underlying biological cause of each disease it tackles:

Key risk factors and potential stock drivers:

  • Successful milestones in the company’ lead candidates would drive the future direction for the company. Any adversities related to this might adversely impact the overall investor sentiments.
  • Axovant is still an early stage entity and has not yet generated meaningful revenue and will likely operate at a loss as it grows its product candidates and seeks ways to monetize it. 
  • The company is offering new securities, which would have the potential dilution risk for the existing shareholders.
  • Axovant has a history of operating losses. Therefore, any time or cost overrun in its ongoing R&D activities and its impact on business & financial profile would continue to remain a key business and stock sensitivity factor. 

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Stock Performance


  • On Friday, March 15th, 2019, AXGT closed at $1.22, with a robust volume of 2.78 million shares exchanging hands. Market capitalization is $189.751 million. The current RSI is 52.12
  • In the past 52 weeks, shares of AXGT have traded as low as $0.05 and as high as $6.59
  • At $1.22, shares of AXGT are trading around its 50-day moving average (MA) at $1.22 and below its 200-day moving average (MA) at $1.94
  • The present support and resistance levels for the stock are at $1.12 & $1.55 respectively. 


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