Envisioning a future with next-gen genetic medicines
Updated August 2024
Market/Symbol
NASDAQ:MGTX
Price
$4.180.48%
Market Cap
$323m
PE Ratio
0
Average Volume
204k
Pricing delayed 2 hours. Sep 16, 2024 5:00 AM
Investment thesis
Envisioning a future with next-gen genetic medicine
MeiraGTx, an emerging leader in genetic medicine, boasts Johnson & Johnson and Sanofi as shareholders, and has a number of late-stage drugs in its clinical pipeline using its first-gen gene technology. MeiraGTx is pioneering future genetic medicines with its riboswitch technology that broadens the application of genetic medicine to a wider range of disease areas.
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Gene regulation could also help to treat obesity, one of the most pressing long-term health issues facing the NHS. Meira's technology works by "switching on" the genes that produce the hormones and peptides that make you feel full
Daniel Keane
Health Reporter at The Standard
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The opportunity to address Parkinson’s disease (PD) lies in the development of AAV-GAD genetic medicine, which targets the specific neurological dysfunction underlying Parkinson’s. PD is a progressive disease characterised by degeneration of neurons involved in motor control. There are currently 10 million people affected by PD worldwide, and the exciting treatments whose efficacy is limited duration. AAV-GAD genetic medicine shows promise in treating PD by regulating brain activity without relying on dopamine. It is a one-time treatment that has proven to be safe and effective in improving motor function, potentially filling a huge unmet need in Parkinson’s treatment.
With more than 180,000 sqft of combined dedicated manufacturing facilities in the UK and Ireland, MeiraGTx has brought all aspects of manufacturing and process development of its genetic medicines in-house. MeiraGTx’s facilities support the supply of its own innovative genetic medicines at all stages of the product development cycle, from pre-clinical stages through clinical trials and commercialization, whilst offering the scale to support drug development for other companies.
In 2019, MeiraGTx and Janssen, a subsidiary of Johnson & Johnson, entered an agreement for the clinical development of MeiraGTx’s leading IRD pipeline. MeiraGTx received an upfront payment of $100 million and the potential of additional milestone payments of up to $340 million, along with untiered royalties. In 2023, MeiraGTx entered into an Asset Purchase Agreement with J&J who acquired full rights for the development, manufacturing and commercialisation of Bota-vec, a drug linked for the treatment of X-linked Retinitis Pigmentosa. MeiraGTx received $130 million upfront, and will receive an additional $285 million based on first commercial sales and manufacturing technology transfer.
The proprietary Riboswitch technology involves the precise control of genetic medicines via a sort of ‘molecular switch’, or a regulator. This is initiated by the patient simply taking a pill that activates the specific genetic medicine to produce the required proteins at the precise time and the precise dosage needed for that particular condition. It improves the potency, safety and specificity of genetic medicines. This technology can activate a combination of drugs, making it versatile for genetic medicine, cell therapy, and gene editing.
In October 2023, MeiraGTx received a $30 million strategic investment from pharma giant Sanofi that reinforces MeiraGTx's financial position but also validates the company's potential in the genetic medicine landscape. This type of investment is a rare occurrence for a young company involved in early-stage biotech. In exchange for the $30 million, Sanofi received a Right of First Negotiation for MeiraGTx's phase 2 Xerostomia program, which treats salivary glands damaged by radiotherapy treatments. In addition to this, Sanofi will get the first look at MeiraGTx's Riboswitch gene regulation technology in certain targets.
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