MeiraGTx: Positive PD Data Moves Needle Forward For Regulatory Meetings

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MeiraGTx Holdings plc's GAD gene therapy for Parkinson's Disease shows promising phase 1/2 results, with significant improvements in UPDRS Part 3 and PDQ-39 scores.The company plans to discuss phase 3...

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This program is still coming along well and the company is expected to still earn a few milestone payments for this program. Plus, phase 3 data from the ongoing study for this program is expected by the end of 2024. Being that Janssen, a subsidiary of Johnson & Johnson (JNJ) took control of this program, the timing of data release will be contingent on its schedule. However, this time around, there were two other developments to note relating to its pipeline. The first new development would be that the company just reported a positive phase 1/2 study targeting patients with Parkinson's Disease [PD] using gene therapy AAV-GAD. While this was good news and may provide a small boost in the stock price today on the back of this news, it is more important to consider the possible regulatory approval pathway that might happen. That's because with this positive data on hand, it now intends to speak with multiple regulatory agencies in Q4 of 2024 around designing a phase 3 study to move forward with. This would not only lead to catalysts of late-stage study initiations, but then eventual data and possibly regulatory filings thereafter. The second and final noteworthy piece of news to mention since the last time I wrote about this company would be that it received $50 million in funding through an offering. More significant than that, about $30 million of that came from Sanofi (SNY) as an equity investment. Lastly, this company is looking to change the scope of metabolic disorder and obesity treatment with its riboswitch platform technology. With BLA filings expected each year for the next several years based on its pipeline, plus the PD program moving forward with positive data, I believe that investors could benefit with any potential gains made here.  AAV-GAD Moves Needle Forward In Tested Efficacy Measurements  As I noted above, the company was able to report positive results from its phase 1/2 bridging study, known as MGT-GAD-025, for the treatment of patients with Parkinson's Disease. It moved the needle forward in terms of clinical improvement for these patients, especially now that it has proven to be a disease modifying therapy. Before going over the clinical study data, plus any catalysts that are expected to be released from this program, it is first important to understand what this disorder is and what the possible market opportunity for it could be. Parkinson's Disease [PD] is a type of central nervous system [CNS] disorder whereby patients have a problem with movement problems, mental health and other neurological issues. A major concern with these patients is that they lack the proper dopamine in the brain for movement, thus levadopa [being a precursor to dopamine] is used as a treatment option in an attempt to halt the progression of PD. Some symptoms that these patients with PD experience are as follows:  Slowed movements  Muscle stiffness Unsteady posture  Abnormal gait [change in walking pattern].  The global Parkinson's Disease Therapeutics' market is projected to grow to $6.7 billion by 2034. This is a massive market opportunity, but to capture it, this company has to move the program towards the finish line. Having said that, everything is going right thus far with this program. What makes me say that? Well, that's because, first, it recently released positive data from its phase 1/2 MGT-GAD-025 study. This trial deployed three arms for patients as follows:   Low-dose AAV-GAD [5 patients]  High-dose AAV-GAD [5 patients] Sham Control [4 patients].  One important thing to note is that there were some recruitment requirements that had to be met for idiopathic PD patients to be recruited into this trial. First, they had to have a history of being responsive to levodopa therapy for at least 12 months, along with needing a UPDRS Part 3 score of ≥25 points in the “off” state. What is this “off” period in PD? This is where, despite a patient taking their prescribed medication, they have the return of symptoms that affect their daily lives. These patients were treated and evaluated over a 6-month period after being given AAV-GAD via subthalmic nuclei administration.  The data presented was highly positive, especially when you consider that the high dose of AAV-GAD achieved what it needed to. The higher dose of the drug at Week 26 was able to obtain a statistically significant 18-point average improvement from baseline in UPDRS Part 3 “off” medication score. This was achieved with a statistically significant p-value of p=0.03. However, there was no change observed in the sham control or low dose AAV-GAD group. It is good to see that at least the high dose group yielded the necessary efficacy to move forward with. Besides needing efficacy endpoints for approval, another important item for any biotech to accomplish would be to improve quality of life [QoL] for patients. Having said that, the PDQ-39 score was shown to have improved by 8 points from baseline with high-dose AAV-GAD being given to PD patients. This was also a statistically significant change, with a p-value of p=0.02. The other dosing groups failed to achieve significance, as shown here below:   Low-dose AAV-GAD : 6 points from baseline  Sham surgery group : -0.2 points from baseline [worsening].  Okay, so the high dose of AAV-GAD achieved both of these efficacy measures. However, there is one other critical item to consider here, which is changing the scope of treatment for these patients. How so? Well, consider that the 18 points change in UPDRS Part 3 “off” score is much better than the 5 to 10 points needed to achieve clinical significance. The same can be said in PDQ-39 score as well, where patients given other treatment options only see a 2 to 4 point improvement. In this case, with this phase 1/2 MGT-GAD-025 trial, the high dose group achieved an 8 point improvement regarding this score. The potential for MeiraGTx is to offer a new possible disease-modifying PD treatment option for patients. Plus, it is pretty neat in that patients only need to go through a minimally invasive procedure to receive only one infusion of one drop of gene therapy into the subthalmic nucleus. Why is this gene therapy delivered to this? It is because this portion of the brain that is the main component of motor control and function. Thus, affecting the circuitry here of the patient, it is believed that this gene therapy provides the necessary GABA to prevent disease progression and help the patient improve in terms of QoL. This program specifically brings about several catalysts in one. By that, I mean the company intends to meet with regulators to discuss a phase 3 study for this program in Q4 of 2024. Should discussions go well, then it could start a few or more late-stage studies in 2025. Not only that, but it would set up AAV-GAD to be commercialized across several territories.  Financials According to the 10-Q SEC Filings, MeiraGTx had cash, cash equivalents and restricted cash of $101 million as of June 30th of 2024. As I stated in the beginning above, the company was also able to raise cash through an offering. It agreed to sell a total of 12.5 million ordinary shares of its common stock at a price of $4 per share. It generated roughly $50 million with this financing. Even better, Sanofi wanted to take part in this financing that was obtained. Thus, this big Pharma decided to make a $30 million equity investment into the company through the offering. This financing was necessary, especially when you consider its cash runway projection before it. Beforehand, the company believed that it would have enough cash on hand to fund its operations, or cash runway, for at least 12 months. However, with this new cash infusion it should be in good shape to fund itself for an extended period of time. The company cash burn roughly $46.2 million in cash per quarter. This is broken down into $11.3 million in G&A expenses and then $34.9 in R&D expenses.  Risks To Business  There are several risks for investors to consider before investing in MeiraGTx. The first risk would be regarding the development of AAV-GAD for the treatment of patients with Parkinson's Disease. With the positive data on hand for this program, the goal is to bring it to multiple regulatory agencies in the hopes of being able to initiate phase 3 studies. The risk is that there is no guarantee that the FDA or any other regulatory agency will feel that the data is enough to warrant further investigation into a phase 3 study. If that is the case, then it might be inclined to run another mid-stage study, setting it back.  The second risk to consider would be in terms of the ongoing phase 3 study being done by Janssen of Bota-Vec, which is being developed to treat patients with XLRP. Data from this study is expected before the end of 2024 and there is no assurance that the primary efficacy endpoint will be met with statistical significance. A failure would not only mean an inability to further develop this program, but it would also result in the partnership being terminated as well.  The third and final risk to consider would be the company's development of its riboswitch platform to develop small molecules targeting metabolic and obesity disorders. It is believed that this technology can be used to control messenger RNA [mRNA] production through the use of small molecules. However, achieving this function with similar efficacy to that of a biologic. What the advantage of this? Developing biologically like small molecules capable of not needing to be developed in an ex-vivo fashion or the need for repeat dosing. The risk here is that there is no assurance that the use of this technology will be proven in human testing. Nor, that the company will be able to overcome all the other competing drugs in the metabolic and/or obesity space.  Conclusion  MeiraGTx has done well to develop several of its drugs in its pipeline. Most notably, it achieved a few recent milestones to boost shareholder value. The first of which would be that it was able to obtain a $30 million equity investment from Sanofi. Thus, it raised a significant amount of cash to fund several of its gene therapy products, along with the ability to use some cash to help develop the gene splicing riboswitch platform. If this works out, then it could allow the company to develop drugs as biologics but in small molecule forms. The second of which is achieving positive data from the phase 1/2 MGT-GAD-025 study. This now brings about a collection of possible regulatory milestones. That is, the company might receive a green light from these agencies to begin a phase 3 study using AAV-GAD for the treatment of patients with PD. Beyond these candidates I discussed above, the biotech has an extensive pipeline with other gene therapies. There are a total of 4 clinical programs with regulatory filings expected each year in 2024, 2025, 2026 and 2027 respectively.   Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. 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