Top Stocks Targeting the Underserved $5.7B Parkinson’s Opportunity (PRTA, ITCI, ATRX, QURE, GMAB, IMPL, AMRX)

According to a recent report from, the global Parkinson’s disease treatment market is expected to reach $5.69 billion by 2022, growing from $4.24 billion in 2017, expanding at a CAGR of 6.1%. 

The report notes that this market is being driven by rising geriatric population and expanding government funding for research on the disease.

This is a critical potential opportunity for investors because this large market is still technically underserved as a healthcare end target. Resources have begun to flow in, but healthcare workers have few strong solutions to bring to bear for those suffering from this debilitating condition.

With that in mind, we take a look at a few of the most interesting stocks targeting Parkinson’s, along with some of the key recent catalysts impacting these players.

Intra-Cellular Therapies Inc (NASDAQ:ITCI) operates as a biopharmaceutical company that focuses on the discovery and clinical development of small molecule drugs that address underserved medical needs in neuropsychiatric and neurological disorders by targeting intracellular signaling mechanisms within the central nervous system, or CNS, including a focus on Parkinson’s, Alzheimer’s, and schizophrenia.

The company was founded on Nobel prize-winning research that allows it to “understand how therapies affect the inner workings of cells in the body”. The company leverages this intracellular approach to develop innovative treatments for people living with complex psychiatric and neurologic diseases. 

Intra-Cellular Therapies Inc (NASDAQ:ITCI) recently highlighted the publication of four manuscripts that report on the mechanism of action of lenrispodun (ITI-214) and the potential cardiovascular therapeutic effects of Phosphodiesterase Type I (PDE1) inhibition.

“PDE1 inhibition restores intracellular signaling by elevating the second messengers cAMP and cGMP in certain pathological states associated with low levels of these signaling molecules. PDE1 enzymes require calcium and calmodulin for their activation and are expressed in multiple tissues including smooth and cardiac muscle, neurons, and macrophages/microglia providing opportunities to treat multiple diseases,” said Robert Davis, PhD, Senior Vice President and Chief Scientific Officer, Intra-Cellular Therapies.

The context for this announcement is a bit of a bid, with shares acting well over the past five days, up about 13% in that timeframe. 

Intra-Cellular Therapies Inc (NASDAQ:ITCI) managed to rope in revenues totaling $20M in overall sales during the company’s most recently reported quarterly financial data — a figure that represents a rate of top line growth of 951.4%, as compared to year-ago data in comparable terms. In addition, the company has a strong balance sheet, with cash levels exceeding current liabilities ($556.2M against $50.4M).

Adhera Therapeutics Inc (OTC US:ATRX) is another emerging core player in the Parkinson’s space. The company recently provided its shareholders with an update on its corporate strategy and planned clinical development of MLR-1019 (armesocarb) and MLR-1023 (tolimidone) that offers a ton of insight into the path ahead for this small but very promising player.

For example, on July 29 and August 24, respectively, the Company announced executing exclusive license agreements for MLR-1019 for Parkinson’s disease (PD) and MLR-1023 for Type 1 diabetes.

Adhera Therapeutics Inc (OTC US:ATRX) notes that it plans to develop MLR-1019 as a new class of drug for PD and, to the best of the Company’s knowledge, as the only drug candidate designed to address both movement and non-movement symptoms of PD, which places ATRX as both the smallest and potentially the most promising name in this space.

According to the company’s release, “Armesocarb is the active enantiomer in mesocarb, a drug marketed in Europe for 37 years for various psychiatric and central nervous system indications. Based upon previous work conducted by licensing partner Melior Pharmaceuticals II, LLC, a clear understanding of MLR-1019’s mechanism of action, abundant pre-clinical research and decades of clinical data on mesocarb, which is chemically related to armesocarb, Adhera intends to initiate a Phase 2a clinical trial in an Eastern European jurisdiction where mesocarb still has marketing registration. The Company will undertake a parallel process in the U.S. with Investigational New Drug (IND)-enabling studies. Adhera believes that the comprehensive historical data in combination with new data from clinical trials in Europe will facilitate an accelerated development pathway in the U.S.”

“We are very pleased about the positioning of our company today with two Phase 2-ready drug candidates indicated for extremely large market opportunities, as existing therapeutics for PD and Type 1 diabetes are lacking,” commented Andrew Kucharchuk, Chief Executive Officer at Adhera Therapeutics. “We are particularly excited about the pivot to Type 1 diabetes, which we model to positive Net Present Value (NPV), versus a flat NPV for Type 2. Given the dearth of oral medications for Type 1 diabetes, we are optimistic that we will garner attention from institutions and potentially large pharma as we advance our clinical work of MLR-1023.”

Adhera Therapeutics Inc (OTC US:ATRX) notes that it is beginning the process to graduate to OTC Markets’ OTCQB marketplace with the ultimate goal of a NASDAQ listing. The company is also targeting greater transparency through this uplist course, which is a promising sign. Shares have been finding a strong and consistent bid over recent months, up nearly 200% since early June.

Uniqure NV (NASDAQ:QURE) bills itself as a company that engages in the research, development, and commercialization of gene therapies intended to treat hemophilia, Huntington’s disease, glybera, and cardiovascular problems, with research highly applicable to the Parkinson’s space. 

The company sees itself as leveraging its modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with critical underserved conditions.

Uniqure NV (NASDAQ:QURE) recently announced that two additional patient procedures have been completed in the second, higher-dose cohort in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. These latest procedures follow a positive recommendation by the independent Data Safety Monitoring Board (DSMB) after reviewing available follow-up data from all participants in the trial. A total of 14 blinded administration procedures have now been completed, including four patients in the second dose cohort. In the study to date, eight patients have been treated with AMT-130, and six patients received imitation surgery.

“We continue to be very pleased with the enrollment of this important study and look forward to having the trial fully enrolled by the middle of next year,” said David Cooper, M.D., vice president of clinical development at uniQure. “We also look forward to sharing preliminary imaging and biomarker data from initial patients in the U.S. clinical trial before the end of the year and initiating a separate open-label study of AMT-130 in Europe later this year.”

If you’re long this stock, then you’re liking how the stock has responded to the announcement. QURE shares have been moving higher over the past week overall, pushing about 5% to the upside on above average trading volume. 

Uniqure NV (NASDAQ:QURE) managed to rope in revenues totaling $463.9M in overall sales during the company’s most recently reported quarterly financial data — a figure that represents a rate of top line growth of 30119.4%, as compared to year-ago data in comparable terms. In addition, the company has a strong balance sheet, with cash levels exceeding current liabilities ($677.3M against $46.1M).

Other core names in the Parkinson’s space include Prothena Corporation PLC (NASDAQ:PRTA), Genmab 10 Sponsored ADR Ord Shs (NASDAQ:GMAB), Impel Neuropharma Inc (NASDAQ:IMPL), and Amneal Pharmaceuticals Inc (NYSE:AMRX).

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Published by Benjamin Roussey

Benjamin Roussey is from Sacramento, California. He has two master’s degrees and served four years in the U.S. Navy. His bachelor’s degree is from CSUS (1999) where he was on a baseball pitching scholarship. His second master’s degree is an MBA in Global Management from the University of Phoenix (2006). He has worked for small businesses, public agencies, and large corporations. He has lived in Korea and Saudi Arabia where he was an ESL instructor. Benjamin spends his time in between Northern California and Cabo San Lucas, Mexico, committing himself to his craft of freelance and website writing.

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