Drug Discovery and Development

Type: Cure

Overview: We’re working with EndLyz Therapeutics, Inc. to help find therapeutic ways to clear cells of damaging or unwanted materials that might contribute to the causes of Parkinson’s. Recent research suggests that lysosomes, packets of digestive chemicals that help break down and recycle unwanted material inside cells, may be central to the development and progression of Parkinson’s. When lysosomes don’t work properly, brain cells can’t get rid of old and damaged proteins, so these build up and clump together, slowly choking cells.

Key Takeaway: This project will focus on developing new therapies to restore efficient lysosomal function, which may have the potential to slow or stop Parkinson’s.

Type: Cure

Overview: CU-13001 is a molecule that has shown exciting potential for protecting the brain cells that are lost in Parkinson’s in laboratory-based tests. It targets an enzyme called 15-lipoxygenase, which emerging research suggests may play an important role in the death of dopamine-producing cells in PD.

Key Takeaway: We are supporting the final stages of testing and development of CU-13001 ahead of clinical trials. This promising molecule could slow or stop Parkinson’s. 

Type: Life (Symptom)

Overview: It is estimated that around 75% of people with Parkinson’s experience hallucinations during the course of PD. However, current treatment options are limited. We’ve partnered with University College London to explore the potential of ondansetron as a treatment for visual hallucinations in people with Parkinson’s or Lewy body dementia. Ondansetron is currently used to treat sickness following operations or during chemotherapy.

Key Takeaway: This study is investigating whether ondansetron is beneficial and safe as a treatment for hallucinations in 306 people with Parkinson’s or Lewy body dementia.

Type: Life (Symptom)

Overview: There are many symptoms of Parkinson’s and not everyone will experience the same ones. Evidence shows up to 75% of people with Parkinson’s go on to develop hallucinations or delusions as their condition progresses. We’re partnering with King’s College London to carry out a clinical trial to see whether CBD is safe and effective for treating symptoms of Parkinson’s psychosis. The first stage of the study, a six-week pilot to find the ideal dosage of oral CBD capsules, is complete.

Key Takeaway: In 2023, the second stage of the trial started and has now completed recruitment. For this trial, 103 people with Parkinson’s who experience problems with these symptoms take part in a 12-week, double-blind, placebo-controlled study — the gold standard for testing new treatments. Results are expected in mid-2026.

Type: Cure/Life (Symptom)

Overview: Inflammation is vital for defending the body against harm from things like infection and toxins. Researchers believe that inflammation may be linked to the causes and progression of Parkinson’s. We’re working with Syntara to investigate whether a drug called PXS-4728 can reduce inflammation in the early stages of Parkinson’s. This study will investigate PXS-4728 in 40 people who experience sleep disorders known as isolated rapid eye movement sleep behavior disorder (iRBD). Studies suggest that as many as 70% of people with iRBD go on to develop Parkinson’s.

Key Takeaway: The hope is this drug might be able to slow the onset of Parkinson’s symptoms in this group of people that are at a high risk of developing the condition. The first participant was recruited in Australia in November 2023, and in the UK in April 2025. By January 2026, all 40 participants had been successfully recruited. Results are expected by mid-2026.

Type: Cure

Overview: We partnered with research charity Cure Parkinson’s, Van Andel Institute and John Black Charitable Foundation to co-fund a trial looking at the potential of ambroxol, a drug found in a cough medicine which has been used for many years, to slow the progression of Parkinson's. Results from phase 2 of the clinical trial show that ambroxol increases a protein called GCase. A deficiency in GCase can contribute to Parkinson’s.

Key Takeaway: A world-first phase 3 trial began recruiting in 2025. This is the first large phase 3 study in the portfolio, and it offers hope that a drug to slow the progression of Parkinson’s may be on the horizon.

Type: Cure

Overview: Mitochondria are small structures responsible for producing the energy cells need to function properly, including brain cells. In Parkinson’s, mitochondria inside dopamine-producing brain cells stop working properly. MTX325 is a compound that has been designed to help get rid of faulty mitochondria. By removing ‘problem mitochondria’ the team hopes to protect dopamine-producing brain cells and ultimately slow or even stop the progression of the condition. 

Key Takeaway: After successful studies in the lab, MTX325 is currently being trialed in healthy individuals to understand its safety. In 2024, we announced plans to fund the next stage of research through the Parkinson’s Virtual Biotech – investigating the safety and benefits of the potential drug in people with Parkinson’s. The trial will recruit people with Parkinson’s to take part in an early phase clinical trial in early 2026 across multiple sites in the UK and other European countries.

Type: Cure

Overview: HER-096 is a new treatment that aims to protect and restore the dopamine-producing brain cells that are lost in Parkinson’s. It has been developed based on a growth factor the brain naturally produces called Cerebral Dopamine Neurotrophic Factor (CDNF). Research in the lab shows CDNF can help damaged dopamine cells survive and recover. However, CDNF is a large protein that can’t easily cross from the blood into the brain, so giving it to patients would require complex and invasive surgery. HER-096 has similar properties to CDNF but is smaller, can access the brain and be given as a simple injection. 

Key Takeaway: HER-096 has already been tested in a study with 60 healthy participants and was shown to be safe with no serious side effects. In October 2025, results suggested the treatment is safe, well-tolerated and effective at reaching the brain. Additional biomarker data in January 2026 provided further confidence that the drug is acting as expected. In early 2026, Herantis Pharma received funding from the European Union to run a phase 2 trial of HER-096, which is now preparing to start as soon as possible.

Type: Life (Symptom)

Overview: This project investigated a drug called NLX-112 for the treatment of involuntary movements (dyskinesia). This is a common side effect experienced by people with Parkinson’s who have been taking levodopa-based medications for several years. Studies investigated NLX-112 in the lab and secured approval from regulatory authorities to take the drug into clinical trials in people with Parkinson’s. In November 2020, a new phase 2a trial in 22 people with Parkinson’s was announced. Results showed the drug was safe and was beneficial for those who experience dyskinesia. The trial also showed that the drug had a positive effect on other motor symptoms of Parkinson’s. This was an early-stage trial, and its main aim was to prove the safety and understand how the body reacts to the drug, so that it can be tested on a larger scale.

Key Takeaway: Neurolixis is currently planning how to best take this project forward to understand the effectiveness of the drug in more people and over a longer period of time.

Type: Life (Symptom)/Cure

Overview: Eurofins aims to create new molecules to increase the activity of a selection of genes to both increase dopamine production, and boost the production of protective proteins to slow or halt the damage and loss of brain cells in Parkinson’s.

Key Takeaway: There is still potential in this work, but after an independent review, we decided not to invest further at this time.

Type: Cure

Overview: Through our partnership with Sheffield University, drug-like molecules were tested to see if they can help protect brain cells and boost the function of mitochondria, the batteries of the cell.

Key Takeaway: As the project progressed, we made new discoveries that provided valuable insights. We have no current active investment in this work.

Type: Cure

Overview: Inflammation is a vital process for defending the body against harm from things like infections, injuries and toxins. It should only be activated when there’s a threat. If inflammation is active when it shouldn’t be, it can cause harm to healthy cells. There’s increasing evidence that this might be the case in Parkinson’s. Announced in December 2021, this project looked to uncover ways to dial down inflammation in the brain to protect brain cells.

Key Takeaway: The project was stopped because it looked increasingly unlikely to produce a new treatment. 

Type: Cure

Overview: Glial cell-derived neurotrophic factor (GDNF) is a special protein that is naturally produced inside the brain. When GDNF is given to damaged dopamine brain cells in the lab it helps them to regenerate. It may be able to do the same in people with Parkinson’s if it can be delivered to the right part of the brain in the right way. This project aimed to move forward a clinical trial of device delivered GDNF that has potential to help slow, stop or reverse the progression of Parkinson’s.

Key Takeaway: We decided not to continue investing in device delivered GDNF as the costs and difficulties became too high. There are other trials in progress that continue to investigate the potential of GDNF. We’re also now funding a clinical trial of an injectable form of a growth factor that has similar potential to GDNF.

Type: Life (Symptom)

Overview: A drug called ENT-01 has the potential to reduce the effects of alpha-synuclein, a protein thought to play an important role in the development of Parkinson’s, in the gut. Early-stage clinical trials in people with Parkinson’s suggested that the drug improved constipation and revealed it may hold promise for treating other important aspects of Parkinson’s, including dementia.

Key Takeaway: Unfortunately, despite their best efforts, the company has not been able to raise enough investment to take the study forward at this time. However, Enterin remains fully committed to developing ENT-01 for Parkinson’s constipation.

Type: Cure

Overview: In June 2024, we announced a partnership with Lucy Therapeutics to drive forward research looking at new drugs to restore mitochondria. Mitochondria are responsible for producing the energy cells need to function properly. Lucy Therapeutics has developed molecules that early experiments show can target a key part of the mitochondria and improve its function. With investment from this partnership, they looked to see in more detail how the molecules might be able to protect brain cells. This involved exploring whether the molecules can reduce clumps of a protein thought to damage brain cells.

Key Takeaway: The data was not strong enough for further investment and the company has no funds to take this work forward.

Type: Cure

Overview: Keapstone is a company co-founded by researchers at the University of Sheffield and Parkinson’s UK. This project looked at developing drugs that act on multiple pathways believed to be important in the development of Parkinson’s.

Key Takeaway: The company reached the end of our funding. Recent findings have provided interesting avenues for further studies, and the company and University of Sheffield are currently evaluating strategic options to progress the program.

Type: Cure

Overview: Research shows that there’s more inflammation in the areas of the brain impacted by Parkinson’s. This is thought to play a potential role in damaging the dopamine-producing cells in the brain, causing Parkinson’s to progress faster. We partnered with Neumora Therapeutics Inc., a US-based company, to help fund and accelerate the final lab-based research needed to advance a potential new drug that targets inflammation. The drug aims to protect brain cells impacted by Parkinson’s by stopping inflammation being triggered in the brain.

Key Takeaway: Our investment was to help safety test a new treatment that targets neuroinflammation in PD in animal models to progress it toward clinical trials. In 2025, the company shared it will be focusing on a lead indication of obesity. The company has not ruled out working on Parkinson’s again in the future.

Type: Cure

Overview: We partnered with NRG Therapeutics Ltd to find ways to boost the functioning of mitochondria in Parkinson’s. Mitochondria (the power plants of the cell) play an important role in both sporadic and inherited forms of Parkinson’s. With the help of the initial investment, NRG Therapeutics Ltd designed small molecules that are able to pass across the blood brain barrier and patch up a hole in the mitochondria wall to help prevent the loss of energy and cell death. In October 2024, more funding was secured and NRG selected NRG5051 as the molecule to focus on and do the final preparations before it can be tested in clinical trials. 

Key Takeaway: In September 2025, NRG successfully raised enough funding to take the drug into clinical trials in people with Parkinson’s and motor neuron disease. This offers hope as a potential way to protect mitochondria and help stop brain cells from dying.