Research News
The most important results from the last 12 months (18 months for conditions with few trials): what was tested, whether it worked, and what it means for you now.
Recent FDA approvals
- FDA approved Parkinson’s Disease
Juvmo (Tavapadon)
- What it is
- Juvmo is a new pill for Parkinson’s Disease, taken once a day. It is a new kind of Dopamine Agonist, the first approved one that works on only certain dopamine receptors in the brain.
- Who it is for
- Adults with Parkinson’s Disease. It can be used alone in early Parkinson’s Disease, or added to Levodopa for people whose medicine wears off between doses.
- How it works
- It acts like dopamine on two types of dopamine receptors (called D1 and D5) that help control movement. Older Dopamine Agonists act mostly on other dopamine receptors, so this is a different approach.
- What the approval is based on
- Three Phase 3 trials called TEMPO. In two trials of people with early Parkinson’s Disease not taking Levodopa, daily activities improved more than with a placebo after 26 weeks. In a third trial of people taking Levodopa whose medicine wore off, Juvmo added about 1.7 hours a day of good movement time without troublesome extra movements, compared with about 0.6 hours on placebo.
- What this means for you now
- AbbVie expects it in U.S. pharmacies in October 2026. The most common side effects in the trials included nausea, headache, and lightheadedness. Ask us whether it is right for you.
Source: FDA, Novel Drug Approvals for 2026 (Juvmo, 9/25/2026) · AbbVie press release, September 28, 2026 · Drugs@FDA, NDA 220415
- FDA approved Alzheimer’s Disease
Leqembi Iqlik (Lecanemab) starting dose by injection
- What it is
- The FDA approved starting Lecanemab treatment with a weekly shot under the skin at home, using an autoinjector pen. Before this, treatment had to start with IV infusions every two weeks at an infusion center.
- Who it is for
- Adults with Mild Cognitive Impairment or mild Dementia due to Alzheimer’s Disease who have confirmed amyloid plaques and are starting Lecanemab.
- How it works
- Lecanemab is an Anti-Amyloid antibody that helps clear amyloid plaques from the brain and modestly slows decline. The starting dose is two quick shots once a week, each taking about 15 seconds; after 18 months the dose can drop to one weekly shot.
- What the approval is based on
- Sub-studies of the Phase 3 Clarity AD extension showed that the weekly shots put about the same amount of medicine in the blood as IV infusions, so the benefit is expected to be similar. Side effects were similar to IV treatment, mostly mild skin reactions where the shot was given.
- What this means for you now
- It became available in the U.S. in late August 2026 through specialty pharmacies. Regular MRI scans to check for brain swelling or bleeding are still required. People can switch between shots and IV. Ask us whether this option is right for you.
Source: Eisai and Biogen press release, July 13, 2026 · Eisai, availability announcement, August 24, 2026 · Drugs@FDA, BLA 761375
- FDA approved Alzheimer’s Disease
Auvelity (Dextromethorphan and Bupropion)
- What it is
- Auvelity, a pill already approved for depression, is now also approved to treat agitation in people with Dementia due to Alzheimer’s Disease. It is the first medicine for this use that is not an Antipsychotic.
- Who it is for
- Adults with Dementia due to Alzheimer’s Disease who have agitation, such as restlessness, pacing, shouting, or aggressive behavior.
- How it works
- It combines two older medicines. Dextromethorphan acts on brain chemical signals (glutamate and sigma-1 receptors), and Bupropion keeps Dextromethorphan in the body longer.
- What the approval is based on
- Two Phase 3 trials. In ADVANCE-1, agitation improved more with Auvelity than with placebo after 5 weeks. In ACCORD-2, people who had improved and kept taking Auvelity went much longer before agitation returned than those switched to placebo.
- What this means for you now
- It treats agitation, not memory loss, and does not slow Alzheimer’s Disease. Ask us whether it is right for you or your loved one.
Source: Axsome press release, April 30, 2026 (SEC filing) · Drugs@FDA, NDA 215430
Parkinson’s Disease
Only 2 Phase 3 results were reported for Parkinson’s Disease in the past 12 months.
- Positive
Solengepras (CVN424)
- What it is meant to treat
- “Off” periods (times when Levodopa wears off and movement symptoms return) in Parkinson’s Disease, added to Levodopa.
- Dose
- Solengepras was taken as a pill by mouth once a day for 12 weeks, at one of two doses: 75 mg or 150 mg (or a placebo). Everyone kept taking their usual Levodopa and other Parkinson’s Disease medicines. The result we report (about 37 minutes less “off” time per day than placebo) is for the 150 mg dose only. The company has not released the main result for the 75 mg dose.
- How it works
- Solengepras is a once-a-day pill that blocks a brain receptor called GPR6. This receptor sits on nerve cells in a movement circuit that becomes overactive when dopamine is low. Calming this circuit may ease movement symptoms without adding more dopamine, so it is taken along with Levodopa.
- Why it is positive
- People with Parkinson’s Disease who had “off” periods while taking Levodopa took Solengepras or a placebo (sugar pill) for 12 weeks. The higher dose cut “off” time by about 37 minutes more per day than the placebo, and “good on” time grew by about the same amount. People also reported doing better with daily activities, and daytime sleepiness improved.
- What it proves and what it does not
- It shows that a medicine that works without adding dopamine can shorten “off” time when taken with Levodopa, and side effects caused few people to stop. It does not prove long-term safety, the benefit was modest, and the lower dose was not reported as a clear success.
- What’s next
- The company plans to meet with the FDA to discuss applying for approval. No application date has been announced.
- What this means for you now
- Not available yet. Do not change your medicines. If you have “off” periods, ask us at your next visit about the options available today.
Source: Cerevance press release, Oct 7, 2026 · ClinicalTrials.gov NCT06553027 · Cerevance first-patient-dosed release (Nov 18, 2024), listed on news page
- Did not work
Nicotinamide Riboside (a Vitamin B3 supplement)
- What it is meant to treat
- To slow the progression of early Parkinson’s Disease.
- Dose
- Nicotinamide Riboside was taken as capsules by mouth, 500 mg twice a day (1,000 mg a day in all), for one year (52 weeks). There was only one dose, so the result applies to 500 mg twice a day.
- How it works
- Nicotinamide Riboside is a form of Vitamin B3 sold as a supplement. The body turns it into NAD, a molecule cells need to make energy. Researchers hoped that raising NAD would protect the brain’s energy factories and slow Parkinson’s Disease.
- Why it did not work
- People diagnosed with Parkinson’s Disease within the past two years took Nicotinamide Riboside or a placebo for one year. Symptoms did not improve, and people taking the supplement actually did slightly worse than those on placebo, though the difference was small. Brain scans showed no slowing of the disease.
- What it proves and what it does not
- It shows that Nicotinamide Riboside at this dose does not slow early Parkinson’s Disease, even though it raised NAD in the body. It does not show that the supplement is dangerous, since serious side effects were not more common, but it gives no reason to take it for Parkinson’s Disease.
- What’s next
- The researchers conclude the results do not support using Nicotinamide Riboside as a disease-slowing treatment. A separate, smaller dose-finding study (N-DOSE) is a different trial and does not change this result.
- What this means for you now
- We do not recommend buying Nicotinamide Riboside supplements for Parkinson’s Disease. If you already take it, talk with us at your next visit before stopping or continuing any supplement.
Source: JAMA, Oct 8, 2026 (PubMed 42848371) · ClinicalTrials.gov NCT03568968
Multiple System Atrophy
Only 1 Phase 3 result was reported for Multiple System Atrophy in the past 18 months.
- Did not work
Ampreloxetine
- What it is meant to treat
- Symptoms of Neurogenic Orthostatic Hypotension (blood pressure that drops on standing, causing lightheadedness) in Multiple System Atrophy.
- Dose
- Ampreloxetine was taken as a 10 mg tablet by mouth once a day. Everyone took it for the first 12 weeks; then people who improved were split to keep taking 10 mg once a day or switch to a placebo for 8 weeks. There was only one dose, so the result applies to 10 mg once a day.
- How it works
- Ampreloxetine is a once-a-day pill that keeps more norepinephrine, the body’s own blood pressure chemical, active around the nerves that tighten blood vessels. In Multiple System Atrophy the damage is mostly in the brain, so these outer nerves often still work. The goal was to raise standing blood pressure and ease the lightheadedness of Neurogenic Orthostatic Hypotension.
- Why it did not work
- Everyone first took Ampreloxetine for 12 weeks. People who felt better were then split by chance to keep taking it or switch to a placebo (a dummy pill) for 8 weeks. After 8 weeks, lightheadedness and other symptoms on standing had worsened about the same in both groups, so the drug did not beat placebo on the main goal. That is why we say it did not work.
- What it proves and what it does not
- It shows the drug does act on the body: blood pressure and heart rate went up as expected, and it was generally well tolerated without raising blood pressure too much when lying down. It does not prove that people feel better or function better on it. The company's later re-analyses of smaller groups looked more favorable, but those were done after the fact and are not proof.
- What’s next
- The company stopped the program after these results. In May 2026 the FDA said the current data would not support approval but that one more large trial could be a possible path, and the company asked the FDA for another meeting about the design of that trial. No new trial has been announced.
- What this means for you now
- Not available. Proven treatments for Neurogenic Orthostatic Hypotension already exist, including Midodrine, Droxidopa and Fludrocortisone, plus steps like more fluids, salt, compression garments and raising the head of the bed. Do not change your medicines. Ask us at your next visit.
Source: Theravance Biopharma press release (SEC Form 8-K, Exhibit 99.1), March 3, 2026 · Theravance Biopharma CYPRESS Study 0197 Update (SEC Form 8-K, Exhibit 99.1), August 25, 2026 · ClinicalTrials.gov NCT05696717
Trials under way for Multiple System Atrophy
Phase 3 trials of treatments not yet approved that are under way now. Checked on ClinicalTrials.gov on October 15, 2026.
- Phase 3 Enrollment closed; trial in progress
Amlenetug (Lu AF82422)
- What it is meant to treat
- To slow the progression of Multiple System Atrophy.
- Dose
- Amlenetug is given as an IV infusion (through a vein) about every 4 weeks for 72 weeks. People are assigned to a high dose, a low dose, or a placebo (salt water). The company has not made the exact doses public.
- How it works
- Amlenetug is an antibody that attaches to alpha-synuclein, the protein that clumps inside brain cells in Multiple System Atrophy, to help clear it and stop it from spreading.
Source: ClinicalTrials.gov NCT06706622
- Phase 3 Not yet enrolling
ONO-2808
- What it is meant to treat
- To slow the progression of the parkinsonian type of Multiple System Atrophy (MSA-P).
- Dose
- ONO-2808 will be taken as a pill by mouth once a day for 48 weeks, at a low dose or a high dose (or a placebo). The exact doses have not been made public.
- How it works
- ONO-2808 activates a receptor (S1P5) on oligodendrocytes, the support cells that are damaged first in Multiple System Atrophy. The goal is to protect these cells and slow the disease. In its earlier Phase 2 study, signs of slower worsening were encouraging but not yet proven.
Source: ClinicalTrials.gov NCT07825402 · Ono Pharmaceutical, May 28, 2026
- Phase 3 Enrolling now
MSA-01 (Ubiquinol, a form of Coenzyme Q10)
- What it is meant to treat
- To slow the worsening of movement problems in Multiple System Atrophy.
- Dose
- MSA-01 (Ubiquinol) is taken by mouth for 52 weeks, compared with a placebo. The dose and how often it is taken have not been made public. The earlier Phase 2 study used 1,500 mg a day, but it is not confirmed that this new study uses the same dose.
- How it works
- Ubiquinol is a well-absorbed form of Coenzyme Q10, which brain cells use to make energy. Low Coenzyme Q10 may play a part in Multiple System Atrophy, and an earlier Phase 2 study was encouraging.
Source: ClinicalTrials.gov NCT07446894
Progressive Supranuclear Palsy
No Phase 3 results were reported for Progressive Supranuclear Palsy in the past 18 months.
Trials under way for Progressive Supranuclear Palsy
Phase 3 trials of treatments not yet approved that are under way now. Checked on ClinicalTrials.gov on October 15, 2026.
- Phase 3 Enrolling now
NIO752
- What it is meant to treat
- To slow the progression of Progressive Supranuclear Palsy.
- Dose
- NIO752 is given by injection into the spinal fluid (intrathecal), or a placebo, with 2 out of every 3 people getting NIO752. The main comparison is at 72 weeks. The company has not made the dose or the injection schedule public.
- How it works
- NIO752 is a small piece of genetic material (an antisense oligonucleotide) that lowers the making of tau, the protein that builds up and damages brain cells in Progressive Supranuclear Palsy. It is given into the fluid around the spinal cord.
Source: ClinicalTrials.gov NCT07498426 · CurePSP, Novartis PRESERVE update, May 7, 2026
Alzheimer’s Disease
Only 2 Phase 3 results were reported for Alzheimer’s Disease in the past 12 months.
- Positive
Rivastigmine twice-weekly skin patch (TW-4752N)
- What it is meant to treat
- Memory and thinking symptoms of mild to moderate Alzheimer’s Disease.
- Dose
- The new Rivastigmine patch (TW-4752N) was put on the skin and changed every 3 or 4 days (twice a week). People started on a smaller patch holding 25.92 mg for 4 weeks, then moved up to a larger patch holding 51.84 mg for the next 20 weeks. The other group used the standard once-a-day Rivastigmine patch, starting at 4.6 mg per 24 hours for 4 weeks, then 9.5 mg per 24 hours. The main result (memory and thinking at least as good as the daily patch at 24 weeks) compares the 51.84 mg twice-a-week patch with the 9.5 mg per 24 hours daily patch. The highest daily patch sold in the U.S., 13.3 mg per 24 hours, was not tested.
- How it differs from what is available now
- It is the same medicine (Rivastigmine) given the same way, through the skin, and it is not a higher dose. In an earlier study, the 51.84 mg twice-a-week patch put about the same amount of Rivastigmine into the blood as the daily 9.5 mg per 24 hours patch. The difference is the patch itself: it holds more medicine and releases it slowly over 3 to 4 days, so it is changed twice a week instead of every day. The 51.84 mg is the total medicine stored in the patch, not the amount given each day. There is no twice-a-week version of the highest daily dose (13.3 mg per 24 hours). Skin itching was more common with the new patch.
- How it works
- Rivastigmine is a Cholinesterase Inhibitor, the same kind of medicine as Donepezil. It slows the breakdown of a brain chemical needed for memory and thinking. This new skin patch holds enough medicine to be changed only every 3 to 4 days (twice a week) instead of every day.
- Why it is positive
- In 46 clinics in Japan, people with mild to moderate Alzheimer’s Disease used either the new twice-a-week patch or the standard once-a-day Rivastigmine patch for 24 weeks. Memory and thinking scores were at least as good with the new patch as with the daily patch, which was the main goal of the study. It is called positive because it met that goal and means half as many patch changes each week for patients and caregivers.
- What it proves and what it does not
- It shows the twice-a-week patch works about as well as the daily patch over 6 months, with similar side effects, though skin itching at the patch site was more common (about 28 in 100 people versus 17 in 100). It does not show any slowing of Alzheimer’s Disease; like the daily patch, it only helps symptoms. It was tested only in Japanese patients and only against another Rivastigmine patch, not against placebo.
- What’s next
- The study was run by a Japanese company and the report did not say when or where it will apply for approval. No U.S. plans have been announced.
- What this means for you now
- Not available in the U.S. The daily Rivastigmine patch and other Cholinesterase Inhibitors are available now. Do not change your medicines. Ask us at your next visit if daily patches are hard to manage.
Source: Journal of Alzheimer’s Disease, Dec 2025 (PubMed 41364455) · Full text, PubMed Central · Japan Registry of Clinical Trials jRCT2031210496
- Did not work
Oral Semaglutide (Rybelsus, approved for Type 2 Diabetes; not approved for Alzheimer’s Disease)
- What it is meant to treat
- To slow early Alzheimer’s Disease (Mild Cognitive Impairment or mild Dementia due to Alzheimer’s Disease).
- Dose
- Semaglutide was taken as a pill by mouth once a day. The dose was raised step by step up to 14 mg once a day; the dose was flexible, so people who could not handle 14 mg could stay on a lower dose. People took it for up to 3 years. There was one Semaglutide group in each trial, and the result (no slowing of Alzheimer’s Disease) applies to this up-to-14 mg daily pill.
- How it differs from what is available now
- It is the same medicine already sold as Rybelsus for Type 2 Diabetes, and 14 mg once a day is the highest Rybelsus dose. The study did not use a new pill or a higher dose. It did not test the Semaglutide shots (Ozempic, Wegovy) or the higher-dose Wegovy pill (25 mg) used for weight loss. Note: the newer Ozempic pills (1.5, 4 and 9 mg) are a different tablet and their mg numbers cannot be compared directly with Rybelsus.
- How it works
- Semaglutide is a GLP-1 medicine already used for Type 2 Diabetes and weight loss (Ozempic, Wegovy, Rybelsus). It may calm inflammation in the brain and protect nerve cells. Large studies of people with diabetes had suggested that people taking these medicines got Dementia less often, so it was tested as a daily pill in early Alzheimer’s Disease.
- Why it did not work
- People aged 55 to 85 with Mild Cognitive Impairment or mild Dementia due to Alzheimer’s Disease took a Semaglutide pill or a placebo (sugar pill) every day for 2 years. In both trials, memory, thinking and daily function got worse at the same rate on Semaglutide as on placebo. We say it did not work because it did not slow the disease at all.
- What it proves and what it does not
- It proves that Semaglutide pills do not slow early Alzheimer’s Disease, even though some blood and spinal fluid markers of the disease improved slightly. It does not answer whether other GLP-1 medicines, higher doses, injections, or starting much earlier (before memory problems) could help. Side effects were the same kind seen when it is used for diabetes, mainly stomach upset.
- What’s next
- Novo Nordisk stopped the planned third year of both trials because of the results, and no plan to apply to the FDA for Alzheimer’s Disease has been announced. Researchers are studying the data to understand why the earlier hopeful signals did not hold up.
- What this means for you now
- Do not start Semaglutide to prevent or treat Alzheimer’s Disease. If you already take it for diabetes or weight, it is still helpful for those reasons, so do not stop it. Ask us at your next visit about treatments that are proven for memory.
Source: Novo Nordisk company announcement, Nov 24, 2025 · The Lancet, March 2026 (PubMed 41865758) · ClinicalTrials.gov NCT04777396 (EVOKE) · ClinicalTrials.gov NCT04777409 (EVOKE+)
Huntington’s Disease
No Phase 3 results were reported for Huntington’s Disease in the past 18 months.
Trials under way for Huntington’s Disease
Phase 3 trials of treatments not yet approved that are under way now. Checked on ClinicalTrials.gov on October 15, 2026.
- Phase 3 Enrolling now
Votoplam (HTT227, formerly PTC518)
- What it is meant to treat
- To slow the progression of early symptomatic Huntington’s Disease.
- Dose
- Votoplam is taken by mouth at a dose of 10 mg, compared with a placebo, for up to 3 years. There is only one dose in this study. How often it is taken is not listed in the trial record; in the earlier study, the same 10 mg dose was taken once a day.
- How it works
- Votoplam changes how the huntingtin gene’s message is read, so the body makes less of the harmful huntingtin protein.
Source: ClinicalTrials.gov NCT07326709 · PTC Therapeutics press release, Apr 28, 2026; ClinicalTrials.gov NCT07326709
- Phase 3 Enrolling now
Pridopidine
- What it is meant to treat
- To slow the overall worsening of Huntington’s Disease, including daily function.
- Dose
- Pridopidine is taken as a 45 mg capsule by mouth. For the first 2 weeks it is taken once a day (in the morning); after that it is 45 mg twice a day (morning and afternoon, 7 to 10 hours apart) for 50 more weeks, compared with a placebo. There is only one dose in this study.
- How it works
- Pridopidine activates the sigma-1 receptor, which helps brain cells handle stress and stay healthy. An earlier Phase 3 study (PROOF-HD) did not meet its main goal, so this new study tests it again in a more selected group.
Source: ClinicalTrials.gov NCT07609108
- Phase 2/3 Enrolling now
SKY-0515
- What it is meant to treat
- To slow the progression of Huntington’s Disease and improve its symptoms.
- Dose
- SKY-0515 is taken as a pill by mouth once a day for 18 months, at one of three doses (low, middle or high) or a placebo. The exact doses have not been made public.
- How it works
- SKY-0515 changes how the huntingtin gene’s message is read, so less of the harmful protein is made.
Source: ClinicalTrials.gov NCT07378644
Essential Tremor
Only 1 Phase 3 result was reported for Essential Tremor in the past 18 months.
- Positive
Ulixacaltamide (PRAX-944)
- What it is meant to treat
- Essential Tremor in adults (shaking of the hands and arms that gets in the way of daily tasks).
- Dose
- Ulixacaltamide was taken as a pill by mouth once a day in the morning. The dose was raised over 2 weeks: 20 mg for 1 week, 40 mg for 1 week, then 60 mg a day. The first study lasted 12 weeks, and its main result was measured at week 8. In the second study everyone took the pill for 8 weeks, and then those who improved were randomly kept on 60 mg or switched to placebo for 4 more weeks. Both results apply to the 60 mg daily dose.
- How it works
- Ulixacaltamide is a once-a-day pill that blocks T-type calcium channels, tiny gates on nerve cells. In Essential Tremor, brain circuits that control movement fire in a rhythm that is too strong. Blocking these gates may calm that rhythm and ease the shaking.
- Why it is positive
- Adults with Essential Tremor took Ulixacaltamide or a placebo (sugar pill) at home, in a study run by video and mail. In the first study, people on Ulixacaltamide had less trouble with everyday tasks like eating, drinking, and writing after 8 weeks than people on placebo. In the second study, people who had improved on the pill and then stayed on it were more likely to keep their benefit than people switched to placebo (just over half versus about one in three).
- What it proves and what it does not
- It shows that Ulixacaltamide can lessen how much tremor gets in the way of daily tasks, and that the benefit lasts while people keep taking it. It does not prove long-term safety. About 1 in 4 people on the pill stopped because of side effects, mostly lightheadedness and foggy thinking, compared with very few on placebo. Other common side effects were constipation, headache, tingling, and trouble sleeping.
- What’s next
- The FDA has accepted Praxis’s application and set a decision date of January 29, 2027. This is not an approval, and the FDA may approve it, ask for more information, or decline.
- What this means for you now
- Not available yet. Do not change your medicines. Ask us at your next visit about the treatments for Essential Tremor that are available today.
Source: Praxis Precision Medicines topline press release, Oct 16, 2025 (SEC Form 8-K exhibit) · Praxis Precision Medicines topline press release (full text, BioSpace copy) · ClinicalTrials.gov NCT06087276 · Praxis press release, April 14, 2026 (FDA accepts application)
Why we only report Phase 3 trials
Most treatments that look promising in smaller, earlier studies do not work when tested in larger ones. For brain and nervous system drugs, about 9 out of 10 that reach Phase 2 are never approved by the FDA. Phase 3 trials are the large studies the FDA relies on to decide whether a treatment works and is safe. Even so, about half of Phase 3 neurology drugs do not succeed, so we explain each result carefully.
| Brain and nervous system (neurology) drugs | Exact figure |
|---|---|
| Drugs in Phase 2 that never move on to Phase 3 | 73.2% (only 26.8% advance) |
| Drugs entering Phase 2 that never get FDA approval | 87.7% (only 12.3% are approved) |
| Drugs entering Phase 3 that never get FDA approval | 54% (46% are approved) |
Source: BIO, Informa Pharma Intelligence, and QLS, Clinical Development Success Rates 2011–2020.
Find a clinical trial
Two places to find clinical trials you may be able to join:
-
ClinicalTrials.gov
Search ClinicalTrials.gov, the U.S. National Library of Medicine’s registry of clinical studies around the world.
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SiteRx
SiteRx is a healthcare research organization that connects people with clinical studies through the doctors they already see. With your permission, SiteRx requests your health records from your doctor and uses them to check whether you may qualify for a study. You may opt out at any time, and SiteRx encourages you to talk with your doctor first.