Analyzing the Parkinson's disease clinical trial pipeline to facilitate patient collaboration in research.
Begun recruiting or been newly listed?
Recruiting began for three trials:
- Phase 2 trial of Intracellular Therapies’ PDE-1 inhibitor lenrispodun as an adjunctive therapy for motor fluctuations
- Phase 2 trial of Kainos Medicine’s FAF 1 inhibitor KM819
- Phase 1 trial of Shanghai iCELL Biotechnology’s human amniotic epithelial stem cells
Six other trials were newly listed but not yet recruiting at quarter end -- most notable is the Ambroxol Phase 3 study on slowing disease progression.
Completed enrollment?
Six trials completed enrollment including two Phase 2 trials from the UCB & Neuropore collaboration (aSN misfolding inhibitor UCB0599 & aSN antibody UCB7853). Also of note was completion of enrollment for the Phase 1 GDNF gene therapy trial from Bayer / Ask Bio.
Reached clinical completion?
Another nine trials reached clinical completion including a Phase 3 trial of Pharma Two B’s combination product of rasagiline & pramipexole. Amongst earlier stage trials of note were Phase 1 trials of Inhibikase Therapeutics c-Abl kinase inhibitor iKT-148009 and Vaxxinity’s “endobody” to aSN.
Been delayed (or accelerated)?
Delays in completion date were disclosed for 17 studies (for 10 the previous completion date had already passed suggesting the sponsor was behind on updating clinicaltrials.gov) with virtually all the delays being at least 6 months. Most notable was the entire Phase 3 program (4 studies) for Cerevel’s tavapadon (a dopamine D1/5 partial agonist) which were delayed 12-16 months. In contrast, just three studies reported an accelerated completion date (by 3 to 6 months).
Completion projected within the next six months?
Twenty-four trials are projected to complete by Q3 2023 with 12 to complete in Q2 2023
• Included are the Neurocrine Phase 3 trial of the COMT inhibitor opicapone and a large (400 patient) Norwegian trial of the supplement nicotinamide riboside
• Earlier stage highlights are a Phase 2 trial of pimavanserin for impulse control disorder and a Phase 1 stem cell trial at Memorial Sloan Kettering
Q1 2023 Trial Results Headlines (ref. dashboard 4)
What trials have . . .
Q1 2023 Trial Status Headlines (ref. dashboards 1-3)
What trials have . . .
Note that clinical trial results are often disclosed for the first time via company press releases and/or investor presentations. For public companies this is often driven by requirements for timely disclosure of material events deemed likely to inform investment decisions. These corporate disclosures typically include only the key top-line results. More detailed trial results may be disclosed via posters or presentations at scientific conferences. Eventually comprehensive trial results are generally published in medical journals sometimes as a “pre-print” (that has not yet been reviewed by experts not involved in the trial) and ultimately as a final peer-reviewed manuscript. Trial results can also be posted by sponsors in the clinicaltrials.gov database, but this often lags behind other routes of disclosure.
quarterly trial dashboards
Had results disclosed for the first time?
Results for eight trials were disclosed for the first time. Only one trial was positive on a primary efficacy endpoint – the Mitsubishi Tanabe / Neuroderm Phase 3 trial of ND0612 (a sub-cutaneous pump of LD/CD) which demonstrated reduced motor fluctuations relative to oral LD/CD (when used on top of otherwise optimized oral therapy).
Three smaller studies (not designed with a primary efficacy endpoint) were described as having safety supporting further development and favorable trends on secondary efficacy endpoints:
- Phase 2 study (27 patients) of bifiradol (NLX-112 from Neurolixis) a selective serotonin 5HT1A full agonist. Study results indicated potential to reduce levodopa-induced dyskinesias.
- Phase 1 study (11 patients) of the GDNF gene therapy (AAV2-GDNF) from Bayer / Ask Bio suggested some symptom improvement in patients with moderate disease although not in the cohort with mild disease.
- Phase 1 study (12 patients) of a lyophilized fecal extract (PRIM-DJ2727) with trends indicating possible improvement in both GI and non-GI symptoms.
Four Phase 2 trials had negative results disclosed on their primary efficacy endpoints. For the Aptinyx NYX-458 (NMDA receptor modulator) program, the negative results signaled the end of development of this agent for cognitive impairment in PD or Lewy Body Dementia. For the other three negative Phase 2 studies, favorable data on secondary endpoints or in patient sub-groups was suggested as supporting further studies. Notable here was the Phase 2 study with Neuraly’s GLP-agonist NLY-01 which did not show a benefit in the study overall but had favorable data in the sub-set of patients under 60 with early PD.
Had additional detail on results disclosed?
Additional results were disclosed for ten trials for which at least top-line results had been previously reported. Notable was a peer-reviewed manuscript on the Phase 1 study with DNL-151 (LRRK2 inhibitor from Denali Therapeutics & Biogen) which led to the ongoing Phase 2 program for this agent. Also of interest was a conference presentation on CVN424 (selective GPR6 inverse agonist from Cerevance) for a large (132 patient) Phase 2 study showing reduced motor fluctuations. Further studies with this agent are also ongoing.
Dashboard Methodology
- Trial data for Parkinson's disease Phase 1, 2, 3 trials downloaded from ClinicalTrials.gov on first and last days of quarter.
- "Status" parameter compared at beg. and end of quarter to identify trials that: were registered, started or completed recruitment, or were completed or withdrawn during the quarter. "Primary completion date" parameter compared at beg. and end of quarter to determine trials with a change in expected primary completion date. Trials with "Primary completion date" within 6 months were also identified.
- Dashboards limited to Phase 1 to Phase 3 trials evaluating pharmaceuticals, including biologics, cell and gene therapies. The "Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline" reports for 2020, 2021, 2022 and The Hope List were references in identifying pharma vs. non-pharma trials within the Phase 1-3 trials, along with some information in the "agent description" and "company/sponsor" fields.
- Dashboards on Trial Results based on alerts from PubMed.gov, review of conference abstract books, daily emails from Parkinson’s News Today, the Science of Parkinson’s Disease blog, pre-print servers, and the Parkinson’s Research Interest Group on Facebook. It is challenging to capture all results disclosures, so if anything is missing, please let us know at PDTrialTracker@outlook.com.
Q1 2023 Trial Dashboards
Clinical Trials of PD Drug Therapies: What Happened During Q1 2023?
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