sasineprocel (ANPD001) / Aspen Neurosci - LARVOL DELTA

Aspen Neuroscience Announces Publication of Sasineprocel Preclinical Data in 'Cell Stem Cell' (GlobeNewswire) - "The peer-reviewed paper describes the scientific and translational foundation for sasineprocel (ANPD001), Aspen’s investigational autologous induced pluripotent stem cell (iPSC)-derived dopamine neuron replacement therapy for Parkinson’s disease (PD), designed to restore dopamine and underlying neural circuitry lost in the progression of PD....Additionally, improvement in motor function was observed in these preclinical PD models, and there were no treatment-related adverse findings in a nine-month Good Laboratory Practice (GLP) safety study in a rodent model."

Preclinical • Parkinson's Disease

Aspen Neuroscience Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Sasineprocel (ANPD001) to Treat Parkinson's Disease (PRNewswire) - "The RMAT designation for sasineprocel is based on results from the ongoing Phase 1/2a ASPIRO trial, which has demonstrated encouraging early clinical activity and a favorable safety profile."

FDA event • Parkinson's Disease

Aspen Neuroscience…announced completion of dosing in Cohort 3 and Cohort 4 of its ongoing Phase 1/2a ASPIRO clinical trial evaluating sasineprocel (ANPD001) for the treatment of Parkinson's disease (PD) (PRNewswire) - "With the completion of these cohorts, a total of 15 patients have been dosed in the ASPIRO study to date, representing one of the largest clinical experiences reported for an autologous cell therapy in Parkinson's disease....The ASPIRO study is an open-label Phase 1/2a clinical trial designed to evaluate the safety, tolerability, and potential efficacy of sasineprocel, an autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell (DANPC) therapy."

Trial status • Parkinson's Disease

ASPERO: Phase 1/2a Study of ANPD001 in Parkinson Disease (clinicaltrials.gov) - P1 | N=9 | Enrolling by invitation | Sponsor: Aspen Neuroscience | Trial primary completion date: Oct 2025 ➔ Mar 2027

Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease

ASPIRO TRIAL – TWELVE-MONTH SAFETY, TOLERABILITY, AND EFFICACY OF INTRACRANIAL DELIVERY OF AUTOLOGOUS IPSC-DERIVED DOPAMINERGIC PRECURSORS (SASINEPROCEL) IN MODERATE TO ADVANCED PARKINSON'S DISEASE (ADPD 2026) - "Twelve-month data show that the surgical procedure and cell product were safe and well tolerated. MRI confirmed accurate cell placement. At one year, improvements were seen in Good ON time, MDS-UPRDRS III OFF scores, MDS-UPDRS II scores, and PDQ-39 scores across both dosing cohorts."

Clinical • First-in-human • Metastases • CNS Disorders • Hematological Disorders • Movement Disorders • Parkinson's Disease

Aspen Neuroscience Announces Positive 12‑Month Data from its ASPIRO Clinical Trial in a Late‑Breaking Oral Presentation at the AD/PD 2026 International Conference on Alzheimer's and Parkinson's Diseases (PRNewswire) - "The presentation represents data from the first eight treated patients – four of whom received a low dose and four of whom received a higher dose of the therapy. At twelve months, both dosing cohorts showed numerical improvements in function, physician- and patient-reported outcomes, and quality-of-life, including: mean Good ON time (+2.1 hours in low dose (LD), +2.4 hours in high dose (HD)), mean MDS-UPDRS III OFF scores (-15.5 points in LD, -13.5 points in HD), mean MDS-UPDRS II scores (-5.3 points in LD, -2.3 points in HD), and mean improvement in PDQ-39 scores (51.6% in LD, 28.5% in HD)...'look forward to advancing Sasineprocel to a Phase 3 study later this year.'"

Late-breaking abstract • New P3 trial • P1/2 data • Parkinson's Disease

Aspen Receives Adoption of Sasineprocel as the Official Nonproprietary Naming of ANPD001, its Investigational Autologous Cell Therapy for the Treatment of Parkinson's Disease (PRNewswire) - "Sasineprocel is being evaluated in Aspen's Phase 1/2a ASPIRO study for moderate to advanced Parkinson's disease."

Commercial • Trial status • Parkinson's Disease

Aspen Neuroscience Announces $115 Million Series C Financing to Accelerate Personalized Cell Therapy Programs (PRNewswire) - "This significant investment will fuel the continued clinical development of Aspen's lead program, ANPD001, for moderate to advanced Parkinson's disease....The Series C financing will be used to: Support ongoing clinical trials of ANPD001, Aspen's lead autologous cell therapy candidate for Parkinson's disease; Scale manufacturing capabilities to meet clinical and future commercial demand; Advance Aspen's pipeline of autologous iPSC-derived therapies for additional neurological indications."

Financing • Parkinson's Disease

Aspen Neuroscience Initiates Cohort 3 in ASPIRO Phase 1/2a Trial, Highlighting Commercial Formulation of ANPD001 and Its Transformative Potential for Parkinson’s Disease (PRNewswire) - "The Cohort 3 formulation has been deemed preclinically comparable to the formulation used in the first two Cohorts, ensuring continuity as we advance to this next phase. In earlier cohorts, positive six-month data reported at IAPRD demonstrated strong safety and tolerability, along with clinician- and patient-reported improvements, achieved without the need for immunosuppression."

P1/2 data • Trial status • Parkinson's Disease

Aspen Neuroscience to Present at the International Society for Stem Cell Research (ISSCR) 2025 Annual Meeting (PRNewswire) - "Aspen Co-founder and Senior Vice President, Research and Development, Andrés Bratt-Leal, PhD will deliver a presentation, Safety, tolerability, and efficacy of transplanted autologous iPSC-derived dopaminergic precursors in moderate to advanced Parkinson's Disease....Dr. Bratt-Leal's presentation will cover data for the first four patients of the ASPIRO Study*, a Phase 1/2a trial of the company's iPSC-derived autologous cell therapy (ANPD001)."

Clinical data • P1/2 data • Parkinson's Disease

Aspen Neuroscience Announces 6-Month ASPIRO Phase 1/2a Clinical Trial Results of Personalized Cell Therapy for Parkinson's Disease (PRNewswire) - P1/2a | N=9 | ASPIRO (NCT06344026) | Sponsor: Aspen Neuroscience | "Aspen Neuroscience has released 6-month data from the first three patients dosed in the ASPIRO Phase 1/2a trial of ANPD001, an investigational autologous dopaminergic neuronal precursor cell (DANPC) therapy being studied in patients with moderate to advanced Parkinson's Disease (PD)....No hemorrhages nor serious intra-operative or post-operative complications were observed. No graft-induced dyskinesia have been observed. The most common adverse events in the first three patients were incision pain and tongue swelling, the latter of which is related to the prone position during surgery....At six months there was an average improvement of 45% in the physician-reported MDS-Unified Parkinsons Disease Part III 'off' score compared to baseline, and an average improvement of 71% in patient-reported MDS-UPDRS Part II scores....Detailed Phase 1/2a trial data will be presented this week at IAPRD 2025."

P1/2 data • Parkinson's Disease

Aspen Neuroscience Announces Completion of First and Second Cohorts in ASPIRO Phase 1/2a Trial for Parkinson's Disease (PRNewswire) - "Aspen Neuroscience, Inc. has successfully completed dose escalation and the first two cohorts of patients in the ASPIRO study, an ANPD001 Phase 1/2a clinical trial in Parkinson's Disease (PD)....We are happy to announce that to date, ANPD001 and its delivery have been well tolerated, and no serious adverse events have been observed in the first two cohorts of patients in the ASPIRO study."

Trial status • Parkinson's Disease

Mytos Announces Manufacturing Collaboration with Aspen Neuroscience in Parkinson’s Disease (Businesswire) - "Mytos...has entered into a manufacturing collaboration with Aspen Neuroscience, Inc. to automate the manufacturing of autologous dopaminergic neuronal precursor cells (DANPCs) for ANPD001, Aspen’s investigational cell therapy program for Parkinson’s disease (PD)."

Commercial • CNS Disorders • Parkinson's Disease

Aspen Neuroscience Announces Dosing Completion of First Cohort in ASPIRO Phase 1/2a Trial for Parkinson's Disease (PRNewswire) - "Aspen Neuroscience announced today that it has successfully completed dosing of the first cohort of patients in the ASPIRO study. Surgical safety data from this cohort has been accepted for late breaking presentations this week at the MDS International Congress of Parkinson's Disease and Movement Disorders, taking place in Philadelphia September 27 - October 1...The Autologous-derived Study of a Parkinson's Investigational Regenerative therapy in an Open-label trial (ASPIRO) is a Phase 1/2a clinical trial to assess the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's disease (PD). The primary study endpoint is safety and tolerability of ANPD001....In addition to multiple scientific presentations, Aspen will participate as a Bronze Level sponsor, showcasing company highlights at booth #1003."

Late-breaking abstract • P1/2 data • Trial status • CNS Disorders • Parkinson's Disease

ClearPoint Neuro Congratulates Partner Aspen Neuroscience on Use of the ClearPoint® Navigation System for All Enrolled Patients in ASPIRO Clinical Trial (GlobeNewswire) - "ClearPoint Neuro, Inc...today congratulates its partner Aspen Neuroscience, Inc. on use of the ClearPoint Neuro Navigation System to transplant dopaminergic neuron precursor cells (DANPCs) for all enrolled patients with Parkinson’s Disease (PD) in its recently launched ASPIRO Phase 1/2a clinical trial."

Clinical • CNS Disorders • Parkinson's Disease

Aspen Neuroscience to Present at International Society for Cell & Gene Therapy (ISCT) Annual Meeting (PRNewswire) - "Aspen Neuroscience will present this week to a global audience of clinicians, regulators, researchers, technologists and industry partners at the 2024 annual meeting of the International Society for Cell & Gene Therapy (ISCT) in Vancouver, Canada, May 29-June 1...As part of the ISCT 2024 Translational Pathway Sessions, as well as the ISCT main program, Aspen's Chief Scientific Officer Xiaokui Zhang, Ph.D., and Chief Technology Officer Kim Raineri will discuss developments in autologous research and development, including for ANPD001...Daniel Fremgen, Director of Analytical Development, will present regarding Aspen analytical development strategy including machine learning genomic models and characterization of autologous iPSCs and dopaminergic neuron precursor cells."

Clinical • CNS Disorders • Parkinson's Disease

Aspen Neuroscience Receives CLIN2 Grant for ANPD001 from California Institute for Regenerative Medicine (CIRM) (PRNewswire) - "Aspen Neuroscience, Inc...has received a CLIN2 grant award of $8 million from the California Institute for Regenerative Medicine (CIRM), the world's largest institution dedicated to regenerative medicine, to support clinical research aimed at treating Parkinson's disease (PD). The grant, a first for an autologous therapeutic for degenerative neurological conditions, will help advance the development of ANPD001, an investigational iPSC-derived dopaminergic neuron replacement therapy....'Our Phase 1/2a study has completed enrollment, the first patient has been dosed and we will continue dosing patients this year.'"

Enrollment closed • Financing • Trial status • CNS Disorders • Parkinson's Disease

Aspen Neuroscience Announces First Patient Dosed in First-in-Human Phase 1/2a Clinical Trial of Autologous Neuronal Cell Replacement Therapy for Parkinson's Disease (PRNewswire) - "Aspen Neuroscience...announced that the first patient has been dosed in the ASPIRO trial, a Phase 1/2a open label clinical trial to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to severe Parkinson's disease (PD). Aspen Neuroscience is a private biotechnology company developing personalized regenerative therapies....The hospital is one of five clinical sites for the Phase 1/2a trial....'Our 2022 Trial Ready Cohort Screening Study has completed enrollment, and we plan to dose patients in the ASPIRO Phase 1/2a study this year'."

Trial status • Parkinson's Disease

ASPERO: Phase 1/2a Study of ANPD001 in Parkinson Disease (clinicaltrials.gov) - P1 | N=9 | Enrolling by invitation | Sponsor: Aspen Neuroscience

New P1 trial • CNS Disorders • Movement Disorders • Parkinson's Disease

Aspen Neuroscience to Present at Future of Parkinson's Disease Conference (PRNewswire) - "This week, Aspen Neuroscience will present to the Parkinson's community at the Future of Parkinson's Disease conference, held November 30 – December 3, in Austin. The joint annual conference is hosted by the Parkinson's Foundation and the Parkinson Study Group. In addition to multiple scientific presentations, Aspen Chief Executive Officer Damien McDevitt, PhD, and co-founder and Senior Vice President of R&D, Andres Bratt-Leal, PhD, will present updates on company progress for upcoming Phase 1 clinical trials for ANPD001, a personalized (autologous) cell therapy under investigation to treat PD by replacing lost dopamine neurons."

Clinical • CNS Disorders • Parkinson's Disease

FDA Grants Fast Track Designation to ANPD001, Autologous Investigational Cell Therapy for the Treatment of Parkinson's Disease (PRNewswire) - "Aspen Neuroscience today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for ANPD001 for the treatment of Parkinson's disease (PD) to improve motor function. ANPD001, is a personalized (autologous) cell therapy under investigation to treat PD by replacing lost dopamine neurons."

Fast track designation • CNS Disorders • Parkinson's Disease

Aspen Neuroscience to Present at 2023 Cell & Gene Meeting on the Mesa (PRNewswire) - "Aspen Neuroscience announced today that Chief Technology Officer Kim Raineri and Chief Scientific Officer Xiaokui Zhang, Ph.D. will present at the annual Cell & Gene Meeting on the Mesa, held this week in Carlsbad, Calif. and live-streamed globally. CTO Kim Raineri will present a corporate overview of Aspen Neuroscience, highlighting its work in the development of iPSC-derived cell therapies for the treatment of CNS disorders, beginning with Parkinson's Disease (PD)."

Preclinical • CNS Disorders • Parkinson's Disease

Aspen Neuroscience to Partner with Rune Labs and Emerald Innovations to Incorporate both Active and Passive Digital Health Monitoring in Trial Ready Cohort Screening Study (PRNewswire) - "Aspen Neuroscience today announced that it is partnering with Emerald Innovations, a pioneer in the creation of 'invisible' off-body sensors for measuring health analytics, and Rune Labs, a software and data analytics company for precision neurology, to incorporate digital health (DHT) modalities in the company's Trial-Ready Screening Cohort Study. The Trial Ready Screening Cohort study was launched in 2022 to screen, enroll and begin manufacturing cells for potential patient candidates for the future Phase 1/2a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's disease (PD) by replacing lost dopamine neurons."

Licensing / partnership • CNS Disorders • Parkinson's Disease

Aspen Neuroscience Announces FDA Clearance of Investigational New Drug Application for ANPD001, Autologous Cell Therapy for the Treatment of Parkinson's Disease (PRNewswire) - "Aspen Neuroscience today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application, enabling the company to proceed with a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's Disease by replacing lost dopamine neurons. Next Aspen plans to initiate a first-in-patient Phase 1/2a clinical trial for people with moderate to severe Parkinson's Disease (PD). This follows the company's 2022 Trial-Ready Screening Cohort Study to screen, enroll and begin manufacturing cells for potential patient candidates for the clinical trial. This study will be the first multicenter Phase 1/2a trial of an autologous iPSC-derived therapy in the U.S."

IND • New P1/2 trial • CNS Disorders • Parkinson's Disease

Preclinical Safety and Efficacy Data for ANPD001 Presented by Dr. Andres Bratt-Leal at 2023 World Parkinson Congress in Barcelona (PRNewswire) - "On Wednesday at the 2023 World Parkinson Congress in Barcelona, Aspen Neuroscience co-founder Andres Bratt-Leal, PhD...presented preclinical safety and efficacy data for the first time for ANPD001, Aspen's iPSC-derived lead candidate for treatment of Parkinson's Disease (PD)....The data suggest that after transplantation there is no evidence of migration or tumor formation, and that PD neurons function in vitro and restore dopamine signaling deficits in rodents. The results demonstrate the ability to manufacture patient specific DANPCs which are safe and efficacious in support of clinical investigation."

Preclinical • CNS Disorders • Parkinson's Disease