Aspen Neuroscience Receives CLIN2 Grant for ANPD001 from California Institute for Regenerative Medicine (CIRM)

Award Will Support the First U.S. Multi-center, Multi-patient Phase 1/2a Trial of an Autologous Neurological Therapy 

SAN DIEGO, May 15, 2024 /PRNewswire/ -- Aspen Neuroscience, Inc., a California-based private biotechnology company developing personalized regenerative therapies, 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.

ANPD001 is being studied in a First in Human Phase 1/2a clinical trial for patients with moderate to advanced PD, to assess safety and tolerability.  This is the first use of the autologous approach in a multi-patient and multi-center clinical trial.

"This clinical award represents a significant step forward in the treatment landscape of Parkinson's disease by advancing individualized therapy, which has the potential to restore motor function in patients impacted by this devastating condition," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. 

"We are honored to receive support from CIRM, an incredible sponsor of innovation.  ANPD001 was developed in California, and is now being produced and studied here," said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience, Inc. "Providing patients in this study with dopamine neurons made from their own cells is a huge leap forward for personalized medicine, and has the potential to impact the entire field of neurodegenerative disorders."

Affecting more than one million Americans, PD is a neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the underlying disease process, leading to a significant unmet medical need for those suffering from this chronic condition.

"Parkinson's disease is the most common neurodegenerative movement disorder, primarily by depleting dopamine neurons in the midbrain.  By the time of diagnosis, it is common for people with Parkinson's to have lost the majority of dopaminergic (DA) neurons, leading to progressive loss of motor and neurological function," explained Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience. "Our Phase 1/2a study has completed enrollment, the first patient has been dosed and we will continue dosing patients this year."

About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.

To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.  For more information go to www.cirm.ca.gov

About the ASPIRO Trial
ASPIRO is an open-label Phase 1/2a clinical trial to assess the safety and tolerability of ANPD001 in patients with moderate to severe Parkinson's disease. The trial includes patients 50–70 years of age, and excludes patients with cognitive impairment and other comorbidities that could preclude treatment. All enrolled patients are under the care of a movement disorder specialist.

The primary study endpoint is safety and tolerability of two sequential escalating doses of ANPD001.  Secondary endpoints include improvement in "on" time, when patients experience periods of good symptom control, and improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales.  

About Aspen Neuroscience 
Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease. 

Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control. For more information and important updates, please visit https://www.aspenneuroscience.com.

SOURCE Aspen Neuroscience, Inc.