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FDA Fast Tracks Parkinson’s Treatment Application

BIOTECH: Aspen Neuro Initiates Clinical Trial

Local biotech company Aspen Neuroscience announced that the U.S. Food and Drug Administration (FDA) has granted clearance of an investigational cell therapy drug application for the treatment of Parkinson’s disease (PD) to treat motor function. The FDA’s Fast Track Designation speeds up the review process for ANPD001, a personalized (autologous) option that works by replacing lost dopamine neurons.

Dr. Damien McDevitt, PhD
President & CEO
Aspen Neuroscience

“We are pleased that the FDA has granted Fast Track Designation, underscoring the potential of an autologous dopamine replacement therapy such as ANPD001 to serve as a meaningful treatment option,” said Aspen Neuroscience CEO and President Dr. Damien McDevitt, PhD. “People with Parkinson’s disease have a significant unmet medical need and have been waiting for many years for more advanced treatment options.”

Tremors, Slow Movements, Limb Stiffness

PD is a progressive disorder that affects the nervous system and the parts of the body controlled by the nerves. Nearly 1 million people in the U.S. are living with PD, and that number is expected to hit 1.2M by 2030, according to the Parkinson’s Foundation. It is the second most common neurodegenerative disease after Alzheimer’s disease. Symptoms typically include tremors, slow movements, limb stiffness and problems with balance and gait. The cause remains largely unknown, although genetics are believed to be a factor in about 10% to 15% of cases. There is currently no cure.

Angelyn Lowe
Senior VP of Corporate Affairs
Aspen Neuroscience

With its headquarters in Torrey Pines, Aspen Neuroscience is currently VC-funded and reports that it has raised almost $225 million to date.

“When I started in 2021, we had 27 employees,” Aspen Neuroscience Senior VP of Corporate Affairs’ Angelyn Lowe said. “Now, we have more than 100 employees.”

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Aspen recently expanded into a second building in Torrey Pines to accommodate its increased manufacturing needs.

The company was founded in 2018 by a group of research scientists from Dr. Jeanne Loring’s lab at The Scripps Research Institute, and a group of patient advocates from the Summit for Stem Cell Foundation, who believe in the potential benefits of cell replacement for PD.

Using a Patient’s Own Stem Cells

Aspen collects a patient’s own stem cells by means of biopsy to stimulate neuron replacement for both sporadic and genetic forms of PD.

“We are using the patient’s own iPSCs (so-called induced ‘pluripotent’ stem cells, meaning they have the capacity to develop into several different cell types) to create dopamine neurons. This is a cell replacement therapy. We are planning to give the patients their cells back through a neurosurgical procedure,” Lowe said. “We use AI tools in our steps –to create the iPSCs and select the best iPSC clone to create dopamine neurons and select the best neurons before they are transplanted.”

Aspen plans to initiate a first in-patient Phase 1/2a clinical trial of ANPD001 for participants with moderate to severe PD.

“The trial is a small, open label study with nine patients. It’s aimed primarily at studying safety,” she added.

The company plans to start dosing patients in the first half of 2024. There is no timeline for when the study will be completed, although each phase typically takes a few years.

“We’re hoping that our phases will be faster than that, but that is a pretty average timeline,” Lowe said. Following this phase, researchers will initiate a second study where they’ll look for signs of efficacy in several more patients.

“In the next five years, we’ll hopefully have very strong signals of how the therapeutic is working in PD patients. Hopefully, we’ll also be exploring other cell types as other potential therapeutics in other parts of the central nervous system,” she added.

Aspen Neuroscience

FOUNDED: 2018
PRESIDENT & CEO: Dr. Damien McDevitt, PhD
HEADQUARTERS: Torrey Pines
EMPLOYEES: 100+
BUSINESS: biotech
FUNDING: $217.5 million (Series A and B)
WEBSITE: aspennueroscience.com
NOTABLE: The company was founded by a group of research scientists from Dr. Jeanne Loring’s lab at The Scripps Research Institute and a group of patient advocates from Summit for Stem Cell.

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