Aruna Bio’s AB126, an investigational unmodified exosome derived from proprietary neural stem cells, has received clearance of an investigational new drug (IND) application from the FDA for a phase 1b/2a clinical (NCT identifier pending) in acute ischemic stroke.1
Aruna Bio stated that it expects to begin the clinical trial within the first half of this year. The company also noted that no other exosome therapy has entered in-human clinical trials for a neurological indication. The planned clinical trial will recruit patients with acute ischemic stroke who have a poor prognosis after having received a thrombectomy. AB126 will be administered to patients via 3 intravenous injections at a low, medium, and high dose.
Aruna Bio stated that AB126 is capable of crossing through the blood brain barrier and that it has capacity to modulate the inflammasome in the central nervous system (CNS) via mechanisms of antiinflammation and neuroprotection.1,2 Aruna Bio intends to make use of its in-house good manufacturing practice (GMP) manufacturing facility, unique purification techniques, protocols for analytical methods, and assays with the goal of ensuring batches of AB126 are produced under conditions for optimal and scalable consistency, with controlled critical quality attributes and therapeutic consistency.
“We are thrilled with this validation from the FDA, which not only positions AB126 as the first exosome to enter human clinical trials for a neurological indication, but underscores the therapeutic feasibility of our platform,” Steven Stice, PhD, the cofounder and chief scientific officer of Aruna Bio, said in a statement.1 “Further, maintaining cells under controlled conditions is a key aspect of exosome manufacturing and we look forward to leveraging our in-house GMP manufacturing expertise to support clinical advancement. Simultaneously, we are expanding AB126’s applications across other indications, including amyotrophic lateral sclerosis (ALS), and exploring the broader potential of our neural exosome platform in overcoming existing challenges in CNS treatments.”
- Aruna Bio's AB126, an investigational exosome derived from neural stem cells, has received FDA clearance for a phase 1b/2a clinical trial in acute ischemic stroke.
- The clinical trial will recruit patients with acute ischemic stroke who have a poor prognosis after thrombectomy.
- Aruna Bio is exploring other neurological applications for AB126 beyond stroke, including amyotrophic lateral sclerosis (ALS).
Proof-of-concept data regarding AB126’s potential in multiple different neurological indications was previously presented at the Exosome Based Therapeutic Development Summit, held in Boston, MA, on October 4-6, 2022.3 Stice commented on the data in an October 2022 statement:
“We are encouraged by our growing dataset demonstrating the therapeutic potential of AB126 to overcome existing challenges of CNS treatments, as well as the opportunity to accelerate the development of specialized exosomes through our proprietary neural stem cell line. Our proof-of-concept data in preclinical models further validate the ability of AB126 in its native form to reduce neuro-inflammation, provide neuroprotection, and stimulate neuro-regeneration. We are on track to file our first IND in Q2 2023 for stroke as a model indication for acute neurological conditions and will continue to explore the applications of AB126 across a potentially wide range of inflammatory and degenerative diseases of the brain.”3
More recently, in September 2023, announced results from a mouse model pilot study assessing AB126 for the treatment of superoxide dismutase 1 (SOD1) ALS.2 Compared to SOD1 ALS mice who were treated with a vehicle, the SOD1 ALS mice who were treated with a weekly dose of AB126 beginning at the onset of disease symptoms showed prolonged survival, decreased inflammation, and decreased levels of neurofilament light chain levels in serum.
“We are encouraged by our growing dataset demonstrating the potential of AB126 in its native form to address both acute and chronic neurological indications, including ALS, a progressive neurodegenerative disease with an average survival of 3 to 5 years,” Stephen From, BS, the chief executive officer of Aruna Bio, said in a September 2023 statement.2 “In the current pilot study, we observed prolonged survival in AB126 treated mice due to significantly reduced inflammatory mediators in the spinal cord, as well as significant reductions in serum neurofilament light chain, an important biomarker of neurodegeneration. We look forward to building upon this pilot data and further exploring the therapeutic potential of AB126 for the treatment of ALS and expanding our AB126 platform to chronic CNS indications.”
1. Aruna Bio announces FDA clearance of IND for lead program AB126, enabling the first exosome to enter in human clinical trials for a neurological indication. News release. Aruna Bio, Inc. January 16, 2024. Accessed January 23, 2024. https://www.globenewswire.com/en/news-release/2024/01/16/2809846/0/en/Aruna-Bio-Announces-FDA-Clearance-of-IND-for-Lead-Program-AB126-Enabling-the-First-Exosome-to-Enter-in-Human-Clinical-Trials-for-a-Neurological-Indication.html
2. Aruna Bio announces preclinical results from pilot study evaluating AB126 for the treatment of amyotrophic lateral sclerosis. News release. Aruna Bio, Inc. September 7, 2023. Accessed January 23, 2024. https://www.globenewswire.com/en/news-release/2023/09/07/2739775/0/en/Aruna-Bio-Announces-Preclinical-Results-from-Pilot-Study-Evaluating-AB126-for-the-Treatment-of-Amyotrophic-Lateral-Sclerosis.html
3. Aruna Bio announces presentation on neural exosome treatment opportunities and lead program, AB126 at exosome based therapeutic development summit. News release. Aruna Bio, Inc. October 6, 2022. Accessed January 23, 2024. https://www.globenewswire.com/news-release/2022/10/06/2529435/0/en/Aruna-Bio-Announces-Presentation-on-Neural-Exosome-Treatment-Opportunities-and-Lead-Program-AB126-at-Exosome-Based-Therapeutic-Development-Summit.html