FDA Clears Aruna Bio’s Exosome AB126 for Clinical Trials in Neurological Indication


Aruna Bio's investigational treatment, AB126, is now set for a phase 1b/2a clinical trial targeting acute ischemic stroke, anticipated to begin in the first half of 2024.

Sean Savitz, MD, professor of neurology and the director of the Institute for Stroke and Cerebrovascular Disease at The University of Texas Health Science Center at Houston (UTHealth

Sean Savitz, MD

The FDA has cleared Aruna Bio’s investigational new drug (IND) application for the company’s lead candidate AB126, an unmodified neural-derived exosome, according to a new announcement.1 This clearance by the agency paves the way for investigating AB126 in a phase 1b/2a clinical trial among patients with acute ischemic stroke, which the company noted is expected to initiate in the first half of 2024.

AB126 has intuitive ability to cross the blood-brain barrier and modulate inflammasome in the central nervous system (CNS) by action of anti-inflammatory and neuroprotective mechanisms, giving it the potential to treat a range of neurodegenerative disorders, according to Aruna Bio. In addition, the company noted that its neural exosome platform can be combined with therapeutics, such as small molecules, small interfering RNAs, and proteins.

Top Clinical Takeaways

  • Aruna Bio's AB126, is set to enter a phase 1b/2a clinical trial, offering a potential novel approach for acute ischemic stroke treatment.
  • The neural-derived exosome, AB126, not only shows promise in treating acute conditions but also demonstrates potential for chronic neurological disorders, including ALS.
  • The FDA validation positions Aruna Bio's platform as a potential pioneering force in CNS therapies, emphasizing the significance of AB126's journey into human clinical trials.

“We look forward to building on the preclinical findings that showed AB126 may diminish neuroinflammation, and potentially foster neuroprotection, and promote neuroregeneration,” principal investigator Sean Savitz, MD, professor of neurology and the director of the Institute for Stroke and Cerebrovascular Disease at The University of Texas Health Science Center at Houston, said in a statement.1

READ MORE: FDA Approves Takeda’s Immune Globulin Infusion, Hyqvia, for Chronic Inflammatory Demyelinating Polyneuropathy

In the planned phase 1b/2a dose-ascending trial, investigators will assess the safety, tolerability, and preliminary efficacy of AB126 among patients with an acute ischemic stroke who had undergone a thrombectomy. Notably, only those with a poor prognosis post thrombectomy will be enrolled into the trial. The participants in the trial will receive 3 intravenous injection treatments of AB126 at low, medium, and high doses.

“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, cofounder and chief scientific officer at Aruna, 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, and exploring the broader potential of our neural exosome platform in overcoming existing challenges in CNS treatments.”

Previously, a pilot study on SOD1 amyotrophic lateral sclerosis (ALS) mouse model demonstrated that a weekly dose of AB126, starting at the onset of ALS symptoms and extending through the study end point, had prolonged survival and reduced inflammation in the spinal cord compared with vehicle.2 In addition, AB126-treated animals also showed reduced levels of neurofilament light chain levels in serum compared with vehicle-treated animals.

“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, Chief Executive Officer at Aruna Bio, said in a statement.2 “In the current pilot study, we observed prolonged survival in AB126 treated mice because of 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. Published January 16, 2024. Accessed January 16, 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. Published September 7, 2023. Accessed January 16, 2024. https://www.globenewswire.com/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
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