Seelos Therapeutics Inc (NASDAQ:SEEL) stock extended gains in premarket trade Friday after the biotech said it has exclusively in-licensed the rights to a potential disease modifying Parkinson’s disease therapy created by researchers at the University of California, Los Angeles (UCLA).
Parkinson’s disease is a progressive nervous system disorder that affects movement, and symptoms include tremors and changes in speech and gait.
The news was met with enthusiasm by investors and as a result, Seelos stock nearly doubled Thursday. Shares in the company were rising again, up 61.1% to $4.90 before the opening bell Friday.
READ: Seelos Therapeutics stock doubles on license of potential Parkinson's candidate created at UCLA
The New York-based biotech has exclusively in-licensed the rights to a family of peptide inhibitors that target the aggregation of alpha-synuclein (α-synuclein).
The function of α-synuclein proteins in the healthy brain is unknown, but Parkinson's researchers are interested in it because it is a major constituent of Lewy bodies, protein clumps that are the pathological hallmark of the neurological disorder.
The company has designed the new program as SLS-007, with an initial focus on Parkinson's disease.
Pre-clinical data provides supportive evidence to slow progression, an early sign of disease-modifying potential in Parkinson’s disease.
Seelos will evaluate the potential for in-vivo delivery of SLS-007 in a Parkinson’s disease transgenic mice model.
“Alpha-synuclein is a normal protein that all of us have,” Seelos Therapeutics CEO Raj Mehra told Proactive Investor’s Christine Corrado. “But it turns out that in Parkinson’s patients the accumulation and aggregation of alpha-synuclein is always present and that has been identified as a key target.”
Pivotal role in Parkinson's
Though its role is not completely understood, researchers say alpha-synuclein appears pivotal in the pathogenesis of Parkinson’s and other alpha-synucleinopathies such as dementia. Researchers have been working to definitively characterize the protein's role in Parkinson's and its potential as a target for neuroprotective therapies.
“It is a top priority for researchers to focus on αlpha-synuclein aggregates. If you look at Big Pharma — Roche Holdings AG (OTCMKTS:RHHBY) and Biogen Inc (NASDAQ:BIIB) are focused on creating antibodies for alpha-synuclein aggregates,” said Mehra. “Now these aggregates are psychotoxic, but the antibody approach from Big Pharma is focused on the aggregates outside the cell. However, these aggregates are seeded and formed within the cell, so our focus was to find a smaller molecule approach that works inside the cell for these αlpha-synuclein aggregates as well as the outside.”
Mehra said the “entire goal” for Seelos and Big Pharma was the same to the extent that it focused on reducing the levels of pathological forms of αlpha-synuclein in the hopes that it may slow the progression of Parkinson’s.
“It is a huge unmet need that’s why we are focused on it,” said Mehra.
Tim Whitaker, who is the head of R&D at Seelos, said that the current available treatments for Parkinson’s including dopamine-promoter Levodopa and deep-brain stimulation have poor long-term outcomes for patients.
"With no disease-modifying treatments, and long-term use of established dopaminergic therapies resulting in both adverse events and side effects, significant need remains to develop not only a better means of restoring striatal dopamine but a safe and effective treatment that slows progression of the disease in patients with Parkinson’s,” said Whitaker.
Under the terms of the license, Seelos paid the UC Regents and UCLA $100,000 up front and will pay royalties on net sales.
Seelos Therapeutics works on novel therapeutics for patients with central nervous system disorders and has a diversified pipeline of late-stage clinical assets in areas of high unmet need targeting psychiatric and movement disorders, including orphan diseases.
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Contact Uttara Choudhury at [email protected]