Seelos Therapeutics Doses First Patient in a Registrational Phase II/III study of SLS-005 in Spinocerebellar Ataxia
Seelos Therapeutics (Nasdaq: SEEL) has initiated dosing the first patient in a registrational phase II/III study of SLS-005 (trehalose injection) targeting spinocerebellar ataxia type 3 (SCA3). This trial aims to enroll up to 245 participants globally, assessing the treatment's efficacy over a 52-week period. Seelos will present findings at the International Congress for Ataxia Research in Dallas, Texas, in November. SLS-005 has received Orphan Drug Designation from the FDA and EMA, representing a potential breakthrough for a condition lacking approved treatments.
- Initiated first patient dosing in a phase II/III study for SLS-005.
- Phase II/III trial to enroll up to 245 participants, indicating strong study design.
- SLS-005 received Orphan Drug Designation from FDA and EMA, enhancing development prospects.
- None
- Seelos will also present at the International Congress for Ataxia Research in November
NEW YORK, Oct. 25, 2022 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced dosing of the first patient in a registrational phase II/III study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of spinocerebellar ataxia focusing on participants with type3 (SCA3, also known as Machado-Joseph disease) and also announced that it will participate in the International Congress for Ataxia Research (ICAR) in Dallas, Texas November 1st – 4th, 2022.
Dr. David Biondi, DO, FAAN, from Seelos will present: Autophagy as a potential treatment pathway in Spinocerebellar Ataxia: SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) at ICAR on Thursday, November 3rd at 4:45pm CT. For more information, visit https://ataxiacongress.org/.
"We are delighted to take part in this potentially ground-breaking and innovative method to treat SCA3, a devastating disease with no FDA-approved treatment or cure", said Dr. Theresa Zesiewicz of the University of Southern Florida.
Seelos' Phase II/III trial (NCT05490563) plans to enroll up to 245 participants globally with spinocerebellar ataxia type-3 in a double-blind, randomized, placebo-controlled trial. Eligible participants will be randomized to treatment with SLS-005 or placebo in 1 of 2 dosage arms and assessed with a primary efficacy endpoint measuring change from baseline in the Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52. Secondary endpoints include change from baseline in a blood-based biomarker for neurodegeneration, clinical global impression of severity, patient global impression of severity and an activities of daily living score. Safety and tolerability of SLS-005 will be monitored and assessed throughout the trial.
About SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion)
SLS-005 is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, SLS-005 has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. SLS-005 has previously received Orphan Drug Designation for the treatment of SCA type 3 from the U.S. Food and Drug Administration and from the European Medicines Agency in the EU. SLS-005 is an investigational treatment and is not currently approved by any health authority for medicinal use.
About Spinocerebellar Ataxia
Spinocerebellar Ataxia is a serious disease caused by degeneration of the cerebellum with an onset usually in adult life. Clinically, it is characterized by progressive unsteadiness of gait and stance, impaired coordination of limb movements, slurred speech, and abnormal eye movements. Spinocerebellar ataxia type-3, also known as Machado-Joseph disease (MJD), is characterized by progressive cerebellar ataxia and is known to cause progressively severe disability and often premature death approximately 10-20 years from onset of symptoms.
Forward Looking Statements
Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding Seelos' registrational phase II/III study of SLS-005 for the treatment of spinocerebellar ataxia, including SLS-005's prospects and potential insights from the registrational phase II/III study, as well as statements regarding the anticipated enrollment and timing of the study and the potential for SLS-005 to be a ground-breaking and innovative method to treat SCA3. These statements are based on Seelos' current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business and plans described herein include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies, and not gaining marketing approvals for its product candidates, the risk that prior clinical results may not be replicated in future studies and trials, the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval, the risks associated with the implementation of a new business strategy, the risks related to raising capital to fund its development plans and ongoing operations, risks related to Seelos' current stock price, risks related to the global impact of COVID-19, as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.
Contact Information:
Anthony Marciano
Chief Communications Officer
Seelos Therapeutics, Inc. (Nasdaq: SEEL)
300 Park Ave., 2nd Floor
New York, NY 10022
(646) 293-2136
anthony.marciano@seelostx.com
https://seelostherapeutics.com/
https://twitter.com/seelostx
https://www.linkedin.com/company/seelos
Mike Moyer
Managing Director
LifeSci Advisors, LLC
250 West 55th St., Suite 3401
New York, NY 10019
(617) 308-4306
mmoyer@lifesciadvisors.com
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FAQ
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