Seelos Therapeutics Announces FDA Acceptance of Investigational New Drug Application and Grant of Fast Track Designation for SLS-005 (IV Trehalose) for the Treatment of Spinocerebellar Ataxia

-Seelos Therapeutics is an Initial Member of the National Ataxia Foundation Drug Development Collaborative

-Global Phase IIb/III Study for SLS-005 to be Initiated in Early 2022

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Nov 08, 2021, 08:00 ET

NEW YORK, Nov. 8, 2021 /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, announced today that the U.S. Food and Drug Administration (FDA) has accepted Seelos' Investigation New Drug (IND) application to study SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of spinocerebellar ataxia (SCA). The FDA has also granted the program Fast Track designation in the U.S. for SCA, and SLS-005 has previously received Orphan Drug designation for spinocerebellar ataxia type 3 (SCA3) from the FDA and from the European Medicines Agency in the EU.

"SCA is a highly debilitating neurodegenerative disease that currently lacks a cure or an approved therapeutic and as such, patients manage symptoms through physical therapy and other symptomatic treatments," said Raj Mehra, Ph.D., Chairman and CEO of Seelos. "SLS-005 has already displayed encouraging open label human data in SCA3, the most common type of SCA, and our team has taken that experience and knowledge into the design and plans for our global Phase IIb/III placebo-controlled study. We look forward to initiating this study in early 2022 and our recent capital raises have accounted for the expected development costs for initiating this study."

Prior to Seelos acquiring the program, SLS-005 had already been studied in a six-month open label Phase IIa study that also included an additional six-month follow up in patients with SCA3, also known as Machado-Joseph Disease (Zaltzman 2020). The open label study evaluated 14 patients with SCA3 over a six-month period and found the average score on the Scale for Assessment and Rating of Ataxia (SARA), a well-recognized clinical tool for measuring functional impairment associated with the disease, remained stable. Six patients received treatment for as long as 12 months and continued to maintain stable SARA scores. In comparison, natural history data suggests that individuals with SCA3 would be expected to show a measurable increase on SARA within a 12-month period, which is indicative of disease progression and worsening of symptoms.

Additionally, Seelos was named as one of the initial members of the National Ataxia Foundation (NAF) Drug Development Collaborative, an industry consortium that has the principal goal of accelerating the development of treatments for Ataxia.

"On behalf of the National Ataxia Foundation and the ataxia community we represent, we're thrilled that Seelos Therapeutics has received both IND acceptance and Fast Track designation from the FDA," said Andrew Rosen, NAF Executive Director. "Seelos is a founding member of the NAF Drug Development Collaborative and NAF looks forward to working closely with Seelos to support the company as its program progresses."

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.

About the National Ataxia Foundation (NAF) and the NAF Drug Development Collaborative

NAF is a nonprofit organization established in 1957 to help persons with ataxia and their families. The Foundation's mission is to accelerate the development of treatments and a cure while working to improve the lives of those affected by ataxia. NAF is the only organization in the United States dedicated to the disease that serves all types of ataxias. NAF works closely with the world's leading ataxia researchers, promoting exchanges of ideas and innovation in ataxia discovery.

The NAF Drug Development Collaborative provides a centralized source for access to resources needed to support research and development of ataxia therapies. Members will benefit from the integration of the patient experience with sound ataxia scientific and clinical expertise. NAF brings more than 60 years of experience in supporting patients and caregivers and connecting them with research and clinical trial opportunities. NAF has also funded a network of ataxia clinicians at sites around the US that will be a critical component of the Collaborative's work. Specific objectives of the Collaborative include natural history and biosample data collection, development of biomarkers, validation of rating scales, clinical trial design, patient-reported outcomes, and other data necessary for the development and approval of safe and effective therapies.

For more information on NAF: https://www.ataxia.org/

About Trehalose

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy, which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates 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.

About Seelos Therapeutics

Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system (CNS) disorders and other rare diseases. The Company's robust portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder (MDD) or Post-Traumatic Stress Disorder (PTSD), Amyotrophic lateral sclerosis (ALS), Spinocerebellar ataxia (SCA), Sanfilippo syndrome, Parkinson's disease, other psychiatric and movement disorders plus orphan diseases.

For more information, please visit our website: http://seelostherapeutics.com, the content of which is not incorporated herein by reference.

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 the design and plans for the global Phase IIB/III placebo-controlled study of SLS-005 in SCA (the "Study"), the expected timeline for commencing the Study and Seelos' available capital and expected development costs to initiate the Study. 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, or initiating the Study, and not gaining marketing approvals for its product candidates, the risk that prior clinical results may not be replicated in future studies and trials (including the risk that the results from the prior studies of SLS-005 may not be replicated or may be materially different from the results of the Study or other future trials and studies of SLS-005), 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.

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Zaltzman R, Elyoseph Z, Lev N, Gordon CR. Trehalose in Machado-Joseph Disease: Safety, Tolerability, and Efficacy. Cerebellum. 2020 Oct; 19(5):672-679. doi: 10.1007/s12311-020-01150-6. PMID: 32514820.

Contact Information
Anthony Marciano
Chief Communications Officer
Seelos Therapeutics, Inc. (Nasdaq: SEEL)
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(646) 293-2136
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www.seelostherapeutics.com
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