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Seelos Therapeutics – FDA Acceptance of IND for SLS-005 for Sanfilippo Syndrome a shot in the arm for the Company

31 Aug Seelos Therapeutics – FDA Acceptance of IND for SLS-005 for Sanfilippo Syndrome a shot in the arm for the Company

Seelos Therapeutics (NASDAQ: SEEL), a clinical-stage biotechnology Company, announced the acceptance of its Investigation New Drug (IND) application for SLS-005 (trehalose) by the FDA. The company is planning to initiate a combined Phase IIb/III, multicenter study to assess the safety, tolerability and efficacy of trehalose IV in Sanfilippo A and B and will evaluate the results based on functional outcomes, biomarkers, neuro-cognitive assessments and quality of life measurements. The company has received the FDA’s approval for initiating its clinical trials.

Mr. Raj Mehra Ph.D., Chairman and CEO of Seelos Therapeutics opines

“We are grateful for the detailed guidance from the regulatory agency (FDA) and thank our financial supporter and collaborator, TSF, which has brought a singular focus to helping the Sanfilippo community.”

Trehalose is a low molecular weight disaccharide (.342 kDa), which is capable of crossing the blood brain barrier, stabilising proteins and activating autophagy, through the activation of Transcription Factor EB (TFEB), a cells internal clearance mechanism. In preclinical studies, the compound has demonstrated the ability to reduce aggregation of misfolded proteins and lower the accumulation of pathologic material as per the company.

Trehalose has two U.S patents for parental administration for patients with Oculopharyngeal Muscular Dystrophy (OPMD) and Spinocerebellar AtaxiaType 3 (SCA3)3 SCA3.

The company plans to extend the trial, and provide expanded patient access to those suffering from Sanfilippo type C and D, as well as type A and B who do not qualify for the proposed trial.

In addition, Seelos has also entered into a securities purchase agreement, for the purchase and sale of 4,475,000 shares of common stock at a price of $1.50 per share, for total gross proceeds of approximately $6.7 million. The company will also issue unregistered warrants to purchase up to 2,237,500 shares of common stock in a concurrent private placement, at a price of $1.78 per share of common stock, to be exercisable six months from the date of issuance and will expire four years following the date of issuance as per a company release.

The proceeds from the offering are expected to be utilised for the advancement of its clinical pipeline and for general corporate purposes.

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Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction in any of these genes hinders the breakdown of heparan sulfate, leading to its accumulation inside the lysosomes.

This disease primarily affects the brain and the central nervous system and is manifested in early childhood in the form of behavioural problems, sleep disturbances, developmental regression, motor disability and seizures. Those affected with MPS III exhibit macrocephaly, umbilical or inguinal hernia and skeletal abnormalities.

According to the NIH, the estimated incidence of all forms of mucopolysaccharidosis is 1 in 70000 new borns. The disease is inherited through an autosomal recessive pattern and can be diagnosed through a urine test and measurement of enzyme activity through blood and skin samples. In addition, genetic screening can provide information as to which gene is malfunctioning.

The present treatment option for this disease is aimed at alleviating symptoms and is multidisciplinary in nature. However, there are companies that are engaged in extensive research and development activities in trying to create novel therapeutics for this disease. Gene Therapy, Enzyme replacement therapy, stem cell and substrate reduction therapy are some of the emerging fields, which are offering hope to both the patients and caregivers. 

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The Company
Seelos acquired Trehalose, an investigational molecule for treatment of two rare diseases, from Bioblast Pharma, in February 2019. The deal was for a total consideration of $3.5 million, to be payable in two installments. Seelos will pay $1.5 million upon closing of the sale and the other $2.0 million will be paid upon the first anniversary of the closing of the sale. SLS-005, its lead candidate for the Sanfilippo syndrome, is being developed in collaboration with the Team Sanfilippo Foundation (TSE).

Team Sanfilippo Foundation (TSE) is sponsoring an open label, 2b clinical trial in up to 20 patients, for assessing the safety and efficacy of SLS-005 (Trehalose). This would be in addition to prior phase 2 clinical studies in over 70 patients, which demonstrated positive safety profile and promising efficacy signal.

The company has recently acquired the license to the Intellectual Property Rights (IPR) of a peptide-inhibitor technology that prevents the aggregation of alpha-synuclein (α-synuclein) created by researchers at the University of California, LA (UCLA), which it intends to evaluate for its efficacy in treating Parkinson’s and other CNS disorders. SL-007 is a peptide-based program that targets NACore (nonamyloid component core), and has shown to slow down the propagation and seeding of α-synuclein aggregates in patients diagnosed with Parkinson’s disease or Lewy Body Dementia.

The company is also developing SLS-006, a small molecule partial dopamine agonist for the treatment of early stage and late stage Parkinson’s as monotherapy and adjunctive therapy, which is currently undergoing phase II trials. This is intended for use in conjunction with low dosage of L-Dopa. It will soon meet with the FDA and EMA to discuss its plans for the registrations for its phase III clinical trials that start in 2019.

In the pipeline is SLS-002, for treatment of Suicidality in PTSD and MDD and SLS-008 in pediatric esophagitis and other undisclosed indications.

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The company has a multi-program partnership with Ligand Pharmaceuticals (NASDAQ: LGND) for worldwide rights to develop and commercialize the SLS-006, SLS-008, SLS-010 and SLS-012 programs from Ligand. It is a shareholder in Seelos pursuant to a cash investment and will further receive an equity milestone payment in addition to receiving potential future milestone payments and royalties for licensed programs.

In January 2019, the company completed a previously announced with merger with Apricus Biosciences, Inc., the combined entities will henceforth be known as Seelos Therapeutics, Inc.

Risk Assessment

    • The company has a diverse pipeline of candidates, which helps mitigate its risk and make optimal use of its resources. Seelos has two programs that have completed phase II and two more that have finished phase I clinical trial stage. The company is set to launch a pharmacokinetic/pharmacodynamic modeling for PTSD and MDD in Q3 2019. while initiating dosing for patients in its Sanfillipo study. Advancing multiple products simultaneously offers economies of scale.


    • Strategic collaborations which focus on late stage drug candidates, such as the acquisition of Trehalose from Bioplast, helps the company save time and effort in establishing mechanism of action in preclinical studies.


    • The company’s Sanfillipo trial is being funded by a grant from the Team Sanfilippo Foundation (TSE). In addition, the company is planning to raise over $6.7 million through a registered direct offering. These funds will help offer stability to run its operations and advance its clinical pipeline.


    • The company is building a robust portfolio of intellectual property rights covering a broad spectrum of its drug candidates, which will afford it protection and stave away competition. Most recently the company was given a notice of allowance from the USPTO for SLS-005 (trehalose) for treating Friedreich Ataxia (FA).


    • Seelos is on an acquisition spree to amass synergistic assets, in the Central Nervous system therapy space through licensing and partnership deals, especially with focus on strategic collaborations in the European and Asian markets, in order to expand its reach and overcome regulatory hurdles.



We believe that Seelos, given its focus on diverse yet relevant indications in the Central Nervous Disorder space, especially diseases with a large unmet need is on an accelerated path to success. The company has the ability to leverage its technology across a broad spectrum of indications, and find unique and effective drug targets for development, which augurs well for its future growth.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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