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Spinogenix is developing a new class of therapeutics to restore memory and motor functions lost in neurodegenerative and psychiatric diseases.

Who we are

In most neurodegenerative diseases we lose connections - synapses – between neurons, a process that contributes to declines in cognition and motor function that characterize these diseases. At Spinogenix we are developing first-in-class, novel small molecule drugs (SPGs) that restore synapses to improve and potentially restore cognitive and motor function. Our approach is unique for diseases of the central nervous system.

Mission

Our mission is to create transformative therapeutics for diseases involving synaptic loss and dysfunction. Our approach is focused on regenerating synapses to reverse declines in cognitive and motor function. This approach is unique among the many therapeutics that are being developed for neurodegenerative conditions, which mostly aim to slow the degenerative process. We envision the use of SPGs as a monotherapy or in combination with other therapeutics that target disease-specific degenerative processes.

Our Science

In contrast to much research focused on alleviating the symptoms of neurodegenerative disease, we seek to develop therapies that impact the underlying disease and fundamentally change treatment paradigms by restoring neuronal connections regardless of the underlying cause of synapse loss. Spinogenix has discovered compounds (SPGs) that restore lost synapses. We have demonstrated the effectiveness of these “spinogenic” molecules in cells and in multiple animal models of neurodegeneration. SPGs have a novel molecular target and their mechanism of action is well understood and unique. This mechanism would be operative in all diseases where synapse loss occurs, irrespective of the pathological basis of synapse loss.


Dendritic spine from an AD mouse model showing the effect of treatment with one of our molecules

Clinical Lead

Our lead compound is a small, orally bioavailable, brain penetrant molecule which works at nanomolar concentrations and has a wide therapeutic window. The lead compound has shown efficacy in multiple animal models as well as in human patient-derived iPSC cell lines.

Lead Disease Indication

Our initial indication is ALS (a.k.a. Lou Gehrig’s disease), a devastating and almost always fatal neurodegenerative condition. ALS primarily affects the motor system, but it can also lead to cognitive decline in up to 50% of patients. Recent data indicate that a loss of dendritic spine synapses is an early and progressive feature of ALS pathogenesis that may contribute to both motor and cognitive symptoms. Current therapies are not disease modifying and afford only a modest extension of lifespan, with no reversal of symptom progression. We are planning to evaluate our lead compound for the treatment of motor and cognitive function of ALS, an FDA designated orphan disease.

Platform

Accumulating evidence suggests that deficits in dendritic spine synapses are associated with variety of neurological and psychiatric diseases, such as Alzheimer’s disease, Parkinson’s disease, ALS, schizophrenia, and depression. Therefore, promoting dendritic spine genesis may represent a promising therapeutic strategy for the treatment of multiple diseases:

Franz Hefti, Ph.D. - Chairman of the Board

Dr. Hefti brings more than 15 years of experience in the biotech industry as an executive, board member, co-founder and scientific advisor of many early-stage neurology companies. He is currently the Chief Development Officer of Prevail and was previously Chief Operations Officer at Proclara Biosciences, President and Chief Executive Officer of Acumen Pharmaceuticals, Chief Scientific Officer at Avid Radiopharmaceuticals (acquired by Eli Lilly and Company), and was Executive Vice President of Drug Development at Rinat Neuroscience (acquired by Pfizer).

Earlier in his career, Franz was Senior Vice President of Neuroscience Research at Merck & Co. and Head of Neuroscience Research at Genentech. He also held several positions in academia as a Professor at the University of Southern California and Associate Professor at the University of Miami, where he carried out discovery research on therapeutic approaches to neurodegenerative diseases. He has published more than 250 papers on neurotrophic factors and topics in neuropharmacology. He has been a member of the Scientific Advisory Board of the Alzheimer’s Disease Imaging Initiative since 2013.

Franz holds a Ph.D. from the University of Zurich and completed his postdoctoral research at the Massachusetts Institute of Technology.

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Stella Sarraf, Ph.D. – Founding Chief Executive Officer

Dr. Sarraf founded Spinogenix, Inc. in 2016 after seeing a clinical need for novel drugs to treat neurodegenerative diseases. She is viewed as a visionary with a deep understanding of product life cycle and the necessary components to achieve success in product commercialization. Previously, she founded Amydis, a privately held company focused on developing novel ocular diagnostic probes to identify patients at risk for neurodegenerative diseases, such as Alzheimer’s and Parkinson’s Disease.

Prior to becoming a serial entrepreneur, she spent 10 years in venture capital at Foresite Capital Management and Prospect Venture Partners, both growth capital health care funds with over $1 billion in assets under management. She led scientific, regulatory and commercial diligence on more than 400 investment opportunities from companies in developing therapeutics and diagnostics in the areas including cognitive disorders and neurology. Earlier in her career, Dr. Sarraf worked at Merck Research Laboratories in Rahway, New Jersey, as a senior research chemist responsible for creating novel drugs.

Stella holds a doctorate in organic chemistry from Columbia University and a bachelor's degree in biochemistry and molecular biology from the University of California, Berkeley.

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Vincent F. Simmon, Ph.D. - Chief Operating Officer

Dr. Simmon joined Spinogenix soon after it was founded in early 2016. Vince is an accomplished senior executive with broad experience in the biotech industry since 1979. Dr. Simmon was previously CEO of publicly traded companies, Alpha 1 Biomedicals and Cortex Pharmaceuticals. In addition, he was the CEO of privately-held companies, Xytis Inc. and COO of Merrimack Pharmaceuticals.

As a senior member of management or CEO, he has successfully led research and clinical development in small biotech companies as well as WR Grace&Co. His experience includes leading multinational clinical trials in mild cognitive impairment and traumatic brain injury, Phase 1 and 2 clinical trials in Alzheimer’s disease, schizophrenia, rheumatoid arthritis and psoriasis as well as clinical trials in AIDS, hepatitis B and hepatitis C. He has negotiated R&D and licensing deals with Organon (ultimately acquired by Merck), Servier, Green Cross and Yoshitomi in Japan, Shire and Pharmacia (acquired by Pfizer).

Vince earned his Ph.D from Brown University in Molecular and Microbiology and received his BA from Amherst College. He was a post-doctoral fellow at Stanford University and received Executive MBA training at Stanford and WR Grace.

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Peter Whitney Vanderklish, Ph.D. - Chief Scientific Officer

Dr. Vanderklish has a track record of expertise and significant contributions in synaptic neurobiology, intellectual disability, and the regulation of local protein synthesis within neurons. During graduate work at UC Irvine, he helped establish that long term potentiation is expressed by changes in the postsynaptic element – that is, in dendritic spines – and that postsynaptic adhesion molecules called integrins play a key role in consolidating such changes via the restructuring of synapses.

Dr. Vanderklish has conducted particularly impactful work on the molecular basis of Fragile X syndrome – the most common form of inherited intellectual disability, and leading known cause of autism – which has identified synaptic and molecular defects and potential new targets for therapeutic intervention. In other work, he has made significant contributions to our understanding of how the profile and rate of protein synthesis at synapses is regulated by neurotrophic factors, cytoskeletal and adhesion molecule determinants of synaptic structure, RNA-binding proteins, and microRNAs.

Peter holds a Ph.D. in Neurobiology from University of California, Irvine. He was previously Associate Professor in the Dept. of Molecular Medicine at The Scripps Research Institute, as a Visiting Professor in Anatomy and Neurobiology at UC Irvine, and Lecturer at The University of San Diego.

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Elizabeth Vadas, Ph.D, FAAPS - Head of R&D

Dr. Vadas joined Spinogenix as Head of R&D in 2017. She started her pharmaceutical career at Merck Frosst, the Canadian subsidiary of Merck & Co. in 1980, where she spent 22 years in the department of Pharmaceutical R&D. She served as Executive Director of Pharmaceutical Research and Development at Merck Frosst from 1991 to 2002 and was involved in the early characterization and formulation development of many new chemical entities discovered at the Merck Frosst Centre for Therapeutic Research.

Leadership accomplishments include rapid characterization and selection of viable development candidates, which she established through close collaboration with the discovery groups in basic research. Her product development efforts, which lead to worldwide regulatory approval of several products, include SINGULAIR®, Merck’s oral asthma therapy, and VIOXX® and ARCOXIA™, two Cox-2 inhibitors for the treatment of pain, osteoarthritis, and rheumatoid arthritis. In addition to her responsibilities in the Canadian research labs Dr. Vadas was also responsible for several years for Merck's ophthalmic drug development group, located in France. Dr. Vadas' main scientific interests are in the area of pharmaceutics. She served as a Member of the Advisory Board at Hungarian Innovative Technologies Fund. She was an Adjunct Professor of Pharmaceutics at the Faculty of Pharmacy, University of Montreal 1995 to 2005 and currently she is an Independent Consultant.

Elizabeth earned her Undergraduate degree in Colloid and Surface Chemistry in Budapest, Hungary and a Ph.D. in Physical Chemistry from McGill University in Montreal and was a postdoctoral fellow in the Department of Biochemistry at McGill University and in the Montreal Neurological Institute prior to joing Merck. Dr. Vadas is a Fellow of the American Association of Pharmaceutical Scientists and a Fellow of the Canadian Society for Pharmaceutical Sciences.

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Ron Newbold, Ph.D. - Chief Business Officer

Dr. Ron Newbold brings over 25 years of Business Development and Alliance Management experience leading business teams in negotiating over $1.5 billion in announced pharma & biotech deals. He is experienced leading search & evaluation and due diligence both in big pharma and in smaller biotechnology companies to facilitate corporate licensing and M&A transactions, collaborative academic partnerships and start-up company investments

He has experience from senior roles at Merck and Pfizer, as well as leading business development for a series of startup companies, and advisory board and SAB experience with over 10 companies.

Ron holds a PhD in organic chemistry from the University of Rochester, completed a postdoctoral fellowship at Harvard University, and received a MBA from Columbia University.

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Masoud Mokhtarani, M.D. - Chief Medical Officer

Dr. Masoud Mokhtarani has extensive experience in clinical development from bench to market with a focus on proof of concept, translational, and early-stage development programs ranging multiple therapeutic areas including neurodegenerative disease, systemic sclerosis, genetic and inherited inborn errors of metabolism, IPF, NASH, and pain. Previously, Dr. Mokhtarani served as Chief Medical Officer of NeuroVia Inc. and Curzion Pharmaceuticals (acquired by Horizon), successfully building clinical development plans for X-linked adrenoleukodystrophy, adrenomyeloneuropathy, and fibrotic diseases.

In addition, Dr. Mokhtarani served in executive clinical roles at Hyperion Therapeutics (acquired by Horizon), Limerick Neuroscience, Rinat Neuroscience (acquired by Pfizer), and Immune Tolerance Network. He is a co-inventor of multiple issued patents and an author/co-author of several peer-reviewed publications.

Masoud holds a M.D. from University of Tehran and completed his clinical research Fellowship at UCSF.

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Mervyn Turner, Ph.D. - Advisor

Dr. Mervyn Turner has over 25 years of experience in pharmaceuticals drug discovery, research and development, licensing and business development, emerging markets, strategy development and implementation. He is recognized for his deep industry perspective and for his global connections within the pharmaceuticals sector, biotech, and venture capital. In his last role prior to his retirement from Merck & Co. Inc., he was the company’s first Chief Strategy Officer, and drove strategy planning and resource planning discussions.

He also worked with the research division, the commercial organization and the manufacturing division to develop a coherent, overarching strategy for Merck & Co. Inc.’s investments in the emerging markets. Prior to that, he led the transformation of Merck & Co., Inc. from an inward-facing to an outward looking organization as head of World Wide Licensing & External Research. He was personally involved in more than 200 strategic transactions, including mergers and acquisitions. Before that, he was the Site Head for both Merck & Co. Inc.’s largest R&D facility (Rahway, NJ), and it’s most productive labs (Merck Frosst, Canada). In that role, he oversaw the introduction of multiple development candidates. Dr. Turner has published over 80 articles in peer reviewed journals.

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Justin Ichida, Ph.D. – Advisor

Dr. Ichida is an Associate Professor of Stem Cell Biology and Regenerative Medicine at the Keck School of Medicine of USC, and a Robertson Investigator at the New York Stem Cell Foundation. Hi research focuses on the etiology, pathogenesis, genetics, and treatment of neurodegenerative diseases, with a focus on amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. He is a recipient of the Champion for a Cure Award from the ALS Association, the highest honor the ALS Association bestows upon scientists for distinguished intellectual achievements.

Justin earned his Ph.D. at Harvard Medical School studying the origins of life with Jack Szostak and did postdoctoral work on cellular reprogramming with Kevin Eggan at Harvard University.

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Rita Sattler, Ph.D. - Advisor

Dr. Sattler is Associate Professor of Neurobiology and Neurology at the Barrow Neurological Institute, Phoenix, AZ. She received her masters and doctorate degree from the University of Toronto, Toronto, Canada and performed her postdoctoral training in the Department of Neuroscience at Johns Hopkins University. She then served as the lead scientist for a small startup Hopkins-initiated biotech company for 4 years (Psyadon Pharmaceuticals, Inc., former Ruxton Pharmaceuticals, Inc.), overseeing assay development, SAR and biomarker studies of lead compounds for transcriptional activation of glutamate transporters as therapeutic targets for ALS.

From there, Dr. Sattler joined the Johns Hopkins University Drug Discovery Center to strengthen her expertise in preclinical drug discovery. With this strong translational neuroscience background, Dr. Sattler started her first faculty position as Assistant Professor in the Department of Neurology in 2012 focusing on mechanisms of neurodegeneration in ALS and FTD using varying disease models, including human patient-derived induced pluripotent stem cells (iPSCs) differentiated into different subtypes of neurons and glia cells. Dr. Sattler is funded through numerous federal grants (DOD, NIH) and awards from disease foundations and private donors, as well as the Barrow Neurological Foundation.

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Spinogenix Granted FDA Orphan Drug Designation for SPG302 for the Treatment of Amyotrophic Lateral Sclerosis

SAN DIEGO, Calif., June 07, 2021 (GLOBE NEWSWIRE) - Spinogenix, Inc. a pharmaceutical company focused on the development of novel synaptogenic small molecule therapies for central nervous system disorders and rare diseases, today announced it has been granted Orphan Drug Designation (ODD) for SPG302 in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) by the U.S. Food and Drug Administration (FDA). By obtaining orphan drug designation, SPG302 is now eligible for such benefits as exemption from FDA user fees and tax credits for clinical research.

In addition, Spinogenix reports the successful completion of pre-IND (Investigational New Drug) interaction with the FDA regarding the current development plan for SPG302. The FDA agreed to the overall development program including the first-in-human Phase 1/2 clinical study in ALS patients followed by a Phase 2/3 Pivotal trial based on safety/PK/biomarker data.

“Receiving orphan drug designation from the FDA for the treatment of ALS is an important milestone,” stated Stella Sarraf, Founding CEO at Spinogenix. “We are also pleased with the guidance from the FDA that now provides us with a clear clinical plan to rapidly advance our first-in-class drug to help ALS patients.”

SPG302 is an orally bioavailable, blood-brain barrier penetrating small molecule. Its mode of action regenerates lost synapses and has demonstrated improvements in cognitive and motor behaviors in multiple animal models of neurodegenerative disorders.

Dr. Merit Cudkowicz, Director of the Sean M. Healey and AMG Center for ALS at Mass General Hospital, commented, “We are thrilled that the FDA granted ODD to Spinogenix for their novel drug SPG302 and are excited to collaborate with them as they advance toward the clinic.”

About Spinogenix

Spinogenix was founded in 2016 with the mission to develop transformative therapeutics for diseases involving synaptic loss and dysfunction. Our drugs are designed to regenerate synapses, regardless of the underlying cause of synapse loss, and thereby reverse declines in cognitive and motor function to impact the disease and fundamentally change treatment paradigms. Synapse loss is associated with a variety of neurological and psychiatric diseases, such as ALS, Alzheimer’s disease, Parkinson’s disease, schizophrenia, and depression.

Spinogenix Announces Grant by U.S. Department of Defense to Advance Novel Drug Candidate in Amyotrophic Lateral Sclerosis (ALS)

SAN DIEGO, Calif., Jan. 05, 2021 (GLOBE NEWSWIRE) - Spinogenix, Inc. a preclinical-stage biopharmaceutical company developing novel small molecule drugs for neurological conditions, today announced that it will be collaborating with Dr. Rita Sattler at the Barrow Neurological Institute and Dr. Justin Ichida at the USC Keck School of Medicine on a grant awarded from the U.S. Department of Defense’s (“DOD”) Congressionally Directed Medical Research Programs (“CDMRP”) to evaluate its lead development candidate in ALS. The DOD grant will be used to study the effects of Spinogenix’s lead compound in human iPSCs (induced pluripotent stem cells) from patients with ALS and from healthy volunteers. Additional experiments will be conducted in animal models of ALS.

Spinogenix’s lead drug candidate has a unique mechanism of action wherein it induces an increase in synapses, the key connections between neurons that allow us to think, plan, remember and control motor functions, faculties that are diminished in neurodegenerative diseases including ALS. “We are pleased that the DoD has recognized the potential of our novel drug candidate to change the course of disease progression in ALS,” stated Stella Sarraf, Ph.D., Founding Chief Executive Officer at Spinogenix.

There is an unmet need for new innovative therapeutics for ALS (Lou Gehrig’s disease) which is almost invariably fatal within 3-5 years of diagnosis. The therapies that are currently approved for ALS provide very modest extension of life of several months and are not well tolerated by all patients.

Dr. Merit Cudkowicz, Director of the Sean M. Healey and AMG Center for ALS at Mass General Hospital, commented, “Spinogenix’s novel approach has the potential to demonstrate that replacing lost synapses may result in drugs that can provide a meaningful benefit for patients with ALS.”

About Spinogenix

Spinogenix was founded in 2016 with the mission to develop transformative therapeutics for diseases involving synaptic loss and dysfunction. Our drugs are designed to regenerate synapses to reverse declines in cognitive and motor function to impact the disease and fundamentally change treatment paradigms by restoring neuronal connections regardless of the underlying cause of synapse loss. Synapse loss is associated with a variety of neurological and psychiatric diseases, such as ALS, Alzheimer’s disease, Parkinson’s disease, schizophrenia, and depression.

Spinogenix Awarded NIH Grant to Develop Novel Neurorestorative Therapeutics for Alzheimer’s Disease

San Diego, CA, May 1 2018: Spinogenix, Inc., a preclinical stage, privately-held pharmaceutical company developing first-in-class drugs to reverse synapse loss and restore connections lost to neurodegenerative diseases, today announced a grant award by the National Institute of Health (NIH). The grant award will fund research and development of a novel family of compounds to help restore the brain connections and functions lost in Alzheimer’s disease (AD) and related neurodegenerative disorders.

AD is a degenerative brain disease and the most common cause of dementia - accounting for an estimated 60 to 80 percent of dementia cases. The Alzheimer’s Association 2018 Report estimates that there will be 5.7 million people with dementia in the United States in 2018 with an estimated 14 million new cases of dementia by 2050, equivalent to one person every 3.2 seconds.

“Spinogenix is extremely pleased to receive this grant award which provides rigorous peer-reviewed validation of our innovative approach to tackle AD. This funding will help us further develop our drug candidates and bring a promising new class of therapeutics to the marketplace to treat not only AD but also related diseases that involve a loss of dendritic spine synapses”, said Stella Sarraf, Ph.D., Founder and Acting Chief Executive Officer.

“Loss of dendritic spines is a major driver of cognitive decline in all neurodegenerative diseases as well as normal aging,” said Franz Hefti, Chairman of the Board. “Spinogenix is targeting the loci of changes underlying memory formation.”

Currently, none of the available treatments for AD or other neurodegenerative diseases reverse synapse loss. With the support of this NIH grant, Spinogenix aims to further develop a new class of neurorestorative therapeutics to help treat patients with AD.

About Spinogenix

Spinogenix was founded in 2016 to address the urgent clinical need for novel drugs to combat diseases and conditions involving synaptic aging and loss. Our neurorestorative compounds are designed to restore synapses in order to enhance brain connections and improve memory. Current development is focused on therapeutic treatments for AD and traumatic brain injury/concussion. More information on Spinogenix can be found at www.spinogenix.com.

Disclosure

This project is supported by the National Institute on Aging of the National Institutes of Health under award number R43AG058278. The content is solely the responsibility of the Company and Principal Investigators and does not necessarily represent the official views of the National Institutes of Health.