SiteOne Therapeutics Awarded up to $15M Grant to Develop NaV1.8 Inhibitor as Non-Opioid Therapeutic for Pain

The National Institute on Drug Abuse through the NIH HEAL Initiative will fund development of SiteOne’s selective NaV1.8 inhibitor for the treatment of pain

SOUTH SAN FRANCISCO, CALIF., May 18, 2023 – SiteOne Therapeutics, Inc., a clinical-stage biopharmaceutical company developing isoform-selective inhibitors of the voltage gated sodium channel (NaV) for the treatments of pain, cough, and other conditions involving hypersensitivity of the nervous system, today announced that it has received a grant (UG3DA058552) for up to $15 million from the National Institute on Drug Abuse (NIDA) for the development of STC-004, an investigational, non-opioid treatment for acute and chronic pain conditions. The grant was awarded through the National Institutes of Health (NIH) Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, a trans-agency effort to accelerate the development of scientific solutions to the national opioid public health crisis.

The award, titled “Development of a Potent and Selective Nav1.8 inhibitor for the Treatment of Acute and Chronic Pain with the Goal of Reducing Opioid Use and Preventing Opioid Use Disorders,” is a UG3/UH3 Phase Innovation Awards Cooperative Agreement involving two phases. The UG3 phase is to support a project with specific milestones to be accomplished by the end of a two-year period. The UH3 phase will provide funding for a further three years to a project that successfully completed the milestones set in the UG3 phase. Application budgets are limited to $3 million direct costs per year and UG3 projects that have met their milestones will be considered by NIDA and prioritized for transition to the UH3 phase, with the total funding currently expected to be available under both the UG3 and UH3 phases to be a maximum of $15 million in direct costs.

“SiteOne Therapeutics is honored to receive funding from  the National Institute on Drug Abuse to develop innovative solutions for the treatment of pain and aim to reduce our health care system’s reliance on opioids,” commented John Mulcahy, President and Chief Executive Officer of SiteOne Therapeutics. “This award validates our approach to addressing pain by targeting individual sodium channel isoforms, NaV1.7 and NaV1.8, that are predominantly expressed in pain-sensing nerve fibers.”

“The perception of pain is a complex neurological process, and the key to providing solutions to address both acute and chronic pain is to selectively target pathways that lead to this sensation. Both opioids and over-the-counter NSAIDs can sometimes pose major health risks to patients and are ineffective in blocking certain types of pain, particularly neuropathic pain,” noted John Hunter, Chief Scientific Officer of SiteOne Therapeutics. “Our therapeutic approach of inhibiting sodium channel function aims to limit the hypersensitivity reaction to pain by regulating the misfiring of peripheral nerves that transmit the perceived pain to the central nervous system. This grant is essential in funding the clinical development of our NaV1.8 inhibitors with the goal to provide relief to patients across a variety of pain conditions.”

This research is supported by the National Institute on Drug Abuse of the National Institutes of Health and the NIH Helping to End Addiction Long-term Initiative under award number UG3DA058552. The content presented in this release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.  

About SiteOne Therapeutics

SiteOne Therapeutics is a clinical-stage biopharmaceutical company advancing a novel class of highly selective small molecule inhibitors targeting NaV1.7, NaV1.8, and other voltage-gated sodium ion channels to treat peripheral hypersensitivity disorders such as chronic cough, itch, and pain. Since its inception, SiteOne has been dedicated to the development of safe and effective pain therapeutics without the significant addiction potential and side effects of opioids. The company is also advancing additional novel drug candidates that exhibit precise selectivity for individual sodium channel subtypes to treat other hypersensitivity disorders such as chronic cough and chronic ocular surface pain. For more information, visit www.siteonetherapeutics.com.

About the NIH HEAL Initiative

The Helping to End Addiction Long-term® Initiative, or NIH HEAL Initiative®, is an aggressive, trans-NIH effort to speed scientific solutions to stem the national opioid public health crisis. Launched in April 2018, the initiative is focused on improving prevention and treatment strategies for opioid misuse and addiction, and enhancing pain management. For more information, visit: https://heal.nih.gov.

Media Contact:
Maddie Stabinski
Russo Partners, LLC
Madeline.stabinski@russopartnersllc.com

SiteOne Therapeutics Announces Collaboration and License Agreement with Vertex Pharmaceuticals to Advance NaV1.7 Inhibitors for the Treatment for Pain

- SiteOne will be responsible for research and early preclinical development activities; Vertex will be responsible for development, and commercialization  
- Terms include an upfront payment, milestone payments, and royalties

SOUTH SAN FRANCISCO, CALIF., January 19, 2022 – SiteOne Therapeutics, Inc., a clinical-stage biopharmaceutical company developing non-opioid treatments to address pain, cough and other conditions involving hypersensitivity of the nervous system, today announced that it has entered into a strategic collaboration and licensing agreement with Vertex Pharmaceuticals. Under the terms of the agreement, the companies will work together to advance potent and selective inhibitors of sodium ion channel 1.7 (NaV1.7) as non-opioid therapeutics for the treatment of pain. SiteOne will be responsible for research and early preclinical development activities, with Vertex responsible for subsequent development, and commercialization. SiteOne will receive an upfront payment and is eligible for development, regulatory and commercial milestone payments, and royalties from Vertex if the program succeeds.

“Pain is an extremely common medical condition that severely affects quality of life. The existing standard of care for pain treatment relies heavily on opioid analgesics, which are not always effective and carry a high risk of side-effects, dependence, and abuse. A core mission of SiteOne is to identify non-opioid pain medicines that will be able to safely and effectively treat many different types of pain and other neurological conditions without the risk of dependence or abuse,” said John Mulcahy, Ph.D., CEO of SiteOne. “We are excited to begin this partnership with Vertex, which has a long-standing interest in non-opioid analgesic drug development. This agreement will support the advancement of SiteOne’s portfolio of exquisitely-selective NaV1.7 inhibitors across multiple indications.”

NaV1.7 is a type of sodium channel that is highly expressed in peripheral nerve fibers that play a critical role in the generation and conduction of pain signals. The peripheral nervous system (PNS) is responsible for alerting the body against surrounding harmful stimuli. However, following insult or injury, the PNS often becomes sensitized, resulting in acute or chronic hypersensitivity disorders such as, pain, itch, and cough.

About SiteOne Therapeutics
SiteOne Therapeutics is a clinical-stage biopharmaceutical company advancing novel therapeutics targeting NaV1.7, NaV1.8 and other ion channels to treat hypersensitivity disorders. Since its inception, SiteOne has been dedicated to the development of safe and effective pain therapeutics without the significant addiction potential and side effects of opioids. The company is also advancing additional novel drug candidates that exhibit precise selectivity for individual sodium channel subtypes to treat other hypersensitivity disorders such as chronic cough and chronic ocular surface pain. SiteOne's therapeutic candidates are highly selective ion channel modulators. For more information, visit www.siteonetherapeutics.com

Media Contacts:
David Schull
Russo Partners, LLC
David.schull@russopartnersllc.com
858-717-2310

SiteOne Therapeutics Announces $1.73M Grant from NIH/NHLBI and Adds Respiratory Advisory Team

– Grant award to fund formulation of inhaled therapy for chronic cough –

– Cough advisory team includes 4 acclaimed researchers –

SOUTH SAN FRANCISCO, CALIF., May 11, 2021 – SiteOne Therapeutics, Inc., a clinical-stage biopharmaceutical company developing new treatments to address conditions involving hypersensitivity of the nervous system, today announced that it has received a Notice of Award from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) for $1.73 million in grant funding to support the development of an inhaled formulation of SiteOne’s drug candidate for chronic cough. In conjunction with the grant funding, SiteOne has added an advisory team to advance the development of its drug candidates for chronic cough and other respiratory disorders.

 “The funding from this award will allow us to advance an inhaled formulation of our development candidate for chronic cough through IND-enabling studies and toward the clinic,” stated John Hunter, Ph.D., Principal Investigator for the award and chief scientific officer of SiteOne. “Chronic cough is a prevalent respiratory disorder that is often unexplained or refractory, and severely impacts quality of life. We aim to develop a safe and effective local-acting inhaled antitussive to address the hypersensitivity of vagal afferents that is thought to drive chronic cough.”

Chronic cough is a cough that lasts eight weeks or longer.  The condition often causes individuals to cough 100 to 1,000 times per day. Cough is mediated by sensory nerve fibers of the vagus nerve that terminate in the upper airways and lungs. SiteOne’s drug candidate aims to reduce the sensitivity of these nerve fibers by inhibiting NaV1.7, a type of sodium channel that is highly expressed. By selectively targeting these peripheral nerve fibers, SiteOne’s drug candidate aims to suppress the electrical signals involved in the pathological cough reflex and reduce the urge to cough.

 SiteOne is also adding four experts to an advisory board to guide the development of SiteOne’s drug candidate for chronic cough.

 Brendan Canning, Ph.D., is a Professor of Medicine in the Division of Allergy and Clinical Immunology at the Johns Hopkins University School of Medicine in Baltimore, Maryland. He has been or is currently Co-Editor-in-Chief or Associate Editor for six medical research journals. Dr. Canning has served as a scientific advisory board member and a consultant for many pharmaceutical and biotech companies.

 Peter Dicpinigaitis, M.D., is a Professor of Medicine at the Albert Einstein College of Medicine and is active in the field of cough treatment and research. Dr. Dicpinigaitis and colleagues have developed methods of cough reflex sensitivity measurement that have been adopted in international guidelines as the proposed standard method of cough challenge testing. Using this methodology, Dr. Dicpinigaitis has performed numerous studies to evaluate potential antitussive drugs. He is Director of the Montefiore Cough Center in New York, one of the few subspecialty cough centers in the United States, and is the Editor-in-Chief of the journal LUNG.

 Anthony Ford, Ph.D., has more than 30 years of neuroscience research, pharmacology, drug discovery and development experience from leading biopharmaceutical companies to his role as co-founder and chief executive officer. He is chief executive officer and a co-founder of CuraSen. Dr. Ford also holds senior advisory roles with PharmNovo and for New Leaf Venture Partners, where he had previously held the title of venture partner. He is also an advisor to the Stanford SPARK program. Prior to CuraSen, Dr. Ford co-founded Afferent Pharmaceuticals, and as chief scientific officer led the discovery and development of P2X3 antagonists, including AF-219/MK-7264 (gefapixant), for chronic cough through Phase 3 readiness until the company’s acquisition by Merck in 2016.

 Bradley J. Undem, Ph.D. is a Professor of Medicine in the Division of Allergy and Clinical Immunology at the Johns Hopkins University School of Medicine, with a joint appointment in Physiology in the Bloomberg School of Public Health, in Baltimore, Maryland. He currently serves on the Editorial Boards of several journals and has served as a consultant and on the scientific advisory boards for several pharmaceutical companies.

 “The addition of this team of experts in cough will allow us to bring together knowledge and experience to develop and guide a clinical strategy for our chronic cough therapy,” said John Mulcahy, Ph.D., chief executive officer of SiteOne.

 About SiteOne Therapeutics
SiteOne Therapeutics is a clinical-stage biopharmaceutical company advancing novel therapeutics to treat hyperexcitability and hypersensitivity disorders. Since its inception, SiteOne has been dedicated to developing novel pain therapeutics to treat acute and chronic pain safely, effectively, and efficiently without the significant addiction potential and side effects of opioids. The company is also advancing additional novel drug candidates that exhibit precise selectivity for individual sodium channel subtypes to treat hypersensitivity disorders of both the peripheral and central nervous systems. SiteOne's therapeutic candidates are highly selective sodium ion channel modulators. For more information, visit www.siteonetherapeutics.com

 Media Contacts:
David Schull
Russo Partners, LLC
David.schull@russopartnersllc.com
858-717-2310