SiteOne Therapeutics Announces Initiation of Phase 1 Clinical Trial for its Non-Opioid Analgesic for Acute Pain

 SiteOne’s Study of ST-2427 Examines Safety, Tolerability, and Pharmacokinetics

 SOUTH SAN FRANCISCO, CALIF., April 20, 2021 – SiteOne Therapeutics, Inc., a clinical-stage biopharmaceutical company developing new treatments to address conditions involving hypersensitivity of the nervous system, today announced that dosing has commenced in the company’s Phase 1, dose-escalation study of ST-2427, a highly selective inhibitor of NaV1.7 for the management of moderate-to-severe pain.

 “The initiation of this study represents a significant milestone for the company as it is our first drug candidate to enter the clinic,” stated John Mulcahy, Ph.D., chief executive officer of SiteOne. “There is an urgent, unmet need to develop effective non-opioid therapies for pain. Our lead compound, ST-2427, addresses that need by targeting the peripheral nerve fibers that conduct pain signals without the potential for CNS side effects, addiction and abuse liability of opioid medications.”

 ST-2427 blocks sodium ion channel 1.7 (NaV1.7). NaV1.7 is a subtype of sodium channel that is highly expressed in peripheral nerve fibers, responsible for pain signal transmission, and is validated as a target for pain treatment by human genetics. Individuals lacking this protein are unable to experience pain. By selectively targeting NaV1.7, ST-2427 aims to stop the electrical signals responsible for pain before they reach the central nervous system.

 “SiteOne has discovered exquisitely selective modulators of multiple sodium channel subtypes, and we are now advancing development candidates for a number of indications, starting off by addressing acute pain,” said Debra Odink, Ph.D., SiteOne’s chief development officer. “We look forward to working with the medical community to bring the promise of this new approach to patients and anticipate initial data will drive our future development plans.”

 The phase 1 study is a randomized, double-blind, placebo-controlled study in healthy adults to evaluate the safety, tolerability, and pharmacokinetics of ST-2427. The study is enrolling subjects at AltaSciences in Overland Park, Kansas. Subjects in each group will be randomized to receive a single dose of ST-2427 or placebo.

 Additionally, SiteOne recently announced the expansion its technology platform and clinical development activities to address hypersensitivity disorders more broadly in both the peripheral and central nervous systems. These disorders include chronic cough, neuropathic pain, ocular discomfort, and pruritus.

About the ST-2427 Phase 1 Study
The phase 1, randomized, double-blind, placebo-controlled study in healthy adult males and females will evaluate the safety, tolerability, and pharmacokinetics (PK) of ST-2427. The trial will include careful assessments of treatment effects on vital signs including cardiac and respiratory function and body temperature over a range of doses of ST-2427. SiteOne Therapeutics, Inc. plans to use the safety, tolerability, and PK findings from this study to inform the doses and study design for phase 2 clinical studies in subjects with acute pain. Please refer to clinicaltrials.gov NCT04475198 for additional clinical trial information.

 About ST-2427
ST-2427 is an experimental intravenous, non-opioid analgesic for the management of moderate-to-serve severe pain. ST-2427 blocks sodium ion channel 1.7 (NaV1.7).  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. As ST-2427 does not enter the brain, it is not expected to cause the central nervous system adverse effects (e.g. sedation, nausea, dependence and addition) that plague existing pain therapeutics such as the opioids. The development of ST-2427 was supported by the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH) under Award Number UG3DA049599. The content 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 novel therapeutics 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. 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

Evan Wicker, Ph.D.
Russo Partners, LLC
evan.wicker@russopartnersllc.com
212-845-4235

SiteOne Therapeutics Names Scientific Founder John Mulcahy, Ph.D., as Chief Executive Officer

Company Broadens Drug Development Focus on Platform That Addresses Hypersensitivity Disorders of Peripheral and Central Nervous Systems

SOUTH SAN FRANCISCO, Calif., March 23, 2021—SiteOne Therapeutics Inc., a private biopharmaceutical company developing new treatments to address conditions involving hypersensitivity of the nervous system, today announced the appointment of John Mulcahy, Ph.D., as chief executive officer. Dr. Mulcahy is one of the scientific co-founders of the company and has led the strategic as well as technical drug discovery efforts at SiteOne since its founding. He replaces Stan Abel, who becomes SiteOne’s board chairman.  

The move comes as SiteOne expands its technology platform and clinical development activities to address hypersensitivity disorders more broadly in both the peripheral and central nervous systems. These disorders include chronic cough, neuropathic pain, ocular discomfort, and pruritus. Clinical trials are expected to start for SiteOne’s lead drug candidate this year, while the company works to expand its pipeline.

“As part of the heart and foundation of our company, John was a clear choice to lead SiteOne,” Abel said. “With his deep knowledge of the science, success in securing funding and experience leading our company’s research efforts, John will drive the vision of SiteOne. We are creating a robust pipeline of potent and highly selective drug candidates targeting voltage-gated sodium ion channels that show vast potential for treating a wide array of disorders involving hypersensitivity of the nervous system.”

Dr. Mulcahy was previously the vice president of research at SiteOne. He is the co-inventor and has co-authored the core patents and publications of the company’s technology platform. In addition, Dr. Mulcahy has secured and served as Principal Investigator on multiple grants from the US Department of Defense and National Institutes of Health. Dr. Mulcahy earned his Ph.D. in Organic Chemistry from Stanford University and his A.B. in Chemistry, Physics, and Classics from Harvard University.

“Our approach, which leverages high quality structural information on the voltage-gated sodium channel and targets specific molecular differences between isoforms, has transformative potential for the treatment of common and rare neurological disorders,” said Dr. Mulcahy. “While industry has struggled to develop drugs that selectively engage sodium channels for the past 20 years, recent advances in our understanding of sodium channel structure and pharmacology have enabled discovery of drug candidates with exquisite subtype specificity. With our lead drug candidate, ST-2427, a novel non-opioid analgesic, headed toward clinical trials and additional novel therapeutics in the pipeline to treat chronic cough and ocular discomfort, I look forward to leading SiteOne.”

Clinical trials for ST-2427, which selectively blocks sodium channel NaV1.7 to inhibit pain signals, will start later this year. By interfering with pain signals in the peripheral nervous system, ST-2427 seeks to avoid the significant side effects and addiction potential of opioid pain medications. Another SiteOne drug candidate, ST-2578, for ocular surface discomfort, is undergoing IND-enabling studies.

About SiteOne Therapeutics
SiteOne Therapeutics is a private 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

SiteOne Announces Award of $5.3M Federal Grant from NIH / NIDA for the Development of a Novel Non-Opioid Analgesic

Award intended to fund non-clinical and clinical activities through Phase 2 Proof of Concept in Post-operative Pain Management

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BOZEMAN, Mont., October 10, 2019 /PRNewswire/ -- SiteOne Therapeutics, a privately-held biopharmaceutical company advancing novel non-opioid pain therapeutics, today announced that it has received a Notice of Award from the National Institute on Drug Abuse (NIDA) of the National Institutes of Health (NIH) for grant funding under the Helping to End Addiction Long-term, or the NIH HEAL Initiative, to support development of the Company’s lead therapeutic candidate, ST-2427, a novel NaV1.7 sodium channel inhibitor for the treatment of acute post-operative pain. 

“This grant represents a significant milestone for SiteOne as we continue to advance the ST-2427 program toward the clinic,” said Stan E. Abel, Chief Executive Officer of SiteOne Therapeutics. “ST-2427 selectively blocks sodium channel NaV1.7, which has strong validation as a target for analgesic drug development based on human genetics. Importantly, ST-2427 will potentially block pain signals without affecting the central nervous system, thus avoiding the significant side effects and addiction potential of opioid pain medications. We are very grateful to NIDA and the NIH for this grant, which provides non-dilutive funding that will allow us to assess the potential of ST-2427 for the treatment of post-operative pain through Phase 2 proof-of-concept studies.”

This grant 1UG3DA049599-01, titled “Development of a Potent and Highly Selective NaV1.7 Inhibitor for the Treatment of Acute 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 2-year period. The UH3 phase will provide funding for a further 3 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.

“It’s clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain and provide more flexible and effective options for treating addiction to opioids,” said NIH Director Francis S. Collins, M.D., Ph.D., who launched the initiative in early 2018. “This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.”

SiteOne’s Co-Principal Investigators for the grant are Dr. John Hunter, Chief Scientific Officer and Dr. Debra Odink, Chief Development Officer.

“The development of drugs targeting NaV1.7 has been challenging, due in part to the difficulty of achieving adequate selectivity for NaV1.7 over other closely related sodium ion channel isoforms,” Dr. Hunter added. “Our approach, which leverages natural sodium channel inhibitors, has enabled the development of a robust pipeline of potent and selective drug candidates targeting NaV1.7. We believe that our lead compound, ST-2427 has transformative potential for the treatment of pain without opioids, and we look forward to beginning clinical studies, which we expect to initiate in 2020.”

About SiteOne Therapeutics, Inc.
SiteOne Therapeutics is a private biopharmaceutical company headquartered in Bozeman, Montana with a research laboratory in South San Francisco, California.  Since its inception, SiteOne has been dedicated to developing novel pain therapeutics to safely, effectively and efficiently treat acute and chronic pain without the significant addiction potential and side-effects of opioids. The company's therapeutic candidates are highly selective sodium ion channel 1.7 (Nav1.7) inhibitors based on naturally occurring small molecules. Given the urgent need for new, non-opioid solutions for managing pain, SiteOne is focused on advancing its lead product candidates for multiple therapeutic applications.

About the NIH HEAL Initiative

The Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM, 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.

Contact:
SiteOne
info@site1therapeutics.com

Investors:
Burns McClellan
Lee Roth, 212-213-0006
lroth@burnsmc.com