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PRN-001-01 (tezampanel) is a novel antagonist of glutaminergic signaling with potential to suppress opiate withdrawal symptoms
First-in-human study to evaluate the safety, tolerability, PK profile, and efficacy in patients with Opioid Use Disorder
Dosing of first patient represents key advancement in Proniras’ clinical pipeline

November 14, 2024, Seattle – Proniras Corporation (“Proniras”), a clinical-stage biotechnology company developing novel small-molecule therapeutics for the treatment of substance use and specialty central nervous system disorders, today announced that the first patient has been dosed in the Phase 1 clinical trial evaluating its lead compound, PRN-001-01 (tezampanel) as a therapy for patients recovering from opioid addiction and related conditions. PRN-001-01 (tezampanel) is a competitive antagonist with selectivity for the AMPA and kainate subtypes of the ionotropic glutamate receptor family.

The Phase 1 trial is being conducted at Indiana University (IU) School of Medicine and is designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of PRN-001-01 (tezampanel) in the treatment of opioid withdrawal syndrome. The trial is anticipated to enroll approximately 40 patients with Opioid Use Disorder (OUD). The study will also provide insights into the effectiveness of blocking glutamate receptors to alleviate withdrawal symptoms, utilizing the Clinicians Assessment of Opiate Withdrawal Severity (COWS) and related measures. The results may show that PRN-001-01 (tezampanel) can relieve patients from withdrawal symptoms, potentially alleviating a crucial impediment in progression to long-term sobriety. Additional information about the study is available at clinicaltrials.gov (NCT06538558).

“Treating the first patient is a significant milestone for Proniras as we seek to address the needs of underserved patients suffering from opioid use disorder,” said Thong Q. Le, CEO of Proniras and Senior Managing Director of Accelerator Life Science Partners. “Supported by encouraging preclinical results, the Phase 1 clinical trial will formally assess the potential of tezampanel to make a meaningful difference for people suffering the symptoms of opioid withdrawal syndrome. This debilitating set of symptoms traps individuals and thwarts their attempts to escape dependence on opioids.”

“Understanding the role of glutamate pathways in addiction has been a significant area of research, and tezampanel is the first glutamate antagonist to enter investigational use for opioid use disorders,” said R. Andrew Chambers, MD, Professor of Psychiatry and Director of Addiction Psychiatry Training at IU School of Medicine. “IU School of Medicine is well-positioned to spearhead the effort to evaluate tezampanel’s impact on opioid withdrawal syndrome, given the devastating impact of the opioid crisis in the American Midwest. We are excited to have the first patient enrolled in this initial trial.”

About Opioid Withdrawal Syndrome
Opioid Withdrawal Syndrome is a psychologically and physically punishing condition that occurs when a person who has developed a physical dependence on opioids suddenly stops or significantly reduces their use of opioids. It is characterized by a range of uncomfortable symptoms including anxiety, agitation, muscle aches, sweating, nausea, vomiting, diarrhea, and intense drug cravings. Opioid withdrawal can be severe, often thwarting the best intentions of individuals striving for an opiate-free life. The syndrome affects a significant number of people. A recent assessment (2020-2024) of opioid withdrawal syndrome in the developed world reported annual case rates of approximately 8 million, with 85% occurring in the United States—an alarming statistic expected to persist over the next decade.

About Proniras Corporation
Proniras Corporation is a clinical-stage biotechnology company dedicated to developing novel small-molecule therapeutics for the treatment of substance use disorders and specialty central nervous system conditions. Leveraging cutting-edge research and innovative approaches, Proniras aims to address the unmet medical needs of patients suffering from these challenging conditions. The company’s lead compound, PRN-001-01 (tezampanel), is a novel antagonist of glutaminergic signaling that is currently in a Phase 1 clinical trial for the treatment of opioid withdrawal syndrome and other related disorders. Proniras is committed to improving patient outcomes and enhancing the quality of life through pioneering scientific breakthroughs. Proniras was founded in 2017 by Accelerator Life Science Partners. For more information, please visit www.proniras.com.

Yibin Kang, PhD, chairs Scientific Advisory Board that includes experts in translational and early-phase clinical development activities

Seattle, WA — October 22, 2024, Kayothera, Inc., a preclinical-stage therapeutics company developing first-in-class, oral, small molecule inhibitors of the retinoid nuclear receptor pathway, announced the expansion of its Scientific Advisory Board (SAB) to support the development of its first-in-class, oral, small molecule inhibitors of the retinoid nuclear receptor pathway.

Chaired by Yibin Kang, PhD, Warner-Lambert/Parke-Davis Professor of Molecular Biology at the Ludwig Institute of Princeton University, the SAB will work closely with Kayothera’s executive team to provide strategic guidance and expert insights to advance its mission of creating the best therapies to treat serious and life-threatening diseases.

Kayothera’s SAB includes the following members:

• Domenico Accili, MD, the Russell Berrie Foundation Professor of Diabetes at Columbia University and the Director of the Columbia University Diabetes Research Center. Dr. Accili is the 2017 Banting Medal Recipient and is highly recognized for his pioneering work on beta cell failure in type 2 diabetes. Dr. Accili pioneered the theory of pancreatic islet dedifferentiation and identified ALDH1a3 as a key marker of diseased pancreatic islets.
• Shridar Ganesan, PhD/MD, a prominent physician-scientist and medical oncologist at the NYU Perlmutter Cancer Center, where he serves as the Director for the Center for Molecular Oncology and Professor of Medicine. Dr. Ganesan is well-known for pioneering breast cancer research and guides Kayothera’s early clinical program in oncology.
• Suzanne George, MD, a renowned clinical expert in sarcoma, serving as the clinical director of the Center for Sarcoma and Bone Oncology at Dana-Farber Cancer Institute and an associate professor of Medicine at Harvard Medical School; Dr. George has extensive expertise in soft tissue sarcoma, bone sarcomas, and gastrointestinal stromal tumors (GIST); and
• Kendall Mohler, PhD, was previously co-founder and chief scientific officer at Trubion Pharmaceuticals, the founding CSO at Juno Therapeutics, and a senior leader at Immunex, where he played a key role in the development of Enbrel (etanercept).

“I am truly excited to collaborate with this exceptional team at Kayothera,” said Dr. Kang. “The collective expertise and experience of our Scientific Advisory Board members will be crucial as we push the frontiers of innovation. Together, we are set to make significant progress in developing first-in-class, oral, small-molecule inhibitors of the retinoid nuclear receptor pathway. I eagerly anticipate the profound impact we will have on treating serious and life-threatening diseases.”

About Kayothera. Kayothera, Inc. is an early-stage therapeutics company focused on the development of first-in-class, oral, small-molecule inhibitors of the retinoid nuclear receptor pathway. This pathway is pathogenically activated in several cancers as well as multiple cardiometabolic diseases, including diabetes and obesity. Kayothera is developing three distinct therapies, with KAYO-1732 in development as a disease-modifying treatment that controls blood glucose and lipid levels to prevent multiorgan degeneration in cardiometabolic syndrome and diabetes. KAYO-1609 has completed IND-enabling studies and is in development to treat late-stage and metastatic cancers, including pancreatic, prostate, gastric, and multiple other cancers. Kayothera is also developing a next-generation obesity therapy based on a genetically validated target in the retinoid nuclear receptor pathway. The company was founded based on discoveries from Dr. Mark Esposito’s post-doctoral research at Princeton University and Professor Yibin Kang, PhD. For more information, visit www.kayothera.com.

Bruce L.A. Carter, PhD, and Paul Sekhri join Board of Directors, adding drug development and operating experience

Recent board additions highlight Kayothera’s evolution into pipeline-focused organization with meaningful translational efforts

Seattle, WA — October 14, 2024, Kayothera, Inc., a preclinical-stage therapeutics company developing first-in-class, oral, small molecule inhibitors of the retinoid nuclear receptor pathway, announced the appointment of Bruce L.A. Carter, PhD (Chairman), and Paul Sekhri to its Board of Directors.

“We are pleased to welcome Dr. Bruce Carter and Paul Sekhri to the Board of Kayothera,” stated Thong Q. Le, President & CEO. “Bruce and Paul bring substantial drug development expertise and have demonstrated a commitment to advancing therapeutic innovations that benefit patients in need. We look forward to their contributions, which will help ensure Kayothera’s long-term success.”

Bruce Carter, PhD, was the Chairman of the Board and former Chief Executive Officer of ZymoGenetics Inc., USA. He has also served as the Corporate Executive Vice President and Chief Scientific Officer for Novo Nordisk A/S, the former parent company of ZymoGenetics. From 1982 to 1986, Dr. Carter held various positions of increasing responsibility at G.D. Searle & Co., Limited, including Head of Molecular Genetics. He was a lecturer at Trinity College, University of Dublin, from 1975 to 1982. Dr. Carter holds directorship in Enanta Pharmaceuticals, TB Alliance, and Aurigene Discovery Technologies Limited (India). Dr. Carter received his BS with Honors in Botany from the University of Nottingham, England, and his PhD in Microbiology from Queen Elizabeth College, London University, UK.

“I am thrilled to chair the Kayothera Board,” said Dr. Carter. “This is an exciting time as Kayothera’s KAYO-1732 candidate for type 2 diabetes progresses to IND and its candidate for solid tumors, KAYO-1609, enters Phase 1 trials. I look forward to working closely with Kayothera’s board and executive team as the company progresses its promising drug candidates.”

Paul Sekhri has over 30 years of experience in the Life Science Industry. His experience encompasses senior management in large corporate pharmaceutical and biotechnology companies and private equity and venture capital. Mr. Sekhri is currently President and Chief Executive Officer and Chairman of vTv Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on diabetes. He most recently held the positions of President, CEO, and Chairman at eGenesis, Inc., from 2019 to 2022. Prior to eGenesis, he was President and CEO of Lycera Corp. from 2015 to 2019. Prior to Lycera, he served as Senior Vice President, Integrated Care for Sanofi from 2014-2015. Previously, he served as Chief Strategy Officer and Group Executive Vice President, Global Business Development for Teva Pharmaceutical Industries, Ltd. Prior to joining Teva, Mr. Sekhri spent five years as Operating Partner and Head of the Biotechnology Operating Group at TPG Biotech, the life sciences venture capital arm of TPG Capital. From 2004-2009, Mr. Sekhri was Founder, President, and Chief Executive Officer of Cerimon Pharmaceuticals, Inc. Before founding Cerimon, Mr. Sekhri was President and Chief Business Officer of ARIAD Pharmaceuticals, Inc. Previously, Mr. Sekhri spent five years at Novartis as Senior Vice President, and Head of Global Search and Evaluation, Business Development and Licensing for Novartis Pharma AG.

Mr. Sekhri has been a director on more than 35 private, public company, and non-profit boards and is currently a member of the Board of Directors of AdhereTech, Spring Discovery, and Veeva Systems Inc. Mr. Sekhri is also the Chairman of the Boards of Directors of Resolution Therapeutics, Compugen Ltd., Longboard Pharmaceuticals, and Kaerus Bioscience. Additionally, he is on the Board of Directors of The Metropolitan Opera, The English Concert in America, and was a member of the Board of Trustees of Carnegie Hall. Most recently, he was nominated as Chairman of the Board of Trustees of Young Concert Artists, Inc.

“I look forward to collaborating with the Kayothera Board to guide the development of innovative medicines that address underserved patients in need,” stated Mr. Sekhri. “Kayothera’s focus on developing novel, first-in-class antagonists of the retinoid nuclear signaling pathway and dedication to bringing best-in-class drug candidates forward is a mission that I look forward to supporting.”

About Kayothera. Kayothera, Inc. is an early-stage therapeutics company focused on the development of first-in-class, oral, small-molecule inhibitors of the retinoid nuclear receptor pathway. This pathway is pathogenically activated in several cancers as well as multiple cardio-metabolic diseases, including diabetes and obesity. Kayothera is developing three distinct therapies, with KAYO-1732 in development as a disease-modifying treatment that controls blood glucose and lipid levels to prevent multiorgan degeneration in cardiometabolic syndrome and diabetes. KAYO-1609 has completed IND-enabling studies and is in development to treat late-stage and metastatic cancers, including pancreatic, prostate, gastric, and multiple other cancers. Kayothera is also developing a next-generation obesity therapy based on a genetically validated target in the retinoid nuclear receptor pathway. The company was founded based on discoveries from Dr. Mark Esposito’s post-doctoral research at Princeton University and Professor Yibin Kang, PhD. For more information, visit www.kayothera.com.

Significant Progress Made in Advancing the Development of PRN-001-01(Tezampanel);
Appoints New Board Members in Preparation for Launch of Phase 1 Clinical Study

Series B Financing co-led by Accelerator Life Science Partners and ARCH Venture Partners

Seattle, WA — February 01, 2024, Proniras Corporation (“Proniras”), a clinical-stage biotechnology company developing novel small-molecule therapeutics for the treatment of substance use and specialty central nervous system disorders, today announced the successful completion of a $4.65 million funding round, marking a significant milestone in its ongoing Series B financing, which aims to secure up to $9 million. The new Series B funding will be used to support a Phase 1 clinical study of the Company’s lead program PRN-001-01 (tezampanel), a novel modulator of glutaminergic signaling designed to treat patients seeking to recover from opioid addiction and associated conditions. The Phase 1 study will apply the unique and groundbreaking mechanism of action of PRN-001-01 (tezampanel) to reduce brain over-activation in circuits involved in drug withdrawal, addiction, and mental illness without relying on direct stimulation or antagonism of the opioid system. PRN-001-01 (tezampanel) has the potential to provide significant improvement in the treatment of opiate withdrawal syndrome, opioid overdoses, and conversion to long term medications that can help patients addicted to opiates to achieve full remission from their illness.

The Series B financing was co-led by funds managed by Accelerator Life Science Partners and ARCH Venture Partners, with participation from both new and existing investors, including Watson Fund, LP, WRF Capital (the investment vehicle for Washington Research Foundation), and other undisclosed investors.

PRN-001-01 (tezampanel) is a novel small molecule compound that will be the first parenteral therapeutic to be evaluated for the treatment of symptoms associated with opiate withdrawal in a Phase 1 clinical study. Previously investigated for its potential in acute migraine therapy with positive outcomes observed in over 400 human subjects, PRN-001-01 (tezampanel) has also demonstrated encouraging results in various preclinical models of seizure disorders, suggesting the use of the drug in other therapeutic applications with high unmet medical need. Proniras licensed exclusive global rights to PRN-001-01 (tezampanel) from Eli Lilly and Company in 2017.

In conjunction with the Series B financing, Proniras has appointed Steven Gillis, Ph.D. as Chairman and Atul Saran as an independent member to the Company’s Board of Directors. Dr. Gillis and Mr. Saran join Thong Q. Le, Senior Managing Director from Accelerator Life Science Partners, on the Proniras Board of Directors. With the insights gained throughout their professional careers, Dr. Gillis and Mr. Saran will positively impact guidance on the Company’s strategic decision-making and product development plans. In addition, their extensive networks in the industry will help Proniras on multiple fronts.

Dr. Gillis has more than 30 years of experience in the life sciences industry and is focused on the evaluation of new life science technologies and also on the development and growth of ARCH’s biotechnology portfolio companies. In addition to serving as Chairman of the Board for Proniras, he serves as a Director of multiple ARCH biotechnology portfolio companies, including Homology Medicines (FIXX), 48 Bio, Pheast Therapeutics, BE Biopharma, SonoThera, Dispatch Biotherapeutics, and Carrick Therapeutics. He serves as Chairman of Faraday Pharmaceuticals, eGenesis, HiberCell, hC Bioscience, Skylark Bio, Walden Biosciences, OncoResponse, Mozart Therapeutics, Bitterroot Bio, Vilya, TFC Therapeutics, and VBI Vaccines (VBIV). Dr. Gillis also serves as a director of Takeda Pharmaceuticals (TAK). Dr. Gillis was a founder and director of Corixa Corp. and served as the company’s Chief Executive Officer from its inception and as its Chairman from 1999 until its acquisition in 2005 by GlaxoSmithKline. Prior to Corixa, Dr. Gillis held numerous positions at Immunex Corp., including founder and director. Dr. Gillis is a trained immunologist with over 300 peer-reviewed publications in the areas of molecular and tumor immunology.

Atul Saran currently serves as Chief Executive Officer of IntegerBio a privately held biotechnology company focused on developing immunovirology therapeutics. Prior to IntegerBio, Mr. Saran served in various roles at Emergent BioSolutions, a specialty biopharmaceutical company focused on opioid overdose, biodefense, and other public health threats, most recently as Executive Vice President & Chief Scientific and Development Officer with responsibility for the company’s research, development, medical, and regulatory activities as well as corporate strategy and M&A. Before that, Mr. Saran was Emergent’s General Counsel and Corporate Secretary with responsibility for corporate strategy, M&A, legal, public affairs, and government affairs. Prior to Emergent, Mr. Saran served as Senior Vice President and General Counsel at MacroGenics, and before that he held positions of increasing responsibility at MedImmune/AstraZeneca culminating with Senior Vice President of Corporate Development and Ventures for MedImmune and Vice President, Corporate Development & Strategy for AstraZeneca. Mr. Saran previously served as a Director on several boards, including Arriva, VentiRx, and Xencor.

“We are honored to welcome Dr. Steve Gillis as our Chairman and Atul Saran as a new independent director to our Board of Directors,” said Thong Q. Le, Senior Managing Director of Accelerator Life Science Partners, and CEO of Proniras. “Their wealth of knowledge and industry experience will be instrumental in guiding Proniras through its next phase of growth. We look forward to their leadership and contributions as we continue to advance Proniras through the next stages of product development.”

About Proniras Corporation

Proniras Corporation is a clinical-stage biotechnology developing novel small-molecule therapeutics for the treatment of substance use and specialty central nervous system disorders. The Company’s lead program PRN-001-01 (tezampanel) is being clinical developed as a treatment option for opioid withdrawal and related disorders of addiction. Although effective treatments exist for opioid addiction, painful and difficult withdrawal is one of the primary reasons why current treatment fails, and relapse occurs. By alleviating the symptoms associated with opioid withdrawal, Proniras hopes to help patients successfully discontinue opioid usage and sustain an opioid-free life. Proniras was formed in 2017 by Accelerator Life Science Partners. For more information, please visit www.proniras.com.

Automera unlocks the full potential of autophagy-based targeted protein degradation for oncology and other indications

Seattle, WA & SINGAPORE — September 27, 2023 — Accelerator Life Science Partners (ALSP), an early-stage life science accelerator and investment firm that creates and builds next-generation biotechnology companies centered on innovative science, today announced the Series A financing of Automera, a new biotechnology company that focuses on the development of autophagy-targeting chimera small molecules (AUTACs). Augmented with quantum chemistry and artificial intelligence (AI) enabled drug discovery tools, Automera’s AUTACs have the broad potential to rapidly generate novel therapeutic leads for a range of diseases. Automera will initially focus its product development efforts to treat cancer. The company was established by co-founders Associate Professor Michael Lazarou, Loong Wang, and Taiyang Zhang at Talo Labs Pte Ltd. in collaboration with ALSP. Automera has secured $16M in Series A funding, a co-led endeavor by ALSP and ClavystBio, with participation from Singapore-based global investor EDBI, Xora Innovation, and other undisclosed investors. Joining the Board of Directors are Loong Wang, from Automera, Dr. Alice Chen and Thong Q. Le, from ALSP, and Dr. Wen Qi Ho, from ClavystBio.

“ALSP has long been interested in helping to drive biopharma innovation emerging in Singapore,” said Alice Chen, Executive Vice President at ALSP and Automera board member. “As our first investment in Singapore, Automera exemplifies the innovative scientific and translational research ongoing in the region. We plan to leverage ALSP’s unique company-building capabilities in Singapore, and we look forward to working with the broader Singapore community to enhance the platform technologies and early-stage therapeutic programs that Automera will develop. We are honored to co-lead Automera’s Series A financing with our partners at ClavystBio and are excited about expanding Automera’s operations in the months ahead.”

Automera’s AUTAC platform is a next-generation approach to realizing the potential of targeted protein degradation (TPD) as a therapeutic modality. TPD, an emerging field of novel therapeutics, catalyzes the degradation of disease-related proteins while retaining the benefits of small molecules. Automera’s AUTAC platform is designed to generate novel therapies that degrade not only disease-related proteins but also protein aggregates, organelles, and pathogens. AUTACs have the potential to provide a more flexible approach to protein degradation than existing approaches such as proteolysis-targeting chimeras (PROTACs) to trigger protein degradation.

“Automera was founded on the premise that improving health outcomes for people with serious diseases requires new technologies that make it easier, faster, and less expensive to develop safe and effective medicines,” said Loong Wang, co-founder of Automera. “We believe that our AUTAC platform is one such technology, with broad potential across multiple disease indications that are difficult to treat with current therapeutic approaches. We greatly value the business and financial support that ALSP provides and are proud that leading healthcare investors are participating in Automera’s Series A financing. With these resources, Automera is well positioned to deploy our AUTAC platform toward the development of potentially transformative therapies.”

Automera has assembled a distinguished scientific advisory group that includes Associate Professor Michael Lazarou, Automera’s Scientific Co-Founder at the Walter and Eliza Hall Institute of Medical Research (WEHI) and the Monash Biomedicine Discovery Institute, Dr. Nicholas Ktistakis from Babraham Institute, Dr. Gene Yeo from the University of California San Diego (UCSD) and Dr. Kendall Mohler from ALSP.

“ClavystBio is excited to be building new ventures in Singapore through partnerships with young entrepreneurs like Loong Wang and his team at Automera, and smart capital investors like ALSP,” said Wen Qi Ho, Therapeutics Lead at ClavystBio. “Our mission is to accelerate the commercialization of breakthrough innovations like the AUTAC platform for health impact, and we welcome Automera and ALSP to Singapore’s growing life sciences translational community.”

About Automera

Automera is an early-stage company focused on the development of a novel therapeutic approach via autophagy-based targeted protein degradation. Automera aims to leverage its unique understanding of autophagy, drug development capabilities, and access to quantum chemistry and generative AI-enabled insights to enhance its drug development programs. Automera’s AUTAC platform has broad potential across cancer and other disease areas, with oncology being the initial lead program. The company was founded by co-founders Associate Professor Michael Lazarou, Loong Wang, and Taiyang Zhang at Talo Labs Pte Ltd. For more information, visit www.automeratx.com.

 About Accelerator Life Science Partners

Accelerator Life Science Partners (ALSP) is an early-stage life science accelerator and investment firm that creates and builds next generation biotechnology companies centered on innovative science. ALSP catalyzes the development and commercialization of breakthrough biotechnology innovations by providing a holistic toolkit and leveraging its network and entrepreneurial expertise to accelerate the establishment and operation of early-stage biotechnology companies. ALSP’s portfolio companies are backed by renowned life science investors and are comprised of industry-leading, transformative companies, including KayoThera, Inc., Lodo Therapeutics (acquired by Zymergen, now Ginkgo BioWorks), Petra Pharma (acquired by a global pharmaceutical company), Proniras Corporation, and Rodeo Therapeutics (acquired by Amgen Inc.). For more information, please visit www.acceleratorlsp.com.

About ClavystBio

ClavystBio’s mission is to accelerate the commercialization of life science breakthroughs into health impact. We invest and partner with innovators, entrepreneurs, and founders to launch and grow global companies in Singapore. We foster partnerships among academics, industry, and investors, and convene life sciences communities at our collaborative innovation space Node 1. Together, we advance Singapore as a global life sciences translational hub.

ClavystBio was conceived and launched by Temasek to be a venture builder, trusted partner, and keystone to catalyze global health solutions from Singapore. Since our inception in 2022, ClavystBio has committed over US$220m to biotech, diagnostics, and digital health companies, as well as early-stage VC funds. For more information on ClavystBio, please visit www.clavystbio.com.

About EDBI

Investing since 1991, EDBI is a Singapore-based global investor in select high growth technology sectors ranging from Information & Communication Technology (ICT), Emerging Technology (ET), Healthcare (HC) and promising Singapore SMEs in strategic industries. As a value-creating investor, EDBI assists companies achieve their ambitious goals by leveraging our broad network, resources, and expertise. With our growth capital, EDBI supports companies seeking to grow in Asia and globally through Singapore. For more information, visit https://www.edbi.com.

About Xora Innovation
Xora Innovation invests in disruptive, world-changing ventures forged by ambitious founders and powered by compelling scientific breakthroughs. Headquartered in Singapore, Xora is an early-stage deep tech investing platform of Temasek. For more information, visit https://xora.vc.

Contact
Jessica Burback
Email: jburback@acceleratorlsp.com
Phone: 206-957-7302

Unique safety profile positions KAYO-1609 as the first retinoid pathway inhibitor to enter Investigational New Drug (IND)-enabling studies

 Company expects to file IND application with the U.S. Food & Drug Administration (FDA) by the end of 2024

Seattle, WA — September 18, 2023, KayoThera, Inc. (“KayoThera”), an early-stage therapeutics company developing first-in-class, oral, small molecule inhibitors of the retinoid pathway, today announces that it has selected KAYO-1609 as its first drug development candidate. KAYO-1609 has demonstrated a differentiated safety profile across a variety of species with ideal drug-like properties. Preclinical studies in multiple models of cancer support the potential of KAYO-1609 in the treatment of genetically-defined cancers based on the molecule’s mechanism of action. With the nomination of KAYO-1609 as the development candidate, KayoThera is well-positioned for IND-enabling studies, clinical planning, and partnering activities.

Retinoid signaling is known to both inhibit helper CD4 T cells that mediate tumor cell destruction, and cause maturation of regulatory T cells that dampen the immune system’s response to cancer. Retinoid signaling further forces differentiation of monocytes into tumor-associated macrophages that can promote tumor growth and metastasis. Collectively, these activities reduce the immune system’s ability to detect and eliminate malignant cells. Inhibition of the amplified retinoid signaling found in unique tumor types could potentially restore or enhance the anti-tumor immune activity, enabling a unique approach to cancer immunotherapy in patients with few therapeutic options.

“Despite its known roles in blunting the body’s anti-tumor immune defense, previous efforts to develop retinoid signaling antagonists have failed due to unacceptable toxicity in preclinical development,” said Mark Esposito, Ph.D., vice president of R&D, and co-founder of KayoThera. “The discoveries on which KayoThera was founded enable the development of orally available, small molecule inhibitors of this important signaling pathway that have very favorable safety profiles. The preclinical safety and efficacy data generated to date in studies of KAYO-1609 are exceptionally promising, and we intend to move as rapidly as possible toward initiating human clinical trials of this novel molecule in genetically-defined cancers.”

KAYO-1609 has demonstrated excellent safety and tolerability, with no dose-limiting toxicities observed in studies across five different species. Preclinical studies also have identified a dose-responsive biomarker for target engagement that is expected to facilitate the IND-enabling studies. KayoThera expects to initiate IND-enabling studies of KAYO-1609 in the third quarter of 2023 and anticipates filing an IND application with the FDA by the end of 2024. Following acceptance of the IND, the company plans to initiate a human clinical trial of KAYO-1609 in patients with genetically-defined cancers that are most likely to demonstrate clinical benefit based on the mechanism of action of KAYO-1609.

“The selection of KAYO-1609 as our lead oncology development candidate is the most recent demonstration of the significant progress, we have made over the past few months to position KayoThera for success,” said Kendall Mohler Ph.D., chief development officer and board member of KayoThera. “In May we announced the expansion of our pipeline into the cardiometabolic disease space, with a focus on Type 2 diabetes and in July we strengthened our financial position with multiple grants and the expansion of our Series A financing. Taken together, we now have the financial resources to advance exciting and differentiated candidates in cancer and Type 2 diabetes, diseases with significant unmet clinical need that are associated with significant mortality and morbidity. We are committed to developing retinoid signaling pathway inhibitors as novel therapeutic modalities that can improve outcomes for patients living with these and other serious diseases.”

About KayoThera Inc
KayoThera, Inc. is an early-stage therapeutics company focused on the development of first-in-class, oral, small molecule inhibitors of the retinoid pathway. This pathway plays a critical role in a variety of serious diseases, including cardiometabolic diseases and cancer. KayoThera is developing therapies to treat diabetes and late-stage and metastatic cancers including breast, lung, pancreatic, colorectal, brain, and kidney cancers. The company was founded based on discoveries from Dr. Mark Esposito’s post-doctoral research at Princeton University and professor Yibin Kang, PhD. For more information, visit www.kayothera.com.

About Accelerator Life Science Partners

Accelerator Life Science Partners (ALSP) is an early-stage life science accelerator and investment firm that creates and builds next generation biotechnology companies centered on innovative science. ALSP catalyzes the development and commercialization of breakthrough biotechnology innovations by providing a holistic toolkit and leveraging its network and entrepreneurial expertise to accelerate the establishment and operation of early-stage biotechnology companies. ALSP’s portfolio companies are backed by renowned life science investors and are comprised of industry-leading, transformative companies, including KayoThera, Inc., Lodo Therapeutics (acquired by Zymergen, now Ginkgo BioWorks), Petra Pharma (acquired by a global pharmaceutical company), Proniras Corporation, and Rodeo Therapeutics (acquired by Amgen Inc.).  For more information, please visit www.acceleratorlsp.com.

Contact
Jessica Burback
Email: jburback@acceleratorlsp.com
Phone: 206-957-7302

New funding comprised of grants from the National Institutes of Health, The Andy Hill Cancer Research Endowment (CARE) Fund, and additional Equity Funding

 Additional funding will support advancement of KayoThera’s development programs in diabetes and oncology

 Seattle, WA — July 19, 2023, KayoThera, Inc. (“KayoThera”), an early-stage therapeutics company developing first-in-class, oral, small molecule inhibitors of the retinoid pathway, today announces the strengthening of its financial resources through multiple grant awards and expansion of its previously announced Series A financing. The expanded financing was led by Accelerator Life Science Partners (ALSP), who also provided Series A funding for KayoThera. With the additional $5.2 million in new equity investments, non-dilutive grant funding, and initial seed funding from the New Jersey Health Foundation, KayoThera’s total financial support to date is $14 million.

KayoThera has received grant funding from the National Institutes of Health Small Business Innovation Research (SBIR) program^1,2 and the Andy Hill Cancer Research Endowment (CARE) Fund³. In addition, KayoThera secured additional Series A financing in an expansion round led by ALSP that also included participation from BioAdvance, Pier 70 Ventures, and WRF Capital. The additional resources secured by KayoThera will further support the Company’s therapeutic development programs.

“The grant awards are important validation of our development programs, and the expanded Series A financing reflects our investor’s confidence in our ability to innovate oral, small molecule inhibitors of the retinoid pathway,” said Mark Esposito, Ph.D., Vice President, R&D, and Co-Founder of KayoThera. “With the additional financial resources announced today, we are well-positioned to advance our lead diabetes and oncology programs, each of which has first-in-class potential in disease indications with large patient populations and significant unmet medical need.”

“Our belief that KayoThera offers a compelling investment opportunity and has the potential to transform the treatment of serious metabolic diseases and cancer was the key driver for our initial investment in the Company,” said Thong Q. Le, Senior Managing Director of ALSP and CEO of KayoThera. “Since the initial Series A financing in March 2022, KayoThera has made significant progress in advancing its core technology and development programs. The additional funding announced today will allow the Company to continue making exciting advances in innovating small molecule inhibitors of the retinoid pathway and position itself for strategic partnering activity that will further help realize the commercial and clinical value of its science.”

The retinoid pathway is known to play critical roles in several serious diseases. For example, the clinical use of retinoid activators leads to both adverse cardiometabolic events such as hyperlipidemias as well as increased cancer rates and faster cancer progression. KayoThera’s unique approach to drugging this pathway is based on biology discovered at Princeton University and represents the first platform to create drug-like retinoid inhibitors.

References

¹ NIH NIDDK (National Institute of Diabetes and Digestive and Kidney Diseases) R43DK136420. Grant title: Development of first-in-class antagonists of the retinoid pathway as novel oral therapies for Type 2 Diabetes.
²NIH NCI (National Cancer Institute) R43CA278127. Grant title: Development of first-in-class antagonists of the retinoid pathway as novel oral immunotherapies for solid cancers.
³The Andy Hill Cancer Research Endowment (CARE) Fund FY23-LS-05. Grant title: Development of a First-in-Class Immunotherapy to Treat Advanced Solid Tumors Through IND-Enabling Safety Studies.
The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

About KayoThera, Inc
KayoThera, Inc. is an early-stage therapeutics company focused on the development of first-in-class, oral, small molecule inhibitors of the retinoid pathway. This pathway plays a critical role in a variety of serious diseases, including cardiometabolic diseases and cancer. KayoThera is developing therapies to treat diabetes and late-stage and metastatic cancers including breast, lung, pancreatic, colorectal, brain, and kidney cancers. The company was founded based on discoveries from Dr. Mark Esposito’s post-doctoral research at Princeton University and professor Yibin Kang, Ph.D. For more information, visit www.kayothera.com.

About Accelerator Life Science Partners
Accelerator Life Science Partners is an early-stage life science accelerator and investment firm that creates and builds next generation biotechnology companies centered on innovative science. ALSP catalyzes the development and commercialization of breakthrough biotechnology innovations by providing a holistic toolkit and leveraging its network and entrepreneurial expertise to accelerate the establishment and operation of early-stage biotechnology companies. ALSP’s portfolio companies are backed by some of the world’s leading life science investors and are comprised of industry-leading, transformative companies, including Petra Pharma (acquired by a global pharmaceutical company), Rodeo Therapeutics (acquired by Amgen Inc.) and Lodo Therapeutics (acquired by Zymergen). For more information, please visit www.acceleratorlsp.com.

 About Andy Hill Cancer Research Endowment (CARE) Fund
The Andy Hill Cancer Research Endowment (CARE) Fund invests in public and private entities to promote cancer research in Washington. Through research grants and strategic partnerships, the CARE Fund aims to improve health outcomes by advancing transformational research in the prevention and treatment of cancer. The Washington State Legislature created the CARE Fund in 2015 and this public investment in cancer research is maximized by private and nonstate matching funds. For additional information, please visit www.wacarefund.org.

About BioAdvance
BioAdvance is a $85 million early-stage life sciences fund with a focus in the mid-Atlantic region, investing up to $1.5 million initially and up to $5 million over the lifetime of companies that have the potential to improve human health. Since making its first investments in 2003, BioAdvance has committed more than $50 million in funding to approximately 100 organizations developing more than 160 products in the therapeutics, medtech, diagnostics, research tools and digital health sectors. BioAdvance portfolio companies have leveraged $3.7 billion in subsequent capital, including proceeds from acquisitions. Eleven products have received FDA approval. For more information, please visit www.bioadvance.com.

 About Pier 70 Ventures
Pier 70 Ventures is a US-based venture capital firm investing in impact-driven innovation. The firm’s debut fund is the Pier 70 INpact Fund. Looking for brilliance beyond the usual places, Pier 70 Ventures finds and funds technologies with the potential to disrupt the future of healthcare. We invest in talented entrepreneurs pushing humanity forward with innovation. We VENTURE BOLDLY by empowering bright, enthusiastic leaders with capital, mentorship, and access to our global networks. For additional information, please visit www.pier70ventures.com.

About WRF and WRF Capital
Washington Research Foundation (WRF) supports research and scholarship in Washington state, with a focus on life sciences and enabling technologies. WRF was founded in 1981 to assist universities and other nonprofit research institutions in Washington with the commercialization and licensing of their technologies. WRF is one of the foremost technology transfer and grant-making organizations in the nation, having earned more than $445 million in licensing revenue for the University of Washington and providing over $145 million in grants to the state’s research institutions to date.

WRF Capital, the investment vehicle for Washington Research Foundation, has backed 122 local startups since 1996. Returns support the Foundation’s investment and grant-making programs. For additional information, please visit www.wrfseattle.org.

Contact
Jessica Burback
Email: jburback@acceleratorlsp.com
Phone: 206-957-7302