BioTheryX Announces $35 Million Series D Financing

SAN DIEGO, CA, July 28, 2020 — BioTheryX, Inc., a clinical stage biotechnology company harnessing the power of protein modulation to treat difficult diseases, today announced it has closed a $35 million Series D financing. The round was led by MSD Partners, L.P.

“We are pleased to have closed this financing that will enable us to accelerate expansion of our ongoing clinical trial of BTX-A51, which I believe may ultimately become one of the most significant innovations in the past 40 years for AML patients and their families,” said David Stirling, Ph.D., Chairman and CEO of BioTheryX. “Additionally, and equally as important, this funding will support our ongoing work in protein modulation and degradation utilizing proprietary Protein Homeostatic Modulators (PHMs™) and PHM®-based Proteolysis Targeting Chimeras, where I believe we have a superior probability of success driven by the large diversity of our molecular binders that afford us advanced structural control in drug design.”

In addition to general corporate development purposes, proceeds from the financing will be used to accelerate the timeline of the BTX-A51 Phase 1 clinical trial through the addition of clinical sites beyond Memorial Sloan-Kettering Cancer Center, continue IND-enabling studies for therapeutic candidates utilizing both PHMs™ and PHM®-based Proteolysis Targeting Chimeras, and support more rapid development of the Company’s pre-clinical pipeline.

Louis J. DeGennaro, Ph.D., President and CEO, The Leukemia & Lymphoma Society (LLS), and BioTheryX Board Member, stated, “LLS has been pioneering breakthrough blood cancer research since 1949, and through innovative initiatives like our Therapy Acceleration Program® (TAP) we’re doing more than any cancer nonprofit to advance cutting-edge research and cures. As the first equity investment made through TAP, I am pleased to see BioTheryX’s continued progress on promising approaches to treat blood cancers that may make meaningful differences in patients’ lives.”

In conjunction with the financing, Robert Platek, a Partner and Portfolio Manager at MSD Partners L.P., will be joining BioTheryX’s Board of Directors.

Raymond James’ Alex Brown division served as the sole placement agent for the financing.

About BioTheryX, Inc.
BioTheryX is a clinical-stage biopharmaceutical company focused on restoring protein homeostasis, including protein degradation and modulation, and multi-kinase inhibition to develop treatments intended to extend and improve the quality-of-life of patients with life-threatening diseases. Our principal technology platform centers on targeted protein degradation, as well as PHM® ‘molecular glues’ that enable the design of small molecules to regulate protein equilibrium. This technology is designed to utilize the body’s own protein disposal system to selectively degrade and remove disease-causing proteins. It has potential applicability to a broad range of diseases, including targets that have to date been considered ‘undruggable’. For more information, please visit www.biotheryx.com.

About MSD Partners, L.P.
MSD Partners, L.P. is an SEC-registered investment adviser that was formed in 2009 by the principals of MSD Capital, L.P. to enable a select group of investors to invest in strategies that were developed by MSD Capital (the private investment firm for Michael Dell and his family). MSD Partners utilizes a multi-disciplinary investment strategy focused on maximizing long-term capital appreciation by making investments across the globe in the equities of public and private companies, credit, real estate and other asset classes and securities. MSD Partners is headquartered in New York with an additional office in Santa Monica. Additional information regarding MSD Partners may be found at msdpartners.com.

Media Contact for BioTheryX
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BioTheryX, Inc. Announces the Appointment of Professor Yinon Ben- Neriah, M.D., Ph.D., to its Scientific Advisory Board

Accomplished scientist who created BioTheryX’s promising lead clinical candidate targeting AML adds significant expertise in oncology & immunology to distinguished SAB

CHAPPAQUA, N.Y., March 6, 2020 /PRNewswire/ — BioTheryX, Inc., a clinical stage biotechnology company harnessing the power of protein modulation to treat difficult diseases, today announced the appointment of Professor Yinon Ben-Neriah, M.D., Ph.D., to its distinguished Scientific Advisory Board (SAB) comprised of key opinion leaders in ubiquitin-mediated protein degradation, immunology and oncology. Dr. BenNeriah is Professor of Immunology and Cancer Research at the Lautenberg Center of Immunology of the Hebrew University, Hadassah Medical School in Jerusalem, Israel.

David Stirling, Ph.D., CEO of BioTheryX stated, “As a company dedicated to the development of novel approaches to protein modulation for targeting numerous diseases, we are fortunate to welcome Dr. BenNeriah to our SAB. He and his research team’s discovery of BTX-A51 and its unique mechanism of action, potentially inhibiting several key oncogenic targets simultaneously, eradicating leukemic stem cells, and driving activation of the key tumor suppressing oncogene p53, may be one of the most significant innovations in the past 40 years for AML patients and their families.”

Dr. Ben-Neriah said, “I am honored and proud to be joining the SAB of BioTheyrX. Having the privilege of interacting with the entire team for the past 5 years, I must say it is truly a visionary company. I believe they are well on their way to improve human health, and hopefully I will be able to contribute to their vision.”

Dr. Ben-Neriah received his M.D. from Tel Aviv University and Ph.D. from the Weizmann Institute of Science, and was a postdoctoral fellow in Nobel Laureate Dr. David Baltimore’s lab at the Whitehead Institute-MIT. His research work focuses on signaling pathways regulating innate immunity and inflammation, particularly in the context of cancer. His lab deciphered key steps in the activation of the NFkB and Wnt signaling pathways and studied them in animal models of cancer. Dr. Ben-Neriah is an elected member of the European Molecular Biology Organization (EMBO), a spokesman of the International German Israeli Graduate Student program SignGene, Chair of the advisory board of the BIOSS Excellence Center of Freiburg University (Germany), and Adjunct Professor in Shanghai Jiao Tong University.

In December 2019, Dr. Ben-Neriah was awarded the EMET Prize, also known as ‘Israel’s Nobel Prize’, in the Life Sciences category for his “important scientific contribution to understanding immune signaling processes and decoding abnormal signal transmission mechanisms in cancer cells”. In addition to this prestigious honor, Dr. Ben-Neriah has also been awarded the Rappaport Prize, for his biomedical research; Columbia University’s Schaefer Research Scholar Award; the Teva Founders Prize, and the Landau Prize.

BioTheryX Announces the Initiation of Patient Dosing in a First-in-Man Phase 1 Clinical Trial of BTX-A51

Study is evaluating the safety/tolerability of BTX-A51 in patients with relapsed/refractory acute myeloid leukemia (AML), as well as high risk myelodysplastic syndrome patients.

CHAPPAQUA, N.Y., Jan. 16, 2020 /PRNewswire/ — BioTheryX, Inc., a clinical stage biotechnology company creating new classes of compounds based on multi-kinase inhibition and targeted protein degradation, today announced the initiation of patient dosing in its first clinical program. The Phase 1 study of BTX-A51, a small molecule, oral multi-kinase inhibitor will evaluate the safety, pharmacokinetics and tolerability of BTX-A51 in patients with relapsed/refractory AML, as well as high risk myelodysplastic syndrome patients.

BTX-A51 appears to block a specific leukemic stem cell target (CK1-alpha) as well as super enhancer targets (CDK7/CDK9) preventing transcription of key oncogenic genes. BTX-A51 has demonstrated remarkable preclinical animal efficacy implying the eradication of AML stem cells and the potential for use in multiple malignancies.

“As I stated when the Investigational New Drug application for BTX-A51 was accepted by the FDA, the novel mechanism of BTX-A51 may become one of the most important new treatments for AML in the last 40 years, and has the potential to significantly improve the lives of AML patients and their families,” said David Stirling, Ph.D., CEO of BioTheryX.

In addition to its multi-kinase inhibition program, BioTheryX’s other technology platform is in the field of targeted protein degradation. This technology utilizes the body’s own protein disposal system to selectively degrade and remove disease-causing proteins. It has potential applicability to a very broad range of disease targets, including a wide range of targets that have to date been considered “undruggable.”

In this area, BioTheryX’s preclinical assets include a large and growing library of novel, small molecule, orally available, cereblon-binding targeted protein degraders which BioTheryX has termed Protein Homeostatic Modulators (“PHMs®”). These IP-protected compounds are biologically active against a number of high value therapeutic targets in oncology, inflammation and other diseases. In addition to the therapeutic potential of these “molecular glue” molecules in their own right, these compounds also have a broad range of molecular orientations when bound to cereblon, providing a new level of structural control in the creation of bifunctional chimeric molecules that degrade high-value targets with great specificity. Recognizing this potential, BioTheryX has created a library of PHM-linked, biologically active chimeric molecules, including several that degrade the oncogenic targets of BTX-A51, thus dovetailing BioTheryX’s two major programs.