BioTheryX Announces FDA Approval of IND Application to Initiate Phase 1 Clinical Trial in Relapsed or Refractory Acute Myeloid Leukemia

Expect Enrollment of Phase 1 Trial to Begin in the Third Quarter of 2019

CHAPPAQUA, N.Y., May 29, 2019 /PRNewswire/ — BioTheryX, Inc. (“BioTheryX”), a biotechnology company creating new classes of drugs based on multi-kinase inhibition and targeted protein degradation, today announced that the U.S. Food and Drug Administration (“FDA”) has cleared BioTheryX’s investigational new drug application (“IND”) for BTX-A51, an oral multi-kinase inhibitor, for the treatment of patients with relapsed/refractory acute myeloid leukemia (“AML”) or high-risk myelodysplastic syndrome (“MDS”). BioTheryX anticipates dosing of first patients to begin during the third quarter of this year.

BTX-A51 is a small molecule, multi-kinase inhibitor that 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 This molecule has demonstrated remarkable preclinical animal efficacy implying the eradication of AML stem cells, and the potential for use in multiple malignancies.

“The acceptance of our IND is a major milestone in transitioning BioTheryX from a pre-clinical to a clinical-stage biotechnology company,” said David Stirling, Ph.D., CEO of BioTheryX. “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. In short, BTX-A51 seeks to address a truly unmet medical need in very novel ways.”

In addition to the multi-kinase program described above, BioTheryX’s other technology platform is in the field of protein homeostasis, also known as targeted protein degradation. This technology uses 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 pre-clinical assets include a large and growing library of novel small molecule, cereblon-binding targeted protein degraders, which BioTheryX has termed Protein Homeostatic Modulators (PHM®). 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.

BioRap and BioTheryX Sign Strategic Collaborative Agreement for the Development of Novel Protein Degradation Compounds for the Treatment of Cancer and Immune Disorder

CHAPPAQUA, N.Y., Sept. 08, 2006 /PRNewswire/ — BioTheryX, Inc. (BTX), is developing next-generation drugs that selectively regulate the stability of disease-causing proteins to treat a broad range of diseases, including cancer and immune disorders. BioTheryX’s innovative approach to drug discovery exploits the exquisite specificity of the ubiquitin proteasome system (UPS). BioTheryX has entered into a strategic collaboration with Nobel laureate Dr. Aaron Ciechanover at the Rappaport Institute to employ advanced ubiquitin-directed proteomics to assist in elucidating direct protein targets and prognostic biomarkers for BTX’s proprietary small molecule Protein Homeostatic Modulators™ (PHMs™). This powerful new class of small molecule drugs will selectively modulate key therapeutically validated protein targets in oncology, immunology, inflammation, as well as emerging targets in the fields of immune-oncology and cancer stem cells.

Dr. Ciechanover is a Distinguished Research Professor at the Rappaport institute for Biomedical Research-Technion-Israel Institute of Technology, and BioRap Technologies is its technology transfer arm. Professor Ciechanover, together with colleagues Drs. Avram Hershko and Irwin Rose, won the 2004 Nobel Prize in chemistry for the discovery of ubiquitin mediated protein degradation. Along with Hershko, he was also recognized with the prestigious Albert Lasker Award for Basic Medical Research (2000). Additionally, among many world renowned organizations, Dr. Ciechanover is a member of the USA National Academies of Sciences (NAS) and Medicine (NAM).

The BioTheryX team, led by Celgene Corp. founder David Stirling, were the developers of the remarkable IMiD® family of compounds while at Celgene. It is now known that the IMiDs® (a franchise of blood cancer compounds now exceeding $35 billion) work by promoting ubiquitin degradation of key protein targets in oncology, giving the BTX team the most commercial experience in the protein regulation field.

David Stirling, Chief Executive Officer, BioTheryX, commented, “BioTheryX is pleased and excited to be working with Professor Ciechanover, the “father” of the ubiquitin-proteasome field, to advance these exciting compounds into the clinic. Our proprietary PHMs are orally available, small molecule compounds which display potent and selective biological activities and excellent safety profiles. We believe BTX’s PHMs will have clinical utility in both cancer and immune disorders. Indeed, the lead compound in this series promotes selective degradation of a well-documented, clinically validated drug target more effectively than currently approved drugs, and this trajectory could represent a significant fast track to the clinic.”

Professor Aaron Ciechanover, Tumor and Vascular Biology Research Center, The Rappaport Faculty of Medicine and Research Institute, Technion-Israel Institute of Technology, commented, “The platform discovered by BioTheryX is exciting and broad, and can be applied to any substrate at will. It opens a wide window to the development of novel therapeutic modalities to a broad array of diseases, including malignancies and neurodegenerative disorders. Therefore, we are excited to be part of this novel and promising emerging approach.”

Yissum and BioTheryX Sign Licensing Agreement for the Development of Next-Generation Protein Degradation and Immunomodulatory Treatment for Hematological Cancers

CHAPPAQUA, N.Y., May 04, 2016 /PRNewswire/ — Yissum Research Development Company of the Hebrew University of Jerusalem announced today that it had signed an exclusive world-wide licensing and research agreement with BioTheryX, Inc., developer of novel protein degradation and immunomodulatory drugs for cancer and immune dysfunction, for the development and commercialization of drug candidates representing first-in-class therapy for both hematologic and solid malignancies. Financial terms of the license were not disclosed.

The novel technology was invented by Yinon Ben-Neriah, MD, PhD, Blumenthal Professor of Cancer Research, Lautenberg Center for Immunology, Hebrew University-Hadassah Medical School, with generous support by AMRF (The Dr. Miriam and Sheldon G. Adelson Medical Research Foundation). Dr. Ben-Neriah’s and his team showed that inhibition of the clinically validated enzyme CKI-alpha, induces several tumor suppressor pathways, including a new type of DNA damage response and p53 activation. This provides a novel approach to treat a wide range of cancers, in particular selective types of hematological malignancies.

Prof. Yinon Ben-Neriah has recently received the 2016 Rappaport Prize for excellence in biomedical research, among others, for his ground breaking research on the relationship between chronic inflammation and cancer and the treatment of leukemia.

Based on their complementing expertise, BioTheryX and Yissum have agreed to join forces and focus on the selection and advancement of clinical candidates designed to inhibit CKI-alpha. Initial clinical focus for these candidates will be selective subtypes of myelodysplastic syndrome and acute myeloid leukemia that are not responsive to available cancer therapy.

In preclinical studies of acute leukemia, these clinical candidates showed a far greater therapeutic potential than any previously reported studies. Treatment of genetically-modified leukemic mice modeling poor-prognosis human acute myeloid leukemia, abolished the disease signs in the majority of the animals, without compromising the normal bone marrow, demonstrating that CKI-alpha inhibitors have a large therapeutic window and are unique in their capacity to specifically eliminate leukemia stem cells, including otherwise treatment-resistant stem cells – a strong indication of cancer cure.

Yaacov Michlin, President and Chief Executive Officer, Yissum, commented, “The treatment that was developed in Prof. Ben-Neriah’s lab is very different from other available therapies, both in its mechanism of action and its ability to eliminate leukemic stem cells, and thus in its therapeutic potential. In light of the successful pre-clinical studies, we believe that it offers a significant breakthrough, and we are very pleased to partner with BioTheryX in the development of new drug candidates for potential treatment of a variety of hematological indications. We believe that the combined know-how and research efforts of the teams at BioTheryX and the Hebrew University will facilitate new drug development, leading to significant advancement in the therapy of this class of devastating cancer diseases.”

David Stirling, Chief Executive Officer, BioTheryX, commented, “BioTheryX is particularly pleased to partner with the Hebrew University on this important project. Our team, having developed the remarkable IMiD® family of drugs while at Celgene, which have improved the quality of life of so many cancer patients and their loved ones, brings a deep wealth of experience to developing novel cancer therapies that modulate protein degradation and the immune system to target cancer causing proteins for destruction. We believe that these unique drug candidates from the Hebrew University will not only bring new treatment modalities to a variety of hematological cancers, but may provide the only option for those patients that relapse and become resistant to currently available therapies.”

The Leukemia & Lymphoma Society® Provides Equity Financing to BioTheryX, Inc. to Accelerate Drug Development for Blood Cancer

WHITE PLAINS, N.Y. and CHAPPAQUA, N.Y., Nov. 30, 2010 /PRNewswire/ — The Leukemia & Lymphoma Society (LLS) and BioTheryX, Inc. announced today that they have entered into a partnership to accelerate the development of new therapies for hematological malignancies with an initial focus on acute myelogenous leukemia (AML). The partnership marks the first time that LLS has provided funds through the acquisition of equity.

LLS has provided $4.5 million in financing through its Therapy Acceleration Program to support preclinical studies needed for an Investigational New Drug Application (IND) and a clinical trial of BTX-10504, BioTheryX’s lead compound. The investment also supports the development of BTX-10504 analogs and follow-on compounds.

The activity of BTX-10504 in AML was identified simultaneously by BioTheryX and by a LLS-funded academic researcher. Joint validation of the potential of BTX-10504 to treat AML and other blood cancers led to the partnership.

“Our alliance with BioTheryX represents the latest step in the evolution of LLS toward becoming a fully integrated partner in the process of blood cancer therapy development,” said John Walter, LLS chief executive officer. “The project capitalizes on research funded through our biomedical research grant program and recognizes the important role of industry in bringing new therapies to the patients we care about.”

“We are honored that LLS recognizes the potential of BTX-10504 and is making a substantial investment to bring this important new potential product to AML patients,” said David Stirling, PhD, CEO of BioTheryX. “To achieve this ambitious goal, BioTheryX has assembled an interdisciplinary team of leading research scientists and clinicians in the USA, Canada, Asia, and Europe who share a strong commitment and sense of urgency to bring treatments to patients.”

Louis J. DeGennaro, PhD, LLS Chief Mission Officer, will join the BioTheryX board of directors as a result of the partnership.

About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the world’s largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin’s disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world and provides free information and support services. Founded in 1949 and headquartered in White Plains, NY, LLS has chapters throughout the United States and Canada. To learn more, visit or contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 6 p.m. ET.

About BioTheryX, Inc.
BioTheryX is led by CEO Dr. David Stirling, a founder and former Chief Scientific Officer of Celgene Corporation. While at Celgene, Dr. Stirling was the chief architect of Celgene’s successful Thalidomide strategy, as well as the analog program which led to FDA approval of Revlimid in 2005. Incorporated in 2007, and headquartered in Chappaqua, NY, BioTheryX leverages its extensive experience and proven commercial success to deliver efficacious therapies to patients with unmet medical needs more quickly and less expensively than the biomedical industry traditionally can and does. The company is exploiting enhanced biology-driven models to identify effective core molecules that can be rapidly exploited using focused and prioritized chemistry approaches coupled with clinically proven translational approaches targeted for cancer and immune dysfunction. To learn more, visit or contact BioTheryX President Lawrence Zaslow at 914-490-5425.

Lawrence Zaslow
(914) 490-5425
[email protected]

Andrea Greif
The Leukemia & Lymphoma Society
(914) 821-8958
[email protected]