BioTheryX to Present at the Barclays Global Healthcare Conference and the Credit Suisse Solebury Trout Healthcare Innovators Conference

SAN DIEGO, March 3, 2021 /PRNewswire/ —

BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced that Rob Williamson, President and CEO, will present a company overview and will participate virtually in the following investor conferences:

  • Barclays Global Healthcare Conference
    • Tuesday, March 9th, 4:10 pm EST
  • Credit Suisse Solebury Trout Healthcare Innovators Private Company Showcase
    • Thursday, March 11, 2021, 12:20 pm PST

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, 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 and engage with us on LinkedIn.

Contact

Media and Investor Contact:
Amy Conrad, Juniper Point
(858) 366-3243
[email protected]

BioTheryX to Participate in Upcoming Scientific and Investor Events

SAN DIEGO, Feb. 15, 2021 /PRNewswire/ — BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced that the company will participate virtually in the following scientific and investor events:

  • Targeted Protein Degradation & Proteolysis Targeting Chimeras Symposium
    • Tuesday, February 16, 2021, 16:30 GMT
      • Aparajita Hoskote Chourasia, Ph.D., Senior Director of Biology, will give a presentation entitled, “Developing Novel Cereblon-Directed PHMs® as First-in-Class Targeted Protein Degradation Therapies”.
  • North American Protein Degradation Congress
    • Tuesday, February 23, 2021, 3:00 pm ET
      • Leah Fung, VP, Medicinal Chemistry, will give a presentation entitled, “BioTheryX is Leveraging Novel Cereblon-Directed Molecular Glues to Discover First-in-Class TPD Therapies for Oncology”.
  • LifeSci Partners Precision Oncology Day
    • Wednesday, February 17, 2021, 1:00 PM ET
      • Rob Williamson, President and CEO, will present an overview on the company’s targeted protein degradation platform and the management team will participate in 1x1s with investors.
  • BMO BioPharma Spotlight Series: Targeted Protein Degradation and Other Next Gen Protein Technologies
    • Thursday, February 18, 2021, 10:30 AM ET
      • Rob Williamson, President and CEO, will participate in a panel entitled “Ligating Competitors for Collaboration in the Protein Degradation Space”, moderated by BMO analysts Matt Luchini and Nick Lenard.
      • Additional panel participants include Ian Taylor, CSO, Arvinas; Stew Fisher, CSO, C4 Therapeutics; and Jared Gollob, CMO, Kymera. In addition to the panel, the BioTheryX management team will also participate in 1x1s with investors during the event.
  • 2021 SVB Leerink Global Healthcare Conference
    • Tuesday, February 23, 2021
      • The BioTheryX management team will participate in 1x1s with investors.

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, 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 and engage with us on LinkedIn.

BioTheryX Appoints Jamie Donadio as Chief Financial Officer

SAN DIEGO, Jan. 7, 2021 /PRNewswire/ —

BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced the appointment of Jamie Donadio as Chief Financial Officer.  Mr. Donadio brings more than twenty years of experience as a financial leader to BioTheryX, with a strong background in capital raising, financial strategy and financial planning.

“Jamie is a proven leader who has significant expertise in building and growing a company’s financial infrastructure and I’m thrilled to welcome him as Chief Financial Officer,” said Rob Williamson, President and CEO of BioTheryX.  “Jamie’s recent experience in growing Mirati, coupled with his track record of successful financings for companies during key periods of growth, will be invaluable to us as we seek to fund our pipeline of targeted protein degraders and modulators.”

Mr. Donadio joins BioTheryX from Mirati Therapeutics, Inc., where he served most recently as Senior Vice President and Chief Financial Officer and was responsible for all corporate finance and accounting activities since 2013.  While at Mirati, he played an integral executive role during a period of significant growth including expansion to over 100 employees and an increase in market capitalization from less than $100 million to over $4 billion.  Prior to Mirati, Mr. Donadio spent twelve years at Amylin Pharmaceuticals, Inc., where he was most recently responsible for corporate finance activities including financial planning and analysis, treasury and capital formation strategy.  Before Amylin, Mr. Donadio held financial roles at Novatel Wireless and Ernst & Young.

Mr. Donadio added, “I couldn’t imagine a better time to join Rob and the team at BioTheryX.  I look forward to leveraging my experience building and leading corporate finance teams to help BioTheryX execute on its bold strategy and solidify the company as a pioneer in targeted protein degradation.”

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, 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 and engage with us on LinkedIn.

Contact

Media and Investor Contact:
Amy Conrad, Juniper Point
(858) 366-3243
[email protected]

BioTheryX Appoints Robert F. Williamson, III as President and Chief Executive Officer

SAN DIEGO, Jan. 7, 2021 /PRNewswire/ — BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced the appointment of experienced biotechnology leader Robert F. Williamson, III as President and Chief Executive Officer and a member of the BioTheryX Board of Directors.

“Rob is an extremely talented biotech executive with more than two decades of experience in building and financing companies through critical phases of growth.  His proven track record in leading high-functioning teams and executing partnerships and financings, position us well to advance our pipeline of protein degraders and modulators to create life-saving medicines for patients,” said David Stirling, Ph.D., Executive Chairman and Chief Development Officer of BioTheryX.  “The Board and I welcome him as CEO, and we are excited to have him lead our company as we build on our expertise in protein modulation based drug development.”

Prior to joining BioTheryX, Mr. Williamson served as Chief Executive Officer of both PharmAkea and ATXCo, oncology and fibrosis drug development companies financed through a partnership with Celgene, through PharmAkea’s acquisition by Galecto and ATXCo’s acquisition by Blade Therapeutics.  Prior to PharmAkea and ATXCo, Mr. Williamson was Chief Executive Officer of Arriva Pharmaceuticals, President and COO of Eos Biotechnology, which was acquired by Protein Design Labs, and COO of DoubleTwist, Inc. through its acquisition by Merck and Hitachi. In addition to his executive leadership roles, Mr. Williamson has served as a Board member for privately held and publicly traded companies.  Most notably, Mr. Williamson served as a member of the Board of Directors of Pharmasset, Inc., where he helped finance, grow, and advance the company to the public markets through its acquisition by Gilead in 2011 for $11 billion.  He also served as a member of the Board of Directors of Progen Pharmaceuticals.  Currently, Mr. Williamson serves as Chairman of HAYA Therapeutics SA and as a member of the Board of Directors of the Coulter Center in Miami.

Prior to his management roles in industry, Mr. Williamson was a partner with The Boston Consulting Group and was a research assistant for the Federal Reserve Board.  He received a B.A. in Economics from Pomona College and an M.B.A. from Stanford GSB.

“I am thrilled to assume this role and for the future of BioTheryX, which is poised for rapid growth in the fast-paced category of protein degradation and modulation,” said Mr. Williamson.  “The BioTheryX team is truly exceptional, composed of decorated scientists who created one of the most successful anti-cancer drug franchises in history and are the pioneers in the protein degradation space.  I look forward to leading the team to bring this unmatched expertise to bear and to advance our clinical-stage program for liquid and solid tumors, and to expand our PHM® platform and develop first-in-class therapies for oncology, inflammatory and other diseases.”

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 core technology platform centers on targeted protein degradation with novel PHM ‘molecular glues,’ as well as PHM-based proteolysis targeting chimeras that restore 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 and engage with us on LinkedIn.

CONTACT: Media and Investor Contact: Amy Conrad, Juniper Point, (858) 366-3243, [email protected]

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
[email protected]

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.

BioTheryX, Inc. Announces the Appointment of BioPharma Finance Veteran Peter N. Crnkovich to Board of Directors

Part of BioTheryX’s previously announced plan to continue building out its Board of Directors with key industry leaders now that Company has transitioned from preclinical to clinical with lead Acute Myeloid Leukemia (“AML”) compound BTX-A51

CHAPPAQUA, N.Y., Nov. 11, 2019 /PRNewswire/ — BioTheryX, Inc. (“BioTheryX”), a clinical stage biotechnology company creating new classes of drugs for unmet medical needs based on targeted protein degradation and multi-kinase inhibition, today announced the appointment of Peter N. Crnkovich to its Board of Directors. Since September 2018 Mr. Crnkovich has served as a Senior Consultant at the Company. Mr. Crnkovich currently serves as a Senior Advisor at Morgan Stanley, and prior to his retirement from Morgan Stanley after 34 years, he held a variety of positions there, including most recently as Chairman of the Global Health Care Group in investment banking for over a decade. Mr. Crnkovich is also a Senior Advisor at Riboscience LLC, an emerging biopharmaceutical company. Mr. Crnkovich received a B.S. from Georgetown University and an MBA from the Stanford Graduate School of Business.

“We are delighted to add Peter Crnkovich as a Director of BioTheryX,” said David Stirling, CEO and Founder of BioTheryX. “His vast experience in the biopharma capital markets and as a strategic advisor to companies in the biopharma industry will be of tremendous value as BioTheryX enters the clinic with its lead multi-kinase inhibitor, and drives forward its first-in-class “molecular glue” Protein Homeostatic Modulators (“PHMs®”) and bifunctional protein degraders.”

In addition to Mr. Crnkovich, the BioTheryX Board currently includes:

  • David Stirling, PhD – CEO/Founder, Co-founder and former CSO of Celgene, repositioned thalidomide as a breakthrough therapy for cancer, myelodysplastic syndrome and related conditions. Developed Celgene into a top-tier pharmaceutical business. Led effort to develop a class of antiproliferative and immunomodulatory drugs such as the targeted protein degrading “molecular glues” THALOMID®, REVLIMID®, POMALYST®, as well as the psoriatic arthritis drug OTEZLA® and the ADHD drug FOCALIN®
  • Lawrence Zaslow, MBA – President/Founder, founding Managing Director of Amphion Innovations
  • Louis DeGennaro, PhD – CEO/President of Leukemia & Lymphoma Society
  • Jeffrey L. Edwards – Former CFO of Allergan, Inc.

BioTheryX, Inc. Announces the Appointment of Biopharma Veteran Jeffrey L. Edwards to Board of Directors

BioTheryX plans to continue building out Board of Directors with key biopharma industry leaders as Company transitions from preclinical to clinical stage with lead Acute Myeloid Leukemia (“AML”) compound BTX-A51

CHAPPAQUA, N.Y., Sept. 18, 2019 /PRNewswire/ — BioTheryX, Inc. (“BioTheryX”), a biotechnology company creating new classes of drugs for unmet medical needs based on multi-kinase inhibition and targeted protein degradation, today announced the appointment of Jeffrey Edwards to its board of directors. Mr. Edwards retired from Allergan, Inc. in February 2015 after nearly 22 years at the company. Mr. Edwards served as Executive Vice President, Finance and Business Development, Chief Financial Officer of Allergan, Inc. from September 2005 to August 2014. From 2003 to 2005, Mr. Edwards served as Allergan’s Corporate Vice President, Corporate Development and previously served as Senior Vice President, Treasury, Tax and Investor Relations. Mr. Edwards currently serves on the board of directors and as Compensation Committee chairman and a member of both the Audit and Compliance Committees of Bio-Rad Laboratories, Inc., a publicly traded medical device diagnostics company. Mr. Edwards also serves on the board of directors and as Audit Committee chairman of FibroGen, Inc, a publicly traded biopharmaceutical company. He is also a director of Clearside Biomedical Inc., a publicly traded pharmaceutical company, where he is a member of both the Compensation Committee and Audit Committee. Mr. Edwards received a Bachelor of Arts degree in Sociology from Muhlenberg College and completed the Advanced Management Program at the Harvard Business School.

“We believe that Jeff’s track record, experience and reputation in the business of biopharma are a great fit for BioTheryX as we transition from being a preclinical to a clinical stage biotechnology company with our lead compound, BTX-A51, on the cusp of human clinical trials,” said David Stirling, CEO of BioTheryX. “We are delighted to have him on our Board, and we’re looking forward to working with him as we also drive the lead compounds from our broad platform Protein Homeostatic Modulator (“PHM®”) program toward the clinic in the burgeoning field of Targeted Protein Degradation.”

In addition to Mr. Edwards, the BioTheryX Board currently includes:

  • David Stirling, PhD – CEO/Founder, Co-founder and former CSO of Celgene, repositioned thalidomide as a breakthrough therapy for cancer, myelodysplastic syndrome and related conditions. Developed Celgene into a top-tier pharmaceutical business. Led effort to develop a class of antiproliferative and immunomodulatory drugs such as the targeted protein degrading “molecular glues” THALOMID®, REVLIMID®, POMALYST®, as well as the psoriatic arthritis drug OTEZLA® and the ADHD drug FOCALIN®
  • Lawrence Zaslow, MBA – President/Founder, founding Managing Director of Amphion Innovations
  • Louis DeGennaro, PhD – CEO/President of Leukemia & Lymphoma Society

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.