On August 23, 2022 K36 Therapeutics, Inc. ("K36"), a privately held biotechnology company developing novel targeted therapies for the unmet medical needs of cancer patients, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) Application for the Company’s lead program KTX-1001. KTX-1001 is an investigational small molecule methyltransferase inhibitor of multiple myeloma SET domain known as MMSET, a major regulator of chromatin structure and transcription in multiple myeloma (Press release, K36 Therapeutics, AUG 23, 2022, View Source [SID1234618585]). K36 plans to initiate a Phase 1 clinical trial in the second half of 2022 to establish the safety, tolerability and preliminary efficacy of KTX-1001 in patients with relapsed and refractory multiple myeloma, enriching for patients who have the genetic translocation t(4;14).

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K36 also announced the formation of its Clinical Advisory Board comprised of Drs. Jesus Berdeja, Shaji Kumar, Thomas Martin, María-Victoria Mateos, Noopur Raje, David Siegel and Suzanne Trudel.

"We are proud to announce the clearance of our first IND following the initial 30-day FDA review, officially marking our transition to a clinical-stage company in only 16 months since company inception. This achievement demonstrates our team’s efficiency and focus toward our goal of advancing KTX-1001 and positively impacting the lives of patients and families suffering from multiple myeloma," said Terry Connolly, Ph.D., President and Chief Executive Officer of K36. "Along with this significant milestone, I am delighted to announce the establishment of our Clinical Advisory Board, bringing together a group of leading experts with deep expertise in developing breakthrough therapies for the treatment of multiple myeloma. This Board will complement the exceptional experience of our internal team and Dr. Lori Kunkel, our Independent Director."

K36’s Clinical Advisory Board includes:

Jesus Berdeja, M.D., Director of Myeloma Research, Sarah Cannon Research Institute at Tennessee Oncology, Nashville, TN

Shaji Kumar, M.D., Consultant, Division of Hematology, Professor of Medicine, Mayo Clinic, Rochester, MN

Thomas Martin, M.D., Clinical Professor of Medicine, Adult Leukemia and Bone Marrow Transplantation Program, and Associate Director, Myeloma Program, University of San Francisco, Helen Diller Family Comprehensive Cancer Center, San Francisco, CA

María-Victoria Mateos, M.D. Ph.D., Consultant Physician in the Haematology Department and Associate Professor of Medicine at the University of Salamanca, Spain

Noopur Raje, M.D., Director, Center for Multiple Myeloma, Professor of Medicine, Harvard, Medical School, Massachusetts General Hospital Cancer Center, Boston, MA

David Siegel, M.D., Ph.D., Chief of the Multiple Myeloma Division, John Theurer Cancer Center, Hackensack University Medical Center, Hackensack, NJ

Suzanne Trudel, M.D., M.Sc., Associate Professor of Medicine, University of Toronto, Clinician Scientist, Princess Margaret Hospital Cancer Centre, Toronto, Canada

"There remains a significant unmet need for new and more effective therapies that can benefit high-risk myeloma patients such as those with translocation t(4;14)," said Noopur Raje, M.D. "As we use expanded combinations in early lines, there is a need for new classes of agents like KTX-1001."

"BCMA T cell redirecting therapies and the use of quadruplet therapy in myeloma are incredible advances but still have not leveled the playing field for high-risk multiple myeloma patients," added Jesus Berdeja, M.D. "There is still a need for novel precision therapeutics such as KTX-1001, and we are excited to be a part of bringing this first-in-class agent into Phase 1 testing."

K36 expects to enroll approximately 50 patients in the Phase 1 study, including an expansion cohort that will recruit patients with the genetic translocation t(4;14).

About KTX-1001
KTX-1001 is a novel, first-in-class, potent, and selective methyltransferase inhibitor of the catalytic activity of lysine H3K36. It is an investigational and orally administered small molecule being developed initially for the treatment of relapsed and refractory multiple myeloma, with a focus on patients with the t(4;14) translocation.

About the KTX-1001 Phase 1 Clinical Trial
The Phase 1 clinical trial is a single-arm, open-label study in subjects with relapsed and refractory multiple myeloma. It is a multi-part clinical trial with dose escalation followed by an expansion cohort in patients with the genetic translocation t(4;14) to evaluate the safety, tolerability, and preliminary efficacy of different doses of KTX-1001.