Kalytera Therapeutics, Inc. (TSX VENTURE: KALY and OTCQB: KALTF) (the “Company” or “Kalytera”) today provided an update on its lead clinical development program evaluating cannabidiol (“CBD”) for the prevention and treatment of acute graft versus host disease (“GVHD”).
GVHD is a life-threatening complication commonly occurring after bone marrow transplant procedures. GVHD occurs when the transplanted donor cells attack the patient’s organs, including the skin, GI tract, liver, lungs, and eyes. GVHD is associated with acute and chronic illness, infections, disability, reduced quality of life, and death.
It is estimated that up to 50% of patients who undergo a bone marrow transplant from a fully matched sibling donor, and up to 70% of patients who undergo a bone marrow transplant from an unrelated donor, will develop some level of GVHD. There are currently no FDA approved therapies for either the prevention or treatment of acute GVHD.
The commercial opportunity for Kalytera’s CBD products for the prevention and treatment of GVHD is large. According to the January 2018 Market Forecast Report by DelveInsight Perspective, projected annual sales for prevention and treatment of GVHD in the 7 major markets (the U.S., Germany, France, Italy, Spain, the U.K. and Japan) are estimated to be more than USD $408 million in 2018, and could grow to approximately USD $1.3 billion by 2027.
Kalytera is the exclusive licensee of two issued U.S. patents covering the use of CBD in the prevention and treatment of GVHD, and is also the exclusive licensee of pending patent applications in other jurisdictions for the use of CBD in the prevention and treatment of GVHD.
The U.S. FDA has recommended that Kalytera apply for both Breakthrough Therapy and Fast Track Designations for our CBD products for prevention and treatment of acute GVHD, each of which could accelerate the approval process for these products.
CBD is a non-psychotropic ingredient of cannabis sativa that possesses potent immunomodulatory and anti-inflammatory properties.
Kalytera’s Program Evaluating CBD in Prevention of Acute GVHD
Our ongoing Phase 2b clinical study evaluating the use of CBD in the prevention of GVHD is expected to be completed early next year. We have enrolled patients at clinical sites in Israel, and, in order to expedite the enrollment process, we are in the process of expanding this study to include two additional sites in Australia. Upon completion of this Phase 2b clinical study, we will begin preparations for the pivotal Phase 3 clinical study that will be required for FDA approval.
The ongoing Phase 2b clinical study is a 36-patient randomized, multicenter study, which will evaluate the safety, pharmacokinetics, pharmacodynamics, efficacy and dose ranging of CBD for the prevention of GVHD following bone marrow transplantation procedures. The Principal Investigator of the study is Daniel Couriel, M.D., M.S., Director of the Bone Marrow Transplant Program at the University of Utah Health Sciences, School of Medicine.
Prior to our ongoing Phase 2b clinical study, we completed two Phase 2a clinical studies evaluating CBD in the prevention of GVHD. Data from both of these Phase 2a clinical studies were positive and highly encouraging. Dr. Moshe Yeshurun, Kalytera’s Chief Medical Officer, was the Principal Investigator for both of these studies, which were conducted at the Institute of Hematology, Davidoff Center, Rabin Medical Center, Petah Tikva, Israel.
The results from the first of these Phase 2a clinical studies were published in Biology of Blood and Marrow Transplantation (Cannabidiol for the Prevention of Graft-versus-Host-Disease after Allogeneic Hematopoietic Cell Transplantation: Results of a Phase II Study, M. Yeshurun et al. / Biol Blood Marrow Transplant 21 (2015) 1770 – 1775). The data from this study, in which 48 patients received daily doses of 150 mg of CBD administered twice daily for 7 days prior to the bone marrow transplant procedure and for 30 days after, demonstrated the following results:
- No patients developed acute GVHD while being treated with CBD;
- The rates of grades 2-4 acute GVHD by day 100 were 12.1%, compared with 46% in 101 historical control subjects who received standard GVHD prophylaxis at the same institution in Israel (the Institute of Hematology, Davidoff Center, Rabin Medical Center); and
- CBD was found to be safe and well tolerated
In the second of the two prior Phase 2a clinical studies, 12 patients were administered CBD at a dose of 150 mg administered twice daily from 7 days prior to the bone marrow transplant procedure until up to 100-days post transplantation. In that study, no safety issues were observed, and only 15% of patients in the CBD treatment group developed grades 2-4 acute GVHD, compared to a 60-70% incidence predicted by historical data from patients treated at the same institution in Israel (the Institute of Hematology, Davidoff Center, Rabin Medical Center) that were used as a control.
“Acute GVHD is a severe complication of bone marrow transplant procedures that can lead to permanent impairment of quality of life and even death,” said Robert Farrell, President and CEO of Kalytera. “With no FDA approved therapy for the prevention of acute GVHD, there exists an important unmet medical need that we believe we can address. Based on the positive and encouraging results from the two prior Phase 2a clinical studies that we completed in prevention of GVHD, we are optimistic that our CBD product will address this unmet need, and will be demonstrated to be a safe and effective means to prevent the onset of acute GVHD.”
Kalytera’s Program Evaluating CBD in the Treatment of Acute GVHD
We are planning to initiate a seamless Phase 2-3 pivotal registration study in the treatment of acute GVHD later this year. There are currently few options to treat patients with acute GVHD, a critically underserved market.
Our Phase 2-3 pivotal registration study will enroll approximately 135 patients and will assess the safety and efficacy of multiple doses of CBD for the treatment of acute GVHD. The study will be a multicenter, multinational, placebo controlled, randomized clinical trial, for the evaluation of the safety and efficacy of CBD for the treatment of grade 3-4 acute GVHD. The study will be conducted in up to 25 sites in Australia, Israel, The United Kingdom and the U.S. The Principal Investigator of the study is Edmund Waller, M.D., PhD., Professor, Hematology and Medical Oncology, Medicine, and Pathology at Emory University School of Medicine, and Director, Division of Stem Cell Transplantation and Immunotherapy at Winship Cancer Institute of Emory University.
Kalytera previously completed a Phase 2a clinical study in the treatment of acute GVHD entitled, Cannabidiol for the Treatment of Severe (Grades 3-4) Acute Graft versus-Host Disease. Dr. Moshe Yeshurun, Kalytera’s Chief Medical Officer, was the Principal Investigator for this study, which was conducted at the Institute of Hematology, Davidoff Center, Rabin Medical Center, Petah Tikva, Israel.
All ten patients in the Phase 2a clinical study were steroid refractory, meaning that they had not responded to previously administered standard of care steroid treatment. The study design was an open label, single arm, addition to standard of care treatment. Ten subjects, 18 years of age or older, participated in the study. The patients were administered a fixed oral dose of a highly pure formulation of CBD that was extracted from cannabis and dissolved in olive oil. The fixed dose of 150 mg of CBD was administered twice daily for up to three months, alongside standard of care therapy.
The results of this Phase 2a study were positive and highly encouraging. Nine of the ten patients responded to treatment, with seven achieving complete remissions, and with two achieving near-complete responses. Six patients are still alive with a median follow-up period of 13 months (range 5-30 months). These results compare favorably with the results of the historical control group of 29 patients with steroid-refractory grades 3-4 GVHD who were treated at the same institution in Israel (the Institute of Hematology, Davidoff Center, Rabin Medical Center), among which 26 patients died from GVHD and its complications.
“The ability to treat GVHD is a major unmet need,” stated Robert Farrell, Kalytera’s President and CEO. “With no FDA approved therapy, acute GVHD remains a major cause of morbidity and mortality following bone marrow transplantation procedures. Based on the very positive data from our Phase 2a clinical study, we believe that our CBD product may provide a major advance in the treatment of acute GVHD.”
Kalytera Therapeutics, Inc. is pioneering the development of CBD therapeutics. Through its proven leadership, drug development expertise, and intellectual property portfolio, Kalytera seeks to establish a leading position in the development of CBD medicines for a range of important unmet medical needs, with an initial focus on GVHD and treatment of acute and chronic pain.
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