Kalytera Provides Update on Phase 2 Clinical Study Evaluating CBD in Prevention of Acute GVHD

No Patients Have Developed Grades 3-4 Acute GVHD While Receiving CBD Treatment

SAN FRANCISCO and TEL AVIV, Israel, Dec. 20, 2018 (GLOBE NEWSWIRE) -- Kalytera Therapeutics, Inc. (TSX VENTURE: KALY and OTCQB: KALTF) (the "Company" or "Kalytera") today announced positive interim data from its ongoing Phase 2 clinical study evaluating cannabidiol (“CBD”) for the prevention of acute graft versus host disease (“GVHD”) following bone marrow transplant.

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. There are currently no FDA approved therapies for either the prevention or treatment of acute GVHD.

It is estimated that up to 50% of patients who undergo a bone marrow transplant from a sibling donor, and up to 70% of patients who undergo a bone marrow transplant from an unrelated donor, will develop some level of GVHD.

Key Findings to Date:
Interim data from the initial 12-patient cohort support the following key findings to date:

  • No patients receiving oral CBD at the lowest study dose of 75 mg twice daily (“BID”) have developed grades 3 or 4 acute GVHD, the most serious forms of the disease.
  • One patient developed grade 2 acute GVHD, the least serious form of the disease.
  • CBD has demonstrated a good safety and tolerability profile, with no significant adverse events relating to its use.

Kalytera’s ongoing Phase 2 clinical study is an open label, multicenter study to evaluate multiple doses of CBD for the prevention of acute GVHD following allogeneic hematopoietic cell transplantation, commonly referred to as bone marrow transplantation. The study will evaluate the PK profile, safety, and efficacy of CBD at doses of 75, 150, and 300 mg BID. All patients in the study receive CBD treatment for 7-days prior to their bone marrow transplant procedure, and for 98-days following the procedure.

The study will enroll a total of 36 patients in three 12-patient cohorts. The first cohort is the low dose cohort, with patients in this cohort receiving the 75 mg BID dose. Enrollment of the first cohort is complete, although all 12 patients in this cohort have not yet completed the entire 105-day course of treatment with CBD. It is anticipated that all of the patients in the first cohort will have completed the entire 105-day course of treatment with CBD by February 28, 2019.

To date, two patients in the first cohort have died. Significantly, however, these patients died from causes unrelated to GVHD, and neither of these patients developed GVHD while in the study. One of the patients in the first cohort did develop stage 2 acute GVHD, which is the least serious form of the disease, after 91 days on study.

CBD is a non-psychotropic ingredient of cannabis that does not cause euphoria or cognitive effects. The formulation of CBD that Kalytera is evaluating for the prevention of acute GVHD is a proprietary formulation that is designed to improve product stability and absorption after oral dosing. 

“We are very pleased with these interim results to date, which show compelling safety, tolerability and PK data, as well as evidence of potential efficacy. The fact that these positive results were seen in patients in the lowest dose cohort is particularly encouraging," said Robert Farrell, President and CEO of Kalytera. “These clinical data confirm our expectation that CBD has potential to become the first pharmaceutical approved for prevention of acute GVHD following bone marrow transplantation.”

Mr. Farrell further stated that, “With no FDA approved therapy for the prevention of acute GVHD, there exists an important unmet medical need that we believe we can address. 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. The complete Phase 2 data set should provide strong support for our planned Phase 3 study, which we expect to initiate in 2019.”

The Potential Commercial Opportunity is Large
The commercial opportunity for Kalytera’s CBD product for prevention of acute GVHD is large. Because acute GVHD is a serious and life threatening orphan disease, Kalytera believes that it will be able to obtain premium pricing for a course of treatment. The incidence of acute GVHD following bone marrow transplantation in the six major markets of the U.S., Germany, the U.K., France, Spain and Japan is above 20,000 patients per year. Given this addressable market, and the expected premium pricing that Kalytera believes it will obtain, the commercial opportunity for the Company’s CBD product in prevention of acute GVHD is potentially more than USD $1 billion annually.

Generic Competition
The Company believes that it will be able to successfully overcome the threat of competition from non-prescription generic CBD for the following reasons:

  • Kalytera’s CBD product is covered by two issued U.S. patents, which provide market exclusivity through approximately 2034. Kalytera has also applied for patents in the EU and other jurisdictions.
  • Kalytera has been granted orphan drug designation (“ODD Designation”) for its CBD product for the prevention of acute GVHD in both the U.S. and the EU. These ODD Designations will provide another form of market exclusivity: 7-years in the U.S. and 10-years in the EU.
  • Most importantly, patients who have undergone a bone marrow transplantation procedure are at high risk of developing acute GVHD, which is a serious and life threatening disorder. These patients are often in isolation in the intensive care unit, and it is highly unlikely that a treating physician would prescribe any form of CBD other than an approved formulation. Of secondary importance is the fact that in all jurisdictions where the Company intends to market its CBD product, neither private insurance nor government provided healthcare reimbursement would be available for non-prescription generic CBD.

New Results Consistent with Previously Announced Positive Data from Earlier Studies in Prevention of Acute GVHD
Prior to the ongoing Phase 2 clinical study in prevention of acute GVHD, two Phase 2a clinical studies were completed evaluating CBD in the prevention of GVHD. Kalytera has previously announced the results from these two studies. Data from both of these 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. These studies were carried out by Dr. Yeshurun prior to the date he joined the Company, and the Company subsequently acquired the relevant technology through its acquisition of Talent Biotechs Ltd. in February 2017.

“I am very enthusiastic about the results to date from our ongoing Phase 2 study,” commented Dr. Moshe Yeshurun. “These data are consistent with our two previous Phase 2a clinical studies evaluating CBD in the prevention of acute GVHD. Data from both of those clinical studies were positive and highly encouraging.”

The results from the first of these previous clinical studies were published in May 2015 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. / Biology Blood Marrow Transplant 21 (2015) 1770 - 1775).

As previously announced, 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 a group of all 101 hematopoietic stem cell transplant recipients who received standard GVHD prophylaxis at the same institution in Israel (the Institute of Hematology, Davidoff Center, Rabin Medical Center at Beilinson Hospital) directly prior to the onset of this study; and
  • CBD was found to be safe and well tolerated

Kalytera also previously announced the results from the second of the two previous clinical studies, in which 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 46% incidence at the same institution in the historical group of 101 patients described above.

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 its CBD products for prevention and treatment of acute GVHD, each of which could accelerate the approval process for these products.

About Kalytera Therapeutics
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.

Cautionary Statements
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This press release may contain certain forward-looking information and statements ("forward-looking information") within the meaning of applicable Canadian securities legislation, that are not based on historical fact, including without limitation in respect of its product candidate pipeline, planned clinical trials, regulatory approval prospects, intellectual property objectives and other statements containing the words "believes", "anticipates", "plans", "intends", "will", "should", "expects", "continue", "estimate", "forecasts" and other similar expressions. Readers are cautioned to not place undue reliance on forward-looking information. Actual results and developments may differ materially from those contemplated by these statements depending on, among other things, the risk that future clinical studies may not proceed as expected or may produce unfavourable results. Kalytera undertakes no obligation to comment on analyses, expectations or statements made by third parties, its securities, or financial or operating results (as applicable). Although Kalytera believes that the expectations reflected in forward-looking information in this press release are reasonable, such forward-looking information has been based on expectations, factors and assumptions concerning future events which may prove to be inaccurate and are subject to numerous risks and uncertainties, certain of which are beyond Kalytera's control. The forward-looking information contained in this press release is expressly qualified by this cautionary statement and is made as of the date hereof. Kalytera disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking information, whether as a result of new information, future events or otherwise.

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