Kalytera Therapeutics, Inc., a pharmaceutical company developing a portfolio of proprietary cannabinoid and endocannabinoid-like medicines, has signed a research agreement with Shaare Zedek Medical Center to identify and characterize genetic abnormalities in persons with Prader-Willi syndrome (“PWS”) that may contribute to the development of low bone mineral density (“BMD”) and osteoporosis.
The research will be led by Professor Varda Gross-Tsur, M.D., a Professor at the Hebrew University and the Director of the national multidisciplinary PWS Clinic at the Shaare Zedek Wilf Children’s Hospital. The Clinic treats the majority of individuals with a genetic diagnosis of PWS in Israel.
“Diminished bone mineral density is common in PWS and may contribute to the increased morbidity and poor quality of life,” said Professor Gross-Tsur. “Kalytera has previously demonstrated that KAL671 prevents bone loss when administered to Magel2-null mice, a mouse model that recapitulates some of the features of PWS. Our research seeks to build upon these findings by examining PWS patients to determine the extent to which the Magel2 mutation, and other genetic abnormalities, are associated with reduced bone mineral density and osteoporosis.”
To investigate the genetic and epigenetic cause or causes of low bone mineral density, researchers plan to perform genetic testing on 30 individuals with PWS, including ten with normal BMD, ten with abnormal BMD, and ten with osteoporosis. Researchers will conduct a chromosomal microarray on DNA for each patient in order to define genetic abnormalities in a region that contains at least two genes that are associated with BMD regulation, including Magel2. The researchers also plan to assess global RNA expression patterns using RNA sequencing.
“This new research could greatly improve our understanding of the genetic mutations underlying the musculoskeletal defects often observed in persons with PWS,” said Dr. Yossi Tam, D.M.D, Ph.D., Head of the Obesity and Metabolism Laboratory at the Hebrew University of Jerusalem and a member of Kalytera’s Scientific Advisory Board. “Further, this research could support the advancement of therapeutics which target the effects of specific mutations, including novel compounds like KAL671.”
KAL671 is a unique synthetic fatty acid amide and an endocannabinoid-like molecule. KAL671 may offer advantages over existing osteoporosis therapies, which are commonly either anti-resorptive or proformative; KAL671 is a novel treatment approach that may be both anti-resorptive and proformative. Studies in mice have shown that KAL671 increases bone volume density with a favorable side effect profile. Kalytera has exclusively licensed KAL671 from Yissum Research Development Company, the technology transfer arm of the Hebrew University of Jerusalem.
About Prader-Willi Syndrome
Prader-Willi syndrome is a complex orphan genetic disorder caused by abnormalities in the 15th chromosome. Decreased bone mineral density and an increased fracture risk associated with osteoporosis are common features of individuals with PWS. There are an estimated 25,000 persons living with PWS in the U.S.
Kalytera Therapeutics is developing a portfolio of non-psychoactive cannabinoid and endocannabinoid-like medicines that we believe will address large unmet market needs. Kalytera seeks to commercialize its cannabinoid therapies across a range of disease states, with an initial focus on bone health.
This press release contains certain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding the efficacy of potential products, the timelines for bringing such products to market, and the availability of funding sources for continued development of such products. Forward-looking statements are based on management’s estimates, assumptions, and projections, and are subject to uncertainties, many of which are beyond the control of Kalytera. Actual results may differ materially from those anticipated in any forward-looking statement. Factors that may cause such differences include the risks that potential products that appear promising to Kalytera cannot be shown to be efficacious or safe in subsequent preclinical or clinical trials, Kalytera will not obtain appropriate or necessary governmental approvals to market these or other potential products, Kalytera may not be able to obtain anticipated funding for its development projects or other needed funding, and Kalytera may not be able to secure or enforce adequate legal protection, including patent protection, for its products. All forward-looking statements included in this press release are made only as of the date of this press release, and Kalytera does not undertake any obligation to publicly update or correct any forward-looking statements to reflect events or circumstances that subsequently occur or of which we hereafter become aware.