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"This is an important strategic milestone in the development of ATIR as anovel approach which may enable a safe and potentially life-saving mismatchedbone marrow transplantation as a treatment option for end-stage blood cancerpatients" said Dr. Manja Bouman, Chief Executive Officer of Kiadis Pharma.
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The FDA's orphan drug designation is reserved for new therapies beingdeveloped to treat diseases or conditions that affect fewer than 200,000people in the United States. The orphan drug designation provides for anaccelerated review process, tax benefits, exemption from user fees and aseven-year period of market exclusivity in the US after product approval.
ATIR is under development to prevent life-threatening GvHD, a majorcomplication of allogeneic bone marrow transplantations. GvHD is a conditioncaused by the donor immune cells attacking the patient's tissues and organs.Allogeneic bone marrow transplantations today require that the patient anddonor immune systems (HLA type) are highly similar in order to reduce the riskof GvHD and therefore rely on matching donors. By preventing the occurrence ofGvHD, ATIR enables the use of a mismatched donor and consequently addresses asignificant limitation in bone marrow transplantation, the timely availabilityof a donor.
Every year between 50,000 and 60,000 patients die of blood cancers in theUnited States alone. For these end-stage and high-risk blood cancer patientsbone marrow transplantation is the only treatment option. In addition, anumber of other hematological diseases can be cured by bone marrowtransplantations. However, annually only about 2,500 patients in the UnitedStates receive a transplantation with bone marrow from an unrelated matcheddonor despite the approximately 60,000 worldwide initiated searches for amatching donor in the donor registries.
About ATIR
ATIR is designed to prevent GvHD by selectively eliminating the immunecells that otherwise attack the patient's body. ATIR is also designed to spareuseful immune cells that can fight infections and remaining tumor cells thatwould allow rapid and safe immune reconstitution post transplantation.Prevention of GvHD using ATIR could therefore make the safe use of mismatchedbone marrow donors possible. Moreover, it could improve the outcome of matchedbone marrow transplants.
About Kiadis Pharma
Kiadis Pharma is an oncology focused pharmaceutical development companywith four products in different phases of clinical development. The companydevelops products that offer novel treatment options for terminally ill cancerpatients and address significant unmet medical needs. The key focus indicationfor Kiadis Pharma is limitations and complications of bone marrowtransplantation procedures performed in blood cancer patients. Kiadis Pharmais headquartered in Amsterdam, The Netherlands with facilities in Groningen,The Netherlands and Montreal, Canada. For more information about KiadisPharma, please visit http://www.kiadis.com.
This announcement is not an offer to sell or a solicitation of any offerto buy the securities of Kiadis Pharma B.V. (the "Company", and suchsecurities, the "Securities") in the United States or in any otherjurisdiction.
The Securities have not been and will not be registered under the USSecurities Act of 1933, as amended (the "Securities Act") and may not beoffered or sold in the United States unless registered under the SecuritiesAct or an exemption from such
