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Developing Therapeutics That Target RAD To Treat Leukemia and Lymphoma

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 2R42CA183197-02A1
Agency Tracking Number: R42CA183197
Amount: $1,999,998.00
Phase: Phase II
Program: STTR
Solicitation Topic Code: NCI
Solicitation Number: PA15-270
Solicitation Year: 2015
Award Year: 2016
Award Start Date (Proposal Award Date): 2016-06-01
Award End Date (Contract End Date): 2019-05-31
Small Business Information
Cambridge, MA 02139-3543
United States
DUNS: 078512533
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (207) 288-6820
Business Contact
Phone: (207) 288-6820
Research Institution
BAR HARBOR, ME 04609-1500
United States

 Domestic Nonprofit Research Organization

DESCRIPTION provided by applicant Genomic instability is a hallmark of cancer and represents a targetable vulnerability yet is underdeveloped as a therapeutic area The central goal of this Phase program is to complete the early preclinical development of a new class of new cancer therapeutics that uniquely target RAD delivering effective cancer cell selective therapy in subsets of biomarker defined leukemia and lymphoma Two key challenges in current cancer therapy are overcoming tumor cell andquot evolutionandquot that drives cancer progression and therapy resistance and minimizing the side effects due to off target toxicity Therapies that target genomic instability mechanisms have the potential to meet these critical clinical challenges In recent years the B cell specific DNA mutase recombinase Activation Induced Cytidine Deaminase AID has been implicated as a driver of oncogenic genomic instability While its expression is normally restricted to activated germinal center B cells AID is also overexpressed in a range of human neoplasms especially B cell non Hodgkinandapos s lymphomas NHL and chronic lymphocytic leukemia CLL Cyteir Therapeutics Inc in partnership with The Jackson Laboratory has demonstrated the feasibility of targeting RAD in these cancers and has developed new lead candidate compound Our novel therapeutic approach takes advantage of the discoveries that AID is both a biomarker and a DNA damage driver creating widespread DNA double strand breaks DSBs throughout the genome and RAD has a unique role in the repair of these malignant AID induced DSBs and is therefore critical for viability in transformed AID cells Cyteirandapos s lead RAD inhibitor is potent highly selective
for cells that are AID effective against NHL and CLL cells in vitro and in vivo and is extremely
well tolerated in preclinical animal models The aims of this Phase study are to complete early preclinical testing and development required prior to the filing of investigational new drug IND application and commencement of phase I clinical trials We will develop a clinical dosage form for our lead compound carry out single and multiple dose tolerability and range finding studies establish pharmacokinetics and toxicokinetics for the clinical dose form and generate comprehensive comparative efficacy data using human to mouse xenograft models of NHL and CLL We have assembled an impressive team of academic and industry leaders complemented by an advisory panel of top thought leaders to advance this program and build the Cyteir drug discovery engine One of our strengths is the continuing partnership with The Jackson Laboratory providing both an academic foundation for our Randamp D efforts and a platform of unique in vivo testing technologies to enable rapid translation to the clinical phase Our commercialization plan for this program calls for completion of the proposed preclinical studies commencement of clinical trials and partnership or out licensing after either phase I or phase II trials to create company and investor value and generate revenue to continue building a sustainable drug development pipeline PUBLIC HEALTH RELEVANCE There are roughly new cases of lymphoma leukemia and multiple myeloma in the United States annually and in excess of deaths Although therapies have improved somewhat these cancers are still increasing in incidence worldwide Owing to the rigors of chemotherapy there are significant long term health concerns relating to survivorship The conventional first line therapies for lymphoid cancers can produce difficult and sometimes life threatening side effects but are often non curative Thus there is a significant unmet need for new therapeutics that effectively treat the cancer but elicit fewer side effects Our novel therapeutic strategy promises to selectively target lymphoid cancer cells for self destruction and minimize the side effects associated with conventional chemotherapies Given the continued increase in global lymphoma and leukemia incidence and the insufficiency of current therapies there is a significant clinical and commercial market for effective and selectiv therapeutics both domestically and worldwide In this context we have potential to positively impact a substantial cancer patient population and to improve the quality of life of many who require chemotherapy treatment

* Information listed above is at the time of submission. *

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