DBM Anti-Proliferative Lead Small Molecules for Polycystic Kidney Disease

DESCRIPTION provided by applicant An important discovery and validation event occurred recently within human cell based drug discovery programs at DiscoveryBioMed Inc DBM yielding a lead class of cytostatic anti proliferative small molecules that display nanomolar potency and marked efficacy against hyperproliferative human PKD cells that create and line remodeled PKD cysts within emergent cystic kidney tissue DBMandapos s strength for this program is our ability to culture and implement primary human cells from normal and diseased kidneys and the deep expertise and record of publication in the PKD field by DBMandapos s Founder DBM implemented these primary human PKD cell systems in the Critical Path for drug discovery and validation and will continue to implement said human diseased cell platforms in continued proposed work There are no PKD specific therapeutics emerging as yet or that are approved by the FDA Existing PKD drugs in development and trials are andapos re purposedandapos from other disease programs Therefore there is significant and critical unmet clinical need for small molecule therapies for PKD DBM also sees this drug class as a PKD preventative a drug against ADPKD development would be particularly effective given the slow progression enlargement of kidneys over several decades that is monitored by imaging within families historically afflicted by this autosomal dominant disease Medicinal chemistry driven modification is this lead drug class is on going and has yielded nanomolar potency against PKD and across a wide spectrum of cancers and is specific in effect to cystic or cancer cells versus normal cells This DBM H program is accelerated for typical Phase SBIR status Because of this because of a critical subcontracted collaboration with the Johns Hopkins PKD Center and because of the critical unmet clinical need for new PKD specific therapeutics DBM applies for a Phase Phase andapos Fast Trackandapos award to accelerate this program from its current pace into clinical trials Over arching goals and milestones for Phase of the program include to a advance and finalize basic medicinal chemistry derivatization of lead small molecules b perform comprehensive cellular and molecular mechanism of action MoA assessment and c generate proof of concept in vivo efficacy in a novel PKD mouse model Lead drugs will be administered and assessed in PKD mice in collaboration with the Johns Hopkins PKD Center Armed with these Phase achievements already in progress on this DBM H lead drug class planned Phase milestones are scripted to d refine medicinal chemistry for in vivo andapos druggabilityandapos e define cellular and molecular MoA s fully and specifically f perform ADME DMPK for a full pre clinical profile and g select lead clinical candidates for an IND filing and PKD clinical trials planning The best clinical candidate drug will be developed forward by DBM in conjunction with Johns Hopkins PKD Center a medicinal chemistry CRO and an ADME DMPK CRO in envisioned Phase efforts for a future out license partnership with a BioPharmaceutical company BM Ic PUBLIC HEALTH RELEVANCE DiscoveryBioMed Inc DBM wishes to capitalize on the discovery of a potent lead class of cytostatic anti proliferative small molecules as clinical candidate drugs to attenuate or block the initial hyperproliferative phases of autosomal dominant and autosomal recessive polycystic kidney disease PKD PKD is of particular scientific interest to the Founder of DBM who has studied the basic science of this chronic hyperproliferative disorder for over years DBM is also a partner within the Johns Hopkins PKD Center a subcontracted collaborator in this PKD drug discovery and development program As such we see this lead drug validation and progression program as a personal crusade for the company to offer a new therapeutic for PKD in the near future There are no PKD specific therapeutics that have emerged at present thus we seek andapos Fast Trackandapos funding to accelerate our collective effects to develop a drug in haste that was discovered in a human PKD cell platform that is potent and specific to hyperproliferative cells without affecting normal cells and that is viable for BioPhara partnership and for rapid entrance into clinical trials