LAUR-201 (V-Smart®-TNV for Neuro-HIV): Novel V-Smart® Nanomedicine for Treatment of HAND

Lauren Sciences goal is to advance development of its innovative V-Smart® Nanomedicine for Neuro-HIV, LAUR- 201 (V-Smart®-Tenofovir for Neuro-HIV), as a disease-modifying therapeutic for the critical unmet medical needs of 50% of the 40 million HIV patients worldwide who, despite suppressive antiretroviral therapy (ART), suffer from HIV-associated neurocognitive disorders (HAND). Currently, there are no FDA-approved therapies for HAND, and clinical symptoms include quantifiable cognitive deficits hallmarked by impaired memory. A major shortcoming of first-line HIV therapies is lack of sustained potent delivery to the central nervous system (CNS), which LAUR-201 is designed to solve. Tenofovir (TNV), a component of first-line combination antiretroviral (ARV) pills Truvada and Atripla, is widely used and effective in HIV-infected individuals; however, TNV hardly crosses the blood brain barrier (BBB). Since TNV has the poorest BBB penetration of typical ARV drugs, and is contained in the most widely prescribed first-line therapy regimens, delivering TNV into the CNS may be the most useful to eliminate HIV in the CNS. TNV is particularly effective in penetrating and eliminating HIV from the predominant cells harboring HIV in the brain, and ongoing low-level viral replication in the brain confers a unique sanctuary for HIV, which further contributes to HAND pathogenesis and the CNS HIV reservoir. Lauren Sciences used its novel cutting edge V-Smart® Platform to develop LAUR-201, proven in preliminary data to: (1) encapsulate high TNV concentrations, (2) retain TNV chemical structure, (3) deliver therapeutic TNV concentrations into mouse brain after systemic administration, without apparent toxicity, and (4) reach TNV brain levels in mice well above those attained in ART-treated patients’ blood. This SBIR Phase I is critical to prove feasibility of LAUR-201 to: (1) maintain TNV efficacy in vitro and therapeutic levels in mice brain, and (2) be effective in the murine HAND model, i.e., suppress persistent HIV in the CNS and ameliorate cognitive deficits conferred by HIV, without toxicity, compared to free TNV, after systemic administration. LAUR-201 is a breakthrough solution for HAND, solves the TNV brain delivery issue, targets persistent HIV within the CNS and can impact, and exert a sustained, powerful influence on, this high unmet medical need. LAUR-201 is intended to become the first FDA-approved drug to safely prevent HAND and overcome Neuro-HIV as part of the standard treatment for HIV patients.Lauren Sciences is developing its innovative V-Smart® Nanomedicine for Neuro-HIV, LAUR-201 (V-Smart®- Tenofovir for Neuro-HIV), as a disease-modifying therapeutic for HIV-associated neurocognitive disorders (HAND), and expects LAUR-201 to become the 1st FDA-approved drug for this critical unmet medical need of 50% of the 40 million HIV patients worldwide for whom, despite suppressive antiretroviral therapies (ART), no approved disease-modifying therapies currently exist, and, hence, HAND, with its quantifiable cognitive deficits hallmarked by impaired memory, prevails, due to current HIV drugs’ lack of sustained potent delivery to the central nervous system (CNS). Tenofovir (TNV), a component of first-line combination antiretroviral (ARV) pills Truvada and Atripla, is widely used and effective in HIV-infected individuals; however, TNV hardly crosses the blood brain barrier (BBB) and, in fact, has the poorest BBB penetration of typical ARV drugs, although it is contained in the most widely prescribed first-line therapy regimens; hence, TNV may be the most useful to eliminate HIV in the CNS, and it is particularly effective in penetrating and eliminating HIV from the predominant cells harboring HIV in the brain, where ongoing low-level viral replication confers a unique sanctuary for HIV, which further contributes to HAND pathogenesis and the CNS HIV reservoir. LAUR-201 is designed with both: (1) the disease-modifying TNV, that is an effective ART, but, due to its poor brain penetrability, is ineffective for HAND, and (2) the novel cutting edge V-Smart® Platform, that has demonstrated safe and successful non- invasive, delivery of extremely high therapeutic TNV concentrations into the mouse brain.