Ashvattha Therapeutics Announces Presentation of Preclinical Data at the 2022 Neurofibromatosis (NF) Conference Demonstrating the Potential of Hydroxyl Dendrimer Therapeutics to Reduce Toxicity in Targeted Treatment of Plexiform Neurofibroma

  • Scientific collaborators at Indiana University School of Medicine presented preclinical data showing Ashvattha’s hydroxyl dendrimers (HDs) were selectively taken up only within tumor-associated microglia/macrophages in a mouse model of NF1
  • These findings suggest that therapeutic conjugation of HDs to target cells of plexiform neurofibroma (PNF) tumor microenvironment may be efficacious and minimize toxicity in the treatment of NF1-associated PNF

REDWOOD CITY, Calif., June 21, 2022 – Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company developing novel hydroxyl dendrimer therapeutics, today announced a poster presentation of preclinical data demonstrating the potential of hydroxyl dendrimer therapeutics (HDTs) to reduce toxicity in the targeted treatment of plexiform neurofibroma, a slow-growing tumor associated with neurofibromatosis type 1 (NF1), at the 2022 Neurofibromatosis (NF) Conference hosted by the Children’s Tumor Foundation and taking place at Loews Philadelphia Hotel in Philadelphia, PA, June 18–21, 2022.

The data was presented by Emma C. Mazurek, a scientific collaborator at Indiana University School of Medicine, in a poster titled, “Hydroxyl Dendrimer Therapeutics Reduce Toxicity in Targeted Delivery to Plexiform Neurofibroma.”

The study investigated the uptake of HDs varying in size. The data showed selective uptake of HDs only by tumor associated macrophages and microglia, but not adjacent neurons, in an NF1-associated PNF mouse model. Notably, the smaller HD (~14 kDa) showed greater uptake in tumor associated macrophages and microglia, than the larger HD (~56 kDa). In vivo evaluation of HD therapeutics is currently underway in an NF1 mouse model of PNF to assess efficacy as measured by a reduction in tumor burden. These results lay the groundwork for future HD therapies towards treatment of NF1-associated PNF.

“Systemic toxicity remains a challenge in treating PNF, limiting the use of pharmacologic agents. These results suggest that by conjugating drugs to HDs there’s potential to improve safety and reduce toxicity associated with current treatments for NF. HDs allow for the uptake of highly toxic drugs specifically in immune cells within the PNF microenvironment impairing tumor growth,” said Jeffrey Cleland, Ph.D., Chairman, CEO and President of Ashvattha Therapeutics. “Studies are currently underway to evaluate novel HDTs in this mouse model of disease to further demonstrate the potential of HDTs in NF1-associated PNF.”

Chronic activation of macrophages and microglia plays a critical role in the progression of many neurological diseases, including NF1. Due to the high inoperability of neurofibromas, pharmacological therapeutics are the main strategy in treatment. Current therapies cause systemic toxicity that is not tolerated by patients leading to discontinuation of treatment. For patients with NF1, there is a need for safer treatments to achieve greater efficacy and durability. Previous data suggests that the combined targeting of tumorigenic pathways and immunosuppressive cells within the PNF tumor microenvironment (TME) may be an effective strategy to overcome these challenges. HDs can be conjugated to more than one drug, enabling one HDT to affect multiple pathways.

About Neurofibromatosis Type 1 (NF1)
NF1 is the most common cancer predisposition syndrome. It is characterized by changes in skin coloring (pigmentation) and the growth of tumors along nerves in the skin, brain, and other parts of the body known as plexiform neurofibromas (PNF). According to the American Academy of Pediatrics, NF1 is one of the most common inherited disorders affecting 1 in every 3000 individuals. The signs and symptoms of this condition vary widely among affected people. Current treatments are limited and somewhat effective in reducing tumor growth but are associated with toxic side effects.

About Hydroxyl Dendrimers Therapeutics (HDTs)
Hydroxyl dendrimers (HDs) are small hydrophilic compounds that can be conjugated to therapeutics to bring small molecule drugs including highly toxic drugs specifically to active inflammatory cells localized to the tumor microenvironment. Prior HDTs have demonstrated excellent safety and tolerability in human clinical trials. HDTs provide a novel approach to treating tumors in their specificity to tumor-associated macrophages (TAMs), further differentiating Ashvattha’s precision medicine approach.

About Ashvattha Therapeutics
Ashvattha Therapeutics is a clinical-stage biotech company developing novel hydroxyl dendrimer therapeutics (HDTs) targeting unmet medical needs in neurology, ophthalmology, inflammatory diseases and neuro-oncology. The therapies are based on hydroxyl dendrimers (HDs), a targeted platform technology exclusively licensed from our founders, Kannan Rangaramanujam and Sujatha Kannan at Johns Hopkins University. HDs chemically conjugated to disease modifying drugs create novel proprietary HD therapeutics (HDTs) selectively targeting reactive inflammatory cells in disease tissue with localized sustained effects. Ashvattha has initiated multiple programs with HDTs focused on neurology, ocular neovascular disease including neovascular age-related macular degeneration (AMD) and diabetic macular edema (DME), and hyperinflammation in diseases. For more information, visit:

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