Philanthropic support from Dr. Marie and Mr, Vijay Goradia is enabling a research collaboration between Goldenrod Therapeutics and investigators at the Glenn Biggs Institute for Alzheimer’s and Neurodegenerative Diseases at UT San Antonio’s Health Science Center (The Glenn Biggs Institute for Alzheimer’s and Neurodegenerative Diseases at UT Health San Antonio is a comprehensive center dedicated to advancing the understanding, diagnosis, treatment and care of Alzheimer’s disease and other neurodegenerative disorders through research, clinical care and education) to evaluate Goldenrod’s investigational small molecule 11h in preclinical models of Alzheimer’s disease.
The philanthropic support provides the resources needed to conduct the academic research program and explore whether the biological effects of 11h may have relevance to Alzheimer’s disease.
The study, known as the Alzheimer’s Disease anti-Amyloid & PDE4 inhibitor Therapy (ADAPT) Study, is being led by Sarah Hopp, PhD, Principal Investigator and Associate Professor whose research focuses on microglia neuroimmunology in Alzheimer’s disease, with Sam Houle, PhD, a postdoctoral research fellow at the Biggs Institute, contributing to the experimental program. Goldenrod Therapeutics is providing 11h and contributing scientific and drug-development expertise to the collaboration.
Understanding Alzheimer’s Disease and Its Impact
“Support for rigorous early-stage research is critical to determining which scientific ideas have the potential to become meaningful new therapies,” said Dev Chatterjee, CEO of Goldenrod Therapeutics. “We are deeply grateful to Dr. Marie and Mr. Vijay Goradia for making the ADAPT Study possible and for supporting research that brings together academic expertise and emerging therapeutics.”
11h is an investigational, orally bioavailable, brain-penetrant phosphodiesterase-4 (PDE4) inhibitor being developed by Goldenrod to modulate neuroinflammation and other disease-relevant pathways in the central nervous system. PDE4 regulates intracellular cyclic AMP signaling, a pathway involved in inflammatory responses, neuronal function and synaptic biology. Goldenrod is developing 11h with the goal of capturing the therapeutic potential of PDE4 inhibition while improving upon limitations that have historically constrained the use of this drug class.
Chronic neuroinflammation and dysfunctional microglial activity are increasingly recognized as important components of Alzheimer’s disease biology. The ADAPT Study is evaluating 11h in disease-relevant experimental models to determine whether its pharmacological profile translates into beneficial effects on Alzheimer’s-related biology and function.
“Dysfunctional microglia and chronic changes in immune signaling are increasingly recognized as important components of Alzheimer’s disease biology,” said Dr. Hopp. “The ADAPT Study allows us to examine a pharmacological approach directed at these mechanisms in relevant preclinical models, and to understand how combining this kind of new therapy with existing anti-amyloid therapeutics might be beneficial. Our goal is to generate rigorous data that can help determine whether further translational research is warranted.”
The ADAPT Study was originally conceived with Kevin Bieniek, PhD, formerly of the Biggs Institute, who helped develop the scientific rationale and establish the collaboration before transitioning to the biotechnology industry.
“When we first began discussing this study, what was particularly compelling was the opportunity to examine a pharmacological approach that could address neuroinflammation alongside the established focus on amyloid pathology,” said Dr. Bieniek. “I’m pleased to see the ADAPT Study moving forward and look forward to seeing what the research tells us about the potential of this approach in Alzheimer’s disease.”
The collaboration reflects Goldenrod’s strategy of working with disease-area experts to evaluate 11h across neurological disorders in which chronic neuroinflammation may contribute to disease progression. Findings from the ADAPT Study are intended to inform both the potential role of 11h in Alzheimer’s disease and Goldenrod’s broader neurodegenerative disease development strategy.
The ADAPT Study also highlights the role philanthropy can play in bridging early scientific discovery and translational drug development. By supporting work at the intersection of academic neuroscience and therapeutic development, Marie and Vijay Goradia are helping enable the generation of experimental evidence needed to evaluate new approaches to Alzheimer’s disease.
About the ADAPT Study: The ADAPT Study is an ongoing preclinical research collaboration evaluating Goldenrod Therapeutics’ investigational compound 11h in mouse models relevant to Alzheimer’s disease. The work is being conducted at the Glenn Biggs Institute for Alzheimer’s and Neurodegenerative Diseases at UT San Antonio’s Health Science Center under the direction of Sarah Hopp, PhD, with Sam Houle, PhD.
The study is made possible through philanthropic support from Marie and Vijay Goradia. Goldenrod Therapeutics is providing 11h and related scientific and drug-development expertise.
Goldenrod Therapeutics, Inc. is a Fannin-founded precision therapeutics company developing novel brain-penetrant therapies that target neuroinflammation and neuronal signaling pathways implicated in neurodegenerative and neurological disorders. Goldenrod’s lead candidate, 11h, is a small-molecule drug that inhibits the enzyme PDE4. The company is advancing differentiated programs designed to address significant unmet medical needs through innovative CNS-targeted approaches. In addition to funding from Fannin, the development of 11h has been supported by grants from the NIH and the CDMRP.
Fannin was Established in 2014, Houston-based Fannin is among the most active early-stage product development groups in the life sciences with a dozen programs/platforms at different stages of development including four in the clinic. Fannin advances its pipeline both internally and through Fannin-founded entities with a combination of investor and grant funding. In the last decade, Fannin has brought in more than 35 programs, of which a dozen are active, including three in the clinic. More than $295 million has been invested across the Fannin portfolio, including over $85 million from grant funding. An additional critical element to our model is helping develop life sciences entrepreneurs locally through our talent development programs.