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Lucy Therapeutics Debuts Novel Therapeutic Agents for Parkinson’s Disease at Michael J. Fox Foundation Annual Conference

Lucy Therapeutics’ proprietary compounds demonstrate promising preclinical efficacy, safety profiles via novel mechanism of action, validating new mitochondrial-based therapeutic approach for Parkinson’s Disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Lucy Therapeutics, Inc., a private biotech company developing proprietary mitochondrial-based small molecule therapies and biomarkers for neurological diseases, today announced promising preclinical data for multiple compounds in its lead program for Parkinson's disease, during an oral presentation at the Michael J. Fox Foundation's 14th annual Parkinson's Disease Therapeutics Conference on October 13, 2022, in New York City.

“The high selectivity and potency of two of our lead small molecule agents, LucyTx-1209 and LucyTx-1212, in inhibiting a target we believe is at the nexus of several mitochondrial pathways contributing directly to Parkinson’s disease represents a significant advance for our mission to develop first-in-class therapies for neurological diseases,” said Roland Staal, Ph.D., director of pharmacology at Lucy Therapeutics, during the conference presentation. “We have demonstrated that our compounds can rescue neurons in cellular models of Parkinson’s disease, validating our therapeutic approach. We did not observe any significant safety issues with the use of LucyTx-1209 or LucyTx-1212 in pre-clinical laboratory or rodent studies, so we will now focus on studies to select the best candidate to advance for clinical trials.”

The development of LucyTx-1209 and LucyTx-1212 resulted from Lucy Therapeutics’ proprietary research strategy and platform. By understanding the function and dysfunction of the mitochondria, which generates energy within the cell, Lucy Therapeutics identified critical, potent targets that modulate rate-limiting steps – the bottlenecks – in biological pathways that can cause disease. The scientists recognized a key target for drug discovery and development within the mitochondria’s electron transport chain (ETC), which is a known driver of multiple dysfunctions specific to Parkinson’s disease. Specifically, LucyTx-1209 and LucyTx-1212 are designed to target one of the functions of the protein complex F1F0-ATPase, which acts within the ETC.

In their preclinical studies, the Lucy Therapeutics investigators demonstrated the investigational agents inhibited F1F0-ATPase to reverse mitochondrial dysfunctions known to lead to many key characteristics of Parkinson’s disease biology, including decreased levels of the cellular energy molecule ATP and death of neurons. The Lucy Therapeutics compounds also reduced levels of α-synuclein, the major constituent of Lewy bodies, which accumulate and are a pathological hallmark of PD.

"Lucy Therapeutics wants to create a world in which mitochondrial-involved neurological diseases like Parkinson’s or Rett syndrome can be halted when diagnosed,” said Amy Ripka, Ph.D., founder and CEO of Lucy Therapeutics. “The grant and support from the Michael J. Fox Foundation has helped our scientists significantly speed our investigations. The very encouraging preclinical data we debut today document that our platform and small molecule agents, have great potential for further development.”

Including an April 2022 grant of $4.9 million from The Michael J. Fox Foundation for Parkinson’s Research, Lucy Therapeutics has received support tallying $24 million as of October 2022, from Pivotal bioVenture Partners, Safar Partners, The Engine and the Massachusetts Life Science Center. Lucy Therapeutics also received two Golden Tickets from Biogen and Bristol Myers-Squibb to support work at Lab Central.

About Lucy Therapeutics

Lucy Therapeutics is a private biotechnology company in Cambridge, MA, founded by Amy Ripka, PhD., in 2017, that is dedicated to unraveling the complex and interrelated biological processes driving mitochondrial dysfunction and addressing them to slow and stop the diseases they cause. Sitting at the center of genetics and metabolism, Lucy Therapeutics is leading the change in how we think about mitochondria and the intersecting pathways that play critical roles in human health and disease, particularly those of the central nervous system including Parkinson’s disease and Rett syndrome. For more information, visit:


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Last Updated: 13-Oct-2022