News

Foundation to Continue Advancement of TRPML1 Agonists for GBA-Parkinson’s Disease

CAMBRIDGE, MA – January 26, 2022 – Caraway Therapeutics today announced that the Company has been awarded a third research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF). This latest grant enables Caraway to further explore the role of TRPML1 activation in alpha-synuclein processing and clearance. It also supports efforts to identify biomarkers for use in clinical trials of the Company’s proprietary TRPML1 agonists in GBA-Parkinson’s disease (GBA-PD). Mutations in the GBA gene disrupt the function of the lysosomal lipid processing enzyme it encodes, glucocerebrosidase (GCase), and are among the most significant genetic risk factors for the development of Parkinson’s disease. Severe homozygous mutations in GBA cause Gaucher disease, a lysosomal storage disorder.

TRPML1, a lysosomal cation channel, regulates the activity of the lysosome, the organelle responsible for recycling cellular debris, such as aggregated alpha-synuclein and toxic lipids. Data suggests that increasing TRPML1 activity improves lysosomal function, addressing the underlying defect in GBA-PD. In previous research, Caraway’s TRPML1 activators have impacted the production of various lipids and alpha-synuclein in neurons derived from patients with GBA-PD. The Company will use the grant funding to further evaluate these effects and characterize potential biomarkers which may be used in future clinical trials of TRPML1 activators.

“It has been a pleasure to work with The Michael J. Fox Foundation and we are thrilled to continue our partnership with this third grant,” said Martin D. Williams, Chief Executive Officer at Caraway Therapeutics. “Understanding the role TRPML1 plays in alpha-synuclein clearance will be important as we advance our TRPML1 program towards the clinic and further our understanding of how our TRPML1 agonists impact disease processes in GBA-PD, one of the most common genetically-defined forms of PD.”

“TRPML1 activation leads to enhanced activity of the GCase pathway which is compromised in GBA-PD patients,” added Magdalene Moran, Chief Scientific Officer at Caraway Therapeutics. “GBA-PD is characterized by lysosomal dysfunction. By agonizing TRPML1, we aim to restore the activity of the lysosome and improve cell health.”

“MJFF continues to invest in research that will improve our understanding of lysosomal function in PD. We are pleased to award this third grant to Caraway to increase our understanding of TRPML1, a target of high interest to the Foundation,” said Marco Baptista, MJFF Vice President of Research Programs.

 

About Caraway Therapeutics

Caraway Therapeutics is a biopharmaceutical company pursuing novel approaches for the treatment of genetically defined neurodegenerative and rare diseases. The company is a leader in the cutting-edge science of activating cellular recycling processes to clear toxic materials and defective cellular components by modulating lysosomal function. Caraway is utilizing its unique

product engine to develop proprietary insights into lysosomal function and small molecule ion channel modulation and advance a robust pipeline of precision therapeutic candidates with disease-modifying potential for patients. The company is backed by top-tier investors, including SV Health Investors, AbbVie Ventures, MRLV Fund, Amgen Ventures, Dementia Discovery Fund, Alexandria Venture Investments and Eisai Innovation.

Caraway is based in Cambridge, MA. For more information, please visit www.carawaytx.com.

 

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