Advancing Neurodegenerative Care: Kasper Roet from QurAlis Corporation in a Stimulating Conversation with PharmaShots

Shots:

Recently, QurAlis signed an exclusive license agreement with Lilly for QRL-204 for patients with ALS and FTD

Under the terms of the agreement, QurAlis granted Lilly an exclusive worldwide license to develop and commercialize QRL-204 and other UNC13A targeting compounds.

QurAlis received an up front payment of $45M plus an additional equity investment. The company is eligible for future milestones payments of up to $577M and tiered royalties on net sales

Saurabh: QurAlis' partnership with Eli Lilly is a significant step forward. Can you explain how this collaboration will accelerate the development of QRL-204 for patients with ALS and FTD?

Kasper: This partnership combines the ASO and ALS drug development expertise of QurAlis with the deep expertise of Lilly in dementia disorders such as FTD. The combined resources of both companies also enable the most rapid development path which is to the benefit of ALS and FTD patients.

Saurabh: For someone unfamiliar with ALS and FTD, can you explain the role of UNC13A and how QRL-204 targets it?

Kasper: Neurons communicate through small vesicles which contain neurotransmitters. UNC13A is crucial for vesicle release which enables this communication. In many ALS and FTD patients, UNC13A protein expression is lost because of a mis-splicing of the UNC13A pre mRNA. QRL-204 corrects this mis-splicing and restores UNC13A function through normalizing vesicle release in neurons derived from patients.

Saurabh: The preclinical data on QRL-204 are encouraging. What are the next steps to move this treatment into clinical trials?

Kasper: The QurAlis and Lilly teams are working together to determine the next steps toward clinical development.

Saurabh: The agreement includes developing other UNC13A-targeting drugs. Can you share some insights into QurAlis' pipeline beyond QRL-204?

Kasper: QurAlis is developing QRL-201, a potential STMN2 ASO therapy for ALS; and QRL-101, a potential small molecule Kv7.2 therapy for ALS. Both programs are in Phase 1 clinical trials.

Saurabh: TDP-43 pathology is linked to ALS and FTD. Can you explain the significance of this connection for the development of QRL-204?

Kasper: TDP-43 pathology is present in more than 90 percent of ALS patients and up to 50 percent of FTD patients. Loss of TDP-43 from the nucleus in neurons of the brain and spinal cord drives UNC13A pre-mRNA mis-splicing leading to loss of UNC13A protein expression. 

Saurabh: This partnership brings renewed hope to ALS and FTD patients. What message would you like to give them about the future of treatment options? 

Kasper: QurAlis believes that ALS and FTD are treatable diseases. We believe that the success of therapy development depends on the quality of the targets and a precision medicine approach. We believe that UNC13A, as well as STMN2 and Kv7.2, all of which are targets of QurAlis’ programs, are the most promising genetic targets for therapy development for ALS, FTD, and other neurodegenerative diseases.

Saurabh: QurAlis' mission is to find cures for neurodegenerative diseases. Can you share your vision for the future of QurAlis and its impact on patients? 

Kasper: We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases.

Image Source: Canva

About the Author:

Kasper Roet

Kasper Roet, Ph.D., is CEO and co-founder of QurAlis Corporation. He is also co-founder and serves on the board of EnClear Therapies. Kasper is a passionate neuroscientist and therapy developer who specializes in genetic medicine and stem cell technology-based precision medicine solutions for serious neurodegenerative and neurological diseases. He co-founded QurAlis with two visionary leaders in ALS stem cell disease modeling, Harvard professors Clifford Woolf and Kevin Eggan. Kasper was awarded the Milton Safenowitz postdoctoral fellowship from the ALS Association. With QurAlis, Kasper won two Golden Tickets (Amgen 2017 and Pfizer 2018), became a JLabs member in 2018, the 2020 NEVY Award for Best Emerging Life Science Company, the 2020 Fierce15 Biotech award, and was awarded the 2022 Henri Termeer Transatlantic Connections Award. He holds a Ph.D. in neuroscience from the Graduate School Neurossciences of Amsterdam and Rotterdam and a M.Sc. In biomedical sciences with a focus on neuroscience from the University of Amsterdam.