QurAlis Scores Major Investment with Biomarker-Fueled Strategy

QurAlis, a biotech company pioneering a biomarker-driven approach to treating ALS, announced a successful conclusion to its oversubscribed Series B round on Thursday, raising a whopping $88 million. The funding marks a major milestone in the company’s mission to make a real difference in the lives of those living with ALS.

QurAlis, a Cambridge, Massachusetts-based biotech company, has received a significant boost with a new round of financing. Led by EQT Life Sciences, the financing brings the company’s total funds to a whopping $143.5 million. Along with the financing, two new board members have been appointed: Cillian King, Ph.D., managing director at EQT Life Sciences, and Laia Crespo, Ph.D., partner at Sanofi Ventures. This is exciting news for QurAlis and will undoubtedly give them the resources they need to continue their innovative work in the biotech space.

In May 2020, the company made a major breakthrough, closing a $42 million Series A round of funding. This funding will enable the company to take its operations to the next level, furthering its growth and impact.

QurAlis is revolutionizing the development of precision medicines for neurodegenerative diseases such as ALS, with a strong focus on genetically validated targets and a biomarker-driven approach. According to CEO Thomas King, this is the way forward for creating effective and targeted treatments.

QurAlis is focused on a groundbreaking target – the protein stathmin-2. Encoded by the STMN2 gene, stathmin-2 is a crucial factor in neural repair and axonal stability, and its downregulation has been linked to Amyotrophic Lateral Sclerosis (ALS). If successful, QurAlis’ research could have far-reaching implications for those affected by this devastating neurological condition.

The company has just acquired new funds to further progress their two clinical-stage programs, signalling an exciting step forward in their development.

QRL-201 is a revolutionary development in the fight against ALS, with the potential to rescue STMN2 loss of function in patients with TDP-43 pathology. This pathology is the primary driver of ALS in 97% of patients, making QRL-201 a major breakthrough in the search for a cure.

In ALS, a mysterious exon in the pre-mRNA of STMN2 is revealed, halting transcription and leading to a deficiency of the STMN2 protein. This unfortunate event causes nerve cells to degenerate, leading to devastating effects on the person afflicted.

QRL-201 is an innovative splice switching ASO that works to protect motor neurons from neurodegeneration. By binding to pre-mRNA, it restores the STMN2 protein, giving hope to those affected by diseases that cause motor neuron degeneration. Developed by QurAlis founder and CEO, Kasper Roet, Ph.D., QRL-201 is a promising solution for those in need of a breakthrough in this space.

QurAlis is set to begin a clinical trial on its asset in December 2022, after the application was authorized by Health Canada. According to CEO Roet, the two and a half year trial will evaluate safety, tolerability and proof of concept. With this trial, QurAlis hopes to make a major breakthrough in the medical field.

QurAlis is embarking on an ambitious project to explore the issue in depth across the globe, with a focus on the U.S., U.K. and Europe. This comprehensive effort will provide invaluable insights and help to inform our understanding of the topic.

QRL-101 is the company’s other clinical-stage asset, a unique ion channel opener that has the potential to dramatically reduce motor neuron degeneration in ALS patients. Currently, the compound is being assessed in a safety and tolerability study of healthy volunteers to determine its efficacy. If successful, this revolutionary treatment could drastically improve the quality of life for ALS sufferers.

QurAlis began Phase I of their study in January 2023 and are on track to conclude the phase in the third quarter of the year. They plan to transition to patient trials in the fourth quarter, bringing them one step closer to achieving their goal.

Roet revealed that both of these targets are precision medicine targets which have been identified as having missplicing in large cohorts of sporadic ALS patients. This means that target engagement and efficacy biomarkers can be combined with functional readouts to gain a more comprehensive understanding of their effects.

King is absolutely thrilled about QurAlis’ FlexASO platform, as it stands apart from other products in the market. He believes it is a truly unique and innovative solution.

In addition to the pharmacological kinetics, King highlighted the platform’s remarkable biodistribution capabilities, emphasizing that its penetration of deep brain tissue could enable the treatment of a wealth of neurodegenerative diseases beyond ALS and motor neuron disease. This opens up a whole new world of therapeutic possibilities.

QurAlis has positioned itself as a platform company, with the innovative FlexASO product leading the way. King believes this product can revolutionize the industry, offering a unique and powerful tool to businesses of all sizes.

Biomarkers Gain Traction

QurAlis is keeping pace with the FDA’s evolving perspective on neurodegenerative diseases, offering a progressive approach to treatment and prevention. With their innovative approach, they’re helping to shape the future of healthcare in the field.

The FDA recently gave the green light to Eisai and Biogen’s Leqembi (lecanemab), marking a major milestone in the fight against Alzheimer’s disease. With this approval, the FDA has demonstrated their willingness to approve treatments that target amyloid beta plaques, which are believed to be a major cause of the disease. This is a major step forward in the quest to find effective treatments for Alzheimer’s and hope for those affected by the condition.

“I’m thrilled to see this move made and I’m hopeful that the field is heading in this direction,” enthused King. “It’s a great step forward and I’m optimistic about the possibilities.”

The FDA is nearing a decision on Biogen and Ionis’ tofersen for superoxide dismutase 1 (SOD1) ALS, with an upcoming advisory committee meeting set to take place. Roet highlighted the significance of this potential breakthrough, which could offer hope to those suffering from ALS.

On March 22, the FDA’s Peripheral and Central Nervous System Drugs advisory committee will evaluate the potential of neurofilament light chain (NfL) as a reliable biomarker for predicting clinical benefit. This important test could open up a new world of possibilities for those suffering from various neurological conditions.

QurAlis is leading the way in accelerating drug development in ALS by focusing on biomarkers. We are thrilled to see this field mature and grow, and are confident that it will make a major impact in the future.

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