Ensuring Excellence from Lab to Life
At Sarcomatrix, clinical and regulatory excellence is at the heart of our mission to bring transformative therapies to patients.
Preclinical Progress
Our culture is rooted in a deep commitment to patients, guided by science, and powered by data-driven decision-making. We hold ourselves to the highest ethical standards, always striving to do what is right. United by dedication, resilience, and a shared purpose to make a meaningful impact on patients’ lives, we operate as a cohesive, mission-driven team inspired by the values of integrity, innovation, and collaboration.
IND Submission
Our team’s unique skill sets include industry-leading muscle research expertise originating from the Dean Burkin Lab, and the development of a novel drug discovery platform and screening system for integrin activators. Our scientific foundation is backed by comprehensive expertise spanning the full spectrum of drug development and commercialization. From translational research and IND-enabling studies to the strategic design and execution of clinical trials, our team brings the capabilities needed to advance programs efficiently and effectively.
Clinical & Regulatory
Advancing with Purpose, Aligned with Global Standards
Clinical Trial Plan
In collaboration with patients, families, and physicians, we plan to initiate a Phase 1 human clinical safety trial for S-969. Launching this study within the next 12–18 months marks a key milestone. The planned Phase I/II proof-of-concept study represents a pivotal inflection point in the advancement of our program.
Use of Funds
Our near-term goal is to generate human proof-of-concept data through Phase 2 studies. We project that achieving accelerated approval—along with fulfilling post-marketing (Phase 4) commitments—will require approximately $180 million over the next three to five years.
Regulatory Designations
We are pursuing multiple FDA designations for S-969, including Orphan Drug, Breakthrough Therapy, Fast Track, and Priority Review. The FDA has responded positively to our innovative approach, and a recent NIH assessment confirmed our readiness to submit an IND application.
Specific Trials
Like several companies before us—Sarepta Therapeutics (Exondys 51, Vyondys 53, Amondys 45, Elevidys), NS Pharma (Viltepso), and Wave Life Sciences—we expect to advance our lead oral drug, S-969, through the FDA’s accelerated approval pathway. This process is supported by the use of surrogate biomarkers, and we are focused on generating robust data to meet these regulatory requirements while expediting patient access to urgently needed treatment.