Novel SA-53 MDM2 Inhibitor Therapy Has Potential to be a Breakthrough in Genetically Targeted Cancer Treatment
(CLEVELAND, OH) – Steady progress for Lamassu Biotech’s potentially transformative cancer treatment. The private, pioneering pharmaceutical company today announced the latest significant clinical milestone in its Phase 1/2 trial investigating SA-53, a first-in-class, small-molecule MDM2 inhibitor. SA-53 has now successfully cleared the previous dose level and is now escalating to a new cohort at 16 times the initial starting dose. This represents a major safety threshold for the program, and the data continues to distinguish SA-53 within the MDM2 landscape.
SA-53 is a genetically targeted therapy designed to reactivate the tumor suppressor protein p53 commonly referred to as the “Guardian of the Genome. It does this by inducing highly targeted tumor cell death and inhibit growth without the systemic toxicity often associated with traditional chemotherapy. The potential for impacts on patients is exceptionally broad since the p53 pathway is functional (p53 wild type) in approximately half of all human cancers.
Key clinical highlights to date include:
- Clean Safety Profile: All safety data to date is excellent. We have observed zero clinically significant Dose-Limiting Toxicities (DLTs) across all cohorts studied.
- Competitive Differentiation: Prior MDM2 inhibitors encountered Grade 4 toxicity events and failed to escalate beyond 8× their starting dose. In contrast, SA-53 passed the equivalent 8× level with only Grade 1 toxicity, escalating and enrolling to ongoing cohort of 16× without significant safety concerns.
- Manageable Hematological Profile: Within the safety period we are observing a maximum thrombocytopenia (platelet reduction) of only 30% a known pharmacodynamic marker of target engagement. Platelet counts have consistently recovered within 10 days, and critically, all nadir (lowest) counts have remained within the normal range.
- Efficacy Projections: Based on our preclinical models, we believe we have now reached a dose level capable of achieving meaningful tumor growth inhibition in sarcoma—a signal we expect to see reflected in emerging clinical data.
Reaching this safety threshold is a significant milestone for Lamassu Biotech’s innovative new cancer protocol.
“The lack of traditional MDM2-related toxicity at these levels gives us great confidence as Cleveland Clinic begins this new cohort and approach the Phase 2 efficacy stage,” stated Lamassu CEO, Dr. Gabi Hanna.
“The safety evaluation of SA-53 continues to be highly encouraging, which is the paramount goal of any clinical study,” said Greg Palmer, Ph.D., co-founder and Chief Scientific Officer for Lamassu Biotech. “We are especially pleased to have no significant DLTs to date, and no significant safety concerns as we move rapidly through Phase 1. We continue to be well positioned to advance to the Phase 2 efficacy assessment in an expedited fashion.”
“Our team and our partners at Cleveland Clinic continue to be pleased with the progress being made as we advance SA-53 through Phase 1 and into Phase 2. The latest favorable safety data further validates our preclinical assessments and affirms the FDA’s decision to grant an accelerated study protocol design,” Dr. Hanna continued.
The ongoing Phase 1/2 trial, conducted in collaboration with the National Institutes of Health (NIH) and Cleveland Clinic, is designed to determine the safety, tolerability, pharmacokinetics, and preliminary efficacy of SA-53. The therapy is being investigated in adult patients with sarcoma and other solid tumors who possess the p53 wild-type gene and who currently have limited therapeutic options. The trial is overseen by Principal Investigator Peter Anderson, MD, PhD, and Joseph Wooley, MD, at the Cleveland Clinic.
New patients continue to be screened and will be added to the trial on an ongoing basis. Complete details about the trial design, locations, and participation criteria can be found HERE.
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About Lamassu Biotech
Lamassu Biotech is a private clinical stage, pioneering pharmaceutical company focused on developing novel therapeutics for severe and unmet medical needs. Leading assets include SA-53, a molecular targeted MDM-2 inhibitor that targets P53 wild-type cancers currently at Phase 1/2 clinical trial, and RABI-76,7 a lipase inhibitor at Phase 2 clinical trial. With decades of experience, Lamassu specializes in advancing transformational treatments from concept to bedside with efficiency and precision. Lamassu partners and collaborates with top-flight institutions like Mayo Clinic, Cleveland Clinic, and NIH, leveraging the best intellectual capital to accelerate the application of treatments that bring hope and healing. Learn more at LamassuBiotech.com.