Spine BioPharma, Inc. announced today the Phase 3 MODEL trial (MOderate – Severe Degenerative Disc Disease Evaluation of the Lumbar Spine) evaluating SB-01, a TGF-β antagonist for intradiscal treatment of patients with Chronic Low Back Pain (CLBP) associated with Degenerative Disc Disease (DDD), did not meet its primary endpoint of pain intensity and pain-related function at Month 6 post treatment compared to sham control.

A single intradiscal injection of SB-01 in CLBP patients associated with DDD demonstrated:

• A robust safety profile consistent with the previous Phase 2 study.
• SB-01 patients saw a numerical and clinically meaningful improvement in pain intensity and pain-related function.
• The SB-01 response was observed at all timepoints out to Month 6 and was durable at one year.
• There was no difference in success at any timepoint between 1- and 2-level patients.
• There was an inconsistent sham control response among sites, with some sites having much higher than anticipated sham control rates.
• In sites with an anticipated sham control response, SB-01 outperformed the sham control, almost achieving nominal statistical significance p=0.051.

The clinical trial was prospective, 1:1 randomized with 417 patients utilizing a single 1.5 ml intradiscal injection of SB-01 or a sham control. Both 1- and 2-level DDD patients were enrolled across 30 sites in the United States. Patients were followed at Week 2, Week 6, Month 3 and to the primary endpoint at Month 6 with an additional follow-up at Month 12. Patients were evaluated for pain intensity utilizing Numerical Rating Scale (NRS) and pain-related function using Oswestry Disability Index (ODI). Patients had to achieve both a 2/10-point improvement in NRS and 15/100-point improvement in ODI to be considered an overall composite success (primary endpoint).

As previously observed in the Phase 2 clinical trial of 325 patients, and in this Phase 3 clinical trial of 417 patients, SB-01 demonstrated a robust safety profile.

In the Intent-to-Treat (ITT) analysis of the 417 patients, the SB-01 group achieved primary endpoint success (ODI+NRS) of 67% at Month 6, which although clinically meaningful, did not reach statistical significance compared to the sham control group. The SB-01 response was present at the early timepoints. For the patients that reached the Month 12 follow-up at database lock (N=281), 62% of the SB-01 group achieved composite success (ODI+NRS). There was no difference in SB-01 success at any timepoint between 1- and 2-level patients.

In a secondary endpoint ITT analysis of pain-related function (ODI), the SB-01 group achieved a clinically meaningful success rate of 75% at Month 6, and 71% at Month 12 but was not statistically significant compared to the sham control.

Fran Magee, DVM, CTO, stated “The SB-01 patients responded as anticipated and consistent with the Phase 2 study. In contrast, the sham control response was statistically significantly higher than observed in the Phase 2 study. The statistical design of this Phase 3 study anticipated a high sham control success, as observed in the Phase 2 study and typically seen in CLBP studies. We are surprised by the very high sham control response in this study that kept us from achieving statistical significance.”

In this Phase 3 study, the very high sham response was not observed at all investigational sites. There is statistically reliable evidence of site-to-site heterogeneity in the sham control response with some sites demonstrating exceedingly high sham control response that was not observed in the homogenous SB-01 response across sites.

Given the site-to-site sham control response variability, a subset analyzing only patients (n=227) at sites that had an anticipated sham response, consistent with the previous Phase 2 study was performed. For the ITT analysis, utilizing the composite primary endpoint (ODI+NRS) the response in the SB-01 and sham control group was 70% and 59% respectively, nominal p=0.051. In the same patient subset, the secondary endpoint of ODI response in the SB-01 and sham control group was 79% and 69% respectively and compared to sham control with a nominal p=0.040. These analyses demonstrate that in the sites that had the anticipated sham control response rate, SB-01 outperformed the sham control patients.

Marc Viscogliosi, CEO, stated “Despite our disappointment in missing the primary endpoint, we are quite proud of enrolling and completing this trial under strict FDA guidelines. We ran a solid trial and had we achieved our anticipated sham control group response, the results would have been statistically significant in favor of SB-01.”

Christopher Gilligan, MD, Principal Investigator of the MODEL trial stated “This was a landmark, well-designed clinical trial with strict enrollment criteria and very high follow-up rates. The study employed a rigorous composite endpoint requiring patients to achieve both clinically meaningful improvement in both pain intensity and pain-related function. Pain studies are challenging and are susceptible to a sham control response, which was particularly high in this study. We are thankful to the patients, their families, investigators, clinical coordinators, and research associates who participated in this clinical trial for CLBP.”

There remains a significant need for less invasive, safe and effective treatment for CLBP associated with DDD. Spine BioPharma will complete the data analysis. We plan to meet with the FDA to discuss the results from this Phase 3 trial together with the earlier Phase 1 and 2 randomized trials and explore potential approval pathways for SB-01 as a treatment for CLBP associated with moderate-severe DDD.

About the MODEL Clinical Trial

SB-01 MODEL clinical trial is a U.S. multi-center, randomized, double-blind, placebo-controlled Phase 3 trial being conducted to establish the safety and effectiveness of SB-01 For Injection in adult patients with chronic low back pain and its associated impairment in pain-related function due to Lumbar DDD.

About SB-01 For Injection

SB-01 is a 7-amino acid synthetic peptide that binds to and antagonizes TGF-Beta activity. TGF-Beta is a pleiotropic cytokine expressed by almost every tissue and cell type, is stored in abundance in the extracellular matrix, and possesses suppressive and stimulatory signaling pathways. In many diseases, there are high concentrations of TGF-Beta that result in a spectrum of negative downstream effects including inflammation, fibrosis, neoinnervation, hyperexcitability of nerves and cell proliferation. SB-01 modulates TGF-Beta concentration, without eliminating it, mitigating the negative downstream effects.

About Spine BioPharma

Spine BioPharma is committed to developing non-opiate, non-surgical treatments that will reduce pain, restore function, and slow or stop pathological disease progression. Spine BioPharma’s lead candidate, SB-01 For Injection, is a first-in-class treatment of DDD, offering potential clinical benefits of pain relief, restoration of function, and prevention of disease progression. To learn more about Spine BioPharma, visit www.spinebiopharma.com.

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