FDA Accepts IND Application for ST-001 in Relapsed/Refractory SCLC

Developers anticipate launching the phase 1a/1b SCLC trial in the second or third quarter of 2025.

The FDA has approved an investigational new drug application (IND) for ST-001, a novel fenretinide phospholipid suspension, as an intravenous therapy for patients with relapsed/refractory small cell lung cancer (SCLC), according to a press release from the developer, SciTech Development, Inc.¹

Following a thorough safety review from the FDA, the agency indicated that developers may proceed with a proposed clinical investigation of fenretinide phospholipid suspension for patients with SCLC. Investigators will evaluate the safety profile, pharmacology, and maximum tolerated dose of ST-001 in this patient population as part of a phase 1a/1b trial.

Developers anticipate launching the SCLC trial in the second or third quarter of 2025.

“[SCLC] remains a formidable challenge with few effective therapies for patients who relapse or don't respond to initial treatment,” Gregory Peter Kalemkerian, MD, a clinical professor and thoracic oncologist at The University of Michigan, stated in the press release.¹ “ST-001 represents a promising approach, and I'm excited to oversee this trial to evaluate its safety and potential for [patients with] SCLC who desperately need new options.”

Developers designed ST-001 nanoFenretinide to harness a patented drug delivery system, which may overcome historical bioavailability challenges associated with fenretinide while administering high intravenous doses with enhanced efficacy and minimized toxicity. Once the fenretinide infiltrates the cancer cell, investigators hypothesize that the agent may affect multiple biochemical pathways to ultimately elicit cell death through apoptosis.² Such biochemical pathways include retinoid receptors, oxygen radicals, and ceramide inhibition.

“We are thrilled about the FDA's approval of our IND for ST-001 in SCLC. This is a pivotal moment that brings us closer to offering a transformative treatment option for patients [with] this devastating disease,” Earle T. Holsapple, chief executive officer at SciTech Development.¹ “Our team's dedication to advancing ST-001's potential through innovative nanotechnology is showing promising results, and we're eager to see its impact in the clinic.”

Investigators are also evaluating treatment with ST-001 for patients with T-cell non-Hodgkin lymphoma in a phase 1a/1b study (NCT04234048). According to preliminary results shared in December 2024, ST-001 yielded stable disease and partial responses—1 initial and 1 confirmed response—at earlier times than anticipated.³ Additionally, the nanoparticle delivery system demonstrated the desired pharmacokinetic profile.

“The early findings from the phase 1a trial of ST-001 nanoFenretinide are incredibly promising. Achieving a partial response at this stage is a remarkable step forward and highlights the potential of this innovative treatment to address significant gaps in care for patients with cutaneous T-cell lymphoma,” Larisa J. Geskin, MD, a professor of dermatology at Columbia University Medical Center and director of the Comprehensive Skin Cancer Center at the Division of Cutaneous Oncology in the Department of Dermatology, stated in a press release on these findings.³

Investigators of this phase 1 trial assessed the safety profile, pharmacology, and maximum tolerated dose of ST-001 among patients with relapsed/refractory T-cell non-Hodgkin lymphoma.⁴ The trial’s primary end point was the maximum tolerated dose. Secondary end points included complete responses and partial responses, incidence of treatment-related adverse effects, fenretinide half-life, and activation of T-lymphocytes and natural killer cells following treatment.

Patients 18 years and older with cutaneous T-cell lymphoma, peripheral T-cell lymphoma not otherwise specified, angioimmunoblastic T-cell lymphoma, or follicular T-cell lymphoma were eligible for enrollment on the trial. Other requirements for study entry included having an ECOG performance status of 0 or 1, a life expectancy of more than 6 months, and normal organ and marrow function.

References

1. SciTech Development announces 2nd FDA approval of a phase 1 a/b IND For ST-001. New IND targets the treatment of relapsed/refractory small cell lung cancer following Its previous approval in the treatment of T-cell NHL. News release. SciTech Development, Inc. April 8, 2025. Accessed April 9, 2025. https://tinyurl.com/43eas9ra
2. ST-001: nanoFenretinide therapeutic profile. SciTech Development, Inc. Accessed April 9, 2025. https://tinyurl.com/3fym5jtv
3. SciTech Development announces promising preliminary results in phase 1a trial for T-cell non-Hodgkin lymphoma. News release. SciTech Development, Inc. December 3, 2024. Accessed April 9, 2025. https://tinyurl.com/mryakebh
4. Phase 1 trial of ST-001 nanoFenretinide in relapsed/​refractory T-cell non-Hodgkin lymphoma. ClinicalTrials.gov. Updated March 26, 2025. Accessed April 9, 2025. https://tinyurl.com/3sf4snna