FDA Grants Orphan Drug Designation for SM-88 for Treatment of Pancreatic Cancer

The FDA has granted an Orphan Drug designation to SM-88 for the potential treatment of patients with pancreatic cancer.

The FDA has granted an Orphan Drug designation to SM-88 for the potential treatment of patients with pancreatic cancer, Tyme Technologies, Inc, announced in a press release.

“Receiving Orphan Drug designation for SM-88 is another important milestone to emerge from our innovative pipeline of cancer metabolism-based compounds,” said Steve Hoffman, chairman and chief executive officer of TYME, in a statement. “We are pleased with the progress we are making in the clinic and believe that SM-88, which is now in multiple late-stage clinical trials offers the potential to play a significant role in establishing a new treatment paradigm for more than 57,000 patients who are diagnosed with pancreatic cancer annually within the United States.”

The pivotal TYME-88 study has demonstrated encouraging tumor responses in 15 different cancer types in 4 clinical trials. Minimal serious grade 3 or higher adverse events (AEs) were observed as well in these studies.

The agent will be evaluated in the pivotal TYME-88-Panc study, which will assess the efficacy and safety of this treatment in patients with metastatic pancreatic cancer who have failed at least 2 prior lines of systemic therapy. SM-88 is used in combination with methoxsalen, phenytoin, and sirolimus (MPS).

This is a randomized multicenter, open-label phase 2/3. Patients enrolled in part 1 will receive SM-88 with MPS daily while part 2 will treat patients with the combination until unacceptable toxicity, disease progression, or treatment discontinuation criteria are met. In the control arm, patients will receive the physician’s choice of therapy or capecitabine, gemcitabine, and 5-FU.

The primary end point of the study is overall survival, and the secondary end point is progression-free survival. Other end points include relevant biomarkers, quality of life, safety, and overall response rate.

Patients in part 1 of the study will be given 1 of 2 dose levels, and part 2 will be a subsequent expansion of the study to further evaluate the safety and efficacy of the recommended phase 2 dose from part 1. Overall, 250 patients are planned to be enrolled in the second part of the trial, who will then be randomized 1:1 to either the SM-88 or control arm.

To be included in part 1 of the study, patients had to have a biopsy-proven diagnosis of metastatic pancreatic adenocarcinoma with radiographic disease progression on or after 1 or more systemic therapies. Chemotherapy that was given as part of a chemoradiation treatment in the non-metastatic disease setting does not count as a line of therapy in the study, but adjuvant chemotherapy given for at least 4 months would be considered the first line of therapy.

In part 2 of the trial, patients must have biopsy-proven disease on or after 2 prior lines of systemic therapy, in which chemotherapy in a prior chemoradiation regimen for nonmetastatic disease is not counted unless metastases develop within 6 months of completing the chemosensitization. Patients must also be eligible to receive 1 or more of the physician’s choice treatment options and have completed any prior investigational therapy at least 30 days prior to the study’s first dose. Patients had to have completed any other cancer treatments at least 14 days prior to the first study dose and have recovered from any major side effects. In addition, patients must have an ECOG performance status of 0 to 2 and adequate organ function.

Patients with an unacceptable risk in the opinion of the investigator and those with a history or evidence of any clinically significant disorders, conditions, or disease that would pose a risk to the patient’s safety or interfere with study evaluations are ineligible to enroll to the study. Patients also cannot enroll if they have a history of a concurrent or second malignancy, microsatellite instability-high disease with no prior pembrolizumab (Keytruda) therapy, actionable mutations that have not been treated with an approved therapy for the mutation, radiation to all target lesions within 12 weeks of the study baseline, or no measurable target lesions.

SM-88 is an investigational modified proprietary tyrosine derivative administered orally. By breaking down the cells’ key defenses that lead to cell death with oxidative stress and exposure to the immune system, this agent is believed to interrupt the metabolic processes of cancer cells. SM-88 is not yet approved for any indication in any disease type at this time.


TYME announces orphan drug designation for SM-88 as potential treatment for patients with pancreatic cancer. News Release. August 3, 2020. Accessed August 3, 2020. https://bwnews.pr/3k9LsMo

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