On January 19, 2023) Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has issued the Adoption of Commission Implementing Decision relating to the designation of ivospemin (SBP-101) as an orphan medicinal product in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma (PDA) (Press release, Panbela Therapeutics, JAN 19, 2023, View Source;utm_medium=rss&utm_campaign=panbela-announces-adoption-of-commission-implementing-decision-from-the-ema-for-the-orphan-designation-of-ivospemin-sbp-101 [SID1234626390]). The Commission adopted the decision on January 13, 2023 and it will be published for information in all official languages of the EU in the Community Register of Orphan Medicinal Products

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Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to SBP-101.

"We are pleased to receive notice that the European Commission has adopted the positive decision for orphan drug designation from EMA’s Committee for Orphan Medicinal Products," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "The designation of orphan drug status within the EU is an important achievement as we continue to advance the global ASPIRE trial with the potential that this may be an option for patients with first line metastatic pancreatic cancer in the future."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies’ developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria may qualify for financial and regulatory incentives, including a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.

Panbela is continuing to focus on site initiation and enrollment in the ASPIRE trial to ultimately deliver a more effective treatment for pancreatic cancer, a deadly disease with few treatment options. The Company expects that the full complement of sites will be open by mid 2023.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101 Ivospemin

Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. For more information, please visit View Source

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigatorinitiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.