Panbela Announces Publication of Clinical Data Titled: Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial
- Positive clinical data from a Phase 1 study
- DFMO treatment was well tolerated
- Median time-to-progression of 19.8 months
- Overall objective response rate of 9%
- Progression-free survival at 2 years was 29.5%
- None.
Insights
The recent findings from the clinical study on CPP-1X, or DFMO, in combination with chemotherapy for neuroblastoma patients, indicate a potential advancement in the treatment of this aggressive childhood cancer. Neuroblastoma, a cancer arising from immature nerve cells, is notorious for its poor prognosis in relapsed and refractory cases. The median time-to-progression of 19.8 months is a significant metric, as it suggests a prolonged period during which the disease does not advance. This is particularly noteworthy in a heavily pretreated population where treatment options are limited and often ineffective.
The overall response rate of 9% and the 26% rate for any response, while modest, are encouraging when considering the historical challenges associated with treating this disease. Furthermore, the 29.5% two-year progression-free survival (PFS) rate for the entire cohort is a critical indicator of the treatment's potential efficacy. The fact that three patients have remained disease-free for over four years post-treatment without additional therapy could signify a durable response in a subset of patients, which is a remarkable outcome in the context of high-risk neuroblastoma.
The tolerability of DFMO is a crucial aspect of its potential as a therapeutic option. In diseases like neuroblastoma, where patients are often young and have already undergone extensive treatment, a well-tolerated oral medication can significantly improve the quality of life. This aspect, coupled with the reported responses, positions DFMO as a promising candidate in the oncology pipeline.
From an investment perspective, the safety profile and efficacy data could positively influence Panbela Therapeutics' market valuation. The ongoing ANBL-1821 Phase 2 trial will be pivotal in confirming these results and potentially leading to a broader adoption of the treatment. Investors should monitor the progression of this trial closely, as positive Phase 2 results could lead to increased confidence in the company's prospects and potentially attract partnership opportunities or interest from larger pharmaceutical companies.
Neuroblastoma treatments represent a niche but critical market within oncology. The recent FDA approval of DFMO for reducing the risk of relapse in high-risk neuroblastoma patients has already positioned Panbela Therapeutics as a key player in this space. The current study's positive results could further strengthen the company's market position. As new treatment options are direly needed for relapsed refractory neuroblastoma, a drug like DFMO that shows promise in early trials could capture significant market share if later-phase trials confirm its efficacy and safety.
Analysts should evaluate the competitive landscape, considering that the neuroblastoma treatment market is relatively small but with high unmet needs. The potential commercial success of DFMO hinges not only on clinical outcomes but also on reimbursement strategies, market access and adoption rates among oncologists. A detailed market analysis post-Phase 2 trial results will be essential to forecast the drug's market potential accurately.
A Phase 1 clinical study showed that high dose DFMO treatment in combination with chemotherapy may provide therapeutic benefits to heavily pretreated patients with neuroblastoma.
MINNEAPOLIS, Jan. 18, 2024 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs announces the publication of clinical data from studies of CPP-1X (also known as α-Difluoromethylornithine (DFMO) or Eflornithine) in neuroblastoma. According to Hogarty et al, children with relapsed refractory neuroblastoma have dismal outcomes and new therapeutic options are needed. Data published in the British Journal of Cancer investigated the tolerability and activity of depleting polyamines by high dose CPP-1X and celecoxib in combination with standard of care chemotherapy in heavily pretreated neuroblastoma patients. Results showed that DFMO treatment was well tolerated, and the median time-to-progression was 19.8 months. The work reflects the Company’s previous collaboration with New Advances in Neuroblastoma Therapy Consortium (NANT) (https://www.nant.org/). A link to the publication can be found here: https://www.nature.com/articles/s41416-023-02525-2.
From the Phase 1 dose range finding study of CPP-1X in heavily pretreated neuroblastoma patients, CPP-1X was well tolerated. The best overall response included 2 partial responses (PR), 4 minor responses (MR), 10 Stable disease (SD), 7 progressive disease (PD) and 1 unevaluable. All patients with an overall response of PR or MR sustained this response until stopping or completing protocol therapy. The overall objective response rate (CR+PR) was
These results build upon the recent FDA approval of CPP-1X or DFMO to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Results from these studies suggest that CPP-1X is a safe, oral treatment option that may improve response rates in heavily pretreated relapsed refractory neuroblastoma patients and are the basis for the ongoing ANBL-1821 Phase 2 trial.
“We are excited about the publication of these Phase 1 trial results in light of the recent DFMO FDA approval for patients in maintenance therapy. From this dose escalation study, our collaborators were able to demonstrate high dose DFMO is well tolerated and demonstrated activity in patients with heavily pretreated neuroblastoma,” said Elizabeth Bruckheimer, PhD, Vice President & Chief Scientific Officer of Panbela. “Moreover, three patients remain alive over four years from treatment end without any additional therapy which suggests that high dose DFMO treatment in combination with chemotherapy may be a potential treatment option for this high unmet need population.”
"Overall, these results in addition to the recent approval of DFMO as a maintenance therapy, suggests a role for polyamine inhibition therapy for neuroblastoma that may impact other cancer types such as prostate cancer. We are excited by these results and the potential role for CPP-1X in the clinical management of neuroblastoma and cancer as a whole,” said Dr. Bruckheimer. “These studies were the basis for the ongoing Children’s Oncology Group Phase II trial in relapsed refractory neuroblastoma to support the goal of developing effective novel therapies for patients with unmet medical needs.”
First author Michael Hogarty, MD, Professor of Pediatrics at the University of Pennsylvania, and Children’s Hospital of Philadelphia said, "The results from the Phase 1 study have built upon the preclinical work performed in my laboratory demonstrating a role of deep polyamine depletion as a potential therapeutic target for relapsed or refractory neuroblastoma. By understanding the underlying biology and role of MYC signaling and the polyamine pathway in neuroblastoma, we are able to show the potential impact of high-dose DFMO in neuroblastoma."
About Panbela
Panbela Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing disruptive therapeutics for patients with urgent unmet medical needs. Panbela’s lead assets are Ivospemin (SBP-101) and Flynpovi. Further information can be found at www.panbela.com . Panbela’s common stock is listed on The Nasdaq Stock Market LLC under the symbol “PBLA”.
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Contact Information:
Investors:
James Carbonara
Hayden IR
(646) 755-7412
james@haydenir.com
Media:
Tammy Groene
Panbela Therapeutics, Inc.
(952) 479-1196
IR@panbela.com
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