Processa Pharmaceuticals Announces Positive Efficacy Results from Preliminary Evaluation of Phase 1b Dose-escalating Trial with NGC-Cap in Gastrointestinal Cancer
Processa Pharmaceuticals has announced positive results from their Phase 1b trial of NGC-Cap in gastrointestinal cancer patients. The trial involved 12 patients, with 66.7% achieving progression-free survival (PFS) ranging from 5 to 11 months. At the highest dose, all patients showed PFS, with two partial responses and one stable disease. Compared to monotherapy capecitabine, 5-FU exposure was greater, and FBAL exposure was lower, with better or similar side effects. These promising results support further development, with a Phase 2 trial in breast cancer slated for the third quarter of 2024.
- 66.7% of patients achieved PFS ranging from 5 to 11 months.
- At the highest dose, 100% of evaluable patients showed PFS.
- Two patients had partial responses, and one had stable disease at the highest dose.
- Greater 5-FU exposure and lower FBAL exposure compared to monotherapy capecitabine.
- Similar or better side-effect profile compared to monotherapy capecitabine.
- MTD and RP2DR defined for Phase 2 trials.
- Phase 2 trial in breast cancer scheduled for Q3 2024.
- Results are preliminary and based on a small sample size of 12 patients.
- Efficacy data are early and need further validation in larger trials.
- Previous treatments were unsuccessful, indicating a challenging patient population.
The preliminary results from Processa Pharmaceuticals' Phase 1b trial for NGC-Cap show promising early efficacy in patients with advanced gastrointestinal cancer. The observed 66.7% progression-free survival (PFS) rate indicates that NGC-Cap may be more effective than current treatments. The higher exposure to 5-Fluorouracil (5-FU) and lower exposure to fluoro-beta-alanine (FBAL) could potentially translate to better treatment outcomes and reduced toxicity, which are critical for patients who have relapsed or are resistant to existing therapies. The use of RECIST 1.1 criteria for evaluating anti-tumor activity adds a standardized benchmark to the results, making the findings more robust and comparable to other studies.
In the context of oncology, the defined Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose Range (RP2DR) are significant as they provide a clear path for further clinical development. Early efficacy signals, along with a favorable safety profile, are encouraging for the ongoing development of NGC-Cap and suggest potential for use in other cancers where capecitabine and 5-FU are standard treatments.
From a financial perspective, the positive results from Processa Pharmaceuticals' Phase 1b trial could have a beneficial impact on the company's valuation. The promising efficacy and safety profile of NGC-Cap enhances its market potential, which can attract investor interest and possibly lead to increased stock prices. The comparison to existing treatments shows a potential market differentiation, which is essential for securing approval and adoption within the medical community.
The definition of the MTD and RP2DR is a critical milestone for advancing to Phase 2 trials, which signals the company's commitment to moving forward with NGC-Cap development. This progression reduces uncertainty and can be viewed positively by investors looking for tangible progress in the pipeline.
However, it's important to note that these are preliminary results and the transition to Phase 2 trials does not guarantee ultimate success. Investors should remain cautious about the inherent risks in clinical development despite the encouraging initial data.
The data indicating higher 5-FU exposure and lower FBAL exposure with NGC-Cap compared to monotherapy capecitabine suggests an improved pharmacokinetic profile. This can lead to enhanced efficacy and reduced side effects, which are important for improving patient quality of life and compliance with treatment regimens. The observed progression-free survival in a significant portion of patients (66.7%) and the establishment of the MTD and RP2DR mark essential steps in the development of a new chemotherapeutic option.
The trial results imply that NGC-Cap could potentially set a new standard in the treatment of gastrointestinal cancers, especially for patients who have exhausted other treatment options. The upcoming Phase 2 trials in breast cancer will be pivotal in validating these early findings and identifying the optimal dosage. The strategic focus on different cancer types reflects a broad applicability, further enhancing the drug's marketability and potential impact on clinical practice.
Eight of 12 evaluable patients (
At the highest NGC-Cap dose, all three evaluable patients had PFS with two partial responses (PR) and one stable disease (SD)
For all NGC-Cap doses, 5-Fluorouracil (5-FU) exposure was greater and fluoro-beta-alanine (FBAL) exposure was lower with a better or similar side-effect profile than monotherapy capecitabine
HANOVER, MD., June 11, 2024 (GLOBE NEWSWIRE) -- Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) (Processa or the Company), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs with improved efficacy and safety, today announced positive efficacy results from the preliminary evaluation of its recently completed Phase 1b clinical trial which defined the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose Range (RP2DR) for Next Generation Capecitabine (NGC-Cap) administered to patients with Stage III or IV gastrointestinal tract (GI) cancer.
“We are encouraged by the preliminary efficacy analysis from our NGC-Cap Phase 1b dose-escalating safety/tolerability trial demonstrating some anti-tumor activity in patients with advanced GI cancer who have progressive cancer after relapsing or not responding to prior therapy. The favorable response is likely due to NGC-Cap’s ability to distribute more 5-FU to cancer cells than monotherapy capecitabine. The promising Phase 1b safety and tolerability profile plus these early efficacy signals provide validation for further development of NGC-Cap,” stated David Young, PharmD, Ph.D., President of Research and Development at Processa. “From this Phase 1b trial, we have been able to define the MTD and the RP2DR to use in our Phase 2 Optimal Dosage Regimen trial in breast cancer in the third quarter of 2024.”
Dr. Young added, “Given the need for more effective chemotherapy treatment with improved tolerability across multiple types of cancer, we believe that NGC-Cap has the potential to provide a safer, more efficacious option to treat the different cancers for which capecitabine and 5-FU are presently used.”
The NGC-Cap Phase 1b trial is evaluating ascending doses of capecitabine when administered after a single dose of PCS6422 in Stage III or IV patients with advanced, relapsed or refractory progressive GI cancer. All patients relapsed from or failed all other treatments, including prior treatment with capecitabine or 5-FU. For all doses of capecitabine in the Phase 1b NGC-Cap trial, exposure to 5-FU was greater and exposure to FBAL was lower with a better or similar side effect profile compared with monotherapy capecitabine. In addition, preliminary analysis of the efficacy data demonstrated early evidence of anti-tumor activity of capecitabine combined with PCS6422, which was assessed using RECIST 1.1 evaluations (Response Evaluation Criteria in Solid Tumors) by scans every eight weeks.
Key Efficacy Findings from Preliminary Analysis of NGC-Cap Phase 1b Clinical Trial (NCT04861987)
- In all evaluable patients receiving one dose of PCS6422 and seven days of capecitabine, PR or SD was observed in
66.7% (8 out of 12) of evaluable patients, including two with PR and six with SD. The length of PFS was approximately 5 to 11 months across these patients. - At the MTD of 225 mg of capecitabine dosed twice daily after a single dose of PCS6422, all three evaluable patients (
100% ) had PFS with the time to progression being approximately 5 to 7 months. - At the second highest dose of 150 mg capecitabine dosed twice daily after a single dose of PCS6422,
66.7% (2 out of 3) of evaluable patients had SD with the time to progression of approximately 3 to 7 months. - These two dosage regimens will be further evaluated in the Phase 2 trial in breast cancer patients to determine the optimal dosage regimen for the pivotal trial.
- By comparison, in the capecitabine product label, 301 metastatic colorectal cancer patients treated with monotherapy capecitabine had an overall response rate of approximately
21% and the time to progression of approximately 4.5 months.
About Capecitabine Administered with PCS6422 (NGC-Cap)
NGC-Cap combines the administration of PCS6422, the Company’s irreversible dihydropyrimidine dehydrogenase (DPD) enzyme inhibitor, with low doses of capecitabine. Capecitabine is the oral form of 5-FU, and along with 5-FU is among the most widely used chemotherapy drugs, particularly for the treatment of solid tumors. When metabolized (after oral ingestion) it becomes 5-FU in the body, which, in turn, metabolizes to molecules called anabolites that actively kill duplicating cells, such as cancer cells, and to molecules called catabolites that only cause side effects. The presence of the DPD enzyme plays an integral role in the undesirable conversion of 5-FU to catabolites.
PCS6422 irreversibly inhibits DPD. PCS6422 is neither toxic nor active as a single agent in animals at comparable dose levels. However, when administered in combination with capecitabine or 5-FU, PCS6422 decreases the metabolism of 5-FU to the catabolites that only cause side effects, allowing more of the 5-FU to reach the cancer cells.
About Processa Pharmaceuticals, Inc.
Processa is a clinical-stage pharmaceutical company focused on developing the Next Generation Chemotherapy (NGC) drugs to improve the safety and efficacy of cancer treatment. By combining its novel oncology pipeline with proven cancer-killing active molecules and the Processa Regulatory Science Approach, as well as experience in defining Optimal Dosage Regimens for FDA approvals, Processa not only will provide better therapy options to cancer patients but will also increase the probability of FDA approval for its NGC drugs following an efficient path to approval. Processa’s NGC drugs are modifications of existing FDA-approved oncology drugs resulting in an alteration of the metabolism and/or distribution of these drugs while maintaining the existing mechanisms of killing the cancer cells. The Company’s approach to drug development is based on more than 30 years of expertise to efficiently design and conduct clinical trials that demonstrate a positive benefit/risk relationship. The Processa team has a track record of obtaining over 30 indication approvals across almost every division of the FDA. Using its proven Regulatory Science Approach, the Processa Team has experience defining the Optimal Dosage Regimen using the principles of the FDA’s Project Optimus Oncology initiative. The advantages of Processa’s NGCs are expected to include fewer patients experiencing side effects that lead to dose discontinuation, more significant cancer response and a greater number of patients – in excess of 200,000 for each NGC drug – who will benefit from each NGC drug. Processa is currently 1) starting to initiate sites for the Phase 2 study that will identify the optimal dosage regimen for Next Generation Capecitabine (PCS6422 and capecitabine to treat breast, metastatic colorectal, gastrointestinal, pancreatic and other cancers), 2) defining the design of the Next Generation Gemcitabine (PCS3117 to treat pancreatic, biliary, lung, ovarian, breast and other cancers) Phase 2 optimal dosage regimen study to discuss with FDA, and 3) defining the formulation and toxicology program for Next Generation Irinotecan (PCS11T to treat lung, colorectal, gastrointestinal, pancreatic and other cancers).
For more information, visit our website at www.processapharma.com.
Forward-Looking Statements
This release contains forward-looking statements. The statements in this press release that are not purely historical are forward-looking statements which involve risks and uncertainties. Actual future performance outcomes and results may differ materially from those expressed in forward-looking statements. Please refer to the documents filed by Processa Pharmaceuticals with the SEC, specifically the most recent reports on Forms 10-K and 10-Q, which identify important risk factors which could cause actual results to differ from those contained in the forward-looking statements.
Company Contact:
Patrick Lin
(925) 683-3218
plin@processapharma.com
Investor Relations Contact:
Yvonne Briggs
LHA Investor Relations
(310) 691-7100
ybriggs@lhai.com
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