BPTH.OTC.PinkBio-path Holdings, INC
10-K: Bio-Path Holdings Reports Promising Interim Data in AML Trial, Advances Pipeline
Sentiment:
Annual Report
Bio-Path Holdings announces positive interim results from its Phase 2 AML clinical trial and provides updates on its other drug candidates.
Capital raiseThe company has determined that its available cash at December 31, 2023 will not be sufficient to fund current liabilities and capital expenditure requirements.The company expects to finance its foreseeable cash requirements through cash on hand, cash from operations, debt financings and public or private equity offerings.The company may seek to access the public or private equity markets whenever conditions are favorable.
Better than expectedThe complete remission rates for prexigebersen in combination with decitabine and venetoclax are significantly higher than the rates reported for decitabine and venetoclax alone in both newly diagnosed and relapsed/refractory AML patients.
Summary
- Bio-Path Holdings is a clinical-stage oncology company focused on RNAi nanoparticle drug development.
- Their lead drug candidate, prexigebersen, is being evaluated in a Phase 2 clinical trial for acute myeloid leukemia (AML).
- Interim data from the trial showed a 86% complete remission rate in newly diagnosed AML patients and a 57% complete remission rate in relapsed/refractory AML patients when prexigebersen was combined with decitabine and venetoclax.
- The company is also developing BP1002, targeting Bcl-2, which is in Phase 1 trials for lymphoma, CLL and AML.
- BP1003, targeting STAT3, is in IND enabling studies for pancreatic cancer, non-small cell lung cancer (NSCLC) and AML.
- BP1001-A, a modified version of prexigebersen, is in a Phase 1/1b trial for advanced solid tumors.
- The company's DNAbilize technology is available for out-licensing and partnering.
Sentiment
Score: 7
Explanation: The document presents positive clinical trial results and pipeline advancements, but the company's financial situation and dependence on future capital raises temper the overall sentiment. The company has a strong technology platform and is showing promising results, but the financial risks are significant.
Positives
- The interim data for prexigebersen in AML is significantly higher than the CR/CRi rates of 62% for newly diagnosed patients and 21% for refractory/relapsed patients treated with the combination treatment of decitabine and venetoclax alone.
- The company has successfully completed the first dose cohorts for BP1002 in lymphoma/CLL and AML, and BP1001-A in solid tumors, with no dose limiting toxicities.
- Preclinical studies suggest that BP1002 in combination with decitabine is efficacious in venetoclax-resistant leukemia and lymphoma cells.
- Preclinical studies have shown BP1003 to inhibit cell viability and STAT3 protein expression in NSCLC and AML cell lines.
- BP1003 successfully penetrated pancreatic tumors ex vivo and enhanced the efficacy of gemcitabine in a pancreatic cancer model.
Negatives
- The company has incurred significant operating losses since its inception and expects to continue to incur losses.
- The company has a limited cash balance of $1.1 million as of December 31, 2023, and has stated that substantial doubt exists about the Companys ability to continue as a going concern.
- The company is dependent on third-party manufacturers for its drug supplies.
- The company has no commercial products and no sales and marketing organization.
Risks
- The company needs substantial additional capital and may be forced to delay, reduce or eliminate its drug development programs if it cannot raise additional capital.
- The pharmaceutical and biotechnology industry is highly competitive.
- Clinical trials may be delayed or terminated.
- The company may not be able to obtain regulatory approval for its drug candidates.
- The company relies on third parties to conduct clinical trials and manufacture drug supplies.
- The company may not be able to establish sales and marketing capabilities.
- The company's drug candidates may not achieve market acceptance.
- The company's patent position may not adequately protect its drug candidates.
- The company's stock price has been volatile and is thinly traded.
Future Outlook
The company plans to pursue FDA expedited programs for Fast Track designation for prexigebersen and is evaluating whether to expand the Phase 2 clinical trial in Europe. The company also intends to apply its drug delivery technology to new disease-causing protein targets and expand into diseases other than cancer.
Management Comments
- The company believes that adding prexigebersen to the treatment combination of decitabine and venetoclax could lead to improved efficacy in AML patients.
- The company believes that BP1002 may be a potential treatment for venetoclax-relapsed AML patients.
- The company believes that the excellent safety profile of the DNAbilize chemistry, the novel lipid formula that allows for penetration of the tumor stroma, and the ability to target a single protein with precision, makes BP1003 an ideal candidate for combination with approved treatments to extend survival while maintaining quality of life for the patient.
Industry Context
The company is operating in a highly competitive pharmaceutical and biotechnology industry, with many companies pursuing novel drugs for AML, lymphoma, ovarian cancer, pancreatic cancer and other cancers. The company's focus on targeted therapy using antisense technology and its unique DNAbilize delivery system positions it to potentially address unmet needs in these areas.
Comparison to Industry Standards
- The CR/CRi rates of 86% for newly diagnosed AML patients treated with prexigebersen, decitabine and venetoclax is significantly higher than the CR/CRi rates of 62% for newly diagnosed patients treated with the frontline combination treatment of decitabine and venetoclax alone.
- The CR/CRi rates of 57% for relapsed/refractory AML patients treated with prexigebersen, decitabine and venetoclax is significantly higher than the CR/CRi rates of 21% for refractory/relapsed patients treated with the combination treatment of decitabine and venetoclax alone.
- Recent publications provide that response (CR + CRi) rates to combination treatment with decitabine and venetoclax (but without prexigebersen) are 42 to 52% for relapsed/refractory AML patients and 0 to 39% for relapsed/refractory secondary AML patients.
- Response rates to frontline treatment with decitabine and venetoclax (but without prexigebersen) are 62 to 71% for newly diagnosed AML patients.
Stakeholder Impact
- Shareholders may benefit from the positive clinical trial results and pipeline advancements, but face risks due to the company's financial situation and potential dilution from future capital raises.
- Employees may benefit from the company's progress, but face risks due to the company's financial situation.
- Patients may benefit from the development of new treatments for AML, lymphoma, CLL, pancreatic cancer, NSCLC and solid tumors.
- Creditors face risks due to the company's financial situation.
Next Steps
- The company plans to pursue FDA expedited programs for Fast Track designation for prexigebersen.
- The company is evaluating whether to seek to expand Stage 2 of the Phase 2 clinical trial in Europe.
- The company plans to file an IND application and initiate the first-in-humans Phase 1 study of BP1003 in patients with refractory, metastatic solid tumors.
- The Phase 1b portion of the BP1002 study is expected to commence after completion of BP1002 monotherapy cohorts.
- The Phase 1b portion of the BP1001-A study is expected to commence after successful completion of BP1001-A monotherapy cohorts.
Key Dates
| Date | Description |
|---|---|
| 2000-05 | The Company was incorporated as a Utah corporation. |
| 2007-09-27 | Bio-Path was established. |
| 2008-02 | Bio-Path Subsidiary completed a reverse merger with the Company. |
| 2014-03-10 | The Company's common stock commenced trading on the Nasdaq Capital Market. |
| 2014-12-31 | The Company changed its state of incorporation from Utah to Delaware. |
| 2016-04 | The Company entered into a lease agreement for lab space in Bellaire, Texas. |
| 2016-10 | Prexigebersen received orphan drug designation for AML in the E.U. from the EMA. |
| 2017-08-29 | US Patent 9,744,187 issued for P-ethoxy nucleic acids for liposomal formulation. |
| 2018-02-01 | Paul D. Aubert was appointed to the Board. |
| 2018-12 | The Company extended the term of the lab space lease agreement to April 30, 2022. |
| 2019-05 | The Company extended the term of the office space lease agreement to October 31, 2024. |
| 2020-08-13 | The Company announced the enrollment and dosing of the first patient in the amended Stage 2 of the Phase 2 clinical trial. |
| 2021-01-26 | US Patent 10,898,506 issued for P-ethoxy nucleic acids for liposomal formulation. |
| 2021-02-23 | US Patent 10,927,379 issued for combination therapy with liposomal antisense oligonucleotides. |
| 2021-06-22 | US Patent 11,041,153 issued for P-ethoxy nucleic acids for STAT3 inhibition. |
| 2022-01 | The Company exercised an option to extend the term of the lab space lease to April 30, 2025. |
| 2022-03-31 | Aline B. Sherwood was appointed to the Board. |
| 2022-05-27 | The Company filed a shelf registration statement on Form S-3 with the SEC. |
| 2022-06-14 | The Company's shelf registration statement on Form S-3 was declared effective by the SEC. |
| 2022-07-27 | EP Patent 3 512 525 issued for combination therapy with liposomal antisense oligonucleotides. |
| 2022-08-30 | JP Patent 7132911 issued for combination therapy with liposomal antisense oligonucleotides. |
| 2022-11-09 | The 2022 Registered Direct Offering and the 2022 Private Placement closed. |
| 2022-12-01 | JP Patent 7186721 issued for P-ethoxy nucleic acids for IGF-1R inhibition. |
| 2022-12-14 | The Company announced the successful completion of the first dose cohort of the dose escalation portion of the Phase 1/1b clinical trial of BP1002. |
| 2022-12-16 | CN Patent ZL 201880033244.6 issued for P-ethoxy nucleic acids for STAT3 inhibition. |
| 2022-12-19 | EA Patent 041953 issued for combination therapy with liposomal antisense oligonucleotides. |
| 2023-01-05 | AU Patent 2016340123 issued for P-ethoxy nucleic acids for liposomal formulation. |
| 2023-03-02 | JP Patent 7237009 issued for P-ethoxy nucleic acids for STAT3 inhibition. |
| 2023-03-09 | EA Patent 042663 issued for P-ethoxy nucleic acids for STAT3 inhibition. |
| 2023-04-06 | HK Patent 400 11951 issued for combination therapy with liposomal antisense oligonucleotides. |
| 2023-05-23 | JP Patent 7284709 issued for P-ethoxy nucleic acids for BCL2 inhibition. |
| 2023-06-20 | MX Patent 403603 issued for P-ethoxy nucleic acids for liposomal formulation. |
| 2023-07-17 | The Company announced successful completion of the first dose cohort of BP1001-A in the Phase 1/1b study. |
| 2023-08-01 | The Company announced interim data for the first two cohorts of the amended Stage 2 of the Phase 2 clinical trial. |
| 2023-08-07 | The 2023 Public Offering closed. |
| 2023-09-19 | EA Patent 044637 issued for P-ethoxy nucleic acids for BCL2 inhibition. |
| 2023-11-24 | IN Patent 472686 issued for P-ethoxy nucleic acids for liposomal formulation. |
| 2023-12-07 | MX Patent 408790 issued for P-ethoxy nucleic acids for STAT3 inhibition and MX Patent 408785 issued for P-ethoxy nucleic acids for BCL2 inhibition. |
| 2024-01-10 | The Company announced the successful completion of the first dose cohort in the Phase 1 clinical trial evaluating BP1002 for lymphoma and CLL. |
| 2024-02-22 | The Company effected a reverse stock split of its outstanding shares of common stock at a ratio of 1-for-20. |
| 2024-02-23 | The Company's common stock began trading on the split-adjusted basis on the Nasdaq Capital Market. |
Keywords
AML, Prexigebersen, DNAbilize, BP1002, BP1003, BP1001-A, Oncology, RNAi, Clinical Trial, Liposomal, Antisense, Grb2, Bcl-2, STAT3, Pancreatic Cancer, NSCLC, Lymphoma, CLL
Disclaimer:The information provided here is for general informational purposes only and does not constitute financial advice, recommendation, or endorsement of any kind. It may contain errors or omissions. You should not rely on this information to make financial decisions. Always seek the advice of a qualified financial professional before making any investment or financial decisions. Use of this information is at your own risk.