APRE Aprea Therapeutics Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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Eprenetapopt (APR-246) / venetoclax and azacitidine
TP53 mutant AML
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Phase 1
Phase 1
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
|
Eprenetapopt (APR-246) and venetoclax and rituximab
Chronic lymphoid leukemia (CLL) and Mantle cell lymphoma (MCL)
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Phase 1
Phase 1
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Phase 1 trial enrollment commenced 1Q 2021.
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Eprenetapop (APR-246)
Solid tumors
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Phase 1/2
Phase 1/2
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Phase 1/2 enrollment commenced August 2020.
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APR-548
TP53 mutant myelodysplastic syndromes (MDS)
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Phase 1
Phase 1
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Phase 1 enrollment to commence 2Q 2021.
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Eprenetapopt (APR-246) and azacitidine
TP53 mutated myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) - post transplant
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Phase 2
Phase 2
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Phase 2 initial data due 2Q 2021.
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APR-246 (eprenetapopt) and azacitidine
TP53 mutant Myelodysplastic syndromes (MDS)
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Phase 3
Phase 3
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Phase 3 top-line data did not meet primary endpoint - December 28, 2020.
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Latest News
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BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (NASDAQ:APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to eprenetapopt for treatment of AML.
"We are pleased to have been granted Orphan Drug designation by FDA for eprenetapopt in AML, building on the Fast Track designation in AML that was granted in November 2020," said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "We look forward to continued productive dialogue with FDA and bringing eprenetapopt to patients as soon as possible."
Orphan Drug designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, tax credits related to clinical trial expenses, and an exemption from FDA user fees.
About Aprea Therapeutics, Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Company's lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies and solid tumors. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for myelodysplastic syndromes (MDS), Orphan Drug and Fast Track designations from the FDA for acute myeloid leukemia (AML), and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
About p53, eprenetapopt and APR-548
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with eprenetapopt in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with eprenetapopt has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
A pivotal Phase 3 clinical trial of eprenetapopt and azacitidine for frontline treatment of TP53 mutant MDS has been completed and failed to meet the primary endpoint of complete remission. Additional clinical trials in hematologic malignancies and solid tumors are ongoing. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Orphan Drug and Fast Track designations from the FDA for AML, and Orphan Drug designation from the European Medicines Agency for MDS, AML and ovarian cancer.
APR-548 is a next-generation small molecule p53 reactivator. APR-548 has demonstrated high oral bioavailability, enhanced potency relative to eprenetapopt in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice. Enrollment in a Phase 1 clinical trial of APR-548 is anticipated to begin early in the second quarter of 2021.
About AML
AML is the most common form of adult leukemia, with the highest incidence in patients aged 60 years and older. AML is characterized by proliferation of abnormal immature white blood cells that impairs production of normal blood cells. AML can develop de novo or may arise secondary to progression of other hematologic disorders or from chemotherapy or radiation treatment for a different, prior malignancy; secondary AML carries a worse prognosis than de novo AML. Mutations in TP53, which are associated with poor overall prognosis, occur in approximately 20% of patients with newly diagnosed AML, more than 30% of patients with therapy-related AML and approximately 70-80% of patients with complex karyotype.
Forward-Looking Statement
Certain information contained in this press release includes "forward-looking statements", within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our study analyses, clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as "future," "predicts," "believes," "potential," "continue," "anticipates," "estimates," "expects," "plans," "intends," "targeting," "confidence," "may," "could," "might," "likely," "will," "should" or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. Coiante
Sr. Vice President and Chief Financial Officer
617-463-9385
Gregory A. Korbel
Chief Business Officer
617-463-9385
-
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BOSTON, March 16, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (NASDAQ:APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today reported financial results for the three months and year ended December 31, 2020 and provided a business update.
"Though disappointed the topline complete remission rate from the Phase 3 clinical trial narrowly missed its primary endpoint, we continue to analyze the totality of the data from the study to understand those differences from our prior Phase 2 experience in frontline MDS patients and expect to present these findings in the second quarter of 2021," said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "Our dedicated team remains committed to the clinical development of eprenetapopt and our next generation, oral p53 reactivator, APR-548, in hematological and solid tumor malignancies. In 2021, we look forward to sharing data from our current clinical studies as well as our plans to expand the clinical pipeline to include new indications."
Business Operations Update:
The Company is conducting, supporting, and planning multiple clinical trials of eprenetapopt (APR-246) and APR-548:
- Pivotal Phase 3 MDS Trial—In December 2020, the Company announced its pivotal Phase 3 randomized, controlled trial evaluating eprenetapopt with azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes (MDS) patients failed to meet its predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a 53% higher number of patients achieving a CR in the experimental arm receiving eprenetapopt with azacitidine versus the control arm receiving azacitidine alone but did not reach statistical significance. The Company is completing analysis from this Phase 3 clinical trial and expects to present additional information in the second quarter of 2021.
- Phase 2 MDS/AML Post-Transplant Trial – The Company has completed enrollment of 33 patients in a single-arm, open-label Phase 2 clinical trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and AML patients who have received an allogeneic stem cell transplant. The Company anticipates initial results from the primary endpoint of relapse-free survival at 12 months in the second quarter of 2021.
- Phase 1/2 AML Trial – The Company is currently enrolling a Phase 1/2 clinical trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in 12 patients receiving either regimen. Based on these results, the Company has expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. In the 19 frontline AML patients who are evaluable for efficacy with the triplet regimen, the Company has observed a 63% CR + CRi composite response rate and a 31% CR rate. The Company anticipates completion of enrollment in the triplet regimen expansion cohort during the second quarter of 2021 with availability of preliminary response rate data from the cohort also in the second quarter of 2021.
- Phase 1 NHL Trial – The Company is currently enrolling a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab and eprenetapopt with ibrutinib in order to further assess eprenetapopt in hematological malignancies. The first patient was enrolled in the first quarter of 2021. The Company is also planning to evaluate the combination of eprenetapopt with venetoclax in relapsed/refractory mantle cell lymphoma.
- Phase 1/2 Solid Tumor Trial – The Company is currently enrolling a Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. The dose-escalation phase of the trial enrolled 6 patients with advanced solid tumors and no dose-limiting toxicities were observed. Based on these results, the Company is enrolling expansion cohorts for patients with advanced gastric, bladder and non-small cell lung cancers and has currently enrolled 8 patients across these expansion arms.
- APR-548 -- The Company's second product candidate, APR-548, is a next-generation p53 reactivator that is being developed in an oral dosage form. The Company has planned a Phase 1 dose-escalation clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-548 with azacitidine in frontline and relapsed/refractory MDS patients. The Company anticipates the first patient to be enrolled early in the second quarter of 2021.
Fourth Quarter Financial Results
- Cash and cash equivalents: As of December 31, 2020, the Company had $89.0 million of cash and cash equivalents compared to $130.1 million of cash and cash equivalents as of December 31, 2019. The Company expects cash burn for the full year 2021 to be between $30.0 million $35.0 million. The Company believes its cash and cash equivalents as of December 31, 2020 will be sufficient to meet its current projected operating requirements into 2023.
- Research and Development (R&D) expenses: R&D expenses were $9.3 million for the quarter ended December 31, 2020, compared to $8.0 million for the comparable period in 2019. The increase in R&D expenses was primarily related to the continued development of the Company's lead product candidate, eprenetapopt, in the following ongoing clinical trials; its pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for frontline treatment of TP53 mutant MDS, its Phase 1/2 clinical trial for the treatment of TP53 mutant AML with venetoclax and azacitidine, its Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy, its Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib and its Phase 2 post-transplant MDS/AML clinical trial.
- General and Administrative (G&A) expenses: G&A expenses were $4.9 million for the quarter ended December 31, 2020, compared to $3.9 million for the comparable period in 2019. The increase in G&A expenses was primarily due to increases in non-cash stock-based compensation, insurance expense and commercial development expense.
- Net loss: Net loss was $15.4 million, or $0.73 per share for the quarter ended December 31, 2020, compared to a net loss of $13.1 million, or $0.64 per share for the quarter ended December 31, 2019. The Company had 21,186,827 shares of common stock outstanding as of December 31, 2020.
About Aprea Therapeutics, Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Company's lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Fast Track designation from the FDA for AML, and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
About p53, eprenetapopt and APR-548
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with eprenetapopt in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with eprenetapopt has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
A pivotal Phase 3 clinical trial of eprenetapopt and azacitidine for frontline treatment of TP53 mutant MDS is ongoing but at the primary data cut, it failed to meet its predefined primary endpoint of complete remission rate. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the European Medicines Agency for MDS, AML and ovarian cancer.
APR-548 is a next-generation small molecule p53 reactivator. APR-548 has demonstrated high oral bioavailability, enhanced potency relative to eprenetapopt in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice. A Phase 1 clinical trial of APR-548 in TP53 MDS is planned.
Forward-Looking Statement
Certain information contained in this press release includes "forward-looking statements", within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions, and projected cash position. We may, in some cases use terms such as "predicts," "believes," "potential," "continue," "anticipates," "estimates," "expects," "plans," "intends," "targeting," "confidence," "may," "could," "might," "likely," "will," "should" or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward-looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. Coiante
Sr. Vice President and Chief Financial Officer
617-463-9385Gregory A. Korbel
Chief Business Officer
617-463-9385Aprea Therapeutics, Inc.
Condensed Consolidated Balance SheetsDecember 31, 2020 December 31, 2019 Assets Current assets: Cash and cash equivalents $89,017,686 $130,088,869 Prepaid expenses and other current assets 3,399,019 2,955,878 Total current assets 92,416,705 133,044,747 Property and equipment, net 38,515 41,639 Right of use lease and other noncurrent assets 349,999 521,499 Total assets $92,805,219 $133,607,885 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $4,503,619 $2,176,852 Accrued expenses 10,571,237 6,642,553 Lease liability—current 256,309 242,329 Total current liabilities 15,331,165 9,061,734 Lease liability—noncurrent 78,847 302,621 Total liabilities 15,410,012 9,364,355 Commitments and contingencies Stockholders' equity: Common stock, par value $0.001; 21,186,827 and 21,022,752, shares issued and outstanding at December 31, 2020 and 2019, respectively. 21,187 21,023 Additional paid-in capital 231,418,356 226,284,548 Accumulated other comprehensive loss (10,037,261 ) (11,533,778 ) Accumulated deficit (144,007,075 ) (90,528,263 ) Total stockholders' equity 77,395,207 124,243,530 Total liabilities and stockholders' equity $92,805,219 $133,607,885 Aprea Therapeutics, Inc.
Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)Three Months Ended December 31, Year Ended December 31, 2020 2019 2020 2019 Operating expenses: Research and development $ 9,328,079 $ 8,041,993 $ 37,879,325 $ 20,950,672 General and administrative 4,895,323 3,937,765 14,931,887 8,593,626 Total operating expenses 14,223,402 11,979,758 52,811,212 29,544,298 Other income (expense): Interest income 4,744 169,888 222,652 156,351 Foreign currency (loss) gain (1,173,888 ) (1,262,868 ) (890,252 ) 1,328,140 Total other income (expense) (1,169,144 ) (1,092,980 ) (667,600 ) 1,484,491 Net loss $ (15,392,546 ) $ (13,072,738 ) $ (53,478,812 ) $ (28,059,807 ) Other comprehensive income (loss): Foreign currency translation 2,333,369 2,154,388 1,496,517 (2,772,453 ) Total comprehensive loss (13,059,177 ) (10,918,350 ) (51,982,295 ) (30,832,260 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.73 ) $ (0.64 ) $ (2.53 ) $ (4.67 ) Weighted-average common shares outstanding, basic and diluted 21,186,827 20,318,040 21,133,651 6,002,486 -
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BOSTON, Jan. 08, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (NASDAQ:APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that Christian S. Schade, Chairman and Chief Executive Officer, will present a corporate update at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 2:50 p.m. ET.
A live webcast of the presentation can be accessed under the "Events Calendar" in the Investors section of the Aprea website at Link.
About Aprea Therapeutics, Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Company's lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies and solid tumors. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for myelodysplastic syndromes (MDS), Fast Track designation from the FDA for acute myeloid leukemia (AML), and Orphan Drug designation from the European Commission for MDS and AML. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
Forward-Looking Statement
Certain information contained in this press release includes "forward-looking statements", within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our study analyses, clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as "future," "predicts," "believes," "potential," "continue," "anticipates," "estimates," "expects," "plans," "intends," "targeting," "confidence," "may," "could," "might," "likely," "will," "should" or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. Coiante
Sr. Vice President and Chief Financial Officer
617-463-9385
Gregory A. Korbel
Vice President of Business Development
617-463-9385
-
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- The trial failed to meet its primary endpoint of complete remission (CR) rate
- CR rate was 53% higher in eprenetapopt with AZA arm compared to AZA alone, but did not reach statistical significance
BOSTON, Dec. 28, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (NASDAQ:APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced results of the primary data cut from its Phase 3 clinical trial evaluating the safety and efficacy of eprenetapopt with azacitidine (AZA) versus AZA alone in TP53 mutant myelodysplastic syndromes (MDS). The trial did not meet the predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a higher CR rate in the experimental arm receiving eprenetapopt with AZA versus the control arm receiving AZA alone, but did not reach statistical significance. In the intention-to-treat population of 154 patients, the CR rate in the eprenetapopt with AZA arm was 33.3% (95% CI: 23.1% - 44.9%) compared to 22.4% (95% CI: 13.6% - 33.4%) in the AZA alone arm (P = 0.13).
While analysis of certain secondary endpoints (ORR and duration of responses) appears to favor the experimental arm at this data cut, they are not significantly different. The median duration of overall survival at the primary data cut was similar between the arms. Additional patients in the study who have not achieved a CR remain on study treatment and the data will be analyzed at future pre-specified timepoints as set forth in the statistical analysis plan. The combination of eprenetapopt with AZA appeared well-tolerated, with an adverse event profile that was similar to the Company's prior Phase 2 clinical trials. Subsequent analyses of the trial data, including secondary endpoints, will be conducted as the duration of patient follow-up increases. The Company expects to present the data at a future scientific conference.
"Though we are disappointed the topline results did not reach statistical significance, we continue to believe that eprenetapopt can offer clinical benefit to patients with TP53 mutant malignancies," said Dr. Eyal Attar, Chief Medical Officer of Aprea. "We will continue to analyze data as it matures and follow patients who are still receiving study treatment. Our other clinical trials continue to progress and we remain committed to pursuing our clinical development programs."
About the Phase 3 Trial in TP53 Mutant MDS
The Phase 3 trial enrolled 154 TP53 mutant MDS patients, randomized 1:1 to either the eprenetapopt with AZA arm or the AZA alone arm. Response criteria are those defined by International Working Group 2006 (IWG 2006) and include measures of peripheral blood counts and bone marrow blasts.
About Aprea Therapeutics, Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Company's lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies and solid tumors. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for myelodysplastic syndromes (MDS), Fast Track designation from the FDA for acute myeloid leukemia (AML), and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
About p53, eprenetapopt and APR-548
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with eprenetapopt in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with eprenetapopt has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
A pivotal Phase 3 clinical trial of eprenetapopt and azacitidine for frontline treatment of TP53 mutant MDS has been completed and additional clinical trials in hematologic malignancies and solid tumors are ongoing. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Fast Track designation from the FDA for AML, and Orphan Drug designation from the European Medicines Agency for MDS, AML and ovarian cancer.
APR-548 is a next-generation small molecule p53 reactivator. APR-548 has demonstrated high oral bioavailability, enhanced potency relative to eprenetapopt in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice. Enrollment in a Phase 1 clinical trial of APR-548 is anticipated to begin in the first quarter of 2021.
Forward-Looking Statement
Certain information contained in this press release includes "forward-looking statements", within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our study analyses, clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as "future," "predicts," "believes," "potential," "continue," "anticipates," "estimates," "expects," "plans," "intends," "targeting," "confidence," "may," "could," "might," "likely," "will," "should" or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. Coiante
Sr. Vice President and Chief Financial Officer
617-463-9385Gregory A. Korbel
Vice President of Business Development
617-463-9385 - The trial failed to meet its primary endpoint of complete remission (CR) rate
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BOSTON, Nov. 30, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (NASDAQ:APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for eprenetapopt in the treatment of patients with TP53 mutant acute myeloid leukemia (AML). The Company previously received Breakthrough Therapy, Orphan Drug and Fast Track designations for eprenetapopt in the treatment of patients with TP53 mutant myelodysplastic syndromes (MDS).
The FDA's Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need. A drug candidate that receives Fast Track designation may be eligible for more frequent interaction with the FDA to discuss the drug candidate's development plan as well as eligibility for accelerated approval and priority review.
"We are pleased to have received Fast Track designation for eprenetapopt in the treatment of TP53 mutant AML, a cancer for which outcomes are poor and there are no current therapeutic options specifically for these patients," said Eyal C. Attar, M.D., Chief Medical Officer of Aprea. "Emerging data from our AML trials evaluating eprenetapopt with azacitidine, and with eprenetapopt, azacitidine and venetoclax, are promising and we continue to enroll patients to identify the best treatment regimen. As these data mature in 2021, we look forward to continued interaction with FDA as we map out opportunities for an accelerated pathway to potential approval."
About Aprea Therapeutics, Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Company's lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Fast Track designation from the FDA for AML,and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
About p53, eprenetapopt and APR-548
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with eprenetapopt in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with eprenetapopt has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
A pivotal Phase 3 clinical trial of eprenetapopt and azacitidine for frontline treatment of TP53 mutant MDS is ongoing. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Fast Track designation from the FDA for AML, and Orphan Drug designation from the European Medicines Agency for MDS, AML and ovarian cancer.
APR-548 is a next-generation small molecule p53 reactivator. APR-548 has demonstrated high oral bioavailability, enhanced potency relative to eprenetapopt in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice. Enrollment in a Phase 1 clinical trial of APR-548 is anticipated to begin in the first quarter of 2021
Forward-Looking Statement
Certain information contained in this press release includes "forward-looking statements", within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as "predicts," "believes," "potential," "continue," "anticipates," "estimates," "expects," "plans," "intends," "targeting," "confidence," "may," "could," "might," "likely," "will," "should" or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. Coiante
Sr. Vice President and Chief Financial Officer
617-463-9385Gregory A. Korbel
Vice President of Business Development
617-463-9385