Aptose Reports Results for the Fourth Quarter and Full Year 2023
Aptose Biosciences Inc. announces financial results for the year ended December 31, 2023, highlighting positive clinical data for tuspetinib in AML and completion of Luxeptinib G3 formulation evaluation. The company also completed a public offering and private placement to strengthen its financial position.
Positive
Positive clinical data for tuspetinib in AML, showing broad activity and favorable safety profile.
Completion of Luxeptinib G3 formulation evaluation, demonstrating desired plasma exposure levels for future studies.
Successful completion of a public offering and private placement to raise funds for research and development.
Stable financial position with sufficient capital to fund operations through August 2024.
Negative
Net loss increased by $9.4 million to $51.2 million for the year ended December 31, 2023, compared to the previous year.
Cash and cash equivalents decreased to $9.3 million as of December 31, 2023, from $47.0 million at the end of 2022.
Research and development expenses increased by $8.7 million in 2023, primarily driven by higher program costs for tuspetinib.
The advancement of tuspetinib into a triplet therapy pilot study represents a significant stride in the treatment landscape for acute myeloid leukemia (AML), particularly for newly diagnosed patients ineligible for induction chemotherapy. The study's focus on a combination of tuspetinib, venetoclax and a hypomethylating agent (TUS/VEN/HMA) could potentially address a substantial unmet medical need, given the limited options for these patients.
From a research perspective, the reported broad activity of tuspetinib across mutational subtypes and its favorable safety profile are noteworthy. These factors, combined with the drug's unique kinase targeting pattern, suggest a differentiated approach that could offer therapeutic benefits over existing treatments. Moreover, the reported complete remission (CR/CRh) rate of 36% in certain patient subsets is encouraging and warrants further investigation in larger, controlled studies.
The financial implications for Aptose Biosciences and its stakeholders are multifaceted. On one hand, the progression into clinical studies is a resource-intensive endeavor that will likely increase R&D expenses. On the other hand, the potential for a successful therapy could lead to significant revenue generation. The recent public and private financings reflect an effort to support these initiatives, although the premium pricing in the private placement with Hanmi Pharmaceutical suggests confidence in the company's prospects.
Analyzing the financial results of Aptose Biosciences, the decrease in cash and cash equivalents from $46.9 million in 2022 to $9.3 million in 2023 is quite stark. This highlights the burn rate of the company as it invests heavily in its R&D programs. However, the subsequent financings in January 2024, which raised approximately $13.7 million, provide a lifeline that extends the company's operational runway through August 2024.
Investors should note the dilutive effect of the common share offerings, which could potentially impact share value. The increase in R&D expenses by $8.7 million underscores the company's commitment to advancing its drug candidates, but also reflects the high costs associated with clinical development. The decrease in general and administrative expenses, particularly in stock-based compensation, might be seen as a cost-containment measure in response to the increased R&D spend.
It is important to monitor the company's cash position and burn rate closely, as these will be key indicators of its ability to sustain operations and reach critical milestones without requiring additional dilutive financing. The company's strategic partnership with Hanmi Pharmaceutical also introduces an element of regional market interest and potential future collaboration benefits.
Within the pharmaceutical industry, especially in the oncology segment, the development of new therapies like tuspetinib can significantly disrupt market dynamics. The current AML treatment market is competitive, with a need for therapies that can offer both efficacy and safety for a broad patient population. Tuspetinib's advancement into a triplet therapy pilot study signals potential market entry that could capture a share of the AML treatment space, particularly if the therapy proves effective in a broader range of mutational subtypes.
The company's strategic moves, such as the partnership with Hanmi Pharmaceutical and the successful public and private offerings, indicate a proactive approach to financing and collaboration. These actions not only provide necessary capital but also potentially pave the way for market expansion and penetration, especially in Asia with Hanmi's involvement.
Given the early stage of the clinical studies, the long-term market impact remains speculative. However, the progression of tuspetinib into further studies is a positive signal for investors monitoring the company's pipeline. The upcoming milestones, such as the initiation of the TUS/VEN/HMA pilot study and early data presentations at major conferences, will be critical in shaping market perceptions and investor confidence.
03/26/2024 - 04:01 PM
Tuspetinib to Advance to TUS+VEN+HMA Triplet Therapy Pilot Study in 1L AML Tuspetinib Continues Broad Activity Across Mutations with Excellent Safety Profile Luxeptinib G3 Formulation Achieves Desired Levels and Positions for Future Trials Conference Call and Webcast at 5:00 pm ET Today SAN DIEGO and TORONTO, March 26, 2024 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, today announced financial results for the three months and year ended December 31, 2023, and provided a corporate update.
“The data we have generated from tuspetinib thus far – as a single agent and in combination therapy with venetoclax in relapsed and refractory AML – have demonstrated a distinctly favorable safety profile and broad activity for tuspetinib across mutational subtypes. This profile also extends to FLT3 wildtype AML, which represents the majority of AML patients, and in which few agents have shown such broad activity,” said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. “These data have propelled us to initiate a clinical study of tuspetinib in a triplet combination with venetoclax and azacitidine in frontline therapy for newly diagnosed AML, including both FLT3 wild type and FLT3 mutated subtypes.”
Key Corporate Highlights
Aptose Completes Public Offering – On January 30, 2024, Aptose closed a public offering of 5,649,122 common shares of the Company and warrants at a combined offering price of US $1.71 per share. This included 736,842 Common Shares and warrants pursuant to a full exercise by the underwriter of its over-allotment option. Total gross proceeds from the public offering were approximately $9.7 million before deducting underwriting costs, placement agent commissions and other offering-related expenses.Private Placement – On January 31, 2024, Aptose closed a US $4 million private placement of common shares with strategic partner Hanmi Pharmaceutical. Under the terms of the strategic investment, Hanmi purchased each common share at a price of US $1.90 , representing an 11% premium over the price of the common shares issued as part of the public offering. The Company also issued Hanmi warrants to purchase common shares at an exercise price of US $1.71 per warrant share. Total gross proceeds from the private placement were approximately $4 million , excluding underwriting discounts, placement agent commissions and other offering-related expenses.Tuspetinib Advancing to Triplet Therapy Pilot Study – Tuspetinib (TUS), a once daily oral agent with a unique kinase targeting pattern, is being developed for the treatment of patients with acute myeloid leukemia (AML). More than 170 patients to date received TUS alone or in combination with the BCL-2 inhibitor venetoclax (VEN) during the Phase 1/2 clinical program in the very ill relapsed or refractory (R/R) AML patient population. At the single agent 80 mg recommended Phase 2 dose, TUS achieved a favorable safety profile and a CR/CRh rate of 36% among patients who were naive to VEN. The safety profile of TUS remained favorable when TUS was combined with VEN in R/R AML patients, and responses were achieved in both patients naive to VEN and those who failed prior therapy with VEN. TUS avoids many typical toxicities observed with other agents and achieves broad activity across AML patients with a diversity of adverse genetics. Tuspetinib is now being advanced to a triplet combination therapy of tuspetinib, venetoclax and a hypomethylating agent (TUS/VEN/HMA) for the frontline treatment of newly diagnosed AML patients ineligible for induction chemotherapy.Luxeptinib G3 Evaluation Completed – During 2023 and early 2024, clinical evaluation of the new generation 3 (G3) formulation of luxeptinib (LUX) was completed. The G3 formulation was tested in a single dose bioavailability study in 20 patients, including both B-cell cancer and AML patients, and across 5 dose levels (10mg to 200mg). The G3 formulation then was evaluated in R/R AML patients with continuous dosing using two different dose levels (50mg BID and 200mg BID) in a total of 11 patients. Data show the G3 formulation dosed at 200mg twice daily can achieve 2-3uM steady state plasma levels, with approximately 10-fold better absorption, and interestingly even better tolerability, than the original G1 formulation. Thus, the G3 formulation achieved the desired plasma exposure benchmark and can serve as the formulation of choice for future studies with LUX. Aptose is exploring alternative development paths and collaborations to advance LUX as a single agent or in combination with VEN to treat defined R/R patient populations of high unmet need.Multiple Planned Value-creating Milestones Ahead
TUS/VEN doublet synopsis in R/R AML: EHA 2024 TUS/VEN/HMA planned initiation of pilot triplet study in 1L AML: Summer 2024 Triplet pilot dose escalation planned with early CR/MRD/safety data in 1L AML: ASH 2024 Triplet pilot completed with CR/MRD data and dose selection: EHA 2025 Triplet initiation of Ph2/Ph3 pivotal program: 2H 2025 FINANCIAL RESULTS OF OPERATIONS
Balance Sheet Data December 31, December 31, 2023 2022 Cash, cash equivalents and short-term investments $ 9,252 $ 46,959 Working capital (3,375 ) 37,235 Total assets 12,989 51,027 Non-current liabilities 621 1,002 Deficit (515,537 ) (464,330 ) Total shareholders’ equity (2,901 ) 37,741
Cash and cash equivalents, January 31, 2024 (unaudited) was $18.6 million , after gross proceeds from January 2024 financings (unaudited) of $13.7 million . Total cash and cash equivalents and investments as of December 31, 2023, were $9.3 million , a decrease of $37.7 million as compared to $47.0 million at December 31, 2022. Based on current operations, the Company expects that cash on hand and available capital provide the Company with sufficient resources to fund planned Company operations including research and development through August of 2024. Working capital is a non-GAAP measure and represents cash, cash equivalents, investments, prepaid expenses and other current assets less current liabilities. Common shares issued and outstanding as at March 26, 2024 were 15,717,701. Statements of Operations Data Year ended December 31, (in thousands except per Common Share data) 2023 2022 Revenues $ - $ - R&D, related party 3,492 3,556 Research and development expenses 33,273 24,532 General and administrative expenses 15,591 14,514 Net finance income 1,149 779 Net loss $ (51,207 ) $ (41,823 ) Basic and diluted loss per Common Share $ (7.58 ) $ (6.80 ) Weighted average number of common shares outstanding used in the calculation of basic loss per share 6,755 6,151
Net loss for the year ended December 31, 2023 increased by $9.4 million to $51.2 million , as compared to $41.8 million for the comparable period in 2022.
Research and Development Expenses
The research and development expenses for years ended December 31, 2023, and 2022 are as follows:
Twelve months ended December 31, (in thousands) 2023 2022 Program costs – Tuspetinib $ 24,925 $ 10,083 Program costs – Luxeptinib 3,510 8,426 Program costs – APTO-253 40 141 Personnel related expenses 6,878 7,181 Stock-based compensation 1,373 2,218 Depreciation of equipment 39 39 Total $ 36,765 $ 28,088
R&D expenses increased by $8.7 million to $36.8 million for the year ended December 31, 2023, as compared with $28.1 million for the comparative period in 2022. Changes to the components of our R&D expenses presented in the table above are primarily related to the following activities:
Program costs for tuspetinib increased by $14.8 million . The higher program costs for tuspetinib in 2023 represent the enrollment of patients in our APTIVATE clinical trial, our healthy volunteer trial, manufacturing activities to support clinical development, purchase of clinical trial materials, and related expenses. Program costs for luxeptinib decreased by approximately $4.9 million , primarily due to lower clinical trial costs and lower manufacturing costs as a result of the current formulation requiring less API than the prior formulation. Program costs for APTO-253 decreased by approximately $101 thousand due to the Company's decision on December 20, 2021, to discontinue further development of APTO-253. Personnel-related expenses decreased by $0.3 million due to lower headcount in 2023. Stock-based compensation decreased by approximately $845 thousand in the year ended December 31, 2023, compared with the year ended December 31, 2022, primarily due to stock options granted with lower grant date fair values in the current period. General and Administrative Expenses
General and administrative expenses consist primarily of salaries, benefits, and travel, including stock-based compensation for our executive, finance, business development, human resources, and support functions. Other general and administrative expenses and professional fees include auditing, and legal services, investor relations and other consultants, insurance. and facility related expenses.
We expect that our general and administrative expenses will increase for the foreseeable future as we incur additional costs associated with being a publicly traded company and to support our pipeline of activities. We also expect our intellectual property related legal expenses to increase as our intellectual property portfolio expands.
The general and administrative expenses for the years ended December 31, 2023 and 2022 are as follows:
Year ended December 31, (in thousands) 2023 2022 General and administrative, excluding items below: $ 13,262 $ 11,444 Stock-based compensation 2,280 2,989 Depreciation of equipment 49 81 Total $ 15,591 $ 14,514
General and administrative expenses increased by approximately $1.1 million $15.6 million for the year ended December 31, 2023, as compared with $14.5 million for the comparative period in 2022. The increase was primarily as a result of higher salaries expenses, higher professional fees, and higher travel expenses, partly offset by a decrease in stock-based compensation costs.
Stock-based compensation decreased by $709 thousand mostly as a result of options having a lower grant date fair value as compared with the options granted in the comparative period.
*Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a unique PIN, which are required to access the conference call. They also will have the option to take advantage of a Call Me button and the system will automatically dial out to connect to the Q&A session.
The audio webcast also can be accessed through a link on the Investor Relations section of Aptose’s website here . A replay of the webcast will be available on the company’s website for 30 days.
The press release, the financial statements and the management’s discussion and analysis for the year ended December 31, 2023 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml .
About Aptose
Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company's small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. The Company has two clinical-stage oral kinase inhibitors under development for hematologic malignancies: tuspetinib (HM43239), an oral, myeloid kinase inhibitor being studied as monotherapy and in combination therapy in the APTIVATE international Phase 1/2 expansion trial in patients with relapsed or refractory acute myeloid leukemia (AML); and luxeptinib (CG-806), an oral, dual lymphoid and myeloid kinase inhibitor in Phase 1 a/b stage development for the treatment of patients with relapsed or refractory hematologic malignancies. For more information, please visit www.aptose.com
Forward Looking Statements
This press release contains forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements regarding the Company’s clinical development plans, the clinical potential, anti-cancer activity, therapeutic potential and applications and safety profile of tuspetinib and luxeptinib, clinical trials, alternative development paths and collaborations to advance luxeptinib, upcoming milestones, expected increase of expenses and statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “hope” “should”, “would”, “may”, “potential” and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us, are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to obtain the capital required for research and operations; the inherent risks in early stage drug development including demonstrating efficacy; development time/cost and the regulatory approval process; the progress of our clinical trials; our ability to find and enter into agreements with potential partners; our ability to attract and retain key personnel; changing market and economic conditions; inability of new manufacturers to produce acceptable batches of GMP in sufficient quantities; unexpected manufacturing defects and other risks detailed from time-to-time in our ongoing current reports, quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission.
Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled "Risk Factors" in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein.
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The press release highlighted positive clinical data for tuspetinib in AML, showcasing broad activity and a favorable safety profile.
Aptose completed a public offering and a private placement to raise funds for research and development, enhancing its financial position.
The net loss for the year ended December 31, 2023, increased by $9.4 million to $51.2 million compared to the previous year.
Research and development expenses increased by $8.7 million in 2023, primarily due to higher program costs for tuspetinib.
Cash and cash equivalents were $9.3 million as of December 31, 2023, a decrease from $47.0 million at the end of 2022.
