OnKure Therapeutics Reports First Quarter 2025 Financial Results and Business Highlights
- Strong cash position of $96.7M sufficient to fund operations through Q4 2026
- Promising preliminary safety data from PIKture-01 trial with minimal adverse events
- No dose limiting toxicities observed in combination trial with fulvestrant to date
- Successful completion of dose escalation up to 1500 mg BID in Part A of PIKture-01 trial
- Increased net loss to $15.9M in Q1 2025 from $9.5M in Q1 2024
- Higher R&D expenses at $13.0M, up from $8.6M year-over-year
- Increased G&A expenses to $4.0M from $1.3M in Q1 2024
Insights
OnKure reports continued development of PI3Kα inhibitors with promising safety profile; multiple clinical data readouts expected in H2 2025.
OnKure's lead compound OKI-219 targets the PI3KαH1047R mutation, an important oncogenic driver in certain cancers, particularly HR+/HER2- breast cancer. The preliminary safety data revealed in the PIKture-01 trial is particularly noteworthy - unlike many other PI3K inhibitors that typically cause significant adverse events, OKI-219 demonstrated a favorable tolerability profile with only grade 1 treatment-related adverse events and notably no hyperglycemia, stomatitis, or rash.
The company has completed dose escalation up to 1500 mg BID with dose-limiting toxicities observed in only one patient at the highest dose level. This suggests a potentially wide therapeutic window, which is uncommon for this class of drugs. The tolerability profile could represent a significant competitive advantage if maintained in larger patient cohorts.
OnKure's clinical strategy is scientifically sound, evaluating OKI-219 both as monotherapy and in combination with fulvestrant, a standard endocrine therapy for HR+/HER2- metastatic breast cancer. The company expects to report mature monotherapy data and initial combination data in H2 2025.
The expansion into a pan-mutant PI3Kα program addresses an unmet need by targeting multiple common oncogenic mutations (H1047X, E542K, and E545K) with a single agent. Their stated selectivity goal of 10-fold selectivity against mutant versus wild-type PI3Kα is ambitious but would help minimize off-target effects if achieved. This diversification of their pipeline leverages their expertise in PI3K biology while expanding their potential market opportunity beyond H1047R-mutated cancers.
The coming quarters will be critical for OnKure as they report clinical data that could validate their selective targeting approach in a pathway that has historically been challenging to drug effectively due to toxicity concerns.
OnKure reports increased R&D investment with $96.7M cash runway into Q4 2026; financial position supports multiple clinical milestones.
OnKure's Q1 2025 financial results reflect a company in active clinical development phase with strategic resource allocation. Their cash position of
R&D expenses increased
G&A expenses saw a more dramatic increase of
The quarterly net loss of
With a current quarterly cash burn of approximately
From a financial perspective, OnKure has positioned itself with sufficient capital to reach multiple value-creating milestones without immediate financing pressure, providing operational flexibility during a critical phase of clinical development.
-- Continued progress in the PIKture-01 trial; on track to report additional data in the second half of 2025, including mature single agent and initial combination data
-- Expansion of the Company’s PI3Ka franchise with the planned announcement of a pan-mutant selective development candidate in Q2 2025
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BOULDER, Colo., May 06, 2025 (GLOBE NEWSWIRE) -- OnKure Therapeutics, Inc. (Nasdaq: OKUR), a clinical-stage biopharmaceutical company focused on developing novel precision medicines in oncology, today reported financial results for the quarter ended March 31, 2025, and provided business highlights.
“OnKure looks to define a new standard of performance for precision targeted agents. We designed OKI-219 and our ongoing PIKture-01 trial to set a high bar for achieving considerable selectivity in targeting oncogenic PI3KαH1047R. Late last year, we announced preliminary data that showed OKI-219 was well tolerated across all dose levels, allowing us to complete the dose escalation portion of the study up to 1500 mg BID. We anticipate presenting a mature clinical update in the second half of 2025 that could demonstrate a clear and meaningful benefit in treating this very challenging patient population. This exciting next step in our program would further solidify OnKure’s place as a leader in developing PI3Ka inhibitors,” said Nick Saccomano, Ph.D., President and Chief Executive Officer of OnKure.
“Building on the advancement of our initial candidate, our research and development team is advancing towards a second development candidate in the second quarter of 2025; a true pan-mutant inhibitor with a selectivity profile that augurs well for rendering all major PI3Ka mutants actionable.”
Business Highlights and Upcoming Anticipated Milestones
- PIKture-01 trial Part A Monotherapy – The Company has completed dose escalating and closed enrollment in Part A with dose limiting toxicities observed in only one patient to date and at the highest dose level. OnKure expects to provide a mature clinical update in the second half of 2025.
OnKure previously announced encouraging preliminary safety, tolerability, and pharmacokinetic (“PK”) data from the part A single-agent arm of the PIKture-01 trial, with a data cut-off of October 28, 2024. These preliminary data showed OKI-219 was well tolerated across all dose levels, with no hyperglycemia, stomatitis, or rash observed. Additionally, only grade 1 treatment-related adverse events (“TRAEs”) were reported with no dose interruptions, delays, reductions, or discontinuations reported for any adverse events.
- PIKture-01 Part B Fulvestrant Combination – OnKure is actively enrolling patients in Part B of the PIKture-01 trial, evaluating OKI-219 in combination with fulvestrant in patients with PI3KαH1047R mutated HR+/HER2- metastatic breast cancer. The Company is nearing the end of the dose escalation portion of Part B, with no dose limiting toxicities observed to date. OnKure expects to report initial combination data with fulvestrant in the second half of 2025. It is anticipated that the dose escalation portion of Part B will be completed at the time of the presentation.
- Pan-mutant Program – OnKure believes that to be truly “pan-mutant”, a candidate should be highly selective against each of the most common PI3Ka mutations with a favorable safety and tolerability profile. OnKure is targeting approximately 10-fold selectivity against each of the most common mutations (PI3KαH1047X, PI3KαE542K, and PI3KαE545K) over wild type with its pan-mutant development candidate, which it expects to announce in the second quarter of 2025.
First Quarter 2025 Financial Results
Cash and cash equivalents were approximately
Research and development (R&D) expenses were
General and Administrative (G&A) expenses were
Net loss and net loss per share for the first quarter of 2025 were
About OnKure Therapeutics
OnKure Therapeutics, Inc. (Nasdaq: OKUR) is a clinical-stage biopharmaceutical company focused on the discovery and development of best-in-class precision medicines that target biologically validated drivers of cancers that are underserved by available therapies. Using a structure-based drug design platform, OnKure is building a pipeline of tumor-agnostic candidates that are designed to achieve optimal efficacy and tolerability. OnKure is currently developing OKI-219, a selective PI3KαH1047R inhibitor, as its lead program. OnKure aims to become a leader in targeting oncogenic PI3Kα and has multiple programs designed to enable best-in-class targeting of this key oncogene.
For more information about OnKure, visit us at www.onkure.com and follow us on LinkedIn.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this press release, including statements regarding our future financial condition, results of operations, business strategy and plans, and objectives of management for future operations, as well as statements regarding industry trends, are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding, OnKure’s current and potential future product candidates and programs, including OKI-219 and the pan-mutant program; OnKure’s ability to advance additional programs; expected milestones and timing of such milestones, including additional data for OKI-219 from the PIKture-01 trial, anticipated development candidate announcements and advancement of OnKure’s discovery stage programs; and statements regarding OnKure’s financial position, including its liquidity, cash runway and the sufficiency of its cash resources. In some cases, you can identify forward-looking statements by terminology such as “estimate,” “intend,” “may,” “plan,” “potentially” “will” or the negative of these terms or other similar expressions.
We based these forward-looking statements largely on our current expectations and projections about future events and trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, among other things, OnKure’s limited operating history; the significant net losses incurred since inception; the ability to raise additional capital to finance operations; the risk that actual uses of cash and cash equivalents differ from the assumptions underlying our expected cash runway; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, OnKure’s product candidates; the outcome of preclinical testing and early clinical trials for OnKure’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements, timing of regulatory reviews and approvals, and the potential that the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials; OnKure’s limited resources; the risk of adverse events, toxicities or other undesirable side effects; potential delays or difficulties in the enrollment or maintenance of patients in clinical trials; the decision to develop or seek strategic collaborations to develop OnKure’s current or future product candidates in combination with other therapies and the cost of combination therapies; OnKure’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the substantial competition OnKure faces in discovering, developing, or commercializing products; OnKure’s ability to protect its intellectual property and proprietary technologies; developments relating to OnKure’s competitors and its industry, including competing product candidates and therapies; reliance on third parties, contract manufacturers, and contract research organizations; legislative, regulatory, political and economic developments and general market conditions; and those risks described in the section entitled “Risk Factors” in documents that OnKure files from time to time with the Securities and Exchange Commission (“SEC”), including our Annual Report on Form 10-K filed with the SEC on March 10, 2025 and any subsequent filings with the SEC. These risks are not exhaustive. New risk factors emerge from time to time, and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in, or implied by, any forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. Except as required by law, we undertake no obligation to update publicly any forward-looking statements for any reason after the date of this press release.
Contact:
Dan Ferry
LifeSci Advisors
daniel@lifesciadvisors.com
| ONKURE THERAPEUTICS, INC. | ||||||||
| Condensed Consolidated Balance Sheets | ||||||||
| (In thousands, unaudited) | ||||||||
| March 31, | December 31, | |||||||
| 2025 | 2024 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 96,661 | $ | 110,761 | ||||
| Prepaid expenses and other current assets | 1,511 | 2,242 | ||||||
| Total current assets | 98,172 | 113,003 | ||||||
| Property and equipment, net | 925 | 1,025 | ||||||
| Operating lease right-of-use asset | 676 | 770 | ||||||
| Other assets | 109 | 109 | ||||||
| Total assets | $ | 99,882 | $ | 114,907 | ||||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,344 | $ | 2,968 | ||||
| Accrued expenses | 5,895 | 7,026 | ||||||
| Operating lease liabilities, current portion | 546 | 536 | ||||||
| Total current liabilities | 8,785 | 10,530 | ||||||
| Long-term operating lease liabilities | 409 | 549 | ||||||
| Other long-term liabilities | 40 | — | ||||||
| Total liabilities | 9,234 | 11,079 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Common stock, Class A, | 1 | 1 | ||||||
| Common stock, Class B, | — | — | ||||||
| Additional paid-in capital | 261,296 | 258,551 | ||||||
| Accumulated deficit | (170,649 | ) | (154,724 | ) | ||||
| Total stockholders’ equity | 90,648 | 103,828 | ||||||
| Total liabilities and stockholders’ equity | $ | 99,882 | $ | 114,907 | ||||
| ONKURE THERAPEUTICS, INC. | |||||||
| Condensed Consolidated Statements of Operations and Comprehensive Loss | |||||||
| (In thousands, except share and per share data, unaudited) | |||||||
| Three Months Ended | |||||||
| March 31, | |||||||
| 2025 | 2024 | ||||||
| Operating expenses: | |||||||
| Research and development | $ | 13,012 | $ | 8,566 | |||
| General and administrative | 3,988 | 1,265 | |||||
| Total operating expenses | 17,000 | 9,831 | |||||
| Loss from operations | (17,000 | ) | (9,831 | ) | |||
| Other income: | |||||||
| Interest income | 1,075 | 295 | |||||
| Net loss and comprehensive loss | $ | (15,925 | ) | $ | (9,536 | ) | |
| Net loss per share attributable to common stockholders: | |||||||
| Basic and diluted | $ | (1.19 | ) | $ | (30.37 | ) | |
| Weighted average shares outstanding: | |||||||
| Basic and diluted | 13,424,335 | 314,016 | |||||
FAQ
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