Fam-trastuzumab deruxtecan-NXKI

Announced positive proof of concept data from Phase 1 clinical trial of ELVN-001 in CML, achieving an initial cumulative MMR rate of 44% (7/16) by 12 weeks in response-evaluable patients Strong balance sheet with $321 million in cash, cash equivalents and marketable securities, which is expected to provide cash runway into late 2026 BOULDER, Colo., May 14, 2024 (GLOBE NEWSWIRE) -- Enliven Therapeutics, Inc. (Enliven or the Company) (Nasdaq: ELVN), a clinical-stage precision oncology company focused on the discovery and development of next-generation small molecule kinase inhibitors, today reported financial results for the first quarter ended March 31, 2024, and provided a business update, including highlights of pipeline progress. “The first quarter of 2024 was a pivotal quarter for Enliven. We released positive proof of concept data from our Phase 1 clinical trial of ELVN-001, which was a significant milestone for the Company,” said Sam Kintz, MBA, Enliven’s Co-founder and Chief Executive Officer. “We are thrilled by the initial ELVN-001 data, particularly the tolerability profile and evidence of activity in heavily pre-treated patients, including in patients with asciminib-resistant chronic myeloid leukemia. We also continued to advance our trials of ELVN-002, including the activation of the first site to evaluate ELVN-002 in combination with trastuzumab in patients with HER2+ cancers, which we believe is an area of significant unmet need for patients. Additionally, we extended our cash runway into late 2026 thanks to the support of new and existing investors.” Corporate Updates Entered into a securities purchase agreement for a private investment in public equity (PIPE) financing resulting in gross proceeds of $90 million. The financing, from both new and existing investors, is expected to extend the Company’s cash runway into late 2026 and through multiple key milestones for ELVN-001 and ELVN-002.Appointed to the Board of Directors, Lori Kunkel, MD, an accomplished industry executive and board member with a track record of success in corporate strategy, clinical development and commercialization. Pipeline Updates ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR-ABL gene fusion, the oncogenic driver for patients with chronic myeloid leukemia (CML). Announced positive proof of concept data from the Phase 1 clinical trial evaluating ELVN-001 in patients with CML who are relapsed, refractory or intolerant to available tyrosine kinase inhibitors (TKIs). As of the cutoff date of March 18, 2024, 44% (7/16) of response-evaluable patients achieved a cumulative major molecular response (MMR) rate by week 12, and patients with prior exposure to asciminib and/or who were TKI-resistant also demonstrated responses, with a 44% and 40% cumulative MMR rate, respectively. Among response-evaluable patients, all had improved or stable BCR::ABL1 transcript levels by 12 weeks. ELVN-001 was well tolerated with no Grade 3 or higher non-hematologic treatment-related adverse events and no dose reductions reported.These data compare favorably to precedent Phase 1 cumulative MMR rates for approved BCR::ABL1 TKIs, particularly given the more heavily pre-treated patient population. Further details can be found here.Phase 1b data is expected in 2025 and is expected to include between approximately 60-100 patients across various lines of therapy with significant follow-up. ELVN-002 is a potent, highly selective, central nervous system penetrant and irreversible HER2 inhibitor with activity against wild type HER2 and various HER2 mutations. Following U.S. Food and Drug Administration approval of its Investigational New Drug application, the Company activated the first site to evaluate ELVN-002 in combination with trastuzumab +/- chemotherapeutic agents in HER2+ metastatic breast cancer (MBC) and colorectal cancer (CRC). First patient dosing for the combination trial is expected in Q2 2024.The combination trial in patients with HER2+ cancers is supported by the initial data from the ongoing monotherapy trial, which includes: Investigator reported responses (including unconfirmed) in both HER2+ and HER2 mutant tumors, including in patients who have progressed on Enhertu and patients with brain metastases, at doses that have been well tolerated.Importantly, at the clinically predicted optimal monotherapy dose, ELVN-002 had >10x target coverage based on pharmacokinetics in cancer patients and preclinical HER2+ efficacy compared to tucatinib. Phase 1 monotherapy data, Phase 1a/b Herceptin combination data in HER2+ CRC, and initial Phase 1a combination data in HER2+ MBC are expected in 2025. First Quarter 2024 Financial Results Cash Position: As of March 31, 2024, the Company had cash, cash equivalents and marketable securities totaling $320.5 million, which is expected to provide cash runway into late 2026.Research and development (R&D) expenses: R&D expenses were $20.0 million for the first quarter of 2024, compared to $11.9 million for the first quarter of 2023.General and administrative (G&A) expenses: G&A expenses for the first quarter of 2024 were $6.0 million, compared to $4.5 million for the first quarter of 2023.Net Loss: Enliven reported a net loss of $22.7 million for the first quarter of 2024, compared to a net loss of $14.7 million for the first quarter of 2023. About Enliven TherapeuticsEnliven Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule inhibitors to help people with cancer not only live longer, but live better. Enliven aims to address existing and emerging unmet needs with a precision oncology approach that improves survival and enhances overall well-being. Enliven’s discovery process combines deep insights in clinically validated biological targets and differentiated chemistry to design potentially first-in-class or best-in-class therapies. Enliven is based in Boulder, Colorado. Forward-Looking StatementsThis press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended) concerning Enliven and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Enliven, as well as assumptions made by, and information currently available to, management of Enliven. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements regarding the potential of, and plans and expectations regarding Enliven’s programs, including ELVN-001 and ELVN-002; Enliven’s pipeline of product candidates; expected milestones for ELVN-001 and ELVN-002, including the expected timing of dosing of ELVN-002 for the combination trial and of data from the clinical trials of ELVN-001 and ELVN-002; statements relating to Enliven’s expected cash runway; and the anticipated use of proceeds from the PIPE financing; and statements by Enliven’s Co-founder and Chief Executive Officer. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various risks and uncertainties, including, without limitation: the limited operating history of Enliven; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, product candidates; the outcome of preclinical testing and early clinical trials for product candidates and the potential that the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials; Enliven’s limited resources; the risk of failing to demonstrate safety and efficacy of product candidates; Enliven’s limited experience as a company in designing and conducting clinical trials; the potential for interim, topline and preliminary data from Enliven’s preclinical studies and clinical trials to materially change from the final data; potential delays or difficulties in the enrollment or maintenance of patients in clinical trials; developments relating to Enliven’s competitors and its industry, including competing product candidates and therapies; the decision to develop or seek strategic collaborations to develop Enliven’s current or future product candidates in combination with other therapies and the cost of combination therapies; the ability to attract, hire, and retain highly skilled executive officers and employees; the ability of Enliven to protect its intellectual property and proprietary technologies; the scope of any patent protection Enliven obtains or the loss of any of Enliven’s patent protection; reliance on third parties, including contract manufacturing organizations, contract research organizations and strategic partners; general market or macroeconomic conditions; Enliven’s ability to obtain additional capital to fund Enliven’s general corporate activities and to fund Enliven’s research and development; and other risks and uncertainties, including those more fully described in Enliven’s filings with the Securities and Exchange Commission (SEC), which may be found in the section titled “Risk Factors” in Enliven’s Annual and Quarterly Reports on Form 10-K and 10-Q filed with the SEC and in Enliven’s future reports to be filed with the SEC. Except as required by applicable law, Enliven undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. Head-to-Head ComparisonsThe Company has not performed any head-to-head trials for ELVN-001. As a result, the data referenced in this press release is derived from different clinical trials at different points in time, with differences in trial design and patient populations. As a result, conclusions from cross-trial comparisons cannot be made. Contact:Investorsir@enliventherapeutics.com Mediamedia@enliventherapeutics.com Enliven Therapeutics, Inc.Selected Condensed Consolidated Financial Information(in thousands, except per share data)(unaudited) Statements of OperationsThree Months Ended March 31, 2024 2023 Operating expenses: Research and development$19,970 $11,880 General and administrative 6,017 4,538 Total operating expenses 25,987 16,418 Loss from operations (25,987) (16,418)Other income (expense), net 3,249 1,694 Net loss$(22,738) $(14,724)Net loss per share, basic and diluted$(0.54) $(0.80)Weighted-average shares outstanding, basic and diluted 42,046 18,515 Balance SheetsMarch 31, December 31, 2024 2023 Assets Current assets: Cash, cash equivalents and marketable securities$320,504 $253,148 Restricted cash 54 54 Prepaid expenses and other current assets 6,641 2,949 Contingent value right asset 10,000 10,000 Total current assets 337,199 266,151 Property and equipment, net 683 742 Operating lease right-of-use assets 241 320 Deferred offering costs 563 563 Other long-term assets 4,091 4,091 Total assets$342,777 $271,867 Liabilities and Stockholders' Equity Current liabilities: Accounts payable$2,727 $532 Accrued expenses and other current liabilities 12,304 15,362 Contingent value right liability 10,000 10,000 Total current liabilities 25,031 25,894 Long-term liabilities 34 67 Total liabilities 25,065 25,961 Stockholders' equity 317,712 245,906 Total liabilities and stockholders' equity$342,777 $271,867

Vincerx continued to progress Phase 1 dose-escalation studies for VIP943, a potentially best-in-class anti-CD123 antibody-drug conjugate (ADC), and VIP236, a first-in-class small molecule-drug conjugate (SMDC) The National Institutes of Health (NIH) and Vincerx announced positive results from enitociclib Phase 1 combination study, reporting a fourth partial response (PR) in a patient with peripheral T-cell lymphoma (PTCL) Vincerx reported the first PR in a patient with transformed follicular lymphoma (tFL) in its Phase 1 dose-escalation study of enitociclib as a monotherapy Recent financing provides expected cash runway through 2024 PALO ALTO, Calif., May 14, 2024 (GLOBE NEWSWIRE) -- Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today reported financial results for the first quarter of 2024 and provided a corporate update. “During the first quarter, we maintained momentum across our highly differentiated pipeline and our VersAptx™ platform,” said Ahmed Hamdy, M.D., Chief Executive Officer. “Our recent financing provides capital to support the dose-escalation studies for our potentially best-in-class ADC, VIP943, and first-in-class SMDC, VIP236. We look forward to sharing an update on VIP236 by the end of Q3 and on VIP943 by the end of Q4. This timing will enable us to present more advanced dose-escalation data for both programs.” “We also continue to be excited by the clinical progress of enitociclib,” continued Dr. Hamdy. “We have one patient with tFL who has achieved a metabolic PR and continues on enitociclib monotherapy therapy after 33 cycles. In addition, in the NIH study of enitociclib in combination with venetoclax and prednisone, two-thirds of patients have achieved a PR. We believe these clinical results show enitociclib is a best-in-class CDK9 inhibitor and has the potential to be a preferred partner for innovative combination therapies for hard-to-treat cancers.” FIRST QUARTER 2024 CLINICAL PROGRAM HIGHLIGHTS VIP236 VIP236 is an αVβ3 SMDC conjugated to an optimized camptothecin (CPT) payload, created from Vincerx’s VersAptx platform. VIP236 is a first-in-class drug designed to deliver its optimized CPT payload directly to tumor tissues to overcome chemotherapy-related side effects. Preclinical studies have shown 11 times more optimized CPT is delivered to the cancerous tissues than found circulating in the blood. In addition, the optimized CPT is designed to limit drug transporter liabilities, a common mechanism for cancer resistance to chemotherapy. At the 2024 American Association for Cancer Research (AACR) Annual Meeting, Vincerx reported positive preliminary monotherapy data on VIP236 from a Phase 1 dose-escalation study demonstrating signs of clinical activity, including tumor reduction, and an improved safety pro heavily pretreated patients with metastatic solid tumors. As of March 25, 2024, the VIP236 open-label, multicenter, Phase 1 dose-escalation study (NTC05371054) had enrolled 20 patients with advanced or metastatic cancers unresponsive to standard therapies. Vincerx looks forward to sharing additional Phase 1 data by the end of Q3 2024. VIP943 VIP943, a novel CD123-targeted ADC created from Vincerx’s VersAptx platform, consists of an anti-CD123 antibody, a unique linker cleaved intracellularly by legumain, and a novel kinesin spindle protein inhibitor (KSPi) payload enhanced with Vincerx’s CellTrapper® technology. Its next-generation effector chemistry was designed to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities. Enrollment has begun in the fourth cohort of the Phase 1 dose-escalation study of VIP943 in relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and B-cell acute lymphoblastic leukemia (B-ALL) (NCT06034275). Preliminary pharmacokinetic (PK) results on the first two cohorts were presented at the 2024 AACR Annual Meeting and as expected, showed very little payload circulating in the blood. In addition, no dose-limiting toxicities have occurred in the 11 patients who have received VIP943 so far. The preliminary PK and early observations of a favorable safety pro consistent with VIP943 preclinical data. Vincerx expects to present updated Phase 1 dose-escalation data for VIP943 by the end of Q4 2024. Enitociclib Enitociclib is a highly selective CDK9 inhibitor designed to block the activation of RNA polymerase II, leading to the reduction of oncogenes, including MYC and MCL1. Enitociclib is currently in a Phase 1 dose-escalation study (NTC05371054) evaluating the combination of enitociclib, venetoclax and prednisone in diffuse large B-cell lymphoma (DLBCL) and PTCL. This study is being conducted in collaboration with the NIH. In January 2024, Vincerx and the NIH reported two PRs in patients with PTCL with tumor reductions ranging from 86% to 91%. Additionally, one PR was reported in a patient with double-hit diffuse large B-cell lymphoma (DH-DLBCL) with an 80% reduction in tumor burden. Most recently, an additional PR was announced in PTCL with a reduction in lymph node size and skin lesions, totaling four PRs observed to date. Notably, these results were obtained with enitociclib doses below the anticipated efficacious levels. Currently, the study is enrolling patients for the third dose level (30 mg), which is the recommended dose established in the enitociclib monotherapy study. These data will be presented by the NIH at the upcoming AACR Advances in Malignant Lymphoma meeting in June 2024. In a Phase 1 dose-escalation study (NCT02635672) of enitociclib as a monotherapy, one newly confirmed metabolic PR was observed with 63% tumor reduction in a tFL patient who has been enrolled in the study for just under two years. This is particularly notable because outcomes of patients with tFL are historically poor. In total, this study enrolled 63 patients in the dose-escalation and expansion cohorts. Enitociclib showed a favorable safety profile, dose-proportional pharmacokinetics, and on-target pharmacodynamic activity. Significant clinical benefit across various indications includes two patients with DH-DLBCL who experienced durable complete metabolic remissions (3.7 and 2.3 years), which continued more than two years after stopping treatment. This long duration of treatment and response for patients with DH-DLBCL and tFL highlight enitociclib’s favorable safety pro monotherapy efficacy in hard-to-treat hematologic malignancies. Additionally, 13 patients with solid tumors achieved stable disease as their best response to monotherapy treatment. Notably, of these, five were patients with ovarian cancer, providing a promising signal for future combination studies in this indication. Research collaborations continue with the University of Calgary and the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium (POETIC) to discover combination strategies for pediatric leukemia and central nervous system tumors. To date, these collaborations have shown that enitociclib has monotherapy and combination activity in preclinical models of rhabdomyosarcoma, neuroblastoma and KMT2A-rearranged pediatric leukemia. VIP924 VIP924 is a first-in-class CXCR5-targeted ADC created from Vincerx’s VersAptx platform. VIP924 can be evaluated in B-cell malignancies, including MCL, FL, DLBCL, and CLL and monotherapy and in combination. IND application is anticipated in late 2025 or early 2026, pending funding. VersAptx™ Platform VersAptx is Vincerx’s versatile and adaptable, next-generation bioconjugation platform. The modular nature of this platform enables the combination of different targeting, linker and payload technologies to develop bespoke bioconjugates to address different cancer biologies. At the AACR Annual Meeting, Vincerx reported data from preclinical studies applying the next-generation effector chemistry of its VersAptx platform to TRODELVY® and ENHERTU®, two marketed ADCs, demonstrating the potential to improve tumor toxicity of ADCs by orders of magnitude, while improving on safety and tolerability. These findings further support the versatility of VersAptx to address multiple cancer types and increase the efficacy and safety of ADCs. FIRST QUARTER 2024 FINANCIAL RESULTS Vincerx had approximately $5.1 million in cash and cash equivalents as of March 31, 2024, which does not include the proceeds from our recent financing in April, as compared to approximately $12.8 million as of December 31, 2023. Based on its current business plans and assumptions, Vincerx believes its available capital, including the recent financing proceeds of approximately $17.8 million, will be sufficient to meet its operating requirements through the end of 2024. Research and development expenses for the first quarter ended March 31, 2024 were approximately $4.6 million, as compared to approximately $10.9 million for the same period in 2023. This decrease is primarily the result of decreases in manufacturing services associated with our ADC programs of approximately $2.6 million, research services of approximately $2.6 million, and personnel-related expenses of approximately $1.1 million. General and administrative expenses for the first quarter ended March 31, 2024 were approximately $2.9 million, as compared to approximately $4.5 million for the same period in 2023. This decrease is primarily driven by decreases in personnel-related expenses of approximately $0.6 million, professional services of $0.5 million and facilities and other corporate overhead expenses of $0.3 million. For the first quarter ended March 31, 2024, Vincerx reported a net loss of approximately $12.4 million, or $0.58 per share. For the first quarter ended March 31, 2023, Vincerx reported a net loss of approximately $14.6 million, or $0.69 per share. About Vincerx Pharma, Inc. Vincerx Pharma, Inc. is a clinical-stage biopharmaceutical company committed to developing differentiated and novel therapies to address the unmet medical needs of patients with cancer. Vincerx has assembled a seasoned management team with a proven track record of successful oncology drug development, approvals, and value creation. Vincerx’s diverse pipeline consists of the next-generation antibody-drug conjugate, VIP943, in Phase 1; small molecule-drug conjugate, VIP236, in Phase 1; preclinical antibody-drug conjugate, VIP924; CDK9 inhibitor, enitociclib, in an NIH-sponsored Phase 1; and VersAptx, its versatile and adaptable, next-generation bioconjugation platform. Vincerx is based in Palo Alto, California, and has a research facility in Monheim, Germany. For more information, please visit and follow Vincerx on LinkedIn. Forward-Looking Statement This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended, that are intended to be covered by the “safe harbor” created by those sections. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, expectations and events, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “suggest,” “seek,” “intend,” “plan,” “goal,” “potential,” “on-target,” “on track,” “project,” “estimate,” “anticipate,” or other comparable terms. All statements other than statements of historical facts included in this press release are forward-looking statements. Forward-looking statements include, but are not limited to, Vincerx’s business model, cash runway, pipeline, strategy, timeline, product candidates and attributes, and preclinical and clinical development, timing, and results. Forward-looking statements are neither historical facts nor assurances of future performance or events. Instead, they are based only on current beliefs, expectations, and assumptions regarding future business developments, future plans and strategies, projections, anticipated events and trends, the economy, and other future conditions. Forward-looking statements are subject to inherent uncertainties, risks, and changes in circumstances that are difficult to predict, many of which are outside Vincerx’s control. Actual results, conditions, and events may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results, conditions, and events to differ materially from those indicated in the forward-looking statements include, but are not limited to, general economic, financial, legal, political, and business conditions; risks associated with preclinical or clinical development and trials, including those conducted prior to Vincerx’s in-licensing; failure to realize the benefits of Vincerx’s license agreement with Bayer; risks related to the timing of expected business and product development milestones; changes in the assumptions underlying Vincerx’s expectations regarding its future business or business model; Vincerx’s ability to successfully develop and commercialize product candidates; Vincerx’s capital requirements, availability and uses of capital, and cash runway; and the risks and uncertainties set forth in Form 10-K for the year ended December 31, 2023 and subsequent reports filed with the Securities and Exchange Commission by Vincerx. Forward-looking statements speak only as of the date hereof, and Vincerx disclaims any obligation to update any forward-looking statements. Vincerx, the Vincerx logo, CellTrapper, and VersAptx are our trademarks. This press release also contains trademarks and trade names that are the property of their respective owners. Contacts Gabriela Jairala Vincerx Pharma, Inc. gabriela.jairala@vincerx.com Totyana Simien Inizio Evoke Comms totyana.simien@inizioevoke.com Vincerx Pharma, Inc. Condensed Consolidated Balance Sheets (in thousands) March 31, 2024 December 31, 2023 (unaudited) ASSETS Current assets: Cash and cash equivalents $ 5,110 $ 12,782 Prepaid expenses 700 51 Grant receivable 1,025 1,044 Other current assets 842 856 Total current assets 7,677 14,733 Right-of-use assets 1,950 2,201 Property, plant and equipment, net 111 125 Other assets 1,345 1,158 Total assets $ 11,083 $ 18,217 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 2,314 $ 2,497 Accrued expenses 1,727 1,755 Lease liability 1,198 1,162 Common stock warrant liabilities 5,395 191 Total current liabilities 10,634 5,605 Lease liability, net of current portion 1,021 1,340 Other noncurrent liabilities 50 50 Total liabilities 11,705 6,995 Total stockholders' equity (deficit) (622 ) 11,222 Total liabilities and stockholders' equity $ 11,083 $ 18,217 Vincerx Pharma, Inc. Condensed Consolidated Statements of Operations (unaudited) (in thousands, except per share amounts) For the three months ended March 31, 2024 2023 Operating expenses: General and administrative $ 2,922 $ 4,497 Research and development 4,556 10,911 Total operating expenses 7,478 15,408 Loss from operations (7,478 ) (15,408 ) Other income (expense) Change in fair value of warrant liabilities (5,204 ) 18 Interest income 99 466 Other income 154 274 Total other income (expense) (4,951 ) 758 Net loss $ (12,429 ) $ (14,650 ) Net loss per common share, basic and diluted $ (0.58 ) $ (0.69 ) Weighted average common shares outstanding, basic and diluted 21,400 21,188