On March 25, 2025 X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, reported financial results for the fourth quarter and full year ended December 31, 2024, and highlighted key 2024 and recent events and expected upcoming milestones (Press release, X4 Pharmaceuticals, MAR 25, 2025, View Source [SID1234651397]).

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"As expected, 2024 was a transformative year for the company and our momentum has continued into 2025," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "With the U.S. approval and launch of our first product, XOLREMDI (mavorixafor) in WHIM syndrome, we are now a fully integrated company advancing our mission to serve those with rare immunodeficiencies and few treatment options. As we await word from the EU regulatory authority on the potential approval of mavorixafor for WHIM in that region, we’ve continued to expand our potential reach to the global WHIM community through our recently completed commercialization partnerships in the EU, Australia, New Zealand, and the Middle East and North Africa (MENA) territories."

Dr. Ragan continued: "In addition, we continue to make meaningful progress in our efforts to develop mavorixafor for the larger immunodeficiency population with chronic neutropenia (CN). Following positive results from our Phase 2 trial of mavorixafor in certain CN populations, we are currently conducting a global, pivotal Phase 3 trial in CN. With a large number of sites now activated and global screening ongoing, we expect full trial enrollment in the third or fourth quarter of this year and top-line trial data in the second half of 2026."

Dr. Ragan concluded: "Market research and patient testimonials continue to strengthen our belief that there remains significant untapped potential for XOLREMDI for the U.S. and possibly global WHIM patient populations. We are equally confident in mavorixafor’s future potential to address unmet needs in the global CN community."

Key 2024 and Recent Corporate Highlights

Commercializing XOLREMDI (mavorixafor) in WHIM Syndrome, a Rare Primary Immunodeficiency
•FDA Approval and Launch of XOLREMDI: In May 2024, X4 launched XOLREMDI (mavorixafor) following approval by the U.S. Food and Drug Administration (FDA) for its use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Concurrent with the approval of XOLREMDI, X4 received a priority review voucher (PRV), which it subsequently sold to another drug developer for $105 million.
•U.S. Launch Update: X4 is continuing to execute on its commercialization of XOLREMDI in the U.S., generating $2.6 million in sales from its mid-May launch through December 2024. During the year, the company advanced disease awareness by engaging with physicians and rare disease patient advocacy groups through a combination of in-person and targeted digital education campaigns. The company’s suite of patient services, including its X4Connect and nurse educator programs, continue to provide access and support for patients prescribed XOLREMDI.
•Clinical Data Publications and Presentations: Journal publications and presentations of clinical data results at top medical meetings, including those of the American Society of Hematology (ASH) (Free ASH Whitepaper), the American Academy of Allergy, Asthma, and Immunology (AAAAI), and the Clinical Immunology Society (CIS), have provided further visibility on the XOLRMEDI approval.
•Maximizing the Global Opportunity for Mavorixafor in WHIM Syndrome:
MAA Acceptance. In January 2025, X4 announced that its submitted Marketing Authorization Application (MAA) for mavorixafor in the treatment of WHIM syndrome was validated for review by the European Medicines Agency (EMA), meeting an important corporate milestone. Given a typical 12- to 15-month review process, the company believes approval to be possible in the first half of 2026.
EU/ANZ Partnership. Also in January 2025, the company announced that it had entered into an exclusive licensing and supply agreement under which Norgine Pharma UK will commercialize mavorixafor in Europe, Australia, and New Zealand following any regulatory approvals in those territories. X4 received €28.5 million as an upfront payment and is eligible to receive up to €226 million in potential regulatory and commercial milestone payments in addition to tiered, double-digit royalties.
MENA Partnership. In addition, X4 announced in February 2025, that it had entered into an agreement with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in Saudi Arabia, United Arab Emirates, Qatar, Oman, Kuwait, Bahrain, and Egypt, following any approvals in those countries. Pending regulatory approvals in the region, taiba expects to be able to make XOLREMDI available to WHIM patients through a named-patient (compassionate use) program that allows physicians to prescribe medicines approved in other countries to local patients with no other treatment options.

Advancing Mavorixafor in Chronic Neutropenia (CN)
•Phase 2 Clinical Data De-Risk Ongoing Phase 3 4WARD Clinical Trial. Throughout 2024, X4 presented positive interim and full data sets from its Phase 2 trial that evaluated mavorixafor in the treatment of people with chronic neutropenia (CN), as a monotherapy and in combination with injectable granulocyte colony-stimulating factor (G-CSF), the only therapy approved in the U.S. for severe chronic neutropenia. The six-month, open-label clinical trial enrolled a total of 23 participants and demonstrated that:
◦Once-daily oral mavorixafor was generally well tolerated +/- G-CSF, with no drug-related serious adverse events reported, consistent with previous clinical studies;

◦Mavorixafor treatment durably and meaningfully increased participants’ mean absolute neutrophil counts (ANC) across all study populations; and
◦Physicians were willing and able to reduce the use of G-CSF in participants also treated with mavorixafor, maintaining mean ANC levels in the normal range.
•Ongoing Phase 3 4WARD Trial. In June 2024, the company announced the initiation of its global, pivotal Phase 3 clinical trial (NCT06056297), evaluating oral, once-daily mavorixafor (+/- G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week 4WARD trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants.
•4WARD Trial Updates. X4 announced today that this first-of-its-kind trial is now activated at ~90% of targeted sites worldwide.
Refining the 4WARD Protocol: Based on FDA and EMA guidance, an amendment to the 4WARD protocol has been implemented, focusing enrollment on those with the highest unmet needs and refining a co-primary endpoint. The trial is now only enrolling participants with moderate and severe neutropenia (ANC below 1,000 cells per microliter), an ANC level consistent with the company’s targeted patient population for mavorixafor, if approved. In addition, the ANC component of the primary endpoint will now be uniform across all participants, seeking to demonstrate infection benefit from an ANC increase of at least 500 cells per microliter in study participants on active drug.
Enrollment Update: Given the average screening rates currently observed, the company expects the trial to be fully enrolled in the third or fourth quarter of 2025, and disclosure of top-line data in the second half of 2026.

Strategic Restructuring
In February 2025, X4 announced a strategic restructuring to sharpen focus and maximize the opportunity for mavorixafor in chronic neutropenia. Related activities included reducing overall headcount, discontinuing research efforts, pausing pre-clinical drug candidate programs, and closing the company’s facility in Vienna, Austria, as well as right-sizing its U.S. commercial field team and streamlining other spending. X4 continues to expect that these efforts will decrease spending by $30-35 million annually.

Fourth-Quarter and Full-Year 2024 Financial Results
•Cash position: X4 had $102.8 million in cash, cash equivalents, restricted cash, and marketable securities as of December 31, 2024. Pro-forma for the €28.5 million payment received from Norgine in January 2025 and the expected financial impact of the strategic restructuring announced in February 2025, the company believes it has sufficient funds to support operations into the first half of 2026.
•Revenue and Cost of Revenue: For the fourth quarter and full year ended December 31, 2024, X4 reported net product revenue of $1.4 million and $2.6 million, respectively, and cost of revenue of $0.3 million and $0.8 million, respectively, related to sales of XOLREMDI.
•Research and Development (R&D) Expenses were $21.7 million and $81.6 million for the fourth quarter and full year ended December 31, 2024, respectively, as compared to $15.3 million and $72.0 million for the comparable periods in 2023. R&D expenses included $1.2 million and $4.3 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2024, respectively.
•Selling, General, and Administrative (SG&A) Expenses were $15.1 million and $61.5 million for the fourth quarter and full year ended December 31, 2024, respectively, as compared to $9.9 million and $35.5 million for the comparable periods in 2023. SG&A expenses included $1.0 million and $3.9 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2024, respectively.
•Gain on Sale of Non-Financial Asset: For the year ended December 31, 2024, X4 recognized a gain on the sale of a priority review voucher (PRV) to a third party for $105.0 million in cash. The PRV was awarded to X4 by the FDA under its Rare Pediatric Disease program upon the approval of XOLREMDI. Under this program, the FDA awards PRVs to sponsors of rare pediatric disease product applications that meet certain criteria to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
•Net Loss: X4 reported a net loss of $39.8 million and $37.5 million for the fourth quarter and full year ended December 31, 2024, respectively, as compared to $19.1 million and $101.2 million for the comparable periods in 2023. Net loss for the year ended December 31, 2024 includes the sale of a PRV for $105.0 million, as noted above. Net loss included $2.2 million and $8.2 million of stock-based compensation expenses for the fourth quarter and full year ended December 31, 2024, respectively.

Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights. The conference call can be accessed by dialing 1-800-267-6316 from the United States or 1-203-518-9783 internationally, followed by the conference ID: X4PHARMA. The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay will be available on the website.

About WHIM Syndrome
WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilization of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations in the acronym. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections.

About XOLREMDI (mavorixafor)
XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. as a once-daily oral treatment for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. XOLREMDI is the only treatment specifically approved for patients with WHIM syndrome in the U.S.

About Chronic Neutropenia and Mavorixafor
Chronic neutropenia is a primary, rare blood condition lasting more than three months, persistently or intermittently, and characterized by increased risk of infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize functional neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC).

About the 4WARD Clinical Trial
The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants with confirmed trough ANC levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response.

On March 25, 2025 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company developing innovative treatments that exploit specific cancer cell vulnerabilities while minimizing damage to healthy cells, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update (Press release, Aprea, MAR 25, 2025, View Source [SID1234651393]).

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"We made excellent progress across our pipeline in 2024, laying a strong foundation for the year ahead," said Oren Gilad, Ph.D., President and Chief Executive Officer of Aprea. "We continue to enroll patients in the ACESOT-1051 trial evaluating our WEE1 kinase inhibitor, APR-1051, which we believe has best in class potential. The compound appears safe and well tolerated to date with no hematologic toxicity. We look forward to reporting open label data from ACESOT-1051 in the second half of the year. We are also advancing ATRN-119, our highly selective first-in-class macrocyclic ATR inhibitor. The ongoing ABOYA-119 trial is now evaluating ATRN-119 as continuous once daily and twice daily monotherapy in order to maximize therapeutic benefit. Our ultimate goal is to transform the treatment paradigm for difficult to treat cancers by unlocking the full potential of DDR-based therapies."

Key Business Updates and Potential Upcoming Key Milestones

ACESOT-1051: A Biomarkers Focused, Phase 1 Trial of Oral WEE1 inhibitor, APR-1051

● APR-1051 is a potent and selective small molecule that has been designed to potentially solve tolerability challenges of the WEE1 class and may achieve greater clinical activity than other programs currently in development. Aprea is advancing APR-1051 as monotherapy in cancers with Cyclin E over-expression, as well as other biomarkers that may predict sensitivity to WEE1 inhibition. Cancers over-expressing Cyclin E represent a high unmet medical need. Patients with Cyclin E over-expression have poor prognosis and, currently, have no effective therapies available.
● Patients are now being enrolled in Cohort 5 (70 mg dose) of the ACESOT-1051 (A Multi-Center Evaluation of WEE1 Inhibitor in Patients with Advanced Solid Tumors, APR-1051) trial. This Phase 1 clinical trial is evaluating single-agent APR-1051 in advanced solid tumors harboring cancer-associated gene alterations. No hematological toxicities have been observed to date. The primary objectives of the Phase 1 study are to measure safety, dose-limiting toxicities (DLTs), maximum tolerated dose or maximum administered dose (MTD/MAD), and recommended Phase 2 dose (RP2D); secondary objectives are to evaluate pharmacokinetics and preliminary efficacy according to RECIST or PCWG3 criteria; pharmacodynamic parameters are exploratory objectives.

● In October 2024, preliminary findings from the ACESOT-1051 trial were reported in a poster at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, in Barcelona, Spain. The poster can be viewed on Aprea’s corporate website here.
● Preliminary efficacy data from ACESOT-1051 are expected in the second half of 2025. For more information, refer to ClinicalTrials.gov NCT06260514.

ABOYA-119: Ongoing Clinical Trial Evaluating ATR inhibitor, ATRN-119

● ATRN-119 is a potent and highly selective first-in-class macrocyclic ATR inhibitor, designed to be used in patients with mutations in DDR-related genes. Cancers with mutations in DDR-related genes represent a high unmet medical need. Patients with DDR-related gene mutations have a poor prognosis and, currently, there are no effective therapies available for them.
● ATRN-119 is being evaluated in the open-label Phase 1/2a clinical trial of ABOYA-119 as monotherapy in patients with advanced solid tumors having at least one mutation in a defined panel of DDR-related genes. Patients are currently being enrolled at Dose Level 7, with both 1100 mg once daily and 550 mg twice daily doses being evaluated independently and in parallel. The addition of twice daily dosing was implemented to potentially optimize ATRN-119’s activity across a 24-hour cycle thereby providing better target coverage and maximal clinical benefit. This is expected to increase the likelihood of achieving superior clinical outcomes and may potentially accelerate the path to regulatory approval and commercialization.
● An update from the ABOYA-119 trial was provided in a poster at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics in October, 2024. A copy of the poster can be viewed here.
● For more information on ABOYA-119, please refer to clinicaltrials.gov NCT04905914.

Corporate

● Aprea engaged Philippe Pultar, MD in October 2024 as senior medical advisor to support the development and advancement of APR-1051. Dr. Pultar is a seasoned pharmaceutical executive with extensive experience in oncology. He was most recently employed at Zentalis Pharmaceuticals where he played a key role in the strategy and execution of the global clinical development of azenosertib, a WEE1 inhibitor.

Select Financial Results for the Fourth Quarter ended December 31, 2024

● For the quarter ended December 31, 2024, the Company reported an operating loss of $3.2 million, compared to an operating loss of $3.7 million in the fourth quarter of 2023.
● Research and Development (R&D) expenses were $2.4 million for the quarter ended December 31, 2024, compared to $2.0 million for the fourth quarter of 2023. The increase in R&D expense was primarily related to an increase in personnel costs primarily related to new hires and severance.
● General and Administrative (G&A) expenses were $1.1 million for the quarter ended December 31, 2024, compared to $1.6 million for the comparable period in 2023.
● The Company reported a net loss of $2.9 million ($0.49 per basic share) on approximately 6.0 million weighted-average common shares outstanding for the quarter ended December 31, 2024, compared to a net loss of $3.4 million ($0.92 per basic share) on approximately 3.7 million weighted average common shares outstanding for the comparable period in 2023.

Select Financial Results for the Year ended December 31, 2024

● As of December 31, 2024, the Company reported cash and cash equivalents of $22.8 million compared to $21.6 million as of December 31, 2023. The Company believes its cash and cash equivalents as of December 31, 2024 will be sufficient to meet its currently projected operating expenses and capital expenditure requirements into the first quarter of 2026.
● For the year ended December 31, 2024, the Company reported an operating loss of $14.3 million, compared to an operating loss of $15.5 million for the year ended December 31, 2023.
● R&D expenses were $9.4 million for the year ended December 31, 2024, compared to $7.6 million for the year ended December 31, 2023. The increase in R&D expense was primarily related to the ABOYA-119 clinical trial to evaluate ATRN-119, the initiation of the ACESOT-1051 clinical trial to evaluate APR-1051 and an increase in personnel costs primarily related to new hires and severance.
● G&A expenses were $6.5 million for the year ended December 31, 2024, compared to $8.4 million for the year ended December 31, 2023. The decrease in G&A expenses was primarily due to a decrease in personnel costs primarily related to severance expense for former executives and insurance premiums.
● The Company reported a net loss of $13.0 million ($2.35 per basic share) on approximately 5.5 million weighted-average common shares outstanding for the year ended December 31, 2024, compared to a net loss of $14.3 million ($3.95 per basic share) on approximately 3.6 million weighted average common shares outstanding for the comparable period in 2023.

On March 25, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported the first patient has been successfully dosed in the Company’s pivotal TACTI-004 Phase III trial (Press release, Immutep, MAR 25, 2025, View Source [SID1234651375]). TACTI-004 will evaluate Immutep’s eftilagimod alfa, a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab) and chemotherapy as first line treatment for patients with advanced or metastatic non-small cell lung cancer (1L NSCLC).

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Dr. Ina Nordman, who treated the first patient at Calvary Mater Newcastle Hospital in Australia, stated, "We are very excited to participate in this important Phase III trial. Despite advancements in the treatment landscape for non-small cell lung cancer, there remains a high unmet need for new approaches that can safely extend patients’ lives. The anti-cancer immune response driven by efti’s unique mechanism of action as an MHC Class II agonist in combination with KEYTRUDA has led to strong efficacy across all PD-L1 levels with favourable safety in multiple lung cancer trials. We hope to see this study confirm the promise of this novel combination to provide patients with a powerful new treatment option."

Immutep CEO, Marc Voigt, said, "Dosing the first patient in our pivotal Phase III trial ranks among the most significant milestones in the Company’s history. We are excited about the potential of the TACTI-004 study to deliver a new standard-of-care therapy for patients with metastatic or advanced non-small cell lung cancer that includes efti in combination with KEYTRUDA. If successful, the study will result in a clinically meaningful and statistically improved survival benefit and thus could potentially be practice changing."

Immutep CSO, Frédéric Triebel, M.D., Ph.D, commented, "As a result of all global regulatory interactions to date including previous discussion with US FDA under Project Optimus and tolerability issues at 90 mg1, we are moving forward with 30 mg subcutaneous efti dosing used in previous studies. The ability of 30 mg efti in combination with KEYTRUDA to activate the immune system and fight non-small cell lung cancer regardless of PD-L1 expression has been demonstrated across multiple clinical trials.2 Importantly, this novel approach has an excellent safety profile while delivering strong efficacy that compares favourably to standard-of-care therapies, including high rates of durable responses and compelling progression-free survival and overall survival."

Recruitment in TACTI-004 is underway at a growing number of activated clinical sites and countries with approvals from all regulatory authorities continues to expand including Australia, Austria, Belgium, Bulgaria, Canada, Germany, Greece, Hungary, India, Ireland, Italy, Latvia, Lithuania, Portugal, Spain, and the United Kingdom. Further regulatory clearances in three additional countries are expected shortly, with the remaining countries anticipated in the weeks and months ahead.

Lung cancer is the leading cause of death among all cancer types and the incidence is set to increase to approximately 3 million cases worldwide by 2030.3 NSCLC is the most common type of lung cancer representing ~80-85% of all diagnoses.4 The condition is often diagnosed at a late stage, and less than 30% of patients are alive five years after diagnosis.5,6 There remains a high unmet need for additional treatment options for people living with NSCLC.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About TACTI-004
TACTI-004 (Two ACTive Immunotherapies) is a randomised, double-blind, controlled Phase III study evaluating eftilagimod alfa (efti), a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab) and chemotherapy as first line therapy for patients with advanced or metastatic non-small cell lung cancer with no EGFR, ALK or ROS1 genomic tumour aberrations. The global trial will enrol approximately 756 patients regardless of PD-L1 expression and with non-squamous or squamous tumours at over 150 clinical sites in over 25 countries. Patients will be randomised 1:1 to receive either efti in combination with pembrolizumab and chemotherapy in the treatment arm or pembrolizumab in combination with chemotherapy and placebo in the control arm. The study’s dual primary endpoints are progression-free survival and overall survival.

On March 24, 2024, Hoth Therapeutics, Inc. ("Hoth" or the "Company") entered into a Project Order Agreement (the "Agreement") with OnTargetx R&D Inc. ("OnTargetx") pursuant to the terms of an existing Master Service Agreement originally entered into on November 8, 2024 (Filing, Hoth Therapeutics, MAR 24, 2025, View Source [SID1234651459]).

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Under the Agreement, OnTargetx, in collaboration with ITR Laboratories, will perform a 4-week intravenous injection toxicity study followed by a 14-day recovery period in C57BL/6 mice, referred to as Study No. 701821 (P210614002-B). The study includes dose formulation, toxicokinetic analysis, clinical pathology, necropsy, histopathology, and detailed reporting. A total of 448 main study animals and 44 spares will be used.

The study has been designed to test the Company’s proposed treatment for mast-cell derived cancers (HT-KIT), with respect to which the Company has an exclusive, worldwide, royalty bearing license to certain intellectual property to, among other things, discover, develop, make, have made, use and sell certain licensed products and sell, use and practice certain licensed services with respect to cancer (and anaphylaxis) from North Carolina State University ("NC State").

Recently, the Company, in conjunction with NC State, filed amendments to a pending patent application related to its antisense oligonucleotide (ASO) technology platform. The amended claims broaden and clarify the scope of intellectual property protections around antisense oligomers targeting specific pre-mRNA sequences involved in gene expression and immune response pathways.

The amended claims include compositions and methods involving antisense oligomers designed to modulate splicing of specific genes and reduce expression of immune-related cell surface receptors. The claims also describe potential pharmaceutical compositions and therapeutic uses across various species.

These amendments reflect routine practice and are intended to strengthen the Company’s patent position in anticipation of further preclinical development and potential licensing discussions.

On March 24, 2025 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported an update on the company’s development pipeline and announced financial results for the fiscal year ended December 31, 2024 (Press release, Quince Therapeutics, MAR 24, 2025, View Source [SID1234651396]).

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Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "Our Phase 3 pivotal NEAT clinical trial has exceeded 50% enrollment with 71 participants screened and 61 randomized. Recent and upcoming new site activations are expected to accelerate enrollment through our target completion date in the second quarter of 2025. Assuming target enrollment completion, topline results from the NEAT study are anticipated in the fourth quarter of 2025."

Pivotal Phase 3 NEAT Clinical Trial

Enrolled 61 participants to date in the company’s Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial to evaluate the neurological effects of eDSP (previously referred to as EryDex) in patients with Ataxia-Telangiectasia (A-T), including 54 participants in the six to nine year-old primary analysis population.
Transitioned 24 participants to date to the NEAT open label extension study (NCT06664853/IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to an open label extension study.
Expect screening and randomization to accelerate in the coming weeks due to several new geographic regions and sites scheduled for activation.
Quince plans to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older.
Pivotal Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration (FDA).
Expect to report Phase 3 NEAT topline results in the fourth quarter of 2025 with a New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results.
NEAT is an international, multicenter, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of the company’s lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells), in patients with A-T.
Participants will be randomized (1:1) between eDSP or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in a rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo as per the SPA agreement with the FDA.
Pipeline and Corporate Updates

Generating Phase 2 clinical trial study designed to evaluate eDSP for the potential treatment of patients with Duchenne muscular dystrophy (DMD). Quince plans to initiate a DMD Phase 2 study in 2025, which the company expects to conduct utilizing capital efficient study approaches and with financial support from grant and/or opportunistic funding opportunities.
Published long-term safety data from patients with A-T treated with eDSP for a minimum of 24 months in Frontiers in Neurology in January 2025. The online publication titled Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with Ataxia-Telangiectasia can be accessed here.
Appointed Dr. William Whitehouse, Honorary Clinical Associate Professor of the School of Medicine at the University of Nottingham and recently retired Consultant Pediatric Neurologist at Nottingham Children’s Hospital, Nottingham University Hospitals NHS Trust, to the company’s Scientific Advisory Board (SAB).
Strengthened Quince’s growing intellectual property portfolio and long-term market position with the issuance of a Notice of Allowance from the U.S. Patent and Trademark Office’s (USPTO) in February 2025. This extends the company’s patent claims into 2036 in the U.S., supplementing its existing U.S. patent with coverage of patent claims related to method of treating patients with A-T using Quince’s proprietary process to encapsulate DSP in autologous red blood cells.
Held an investor webinar in February 2025 featuring key opinion leader (KOL) Dr. Mary Kay Koenig from UTHealth Houston with a discussion focused on addressing the high unmet need in A-T. During the investor webinar, Dr. Koenig 1) provided an A-T natural history overview, 2) detailed current symptomatic treatment approaches for patients with A-T, 3) discussed the competitive therapeutic A-T landscape, and 4) provided an overview of Quince’s Phase 3 NEAT clinical trial of eDSP for the treatment of A-T. A replay of the A-T KOL investor webinar is available here.
Fiscal Year 2024 Operating Highlights

Granted Fast Track designation from the FDA for Quince’s eDSP System for the treatment of patients with A-T in June 2024.
Published efficacy and safety results from the company’s Phase 3 ATTeST clinical trial evaluating eDSP for the treatment of A-T in medical journal The Lancet Neurology.
Participated at notable scientific meetings, including poster presentations at the 53rd Child Neurology Society (CNS) Annual Meeting and the 2024 International Congress for Ataxia Research (ICAR), where Quince presented data from its Phase 3 ATTeST clinical trial.
Completed an initial patient sizing project based on third-party analysis from IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics, which confirmed that the number of diagnosed patients with A-T in the U.S. is estimated to be to approximately 4,600.
Established a SAB comprised of leading experts in biochemistry, neurology, immunology, hematology, pharmacology, and clinical practice uniquely positioned to provide Quince with deep insights and advice to support advancement of the company’s drug programs.
Fiscal Year 2024 Financial Results

Reported cash, cash equivalents, and short-term investments of $40.8 million for the fiscal year ended December 31, 2024. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results and into 2026. The company plans to be opportunistic from a financing perspective to extend its existing cash runway.
Reported research and development (R&D) expenses of $18.6 million for the fiscal year ended December 31, 2024. R&D expenses primarily included costs related to ongoing Phase 3 NEAT clinical trial activities and related manufacturing costs.
Reported general and administrative (G&A) expenses of $17.6 million for the fiscal year ended December 31, 2024. G&A expenses primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.
Reported a net loss of $56.8 million, or a net loss of $1.31 per basic and diluted share, for the fiscal year ended December 31, 2024. Weighted average shares outstanding for the year were 43.3 million.
Reported net cash used in operating activities of $31.9 million for the year ended December 31, 2024. Cash used in operating activities was primarily due to a net loss of $56.8 million for the period, adjusted for $25.4 million of non-cash items, including a $17.1 million goodwill impairment charge, $4.7 million in stock-based compensation, $4.0 million change in the fair value of contingent consideration liabilities, $1.7 million change in the fair value of long-term debt, and a net increase in operating assets of $2.6 million, offset by a net increase in accounts payable, and accrued expenses and other current liabilities of $ 2.1 million.
Made a cash milestone payment of $5.0 million to EryDel shareholders in the third quarter of 2024 following the achievement of the first patient enrolled in the NEAT study in the second quarter of 2024.