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.

On March 24, 2025 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, reported financial results for the fourth quarter and year ended December 31, 2024, and provided recent business highlights (Press release, Aura Biosciences, MAR 24, 2025, View Source [SID1234651395]).

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"We believe that bel-sar has the potential to transform the treatment paradigm in multiple rare oncology indications starting with ocular cancers and more broadly across many solid tumors such as bladder cancer. The data presented from the Phase 2 trial in early-stage choroidal melanoma and the Phase 1 trial in NMIBC demonstrated bel-sar’s potential as a front-line treatment option across multiple tumor types," said Elisabet de los Pinos, Chief Executive Officer of Aura Biosciences. "These data highlight the potential clinical benefit of a novel dual mechanism of action driven by highly targeted cytotoxicity and robust cell-mediated immunity. We look forward to continuing to advance our pipeline across multiple indications with high unmet patient need."

Recent Pipeline Developments

Early-Stage Choroidal Melanoma

Early-stage choroidal melanoma represents an area of high unmet need with no drugs approved. The Company previously received Orphan Drug Designation from the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and Fast Track designation from the FDA for the treatment of early-stage choroidal melanoma. The CoMpass trial is under a Special Protocol Assessment (SPA) agreement with the FDA.

Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma. The study is a global, Phase 3, randomized trial evaluating bel-sar treatment against a sham control arm and includes an enrichment strategy to enroll approximately 100 patients with documented tumor growth.

The CoMpass trial is actively enrolling globally. To identify appropriate patients to meet the enrichment strategy of documented growth, the Company has enabled a pre-screening ‘run in’ period. Globally, since June 2024, investigators have registered over 175 patients in pre-screening as having met initial enrollment criteria for the study. The acceleration in pre-screening is driven by increasing momentum in the United States and European Union.

Additional Ocular Oncology Indications:

In addition to early-stage choroidal melanoma, bel-sar is being explored for metastases to the choroid and cancers of the ocular surface. These three ocular oncology indications have a collective incidence of greater than 60,000 patients annually in the United States and Europe.

Metastases to the Choroid

The Company has initiated a Phase 2 clinical trial in metastases to the choroid and has sites activated with patients in prescreening. Metastases to the choroid is an indication with high unmet medical need and no approved therapies. The Company expects initial data from this trial in 2025.

Metastases to the choroid represents the second potential ocular oncology indication for bel-sar, affecting approximately 20,000 patients annually in the United States and Europe. The Company previously received FDA Fast Track designation for bel-sar in this indication.

Cancers of the Ocular Surface

The Company’s third potential ocular oncology indication is cancers of the ocular surface, which affects approximately 35,000 patients in the United States and Europe annually. The Company continues to advance its preclinical work designed to be IND-enabling in cancers of the ocular surface.

Bladder Cancer

Positive additional data from the Company’s Phase 1 trial of bel-sar in patients with NMIBC was presented at the 40th Annual European Association of Urology Congress. In totality, the data demonstrate clinical complete responses as well as robust cell mediated immunity across the intermediate- and high-risk disease spectrum.

These data now include the histopathological assessment of all 10 patients after treatment with light activation and the full evaluation of safety and tumor response in two additional patients with high-risk NMIBC. One patient with high-risk disease (due to BCG failure) demonstrated a clinical complete response. Further, to evaluate the local immune response after the treatment with bel-sar in the TME, multiplex immunofluorescence staining for key immune cell types was performed on tumor biopsies from three patients. These early observations show induction of effector immunity and the development of local active immunosurveillance, highlighting key features of bel-sar’s dual mechanism of action and the potential to translate into durable treatment responses. The Company previously announced early data from this Phase 1 trial in October 2024. Details of the updated results of the Phase 1 NMIBC trial can be found here.

Corporate Updates

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The Company will host a virtual urologic oncology investor event today, at 4:30 pm ET, featuring Neal Shore, MD, FACS (Carolina Urologic Research Center), Gary Steinberg, MD, FACS (Rush University) and Jennifer A. Linehan, MD (Saint John’s Cancer Institute), to discuss the data from the Phase 1 trial in NMIBC, as well as a bladder cancer program update including the Phase 1b/2 trial and future development plans. A replay of the webcast will be available following the event on the "Investors & Media" page under the "Events & Presentations" section of Aura’s website at View Source

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Tony Gibney joined the Company as Senior Finance and Strategy Advisor. Mr. Gibney is an experienced biotechnology leader and former investment banker who brings over 30 years of experience dedicated to advising biotechnology companies in the United States and Europe across their businesses, including corporate strategy, business development, finance and investor relations, among many others. Following his investment banking career, he has worked as Chief Business Officer at Achillion Pharmaceuticals, Inc. and Iveric Bio, Inc. and as Chief Business and Financial Officer at Fog Pharmaceuticals, Inc.

Full Year and Fourth Quarter 2024 Financial Results

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As of December 31, 2024, Aura had cash and cash equivalents and marketable securities totaling $151.1 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the second half of 2026.

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Research and development expenses increased to $22.3 million and $73.3 million for the three months and full year ended December 31, 2024, respectively, from $20.3 million and $65.2 million for the three months and full year ended December 31, 2023, respectively, primarily due to ongoing clinical and contract research organization costs associated with the progression of the Company’s Phase 3 global trial and manufacturing and development costs for bel-sar.

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General and administrative expenses increased to $5.5 million and $22.8 million for the three months and full year ended December 31, 2024, respectively, from $4.5 million and $19.8 million for the three months and full year ended December 31, 2023, respectively. General and administrative expenses include $1.4 million and $1.2 million of stock-based compensation for the three months ended December 31, 2024 and 2023, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to the global growth of the Company.

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Net loss for the three months and full year ended December 31, 2024, was $25.8 million and $86.9 million, respectively, compared to $22.1 million and $76.4 million for the three months and full year ended December 31, 2023, respectively.

On March 24, 2025 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, reported financial results for the full year ended December 31, 2024, and highlighted recent progress (Press release, Artiva Biotherapeutics, MAR 24, 2025, View Source [SID1234651394]).

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"2024 was a transformational year for Artiva including initiating dosing of AlloNK in patients with autoimmune disease across our trials, a successful initial public offering strengthening our balance sheet, and expanding key leadership across the organization with expertise in cell therapy and autoimmune disease," said Fred Aslan, M.D., CEO of Artiva. "We look forward to sharing initial data from our AlloNK program in autoimmune disease this year. We also look forward to sharing updated clinical data from our non-Hodgkin’s lymphoma (NHL) trial with AlloNK which continues to mature as one of the strongest data sets for the allogeneic field, demonstrating deep B-cell depletion, continued durability of response, and the compatibility of our treatment regimen with outpatient administration."

Recent Business Highlights

Corporate and Financial Updates

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Expanded Board of Directors: In January 2025, Artiva appointed Dan Baker, M.D., as an independent member of its Board of Directors. Dr. Baker brings over two decades of drug development experience in the pharmaceutical industry. He is currently the interim Chief Development Officer of Cue Biopharma, Inc., and previously held a 19-year tenure at Johnson & Johnson (Janssen/Centocor) most recently as the Vice President of Immunology R&D.
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Bolstered Key Development Leadership: Artiva appointed key leadership with cell therapy and autoimmune expertise across the development organization, including Benjamin Dewees as Senior Vice President (SVP), Regulatory Affairs, David Moriarty, Ph.D., as SVP, Clinical Operations, and Feng Xu as SVP, Biometrics. Collectively, the leadership team brings experience developing therapies targeting autoimmune indications including systemic lupus erythematosus (SLE), lupus nephritis (LN), rheumatoid arthritis (RA) and Sjogren’s disease from their tenures at companies such as Kyverna Therapeutics, Inc., Horizon Therapeutics plc and IGM Biosciences, Inc.
Upcoming Milestones

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Initial data for AlloNK (also known as AB-101) on autoimmune indications from at least one of the following trials expected in H1 2025:
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Artiva Sponsored Trial in SLE/ LN: Ongoing Phase 1/1b trial evaluating AlloNK in combination with rituximab or obinutuzumab in patients with SLE with or without LN.
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Ongoing IIT Basket Trial: Investigator-initiated basket trial (IIT) assessing the safety, tolerability, and clinical activity of AlloNK plus rituximab in patients with RA, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists, a community rheumatology clinic.
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Updated clinical data from the Phase 1/2 trial exploring AlloNK + rituximab in patients with relapsed/refractory B-cell NHL showing continued durability of response to be presented at a medical conference in 2025

Full Year 2024 Financial Results

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Cash, Cash Equivalents and Investments. As of December 31, 2024, Artiva had cash, cash equivalents, and investments of $185.4 million. This includes $179.0 million in gross proceeds from Artiva’s completed initial public offering in July 2024 in which it sold 14,920,000 shares of its common stock, including partial exercise of the overallotment option. Existing cash, cash equivalents, and investments as of December 31, 2024, are expected to fund operations at least through the end of 2026.
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Collaboration Revenue. Collaboration revenue was zero for the year ended December 31, 2024, compared to $32.9 million for the year ended December 31, 2023.
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Research and Development Expenses. Research and development expenses were $50.3 million for each of the years ended December 31, 2024 and 2023.
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General and Administrative Expenses. General and administrative expenses were $17.2 million for the year ended December 31, 2024, compared to $13.9 million for the year ended December 31, 2023.
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Other Income, net. Other income, net, was $1.9 million for the year ended December 31, 2024, compared to other income, net, of $2.0 million for the year ended December 31, 2023.
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Net Loss. Net loss totaled $65.4 million for the year ended December 31, 2024, as compared to net income of $28.7 million for the year ended December 31, 2023, with non-cash stock-based compensation expense of $7.0 million and $7.1 million for the years ended December 31, 2024 and 2023, respectively.

On March 24, 2025 Agilent Technologies Inc. (NYSE: A) reported its role in supporting Autolus Therapeutics’ FDA approval for AUCATZYL, a recently approved CAR T therapy. Agilent’s advanced cell analysis instrumentation, specifically the xCELLigence Real-Time Cell Analysis (RTCA) technology, was applied in developing and validating AUCATZYL. The xCELLigence RTCA technology supported the development and implementation of the potency assay (Press release, Agilent, MAR 24, 2025, View Source [SID1234651384]). By providing precise and reliable cell analysis solutions, Agilent assisted Autolus in achieving the high standards required for FDA approval.

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Dr. Xiaobo Wang, Vice President and General Manager of the Cell Function and Phenotyping Business at Agilent stated, "Agilent is honored to have contributed to this significant milestone in cancer treatment. The collaboration between Agilent and Autolus highlights the importance of innovative technologies and cooperative efforts in advancing cancer therapies."

David Brochu, Chief Technical Officer at Autolus Therapeutics added, "Agilent’s support was invaluable in our journey to FDA approval. Agilent’s state-of-the-art xCELLigence RTCA technology facilitated the accuracy, reliability and precision of our potency analytical procedure."

The xCELLigence Real-Time Cell Analysis (RTCA) technology is a cutting-edge platform that continuously monitors cell behavior in real-time without using labels or dyes. This technology measures electrical impedance to provide dynamic information on cell numbers, viability, and morphology. Unlike traditional endpoint assays, xCELLigence RTCA offers real-time insights, enabling more accurate and timely decision-making in drug development. Its ability to deliver continuous, real-time data sets it apart in the market, providing researchers with a deeper understanding of cellular responses and enhancing the development of effective therapies.

Agilent and Autolus will attend the 2025 Hybrid US Bioassay Conference from March 24-25 in Tucson, Arizona. Mike Merges, Vice President of Analytical Development, QC Control Operations, Automation, and CMC (Chemistry, Manufacturing, and Controls) at Autolus, along with Jakub Dragun, Quality Control Consultant at Autolus, and Xiaobo Wang, VP/GM of the Cell Function and Phenotyping Business at Agilent, will present a Main Session Podium Talk titled: ‘Lessons Learned in CAR T Cell Product Potency Measurement: A Cross-Functional Investigation and the Importance of Partnership.’ The talk will be held Monday, March 24, from 1:30-2:00 PM in the Sabino Room at the Marriott Tucson University Park Hotel and is open to all conference attendees.

Agilent is committed to advancing scientific innovation and supporting groundbreaking therapies that enhance health outcomes. To learn more about Agilent’s real-time cell analysis solutions, visit Agilent Cell Analysis.

On March 24, 2025 CEL-SCI Corporation (NYSE American: CVM) reported new data has been published from its prior Phase 3 study of Multikine* (Leukocyte Interleukin, Injection) in newly diagnosed, treatment naïve, resectable, locally advanced head and neck cancer patients in the highly regarded peer reviewed journal Pathology and Oncology Research (POR) (Press release, Cel-Sci, MAR 24, 2025, View Source [SID1234651383]). The article titled "Neoadjuvant Leukocyte Interleukin Injection Immunotherapy Improves Overall Survival in Low-risk Locally Advanced Head and Neck Squamous Cell Carcinoma -The IT-MATTERS Study" included a comprehensive presentation of results from CEL-SCI’s Phase 3 trial, the largest study ever conducted for newly diagnosed locally advanced head and neck cancer.

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"We are pleased that the wealth of data resulting from our completed Phase 3 study is now published in this international oncology journal," stated CEL-SCI’s Chief Scientific Officer, Dr. Eyal Talor. "We believe the marked improvement in quality of life offered by Multikine neoadjuvant treatment appeared to have had a positive impact on patients’ quality of life, and in addition to its favorable safety profile, tolerability and efficacy, it is likely to improve adoption rates for Multikine following regulatory approval."

The new, previously unpublished findings included the following patient quality of life data:

Quality of life (QoL) was assessed and validated through use of two instruments, EORTC QLQ-C30 and EORTC QLQ-H&N 35 across all clinical sites.
EORTC QLQ-C30 is a 30-item questionnaire developed by the European Organisation for Research and Treatment of Cancer (EORTC) to assess the health-related QoL of cancer patients.
EORTC QLQ-H&N 35 is a questionnaire designed to assess the QoL of head and neck cancer patients in conjunction with the general cancer-specific EORTC QLQ-C30.
QoL variables were assessed at baseline, before and after the Multikine treatment, and periodically during long-term follow up. These assessments included questions regarding pain in the mouth, jaw, and throat, problems swallowing, sense of smell and taste, ability for selfcare and mobility including walking, using the toilet, shortness of breath, emotional wellbeing including irritability and depression, and many other daily health assessment factors.
As a neoadjuvant therapy, patients were treated with Multikine before surgery. Pre-surgery objective early response to treatment with Multikine was confirmed by pathology at surgery. There were 45 objective early responders (which included 5 complete responders following 3-weeks of Multikine treatment) in the Multikine treated + standard of care group and zero (none reported by investigators) in the control group, which received the standard of care treatments only (i.e., surgery plus radiotherapy or surgery plus chemoradiotherapy; with cisplatin as the chemotherapeutic agent per the study protocol and NCCN Guidelines).
95.1% of complete responders to Multikine reported improved QoL
Complete responders reported a 100% (wherein all respondents scored the highest possible improvement from baseline) on 60% (39/65) of the QoL measures assessed including sleep, appetite, pain, emotional state, condition of mouth, sense of smell and taste, and social, family and public interactions.
QoL results for complete responders were measured and sustained for over 3 years following treatment with Multikine.
89.4% of partial responders to Multikine (those exhibiting greater than 30% reduction in tumor – confirmed by pathology at surgery) also reported improved assessed QoL measures from baseline.
About the Completed Phase 3 and Upcoming Confirmatory Registration Study

Based on the exceptional efficacy results and favorable safety profile for Multikine in a cohort of patients in the Phase 3 study, the U.S. Food and Drug Administration has given CEL-SCI the go ahead to initiate a confirmatory Registration Study of Multikine in newly diagnosed, previously untreated resectable stage 3 and 4 head and neck cancer patients who had no lymph node involvement and low PD-L1 tumor expression. There is a high unmet need in this patient population, for which no advancement in overall survival has been forthcoming in decades, despite many previous attempts by others.

Upon the Registration Study achieving full enrollment, CEL-SCI plans to seek early approval based on early tumor responses which were shown to correspond with survival rates. During the completed Phase 3 clinical trial, the 5-year overall survival rate of the target patient population (disease stage 3 and 4 patients who had no lymph node involvement and low PD-L1 tumor expression), which is the same population that will participate in the confirmatory study, increased to 73% when patients were treated with Multikine vs 45% for control patients who received only standard of care treatments.

About Pathology and Oncology Research (POR)

A Switzerland-based highly regarded international journal, POR is dedicated to keeping scientists informed of developments in its focused biomedical fields which span the gap between basic research and clinical medicine.