On May 9, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL siRNA gene silencing technology is designed to make immune cells more effective in killing tumor cells, reported its Q1 2024 financial results and provided a business update (Press release, Phio Pharmaceuticals, MAY 9, 2024, View Source [SID1234643036]).

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Recent Corporate Updates

· Phio’s INTASYL compound PH-762 is currently being investigated in an open-label Phase 1b clinical study (NCT 06014086) to evaluate the safety and tolerability of intratumoral PH-762 in cutaneous squamous cell (cSCC), melanoma, or Merkel cell carcinoma. Two patients have already completed treatment. Four sites across the US are now engaged in the Phase 1b study. The sites are:
o George Washington University in Washington DC
o Banner MD Anderson in Arizona
o Centricity Research in Ohio
o Integrity Research in Florida.
· Presented new data on the immunotherapeutic activity of INTASYL at:
o 10th Annual Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (ITOC10): this preclinical data demonstrates the potential of the INTASYL compound PH-905 targeting Cbl-b to improve the function of natural killer (NK) cells.
o 27th Annual American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper): this preclinical data demonstrates that intratumoral injection of PH-762 significantly inhibits tumor growth in murine cells and may generate memory-specific T cells.
· The INTASYL siRNA platform is the focus of a National Spotlight on PBS Viewpoint, a national program hosted by Dennis Quaid, and on Fox Business Network; both are airing through May.
· Patent granted by USPTO for two of its INTASYL Compounds, RXI-185 and RXI-231 that treat age-related skin disorders including photo-aging and dermal hyperpigmentation targeting down-regulation of the Matrix metalloproteinase-1(MMP-1) and Tyrosinase (TYR) proteins.

Financial Results

Cash Position

At March 31, 2024, we had cash of $6.5 million as compared with $8.5 million at December 31, 2023.

Research and Development Expenses

Research and development expenses were $1.1 million for the three months ended March 31, 2024 as compared with $2.1 million for the three months ended March 31, 2023, a decrease of 46%. The decrease was primarily driven by the Company’s cost rationalization measures in transitioning from a discovery research company to a product development company resulting in decreased costs for the wind-down of preclinical studies, salary-related costs and lab supplies. Additional decreases in research and development expenses were due to clinical consulting fees incurred in connection with the Company’s IND filing and manufacturing fees for PH-762 in the prior year period.

General and Administrative Expenses

General and administrative expenses were $1.1 million for the three months ended March 31, 2024 as compared with $1.5 million for the three months ended March 31, 2023, a decrease of 28%. The decrease was primarily due to decreases in consulting expenses and legal expenses as compared to the prior year period.

Net Loss

Net loss was $2.2 million for the three months ended March 31, 2024 as compared with $3.6 million for the three months ended March 31, 2023. The decrease in net loss was primarily due to the changes in research and development expenses, as described above.

On May 9, 2024 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported financial results for the first quarter 2024, recent business highlights, and key upcoming milestones for 2024 (Press release, Atara Biotherapeutics, MAY 9, 2024, View Source [SID1234642977]).

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"Our lead CAR T program, ATA3219, is advancing as a promising new therapeutic option for oncology and autoimmune diseases, where it is positioned to benefit from the unique characteristics of our proven allogeneic EBV T-cell platform," said Pascal Touchon, President and Chief Executive Officer of Atara. "This is anticipated to provide multiple near-term clinical milestones for ATA3219, including initial non-Hodgkin’s lymphoma data expected in the fourth quarter 2024, and initial lupus nephritis data in the first half of 2025, with plans to expand into a new SLE cohort without lymphodepletion. In addition, our partnership with Pierre Fabre continues to generate value, as we received our first milestone payment related to tab-cel regulatory progress in the U.S. in April, with the potential for additional near-term payments related to the BLA acceptance and approval."

Tabelecleucel (tab-cel or Ebvallo) for Post-Transplant Lymphoproliferative Disease (PTLD)

Atara plans to submit a Biologics License Application (BLA) in Q2 2024 for tab-cel indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate
The data package for the filing includes pivotal and supportive data covering more than 430 patients treated with tab-cel across multiple life-threatening diseases
The BLA will include the latest pivotal ALLELE study data-cut that demonstrated a statistically significant 48.8% Objective Response Rate (ORR) (p<0.0001) and favorable safety profile consistent with previous analyses
Ebvallo received regulatory approval from Swissmedic, the regulatory authority in Switzerland, further expanding its potential in Europe
Atara continues to advance the Pierre Fabre Laboratories expanded global partnership, which included approximately $27 million in cash upfront at the closing of the deal and the potential to receive up to $640 million in additional payments and significant double-digit tiered royalties on net sales, including up to $100 million in potential regulatory milestones through BLA approval
ATA3219: CD19 Program in Lupus Nephritis (LN)

Atara expects to initiate a Phase 1 study of ATA3219 as a monotherapy for the treatment of systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]) in Q4 2024 with initial clinical data anticipated in H1 2025
The Phase 1 open-label, dose-escalation study is designed to evaluate safety, preliminary efficacy, pharmacokinetics, and biomarkers of a single dose of ATA3219 administered to LN subjects refractory to one or more lines of treatment. Subjects will receive lymphodepletion treatment followed by ATA3219 at a dose of 40, 80, or 160 x 106 CAR+ T cells. Each dose level is designed to enroll 3-6 patients
Atara is positioned to potentially expand ATA3219 Phase 1 study into additional autoimmune indications via the same Investigational New Drug (IND) application previously cleared for the LN study
Preclinical data supporting the potential of ATA3219 in SLE will be presented in poster presentation at the International Society for Cell & Gene Therapy meeting, which is being held May 29-June 1, 2024. The data demonstrate that ATA3219 CAR T cells led to complete CD19-specific B-cell depletion against SLE or multiple sclerosis patient peripheral blood mononuclear cells
Additional preclinical data also presented in the poster show that ATA3219 CAR T cells, which use the next-generation 1XX costimulatory domain, release lower levels of pro-inflammatory cytokines while maintaining cytotoxic function as potent as observed with autologous CAR T controls in response to stimulation with CD19+ target cells. Mitigating inflammatory cytokine release that is typically seen with standard CD19 CAR T signaling may lead to reduced toxicity and better tolerability if confirmed in clinical trials
ATA3219: CD19 Program in Severe Systemic Lupus Erythematosus (SLE) Without Lymphodepletion

Atara plans to expand the Phase 1 LN study of ATA3219 and add a new cohort in severe SLE without lymphodepletion (LD) in Q4 2024 with initial clinical data anticipated in H2 2025
The elimination of LD is designed to further simplify the treatment regimen and to potentially provide a differentiated safety profile to patients without comprising efficacy which may improve patient access
There is compelling clinical and scientific rationale supporting the potential to eliminate the need for LD based on the EBV T-cell backbone and unique features of ATA3219, including: 1) low alloreactivity risk and favorable safety in over 600 patients treated without LD, due to T-cell receptor EBV specificity and partial human leukocyte antigen matching; 2) expansion and persistence data without LD correlating to efficacy in patients treated with tab-cel; and 3) the inclusion of clinically validated features into ATA3219 such as the 1XX costimulatory domain and memory phenotype that increase potency and persistence
ATA3219: CD19 Program in Non-Hodgkin’s Lymphoma (NHL)

Atara initiated enrollment of a multi-center, Phase 1 open-label, dose-escalation clinical trial of ATA3219 in NHL, including large B-cell lymphomas, follicular lymphoma, and mantle cell lymphoma, with initial clinical data anticipated in Q4 2024
Study designed to evaluate safety, preliminary efficacy, pharmacokinetics, and biomarkers. Subjects will receive LD treatment followed by ATA3219 at a dose of 40, 80, 240, or 480 x 106 CAR+ T cells. Each dose level is designed to enroll 3-6 patients
Previously presented preclinical data demonstrated superior in vivo persistence and CD19-specific anti-tumor efficacy compared to an autologous CD19 CAR T benchmark with no observed toxicity or alloreactivity
ATA3431: CD19/CD20 Program for B-Cell Malignancies

Preclinical data presented at ASH (Free ASH Whitepaper) 2023 demonstrated early evidence of potent antitumor activity, long-term persistence, and superior tumor growth inhibition compared to an autologous CD19/CD20 CAR T benchmark
Dual CD19 and CD20 targeting designed to address CD19 escape and tumor variability and may provide additional efficacy in lymphoma
Atara is progressing toward an IND submission in H2 2025
First Quarter 2024 Financial Results

Cash, cash equivalents and short-term investments as of March 31, 2024 totaled $46.2 million, as compared to $51.7 million as of December 31, 2023
Q1 2024 accounts receivable totaled $35.8 million and include a $20 million milestone payment related to the positive tab-cel pre-BLA meeting and approximately $12 million for the reimbursement of tab-cel global development costs from Pierre Fabre. The $20 million milestone payment was received in April 2024 and the approximate $12 million payment is expected to be received in May 2024
Together, cash, cash equivalents, short-term investments, and accounts receivable as of March 31, 2024 totaled $82.1 million
Net cash used in operating activities was $29.6 million for the first quarter 2024, as compared to $38.4 million in the same period in 2023
Q1 2024 net cash used in operating activities included 2023 annual employee bonus payments and cash disbursements related to the November 2023 and January 2024 reductions in force of approximately $13 million in aggregate
Total revenues were $27.4 million for the first quarter 2024, as compared to $1.2 million for the same period in 2023. Total revenues increased by $26.2 million year over year, primarily due to revenue recognized as a result of additional obligations for the expanded partnership with Pierre Fabre and accelerated recognition of existing deferred revenue due to the planned transition of substantially all activities relating to tab-cel at the time of BLA approval and transfer to Pierre Fabre
Total costs and operating expenses include non-cash stock-based compensation, depreciation and amortization expenses of $9.8 million for the first quarter 2024, as compared to $13.0 million for the same period in 2023
Total costs and operating expenses include restructuring expense of $4.8 million for the first quarter 2024 related to the reduction in force Atara announced in January 2024, and which reduced its headcount at that time by approximately 25% to 170 employees
Research and development expenses were $45.5 million for the first quarter 2024, as compared to $62.2 million for the same period in 2023
Research and development expenses include $4.7 million of non-cash stock-based compensation expenses for the first quarter 2024, as compared to $6.8 million for the same period in 2023
General and administrative expenses were $11.1 million for the first quarter 2024, as compared to $13.9 million for the same period in 2023
General and administrative expenses include $3.7 million of non-cash stock-based compensation expenses for the first quarter 2024, as compared to $5.0 million for the same period in 2023
Atara reported net losses of $31.8 million, or $0.23 per share, for the first quarter 2024, as compared to $74.8 million, or $0.72 per share, for the same period in 2023
Outlook and Cash Runway

Atara expects full year 2024 operating expenses to decrease by approximately 35% from 2023
The large majority of the year-over-year operating expense reduction is expected to begin in Q2 2024 and continue for the remainder of the year
Atara expects that cash, cash equivalents, short-term investments, and accounts receivable as of March 31, 2024, plus the items noted below, in total will enable funding of planned operations into 2027:
anticipated payments of $20 million and $60 million from Pierre Fabre contingent upon the successful acceptance and approval of the tab-cel BLA, respectively;
anticipated purchases of tab-cel inventory through the manufacturing transfer date by Pierre Fabre;
anticipated reimbursement for tab-cel global development costs through the BLA transfer by Pierre Fabre;
operating efficiencies resulting from completed workforce reductions;
the planned transition of substantially all activities relating to tab-cel at the time of the BLA transfer to Pierre Fabre potentially as early as Q1 2025, which will further reduce quarterly operating expenses; and
anticipated royalties from sales of tab-cel by Pierre Fabre in the U.S. post BLA approval
About ATA3219

ATA3219 combines the natural biology of unedited T cells with the benefits of an allogeneic therapy. It consists of allogeneic Epstein-Barr virus (EBV)-sensitized T cells that express a CD19 CAR construct for the treatment of CD19+ relapsed or refractory B-cell malignancies, including B-cell non-Hodgkin’s lymphoma and B-cell mediated autoimmune diseases including systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]). ATA3219 has been optimized to offer a potential best-in-class profile, featuring off-the-shelf availability. It incorporates multiple clinically validated technologies including a modified CD3ζ signaling domain (1XX) that optimizes expansion and mitigates exhaustion, enrichment during manufacturing for a less differentiated phenotype for robust expansion and persistence and retains the endogenous T-cell receptor without gene editing as a key survival signal for T cells contributing to persistence.

About ATA3431

ATA3431 is an allogeneic, bispecific CAR directed against CD20 and CD19, built on Atara’s EBV T-cell platform. The design consists of a tandem CD20-CD19 design, with binders oriented to optimize potency. Dual targets address the limitations of single antigen loss and tumor variability. ATA3431 features a novel 1XX costimulatory domain, memory phenotype, and retained, unedited T-cell receptor. Preclinical data have demonstrated early evidence of antitumor activity, long-term persistence, and superior tumor growth inhibition compared to an autologous CD19/CD20 CAR T benchmark.

Next-Generation Allogeneic CAR T Approach

Atara is focused on applying Epstein-Barr virus (EBV) T-cell biology, featuring experience in over 600 patients treated with allogeneic EBV T cells, and novel chimeric antigen receptor (CAR) technologies to meet the current limitations of autologous and allogeneic CAR therapies head-on by advancing a potential best-in-class CAR T pipeline in oncology and autoimmune disease. Unlike gene-edited approaches aimed at inactivating T-cell receptor (TCR) function to reduce the risk for graft-vs-host disease, Atara’s allogeneic platform maintains expression of the native EBV TCR that promote in vivo functional persistence while also demonstrating inherently low alloreactivity due to their recognition of defined viral antigens and partial human leukocyte antigen (HLA) matching. A molecular toolkit of clinically-validated technologies—including the 1XX costimulatory domain designed for better cell fitness and less exhaustion while maintaining stemness—offers a differentiated approach to addressing significant unmet need with the next generation CAR T.

On May 9, 2024 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported financial results for the first quarter ended March 31, 2024 and recent corporate highlights (Press release, In8bio, MAY 9, 2024, View Source [SID1234642993]).

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"We continued to make significant progress advancing our gamma-delta T cell programs in the first quarter of 2024," said William Ho, CEO and co-founder of IN8bio. "We presented new preclinical data on our nsCAR platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024 Annual Meeting demonstrating its potential to target and kill various acute myeloid leukemia (AML) cells by targeting CD33 and/or CD123, while preserving healthy bone marrow cells. These findings reinforce our technology’s ability to precisely target "undruggable" cancer targets that have historically been challenging due to on-target, off-tumor toxicity. We will provide an update from our Phase 1 study of INB-100 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting in June, including patient status and survival rate data. We anticipate enrolling ten additional patients in an expansion cohort at the recommended Phase 2 dose, and could potentially submit an investigational new drug (IND) application for a Phase 2 randomized control trial this year. In addition, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, we will provide an update on our Phase 1 INB-200 study in GBM which generated an initial efficacy signal supporting the INB-400 trial."

Corporate Highlights and Recent Developments

Presented data at AACR (Free AACR Whitepaper) 2024, supporting the potential of proprietary constructs targeting CD33 and/or CD123 for in vitro evaluation against various types of leukemia, including AML and chronic myeloid leukemia (CML).
Demonstrated significant differences between cells expressing traditional signaling chimeric antigen receptors (CARs) and those expressing nsCAR constructs, which include a reduction in activation-induced cell death with nsCAR constructs.
Peer-reviewed publication of "Adoptive Cell Therapy for High Grade Gliomas using Simultaneous Temozolomide and Intracranial MGMT-Modified γδ T cells Following Standard Post-Resection Chemotherapy and Radiotherapy: Current Strategy and Future Directions" in Frontiers in Immunology detailing IN8bio’s DeltEx Drug Resistant Immunotherapy (DRI) as a rational therapeutic approach for newly diagnosed GBM.
Announced first patient dosed in the Phase 2 autologous arm of INB-400 in patients with newly diagnosed GBM.
Upcoming Anticipated Pipeline Milestones and Events

American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting (May 10, 2024): Upcoming oral presentation: "Healthy Donor vs Patient Manufactured Autologous DeltEx DRI Product; Immunophenotyping Gene Expression," will unveil new data highlighting the characterization of our clinical manufactured DeltEx DRI product. The presentation will explore the impact of manufacturing on the final cell product from healthy donors and those manufactured from cancer patients, showcasing IN8bio’s robust capabilities and know-how in complex cell therapy process development and manufacturing.
INB-100: Report updated interim results from the ongoing Phase 1 investigator-sponsored trial at the 2024 EHA (Free EHA Whitepaper) Annual Meeting, held June 13-16 in Madrid, Spain. In addition, we will potentially submit an IND application for a Phase 2 registrational trial in 2024 in the AML and myelodysplastic syndrome (MDS) patient setting.
INB-200: Report interim Phase 1 long-term follow up results in GBM at multiple medical meetings in 2024 including at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.
INB-400: Initiated patient dosing in the Phase 2 autologous arm of INB-400 in newly diagnosed GBM. IN8bio expects to treat up to a total of 40 patients in arm A at multiple sites across the United States.
First Quarter 2024 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $4.9 million, compared to $4.4 million for the comparable prior year period. The increase was primarily due to (i) increased personnel-related costs, including salaries and stock-based compensation due to increased headcount and (ii) direct clinical costs for INB-100, INB-200 and INB-400.
General and administrative expenses: General and administrative expenses were $3.7 million, compared to $3.5 million for the comparable prior year period. The increase was primarily due to increased personnel-related costs, including stock-based compensation and rent, offset by cost savings related to directors’ and officers’ insurance premiums and a reduction in professional services.
Net loss: Net loss was $8.6 million, or $0.20 per basic and diluted common share, compared to a net loss of $7.5 million, or $0.30 per basic and diluted common share, for the comparable prior year period.
Cash position: As of March 31, 2024, the Company had cash of $13.0 million, compared to $21.3 million, as of December 31, 2023.

On May 9, 2024 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the first quarter ended March 31, 2024 (Press release, Nektar Therapeutics, MAY 9, 2024, View Source [SID1234643009]).

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Cash and investments in marketable securities at March 31, 2024 were $326.0 million as compared to $329.4 million at December 31, 2023. Nektar’s cash and marketable securities are expected to support strategic development activities and operations into the third quarter of 2026.

"In the first quarter, we made significant progress with our highly promising immunology and inflammation pipeline," said Howard W. Robin, President and CEO of Nektar. "REZPEG is advancing in the clinic in our Phase 2b study in patients with atopic dermatitis and in our Phase 2b study in patients with alopecia areata. Enrollment for both studies is on-track, and we expect to report topline data from these trials in the first half of 2025. Building out our Treg pipeline, our novel bivalent antibody targeting the TNFR2 receptor is progressing through IND-enabling studies to support entering the clinic next year."

Summary of Financial Results

Revenue in the first quarter of 2024 was $21.6 million as compared to the same $21.6 million in the first quarter of 2023.

Total operating costs and expenses in the first quarter of 2024 were $57.1 million as compared to $156.3 million in the first quarter of 2023. Operating costs and expenses for the first quarter of 2023 included a one-time $76.5 million non-cash goodwill impairment charge. Operating costs and expenses for the first quarter of 2024 further decreased as compared to 2023 due to decreases in restructuring, impairment and costs of terminated program, as well as decreases in R&D and G&A expense.

R&D expense in the first quarter of 2024 was $27.4 million as compared to $30.5 million for the first quarter of 2023. R&D expense for the first quarter of 2024 decreased primarily due to a decrease in employee costs and related facilities costs, partially offset by an increase in expense for the development of rezpegaldesleukin and NKTR-0165, our TNFR2 agonist antibody.

G&A expense was $20.1 million in the first quarter of 2024 as compared to $21.1 million in the first quarter of 2023.

Restructuring, impairment and other costs of the terminated program were $1.0 million in the first quarter of 2024 as compared to $21.2 million in the first quarter of 2023. Restructuring, impairment and other costs of terminated program decreased primarily due to $13.2 million in non-cash lease and equipment impairment charges and $5.5 million in severance expense recognized in the first quarter of 2023.

Net loss for the first quarter of 2024 was $36.8 million or $0.19 basic and diluted loss per share as compared to a net loss of $137.0 million or $0.73 basic and diluted loss per share in the first quarter of 2023.

First Quarter 2024 and Recent Business Highlights

● In March 2024, Nektar initiated a Phase 2b study of rezpegaldesleukin in patients with severe-to-very severe alopecia areata. The Company expects topline data from this study in the first half of 2025.

● Enrollment is ongoing in the Phase 2b study of rezpegaldesleukin in patients with moderate-to-severe atopic dermatitis. The Company expects topline data from this study in the first half of 2025.

● In March 2024, we entered into a securities purchase agreement with TCG Crossover Fund, an institutional accredited investor, to sell securities in a private placement financing for gross proceeds to the Company of approximately $30 million, before deducting expenses.

Conference Call to Discuss First Quarter 2024 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, May 9, 2024.

This press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the Home Page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through June 9, 2024.

To access the conference call, follow these instructions:

Dial: (800) 715-9871 (U.S & Canada)

Conference ID: 4855448

On May 9, 2024 Kite, a Gilead Company (NASDAQ: GILD), and Arcellx, Inc. (NASDAQ: ACLX) reported several key operational updates on their partnered anitocabtagene autoleucel (anito-cel) multiple myeloma program (Press release, Gilead Sciences, MAY 9, 2024, View Source [SID1234643040]). Anito-cel is the first BCMA CAR T to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact D-Domain binder.

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The companies shared the design of a global, Phase 3 randomized controlled clinical trial, iMMagine-3, which Kite expects to start in the second half of this year. The trial will compare the efficacy and safety of anito-cel randomized against the standard of care (SOC) in patients with relapsed and/or refractory multiple myeloma (rrMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

Kite’s facility in Frederick, Maryland will manufacture anito-cel for this trial. This follows the completion of the technical transfer from a third-party contract manufacturing organization to Kite, as well as the transfer of the Investigational New Drug (IND) application for anito-cel, which has been cleared by the U.S. Food and Drug Administration.

"We are pleased to start the Phase 3 pivotal trial, iMMagine-3, in the second half of this year given the tremendous unmet need that remains in patients with relapsed and/or refractory multiple myeloma," said Cindy Perettie, Executive Vice President, Kite. "As we prepare for this pivotal program, we look forward to leveraging our manufacturing expertise to further position anito-cel as a potential best-in-class cell therapy. We know manufacturing quality, reliability and speed are critically important as every day matters for these patients."

"Our global iMMagine-3 trial will evaluate anito-cel as a second through fourth line treatment in patients with multiple myeloma who were previously exposed to both an immunomodulatory drug and an anti-CD38 monoclonal antibody," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "The iMMagine-3 study allows us to maximize the impact of anito-cel as it captures what will become the largest second line patient population based on the current treatment paradigm, as anti-CD38 therapies move to front line treatment. This population represents an emerging significant unmet clinical need allowing us to provide access to a unique patient population. In addition, the completion of the technical transfer to Kite allowed us to accelerate our development program and launch iMMagine-3 globally, which will enable broader and earlier patient access to anito-cel."

About iMMagine-3 Global Phase 3 Randomized Controlled Clinical Trial

iMMagine-3 is a phase 3, randomized controlled trial designed to compare the efficacy and safety of anitocabtagene autoleucel (anito-cel) with SOC in patients with relapsed and/or refractory multiple myeloma (rrMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

iMMagine-3 will enroll approximately 450 adult patients. Prior to randomization, investigator’s choice of SOC regimens include: pomalidomide, bortezomib, and dexamethasone (PVd); daratumumab, pomalidomide, and dexamethasone (DPd); carfilzomib, daratumumab and dexamethasone (KDd); or carfilzomib and dexamethasone (Kd). Patients in the anito-cel arm will undergo leukapheresis and optional bridging therapy (with the SOC regimen selected by the investigator prior to randomization) followed by lymphodepleting chemotherapy (fludarabine 30 mg/m2/d and cyclophosphamide 300 mg/m2/d for 3 days) and one infusion of anito-cel (115×106 CAR+ T cells) on Day 1.

The primary endpoint is progression free survival (PFS) per blinded independent review according to the 2016 IMWG uniform response criteria for MM with the hypothesis that anito-cel will prolong PFS compared to SOC. Key secondary endpoints include complete response rate (CR/sCR), minimal residual disease negativity, overall survival, and safety.

The iMMagine-3 trial is expected to initiate in the second half of 2024 at ~130 study sites across North America, Europe, and rest of world.