On May 9, 2024 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer, reported financial results for the first quarter ended March 31, 2024 and provided a business update (Press release, Kezar Life Sciences, MAY 9, 2024, View Source [SID1234642997]).

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"We continued to make meaningful progress this past quarter on our mission to develop first-in-class small molecule therapeutics in immunology and oncology. We are focused this year on clinical execution in our PALIZADE and PORTOLA trials and are excited by the strong enrollment activity we have seen to date." said Chris Kirk, Kezar’s Co-Founder and Chief Executive Officer. "We are also looking forward to sharing initial results from the dose escalation and dose expansion portions of the KZR-261 study in the fourth quarter of this year."

Zetomipzomib: Selective Immunoproteasome Inhibitor

PALIZADE – Phase 2b clinical trial of zetomipzomib in patients with active lupus nephritis (LN) (ClinicalTrials.gov: NCT05781750)

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PALIZADE is a global, placebo-controlled, randomized, double-blind Phase 2b clinical trial evaluating the efficacy and safety of two dose-levels of zetomipzomib in patients with active LN. Target enrollment will be 279 patients, randomly assigned (1:1:1) to receive 30 mg of zetomipzomib, 60 mg of zetomipzomib or placebo subcutaneously once weekly for 52 weeks, in addition to standard background therapy. Background therapy can, but will not be mandated to, include standard induction therapy. Over the initial 16 weeks, there will be a mandatory corticosteroid taper to 5 mg per day or less. End-of-treatment assessments will occur at Week 53. The primary efficacy endpoint is the proportion of patients who achieve a complete renal response (CRR) at Week 37, including a urine protein-to-creatine ratio (UPCR) of 0.5 or less without receiving rescue or prohibited medications.
PORTOLA – Phase 2a clinical trial of zetomipzomib in patients with autoimmune hepatitis (AIH) who have not benefited from standard-of-care treatment (ClinicalTrials.gov: NCT05569759)

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PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or have relapsed. Target enrollment will be 24 patients, randomized (2:1) to receive 60 mg of zetomipzomib or placebo in addition to background corticosteroid therapy for 24 weeks, with a protocol-mandated steroid taper by Week 14. The primary efficacy endpoint will measure the proportion of patients who achieve a complete response measured as normalization of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels with a successful corticosteroid taper by Week 24.
KZR-261: Broad-Spectrum Sec61 Translocon Inhibitor

KZR-261-101 – Phase 1 clinical trial of KZR-261 in patients with locally advanced or metastatic solid malignancies (ClinicalTrials.gov: NCT05047536)

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The Phase 1 clinical trial of KZR-261 is being conducted in two parts: dose escalation and dose expansion in tumor-specific solid tumors. The study is designed to evaluate safety and tolerability, pharmacokinetics and pharmacodynamics, identify a recommended Phase 2 dose and to explore the preliminary anti-tumor activity of KZR-261 in patients with locally advanced or metastatic disease.
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The dose escalation part of the trial is completing Cohort 9 and reached a maximum tolerated dose of 80mg/m2 based on the presence of reversible neutropenia. A total of 43 patients have been enrolled in the dose escalation part of the trial to date and additional backfill patients may be added to better understand the safety, pharmacokinetics, and pharmacodynamics of KZR-261 at specific dose levels.
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We are currently enrolling the dose expansion part of the study with a cohort of melanoma patients at a dose level of 60 mg/m2. To date, KZR-261 has shown dose-proportional exposure and no signs of accumulation or altered pharmacokinetics with repeated dosing.
Financial Results

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Cash, cash equivalents and marketable securities totaled $179.8 million as of March 31, 2024, compared to $201.4 million as of December 31, 2023. The decrease was primarily attributable to cash used in operations to advance clinical-stage programs.
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Research and development (R&D) expenses for the first quarter of 2024 decreased by $1.1 million to $17.2 million, compared to $18.3 million in the first quarter of 2023. This decrease was primarily due to the Company’s strategic restructuring in October 2023 to prioritize its clinical-stage programs, reducing personnel-related costs and spending in its early-stage research activities. The decrease was partially offset by the increased clinical trial costs related to the PALIZADE and PORTOLA trials, as well as manufacturing expenses.
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General and administrative (G&A) expenses for the first quarter of 2024 increased by $0.3 million to $6.5 million compared to $6.2 million in the first quarter of 2023. The increase was primarily due to an increase in non-cash stock-based compensation and personnel-related expenses, offset by a decrease in legal and professional service expenses.
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Net loss for the first quarter of 2024 was $21.7 million, or $0.30 per basic and diluted common share, compared to a net loss of $22.2 million, or $0.31 per basic and diluted common share, for the first quarter of 2023.

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Total shares of common stock outstanding were 72.8 million shares as of March 31, 2024. Additionally, there were options to purchase 15.6 million shares of common stock at a weighted-average exercise price of $2.27 per share and 0.2 million restricted stock units outstanding as of March 31, 2024.

On May 9, 2024 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported first quarter 2024 financial results and corporate updates (Press release, Iovance Biotherapeutics, MAY 9, 2024, View Source [SID1234642996]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "The first quarter of 2024 was transformative for Iovance following our first FDA approval and our strong start for the U.S. commercial launch of Amtagvi for patients with advanced melanoma. Immediate demand for Amtagvi is very high and continues to significantly increase across initial ATCs. As of today, more than 100 patients have already enrolled for Amtagvi therapy. We have successfully manufactured and delivered Amtagvi to many ATCs where commercial patients are being treated. We expect our launch momentum to remain strong and continue to build as we ramp up the U.S. launch throughout 2024 with the authorization of additional ATCs. We also continue to execute across our broad clinical pipeline. As a fully integrated company, Iovance is well positioned to remain the global leader in innovating, developing, and delivering TIL cell therapy for patients with cancer."

Recent and First Quarter 2024 Highlights and Corporate Updates

Amtagvi (Lifileucel) U.S. Approval and Launch Highlights in Advanced Melanoma

The U.S. FDA approved Amtagvi (lifileucel) on February 16, 2024, as the first treatment option for advanced melanoma after anti-PD-1 and targeted therapy. Amtagvi is also the first and only FDA-approved T cell therapy for a solid tumor indication.
Since approval, more than 100 patients have enrolled for Amtagvi therapy. The first patients have been successfully treated and the balance are moving through the stages of the journey, which includes surgery for cell collection, manufacturing, and the Amtagvi treatment regimen.
Onboarding is complete at more than 40 U.S. ATCs, up from 30 initial ATCs at approval. Iovance remains on track to onboard approximately 50 ATCs by the end of May 2024 and expects to have more than 70 ATCs onboarded by the end of 2024.
Manufacturing turnaround time has been on-target with initial launch expectations of approximately 34 days from inbound to return shipment to ATCs. The commercial manufacturing experience to date is consistent with prior clinical experience.
The U.S. launch of Amtagvi, and additional sales of Proleukin used with the treatment regimen, are expected to drive significant revenue for Iovance in 2024.
Amtagvi was added as a preferred second-line or subsequent therapy in the National Comprehensive Cancer Network guidelines for treatment of cutaneous melanoma.
Reimbursement remains strong and on track at the ATCs with progress toward coverage policies successful in many cases. As anticipated, more than 75% of enrolled Amtagvi patients are covered by private payers. To date, payers covering more than 200 million lives have already authorized Amtagvi treatment for their patients, setting a strong precedent for reimbursement success.
Lifileucel Launch Expansion into New Markets and Indications

Geographic expansion can more than double the total addressable patient population for Amtagvi in advanced melanoma. Regulatory dossiers remain on track for submission in the following markets with significant populations of advanced melanoma patients:
EU in the second quarter of 2024
UK and Canada in the second half of 2024
Australia and additional countries in 2025
Iovance TIL Cell Therapy Pipeline Highlights

Lifileucel in Frontline Advanced Melanoma
The registrational Phase 3 TILVANCE-301 trial is well underway to support accelerated and full U.S. approvals of Amtagvi in combination with pembrolizumab in frontline advanced melanoma as well as regular approval of Amtagvi in post-anti-PD-1 melanoma.
Global site activation and patient enrollment continue with strong momentum in the U.S., Europe, Australia, Canada, and additional countries.
An oral presentation of updated clinical data from Cohort 1A of the IOV-COM-202 trial, which strongly supports the rationale for TILVANCE-301 and the frontline melanoma opportunity, will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on May 31, 2024.
Lifileucel in Non-Small Cell Lung Cancer (NSCLC)
Enrollment resumed for new patients in the IOV-LUN-202 registrational Phase 2 trial in post-anti-PD-1 NSCLC soon after the U.S. FDA lifted a partial clinical hold in the first quarter. The IOV-LUN-202 trial includes clinical sites in the U.S., Canada, and Europe, with plans to include additional regions with strong track records for enrollment in lung cancer studies over the next few months. Enrollment has restarted with high demand and the registrational cohorts are expected to be fully enrolled in 2025.
At a recent Type D meeting, the FDA provided positive regulatory feedback on the proposed potency matrix for lifileucel in NSCLC. The FDA previously provided positive regulatory feedback that the design of the single-arm IOV-LUN-202 trial may be acceptable for approval of lifileucel in post-anti-PD-1 NSCLC.
Lifileucel in Endometrial Cancer
A Phase 2 trial of lifileucel in advanced endometrial cancer patients is on track to initiate in the second quarter of 2024. The trial will enroll patients with advanced endometrial cancer who progressed after platinum-based chemotherapy and anti-PD-(L)1 therapy regardless of mismatch repair (MMR) status. This clinical program and trial design are supported by preclinical and manufacturing success data to be presented at a conference in 2024 and has received positive feedback from gynecological oncology experts.
In 2024, an estimated 67,880 new cases of uterine cancer (>90% of which are endometrial cancer) are expected to be diagnosed, with 13,250 deaths expected in the US.1 There are no currently approved therapies in the emerging second-line setting after frontline post-anti-PD1 therapy and chemotherapy. Endometrial cancer represents a significant opportunity for TIL cell therapy to address an additional unmet medical need in the post-anti-PD-1 treatment setting and may address both MMR deficient and proficient patients.
Next Generation TIL Pipeline
IOV-4001 (PD-1 Inactivated TIL Cell Therapy): The Phase 1 safety portion concluded in the first in human IOV-GM1-201 trial to investigate PD-1 inactivated TIL cell therapy (IOV-4001) in previously treated advanced melanoma and NSCLC, and the trial is progressing successfully into the multi-center Phase 2 efficacy stage. Iovance continues to utilize the TALEN technology licensed from Cellectis to develop other investigational gene-edited TIL cell therapies with multiple knockout targets to potentially improve efficacy.
Next Generation IL-2 for TIL Treatment Regimen: Iovance plans to submit an Investigational New Drug application (IND) for a Phase 1/2 clinical trial of IOV-3001, a modified interleukin-2 (IL-2) fusion protein, for use in the TIL therapy treatment regimen in the third quarter of 2024. Results from non-human primate and IND-enabling studies of IOV-3001 will be presented in a poster at ASCO (Free ASCO Whitepaper) 2024 and demonstrate the potential for improved safety with strong effector T cell expansion.
Next Generation, Cytokine-Tethered TIL Therapy: A genetically engineered, inducible, and tethered IL-12 TIL cell therapy, designated IOV-5001, is in IND-enabling studies. In preclinical studies, IOV-5001 augmented anti-tumor activity in vitro, and a clinical trial of a prior generation IL-12 TIL therapy at the National Cancer Institute showed improved efficacy. An INTERACT meeting is planned with the FDA to discuss IOV-5001 in the third quarter of 2024, followed by an IND submission in early 2025.
Manufacturing Capacity Expansion

The Iovance Cell Therapy Center (iCTC), and a nearby FDA-approved contract manufacturer, are built today for capacity for several thousands of patients annually. Capacity expansion is currently underway at iCTC to supply TIL cell therapies for more than 5,000 patients annually in the next few years.
Upcoming ASCO (Free ASCO Whitepaper) 2024 Highlights for Iovance

Oral Presentation: Efficacy and safety of lifileucel, an autologous tumor-infiltrating lymphocyte cell therapy, and pembrolizumab in patients with immune checkpoint inhibitor-naive unresectable or metastatic melanoma: updated results from IOV-COM-202 Cohort 1A
Session: Melanoma/Skin Cancers, Friday, May 31, 2024, 2:45 – 5:45 p.m. CDT
Poster: IOV-3001, a modified interleukin-2 fusion protein, for potential use in tumor-infiltrating lymphocyte cell therapy regimens
Session: Developmental Therapeutics – Immunotherapy – Saturday, June 1, 2024; 9:00 a.m. – 12:00 p.m. CDT
Poster: Dynamics of circulating cytokines and chemokines during and after tumor-infiltrating lymphocyte cell therapy with lifileucel in advanced melanoma patients
Session: Melanoma/Skin Cancers: Saturday, June 1, 2024, 1:30-4:30 p.m. CDT
Corporate Updates

As of March 31, 2024, Iovance had cash, cash equivalents, investments, and restricted cash of approximately $362.6 million, compared to $346.3 million at December 31, 2023. The current cash position and anticipated revenue from Amtagvi and Proleukin are expected to be sufficient to fund current and planned operations well into the second half of 2025.
Iovance is now recognizing all Proleukin revenues for commercial and clinical use and has recently commenced significant sales to distributors. In the first quarter of 2024, Iovance completed the transfer of marketing authorizations and began distribution of Proleukin. Beginning in the second quarter of 2024, Iovance expects to recognize significant incremental revenues for Proleukin as part of the Amtagvi treatment regimen.
Iovance currently owns more than 120 granted or allowed U.S. and international patents for TIL-related technologies, including TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038 and additional patent rights, including patents related to potency assays expected to provide exclusivity into 2042. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
First quarter 2024 Financial Results

Net loss for the first quarter ended March 31, 2024, was $113.0 million, or $0.42 per share, compared to a net loss of $107.4 million, or $0.50 per share, for the first quarter ended March 31, 2023. The net loss for the first quarter 2024 includes amortization of intangible assets acquired as part of the Proleukin transaction.

Revenue for the first quarter ended March 31, 2024, was $0.7 million from sales of Proleukin in licensed markets outside of the U.S. Cost of sales was $7.3 million, primarily related to inventoriable costs associated with sales of Proleukin and non-cash amortization expense for the acquired intangible asset for developed technology during the first quarter of 2024. No revenue or cost of sales were incurred for the first quarter ended March 31, 2023.

Research and development expenses were $79.8 million for the first quarter ended March 31, 2024, a decrease of $2.9 million compared to $82.7 million for the first quarter of 2023. The decrease was primarily attributable to the transition to commercial Amtagvi manufacturing in the most recent quarter, partially offset by increased costs associated with the purchases of raw materials, clinical trials driven primarily by the Phase 3 TILVANCE-301 clinical trial, and the planned EU regulatory submissions for lifileucel.

Selling, general and administrative expenses were $31.4 million for the first quarter ended March 31, 2024, an increase of $3.3 million compared to $28.1 million for the first quarter of 2023. The increase was primarily attributable to increases in headcount and related costs, including stock-based compensation, to support the growth in the overall business and related corporate infrastructure, as well as costs incurred to support the commercialization of Amtagvi and Proleukin, partially offset by a decrease in legal costs.

For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com for one year.

1. National Cancer Institute Surveillance, Epidemiology and End Results (SEER) Program. 2024 Estimates. View Source

On May 9, 2024 Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported first quarter 2024 financial results and provides a corporate update (Press release, Intensity Therapeutics, MAY 9, 2024, View Source [SID1234642995]).

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Corporate Update

•In mid-2024, the Company intends on initiating a Phase 3 open-label, randomized study, or the INVINCIBLE-3 Study, testing INT230-6 as a monotherapy compared to the standard of care ("SOC") drugs in second and third line treatment for certain soft tissue sarcoma subtypes. We plan to enroll 333 patients with an endpoint of overall survival and have screened and qualified over 30 sites for the INVINCIBLE-3 Study. Contract negotiations to approve and activate these sites are in process. We estimate the sites could take between two to six months to complete their contracting processes.

•In mid-2024, the Company intends on initiating a Phase 2/3 program testing INT230-6 in combination with the SOC treatment (chemotherapy/immunotherapy) compared to SOC alone in women with triple negative breast cancer in presurgical (neoadjuvant) breast cancer. The endpoint for the Phase 2 portion of the study, or the INVINCIBLE-4 Study, is the change in the pathological complete response rate for the combination compared to the SOC alone. We expect to initiate the INVINCIBLE-4 Study in mid-2024, which will provide data to size a Phase 3 study. We are in the process of screening and qualifying sites for the INVINCIBLE-4 Study.

"We continue to make excellent progress towards the initiation of our INVINCIBLE-3 Study," said Lewis H. Bender, Founder, President and CEO of Intensity Therapeutics, Inc. "In the first quarter, we were able to release our INT230-6 Phase 3 clinical supplies, successfully ship the entire batch to our main depot, and complete labeling of a portion of the vials for the United States sites. During the quarter, we completed evaluation of dozens of sites and are now in various stages of contract and budget discussions with over 30 different high-quality sarcoma centers. We continue to qualify new sarcoma specialty hospitals in the US and internationally, and top enrolling centers from our previous metastatic study have expressed interest in participating in the INVINCIBLE-3 trial." Mr. Bender continued, "Our INVINCIBLE-4 Study also continues to make excellent progress. Sites are being recruited and we continue to work towards initiating the study in mid-2024."

First Quarter 2024 Financial Results

Research and development expenses were $2.8 million for the three months ended March 31, 2024, compared to $0.8 million for the same period in 2023. The increase was primarily due to preliminary work related to the INVINCIBLE-3 Study, and to a lesser extent, costs for manufacturing a new batch of INT230-6 and increased expenses related to salary, benefits, and stock-based compensation.

General and administrative expenses were $1.9 million for the three months ended March 31, 2024, compared to $0.5 million for the same period in 2023. The increase was primarily due to increased expenses related to salary, benefits and stock-based compensation, higher legal, audit, and consulting fees, and higher directors and officers insurance.

Overall, net loss was $4.6 million for the three months ended March 31, 2024, compared to $1.3 million for the three months ended March 31, 2023.

As of March 31, 2024, cash, cash equivalents and marketable debt securities totaled $10.5 million, which the Company expects will be sufficient to fund operations through the end of the first quarter in 2025.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is composed of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor resulting in a favorable safety profile. In addition to local disease control, direct killing of the tumor by INT230-6 releases a bolus of neoantigens specific to the patient’s malignancy, leading to engagement of the immune system and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression that so often occurs with systemic chemotherapy.

On May 9, 2024 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported operational highlights and financial results for the first quarter ended March 31, 2024 (Press release, Intellia, MAY 9, 2024, View Source [SID1234642994]).

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"Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies. With one ongoing and two soon-to-be-initiated pivotal Phase 3 trials, Intellia is undoubtedly at the forefront of a new era in medicine," said Intellia President and Chief Executive Officer John Leonard, M.D. "We have been extremely pleased with the speed of enrollment in the Phase 3 MAGNITUDE trial for patients with ATTR amyloidosis with cardiomyopathy, which is tracking ahead of our initial projections. In addition, we now expect to start a pivotal Phase 3 trial of NTLA-2001 for patients with polyneuropathy by year-end, based on productive discussions with the FDA. Moving to NTLA-2002, we expect to report key data readouts from the Phase 1/2 study this year. These data will support the dose selection for the Phase 3 trial and highlight what we believe is the potential for NTLA-2002 to dramatically change the HAE treatment paradigm. Building on our success with in vivo gene inactivation, we are excited to initiate the first-in-human study of NTLA-3001 for AATD this year, positioning us to be the first to clinically validate CRISPR-based gene insertion. We look forward to continuing our strong execution, with many notable milestones to mark the progress against our strategic priorities."

First Quarter 2024 and Recent Operational Highlights

Transthyretin (ATTR) Amyloidosis

NTLA-2001: NTLA-2001 is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in the liver and thereby prevent the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Intellia leads development and commercialization of NTLA-2001 in collaboration with Regeneron.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
The pivotal Phase 3 MAGNITUDE trial is rapidly enrolling. In March, the first patients in the U.S. and globally were dosed. Enrollment is currently tracking well ahead of the Company’s initial projections, with over 30 patients dosed to date and more than 40 additional patients in screening. Many additional sites are expected to open in the weeks and months ahead to further accelerate enrollment.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
Intellia announced today alignment with the U.S. Food and Drug Administration (FDA) on a pivotal Phase 3 trial design to support a biologics license application (BLA) filing for NTLA-2001 as a single-dose treatment for people living with ATTRv-PN, subject to review of its investigational new drug (IND) application. The study is expected to be a small, placebo-controlled trial conducted at ex-U.S. sites with approximately 50 ATTRv-PN patients. The Company plans to initiate the study by year-end.
The Company plans to present updated data from the ongoing Phase 1 study in the second half of 2024.
Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
Intellia plans to initiate the global pivotal Phase 3 study, including U.S. patients, in the second half of 2024, subject to regulatory feedback.
The Company will present updated data from the Phase 1 study at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, taking place May 31 – June 3 in Valencia, Spain. Long-term data from the Phase 1 portion of the Phase 1/2 study will include safety, kallikrein reduction and attack rate data, including number of patients who continue to be completely attack free through the latest follow-up. Additionally, Intellia plans to report topline results from the Phase 2 portion in mid-2024 and present full results at a medical meeting in the second half of 2024.
In January, landmark findings from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published in the New England Journal of Medicine (NEJM).
Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

NTLA-3001: NTLA-3001 is a first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert the wild-type SERPINA1 gene, which encodes the alpha-1 antitrypsin (AAT) protein, with the potential to restore permanent expression of fully functional AAT protein to normal levels after a single dose. This is Intellia’s first wholly owned gene insertion program.
Intellia expects to dose the first patient in a Phase 1 study of NTLA-3001 in 2024.
In Vivo Platform Expansion

Intellia is expanding the range of diseases that can be targeted with its CRISPR-based technologies by deploying new editing and delivery innovations. This includes advancing gene editing programs in five different tissues outside the liver, either independently or in collaboration with partners. These research and preclinical programs are targeting diseases that originate in the bone marrow, brain, muscle, lung and eye, which, if successful, could dramatically expand the opportunities for CRISPR-based treatments.
In February, Intellia and ReCode announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF). The collaboration will leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle delivery platform to precisely correct one or more CF disease-causing gene mutations.
Ex Vivo Program Updates

Intellia is advancing multiple programs, wholly owned and in collaboration with partners, utilizing its allogeneic platform for the treatment of immuno-oncology and autoimmune diseases. The Company’s proprietary allogeneic cell engineering platform avoids both T cell- and NK cell-mediated rejection in preclinical models, a key unsolved challenge with other investigational allogeneic approaches. Cell therapies engineered with Intellia’s allogeneic platform, combined with edits to enhance cell function, offer a new approach to target solid tumors.
Corporate Updates

Corporate Responsibility Report: In April, Intellia published its 2024 Corporate Responsibility Report. The report highlights the Company’s Environmental, Social and Governance (ESG) principles and practices as part of its objective to build a sustainable company, while delivering on its commitments to patients, employees and shareholders.
Upcoming Events

The Company will participate in the following events during the second quarter of 2024:

Bank of America Health Care Conference, May 14, Las Vegas
RBC Capital Markets Global Healthcare Conference, May 14, New York
EAACI Congress 2024, May 31 – June 3, Valencia, Spain
Goldman Sachs 45th Annual Global Healthcare Conference, June 10, Miami
First Quarter 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $953.4 million as of March 31, 2024, compared to $1.0 billion as of December 31, 2023. The decrease was driven by cash used to fund operations of $137.2 million. The decrease was offset in part by $58.0 million of net equity proceeds from the Company’s "At the Market" (ATM) program, $12.6 million of interest income, $5.9 million of reimbursement from its collaborators, and $2.0 million in proceeds from employee-based stock plans. The cash position is expected to fund operations into late 2026.
Collaboration Revenue: Collaboration revenue was $28.9 million during the first quarter of 2024, compared to $12.6 million during the first quarter of 2023. The $16.3 million increase was mainly driven by a $21.0 million non-cash revenue recognition adjustment related to the AvenCell collaboration.
R&D Expenses: Research and development expenses were $111.8 million during the first quarter of 2024, compared to $97.1 million during the first quarter of 2023. The $14.7 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in research and development expenses was $20.2 million for the first quarter of 2024.
G&A Expenses: General and administrative expenses were $31.1 million during the first quarter of 2024, compared to $27.4 million during the first quarter of 2023. The $3.7 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in general and administrative expenses was $14.0 million for the first quarter of 2024.
Net Loss: Net loss was $107.4 million for the first quarter of 2024, compared to $103.1 million during the first quarter of 2023.
Conference Call to Discuss First Quarter 2024 Results

The Company will discuss these results on a conference call today, Thursday, May 9, at 8 a.m. ET.
To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on May 9 at 12 p.m. ET.

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.