On May 9, 2024 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported its first quarter 2024 financial and operating results and will host a conference call at 4:30 p.m. Eastern Time to discuss these results and to provide a business update (Press release, Lineage Cell Therapeutics, MAY 9, 2024, View Source [SID1234643000]).

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"The quarter was highlighted by significant milestones and data updates on our lead program," stated Brian M. Culley, Lineage CEO. "A key area of attention for investors is our partnership with Roche and Genentech, and we are pleased to announce a new services agreement which reflects an additional commitment by Genentech for the benefit of the OpRegen program. We believe this agreement will enable our partners to take advantage of our cell transplant expertise to more fully investigate the promising potential of the OpRegen program and do so in a cost-effective manner. We also are planning to bring our second cell transplant program, OPC1, into the clinic this year for a condition with growing awareness of its unmet need and commercial opportunity. Lastly, we continue to build value through the advancement of our early-stage pipeline, which can help create value by capitalizing on the continued validation of our cell transplant approach."

Recent Operational Highlights

RG6501 (OpRegen)
Established new services agreement with Genentech, a member of the Roche Group, to support ongoing development of OpRegen. Under this new agreement, Lineage will provide additional clinical, technical, training and manufacturing services that further support the ongoing advancement and optimization of the OpRegen program. These additional services will be fully funded by Genentech and include: (i) activities to support the ongoing Phase 1/2a study and currently-enrolling Phase 2a study; and (ii) additional technical training and materials related to Lineage’s cell therapy technology platform to support commercial manufacturing strategies.
Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including support for the ongoing Phase 2a clinical study in patients with GA secondary to AMD.
Positive clinical data from long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen presented by David Telander, MD, PhD, Retinal Consultants Medical Group, at the 2024 Retinal Cell & Gene Therapy Innovation Summit.
Mean BCVA gain of 5.5 letters at 24 months in Cohort 4 patients (less advanced geographic atrophy)
Mean BCVA gains greater among patients with improvement in outer retinal structure (+7.4 letters)
Maintenance or increases in external limiting membrane (ELM) and retinal pigment epithelium (RPE) layers at 24 months observed in patients with extensive coverage of OpRegen across the areas of GA
Data suggests OpRegen may counteract RPE cell dysfunction and cell loss secondary to geographic atrophy by providing support to remaining retinal cells, with multi-year effects observed following a single administration
Preclinical results from a surgical development study of OpRegen presented by Rachel N. Andrews, DVM, PhD, DACVP, Genentech, a member of the Roche Group, at 2024 Association for Research in Vision and Ophthalmology Annual Meeting (2024 ARVO).
OPC1
DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study for the treatment of subacute and chronic spinal cord patient start-up activities underway.
Received an Education Conference II Grant from the California Institute for Regenerative Medicine (CIRM), to support the 2nd Annual Spinal Cord Injury Investor Symposium, hosted in partnership with the Christopher & Dana Reeve Foundation, to be held on June 26 and 27, 2024 at the Sanford Consortium for Regenerative Medicine in La Jolla, CA.
Corporate Updates
Appointed veteran industry executive Dr. Charlotte Hubbert as Vice President of Corporate Development. Dr. Hubbert previously served as Partner and Head of Gates Foundation Venture Capital, an initiative at the Bill and Melinda Gates Foundation Strategic Investment Fund, and most recently served in the leadership team at NanoString Technologies. She currently serves on the Board of Directors of the Beckman Research Institute at the City of Hope and is a Strategic Director at Madrona Venture Group.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities of $43.6 million as of March 31, 2024, is expected to support planned operations into Q3 2025.

First Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from collaboration revenues and royalties. Total revenues for the three months ended March 31, 2024 were approximately $1.4 million, a net decrease of $1.0 million as compared to $2.4 million for the same period in 2023. The decrease was primarily driven by lower collaboration and licensing revenue recognized from deferred revenues under the collaboration and license agreement with Roche.

Operating Expenses: Operating expenses are primarily comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended March 31, 2024 were $8.1 million, a decrease of $0.9 million as compared to $9.0 million for the same period in 2023.

R&D Expenses: R&D expenses for the three months ended March 31, 2024 were $3.0 million, a net decrease of $1.2 million as compared to $4.2 million for the same period in 2023. The net decrease was primarily driven by $0.4 million for our OPC1 program, $0.3 million for our preclinical programs, and $0.2 million for our OpRegen program. Another $0.3 million of the decrease was attributable to other research and development expenses, primarily related to reduced manufacturing activities.

G&A Expenses: G&A expenses for the three months ended March 31, 2024 were $5.0 million, a net increase of $0.3 million as compared to approximately $4.7 million for the same period in 2023. The increase was primarily driven by $0.2 million in stock-based compensation expenses, and an overall increase in costs incurred for consulting services.

Loss from Operations: Loss from operations for the three months ended March 31, 2024 were $6.7 million, an increase of $0.1 million as compared to $6.6 million for the same period in 2023.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended March 31, 2024 was comprised of other income of $0.1 million, compared to other income of $0.4 million for the same period in 2023. The net decrease was primarily driven by the employee retention credit recognized in the prior year, partially offset by exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2024 was $6.5 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $4.4 million, or $0.03 per share (basic and diluted), for the same period in 2023.

Conference Call and Webcast

Interested parties may access the conference call on May 9th, 2024, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 15th, 2024, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 1330332.

On May 9, 2024 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer therapies using its proprietary RADR AI and machine learning ("ML") platform with multiple clinical-stage drug programs, reported operational highlights and financial results for the first quarter 2024, ended March 31, 2024 (Press release, Lantern Pharma, MAY 9, 2024, View Source [SID1234642999]).

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"Our company made meaningful progress across multiple clinical trials and in furthering our AI platform this past quarter while advancing our internal capabilities to both support data-driven, precision oncology trials and accelerate the cost-effective development of drug-conjugates. Our team is at the forefront of demonstrating how combining emerging AI technologies, cancer biology and biomarker expertise along with focused clinical operations holds the promise of transforming timelines and costs in biopharma development." said Panna Sharma, President and CEO of Lantern Pharma.

Sharma continued, "Over the past several months, we have experienced growing interest in machine-learning enabled drug development and, in our RADR, AI platform. Our team is energized by the growing desire to adopt and leverage AI-driven innovations in biopharma and in the meaningful progress we are making with our own drug-candidates. We are excited about the opportunities we have in front of us to drive increased collaborations on our AI platform this year, and also propel the streamlined development of our own portfolio of high-value, high-impact drug-candidates."

Highlights of AI-Powered Pipeline:

LP-184 – Five cohorts of patients, comprised of dose levels 1 thru 5, have been enrolled and dosed – in escalating doses – in the ongoing Phase 1A clinical trial. This is a first-in-human Phase 1 trial across multiple solid tumor indications that are advanced and refractory to existing standard-of-care therapies. The trial is actively enrolling and dosing patients at dose level 6 that have relapsed/refractory advanced solid tumors, such as pancreatic cancer, glioblastoma (GBM), lung, triple-negative breast cancer, and multiple other solid tumor types. There have not been any observed dose-limiting toxicities to date.

The company believes that enrollment should be complete this summer and on-track for a readout of data in late summer or early fall. Current efforts are underway to focus enrollment efforts on cancer patients with tumors that have DDR(1) (DNA damage repair) deficiency. DDR deficient tumors have been observed to have higher sensitivity to LP-184. The company has also submitted a dose optimization and expansion protocol (Supplement A) to the FDA related to LP-184 in non-CNS solid tumors, including TNBC (triple negative breast cancer) with DDR alterations. Additionally, the company in collaboration with Starlight Therapeutics, has also submitted to the FDA a dose optimization and expansion protocol in recurrent IDH wild-type high grade gliomas (Supplement B).

The dosage and safety data obtained in the Phase 1a and 1b trials are expected to be used to advance the central nervous system (CNS) indications for a future Phase 2 trial to be sponsored by Lantern’s wholly owned subsidiary, Starlight Therapeutics. The Phase 1a data will also inform other anticipated later phase trials in select solid tumors, most likely with genomic signatures signifying DDR (DNA damage repair) deficiency, that have shown responsiveness to LP-184. Genomic identification of these patients and biomarker characterization of their underlying tumor is central to our focus of personalizing treatment and developing efficient later stage clinical trials. To further this effort, Lantern has also initiated the development of a PCR-based molecular diagnostic test that may help in identifying cancer patients with the best likelihood of response and benefit from treatment with LP-184.

AI and preclinical studies are ongoing to further refine drug combination studies supporting the use of LP-184 to improve the durability or overall response rates in combination with FDA approved drugs that are widely used in cancer treatment. Globally, the aggregate annual market potential of LP-184’s target indications is estimated to be approximately $12+ billion, consisting of $4.5+ billion for CNS cancers and $7.5+ billion for solid tumors.

LP-284 – The initial two cohorts of patients have been dosed, and no dose-limiting toxicities have been observed in the Phase 1a clinical trial. The company expects to open additional sites in the US throughout the second quarter with the potential to advance to Phase 1b/2 by the close of 2024. LP-284 has shown nanomolar potency across multiple published in vitro and in vivo studies, including mantle cell lymphoma (MCL), double hit lymphoma (DHL), and other advanced NHL cancer subtypes and certain sarcomas with DDR deficiencies, notably those with compromised functioning of the ataxia-telangiectasia mutated (ATM) gene due to mutations or deletions. Nearly all MCL, DHL, and HGBL patients relapse from the current standard-of-care agents and there is an urgent and unmet need for novel improved therapeutic options for these patients. In the US and Europe, MCL, DHL, and HGBLs are diagnosed in 16,000-20,000 patients each year and have an estimated annual market potential of over USD 3+ billion.

LP-300 – The phase 2 Harmonic clinical trial sites in the US are continuing to screen for patients and have also increased the pace of enrollment. This past quarter we also received approval to proceed with the Phase 2 clinical trial in Japan and Taiwan. This is expected to accelerate the collection of patient and response data needed for the next-stage of evaluation and development of LP-300, an investigational therapeutic for the treatment of relapsed and inoperable primary adenocarcinoma of the lung given in combination with chemotherapy Additionally, it may also bring a needed therapeutic option for LCINS (Lung Cancer In Never Smokers) diagnosed patients in Japan and Taiwan, where one-third of all lung cancer diagnoses are made among those who have never smoked.

Dr. Yashushi Goto, a physician and researcher focused on lung cancer at the National Cancer Center of Japan, will be leading the phase 2 trial in Japan, where the incidence of non-small cell lung cancer (NSCLC) in never-smokers is double or more than that of the United States. Lantern believes that this improves the positioning for drug-candidate LP-300 to develop collaborative and co-development partnerships with global biopharma companies with a primary focus in serving the Asian markets.

The Harmonic trial is assessing the effect of LP-300 in combination with standard-of-care chemotherapy (carboplatin and pemetrexed) in LCINS patients with relapsed NSCLC. Globally, LCINS patients are a growing population of patients and do not respond well to PD-1/PD-L1-based therapies or the available chemotherapy doublets, leaving them with reduced treatment options. In the US it is estimated that LP-300 has an annual market potential of $1.5 billion, and a global estimated annual market potential of over $2.6 billion. LCINS is the eighth leading cause of cancer-related mortality in the USA and the fifth most common cause of cancer-related deaths worldwide.

RADR Platform Growth and Development:

RADR continues to advance in size, scope, and capabilities and is progressing towards becoming a standard for AI-driven drug development in oncology – for both early-stage development and later-stage patient biomarker and combination therapy identification. The company recently announced a artificial intelligence (AI)-driven collaboration to optimize the development of a protein disulfide isomerase (PDI) inhibitor drug candidate, XCE853, for a variety of novel and targeted cancer indications. The collaboration is leveraging RADR’s AI-based capabilities, including 200+ machine learning (ML) algorithms and foundational models for oncology drug development to uncover biomarkers and molecular correlates of efficacy and define potential combination regimens to sharpen and accelerate XCE853’s drug development strategy. Lantern Pharma is receiving equal IP co-ownership and drug development rights in newly discovered biomarkers, novel indications, and use for new pharmacological strategies for XCE853.

The scope of RADR’s data has broadened with a strategic focus on additional classes of compounds, including drug-conjugates such as ADCs and inclusion of detailed data on chemical and biochemical features and drug-interaction data. Additionally, data from clinical studies such as those being obtained from liquid biopsy, and data from preclinical combination studies that aim to define drug interaction and optimal dosage are being incorporated into the datapoints and data sets powering RADR. Lantern expects to pursue additional biopharma and technology partnerships during 2024 to further advance and commercialize the RADR AI platform.

Starlight Therapeutics:

Starlight Therapeutics, a wholly owned subsidiary of Lantern Pharma focused on CNS and brain cancers with STAR-001, continues advancements with the filing of a clinical trial protocol for the Phase 1B dose optimization and expansion cohort in recurrent IDH wild-type high grade gliomas. IDH wild-type glioblastomas are the most malignant glial tumors with median survival of only 14–16 months after diagnosis; patients aged ≥ 65 years have reportedly worse outcomes.(3) Lantern formed a wholly-owned subsidiary, Starlight Therapeutics Inc. ("Starlight"), in early 2023 for the clinical development of drug candidate LP-184’s central nervous system (CNS) and brain cancer indications – including GBM, brain mets., and several rare pediatric CNS cancers. Starlight will refer to the molecule LP-184, as it is developed in CNS indications, as "STAR-001". The indications and mechanistic insights powering the creation of Starlight and the identification of multiple CNS tumors that can be potentially impacted were largely driven by insights and analysis from the RADR AI platform.

During Q4 of 2023 Lantern announced that it had hired a CMO, Dr. Marc Chamberlain, who will focus on Starlight’s clinical trials, development of personnel to execute on the planned clinical trials and overall support in corporate development activity. Starlight and Lantern expect to initiate Phase 1b/2 clinical trials during the second half of 2024. The market potential for the currently planned indications for Starlight’s synthetically-lethal, cancer-cell DNA damaging agent – STAR-001 – is estimated to be 4.5 billion to 5+ billion USD across both adult and pediatric primary and secondary CNS cancers.

ADC & Drug Conjugate Programs:

During the first quarter, Lantern, in collaboration with academic research partners in Germany, advanced the development, synthesis, and preclinical proof-of-concept of a novel, highly potent, cryptophycin-based ADC (cpADC). The cpADC has shown picomolar potency in a wide range of solid tumors tested in preclinical development and is being further evaluated for clinical potential in six solid tumor indications. In preclinical work, the cpADC produced an 80% cancer cell kill rate which was more than other commonly used approved ADCs, including in a cancer sub-type, medium and low HER-2 expression cancers, which is an area of critical patient need. Lantern expects to move towards IND development of its ADC program during 2024 with a focus on select solid tumors that are unresponsive or refractory to current therapies.

Additionally, Lantern has advanced its AI module for differentiated, machine-learning based ADC development, characterization, analysis and bioactivity prediction. The ADC module is being developed as an extension to RADR and leverages the data and biomarker insights curated by and generated in RADR. Lantern has plans to further advance the development through partnerships and collaborations with both technology and biopharma companies.

Additional Operational Highlights:

A new publication on the lethal activity of LP-184, inducing elevated levels of DNA double-strand breaks, in HR deficient (HRD) cancer cells was published by research scientists at Lantern Pharma in collaboration with Georgetown University Medical Center in Cancer Research Communications. The publication showcased that depletion of key HR components BRCA2 or ataxia telangiectasia mutated (ATM) in cancer cells conferred up to 12-fold increased sensitivity to LP- 184 and that LP-184 showed nanomolar potency in a diverse range of HRD cancer models. A link to the publication titled "LP-184, a Novel Acylfulvene Molecule, Exhibits Anticancer Activity against Diverse Solid Tumors with Homologous Recombination Deficiency" can be accessed here.

New data and scientific findings for LP-284 and the ongoing clinical trial were presented at AACR (Free AACR Whitepaper) (American Association for Cancer Research) during the 2024 Annual Meeting in San Diego – Phase 1a/1b clinical trial of LP-284, a highly potent TP53 mutation agnostic DNA damaging agent, in patients with refractory or relapsed lymphomas and solid tumors (NCT06132503)

The company also announced during April that it will hosting a series of educational and informative webinars focused on updates on Lantern’s areas of research, clinical trials and AI efforts titled Webinar Wednesdays. The first of these webinars was held on April 24th and featured Dr. Joseph Treat, a Professor in the Department of Hematology and Oncology, Vice Chair of Education, and Medical Director of Ambulatory Care at Fox Chase Cancer Center discussing LP-300 and the Harmonic clinical trial focused on LCINS patients.

First Quarter 2024 Financial Highlights

Balance Sheet: Cash, cash equivalents, and marketable securities were approximately $38.4 million as of March 31, 2024, compared to approximately $41.3 million as of December 31, 2023. The quarterly cash burn rate continues to reflect our capital-efficient, collaborator-centered business model.

R&D Expenses: Research and development expenses were approximately $4.3 million for the quarter ended March 31, 2024, compared to approximately $2.6 million for the quarter ended March 31, 2023. This increase was largely driven by an increase in clinical trial activity and clinical trial site activations.

G&A Expenses: General and administrative expenses were approximately $1.5 million for the quarter ended March 31, 2024, compared to approximately $1.7 million for the quarter ended March 31, 2023.

Net Loss: Net loss was approximately $5.4 million (or $0.51 per share) for the quarter ended March 31, 2024, compared to a net loss of approximately $3.9 million (or $0.36 per share) for the quarter ended March 31, 2023.

Warrant Exercises: Lantern issued 20,132 shares of common stock during Q1 2024, relating to the cashless exercise of warrants to purchase 79,021 shares. Also, in Q1 2024, Lantern issued 17,481 shares of common stock for aggregate proceeds of approximately $55,000, relating to the exercise of warrants for cash. Following these exercises, there remain 81,496 warrants outstanding to purchase Lantern common stock at a weighted average exercise price of $16.55 per share.

Earnings Call and Webinar Details:

Lantern will host its 1st quarter 2024 earnings call and webinar today, May 9, 2024, at 4:30 p.m. ET. A link to register can be accessed at: Lantern 1st Quarter 2024 Earnings Call & Webinar Link

Related presentation materials will be accessible at: View Source
A replay of the 1st quarter 2024 earnings call and webinar will be available at: View Source

On May 9, 2024 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to developing small molecule therapeutics that address cancers and other diseases driven by deregulated transcription, reported recent business progress and financial results for the first-quarter of 2024 (Press release, Kronos Bio, MAY 9, 2024, View Source [SID1234642998]).

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"We have continued to make good progress towards our goals. We are looking forward to sharing a clinical update on the KB-0742 data to date at ASCO (Free ASCO Whitepaper) in June," said Nobert Bischofberger, Ph.D., president and chief executive officer of Kronos Bio, Inc. "We expect to complete the IND-enabling studies for KB-9558 by the end of 2024 and expect to dose our first patients in the first half of 2025. Our collaboration with Genentech and our internal discovery programs continue to advance and we look forward to sharing our progress later this year."

Recent Company News
•At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2024, the Company presented three posters, including the first public presentation of data from its p300 program. The posters include:
◦p300 catalytic inhibition selectively targets IRF4 oncogenic activity in multiple myeloma (link to poster)
◦A dose escalation and cohort expansion study of the CDK9 inhibitor KB-0742 in relapsed, refractory and transcriptionally addicted solid tumors (link to poster)
◦KB-0742, an oral highly selective CDK9 inhibitor, demonstrates preclinical activity in transcription factor fusion driven adenoid cystic carcinoma patient-derived models (link to poster)

•The Company announced that they will be presenting updated study data from KB-0742-1001, the ongoing Phase 1/2 trial of KB-0742 at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper):
Title: Study update of the oral CDK9 inhibitor KB-0742 in relapsed or refractory transcriptionally addicted advanced solid tumors
Presenter: Brian A. Van Tine, M.D., Ph.D., Washington University in St. Louis
Abstract ID#: 3102
Poster Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Location: Hall A, McCormick Place, Chicago, Illinois
Poster Board #: 247
Date and Time: Saturday, June 1, 2024, from 9:00 a.m. to 12:00 p.m. CDT

First Quarter 2024 Financial Highlights

•Cash, Cash Equivalents and Investments: With its ongoing and currently planned clinical programs and $152.0 million in cash, cash equivalents and investments as of March 31, 2024, the Company anticipates sufficient resources to fund its planned operations into the second half of 2026.

•R&D Expenses: Research and development expenses were $14.2 million for the first quarter of 2024, which includes non-cash stock-based compensation expense of $1.2 million.

•G&A Expenses: General and administrative expenses were $7.5 million for the first quarter of 2024, which includes non-cash stock-based compensation expense of $2.2 million.

•Impairment of Long-lived Assets and Restructuring: The Company incurred impairment of long-lived assets expense of $6.6 million for the first quarter of 2024. The Company also incurred restructuring expense of $6.2 million for the first quarter of 2024, which includes non-cash stock-based compensation of $4.4 million.

•Net Loss: Net loss for the first quarter of 2024 was $30.0 million, or $0.50 per share, including non-cash stock-based compensation expense of $7.7 million.

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)

•
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)

•
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)

•
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.
•
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.
•
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

•
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.
•
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.
•
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.
•
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.

•
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.

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