On May 5, 2022 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company that aims to develop the world’s most potent vaccines, reported financial results for the first quarter ended March 31, 2022 and reviewed business highlights (Press release, Gritstone Oncology, MAY 5, 2022, View Source [SID1234613753]).

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"Clinical data out of our cancer and viral disease vaccine programs are expected to be flowing steadily through year-end and over the next 18 months," said Andrew Allen, M.D., Ph.D., Co-founder, President and Chief Executive Officer of Gritstone. "Our individualized neoantigen vaccine candidate for solid tumors, GRANITE, is now in a randomized Phase 2/3 trial in newly diagnosed metastatic colorectal cancer, building on the exciting molecular response/survival data shown at ESMO (Free ESMO Whitepaper) 2021 in end-stage colorectal cancer patients. Multiple Phase 1 trials are ongoing in CORAL (2nd generation COVID-19 vaccine program) with data from all studies expected throughout 2H2022, and initial data from the Phase 2 study of SLATE-KRAS (KRAS-specific ‘off-the-shelf’ vaccine candidate) is also expected in 2H2022. Our recent presentations at AACR (Free AACR Whitepaper) underscore the unique capabilities of our novel self-amplifying mRNA (samRNA) vector, which has demonstrated potency and dose sparing potential along with a favorable safety and tolerability profile in the clinic. The data we are generating with SARS-CoV-2 as the target are providing validation for the entire samRNA platform and provide clear rationale to pursue additional viral pathogens. We look forward to sharing more data and continuing to demonstrate the value we are bringing to patients and all stakeholders as the year progresses."

Clinical Program Updates

Tumor-Specific Neoantigen (TSNA) Oncology Programs

GRANITE – Individualized, TSNA-directed vaccine-based immunotherapy using an adenoviral priming vector and samRNA boost vector to deliver relevant neoantigens. Following success in late-line studies, Gritstone intends to continue advancing GRANITE through randomized, controlled trials and evaluate these candidates in earlier lines of treatment, where immune responses may be stronger and the potential benefits could be further accentuated.

In January, Gritstone announced the first patient was enrolled for inclusion in GRANITE-CRC-1L, a randomized, controlled Phase 2/3 trial evaluating GRANITE in combination with immune checkpoint blockade for frontline maintenance treatment of newly diagnosed patients with metastatic, microsatellite-stable colorectal cancer (MSS-CRC). Preliminary data (molecular response and progression-free survival) from the Phase 2 portion of the trial are expected in 2H2023.
In March, Gritstone announced the first patient was enrolled for inclusion in GRANITE-CRC-ADJUVANT, a randomized, controlled Phase 2 trial in patients with high risk MSS-CRC and stage II/III disease who are circulating tumor DNA (ctDNA)+ after definitive surgery.
In April, Gritstone shared two poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting:
Poster 1238 further demonstrated the correlation between patient survival and ctDNA in metastatic CRC.
Poster 4149 demonstrated lower doses of samRNA drive superior neoantigen-specific CD8+ T cell responses in cancer patients versus high doses in Phase 1/2 trials for GRANITE and SLATE in patients with advanced solid tumors.
SLATE – "Off-the-shelf" shared neoantigen-directed vaccine-based immunotherapy using an adenoviral priming vector and samRNA boost vector to deliver a cassette of shared TSNA. Gritstone intends to continue advancing its existing candidate, SLATE-KRAS, and has a long-term objective of developing a suite of "off-the-shelf" product candidates that target tumor-specific targets across a number of patient populations and cancer types.

In April, Gritstone presented an oral presentation at AACR (Free AACR Whitepaper) detailing how translational immunology data and the company’s cassette design capabilities enabled development of SLATE-KRAS, an optimized, KRAS-specific version of SLATE that is now in Phase 2 study in patients with advanced non-small cell lung cancer (NSCLC) and CRC.
Early signals from the ongoing Phase 2 study support the potential of SLATE-KRAS to drive stronger CD8+ T cell responses to mutant KRAS than our original candidate, SLATE v1.
Initial data from the ongoing Phase 2 study of SLATE-KRAS trial are expected in 2H2022.
Infectious Disease Programs

Gritstone’s infectious disease programs aim to deliver vaccine candidates that drive both B cell and T cell immunity with the potential to provide either a protective or therapeutic effect across a broad array of viral diseases. This approach has demonstrated the ability to generate robust CD8+ T cells and neutralizing antibodies against SARS-CoV-2 in multiple preclinical and clinical studies and is being evaluated against multiple other pathogens in Gritstone-owned and partnered studies.

CORAL – Second-generation SARS-CoV-2 vaccine program delivering both spike and highly conserved non-spike T cell epitopes (TCEs) with a focus on the samRNA vector. This approach offers potential for more durable clinical protection and broader immunity against SARS-CoV-2 variants than first generation products by inducing potent CD8+ T cells in addition to neutralizing antibody responses.

Gritstone is currently evaluating five distinct SARS-CoV-2 product candidates across four different clinical trials containing Spike plus additional non-Spike TCE sequences (and also full-length nucleocapsid). These studies include homologous and heterologous prime-boost regimens. All four of these studies are ongoing, and initial data from all are expected during the second half of 2022.
The CORAL-BOOST study, a Phase 1 study evaluating a T cell enhanced samRNA vaccine as a booster against SARS-CoV-2, is ongoing in the United Kingdom. In January, Gritstone announced positive clinical data from the first cohort and subsequently expanded the study. The data, which demonstrated both strong neutralizing antibody responses to Spike and robust CD8+ T cell responses, provided human proof of concept of the samRNA vector in viral diseases and the company’s approach to infectious disease.
The CORAL-CEPI trial is ongoing in South Africa with support from the Coalition for Epidemic Preparedness Innovations (CEPI) and is evaluating T cell enhanced omicron- and beta-spike constructs in virus-naïve, convalescent, and HIV+ patients.
The CORAL-IMMUNOCOMPROMISED trial is ongoing in the United Kingdom evaluating T cell enhanced samRNA and chimpanzee adenovirus (ChAd) vaccines in B cell deficient subjects.
The CORAL-NIH trial, which is being sponsored and executed by the National Institute of Allergy and Infectious Disease (NIAID), is ongoing in the United States evaluating T cell enhanced samRNA and/or ChAd vaccines in previously vaccinated healthy volunteers.
HIV – Collaboration with Gilead Sciences, Inc (Gilead) under Gilead’s HIV Cure Program to research and develop vaccine-based HIV immunotherapy treatment

An investigational new drug application (IND) was cleared in December 2021.
First Quarter 2022 Financial Results

Cash, cash equivalents, marketable securities and restricted cash were $186.8 million as of March 31, 2022, compared to $223.5 million as of December 31, 2021.

Research and development expenses were $28.2 million for the three months ended March 31, 2022, compared to $24.9 million for the three months ended March 31, 2021. The increase of $3.3 million for the three months ended March 31, 2022 compared to the three months ended March 31, 2021 was primarily due to increases in personnel-related expenses, outside services, and facilities related costs, offset by decreases in laboratory supplies and milestone and license payments.

General and administrative expenses were $8.0 million for the three months ended March 31, 2022, compared to $6.9 million for the three months ended March 31, 2021. The increase of $1.1 million was primarily attributable to increases in personnel-related expenses and in facilities-related costs, offset by a decrease in outside services.

Collaboration and license revenue was $4.7 million for the three months ended March 31, 2022, compared to $39.7 million for the three months ended March 31, 2021. During the three months ended March 31, 2022, we recognized $4.0 million in collaboration revenue related to the 2seventy Agreement, $0.7 million in collaboration revenue related to the Gilead Collaboration Agreement, $2.2 million in grant revenue from the CEPI Funding Agreement, and $0.2 million in grant revenue from the Gates Foundation. During the three months ended March 31, 2021, we recorded $38.6 million in license revenue and $0.3 million in collaboration revenue related to the Gilead Collaboration Agreement and $0.7 million in collaboration revenue related to the 2seventy Agreement.

On May 5, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2022 and reaffirmed its financial guidance for the year (Press release, Ultragenyx Pharmaceutical, MAY 5, 2022, View Source [SID1234613752]).

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"Product based revenue continues to grow driven by increased traction for Crysvita in Latin America," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In the clinical portfolio, we are now dosing patients in three of our four concurrent pivotal programs and are on track for updated data from the Phase 1/2 for Angelman syndrome mid-year."

First Quarter 2022 Financial Results

Net Revenues
For the first quarter of 2022, Ultragenyx reported $79.9 million in total revenue. Ultragenyx recognized $54.6 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $45.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $9.4 million. Total royalty revenue related to European Crysvita sales was $4.8 million. Dojolvi (triheptanoin) product sales in the first quarter of 2022 were $12.4 million. Mepsevii (vestronidase alfa) product sales for the first quarter of 2022 were $4.9 million.

Total revenue for the first quarter of 2022 also includes $3.2 million related to technical assistance following the successful completion of technology transfer activities with Daiichi Sankyo. This compares to total revenue in the first quarter of 2021, which includes $42.8 million related to the technology transfer services which were ongoing at the time.

Operating Expenses
Total operating expenses for the first quarter of 2022 were $216.6 million, including non-cash stock-based compensation of $29.4 million.

Net Loss
For the first quarter of 2022, Ultragenyx reported net loss of $152.3 million, or $2.19 per share basic and diluted, compared with a net loss for the first quarter of 2021 of $136.1 million, or $2.03 per share, basic and diluted. Net cash used in operations for the quarter ended March 31, 2022 was $117.5 million.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were $813.8 million as of March 31, 2022.

2022 Financial Guidance

The company continues to expect 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.

First Quarter 2022 Revenue and Selected Financial Data Tables

Recent Updates

Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): European marketing authorization transitioned to Ultragenyx, reimbursement processes initiated
The European Commission has completed transfer of the marketing authorization of Evkeeza from Regeneron. Ultragenyx is preparing reimbursement dossiers for various national health authorities in Europe. Ultragenyx received rights to commercialize and distribute Evkeeza in countries outside the U.S. in January 2022.

UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing has been initiated for the Phase 2/3 Orbit study; Phase 2 study in children under age five planned for second half of 2022
Ultragenyx has begun dosing patients in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI ages five to <26 years. A dosing update on the Phase 2 portion of the Orbit study and transition to Phase 3 is expected in the second half of 2022.

In addition, Ultragenyx intends to initiate an additional study in children with OI under age 5 years in the second half of 2022 and will continue to evaluate adult patients who were previously treated in the ASTEROID study, a Phase 2b study conducted by our partner Mereo.

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 GlucoGene study dosing patients
Dosing and enrollment of the Phase 3 study of DTX401 is ongoing. The pivotal GlucoGene study has a 48-week primary efficacy analysis period and the company plans to enroll approximately 50 patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control.

UX701 for Wilson Disease: Cyprus2+ pivotal Phase 1/2/3 study dosing patients
The company is dosing patients in the Phase 1/2 stage of the seamless Phase 1/2/3 Cyprus2+ study of UX701. During the first stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary efficacy endpoints are change in 24-hour urinary copper concentration and percent reduction in standard of care medication by Week 52.

DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 eNH3ance study expected to initiate in mid-2022
Ultragenyx expects to initiate the Phase 3 eNH3ance study of DTX301 in patients with OTC in mid-2022. The 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

GTX-102 for Angelman Syndrome: Patients continue to be treated in the Phase 1/2 study in Canada and the U.K. and under a separate protocol in the U.S.
Dosing is ongoing in cohorts 4 and 5 of the Phase 1/2 study in the U.K. and Canada, as well as for four additional patients in the U.S. under a separate protocol. To date, no treatment-related serious adverse events or lower extremity weakness adverse events have been observed in these patients. An interim update on the safety and efficacy of GTX-102 is planned for mid-2022.

UX053 for Glycogen Storage Disease Type III (GSDIII) Debrancher Deficiency: Phase 1/2 study currently dosing patients; Preliminary data from first part of study and initiation of second part of study anticipated in second half of 2022
Ultragenyx has begun to dose patients in the two-part Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of UX053 in adults age 18 years and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. Preliminary data from the Part 1 single ascending dose phase of the study is expected in the second half of the year.

1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 5, 2022, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2022 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 7951356. The replay of the call will be available for one year.

On May 5, 2022 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Celldex Therapeutics, MAY 5, 2022, View Source [SID1234613751]).

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"This quarter, we continued to focus on advancing our clinical programs and are on track to report data from our chronic spontaneous urticaria Phase 1b study early this summer," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "After successfully completing important readiness activities, including the development of a CDX-0159 subcutaneous formulation, we remain excited to initiate our Phase 2 chronic urticaria programs during the second quarter. We are well-positioned to further build on this positive momentum as we anticipate executing on several other significant key milestones across our pipeline in the year ahead."

Recent Program Highlights

CDX-0159 (also referred to as barzolvolimab) – KIT Inhibitor Program

Barzolvolimab is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

Celldex is currently completing enrollment in the Phase 1b multi-center, randomized, double-blind, placebo-controlled study of barzolvolimab in chronic spontaneous urticaria. This study is designed to assess the safety and treatment effects of multiple ascending doses of barzolvolimab in up to 40 patients with chronic spontaneous urticaria who remain symptomatic despite treatment with antihistamines. Data from this study (0.5, 1.5 and 3 mg/kg cohorts) have been submitted for a late breaking presentation at EAACI 2022.

Celldex remains on track to initiate Phase 2 studies in chronic spontaneous urticaria and chronic inducible urticaria (cold urticaria and symptomatic dermographism) in the second quarter of 2022. As previously reported, in the fourth quarter of 2021 and first quarter of this year, Celldex successfully advanced important activities to support the initiation of these studies, including the development of a barzolvolimab subcutaneous formulation and the completion of the in-life dosing portion of a six month chronic toxicology study.

In February 2022, Celldex announced that the development of barzolvolimab will be expanded into eosinophilic esophagitis, the most common type of eosinophilic gastrointestinal disease. Several studies have suggested that mast cells may be an important driver in the disease, demonstrating that the number and activation state of mast cells are greatly increased in eosinophilic esophagitis biopsies and that mast cell signatures correlate with markers of inflammation, fibrosis, pain and disease severity. Given the lack of effective therapies for eosinophilic esophagitis and barzolvolimab’s potential as a mast cell depleting agent, Celldex believes this is an important indication for future study and plans to initiate a Phase 2 trial in the fourth quarter of 2022.

Celldex continues to enroll patients in the Phase 1b multi-center, randomized, double-blind, placebo-controlled study of barzolvolimab in patients with prurigo nodularis, a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin. Enrollment also remains ongoing in the barzolvolimab Phase 1b open label study in inducible urticaria in a third cohort (single dose, 3 mg/kg) in cholinergic urticaria and a fourth cohort at a lower dose (single dose, 1.5 mg/kg) in cold urticaria.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, the monotherapy cohort, the combination cohort with CDX-301 and the safety run-in combination cohort with gemcitabine/nab-paclitaxel have been completed. In late March 2022, Celldex closed enrollment to expansion cohorts in combination with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer and non-small cell lung cancer who have progressed on checkpoint therapy. Patients in these cohorts continue to be dosed and followed for safety and potential treatment effect.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In the Phase 1 dose-escalation study of CDX-527 in patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy, enrollment to the dose escalation portion of the study has been completed and an expansion cohort in ovarian cancer is currently enrolling patients.

First Quarter 2022 Financial Highlights and 2022 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2022 were $380.5 million compared to $408.3 million as of December 31, 2021. The decrease was primarily driven by first quarter cash used in operating activities of $24.5 million. At March 31, 2022, Celldex had 46.8 million shares outstanding.

Revenues: Total revenue was $0.2 million in the first quarter of 2022 compared to $0.7 million for the comparable period in 2021. The decrease in revenue was primarily due to a decrease in services performed under our manufacturing and research and development agreements with Rockefeller University and Gilead Sciences.

R&D Expenses: Research and development (R&D) expenses were $17.1 million in the first quarter of 2022 compared to $12.7 million for the comparable period in 2021. The increase in R&D expense was primarily due to an increase in clinical trial and personnel expenses.

G&A Expenses: General and administrative (G&A) expenses were $6.9 million in the first quarter of 2022 compared to $4.1 million for the comparable period in 2021. The increase in G&A expense was primarily due to higher personnel, legal and commercial planning expenses.

Changes in Fair Value Remeasurement of Contingent Consideration: The gain on fair value remeasurement of contingent consideration was $0.5 million for the first quarter of 2022, primarily due to changes in discount rates.

Net Loss: Net loss was $23.1 million, or ($0.49) per share, for the first quarter of 2022, compared to a net loss of $16.5 million, or ($0.42) per share, for the comparable period in 2021.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2022 are sufficient to meet estimated working capital requirements and fund planned operations through 2025.

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

On May 5, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that it will host a conference call and live audio webcast on Thursday, May 12, 2022 at 4:30 p.m. Eastern Time to discuss financial results for the first quarter ended March 31, 2022 and to provide a business update (Press release, CymaBay Therapeutics, MAY 5, 2022, View Source [SID1234613750]).

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Conference Call Details
To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID#13728967. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On May 5, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported first quarter 2022 financial results and corporate updates (Press release, Iovance Biotherapeutics, MAY 5, 2022, View Source [SID1234613749]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Iovance had a positive start to the year across our lead program lifileucel in metastatic melanoma as well as our growing TIL pipeline. We recently reported favorable feedback from the FDA on our potency assays and assay matrix, which brings us a step closer to our planned BLA submission for lifileucel in metastatic melanoma. We continue to enroll patients in clinical studies to investigate our TIL therapies in multiple solid tumors, with plans to initiate a Phase 3 clinical trial of lifileucel in combination with pembrolizumab in frontline melanoma. In addition, the FDA has allowed an IND to proceed with a clinical trial of our PD-1 inactivated, gene-edited TIL therapy, IOV-4001. Our TIL platform, clinical data, and people are a solid foundation to establish TIL as the next class of paradigm-shifting therapy for cancer patients with significant unmet need."

First Quarter 2022 Highlights and Recent Corporate Updates

Regulatory

Iovance TIL therapy (lifileucel) in metastatic melanoma (post-anti-PD-1): Iovance received positive feedback from the U.S. Food and Drug Administration (FDA) on both its potency assay matrix and its proprietary cell co-culture assay included in the potency assay matrix. Iovance expects to request a pre-BLA meeting in July 2022 and to complete a BLA submission for lifileucel by August 2022.

IOV-4001 (PD-1 inactivated TIL therapy) Investigational New Drug (IND) Application: The FDA allowed an IND to proceed for Iovance’s first genetically modified TIL therapy, IOV-4001, for the treatment of previously treated advanced melanoma or metastatic non-small cell lung cancer (mNSCLC). IOV-4001 leverages the gene editing TALEN technology licensed from Cellectis to inactivate PD-1 expression. A clinical trial of IOV-4001 is expected to begin in 2022.

Clinical

Iovance TIL therapy (lifileucel) in frontline (anti-PD-1 naïve) metastatic melanoma:
Updated clinical data (Cohort 1A in the IOV-COM-202 trial, n=12): Updated clinical data announced in April 2022 demonstrated an overall response rate (ORR) of 67% for lifileucel in combination with pembrolizumab. Eight out of 12 patients had a confirmed objective response, including three complete responses and five partial responses.
Frontline melanoma strategy: Iovance plans to open a Phase 3 trial of lifileucel in combination with pembrolizumab in frontline metastatic melanoma in late 2022. The FDA previously granted Fast Track Designation for lifileucel in combination with pembrolizumab for the treatment of immune checkpoint inhibitor naïve metastatic melanoma.

Iovance TIL therapy (LN-145) in second-line mNSCLC:
Enrollment is ongoing at more than 30 active clinical sites in the U.S., Canada and Europe for the IOV-LUN-202 trial of LN-145 in patients with mNSCLC. A Trial in Progress (TIP) poster on IOV-LUN-202 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting featured updated eligibility criteria to broaden enrollment in reflection of the unmet need in mNSCLC.
Iovance is engaged in discussions with the FDA about the potential for IOV-LUN-202 to serve as a registrational trial for LN-145 in mNSCLC and intends to execute an updated regulatory strategy based on this dialogue and feedback.

Lifileucel in cervical cancer: Iovance is engaged in regulatory discussions about a potential BLA for lifileucel in cervical cancer and intends to execute an updated registrational strategy based on FDA dialogue and feedback.

Next-Generation Research Programs

Data presentations:
AACR 2022 Annual Meeting: A poster highlighting preclinical data for IOV-4001 demonstrated that anti-tumor activity of IOV-4001 was superior to non-edited TIL product whether alone or in combination with an anti-PD-1 antibody in a murine model of melanoma.
2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings: Research posters described TIL products manufactured from cryopreserved tumor samples shipped from Australia and a potential approach to optimize TIL memory-like phenotype and increase functionality during the manufacturing process.

Additional updates:
Several additional targets for genetic modification using the TALEN technology, including double genetic knock-out programs, are advancing in preclinical development.
Additional research and preclinical studies of next generation TIL therapies and related technologies include approaches to increase TIL potency using CD39/69 double negative TILs and gene knock-in targets as well as development of a novel interleukin-2 (IL-2) analog (IOV-3001).

Manufacturing

The Iovance Cell Therapy Center (iCTC) was awarded an Honorable Mention by the International Society for Pharmaceutical Engineering (ISPE) in the 2022 Facility of the Year Awards.
Corporate

Cash position of $516.0 million at March 31, 2022 is expected to be sufficient into 2024.

Iovance currently owns more than 40 granted or allowed U.S. and international patents for 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. More information on Iovance’s patent portfolio can be found on the Intellectual Property page on www.iovance.com.
First Quarter 2022 Financial Results

Iovance had $516.0 million in cash, cash equivalents, investments and restricted cash at March 31, 2022, compared to $602.1 million at December 31, 2021. The cash position is expected to be sufficient to fund current and planned operations into 2024.

Jean-Marc Bellemin, Chief Financial Officer of Iovance, said, "With late-stage clinical assets in our pipeline, as well as a strong balance sheet and investments focused on launch preparations, we are well positioned to execute our mission to innovate, develop and deliver TIL therapy for patients with cancer while enhancing shareholder value."

Net loss for the first quarter ended March 31, 2022, was $91.6 million, or $0.58 per share, compared to a net loss of $75.4 million, or $0.51 per share, for the first quarter ended March 31, 2021.

Research and development expenses were $68.3 million for the first quarter ended March 31, 2022, an increase of $12.4 million compared to $55.9 million for the first quarter ended March 31, 2021. The increase in research and development expenses in the first quarter 2022 over the prior year period was primarily attributable to growth of the internal research and development team, including stock-based compensation expense, as well as facility-related costs.

General and administrative expenses were $23.4 million for the first quarter ended March 31, 2022, an increase of $3.8 million compared to $19.6 million for the first quarter ended March 31, 2021. The increase in general and administrative expenses in the first quarter 2022 compared to the prior year period was primarily attributable to growth of the internal general and administrative and commercial teams, including stock-based compensation expense, facility-related costs associated with the build out of the new corporate headquarters, increases in intellectual property filing and legal expenses and enhancements to the information technology infrastructure.

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

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss first quarter 2022 financial results and corporate updates. The conference call dial-in numbers are 1 (844) 646-4465 (domestic) or 1 (615) 247-0257 (international), the conference ID is #4655146. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.