On August 1, 2024 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported the following update of the expansion of clinical sites into Spain (Press release, Greenwich LifeSciences, AUG 1, 2024, View Source [SID1234645239]).

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As previously announced, the Company has partnered with GEICAM, the largest academic breast cancer research network in Spain, where 38 hospitals have agreed to participate in FLAMINGO-01.

To date, approximately 30 out of 38 sites have conducted site initiation visits (SIVs). An SIV is followed by site activation, HLA type screening, and study treatment. To date approximately 19 sites have been activated. Multiples sites have begun screening patients for HLA type and have started treating patients. The Company has visited and trained in-person at approximately 26 out of the 38 sites to ensure optimal treatment at each site. In the coming months, GEICAM hopes to complete the remaining SIVs and the Company hopes to visit the remaining sites.

"It has been an honor to meet with the excellent study staff, investigators and GEICAM staff across Spain. Their expertise and interest in FLAMINGO-01 is encouraging for the success of the study. We look forward to seeing patient recruitment increase in Spain as other countries in the European Union become active," remarked Jaye Thompson, VP of Clinical and Regulatory Affairs.

CEO Snehal Patel commented, "The remarkable progress in Spain, in just May through July, is a testament to the commitment of GEICAM and our team to collaborate and to train the sites together, which we hope will lead to high quality treatment of patients. With just these 30 sites, we have approximately doubled the number of sites that we have opened in the US."

The Company may provide updates on the status of other countries in Europe as the clinical trial is expanded to up to 150 sites in the US, Spain, Italy, France, Germany, and Poland.

About GEICAM

Founded in 1995, GEICAM is a not-for-profit organization leading academic breast cancer research in Spain. It has conducted more than 140 studies involving more than 67,000 women and men and is comprised of more than 900 experts based in over 200 Spanish hospitals. Its mission is to promote independent clinical, epidemiological, and translational research in oncology, with a multidisciplinary approach and under quality criteria, to improve health outcomes, as well as prevention, medical education, and the dissemination of the knowledge of this disease to patients and general society. More information on GEICAM can be found at View Source

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US clinical sites from university-based hospitals and cooperative networks with plans to expand into Europe and to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients will be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types will be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On August 1, 2024 Instil Bio, Inc. (Nasdaq: TIL, "Instil") and ImmuneOnco Biopharmaceuticals (Shanghai) Inc. (HKEX Code: 1541.HK, "ImmuneOnco"), reported a definitive agreement pursuant to which Instil is in-licensing ex-China development and commercial rights to ImmueOnco’s proprietary PD-L1xVEGF bispecific antibody, IMM2510, as well as its next-generation anti-CTLA-4 antibody, IMM27M (Press release, Instil Bio, AUG 1, 2024, View Source [SID1234645261]).

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IMM2510 is a novel, potentially best-in-class bispecific antibody consisting of an anti-PD-L1 antibody fused to a vascular endothelial growth factor (VEGF) receptor "trap" which binds VEGF. IMM2510 is differentiated from other PD(L)1xVEGF antibodies by its ability to bind multiple VEGF receptor ligands beyond VEGF-A, a smaller molecular weight allowing for potentially better tumor penetration, and enhanced antibody-dependent cellular cytotoxicity (ADCC) designed to improve tumor killing. IMM2510 has completed a dose-escalation clinical trial for advanced solid tumors and demonstrated multiple responses including patients with squamous non-small cell lung cancer (NSCLC) who previously failed PD-1 inhibitors.

IMM27M is a next-generation anti-CTLA-4 antibody with enhanced ADCC activity, which has been designed to promote intratumoral regulatory T cell depletion to enhance the efficacy and reduce the toxicity associated with first-generation anti-CTLA-4 antibodies. IMM27M has completed a dose-escalation clinical trial demonstrating anti-tumor activity in patients with advanced solid tumors and has entered combination studies with IMM2510 in China in July 2024.

Terms of License and Collaboration Agreement
Under the terms of the agreement, a wholly owned subsidiary of Instil will receive global development and commercialization rights for IMM2510 and IMM27M outside of Greater China, while ImmuneOnco will retain development and commercialization rights in Greater China including Taiwan, Macau, and Hong Kong. ImmuneOnco will receive an upfront payment and potential near-term payments of up to $50 million as well as potential additional development, regulatory, and commercial milestones exceeding $2 billion plus single digit to low double-digit percentage royalties on global ex-China sales.

On August 1, 2024 Moderna, Inc. (NASDAQ:MRNA) reported financial results and provided business updates for the second quarter of 2024 (Press release, Moderna Therapeutics, AUG 1, 2024, View Source/news/news-details/2024/Moderna-Reports-Second-Quarter-2024-Financial-Results-and-Provides-Business-Updates/default.aspx" target="_blank" title="View Source/news/news-details/2024/Moderna-Reports-Second-Quarter-2024-Financial-Results-and-Provides-Business-Updates/default.aspx" rel="nofollow">View Source [SID1234645279]).

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"During the second quarter, we marked the approval of our second mRNA product and significantly lowered our operating costs. We remain focused on execution for the 2024-25 COVID season and the launch of our RSV vaccine in the U.S.," said Stéphane Bancel, Chief Executive Officer of Moderna. "With continued positive Phase 3 data across our respiratory portfolio, we are using our mRNA platform to address significant unmet medical needs and advance public health. Our platform is poised to reach millions globally this year and we are excited by its continued positive impact on patients."

Recent progress includes:

Commercial Updates

COVID-19: The Company reported $184 million in Spikevax (COVID-19 vaccine) sales in the second quarter of 2024, which includes $162 million of U.S. sales and $22 million of international sales.

Contracting is mostly complete in a highly competitive COVID market. Moderna is ready for the upcoming vaccination season with ample and timely supply. In the U.S., the majority of contracts are finalized and Moderna’s focus is on working with public health officials, health care providers and pharmacies to drive vaccination coverage rates to reduce the substantial burden of COVID-19. In the European Union (EU), Moderna is negotiating a tender framework agreement to provide market access; however, based on recent discussions, the Company now expects very low EU sales in 2024. In the Rest of World, the Company has multiple signed contracts in place, with some potential revenue deferrals into 2025.

RSV: Following U.S. Food and Drug Administration (FDA) approval in May and the Advisory Committee on Immunization Practices (ACIP) recommendation in June, Moderna’s RSV vaccine, mRESVIA, has launched in the U.S. and deliveries are underway as of July 2024. The Company is targeting the pharmacy segment in the U.S. and its commercial focus is on reaching vaccinators in a highly competitive market. Additionally, the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization in the EU for mRESVIA.

Second Quarter 2024 Financial Results

Revenue: Total revenue for the second quarter of 2024 was $241 million, compared to $344 million in the same period in 2023. This decline was primarily attributable to decreased sales of the Company’s COVID-19 vaccine. Net product sales for the second quarter of 2024 were $184 million, reflecting a 37% decrease compared to the same period in 2023. This reduction aligns with the expected shift to a seasonal COVID-19 vaccine market, with higher demand anticipated in the fall and winter. In the prior year period, product sales were largely recognized from doses delivered and deferred from 2022.

Cost of Sales: Cost of sales for the second quarter of 2024 was $115 million, which included third-party royalties of $10 million, unutilized manufacturing capacity and wind-down costs of $55 million, and inventory write-downs of $14 million. Compared to the same period in 2023, the cost of sales decreased by $616 million, or 84%. Cost of sales as a percentage of net product sales was 62% for the second quarter of 2024, down from 249% in the second quarter of 2023. The reduction in cost of sales was primarily due to a lower sales volume and decreased unutilized manufacturing capacity, inventory write-downs, and losses on firm purchase commitments and related cancellation fees. The reduction in cost of sales as a percentage of net product sales in 2024 was mainly driven by lower costs, partially offset by the reduced sales volume, reflecting a decline in product demand and increased product seasonality.

Research and Development Expenses: Research and development expenses for the second quarter of 2024 increased by 6% to $1.2 billion, compared to the second quarter in 2023. This increase was mainly due to higher personnel-related costs, driven by an increased headcount to support ongoing research and development efforts. Research and development expenses for the quarter also include the purchase of a priority review voucher. The increase was partly offset by a decrease in clinical development and manufacturing expenses, resulting from lower spending on clinical trials for the Company’s COVID-19 and seasonal flu programs, in line with its planned trial schedules.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for the second quarter of 2024 decreased by 19% to $268 million, compared to the second quarter in 2023. This decrease is a result of cost discipline and the efficiencies resulting from investments the Company made in foundational capabilities over the last year, which allowed for a significant reduction of purchased services and the use of external consultants. The Company continues to invest in digital and commercial capabilities and has intensified its focus on building and utilizing AI technologies to further streamline operations and enhance productivity.

Income Taxes: The Company did not recognize an income tax expense for the second quarter of 2024, in contrast to an income tax benefit of $369 million in the same period last year. The shift primarily resulted from the continued application of a valuation allowance on the majority of the Company’s deferred tax assets, first established in the third quarter of 2023. The Company only maintained a valuation allowance on certain state deferred tax assets prior to the third quarter of 2023.

Net Loss: Net loss was $(1.3) billion for the second quarter of 2024, compared to $(1.4) billion for the second quarter of 2023.

Net Loss Per Share: Net loss per share was $(3.33) for the second quarter of 2024, compared to $(3.62) for the second quarter of 2023.

Cash Position: Cash, cash equivalents and investments as of June 30, 2024, were $10.8 billion, compared to $12.2 billion as of March 31, 2024. The decrease in the cash position during the second quarter of 2024 was largely attributable to research and development expenses and operating activities.

2024 Financial Framework

Net Sales: The Company revises its 2024 expected net product sales to $3.0 to $3.5 billion from its respiratory franchise. For the second half of the year, it expects a sales split of 40-50% in the third quarter with the balance in the fourth quarter of 2024, subject to the timing of regulatory approvals. The update in product sales is driven by three primary factors: very low EU sales in 2024, potential revenue deferrals for certain international sales into 2025, and an increasingly competitive environment for respiratory vaccines in the U.S.

Cost of Sales: Cost of sales is expected to be in the range of 40-50% of product sales for the year.

Research and Development Expenses: Full-year 2024 research and development expenses are anticipated to be approximately $4.5 billion.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for 2024 are projected to be approximately $1.3 billion.

Income Taxes: The Company continues to expect its full-year tax expense to be negligible.

Capital Expenditures: Capital expenditures for 2024 are expected to be approximately $0.9 billion.

Cash and Investments: Year-end cash and investments for 2024 are projected to be approximately $9 billion.

Recent Progress and Upcoming Late-Stage Pipeline Milestones

Respiratory vaccines:

Respiratory syncytial virus (RSV) vaccine: After Moderna received U.S. FDA approval for its RSV vaccine (mRNA-1345), ACIP issued a recommendation for all unvaccinated people 75 years of age and older and unvaccinated people ages 60-74 who are at increased risk for RSV to receive the vaccine for the prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD). Additionally, the EMA adopted a positive opinion recommending marketing authorization in the EU for mRESVIA. The Company is awaiting regulatory approvals in additional countries.

Seasonal flu vaccine: Moderna’s seasonal flu vaccine (mRNA-1010) demonstrated consistently acceptable safety and tolerability across three Phase 3 trials. The Company is engaging with regulators and intends to file in 2024.

Next-generation COVID-19 vaccine: A recent announcement of positive interim results from the NEXTCove Phase 3 trial showed that Moderna’s next-generation COVID-19 vaccine (mRNA-1283) has met its primary efficacy endpoint, demonstrating non-inferior vaccine efficacy against COVID-19 compared to Spikevax (mRNA-1273) in participants 12 years of age and older. Higher efficacy was also observed compared to Spikevax in adults 18 years of age and older. The Company is engaging with regulators and intends to file in 2024.

Seasonal flu + COVID vaccine: Moderna recently announced the Phase 3 trial of its combination vaccine against seasonal flu and COVID-19 (mRNA-1083) has met its primary endpoints, eliciting higher immune responses against influenza virus and SARS-CoV-2 than licensed flu and COVID vaccines in adults 50 years and older, including an enhanced influenza vaccine in adults 65 years and older. The Company is engaging with regulators on next steps.

Oncology therapeutics:

Individualized Neoantigen Therapy (INT): Moderna and Merck announced additional 3-year data for mRNA-4157 in combination with KEYTRUDA (pembrolizumab), demonstrating sustained improvement in recurrence-free survival and distant metastasis-free survival versus KEYTRUDA in patients with high-risk stage III/IV melanoma following complete resection. At a median planned follow-up of the Phase 2b study at 34.9 months, mRNA-4157 in combination with KEYTRUDA reduced the risk of recurrence or death by 49% and the risk of distant metastasis or death by 62% compared to KEYTRUDA alone in these patients. The 2.5-year recurrence-free survival rate of mRNA-4157 in combination with KEYTRUDA was 74.8% as compared to 55.6% for KEYTRUDA alone, with the benefit observed across exploratory subgroups.

Rare disease therapeutics:

Methylmalonic acidemia (MMA) therapeutic: Moderna’s investigational therapeutic for MMA (mRNA-3705) has been selected by the U.S. FDA for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. mRNA-3705 was chosen by the Center for Biologics Evaluation and Research (CBER) as one of four investigational medicines for accelerated development to address unmet medical needs for rare diseases. The Company expects to advance its MMA program into a registrational study in 2024.

Moderna Corporate Updates

Announced that David M. Rubenstein, Co-Founder and Co-Chairman of The Carlyle Group, will join Moderna’s Board of Directors, effective August 5, 2024, when directors Robert Langer and Stephen Berenson will retire from the Board.

Entered into an agreement with Mitsubishi Tanabe Pharma Corporation to promote Moderna’s mRNA respiratory vaccine portfolio in Japan.

Received a project award of $176 million through BARDA’s Rapid Response Partnership Vehicle Consortium to accelerate the development of an mRNA-based pandemic influenza vaccine.

Published third annual ESG Report, Impacting Human Health, on June 25, 2024.

Company Accolades

Moderna was named to the Boston Business Journal’s annual list of the Most Charitable Companies in Massachusetts (second consecutive year)

Moderna was recognized as a top-scoring company on Disability:IN’s Disability Equality Index and a Best Place to Work for Disability Inclusion (third consecutive year)

Moderna was recognized as a Great Place to Work in the U.S. by Great Place To Work (second consecutive year)

Key 2024 Investor and Analyst Event Dates

R&D Day: September 12

Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on August 1, 2024. To access the live conference call via telephone, please register at the link below. Once registered, dial-in numbers and a unique pin number will be provided. A live webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website.

Telephone: https://register.vevent.com/register/BI2ef72dcc8f6e4477bf2f61ab92054abb

Webcast: View Source

The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.

On August 1, 2024 ARCA biopharma, Inc. (Nasdaq: ABIO), (the "Company") a biopharmaceutical company applying a precision medicine approach to developing genetically targeted therapies for cardiovascular diseases, reported second quarter 2024 financial results and provided a corporate update (Press release, Arca biopharma, AUG 1, 2024, View Source [SID1234646051]).

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In April 2022, ARCA established a Special Committee of the board of directors (the "Board") of ARCA to conduct a comprehensive review of strategic alternatives. As part of the strategic review process, the Company explored potential strategic alternatives that included, without limitation, an acquisition, merger, business combination or other transactions. The Company has and is continuing to explore strategic alternatives related to its product candidates and related assets, including, without limitation, licensing transactions and asset sales.

On April 3, 2024, following a comprehensive review of strategic alternatives, the Company, Atlas Merger Sub Corp., a Delaware corporation and a wholly-owned subsidiary of ARCA ("Merger Sub I"), Atlas Merger Sub II LLC, a Delaware limited liability company and a wholly-owned subsidiary of ARCA ("Merger Sub II") and Oruka Therapeutics, Inc., a Delaware corporation ("Oruka"), entered into an Agreement and Plan of Merger and Reorganization (the "Merger Agreement"), pursuant to which, among other matters, and subject to the satisfaction or waiver of the conditions set forth in the Merger Agreement, Merger Sub I will merge with and into Oruka, with Oruka continuing as a wholly owned subsidiary of ARCA and the surviving corporation of such merger (the "First Merger") and as part of the same overall transaction, the surviving corporation in the First Merger will merge with and into Merger Sub II with Merger Sub II continuing as a wholly owned subsidiary of ARCA and the surviving entity of such merger (the "Second Merger" and together with the First Merger, the "Merger"). The Merger is intended to qualify for federal income tax purposes as a tax-free reorganization under the provisions of Section 368(a) of the Internal Revenue Code of 1986, as amended.

Additional descriptions about the Merger Agreement and related agreements were previously disclosed on a Current Report on Form 8-K filed with the U.S. Securities and Exchange Commission (the "SEC") on April 3, 2024. In connection with the proposed Merger, the Company has filed relevant materials with the SEC, including a registration statement on Form S-4 that contains a definitive proxy statement and prospectus of the Company.

In connection with the Merger, the Company will dispose of (or is in the process of disposing of) its legacy technology and intellectual property, including those related to Gencaro (bucindolol hydrochloride) and Recombinant Nematode Anticoagulant Protein c2 ("rNAPc2"). Any such disposal of legacy technology and intellectual property will be contingent upon obtaining stockholder approval for the Merger and is expected to occur immediately prior to or concurrently with the closing of the Merger. In the event that the Company shall enter into an agreement for any such sale or other disposition of its legacy assets at or prior to the closing of the Merger, the net proceeds received at or prior to the closing of the Merger will be included in the calculation of the net cash of the Company as of the closing.

The Company’s future operations are highly dependent on the success of the Merger and there can be no assurances that the Merger will be successfully consummated. In the event that the Company does not complete the Merger, the Company may explore strategic alternatives, including, without limitation, another strategic transaction and/or pursue a dissolution and liquidation of the Company.

Second Quarter 2024 Summary Financial Results

Cash and cash equivalents were $33.3 million as of June 30, 2024, compared to $37.4 million as of December 31, 2023. ARCA believes that its current cash and cash equivalents, consisting primarily of money market funds, will be sufficient to fund its operations through the end of 2025. Our future viability beyond that point is dependent on the results of the strategic review process and our ability to raise additional capital to fund our operations. We expect to continue to incur costs and expenditures in connection with the process of evaluating strategic alternatives. There can be no assurance, however, that we will be able to successfully consummate any particular strategic transaction, including the Merger. The process of continuing to evaluate these strategic options may be very costly, time-consuming and complex and we have incurred, and may in the future incur, significant costs related to this continued evaluation, such as legal, accounting and advisory fees and expenses and other related charges.

General and administrative (G&A) expenses were $3.0 million for the quarter ended June 30, 2024, compared to $1.7 million for the corresponding period in 2023, an increase of approximately $1.3 million. During the three months ended June 30, 2024, we recorded $370,000 for one-time termination benefits related to the separation of Dr. Michael Bristow, the former Chief Executive Officer of ARCA, effective April 3, 2024. The increase for the three month period was primarily the result of a $0.9 million increase in professional fees primarily related to the Merger Agreement discussed above and $0.4 million higher one-time termination benefits from the termination discussed above in 2024. G&A expenses in 2024 are expected to be higher than those in 2023 as we incur professional fees related to the Merger Agreement discussed above and maintain administrative activities to support our ongoing operations. We expect to incur significant costs related to our exploration of strategic alternatives and the Merger, including legal, accounting and advisory expenses and other related charges.

Research and development (R&D) expenses were $0.1 million for the quarter ended June 30, 2024, compared to $0.3 million for the corresponding period in 2023. Of the $0.2 million decrease in R&D expenses in the second quarter of 2024 as compared to the second quarter of 2023, $0.1 million was primarily related to decreased headcount and $0.1 million was primarily related to the unrestricted research grants with ARCA’s former President and Chief Executive Officer’s academic research laboratory at the University of Colorado. There was no expense under these arrangements for the three months ended June 30, 2024. Total expense under these arrangements for the three months ended June 30, 2023 was $0.1 million. In December 2023, the Company made a payment of $125,000 for the grant period July 2022 through December 2023 under these arrangements. As discussed above, the former President and Chief Executive Officer resigned in April 2024. R&D expense in 2024 is expected to be lower than 2023 while we explore strategic alternatives. Should we resume clinical trials of product candidates, we expect research and development costs to increase significantly for the foreseeable future as our product candidate development programs progress.

Total operating expenses for the quarter ended June 30, 2024 were $3.1 million compared to $2.0 million for the second quarter of 2023.

Net loss for the quarter ended June 30, 2024 was $2.7 million, or $0.18 per basic and diluted share, compared to $1.5 million, or $0.10 per basic and diluted share in the second quarter of 2023.

On August 1, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, gene insertion, and gene excision, reported financial results for the second quarter ended June 30, 2024 and provided a business update (Press release, Precision Biosciences, AUG 1, 2024, View Source [SID1234645240]).

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"During the first half of 2024 we have made excellent progress against our key priorities. We rapidly advanced PBGENE-HBV toward planned investigational new drug (IND) submission and/or clinical trial application (CTA) in 2024. We believe we are in a strong position to advance our first wholly owned in vivo gene editing program into the clinic. PBGENE-HBV is expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to provide a functional cure for chronic Hepatitis B by eliminating the root source of viral replication. I’m proud of our team’s operational progress this year, including nearing completion of final toxicology studies, production of final clinical trial material, onboarding global clinical study sites in multiple countries to conduct the Phase 1 trial, and adding world-class clinicians to our Hepatitis Scientific Advisory Board," said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences.

"While advancing PBGENE-HBV as our first operational priority in 2024, we have also made steady progress on our second wholly owned program PBGENE-PMM for 3243 mutated (m.3243) mitochondrial disease, which is targeted for IND and/or CTA filing in 2025."

"As we enter the second half of the year, we are sufficiently capitalized based on our expected cash runway as a result of multiple business development deals combined with a $40 million equity raise to propel our two wholly owned programs to Phase 1 data readouts in 2025 and 2026," emphasized Mr. Amoroso.

Wholly Owned Portfolio

PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic Hepatitis B. Currently, it is estimated that approximately 300 million people worldwide are afflicted with chronic Hepatitis B. PBGENE-HBV is expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA.

In June 2024, Precision participated in a panel discussion on the application of gene editing to the treatment of chronic Hepatitis B during the HBV Forum and presented new preclinical safety data for PBGENE-HBV at the European Association for the Study of the Liver. This data further supported the ability of PBGENE-HBV to specifically target and cut HBV DNA, leading to the elimination of cccDNA and integrated HBV DNA. The data also demonstrated a lack of detectable off-target editing for PBGENE-HBV at therapeutically relevant doses, including no editing-associated translocations in HBV infected primary human hepatocytes. Importantly, new non-human primate data showed that PBGENE-HBV was well-tolerated across multiple dose administrations. PBGENE-HBV is advancing through final toxicology studies and drug for the planned Phase 1 clinical trial has been manufactured. Precision expects to submit an IND and/or CTA for this program in 2024 and plans to provide an additional program update later this year.

PBGENE-PMM (Mutant Mitochondrial DNA Elimination Program): PBGENE-PMM is a first of its kind potential treatment for m.3243-mitochondrial disease designed to target mutant mitochondrial DNA while having no adverse impact on wild type (normal) mitochondrial DNA. Mitochondrial diseases are the most common hereditary metabolic disorder in the world. The m.3243 mitochondrial disease population that the program intends to address is large, affecting approximately 20,000 people in the US alone. The highly specific ARCUS nuclease is designed to shift heteroplasmy by editing and eliminating mutant mitochondrial DNA while allowing wild type mitochondrial DNA to repopulate in the mitochondria, thus improving cellular function. Unlike CRISPR/Cas, base editors, and prime editors, ARCUS single-component nucleases do not require a guide RNA and are therefore differentiated amongst gene editing modalities due to their ability to penetrate mitochondrial membranes.

In June 2024, Precision presented additional data from the PBGENE-PMM program at the United Mitochondrial Disease Foundation Mitochondrial Medicine 2024 Conference. The presentation highlighted the ability of PBGENE-PMM to localize exclusively to mitochondria, avoiding any detectable off-target editing in the nuclear genome, while generating substantial shifts in heteroplasmy and improvements in mitochondrial function. The Company anticipates filing an IND and/or CTA for this program in 2025.

Wholly Owned Portfolio – Under Assessment

In July 2024, Precision regained control of three programs developed under its collaboration with Prevail Therapeutics Inc. The Company has received inbound interest from potential partners regarding these programs and is in the process of conducting a portfolio assessment for these returned programs for internal development and/or development through new partners and expects to provide an update as decisions are finalized. These programs include:

PBGENE-DMD – novel gene excision approach for treatment of Duchenne Muscular Dystrophy utilizing a pair of ARCUS nucleases, delivered by a single adeno-associated virus (AAV), that are designed to excise an approximately 500,000 base pair mutation "hot spot" region from the dystrophin gene to restore a functionally competent variant of the native dystrophin protein. We believe this approach is unique when compared with microdystrophin treatment and is the first in class gene editing application for Duchenne Muscular Dystrophy.
PBGENE-LIVER – liver target for gene insertion with data demonstrating that ARCUS achieved 40% to 45% high efficiency and durable gene insertion at 1- and 3-months in nondividing cells in adult nonhuman primates (NHPs), the most challenging context for gene insertion. To our knowledge, ARCUS is the only gene editor presented at a conference showing high efficiency gene insertion in non-dividing cells in NHPs. This opens therapeutic application for both pediatric patients whose cells divide quickly, as well as adult patients whose cells divide much less frequently than pediatric patients.
PBGENE-CNS – gene editing program targeting neurons to address a disease of the central nervous system. Precision is the first to demonstrate successful in vivo editing of neurons in both mice and NHPs. This remains a very attractive program for Precision or for partners focused on neurodegenerative diseases.
Partnered Programs:

iECURE-OTC (Gene Insertion Program for OTC deficiency): Led by iECURE, ECUR-506 is the first ARCUS-mediated in vivo gene editing program to advance into the clinic following regulatory approvals in the US, the United Kingdom, and Australia for initiation of the OTC-HOPE study. The OTC-HOPE study is a first-in-human Phase 1/2 trial evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency and has begun recruiting patients at two sites in the United Kingdom. In May 2024, iECURE announced that it had received Fast Track designation from the FDA for ECUR-506 and expects initial data from this trial to be available in late 2024 or in 2025.

PBGENE-NVS (Gene Insertion Program for Sickle Cell Anemia and Beta Thalassemia): Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic transgene as a potential one-time transformative treatment. We believe this is the only in vivo approach in development that can be administered directly to the patient to overcome disparities in patient treatment access with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach.

Corporate Updates

Expansion of Hepatitis Scientific Advisory Board to include world-class clinical investigators: In June 2024, Precision announced the appointment of Mark Sulkowski, M.D. and Jordan Feld, M.D., M.P.H. to its Hepatitis Scientific Advisory Board. They join inaugural member Raymond Schinazi, Ph.D., DSc, to provide counsel and deepen the Company’s scientific and clinical expertise ahead of Precision’s anticipated IND and/or CTA submission for PBGENE-HBV.

Addition to the Russell Microcap Index: As of the close of U.S. markets on June 28, 2024, Precision BioSciences was added to the Russell Microcap Index as part of the index’s annual reconstitution.

Common Stock purchase by members of management for $300,000: In May 2024, Precision entered into a definitive subscription agreement, pursuant to which the Company issued and sold in a non-brokered private placement to members of its senior leadership team, including the Chief Executive Officer, 25,000 shares of its common stock at a price of $12.00 per share, representing a 13.5% premium to the closing price of its common stock immediately preceding the signing of the subscription agreement, for an aggregate amount of $300,000.

Banc of California Loan and Security Agreement: On July 31, 2024, the Company entered into an amended and restated loan and security agreement (the 2024 Loan and Security Agreement) with Banc of California (formerly known as Pacific Western Bank) pursuant to which Banc of California provided the Company with a term loan with a principal amount of $22.5 million. The proceeds from the term loan were used to repay the $22.5 million outstanding principal balance under the prior revolving line of credit with Banc of California, and pursuant to the terms of the 2024 Loan and Security Agreement, the revolving line of credit was terminated. The maturity date under the 2024 Loan and Security Agreement is June 30, 2027. The term loan bears interest at an annual rate equal to the greater of (i) 1.50% below the Prime Rate then in effect or (ii) 4.50%.

Quarter Ended June 30, 2024 Financial Results:

Cash and Cash Equivalents: As of June 30, 2024, Precision had approximately $123.6 million in cash and cash equivalents. Existing cash and cash equivalents, upfront and potential near-term cash from CAR T transactions, along with expected operational receipts, continued fiscal and operating discipline, availability of Precision’s at-the-market (ATM) facility, and available credit are expected to extend Precision’s cash runway into the second half of 2026.

Revenues: Total revenues for the quarter ended June 30, 2024 were $49.9 million, as compared to $19.8 million for the same period in 2023. The increase of $30.1 million in revenue during the quarter ended June 30, 2024 was primarily the result of recognizing deferred revenue related to the termination of the amended and restated development and license agreement with Prevail Therapeutics.

Research and Development Expenses: Research and development expenses were $17.2 million for the quarter ended June 30, 2024, as compared to $13.1 million for the same period in 2023. The increase of $4.1 million was primarily due to an increase in external development costs for the PBGENE-HBV and PBGENE-PMM programs as they continue to advance towards the clinic.

General and Administrative Expenses: General and administrative expenses were $8.5 million for the quarter ended June 30, 2024, as compared to $9.8 million for the same period in 2023. The decrease of $1.3 million was primarily due to decreased employee and share-based compensation expense from a decrease in headcount.

Net Income from Continuing Operations: Net income from continuing operations was $32.7 million for the quarter ended June 30, 2024, inclusive of a $7.8 million non-cash gain on the fair value of our warrant liability which does not impact our cash runway, as compared to a net loss from continuing operations of $11.9 million, for the same period in 2023. The improvement was primarily related to recognition of deferred revenue from termination of the Prevail Agreement as well as the non-cash gain related to the fair value adjustments of our warrant liability.

Net Income: Net income was $32.7 million, or $4.70 per share (basic) and $4.67 per share (diluted), for the quarter ended June 30, 2024, as compared to a net loss of $11.9 million, or $(3.13) per share (basic and diluted), for the same period in 2023.

Shares: Basic and diluted weighted-average common shares outstanding for the second quarter of 2024 were 6,966,680 and 7,011,630, respectively, compared to 3,803,083 (basic and diluted) for the same period in 2023.