Precision BioSciences Reports Second Quarter 2024 Financial Results and Provides Business Update

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.

Greenwich LifeSciences Provides Update on Expansion of Flamingo-01 into Spain

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.