PRECISION BIOSCIENCES INC
For the period ended Mar 31, 2025. Filed May 15, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 15, 2025
DTIL$10.60+7.4%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-04.
0000950170-24-120279
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-15.
0000950170-25-071991
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 152 previous paragraphs and 135 current paragraphs.
72 unchanged; 27 reworded; 32 added; 46 removed; 7 previous and 4 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (32)
Current report
PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B (“HBV”) infection. PBGENE-HBV is 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, the root cause chronic Hepatitis B. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B.
Current report
In February 2025, we announced initial results from the first administration of PBGENE-HBV in cohort 1, the lowest dose level of the ELIMINATE-B trial. PBGENE-HBV was safe and well tolerated in all three participants in cohort 1 after the first administration of a 0.2 mg/kg dose. In addition to safety, PBGENE-HBV demonstrated a substantial reduction in Hepatitis B surface antigen (“HBsAg”) in two of the three participants following the first administration at the lowest dose level.
Current report
In March 2025, the U.S. Food and Drug Administration (“FDA”) cleared PBGENE-HBV to commence Phase 1 clinical trials in the United States, and in April 2025, the FDA granted Fast Track designation to PBGENE-HBV for chronic Hepatitis B. Throughout 2025, we plan to share updates on the full cohorts including antiviral efficacy at different dose levels as each cohort is completed following three dose administrations and appropriate follow up. Also, in April 2025, the U.K. Medicines and Healthcare products Regulatory Agency (“MHRA”) cleared PGENE-HBV for a Phase 1 trial, making it the fifth country to clear a clinical trial application (“CTA”) or investigational new drug (“IND”) for PBGENE-HBV.
Current report
On May 8, 2025, we presented initial safety for PBGENE-HBV at the European Association for the Study of the Liver Congress (“EASL”), which indicated translation of PBGENE-HBV nonclinical pharmacokinetic and safety data from non-human primates into the clinic. These data support pre-planned repeat dosing as well as dose escalation of PBGENE-HBV with the goal of moving appropriate dose and schedule into Phase 2 expansion.
Current report
PBGENE-DMD is our development program for the treatment of Duchenne muscular dystrophy (“DMD”). DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the United States alone. There are currently no approved therapies with curative intent that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function over time and addresses more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit.
Current report
In preclinical data presented at the Muscular Dystrophy Association (“MDA”) in March 2025 and additional new data presented at the American Society of Gene and Cell Therapy (“ASGCT”) on May 14, 2025, PBGENE-DMD demonstrated the ability to target the key muscle types involved in the progression of DMD and significant, durable functional improvement in a humanized DMD mouse model. PBGENE-DMD restored the body’s ability to produce a near full length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement.
Current report
Based on these compelling data, the significant unmet need in DMD, and the clear regulatory guidance established for new therapeutics for DMD, we believe that PBGENE-DMD holds significant potential for the majority of patients with DMD. As a result, we are prioritizing and accelerating the development of PBGENE-DMD as our second wholly owned clinical program and target filing an IND and/or CTA for PBGENE-DMD in 2025 with clinical data expected in 2026.
Current report
The PBGENE-3243 is a first of its kind potential treatment for m.3243 associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA (“mtDNA”), thereby eliminating the root cause of the disease. Currently, there are no curative treatments for m.3243-associated mitochondrial disease, which affects approximately 20,000 people in the U.S. alone and an even larger prevalent population globally. PBGENE-3243 is designed to alleviate muscular myopathy symptoms, providing a significant quality of life and functional improvement for patients.
Current report
New preclinical data presented at ASGCT on May 14, 2025 highlight the ability of an ARCUS nuclease to eliminate mutant mtDNA and achieve therapeutically meaningful heteroplasmy shiftsin vivo. Reductions of mutant mtDNA exceed the baseline cited in the literature to be sufficient to alleviate the clinical symptoms in patients with m.3243-associated disease.
Current report
To accelerate development of PBGENE-DMD and maintain operational capability to deliver PBGENE-HBV and PBGENE-DMD data through Phase 1 clinical results, we plan to pause development of PBGENE-3243 and will stage future development of PBGENE-3243 alone or with partners following completion of the Phase 1 ELIMINATE-B HBV trial and after the PBGENE-DMD program reaches the clinic.
Current report
In partnership with iECURE, Inc. (“iECURE”), an ARCUS-mediated gene insertion approach is being pursued as a potential treatment option for neonatal onset ornithine transcarbamylase (“OTC”) deficiency. In January 2025, iECURE reported clinical efficacy and safety data in the first patient dosed showing a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year old and is eating age-appropriate levels of protein for a child of this age. Clinical data from the first patient in the ongoing OTC-HOPE trial was presented at the 2025 ACMG Annual Clinical Genetics Meeting in March 2025 and additional data has been accepted for presentation at ASGCT 2025. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026.
Current report
On January 7, 2024, we entered into a license agreement (the “TG License Agreement”) with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc. (“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”), pursuant to which we granted TG Subsidiary certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize azercabtagene zapreleucel (“azer-cel”) for autoimmune diseases and other indications outside of cancer. We received a deferred cash payment of $2.5 million from TG Therapeutics on January 6, 2025 in exchange for 220,712 shares of our common stock at a price of $11.33, which represented a 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of payment. We recognized no revenue under the TG License Agreement during the three months ended March 31, 2025, and we recognized $7.0 million of revenue under the TG License Agreement in the three months ended March 31, 2024.
Current report
On November 19, 2020, we entered into a development and license agreement with Eli Lilly and Company (“Lilly”) to collaborate, discover and developin vivo gene editing products incorporating ARCUS nucleases for the research and development of potentialin vivo therapies for genetic disorders, which was subsequently assigned to Prevail Therapeutics Inc., a wholly-owned subsidiary of Lilly (“Prevail”), effective November 1, 2022 and amended on June 30, 2023 (the “Prevail Agreement”). For the three months ended March 31, 2025, we did not recognize any revenue under the Prevail Agreement as it was terminated in July 2024, and we subsequently exercised our right to the return of our three programs. We recognized revenue of $4.5 million for the three months ended March 31, 2024. We have no deferred revenue related to the Prevail Agreement as of March 31, 2025 or December 31, 2024.
Current report
iECURE
Current report
In August 2021, we entered into a development and license agreement with iECURE (the “iECURE DLA”) under which iECURE was to advance our PBGENE-PCSK9 candidate through preclinical activities as well as a Phase 1 clinical trial in order to gain access to a license to use our PCSK9-directed ARCUS nuclease to insert genes into the PCSK9 locus to develop treatments for four pre-specified rare genetic diseases, including OTC deficiency (the “PCSK9 License”). Simultaneously with the entry into the iECURE DLA, we and iECURE entered into an equity issuance agreement (the “iECURE Equity Agreement”), pursuant to which iECURE issued us common stock in iECURE as additional consideration for the PCSK9 license. Additionally, we are eligible to receive milestone and mid-single digit to low double digit royalty payments on sales of iECURE products developed with ARCUS.
Current report
Research and Development Expenses
Current report
General and Administrative Expenses
Current report
Gain from Equity Method Investment
Current report
Interest Expense
Current report
Interest Income
Current report
Research and Development Expenses
Current report
The direct expense increases of $2.3 million for PBGENE-DMD and $1.0 million for PBGENE-3243 were driven by development costs to continue advancing these programs. These increases were partially offset by a $1.5 million decrease in PBGENE-HBV development costs, which was driven by the program’s conclusion of non-clinical work and the program’s transition to the clinic. Contributing to the decrease in platform development and development expenses was a $0.6 million decrease in depreciation and amortization, a $0.3 million decrease in licensing fees and other research and development costs, and a $0.3 million decrease in laboratory supplies and services expenses.
Current report
General and Administrative Expenses
Current report
General and administrative expenses were $8.6 million for the three months ended March 31, 2025 compared to $8.4 million for the three months ended March 31, 2024. The increase of $0.2 million was primarily due to a $0.5 million increase in employee-related costs, partially offset by decreases in depreciation and amortization and other operating expenses, specifically taxes and insurance.
Current report
Gain from Equity Method Investment
Current report
Gain from equity method investment was $1.3 million during the three months ended March 31, 2025 which was primarily driven by a $2.3 million gain on dilution from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s net loss of $0.9 million. Gain from equity method investment was $1.7 million during the three months ended March 31, 2024, which was primarily the result of a $2.7 million gain recorded from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s loss of $1.0 million.
Current report
Interest Expense
Current report
The increase in non-cash adjustments was primarily driven by the net change in fair value of warrants for the three months ended March 31, 2025 compared to the three months ended March 31, 2024.
Current report
Cash Used in Investing Activities
Current report
Smaller Reporting Company Status
Current report
We are a “smaller reporting company” as defined under applicable regulations promulgated by the SEC. We will continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250 million or (ii) our annual revenue is less than $100 million during the most recently completed fiscal year and the market value of our stock held by non-affiliates is less than $700 million. As a smaller reporting company, we are able to take advantage of certain exemptions from disclosure requirements, including presenting only the two most recent fiscal years of audited financial statements and reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements.
Current report
We cannot predict whether investors will find our common stock less attractive if we rely on the exemptions available to smaller reporting companies. If investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be reduced or more volatile. To the extent we take advantage of such reduced disclosure obligations, it may also make comparison of our financial statements with other public companies difficult or impossible.
Removed paragraphs (46)
Previous report
Business Updates
Previous report
In January 2024 we entered into a license agreement with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc. (“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”) for non-oncological applications of azercabtagene zapreleucel (“azer-cel”) (the “TG License Agreement”) in January 2024. In connection with the TG License Agreement, we received upfront, and are also entitled to receive potential near-term, economics valued in the aggregate at $17.5 million. We are also entitled to receive additional payments upon the achievement of additional specified milestones of up to $288.6 million. If a licensed product under the TG License Agreement is approved and sold, TG Therapeutics is also required to pay us tiered royalties ranging from high-single-digit to low-double-digit percentages on net sales of the licensed product. In August 2024, we announced TG Therapeutics received U.S. Food and Drug Administration (“FDA”) clearance for its investigational new drug (“IND”) application to investigate azer-cel in human clinical trials for the treatment of progressive forms of multiple sclerosis. TG Therapeutics anticipates commencing a phase 1 clinical trial in 2024.
Previous report
In February 2024, we announced that we had granted Caribou Biosciences, Inc. (“Caribou”), a leading CRISPR genome-editing cell therapy company, a non-exclusive, worldwide license, with the right to sublicense, to one of our foundational cell therapy patent families for use with CRISPR-based therapies in the field of human therapeutics. Under the terms of the agreement, we received an upfront payment and, upon commercialization by Caribou, will receive royalties on net sales of licensed products. In addition, for each occurrence of certain strategic transactions involving Caribou, we are entitled to receive a specific tiered milestone payment.
Previous report
In April 2024, we received written notice from Prevail Therapeutics, Inc. (“Prevail”), a wholly-owned subsidiary of Eli Lilly and Company, of its termination of the amended and restated development and license agreement between the Company and Prevail (the “Prevail Agreement”). Prevail’s notice informed us that Prevail was exercising its right pursuant to Section 15.3.2 of the Prevail Agreement to terminate the Prevail Agreement in its entirety without cause upon 90 days’ prior written notice to us. We subsequently exercised our rights to the return of the three programs. We are continuing to evaluate these returned programs to determine the appropriate next steps with each of them, including a novel gene excision approach for treatment of Duchenne Muscular Dystrophy (“DMD”), a liver target for gene insertion, and a gene editing program targeting neurons to address a disease of the central nervous system.
Previous report
In July 2024, we 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 us with a term loan with a principal amount of $22.5 million (the “2024 Term Loan”). The proceeds from the 2024 Term Loan were used to repay the $22.5 million outstanding principal balance under our revolving line of credit with Banc of California (the “Revolving Line”), and pursuant to the terms of the 2024 Loan and Security Agreement, the Revolving Line was terminated.
Previous report
Corporate Updates
Previous report
In October 2024, we received approval of our clinical trial application (“CTA”) in Moldova for our wholly-ownedin vivo gene editing program PBGENE-HBV for the potential treatment of chronic hepatitis B virus (“HBV”) and are working towards dosing patients. We have submitted additional global regulatory applications which are pending approval.
Previous report
HBV causes inflammation and damage to the liver, leading to chronic infection and increased risk of death from liver cancer or cirrhosis. There is no cure for chronic hepatitis B and current treatments rarely result in functional cure, primarily due to persistence of viral DNA in the liver. In patients with chronic HBV, genetic material of the virus is converted within infected liver cells into cccDNA that acts as a template to make HBV copies. HBV also inserts its DNA into the human genome of infected liver cells. This integrated HBV DNA produces the viral protein, hepatitis B surface antigen (“HBsAg”), which is secreted in the blood. Presence of HBsAg is associated with poorer outcomes and suppression of HBsAg is necessary for functional cure of chronic hepatitis B.
Previous report
PBGENE-HBV is designed to eliminate cccDNA with direct edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in HBsAg. We believe specificity is of particular importance for developing a safe gene editing approach to eliminating HBV, as a lack of nuclease specificity can lead to unfavorable off-target results including increased integrations of HBV genomes into the human genome, as well as translocations between integrations. Preclinical data from the PBGENE-HBV program was presented in June 2024 at a poster presentation and panel discussion at the European Association for the Study of the Liver Congress, highlighting the ability of ARCUS to make efficient, durable, and targeted elimination edits. Data presented further supported the ability of PBGENE-HBV to specifically target and eliminate cccDNA and inactivate 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. PBGENE-HBV was well-tolerated in non-human primates (“NHP”) across multiple dose administrations. Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potentially curative treatment for chronic hepatitis B.
Previous report
The PBGENE-3243 program, previously known as PBGENE-PMM, is our wholly-owned, first of its kind potential treatment for m.3243 associated mitochondrial disease. The program's updated nomenclature more accurately describes its intended target patient population – those who have the m.3243 mutation and muscle-related symptoms. Mitochondrial diseases are the most common hereditary metabolic disorder, affecting 1 in 4,300 people. In particular, the m.3243 associated mitochondrial disease that our program intends to address affects about 20,000 people in the United States alone. The highly specific mitochondria-targeted ARCUS nucleases are designed to shift heteroplasmy by editing and eliminating mutant mitochondrial DNA while allowing normal (wild-type) mitochondrial DNA to repopulate in the mitochondria, thus improving cellular function. Preclinical data from the PBGENE-3243 program presented in June 2024 at the United Mitochondrial Disease Foundation’s Mitochondrial Medicine 2024 Conference and in March 2024 at a poster presentation at the Mitochondrial Medicine – Therapeutic Development Annual Conference demonstrated ARCUS’ ability to efficiently eliminate mutant mitochondrial DNA without nuclear off-target editing. We expect to submit an IND and/or CTA application in 2025 with respect to PBGENE-3243.
Previous report
In October 2024, we presented a poster at the European Society of Gene & Cell Therapy 31st Annual Congress. The poster highlighted preclinical data demonstrating the ability of ARCUS to achieve high-efficiency gene insertion, gene replacement, and base correction via homology-directed repair (“HDR”). In the preclinical work presented, we showed that targeted gene insertion can be achieved using ARCUS in greater than 85% of T-cells and 39% of non-dividing primary human hepatocytes. These high rates of gene insertion were accomplished primarily through HDR, which the research demonstrated was dependent on homology arms in the repair template and on the characteristic ARCUS 3’ overhang cut in the direction of DNA replication.
Previous report
We, along with our collaboration partners, intend to continue to evaluate the ARCUS platform with regards to safety, on-target editing, gene insertion, complex gene edits, and compatibility with viral and non-viral delivery.
Previous report
In partnership with iECURE, Inc. (“iECURE”), an ARCUS-mediated gene insertion approach is being pursued as a potential treatment option for neonatal onset ornithine transcarbamylase (“OTC”) deficiency. NHP data presented by researchers from the University of Pennsylvania’s Gene Therapy Program demonstrated sustained gene insertion of a therapeutic OTC transgene one-year post-dosing in newborn and infant NHPs with high efficiency. iECURE has received regulatory approvals in the United States, the United Kingdom, Spain, and Australia for initiation of the OTC-HOPE study, a first-in-human Phase 1/2 trial evaluating ECUR-506 as a potential treatment for OTC deficiency, and has begun recruiting patients. 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 2025.
Previous report
In June 2022, we announced we entered into an exclusivein vivo gene editing research and development collaboration and license agreement (the “Novartis Agreement”) with Novartis Pharma AG (“Novartis”). In connection with this partnership, we are developing a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a “safe harbor” location in the genome as a potential one-time transformative treatment option for diseases including certain hemoglobinopathies such as sickle cell disease and beta thalassemia. Under the terms of the Novartis Agreement, we will develop an ARCUS nuclease and conductin vitro characterization, with Novartis then assuming responsibility for all subsequent research, development, manufacturing and commercialization activities.
Previous report
Reverse Stock Split
Previous report
On January 18, 2024, our stockholders approved a proposal to amend our amended and restated certificate of incorporation to effect a reverse stock split of our common stock at a ratio of not less than 1-for-10 and not more than 1-for-30, with such ratio and the implementation and timing of such reverse stock split to be determined by our board of directors in its sole discretion. On February 6, 2024, our board of directors approved a 1-for-30 reverse stock split of our issued and outstanding common stock, and on February 13, 2024, we filed with the Secretary of State of the State of Delaware a certificate of amendment to our amended and restated certificate of incorporation in order to effect the reverse stock split. Trading of our common stock on The Nasdaq Capital Market commenced on a split-adjusted basis on February 14, 2024. As a result of the reverse stock split, every 30 shares of our common stock issued or outstanding were automatically reclassified into and became one new share of common stock, and the number of our issued and outstanding shares of common stock was reduced to 4,191,053 and 4,164,038, respectively. All references to numbers of shares of common stock and per-share information in this Quarterly Report on Form 10-Q have been adjusted retroactively, as appropriate, to reflect the reverse stock split.
Previous report
During the nine months ended September 30, 2024, we recognized revenue under the TG License Agreement of $8.0 million. There was no revenue recognized under the TG License Agreement in the three months ended September 30, 2024. A contract asset related to the TG License Agreement amounted to $1.4 million as of September 30, 2024.
Previous report
For the three months ended September 30, 2024, we did not recognize any revenue under the Prevail Agreement as it was terminated in the prior quarter. We recognized revenue of $5.5 million for the three months ended September 30, 2023. During the nine months ended September 30, 2024 and 2023, we recognized revenue under the Prevail Agreement of $52.7 million and $23.4 million, respectively.We have no deferred revenue related to the Prevail Agreement as of September 30, 2024 due to termination of the agreement. Deferred revenue related to the Prevail Agreement amounted to $52.7 million as of December 31, 2023, of which $4.7 million was included in current liabilities within the condensed balance sheet.
Previous report
Impairment Charges
Previous report
Impairment charges represent our impairment of intangible assets and long-term prepaid assets. An impairment loss is assessed when future undiscounted cash flows are less than the assets’ carrying value and is recognized when the carrying value of the asset exceeds fair value. An impairment charge is recognized for the amount by which the carrying amount exceeds the fair value of the asset.
Previous report
Income from Discontinued Operations
Previous report
Gain from discontinued operations represents the gain on the sale of our CAR T infrastructure to Imugene and the results of operations of our historical cell therapy operations.
Previous report
Contributing to the decrease in platform development and development expenses was a $0.9 million decrease in outsourced research and development costs, a $0.7 million decrease in laboratory supplies and services expenses, and a $0.4 million decrease in share based compensation expense. Direct expenses increased due to a $2.4 million increase in PBGENE-3243 development costs as it continues to advance toward an IND and/or CTA in 2025, partially offset by a $2.7 million decrease in PBGENE-HBV primarily due to a decrease in expenses for nonclinical studies.
Previous report
General and administrative expenses were $8.8 million for the three months ended September 30, 2024 compared to $9.6 million for the three months ended September 30, 2023. The decrease of $0.8 million was primarily due to a $0.4 million decrease in consulting fees in addition to decreases in tax and insurance expenses.
Previous report
Loss from equity method investment was $0.9 million during the three months ended September 30, 2024 compared to $1.4 million during the three months ended September 30, 2023, which represented our proportionate share of Elo’s net loss in each period.
Previous report
Income (Loss) from Discontinued Operations
Previous report
There was no income from discontinued operations during the three months ended September 30, 2024 compared to income of $4.0 million during the three months ended September 30, 2023. The $4.0 million of income during the three months ended September 30, 2023 consisted of the $8.4 million gain on disposal of our CAR T infrastructure to Imugene, partially offset by research and development expenses for our historical cell therapy operations.
Previous report
Comparison of the Nine Months Ended September 30, 2024 and September 30, 2023
Previous report
Revenue
Previous report
Revenue for the nine months ended September 30, 2024 was $68.1 million, compared to $41.7 million for the nine months ended September 30, 2023. The increase of $26.4 million in revenue during the nine months ended September 30, 2023 was primarily the result of a $29.3 million increase in revenue related to the Prevail Agreement as the remaining revenue under the agreement was recognized during the nine months ended September 30, 2024 following conclusion of the collaboration in April 2024. Additionally, there was a $9.5 million increase in revenue in the nine months ended September 30, 2024 as a result of the TG License Agreement and the license agreement with Caribou. Partially offsetting the increase was a $12.4 million decrease in revenue recognized under the Novartis Agreement during the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 as we near the completion of our pre-clinical workplan.
Previous report
Research and development expenses for the nine months ended September 30, 2024 were $43.7 million, compared to $40.0 million for the nine months ended September 30, 2023. The increase of $3.7 million was primarily due a $7.1 million increase in PBGENE-HBV external development costs and a $4.6 million increase in PBGENE-3243 external development costs as the programs continue to advance towards the clinic. The increase in direct research and development expenses was offset by an $8.0 million decrease in platform development and unallocated expenses, including a $2.9 million decrease in laboratory supplies and services expenses, a $2.8 million decrease in outsourced research and development costs, and a $2.1 million decrease in employee-related costs and share based compensation expense.
Previous report
General and administrative expenses were $25.7 million for the nine months ended September 30, 2024 compared to $30.5 million for the nine months ended September 30, 2023. The decrease of $4.8 million was primarily due to a $2.1 million decrease in employee-related costs and share based compensation expense as a result of a reduced headcount, a $1.0 million decrease in consulting fees, and decreases in insurance expense and legal fees.
Previous report
Loss from equity method investment was $0.1 million during the nine months ended September 30, 2024. Loss from equity method investment was $4.1 million during the nine months ended September 30, 2023, which represented our proportionate share of Elo’s net loss for such periods.
Previous report
Gain on changes in fair value was $0.9 million for the nine months ended September 30, 2024, which was primarily attributed to an increase during such period in the fair value of the Imugene Convertible Note and the ordinary shares issued thereunder and still held by us at the end of the period. Loss on changes in fair value was $0.5 million for the nine months ended September 30, 2023, which was attributed to a decrease in the assessed fair value of our equity investment in iECURE as a result of dilution from iECURE’s Series A-1 equity raise in such period, partially offset by an increase in the fair value of the Imugene Convertible Note.
Previous report
Gain from change in fair value of the warrant liability of $21.8 million for the nine months ended September 30, 2024 represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering. There was no warrant liability in the nine months ended September 30, 2023.
Previous report
Interest expense was $1.4 million and $1.7 million for the nine months ended September 30, 2024 and 2023, respectively. The $0.3 million decrease in interest expense was the result of lower interest rates during the nine months ended September 30, 2024 as compared to the nine months ended September 30, 2023.
Previous report
Interest income was $5.3 million during the nine months ended September 30, 2024 compared to $5.9 million during the nine months ended September 30, 2023. The $0.6 million decrease in interest income was the result of lower interest rates during the nine months ended September 30, 2024 as compared to the nine months ended September 30, 2023.
Previous report
Loss from Discontinued Operations
Previous report
There was no loss from discontinued operations during the nine months ended September 30, 2024 compared to a loss of $15.9 million during the nine months ended September 30, 2023. The $15.9 million loss during the nine months ended September 30, 2023 consisted of the $8.4 million gain on disposal of our CAR T infrastructure to Imugene, partially offset by research and development expenses for our historical cell therapy operations.
Previous report
The decrease in non-cash adjustments was primarily driven by decreases in the change in fair value of warrants which we did not have in the nine months ended September 30, 2023, loss from equity method investment, depreciation and amortization, and share-based compensation. These decreases were partially offset by a gain on disposal of the cell therapy business which occurred in the nine months ended September 30, 2023.
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Reworded paragraphs (27)
Previous wording
Since mid-2023, we have solely focused on leveraging our proprietary ARCUS genome editing platform to advancein vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUS in vivo gene editing in human therapeutics.
Current wording
We are leveraging our proprietary ARCUS genome editing platform to advancein vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUS in vivo gene editing in human therapeutics.
Previous wording
On August 15, 2023 we entered into an asset purchase agreement with Imugene Limited, and its wholly-owned subsidiary Imugene (USA) Inc. (“Imugene US” and together with Imugene Limited, “Imugene”). Additionally, we entered into a license agreement with Imugene (the “Imugene License Agreement”) on August 15, 2023, pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic CAR T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
Current wording
On August 15, 2023 we entered into an asset purchase agreement with Imugene Limited, and its wholly-owned subsidiary Imugene (USA) Inc. (“Imugene US” and together with Imugene Limited, “Imugene”). Additionally, we entered into a license agreement with Imugene (the “Imugene License Agreement”) on August 15, 2023, pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic chimeric antigen receptor (“CAR”) T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
Previous wording
As of September 30, 2024, management has constrained all variable consideration related to milestone payments in the Imugene License Agreement given the level of uncertainty associated with achievement of the milestone payments. Accordingly, no revenue was recognized under the Imugene License Agreement during the three and nine months ended September 30, 2024.
Current wording
As of March 31, 2025, management has constrained all variable consideration related to milestone payments in the Imugene License Agreement given the level of uncertainty associated with achievement of the milestone payments. Accordingly, no revenue was recognized under the Imugene License Agreement during the three months ended March 31, 2025.
Previous wording
During the three months ended September 30, 2024 and 2023, we recognized revenue under the Novartis Agreement of $0.6 million and $7.6 million, respectively. During the nine months ended September 30, 2024 and 2023, we recognized revenue under the Novartis Agreement of $5.9 million and $18.2 million, respectively. Deferred revenue related to the Novartis Agreement amounted to $26.6 million and $32.4 million as of September 30, 2024 and December 31, 2023, respectively, of which $0.2 million and $7.4 million, respectively, was included in current liabilities within the condensed balance sheets.
Current wording
On June 14, 2022, we entered into a collaboration and license agreement (the “Novartis Agreement”) with Novartis Pharma AG (“Novartis”), which became effective on June 15, 2022, to collaborate, discover and developin vivo gene editing products incorporating our custom ARCUS nucleases for the purpose of seeking to research and develop potential treatments for certain hemoglobinopathies, including sickle cell disease and beta thalassemia. During the three months ended March 31, 2025 and 2024, we recognized revenue under the Novartis Agreement of less than $0.1 million and $4.5 million, respectively. Deferred revenue related to the Novartis Agreement amounted to $26.2 million and $26.3 million as of March 31, 2025 and December 31, 2024, respectively, of which $5.7 million and $3.0 million, respectively, was included in current liabilities within the condensed balance sheets.
Previous wording
Loss from equity method investment represents our proportionate share of the net loss of our equity method investee, Elo Life Systems, Inc. (“Elo”).
Current wording
Gain from equity method investment represents our proportionate share of the net loss of our equity method investee, Elo Life Systems, Inc. (“Elo”).
Previous wording
We adjust the carrying value of the ordinary shares issued under the Imugene Convertible Note to its fair value each reporting period with any changes in fair value recorded to other income (expense).
Current wording
We adjust the carrying value of the ordinary shares issued under the Imugene Convertible Note to its fair value each reporting period with any changes in fair value recorded to other income (expense). As of March 31, 2025, we had sold all of our ordinary shares of Imugene Limited.
Previous wording
Gain on Change in Fair Value of Warrant Liability
Current wording
(Loss) Gain on Change in Fair Value of Warrant Liability
Previous wording
The change in fair value of warrant liability represents the mark-to-market fair value adjustments to the outstanding warrants issued in connection with the March 2024 Public Offering.
Current wording
The change in fair value of warrant liability represents the mark-to-market fair value adjustments to the outstanding warrants issued in connection with an underwriting agreement in March 2024 relating to a public offering of (i) 2,500,000 shares of our common stock, par value $0.000005 per share, and (ii) warrants to purchase up to an aggregate of 2,500,000 shares of our common stock (the “March 2024 Public Offering”).
Previous wording
Revenue for the three months ended September 30, 2024 was $0.6 million, compared to $13.1 million for the three months ended September 30, 2023. The decrease of $12.5 million in revenue during the three months ended September 30, 2024 was primarily the result of a $7.0 million decrease in revenue recognized under the Novartis Agreement during the three months ended September 30, 2024 compared to the three months ended September 30, 2023 as we near the completion of our pre-clinical workplan and a $5.5 million decrease in revenue recognized under the Prevail Agreement during the three months ended September 30, 2024 compared to the three months ended September 30, 2023 following conclusion of the collaboration in April 2024.
Current wording
Revenue for the three months ended March 31, 2025 was less than $0.1 million, compared to $17.6 million for the three months ended March 31, 2024. The decrease of $17.6 million in revenue during the three months ended March 31, 2025 was primarily the result of $8.5 million in revenue recognized under the TG License Agreement and a license agreement with Caribou Biosciences, Inc. during the three months ended March 31, 2024, a $4.5 million decrease in revenue recognized under the Novartis Agreement as we near the completion of our pre-clinical workplan during the three months ended March 31, 2025 compared to the three months ended
Previous wording
Research and development expenses for the three months ended September 30, 2024 were $13.1 million, compared to $15.9 million for the three months ended September 30, 2023. The decrease of $2.8 million was primarily due to a reduction in platform development and unallocated expenses of $2.5 million. Additionally contributing to the decrease was a $0.3 million decrease in our direct expenses in PBGENE-HBV and PBGENE-3243 programs.
Current wording
Research and development expenses for the three months ended March 31, 2025 were $13.6 million, compared to $13.3 million for the three months ended March 31, 2024. The increase of $0.3 million was primarily due to a $3.2 million increase in our direct expenses in PBGENE-DMD and PBGENE-3243 programs, partially offset by a $1.5 million decrease in our direct expense in PBGENE-HBV and a reduction in platform development and unallocated expenses of $1.5 million.
Previous wording
Gain on changes in fair value was $0.6 million for the three months ended September 30, 2024, which was primarily attributed to an increase during such period in the fair value of the Imugene Convertible Note and the ordinary shares issued thereunder and still held by us at the end of the period. Gain on changes in fair value was $0.3 million for the three months ended September 30, 2023, which was primarily attributable to an increase during such period in the fair value of the Imugene Convertible Note.
Current wording
Gain on changes in fair value was less than $0.1 million for the three months ended March 31, 2025, which was primarily attributed to an increase during such period in the fair value of the Imugene Convertible Note and the ordinary shares issued thereunder. Loss on changes in fair value was $0.3 million for the three months ended March 31, 2024, which was primarily attributable to a decrease during such period in the fair value of the Imugene Convertible Note.
Previous wording
Gain from change in fair value of the warrant liability of $3.6 million for the three months ended September 30, 2024 represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering. There was no warrant liability in the three months ended September 30, 2023.
Current wording
Loss from change in fair value of the warrant liability was $0.8 million for the three months ended March 31, 2025 compared to a gain of $10.4 million for the three months ended March 31, 2024, which represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering.
Previous wording
Interest expense was $0.3 million for the three months ended September 30, 2024 compared to $0.6 million for the three months ended September 30, 2023. The decrease of $0.3 million of interest expense was primarily driven by lower interest rates during the three months ended September 30, 2024 as compared to the three months ended September 30, 2023 as well as the waived final payment fee on the Revolving Line.
Current wording
Interest expense was $0.4 million for the three months ended March 31, 2025 compared to $0.6 million for the three months ended March 31, 2024. The decrease of $0.2 million of interest expense was primarily driven by lower interest rates during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024.
Previous wording
Interest income was $1.8 million during the three months ended September 30, 2024 compared to $1.9 million during the three months ended September 30, 2023. The $0.1 million decrease in interest income was the result of lower interest rates during the three months ended September 30, 2024 as compared to the three months ended September 30, 2023.
Current wording
Interest income was $1.3 million during the three months ended March 31, 2025 compared to $1.7 million during the three months ended March 31, 2024. The $0.4 million decrease in interest income was the result of lower interest rates during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024.
Previous wording
Loss on disposal of assets was $0.3 million during the nine months ended September 30, 2024 compared to a gain of less than $0.1 million during the nine months ended September 30, 2023.
Current wording
Loss on disposal of assets was less than $0.1 million during each of the three months ended March 31, 2025 and 2024.
Previous wording
We have incurred significant operating losses since our inception and have not generated any revenue from the sale of products. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more of our product candidates or the product candidates of our collaborators or other licensees for which we may receive milestone payments or royalties. As of September 30, 2024, we had an accumulated deficit of $464.7 million.
Current wording
We have incurred significant operating losses since our inception and have not generated any revenue from the sale of products. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more of our product candidates or the product candidates of our collaborators or other licensees for which we may receive milestone payments or royalties. As of March 31, 2025, we had an accumulated deficit of $503.0 million.
Previous wording
contract research organizations (“CROs”) and CMOs, expanding our intellectual property portfolio and providing general and administrative support for our operations. If we obtain regulatory approval for any of our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources.
Current wording
We expect to incur significant expenses and operating losses for the foreseeable future as we advance the development of our product candidates. We expect that our research and development and general and administrative costs will increase over the long term, including in connection with conducting preclinical studies and potential clinical trials for our product candidates, contracting with CROs and CMOs, expanding our intellectual property portfolio and providing general and administrative support for our operations. If we obtain regulatory approval for any of our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources.
Previous wording
As of September 30, 2024, we had cash and cash equivalents of $98.8 million and $22.6 million in restricted cash under the 2024 Term Loan, which replaced our Revolving Line on July 31, 2024. Pursuant to our July 31, 2024 amended and restated loan and security agreement with Banc of California (the “2024 Loan and Security Agreement”), we are not entitled to borrow any additional amounts under the 2024 Term Loan and are required to maintain an aggregate balance in a cash security account with Banc of California (the “Cash Security Account”) at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding.
Current wording
As of March 31, 2025, we had a total cash, cash equivalents and restricted cash balance of $99.8 million. Pursuant to our July 31, 2024 amended and restated loan and security agreement with Banc of California (the “2024 Loan and Security Agreement”) pursuant to which Banc of California provided us with a term loan with a principal amount of $22.5 million (the “2024 Term Loan”), we are not entitled to borrow any additional amounts under the 2024 Term Loan and are required to maintain an aggregate balance in a cash security account with Banc of California (the “Cash Security Account”) at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding.
Previous wording
Our cash, cash equivalents, and restricted cash totaled $121.3 million and $122.2 million as of September 30, 2024 and 2023, respectively.
Current wording
Our cash, cash equivalents, and restricted cash totaled $99.8 million and $137.8 million as of March 31, 2025 and 2024, respectively.
Previous wording
Cash used in operating activities during the nine months ended September 30, 2024 was $39.8 million compared to $75.7 million during the nine months ended September 30, 2023. The $35.9 million decrease in cash used in operating activities was primarily driven by a $69.9 million increase in net income. This was offset by decreases of $23.5 million in non-cash adjustments and $10.5 million in changes in operating assets and liabilities.
Current wording
Cash used in operating activities during the three months ended March 31, 2025 was $19.1 million compared to $19.0 million during the three months ended March 31, 2024. The $0.1 million increase in cash used in operating activities was primarily driven by a $29.2 million decrease in net income offset by increases of $10.0 million in non-cash adjustments and $19.0 million in changes in operating assets and liabilities.
Previous wording
The change in cash used in operating assets and liabilities is primarily driven by a decrease in net changes of deferred revenue as a result of the conclusion of the Prevail collaboration offset by increases from the maturity of the Imugene Convertible Note in the nine months ended September 30, 2024 and net changes of accounts receivable.
Current wording
The change in cash used in operating assets and liabilities is primarily driven by the net changes of deferred revenue as a result of the conclusion of the Prevail collaboration and a decrease in revenue recognized under the Novartis collaboration for the three months ended March 31, 2025 compared to the three months ended March 31, 2024. Additionally driving the change in cash used in operating assets and liabilities is the net change of the contract asset as a result of the TG Therapeutics deferred share payment in three months ended March 31, 2025 and net changes in prepaid expenses.
Previous wording
Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the nine months ended September 30, 2024 was $0.1 million, compared to $5.8 million provided by investing activities in the nine months ended September 30, 2023. The $5.9 million decrease in cash provided by investing activities was primarily the result of the $8.0 million proceeds from the disposal of CAR T infrastructure, partially offset by $2.0 million of property, equipment, and software purchases during the nine months ended September 30, 2023.
Current wording
Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the three months ended March 31, 2025 was $0.3 million, compared to an amount less than $0.1 million used in investing activities in the three months ended March 31, 2024. The increase in cash used in investing activities was the result of a $0.3 million increase in intangible asset purchases during the three months ended March 31, 2025.
Previous wording
Net cash provided by financing activities during the nine months ended September 30, 2024 was $44.5 million, compared to $2.6 million during the nine months ended September 30, 2023. The $41.9 million increase in cash provided by financing activities during the nine months ended September 30, 2024 was primarily due to a $41.2 million increase in net proceeds from offerings of common stock in the nine months ended September 30, 2024 as compared to the nine months ended September 30, 2023 and $0.9 million in net proceeds from the issuance of common stock to TG Therapeutics in the nine months ended September 30, 2024.
Current wording
Net cash provided by financing activities during the three months ended March 31, 2025 was $10.7 million, compared to $40.1 million during the three months ended March 31, 2024. The $29.4 million decrease in cash provided by financing activities during the three months ended March 31, 2025 was primarily due to the Company receiving $29.6 million in net proceeds from an underwritten offering of common stock and warrants in the three months ended March 31, 2024, whereas the Company did not receive any net proceeds from underwritten offerings during the three months ended March 31, 2025.
Previous wording
On July 31, 2024, we replaced the Revolving Line with the 2024 Term Loan with an aggregate principal amount of $22.5 million. The stated interest rate under the 2024 Term Loan is equal to the greater of 1.50% below the Prime Rate or 4.5%. As of July 31, 2024, the stated interest rate was 7.00%. As of September 30, 2024, the outstanding principal balance on the 2024 Term Loan was $22.5 million, the stated interest rate was 6.5% and the effective interest rate was 6.99%.
Current wording
On July 31, 2024, we replaced our revolving line of credit with Banc of California with the 2024 Term Loan with an aggregate principal amount of $22.5 million. The stated interest rate under the 2024 Term Loan is equal to the greater of 1.50% below the Prime Rate or 4.5%. As of March 31, 2025, the outstanding principal balance on the 2024 Term Loan was $22.5 million, the stated interest rate was 6.00% and the effective interest rate was 6.43%.
Previous wording
We believe that, as of the date of this Quarterly Report on Form 10-Q, existing cash and cash equivalents, expected operational receipts, including upfront and potential near-term consideration to be received from licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2026. We expect our cash runway to be sufficient to achieve first-in-human Phase 1 clinical data for two wholly-ownedin vivo gene editing programs. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect. Because of the numerous risks and uncertainties associated with research, development and commercialization of pharmaceutical products, it is difficult to estimate with certainty the amount of our working capital requirements. Our future funding requirements will depend on many factors, including:
Current wording
We believe that, as of the date of this Quarterly Report on Form 10-Q, existing cash and cash equivalents, expected operational receipts, including potential near-term consideration to be received from licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2026. We expect our cash runway to be sufficient to achieve first-in-human Phase 1 clinical data for two wholly-ownedin vivo gene editing programs. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect. There are no assurances that we will be successful in obtaining an adequate level of financing as and when needed to finance our operations on terms acceptable to us or at all, particularly in light of current global macroeconomic conditions. If we are unable to obtain sufficient financing on a timely basis or on favorable terms, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves. We may also be otherwise unable to execute our business plan or growth strategy, or capitalize on business opportunities as desired. Any of these events could materially adversely affect our financial condition and business prospects. Because of the numerous risks and uncertainties associated with research, development and commercialization of pharmaceutical products, it is difficult to estimate with certainty the amount of our working capital requirements. Our future funding requirements will depend on many factors, including:
Previous wording
In March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale (the “March 2024 Public Offering”) of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share. Each warrant has an exercise price per share of $20.00, is immediately exercisable and will expire on March 5, 2029. The offering was made pursuant to a registration statement on Form S-3.
Current wording
In March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share. Each warrant has an exercise price per share of $20.00, is immediately exercisable and will expire on March 5, 2029. The offering was made pursuant to a registration statement on Form S-3.
Previous wording
Our critical accounting policies and estimates are described in “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Use of Estimates” in our Annual Report on Form 10-K. We have reviewed those critical accounting policies and estimates for the nine months ended September 30, 2024. Other than the derivative financial instruments basis of accounting outlined in Note 1, there have been no significant changes in our critical accounting policies and estimates from those disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
Current wording
Our critical accounting policies and estimates are described in “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Use of Estimates” in our Annual Report on Form 10-K. We have reviewed those critical accounting policies and estimates for the three months ended March 31, 2025. There have been no significant changes in our critical accounting policies and estimates from those disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
We adjust the carrying value of the iECURE equity to fair value each reporting period with any changes in fair value recorded to other income (expense). There was no change in the fair value of the iECURE equity for the three months ended March 31, 2025 and three months ended March 31, 2024.
Current report — wording carried from elsewhere
March 31, 2024 and a $4.5 million decrease in revenue recognized under the Prevail Agreement during the three months ended March 31, 2025 compared to the three months ended March 31, 2024 following conclusion of the collaboration in April 2024.
Current report — wording carried from elsewhere
(Loss) Gain on Change in Fair Value of Warrant Liability
Current report — wording carried from elsewhere
As of the filing of this Quarterly Report on Form 10-Q, we are subject to the Baby Shelf Rule, as defined and described in “Risk Factors – We will need substantial additional funding, and if we are unable to raise a sufficient amount of capital when needed on acceptable terms, or at all, we may be forced to delay, reduce or eliminate some or all of our research programs, product development activities and commercialization efforts.” in Part II, Item 1A. of this Quarterly Report on Form 10-Q. As a result of the Baby Shelf Rule, we will be limited in the amount of proceeds we are able to raise by selling shares of our common stock using our Form S-3, including under our ATM facility, until such time as our public float exceeds $75 million.
Rearranged previous paragraphs (7)
Previous report — wording carried elsewhere
Gain on Changes in Fair Value
Previous report — wording carried elsewhere
Change in Fair Value of Warrant Liability
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the nine months ended September 30, 2024 and September 30, 2023, together with the changes in those items:
Previous report — wording carried elsewhere
Change in Fair Value of Warrant Liability
Previous report — wording carried elsewhere
We expect to incur significant expenses and operating losses for the foreseeable future as we advance the development of our product candidates. We expect that our research and development and general and administrative costs will increase over the long term, including in connection with conducting preclinical studies and potential clinical trials for our product candidates, contracting with
Previous report — wording carried elsewhere
There are no assurances that we will be successful in obtaining an adequate level of financing as and when needed to finance our operations on terms acceptable to us or at all, particularly in light of current global macroeconomic conditions. If we are unable to obtain sufficient financing on a timely basis or on favorable terms, we may be required to significantly delay, alter, reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves. We may also be otherwise unable to execute our business plan or growth strategy or capitalize on business opportunities as desired. Any of these events could materially adversely affect our financial condition and business prospects.
Previous report — wording carried elsewhere
In March 2024, we entered into an underwriting agreement relating to the offering, issuance and sale of an aggregate of 2,500,000 shares of our common stock and warrants to purchase up to an aggregate of 2,500,000 shares of our common stock at a combined offering price of $16.00 per share. Each warrant has an exercise price per share of $20.00, is immediately exercisable and will expire on March 5, 2029. The offering was made pursuant to a registration statement on Form S-3. Gross proceeds from the transaction were $40.0 million before deducting underwriting discounts and commissions and offering expenses of approximately $3.0 million. We intend to use the net proceeds of the offering to fund ongoing and planned research and development, and for working capital and other general corporate purposes.