Precision BioSciences Reports Second Quarter 2024 Financial Results and Provides Business Update
- Wholly owned programs PBGENE-HBV for Chronic Hepatitis B virus and PBGENE-PMM for m.3243 mitochondrial disease on track for IND and/or CTA submissions in 2024 and 2025, respectively
- Expanded Hepatitis Scientific Advisory Board with addition of world-class clinical investigators Mark Sulkowski, M.D. and Jordan Feld, M.D., M.P.H.
- Sufficient capital to realize Phase 1 clinical data for multiple in vivo gene editing programs; Expected cash runway into the second half of 2026
- Regained control of three advanced preclinical programs for development internally or with partners, including a novel gene editing approach for Duchenne Muscular Dystrophy
“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
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% to45% 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
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
Common Stock purchase by members of management for
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
Quarter Ended June 30, 2024 Financial Results:
Cash and Cash Equivalents: As of June 30, 2024, Precision had approximately
Revenues: Total revenues for the quarter ended June 30, 2024 were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Net Income from Continuing Operations: Net income from continuing operations was
Net Income: Net income was
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.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV).
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners’ and licensees’ product candidates and gene editing approaches including editing efficiency, and the suitability of ARCUS nucleases for gene insertion, gene elimination and gene excision and differentiation from other gene editing approaches; the expected timing of regulatory processes (including filings and studies for PBGENE-HBV and PBGENE-PMM); expectations about our and our partners’ operational initiatives, strategies, and further development of our programs; expectations and updates around our partnerships and collaborations and our ability to enter into new collaborations, license agreements or other arrangements; our expected cash runway and available credit; the sufficiency of our cash runway extending into the second half of 2026; expectations about achievement of key milestones and receipt of any milestone, royalty, or other payments; expectations regarding our liquidity and capital resources; and anticipated timing of clinical data . In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
| Precision Biosciences, Inc. | ||||||||
| Condensed Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| (unaudited) | ||||||||
| For the Three Months Ended June 30, | ||||||||
2024 |
2023 |
|||||||
| Revenue | $ |
49,898 |
|
$ |
19,789 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
17,225 |
|
|
13,088 |
|
||
| General and administrative |
|
8,527 |
|
|
9,830 |
|
||
| Total operating expenses |
|
25,752 |
|
|
22,918 |
|
||
| Operating income (loss) |
|
24,146 |
|
|
(3,129 |
) |
||
| Other income (expense): | ||||||||
| Loss from equity method investment |
|
(950 |
) |
|
(1,369 |
) |
||
| Gain on changes in fair value |
|
694 |
|
|
— |
|
||
| Gain on change in fair value of warrant liability |
|
7,765 |
|
|
— |
|
||
| Interest expense |
|
(560 |
) |
|
(553 |
) |
||
| Interest income |
|
1,843 |
|
|
1,946 |
|
||
| (Loss) gain on disposal of assets |
|
(189 |
) |
|
72 |
|
||
| Total other income |
|
8,603 |
|
|
96 |
|
||
| Income (loss) from continuing operations | $ |
32,749 |
|
$ |
(3,033 |
) |
||
| Loss from discounted operations |
|
— |
|
|
(8,858 |
) |
||
| Net income (loss) | $ |
32,749 |
|
$ |
(11,891 |
) |
||
| Net income (loss) per share | ||||||||
| Basic | $ |
4.70 |
|
$ |
(3.13 |
) |
||
| Diluted | $ |
4.67 |
|
$ |
(3.13 |
) |
||
| Weighted-average shares of common stock outstanding | ||||||||
| Basic |
|
6,966,680 |
|
|
3,803,083 |
|
||
| Diluted |
|
7,011,630 |
|
|
3,803,083 |
|
||
| Precision Biosciences, Inc. | ||||||
| Condensed Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| (Unaudited) | ||||||
| June 30, 2024 | December 31, 2023 | |||||
| Cash and cash equivalents | $ |
123,571 |
$ |
116,678 |
||
| Working capital |
|
111,078 |
|
86,372 |
||
| Total assets |
|
165,800 |
|
159,781 |
||
| Total liabilities |
|
91,086 |
|
140,920 |
||
| Total stockholders' equity | $ |
74,714 |
$ |
18,861 |
||
| Common stock outstanding |
|
7,123,370 |
|
4,164,038 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20240801910847/en/
Investor and Media Contact:
Naresh Tanna
Vice President, Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
