Precision BioSciences Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Update
Precision BioSciences (Nasdaq: DTIL) reported Q4 and fiscal year 2022 results with revenues of $10.6 million for Q4, up from $6.3 million year-over-year, and $25.1 million for the fiscal year, down from $115.5 million in 2021. Key advancements include clinical progress in CAR T therapies azer-cel and PBCAR19B, with FDA engagement ongoing. Net loss for Q4 was $28.5 million, or $(0.26) per share, while the annual net loss was $111.6 million, or $(1.27) per share. The company maintains a strong cash position of $189.6 million, projected to fund operations through Q1 2025. Notable clinical updates and collaborations with partners like Novartis and Lilly are anticipated in 2023.
- Q4 revenue increased to $10.6 million, a $4.3 million rise from Q4 2021.
- FDA provided favorable Type C feedback on azer-cel, aiding clinical development.
- Cash balance of $189.6 million expected to fund operations through Q1 2025.
- Progress in clinical studies for key therapies like azer-cel and PBCAR19B.
- Fiscal year 2022 revenue decreased by $90.4 million compared to 2021.
- Net loss increased to $111.6 million for the fiscal year from $30.6 million in 2021.
- Impairment of prepaid expenses related to iECURE collaboration impacted financials.
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- Progressed Azer-cel and PBCAR19B Allogenic CAR T Clinical Studies; Program Updates Planned for Azer-cel and PBCAR19B
- Advanced Wholly Owned, Preclinical PBGENE-HBV and Partnered DMD, Sickle Cell Disease, and OTC Programs; New Data Expected to be Presented/Published in 2023
- Cash Balance Provides Expected 2-Year Runway through Q1 2025
“2022 proved to be a significant year for Precision as we made considerable progress on clinical, manufacturing, research, and collaboration fronts toward building an end-to-end gene editing company. Over the last year, Precision has invested in manufacturing optimization with our allogeneic CAR T therapies, which resulted in improved product candidate attributes in azer-cel, a potential first-in-class allogeneic CAR T treatment for patients who have relapsed following autologous CAR T treatment. In addition, following the platform-wide manufacturing optimization in 2022, we are continuing to dose subjects with PBCAR19B in its Phase 1 clinical study,” said
Ex
Favorable Type C feedback from FDA on azer-cel: In late 2022, the FDA provided supportive Type C feedback on the Company’s CMC processes and analytical methods for azer-cel. Responses from the FDA on analytical methods, proposed potency assays, and manufacturing processes are further guiding clinical development. Regulatory dialogue is ongoing as the Company progresses azer-cel.
Trials Progressing for azer-cel and PBCAR19B: The Company plans to progress azer-cel to a decision point for a Phase 2 trial in non-Hodgkin lymphoma (NHL) subjects who have relapsed following autologous CAR T treatment. Precision expects to complete the Phase 1b cohort for azer-cel to identify a dosing schedule for further study and plans to seek feedback from the FDA on the azer-cel clinical program once more data become available. The Company also plans to complete Phase 1 dose escalation for PBCAR19B in the earlier line NHL setting.
CAR T Portfolio Update: Precision plans to provide a CAR T update once investigators complete enrollment of the current azer-cel cohort of six CAR T relapsed subjects with sufficient follow-up to support a meeting with the FDA to discuss clinical plans. Subjects are being treated with optimized azer-cel product at the planned final dose level (500 million CAR T cells following a lymphodepletion regimen consisting of 3 days of fludarabine and cyclophosphamide). Based on current enrollment, the update is now expected to occur in the April/
During the CAR T update, the Company plans to provide additional long-term follow up from the azer-cel cohorts presented at ASH 2021 and ASCO 2022, as well as data from subjects in the current cohort. The CAR T update is also expected to include interim efficacy and safety data from the PBCAR19B Phase 1 trial at Dose Level 2 (540 million CAR T cells following 3 days of fludarabine and cyclophosphamide) with an expectation of durability data to follow this year.
In
Precision believes in vivo gene editing applications are particularly well suited to ARCUS because they require extremely low levels of off-target editing and efficient delivery. Emerging preclinical data from Precision and partners continue to validate that ARCUS may be particularly advantageous for gene insertion and large gene excisions, both intended to restore function. ARCUS is also unique in its relatively small size which potentially allows delivery to a wider range of cells and tissues using both viral and non-viral gene delivery methods.
Chronic Hepatitis B Virus (HBV): For its in vivo gene editing portfolio, Precision is prioritizing nominating the final drug candidate for its wholly owned PBGENE-HBV in vivo program, with the goal of submitting a CTA and/or IND in 2024. Preclinical data published in 2022 showed that ARCUS efficiently targeted and degraded HBV cccDNA in HBV-infected primary human hepatocytes and reduced expression of HBV S-antigen (“HBsAg”) by as much as
Novartis Partnered Program: Precision continues to make significant progress in its 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 administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach.
Lilly Partnered Programs: The partnered PBGENE-DMD program with Lilly targeting Duchenne muscular dystrophy (DMD) highlights the versatility of ARCUS for complex editing. Joint teams continue to make significant progress against preclinical objectives with the goal of using a pair of ARCUS nucleases to excise an often mutated “hot-spot” region of the dystrophin gene to restore dystrophin expression in DMD patients. In addition, the Company plans to present initial preclinical data for the DMD program in 2023.
Ornithine Transcarbamylase (OTC) Deficiency: Led by iECURE, an ARCUS-mediated gene insertion approach is being pursued as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Non-human primate (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 NHP with high efficiency. iECURE is targeting to file a CTA and/or IND in the second half of 2023. If accepted this would be the first ARCUS gene insertion program to be ready for human clinical trials and a major milestone for ARCUS in vivo gene editing.
The strategic prioritization exercise for Precision’s in vivo research pipeline announced in
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Fiscal Year 2022 Financial Results:
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About
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 efficacy and benefit of our product candidates and programs, the expected timing of updates regarding programs in our allogeneic CAR T and in vivo portfolio and ARCUS research, the expected timing of regulatory submissions and other communications, planned development activities with our collaboration partners, expectations about our operational initiatives, our business strategy and portfolio review and expectations regarding our liquidity and capital resources. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “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. Such statements are subject to 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 and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the 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; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators 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.
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Consolidated Statements of Operations |
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(In thousands, except share and per share amounts) |
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For the Three Months Ended |
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2022 |
|
|
2021 |
|
||
Revenue |
|
$ |
10,598 |
|
|
$ |
6,339 |
|
Operating expenses |
|
|
|
|
|
|
|
|
Research and development |
|
|
21,072 |
|
|
|
26,470 |
|
General and administrative |
|
|
10,073 |
|
|
|
10,616 |
|
Total operating expenses |
|
|
31,145 |
|
|
|
37,086 |
|
Loss from operations |
|
|
(20,547 |
) |
|
|
(30,747 |
) |
Other (expense) income, net: |
|
|
|
|
|
|
|
|
Impairment charges |
|
|
(11,438 |
) |
|
|
- |
|
Loss on disposal of assets |
|
|
(48 |
) |
|
|
(3 |
) |
(Loss) gain from changes in fair value |
|
|
(510 |
) |
|
|
2,281 |
|
Gain on deconsolidation of subsidiary |
|
|
- |
|
|
|
5,985 |
|
Income from equity method investment |
|
|
2,604 |
|
|
|
184 |
|
Interest expense |
|
|
(486 |
) |
|
|
(53 |
) |
Interest income |
|
|
1,937 |
|
|
|
63 |
|
Total other (expense) income, net |
|
|
(7,941 |
) |
|
|
8,457 |
|
Net loss |
|
$ |
(28,488 |
) |
|
$ |
(22,290 |
) |
|
|
|
|
|
|
|
|
|
Net loss per share - basic and diluted |
|
$ |
(0.26 |
) |
|
$ |
(0.37 |
) |
Weighted average shares of common stock outstanding - basic and diluted |
|
|
110,957,493 |
|
|
|
60,674,925 |
|
|
|
|
|
|
|
|
|
|
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For the Years Ended |
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|
|
2022 |
|
|
2021 |
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Revenue |
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$ |
25,098 |
|
|
$ |
115,529 |
|
Operating expenses |
|
|
|
|
|
|
|
|
Research and development |
|
|
83,939 |
|
|
|
115,238 |
|
General and administrative |
|
|
41,525 |
|
|
|
39,667 |
|
Total operating expenses |
|
|
125,464 |
|
|
|
154,905 |
|
Loss from operations |
|
|
(100,366 |
) |
|
|
(39,376 |
) |
Other (expense) income, net: |
|
|
|
|
|
|
|
|
Impairment charges |
|
|
(11,438 |
) |
|
|
- |
|
Loss on disposal of assets |
|
|
(106 |
) |
|
|
(26 |
) |
(Loss) gain from changes in fair value |
|
|
(510 |
) |
|
|
2,555 |
|
Gain on deconsolidation of subsidiary |
|
|
- |
|
|
|
5,985 |
|
(Loss) income from equity method investment |
|
|
(1,579 |
) |
|
|
184 |
|
Interest expense |
|
|
(1,111 |
) |
|
|
(132 |
) |
Interest income |
|
|
3,473 |
|
|
|
208 |
|
Total other (expense) income, net |
|
|
(11,271 |
) |
|
|
8,774 |
|
Net loss |
|
$ |
(111,637 |
) |
|
$ |
(30,602 |
) |
|
|
|
|
|
|
|
|
|
Net loss per share - basic and diluted |
|
$ |
(1.27 |
) |
|
$ |
(0.52 |
) |
Weighted average shares of common stock outstanding - basic and diluted |
|
|
87,898,498 |
|
|
|
58,688,102 |
|
|
|
|
|
|
|
|
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Consolidated Balance Sheets Data |
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(In thousands, except share amounts) |
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Cash and cash equivalents |
|
$ |
189,576 |
|
|
$ |
143,663 |
|
Working capital |
|
$ |
139,441 |
|
|
$ |
125,774 |
|
Total assets |
|
$ |
238,169 |
|
|
$ |
211,498 |
|
Total liabilities |
|
$ |
177,736 |
|
|
$ |
120,330 |
|
Total stockholders' equity |
|
$ |
60,433 |
|
|
$ |
91,168 |
|
Common stock outstanding |
|
|
110,964,035 |
|
|
|
60,902,105 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20230309005314/en/
Investor and Media Contact:
Director, Investor Relations and Finance
Mei.Burris@precisionbiosciences.com
Source:
FAQ
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