On March 9, 2023 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2022 (Press release, Prime Medicine, MAR 9, 2023, View Source [SID1234628424]).

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"In 2022, we made important progress in our efforts to build Prime Medicine and demonstrate the promise of Prime Editors as a potentially best-in-class genetic medicine approach," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "In addition to closing our upsized initial public offering in October, we recently disclosed new preclinical proof-of-concept data in multiple indications and announced new preliminary safety analyses demonstrating minimal or no off-target editing in two preclinical models. We believe this progress, coupled with advancements in our chemistry, manufacturing and controls (CMC) and delivery capabilities and Prime Editing platform, provides a tremendous foundation as we aim to maximize Prime Editing’s broad therapeutic potential."

Dr. Gottesdiener continued, "Today, we are delighted to share the achievement of a key milestone, the selection of PM359 as our first development candidate for the treatment of chronic granulomatous disease (CGD). We designed and optimized PM359 to achieve high efficiency and highly precise editing of hematopoietic stem cells (HSC), and have observed long-term in vivo engraftment of Prime Edited HSCs in a mouse model of CGD. We look forward to advancing PM359 into investigational new drug (IND)-enabling studies later this year, while continuing to advance our broader portfolio toward additional preclinical proof-of-concept readouts."

Recent Business Updates

Pipeline

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In February 2023, Prime Medicine nominated PM359 as its first development candidate for the treatment of CGD.

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In January 2023, Prime Medicine announced new preclinical data for several programs, including:

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Friedrich’s ataxia (FRDA): Preclinical proof-of-concept data demonstrated that Prime Editing-mediated removal of pathological repeats in vitro resulted in correction of hypermethylation at the FXN gene, restoring gene expression back to wild-type levels. FRDA is caused by GAA-repeat nucleotide sequence expansions in intron 1 of the FXN gene encoding the frataxin protein, which plays important roles in mitochondria.

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Cystic fibrosis (CF): Preclinical proof-of-concept data demonstrated greater than 70 percent precise editing of the G542X mutational hotspot in vitro, as well as functional restoration of swelling and cystic fibrosis transmembrane conductance regulator (CFTR) protein function in patient-derived intestinal organoids. CF is caused by loss of function mutations in the CFTR gene; F508del and mutations at seven additional hotspots in the CFTR gene, including G542X, are found in 98 percent of CF patients.

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Wilson’s disease (WD) and CGD: Preclinical safety data demonstrated that no guide-dependent Prime Editing activity was detected across hundreds of identified potential off-target sites.

Prime Editing Platform

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In January 2023, Prime Medicine announced new preclinical data utilizing its PASSIGETM (Prime Assisted Site-Specific Integrase Gene Editing) technology in a one-step, non-viral process, which resulted in an approximately 60 percent precise insertion of a 3.5 kilobase transgene of interest at a single targeted site in primary human T cells, resulting in positive expression of the gene product by these cells.

CMC and Delivery

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In January 2023, Prime Medicine announced additional proof-of-concept preclinical data showing lipid nanoparticle (LNP) delivery of Prime Editors to rodent liver, as well as new preclinical data demonstrating that dual adeno-associated virus (AAV) delivery to the central nervous system achieved high efficiency transduction in murine models, with a high level of precise editing in transduced cells.

Corporate

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In October 2022, Prime Medicine completed an upsized initial public offering of common stock at $17.00 per share, raising net proceeds of approximately $180.2 million, after deducting underwriting discounts, commissions and estimated offering expenses.

Anticipated Upcoming Milestones

Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:

Pipeline

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Initiate investigational new drug (IND)-enabling studies of PM359 for the treatment of CGD in 2023.

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Expand preclinical proof-of-concept in vivo, including sharing data from in vivo rodent studies and large animal studies in several programs in the second half of 2023.

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Share in vitro preclinical data in additional liver, eye and neuromuscular programs.

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First IND filing expected as early as 2024 and additional IND filings anticipated in 2025.

Platform

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Continue to develop and optimize non-viral and viral delivery systems and share additional proof-of-concept data from in vivo rodent and large animal studies in the second half of 2023.

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Further explore and demonstrate superior off-target profiles for Prime Editing programs.

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Expand Prime Editing using proprietary recombinase and/or retrotransposon technologies for new and existing programs.

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Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.

Fourth Quarter and Full Year 2022 Financial Results:

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Research and Development (R&D) Expenses: R&D expenses were $29.1 million for the fourth quarter of 2022 and $86.7 million for the year ended December 31, 2022, as compared to $52.9 million for the fourth quarter of 2021 and $70.6 million for the year ended December 31, 2021.

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General and Administrative (G&A) Expenses: G&A expenses were $9.6 million for the fourth quarter of 2022 and $29.8 million for the year ended December 31, 2022, as compared to $7.2 million for the fourth quarter of 2021 and $13.9 million for the year ended December 31, 2021.

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Net Loss: Net loss was $39.3 million for the fourth quarter of 2022 and $121.8 million for the year ended December 31, 2022, as compared to $62.9 million for the fourth quarter of 2021 and $165.4 million for the year ended December 31, 2021.

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Cash Position: As of December 31, 2022, cash, short-term investments and related party short term investments were $293.9 million, as compared to $269.6 million as of December 31, 2021. This increase was primarily due to net proceeds of $180.2 million from Prime Medicine’s initial public offering of stock, which was completed in October 2022, partially offset by cash used to fund operating activities for the year ended December 31, 2022.

Financial Guidance

Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and short-term investments as of December 31, 2022 will be sufficient to fund its anticipated operating expenses and capital expenditure requirements into 2025.

On March 9, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported financial results for the fourth quarter and fiscal year ended December 31, 2022 and provided a business update (Press release, Precision Biosciences, MAR 9, 2023, View Source [SID1234628423]).

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"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 Michael Amoroso, Chief Executive Officer of Precision BioSciences. "I’m very proud of our team’s work to align with the U.S. Food and Drug Administration (FDA) on the azer-cel chemistry, manufacturing and controls (CMC) package in late 2022 and look forward to providing updates on our allogeneic CAR T therapies as we progress towards key decision points on next steps for azer-cel and PBCAR19B."

Mr. Amoroso continued, "We have continued to leverage the ARCUS gene editing platform in progressing multiple in vivo gene editing programs, independently and with select partners such as Novartis, Lilly, and iECURE, with the goal of differentiating ARCUS on safety, high efficiency gene insertion, and complex edits aimed at restoring function for specific genetic diseases. We expect 2023 to be an important year as the first ARCUS nuclease for in vivo gene insertion is planned to move towards a Clinical Trial Application (CTA) and/or an Investigational New Drug (IND) application. We look forward to providing progress updates on our in vivo gene editing pipeline, including meaningful learnings with our partnered programs, and next steps from our strategic prioritization exercise at the R&D Day planned for mid-2023."

Ex Vivo Allogeneic CAR T Portfolio:

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/May 2023 time frame, once appropriate follow-up from the current cohort is available.

During the CAR T update, the Company plans to provide additional long-term follow up from the azer-cel cohorts presented at ASH (Free ASH Whitepaper) 2021 and ASCO (Free ASCO Whitepaper) 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 Vivo Gene Editing Portfolio:

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 95%. Similar levels of HBsAg reduction were observed in a newly developed mouse model of HBV infection following administration of ARCUS mRNA using lipid nanoparticle delivery. The Company plans to present additional data in 2023.

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 November 2022 remains ongoing to assess diseases with highest unmet need in a dynamic regulatory and competitive gene editing landscape.

Quarter Ended December 31, 2022 Financial Results:

Revenues: Total revenues for the quarter ended December 31, 2022 were $10.6 million, as compared to $6.3 million for the same period in 2021. The increase of $4.3 million in revenue during the quarter ended December 31, 2022 was primarily the result of an increase of $6.0 million in revenue recognized under the Novartis Agreement offset by a decrease of $1.0 million from agriculture partnering collaborations and a timing related decrease of $0.7 million under the Lilly Agreement.

Research and Development Expenses: Research and development expenses were $21.1 million for the quarter ended December 31, 2022, as compared to $26.5 million for the same period in 2021. The decrease of $5.4 million was primarily due to a decrease of $4.0 million related to the iECURE agreement and a decrease of $2.9 million in employee-related and other operational costs driven by the separation of Elo in 2021, offset by an increase of $1.5 million in research costs related to our in vivo gene editing preclinical studies.

General and Administrative Expenses: General and administrative expenses were $10.1 million for the quarter ended December 31, 2022, as compared to $10.6 million for the same period in 2021.

Other Income and Expense: Total other expense was $7.9 million for the quarter ended December 31, 2022, as compared to total other income of $8.5 million for the same period in 2021. The increase was primarily driven by a $10.8 million impairment of prepaid expenses related to the iECURE PCSK9 collaboration in the quarter ended December 31, 2022.

Net Loss: Net loss was $28.5 million, or $(0.26) per share (basic and diluted), for the quarter ended December 31, 2022, as compared to net loss of $22.3 million, or $(0.37) per share (basic and diluted), for the same period in 2021. Weighted average shares of common stock outstanding were approximately 111.0 million for the quarter ended December 31, 2022, as compared to approximately 60.7 million for the quarter ended December 31, 2021. The increase in weighted average shares of common stock outstanding was primarily due to a $50 million underwritten offering of common stock and Novartis’ $25 million equity investment in the year ended December 31, 2022.

Fiscal Year 2022 Financial Results:

Cash and Cash Equivalents: As of December 31, 2022, Precision had $189.6 million in cash and cash equivalents. The Company expects that existing cash and cash equivalents, continued operational discipline, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements through Q1 2025.

Revenues: Total revenues for the year ended December 31, 2022 were $25.1 million, as compared to $115.5 million for the same period in 2021. The decrease of $90.4 million in revenue during the year ended December 31, 2022 was primarily the result of the absence of $72.9 million in revenue recognized under the Servier Agreement and a decrease of $17.9 million under the iECURE agreement subsequent to the full satisfaction of the performance obligations in April 2021 and August 2021, respectively, a decrease of $5.4 million under the Lilly Agreement, and a decrease of $3.7 million from agriculture partnering collaborations that were transferred to Elo Life Systems, Inc. ("Elo") upon separation in 2021. These decreases in revenue were partially offset by an increase of $9.5 million in revenue recognized under the Novartis Agreement.

Research and Development Expenses: Research and development expenses were $83.9 million for the year ended December 31, 2022, as compared to $115.2 million for the same period in 2021. The decrease of $31.3 million was primarily due to a decrease of $11.3 million in expense related to the Servier Program Purchase Agreement, a decrease of $8.9 million in external development costs associated with our allogeneic CAR T product candidates, a decrease of $6.7 million in employee-related and other operational costs driven by the separation of Elo in 2021, and a decrease of $4.5 million in clinical manufacturing organization and research costs related to our preclinical studies.

General and Administrative Expenses: General and administrative expenses were $41.5 million for the year ended December 31, 2022, as compared to $39.7 million for the same period in 2021. The increase of $1.8 million was primarily due to increased share-based compensation expense offset by decreases in insurance, legal, and information technology expenses.

Other Income and Expense: Total other expense was $11.3 million for the year ended December 31, 2022, as compared to total other income of $8.8 million for the same period in 2021. The increase was primarily driven by a $10.8 million impairment of prepaid expenses related to the iECURE PCSK9 collaboration.

Net Loss: Net loss was $111.6 million, or $(1.27) per share (basic and diluted), for the year ended December 31, 2022, as compared to net loss of $30.6 million, or $(0.52) per share (basic and diluted), for the same period in 2021. Weighted average shares of common stock outstanding were approximately 87.9 million for the year ended December 31, 2022, as compared to approximately 58.7 million for the year ended December 31, 2021. The increase in weighted average shares of common stock outstanding was primarily due to a $50 million underwritten offering of common stock and Novartis’ $25 million equity investment in 2022.

On March 9, 2023 OPKO Health, Inc. (NASDAQ: OPK) reported that it will hold an R&D Day event on March 20, 2023 beginning at 4:00 p.m. Eastern time (Press release, Opko Health, MAR 9, 2023, View Source [SID1234628422]). The two-hour virtual event will provide an opportunity to hear from OPKO executives about the ModeX Therapeutics multispecific technology platforms and applications in oncology and infectious diseases. Management will also review the MDX-2201 technology underlying the recently announced license and collaboration agreement with Merck for a vaccine against Epstein-Barr virus. Presenters will include:

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Phillip Frost, M.D., Chief Executive Officer and Chairman
Elias Zerhouni, M.D., President and Vice Chairman
Gary Nabel, M.D., Ph.D., Chief Innovation Officer
Ronnie Wei, Ph.D., Head of Biologics Discovery and Development, ModeX Therapeutics
John Mascola, M.D., Chief Scientific Officer, ModeX Therapeutics
Vijay Chhajlani, Ph.D., Chief Technology Officer, ModeX Therapeutics
There is no need to pre-register for the event. A live and archived webcast will be available on OPKO’s Investor Relations website. Following management’s presentations, a Q&A session will be available via the chat function of the webcast.

On March 9, 2023 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported a business update and reported fourth quarter and full year 2022 financial results (Press release, Oncternal Therapeutics, MAR 9, 2023, View Source [SID1234628421]).

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"2022 was an important year for Oncternal. With the recent initiation of our Phase 1/2 clinical study for ONCT-808 in advanced hematological malignancies, and the upcoming filing of our IND for ONCT-534 in advanced prostate cancer, we expect to have three product candidates in the clinic later this year," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "The data presented at ASH (Free ASH Whitepaper) 2022 further validated the rationale for our Phase 3 study for patients with MCL, and also strengthened our belief that zilovertamab may provide a new treatment option for patients with CLL with 17p deletion or TP53 mutations, which is a substantial and highly underserved group of patients. We look forward to a catalyst-rich year ahead while continuing to exercise prudent cash management as we expect our existing cash resources will last into the first quarter of 2024."

Recent Highlights

In December 2022, we announced an interim clinical data update from ongoing Study CIRM-0001, a Phase 1/2 clinical trial of zilovertamab in combination with ibrutinib for the patients with relapsed or refractory (R/R) Mantle Cell Lymphoma (MCL) and treatment naïve or R/R chronic lymphocytic leukemia (CLL) [NCT03088878] at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2022 Annual Meeting.
Objective response rate (ORR) of 89% (25 of 28 evaluable patients) observed for patients with R/R MCL treated with zilovertamab plus ibrutinib, which compares favorably to the historical ORR of 66% for ibrutinib monotherapy
Complete response (CR) rate of 43% for R/R MCL patients treated with zilovertamab plus ibrutinib (12 of 28 evaluable patients), which compares favorably to the historical ORR of 20% for ibrutinib monotherapy
The combination of zilovertamab and ibrutinib continued to be well tolerated, with a safety profile consistent with or improved compared with historical data for ibrutinib monotherapy
Median progression-free survival (PFS) had not been reached for R/R MCL patients with TP53 mutation treated with zilovertamab plus ibrutinib, with a landmark PFS of approximately 85% at 15 months, which compares favorably to the historical ibrutinib monotherapy median PFS of 4 months
Landmark PFS of 100% at 42 months for patients with CLL with 17p deletion or TP53 mutations treated with zilovertamab plus ibrutinib, which compares favorably to a recent data update from the ALPINE study of patients with R/R CLL, with a landmark PFS of 55.7% at 24 months for ibrutinib monotherapy, and 77.6% at 24 months for zanubrutinib monotherapy
Mutation in the TP53 gene is the most commonly acquired mutation in cancer, including hematological malignancies. TP53 aberrations are associated with markedly decreased survival and predict inadequate therapeutic response, thus being among the strongest predictive and prognostic factors guiding treatment decisions in CLL and MCL
In December 2022, we received helpful feedback from the FDA from our pre- Investigational New Drug (IND) meeting for ONCT-534, our novel dual-action androgen receptor inhibitor (DAARI)
In January 2023, we obtained our first Institutional Review Board (IRB) approval for the Phase 1/2 study of ONCT-808, our autologous CAR-T targeting ROR1-expressing hematologic malignancies (NCT05588440)
Expected Upcoming Milestones

Zilovertamab, our ROR1 antibody
Opening of additional countries for global clinical registrational Phase 3 Study ZILO-301
Continuing evaluation of potential to treat patients with CLL & MCL and TP53 mutations and/or 17p deletions
ONCT-808, our autologous ROR1-targeted CAR T cell therapy
Initial clinical data available by the end of 2023
ONCT-534, our dual-action androgen receptor inhibitor
U.S. IND application submission planned in mid-2023
Fourth Quarter and Full Year 2022 Financial Results
Our grant revenue was $0.2 million for the fourth quarter ended December 31, 2022, and was $1.5 million for the full year 2022.
Our total operating expenses for the fourth quarter ended December 31, 2022 were $12.1 million, including $1.8 million in non-cash stock-based compensation expense. Research and development expenses for the quarter totaled $8.8 million, and general and administrative expenses for the quarter totaled $3.3 million. Net loss for the fourth quarter was $11.4 million, or a loss of $0.20 per share, basic and diluted. For the full year 2022, total operating expenses were $46.4 million, including $7.4 million in non-cash stock-based compensation expense, and our net loss was $44.2 million, or a loss of $0.84 per share, basic and diluted.
As of December 31, 2022, we had approximately 57.5 million shares of common stock outstanding, $63.7 million in cash, cash equivalents and short-term investments and no debt. We believe these funds will be sufficient to fund our operations into the first quarter of 2024.

On March 9, 2023 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) ("Merrimack" or the "Company") reported its full year 2022 financial results for the period ended December 31, 2022 (Press release, Merrimack, MAR 9, 2023, View Source [SID1234628420]).

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"We are encouraged by the results of the NAPOLI 3 trial as reported by Ipsen in November 2022 and the guidance that Ipsen has subsequently provided regarding its intention to file a supplemental New Drug Application with the U.S. Food and Drug Administration for Onivyde in previously untreated patients with metastatic pancreatic ductal adenocarcinoma ("mPDAC"), which could lead to a potential future milestone payment from Ipsen" said Gary Crocker, CEO and Chairman of Merrimack’s Board of Directors. "As a result of the exercise of stock options during the fourth quarter of 2022 and continued reductions in our operating expenses during 2022 we expect that our cash and cash equivalents as of December 31, 2022, will be sufficient to continue our operations beyond 2027, which we estimate to be the latest date when the longest-term potential Ipsen milestone may be achieved."

Full Year 2022 Financial Results

Merrimack reported net loss of $1.5 million for the year ended December 31, 2022, or $0.11 per basic share, compared to a net loss of $2.5 million, or $0.18 per basic share, for the same period in 2021.

Merrimack reported a gain on sale of assets for the year ended December 31, 2022, of $0.4 million compared to $0.1 million for the same period in 2021.

General and administrative expenses for the year ended December 31, 2022 were $2.2 million, compared to $2.6 million for the same period in 2021.

As of December 31, 2022, Merrimack had cash and cash equivalents of $19.4 million, compared to $14.2 million as of December 31, 2021 The increase in cash was primarily a result of $6.5 million of proceeds from the exercise of stock options.

As of December 31, 2022, Merrimack had 14.2 million shares of common stock outstanding.

Updates on Programs Underlying Potential Milestone Payments

Ipsen

Metastatic Pancreatic Ductal Adenocarcinoma

In November 2022, Ipsen announced the Phase III NAPOLI 3 trial of Onivyde (irinotecan liposome injection) plus 5-fluorouracil/leucovorin and oxaliplatin (the "NALIRIFOX regimen") met its primary endpoint demonstrating clinically meaningful and statistically significant improvement in overall survival compared to nab-paclitaxel plus gemcitabine in 770 previously untreated patients with mPDAC and key secondary efficacy outcome of progression-free survival (PFS) also showed significant improvement over the comparator arm. Ipsen also announced that the safety profile of Onivyde in the NAPOLI 3 trial was consistent with those observed in the previous phase I/II mPDAC study

In January 2023, Ipsen presented clinical trial results at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium

In February 2023 Ipsen provided guidance to investors that it intends to file a supplemental New Drug Application with the U.S. Food and Drug Administration during the first half of 2023 following the Fast Track Designation granted in 2020 for the use of Onivyde in combination with oxaliplatin plus 5-fluorouracil/leucovorin for the treatment of patients with previously untreated mPDAC.

Small Cell Lung Cancer

In August 2022, Ipsen announced that the Phase III RESILIENT trial did not meet its primary endpoint of overall survival compared to topotecan. The trial is evaluating Onivyde versus topotecan in patients with small cell lung cancer, who have progressed on or after platinum-based first-line therapy treatment. In the announcement, Ipsen indicated that detailed results from the RESILIENT trial would be presented at an upcoming medical conference. The analysis concluded that the primary endpoint overall survival was not met in patients treated with Onivyde versus topotecan. However, a doubling of the secondary endpoint of objective response rate in favor of Onivyde was observed. In the August 2022 announcement, Ipsen reported that the clinical study results would be communicated with the regulatory agency. Ipsen indicated that while the results from the analysis of the RESILIENT trial have not demonstrated an overall survival benefit with Onivyde in patients in second-line small cell lung cancer, Ipsen intends to analyze the data further before decisions regarding next steps are made

To date, there have been no further announcements by Ipsen regarding these matters and it remains unclear as to whether Ipsen will continue to seek approval for the use of Onivyde in the small cell lung cancer application. If Ipsen elects not to proceed with seeking regulatory approval, or if regulatory approval is not obtained, Merrimack would not be entitled to the $150 million milestone payment tied to FDA approval of Onivyde for treatment of small cell lung cancer.

Elevation Oncology

In January 2023, Elevation announced it is pausing further investment in the clinical development of seribantumab and intends to pursue further development only in collaboration with a partner. Elevation also announced that it intends to present additional interim data from its study of seribantumab in the first half of 2023.