On March 2, 2023 Xilio Therapeutics, Inc. (Nasdaq: XLO), a biotechnology company developing tumor-activated immuno-oncology therapies for people living with cancer, reported pipeline progress and business updates and reported financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Xilio Therapeutics, MAR 2, 2023, View Source [SID1234628075]).

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"In 2022, we made significant progress against our goals, advancing the clinical development of all three of our novel tumor-activated immuno-oncology programs. Importantly, we observed promising early clinical evidence of our platform in the first two on-treatment tumor samples from patients treated with XTX202, where both the pharmacodynamics in the tumor and in the patients’ peripheral blood were consistent with our expectations for how our tumor-activated molecules are designed to perform," said René Russo, Pharm.D., chief executive officer of Xilio. "In addition, we look forward to several anticipated milestones in 2023, including reporting clinical data from our trials for XTX101, XTX202 and XTX301."

Pipeline and Business Updates

XTX202: tumor-activated, engineered IL-2

XTX202 is an investigational tumor-activated beta-gamma biased (non-alpha), engineered IL-2 molecule designed to potently stimulate CD8+ effector T cells and natural killer (NK) cells without concomitant stimulation of regulatory T cells when activated (unmasked) in the tumor microenvironment (TME). XTX202 is currently being evaluated in an ongoing Phase 1 clinical trial in patients with advanced solid tumors.

● Xilio recently completed dosing patients at the 1 mg/kg dose level (dose level four) and is currently dosing patients at the 1.4 mg/kg dose level (dose level five) in monotherapy dose-escalation (Part 1A) for the Phase 1 clinical trial.
● As of March 1, 2023, a total of 16 patients have been treated with XTX202 in the outpatient setting at five dose levels ranging from 0.27 mg/kg to 1.4 mg/kg once every three weeks (Q3W) in Part 1A for the Phase 1 clinical trial, and monotherapy dose expansion (Part 1B) is open for enrollment.
● No signs of vascular leak syndrome (VLS), including hypotension or decreases in albumin (an early sign of VLS) or hemodynamic compromise, have been observed in patients to date.
● Xilio recently reported preliminary intra-tumoral pharmacokinetics (PK) and pharmacodynamic (PD) data for two patients treated with XTX202, each of whom had an optional on-treatment tumor biopsy and are the only two patients from whom a tumor sample analysis is available to date. For each patient, the tumor sample featured increased numbers of stromal tumor infiltrating

lymphocytes (TILs) and increased frequency of CD8+ effector T cells among these TILs. At the time of the tumor sample, these changes occurred in each patient in the absence of peripheral changes to CD8+ effector T cells demonstrating preliminary evidence of tumor-selective activation.
● A maximum tolerated dose has not yet been determined, and enrollment in Part 1A and Part 1B of the clinical trial is ongoing.

Xilio anticipates multiple milestones for XTX202 through the end of 2023:

● Initiate patient enrollment in a Phase 2 clinical trial evaluating XTX202 as a monotherapy in patients with unresectable or metastatic melanoma and metastatic renal cell carcinoma in April 2023.
● Report preliminary anti-tumor activity, safety, PK and PD data from the Phase 1/2 clinical trial in the third quarter of 2023. Xilio anticipates the reported data will include approximately 15-20 patients across a range of solid tumors treated at the 1 mg/kg dose or higher across all cohorts in the Phase 1/2 clinical trial.

XTX301: tumor-activated, engineered IL-12

XTX301 is an investigational tumor-activated, engineered IL-12 molecule designed to potently stimulate anti-tumor immunity and reprogram the TME of poorly immunogenic "cold" tumors towards an inflamed, or "hot," state.

● Xilio recently opened clinical trial sites and is actively screening patients for enrollment at a starting dose of 5.0 ug/kg (0.005 mg/kg) in monotherapy dose-escalation for its Phase 1 clinical trial evaluating the safety and tolerability of XTX301 in patients with advanced solid tumors.
● In addition, Xilio anticipates reporting preliminary safety data from the Phase 1 clinical trial into at least the third dose level in the fourth quarter of 2023.

XTX101: tumor-activated anti-CTLA-4

XTX101, an investigational Fc-enhanced, tumor-activated anti-CTLA-4, is currently being evaluated in an ongoing Phase 1 clinical trial in patients with advanced solid tumors.

● As of March 1, 2023, 24 patients have been treated with XTX101 in the Phase 1 clinical trial. Enrollment in monotherapy dose-escalation (Part 1A) has been completed, and enrollment in monotherapy dose-expansion (Part 1B) is ongoing.
● Xilio has determined a recommended Phase 2 dose (RP2D) of 150 mg once every six weeks (Q6W).
● Xilio anticipates reporting preliminary safety, PK, PD and anti-tumor activity data from the Phase 1 clinical trial in the second quarter of 2023.
● In addition, as previously announced, Xilio plans to continue to explore strategic opportunities to advance XTX101 with a partner beyond the current Phase 1 trial.

Upcoming Presentations

Xilio reported the acceptance of the following two abstracts for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 in Orlando, Florida.

Xilio will present a poster highlighting preclinical data demonstrating anti-tumor activity of XTX301 in preclinical mouse models with improved tolerability compared to a systemically active IL-12 molecule.

● Presentation title: A half-life extended, tumor-activated IL-12 increased the infiltration of effector immune cells into the tumor microenvironment and demonstrated anti-tumor activity in syngeneic mouse models.
● Session date and time: Sunday, April 16, 2023, at 1:30 pm to 5:00 pm E.T.
● Abstract number: 587
● Poster Board Number: 21

Xilio will present a poster highlighting preclinical data for a discovery-stage, tumor-activated multi-functional molecule.

● Presentation title: Tumor-activated PD1-directed IL-2 increased antigen specific T cells in tumors and demonstrated anti-tumor activity in mice.
● Session date and time: Sunday, April 16, 2023, at 1:30 pm to 5:00 pm E.T.
● Abstract number: 572
● Poster Board Number: 6

Year-End and Fourth Quarter 2022 Financial Results

● Cash Position: Cash and cash equivalents were $120.4 million as of December 31, 2022, compared to $198.1 million as of December 31, 2021.
● Research & Development (R&D) Expenses: R&D expenses were $15.0 million for the quarter ended December 31, 2022, compared to $11.4 million for the quarter ended December 31, 2021. R&D expenses were $59.2 million for the year ended December 31, 2022, compared to $51.2 million for the year ended December 31, 2021. The year-over-year increase was primarily driven by increased manufacturing, preclinical and clinical development activities for XTX301, increased clinical development activities for XTX202, increased personnel-related costs and increased costs related to the company’s earlier-stage programs. These increases were partially offset by a decrease in manufacturing and preclinical expenses for XTX202 as the program advanced further into clinical development in 2022.
● General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the quarters ended December 31, 2022 and December 31, 2021, respectively. G&A expenses were $29.9 million for the year ended December 31, 2022, compared to $23.9 million for the year ended December 31, 2021. The year-over-year increase was primarily driven by increased personnel-related costs and an increase in expenses associated with operating as a public company.
● Net Loss: Net loss was $22.5 million for the quarter ended December 31, 2022, compared to $19.7 million for the quarter ended December 31, 2021. Net loss was $88.2 million for the year ended December 31, 2022, compared to $75.8 million for the year ended December 31, 2021.
Financial Guidance

Xilio continues to anticipate that its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2024.

About XTX202 (IL-2) and the Phase 1/2 Clinical Trial

XTX202 is an investigational tumor-activated beta-gamma biased (non-alpha), engineered IL-2 molecule designed to potently stimulate CD8+ effector T cells and natural killer (NK) cells without concomitant stimulation of regulatory T cells when activated (unmasked) in the tumor microenvironment (TME). The Phase 1 clinical trial for XTX202 is a first-in-human, multi-center, open-label trial designed to evaluate

the safety and tolerability of XTX202 as a monotherapy in patients with advanced solid tumors. Please refer to NCT05052268 on www.clinicaltrials.gov for additional details.

The planned Phase 2 clinical trial for XTX202 is a multi-center, open-label trial designed to evaluate the safety and anti-tumor activity of XTX202 as a monotherapy in patients with unresectable or metastatic melanoma and metastatic renal cell carcinoma at the recommended Phase 2 dose. Please refer to NCT05052268 on www.clinicaltrials.gov for additional details.

About XTX301 (IL-12) and the Phase 1 Clinical Trial

XTX301 is an investigational tumor-activated, engineered IL-12 molecule designed to potently stimulate anti-tumor immunity and reprogram the tumor microenvironment (TME) of poorly immunogenic "cold" tumors towards an inflamed, or "hot," state. In November 2022, the U.S. Food and Drug Administration cleared Xilio’s investigational new drug application for the evaluation of XTX301 as a potential treatment for patients with advanced solid tumors.

The Phase 1 clinical trial for XTX301 is a first-in-human, multi-center, open-label trial designed to evaluate the safety and tolerability of XTX301 as a monotherapy in patients with advanced solid tumors. Please refer to NCT05684965 on www.clinicaltrials.gov for additional details.

About XTX101 (anti-CTLA-4) and the Phase 1 Clinical Trial

XTX101 is an investigational Fc-enhanced, tumor-activated anti-CTLA-4 monoclonal antibody designed to deplete regulatory T cells when activated (unmasked) in the TME. The Phase 1 clinical trial is a first-in-human, multi-center, open-label trial designed to evaluate the safety and tolerability of XTX101 for the treatment of adult patients with advanced solid tumors. Please refer to NCT04896697 on www.clinicaltrials.gov for additional details.

On March 2, 2023 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases and gene therapies, reported financial results for the fourth quarter and full year ended December 31, 2022 and provided a business update (Press release, Selecta Biosciences, MAR 2, 2023, View Source [SID1234628074]).

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"2022 was a successful year for Selecta. We delivered on key milestones across our pipeline and established strategic collaborations that have allowed us to drive forward our development programs," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. "The Phase 3 DISSOLVE I and II clinical trials investigating SEL-212 in chronic refractory gout remains on track, and we expect to deliver topline data later this quarter. We look forward to the potential for SEL-212 to address the unmet needs of patients suffering from chronic refractory gout. In addition, we are currently enrolling patients in ReiMMAgine, our Phase 1/2 clinical trial of SEL-302 for the treatment of MMA. We intend to continue to leverage learnings from our clinical experience and growing safety database to propel our next-generation programs toward multiple IND filings and advance our wholly owned pipeline using ImmTOR in combination with a proprietary IL-2 (ImmTOR-IL), which we believe has the potential to address the unmet need for precision immunotherapy for autoimmune diseases."

Recent Program Highlights and Anticipated Upcoming Milestones:

Tolerogenic Therapies for Autoimmune Disease:

ImmTOR-IL: In December 2022, the Company selected an identified IL-2 candidate.

The combination of ImmTOR and IL-2 (ImmTOR-IL) represents an evolution of the precision immune tolerance platform, and an opportunity to further advance and expand the Company’s wholly-owned pipeline.
The Company plans to initiate IND enabling studies in 2023, while also exploring multiple autoimmune indications that may be suitable for study with ImmTOR-IL, with an initial focus on diseases of the liver.
Gene Therapies:

SEL-302 for MMA: In December 2022, the Company initiated ReiMMAgine, the Phase 1/2 clinical trial of SEL-302, an adeno-associated virus (AAV) gene therapy combined with ImmTOR for the treatment of MMA.

The ReiMMAgine trial is now enrolling patients and aims to evaluate the safety, tolerability and efficacy of SEL-302.
SEL-018 IgG Protease (Xork) for Late-Onset Pompe Disease: In January 2023, the Company announced an exclusive licensing and development agreement for IdeXork (Xork), a next-generation immunoglobulin G (IgG) protease, to be developed for use with AT845, Astellas Pharma Inc.’s investigational AAV-based treatment for Late-Onset Pompe disease (LOPD) in adults.

Xork is differentiated by its low cross reactivity to pre-existing antibodies in human serum, which the Company believes has the potential to expand access of life-changing gene therapies to more patients.
Under the terms of the agreement, Selecta received a $10M upfront payment and is eligible to receive up to $340M upon the achievement of certain additional development and commercial milestones plus royalties on commercial sales. Selecta is responsible for the early development activities and manufacturing of Xork and will maintain rights to the development of additional indications beyond LOPD.
Biologic Therapies:

SEL-212 for chronic refractory gout: The DISSOLVE I & II Phase 3 clinical trials for SEL-212, which has been licensed to Swedish Orphan Biovitrum AB (publ.) (Sobi), are on track with topline data expected in Q1 2023.

Selecta has been responsible for executing the Phase 3 clinical trials, with reimbursement from Sobi, and Sobi will be responsible for biologics license application filing and commercialization.
ImmTOR with IgA Protease for IgA Nephropathy (IgAN): In December 2022, the Company selected a next generation Immunoglobulin A (IgA) protease from IGAN Biosciences, Inc. to develop in combination with ImmTOR for the treatment of IgAN.

ImmTOR in combination with this new class of IgA protease, which is derived from a commensal bacteria and has low levels of baseline anti-drug antibodies, is designed to address the underlying pathophysiology of IgAN.
The Company plans to initiate IND enabling studies in 2023.
Further Corporate and Partnership Updates:

In November 2022, Blaine Davis was appointed as Chief Financial Officer. Mr. Davis brings more than 25 years of experience in investor relations, business development, corporate affairs and sales and marketing at life sciences companies, with a particular focus on rare diseases.

Fourth Quarter and Full Year 2022 Financial Results:

Cash Position: Selecta had $136.2 million in cash, cash equivalents, marketable securities, and restricted cash as of December 31, 2022, as compared to cash, cash equivalents, marketable securities, and restricted cash of $129.4 million as of December 31, 2021. Selecta believes its available cash, cash equivalents, restricted cash, and marketable securities will be sufficient to meet its operating requirements into mid-2024.

Collaboration and License Revenue: Revenue for the fourth quarter and full year 2022 was $16.8 million and $110.8 million, respectively, as compared to $29.9 million and $85.1 million for the same periods in 2021. Revenue was primarily driven by the license agreement with Sobi resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program.

Research and Development Expenses: Research and development expenses for the fourth quarter and full year 2022 were $19.0 million and $72.4 million, respectively, as compared to $20.3 million and $68.7 million for the same periods in 2021. The quarterly decrease was primarily driven by a decrease in expenses incurred for the SEL-212 clinical program. The annual increase was primarily driven by expenses incurred for preclinical programs, increased personnel expense and stock compensation expense.

General and Administrative Expenses: General and administrative expenses for the fourth quarter and full year 2022 were $6.3 million and $23.9 million, respectively, as compared to $5.5 million and $20.9 million for the same periods in 2021. The quarterly and annual increases were primarily driven by increases in stock compensation and personnel expenses.

Net Income (loss): For the fourth quarter and full year 2022, Selecta reported net income of $5.9 million, or basic net income per share of $0.04, and net income of $35.4 million, or basic net income per share of $0.24, respectively. For the fourth quarter and full year 2021, Selecta reported net income of $12.2 million, or $0.10 per share, and a net loss of $(25.7) million, or $(0.22) per share, respectively.

Conference Call and Webcast Reminder

Selecta’s management will host a conference call at 8:30 AM ET today to provide a corporate update and review the Company’s fourth quarter and full year 2022 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 8768458. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the Company’s website, www.selectabio.com.

On March 2, 2023 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a biopharmaceutical company focused on the localized treatment of solid tumors, reported its financial results for the year ended December 31, 2022, and a pipeline update including the status of the interim analysis of for the Company’s Phase III TIGeR-PaC clinical trial (Press release, Renovorx, MAR 2, 2023, View Source [SID1234628073]).

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RenovoRx anticipates finalizing the interim analysis for the TIGeR-PaC Phase III trial and releasing the results within the next week or so. To date, 47 out of 114 target post-SBRT/chemotherapy patients have been randomized in the TIGeR-PaC clinical trial, and the Company has received reports of 26 events (deaths) in this population. This 26th event represents 30% of the 86 events and thus triggered the first planned interim analysis and a meeting of the study’s Data Monitoring Committee ("DMC") to evaluate the ongoing progression of the Phase III clinical trial. Upon completion of the analysis and assessment of the data by the DMC, and RenovoRx’s subsequent review of their findings, the Company will report the first interim analysis

TIGeR-PaC is a randomized multi-center Phase III open label clinical trial designed to investigate the Company’s first product candidate, RenovoGemTM, which utilizes RenovoRx’s proprietary therapy platform, RenovoTAMP, to provide targeted intra-arterial delivery of FDA-approved chemotherapy, gemcitabine, to treat locally advanced pancreatic cancer (LAPC) following stereotactic body radiation therapy (SBRT). The study is comparing treatment with RenovoTAMP versus standard of care systemic intravenous (IV) administration of gemcitabine and nab-paclitaxel. The study is designed to randomize 114 patients (57 in each arm) with all patients receiving upfront induction chemotherapy and SBRT. Final analysis will be conducted after 86 protocol-specified events have occurred in the SBRT population with two planned interim analyses: the first analysis when 30% of the specified events have been reported (data announcement forthcoming) and the second analysis when 60% of the events have been reported (expected in 2024

RenovoRx has continued to grow in 2022 as we have focused on advancing our innovative therapy platform with the vision of disrupting the current standard of care," said Shaun Bagai, CEO of RenovoRx. "Over the past year, we have advanced our mission to transform oncology therapy with our TIGeR-PaC Phase III study, treating pancreatic cancer, and expanding our clinical pipeline to treat other solid tumors. We have also built a strong internal team, and we look forward to continued progress as we strive to give our patients not just more time but more quality time that they can spend with their loved ones

Fiscal Year 2022 and Subsequent Operational Highlights:

● Announced Initial Results in Pharmacokinetic Substudy at the 2023 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancer Symposium: Data on RenovoGem supports potential for RenovoTAMP therapy platform to increase local gemcitabine delivery and decrease side effects of pancreatic cancer treatment

● Eighth U.S. patent issued extending the intellectual property coverage of the RenovoTAMP therapy platform

● Acceptance of four clinical data abstracts at the 2023 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancer Symposium

● Presented RenovoTAMP therapy platform at the Advanced Interventional Management Symposium

● Presented at Symposium on Clinical Interventional Oncology

● Appointed Angela Gill Nelms as Chief Operating Officer

● Appointed James Ahlers as Chief Financial Officer

● Participated in University of Cambridge Academy of Therapeutic Sciences Gateway to Translation Seminar Series

● Presented preclinical research data demonstrating potential utility for treatment of bile duct cancer

● Enrolled first pancreatic cancer patient at Columbia University’s New York-Presbyterian Hospital Irving Medical Center in TIGeR-PaC clinical trial study

● Presented at 2022 SPECTRUM Conference

Financial Highlights for the Fiscal Year Ended December 31, 2022

● Cash, cash equivalents and marketable securities as of December 31, 2022, were $6.4 million. We estimate that our current capital resources will be sufficient to fund our operating expenses and capital expenditure requirements into the third quarter of 2023.

● Total assets and total liabilities as of December 31, 2022, were $7.3 million and $1.1 million, respectively.

● Total operating expenses were $10.0 million for the year ended December 31, 2022, compared to $5.7 million for the year ended December 31, 2021. The increase was related to higher research and development expenses and general and administrative expenses.

● Research and development expenses were $4.3 million for the year ended December 31, 2022, compared to $3.0 million for the year ended December 31, 2021. The increase was primarily related to higher costs for contracted research development and consulting, including employee related costs and costs associated with a secondary manufacturer for the RenovoCath delivery system. In addition, allocated general administrative support costs increased during the year.

● General and administrative expenses were $5.6 million for the year ended December 31, 2022, compared to $2.6 million for the year ended December 31, 2021. The increase was primarily related to higher professional and consulting costs, including legal expenses, employee-related costs and D&O and liability insurance and other expenses. The increase was partially offset by general and administrative support costs allocated to research and development expenses.

● Net loss was $9.9 million for the ended December 31, 2022, compared to net loss of $6.3 million for year ended December 31, 2021.

● Net loss per share, basic and diluted were ($1.09) for the year ended December 31, 2022, compared to ($1.21) for the year ended December 31, 2021.

● As of December 31, 2022 the Company had 9,097,701 common shares outstanding.

On March 2, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2022 (Press release, Puma Biotechnology, MAR 2, 2023, View Source [SID1234628072]). Unless otherwise stated, all comparisons are for the fourth quarter and full year 2022, compared to the fourth quarter and full year 2021

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Product revenue, net consists entirely of revenue from sales of NERLYNX, Puma’s first commercial product. Product revenue, net for the fourth quarter of 2022 was $53.7 million, compared to $51.0 million in the fourth quarter of 2021. Product revenue, net for the full year 2022 was $200.0 million, compared to $189.1 million in 2021

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $5.6 million, or $0.12 per share, for the fourth quarter of 2022, compared to net income of $4.2 million, or $0.10 per basic and diluted share, for the fourth quarter of 2021. In the fourth quarter of 2022, Puma increased its legal accrual by approximately $12.4 million to reflect the settlement of a lawsuit. Net income for full year 2022 was $0.0 million, or $0.00 per basic and diluted share, compared to a net loss of $29.1 million, or $0.72 per share, for full year 2021

Non-GAAP adjusted net loss was $3.0 million, or $0.07 per share, for the fourth quarter of 2022, compared to non-GAAP adjusted net income of $8.4 million, or $0.21 per basic and diluted share, for the fourth quarter of 2021. Non-GAAP adjusted net income for full year 2022 was $11.8 million, or $0.26 per basic and diluted share, compared to non-GAAP adjusted net income of $3.5 million, or $0.09 per basic share and $0.08 per diluted share, for full year 2021. Non-GAAP adjusted net income/loss excludes stock-based compensation expense. For a reconciliation of GAAP net income/loss to non-GAAP adjusted net income/loss and GAAP net income/loss per share to non-GAAP adjusted net income/loss per share, please see the financial tables at the end of this news release

Net cash provided by operating activities for the fourth quarter of 2022 was $7.7 million, compared to net cash used in operating activities of $5.4 million for the fourth quarter of 2021. Net cash used in operating activities for full year 2022 was $15.8 million, compared to net cash provided by operating activities of $20.7 million for full year 2021. At December 31, 2022, Puma had cash, cash equivalents, and marketable securities of $81.1 million, compared to cash, cash equivalents, and marketable securities of $82.1 million at December 31, 2021

In the fourth quarter of 2022, we are very pleased to have achieved $53.7 million in NERLYNX revenues," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "This is being driven by the U.S. commercial revenues from NERLYNX and our commercial execution, which is designed to support increased patient awareness and access to NERLYNX. Puma also continued to execute on its key milestones during the quarter, which included presentation of updated findings from the Phase II SUMMIT basket trial of neratinib for EGFR exon 18-mutant non-small cell lung cancer (NSCLC) patients at the EORTC/NCI/AACR Molecular Targets and Cancer Therapeutics Symposium, as well as the presentation of data from the TBCRC-022 trial at the 2022 San Antonio Breast Cancer Symposium," said Mr. Auerbach. "We remain committed to supporting patients with HER2-positive breast cancer with NERLYNX and, with the addition of alisertib to our pipeline, we hope to be able to support more cancer patients in the future

Mr. Auerbach added, "We anticipate the following key milestones over the next 12 months: (i) publication of the biomarker studies from the randomized trial of alisertib plus fulvestrant versus alisertib alone in hormone receptor positive, HER2-negative breast cancer (H1 2023); (ii) reporting biomarker data from the randomized trial of alisertib plus paclitaxel versus paclitaxel alone in hormone receptor positive, HER2-negative breast cancer (H1 2023); (iii) reporting data from an ongoing investigator sponsored Phase I/II trial of alisertib plus pembrolizumab for the treatment of patients with Rb-deficient head and neck squamous cell cancer (2023); (iv) conducting a meeting with the FDA to discuss the registration pathway for alisertib in small cell lung cancer (H1 2023); and (v) conducting a meeting with the FDA to discuss the registration pathway for alisertib in hormone receptor positive, HER2-negative breast cancer (H2 2023

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, license revenue and royalty revenue. For the fourth quarter of 2022, total revenue was $65.7 million, of which $53.7 million was product revenue, net, and $12.0 million was royalty revenue. This compares to total revenue of $55.4 million for the fourth quarter of 2021, of which $51.0 million was product revenue, net, $1.5 million was license revenue received from Puma’s sub-licensees, and $2.9 million was royalty revenue. For the year ended December 31, 2022, total revenue was $228.0 million, of which $200.0 million was product revenue, net, and $28.0 million was royalty revenue. This compares to total revenue in 2021 of $253.2 million, of which $189.1 million was product revenue, net, $51.8 million was license revenue received from Puma’s sub-licensees, which included a $50 million upfront payment for providing development, manufacturing and commercial rights to NERLYNX in Greater China to Pierre Fabre, and $12.3 million was royalty revenue

Operating Costs and Expenses

Total operating costs and expenses were $55.7 million for the fourth quarter of 2022, compared to $48.6 million for the fourth quarter of 2021. Total operating costs and expenses were $204.3 million for full year 2022, compared to $251.9 million for full year 2021

Cost of Sales

Cost of sales was $16.8 million for the fourth quarter of 2022, compared to $11.9 million for the fourth quarter of 2021. Cost of sales was $55.1 million for full year 2022, compared to cost of sales of $63.7 million for full year 2021, of which $20.0 million was a termination fee paid to a former sub-licensee for the return of commercial rights to NERLYNX in Greater China

Selling, General and Administrative Expenses

Selling, general and administrative (SG&A) expenses were $25.1 million for the fourth quarter of 2022, compared to $22.5 million for the fourth quarter of 2021. SG&A expenses for full year 2022 were $90.0 million, compared to $116.3 million for full year 2021. The $26.3 million decrease in SG&A expenses for full year 2022 compared to 2021 resulted primarily from a decrease in payroll and related costs of approximately $8.2 million, consisting of $7.6 million from lower headcount and a $2.0 million payroll tax credit under the CARES Act,; and a decrease in stock-based compensation expense of approximately $17.7 million, primarily due to $13.6 million of incremental expense in 2021, which resulted from modification to the term of a warrant held by Mr. Auerbach, offset by an increase of commissions and bonuses of $1.2 million.

Research and Development Expenses

Research and development (R&D) expenses were $13.8 million for the fourth quarter of 2022, compared to $14.2 million for the fourth quarter of 2021. R&D expenses for full year 2022 were $52.2 million, compared to $71.9 million for full year 2021. The $19.7 million decrease in R&D expenses during full year 2022 compared to full year 2021 resulted primarily from a decrease in clinical trial expense of approximately $8.7 million, primarily due to the reduction in the number of patients in certain clinical trials; a decrease in internal R&D expenses of approximately $5.6 million; a decrease in consultant and contractors’ expense of approximately $2.3 million, primarily due to the close of the CONTROL study and a reduction in the number of patients being treated in the SUMMIT study; and a decrease in stock-based compensation expense of approximately $3.2 million, primarily due to the impact of lower headcount combined with the decreased stock price

Acquired In-Process Research and Development Expense

In September 2022, the Company entered into an exclusive license agreement with Takeda Pharmaceutical Company Limited to in-license the worldwide research and development and commercial rights to alisertib. The Company recorded acquired in-process research and development expense related to the up-front payment of $7.0 million during the year ended December 31, 2022.

Total Other Income (Expenses)

Total other expenses were $15.3 million for the fourth quarter of 2022, compared to total other expenses of $2.4 million for the fourth quarter of 2021. Total other expenses were $23.2 million for full year 2022, compared to $30.1 million for full year 2021. The $6.9 million decrease in other expenses in full year 2022 consisted primarily of a net increase in legal verdict expense of $2.9 million, which included a $12.4 million expense related to the legal settlement in the year ended December 31, 2022; $8.1 million in loss on debt extinguishment related to our debt refinancing in July 2021; and a decrease of $1.3 million in lower interest expense related to our outstanding debt.

Full Year 2023 and First Quarter 2023 Financial Outlook

Full Year 2023

First Quarter 2023

Net Product Revenue

$205 – $210 million

$43 -$46 million

Royalty Revenue

$25 – $30 million

$4 – $6 million

Net Income/(Loss)

$20 -$24 million

$(5) – $(2) million

Gross to Net Adjustment

19% – 20%

21% – 21.5%

Conference Call

Puma Biotechnology will host a conference call to report its fourth quarter and full year 2022 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PST/4:30 p.m. EST on Thursday, March 2, 2023. The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international). Please dial in at least 10 minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on Puma’s website for 90 days.

On March 2, 2023 Perrigo Company plc (NYSE: PRGO), a leading provider of Consumer Self-Care Products, reported that President and CEO, Murray S. Kessler and CFO Eduardo Bezerra are scheduled to present at the 12th Annual UBS Global Consumer and Retail Conference in New York City on Wednesday, March 15th at 3:00 PM EST (Press release, Perrigo Company, MAR 2, 2023, View Source [SID1234628071]).

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Interested parties can access the presentation webcast on the Perrigo website at View Source