On March 21, 2024 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class and best-in-class therapies to treat cancer reported a business update and provided full year 2023 financial results (Press release, NextCure, MAR 21, 2024, View Source [SID1234641344]).

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"Clinical data generated in 2023 enabled us to objectively assess each program and set our priorities for 2024. Based on that assessment, we will prioritize NC410 combo and LNCB74 in 2024, while seeking to partner our other portfolio assets," said Michael Richman, NextCure’s president and chief executive officer. "These strategic decisions have led to restructuring our workforce reflecting our reduced need for internal GMP manufacturing operations. We believe we have sufficient inventory to support our currently planned clinical trials. I wish to thank those employees who are impacted today for their contributions and dedication to our mission."

Mr. Richman continued, "We are excited to focus on NC410 combo, which is demonstrating clinical responses in ovarian and colorectal cancers. We are advancing LNCB74 in collaboration with LegoChem Biosciences, a differentiated ADC directed to B7-H4, a clinically validated cancer target. Given our current cash position and revised runway to the second half of 2026, we believe we can advance our two programs through important near-term clinical milestones."

Business Highlights and Near-Term Milestones

Prioritized Programs

NC410 (LAIR-2 fusion)

● The multi-center, multi-arm, first-in-human Phase 1b combination trial is evaluating the efficacy of NC410 in combination with pembrolizumab. Indications include: (i) ICI Naïve and refractory ovarian cancer and (ii) microsatellite stable (MSS) / microsatellite instability low (MSI-L) immune checkpoint inhibitor (ICI) Naïve and refractory colorectal cancer (CRC). The combination has been shown to be
well tolerated up to 200 mg of NC410 with Grade 3 or higher Treatment Related Adverse Events of 3.7%.
NC410 for Ovarian Cancer

● Evidence of early clinical activity and biomarker observations support the proposed mechanism of action for NC410 in relapsed/refractory ICI Naïve ovarian cancer, with/without active liver metastasis for subjects in the 100 mg and 200 mg cohorts. As of February 23, 2024, the findings of the initial 7 evaluable patients based on RECIST 1.1 are summarized in the table below:
Relapsed/Refractory ICI Naïve Ovarian, 100 mg and 200 mg cohorts

Evaluable Patients as of February 23, 2024

n=7

Overall Response rate (ORR)

42.8%, n=3

Disease Control Rate (DCR)

42.8%, n=3

Evidence supporting mechanism of action

Observed in biomarker data

● Additional observations from the initial 7-patient data set as of February 23, 2024, include:
o 3 partial responses (PR) were observed at the initial 9-week scan.
o 1 confirmed PR observed in the 200 mg cohort continues on study beyond 6 months.
o The 2 PRs at the 100 mg cohort are pending confirmatory scans at week 18.
● Biomarker data on blood samples drawn from patients in both the prior NC410 monotherapy trial and, the current NC410 combo trial support our hypothesis regarding the mechanism of action (MOA) and activity in PR patients as follows:
o Decrease in peripheral Granzyme B-expressing CD8+ T cells, which supports our mechanism of action (MOA) that NC410 remodels the extracellular matrix (ECM) allowing activated immune cells to infiltrate into the tumor microenvironment (TME). Generation of collagen-derived product 4GZ fragments is mediated by Granzyme B-expressing T cells and provides direct evidence of ECM remodeling and correlates with responses.
o Decrease in peripheral myeloid-derived suppressor cells reduces suppressive effects and enhances activation of immune cells and anti-tumor activity.
o Decrease in peripheral CCR7+ DC+ T cells which is consistent with chemokine guided migration of immune cells to the TME.
● Taken together, the data demonstrate that NC410 mediates activation of immune cells and migration into the TME through remodeling of the ECM. We believe NC410 combo results in anti-tumor activity and clinical responses in patients that have been shown to respond poorly to or are resistant to checkpoint inhibitors.
● In March 2024, we commenced enrolling an additional 18 patients among the 100 mg and 200 mg cohorts. We plan to present the data from the ovarian cancer patients in the second half of 2024.
NC410 Colorectal Cancer (CRC)

● Preliminary evidence of clinical activity in the 100 mg cohort of patients with MSS/ MSI-L ICI naïve CRC without active liver metastasis (LM-). The findings as of February 23, 2024, of the initial 19 evaluable patients are summarized based on RECIST 1.1 guideline in the table below:
MSS/MSI-L ICI Naïve CRC, LM-, 100 mg cohort

Evaluable Patients as of February 23, 2024

n=19

Overall Response rate (ORR)

10.5%, n=2

Disease Control Rate (DCR)

47.3%, n=9

Median Progression Free Survival (mPFS)

8.1 months

● Additional observations from the 19-patient data set as of February 23, 2024:
o Both responses were observed at the initial 9-week scan in the 100 mg cohort.
o Subjects enrolled had a median of 5 lines of prior treatment.
o The 2 responders remain as PRs, and continue on study for over 10 months and 5 months, respectively.
● Completed enrollment in January 2024 of an additional 20 patients in the 100 mg cohort. We plan to present the data of the CRC patients at a scientific conference within the second quarter of 2024.
LNCB74 (B7-H4 ADC)

● Selected our first antibody drug conjugate (ADC) candidate of a potential of three from our collaboration with LegoChem Biosciences, Inc. (LegoChem). Under the terms of the Agreement, both parties equally share the costs of developing the molecules and profits on commercialized products.
● Commenced development of LNCB74 utilizing a NextCure B7-H4 antibody and LegoChem’s ConjuAllTM ADC technology.
● Differentiated approach leveraging:
o B7-H4 specific antibody with an Fc modification that protects immune cells to improve safety
o Use of a glucuronidase cleavable linker that offers cancer-selective payload release to minimize toxicity in non-tumor cells, and
o Use of a monomethyl auristatin E (MMAE) payload with a drug-to-antibody ratio (DAR) of 4, that has the advantage of bystander killing of surrounding tumor cells.
● Pre-clinical experiments in vitro and in vivo demonstrating potent tumor killing and pilot toxicology studies have been completed.
● Pre-filing feedback from the FDA supports moving forward to planned submission of an IND application by this year-end.
● Ongoing activities associated with GLP toxicity studies, GMP manufacturing, and clinical development planning are in progress.

Assets We Intend to Partner

● NC525 is a novel LAIR-1 antibody that selectively targets Acute Myeloid Leukemia (AML) blast cells and leukemic stem cells, and currently is in a Phase 1a monotherapy dose escalation and safety study evaluating NC525 in AML patients. The trial is now in the fifth dose escalation cohort, and we plan to complete the dose-finding portion of the study to arrive at a predicted biologically active dose and further assess development plans by the fourth quarter of 2024.

● NC605 is an antibody that targets Siglec-15 and has the potential as a treatment for bone disease. Preclinical data show that NC605 treatment reduced bone loss and enhanced bone quality in mice with osteogenesis imperfecta (OI). OI is a rare disorder that results in high bone turnover, abnormal bone formation, bone fragility, and recurrent fractures. NC605 could also have applications in chronic bone diseases such as osteoarthritis and non-union fractures. We are currently conducting toxicology studies in preparation for partnering.
● NC181 is a humanized antibody targeting ApoE4 for the treatment of Alzheimer’s disease (AD). In preclinical AD animal models, NC181 has demonstrated amyloid clearance, prevention of amyloid deposition, plaque clearance and neuroinflammation reduction. Preclinical studies have demonstrated that it reduces microhemorrhages, improves cerebral vascular function and lowers risk of Amyloid Related Imaging Abnormalities (ARIA).

Restructuring of Operations

● Implemented a restructuring plan to reduce operating costs and better align our workforce with the needs of our business. Under the plan, we paused our internal manufacturing operations and reduced our workforce by approximately 37%. We estimate that we will incur one-time restructuring charges of approximately $0.8 million including employee severance, benefits and related termination costs, the majority of which we expect to pay in the second quarter of 2024.

Financial Guidance

● NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditures into the second half of 2026.

Financial Results for Full Year Ended December 31, 2023

● Cash, cash equivalents, and marketable securities as of December 31, 2023, were $108.3 million as compared to $159.9 million as of December 31, 2022. The decrease of $51.6 million was primarily due to cash used to fund operations, and cash used to purchase fixed assets.
● Research and development expenses were $47.9 million for the year ended December 31, 2023, as compared to $54.2 million for the year ended December 31, 2022. The decrease of $6.3 million was primarily due to lower costs related to our clinical programs.
● General and administrative expenses were $19.7 million for the year ended December 31, 2023, as compared to $21.7 million for the year ended December 31, 2022. The decrease of $2.0 million was primarily related to lower personnel-related costs, including $1.2 million of stock compensation, and lower insurance and professional costs.
● Net loss was $62.7 million for the year ended December 31, 2023, as compared with a net loss of $74.7 million for the year ended December 31, 2022. Lower research and development expenses, lower general and administrative expenses and higher other income contributed to the lower net loss.

On March 21, 2024 Milestone Pharmaceuticals Inc. (Nasdaq: MIST) reported financial results for the fourth quarter and year ended December 31, 2023 and provided a regulatory and corporate update (Press release, Milestone Pharmaceuticals, MAR 21, 2024, View Source [SID1234641343]).

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"We look forward to resubmitting our NDA imminently. With the completion of our recent financing and potential future synthetic royalty payment, we believe we are well positioned to advance etripamil through potential approval and launch in PSVT," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We’re excited to continue this positive momentum as we execute on our programs and advance etripamil."

Corporate Updates

• In March 2024, completed a public offering of common shares and pre-funded warrants, raising net proceeds of approximately $32.4 million. Milestone intends to use the proceeds from the Offering to continue the development of etripamil in its lead indication of paroxysmal supraventricular tachycardia (PSVT) and its subsequent indication of atrial fibrillation with a rapid ventricular rate (AFib-RVR), as well as for working capital and other general corporate purposes.

Recent Program Updates

Etripamil for Patients with PSVT

•
Announced Plans to Resubmit New Drug Application (NDA) for Etripamil for PSVT in early 2Q 2024. Milestone held a Type A meeting with the FDA in February 2024 regarding steps required to resolve the items raised in the Refusal to File (RTF) letter received in December 2023. The Company is working to restructure the data sets that capture timing of reported adverse events (AEs) in the clinical etripamil studies and is reformatting certain data files to facilitate FDA’s analyses. The Company expects a standard NDA review period following resubmission of the NDA.

Etripamil for Patients with AFib-RVR

• Phase 3 guidance received from FDA in 1Q2024 meeting. FDA reiterated prior guidance regarding the availability of a single-study supplemental New Drug Application (sNDA) pathway contingent on obtaining approval for the NDA in PSVT. FDA further concurred with respect to key study elements including powering, inclusion criteria, patient population, and statistical analyses, and offered clarification with respect to the endpoints to guide the design of the Phase 3 study. We anticipate progressing to an End of Phase 2 meeting to finalize the registrational study protocol in mid-2024.

• Positive Results from ReVeRA Phase 2 Study of Etripamil in AFib-RVR Presented as Featured Science at the American Heart Association (AHA) Scientific Sessions 2023 and Simultaneously Published in Circulation: Arrhythmia and Electrophysiology. In November 2023, Milestone announced positive data from the ReVeRA Phase 2 study that show that patients with AFib-RVR receiving etripamil demonstrated rapid and statistically superior ventricular rate reduction and improved symptom-relief when compared to placebo. Safety and tolerability reported in the 56-patient safety population who received etripamil was generally consistent with that observed in the Company’s extensive safety database from the PSVT program. The results were presented as a Featured Science presentation at the American Heart Association (AHA) Scientific Sessions 2023 and simultaneously published in Circulation: Arrhythmia and Electrophysiology, which can be found here.

Fourth Quarter and Full Year 2023 Financial Results

• As of December 31, 2023, Milestone had cash, cash equivalents, and short-term investments of $66.0 million, compared to $64.6 million as of December 31, 2022.

• There was no revenue recorded for the fourth quarter of 2023, compared with $3.5 million the fourth quarter of 2022. Revenue for the full year ended December 31, 2023 was $1.0 million compared to $5.0 million in the year ended December 31, 2022. Revenue in 2023 was related to a milestone payment received from Ji Xing Pharmaceuticals, under the Company’s License and Collaboration Agreement. Revenue in 2022 was related to two milestone payments received under the agreement with Ji Xing Pharmaceuticals.

• Research and development expense for the fourth quarter of 2023 was $5.5 million, compared with $10.6 million for the prior year period. For the full year ended December 31, 2023, research and development expense was $31.1 million, compared with $39.8 million for the prior year. The decrease year over year was primarily due to lower clinical expenses as a result of the completion of Phase 3 studies, partially offset by an increase in drug manufacturing consulting costs, drug manufacturing personnel costs and regulatory consulting costs.

• General and administrative expense for the fourth quarter of 2023 was $3.4 million, compared with $4.1 million for the prior year period. For the full year ended December 31, 2023, general and administrative expense was $15.9 million, compared with $15.7 million for the prior year.

• Commercial expense for the fourth quarter of 2023 was $5.0 million, compared with $2.6 million for the prior year period. For the full year ended December 31, 2023, commercial expense was $15.1 million, compared with $9.1 million for the prior year. The increase in commercial expense year over year was a result of additional personnel and professional costs required to expand capabilities and operations in anticipation of potential commercialization.

• For the fourth quarter of 2023, net loss was $13.6 million, compared to $13.2 million for the prior year period. For the full year ended December 31, 2023, Milestone’s net loss was $59.7 million, compared to $58.4 million for the prior year.

For further details on the Company’s financials, refer to Form 10-K for the year ended December 31, 2023, filed with the SEC.

About Paroxysmal Supraventricular Tachycardia

An estimated two million people in the United States are currently diagnosed with PSVT which is a type of arrhythmia or abnormal heart rhythm. PSVT is characterized by episodes of sudden onset rapid heartbeats often exceeding 150 to 200 beats per minute. The heart rate spike is unpredictable and may last several hours. The rapid heart rate often causes disabling severe palpitations, shortness of breath, chest discomfort, dizziness or lightheadedness, and distress, forcing patients to limit their daily activities. The uncertainty of when an episode of PSVT will strike or how long it will persist can provoke anxiety in patients and negatively impact their day-to-day life between episodes. The impact and morbidity from an attack can be especially detrimental in patients with underlying cardiovascular or medical conditions, such as heart failure, obstructive coronary disease, or dehydration. Many health care providers are dissatisfied with the lack of effective treatment options with patients often requiring prolonged, burdensome, and costly trips to the emergency department or even invasive cardiac ablation procedures.

About Atrial Fibrillation with Rapid Ventricular Rate

An estimated five million Americans suffer from AFib, a common arrhythmia marked by an irregular, disruptive and often rapid heartbeat. The incidence of AFib is expected to grow to approximately 10 million by 2025 and up to about 12 million by 2030. A subset of patients with AFib experience episodes of abnormally high heart rate most often accompanied by palpitations, shortness of breath, dizziness, and weakness. While these episodes, known as AFib-RVR, may be treated by oral calcium channel blockers and/or beta blockers, patients frequently seek acute care in the emergency department to address symptoms. In 2016, nearly 800,000 patients were admitted to the emergency department due to AFib symptoms where treatment includes medically supervised intravenous administration of calcium channel blockers or beta blockers, or electrical cardioversion. With little available data for AFib-RVR, Milestone’s initial market research indicates that 30 to 40% of patients with AFib experience one or more symptomatic episodes of RVR per year that require treatment, suggesting a target addressable market of approximately three to four million patients in 2030 for etripamil in patients with AFib-RVR.

About Etripamil

Etripamil is Milestone’s lead investigational product. It is a novel calcium channel blocker nasal spray under clinical development for frequent and often highly symptomatic episodes of PSVT and AFib-RVR. It is designed as a self-administered rapid response therapy for patients thereby bypassing the need for immediate medical oversight. If approved, etripamil is intended to provide health care providers with a new treatment option to enable on-demand care and patient self-management. This portable, self-administered treatment may provide patients with active management and a greater sense of control over their condition. CARDAMYST, the conditionally approved brand name for etripamil nasal spray, is well studied with a robust clinical trial program that includes a completed Phase 3 clinical-stage program for the treatment of PSVT and Phase 2 trial for the treatment of patients with AFib-RVR.

On March 21, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that the Phase 3 KEYLYNK-006 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with maintenance LYNPARZA, a PARP inhibitor, did not meet its dual primary endpoints of overall survival (OS) and progression-free survival (PFS) for the first-line treatment of certain patients with metastatic nonsquamous non-small cell lung cancer (NSCLC) (Press release, Merck & Co, MAR 21, 2024, View Source [SID1234641342]).

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In the KEYLYNK-006 trial, KEYTRUDA in combination with chemotherapy followed by KEYTRUDA plus maintenance LYNPARZA did not meet the study’s pre-specified statistical criteria for OS or PFS compared to KEYTRUDA in combination with chemotherapy (pemetrexed plus carboplatin or cisplatin) followed by KEYTRUDA plus maintenance chemotherapy (pemetrexed). The safety profiles of KEYTRUDA and LYNPARZA in this trial were consistent with those observed in previously reported studies for the individual therapies. A full evaluation of the data from this study is ongoing. Merck will work with investigators to share the results with the scientific community.

"As lung cancer continues to be the leading cause of cancer death worldwide, we are committed to exploring KEYTRUDA-based combinations and novel candidates that may further help improve patient outcomes," said Dr. Gregory Lubiniecki, vice president, global clinical development, Merck Research Laboratories. "These results are an important reminder of how challenging it may be to treat these patients with metastatic nonsquamous non-small cell lung cancer. We sincerely thank the patients and investigators for their important contributions to this study."

About KEYLYNK-006

KEYLYNK-006 is a randomized, open-label, two-phase Phase 3 trial (ClinicalTrials.gov, NCT03976323) evaluating KEYTRUDA in combination with chemotherapy (pemetrexed plus carboplatin or cisplatin) followed by either KEYTRUDA plus maintenance LYNPARZA or KEYTRUDA plus maintenance chemotherapy (pemetrexed) for the first-line treatment of patients with metastatic nonsquamous NSCLC with no epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK) or proto-oncogene tyrosine-protein kinase (ROS1) genomic tumor aberrations. The dual primary endpoints are PFS per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) by blinded independent central review (BICR) and OS. The secondary endpoints include safety and health-related quality of life assessments. The trial enrolled an estimated 1,005 patients in the Induction Phase and randomized 672 patients with a complete or partial response or with stable disease. In the Induction Phase, patients received KEYTRUDA (200 mg intravenously [IV]) in combination with pemetrexed plus investigator’s choice of platinum chemotherapy, consisting of either carboplatin Area Under the Curve (AUC) (5 mg/mL/min IV) or cisplatin (75 mg/m2 IV) every three weeks (Q3W) for four cycles. In the Maintenance Phase, patients with a complete or partial response or with stable disease after completing four cycles of induction therapy were randomized to receive either KEYTRUDA (200 mg IV Q3W for up to 31 cycles) plus maintenance LYNPARZA (300 mg orally twice daily) or KEYTRUDA (200 mg IV Q3W for up to 31 cycles) plus maintenance pemetrexed (500 mg/m2 IV Q3W) until progressive disease, physician decision or intolerable toxicity.

About lung cancer

Lung cancer is the leading cause of cancer death worldwide. In 2022 alone, there were approximately 2.4 million new cases and 1.8 million deaths from lung cancer globally. Non-small cell lung cancer is the most common type of lung cancer, accounting for about 80% of all cases. In 2024, the overall five-year survival rate for patients diagnosed with lung cancer is 25% in the United States. Improved survival rates are due, in part, to earlier detection and screening, reduction in smoking, advances in diagnostic and surgical procedures, as well as the introduction of new therapies. Early detection and screening remain an important unmet need, as 44% of lung cancer cases are not found until they are advanced.

On March 21, 2024 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, reported recent business progress and fourth-quarter and full-year 2023 financial results (Press release, Kronos Bio, MAR 21, 2024, View Source [SID1234641341]).

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"In 2023, we made great progress across our portfolio and business. We demonstrated that KB-0742 has a safety profile that is clearly differentiated from all other CDK9 inhibitors, and we believe our extended dosing schedule will show increased efficacy signals while maintaining a favorable safety profile," said Nobert Bischofberger, Ph.D., president and chief executive officer of Kronos Bio. "We also announced our second development candidate, KB-9558, which is designed to target the KAT domain of p300 and downregulate IRF4 transcription in multiple myeloma. We are looking forward to completing the IND-enabling studies in 2024 and expect to dose our first patients in the first half of 2025."

Clinical Program and Recent Company Updates

KB-0742
•At the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC International Conference, the Company reported target engagement, tumor regressions, and an acceptable safety profile for KB-0742 dosed at 60mg three-days-on, four-days-off in heavily pre-treated patients with transcriptionally addicted solid tumors. Out of fourteen "all-comer" patients, two patients with myxoid liposarcoma exhibited tumor regressions: one (7th line) had a partial response (per RECIST v1.1) lasting 113 days and the second achieved a 26% reduction in tumor diameters with stable disease. KB-0742 also cleared the 80mg three-days-on, four-days-off schedule in dose escalation and is currently enrolling patients in the KB-0742 60mg four-days-on, three-days-off dose escalation cohort of the Phase 1/2 trial.
•At a medical conference in mid-2024, the Company intends to share an update on the clinical data to date from patients with transcriptionally addicted tumors who were dosed with 60mg three-days-on, four-days-off, and from "all-comer" patients in the dose-escalation cohort who received 80mg three-days-on, four-days-off.
•In the third quarter of 2024, the Company expects to clear the 80mg four-days-on, three-days-off dose escalation cohort, and begin enrolling patients in an expansion cohort including one or more of the following: non-small cell lung cancer, small cell lung cancer, ovarian cancer, and triple negative breast cancer.

Exhibit 99.1
•The Company expects to announce topline data from the expansion cohort at the 80mg four-days-on, three-days-off dose in the first half of 2025.

KB-9558
•In December 2023, the Company announced the nomination of KB-9558, a p300 KAT inhibitor, as a development candidate.
•KB-9558 is the second development candidate to emerge from Kronos Bio’s product engine, where the Company identified that inhibition of the lysine acetyltransferase (KAT) domain on p300 inhibits expression of interferon regulatory factor 4 (IRF4) and thus collapses the transcription regulatory network that is required for the survival of multiple myeloma cells.
•The Company plans to present data on KB-9558 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, being held from April 5-10, 2024 in San Diego, California, including how targeting p300’s enzymatic KAT domain can selectively downregulate IRF4.
•Pending completion of IND-enabling studies in 2024, the Company intends to initiate a first-in-human trial in relapsed or refractory multiple myeloma in the first half of 2025.

Lanraplenib
•In December 2023, after a review of data from the Phase 1b portion of its Phase 1b/2 trial of lanraplenib in combination with gilteritinib in FLT3-mutated relapsed/refractory acute myeloid leukemia (AML), the Company decided not to proceed to Phase 2.

Company Update
•Kronos Bio extended its expected cash runway by a year, into the second half of 2026, through restructurings and resource optimization.

Fourth-Quarter and Full-Year 2023 Financial Highlights

▪Cash, Cash Equivalents and Investments: With its ongoing and currently planned clinical programs and $175.0 million in cash, cash equivalents and investments as of December 31, 2023, the Company anticipates sufficient resources to fund its planned operations into the second half of 2026.

▪R&D Expenses: Research and development expenses were $18.7 million for the fourth quarter of 2023, which includes non-cash stock-based compensation expense of $2.5 million. For the full year of 2023, research and development expenses were $86.4 million, which includes non-cash stock-based compensation expense of $12.0 million.

▪G&A Expenses: General and administrative expenses were $10.9 million for the fourth quarter of 2023, which includes non-cash stock-based compensation expense of $2.7 million. For the full year of 2023, general and administrative expenses were $41.7 million, which includes non-cash stock-based compensation expense of $13.0 million.

▪Net Loss: Net loss for the fourth quarter of 2023 was $25.3 million, or $0.43 per share, including non-cash stock-based compensation expense of $5.2 million. Net loss for the full-year 2023 was $112.7 million, or $1.95 per share, including non-cash stock-based compensation expense of $25.0 million.

On March 21, 2024 Kineta, Inc. (Nasdaq: KA), a clinical-stage biotechnology company with a mission to develop next-generation immunotherapies that transform patients’ lives, reported financial results for the full year ended December 31, 2023 and provided a corporate update (Press release, Kineta, MAR 21, 2024, View Source;utm_medium=rss&utm_campaign=kineta-reports-full-year-2023-financial-results-and-provides-corporate-update [SID1234641340]).

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In February 2024, the Company announced a significant corporate restructuring to substantially reduce expenses and preserve cash. The restructuring included a significant workforce reduction and the suspension of enrollment of new patients in its ongoing VISTA-101 Phase 1/2 clinical trial evaluating KVA12123 in patients with advanced solid tumors. Patients currently enrolled in the trial will be permitted to continue to participate. The Company announced the restructuring as a result of certain investors indicating that they will not fulfill their April 2024 funding obligation in the previously disclosed private placement financing. In connection with the restructuring, the Company announced that it is exploring strategic alternatives to maximize stockholder value.

"2023 was a productive year for Kineta where we initiated the Phase 1/2 VISTA-101 trial of KVA12123 as monotherapy and in combination with pembrolizumab in cancer patients, and saw promising results, underscoring the potential of KVA12123 as a next-generation immunotherapy. We truly appreciate the efforts of the healthcare professionals, the patients and their caregivers, and the Kineta employees involved in this trial. We look forward to sharing the clinical update in the second quarter and we will continue to explore strategic alternatives that will allow us to continue to pursue our mission of developing transformative immunotherapies for patients with cancer," said Craig Philips, President of Kineta.

RECENT CORPORATE HIGHLIGHTS

Phase 1/2 VISTA-101 Trial of KVA12123 in Patients with Solid Cancer Tumors

Efficacy

Announced positive KVA12123 monotherapy safety data from its ongoing Phase 1/2 VISTA-101 clinical trial in patients with advanced solid tumors.
Monotherapy Dose Escalation (3-300 mg KVA12123 Q2W)
Of 21 patients enrolled, 12 received at least one baseline and one follow up scan.
Best overall response (BOR) in nine of 12 patients is currently stable disease among patients with at least one follow-up scan with a mean duration of 15 weeks.
One patient with non-small cell lung cancer that failed six prior lines of therapy, including checkpoint inhibitor (CPI) therapy, has experienced a stable disease lasting 28 weeks.
Nine participants remain on-treatment.
Combination Therapy Dose Escalation (30-100 mg KVA12123 Q2W, 400 mg pembrolizumab Q6W).
Of nine patients enrolled, three received at least one baseline and one follow-up scan.
BOR in 2 of 3 patients with at least one follow up scan is:
Stable disease in one CPI-failure renal cell carcinoma patient with a 24% reduction in target lesions.
Partial response in one patient with a PD-L1 negative mucoepidermoid carcinoma and a 54% reduction in target lesions and a complete response in non-target lesions.
Eight patients remain on treatment.
Biomarkers

Dose-dependent induction of on-target pro-inflammatory cytokines and chemokines.
Dose-dependent increases in non-classical monocytes, CD4+ and CD8+ T cells, and NK cells.
Safety

No dose limiting toxicities (DLTs) observed in any patient at any dose level.
No evidence of cytokine release syndrome in any patient at any dose level.
Conference Presentations

Co-organized the 3rd Annual VISTA Symposium, with Hummingbird Bioscience and Dartmouth Giesel School of Medicine, to be held virtually on March 27, 2024.
Presented clinical and preclinical data on VISTA blocking KVA12123 at the Keystone Symposia of Cancer Immunotherapy: Beyond Immune Checkpoint Blockade and Overcoming Resistance.
Presented new preclinical data on KVA12123 in acute myeloid leukemia (AML) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Blood Cancer Discovery Symposium.
ANTICIPATED FUTURE MILESTONES

Additional KVA12123 monotherapy safety and efficacy data in Q2 2024.
Initial KVA12123 and pembrolizumab combination therapy data in Q2 2024.
EXPLORATION OF STRATEGIC ALTERNATIVES

In February 2024, the Company announced that it is pursuing strategic alternatives to maximize shareholder value due to certain investors indicating they will not fulfill their April 2024 funding obligation in the previously disclosed private placement financing. As a result, the Company has suspended new patient enrollment into the Phase 1/2 VISTA-101 trial and will not be recruiting patients into either the sixth cohort in the monotherapy arm or the third cohort in the combination therapy arm. Patients currently enrolled in the trial will be permitted to continue to participate. In connection with the restructuring, the Company implemented a workforce reduction of the Company’s workforce of approximately 64% of the Company’s then-current employee base.
2023 FINANCIAL HIGHLIGHTS

Cash position: As of December 31, 2023, cash was $5.8 million, compared to $13.1 million as of December 31, 2022. The decrease was primarily due to cash used for clinical trial development of KVA12123 as well as general corporate purposes, partially offset by $8.6 million net proceeds received from institutional and individual investors and $5.0 million in cash received from the Merck milestone payment in July 2023. As of December 31, 2023, we had $5.8 million in cash, and there is substantial doubt about our ability to continue as a going concern. Based on our current operating plans, we do not have sufficient cash and cash equivalents to fund our operating expenses and capital expenditures for at least the next 12 months from the filing date of our Annual Report on Form 10-K for the fiscal year ended December 31, 2023, which we expect to file on March 21, 2024.
Revenues: Total revenues were $5.4 million for the year ended December 31, 2023 and $2.0 million for the year ended December 31, 2022. Revenues in 2023 were primarily due to our achievement of a development milestone under the Merck Exclusive License and Research Collaboration Agreement, which triggered a $5.0 million milestone payment. Revenues in 2022 were due to research and development services from the Genentech Option and License Agreement, which was terminated in December 2022, and also due to services provided under a grant that was concluded in December 2022.
Research and development (R&D) expense: R&D expenses were $9.0 million for the year ended December 31, 2023 and $15.9 million for the year ended December 31, 2022. The decreases in R&D expenses were primarily due to lower activities for KVA12123 manufacturing and clinical study start up as the Company began enrolling the first patient in the study, which occurred in April 2023.
General and administrative expense: General and administrative expenses were $12.1 million for the year ended December 31, 2023 and $8.7 million for the year ended December 31, 2022. The increase was primarily due to increases in personnel costs of $2.3 million and insurance and other company expenses of $1.1 million. Personnel costs increased due to higher salaries and benefits of $1.2 million from increased headcount to support public company responsibilities and higher stock-based compensation of $1.0 million, which increased due to options granted during 2023. Insurance and other company expenses increased primarily due to public company directors and officers insurance premiums of $601,000 and board fees of $270,000.
Net loss: Net loss was $14.1 million, or $1.28 per basic and diluted share, for the year ended December 31, 2023 compared to a net loss of $63.4 million, or $12.87 per basic and diluted share, for the year ended December 31, 2022.