On May 11, 2023 MEI Pharma, Inc. (Nasdaq: MEIP), a clinical-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for the quarter ended March 31, 2023, and highlighted recent corporate events (Press release, MEI Pharma, MAY 11, 2023, View Source [SID1234631520]).

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"We are making steady progress in advancing both of our clinical-stage pipeline programs: voruciclib for hematologic malignancies and ME-344 for relapsed colorectal cancer, both expected to report data by around the end of the calendar year," said Dan Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "With the clinical-data expected from these two programs around year-end, funds to support operations for at least two years based upon our current development plans, and the opportunity to further strengthen our value proposition via the pending merger with Infinity Pharmaceuticals, we are well positioned to deliver progress in our mission to deliver improved benefits to patients with cancer, as well as value to our shareholders."

Third Quarter Fiscal Year 2023 and Recent Developments

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Kyowa Kirin has been evaluating whether to continue developing zandelisib in Japan and after meeting with the PMDA has concluded this month that conducting a randomized study consistent with agency guidance to support a marketing application would likely not be feasible to complete within a time period that would support further investment. As a result, Kyowa Kirin decided to discontinue development of zandelisib in Japan. The discontinuation of zandelisib in Japan was a business decision by Kyowa Kirin based on the most recent regulatory guidance from the PMDA and is not related to the zandelisib clinical data generated to date.

In light of Kyowa Kirin’s decision to discontinue development of zandelisib in Japan, the parties intend to terminate the global license, development and commercialization agreement executed in April 2020.

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In April 2023, MEI conducted a 1-for-20 reverse stock split. The reverse stock split was approved by MEI’s stockholders on January 5, 2023, and was implemented with the intent to increase the per share trading price of the Company’s common stock to enable the Company to satisfy the minimum bid price requirement for continued listing on Nasdaq. As per a notice received from the Nasdaq dated May 2, 2023, MEI regained compliance with the Nasdaq minimum bid requirement.

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In March 2023, the Safety Review Committee of the Phase 1 study evaluating voruciclib, MEI’s orally administered cyclin-dependent kinase 9 (CDK9) inhibitor, plus venetoclax (Venclexta) completed a safety assessment of the initial dose escalation cohort evaluating the combination in patients with acute myeloid leukemia (AML) and recommended opening the next cohort. The combination stage of the study started after completing the single-agent dose exploration stage of the Phase 1 study in patients with either AML or B-cell malignancies. CDK9 inhibition disrupts Mcl-1 production and upregulation of Mcl-1 is a known escape mechanism of treatment with venetoclax. Thus, the combination being evaluated presents an opportunity to explore the synergistic potential to disrupt the cell cycle and inhibition of pro-survival cell cycle pathways.
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In February 2023, MEI Pharma and Infinity Pharmaceuticals announced a definitive merger agreement for an all-stock transaction pursuant to which Infinity will become a wholly owned subsidiary of MEI Pharma. The combined company would have a projected cash balance of approximately $100 million at Closing that would be expected to fund operations through mid-2025, and to clinical data over the next 12 to 24 months across three clinical-stage oncology development programs: eganelisib, an oral immuno-oncology macrophage reprogramming product candidate, voruciclib, an oral CDK9 inhibitor, and ME-344, a novel tumor selective mitochondrial inhibitor.

Expected Drug Candidate Pipeline Developments

Voruciclib – Oral CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia
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Report clinical data from the ongoing Phase 1b trial evaluating voruciclib plus Venclexta (venetoclax) in patients with acute myeloid leukemia around calendar year-end 2023.

ME-344 – Tumor selective mitochondrial inhibitor

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Initiate a Phase 1b clinical trial evaluating ME-344 plus Avastin in relapsed colorectal cancer patients in the first half of calendar year 2023.
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Report clinical data from the Phase 1b clinical trial evaluating ME-344 plus Avastin in patients with relapsed colorectal cancer around calendar year-end 2023.

Third Quarter Fiscal Year 2023 Financial Results

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As of March 31, 2023, MEI had $112.0 million in cash, cash equivalents, and short-term investments with no outstanding debt.

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For the quarter ended March 31, 2023, cash used in operations was $12.1 million, compared to $17.0 million used in operations during the quarter ended March 31, 2022. The decrease in cash used in operations is due to a reduction in zandelisib costs as we continued the close down of development activities and other changes in working capital.

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Research and development expenses were $15.1 million for the quarter ended March 31, 2023, compared to $22.3 million for the quarter ended March 31, 2022. The decrease was primarily related to a reduction in zandelisib costs as we continued the close down of development activities in December 2022.

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General and administrative expenses were $7.2 million for the quarter ended March 31, 2023, compared to $8.9 million for the quarter ended March 31, 2022. The decrease primarily relates to personnel costs related to the reduction in workforce.

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MEI recognized revenue of $5.9 million for the quarter ended March 31, 2023, compared to $9.7 million for the quarter ended March 31, 2022. The decrease in revenue comes as a result of the discontinuation of the zandelisib program in December 2022 and the associated reduction in expense reimbursement under our global License, Development and Commercialization Agreement with Kyowa Kirin.

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Net loss was $15.4 million, or $2.32 per share, for the quarter ended March 31, 2023, compared to net loss of $8.7 million, or $1.31 per share for the quarter ended March 31, 2022. The Company had 6,662,857 shares of common stock outstanding as of March 31, 2023, compared with 6,657,602 shares as of March 31, 2022.

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The adjusted net income (a non-GAAP measure) for the quarter ended March 31, 2023, excluding non-cash expenses related to changes in the fair value of the warrants, was $15.4 million, compared to an adjusted net loss of $21.5 million for the quarter ended March 31, 2022.

On May 11, 2023 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the first quarter ended March 31, 2023 and will host a conference call today at 4:30 p.m. Eastern Time to discuss these results (Press release, Lineage Cell Therapeutics, MAY 11, 2023, View Source [SID1234631519]).

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"During the first quarter of this year, the Lineage team executed on multiple fronts, advancing our clinical and preclinical cell transplant programs while continuing support of our existing alliances and establishing new ones," stated Brian M. Culley, Lineage CEO. "A key area of focus was our partnership with Roche and Genentech and supporting the ongoing Phase 2a clinical study of OpRegen in patients with GA secondary to AMD, which is enrolling and treating patients at multiple sites in the U.S. Through presentations at various medical and scientific conferences, we also broadened our efforts to enhance awareness of OpRegen’s potential to provide durable anatomical and functional improvements in dry AMD patients. In addition, we established new collaborations with CIRM and the Christopher & Dana Reeve Foundation to support a new SCI conference which aims to identify, discuss, and address gaps in the product development process and elevate the patient’s voice in the treatment process. Looking ahead, we will be working on regulatory interactions for OPC1 while continuing to maintain our commitment to disciplined spending and making responsible investments in disease settings where we believe the use of differentiated cell transplants can provide a meaningful impact for patients."

Recent milestones and activities included:

– RG6501 (OpRegen)

Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including support for the ongoing Phase 2a multi-center clinical study in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD):
Additional sites expected to come online this year.
Long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen:
Preliminary evidence of durable anatomical and functional improvements following administration of OpRegen cells was presented at 2023 Association for Research in Vision and Ophthalmology Annual Meeting and other medical and scientific meetings.
– Received CIRM grant to support 1st Annual Spinal Cord Injury Investor Symposium

California Institute for Regenerative Medicine (CIRM) awarded Lineage an educational grant with a total award value of $50,000, recognizing this event as an important mission-specific conference, which will allow for the exchange of scientific information, create opportunities to accelerate the development of stem cell therapies to patients, increase the likelihood of successful treatments reaching patients, addresses an unmet clinical need, and can be impactful to CIRM’s overall mission.
– Strengthened OPC1 intellectual property portfolio

United States Patent and Trademark Office has granted a patent for the Company’s U.S. patent application No. 16/750,975, now U.S. Patent No. 11,603,518, entitled "Dorsally-Derived Oligodendrocyte Progenitor Cells From Human Pluripotent Stem Cells," with claims covering proprietary manufacturing processes developed by Lineage for its oligodendrocyte progenitor cell therapy candidate (OPC1) for the treatment of spinal cord injury. The patent has a term that would expire no earlier than 2040.
– Executed option and license agreement with Eterna Therapeutics

Enables development of novel beta 2 microglobulin (B2M)-deficient iPSC lines, which Lineage will evaluate for development into differentiated cell transplant therapies, specifically for the treatment of certain central nervous system disorders and other neurology indications.
– Initiated preclinical testing of ANP1

Preclinical testing underway through a collaboration with the University of Michigan and Yehoash Raphael, Ph.D., The R. Jamison and Betty Williams Professor of Otolaryngology, Department of Otolaryngology-Head and Neck Surgery and Lab Director at the University of Michigan Kresge Hearing Research Institute.
Some of the events anticipated by Lineage include:

– Type B Meeting with FDA to discuss a proposed amendment to the Investigational New Drug Application (IND) for OPC1 to enable clinical testing of a novel spinal cord delivery system.

– Amendment of an IND for OPC1 to enable clinical testing of a novel spinal cord delivery system.

– Submission of an additional OPC1 manuscript describing magnetic resonance imaging (MRI) findings from the subacute studies in both thoracic and cervical spinal cord injury.

– Updates from ongoing ANP1 preclinical testing at the University of Michigan Kresge Hearing Research Institute under a collaboration with the University of Michigan.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $46.8 million as of March 31, 2023, which is expected to support planned operations into Q3 2024.

First Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from licensing fees, royalties, collaboration revenues, and research grants. Total revenues for the three months ended March 31, 2023 were approximately $2.4 million, a net decrease of $2.8 million as compared to $5.2 million for the same period in 2022. The decrease was driven by less collaboration and licensing revenue recognized from deferred revenues from the Roche Agreement.

Operating Expenses: Operating expenses are comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended March 31, 2023 were $8.9 million, a decrease of $2.5 million as compared to $11.4 million for the same period in 2022.

R&D Expenses: R&D expenses for the three months ended March 31, 2023 were $4.2 million, a net increase of $1.2 million as compared to $3.0 million for the same period in 2022. The net increase was primarily driven by $0.5 million for nonclinical-related expenses to support the OPC1 program, and $0.2 million in OpRegen program expenses to support the Roche collaboration. Another $0.4 million and $0.2 million of the increase was related to R&D spending on the new auditory neuron and photoreceptor cell therapy programs, respectively.

G&A Expenses: G&A expenses for the three months ended March 31, 2023 were $4.7 million, a net decrease of $3.7 million as compared to approximately $8.4 million for the same period in 2022. The decrease was primarily driven by $3.5 million in lower litigation and legal expenses, mostly due to the Asterias litigation settlement expense accrued in the prior year, and $0.2 million in lower expense for accounting and tax services.

Loss from Operations: Loss from operations for the three months ended March 31, 2023 was $6.6 million, an increase of $0.2 million as compared to $6.4 million for the same period in 2022.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended March 31, 2023 reflected other income, net of $0.4 million, compared to other expense, net of ($0.7) million for the same period in 2022. The net income was primarily driven by fair market value changes in marketable equity securities, interest income from our marketable debt securities, and other income recorded in the current period related to the employee retention credit program, partially offset by exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2023 was $4.4 million, or $0.03 per share (basic and diluted), compared to a net loss attributable to Lineage of $7.1 million, or $0.04 per share (basic and diluted), for the same period in 2022.

Conference Call and Webcast

Interested parties may access today’s conference call and webcast, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 18, 2023, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 8339383.

On May 11, 2023 LianBio (Nasdaq: LIAN), a biotechnology company dedicated to bringing innovative medicines to patients in China and other major Asian markets, reported financial results for the first quarter ended March 31, 2023 (Press release, LianBio, MAY 11, 2023, View Source [SID1234631518]).

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"We continue to achieve multiple significant milestones, propelling the company towards the anticipated commercial launch of mavacamten in China," said Yizhe Wang, Ph.D., Chief Executive Officer of LianBio. "Our team’s demonstrated proficiency in navigating the Chinese regulatory landscape instills confidence in our cross-border model. With the recent progress we’ve made, we believe we are on track to bring our first drug to market in China next year, while we continue to advance our other key programs through the clinic."
Recent Business Highlights and Clinical Development Updates

Mavacamten late-stage clinical development and launch readiness activities on track in China with NDA on file; mavacamten approved in Macau; NDAs under review in Singapore and Hong Kong
•In January 2023, mavacamten was added to The Joint Committee of Cardiomyopathy Specialty Alliance, National Center for Cardiovascular Diseases/Cardiovascular Precision Medicine Branch of China International Exchange and Promotive Association for Medical and Health Care’s 2023 Guidelines for the Diagnosis and Treatment of Patients with Hypertrophic Cardiomyopathy.
•In April 2023, the China NMPA accepted with priority review the NDA for mavacamten for the treatment of adults with symptomatic oHCM.
•In April 2023, LianBio announced topline results from the Phase 3 EXPLORER-CN trial evaluating mavacamten in Chinese patients with oHCM. EXPLORER-CN met the primary endpoint, demonstrating statistically significant and clinically meaningful improvement in Valsalva left ventricular outflow tract gradient from baseline to week 30 compared to placebo, and a safety profile consistent with previous studies.
•In May 2023, mavacamten was approved for the treatment of adults with symptomatic New York Heart Association Class II-III oHCM in the Macau Special Administrative Region.

Commercial infrastructure build continues in preparation for anticipated 2024 mavacamten launch
•In April 2023, Pascal Qian was promoted to Chief Commercial Officer to oversee the continued growth of the company’s commercial capabilities in preparation for the anticipated mavacamten commercial launch in China. He will continue to also serve as the company’s China General Manager.
Business is well-positioned to achieve anticipated milestones
•Current cash runway is projected to extend through the end of 2024.
Key Anticipated Milestones
Mavacamten
•LianBio plans to present detailed results from EXPLORER-CN at an upcoming medical meeting.
TP-03
•LianBio expects to report topline data from the Phase 3 LIBRA trial of TP-03 in Chinese patients with Demodex blepharitis in the fourth quarter of 2023.
Infigratinib
•LianBio expects to report topline data from the ongoing Phase 2a clinical trial of infigratinib in locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification and other advanced solid tumors with FGFR genomic alterations in the second half of 2023.
•LianBio expects to initiate a pivotal Phase 2 trial of infigratinib in locally advanced or metastatic gastric cancer patients with FGFR2 gene amplification in the first half of 2024 to support regulatory approval in China.
BBP-398
•LianBio expects to initiate a Phase 1 clinical trial of BBP-398 in combination with an EGFR-inhibitor in non-small cell lung cancer in the second half of 2023.

First Quarter 2023 Financial Results

Research & Development Expenses

Research and development expenses were $10.8 million for the first quarter of 2023 compared to $12.3 million for the first quarter of 2022. The decrease was primarily attributable to increased milestone payments in 2022, and was offset by higher development activities to support clinical trials in 2023.
General & Administrative Expenses
General and administrative expenses were $15.1 million for the first quarter of 2023 compared to $16.1 million for the first quarter of 2022. The decrease was primarily attributable to decreases in expenses for legal, consulting and accounting services.

Net Loss

Net loss was $24.0 million for the first quarter of 2023 compared to net loss of $27.7 million for the first quarter of 2022.

Cash Balance
Cash, cash equivalents, marketable securities and restricted cash at March 31, 2023 totaled $286.6 million compared to $302.4 million as of December 31, 2022. LianBio projects its current cash, cash equivalents, marketable securities, and restricted cash will be sufficient to fund its current operating plan through the end of 2024.

On May 11, 2023 Kineta, Inc. (Nasdaq: KA), a clinical-stage biotechnology company focused on the development of novel immunotherapies in oncology that address cancer immune resistance, reported financial results for the first quarter of 2023 and provided a corporate update (Press release, Kineta, MAY 11, 2023, View Source;utm_medium=rss&utm_campaign=kineta-reports-first-quarter-2023-financial-results-and-provides-corporate-update [SID1234631517]).

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"The year began with an exciting start as we opened our first-in-human clinical trial of KVA12123 and dosed our first patient in the Phase 1/2 study in advanced solid tumors. Our team is laser focused on working with clinical partners to enroll patients in this study and report initial clinical data by the end of the year," said Shawn Iadonato, Ph.D., Chief Executive Officer of Kineta. "Additionally, in April 2023, we strengthened our balance sheet with a $6 million financing to provide the company with runway through early 2025."

RECENT CORPORATE HIGHLIGHTS

Dosed first patient in Phase 1/2 clinical study evaluating KVA12123 alone and in combination with pembrolizumab in patients with advanced solid tumors
Closed $6 million registered direct offering priced at-the-market under Nasdaq rules
Presented VISTA biomarker data and KVA12123 Phase 1/2 clinical trial update at the AACR (Free AACR Whitepaper) Annual Meeting 2023
Expanded the company’s Scientific Advisory Board (SAB) with the addition of two internationally-renowned clinical immuno-oncology experts:
Myriam Chalabi, M.D.: Medical oncologist and researcher at the Netherlands Cancer Institute
Evan Y. Yu, M.D.: Section Head for Cancer Medicine, Clinical Research Division at the Fred Hutchinson Cancer Center and Medical Director for Clinical Research at the Fred Hutchinson Cancer Consortium
Hosted Key Opinion Leader Event on KVA12123: VISTA as an Immuno-oncology target with Dr. Michael A. Curran, Ph.D. (MD Anderson Cancer Center Department of Immunology) and Dr. Patricia LoRusso, D.O. (Yale School of Medicine Cancer Center)
ANTICIPATED FUTURE MILESTONES

Dose first patient in the combination arm (Part B) of the ongoing KVA12123 Phase 1/2 clinical study

Report initial clinical data of KVA12123 by end of 2023

FIRST QUARTER 2023 FINANCIAL HIGHLIGHTS

Cash position: As of March 31, 2023, cash was $9.2 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 $0.8 million net proceeds received from the sale of 126,503 shares of our common stock in connection with an at-the-market equity offering program. We believe our cash position as of March 31, 2023, together with the $5.5 million net proceeds received in April 2023 plus the committed proceeds of $22.5 million pursuant to the second closing of the private placement expected in July 2023, will be sufficient to fund operating expenses and capital expenditure requirements into early 2025.

Research and development (R&D) expense: R&D expenses were $2.8 million for the three months ended March 31, 2023 compared to $4.0 million for the three months ended March 31, 2022. The decrease in R&D expenses was primarily due to lower activities for KVA12123 as we began securing clinical trial sites and enrolling patients in advance of enrolling the first patient, which occurred in April 2023. We expect our R&D expenses to increase over time this year as we enroll and dose additional patients.

General and administrative expense: General and administrative expenses were $3.9 million for the three months ended March 31, 2023 compared to $1.6 million for the three months ended March 31, 2022. The increase was primarily due to higher personnel-related costs from non-cash stock-based compensation with performance conditions contingent upon the closing of the reverse merger and increased public company expenses such as professional services fees and insurance.

Net loss: The Company reported a net loss of $6.5 million, or $0.77 per basic and diluted share, compared to a net loss of $5.0 million, or $1.07 per basic and diluted share, for the prior year.

On May 11, 2023 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune mediated and oncologic disorders, reported financial results for the first quarter ended March 31, 2023 and provided a business update (Press release, Kezar Life Sciences, MAY 11, 2023, View Source [SID1234631516]).

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"This quarter, Kezar continued to make meaningful progress on its clinical strategy. We remain focused on actively exploring zetomipzomib’s promise in lupus nephritis and autoimmune hepatitis, in addition to advancing our protein secretion inhibitor pipeline, showcasing our ability to discover and develop first in class small molecule therapeutics against novel targets," said John Fowler, Kezar’s Co-Founder and Chief Executive Officer. "To that end, we recently presented the trial design for PALIZADE, our Phase 2b clinical trial of zetomipzomib in lupus nephritis, and provided an encouraging early update from our Phase 1 oncology study of KZR-261, our Sec61 translocon inhibitor. We look forward to continued strong execution in our programs and are excited to share results from our KZR-261 dose escalation trial in the second half of this year."

Zetomipzomib: Selective Immunoproteasome Inhibitor

PALIZADE – Phase 2b clinical trial of zetomipzomib in patients with active lupus nephritis (LN) (ClinicalTrials.gov: NCT05781750)

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PALIZADE is a global, placebo-controlled, randomized, double-blind Phase 2b clinical trial evaluating the efficacy and safety of two dose-levels of zetomipzomib in patients with active LN. Target enrollment will be 279 patients, randomly assigned (1:1:1) to receive 30 mg of zetomipzomib, 60 mg of zetomipzomib or placebo subcutaneously once weekly for 52 weeks, in addition to standard background therapy. Background therapy can, but will not be mandated to, include standard induction therapy. Over the initial 16 weeks, there will be a mandatory corticosteroid taper to 5 mg per day or less. End-of-treatment assessments will occur at Week 53, and the end-of-study assessments will occur at Week 57. The primary efficacy endpoint is the proportion of patients who achieve a complete renal response (CRR) at Week 37, including a urine protein-to-creatine ratio (UPCR) of 0.5 or less without receiving rescue or prohibited medications. PALIZADE is on track to initiate in the first half of 2023.
PORTOLA – Phase 2a clinical trial of zetomipzomib in patients with autoimmune hepatitis (AIH) who have not benefited from standard-of-care treatment (ClinicalTrials.gov: NCT05569759)

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PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to

standard of care or have relapsed. Target enrollment will be 24 patients, randomized (2:1) to receive 60mg of zetomipzomib or placebo in addition to background corticosteroid therapy for 24 weeks, with a protocol-mandated steroid taper by Week 14. The primary efficacy endpoint will measure the proportion of patients who achieve a complete response measured as normalization of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels with a successful corticosteroid taper by Week 24.
MISSION – Completed open-label Phase 2 clinical trial of zetomipzomib in patients with active LN (ClinicalTrials.gov: NCT03393013)

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An abstract featuring post-hoc analysis across LN biopsy classes from the open-label Phase 2 MISSION clinical trial was presented at the National Kidney Foundation (NKF) Spring Clinical Meeting 2023, which took place April 11-15, 2023 in Austin, Texas.
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An abstract featuring complete MISSION Phase 1b/2 results, along with a post hoc subgroup analysis in the Phase 2 Hispanic/Latino population, was presented at the Pan American League of Associations for Rheumatology (PANLAR) 2023 Congress, which took place April 26-29, 2023 in Rio de Janeiro, Brazil.
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An abstract featuring complete MISSION Phase 1b/2 results has been selected for oral presentation at the upcoming LUPUS & KCR 2023 meeting, taking place May 17-20, 2023 in Seoul, Korea.
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Three abstracts featuring complete MISSION Phase 2 results, MISSION Phase 2 uCD163 data, and the unmet need of European patients with LN have been selected for poster presentations at the upcoming European Alliance of Associations for Rheumatology (EULAR) 2023 Congress, taking place May 31 – June 3, 2023 in Milan, Italy.
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Two abstracts featuring post-hoc analysis of MISSION Phase 2 patients with nephrotic range proteinuria and the unmet need of European patients with LN will be presented as focused oral presentations at the 60th European Renal Association (ERA) Congress, taking place June 15-18, 2023 in Milan, Italy.
Protein Secretion Inhibition Platform

KZR 261: Broad-Spectrum Sec61 Translocon Inhibitor

KZR-261-101 – Phase 1 clinical trial of KZR-261 in patients with locally advanced or metastatic solid malignancies (ClinicalTrials.gov: NCT05047536)

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The Phase 1 clinical trial of KZR-261 is being conducted in two parts: dose escalation and dose expansion in four tumor-specific solid tumors and one all-tumor cohort. The study is designed to evaluate safety and tolerability, pharmacokinetics and pharmacodynamics, identify a recommended Phase 2 dose and to explore the preliminary anti-tumor activity of KZR-261 in patients with locally advanced or metastatic disease. Kezar plans to initiate the dose expansion study in the second half of 2023, with topline data expected starting mid-2024.

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The KZR-261 trial is currently enrolling Cohort 6 (27 mg/m2). To date, KZR-261 has shown dose-proportional exposure and no signs of accumulation or altered pharmacokinetics with repeated dosing. There have been no consistent patterns of safety signals. Kezar plans to report safety and dose escalation data from this trial in the second half of 2023.
Financial Results

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Cash, cash equivalents and marketable securities totaled $257.7 million as of March 31, 2023, compared to $276.6 million as of December 31, 2022. The decrease was primarily attributable to cash used to advance clinical-stage programs and preclinical research and development.
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Research and development expenses for the first quarter of 2023 increased by $7.4 million to $18.3 million compared to $10.9 million in the first quarter of 2022. This increase was primarily due to advancing the zetomipzomib clinical program in multiple indications and the KZR-261 clinical program, and an increase in compensation and related personnel costs, including non-cash stock-based compensation expense, attributable to higher headcount to support the progression of the company’s programs and drug discovery.
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General and administrative (G&A) expenses for the first quarter of 2023 increased by $1.3 million to $6.2 million compared to $4.9 million in the first quarter of 2022. The increase was primarily due to an increase in legal and professional services and an increase in compensation and related personnel costs, including non-cash stock-based compensation expense as a result of an increase in headcount and salaries.
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Net loss for the first quarter of 2023 was $22.2 million, or $0.31 per basic and diluted common share, compared to a net loss of $16.0 million, or $0.26 per basic and diluted common share, for the first quarter of 2022.
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Total shares of common stock outstanding were 70.8 million shares as of March 31, 2023. Additionally, there were outstanding pre-funded warrants to purchase 1.6 million shares of common stock at an exercise price of $0.001 per share, 0.5 million outstanding restricted stock units and options to purchase 12.5 million shares of common stock at a weighted-average exercise price of $7.83 per share as of March 31, 2023.