Lineage Cell Therapeutics Reports Second Quarter 2022 Financial Results and Provides Business Update

On August 11, 2022 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 second quarter of 2022 (Press release, Lineage Cell Therapeutics, AUG 11, 2022, View Source [SID1234618128]). Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its second quarter 2022 financial and operating results and to provide a business update.

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"We are pleased with the progress on RG6501 (OpRegen) product development under our collaboration with Roche and Genentech. During the second quarter this year, we have made progress across multiple functional areas, including clinical, regulatory, and technology transfer activities," stated Brian M. Culley, Lineage CEO. "As we continue to position Lineage as a leader in regenerative medicine through the transplant of specific cell types, our focus is on completing the necessary clinical, regulatory and related activities which can create value and reduce risk across our portfolio of five cell transplant assets. In particular, our efforts have been focused on preparing for OPC1 and VAC2 regulatory interactions to enable their next phases of clinical testing in spinal cord injury and oncology, respectively. In parallel, we are advancing our auditory neuron and photoreceptor programs through preclinical development activities which are necessary to support initial clinical testing. We believe our disciplined use of capital and our increased strategic business development activities can support multiple years of growth and the achievement of important milestones in the months and years to come."

Second quarter milestones and activities included:

– RG6501 (OpRegen)

Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including:
Conducting additional OpRegen manufacturing runs and supporting Chemistry Manufacturing and Controls (CMC) activities.
Continuing technology transfer activities.
Actively participating in both Joint Advisory and Joint Manufacturing Committees, forums for discussion and planning with respect to next steps in clinical development and related activities.
Continuing long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen:
Enrolled patients have continued to do well, supporting multi-year durability of a treatment effect with RG6501 (OpRegen).
– OPC1

Results from a Phase 1/2a clinical study in subacute cervical spinal cord injury were published in the Journal of Neurosurgery: Spine;
OPC1 has demonstrated an excellent safety profile, and at one-year post-treatment, 96% of patients had recovered one or more levels of neurological function on at least one side of their body, and 32% of patients had recovered two or more levels of neurological function on at least one side of their body.
Preclinical testing of a new thaw and inject formulation of OPC1, manufactured via an improved and larger-scale process, has demonstrated functional recovery, improvement in gait coordination and motor performance with a reduction of the area of cavitation.
A majority of the verification and validation activities for the novel parenchymal spinal delivery (PSD) system, and its preclinical testing in support of a regulatory submission have been completed.
Preclinical activities to support upcoming regulatory interactions are near completion.
Engagement with the California Institute of Regenerative Medicine (CIRM), as well as various patient advocacy organizations and patient advocates, is underway.
– VAC2

Following technology transfer of the program from Cancer Research UK (CRUK) to Lineage and improvement of manufacturing processes, production scale was increased and accordingly the cost of goods has been reduced significantly, along with marked improvements in the purity and functionality of the manufactured material.
CRUK continues to follow patients on the Phase 1 NSCLC clinical study; Lineage has received all necessary clinical information from CRUK required to support U.S., or other, regulatory interactions.
– ANP1 & PNC1

Preclinical activities are continuing, including thought leader engagement to support future preclinical testing.
– Business Development

Broadened collaboration with Advanced BioMatrix, a division of BICO Group AB (STO: BICO), for the HyStem delivery technology to include clinical/commercial GMP (Good Manufacturing Practice) material, increasing the milestone payments and royalty percentages due to Lineage upon ABM reaching certain development milestones and/or product sales.
Continued work under our collaboration with our strategic partner, Immunomic Therapeutics ("ITI"); currently awaiting decision on next steps for ITI’s allogeneic cell-based cancer immunotherapy for the treatment of glioblastoma based on the VAC platform.
Some of the key upcoming milestones and activities anticipated by Lineage include:

– Planned interaction with FDA in Q4 2022 to discuss an OPC1 IND amendment submission to enable clinical performance and safety testing of a novel PSD system.

– A pre-IND regulatory interaction in Q4 2022 to seek feedback on a VAC2 CMC, nonclinical, and clinical package to support U.S. clinical development; pre-IND briefing package submission in Q3 2022.

– Submission of a grant application to the California Institute for Regenerative Medicine (CIRM) for the continued support of the clinical development of OPC1.

– Clinical data update from the ongoing VAC2 Phase 1 non-small cell lung cancer (NSCLC) study, pending release from CRUK.

– Preclinical activities for both the ANP1 and PNC1 programs.

– Additional OPC1 publications, including preclinical study results utilizing a new thaw and inject formulation of OPC1, manufactured via an improved and larger-scale process.

– An additional OPC1 manuscript from a Phase 1/2a clinical study in subacute cervical spinal cord injury, focused on MRI data.

– Evaluation of new partnership opportunities and/or expansion of existing collaborations.

– Continued participation in investor and partnering meetings and medical and industry conferences to broaden awareness of our mission and accomplishments.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $72 million as of June 30, 2022, which is expected to support operations through Q2 2024.

Second 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 June 30, 2022 were approximately $4.6 million, a net increase of $4.0 million as compared to $0.5 million for the same period in 2021. The increase was primarily related to licensing fees recognized from deferred revenues in connection with the $50.0 million upfront licensing payment received in the first quarter of 2022 from Roche.

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 June 30, 2022 were $8.6 million, an increase of $1.1 million as compared to $7.5 million for the same period in 2021.

R&D Expenses: R&D expenses for the three months ended June 30, 2022 were $3.3 million, a net increase of $0.4 million as compared to $2.9 million for the same period in 2021. The net increase was driven by $0.1 million in higher OpRegen related expenses to support the Roche Collaboration. Another $0.2 million and $0.1 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 June 30, 2022 were $5.3 million, a net increase of approximately $0.7 million as compared to $4.5 million for the same period in 2021. The increase was primarily attributable to $0.4 million in payroll and related benefits expense, and $0.5 million in share-based compensation, partially offset by $0.2 million in lower investor relations expense.

Loss from Operations: Loss from operations for the three months ended June 30, 2022 was $4.2 million, a decrease of $2.9 million as compared to $7.1 million for the same period in 2021.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended June 30, 2022 reflected other expense, net of ($2.5) million, compared to other income, net of $2.1 million for the same period in 2021. The net change was primarily driven by exchange rate fluctuations related to Lineage’s international subsidiaries, as well as a decrease in the value of marketable equity securities, and partially offset by the gain on extinguishment of debt from Lineage’s Paycheck Protection Program loan forgiveness recognized in the prior year’s quarter.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended June 30, 2022 was $6.8 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $4.8 million, or $0.03 per share (basic and diluted), for the same period in 2021.

Conference Call and Webcast

Interested parties may access today’s conference call by dialing (800) 715-9871 from the U.S. and Canada and (646) 307-1952 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call" or provide conference ID number 6448886. 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 August 18, 2022, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 6448886.

Alpine Immune Sciences Provides Corporate Update and Reports Second Quarter 2022 Financial Results

On August 11, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported financial results for the second quarter ended June 30, 2022 (Press release, Alpine Immune Sciences, AUG 11, 2022, View Source [SID1234618127]).

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"We are pleased with our productive second quarter that included the presentation of encouraging clinical data across our pipeline of novel autoimmune and immuno-oncology candidates and the continued strengthening of our company with the additions of Dr. Sandler to our management team and Dr. Drapa to our board of directors," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "Promising first-in-human data for ALPN-303, our potentially best-in-class dual BAFF/APRIL inhibitor, were presented at the 2022 European Alliance of Associations for Rheumatology (EULAR) Congress. We look forward to sharing a more detailed clinical update later in the year to be followed by multiple patient-based studies. Our lead immuno-oncology candidate, davoceticept, has demonstrated a deepening reduction in tumor volume as a monotherapy and focused expansion cohorts have been initiated. In addition, we are pleased to have had the partial clinical hold removed on the NEON-2 trial of davoceticept in combination with pembrolizumab and we plan to report initial data from this combination later this year."

Second Quarter 2022 and Recent Pipeline Updates

ALPN-303

Preliminary data from the first-in-human, phase 1 study of ALPN-303 in healthy volunteers were included in a poster presentation at the 2022 European Alliance of Associations for Rheumatology (EULAR) Congress. ALPN-303 has significantly improved potency against the B cell cytokines BAFF and APRIL, superior to related inhibitors including wild-type TACI-Fc’s. In addition, the ongoing phase 1 study demonstrates that ALPN-303 has been well tolerated to-date and has exhibited highly encouraging preliminary pharmacodynamic reductions in circulating immunoglobulins.
Initiation of up to three basket trials in hematologic, renal, and dermatologic indications and a phase 2 study in systemic lupus erythematosus (SLE) is anticipated beginning in the first half of 2023, updated from end of 2022 due, in part, to delays in enrollment in our phase 1 study related to the COVID-19 pandemic in Australia earlier this year. We plan to provide a more detailed clinical update from the phase 1 study in the third quarter of 2022 and anticipate initial clinical data from the basket trials in the second half of 2023.
Davoceticept

Davoceticept monotherapy dose escalation data (NEON-1) were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, demonstrating tumor volume reduction in 23% of evaluable participants despite a highly heterogeneous, heavily pretreated, advanced solid tumor population. Monotherapy expansion cohorts are currently open for enrollment with a particular interest in renal cell carcinoma, given the observed preliminary activity in this setting.
FDA removed the partial clinical hold on the NEON-2 trial of davoceticept in combination with pembrolizumab after review of the Company’s Complete Response, which included a comprehensive review of the davoceticept safety database, as well as a revised investigator brochure and study protocol. Preliminary data from the NEON-2 combination trial is anticipated in the second half of 2022.
Corporate

Andrew Sandler, MD was appointed Chief Medical Officer of Alpine effective August 17, 2022. Dr. Sandler brings over 25 years of industry experience and clinical development expertise and leadership to the Alpine team. Dr. Sandler joins Alpine from Kiadis Pharma, a Sanofi Company, where he was Chief Medical Officer.
Jörn Drappa, MD, PhD, a 20-year industry veteran with extensive global clinical development leadership experience in autoimmune and inflammatory diseases, joined the Alpine Board of Directors. Dr. Drappa co-founded Viela Bio, a biotechnology company focused on the discovery, development and commercialization of treatments for autoimmune and severe inflammatory diseases, and served as its Head of R&D and Chief Medical Officer through its acquisition by Horizon Therapeutics in March 2021 for $3.1 billion.
Second Quarter 2022 Financial Results

As of June 30, 2022, Alpine’s cash, cash equivalents, and investments totaled $201.2 million. The Company recorded net losses of $18.1 million and $11.0 million for the quarters ended June 30, 2022 and 2021, respectively.

Collaboration revenue for the second quarter ended June 30, 2022 was $5.3 million compared to $7.2 million for the second quarter ended June 30, 2021. The 2022 amounts were primarily attributable to revenue recognized under the Company’s AbbVie and Horizon Agreements, while 2021 revenue recognized solely relates to the AbbVie collaboration.

Research and development expenses for the second quarter ended June 30, 2022 were $17.6 million compared to $14.6 million for the second quarter ended June 30, 2021. The increase was primarily attributable to the Company’s Synergy, NEON, and ALPN-303 studies, and increased personnel costs and direct research activities. These increases were partially offset by a decrease in contract manufacturing and process development for ALPN-303.

General and administrative expenses for the second quarter ended June 30, 2022 were $4.2 million compared to $3.3 million for the second quarter ended June 30, 2021. The increase was primarily attributable to increases in personnel costs.

The Company expects that its current cash resources will be sufficient to fund its planned operations into 2024.

Vincerx Pharma Reports Second Quarter 2022 Financial Results and Provides a Corporate Update

On August 11, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Vincerx Pharma, AUG 11, 2022, View Source [SID1234618126]).

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"We shared key updates last quarter that are intended to strategically position Vincerx to aggressively advance our pipeline programs while optimizing our cash runway," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "Our unique INN, enitociclib, for our lead asset, VIP152, was recently issued. The wide application and global recognition of INNs suggest our highly selective PTEFb/CDK9 inhibitor is one step closer to becoming a potential therapy for cancer patients. We continue to execute on priority indications in our enitociclib clinical program and remain on track for multiple IND-related catalysts, including IND submission for VIP236 later this year and two additional filings for VIP943 and VIP924 in the second half of 2023 and first half of 2024, respectively. The Vincerx team remains focused on our prioritized studies and look forward to providing additional progress updates in the coming months."

RECENT CORPORATE HIGHLIGHTS

VIP152 recently issued INN, enitociclib
Prioritized enitociclib clinical studies to focus on:
Monotherapy in patients with high grade B-cell lymphoma characterized by translocations of MYC and BCL-2
Monotherapy in patients with high-risk chronic lymphocytic leukemia (CLL)
Combination with Bruton tyrosine kinase (BTK) inhibitor in patients with high-risk CLL
Prioritized enitociclib indications expected to initiate Phase 2 studies in 2023
Continuing to prioritize advancement of first-in-class and potentially best-in-class bioconjugation assets
IND submission for SMDC VIP236 on track for 2H2022
IND filing for ADCs, VIP943 and VIP924, on track for 2H2023 and 1H2024, respectively
Streamlined and realigned resources to support prioritized enitociclib indications, advancement of the bioconjugation programs, and extension of cash runway through late 2024
Presented poster at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, titled "VIP152 is a novel CDK9 inhibitor with improved selectivity, target modulation, and cardiac safety in patients with lymphoma"
SECOND QUARTER 2022 FINANCIAL RESULTS

Vincerx Pharma had $80.9 million in cash as of June 30, 2022, as compared to $111.5 million as of December 31, 2021. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements into late 2024.
Research and development (R&D) expenses for the three- and six-months ended June 30, 2022 were $13.7 million and 29.7 million, respectively, as compared to $10.7 million and $15.5 million for the same periods in 2021, respectively. These increases were primarily driven by manufacturing services in support of our ADC programs and third-party services in support of our ongoing enitociclib clinical trials.
General and administrative (G&A) expenses for the three- and six-months ended June 30, 2022 were $4.7 million and $10.4 million, respectively, as compared to $6.7 million and $11.5 million for the same periods in 2021, respectively. These decreases were primarily the result of declines in stock-based compensation expense.
For the second quarter ended June 30, 2022, Vincerx reported a net loss of $18.4 million, or $0.88 per share, as compared to a net loss of $2.0 million, or $0.12 per share for the same period in 2021. For the six-months ended June 30, 2022, Vincerx reported a net loss of $34.8 million, or $1.66 per share, as compared to a net loss of $8.3 million, or $0.55 per share for the same period in 2021. The net loss for the second quarter and six-months ended June 30, 2021 include gains from the change in fair value of our warrant liabilities of $15.4 million and $18.7 million, respectively.

CymaBay Reports Second Quarter and Six Months Ended June 30, 2022 Financial Results and Provides Corporate Update

On August 11, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the second quarter ended June 30, 2022 (Press release, CymaBay Therapeutics, AUG 11, 2022, View Source [SID1234618125]).

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Sujal Shah, President and CEO of CymaBay, stated, "We are elated to have finished enrollment in RESPONSE, our second, global Phase 3 study of seladelpar for patients with primary biliary cholangitis (PBC). We enrolled 193 patients across more than 20 countries and would like to thank our patients, their families and all of the clinical-site personnel involved for helping us reach this critical milestone. We anticipate releasing topline data for RESPONSE in the third quarter of 2023. Together with the more than 150 patients that are already enrolled in ASSURE, our long-term extension study, we believe our development program for seladelpar is one of the most robust to be conducted in PBC. We were also pleased to present data at EASL this past June, including two-year treatment data with seladelpar showing it improved the GLOBE PBC score and predicted improved transplant-free survival. Finally, we continue to focus on careful management of expenses which has resulted in a strong balance sheet with $170.8 million of cash, cash equivalents and investments as of June 30, 2022."

Recent Corporate Highlights

Completed enrollment in RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study enrolled 193 patients who have an inadequate response or intolerance to ursodeoxycholic acid in a 2:1 ratio to receive once daily oral seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline as assessed by a validated numerical rating scale recorded with an electronic diary.
Continued strong enrollment in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety and efficacy data to support registration. There are now over 150 patients in this study taking daily seladelpar. After completing the RESPONSE study, patients will be able to roll over into ASSURE.
Supported continued enrollment in a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes. The study is being conducted by the AdventHealth Translational Research Institute in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982.

Featured results of our studies at The International Liver Congress 2022 of the European Association for the Study of Liver (EASL) held in London, UK., including a presentation by Bettina Hansen, PhD, Associate Professor Toronto Centre for Liver Disease, University of Toronto, reporting the improvement in GLOBE score following seladelpar treatment over two years and predicted transplant-free survival. The GLOBE score is a validated risk-assessment tool providing an estimate of transplant-free survival for patients with PBC. The presentation reported:

Treatment of 50 patients with oral seladelpar 5 or 10 mg daily for 2 years resulted in a mean (SD) change from baseline in GLOBE score of -0.417 (0.269), resulting in a corresponding hazard ratio of 0.66 for transplantation or death compared to no prior treatment with seladelpar (baseline).
The improvement in GLOBE score and predicted survival did not depend on age. However, an analysis of subpopulations of high-risk patients by GLOBE score (> 0.3) revealed that while patients of all ages improved, the younger patients tended to have numerically greater improvements, suggesting a potential benefit of earlier treatment.
Planning and execution progressed in our manufacturing and supply chain functions to support NDA submission and post-approval launch. Pre-commercial market analysis and planning became an increasing focus to support our planned product launch.

Held $170.8 million in cash, cash equivalents and investments as of June 30, 2022. We believe that cash and investments on hand are sufficient to fund CymaBay’s operating plan through 2023.
Second Quarter and Six Months Ended June 30, 2022, Financial Results

Research and development expenses for the three months ended June 30, 2022, and 2021 were $17.9 million and $16.7 million, respectively. Research and development expenses for the six months ended June 30, 2022, and 2021 were $36.3 million and $29.1 million, respectively. Research and development expenses in the three- and six-month period ended June 30, 2022, were higher than the corresponding periods in 2021 primarily due to an increase in clinical trial activities associated with the ongoing late-stage development of seladelpar in PBC. In particular, cost increases were primarily driven by an expansion of our site activation, patient enrollment, and other clinical trial activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC.

General and administrative expenses for the three months ended June 30, 2022, and 2021 were $5.9 million and $6.5 million, respectively. General and administrative expenses for the six months ended June 30, 2022, and 2021 were $12.0 million and $11.8 million, respectively. General and administrative expenses in the three months ended June 30, 2022, were lower than in the three months ended June 30, 2021, due to a reduction in legal and consulting expenses. General and administrative expenses were higher in the six months ended June 30, 2022, when compared to the six months ended June 30, 2021, due to compensation associated with the hiring of additional personnel, partially offset by a reduction in legal and consulting expenses.

Net loss for the three months ended June 30, 2022 and 2021 was $27.1 million and $23.2 million, or ($0.31) and ($0.34) per share, respectively. Net loss for the six months ended June 30, 2022, and 2021 was $54.9 million and $40.8 million, or ($0.62) and ($0.59) per share, respectively. Net loss was higher due to an increase in clinical operating expenses, as clinical activity related to our late-stage development of seladelpar in PBC continued to expand, as well as an increase in interest expense accretion related to the Abingworth development financing arrangement. We expect our operating expenses to increase in the future as we continue to execute on our development plans for seladelpar in PBC.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss second quarter financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13730902. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

Kezar Life Sciences Reports Second Quarter 2022 Financial Results and Provides Business Update

On August 11, 2022 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 second quarter ended June 30, 2022 and provided a business update (Press release, Kezar Life Sciences, AUG 11, 2022, View Source [SID1234618124]).

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"The second quarter was tremendously productive for Kezar, during which we achieved key clinical milestones, strengthened our balance sheet, and added great talent to our leadership team, marking a major step in our growth," said John Fowler, Kezar’s Co-founder and Chief Executive Officer. "We shared exciting positive topline results from our MISSION Phase 2 study evaluating zetomipzomib in LN patients, and our Phase 1 trial in solid tumors with our novel protein secretion inhibitor, KZR-261 is progressing as planned. We look forward to continued momentum across the company for the rest of 2022 and providing further updates in the coming months."

Zetomipzomib: Selective Immunoproteasome Inhibitor

MISSION – Phase 1b/2 clinical trial of zetomipzomib (KZR-616) in patients with systemic lupus erythematosus with and without active lupus nephritis (LN) (NCT03393013)

In June 2022, Kezar reported topline results from the open-label MISSION Phase 2 clinical trial evaluating zetomipzomib in patients with active LN.
During the 24-week treatment period, patients received 60 mg of zetomipzomib subcutaneously once weekly (first dose of 30 mg) in addition to stable background therapy. End-of-treatment assessments occurred at Week 25, with completion of study at Week 37. Patients in the MISSION Phase 2 clinical trial received zetomipzomib without induction therapy, which represents a difference from other recently published trials in LN. The primary efficacy endpoint for the trial was the proportion of patients achieving an overall renal response (ORR), measured as a 50% or greater reduction in urine protein to creatinine ratio (UPCR) at end of treatment. A key secondary efficacy endpoint was the number of patients with a complete renal response (CRR), measured as an absolute reduction in proteinuria values to a UPCR of 0.5 or less, with preserved renal function (eGFR), and corticosteroid use of 10 mg or less prednisone/prednisone equivalent and no use of prohibited medication.
In the Phase 2 topline analysis, 17 of 21 patients enrolled in the trial reached end of treatment:
11 of 17 patients (64.7%) achieved an ORR measured as a 50% or greater reduction in UPCR at end of treatment compared to baseline, the primary efficacy endpoint of the clinical trial.
6 of 17 patients (35.2%) achieved a CRR of 0.5 UPCR or less, with all other protocol definitions satisfied.
Treatment benefit of zetomipzomib was maintained or deepened following the end of treatment, based on assessments at Week 29.
16 of 17 patients (94.1%) reached an ORR at Week 29, and 6 patients maintained a CRR.
Patients’ mean daily prednisone background dosage was reduced from 19.2 mg at baseline to 9.1 mg at week 25 and was further reduced at Week 29.
Also in June, Kezar presented a poster featuring zetomipzomib as part of the EULAR Science Exhibit session at the Annual European Congress of Rheumatology (EULAR) in Copenhagen, Denmark.
POS0715: Treatment of SLE Patients with Zetomipzomib (KZR-616), a Selective Inhibitor of the Immunoproteasome, Results in Circulating Gene Expression, Protein Level, and Immune Cell Phenotypic Changes with Potential Correlations to Clinical Response, presented by Andrea Fan, Ph.D., Vice President, Head of Biology and Translational Research, Kezar Life Sciences.
KZR-261: Protein Secretion Inhibitor

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

KZR‑261 is a novel, broad-spectrum agent that acts through direct interaction and inhibition of the Sec61 translocon. In preclinical studies, KZR-261 has been shown to induce a direct anti-tumor effect as well as modulate the tumor microenvironment, including enhancing anti-tumor immune responses.
The Phase 1 clinical trial of KZR-261 is being conducted in two parts: dose escalation and dose expansion in subjects with selected tumor types. The trial is designed to evaluate safety and tolerability, pharmacokinetics and pharmacodynamics, as well as to explore the preliminary anti-tumor activity of KZR-261 in patients with locally advanced or metastatic disease.
At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting, held in April 2022 in New Orleans, LA, Kezar presented data on its proprietary small molecule inhibitors of the Sec61 translocon, specifically KZR-834, a working analog of KZR-261.
Appointment of Chief Business Officer

In July 2022, Nick Mordwinkin, Pharm.D., Ph.D., was appointed as Chief Business Officer. Dr. Mordwinkin brings over a decade of experience in leadership, corporate development and strategic partnership roles in the healthcare industry. Dr. Mordwinkin will be responsible for shaping and overseeing the Company’s business development strategy.
Financial Results

Cash, cash equivalents and marketable securities totaled $306.8 million as of June 30, 2022, compared to $208.4 million as of December 31, 2021. The increase was primarily attributable to net proceeds from the issuance of common stock under the "at-the-market" Sales Agreement with Cowen and Company, LLC, net of cash used by the company in operations to advance its clinical-stage programs and preclinical research and development.
Research and development expenses for the second quarter of 2022 increased by $2.0 million to $11.3 million compared to $9.3 million in the second quarter of 2021. This increase was primarily related to advancing the zetomipzomib clinical programs and the KZR-261 Phase 1 clinical trial.
General and administrative expenses for the second quarter of 2022 increased by $1.3 million to $5.0 million compared to $3.7 million in the second quarter of 2021. The increase was primarily due to an increase in personnel expenses, including non-cash stock-based compensation and an increase in professional services.
Net loss for the second quarter of 2022 was $16.2 million, or $0.25 per basic and diluted common share, compared to a net loss of $13.0 million, or $0.25 per basic and diluted common share, for the second quarter of 2021.
Total shares of common stock outstanding were 68.3 million shares as of June 30, 2022. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 9.2 million shares of common stock at a weighted-average exercise price of $8.05 per share as of June 30, 2022.
About Zetomipzomib (KZR-616)

Zetomipzomib (KZR-616) is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1 clinical trials provide evidence that zetomipzomib exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases.

About Lupus Nephritis

Lupus nephritis (LN) is one of the most serious complications of systemic lupus erythematosus (SLE). LN is a disease comprising a spectrum of vascular, glomerular and tubulointerstitial lesions and develops in approximately 50% of SLE patients within 10 years of their initial diagnosis. LN is associated with considerable morbidity, including an increased risk of end-stage renal disease requiring dialysis or renal transplantation and an increased risk of death. There are limited approved therapies for the treatment of LN. Management typically consists of induction therapy to achieve remission and long-term maintenance therapy to prevent relapse.

About KZR-261 and the Inhibition of Protein Secretion

KZR-261 is a first-in-class small molecule compound, derived from Kezar’s research and discovery platform of protein secretion pathway inhibitors. This broad-spectrum anti-tumor agent directly targets the Sec61 translocon and inhibits multiple cancer drivers both within tumor cells and the tumor microenvironment. A Phase 1 clinical trial is underway for the treatment of solid tumor malignancies.

Kezar’s drug discovery platform of protein secretion pathway inhibitors is a novel approach with broad application. The protein secretion pathway is a highly conserved and ubiquitously functioning pathway in all cells in the body and involves a conserved protein complex called the Sec61 translocon, the target of Kezar’s compounds. In preclinical models, Kezar’s library of protein secretion inhibitors have demonstrated broad activity with far-reaching potential in oncology, immune-oncology, and autoimmunity.