On May 12, 2022 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company focused on the discovery and development of innovative gamma-delta T cell therapies utilizing its DeltEx platform, reported financial results and operational highlights for the quarter ended March 31, 2022 (Press release, In8bio, MAY 12, 2022, View Source [SID1234614483]). In addition, the Company provided an overview of recent corporate developments.

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"Our team continues to execute and advance our programs on multiple fronts despite these challenging times in biotech," said William Ho, CEO and co-founder of IN8bio. "Our Phase I clinical trials for both DeltEx gamma-delta product candidates, INB-200 in GBM and INB-100 in leukemia patients, continue to progress with encouraging early data. We added key clinical and regulatory expertise to our senior management team and we will be introducing our iPSC derived gamma-delta T cell platform at ASGCT (Free ASGCT Whitepaper) next week. The IN8bio team will continue to be disciplined with our capital and we will use our resources to continue to accelerate the growth and momentum in our Company."

Business Highlights & Updates

In January 2022, IN8bio provided a clinical update from the Phase 1 clinical trial of its genetically modified gamma-delta T cell therapy candidate, INB-200, in newly diagnosed GBM. In the single ascending dose cohort 1 (n=3), all three
patients showed no dose limiting toxicities (DLTs), no cytokine release syndrome (CRS) or neurotoxicity and demonstrated a manageable safety profile. Cohort 2, which will receive three repeat doses of INB-200, includes one patient who has already received all three doses without any DLTs or significant cell therapy related adverse events. All of the patients treated have exceeded their expected progression-free survival (PFS) interval based on age and MGMT status and exceeded median expected PFS of up to seven months with encouraging trends in overall survival.

IN8bio presented a clinical update from the ongoing Phase 1 clinical trial of INB-100, an allogeneic gamma-delta T cell therapeutic candidate in leukemia patients undergoing hematopoietic stem cell transplantation. All three patients treated remain in morphologic CR with durable remissions ranging from nine to 23 months through March 2022. We observed robust immune reconstitution in the patients including T cells, B cells and gamma-delta T cells. INB-100’s safety profile continues to be manageable with no DLTs, no grade 3 or greater graft vs. host disease (GvHD), no CRS and no neurotoxicity. The data were presented at the EBMT 48th Annual Meeting. These are encouraging results given up to 51% are expected to relapse within a year with standard of care treatment.

In April 2022, IN8bio announced the continued expansion of its clinical and regulatory teams with the appointments of Urvashi Patel, Ph.D., Vice President, Regulatory Affairs, and Stacey Bilinski, Vice President, Clinical Operations. They bring deep clinical and regulatory experience to the IN8bio team, from early to late-stage development across the biotechnology and pharmaceutical industry with more than 45 years of combined experience. Dr. Patel has provided regulatory guidance to biopharmaceutical companies, including WindMIL Therapeutics, Precision for Medicine, Janssen, Elan Pharmaceuticals and Genentech. Ms. Bilinski has initiated numerous first in-human and early-phase clinical development programs at biopharmaceutical companies, including Taiho Oncology, Onconova, Insmed, Amicus Therapeutics and Covance.

Upcoming Milestones and Events

In May 2022, IN8bio unveiled a new preclinical program focused on developing iPSC derived gamma-delta T cells to be presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting, which will be held from May 16-19, 2022. Scalable iPSC-based cellular manufacturing is designed to create for a uniform and renewable therapeutic product available "off-the-shelf" for patients. IN8bio will be holding an investor event via webcast at the conference to introduce the program to the investment community on May 17, 2022, at 6:00 p.m. EDT. A link to the webcast will be available on the Events & Presentations page of the Company’s website.

IN8bio plans to submit an investigational new drug (IND) application for a Phase 1b/2 clinical trial of INB-400 in GBM during the second half of 2022.

Expected Upcoming Presentations

ASGCT 25th Annual Meeting, Washington, D.C., May 2022: introducing a new program on "The Development of Off-the-Shelf" Manufacturing Strategies for iPSC-Based Gamma-Delta T Cells" along with an oral presentation by Dr. Lamb, "Off the Shelf Cell Therapies – Beyond T Cells (Education Session) and The Next Generation of γδ T Cell-based Therapies" by Dr. Lamb.

Advanced Therapies Congress Live, London, May 2022: presenting "New Directions in the Treatment of Solid Tumors with gdT Cells" by Dr. Lamb.

Cambridge Healthcare Immuno-Oncology Therapeutic Development,
London, May 2022: presenting "Genetically Modified gamma delta T cells in
combination with chemotherapy: now in the clinic" by Dr. Lamb

Cell Engager summit, Boston, May 2022: presenting "Gamma-Delta T-cells: Novel approaches to genetic engineering and cell-type specific therapeutic applications" by Dr. Rochlin.

H.C. Wainwright Global Investment Conference, May 2022: Mr. Ho will participate in a fireside chat on May 25, 2022, at 2 p.m. EDT.

American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper), June 2022: presenting "Phase 1 study of drug-resistant immunotherapy (DRI) with gene-modified autologous γδ T cells in patients with newly diagnosed glioblastoma multiforme (GBM) receiving maintenance temozolomide (TMZ)" by Louis B. Nabors, M.D.

Jefferies Healthcare Conference, June 2022: Mr. Ho will present at 1:30 p.m. EDT on Wednesday, June 8, 2022.

First Quarter 2022 Financial Highlights

Cash position: As of March 31, 2022, the Company had cash of $32.1 million, compared to $37.0 million as of December 31, 2021. The decrease in cash was primarily due to cash used by the Company in research and development and continued operations to advance its programs.

Research and Development (R&D) expenses: R&D expenses were $2.4 million for the three months ended March 31, 2022, compared to $1.2 million for the comparable prior year period. The increase in R&D expenses were primarily due to (i) increased third-party clinical trial and IND-related activities, (ii) contract manufacturing costs for the ongoing INB-200 clinical trial and (iii) increased personnel-related costs, including salaries, benefits and stock-based compensation due to increased headcount.

General and administrative expenses: General and administrative expenses were $3.8 million for the three months ended March 31, 2022, compared to $1.1 million for the comparable prior year period. The increase was primarily
due to increased legal expenses, insurance costs and expenses associated with operating as a public company.

Net loss: The Company reported a net loss attributable to common stockholders of $6.1 million, or $0.33 per basic and diluted common share, for the three months ended March 31, 2022, compared to a net loss attributable to common stockholders of $3.1 million, or $0.82 per basic and diluted common share, for the comparable prior year period.

On May 12, 2022 Targovax ASA (OSE: TRVX), a clinical stage biotechnology company developing immune activators to target hard-to-treat solid tumors, reported its first quarter 2022 results (Press release, Targovax, MAY 12, 2022, View Source [SID1234614265]). Members of Targovax’s executive management team will give an online presentation to investors, analysts and the press at 10:00 CET today (details below).

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1Q 2022 HIGHLIGHTS
Business Development
Announced two key collaboration agreements with Agenus:

Free drug supply of two checkpoint inhibitors for ONCOS-102 combination therapy in the upcoming phase 2 melanoma trial
Inclusion of the adjuvant QS-21 STIMULONTM as an immune-stimulatory component of the TG mutant KRAS cancer vaccine
R&D
Announced that Oslo University Hospital will sponsor a study to test the TG01 cancer vaccine in RAS mutant multiple myeloma patients
Announced a research collaboration with Prof Michael Uhlin at Karolinska Institutet in Stockholm for development and characterization of NextGen circular RNA ONCOS viruses
Organization
Appointed circular RNA co-discoverer and pioneer Dr Thomas B Hansen as VP of Research to lead the circular RNA pipeline program
Strengthened the management team with the appointment of industry veteran Dr Lubor Gaal as Chief Financial Officer
Appointed Dr Raphael Clynes and Mr Thomas Falck as new members of the Board of Directors

On May 12, 2022 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions utilizing the latest biotechnology, reported that two abstracts highlighting data and information for zandelisib, an investigational phosphatidylinositol 3-kinase delta ("PI3Kδ") inhibitor in clinical development for the treatment of B-cell malignancies, will be presented at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress to be held June 9 – 17, 2022 (Press release, Kyowa Hakko Kirin, MAY 12, 2022, View Source [SID1234614315]).

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Oral Presentation Title: Efficacy and Safety of Zandelisib Administered by Intermittent Dosing (ID) in Patients with Relapsed or Refractory (r/r) Follicular Lymphoma: Primary Analysis of the Global Phase 2 Study TIDAL
Session Title: Indolent & mantle cell non-Hodgkin lymphoma – Clinical
Presenter: Andrew David Zelenetz, PhD, MD
Time: June 11, 11:30am-12:45pm CEST
Abstract Code: S208

Poster Title: Zandelisib on Intermittent Dosing as a Single Agent or in Combination with Rituximab or Zanubrutinib in Relapsed or Refractory (r/r) Follicular Lymphoma (FL): Results from a Multi-Arm Phase 1b Study
Session Title: Indolent & mantle cell non-Hodgkin lymphoma – Clinical
Time: June 10, 2:30pm-3:45pm CEST
Presenter: Jacob Drobnyk Soumerai, MD
Abstract Code: P1114

The abstracts are available on the EHA (Free EHA Whitepaper) Annual Congress website. All presentations will be made available on the EHA (Free EHA Whitepaper) website for on-demand viewing on June 10, 2022.

On May 12, 2022 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that financial results for the three-month period ended March 31, 2022 and provided a corporate update (Press release, Spectrum Pharmaceuticals, MAY 12, 2022, View Source [SID1234614332]).

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"We anticipate FDA approvals later this year for poziotinib and eflapegrastim. In the first quarter, we initiated a confirmatory study and presented additional positive scientific data for poziotinib. The resubmitted BLA for eflapegrastim was also accepted for review by the FDA," said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals. "We are proud of the progress we’ve made toward our core business objectives and we remain dedicated to making a meaningful difference in the lives of cancer patients."

Pipeline Updates

Eflapegrastim, a novel long-acting G-CSF

The U.S. Food and Drug Administration (FDA) has accepted Spectrum’s resubmitted Biologics License Application (BLA) for eflapegrastim with a Prescription Drug User Fee Act (PDUFA) date of September 9, 2022. The company is working with its partner, Hanmi Pharmaceutical, to support the FDA regulatory review process.
Poziotinib, a Pan ErbB inhibitor targeting HER2 exon20 mutations

The New Drug Application (NDA) for poziotinib is under active review at the FDA with Fast Track designation. The NDA is based on the positive results of Cohort 2 in patients with previously treated locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations. The agency has set a PDUFA date of November 24, 2022. There is currently no FDA approved therapy for patients with NSCLC harboring HER2 exon 20 insertion mutations.
A study for poziotinib has been initiated to confirm the clinical benefit seen in Cohort 2, as required for an accelerated approval. The trial, Study SPI-POZ-301 (PINNACLE), is designed to enroll 268 patients with previously treated NSCLC harboring HER2 exon 20 mutations. Patients are being randomized 2-to-1 into one of two treatment arms using 8mg of poziotinib orally administered BID (twice daily) versus 75mg/m2 of docetaxel administered intravenously every three weeks. The primary endpoint will be Progression Free Survival.
FDA’s Oncologic Drugs Advisory Committee (ODAC) is scheduled to review poziotinib for the treatment of patients with previously treated locally advanced or metastatic NSCLC harboring HER2 exon 20 insertion mutations. The fall ODAC meeting is being held September 22-23, 2022. ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes appropriate recommendations to the FDA. As usual, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.
Data from Cohort 4 of the ZENITH20 study in patients with treatment-naïve NSCLC harboring HER2 exon 20 insertion mutations were presented in an oral session at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress 2022. The results showed a confirmed objective response rate (ORR) of 41%, as evaluated centrally by an independent image review committee using RECIST 1.1 criteria. The evaluable patient population showed an ORR of 50%. The study met its primary endpoint as the observed lower bound of 30% exceeded the pre-specified lower bound of 20%. The safety profile was consistent with the tyrosine kinase inhibitor (TKI) class. Notably, on-target adverse events (AEs) were meaningfully reduced with BID dosing.
The company presented a poster on the predictive ability of circulating tumor DNA (ctDNA) in poziotinib-treated patients with NSCLC harboring HER2 exon 20 insertion mutations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Preliminary results suggest that decreases in plasma ctDNA during poziotinib therapy correlate with clinical response in patients with advanced NSCLC with HER2 exon 20 insertion mutations. Additional data from this study will be presented at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting in early June.
Corporate Updates

Nora E. Brennan was named Chief Financial Officer effective May 25, 2022. Ms. Brennan has served on Spectrum’s Board of Directors since December 2020 and as Chair of the Audit Committee. She will relinquish her board duties to assume her new senior leadership role. Most recently, Ms. Brennan served as Chief Financial Officer of Fore Biotherapeutics, a developer of cancer therapies driven by functional genomics. Prior to Fore, she served as Chief Financial Officer at TELA Bio, Inc. and as Senior Vice President of Treasury and Investor Relations at Integra Life Sciences Holdings Corporation.
Hanmi Pharmaceutical completed a $20 million strategic equity investment in Spectrum in January 2022, which included revisions to the licensing and supply agreements for eflapegrastim and poziotinib.
Two new members of the Board of Directors have been named. In March, Juhyun Lim was appointed to the Board. Ms. Lim currently serves as President, Global Strategy and Planning at Hanmi Science and Hanmi Pharmaceutical, where she leads the execution of corporate strategy and investment. In May, Spectrum named Brittany Bradrick to the Board and she will succeed Ms. Brennan as Chair of the Audit Committee. Ms. Bradrick currently serves as Chief Financial Officer of Neurelis, Inc. Ms. Bradrick is a seasoned executive with 25 years of experience in the life sciences sector including in the areas of mergers and acquisitions, investment banking, finance, strategy and corporate development.
A strategic restructuring with a ~30% staff reduction and ~20-25% reduction in operating cash burn was initiated in January 2022 to focus the company’s development activities on its late-stage assets, poziotinib and eflapegrastim. Development activities for the early-stage pipeline has been deprioritized.
Three-Month Period Ended March 31, 2022 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $15.4 million, or a $0.09 loss per basic and diluted share, in the three-month period ended March 31, 2022, compared to a net loss of $35.7 million, or a $0.25 loss per basic and diluted share, in the comparable period in 2021. Total research and development expenses were $4.2 million in the quarter, as compared to $19.4 million in the same period in 2021. Selling, general and administrative expenses were $9.9 million in the quarter, compared to $14.3 million in the same period in 2021.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $9.6 million, or a $0.06 non-GAAP loss per basic and diluted share, in the three-month period ended March 31, 2022, compared to a non-GAAP net loss of $29.4 million, or a $0.20 non-GAAP loss per basic and diluted share, in the comparable period in 2021. Non-GAAP research and development expenses were $2.1 million, as compared to $18.0 million in the same period of 2021. Non-GAAP selling, general and administrative expenses were $7.5 million, as compared to $11.5 million in the same period in 2021.

Cash Position and Guidance

In January, the company received a $20 million strategic equity investment from Hanmi Pharmaceutical. Together with this strategic investment, Spectrum ended the quarter with cash, cash equivalents, and marketable securities of approximately $89.2 million. The additional cash, combined with the restructuring, is expected to extend the company’s cash runway into 2023.

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: View Source on May 12, 2022 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On May 12, 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 first quarter ended March 31, 2022 and provided a business update (Press release, Kezar Life Sciences, MAY 12, 2022, View Source [SID1234614349]).

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"I want to thank my colleagues at Kezar for their excellent execution across both of our novel programs so far this year," said John Fowler, Kezar’s Co-founder and CEO. "We are excited to present the topline results from our Phase 2 MISSION trial in lupus nephritis this June and remain committed to exploring zetomipzomib’s potential in inflammatory diseases with high unmet need. Our strengthened balance sheet is key in supporting both of our programs, including our Phase 1 trial of KZR-261 in solid tumors."

Zetomipzomib: Selective Immunoproteasome Inhibitor

MISSION – Phase 2 clinical trial of zetomipzomib (KZR-616) in patients with lupus nephritis (LN) (NCT03393013)

In November 2021, Kezar reported interim data from the MISSION Phase 2 open-label trial in patients with active, proliferative LN. The interim data showed that, of the five patients who completed the full 24 weeks of weekly treatment with zetomipzomib 60mg doses, two achieved partial renal responses (PRRs) and two achieved complete renal responses (CRRs). The primary efficacy endpoint for the trial is the number of patients achieving a renal response measured by a 50% or greater reduction in UPCR at the end of treatment compared to baseline.
Kezar reiterates prior guidance and expects to report Phase 2 topline data in June 2022
An abstract featuring zetomipzomib has been selected for poster presentation as part of the EULAR Science Exhibit session at the upcoming Annual European Congress of Rheumatology (EULAR), taking place June 1-4, 2022, in Copenhagen, Denmark. The virtual presentation will be available beginning Wednesday, June 1, 2022, at 2:00 am Eastern Time through July 31, 2022.
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, PhD, Vice President, Head of Biology and Translational Research, Kezar Life Sciences
PRESIDIO – Phase 2 clinical trial of zetomipzomib (KZR-616) in patients with active dermatomyositis (DM) or polymyositis (PM) (NCT04033926)

On May 3, 2022, Kezar reported topline data from the PRESIDIO Phase 2 clinical trial of zetomipzomib in patients with DM (n=13) and PM (n=12). 20 of the 25 patients enrolled in the study completed end-of-treatment (Week 32). Topline results from PRESIDIO showed that most DM and PM patients saw clinically meaningful improvements in total improvement score (TIS), but zetomipzomib demonstrated no significant differentiation from placebo. The overall safety and tolerability of zetomipzomib was favorable and consistent with previous results.
KZR-616-003E (NCT04628936) is an open-label extension (OLE) study available to patients who completed 32 weeks in the PRESIDIO trial. Following completion of PRESIDIO, 18 out of 20 patients enrolled in the OLE. For the first time, patients have the option to self-administer zetomipzomib in the OLE. As of the release date, active participation in the OLE ranged from 2 to 77 weeks, and six patients had discontinued for reasons unrelated to zetomipzomib. No additional safety or tolerability issues have been observed, and mean TIS scores have improved over scores observed at the 32-week conclusion of PRESIDIO.
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.
Financial Results

Cash, cash equivalents and marketable securities totaled $242.6 million as of March 31, 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 first quarter of 2022 increased by $1.7 million to $11.0 million compared to $9.3 million in the first quarter of 2021. This increase was primarily related to advancing the KZR-616 clinical program and the KZR-261 Phase 1 clinical trial.
General and administrative expenses for the first quarter of 2022 increased by $1.1 million to $4.9 million compared to $3.8 million in the first quarter of 2021. The increase was primarily due to an increase in personnel expenses, including non-cash stock-based compensation.
Net loss for the first quarter of 2022 was $16.0 million, or $0.26 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 first quarter of 2021.
Total shares of common stock outstanding were 59.6 million shares as of March 31, 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 8.9 million shares of common stock at a weighted average exercise price of $7.94 per share as of March 31, 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.