On March 17, 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 fourth quarter and year ended December 31, 2021 and provided a business update (Press release, Kezar Life Sciences, MAR 17, 2022, View Source [SID1234610263]).

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"In 2021, we made significant progress in each of our programs, achieving target enrollment in both of our Phase 2 trials with zetomipzomib, sharing positive interim results from our MISSION Phase 2 study, and launching a Phase 1 trial in solid tumors with our novel protein secretion inhibitor, KZR-261," said John Fowler, Kezar’s Co-founder and Chief Executive Officer. "We look forward to continued momentum across the company in 2022, including the presentation of topline results from the MISSION and PRESIDIO trials in the second quarter and preparing for the exciting next phase of development with our first-in-class immunoproteasome inhibitor, zetomipzomib."

Zetomipzomib Assigned as Nonproprietary Name for KZR-616

The International Nonproprietary Name (INN) of zetomipzomib has been selected as the proposed nonproprietary name for KZR-616. The established suffix "-ipzomib" is being utilized to convey the compound’s mode of action to selectively inhibit the immunoproteasome.

KZR-616: Selective Immunoproteasome Inhibitor

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

In November 2021, Kezar presented positive interim results from the MISSION Phase 2 clinical trial of KZR-616, in which five patients had reached the end of treatment, and ten patients had reached week 13 of treatment. The interim results showed a clinically meaningful renal response at the end of treatment for this subset of patients. Four of five patients who completed treatment at week 25 with KZR-616 demonstrated clinically meaningful reduction in proteinuria to less than 0.8 urine protein to creatine ratio (UPCR). Clinically meaningful reductions in UPCR were also observed in five of ten patients at week 13 of treatment and included improvements in key disease biomarkers. KZR-616 was well tolerated over the six-month treatment period.
The MISSION Phase 2 open-label trial in patients with active, proliferative LN reached target enrollment of 20 patients in November 2021. 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 when compared to baseline.
Kezar expects to report topline data in the second quarter of 2022.
PRESIDIO – Phase 2 clinical trial of KZR-616 in patients with active dermatomyositis (DM) or polymyositis (PM) (NCT04033926)

The PRESIDIO Phase 2 randomized, placebo-controlled, double-blind, cross-over clinical trial of KZR-616 in patients with DM or PM reached target enrollment of 24 subjects in August 2021. The primary efficacy endpoint of this clinical trial is the mean change from beginning to end of treatment with KZR-616 in the Total Improvement Score (TIS), which ranges from 0 to 100.
Kezar expects to report topline data in the second quarter of 2022.
Patients completing the PRESIDIO study have an opportunity to enroll into an open-label extension study to continue receiving KZR-616 treatment for up to a total of 96 weeks (NCT04628936). In December 2021, at-home self-administration of KZR-616 was introduced for patients in the open-label extension study.
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.
In October 2021, the first patient was dosed in the open-label Phase 1 clinical trial of KZR-261 in patients with solid tumor malignancies.
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.
An abstract featuring Kezar’s proprietary small molecule inhibitors of the Sec61 translocon, specifically KZR-834, a working analog of KZR 261, has been selected for presentation at the upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting, taking place April 8–13, 2022 in New Orleans, LA. Details for the AACR (Free AACR Whitepaper) presentation are as follows:
Title: Sec61 inhibitor KZR-834, an anti-cancer agent, exhibits immunomodulatory activity and combines with PD-1 blockade to further enhance immune responses
Abstract Number: 5592
Session Title: Immunology, Preclinical Immunotherapy
Date/Time: Available on demand [Friday, April 8, 2022, 8:30 a.m. ET]
Presenter: Jennifer Whang, Associate Director, Biology
Board Appointment

Courtney Wallace, a strategic business development executive, was appointed to Kezar’s Board of Directors in December 2021, bringing over a decade of business experience in healthcare.

Financial Results

Cash, cash equivalents and marketable securities totaled $208.4 million as of December 31, 2021, compared to $140.4 million as of December 31, 2020. The increase in cash, cash equivalents and marketable securities was primarily attributable to the net proceeds from the issuance of common stock under the company’s "at-the-market" sales program, net of cash used by the company in operations to advance its clinical-stage programs.
Research and development expenses for the fourth quarter of 2021 increased by $1.7 million to $9.8 million compared to $8.1 million in the fourth quarter of 2020. Full year R&D expenses increased by $7.9 million to $38.9 million in 2021, compared to $31.0 million in 2020. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the KZR-261 clinical program.
General and administrative expenses for the fourth quarter of 2021 increased by $1.3 million to $4.3 million compared to $3.0 million in the fourth quarter of 2020. Full year G&A expenses increased by $3.7 million to $15.7 million in 2021, compared to $12.0 million in 2020. The increase was primarily due to an increase in stock-based compensation and personnel expenses as a result of an increase in headcount and salaries.
Net loss for the fourth quarter of 2021 was $14.2 million, or $0.25 per basic and diluted common share, compared to a net loss of $10.9 million, or $0.22 per basic and diluted common share, for the fourth quarter of 2020. Net loss for 2021 was $54.6 million, or $1.04 per basic and diluted common share, compared to $41.7 million, or $0.95 per basic and diluted common share, in 2020.
Total shares of common stock outstanding were 56.3 million shares as of December 31, 2021. 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 6.9 million shares of common stock at a weighted-average exercise price of $5.95 per share, each as of December 31, 2021.
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 1a and 1b clinical trials provide evidence that zetomipzomib exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in multiple severe autoimmune diseases.

About KZR-261

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.

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 Dermatomyositis and Polymyositis

Dermatomyositis (DM) and Polymyositis (PM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Approximately 30,000 to 120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

About Inhibition of Protein Secretion

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.

On March 17, 2022 Foundation Medicine, Inc. a pioneer in molecular profiling for cancer, reported it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic to identify patients with non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) EGFR exon 19 deletions or exon 21 (L858R) alterations and are appropriate for treatment with EGFR Tyrosine Kinase Inhibitors (TKI) approved by FDA for this indication (Press release, Foundation Medicine, MAR 17, 2022, View Source [SID1234610281]).

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EGFR mutations are the second most common oncogenic drivers in NSCLC. Deletions in exon 19 and substitutions in exon 21 account for roughly 85 percent of observed EGFR mutations in NSCLC.1 As a companion diagnostic for therapies targeting these mutations in NSCLC, FoundationOneCDx offers oncologists flexibility when selecting the right therapy for their patients and ensures all FDA approved treatment options are considered within this group of therapies.

"Cancer treatment decision-making is growing more complex as we learn more about the mutations that drive cancers to grow and new treatments become available to target those changes," said Mia Levy, M.D., Ph.D., chief medical officer at Foundation Medicine. "We believe securing a third group companion diagnostic approval for FoundationOne CDx is another critical step toward simplifying the decision-making process for oncologists and their patients by allowing them to uncover all FDA approved treatment options for this indication through one test."

In addition to driving efficiency in clinical care, this approval is part of an innovative, efficient regulatory approach that simplifies the companion diagnostic approval process for Foundation Medicine’s biopharma partners developing EGFR inhibitor therapeutics targeting EGFR exon 19 deletions or exon 21 alterations in NSCLC while maintaining rigorous quality standards.

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit View Source

On March 17, 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 fourth quarter and year ended December 31, 2021 (Press release, Alpine Immune Sciences, MAR 17, 2022, View Source [SID1234610303]).

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"2021 was a period of significant growth at Alpine with the advancement of all three of our clinical programs into significant stages of development," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "As a result we secured $176 million in additional capital through a PIPE financing, the achievement of milestones as part of our AbbVie collaboration and an upfront payment from our recently announced discovery partnership with Horizon Therapeutics."

Dr. Gold added: "2022 looks to be a transformative year for Alpine with several important readouts anticipated across our programs, including the completion of dose escalation for davoceticept monotherapy and initiation of expansion cohorts; as well as the completion of the phase 1 healthy volunteer study for ALPN-303 and initiation of one or more patient-based studies in autoantibody-related diseases by the end of the year."
Full Year 2021 and Recent Clinical and Corporate Updates
•Davoceticept (ALPN-202): Conditional CD28 costimulator and dual checkpoint inhibitor
◦Davoceticept monotherapy dose escalation data (NEON-1) will be presented in an oral presentation at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on April 12, 2022. Initial findings of davoceticept’s tolerability and clinical activity were previously reported in an oral presentation at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
◦NEON-1 monotherapy expansion cohorts in patients with cutaneous melanoma and PD-L1+ tumors projected to be initiated in the first half of 2022.
◦Announced partial clinical hold on the NEON-2 trial combining davoceticept with pembrolizumab. Participants previously enrolled can continue to receive davoceticept and pembrolizumab; the NEON-1 monotherapy trial was not affected.
•ALPN-303: Dual APRIL/BAFF inhibitor
◦Began a first-in-human, phase 1 study of ALPN-303 in healthy volunteers in the fourth quarter of 2021; initial safety, pharmacokinetic, and pharmacodynamic findings anticipated by mid-2022.
◦Preclinical data, highlighting the differentiation and potential best-in-class profile of ALPN-303, were presented in two oral presentations at the European Alliance of Associations for Rheumatology and American College of Rheumatology Convergence 2021 meetings.
◦Plan to initiate studies in systemic lupus erythematosus (SLE) and potentially additional autoantibody-related diseases in the second half of 2022.
•Acazicolcept (ALPN-101): Dual CD28/ICOS inhibitor
◦Received $45 million in pre-option exercise development milestones as part of the Option and License Agreement with AbbVie, bringing total upfront and milestone payments received through December 31, 2021, to $105 million.
◦Continue to enroll Synergy, a phase 2 trial of acazicolcept in patients with SLE.

◦With collaborators at the French National Institute of Health and Medical Research (INSERM), demonstrated that acazicolcept decreased manifestations of systemic sclerosis in preclinical mouse models (Arthritis Res Ther 24: 13, 2022)

•General Corporate
◦Announced an exclusive license and collaboration agreement with Horizon Therapeutics plc for the development and commercialization of up to four preclinical candidates generated from Alpine’s unique discovery platform, which included $40 million in upfront payments ($25 million cash and $15 million equity investment at a 25% premium to the 30-day volume-weighted average share price) and eligibility to receive up to $1.5 billion in total, through future milestone-based payments.

◦Raised $91 million in a private placement, led by Frazier Life Sciences Public Fund with participation from Decheng Capital, BVF Partners, TCG X, Avidity Partners, OrbiMed, Omega Fund, and Logos Capital, among others.
Fourth Quarter and Full Year 2021 Financial Results
As of December 31, 2021, we had cash, cash equivalents, and investments totaling $215.4 million. Net cash used in operating activities for the year ended December 31, 2021 was $15.2 million compared to net cash provided by operating activities of $30.1 million for the year ended December 31, 2020. The Company recorded net losses of $50.3 million and $27.9 million for the years ended December 31, 2021 and 2020, respectively.
Collaboration revenue for the fourth quarter ended December 31, 2021 was $4.5 million compared to $5.6 million for the fourth quarter ended December 31, 2020. Collaboration revenue for the year ended December 31, 2021 was $23.4 million compared to $9.3 million for the year ended December 31, 2020. The amounts were primarily attributable to the revenue recognized under our AbbVie Agreement.

Research and development expenses for the fourth quarter ended December 31, 2021 were $15.4 million compared to $9.1 million for the fourth quarter ended December 31, 2020. Research and development expenses for the year ended December 31, 2021 were $58.7 million compared to $27.2 million for the year ended December 31, 2020. The increases were primarily attributable to our Synergy and NEON studies, contract manufacturing and process development of our product candidates primarily for acazicolcept and ALPN-303, increased personnel costs and other direct research activities.

General and administrative expenses for the fourth quarter ended December 31, 2021 were $4.5 million compared to $3.0 million for the fourth quarter ended December 31, 2020. General and administrative expenses for the year ended December 31, 2021 were $14.6 million compared to $10.9 million for the year ended December 31, 2020. The increase was primarily attributable to increases in professional and legal services and personnel costs.
The Company expects that its current cash resources, combined with the $25 million up-front payment received from Horizon in January 2022 and the potential $30 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of acazicolcept, will be sufficient to fund its planned operations into 2024.

On March 17, 2022 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, Aduro Biotech, MAR 17, 2022, View Source [SID1234610242]).

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"2021 was a very productive year for Chinook, as we initiated the phase 3 ALIGN and phase 2 AFFINITY trials for atrasentan, presented compelling phase 1/2 data in patients with IgA nephropathy for BION-1301, successfully completed IND-enabling studies for CHK-336, continued to advance multiple programs within our research pipeline and executed notable strategic partnerships including our Evotec research collaboration, formation of Sairopa B.V. for the development of non-renal assets and establishment of the SanReno Therapeutics joint venture in China," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "With the recent milestones we have achieved and our strong financial position, Chinook is well-positioned to continue building the leading company addressing unmet medical needs in kidney disease. During 2022, we look forward to presenting additional data from both our atrasentan and BION-1301 programs, initiating our phase 1 trial of CHK-336 and continuing to advance our preclinical pipeline for rare, severe chronic kidney diseases."

2021 and Recent Accomplishments

Atrasentan
Atrasentan is a potent and selective endothelin A (ETA) receptor antagonist that has the potential to provide benefit in multiple chronic kidney diseases by reducing proteinuria and having direct anti-inflammatory and anti-fibrotic effects to preserve kidney function. The phase 3 ALIGN trial of atrasentan is currently enrolling patients with IgA nephropathy (IgAN), and the phase 2 AFFINITY basket trial of atrasentan is currently enrolling patients with proteinuric glomerular diseases.

Enrollment of the phase 3 ALIGN trial of atrasentan continues to advance with the activation of new trial sites and expansion into additional countries. Chinook expects to report topline data from the six-month interim proteinuria endpoint analysis in 2023 to support an application for accelerated approval under Subpart H in the United States.

Chinook has completed enrollment of the IgAN patient cohort of the phase 2 AFFINITY trial, and continues to enroll the other three cohorts, including patients with focal segmental glomerulosclerosis (FSGS), Alport syndrome and diabetic kidney disease in combination with SGLT2 inhibitors. Chinook plans to present data from the IgAN patient cohort of this study in an oral presentation at the ERA Congress in May 2022, with data from one or more additional cohorts expected in the second half of 2022.

Several abstracts on atrasentan were delivered as poster presentations at nephrology conferences throughout 2021, including:

Data demonstrating endothelin pathway activation in the kidneys of patients with IgAN has a strong association with clinical progression. Atrasentan was also shown to inhibit endothelin-1 mediated transcriptional networks, including cell proliferation, inflammation and fibrosis in human mesangial cells. This translational research was conducted in collaboration with the laboratory of Matthias Kretzler, M.D., Professor of Nephrology and Professor of Computational Medicine & Bioinformatics at University of Michigan Medical School, and supports the therapeutic potential of ETA receptor blockade with atrasentan in patients with IgAN at high risk of progression. (ASN Kidney Week 2021)

Analysis of three separate single-dose, randomized phase 1 studies of atrasentan demonstrating consistent and predictable safety, tolerability and pharmacokinetic profiles in healthy Chinese, Japanese and North American adults of non-Asian descent. The consistent profile of atrasentan across these ethnicities and geographic regions, supports the inclusion of patients with IgAN in Asia, where there is an increased prevalence and potentially accelerated disease progression, in the ongoing global phase 3 ALIGN study. (ASN Kidney Week 2021)

Data demonstrating atrasentan’s effect to rapidly reduce albuminuria and downregulate intra-renal transcriptional proliferative, inflammatory and fibrotic signaling in the gddY mouse IgAN model, supporting the therapeutic potential of atrasentan in patients with IgAN to reduce proteinuria and kidney inflammation and fibrosis, key drivers of IgAN progression. (ISN WCN 2021)

Data demonstrating atrasentan’s effect to attenuate human renal mesangial cell activation induced by endothelin-1 or IgAN patient immune-derived immune complexes in a translational model system, supporting the therapeutic potential of atrasentan in patients with IgAN, not only via its well characterized effect of reducing proteinuria, but also by potentially reducing mesangial cell activation, a hallmark of IgAN. (ISN WCN 2021)
Atrasentan was granted orphan drug designation for the treatment of primary IgAN by the European Commission.
BION-1301
BION-1301 is a novel anti-APRIL monoclonal antibody currently in phase 1/2 development for patients with IgAN. BION-1301’s potentially disease-modifying approach to treating IgAN by reducing circulating levels of galactose-deficient IgA1 (Gd-IgA1) has been demonstrated preclinically as well as clinically in both healthy volunteers and patients with IgAN.

Chinook presented data from the BION-1301 phase 1 intravenous (IV) to subcutaneous (SC) bioavailability study in healthy volunteers at the ISN World Congress of Nephrology 2021, demonstrating the ability to transition to SC administration of BION-1301 in the ongoing phase 1/2 study of BION-1301.

Chinook completed enrollment of Cohort 1 of Part 3 of the ongoing phase 1/2 study of BION-1301 and presented additional patient data and follow-up from this cohort at ASN Kidney Week 2021, indicating BION-1301 was well-tolerated and caused durable reductions in Gd-IgA1, IgA, IgM, and to a lesser extent, IgG levels in patients with IgAN. BION-1301 demonstrated a greater than 50% geometric mean reduction in 24-hour urine protein creatinine ratio (UPCR) after three (n=6) to six (n=4) months of treatment, with further reductions in two patients through one year of treatment. After at least 24 weeks of treatment, patients in Cohort 1 are transitioning from IV dosing at 450 mg every two weeks to SC dosing at 600 mg every two weeks.

Enrollment of Cohort 2 of Part 3 of the ongoing phase 1/2 study of BION-1301 is ongoing. Patients in Cohort 2 receive a SC dose of 600 mg of BION-1301 every two weeks.

Chinook plans to present additional data from Cohort 1 of Part 3 in a mini-oral presentation at the ERA Congress in May 2022, with data from Cohort 2 of Part 3 expected in the second half of 2022.
CHK-336
CHK-336 is an oral small molecule lactate dehydrogenase A (LDHA) inhibitor with liver-targeted tissue distribution that Chinook is developing for the treatment of patients with primary hyperoxaluria (PH), secondary hyperoxaluria due to increased endogenous oxalate production and idiopathic stone formation.

Chinook has completed IND-enabling studies, submitted an IND and is advancing CHK-336 towards planned initiation of a phase 1 clinical trial in healthy volunteers in the first half of 2022.

CHK-336 has received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for PH.
Precision Medicine Research & Discovery
Chinook is focused on the discovery and development of novel precision medicines for rare, severe chronic kidney diseases (CKDs) with defined genetic or molecular drivers of disease initiation and progression, and efficient development paths.

Chinook has multiple preclinical programs across the discovery, target validation, lead identification and lead optimization stages to generate future clinical pipeline candidates.

Chinook is leveraging its ongoing strategic collaboration with Evotec to identify and validate novel targets and enable patient stratification strategies through access to the NURTuRE CKD Patient Biobank, which provides comprehensive PANOMICS characterization of thousands of CKD patients with prospective clinical follow-up and retained bio-samples of urine and blood for exploratory biomarker analysis.
Corporate

In April 2021, Chinook entered into an agreement for Sairopa B.V. to acquire certain of Chinook’s non-renal assets in exchange for a 44 percent preferred equity position in Sairopa. Any future proceeds resulting from this transaction will be shared equally between Chinook and the CVR holders until October 4, 2030, after which 100 percent of the value will accrue to Chinook.

In November 2021, Chinook closed a $183.5M public offering, which included the exercise in full of the underwriters’ option to purchase additional shares of common stock.

Also in November 2021, Chinook formed SanReno Therapeutics, a 50/50 joint venture with an investor syndicate led by Frazier Healthcare Partners and Pivotal bioVenture Partners China, to develop, manufacture and commercialize kidney disease therapies in mainland China, Hong Kong, Macau, Taiwan and Singapore.
Fourth Quarter and Full Year 2021 Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $355.1 million at December 31, 2021, compared to $250.4 million at December 31, 2020. The increase is primarily due to net proceeds from our public offering in November 2021 and at-the-market (ATM) sales of common stock under our existing ATM facility in the second quarter of 2021, partially offset by cash used in operations.

Revenue – Total revenue increased by $50.4 million and $50.8 million for the quarter and year ended December 31, 2021, respectively, compared to the same periods in 2020. The increase was primarily due to non-cash revenue of $41.2 million recognized under Chinook’s license agreement with SanReno and from a development milestone of $10.0 million recognized under the Exclusive Patent and Know-How License and Research Collaboration Agreement with Merck for MK-5890, an anti-CD27 agonist.

Expenses –
Research and development expenses for the quarter and year ended December 31, 2021 were $24.9 million and $97.0 million, respectively, compared to $21.8 million and $36.1 million, respectively, for the same periods in 2020. The increase was primarily due to external clinical and manufacturing expenses related to the atrasentan and BION-1301 clinical programs; higher employee-related costs from increased staff to build out our clinical and development capabilities; and an increase in facilities and other costs. Research and development expenses for the year ended December 31, 2021 also included an upfront fee under a collaboration agreement with Evotec.
General and administrative expenses for the quarter and year ended December 31, 2021 were $7.7 million and $31.9 million, respectively, compared to $11.0 million and $19.1 million, respectively, for the same periods in 2020. The decrease in the quarter ended December 31, 2021 compared to same period in 2020 was primarily due to higher costs related to the merger with Aduro in 2020. The increase in the year ended December 31, 2021 compared to the prior year was primarily due to higher employee-related costs from increased staff to build out our public-company infrastructure and an increase in facilities and other costs.
Expenses due to the change in fair value of contingent consideration and contingent value rights liabilities for the quarter and year ended December 31, 2021 were $5.8 million and $27.3 million, respectively, compared to $1.5 million for the same periods in 2020. These non-cash expenses are due to the quarterly revaluation of liabilities related to the Sairopa transaction and under the Merck collaboration. During the quarter ended December 31, 2021, we were notified of the achievement of a development milestone under the agreement with Merck, which resulted in an increase to the fair value of the CVR liability.
Other –
The sale of non-renal assets to Sairopa in the second quarter of 2021 resulted in a $7.2 million gain for the year ended December 31, 2021.

The development milestone received under the Merck collaboration, net of permitted deductions, will be paid to CVR holders in the second quarter of 2022.
Net Loss – Net income for the fourth quarter of 2021 was $7.5 million, or $0.15 per basic share, compared to a net loss of $49.9 million, or $1.24 per share for the same period in 2020. Net loss for the year ended December 31, 2021 was $102.9 million, or $2.26 per share, compared to a net loss of $81.6 million, or $6.20 per share for the same period in 2020.

On March 17, 2022 LUMICKS, a leading life science tools company that develops instruments for dynamic single-molecule and cell avidity analysis, reported that a paper uncovering new insights into Chimeric Antigen Receptor (CAR) T cell functionality was published in the February 2022 issue of Frontiers in Immunology (Press release, LUMICKS, MAR 17, 2022, View Source;utm_medium=rss&utm_campaign=new-insights-into-car-t-cell-efficacy-for-cancer-therapies [SID1234610264]). The paper describes innovative research conducted at King’s College London under the auspices of Dr. John Maher, in collaboration with Leucid Bio, using LUMICKS’ z-Movi Cell Avidity Analyzer. This new research suggests that adding a second targeting receptor to CAR T cells enhances their ability to target cancer cells and shows promise to improve immunotherapies for cancer in pre-clinical models

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Said Dr. John Maher, Senior Lecturer at King’s College London in the School of Cancer & Pharmaceutical Sciences, and Chief Scientific Officer of Leucid Bio, "The unique abilities of the z-Movi Cell Avidity Analyzer enabled us to measure the total strength of interaction between our panel of CAR T cells and cancer cells. Using the LUMICKS’ z-Movi instrument, we could easily find the ‘goldilocks’ CARs that do not bind too strongly or too weakly to the target cells and show superior killing in pre-clinical models. New insights obtained using cell avidity measurements can really help improve the way we design cell therapies."

Added Andrea Candelli, Chief Scientific Officer of LUMICKS, "We share the King’s College team’s excitement about the additional evidence shown by this paper of the critical role that cell avidity plays in uncovering and optimizing CAR T cells. Dr. Maher’s powerful research underscores the insights that our revolutionary technology can help researchers around the world discover, and help science better address core challenges in human health, particularly in designing potentially more effective immunotherapies to fight cancer."

CAR-T cell immunotherapies modify T cells in the immune system to target and destroy cancer cells by genetically reprogramming T cells to make the CAR protein, by which T cells bind to, and attack, cancer cells. Such therapies are offered as treatments for certain cancers and many researchers, including Dr. Maher, are investigating new designs for CAR T-cell therapy for solid tumor cancers.

The z-Movi measures the avidity, or level of binding, between immune cells like CAR Ts, and their targets, the cancer cells, enabling researchers to identify the most potent immunotherapeutic effector cells as candidates to target and destroy specific cancer cells. This new technology provides predictive, reproducible, and fast results at a single-cell resolution without compromising cell viability, and ensures safe sample handling. LUMICKS’ cell avidity solutions use acoustics to measure forces and interactions between cells, with the goal of shortening the drug development cycle for adoptive cell therapies and other immunotherapies and reducing failure rates in clinical trials. First introduced in 2020, the z-Movi has found wide appeal in academic and biopharma laboratories around the world, with a rapid uptake in sales in 2021.