On May 12, 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 first quarter ended March 31, 2022 (Press release, Alpine Immune Sciences, MAY 12, 2022, View Source [SID1234614353]).

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"Our clinical pipeline of novel autoimmune and immuno-oncology candidates continues to make significant progress, as highlighted by the promising monotherapy dose escalation data presented at AACR (Free AACR Whitepaper) for davoceticept, a first-in-class CD28 costimulator and dual checkpoint inhibitor, in advanced malignancies and monotherapy expansion cohorts are currently open for enrollment," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "For ALPN-303, our next-generation dual BAFF/APRIL inhibitor, we look forward to sharing preliminary data from our phase 1 healthy volunteer study as part of a poster at the 2022 EULAR congress and to initiating patient-based studies by the end of 2022."
First Quarter 2022 and Recent Pipeline Updates
•Davoceticept monotherapy dose escalation data (NEON-1) presented in an oral mini-symposium at the 2022 American Association for Cancer Research (AACR) (Free AACR 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.
•Key preclinical rationale for davoceticept published in Nature Communications, showing its novel, first-in-class approach to engaging CD28 to enhance activation of T cells and drive anti-tumor activity.
•NEON-2 trial of davoceticept in combination with pembrolizumab placed on a partial clinical hold. Participants previously enrolled in the NEON-2 trial were able to continue to receive study drug; the NEON-1 monotherapy trial was not affected.
•Preliminary data for the first-in-human, phase 1 study of ALPN-303 in healthy volunteers to be presented as part of a poster at the 2022 European Alliance of Associations for Rheumatology (EULAR) Congress. Initiation of a phase 2 study in systemic lupus erythematosus (SLE) and at least one basket study in renal, hematologic and/or dermatologic indications planned by the end of 2022.
First Quarter 2022 Financial Results
As of March 31, 2022, we had cash, cash equivalents, and investments totaling $219.1 million. Net cash provided by operating activities for the quarter ended March 31, 2022 was $5.4 million compared to net cash used in operating activities of $16.0 million for the quarter ended March 31, 2021. Cash provided by operating activities was driven by the receipt of a $25.0 million upfront payment from Horizon, which was recorded as current and noncurrent deferred revenue on our Condensed Consolidated Balance Sheets. The Company recorded net losses of $7.5 million and $10.6 million for the quarters ended March 31, 2022 and 2021, respectively.
Collaboration revenue for the first quarter ended March 31, 2022 was $13.6 million compared to $3.2 million for the first quarter ended March 31, 2021. The 2022 amounts were attributable to revenue recognized under our AbbVie and Horizon Agreements, while 2021 revenue recognized solely relates to AbbVie.
Research and development expenses for the first quarter ended March 31, 2022 were $16.3 million compared to $10.4 million for the first quarter ended March 31, 2021. The increase was primarily attributable to our 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 first quarter ended March 31, 2022 were $4.8 million compared to $3.3 million for the first quarter ended March 31, 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.

On May 12, 2022 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the first quarter ended March 31, 2022 (Press release, Arcellx, MAY 12, 2022, View Source [SID1234614352]).

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"This is an exciting time for our company as we successfully completed our initial public offering in February, raising $142 million in gross proceeds and strengthened our balance sheet as we advance our pioneering platform to treat cancer and other incurable diseases," said Rami Elghandour, Arcellx’s chairman and chief executive officer. "In our first quarter as a public company, we continued to lay the foundation for our near-term and long-term success and our dedicated team made meaningful strides towards our 2022 milestones. We are looking forward to presenting additional patient and longer-term follow-up data on approximately 25 patients for our lead CART-ddBCMA program for patients with relapsed or refractory multiple myeloma (r/r MM) during an oral presentation at ASCO (Free ASCO Whitepaper) in June. Recently, we also advanced our development pipeline programs with the dosing of the first patient in our Phase 1 study evaluating ACLX-001 for patients with r/r MM utilizing our ARC-SparX technology. ARC-SparX has the potential to overcome some of the core challenges in cell therapy by reducing toxicities and addressing antigen heterogeneity, opening up significant market opportunities in harder to treat indications. In the second half of this year, we look forward to initiating our Phase 1 ARC-SparX clinical trial in patients with acute myeloid leukemia and high-risk myelodysplastic syndrome, our Phase 2 CART-ddBCMA pivotal trial in patients with r/r MM, and presenting longer-term patient data from our Phase 1 CART-ddBCMA expansion trial in r/r MM. The capstone of our progress is driven by our D-Domain technology which allows us to choose the right approach for the right indication, and we believe this differentiates our programs from traditional CAR-T therapies."

Recent Business Highlights

Clinical results from CART-ddBCMA Phase 1 study published in Blood Advances. On May 9, 2022, Arcellx announced the publication of clinical results from the dose escalation cohorts of its CART-ddBCMA Phase 1 study in patients with r/r MM in Blood Advances, the open-access journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). The data demonstrate 100% ORR and 75% CR/sCR; and evidence of durable clinical benefit in a population with poor prognostic features were observed in the dose escalation cohorts with CART-ddBCMA. The full online publication can be accessed here.

Dosed first patient in Phase 1 clinical trial evaluating ACLX-001 utilizing the ARC-SparX platform for the treatment of patients with r/r MM. On May 10, 2022, Arcellx announced that the first patient was dosed in its open-label, multicenter Phase 1 clinical trial (NCT04155749) to evaluate the company’s novel dosable and controllable ARC-SparX program in patients with r/r MM. ARC-SparX is comprised of SparX (soluble protein antigen receptor X-linkers) proteins engineered to target BCMA on myeloma cells together with ARC-T (Antigen Receptor Complex-T) cells that are dosed separately and engineered to activate only when engaged with a SparX protein bound to a myeloma cell. Both the ARC cells and the SparX proteins utilize the company’s proprietary novel synthetic binding scaffold called the D-Domain.

CART-ddBCMA accepted as an oral abstract presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. On April 27, 2022, Arcellx announced the presentation of new clinical data from its CART-ddBCMA Phase 1 trial in patients with relapsed or refractory multiple myeloma in an oral abstract session at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place June 3-7, 2022, in Chicago, Illinois.

Oral Presentation Details:

Title: Phase 1 Study of CART-ddBCMA in Relapsed or Refractory Multiple Myeloma
Speaker: Matthew J. Frigault, M.D., Assistant Director of the Cellular Therapy Service at Massachusetts General Cancer Center, and Instructor at Harvard Medical School
Session Type/Title: Oral Abstract Session/Hematologic Malignancies—Plasma Cell Dyscrasia
Session Date: Sunday, June 5, 2022
Session Time: 8:00 a.m. – 11:00 a.m. CDT
Location: McCormick Place Convention Center, Chicago, Illinois
Abstract Number: 8003

Live webcast event with management and panel of clinician experts. On Sunday, June 5, 2022, at 7:00 p.m. CDT, Arcellx will host a live webcast event with an expert panel of clinicians to discuss the clinical results from its CART-ddBCMA study being presented during ASCO (Free ASCO Whitepaper). The event will be accessible from Arcellx’s website at www.arcellx.com in the Investors section. A replay of the webcast will be archived and available for 30 days following the event.

First Quarter 2022 Financial Highlights

Cash, cash equivalents, and marketable securities:
As of March 31, 2022, Arcellx had cash, cash equivalents, and marketable securities of $210.9 million, which is anticipated to fund its operations into the second half of 2023.

R&D expenses:
Research and development expenses were $24.4 million and $8.5 million for the quarters ended March 31, 2022 and 2021, respectively, an increase of $15.9 million. This increase was driven by higher external costs associated with the advancement of our CART-ddBCMA clinical program, preclinical development of our other pipeline candidates, and increased headcount.

G&A expenses:
General and administrative expenses were $8.0 million and $2.8 million for the quarters ended March 31, 2022 and 2021, respectively, an increase of $5.2 million. This increase was driven by increased headcount, professional fees related to consulting and accounting, audit services, and other expenses.

Net loss:
Net loss was $32.4 million and $11.3 million for the quarters ended March 31, 2022 and 2021, respectively.

About the ARC-SparX Platform Technology
The ARC-SparX platform is designed to allow for controllability and adaptability to potentially reduce toxicities that are often associated with serious dose-limiting adverse events and to overcome tumor heterogeneity. It is a modular therapy which utilizes a universal ARC-T cell combined with an off-the-shelf SparX protein to separate the tumor-recognition and tumor-killing functions. SparX (soluble protein antigen-receptor X-linkers) proteins utilize our D-Domain technology engineered to recognize antigens on the surface of diseased cells and flags those cells for detection by the ARC-T cells. ARC-T cells express a D-Domain-based CAR engineered to specifically recognize a unique TAG in the SparX protein. ARC-T cells are dosed separately and only activated to kill the target cell when they encounter a SparX protein bound to the target antigen and thus are controlled through SparX dose modulation. Arcellx has developed a collection of SparX proteins that bind different antigens on the surface of diseased cells. Multiple SparX proteins with different antigen specificity can be administered to potentially address antigen heterogeneity or antigen escape that contribute to relapsed and refractory disease.

On May 12, 2022 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported financial results for the fiscal quarter ended March 31, 2022, and provided business updates (Press release, Erasca, MAY 12, 2022, View Source [SID1234614351]).

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"This quarter, we continued to advance our pipeline well. In addition to progressing our ERK1/2 inhibitor ERAS-007 and our SHP2 inhibitor ERAS-601 in their respective clinical trials, we also achieved first patient dosing for our CNS-penetrant EGFR inhibitor ERAS-801," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "We presented preclinical data in six posters at this year’s AACR (Free AACR Whitepaper) meeting supporting the clinical development of our potentially best-in-class programs, including ERAS-007, ERAS-601, and ERAS-3490 (CNS-penetrant KRAS G12C inhibitor). We were delighted to host a webinar with Dr. Scott Kopetz from MD Anderson Cancer Center discussing the unmet need and therapeutic opportunities targeting cancers driven by the RAS/MAPK pathway, with a particular focus in gastrointestinal cancers."

Dr. Lim continued, "We remain on track to report Phase 1b monotherapy data for ERAS-007 in HERKULES-1 and Phase 1 monotherapy data for ERAS-601 in FLAGSHP-1 in the second half of 2022. These data will include preliminary monotherapy safety and pharmacokinetics to support dose selection for combinations of ERAS-007 and ERAS-601. We also remain on track to file an IND for ERAS-3490 in the second half of 2022. As we look ahead, our pipeline continues to demonstrate encouraging therapeutic potential, our cash position remains strong, and we are highly focused on continued execution throughout the year."

Research and Development Highlights

Dosed First Patient in THUNDERBBOLT-1 Trial: In February 2022, Erasca dosed the first patient in THUNDERBBOLT-1, a Phase 1 trial evaluating ERAS-801 for the treatment of recurrent glioblastoma multiforme (rGBM)

Presented Six Posters at the 2022 AACR (Free AACR Whitepaper) Annual Meeting: In April 2022, Erasca presented six poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. These posters featured data supporting the clinical development of programs with best-in-class potential, including ERK1/2 inhibitor ERAS-007, SHP2 inhibitor ERAS-601, and central nervous system (CNS)-penetrant KRAS G12C inhibitor ERAS-3490

Hosted KOL Investor Webinar on AACR (Free AACR Whitepaper) Data: In April 2022, Erasca hosted an investor webinar highlighting its 2022 AACR (Free AACR Whitepaper) presentations and featuring a presentation by key opinion leader (KOL) Scott Kopetz, M.D., Ph.D., of MD Anderson Cancer Center

Corporate Highlights

Strengthened Executive Leadership: In January 2022, Erasca appointed Lisa Tesvich-Bonora, Ph.D., as Chief People Officer and promoted Robert Shoemaker, Ph.D., to Senior Vice President of Research

Entered into a Clinical Trial Collaboration and Supply Agreement (CTCSA) with Lilly: In March 2022, Erasca announced that it had entered into a CTCSA under which Lilly will supply its EGFR inhibitor cetuximab (ERBITUX) at no cost in connection with a clinical proof-of-concept study evaluating ERAS-007 in combination with the BRAF inhibitor encorafenib and cetuximab for the treatment of patients with BRAF V600E-mutant metastatic colorectal cancer (CRC) as part of the ongoing Phase 1b/2 HERKULES-3 trial. This agreement complements the CTCSA previously entered into with Pfizer for Pfizer’s BRAF inhibitor encorafenib (BRAFTOVI)

Expanded Board of Directors: In April 2022, Erasca appointed Jean Liu to its board of directors and to its audit committee

Opened New Corporate Headquarters: In April 2022, Erasca opened its new corporate headquarters in San Diego, CA, to accommodate and unify an expanded employee base

Key Upcoming Milestones

HERKULES-1: Phase 1b/2 trial for ERAS-007 in patients with advanced solid tumors
o
Initial Phase 1b monotherapy data expected in second half of 2022

HERKULES-3: Phase 1b/2 trial for ERAS-007 in patients with gastrointestinal malignancies

Initial Phase 1b combination data expected between the fourth quarter of 2022 and the first half of 2023

FLAGSHP-1: Phase 1/1b trial for ERAS-601 in patients with advanced solid tumors

Initial Phase 1 monotherapy data expected in second half of 2022

Initial Phase 1b combination data in triple wildtype (KRAS/NRAS/BRAF wildtype) CRC expected between the fourth quarter of 2022 and the first half of 2023

ERAS-3490: CNS-penetrant KRAS G12C inhibitor

IND filing expected in second half of 2022

First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $421.8 million as of March 31, 2022, compared to $459.2 million as of December 31, 2021. Erasca expects its current cash, cash equivalents, and marketable securities to fund operations into the second half of 2024.

Research and Development (R&D) Expenses: R&D expenses were $27.4 million for the quarter ended March 31, 2022, compared to $12.2 million for the quarter ended March 31, 2021. The increase was primarily driven by expenses incurred in connection with clinical trials, preclinical studies, and discovery activities, personnel costs due to increased headcount to support increased development activities, stock-based compensation expense, and outsourced service and consulting fees. Erasca also recorded

$2.0 and $3.7 million of in-process R&D expense during the quarters ended March 31, 2022 and 2021, respectively, for upfront and milestone payments and stock issuances under certain of our acquisition and license agreements.

General and Administrative (G&A) Expenses: G&A expenses were $7.1 million for the quarter ended March 31, 2022, compared to $3.7 million for the quarter ended March 31, 2021. The increase was primarily driven by stock-based compensation expense, insurance costs, personnel costs, and facilities and related costs.

Net Loss: For the quarter ended March 31, 2022, Erasca reported a net loss of $36.5 million, or $(0.31) per basic and diluted share, compared to a net loss of $18.0 million, or $(0.81) per basic and diluted share, for the quarter ended March 31, 2021.

On May 12, 2022 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported its financial results for the quarter ended March 31, 2022 and provided a business update (Press release, Sellas Life Sciences, MAY 12, 2022, View Source [SID1234614350]).

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"SELLAS achieved several key milestones in the first four months of 2022, including expanding our development pipeline and strengthening our balance sheet," said Angelos Stergiou, M.D., Sc.D. h.c., President and Chief Executive Officer of SELLAS. "We entered into an exclusive license agreement with GenFleet Therapeutics (Shanghai), Inc. ("GenFleet") granting us exclusive rights for the development and commercialization of GFH009, a highly selective and potentially first and best in class small molecule cyclin-dependent kinase 9 ("CDK9") inhibitor, across all therapeutic and diagnostic uses worldwide outside of Greater China. We believe that GFH009 is a complementary strategic fit to our overall clinical development plans and provides us with an opportunity to further advance into the market for acute myeloid leukemia ("AML") therapeutics. GFH009 affords us the potential to treat active AML disease while our lead clinical candidate, galinpepimut-S (GPS), is potentially used in the maintenance setting in AML. The in-license of GFH009 also provides us an opportunity to address a very important pediatric market of soft tissue sarcomas as well as other cancers."

Dr. Stergiou added, "During the first quarter, we continued to advance the Phase 3 REGAL trial for our lead asset, GPS and, in April, we also launched an expanded access program for GPS in response to multiple requests from physicians with which we hope to potentially improve clinical outcomes for patients and their families. Subsequent to the quarter, we fortified SELLAS’ balance sheet with the closing of a public offering with gross proceeds of $25 million. The funds position us to advance our clinical programs for both GPS and GFH009. We are pleased by the progress we made over the first quarter of 2022 and remain steadfast in our mission to develop innovative treatments and improve the standard of care available to patients. With GPS and GFH009, we now have two significant clinical assets in our pipeline, with several opportunities to develop and potentially commercialize, cancer drugs that can prolong the lives of patients battling cancer."

Pipeline Updates:

Galinpepimut-S (GPS)
Expanded Access Program: In April 2022, the Company launched a pre-approval access/expanded access program ("EAP") with SELLAS’ lead asset, GPS, for treating patients suffering from AML. For more information on the GPS EAP, please visit the PIPELINE page at www.sellaslifesciences.com.

Phase 3 REGAL Study: SELLAS continued to progress enrollment of patients and activation of additional sites in the United States, Europe, and Asia in the first quarter of 2022. The Company believes that enrollment for the REGAL study will be completed in late 2022 or early in the first quarter of 2023 and the planned interim analysis will occur by the end of the first half of 2023, provided that its statistical assumptions and assumptions regarding the impact of COVID-19 on the operations of clinical sites as well as the duration of the pandemic remain unchanged.

Phase 1/2 GPS Study in Combination with Merck’s KEYTRUDA: In February 2022, the Company completed enrollment in the Phase 1/2 clinical trial of GPS in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in second or third line Wilms Tumor-1 (WT1+) relapsed or refractory metastatic ovarian cancer. Data from 15 patients is currently being reviewed by SELLAS and Merck, with top-line results expected by mid-2022 and a final data analysis for all evaluable patients expected by the end of 2022.

Phase I GPS Study in China: In March 2022, an IND application to initiate the first clinical trial in China for GPS was approved by China’s National Medical Products Administration ("NMPA"), which triggered a milestone payment of $1 million received by the Company in May 2022. The IND, for a small Phase 1 clinical trial investigating safety, was submitted by SELLAS’ partner in China, 3D Medicines Inc. ("3D Medicines"), with 3D Medicines expecting to initiate the trial by mid-2022. 3D Medicines’ current clinical development plan provides for initiation of a Phase 2 clinical trial following receipt of satisfactory safety data from the Phase 1 clinical trial; the initiation of the Phase 2 clinical trial will also trigger a milestone payment to SELLAS.

New Patent Allowance: In February 2022, the U.S. Patent and Trademark Office issued a Notice of Allowance for a patent application covering certain WT1-targeting peptides, in combination with other molecules such as other peptides and immunomodulating compounds, useful for treatment of WT1-expressing cancers. The patent application covers WT1-targeting peptides linked to other molecules and is expected to be granted later this year. The patent will have a term extending to at least 2026. This patent complements the Company’s existing composition of matter patents covering GPS peptides which expire in 2033 not including any potential extensions.
GFH009
In-License: On March 31, 2022 the Company announced it had entered into an exclusive license agreement with GenFleet Therapeutics (Shanghai), Inc. ("GenFleet"), that grants rights to SELLAS for the development and commercialization of GFH009, a highly selective small molecule CDK9 inhibitor, across all therapeutic and diagnostic uses worldwide outside of Greater China (mainland China, Hong Kong, Macau and Taiwan). CDK9 activity has shown a negative correlation with overall survival in a number of cancer types, including hematologic cancers, such as AML and lymphomas, as well as solid cancers, such as osteosarcoma, pediatric soft tissue sarcomas, and melanoma, and endometrial, lung, prostate, breast and ovarian cancer.

Ongoing Phase 1 Clinical Trial: In April 2022, SELLAS announced that initial data from the first four dose levels of the ongoing Phase 1 dose-escalating clinical trial of GFH009 show a significant anti-leukemic effect at the 9mg and 15mg dose levels given twice a week in AML patients resistant to standard-of-care treatments, with two patients refractory to, or relapsed after, venetoclax treatment experiencing greater or equal to a 50 percent decrease in bone marrow blasts following GFH009 monotherapy. There have been no dose-limiting toxicities, including no grade 3/4 neutropenia (an abnormally low count of a type of white blood cells), in the first four dose levels (2.5mg, 4.5mg, 9mg and 15 mg) with the twice-weekly GFH009 dosing. The first AML patient has been enrolled in the fifth dose level (22.5mg twice a week) cohort. The last planned dose level in this clinical trial is 30mg.
Corporate Highlights:

Underwritten Public Offering: On April 5, 2022, the Company consummated an underwritten public offering providing gross proceeds to the Company of $25.0 million, before deducting underwriting discounts and commissions and offering expenses.

Bolstered Leadership Team: In March 2022, SELLAS appointed Robert Francomano as Chief Commercial Officer. In January 2022, the Company promoted John Burns to Senior Vice President, Finance, and Chief Accounting Officer.
Settlement of Legacy Galena Litigations: In February 2022, SELLAS received the final court approval of the settlement of securities litigation relating to the Company’s predecessor, Galena. This marked the end to all litigation related to activities of Galena.
Financial Results for the First Quarter 2022:

Licensing revenue: Licensing revenue for the first quarter of 2022 was $1.0 million, as compared to $5.7 million for the same period in 2021. Licensing revenue in the first quarter of 2022 was related to China’s NMPA approval of an IND application by 3D Medicines and licensing revenue during the first quarter of 2021 was related to the initial transaction price of the license agreement with 3D Medicines, which was recognized over a period of time.

R&D Expenses: Research and development expenses for the first quarter of 2022 were $4.6 million, as compared to $4.3 million for the same period in 2021. The increase was primarily due to an increase in clinical trial expenses for the ongoing Phase 3 clinical trial of GPS in AML and personnel related expenses due to increased headcount, partially offset by a decrease in manufacturing expenses due to the timing of the manufacture of registration batches of GPS in the prior year.

Acquired In-Process Research and Development: Acquired in-process research and development for the first quarter 2022 was $10.0 million, related to the in-licensing of GFH009. There was no acquired in-process research and development during the first quarter of 2021.

G&A Expenses: General and administrative expenses for the first quarter of 2022 were $3.0 million, as compared to $3.6 million for the same period in 2022. The decrease was primarily due to a decrease in amortization expense associated with the capitalized contract acquisition costs of the 3D Medicines license agreement and a decrease in professional service fees, partially offset by an increase in personnel related expenses due to increased headcount.

Net Loss: Net loss was $16.7 million for the first quarter of 2022, or a basic and diluted loss per share of $1.05, as compared to a net loss of $2.4 million for the same period in 2021, or a basic and diluted loss per share of $0.16.

Cash Position: As of March 31, 2022, cash and cash equivalents totaled approximately $14.3 million. Subsequent to March 31, 2022, the Company consummated an underwritten public offering providing gross proceeds to the Company of $25.0 million, before deducting underwriting discounts and commissions and offering expenses, and received a $1.0 million milestone payment from 3D Medicines.

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.