On March 12, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported its financial results for the fourth quarter and full year ended December 31, 2019 (Press release, CTI BioPharma, MAR 12, 2020, View Source [SID1234555486]).

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"In the latter half of 2019 and beginning of 2020, we further advanced our pacritinib development program, including presenting data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting that reinforced the clinical and scientific rationale for our ongoing PAC203 Phase 3 PACIFICA trial evaluating pacritinib at 200 mg BID in severely thrombocytopenic myelofibrosis patients." said Adam R. Craig, M.D., Ph.D. "Severely thrombocytopenic myelofibrosis patients have limited, and often ineffective, therapeutic options. In an effort to advance pacritinib as quickly as possible to these patients, we established an accelerated approval pathway with the U.S. Food and Drug Administration ("FDA") by amending the PACIFICA pivotal Phase 3 trial protocol to allow for the primary analysis of Spleen Volume Reduction ("SVR") rates on the first 168 patients, with an end-of-study analysis of Total Symptom Score ("TSS") and Overall Survival ("OS") following the full enrollment of 348 patients. If the primary endpoint of SVR is met following the planned review of data from the first 168 patients, we intend to submit a New Drug Application ("NDA") under the FDA’s subpart H regulations. We expect to report primary SVR data by the end of 2021, with a potential NDA filing in early 2022. Additionally, we recently raised an additional $59.3 million in a rights offering, which provides us with additional cash runway into Q1 2022 as we continue to develop pacritinib."

Fourth Quarter Financial Results
Operating loss was $9.5 million and $40.7 million for the three months and year ended December 31, 2019, respectively, compared to operating income of $0.2 million and operating loss of $32.9 million for the respective periods in 2018. The operating loss during the three-month period ended December 31, 2019 as compared to the operating income for the comparable period in 2018 resulted primarily from the decrease in license and contract revenue as discussed below, partially offset by a decrease in operating expenses. The increase in operating loss for the year ended December 31, 2019 as compared to the same period in 2018 resulted primarily from a decrease in license and contract revenues between periods as discussed below, partially offset by a decrease in operating expenses. As of December 31, 2019, cash, cash equivalents and short-term investments totaled $33.7 million, compared to $67.0 million as of December 31, 2018. In March 2020, we completed our rights offering and received approximately $59.3 million in net proceeds. We expect current cash, cash equivalents and short-term investments,

when combined with the net proceeds we received from the rights offering, will enable us to fund our operations into the first quarter of 2022.

License and contract revenues for the three months ended December 31, 2018 were $14.1 million while no revenues were recognized during the three months ended December 31, 2019. License and contract revenues for the three months ended December 31, 2018 were primarily related to milestone revenues recognized upon the achievement of a regulatory milestone under the license and collaboration agreement for PIXUVRI with Les Laboratoires Servier and Institut de Recherches Internationales Servier as well as the attainment of a worldwide net sales milestone of TRISENOX (arsenic trioxide) under the agreement with Teva Pharmaceutical Industries Ltd. ("Teva"). License and contract revenues for the years ended December 31, 2019 and 2018 were $3.3 million and $26.3 million, respectively. The decrease between periods primarily resulted from milestone revenues recognized in 2018 from Teva related to the achievement of a milestone for FDA approval of TRISENOX for first-line treatment of acute promyelocytic leukemia, in addition to the license and contract revenues recognized during the three months ended December 31, 2018, as discussed above.

Net loss attributable to common stockholders for the three months ended December 31, 2019 was $8.2 million, or $(0.14) for basic and diluted loss per share, compared to net income attributable to common stockholders of $0.8 million, or $0.01 for basic and diluted income per share, for the same period in 2018. Net loss attributable to common stockholders for the year ended December 31, 2019 was $40.0 million, or $(0.69) for basic and diluted loss per share, compared to net loss attributable to common stockholders of $29.4 million, or $(0.52) for basic and diluted loss per share, for the same period in 2018.

On March 12, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for 2019 (Press release, Y-mAbs Therapeutics, MAR 12, 2020, View Source [SID1234555485]).

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"We are very pleased with our 2019 results, highlighted by notable progress in the preparation of our BLAs for naxitamab and omburtamab, as well as commercial ramp-up for the potential launch of both compounds. We start 2020 with $207 million in cash, and are excited to enter what we believe will be a truly transformational year for Y-mAbs," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "During 2019, we have worked hard to make sure that naxitamab and omburtamab advance towards BLA submissions. We submitted the first portion of the rolling BLA for naxitamab in November 2019, and expect to complete the submission later this month. For omburtamab, we expect the rolling BLA submission to be completed in May."

Fourth Quarter 2019 and Recent Corporate Developments

·After the close of the fourth quarter, on February 26, 2020, Y-mAbs announced a positive Pre-BLA meeting with FDA for omburtamab.

·On December 12, 2019, Y-mAbs announced that its GD2-GD3 Vaccine has been granted a Rare Pediatric Disease Designation by the FDA for the treatment of neuroblastoma.

·On December 11, 2019, Y-mAbs announced positive frontline data for naxitamab at the Company’s R&D event, which took place in New York City. Key opinion leaders discussed the current treatment landscape and unmet medical needs for high-risk neuroblastoma and other solid tumors.

·On December 5, 2019, the Company announced that the European Medicines Agency agreed to the Company’s proposed Pediatric Investigation Plan for omburtamab.

·On November 29, 2019, Y-mAbs announced that it had submitted to the FDA the first portion of its Biologics License Application for naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma under the FDA’s Rolling Review process.

·On November 15, 2019, Y-mAbs announced a clinical update on omburtamab for Desmoplastic Small Round Cell Tumor. The data was presented at the 2019 CTOS Annual Meeting in Tokyo, Japan.

·On November 1, 2019, Y-mAbs announced the pricing of a follow-on shelf public offering, resulting in gross proceeds to the Company of approximately $143.8 million.

·On October 28, 2019, Y-mAbs announced an update on omburtamab data, which was presented at the International Society of Pediatric Oncology conference.

·On October 25, 2019, Y-mAbs announced a naxitamab update, which was presented at the International Society of Pediatric Oncology conference.

Financial Results

Y-mAbs reported a net loss for the year ended December 31, 2019 of $81.0 million, or $2.30 per basic and diluted share, compared to a net loss of $43.3 million, or $1.50 per basic and diluted share, reported for the year ended December 31, 2018.

Operating Expenses

Research and Development

Research and development expenses were $63.5 million for the twelve months ended December 31, 2019, compared to $34.3 million for the twelve months ended December 31, 2018, an increase of $29.2 million. The increase in research and development expenses primarily reflects the following:

·$17.1 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;
·$5.4 million increase in outsourced research and supplies to support expanding development activities;
·$3.3 million increase in personnel costs; and
·$2.5 million increase in clinical trials expenses.

General and Administration

General and administrative expenses were $19.5 million for the twelve months ended December 31, 2019, compared to $9.0 million for the twelve months ended December 31, 2018, an increase of $10.5 million. Such increase in general and administrative expenses primarily reflects the following:

·$5.1 million increase in personnel costs; and
·$2.8 million increase in commercial infrastructure costs;

Cash and Cash Equivalents

The Company had approximately $207.1 million in cash and cash equivalents as of December 31, 2019, compared to $147.8 million as of December 31, 2018. The increase of $59.3 million was primarily attributable to the $134.7 million net proceeds from the secondary public offering, completed in November 2019, which was partially offset by the net loss of $81.0 million for the fiscal year 2019 due to the increased costs of operation as the Company prepares for its submission of rolling BLAs for naxitamab and omburtamab, build-up of the Company’s commercial infrastructure, and increased personnel costs.

Webcast and Conference Call

The Company will host a conference call today at 4:30 pm eastern time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the access code 13699294. A webcast will be available at: View Source

On March 12, 2020 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the full year ended December 31, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 12, 2020, View Source [SID1234555484]).

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"The year is off to a strong start with the initiation of the Phase 1 trial of SRK-181 in immuno-oncology and the completion of enrollment in the SRK-015 TOPAZ Phase 2 trial in SMA," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "We now have two product candidates in the clinic with data expected from both programs later this year that will offer initial insights on their potential to treat patients with SMA and solid tumors, respectively. Scholar Rock is well-positioned to continue to execute and build momentum across its portfolio of product candidates."

Company Highlights and Upcoming Milestones

SRK-015 Program for Spinal Muscular Atrophy (SMA):

Published Structural Insights into the Mechanism by which SRK-015 Targets the Precursor or Latent Form of the Myostatin Growth Factor. In February 2020, the Journal of Biological Chemistry(1) published "Structural basis for specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015" detailing the integrated structural and biochemical approaches used to elucidate the unique molecular mechanism of SRK-015, a highly specific inhibitor of the activation of latent myostatin. The publication offers the first structural insights into the approach of targeting precursor forms to modulate growth factor activation. This approach could be applied to the discovery of specific modulators across members of the TGFβ superfamily or, even more broadly, to other signaling proteins that require precursor processing for activation.

On Track to Report Interim Safety and Efficacy Data from TOPAZ Phase 2 Trial in Mid-2020. In January 2020, Scholar Rock announced the completion of enrollment in the TOPAZ proof-of-concept trial for SRK-015 with a total of 58 patients with Type 2 or Type 3 SMA enrolled across 19 sites in the U.S. and Europe. Scholar Rock plans to report interim safety and efficacy results for all patients enrolled across the three cohorts of the trial following six months of treatment exposure in mid-2020. Top-line data for the full 12-month treatment period are expected beginning in the fourth quarter of 2020 and into the first quarter of 2021.

Presented Preliminary Data Highlighting Patient Demographics and Baseline Characteristics from the TOPAZ Trial at SMA Europe 2nd International Scientific Congress. In February 2020, Scholar Rock presented two posters at the SMA Europe 2nd International Scientific Congress highlighting key preclinical and Phase 1 data, in addition to preliminary pharmacokinetic (PK)/pharmacodynamic (PD) and baseline characteristics from the TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA. Preliminary demographic and baseline characteristic data for patients in the TOPAZ trial are in-line with previously published studies in SMA, including the evaluation of patients treated with nusinersen.

Announced Preliminary Pharmacokinetic and Pharmacodynamic Results from TOPAZ Trial. In November 2019, Scholar Rock announced results from a planned preliminary PK/PD analysis of 29 patients across all three cohorts of the TOPAZ trial. The analysis showed dose-proportional drug exposure and dose-dependent increases of up to 100-fold in serum latent myostatin levels following treatment with SRK-015. These biomarker results confirmed the presence of latent myostatin in patients with SMA and provides the first evidence of successful pharmacologic engagement of a latent growth factor in a human disease setting.

Identification of Second Indication for SRK-015 Planned for 2020. Scholar Rock continues to evaluate multiple potential opportunities beyond SMA, for which the selective inhibition of the activation of myostatin with SRK-015 may offer therapeutic benefit.
SRK-181 Program for Immuno-Oncology:

Initial Clinical Data from SRK-181 Phase 1 Dose Escalation and Dose Expansion Trial in Solid Tumors Expected in the Second Half of 2020. In the first quarter of 2020, Scholar Rock initiated a Phase 1 proof-of-concept trial for SRK-181 in patients with locally advanced or metastatic solid tumors. The two-part trial will consist of a dose escalation portion (Part A) for SRK-181 as both a single-agent and in combination with an approved anti-PD-(L)1 antibody, followed by a dose expansion portion (Part B) evaluating SRK-181 in combination with an approved anti-PD-(L)1 antibody in multiple tumor-specific cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Key objectives of the study include evaluating the safety and pharmacokinetics of SRK-181 and the efficacy of SRK-181 in combination with anti-PD-(L)1 therapy in the treatment of solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Initial clinical results, including biomarker data, from Part A of the Phase 1 trial are expected in the second half of 2020 with clinical response and safety data from Part B anticipated throughout 2021.
RGMc Program for Iron-Restricted Anemias:

Nomination of a Product Candidate from the RGMc Program Planned in 2020. Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody as its third product candidate in 2020. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Corporate Highlight:

Achieved First Milestone of $25 Million in Strategic Fibrosis Collaboration with Gilead Sciences. In December 2019, Scholar Rock achieved a $25 million preclinical milestone under the strategic fibrosis-focused collaboration with Gilead Sciences, Inc. for the successful demonstration of efficacy in preclinical in vivo proof-of-concept studies. This initial milestone advances the collaboration to develop potent and selective inhibitors of latent TGFβ activation for the potential treatment of patients suffering from fibrotic diseases.
Full Year 2019 Financial Results

For the year ended December 31, 2019, net loss was $51.0 million or $1.85 per share compared to a net loss of $49.3 million or $3.15 per share for the year ended December 31, 2018.

Revenue was $20.5 million for the year ended December 31, 2019 and was related to the Gilead Collaboration Agreement that was executed in December 2018.

Research and development expense was $54.2 million for the year ended December 31, 2019 compared to $36.3 million for the year ended December 31, 2018. The increase year-over-year primarily reflects preclinical and manufacturing costs for SRK-181 and higher personnel-related costs, slightly offset by lower manufacturing costs for SRK-015 and early development costs.

General and administrative expense was $20.8 million for the year ended December 31, 2019 compared to $14.4 million for the year ended December 31, 2018. The increase year-over-year was primarily attributable to increased headcount and professional services.
As of December 31, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $157.4 million, compared to $175.6 million as of December 31, 2018. A $25 million payment was received in January 2020 from Gilead for the achievement of the preclinical milestone under the strategic fibrosis-focused collaboration.

Dagbay, K., Treece, E., Streich Jr, F., Carven, G., et al. Structural basis for specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015, Journal of Biological Chemistry, View Source

On March 12, 2020 Champions Oncology, Inc. (Nasdaq: CSBR), engaged in an end-to-end range of research and development technology solutions and services to improve the development and use of oncology drugs, reported its financial results for the third fiscal quarter ended January 31, 2020 (Press release, Champions Oncology, MAR 12, 2020, View Source [SID1234555483]).

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Third Quarter Highlights:

Record quarterly revenue of $9.0 million, an increase of 40% year-over-year

Reported income of $881,000 excluding stock-based compensation and depreciation

Achieved record quarterly bookings

Ronnie Morris, CEO of Champions, commented, "Our third quarter revenue results were record breaking, exceeding $9 million for the first time. We continued to see bookings strength, aided by an increase in end point analysis testing in our studies. The pipeline of opportunities in GCLP flow cytometry services continued to expand and we are confident GCLP flow will contribute to future revenue growth."

David Miller, CFO of Champions added, "As anticipated, we started the second half of the year with very strong results with near record income on both a GAAP and Non-GAAP basis and we anticipate remaining profitable for the remainder of the year."

Third Fiscal Quarter Financial Results

For the third quarter of fiscal 2020, revenue increased 40.6% to $9.0 million compared to $6.4 million for the third quarter of fiscal 2019. The increase in revenue is due to increased sales, both in number and size of studies. Additionally, customers are seeking more complex study designs and end point analysis testing, leading to larger contracts, contributing to the revenue growth. Total costs and operating expenses for the third quarter of fiscal 2020 were $8.6 million compared to $6.8 million for the third quarter of fiscal 2019, an increase of $1.8 million or 26.5%.

Exhibit 99.1

For the third quarter of fiscal 2020, Champions reported income from operations of $433,000, including $229,000 in stock-based compensation and $219,000 in depreciation expenses, an increase of $803,000 compared to the loss from operations of $370,000, inclusive of $335,000 in stock-based compensation and $164,000 in depreciation expenses, in the third quarter of fiscal 2019. Excluding stock-based compensation and depreciation expenses, Champions reported non-GAAP income from operations of $881,000 for the third quarter of fiscal 2020 compared to non-GAAP income from operations of $129,000 in the third quarter of fiscal 2019, an increase of $752,000.

Cost of oncology solutions was $4.3 million for the three-months ended January 31, 2020, an increase of $900,000, or 26.5% compared to $3.4 million for the three-months ended January 31, 2019. For the three- months ended January 31, 2020, gross margin was 52.0% compared to 46.7% for the three-months ended January 31, 2019. Gross margin varies based on timing differences between expense and revenue recognition. The increase in cost of oncology services for the three-month period was mainly due to an increase in compensation and lab supply expenses. The overall expense increase is generally in line with the expected contribution based on the growth in revenue and study volume.

Research and development expense was $1.4 million and $1.3 million for the three-months ended January 31, 2020 and January 31, 2019, respectively. We continued to invest in research and development to offer new PDx models, ex-vivo assays and to expand our endpoint analysis testing. Sales and marketing expense for the three-months ended January 31, 2020 was $1.3 million, an increase of $428,000, or 48.7%, compared to $879,000 for the three-months ended January 31, 2019. The increase was primarily due to compensation expense driven by the continued expansion of the sales force and commissions earned. General and administrative expense was $1.6 million for the three-months ended January 31, 2020 compared to $1.2 million for the three-months ended January 31, 2019, an increase of $400,000 or 33.3%. The increase was mainly due to an increase in compensation expense and a non-recurring bad debt write-off.

Net cash used in operating activities was $41,000. The quarter’s use of cash from operations was primarily due to ordinary course of business timing differences in working capital items for the third quarter of 2020, including an increase in accounts receivable, of $759,000 and a decrease in accounts payable of $893,000.

The Company ended the quarter with $3.3 million of cash. Additionally, subsequent to the quarter end, approximately 700,000 warrants were cash exercised, adding an additional $4 million to the balance sheet. The Company has no debt.

Year-to-Date Financial Results

For the first nine months of fiscal 2020, revenue increased 20.8% to $23.4 million, as compared to $19.3 million for the first nine months of fiscal 2019. For the first nine months of fiscal 2020, total operating expenses increased 22.7% to $23.3 million, as compared to $19.0 million for the first nine months of fiscal 2019. The increase in revenue is due to increased sales, both in number and size of studies, and expanding our customer base.

For the first nine months of fiscal 2020, Champions reported income from operations of $109,000, which includes $437,000 in stock-based compensation and $579,000 in depreciation expenses, a decrease of $277,000 or 71.8%, compared to income from operations of $386,000, inclusive of $498,000 in stock-based compensation and $433,000 depreciation expenses, for the first nine months of fiscal 2019. Excluding stock-based compensation and depreciation, Champions reported non-GAAP operating income of $1.1

Exhibit 99.1

million for the first nine months of fiscal 2020 compared to income of $1.3 million for the same period last year.

Cost of oncology solutions was $12.0 million for the first nine months of fiscal 2020 compared to $10.0 million for the first nine months of fiscal 2019 an increase of $2.0 million or 20.0%. Gross margin was 48.8% for the first nine months of fiscal 2020, consistent with 48.5% for the first nine months of fiscal 2019. The increase in cost of oncology services for the nine-month period was mainly due to an increase in compensation and supply expenses.

Research and development expense was $4.0 million for the first nine months of fiscal 2020 an increase of $485,000, or 13.7% compared to $3.6 million for the first nine months of fiscal 2019. The increase is due to increased compensation and lab supply expenses related to new model development and product expansion. Sales and marketing expense for the first nine months of fiscal 2020 was $3.2 million, an increase of $1.0 million, or 47.6% compared to $2.1 million for the first nine months of fiscal 2019. The increase is the result of the continued expansion of the sales force. General and administrative expense was $4.1 million for the first nine months of fiscal 2020, an increase of $807,000 or 24.4% compared to $3.3 million for the first nine months of fiscal 2019. The increase for the nine-month period was mainly due to an increase in compensation, professional fees, and depreciation expenses offset by a reduction in recruiting costs.

Net cash provided by operations was $360,000 for the first nine months of fiscal 2020 compared to net
cash provided by operations of $1.6 million in 2019, a decrease of $1.3 million or 81.3%. The decrease in net cash provided by operations is primarily due to a decrease in working capital items for the third quarter of 2020 including an increase in accounts receivable and a decrease in accounts payable.

Conference Call Information:

The Company will host a conference call today at 4:30 p.m. EST (1:30 p.m. PST) to discuss its third quarter financial results. To participate in the call, please call 877-407-8035 (domestic) or 201-689-8035 (international) ten minutes ahead of the call and give the verbal reference "Champions Oncology."

On March 12, 2020 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing novel small molecule therapeutics to treat unmet needs in autoimmunity and cancer, reported its fourth quarter and year end 2019 financial results and corporate highlights (Press release, Kezar Life Sciences, MAR 12, 2020, View Source [SID1234555482]).

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"2019 marked several key milestones for Kezar, and I’m grateful for our team’s skill and hard work moving both of our programs forward. Our selective immunoproteasome inhibitor, KZR-616, entered Phase 2 trials in five different autoimmune diseases of high unmet need, building upon encouraging data from our ongoing Phase 1b study. We also nominated KZR-261, the first clinical candidate from our protein secretion program, and expect an IND filing in early 2021", said John Fowler, Chief Executive Officer. "This year we look forward to sharing updates from the Phase 1b portion of our MISSION study, as well as an interim analysis from our MARINA trial in AIHA and ITP. Finally, after our successful financing last month, we have a strong cash position that takes us beyond all of our KZR-616 Phase 2 readouts as well as data with KZR-261 in multiple tumor types."

Recent Clinical and Business Highlights

KZR-616 – Selective Immunoproteasome Inhibitor

MISSION Study

The Phase 1b/2 MISSION study in systemic lupus erythematosus (SLE) patients with and without nephritis is currently ongoing.

Additional data from the Phase 1b portion of MISSION will be presented during various medical conferences throughout the course of 2020.

The Phase 2 portion of MISSION is a randomized, placebo-controlled, double-blind trial to evaluate the safety and efficacy of KZR-616 in patients with active proliferative lupus nephritis. The primary endpoints of this portion of the MISSION trial are safety and tolerability. Secondary and exploratory endpoints include pharmacokinetics (PK), pharmacodynamics (PD), biomarker assessments and additional measures of efficacy. This trial includes four treatment arms evaluating KZR-616 administered subcutaneously once weekly for 24 weeks at dose levels of 30 mg, 45 mg and 60 mg, compared to placebo. The trial is designed to enroll up to 64 patients.

MARINA Study

The Phase 2 MARINA study in patients with autoimmune hemolytic anemia (AIHA) or immune thrombocytopenia (ITP) is currently ongoing.

MARINA is a Phase 2 randomized, dose-blind, multicenter trial designed to evaluate the safety, tolerability, efficacy, PK and PD of KZR-616 in patients with active AIHA or ITP. Patients with AIHA or ITP will be randomized to receive either 30 mg or 45 mg of KZR-616 subcutaneously once weekly for 13 weeks, followed by 12 weeks of follow-up. The trial is designed to enroll up to 40 patients. Kezar believes that whole blood RNASeq data from the Phase 1b portion of MISSION, which showed a decrease in multiple inflammatory gene signature and prolonged increase in erythropoietic gene signatures in SLE patients, support the broad potential anti-inflammatory activity of KZR-616 and therapeutic potential in patients with AIHA.

PRESIDIO Study

The Phase 2 PRESIDIO study in patients with polymyositis (PM) or dermatomyositis (DM) is currently ongoing.

PRESIDIO is a Phase 2 randomized, placebo-controlled, double-blind, crossover, multicenter trial to evaluate the safety, tolerability, efficacy, PK and PD of KZR-616 in patients with active PM or DM. During the 32-week treatment period, patients receive either 45 mg of KZR-616 or placebo subcutaneously once weekly for 16 weeks followed by a crossover to the other treatment arm for an additional 16 weeks. The trial is designed to enroll up to 24 patients. Kezar believes that KZR-616 has the potential to be developed into a treatment for patients with DM and PM, which is in-part supported by preclinical data in a mouse model of PM and DM that demonstrated immunoproteasome inhibition and improved muscle function.

KZR-261 – Protein Secretion Program

Research and discovery efforts targeting the protein secretion pathway have progressed significantly, and this novel program and pathway was featured in four separate presentations during two major medical and scientific conferences in 2019: the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) and the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting & Exposition (ASH) (Free ASH Whitepaper).

KZR-261, our first oncology clinical candidate and a first-in-class protein secretion inhibitor, has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies. Kezar anticipates filing an Investigational New Drug (IND) application for a Phase 1 study of KZR-261 in solid tumors in the first quarter of 2021.

Business Update

On February 4, 2020, Kezar completed an underwritten public offering of 18,965,385 shares of its common stock, which includes the full exercise of the underwriters’ option, and pre-funded warrants to purchase 2,884,615 shares of its common stock at an exercise price of $0.001 per share. The public offering price of the common stock was $2.60 per share and the public offering price of each pre-funded warrant was $2.599 per underlying share. The gross proceeds from the public offering were approximately $56.8 million, before deducting underwriting discounts and offering expenses.

Financial Results

Cash, cash equivalents and marketable securities totaled $78.2 million as of December 31, 2019, compared to $107.4 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities was primarily attributable to cash used by the company in operations to advance its clinical stage programs as well as preclinical research and development.

Research and development expenses for the fourth quarter of 2019 increased by $2.7 million to $7.4 million compared to $4.7 million in the fourth quarter of 2018. Full year R&D expenses increased by $9.2 million in 2019 compared to 2018. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.

General and administrative expenses for the fourth quarter of 2019 increased by $0.8 million to $2.6 million compared to $1.8 million in the fourth quarter of 2018. Full year G&A expenses increased by $3.4 million in 2019 compared to 2018. The increase was primarily due to an increase in personnel expenses and costs related to operating as a public company.

Net loss for the fourth quarter of 2019 was $9.6 million, or $0.50 per basic and diluted common share, compared to a net loss of $5.8 million, or $0.30 per basic and diluted common share, for the fourth quarter of 2018. Net loss for 2019 was $35.1 million, or $1.84 per basic and diluted common share, compared to $23.2 million, or $2.26 per basic and diluted common share, in 2018.

Total shares outstanding were 19.2 million as of December 31, 2019. Additionally, there were outstanding options to purchase 3.1 million shares of common stock at a weighted average exercise price of $7.19 per share as of December 31, 2019.

About KZR-616

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. Phase 1a clinical trial results in healthy volunteers provide evidence that KZR-616 potentially avoids adverse effects caused by currently marketed non-selective proteasome inhibitors, which Kezar believes prevent them from being utilized as a chronic treatment in autoimmune disorders. Phase 2 trials are underway for the treatment of lupus nephritis (MISSION study), autoimmune hemolytic anemia and immune thrombocytopenia (MARINA study), and dermatomyositis and polymyositis (PRESIDIO study).

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathways. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered at Kezar through a robust medicinal chemistry campaign in which several

scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. IND-enabling activities are currently underway, and an IND application in solid tumors is expected to be filed in the first quarter of 2021.