On November 6, 2019 MannKind Corporation (NASDAQ:MNKD) reported preliminary financial results for the quarter and nine months ended September 30, 2019 (Press release, Mannkind, NOV 6, 2019, View Source [SID1234550479]).

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"I am excited to see MannKind continue its transformation, with another quarter of double-digit growth in Afrezza revenue, year over year, and aggregate net revenue of $14.6 million," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "In the third quarter, we completed our recapitalization and achieved several key milestones, such as booking our first international sale of Afrezza to Brazil, seeing the first PAH patient dosed with TreT and progressing two pipeline compounds into nonclinical testing."

Third Quarter 2019 Results

Net revenues were $14.6 million for the third quarter of 2019, reflecting Afrezza net revenue of $6.4 million and collaborations and services revenue of $8.2 million. Afrezza net revenue increased 46% compared to $4.4 million in the third quarter of 2018, primarily driven by higher product demand (including the first shipment to Brazil), a more favorable mix of Afrezza cartridges and price.

Collaborations and services revenue increased $8.1 million compared to the third quarter of 2018, primarily driven by the license agreement with United Therapeutics, which began in the fourth quarter of 2018.

On a GAAP basis, Afrezza gross loss was $0.7 million for the third quarter of 2019 compared to a gross loss of $0.9 million in the same period in 2018. Afrezza cost of goods sold for the third quarter of 2019 included a one-time fee of $2.75 million recorded in connection with the amendment of the Company’s insulin supply agreement with Amphastar in August 2019. As a result, on a non-GAAP basis, gross profit was $2.1 million or 33% for the third quarter of 2019.

Research and development (R&D) expenses for the third quarter of 2019 were $1.6 million compared to $2.0 million for the third quarter of 2018. This 23% decrease was primarily attributable to a $0.4 million decrease in clinical trial spending.

Selling, general and administrative (SG&A) expenses for the third quarter of 2019 were $16.7 million compared to $19.4 million for the third quarter of 2018. This decrease of $2.7 million, or 14%, was primarily attributable to a $0.8 million decrease in personnel-related costs and a $1.9 million decrease in Afrezza marketing costs.

Interest expense on notes for the third quarter of 2019 was $4.1 million compared to $1.0 million for the third quarter of 2018. This $3.1 million increase or 316% was primarily attributable to a $3.4 million charge realized as a result of achieving of a sales milestone in the third quarter of 2019 under the Company’s milestone agreement with Deerfield.

The net loss for the third quarter of 2019 was $10.4 million, or $0.05 per share compared to a $24.2 million net loss in the third quarter of 2018 or $0.16 per share. The decrease was primarily the result of increased total revenues from higher Afrezza commercial demand and from the licensing and research agreements with United Therapeutics.

Nine Months Ended September 30, 2019

Net revenues were $47.0 million for the nine months ended September 30, 2019, reflecting Afrezza net revenue of $17.5 million and collaborations and services revenue of $29.5 million. Afrezza net revenue increased 52% compared to $11.5 million for the nine months ended September 30, 2018, primarily due to higher product demand (including the first shipment to Brazil), a more favorable mix of Afrezza cartridges and price. Collaborations and services revenue increased $29.3 million compared to the nine months ended September 30, 2018, which was primarily attributed to the licensing agreement ($23.3 million) and research agreement ($5.9 million) with United Therapeutics, both of which began in the fourth quarter of 2018.

On a GAAP basis, Afrezza gross profit was $2.1 million for the nine months ended September 30, 2019, an improvement of $5.0 million or 173% compared to a gross loss of $2.9 million in the same period in 2018, primarily due to an increase of $6.0 million in net revenue, a $1.8 million decrease in inventory write-offs, partially offset by increased costs due to higher sales and the Amphastar one-time amendment fee of $2.75 million in the third quarter of 2019. As a result, on a non-GAAP basis, gross profit was $4.9 million or 28% for the nine months ended September 30, 2019.

R&D expenses for the nine months ended September 30, 2019 were $4.9 million compared to $7.7 million for the nine months ended September 30, 2018. This $2.8 million or 36% decrease was primarily attributable to a $1.0 million decrease in personnel-related costs and a $1.1 million decrease in clinical trial spending.

SG&A expenses for the nine months ended September 30, 2019 were $58.9 million compared to $61.7 million for the nine months ended September 30, 2018. This decrease of $2.7 million or 5% was primarily attributable to a $6.1 million decrease in personnel related costs, $1.9 million decrease in professional fees, a $1.0 million decrease in stock-based compensation costs offset by a $6.8 million increase in costs for a television campaign for Afrezza.

Interest income increased by $0.5 million or 160% for the nine months ended September 30, 2019 primarily attributable to a higher balance on money market funds and other short-term investments.

Interest expense on notes for the nine months ended September 30, 2019 was $5.3 million compared to $4.5 million for the nine months ended September 30, 2018. This $0.8 million increase was primarily attributable to a $3.4 million charge realized as a result of achieving of a sales milestone in the third quarter of 2019 under the Company’s milestone agreement with Deerfield, partially offset by a reduction in debt principal balances.

The net loss for the nine months ended September 30, 2019 was $37.6 million, or $0.20 per share compared to a $77.2 million net loss for the nine months ended September 30, 2018 or $0.56 per share. The lower net loss was mainly attributable to a $35.2 million increase in total revenues.

Cash, Cash Equivalents, Restricted Cash and Short Term Investments

Cash, cash equivalents, restricted cash, and short-term investments at September 30, 2019 was $50.4 million compared to $71.7 million at December 31, 2018.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. To participate in the live call by telephone, please dial (866) 548-4713 or (323) 794-2093 and use the participant passcode: 8987532. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 8987532. A replay will also be available on MannKind’s website for 14 days.

On November 6, 2019 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported that new data from across its pipeline will be presented at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release, Magenta Therapeutics, NOV 6, 2019, View Source [SID1234550478]).

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"The breadth of new results we are presenting at ASH (Free ASH Whitepaper) illustrates our progress toward enabling patients with autoimmune diseases, blood cancers and genetic diseases to benefit from curative stem cell transplant. In today’s abstracts we are particularly excited to share the first clinical data from our MGTA-145 program, which we are developing as a first-line stem cell mobilization drug. The data in the abstract show that MGTA-145 in combination with plerixafor was well-tolerated and successfully mobilized the target stem cell dose in 11 of 12 subjects within hours of administration. At ASH (Free ASH Whitepaper) we anticipate sharing initial data from mobilization and collection of the target number of cells in subjects in a single-day procedure for the first time," said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. "We are also pleased to show groundbreaking data from our most advanced patient preparation program, CD117-ADC, in today’s abstract. These data, which will be shared in an oral presentation at ASH (Free ASH Whitepaper) by Dr. John Tisdale of the National Institutes of Health, show the first-ever successful transplant in non-human primates using a targeted, single-agent antibody-drug conjugate, without the use of chemotherapy or radiation. These data provide additional validation for both our CD117-ADC program as well as our entire ADC-based patient preparation portfolio."

First Data from MGTA-145 Clinical Program

Title: Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of MGTA-145 Alone and in Combination with Plerixafor (Abstract #1961)
Presenter: John DiPersio, M.D., Ph.D., Professor of Medicine and Chief of the Oncology Division, Washington University School of Medicine, St. Louis, Missouri
Date and Time: Saturday, December 7th, 5:30 to 7:30 p.m.

Mobilized peripheral blood is used for the majority of the 65,000 stem cell transplants performed each year across the United States and Europe, but the current standard of care, G-CSF, requires at least five days of dosing and is associated with significant side effects, including bone pain that often requires narcotics. Further, patients with autoimmune diseases or sickle cell disease can have severe side effects with G-CSF, including potentially fatal complications.

Magenta is developing MGTA-145 as the new first-line standard of care for stem cell mobilization in a broad range of diseases, including autoimmune diseases and genetic diseases. MGTA-145 works in combination with plerixafor to harness the physiological mechanism of stem cell mobilization. The MGTA-145-based combination has the potential to reliably mobilize robust numbers of high-quality stem cells with single-day dosing and collection. Importantly, the MGTA-145-based combination also avoids the need for G-CSF.

The ASH (Free ASH Whitepaper) abstract includes early results from the Phase 1 study, as of the late July data cut-off. MGTA-145 was safe and well-tolerated as a single agent and in combination with plerixafor, and showed the expected target pharmacology. Eleven of the 12 subjects who received the combination of MGTA-145 and plerixafor mobilized more than 20 CD34+ cells/microliter, the clinically accepted threshold for successful mobilization. This number is considered predictive of collection of at least 2 million CD34+ stem cells/kg through apheresis, the threshold for cell dose for a successful transplant. Magenta anticipates that initial data from additional subjects and apheresis collection results will be presented at ASH (Free ASH Whitepaper).

Data from CD117-ADC Patient Preparation Program

Current methods to condition patients before transplant and gene therapy are dependent on toxic, non-specific chemotherapy or radiation. These pre-transplant treatments are associated with significant side effects, including infertility, cancer, organ damage, and death. Magenta is the only company developing targeted antibody-drug conjugates (ADCs) designed to precisely remove the disease-causing cells in the body, without the need for chemotherapy or radiation.

Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)
Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.
Date and Time: Monday, December 9th, 7:45 a.m.

Magenta’s most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.

Data in the ASH (Free ASH Whitepaper) abstract showed that a single dose of CD117-ADC enabled successful transplant of gene-modified stem cells for gene therapy in non-human primates without the need for chemotherapy or radiation. CD117-ADC removed stem cells in the non-human primates, with a favorable safety profile. The ADC spared the immune system and was cleared rapidly as designed. In a rhesus model of gene therapy, a single dose of the ADC enabled engraftment of stem cells modified with the β-globin gene, the gene that causes sickle cell disease and β-thalassemia when mutated. These proof-of-concept studies validate the use of CD117-ADC for targeted stem cell depletion prior to transplant and support its use as a new conditioning agent for gene therapy and stem cell transplant without toxic chemotherapy or radiation.

Additional Posters and Presentations

Title: A Novel Targeted Approach to Achieve Immune System Reset: Magenta’s CD45-Targeted Antibody-Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS patients, and Achieve Immune Depletion in Non-human Primates (NHP) (Abstract #3208)
Presenter: Geoff Gillard, Ph.D., Magenta Therapeutics, Cambridge, Mass.
Date and Time: Sunday, December 8th, 6:00 to 8:00 p.m.

Title: MGTA-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-vs-Host Disease (GVHD) (Abstract #804)
Presenter: John Wagner, M.D., Executive Medical Director, Bone Marrow Transplant Program, University of Minnesota, Minneapolis, Minn.
Date and Time: Monday, December 9th, 4:00 p.m.

Title: High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome (Abstract #4424)
Presenter: Kevin Goncalves, Ph.D., Magenta Therapeutics, Cambridge, Mass.
Date and Time: Monday, December 9th, 6:00 p.m. to 8:00 p.m.

Title: Ex Vivo Hematopoietic Stem Cell Expansion with E478 Increases the Rate of CRISPR- mediated Homology Directed Repair of the Beta Globin Gene in Human Stem Cells by >10-fold and Improves In Vivo Engraftment by > 120-fold (Abstract #4634)
Presenter: Kevin Goncalves, Ph.D., Magenta Therapeutics, Cambridge, Mass.
Date and Time: Monday, December 9th, 6:00 p.m. to 8:00 p.m.

ASH Investor Event and Webcast to Review ASH (Free ASH Whitepaper) Data and Updated MGTA-456 Phase 2 Clinical Data

Magenta will host a live webcast of an investor and analyst event at 8:30 p.m. ET on Saturday, December 7th, during the ASH (Free ASH Whitepaper) Annual Meeting. During this event, Magenta will review data presented at the ASH (Free ASH Whitepaper) meeting and present updated data from the ongoing Phase 2 clinical trial of MGTA-456 in inherited metabolic disorders.

To access the webcast, please visit the "News & Events" page within the Investors & Media section of the Magenta website at www.magentatx.com. A replay of the webcast will be available on the Magenta website for 90 days following the call.

On November 6, 2019 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported financial results for the quarter ended September 30, 2019 (Press release, MacroGenics, NOV 6, 2019, View Source [SID1234550477]).

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"MacroGenics’ most advanced programs are positioned to initiate or complete registration-directed studies over the next year," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "We look forward to presenting detailed data from the second interim OS analysis of SOPHIA, the Phase 3 study comparing margetuximab to trastuzumab in patients with metastatic HER2-positive breast cancer at the San Antonio Breast Cancer Symposium in December. We believe that our molecule, if approved, will address an important unmet need for patients. We continue our activities in preparation for a BLA submission before the end of the year."

Advances in Key Portfolio Programs

Margetuximab: is an investigational, Fc-optimized monoclonal antibody (mAb) that targets human epidermal growth factor receptor 2 (HER2) being developed for metastatic breast and gastric cancer.

Breast Cancer: MacroGenics reported topline results from the second interim analysis of overall survival (OS) from SOPHIA, the Phase 3 clinical trial of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in patients with HER2-positive metastatic breast cancer. Detailed results are scheduled for an oral presentation at the San Antonio Breast Cancer Symposium (SABCS) on December 11, 2019. MacroGenics expects to submit a Biologics License Application (BLA) to the U.S. FDA before the end of 2019.

Gastric Cancer: MacroGenics recently initiated MAHOGANY, a Phase 2/3 registration-directed clinical trial of margetuximab in combination with either MGA012 (anti-PD-1 mAb) or MGD013 (bispecific PD-1 x LAG-3 DART molecule) in first-line patients with HER2-positive gastroesophageal adenocarcinoma (GEA). The MAHOGANY study is based on results from an ongoing Phase 2 study of margetuximab plus pembrolizumab, an anti-PD-1 mAb, for patients with advanced HER2-positive GEA who have previously been treated with chemotherapy and trastuzumab. These data were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress in September 2019.

Flotetuzumab: is an investigational, bispecific CD123 x CD3 DART molecule being evaluated in acute myeloid leukemia (AML). A Phase 1 monotherapy study enrolled 50 patients with relapsed or refractory AML at the recommended Phase 2 dose. Data from a subset of 30 patients with refractory AML are scheduled for an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 9, 2019. MacroGenics continues to develop flotetuzumab in this refractory patient population. The Company also recently initiated a clinical study with flotetuzumab in combination with MGA012 in relapsed or refractory AML.

Exhibit 99.1
MGA012 (INCMGA0012): is an investigational, anti-PD-1 mAb exclusively licensed to Incyte Corporation on a worldwide basis. Incyte is pursuing development of MGA012 monotherapy in three ongoing, potentially registration-directed trials with initial data expected in 2020. In addition, Incyte and MacroGenics are each conducting studies of MGA012 in combination with other product candidates.

MGD013: is an investigational, first-in-class bispecific PD-1 x LAG-3 DART molecule being evaluated in Phase 1 studies. MacroGenics has enrolled approximately 150 patients as part of a Phase 1 dose expansion study in nine tumor types. The Company expects to submit data from the study for presentation at a scientific conference in the first half of 2020.

Enoblituzumab: is an investigational, Fc-optimized mAb that targets B7-H3, an antigen broadly expressed across many solid tumors. MacroGenics plans to initiate a Phase 2/3 study of enoblituzumab in combination with MGA012 in patients with squamous cell carcinoma of the head and neck (SCCHN) before the end of 2019.

Third Quarter 2019 Financial Results
•Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2019, were $254.4 million, compared to $232.9 million as of December 31, 2018.
•Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $18.7 million for the quarter ended September 30, 2019, compared to $20.8 million for the quarter ended September 30, 2018.
•R&D Expenses: Research and development expenses were $44.9 million for the quarter ended September 30, 2019, compared to $46.2 million for the quarter ended September 30, 2018.
•G&A Expenses: General and administrative expenses were $11.8 million for the quarter ended September 30, 2019, compared to $9.6 million for the quarter ended September 30, 2018.
•Net Loss: Net loss was $44.6 million for the quarter ended September 30, 2019, which included $7.6 million in net unrealized losses recognized on equity securities held, compared to net loss of $34.0 million for the quarter ended September 30, 2018.
•Shares Outstanding: Shares outstanding as of September 30, 2019 were 48,914,284.

Conference Call Information
MacroGenics will host a conference call today at 4:30 p.m. ET to discuss financial results for the quarter ended September 30, 2019 and provide a corporate update. To participate in the conference call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 9298813.

The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On November 6, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that an abstract related to the Company’s lead drug candidate, tipifarnib, has been accepted for oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held from December 7-10, 2019 in Orlando (Press release, Kura Oncology, NOV 6, 2019, View Source [SID1234550476]). The following abstract was published today and is now available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

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Proof of Concept for Tipifarnib in Relapsed or Refractory Angioimmunoblastic T-Cell Lymphoma (AITL) and CXCL12+ Peripheral T-Cell Lymphoma (PTCL): Preliminary Results from an Open-Label, Phase 2 Study (Abstract # 468)
Session Name: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Novel Therapies in Peripheral T-cell Lymphomas
Session Date: Sunday, December 8, 2019
Session Time: 12:00 p.m. – 1:30 p.m. ET
Presentation Time: 1:15 pm ET
Room: Orange County Convention Center, Valencia D (W415D)

A copy of the presentation will be available on Kura’s website at www.kuraoncology.com following presentation at the meeting.

On November 6, 2019 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 third quarter 2019 financial results and corporate highlights (Press release, Kezar Life Sciences, NOV 6, 2019, View Source [SID1234550475]).

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"I commend our team’s continued execution across our clinical and preclinical programs here at Kezar. Our three Phase 2 trials with KZR-616 in severe autoimmune diseases are progressing on track, and we look forward to disclosing additional data from our MISSION study at the ACR meeting next week", said John Fowler, Chief Executive Officer. "Additionally, the nomination of the first clinical candidate from our protein secretion program is a pivotal step for the company and underscores the depth and breadth of our R&D capabilities. Our small molecule approach to targeting the Sec61 translocon represents a highly compelling new therapeutic approach, and the preclinical data generated with KZR-261 gives us confidence that it may prove effective in treating a variety of tumor types. We are excited to share more about this highly novel target and illustrate the broad platform potential of this approach."

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.

On October 1, 2019, we presented a corporate and strategic update, including a protocol amendment for the Phase 2 portion of the MISSION study (NCT03393013). The updated protocol is designed to generate more robust and meaningful data around the safety and efficacy of KZR-616 in lupus nephritis, including the evaluation of three dose levels of KZR-616 (administered with background medication) for a treatment period of six months.

The Phase 1b portion of the MISSION study is ongoing and additional data will be presented during a poster session at the 2019 American College of Rheumatology (ACR) Meeting in Atlanta, GA on November 12, 2019.

PRESIDIO Study

In August 2019, we announced the initiation of the PRESIDIO study (NCT04033926), a Phase 2 randomized, double-blind, placebo-controlled, crossover, multicenter study to evaluate the safety, tolerability, efficacy, PK and PD of treatment with KZR-616 in patients with active dermatomyositis (DM) or polymyositis (PM). This trial is expected to enroll 24 patients with either DM or PM.

MARINA Study

In August 2019, we announced the initiation of the MARINA study (NCT04039477), a Phase 2 randomized, dose-blind, multicenter study to evaluate the safety and efficacy of KZR-616 in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). This trial is expected to enroll 40 patients with either AIHA or ITP.

KZR-261 – Protein Secretion Program

Our research and discovery efforts targeting the protein secretion pathway have progressed significantly, and today, we announce the nomination of KZR-261 as our first oncology clinical candidate. KZR-261 has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies, and we have initiated laboratory studies and manufacturing activities towards an Investigational New Drug (IND) filing for a Phase 1 study in solid tumors, which we anticipate occurring in Q1 2021.

Additionally, this novel program and pathway will be featured in four separate presentations during two major medical and scientific conferences: 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). The abstract titles are summarized below.

SITC: November 6-10, 2019, National Harbor, MD

Abstract Number: 815

Title: Targeting multiple immune checkpoint proteins with novel small molecule inhibitors of Sec61-dependent cotranslational translocation

Date: Friday, November 8, 2019

Time: 12:30pm – 2:00pm

Poster Session: Novel Single-Agent Immunotherapies

ASH: December 7-10, 2019, Orlando, FL

Oral Presentations

Abstract Number: 408

Title: Blocking Protein Secretion with Novel Small Molecule Inhibitors of Sec61 Represents a Potential Treatment Strategy Against Hematologic Malignancies

Date: Sunday, December 8, 2019
Time: 10:45am

Oral Session: 802. Chemical Biology and Experimental Therapeutics: Novel Compounds and Mechanisms of Action

Abstract Number: 805
Title: Protein Translocation Inhibitors Overcome Cytokine-Induced Glucocorticoid Resistance in T-Cell Acute Lymphoblastic Leukemia

Date: Monday, December 9, 2019
Time: 4:30 PM

Oral Session: 605. Molecular Pharmacology, Drug Resistance—Lymphoid and Other

Poster Presentation

Abstract Number: 2076

Title: Proteomic Profiling and Mechanistic Investigating of a Novel Anti-Cancer Small Molecule Inhibitor of Sec61
Date: Saturday, December 7, 2019
Time: 5:30 PM – 7:30 PM

Poster Session: 802. Chemical Biology and Experimental Therapeutics: Poster I

Financial Results

Cash, cash equivalents and marketable securities totaled $85.2 million as of September 30, 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 third quarter of 2019 increased by $2.4 million to $7.1 million compared to $4.7 million in the third quarter of 2018. This increase was primarily related to advancing both the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.

General and administrative expenses for the third quarter of 2019 increased by $1.0 million to $2.6 million compared to $1.6 million in the third quarter of 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 third quarter of 2019 was $9.1 million, or $0.48 per basic and diluted common share, compared to a net loss of $5.7 million, or $0.30 per basic and diluted common share, for the third quarter of 2018.

Total shares outstanding were 19.1 million as of September 30, 2019. Additionally, there were outstanding options to purchase 3.3 million shares of common stock at a weighted average exercise price of $7.39 per share as of September 30, 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 we believe 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), dermatomyositis and polymyositis (PRESIDIO study), and autoimmune hemolytic anemia and immune thrombocytopenia (MARINA study).

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from our research and discovery efforts targeting protein secretion pathways as potential therapies for oncology, immuno-oncology and autoimmune indications. 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 medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary work flow of Sec61 modulation. As a result, Kezar has established a unique and broad library of protein secretion inhibitors and a strong patent position around KZR-261 and its analogs. KZR-261 has demonstrated several encouraging features that lend to its potential to be a new anti-cancer agent for the treatment of solid and hematologic malignancies. IND-enabling studies are currently underway, and an IND filing in solid tumors is expected in Q1 2021.