On August 12, 2021 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Aduro Biotech, AUG 12, 2021, View Source [SID1234586440]).

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"During the second quarter of 2021, we made strong progress across our pipeline of programs for kidney diseases, including driving enrollment in the phase 3 ALIGN and phase 2 AFFINITY trials of atrasentan, presenting interim clinical data for BION-1301 demonstrating initial proof-of-concept in patients with IgA nephropathy and advancing key IND-enabling studies for CHK-336," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "Our solid cash position and resourcing enable us to continue executing on key priorities to advance our pipeline and build Chinook into a leading kidney disease company."

Recent Highlights

Enrolled the first patient in the phase 2 AFFINITY basket trial of atrasentan, a highly potent and selective endothelin A receptor (ETA) antagonist (see www.clinicaltrials.gov, identifier NCT04573920).

Presented interim data from the first several patients with IgA nephropathy (IgAN) enrolled in Cohort 1 of Part 3 of the ongoing phase 1/2 study of BION-1301 at the 58th ERA-EDTA conference, demonstrating durable reductions in Gd-IgA1, IgA, IgM, and to a lesser extent, IgG levels, as well as clinically meaningful reductions in 24-hour proteinuria (UPCR), providing initial proof-of-concept for BION-1301 in IgAN.

Presented Gd-IgA1 biomarker data in healthy volunteers from Part 1 (single ascending dose) and Part 2 (multiple ascending dose) of the ongoing phase 1/2 study of BION-1301 at the ISN World Congress of Nephrology 2021, showing dose-dependent and durable reductions in Gd-IgA1 levels following administration of BION-1301.

Presented data from the BION-1301 phase 1 intravenous to subcutaneous bioavailability study in healthy volunteers at the ISN World Congress of Nephrology 2021.

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

Announced promotions of Tom Frohlich to Chief Operating Officer and Andrew King, D.V.M., Ph.D., to Chief Scientific Officer.
Anticipated Upcoming Catalysts

Chinook expects to complete enrollment in Cohort 1 of Part 3 of the ongoing phase 1/2 study of BION-1301 in the third quarter of 2021 and to present additional patient data from this cohort at the ASN Kidney Week 2021 in November.

Chinook expects to initiate enrollment in Cohort 2 of Part 3 of the ongoing phase 1/2 study of BION-1301 in the third quarter of 2021. Patients in Cohort 2 will receive a subcutaneous dose of 600 mg of BION-1301 every two weeks for up to 52 weeks. Chinook expects to present patient data from Cohort 2 and provide an update on planned later-stage clinical trials of BION-1301 in the first half of 2022.

Chinook expects to present interim data from one or more patient cohorts of the ongoing phase 2 AFFINITY basket trial of atrasentan in the first half of 2022.

CHK-336 is currently in IND-enabling studies and is advancing towards expected initiation of a phase 1 clinical study in healthy volunteers in the first quarter of 2022 for the treatment of primary hyperoxaluria.
Second Quarter 2021 Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $229.8 million at June 30, 2021, compared to $250.4 million at December 31, 2020.

Revenue – Total revenue increased by less than $0.1 million for the second quarter of 2021 and increased by $0.4 million for the six months ended June 30, 2021, compared to the same periods in 2020 due to revenue recognized related to research and development services provided under the collaboration agreement with Lilly, which was acquired under the merger with Aduro.

Expenses –

Research and development expenses were $22.8 million for the second quarter of 2021 and $48.5 million for the six months ended June 30, 2021, compared to $3.9 million and $6.7 million, respectively, for the same periods in 2020. The increase was primarily due to external clinical and manufacturing expenses related to the atrasentan and BION-1301 clinical programs; higher employee-related costs, including salaries, benefits and stock-based compensation expense associated with hiring staff to build out Chinook’s clinical and development capabilities; increased spending for consulting and outside services; and an increase in facilities and other costs. The six months ended June 30, 2021 also includes an upfront fee of $3.3 million due to Evotech International GmbH under a research collaboration and license agreement entered into in February 2021.

General and administrative expenses were $7.8 million for the second quarter of 2021 and $17.3 million for the six months ended June 30, 2021, compared to $3.9 million and $5.2 million, respectively, for the same periods in 2020. The increase was primarily due to higher employee-related costs, including salaries, benefits and stock-based compensation expense associated with the addition of administrative staff to buildout Chinook’s public-company infrastructure; higher legal, consulting and outside services costs; and an increase in facilities and other costs.

Expenses due to the change in fair value of contingent consideration and contingent value rights liabilities were $19.6 million for the second quarter of 2021 and $21.4 million for the six months ended June 30, 2021, compared to nil for the same periods in 2020. These non-cash expenses are due to the quarterly revaluation of assets and liabilities related to the Sairopa transaction and an updated valuation of Chinook’s CVR liability under the Merck collaboration, as a result of the merger with Aduro. In the second quarter of 2021, Merck informed Chinook that it intends to explore the potential benefit of the product candidate MK-5890, previously out-licensed to Merck by Aduro, in a phase 2 clinical study for a new indication. This may result in potential milestone and royalty payments for the benefit of CVR holders.

Other –

The sale of certain of Chinook’s non-renal assets to Sairopa in the second quarter of 2021 resulted in a $7.2 million gain.
Net Loss – Net loss for the second quarter of 2021 was $42.6 million or $0.97 per share and $79.8 million or $1.86 per share for the six months ended June 30, 2021, compared to net loss of $7.7 million or $1.87 per share and $12.9 million or $3.12 per share, respectively, for the same periods in 2020.
Cash Used in Operations – For the six months ended June 30, 2021, cash used in operations totaled $55.5 million.
(Press release, Aduro Biotech, AUG 12, 2021, View Source [SID1234586440])

On August 12, 2021 Applied DNA Sciences, Inc. (NASDAQ: APDN) (the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing and nucleic acid-based technologies, reported consolidated financial results for the three and nine months ended June 30, 2021 (Press release, Applied DNA Sciences, AUG 12, 2021, View Source [SID1234586439]).

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"We delivered excellent year-over-year revenue growth in the fiscal third quarter while laying the groundwork to secure a recently awarded COVID-19 testing services contract that has potential to be the largest contract in the Company’s history," said Dr. James A. Hayward, president and CEO of Applied DNA. "Demand for safeCircle, our pooled COVID-19 testing program, experienced a seasonal decline from the fiscal second quarter, reflecting the start of the summer recess months for our academic clients and progressively higher vaccination rates and lower positivity rates in our operating area. Our recent award from the City University of New York for large-scale turnkey COVID-19 testing services should continue to drive strong year-over-year revenue growth over the period of the contract."

Continued Dr. Hayward, "Our operating activities during the quarter were distinguished by an expansion of our COVID-19 offerings to drive incremental revenue and to drive adoption of LinearDNA as an alternative to plasmids for nucleic acid-based therapies. Following constructive interactions with the U.S. Food and Drug Administration (FDA) as part of a preliminary Emergency Use Authorization application process and the evolving nature of the pandemic, we revised our Linea SARS-CoV-2 Mutation (the "Linea Mutation Panel") (formerly SGS Mutation Panel) to target three SARS-CoV-2 mutations (E48K, L452R, N501Y) that have been designated substitutions (mutations) of therapeutic concern by the Centers for Disease Control and Prevention.

"Should the FDA grant an EUA for the Linea Mutation Panel, we believe that it will offer clinical utility to healthcare systems by enabling precision COVID-19 treatment and commercial utility to monoclonal antibody manufacturers by better characterizing patients before treatments. In recent months, several monoclonal antibody treatments have had their EUA revoked or have demonstrated a reduction in efficacy on a standalone or in combination with other treatments due to mutational impact. Use of the Linea Mutation Panel is tied to our Linea COVID-19 Assay Kit to determine positivity in clinical samples that would drive additional Assay demand if the EUA is granted for our Mutation Panel. We believe that an EUA-authorized Linea Mutation Panel will also provide additional value to our existing COVID-19 testing customers and, when combined with our Whole Genome Sequencing assets, provide data of interest to epidemiologists.

"Concurrently, the launch of our veterinary LinearDNA COVID-19 vaccine trial and the subsequently reported strong immune response that the vaccine candidate elicited, further reinforce the value proposition of LinearDNA, and, longer-term, generates invaluable preclinical data supporting the eventual application of LinearDNA to nucleic acid-based therapies in humans."

Concluded Dr. Hayward, "Looking ahead, the confluence of increasing positivity rates due to the Delta variant, the commingling of vaccinated, partially vaccinated, and unvaccinated individuals, and new mandatory testing requirements for local and state-level employees in our operating area affirm the need for ongoing and consistent COVID-19 screening available through safeCircle. Subject to FDA’s evolving EUA request review priorities, we expect to file shortly our formal request for EUA for our Linea Mutation Panel. In addition, in the coming weeks we intend to launch our COVID-19 veterinary vaccine candidate challenge trial in furtherance of a commercial animal health opportunity.

"Regarding our supply chain security business, we have cautious optimism within the cotton supply chains we serve as we approach the start of the cotton ginning season in the U.S. However, with Asia-Pacific beset by the Delta variant, man-made fiber opportunities remain static. With the tailwind of COVID-19 testing at our back supplemented by continued execution on business development initiatives, we believe we are laying the foundation for sustainable growth."

Fiscal Third Quarter 2021 Financial Highlights:

Revenues increased 294% for the third quarter of fiscal 2021 to $1.7 million, compared with $432 thousand reported in the same period of the prior fiscal year and decreased 36% from $2.7 million for the second quarter of fiscal 2021. The increase in revenues year over year was due primarily to an increase in service revenues of approximately $686 thousand and an increase of $583 thousand in product revenues. The increase in service revenue was primarily from revenues derived from our safeCircle COVID-19 surveillance testing. The increase in product revenue was mainly attributable to an increase in sales of our Linea COVID-19 Assay Kit. The decrease in revenues compared to the second quarter of fiscal 2021 was due to a decline in our safeCircle COVID-19 surveillance testing.
Total operating expenses increased to $4.5 million for the third fiscal quarter of 2021, compared with $3.5 million in the prior fiscal year’s third quarter and decreased from $4.6 million for the second quarter of fiscal 2021. The year-over-year increase is primarily attributable to an increase in total payroll of $535 thousand, of which $325 thousand was for staffing of Applied DNA Clinical Labs, LLC (ADCL). The increase in operating expenses was also the result of an increase of $148 thousand for supplies and equipment to operate the ADCL laboratory. The increase also relates to increases in research and development expenses of $215 thousand and depreciation and amortization of $186 thousand.
Net loss applicable to common stockholders for the quarter ended June 30, 2021, was $3.5 million, or $0.46 per share, compared with a net loss of $3.3 million, or $0.72 per share, for the quarter ended June 30, 2020.
Excluding non-cash expenses, Adjusted EBITDA was negative $2.8 million for both the quarters ended June 30, 2021, and 2020. See below for information regarding non-GAAP measures.
Cash and cash equivalents stood at $12.2 million on June 30, 2021, compared to $7.8 million as of September 30, 2020.
Nine-Month Financial Highlights:

Revenues increased 270% for the first nine-months of fiscal 2021 to $6.0 million, compared with $1.6 million reported in the same period of the prior fiscal year. The increase in revenues year over year was due primarily to an increase in service revenues of approximately $2.7 million and an increase of $1.7 million in product revenues. The increase in service revenue was primarily from revenues derived from our safeCircle COVID-19 surveillance testing. The increase in product revenue was mainly attributable to an increase in sales of our Linea Assay Kit.
Total operating expenses increased to $13.5 million for the first nine-months of fiscal 2021, compared with $9.5 million in the same period of the prior fiscal year. This increase is primarily attributable to an increase in payroll of $790 thousand relating to additional headcount to staff at ADCL. The increase in operating expenses also related to an increase in stock-based compensation expense of $834 thousand primarily relating to officer stock option grants that vested immediately. The increase also relates to increases in research and development expenses of $570 thousand and depreciation and amortization of $341 thousand.
Net loss applicable to common stockholders for the nine-months ended June 30, 2021, was $9.8 million, or $1.45 per share, compared with a net loss of $8.9 million, or $2.54 per share, for the first six months of fiscal 2020.
Excluding non-cash expenses, Adjusted EBITDA was negative $6.7 million for the first nine months of fiscal 2021, compared to negative $7.8 million for the same period in the prior fiscal year. See below for information regarding non-GAAP measures.
Fiscal Third Quarter 2021 Conference Call Information

The Company will hold a conference call and webcast to discuss its fiscal third quarter-end 2021 results on Thursday, August 12, 2021, at 4:30 PM ET. To participate on the conference call, please follow the instructions below. While every attempt will be made to answer investors’ questions on the Q&A portion of the call, not all questions may be answered.

To Participate:

Participant Toll Free:1-844-887-9402
Participant Toll: 1-412-317-6798
Please ask to be joined to the Applied DNA Sciences call
Live webcast: View Source

Telephonic replay (available 1 hour following the conclusion of the live call through August 19, 2021):

Participant Toll Free: 1-877-344-7529
Participant Toll: 1-412-317-0088
Participant Passcode: 10158254
The webcast and accompanying PowerPoint presentation will be archived on the ‘IR Calendar and Corporate Presentations’ page listed under the Investor Relations drop-down menu on the Company’s website.

About safeCircle

ADCL’s high throughput pooled testing program, known as safeCircle, utilizes frequent, high-sensitivity pooled testing to help prevent virus spread by quickly identifying infections within a community, school, or workplace. safeCircle provides rapid results using real-time PCR (RT-PCR) testing.

Click through to learn more about how safeCircle can help your community, school, and workplace: safeCircle

About Linea COVID-19 Assay Kit and Linea SARS-CoV-2 Mutation Panel

The Linea COVID-19 Assay Kit is a real-time RT-PCR test intended for the qualitative detection of nucleic acid from SARS-CoV-2 in respiratory specimens including anterior nasal swabs, self-collected at a healthcare location or collected by a healthcare worker, and nasopharyngeal and oropharyngeal swabs, mid-turbinate nasal swabs, nasopharyngeal washes/aspirates or nasal aspirates, and bronchoalveolar lavage (BAL) specimens collected by a healthcare worker from individuals who are suspected of COVID-19 by their healthcare provider (HCP). The test is also intended for use with anterior nasal swab specimens that are self-collected in the presence of an HCP from individuals without symptoms or other reasons to suspect COVID-19 when tested at least weekly and with no more than 168 hours between serially collected specimens.

The scope of the Linea COVID-19 Assay Kit EUA, as amended, is expressly limited to use consistent with the Instructions for Use by authorized laboratories, certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA) to perform high complexity tests. The EUA will be effective until the declaration that circumstances exist justifying the authorization of the emergency use of in vitro diagnostics for detection and/or diagnosis of COVID-19 is terminated or until the EUA’s prior termination or revocation. The diagnostic kit has not been FDA cleared or approved, and the EUA’s limited authorization is only for the detection of nucleic acid from SARS-CoV-2, not for any other viruses or pathogens.

The Linea SARS-CoV-2 Mutation Panel (formally SGS Mutation Panel) (the "Linea Mutation Panel") is for Research Use Only (RUO) and shall not be used for clinical diagnostic purposes. The Linea Mutation Panel has not been approved or authorized to diagnose, ameliorate and/or detect any disease by any U.S. or international regulatory authority.

On August 12, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported financial results for the second quarter of 2021 and provided clinical and corporate updates (Press release, Sunesis, AUG 12, 2021, View Source [SID1234586438]).

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"We entered the second half of the year with strong momentum thanks to the completion of key milestones across our pipeline," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "We believe Viracta is strongly-positioned to advance our novel therapy for the treatment of EBV-positive cancers forward, with our pivotal NAVAL-1 trial now open for enrollment and the recent clearance of our IND for EBV-positive solid tumors paving the way for a multicenter Phase 1b/2 trial. These two trials are targeting patient populations with a significant unmet medical need, and our expansion into solid tumors could meaningfully broaden our addressable patient market."

Second Quarter 2021 and Recent Highlights

Clinical

NAVAL-1, a global pivotal trial in Epstein-Barr virus (EBV)-positive (EBV+) relapsed/refractory (R/R) lymphoma, now open for enrollment. NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a global, multicenter, open-label Phase 2 basket trial. The trial, which will include multiple subtypes of R/R EBV-positive (EBV+) lymphoma patients, is designed to evaluate the anti-tumor activity of the combination treatment of nanatinostat with valganciclovir and is anticipated to enroll up to approximately 140 patients. The primary endpoint of the trial is objective tumor response rate as assessed by an independent review committee. If successful, Viracta believes this trial could support multiple new drug application (NDA) filings across various EBV+ lymphoma subtypes.
Announced FDA clearance of IND application for a Phase 1b/2 trial in EBV+ solid tumors. The multicenter Phase 1b/2 trial is designed to evaluate the safety and preliminary efficacy of Viracta’s all-oral combination regimen in patients with advanced EBV+ solid tumors, and in combination with the PD-1 inhibitor pembrolizumab in recurrent or metastatic nasopharyngeal carcinoma. Initiation of the trial is expected in the second half of 2021.
Corporate

Strengthened company leadership with the appointment of Ayman Elguindy, Ph.D., as Chief Scientific Officer, and additional key additions to management team. Dr. Elguindy has over 23 years of experience studying the role of viruses in cancer and spent the last decade as a faculty member at the Yale University School of Medicine, most recently as an Associate Professor in the Department of Pediatrics, Section of Infectious Disease, and Department of Pathology. Viracta also recently appointed Patric Nelson, MBA, as Senior Vice President, Business Development and Corporate Strategy and Biljana Nadjsombati, Pharm.D., as Vice President, Pharmaceutical Development.
Added as a member of the Russell 2000 Index and other FTSE Russell indexes. In June 2021, Viracta was included as a member of the small-cap Russell 2000 Index, the all-cap Russell 3000 Index, and the Russell Microcap Index.
Hosted key opinion leader webinar. The webinar featured presentations by key opinion leaders (KOLs) Pierluigi Porcu, M.D. (Thomas Jefferson University) and Kristen Cunanan, Ph.D. (Stanford Medicine Quantitative Sciences Unit) who discussed the current treatment landscape and unmet medical need in R/R EBV+ lymphoma, and the design of NAVAL-1. For a replay of the webinar, click here.
Anticipated 2021 Milestones

Initiation of a global Phase 1b/2 clinical trial in EBV+ solid tumors: H2 2021
Updated data from Phase 1b/2 trial in R/R EBV+ lymphoma (VT3996-201): H2 2021
Second Quarter 2021 Financial Results

Cash position – Cash and cash equivalents totaled approximately $122.7 million as of June 30, 2021. Viracta expects to end 2021 with greater than $100 million in cash and cash equivalents, which it expects will be sufficient to fund its operations into 2024.
Research and development expenses – Research and development expenses were $5.4 million and $9.5 million for the three and six-months ending June 30, 2021, respectively, compared to $3.4 million and $6.8 million for the same periods in 2020. This increase was primarily due to incremental costs associated with the initiation of the NAVAL-1 trial.
General and administrative expenses – General and administrative expenses were $3.9 million and $7.7 million for the three and six-months ending June 30, 2021, respectively, compared to $0.9 million and $1.9 million for the same periods in 2020. This increase was primarily due to expenses associated with operating as a public company and non-recurring, costs related to the merger with Sunesis Pharmaceuticals, as well as non-cash share-based compensation.
Acquired in-process research and development – Non-recurring, non-cash operating expenses of $0 and $84.5 million associated with the write-off of in-process research and development acquired in the merger were recorded for the three and six-months ending June 30, 2021.
Gain on Royalty Purchase Agreement – Gain on Royalty Purchase Agreement for the six-months ending June 30, 2021 was associated with upfront proceeds of $13.5 million received in connection with the multi-license milestone and royalty monetization transaction with XOMA Corporation in March 2021.
Adjusted loss from operations – Adjusted loss from operations for the six-months ended June 30, 2021, excluding the non-recurring and non-cash operating expenses associated with the write-off of in-process research and development acquired in the merger (a non-GAAP measure) was $3.7 million, compared to an unadjusted loss from operations of $88.2 million. There is not a comparative adjustment to loss from operations for the same period in 2020.
Net loss – Net loss was $9.2 million, or $0.25 per share (basic and diluted) for the quarter ended June 30, 2021, compared to a net loss of $4.2 million, or $15.52 per share (basic and diluted), for the same period in 2020. Net loss was $88.4 million, or $3.37 per share (basic and diluted) for the six months ended June 30, 2021, compared to a net loss of $8.7 million, or $32.13 per share (basic and diluted), for the same period in 2020.
About Nanatinostat

Nanatinostat (VRx-3996) is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing latent viral genes which are epigenetically silenced in EBV-associated malignancies. The nanatinostat and valganciclovir combination is being investigated in various subtypes of relapsed/refractory EBV+ lymphoma in multiple clinical trials, including a registration-enabling global, multicenter, open-label basket trial.

On August 12, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the second quarter ended June 30, 2021 (Press release, CymaBay Therapeutics, AUG 12, 2021, View Source [SID1234586437]).

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In July 2021, CymaBay announced a development funding agreement with Abingworth, pursuant to which CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial. In exchange, CymaBay will make fixed payments spread over a six-year period based on regulatory approval in the U.S. or the E.U. after the first such regulatory approval is obtained, as well as pay fixed and capped sales milestones based on U.S. product sales. CymaBay has the ability to accelerate payment at a reduced amount upon regulatory approval and in the event of a change of control of CymaBay. CymaBay retains upside potential for seladelpar in the U.S. along with full worldwide commercial rights.

CymaBay also continued to make progress conducting the development program for seladelpar in primary biliary cholangitis (PBC). Additional clinical sites were activated in North America and Europe during the second quarter in RESPONSE, a global Phase 3 registrational study evaluating seladelpar in patients with PBC, and in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional safety data to support registration. Enrollment of patients in both of these studies continued through the second quarter and is expected to increase as more sites are activated through the rest of 2021.

Sujal Shah, President and CEO of CymaBay, stated, "In addition to adding experienced talent across the functional areas vital to getting seladelpar to patients, we were thrilled to have secured the additional funding needed to complete Phase 3 development of seladelpar in PBC through a non-dilutive, risk-sharing funding agreement with Abingworth. Abingworth has had a long history of funding innovative companies in life sciences and shares our belief that seladelpar has the opportunity to meaningfully improve the care of patients with PBC. This strategic funding agreement further strengthens our balance sheet and allows us to maintain our dedicated focus on completing development of seladelpar to deliver value to patients and shareholders."

Recent Corporate Highlights

Executed a non-dilutive financing transaction with Abingworth for the development of seladelpar in PBC. Under the terms of the agreement, CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial.
Conducted enrollment activities for RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study is targeting enrollment of 180 patients who have an inadequate response to, or intolerance to, ursodeoxycholic acid, in a 2:1 randomization to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline assessed by a numerical rating scale recorded with an electronic diary.
Conducted enrollment activities for ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration.
Initiated enrollment in a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes (T1D). The study is being conducted by the AdventHealth Translational Research Institute in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982.
Completed a Phase 1 single ascending dose and multiple asending dose study of CB-0406, a non-agonist ligand of PPARg that attenuates the expression of inflammatory genes. While the study showed CB-0406 had improved pharmacokinetics versus those historically achieved with the pro-drug arhalofenate, CB-0406’s safety profile did not support continued development as a result of the occurrence of a small number of reversible cases of thrombocytopenia at higher doses.

Held $106.1 million in cash, cash equivalents and short-term investments as of June 30, 2021. We believe that cash and investments, along with the initial $75 million funding raised through the development funding agreement with Abingworth in July 2021 are sufficient to fund CymaBay’s current operating plan into 2023.

Due to the ongoing effects of the global coronavirus pandemic, CymaBay continues to conduct its operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. The recent emergence of the Delta variant has led to uncertainty regarding the duration and effects that the pandemic will have on future operating milestones. CymaBay continues to closely monitor pandemic developments and their associated risks to the business, including the conduct of its clinical development of seladelpar, and will continue to take actions to mitigate them where possible. Further, all CymaBay’s actions will continue to be guided by a commitment to ensuring the health and safety of its employees as well as patients enrolled in its clinical studies.
Second Quarter and Six Months Ended June 30, 2021 Financial Results

Research and development expenses for the three months ended June 30, 2021 and 2020 were $16.7 million and $7.9 million, respectively. Research and development expenses for the six months ended June 30, 2021 and 2020 were $29.1 million and $17.5 million, respectively. Research and development expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 primarily due to an increase in clinical trial activities following our resumption of clinical development of seladelpar in PBC in late 2020. In particular, cost increases were primarily driven by the enrollment activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC. In the three and six months ended June 30, 2020, costs incurred were primarily associated with the termination and shutdown of our Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, after the seladelpar development program was placed on hold from November 2019 through July 2020.

General and administrative expenses for the three months ended June 30, 2021 and 2020 were $6.5 million and $3.2 million, respectively. General and administrative expenses for the six months ended June 30, 2021 and 2020 were $11.8 million and $7.6 million, respectively. General and administrative expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 due to higher employee compensation associated with the hiring of additional personnel and an increase in consulting and other expenses upon resumption of development of seladelpar in the second half of 2020.

Net loss for the three months ended June 30, 2021 and 2020 was $23.2 million and $10.7 million, or ($0.34) and ($0.16) per diluted share, respectively. Net loss for the six months ended June 30, 2021 and 2020 was $40.8 million and $23.8 million, or ($0.59) and ($0.35) per diluted share, respectively. Net loss was higher largely due to increases in clinical operating expenses, which were incurred following the resumption of our clinical development of seladelpar in PBC during the second half of 2020. We expect our operating expenses to increase in 2021 as we continue to execute on our clinical development plans.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss second quarter 2021 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13720877. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On August 12, 2021 Kezar Life Sciences, Inc., (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported its second quarter 2021 financial results and corporate highlights (Press release, Kezar Life Sciences, AUG 12, 2021, View Source [SID1234586436]).

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"We were pleased this quarter to share the final data update from our completed Phase 1b portion of the MISSION study, building on the positive safety, tolerability and early efficacy profile of KZR-616. I commend the entire team at Kezar on another quarter of outstanding execution across both of our programs and look forward to providing meaningful updates from both before the end of the year," said John Fowler, Kezar’s Co-founder and Chief Executive Officer. "We are also pleased to announce that Kezar recently submitted an IND application to the FDA for KZR-261, our first-in-class protein secretion inhibitor. Like the platform potential we see for KZR-616 in autoimmunity, we believe KZR-261 could have broad potential across the oncology landscape. We look forward to sharing more about our planned Phase 1 trial design and the potential of our protein secretion drug discovery platform in the coming months."

Clinical Highlights & Updates

KZR-616: Selective Immunoproteasome Inhibitor

MISSION – Phase 2 clinical trial in patients with lupus nephritis (LN) (NCT03393013)

At the European Congress of Rheumatology (EULAR 2021) in June, Kezar presented final clinical data from the completed Phase 1b dose escalation portion of the MISSION study in 47 patients with systemic lupus erythematous, including two patients with active proliferative LN. KZR-616 demonstrated improvement across all exploratory efficacy measures and was well-tolerated up to 75 mg subcutaneously once weekly for 13 weeks. In the two patients with LN, improvements in renal function correlated with reductions in a key biomarker of kidney inflammation, uCD163.

The safety and tolerability profile observed with KZR-616 was consistent with previously reported data and supports treatment for chronic use. No new safety or tolerability signals were observed from previously reported data.
These data support the development of KZR-616 in multiple immune-mediated diseases.
Doses being investigated in the Phase 2 clinical trials are 60 mg (MISSION Phase 2 in LN) and 45 mg (PRESIDIO Phase 2 in dermatomyositis and polymyositis) with weekly subcutaneous dosing.
The amended Phase 2 open-label portion of the MISSION trial in patients with active, proliferative LN opened for enrollment in August 2020 and is actively recruiting. The primary efficacy endpoint for the trial is the proportion of patients achieving a renal response measured by a 50% or greater reduction in urine protein to creatinine ratio (UPCR) at six months.
Kezar reiterates prior guidance and expects interim data to be reported in Q4 2021, and topline data are expected in the first half of 2022.
PRESIDIO – Phase 2 clinical trial in patients with dermatomyositis (DM) and polymyositis (PM) (NCT04033926)

The PRESIDIO Phase 2, placebo controlled cross-over trial of KZR-616 in DM and PM is actively enrolling. Additionally, a 12-month open-label extension study is open to patients completing the 32-week placebo-controlled trial (NCT04628936).
Topline data are expected in the first half of 2022.
KZR-261: Protein Secretion Inhibitor

KZR-261 is a first-in-class protein secretion inhibitor that targets the Sec61 translocon and has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies.
Kezar submitted an investigational new drug (IND) application for KZR-261 to the U.S. Food and Drug Administration (FDA) in August 2021. The company plans to initiate a Phase 1 clinical trial of KZR-261, which will assess safety, tolerability and preliminary tumor activity of KZR-261 in solid tumors.
At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Virtual Annual Meeting in April, Kezar presented preclinical data on its novel small molecule inhibitors of the Sec61 translocon. These data support the therapeutic potential of inhibiting Sec61 and the protein secretion pathway as a way to generate novel therapies to treat multiple tumor indications.
Corporate Updates

Kezar formed a Clinical Advisory Committee in June, comprised of world-renowned thought leaders in immunology, rheumatology, neurology and nephrology. Appointments to the committee include: Rohit Aggarwal, MD, MS, Professor of Medicine, University of Pittsburgh; Prof. Olivier Benveniste, MD, PhD, Professor of Internal Medicine & Immunology, Sorbonne Université, Pitie Salpetriere Hospital; Mazen Dimachkie, MD, Professor of Neurology, University of Kansas Medical Center; Ingrid Lundberg, MD, PhD, Professor of Medicine, Karolinska Institute; Samir V. Parikh, MD, Assistant Professor of Medicine, Ohio State University Wexner Medical Center; and Onno Teng, MD, PhD, Nephrology Clinical Scientist, Leiden University Medical Center.
Micki Klearman, MD, rheumatologist and internist, was appointed to Kezar’s Board of Directors, bringing over two decades of biopharmaceutical experience with her significant contributions to the fields of immunology and rheumatology.
Second Quarter 2021 Financial Results

Cash, cash equivalents and marketable securities totaled $129 million as of June 30, 2021, compared to $140 million as of December 31, 2020. 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 and preclinical research and development, offset by the net proceeds from the issuance of common stock in February 2021, under the "at-the-market" Sales Agreement with Cowen and Company, LLC.
Research and development expenses for the second quarter of 2021 increased by $2.2 million to $9.3 million compared to $7.1 million in the second quarter of 2020. 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 second quarter of 2021 increased by $1.0 million to $3.7 million compared to $2.7 million in the second quarter of 2020. The increase was primarily due to an increase in stock-based compensation and personnel and recruiting expenses as a result of an increase in headcount and salaries and an increase in the cost of directors’ and officers’ liability insurance.
Net loss for the second quarter of 2021 was $13.0 million, or $0.25 per basic and diluted common share, compared to a net loss of $9.5 million, or $0.22 per basic and diluted common share, for the second quarter of 2020.
Total shares of common stock outstanding were 48.1 million shares as of June 30, 2021. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 7.3 million shares of common stock at a weighted-average exercise price of $5.86 per share as of June 30, 2021.
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. Data generated from Phase 1a and 1b clinical trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

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 pathway. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered by 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. An IND submission in solid tumors was filed in August 2021 and a Phase 1 trial is expected to commence once the IND goes into effect.

About Lupus Nephritis

Lupus nephritis (LN) is one of the most serious complications of systemic lupus erythematosus. LN is a disease comprising a spectrum of vascular, glomerular and tubulointerstitial lesions and develops in approximately 50% of SLE patients within 10 years of their initial diagnosis. LN is associated with considerable morbidity, including an increased risk of end-stage renal disease requiring dialysis or renal transplantation and an increased risk of death. There are limited approved therapies for the treatment of LN. Management typically consists of induction therapy to achieve remission and long-term maintenance therapy to prevent relapse.

About Dermatomyositis and Polymyositis

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