On November 14, 2022 Exicure, Inc. (Nasdaq: XCUR), an early-stage biotechnology company historically focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets, reported financial results for the quarter ended September 30, 2022 and provided an update on its business strategy and corporate progress (Press release, Exicure, NOV 14, 2022, View Source [SID1234623984]).

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Corporate Update

•As previously reported, on September 26, 2022, the Company announced its commitment to a plan to wind down the Company’s existing preclinical programs, including the development of its SCN9A program, to suspend all of its research and development activities, including suspension of all partnered programs, and to implement a reduction in force where the Company reduced approximately 66% of its then-existing workforce, as well as other cost-cutting measures (collectively, the "Plan"). The purpose of the Plan was to decrease expenses, thereby, extending the Company’s cash runway, and enable the Company to maintain a streamlined organization to support key corporate functions while it continues to actively pursue strategic alternatives to maximize stockholder value.

•The reduction in force announced on September 26, 2022 is now substantially complete.

•The Company continues to actively pursue out-license opportunities for its clinical asset, cavrotolimod, as well as for its preclinical candidates, including the SCN9A program for neuropathic pain, and to pursue all strategic alternatives with the goal of maximizing stockholder value.

•As also previously reported, on September 26, 2022, the Company entered into a securities purchase agreement with CBI USA, Inc. ("CBI USA"), pursuant to which it agreed to issue and sell to CBI USA in a private placement an aggregate of 3,400,000 shares of Exicure’s common stock, par value $0.0001 per share, at a purchase price of $1.60 per share (the "Private Placement").

◦The Private Placement is expected to close in the fourth quarter of 2022, subject to the satisfaction of certain closing conditions, including the Company’s stockholders voting in favor of the Private Placement. On November 10, 2022, the Company has filed and mailed its definitive proxy statement in connection with the special meeting to be held on December 15, 2022 at which the Company’s stockholders will be asked to vote on approval of the Private Placement.
◦Immediately following the closing of the Private Placement, CBI USA will hold approximately 50.4% of the shares of the Company’s common stock. The Company expects to receive aggregate gross proceeds from the Private Placement of approximately $5.4 million, before deducting estimated offering expenses payable by Exicure.

"We look forward to the possibility of working with CBI USA to potentially pursue strategic transactions," commented Matthias Schroff, Ph.D., Chief Executive Officer of Exicure. "While awaiting shareholder approval of the private placement transaction with CBI USA, Exicure continues to explore strategic alternatives for its existing clinical and preclinical programs to maximize stockholder value."
1

Third Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and short-term investments, and restricted cash were $16.8 million as of September 30, 2022, as compared to $48.3 million as of December 31, 2021. The Company expects that its existing cash and cash equivalents (which excludes expected proceeds from the Private Placement as the Private Placement has not closed) will enable it to fund its current operations into the second quarter of 2023.

Revenue: Revenue was $2.0 million for the quarter ended September 30, 2022, reflecting an increase of $5.7 million from revenue of $(3.7) million for the quarter ended September 30, 2021. The increase in collaboration revenue of $5.7 million is mostly due to an increase in revenue related to the Company’s collaboration with AbbVie, Inc. ("AbbVie") of $5.1 million, as well as an increase in revenue related to the Company’s collaboration with Ipsen Biopharm Limited of $0.6 million. Revenue recognized under the Company’s collaboration with AbbVie for the three months ended September 30, 2021 reflected the cumulative catchup adjustment (reduction) of revenue of $(4.5) million in connection with the change in estimate that resulted from a change in workplan during the third quarter of 2021.

Research and Development (R&D) Expense: Research and development expenses were $4.8 million for the quarter ended September 30, 2022, as compared to $16.5 million for the quarter ended September 30, 2021. The decrease in R&D expense for the three months ended September 30, 2022 of approximately $11.7 million reflects fewer clinical, preclinical, and discovery program activities and a reduction in headcount resulting from the strategic restructuring activities and discontinuation of cavrotolimod program that were announced in December 2021.

General and Administrative (G&A) Expense: General and administrative expenses were $2.4 million for the quarter ended September 30, 2022, as compared to $2.9 million for the quarter ended September 30, 2021. The decrease in G&A expense of approximately $0.5 million for the three months ended September 30, 2022 was mostly due lower compensation and related costs in connection with a lower headcount during the period resulting from the restructuring activities that were announced in December 2021 and lower accrued bonus expense in the current year period resulting from the reduction of the estimated 2022 bonus liability, as well as lower accounting costs. These lower costs in the current year period were partially offset by higher legal, consultant, and advisory costs incurred.

Net Loss: The Company had a net loss of $5.2 million for the quarter ended September 30, 2022, as compared to a net loss of $23.5 million for the quarter ended September 30, 2021. The decrease in net loss was primarily driven by lower R&D expense and higher non-cash revenue during the period.

Going Concern: Given the Company’s current cash position, operating plans and forecasted negative cash flows from operating activities over the next twelve months, management believes there is substantial doubt regarding the Company’s ability to continue as a going concern within one year after the date that its unaudited condensed consolidated financial statements for the quarter ended September 30, 2022 are issued. The Company will require substantial additional financing to address the Company’s working capital and other financing needs to pursue its business strategy. There is a significant likelihood that, without the consummation of the Private Placement, the Company will need to seek bankruptcy protection in the near term, which may result in its stockholders receiving no or very little value in respect of their shares of the Company’s common stock.

On November 14, 2022 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion" or the "Company), a clinical-stage biotechnology company specializing in the development of AI-driven immunotherapies, reported its third quarter 2022 financial results and provided an operational and business update (Press release, Evaxion Biotech, NOV 14, 2022, View Source [SID1234623983]).

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Per Norlén, CEO of Evaxion, said: "Evaxion continues to advance its drug development pipeline, reaching an important milestone in the third quarter with the enrollment of the first patient in our global Phase 2b clinical trial of EVX-01, our personalized cancer immunotherapy for the treatment of patients with metastatic melanoma. Going forward, we intend to further increase our focus on our clinical lead oncology assets within personalized cancer immunotherapy."

Operational and Business Highlights in the Third Quarter of 2022

Enrolled the first patient in the global Phase 2b clinical trial of EVX-01 for the treatment of melanoma.
In the Company’s first Phase 2b clinical trial, Evaxion is evaluating the efficacy and safety of EVX-01 in adults with metastatic melanoma. The trial is being conducted globally at clinical sites across the US, Europe, and Australia in collaboration with Merck & Co., Inc. Patients enrolled in the Phase 2b clinical trial will receive standard-of-care treatment along with KEYTRUDA in combination with EVX-01. Evaxion is responsible for the conduct of the trial, and Merck will supply the required KEYTRUDA. The Company anticipates interim topline data readout in the second half of 2023.

Announced an increased strategic focus on clinical lead oncology assets and partnering. Evaxion intends to further increase its focus on its clinical lead oncology product candidates, EVX-01 and EVX-02/03, to bring them to clinical proof of concept followed by out-licensing. Additionally, the Company plans preclinical partnering of its early-stage programs under its infectious disease platforms. This was announced by the Company’s new Chief Executive Officer, Per Norlén, who emphasized the importance of prioritizing the Company’s activities in the current market. As previously announced by the Company, the EVX-01 program recently started to enroll patients in the global Phase 2b trial in metastatic melanoma. At the same time, the Phase 1/2a trial of the DNA-based EVX-02 is progressing as planned, with data readout currently expected by mid-2023. The next-generation DNA vaccine, EVX-03, builds on EVX-02 and holds the potential for even stronger results due to an integrated mechanism that boosts the immune system. The Company plans to submit a regulatory filing of EVX-03, following EVX-02 data, which may allow Evaxion to advance EVX-03 faster to clinical proof of concept. Regarding the Company’s early programs for infectious diseases, such as our Staphylococcus aureus vaccine, the Company aims to develop these in partnerships rather than bringing them into clinical development by itself.

Announced NIH Grant for Research Collaboration with UMass Chan Medical School to develop EVX-B2 gonorrhea vaccine product candidate. Evaxion announced a discovery project (EVX-B2) to create a gonorrhea vaccine based on the Company’s artificial intelligence (AI) platform, EDEN. Chief Scientific Officer at Evaxion, Birgitte Rønø, states that the scientific collaboration with UMass Chan and the grant from NIH further reinforces Evaxion’s capabilities within AI-based vaccine design and allow the Company to fast-track the development of a gonorrhea vaccine candidate.

A published peer-reviewed article in Future Oncology on Phase 2b trial design for EVX-01. Evaxion published an article in Future Oncology focusing on the Phase 2b trial design of EVX-01. This trial is designed so that the patients may continue treatment with Evaxion’s immunotherapy, even if the standard-of-care treatment changes. With this new and innovative trial design, Evaxion expands the patients’ otherwise limited treatment opportunities.

Events after the Reporting Period

Per Norlén, M.D., PhD. succeeded Lars Wegner, M.D., as Chief Executive Officer
Dr. Norlén is a board-certified physician and associate professor in clinical pharmacology with more than 20 years in the biotech sector. The last 12 years have been in executive leadership roles. He brings a wealth of experience from being CEO of listed drug development companies. He has a proven business development track record, including major out-licensing deals with biotech and Pharma.

Expected Milestones in the Fourth Quarter of 2022

Selection of the first viral candidate from our RAVEN platform.
Expected Milestones in the First Half of 2023

Clinical readout of Phase 1/2a clinical study to evaluate EVX-02 in patients with resectable melanoma.
Third Quarter 2022 Financial Results

Cash position: As of September 30, 2022, cash and cash equivalents were $17.9 million compared to $32.2 million as of December 31, 2021. The decrease in cash and cash equivalents during the first nine months of 2022 was primarily attributable to an increase in our operating expenses for the first nine months of 2022, partially offset by the proceeds received from the first tranche of our loan from the European Investment Bank.
Research and Development expenses were $4.1 million for the three months that ended September 30, 2022, compared to $4.4 million for the same period in 2021. The decrease was primarily due to lower external costs related to clinical trials.
General and Administrative expenses were $2.0 million for the three months ending September 30, 2022, compared to $1.5 million for the same period in 2021. The increase was primarily due to an increase in fees associated with the expansion of our business as a listed company.
Net loss was $5.7 million for the three months ended September 30, 2022, or ($0.24) loss per basic and diluted share as compared to $5.3 million, or ($0.27) loss per basic and diluted share for the three months ended September 30, 2021.
Guidance

We expect our existing cash and cash equivalents, will be sufficient to fund our operating expenses and capital expenditure requirements into mid-2023.

On November 14, 2022 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company focused on developing novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the third quarter 2022 and provided an update on its clinical development programs (Press release, Equillium, NOV 14, 2022, View Source [SID1234623982]).

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"I am proud of what our team has accomplished in the third quarter, including executing a definitive agreement to acquire Metacrine and announcing interim data from the Type B portion of the EQUALISE study in lupus nephritis. Since then, we have also initiated two new studies – a Phase 2 study of EQ101 in subjects with alopecia areata, and a Phase 1 study of EQ102 that will be positioned to treat patients with celiac disease," said Bruce Steel, chief executive officer at Equillium. "The Metacrine acquisition will meaningfully strengthen our cash position, which at closing is expected to add approximately $35 million to the balance sheet and extend our cash runway into 2024, taking us through multiple data catalysts from three different clinical programs over the next year."

Highlights Since the Beginning of Q3 2022:

Announced definitive merger agreement to acquire Metacrine in an all-stock transaction, anticipated to add $35 million in cash to Equillium’s balance sheet at closing. The acquisition also includes Metacrine’s farnesoid X receptor (FXR) platform, including lead molecule MET642, an orally delivered FXR agonist that is a potential first-in-class, non-immunosuppressive treatment for inflammatory bowel disease.

Announced positive interim results from the Type B portion of the EQUALISE study of itolizumab in subjects with lupus nephritis. Subjects were highly proteinuric with a baseline mean urine protein creatinine ratio (UPCR) of 5.8 g/g. Clinically meaningful responses were observed:

By week 28 (or end of study):

3 of 6 (50%) subjects achieved complete response (UPCR < 0.7 g/g)

2 of 6 (33%) subjects achieved partial response (UPCR > 50% reduction)

4 of 6 (67%) subjects achieved greater than 80% reduction in UPCR

In all subjects receiving more than one dose:

8 of 12 (67%) subjects achieved greater than 50% reduction in UPCR

60% average reduction in UPCR (over 3g of proteinuria)

Subjects titrated steroid dose to < 7.5 mg/day consistent with EULAR/ERA-EDTA recommendations

Initiated a multicenter, Phase 2 open-label, proof-of-concept study of EQ101 in adult subjects between 18 and 60 years of age, with at least 35% scalp hair loss due to alopecia areata. Approximately 30 subjects will be enrolled in the study where they will be dosed intravenously once weekly for 24 weeks.

Initiated a Phase 1 randomized, double-blind, placebo-controlled study of EQ102 administered subcutaneously as single or multiple doses in up to 64 healthy volunteers. The first cohort of eight study participants has been completed and the second cohort has initiated.

Presented data highlighting:

EQ101, a first-in-class, tri-specific cytokine inhibitor selectively targeting IL-2, IL-9 and IL-15 at the receptor level, that may afford significant advantages over other approaches to the treatment of alopecia areata, including JAK inhibition, at the 6th Annual Dermatology Drug Development Summit
Urinary soluble ALCAM (sALCAM) could be a potential biomarker of disease severity in lupus nephritis (LN) and indicative of a patient’s response to treatment at annual meeting of the American College of Rheumatology

Design and development of multi-specific cytokine inhibitors, and the importance of targeting biological synergy to optimize therapeutic outcomes at the 3rd Annual Cytokine-Based Drug Development Summit

Therapeutic potential for multi-cytokine inhibitors such as EQ101 and EQ102, as potentially effective strategies for the treatment of certain autoimmune diseases at the Annual La Jolla Immunology Conference

Anticipated Upcoming Milestones & Catalysts:

Itolizumab – EQUALISE Phase 1b study: topline data from the Type B part of the study in patients with lupus nephritis expected mid-2023

EQ101 – Phase 2 study in subjects with alopecia areata: data expected in 2023

EQ102 – Phase 1 study in healthy volunteers and subjects with celiac disease: data expected in 2023

Third Quarter 2022 Financial Results

Research and development (R&D) expenses for the third quarter of 2022 were $8.8 million, compared with $7.0 million for the same period in 2021. The increase was primarily due to greater clinical development expenses, driven by start-up costs related to the Phase 3 EQUATOR study.

General and administrative (G&A) expenses for the third quarter of 2022 were $4.5 million, compared with $2.9 million for the same period in 2021. The increase was primarily due to higher legal fees related to business development activities and greater employee compensation and overhead expenses.

Net loss for the third quarter of 2022 was $13.7 million, or $(0.40) per basic and diluted share, compared with a net loss of $10.3 million, or $(0.35) per basic and diluted share for the same period in 2021. The increase in net loss was largely attributable to greater operating expenses.

Cash, cash equivalents and short-term investments totaled $44.5 million as of September 30, 2022, compared to $57.6 million as of June 30, 2022. Equillium believes that its cash and investments, together with the additional cash that will be acquired with the expected closing of the Metacrine acquisition, will be sufficient to fund its operations into 2024.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited.

About Multi-Cytokine Platform: EQ101 & EQ102

Our proprietary Multi-Cytokine Platform (MCP) generates rationally designed composite peptides that selectively block key cytokines at the shared receptor level targeting pathogenic cytokine redundancies and synergies while preserving non-pathogenic signaling. This approach provides multi-cytokine inhibition at the receptor level and is expected to avoid the broad immuno-suppression and off-target safety liabilities that may be associated with other therapeutic classes, such as JAK inhibitors. Many immune-mediated diseases are driven by the same combination of dysregulated cytokines, and we believe identifying the key cytokines for these diseases will allow us to target and develop customized treatment strategies for multiple autoimmune and inflammatory diseases.
Current MCP assets include EQ101, a first-in-class, selective, tri-specific inhibitor of IL-2, IL-9 and IL-15, and EQ102, a first-in-class, selective, bi-specific inhibitor of IL-15 and IL-21.

On November 14, 2022 Ensysce Biosciences, Inc. ("Ensysce" or the "Company") (NASDAQ:ENSC)(OTC PINK:ENSCW), a clinical-stage biotech company applying transformative chemistry to improve prescription drug safety to reduce abuse and overdose, reported financial results for the third quarter of 2022 (Press release, Ensysce Biosciences, NOV 14, 2022, View Source [SID1234623981]).

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Dr. Lynn Kirkpatrick, Chief Executive Officer of Ensysce, commented, "We remain focused on progressing our clinical development plans for our programs and achieving each milestone we have identified. I could not be more pleased with the recently reported positive topline results from our human abuse potential (HAP) study for intranasal administration of PF614. These results were in-line with our expectations and importantly, consistent with the unique properties of PF614 as well as prior findings that we believe demonstrate PF614 will provide advantages over currently marketed products. The highly significant results from this study represent a major accomplishment towards our mission of reducing abuse and overdose, ultimately enhancing drug safety."

Dr. Kirkpatrick continued, "Additionally, we were very happy to provide today’s announcement regarding the written guidance we received from the FDA which indicated that a development program for PF614 in acute pain may be appropriate. As a result of this FDA guidance, we believe the non-clinical program for PF614 will be significantly shortened for acute pain. We are pleased we can develop PF614 for acute pain while we also continue with our chronic pain development pathway."

TAAPTM (Opioid Abuse Deterrent Program) Updates

PF614 is the first product in a new class of analgesia, a "Trypsin-Activated Abuse Protection" (TAAPTM) oxycodone product. The Company’s TAAPTM technology is designed to be highly resistant to tampering and abuse and is a unique chemical modification creating a new generation of opioid pain products as compared to traditional Abuse Deterrent Formulations (ADFs). TAAP controls or "turns on" the release of the active ingredient in PF614 providing abuse deterrence.

On October 31, 2022, the Company announced positive topline results from a human abuse potential (HAP) study, PF614-103, with intranasal administration of PF614 as compared to crushed oxycodone or placebo. PF614 demonstrated significantly reduced "drug liking," the primary endpoint, when compared to intranasal crushed immediate-release oxycodone.
On October 28, 2022, the Company announced first subjects dosed in its second HAP study, PF614-104, comparing oral administration of PF614 versus oxycodone or placebo. Data from this study is expected in early 2023. These HAP studies are intended to support abuse-deterrent labeling upon final approval of PF614.
On July 27, 2022, the Company announced positive date from a bioequivalence (BE) study PF614-102 of its novel TAAP opioid PF614 compared to OxyContin which the company believes will support the substitution of PF614 for OxyContin in the market. The Company believes that the BE data from this study will also support the 505(b)(2) regulatory path for clinical development of PF614, an abbreviated pathway to FDA approval. This pathway allows reference to available safety and clinical data from an approved product, and the BE data established by this study will move PF614 closer to registration.
MPARTM (Opioid Abuse Deterrent and Overdose Protection Program) Updates

PF614-MPAR is a combination product designed to have overdose protection along with the abuse protection of TAAP. MPAR (Multi-Pill Abuse Resistance) turns off the release of the opioid in an overdose situation, providing the additional layer of protection to Ensysce’s TAAP pain medications.

On August 31, 2022, the Company announced a partnership with Quotient Sciences on the Development and Clinical Testing of PF614-MPARTM. Quotient Sciences is currently using its integrated Translational Pharmaceutics platform to identify a PF614-MPAR combination that allows conversion into oxycodone within the prescribed dose range but reduces conversion to oxycodone at higher than prescribed dose levels in an overdose scenario.
On June 27, 2022, the Company announced its notice of award for the 4th year of funding for its MPAR platform to support the final part of the ongoing clinical trial PF614-MPAR-101. The amount awarded was $2.8 million and this brings total funding from NIDA under this grant to $10.8 million.
Financial Results

Cash – Cash and cash equivalents were $4.5 million as of September 30, 2022, as compared to $3.7 million as of June 30, 2022.
Federal Grants – Funding from federal grants was $0.3 million for the third quarter of 2022 compared to $1.2 million in the comparable year ago quarter. The decrease is a result of timing of pre-clinical and clinical activities for the MPAR program.
Research & Development Expenses – R&D expenses were $4.8 million for the third quarter of 2022 compared to $1.7 million in the comparable year ago quarter. The increase in expenses results from increased pre-clinical and clinical activities for PF614.
General & Administrative Expenses – G&A expenses were $1.7 million for the third quarter of 2022 compared to $16.4 million for the same period in 2021. The decrease in expenses is largely attributable to significant non-cash expenses recorded in the prior year period related to the valuation of warrants issued.
Other Income (Expense) – Total other income (expense) was expense of $3.7 million in the third quarter of 2022 and expense of $0.3 million in the third quarter of 2021. The change in other expenses is primarily due to non-cash fair value adjustments for convertible notes and warrants.
Net Loss – Net loss for the third quarter of 2022 was $9.9 million compared to net loss of $17.2 million for the comparable year ago period. As a clinical stage biotech company, our continued research and development efforts toward regulatory approvals for our product candidates are expected to result in losses for the foreseeable future.
Corporate Update Conference Call

As previously announced, CEO, Dr. Lynn Kirkpatrick, CFO, Dave Humphrey, and CMO, Dr. William Schmidt, will host a corporate update conference call on Wednesday, November 16, 2022, at 11:00am ET to provide a corporate update and review the recently discussed results from the HAP study of PF614. The call will conclude with Q&A from participants. An accompanying presentation will be posted prior to the call to the Company’s investor relations website.

Please dial in at least 10 minutes before the start of the call to ensure timely participation. A playback of the call will be available through Wednesday, November 16, 2022. To listen, call 1-844-512-2921 within the United States and Canada or 1-412-317-6671 when calling internationally. Please use the replay pin number 13734017.

On November 14, 2022 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 third quarter ended September 30, 2022 (Press release, CymaBay Therapeutics, NOV 14, 2022, View Source [SID1234623979]).

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Sujal Shah, President and CEO of CymaBay, stated, "Early in the third quarter we completed enrollment in RESPONSE, our global registration study of seladelpar in patients with PBC. This achievement was a direct result of the tireless efforts of our teams and the relationships we have been fortunate to leverage among partners, health care providers and patient advocacy groups around the world. In addition to clinical presentations highlighting some of the important benefits of seladelpar for patients, we connected with all of these groups at The Liver Meeting last week and are aligned, coordinated and energized to deliver on transformational catalysts that lie ahead of us in 2023. With over 300 patients with PBC taking seladelpar daily across our active clinical studies, we continue to execute on what we believe is the most robust development program in PBC being conducted today. Our focus at CymaBay is clear and will remain so as we drive towards sharing top-line data from RESPONSE in the third quarter of 2023."

Recent Corporate Highlights

Enrollment was completed in the RESPONSE study, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study has enrolled 193 patients who have an inadequate response or intolerance to ursodeoxycholic acid in a 2:1 ratio to receive once daily oral 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 as assessed by a validated numerical rating scale recorded with an electronic diary.
Continued strong enrollment in ASSURE, the restarted open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety and efficacy data to support registration. There are now over 180 patients in this study taking seladelpar daily. After completing the RESPONSE study, eligible patients will be able to roll over into ASSURE.
Data presented at The Liver Meeting of the American Association for the Study of Liver Diseases, in Washington, DC. The clinical presentations featured:

A poster presentation titled "Seladelpar Improved the Lipid Profile of Patients with Primary Biliary Cholangitis (PBC): Results from Phase 2 and 3 Clinical Studies" delivered by Christopher L. Bowlus MD, the chief of the Division of Gastroenterology and Hepatology at the University of California Davis. This poster highlighted the results of a pooled analysis of the improvements in lipids observed over 6 months from an open-label phase 2 study and a placebo (Pbo) controlled phase 3 study (ENHANCE) assessing seladelpar at a daily dose of 5 mg or 10 mg in PBC patients. This analysis found that of the 373 patients analyzed, total cholesterol, LDL-cholesterol and triglyceride levels were elevated in 77%, 54% and 21% of patients at baseline, respectively. Treatment with seladelpar through 6 months resulted in significant improvements in all three of these parameters. Interestingly, a comparison of lipid lowering in patients with and without background lipid therapy revealed that seladelpar achieved similar treatment effects. Confirmation of the effects of seladelpar treatment on lipid profiles in the RESPONSE and ASSURE studies is of interest given that dyslipidemia is a common feature in patients with PBC.

A second poster presenting clinical data titled "Seladelpar, a PPAR-delta Agonist, Improves Inflammatory Lipid Mediators in the Serum Metabolome in Patients with Primary Biliary Cholangitis (PBC)" examined changes from baseline to 3 months in the serum metabolome of 160 patients in the ENHANCE study dosed daily with placebo, seladelpar 5 mg and seladelpar 10 mg. Seladelpar dose-dependently increased serum markers of mitochondrial and peroxisomal fatty acid oxidation and significantly reduced levels of inflammatory lipid mediators. This study of 1,474 untargeted global serum metabolites provided mechanistic insight into metabolic pathways altered by seladelpar treatment of patients with PBC. These results suggest novel aspects by which the action of seladelpar may improve cholestasis and liver function in patients with PBC.
Held virtual analyst day featuring a presentation from a hepatology key opinion leader, Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD, Director of Liver Institute Northwest and Professor of Medicine at Washington State University, who discussed the current treatment landscape and future goals for treating patients with PBC.
Announced the promotion of Dr. Charles McWherter to President of Research and Development, in addition to his continuing role as Chief Scientific Officer since 2013. Dr. McWherter’s extensive experience in the pharmaceutical industry as an executive focused on discovery, research and development and the depth of knowledge he has attained in our therapeutic areas of focus make him a perfect fit to serve in this expanded capacity.
Planning and execution progressed in our manufacturing and supply chain functions to support NDA submission and post-approval launch. Pre-commercial market analysis and planning became an increasing focus to support our planned product launch.
Held $153.4 million in cash, cash equivalents and investments as of September 30, 2022. We believe that cash and investments on hand are sufficient to fund CymaBay’s operating plan through 2023.
Third Quarter and Nine Months Ended September 30, 2022, Financial Results

Research and development expenses for the three months ended September 30, 2022, and 2021 were $15.5 million and $17.0 million, respectively. Research and development expenses for the nine months ended September 30, 2022, and 2021 were $51.8 million and $46.1 million, respectively. Research and development expenses in the three months ended September 30, 2022 were lower than the corresponding period in 2021 primarily due to the completion of enrollment of our RESPONSE trial in July 2022. Research and development expenses in the nine months ended September 30, 2022 were higher than the corresponding period in 2021 primarily due to the hiring of additional personnel and an expansion of clinical trial activities associated with the ongoing late-stage development of seladelpar in PBC. Specifically, higher costs were primarily driven by site activation, patient enrollment, and other clinical trial activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC.
General and administrative expenses for the three months ended September 30, 2022 and 2021 were $5.9 million and $5.2 million, respectively. General and administrative expenses for the nine months ended September 30, 2022 and 2021 were $17.9 million and $16.9 million, respectively. General and administrative expenses in the three months ended September 30, 2022 were higher than the corresponding period in 2021 due to the hiring of additional personnel to support our corporate growth. General and administrative expenses in the nine months ended September 30, 2022 were higher than the corresponding period in 2021 due to the hiring of additional personnel, partially offset by a reduction in legal and consulting expenses.
Net loss for the three months ended September 30, 2022 and 2021 was $24.5 million and $22.7 million, or ($0.28) and ($0.33) per share, respectively. Net loss for the nine months ended September 30, 2022, and 2021 was $79.4 million and $63.5 million, or ($0.90) and ($0.92) per share, respectively. Net loss in the three months ended September 30, 2022 was higher than the corresponding period in 2021 due primarily to an increase in interest expense accretion related to the Abingworth development financing agreement, partially offset by a decline in research and development expense. Net loss in the nine months ended September 30, 2022 was higher than the corresponding period in 2021 due primarily to an increase in interest expense accretion and to a lesser extent, an increase in research and development and other operating expenses associated with the ongoing late-stage development of seladelpar in PBC. Overall, we expect operating expenses to increase in the future as we continue to execute on our development plans for seladelpar in PBC.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss third quarter financial results and provide a business update. To access the live conference call, please dial 855-327-6837 from the U.S. and Canada, or 631-891-4304 internationally, Conference ID #10020554. 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