On November 10, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported financial results and a business update for the third quarter ended September 30, 2021 (Press release, VolitionRX, NOV 10, 2021, View Source [SID1234595101]). Volition management will host a conference call tomorrow, November 11 at 8:00 a.m. U.S. Eastern Time to discuss these results. Conference call details may be found below.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We have enjoyed an exciting quarter navigating the due diligence process and negotiations with three multi-national companies to advance term sheets for Nu.Q Vet licensing and distribution arrangements and anticipate signing a term sheet with one of those parties before the end of 2021," commented Cameron Reynolds, President and Chief Executive Officer of Volition. "We have also made progress in other key areas including our Nu.Q NETs and Nu.Q Capture programs as we shift gears towards our goal of becoming a commercial company with products."

Watch an interview with Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, Cameron Reynolds, President and Chief Executive Officer of Volition, and Terig Hughes, Chief Financial Officer of Volition: View Source

Company Highlights

Volition Veterinary

In advanced term sheet negotiations with three top global veterinary companies and anticipate signing a term sheet with one of those parties before the end of 2021.
Published two abstracts at the Veterinary Cancer Society Annual Conference including:
the first data published in relation to what will likely be the second Nu.Q Vet product, a disease monitoring tool, on track for a planned launch in 2022; and
the first study using Nu.Q Capture as an enrichment tool in the plasma of dogs with lymphoma.
Financial

Cash and cash equivalents as of September 30, 2021, totalled approximately $22.9 million compared with $19.4 million as of December 31, 2020.
Cash burn rate averaged approximately $1.7 million per month, lower than the rate during the previous two quarters and also below the Company’s expectations.
Net loss for the third quarter of 2021 was $7.2 million versus $5.6 million the prior quarter with this increase primarily due to non-cash items.
Personnel/ Operational

Dr. Gaetan Michel, Chief Operating Officer, has relocated to the U.S. and is tasked with expanding the team with roles expected to be filled in the quality, regulatory and manufacturing functions.
On November 1, Mr. Nick Plummer joined the Company as Group General Counsel.
Intellectual Property

27 patent families (plus three in-licensed families) covering both human and animal use of Volition’s Nucleosomics platform.
82 granted patents (12 in the U.S., 14 in Europe and 56 rest of world).
82 patents pending.
Continued focus on filings during the third quarter of 2021 and expect portfolio to grow in the quarters and years ahead.
Publications

Volition’s list of publications and abstracts continues to grow.
Year to date data for Nu.Q has been presented at four international conferences and Volition has collaborated on four clinical papers.
These publications are another very important step forward for the Company.
Clinical – NETosis including COVID-19

Volition believes the Nu.Q NETs assay will have wide applicability for monitoring diseases with a NETs component (such as COVID-19, influenza, sepsis, autoimmune diseases and cancer) and potentially to risk stratify patients for treatment selection.
Volition expects to register CE marks on its first NETs product, across multiple platforms including ELISA plate, automated beads and a proof of concept on very high throughput platform by the summer of 2022.
Volition intends to register the Nu.Q NETs product with a broad almost C-reactive protein (C.R.P.) style claim "for the detection and evaluation of infection, tissue injury, inflammatory disorders and diseases associated with NETosis".
Posters published this quarter (at the International Society on Thrombosis and Haemostasis Congress) showed that results on admission using the same Nu.Q NETs assay could predict future COVID-19 disease severity and that serial results correlate with disease progression including 28-day mortality.
The Company has further large studies completed in COVID-19 and sepsis which are now awaiting the finalization of data and publication, as well as studies in other diseases in progress with results expected in the coming quarters.
Clinical – Cancer

The Company has completed preliminary analysis of the colorectal cancer studies (both asymptomatic and symptomatic populations) conducted with the National Taiwan University and has submitted these findings for presentation at a conference in early 2022.
The Company has completed preliminary analysis of the lung cancer study also conducted with the National Taiwan University and looks forward to reviewing the data with Professor Chen and his team ahead of publication either through a clinical paper or conference abstract.
Volition has also been in active and continuing negotiations in Asia this year in addition to platform development on its first human cancer launch in China.
Collection for the U.S. EDRN study restarted in June and enrolment has been slow but steady. The EDRN have diverted some efforts to drive recruitment and the Company anticipates study completion in the fourth quarter of 2022.
With regards to Volition’s U.S. blood cancer studies, the timing of expected completion for each has been impacted by the COVID-19 pandemic due to sample collection and protocol issues.
Given the pandemic delays in the larger NHL study (of 1500 subjects) the Company has taken the opportunity to alter the study protocol and are in the process of upgrading the platform to a high-throughput platform which will help facilitate an FDA compliant product. Consequently, Volition now expects this study to initiate recruitment in the first quarter of 2022 with study completion anticipated in 2023.
Upcoming Milestones

Volition expects to achieve the following milestones during the remainder of 2021 and beyond, pandemic permitting:

Drive revenue in the coming quarters in the following key areas:
Licensing of its technology, with a particular but not exclusive focus on Nu.Q Vet, with the aim of signing the first term sheet this year,
Processing samples at Silver One using its Nu.Q Discovery assays, and
Sales of its disease monitoring tests (e.g. COVID-19, sepsis).
Continue to progress the research program for the use of Nu.Q in NETosis, in monitoring disease progression of COVID-19, sepsis and potentially other diseases and as a possible companion diagnostic for a treatment for sepsis.
Continue to advance its previously announced large-scale blood, lung and colorectal cancer trials in Europe, Asia and the U.S.
Publish several abstracts and peer-reviewed scientific papers with clinical results showing the robustness and utility of its Nu.Q platform.
Advance the development of Nu.Q Capture.
Continue to file patents to expand and extend its intellectual property portfolio.
VolitionRx Limited Third Quarter 2021 Financial Results and Business Update

Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, Chief Financial Officer of Volition, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Scott Powell, Executive Vice President, Investor Relations of Volition.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source

In addition, a telephone replay of the call will be available until November 25, 2021. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13725016.

On November 10, 2021 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported financial results for the third quarter and nine months ended September 30, 2021 (Press release, RAPT Therapeutics, NOV 10, 2021, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-reports-third-quarter-2021-financial-results [SID1234595100]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This has been an important year for RAPT as we advance the development of RPT193 in atopic dermatitis and FLX475 in cancer," said Brian Wong, M.D., Ph.D., President and Chief Executive Officer of RAPT Therapeutics. "In the third quarter, we presented additional incremental data for RPT193 at two separate dermatology meetings, allowing us to share our early positive data in atopic dermatitis with key members of the medical community. We are well positioned to advance RPT193 into Phase 2 clinical trials in atopic dermatitis and asthma in 2022. In addition, we have begun to focus development of FLX475 in key indications showing early promise, including EBV+ lymphoma, nasopharyngeal cancer and head and neck cancer. Our goal is to report data from ongoing cohorts in the Phase 1/2 trial for FLX475 at a medical meeting in 2022."

Financial Results for the Third Quarter Ended September 30, 2021

Third Quarter ended September 30, 2021

Net loss for the third quarter of 2021 was $18.7 million, compared to $14.6 million for the third quarter of 2020.

Research and development expenses for the third quarter of 2021 were $15.7 million, compared to $12.9 million for the same period in 2020. This increase was primarily due to increased clinical trial costs for FLX475 and RPT193 and increases in stock-based compensation, personnel costs and facilities costs.

General and administrative expenses for the third quarter of 2021 were $3.8 million, compared to $3.2 million for the same period of 2020. The increase was primarily due to increases in stock-based compensation expense, insurance expense, personnel costs and facilities costs.

Nine Months Ended September 30, 2021

Net loss for the nine months ended September 30, 2021 was $51.3 million, compared to $40.2 million for the same period in 2020.

Research and development expenses for the nine months ended September 30, 2021 were $42.7 million, compared to $34.6 million for the same period in 2020. The increase was primarily due to increases in costs related to the clinical trials of FLX475 and RPT193, as well as increases in stock-based compensation, personnel expenses, facilities costs and laboratory supplies spend.

General and administrative expenses for the nine months ended September 30, 2021 were $11.5 million, compared to $9.3 million for the same period of 2020. The increase in general and administrative expenses was primarily due to increases in stock-based compensation expense, insurance expense and personnel costs.

As of September 30, 2021, the Company had cash and cash equivalents and marketable securities of $210.8 million.

On November 10, 2021 Imago BioSciences, Inc. ("Imago") (Nasdaq: IMGO), a clinical-stage biopharmaceutical company discovering new medicines for the treatment of myeloproliferative neoplasms (MPNs), reported financial results for the third quarter ended September 30, 2021 and highlighted recent corporate updates (Press release, Imago BioSciences, NOV 10, 2021, View Source [SID1234595099]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Over the third quarter we have built upon the momentum of our recent initial public offering and concurrent private placement that resulted in gross proceeds of $174.6 million to advance our novel LSD1 inhibitor, bomedemstat (IMG-7289), for the treatment of hematologic diseases of the bone marrow. We are also pleased to have been added to the Russell 2000 Index in late September, which has driven greater market awareness of Imago," said Dr. Hugh Y. Rienhoff, Jr., M.D, Chief Executive Officer of Imago BioSciences. "We are thrilled to have completed enrollment in our Phase 2 trial of bomedemstat for the treatment of myelofibrosis and look forward to providing data updates on this Phase 2 trial, along with our Phase 2 trial of bomedemstat for the treatment of essential thrombocythemia this year."

Recent Corporate Developments and Pipeline Updates

Announced Oral Data Presentations at the Upcoming 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. In November 2021, Imago announced that two abstracts have been accepted for oral presentation at the ASH (Free ASH Whitepaper), to be held December 11-14, 2021: "A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (bomedemstat) for the Treatment of Advanced Myelofibrosis"; and "A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (bomedemstat) for the Treatment of Essential Thrombocythemia (ET)".
Russell 2000 Index Inclusion: In September 2021, Imago announced its inclusion in the Russell 2000 Index as part of the Index’s quarterly IPO additions.
Granted Orphan Drug Designation for Bomedemstat in Essential Thrombocythemia (ET) from the European Medicines Agency (EMA): In July 2021, Imago announced that its lead asset, bomedemstat, was granted Orphan Drug Designation by the EMA for the treatment of ET. Under Orphan Drug Designation, bomedemstat potentially qualifies for incentives such as 10 years of market exclusivity, reduced fees for regulatory activities, and protocol assistance.
Successful Completion of Initial Public Offering (IPO): In July 2021, Imago completed an initial public offering and concurrent private placement with Pfizer resulting in gross proceeds of $174.6 million.
Third Quarter 2021 Financial Results

Cash and Cash Equivalents: As of September 30, 2021, Imago had cash, cash equivalents, restricted cash and short-term investments of $230.4 million.
Research & Development (R&D) Expenses: R&D expenses for the quarter ended September 30, 2021 were $8.7 million (including stock-based compensation expense of $0.2 million) as compared to $3.6 million for the same period in 2020. The overall increase in R&D expenses was related to the continuation of the Phase 2 clinical studies in ET and MF, as well as startup costs for a Phase 2 extension study to accommodate those patients who wish to continue to receive bomedemstat, continued development of commercial material and material to support the ongoing and new clinical trials, and salaries and non-cash stock-based compensation expense for R&D employees as we ramped up our operations.
General and Administrative (G&A) Expenses: G&A expenses for the quarter ended September 30, 2021 were $3.0 million (including stock-based compensation expense of $0.5 million) as compared to $0.7 million for the same period in 2020.
Net Loss: Net loss for the quarter ended September 30, 2021 was $11.7 million compared to $4.3 million for the same period in 2020.

On November 10, 2021 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage precision oncology company developing targeted drugs to inhibit frontier targets that drive and sustain RAS-addicted cancers, reported its financial results for the third quarter and nine months ended September 30, 2021, and provided a corporate update (Press release, Revolution Medicines, NOV 10, 2021, View Source [SID1234595098]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the third quarter, the company’s unique capabilities and continuing momentum with our innovative RAS(ON) Inhibitors and RAS Companion Inhibitors strengthened Revolution Medicines’ leadership position in the quest to successfully treat RAS-addicted cancers. In our RAS(ON) Inhibitor portfolio, development candidates targeting KRASG12C (RMC-6291) and RASMULTI (RMC-6236) have shown compelling features and continue to advance toward the clinic. Exciting mutant-selective inhibitors of KRASG13C and KRASG12D have also emerged from our RAS(ON) drug discovery platform, and we expect to select another development candidate by year end," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines.

"We have expanded the evaluation of our SHP2 inhibitor, RMC-4630, in combination regimens with direct inhibitors of RAS. In particular, the company recently began recruiting patients in our RMC-4630-03 study under our global partnership with Sanofi and in collaboration with Amgen. This study is intended to further evaluate RMC-4630 and Lumakras in the treatment of lung cancer as a complement to the ongoing CodeBreaK 101c study of these compounds. In addition, we are gratified that Sanofi plans to sponsor a study of RMC-4630 in combination with Mirati’s KRASG12C inhibitor, adagrasib, also under our partnership."

RAS(ON) Inhibitors – Revolution Medicines continues to build on its innovative RAS(ON) Inhibitor platform, producing an expansive collection of tri-complex inhibitors targeting diverse oncogenic RAS variants through highly differentiated chemical and pharmacologic profiles.

RMC-6291 (KRASG12C) – RMC-6291 is a first-in-class, potent, oral and selective tri-complex inhibitor of KRASG12C(ON) with a differentiated preclinical profile designed to address persistent unmet needs for patients with cancers driven by KRASG12C.
The company recently reported data at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) demonstrating superior outcomes with orally administered RMC-6291 as compared to adagrasib in preclinical models of KRASG12C non-small cell lung cancer (NSCLC). In a head-to-head mouse clinical trial of 19 xenograft models, RMC-6291 demonstrated robust anti-tumor activity characterized by a 68% objective response rate, as compared to 42% for adagrasib in the same models. The company believes these findings support the potential for RMC-6291 as a best-in-class KRASG12C inhibitor.
The company remains on track to submit an Investigational New Drug application (IND) for RMC-6291 in the first half of 2022.
RMC-6236 (RASMULTI) – RMC-6236 is a first-in-class, potent, oral RAS-selective tri-complex, RASMULTI(ON) inhibitor designed to treat cancers driven by a variety of KRAS mutations, including those that have emerged in patients following treatment with KRASG12C(OFF) inhibitors.
The company reported data at the AACR (Free AACR Whitepaper)-NCI-EORTC Conference demonstrating RMC-6236’s ability to induce significant regressions of NSCLC, pancreatic cancer and colorectal cancer (CRC) xenograft models driven by KRASG12V, KRASG12D, KRASG12C or KRASG12R. These findings add to the growing body of evidence that the company believes supports the potential of RMC-6236 as a first targeted therapy for treating many cancers for which no targeted treatment is currently available.
The company presented an expanded dataset at the Gastrointestinal Cancer Drug Development Summit demonstrating the ability of both RMC-6236 and RMC-6291 to drive tumor regressions in xenograft models of RAS-mutant CRC. The company believes these findings provide additional support for the potential of RAS(ON) inhibitors to deliver clinical benefit to patients with CRC.
Also during the AACR (Free AACR Whitepaper)-NCI-EORTC Conference, the company presented preclinical data from a combination study evaluating RMC-6236 with a PD-1 inhibitor. Results from this study demonstrated that RMC-6236 alone induces anti-tumor immunity in vivo, and also exhibits additive anti-tumor benefit with checkpoint inhibition as indicated by complete and durable responses.
The company remains on track to submit an IND for RMC-6236 in the first half of 2022.
Continued expansion of other RAS(ON) Inhibitor programs – Revolution Medicines continues to progress its growing portfolio of orally bioavailable, mutant-selective RAS(ON) Inhibitors designed to target RAS variants driving RAS-addicted cancers that are unserved by targeted drugs.
The company reported data at the AACR (Free AACR Whitepaper)-NCI-EORTC Conference showing the capacity of compounds from both its KRASG13C and KRASG12D programs to covalently and selectively modify their respective targets, the first compounds described with these properties. Covalent modification of KRASG12D was also demonstrated in vivo in a tumor xenograft model of KRASG12D-driven pancreatic cancer, with tumor regressions achieved following repeat oral dosing.
The company remains on track to nominate a third development candidate from its RAS(ON) inhibitor portfolio in 2021.
RAS Companion Inhibitors – Revolution Medicines continues to advance and expand multiple clinical studies of its RAS Companion Inhibitors designed to provide maximum clinical benefit in RAS-addicted cancers.

RMC-4630 (SHP2 Inhibitor) – RMC-4630, a potent, oral, selective inhibitor of the SHP2 protein, is being advanced in partnership with, and is primarily funded by, Sanofi.
RMC-4630 and KRASG12C inhibitor Lumakras (sotorasib)
Amgen’s CodeBreaK 101c study continues evaluating RMC-4630 in combination with sotorasib across multiple cancer types. To date this combination has demonstrated acceptable tolerability.
The company is actively recruiting patients for its global Phase 2 clinical trial evaluating the combination of RMC-4630 and sotorasib in patients with advanced NSCLC bearing the KRASG12C mutation. Revolution Medicines is sponsoring the RMC-4630-03 study under its global partnership with Sanofi and conducting the trial in collaboration with Amgen. The RMC-4630-03 study evaluating the combination in NSCLC patients is a complement to Amgen’s ongoing CodeBreaK 101c study exploring this combination across multiple cancer types. The company expects to have preliminary findings from the RMC-4603-03 study in the second half of 2022.
RMC-4630 and KRASG12C inhibitor adagrasib
Sanofi, the company’s global SHP2 development and commercialization partner, plans to sponsor a combination study under its global SHP2 partnership with the company evaluating RMC-4630 (also known as SAR442720) in combination with Mirati’s KRASG12C inhibitor, adagrasib. This study expands the evaluation of the potential benefit of adding RMC-4630 in this class of RAS inhibitors.
RMC-4630 and PD-1 inhibitor pembrolizumab (Keytruda)
The TCD16210 study sponsored by Sanofi continues evaluating RMC-4630 in combination with pembrolizumab, a PD-1 inhibitor. Sanofi is planning an expansion cohort with this combination in front-line PD-L1+ NSCLC.
RMC-5552 (mTORC1/4EBP1 Inhibitor) – RMC-5552 is a potent, selective bi-steric inhibitor of mTORC1 that suppresses phosphorylation and inactivation of 4EBP1.
Enrollment and dosing are underway in a Phase 1 monotherapy dose-escalation study. The company continues to expect initial safety, pharmacokinetic and single agent activity data in 2022.
The company intends to evaluate RMC-5552 in combination with RAS inhibitors for the treatment of tumors driven by co-occurring RAS mutations and genomic activation of the mTORC1 pathway.
RMC-5845 (SOS1 Inhibitor) – RMC-5845 is a potent, selective, oral inhibitor of SOS1, a major switch in the cycling of RAS(OFF) to RAS(ON).
The company expects RMC-5845 to be IND-ready by the end of 2021.
Third Quarter 2021 Financial Highlights

Cash Position: Cash, cash equivalents and marketable securities were $608.7 million as of September 30, 2021, compared to $440.7 million as of December 31, 2020. The increase was primarily due to proceeds from the company’s public equity offering in February 2021.

Revenue: Total revenue consists of revenue from the company’s collaboration agreement with Sanofi. During the third quarter of 2021, the company recorded a non-cash, non-recurring GAAP accounting adjustment that reduced collaboration revenue by $8.5 million. This non-cash revenue adjustment relates to the addition of the RMC-4630-03 study to the RMC-4630 development plan, and deprioritization of the RMC-4630-02 study. As a result of these development plan events, the company revised its estimate of the accounting transaction price and percentage of completion of work performed to date under its agreement with Sanofi, which resulted in a cumulative catch-up adjustment to collaboration revenue in this quarter. Total revenue, including the non-cash revenue adjustment, was $1.1 million for the quarter ended September 30, 2021, compared to $12.7 million for the quarter ended September 30, 2020. The decrease was primarily due to the non-cash revenue adjustment and lower reimbursed manufacturing costs.

R&D Expenses: Research and development expenses were $46.5 million for the quarter ended September 30, 2021, compared to $34.9 million for the quarter ended September 30, 2020. The increase was primarily due to an increase in research expenses associated with the company’s pre-clinical research portfolio, an increase in personnel-related expenses related to additional headcount, and an increase in stock-based compensation.

G&A Expenses: General and administrative expenses were $7.8 million for the quarter ended September 30, 2021, compared to $5.3 million for the quarter ended September 30, 2020. The increase was primarily due to an increase in stock-based compensation, an increase in personnel-related expenses related to additional headcount, and higher legal and accounting fees.

Net Loss: Net loss was $52.9 million for the quarter ended September 30, 2021, compared to a net loss of $27.2 million for the quarter ended September 30, 2020.

2021 Financial Guidance

Revolution Medicines continues to expect full year 2021 GAAP net loss to be between $170 million and $190 million, which includes estimated non-cash stock-based compensation expense of approximately $20 million.

Conference Call

Revolution Medicines will host a conference call and webcast this afternoon, November 10, 2021, at 4:30 PM Eastern (1:30 PM Pacific).

To listen to the conference call, please dial (833) 423-0425 or (918) 922-3069, provide conference ID: 9757524 and request the Revolution Medicines conference call. To listen to the live webcast, or access the archived webcast, please visit: View Source Following the live webcast, a replay will be available on the Company’s website for at least 14 days.

On November 10, 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 third quarter ended September 30, 2021 (Press release, CymaBay Therapeutics, NOV 10, 2021, View Source [SID1234595097]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sujal Shah, President and CEO of CymaBay, stated, "The third quarter of 2021 included key accomplishments that position CymaBay for long-term success. The non-dilutive, risk-sharing development financing agreement signed with Abingworth in July supports the completion of our ongoing phase 3 program for seladelpar in PBC. We now have over 100 sites activated across over 20 countries in our global registration study, RESPONSE, and expect these efforts to continue and ultimately drive the completion of enrollment in the first half of 2022. We are excited to also share additional data from our prior studies in PBC at The Liver Meeting 2021 that demonstrate improved benefit of seladelpar between one to two years of treatment and differentiation from other existing treatments in a population of patients with more advanced disease."

"As we approach the end of 2021, we are well positioned to deliver on our central focus of improving the lives of patients with PBC. Although the pandemic has presented our entire industry with evolving challenges, I am confident we have assembled talent across the organization that will allow us to achieve both our near term goals and our vision of establishing a pipeline of opportunities for patients with unmet need. I’m also proud of the relationships we have established with patient communities, the medical community and high quality investors that all support the work we are dedicated to day in and day out."

Upcoming and Recent Corporate Highlights

An oral presentation titled "Long-Term Safety and Efficacy of Seladelpar in Patients with Primary Biliary Cholangitis" will be delivered by Dr. Marlyn J. Mayo, MD, Professor and Liver Disease Specialist, University of Texas Southwestern Medical Center. This presentation will highlight the efficacy and safety of seladelpar during 2 years of treatment in patients with primary biliary cholangitis (PBC). The mean percent change in alkaline phosphatase (ALP) from baseline was -42% and -50% after 1 and 2 years, respectively. Over 2 years, there were sustained reductions in ALT, AST, and GGT. Total bilirubin and platelet levels remained stable. Seladelpar appeared safe and well-tolerated. These data support that long-term treatment with seladelpar resulted in continued improvement in markers of cholestasis after 1 year.
A clinical presentation titled "Efficacy and Safety of Seladelpar in Patients with Compensated Cirrhosis and Evidence of Portal Hypertension due to Primary Biliary Cholangitis" will be delivered by Dr. Cynthia Levy, MD, Professor of Medicine, University of Miami. This electronic poster presentation will highlight the treatment effects of seladelpar in compensated cirrhotic patients with portal hypertension after 3 months, which led to ALP changes of -30% in the 5 mg and -45% in the 10 mg groups. Total bilirubin, platelets, albumin, and INR either improved or remained stable. Seladelpar appeared safe and well-tolerated. Efficacy, safety, and tolerability in patients with compensated cirrhosis and portal hypertension was comparable to that of non-cirrhotic patients.
CymaBay will host a Post-AASLD KOL Call on seladelpar in PBC on Monday, November 15, at 4:30pm ET. The webinar will feature presentations by Dr. Marlyn J. Mayo, MD, and Dr. Cynthia Levy, MD, who will be reviewing the seladelpar abstracts presented at The Liver Meeting 2021. Dr. Dennis Kim, MD, Chief Medical Officer of CymaBay, will also discuss progress developing seladelpar for patients with PBC. The live and archived webcast will be accessible through this webcast link or through the Events section of the CymaBay website.
Continued enrollment in 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.
Continued enrollment in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration.
Supported enrollment efforts 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. 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.
Executed a non-dilutive financing transaction with Abingworth LLP 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. The first installment of $25 million was received in August and the second installment of $25 million was received in early November. CymaBay expects to receive the third $25 million installment in early February 2022 and 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.
Held $113.8 million in cash, cash equivalents and short-term investments as of September 30, 2021. We believe that cash and investments on hand, together with additional funding amounts we expect to receive through the development financing agreement with Abingworth are sufficient to fund CymaBay’s 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 and other variants 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.
Third Quarter and Nine Months Ended September 30, 2021 Financial Results

Research and development expenses for the three months ended September 30, 2021 and 2020 were $17.0 million and $7.7 million, respectively. Research and development expenses for the nine months ended September 30, 2021 and 2020 were $46.1 million and $25.2 million, respectively. Research and development expenses in the three and nine months ended September 30, 2021 were higher than the corresponding periods in 2020 primarily due to an increase in clinical trial activities associated with the development of seladelpar in PBC. In particular, cost increases were primarily driven by an expansion of our site activation, patient enrollment, and other clinical trial activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC. In the three and nine months ended September 30, 2020, costs incurred were primarily associated with startup activities for RESPONSE and ASSURE as well as costs to shutdown 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 September 30, 2021 and 2020 were $5.2 million and $3.9 million, respectively. General and administrative expenses for the nine months ended September 30, 2021 and 2020 were $16.9 million and $11.5 million, respectively. General and administrative expenses in the three and nine months ended September 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. In addition, general and administrative expenses during the three and nine months ended September 30, 2020, included restructuring charges of $0.6 million and $0.7 million, respectively, and no similar restructuring charges were incurred during the three and nine months ended September 30, 2021.

Net loss for the three months ended September 30, 2021 and 2020 was $22.7 million and $11.4 million, or ($0.33) and ($0.17) per diluted share, respectively. Net loss for the nine months ended September 30, 2021 and 2020 was $63.5 million and $35.2 million, or ($0.92) and ($0.51) 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 the future 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 third 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# 13723498. 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