On November 7, 2023 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, reported corporate updates and financial results for the third quarter ended September 30, 2023 (Press release, CymaBay Therapeutics, NOV 7, 2023, View Source [SID1234637130]).

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Sujal Shah, President and CEO of CymaBay, stated, "This past quarter has been a momentous one for CymaBay where we achieved multiple significant milestones advancing us towards our goal of improving lives of people living with PBC. The consistency and depth of the clinical data set generated from Phase 2, ENHANCE and now RESPONSE demonstrates that seladelpar has the potential to be the first ever approved treatment for patients with PBC to significantly reduce both markers related to the risk of disease progression and symptoms. I am incredibly proud of the team here at CymaBay and commend them for the progress they helped achieve and equally grateful to patients and patient advocacy groups, their caregivers and our investigators for their partnership and support. We are eager to continue the positive momentum and are working diligently on our near-term milestones for seladelpar."

Corporate Updates:

On September 7, 2023, we announced topline results from our seladelpar Phase 3 RESPONSE study. The study evaluated the safety and efficacy of seladelpar for the treatment of PBC. The trial achieved the primary and all key secondary endpoints.

Primary composite endpoint at 12 months of serum alkaline phosphatase and bilirubin was met by 61.7% of patients treated with seladelpar 10 mg vs. 20.0% of placebo treated patients (p<0.0001)
Normalization of alkaline phosphatase at 12 months was achieved by 25.0% of patients treated with seladelpar vs. 0% on placebo (p<0.0001)
In patients having moderate-to-severe itch at baseline, the seladelpar treated group improved their pruritus at 6 months compared to those in the placebo group (p<0.005)
Overall safety and tolerability were comparable between placebo and seladelpar groups and consistent with previous studies
Treatment-emergent adverse events, serious adverse events, and patient discontinuations were generally balanced across the treatment and placebo arms. There were no treatment-related serious adverse events in the study.
On October 23, 2023, we announced that the U.S. Food and Drug Administration (FDA) has revised the originally granted Breakthrough Therapy Designation for seladelpar to now reflect treatment of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) and are inadequate responders to or intolerant to UDCA. Seladelpar is the only potent, selective, orally active PPARδ agonist, or delpar, with Phase 3 results demonstrating a statistically significant improvement in PBC-related cholestatic pruritus.
A late-breaking presentation highlighting results from the RESPONSE Phase 3 study of seladelpar in patients with PBC will be presented at The Liver Meeting of the American Association for the Study of Liver Diseases (AASLD), in Boston, MA (November 10th – 14th).
Announced the initiation of AFFIRM, a randomized, placebo-controlled confirmatory study to evaluate the effect of seladelpar on clinical outcomes in patients with compensated cirrhosis due to PBC. The AFFIRM study is planned to enroll approximately 192 patients with PBC who have compensated cirrhosis (Child-Pugh A or Child-Pugh B) based on prespecified clinical criteria. Patients will be randomly assigned using a 2:1 ratio to oral, once daily seladelpar or placebo for a fixed duration of three years. The primary outcome measure is the time from start of treatment to the first occurrence of clinical events (all-cause death, liver transplant, hospitalization for other serious liver-related events, and progression to Child-Pugh C decompensated cirrhosis). Additional key outcomes include overall survival, liver transplant-free survival, and time to hospitalization for serious liver-related events.
Continued enrollment in ASSURE, an 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 300 patients in this study taking seladelpar daily, including those from our prior studies of seladelpar and patients who have completed RESPONSE.
Findings from post-hoc analysis of the Phase 3 ENHANCE study of seladelpar for the treatment of PBC, showing baseline intensity of patient-reported pruritus was associated with higher levels of serum IL-31 was presented at American College of Gastroenterology (ACG), by Professor Andreas E. Kremer, MD, Ph.D., MHBA, a leading authority in cholestatic pruritus from the University of Zurich. Featured results included novel aspects of the anti-pruritic and anti-cholestatic mechanisms of seladelpar, CymaBay’s first-in-class oral, selective PPARδ agonist, or "delpar," being investigated for the treatment of patients with PBC.
Completed an upsized public equity offering in September 2023, in which we sold 14,521,307 shares of common stock at $17.13 per share and pre-funded warrants to purchase 583,771 shares of common stock at $17.1299 per underlying share. Net proceeds of the offering were $242.8 million, after deducting the underwriting discount and other offering expenses.
The Phase 2a proof-of-pharmacology study to assess whether MBX-2982 can enhance glucagon secretion during insulin-induced hypoglycemia in subjects with type 1 diabetes (T1D) has been completed. The study found that there was no change in glucagon secretion during clamps in subjects with T1D dosed with MBX-2982 versus placebo. In contrast, healthy volunteers showed a glucose dependent increase in glucagon during hypoglycemia. Further, target engagement by MBX-2982 was demonstrated by increases in GLP-1 levels in subjects with T1D. While disappointing, the results demonstrate a well-designed and executed study that demonstrated pharmacodynamic action of MBX-2982 but without demonstrating the pharmacology needed to benefit the T1D population. The scientific questions were answered and CymaBay is not planning any further studies with MBX-2982. We would like to thank Dr. Richard Pratley and his team at the AdventHealth Research Institute in Orlando, Florida as well as ProSciento Inc., California for conducting a well-designed and executed study and The Leona M. and Harry B. Helmsley Charitable Trust for their support of the study through a grant to AdventHealth. We are also grateful to the patients with T1D and healthy volunteers for their participation in the study.
Financial Updates:

Held $438.8 million in cash, cash equivalents and investments as of September 30, 2023. We believe that cash and investments on hand are sufficient to fund CymaBay’s operating expenses into the first half of 2026.
Third Quarter and Nine Months Ended September 30, 2023 Financial Results

Collaboration revenue recognized for the nine months ended September 30, 2023 was $31.0 million and was associated with the collaboration and license agreement with Kaken Pharmaceutical Co., Ltd. (Kaken) entered into in January 2023, to develop and commercialize seladelpar in Japan. As reported earlier, this revenue was recognized upon completion of the initial technology transfer to Kaken in the second quarter of 2023. No incremental collaboration revenue was recognized for the three months ended September 30, 2023. Of the $34.2M upfront payment received from Kaken, $2.7 million remains deferred as of September 30, 2023 and will be recognized upon completion of the CymaBay’s ongoing clinical data delivery and CMC development performance obligations.
Research and development expenses for the three months ended September 30, 2023, and 2022 were $20.0 million and $15.5 million, respectively. Research and development expenses for the nine months ended September 30, 2023 and 2022 were $58.1 million and $51.8 million, respectively. Research and development expenses for the three- and nine-month periods ended September 30, 2023 increased compared to the corresponding periods in 2022 driven by higher clinical activities supporting our clinical studies.
General and administrative expenses for the three months ended September 30, 2023 and 2022 were $12.2 million and $5.9 million, respectively. General and administrative expenses for the nine months ended September 30, 2023 and 2022 were $32.1 million and $17.9 million, respectively. General and administrative expenses for the three and nine months ended September 30, 2023 were higher than the corresponding period in 2022 driven by investments to prepare for potential commercialization of seladelpar in PBC as well as an increase in other corporate expenses.
Net loss for the three months ended September 30, 2023 and 2022 was $33.9 million and $24.5 million, or ($0.32) and ($0.28) per share, respectively. Net loss for the nine months ended September 30, 2023 and 2022 was $63.5 million and $79.4 million, or ($0.62) and ($0.90) per share, respectively. Net loss for the three months ended September 30, 2023 was higher than the three months ended September 30, 2022 primarily due to higher operating expenses. Net loss for the nine months ended September 30, 2023 was lower than the corresponding periods in 2022 due primarily to the recognition of $31.0 million of collaboration revenue related to the Kaken upfront payment during the second quarter of 2023 and higher interest income earned on our investments and other income due to refundable tax credits, offset in part by an increase in operating expenses. Overall, we expect operating expenses to increase in the future as we continue to support our ongoing drug development activities and expand on initiatives to prepare for potential commercialization of 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 1-877-407-0784 from the U.S. and Canada, or 1-201-689-8560 internationally, Conference ID #13740701. 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 November 7, 2023 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported that its product portfolio of FDA-approved brands delivered combined revenues of $10.1 million during the third quarter of 2023 and $30.2 million year to date (Press release, Cumberland Pharmaceuticals, NOV 7, 2023, View Source [SID1234637129]).

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Net loss for the third quarter was $1.0 million, while year-to-date net income was $15,086. Adjusted earnings were $260,146 for the third quarter and $4.2 million, or $0.29 a share, year to date. Cash flow from operations during 2023 has totaled $5.1 million.

The company ended the third quarter with $88 million in total assets, $52 million in total liabilities, and $36 million of shareholders’ equity.

"During 2023 our team has been working diligently to advance our long-term business strategy, building our brands and progressing our clinical programs," said Cumberland Pharmaceuticals CEO A.J. Kazimi. "Moreover we have refined our mission statement, to reflect our collaborative efforts and patient focus – as we provide unique products that improve the quality of their care."
Cumberland will report its full third quarter 2023 financial results and provide a company update via a conference call today at 4:30 p.m. Eastern Time.

Recent Company developments include:

New Bank Credit Facility
On September 5, 2023, Cumberland entered into a new Revolving Credit Loan Agreement with Pinnacle Bank for a three-year term. The agreement provides for an aggregate principal funding amount of up to $25 million. It provides an initial revolving credit line with $20 million of availability, and the ability of Cumberland to increase the amount to $25 million under certain conditions. The interest rate is based on Benchmark (Term SOFR) plus a spread of 2.75%, and Cumberland is subject to one financial covenant, the maintenance of a Funded Debt Ratio, determined on a quarterly basis.

New Vibativ Pediatric Study Publication

In October 2023, Cumberland announced a new publication in Antimicrobial Agents and Chemotherapy detailing the results of the first clinical study investigating the safety and pharmacokinetics of its Vibativ injection in children 2 to 17 years of age. Vibativ is an intravenous antibiotic approved by the FDA for the treatment of hospital-acquired and ventilator-associated bacterial pneumonia as well as complicated skin and skin structure infections caused by certain gram-positive bacteria in adults.

This is the first reported study evaluating Vibativ in pediatric patients. The results of the study suggest that a single dose of Vibativ is safe in children and they experience reduced exposure to Vibativ, compared with the same body weight-based dosing in adults.

Federal NOPAIN Act

Cumberland expects its Caldolor injection, a non-opioid analgesic product, will be eligible for special Medicare reimbursement under the Non-Opioids Prevent Addiction in the Nation Act (the "NOPAIN Act"), which was enacted as part of the Consolidated Appropriations Act of 2023.
The NOPAIN Act requires Medicare to provide separate reimbursement for non-opioid products that are used to manage pain during surgeries conducted in hospital outpatient departments or in ambulatory surgical centers. The NOPAIN Act applies, in part, to products that are indicated to provide analgesia without acting upon the body’s opioid receptors.
The reimbursement for non-opioid pain alternatives under the NOPAIN Act will apply to those products that are furnished between January 1, 2025 and January 1, 2028. It is anticipated that in 2024, the Centers for Medicare & Medicaid Services will issue regulations implementing the NOPAIN Act and detailing the conditions for, and amount of, the separate reimbursement. Cumberland submitted a comment letter along with clinical information in the third quarter, explaining why Caldolor should be included and separately reimbursed.
Caldolor is approved by the FDA for use in adults and pediatric patients 3 months and older for the management of mild to moderate pain as a sole therapy, and for the management of moderate to severe pain as an adjunct to an opioid. A series of published clinical studies have demonstrated that Caldolor significantly reduces patient pain, while also significantly reducing patients’ need for opioids.
Sancuso Acquisition and Approval of New Manufacturing Plant
Early last year, Cumberland acquired the U.S. rights to the FDA-approved oncology-supportive care medicine Sancuso from Kyowa Kirin, Inc., the U.S. affiliate of Japan-based Kyowa Kirin Co., Ltd.
Sancuso is the first and only FDA-approved prescription patch for the prevention of nausea and vomiting in patients receiving certain types of chemotherapy treatment.
As of September 2023, the transition of Sancuso to Cumberland is complete. In late 2022, the FDA approved moving the product’s manufacturer to a new facility, and the production of supplies for Cumberland at that plant is now underway during the fourth quarter of 2023.
Vaprisol Supply Update
Cumberland continues to work with its new manufacturing partner for Vaprisol, Nephron Pharmaceuticals, to provide interim supplies of a special compounded conivaptan product to the market in support of critically ill patients. The companies will share in the sales of this compounded product, which has been successfully manufactured and expected to be available by the end of 2023. Cumberland then plans to file for the approval to manufacture branded Vaprisol once Nephron addresses the FDA’s Form 483 and warning letter issues.
Clinical Development Programs
Cumberland has been evaluating its ifetroban product candidate, a selective thromboxane-prostanoid receptor antagonist, in a series of clinical studies. It has been dosed in nearly 1,400 subjects and has been found to be safe and well tolerated in healthy volunteers and various patient populations.

Patient enrollment is well underway in two company sponsored Phase II clinical programs to evaluate ifetroban in Systemic Sclerosis or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs; and the Cardiomyopathy associated with Duchenne Muscular Dystrophy ("DMD"), a rare and fatal genetic neuromuscular disease that results in deterioration of the skeletal, heart and lung muscles.
Cumberland is sponsoring the FIGHT DMD trial, a multicenter, randomized, placebo-controlled Phase II study evaluating the safety, pharmacokinetics and efficacy of two doses of oral ifetroban for the treatment of the cardiomyopathy associated DMD. The trial is evaluating 12 months of oral ifetroban in 24 subjects with early-stage cardiomyopathy and 24 subjects with advanced-stage heart disease across 10 U.S. centers that specialize in DMD cardiomyopathy.
In May 2023, Cumberland announced that the FDA has cleared the Investigational New Drug Application for a Phase II study in patients with Idiopathic Pulmonary Fibrosis ("IPF"), the most common form of progressive fibrosing interstitial lung disease. As a result, the company is initiating its FIGHTING FIBROSIS trial designed to enroll 128 patients in over 20 medical centers of excellence across the U.S. This Phase II clinical trial will study the safety, tolerability and efficacy of oral ifetroban in patients with IPF. Recent studies have shown ifetroban can both prevent and enhance resolution of lung fibrosis in multiple preclinical models.
Cumberland’s plan going forward is to complete each of its company-sponsored studies, analyze their final data, announce top-line results and decide on the best development path for the registration of ifetroban, which the company continues to believe has the potential to benefit many patients with orphan diseases that represent unmet medical needs.
FINANCIAL RESULTS:
Net Revenue: For the three months ended September 30, 2023, net revenues were $10.1 million and included $3.9 million for Kristalose, $2.8 million for Vibativ, $1.9 million for Sancuso and $1.2 million for Caldolor. Year-to-date 2023 net revenues were $30.2 million and included $12.3 million for Kristalose, $6.8 million for Vibativ, $5.7 million for Sancuso and $3.3 million for Caldolor.
Operating Expenses: Total operating expenses were $12.0 million for the third quarter of 2023 and $33.6 million for the first nine months of the year.
Net Income (Loss): The net loss for the third quarter of 2023 was $1.0 million, while the year-to-date net income was $15,086.
Adjusted earnings: Adjusted earnings for the third quarter of 2023 were $260,146, or $0.02 a share and $4.2 million year to date, or $0.29 a share. The adjusted earnings calculation does not include the benefit of the $0.5 million of Vibativ cost of goods, which was received with the product acquisition. It also does not include the benefit of the $0.3 million of Sancuso cost of goods, which was received with that product’s acquisition.
Balance Sheet: At September 30, 2023, Cumberland had $87.8 million in total assets, including $18.5 million in cash and cash equivalents.
Total liabilities were $52.1 million, including $12.9 million outstanding on the Company’s revolving line of credit. Total shareholders’ equity was $36.0 million.

EARNINGS REPORT CALL:

A conference call will be held on Nov. 7, 2023, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please register at
https://register.vevent.com/register/BI9c23410ae1a342fab9f11ecec96f52fa.
Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.
A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting View Source

On November 7, 2023 Cogent Biosciences reported its quarterly report (Filing, 3 mnth, SEP 30, Cogent Biosciences, 2023, NOV 7, 2023, View Source [SID1234637128]).

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On November 7, 2023 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) reported financial results for the third quarter ended September 30, 2023 (Press release, Crinetics Pharmaceuticals, NOV 7, 2023, View Source [SID1234637127]).

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"The resounding success of paltusotine in our PATHFNDR-1 Phase 3 study for acromegaly is a significant step toward fulfilling our strategic vision of building a premier, fully integrated endocrine company that can consistently create pioneering therapies for people around the world," said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. "We intend to build additional momentum around our investigational compound paltusotine in the next few months. In December, we plan to analyze initial data from approximately half of the study participants in our ongoing Phase 2 study in carcinoid syndrome that should provide a preliminary indication of pharmacokinetic exposure, safety, and efficacy in this important second indication. We anticipate the full Phase 2 dataset from carcinoid to be available in the first half of 2024, and we remain on track to announce Phase 3 data from the PATHFNDR-2 study in the first quarter of 2024. We are enthusiastic about these upcoming key milestones for the paltusotine franchise."

THIRD QUARTER 2023 AND OPERATING HIGHLIGHTS
Phase 3 PATHFNDR-1 study met primary and all secondary endpoints. In September 2023, Crinetics reported positive topline results from its placebo-controlled Phase 3 clinical study of paltusotine in participants with acromegaly switching from standard-of-care injected depot somatostatin analogs which is designed to support an indication for the maintenance of acromegaly treatment. The study met statistical significance (p<0.0001) on the primary endpoint, based on the proportion of participants taking paltusotine (83%) who maintained an insulin-like growth factor 1 (IGF-1) level ≤ 1.0 times the upper limit of normal (xULN) compared to those taking placebo (4%). All secondary endpoints also met statistical significance as compared to placebo: change from baseline in IGF-level (p<0.0001), change from baseline in acromegaly symptoms diary (ASD) total score (p=0.02), and proportion of participants who maintained growth hormone level of <1.0 ng/mL (p=0.0003).
Completed enrollment in paltusotine’s Phase 3 PATHFNDR-2 study. PATHFNDR-2 is a placebo-controlled Phase 3 clinical study of oral paltusotine in participants with acromegaly who are treatment-naïve or not currently receiving medical therapy which is designed to support an indication for the treatment of acromegaly. The study completed enrollment of 112 participants with acromegaly who were either treatment-naïve or untreated for at least four months (Stratum 1: n=82), or who washed out of prior octreotide or lanreotide monotherapy (Stratum 2: n=30). Topline data from the study is expected in the first quarter of 2024.
Hosted Key Opinion Leader (KOL) webinar discussing acromegaly unmet need. In August 2023, Crinetics hosted a webinar featuring presentations by KOLs Beverly MK Biller, M.D. and Karen JP Liebert, R.N., BSN, both of Massachusetts General Hospital, who discussed the current landscape and unmet medical need in acromegaly, as well as the treatment burden associated with standard-of-care injectable somatostatin receptor ligands (SRLs). A replay of the webinar can be accessed here.
Paltusotine NDA submission anticipated in 2024. Pending successful data from the PATHFNDR-2 study, Crinetics plans to submit a new drug application (NDA) to the Food and Drug Administration seeking regulatory approval for both the treatment of acromegaly and maintenance of acromegaly treatment indications.
Initial data from Phase 2 study of paltusotine in carcinoid syndrome expected December 2023. The Phase 2 open-label study of paltusotine in carcinoid syndrome associated with neuroendocrine tumors is completing enrollment. A preliminary analysis from a subset of approximately half of the expected 30 participants is anticipated in December 2023. The study is designed primarily to assess the pharmacokinetic exposure and safety profile of paltusotine in participants with carcinoid syndrome. In addition, the study should provide descriptive efficacy information on the frequency of bowel movements and flushing episodes.
CRN04894 studies for Cushing’s disease and congenital adrenal hyperplasia (CAH). Based on successful Phase 1 studies demonstrating pharmacologic proof-of-concept, Crinetics is advancing clinical studies of CRN04894 in both Cushing’s disease and congenital adrenal hyperplasia. Data from the ongoing studies are expected in the second half of 2024.
Strengthened balance sheet with $350 million public offering. In September 2023, Crinetics announced the pricing of an upsized underwritten public offering of 11,441,648 shares of its common stock at $30.59 per share. Gross proceeds from the offering were $350.0 million.
THIRD QUARTER 2023 FINANCIAL RESULTS
Research and development expenses were $43.8 million for the three months ended September 30, 2023, compared to $32.0 million for the same period in 2022. The change was primarily due to an increase in personnel costs of $8.3 million, increased net spending on manufacturing and development activities of $1.8 million associated with our clinical and nonclinical programs, and increased outside services of $1.2 million.
General and administrative expenses were $15.5 million for the three months ended September 30, 2023, compared to $11.9 million for the same period in 2022. The change was primarily due to an increase in personnel costs of $3.2 million.
Net loss for the three months ended September 30, 2023, was $57.5 million, compared to a net loss of $41.9 million for the same period in 2022.
Revenues were $0.3 million for the three months ended September 30, 2023, compared to $0.5 million for the same period in 2022. Revenues in both periods were primarily derived from the paltusotine licensing arrangement with Sanwa Kagaku Kenkyusho Co., Ltd.
Unrestricted cash, cash equivalents, and investments totaled $554.7 million as of September 30, 2023, compared to $334.4 million as of December 31, 2022. Based on its current projections, the company now expects that its cash, cash equivalents and short-term investments will be sufficient to fund its current operating plan into 2026.
The company had 66,799,257 common shares outstanding as of October 31, 2023.

On November 7, 2023 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported a business update and announced financial results for the third quarter ended September 30, 2023 (Press release, Corvus Pharmaceuticals, NOV 7, 2023, View Source [SID1234637126]).

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"We continue to make progress towards initiating our Phase 3 registrational clinical trial for soquelitinib, including finalizing the study protocol and submitting it to the FDA," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "From a regulatory standpoint, we are clear to initiate the trial. We are delighted by the strong interest from leading academic centers in the U.S. in participating in the clinical trial, which is anticipated to begin enrolling patients by the second quarter 2024. We also plan to initiate a Phase 2 trial in early 2024 with soquelitinib monotherapy in recurrent renal cell cancer and have been encouraged by recent work from other academic groups demonstrating the potential of ITK inhibition in solid tumor animal models. In addition, our internal research team continues to reveal the therapeutic potential of ITK inhibition in several preclinical models of autoimmune and allergic disease, including the inhibition of Th17 cells and secretion of IL-17, which are validated mediators of inflammatory disease."

Business Update and Strategy

Prioritized Program: Soquelitinib (formerly CPI-818, Corvus’ selective ITK inhibitor)

Soquelitinib for T Cell Lymphoma

Corvus continues to enroll patients with relapsed PTCL in a Phase 1/1b clinical trial evaluating single agent therapy with soquelitinib. The latest data from the trial was reported at the International Conference on Malignant Lymphoma, which took place June 13-17, 2023 in Lugano, Switzerland. As of the May 18, 2023 cut-off date, the data showed that a majority of the patients treated with the optimal dose of 200 mg twice per day of soquelitinib experienced tumor regression.
New interim data from the Phase 1/1b clinical trial, along with complementary preclinical data, will be presented in a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2023.
Soquelitinib Preclinical Data in Hematologic and Solid Tumors

In July 2023, Corvus announced the publication of preclinical data on soquelitinib as a preprint at bioRxiv, which highlighted the selective inhibition of ITK being able to potentially enhance anti-tumor immune response to hematologic and solid tumors, indicating its potential as a novel approach to cancer immunotherapy.
In September 2023, a paper was published by an independent academic group in Scientific Reports validating the potential of ITK inhibition for treatment of solid tumors. The preclinical data demonstrated a reduction and reversal of T cell exhaustion markers and an increase in the infiltration of killer T cells into tumors, consistent with soquelitinib’s proposed mechanism of action. The paper highlights the potential of selective ITK inhibition for the treatment of cancers and helps to confirm preclinical and clinical results generated by Corvus.
Soquelitinib Preclinical Data in Autoimmune/Allergy

On November 1, 2023, 2023, Corvus announced the publication of preclinical data on soquelitinib as a preprint at bioRxiv that demonstrated ITK’s selective inhibition which produced therapeutic benefits in several autoimmune and allergy preclinical models including psoriasis, asthma, pulmonary fibrosis, scleroderma and graft versus host disease. The mechanism of action involves the inhibition of Th2 and Th17 cells and the subsequent production of cytokines such as IL-17, IL-4, IL-5 and other cytokines involved in these diseases. The novel mechanism is a result of ITK inhibition and blockade of formation of Th2 and Th17 cells.
Partner Led Programs: Ciforadenant (adenosine 2a receptor inhibitor) and Mupadolimab (anti-CD73)

The Kidney Cancer Research Consortium is enrolling a Phase 1b/2 clinical trial evaluating ciforadenant as a potential first line therapy for metastatic renal cell cancer (RCC) in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The Phase 1b portion of this trial has been completed and patients are now being enrolled in the Phase 2 portion. The clinical trial is expected to enroll up to 60 patients and initial data is anticipated in early 2024.
Angel Pharmaceuticals, Corvus’ partner in China, is enrolling patients in a Phase 1/1b clinical trial of mupadolimab in patients with non-small cell lung cancer (NSCLC) and head and neck squamous cell cancers. In this clinical trial, patients will receive mupadolimab monotherapy or in combination with pembrolizumab.
Financial Results
As of September 30, 2023, Corvus had cash, cash equivalents and marketable securities of $32.2 million as compared to $42.3 million as of December 31, 2022. During the nine months ending September 30, 2023, the Company sold 2,461,903 shares of its common stock through its at-the-market program, generating net proceeds to the Company of approximately $7.8 million. Corvus expects full year 2023 net cash used in operating activities to be between approximately $22 million and $23 million, resulting in a projected cash balance of between $27 million and $28 million as of December 31, 2023. Based on its current plans, Corvus expects its cash to fund operations into late 2024.

Research and development expenses for the three months ended September 30, 2023 totaled $4.0 million compared to $10.4 million for the same period in 2022. The decrease of $6.4 million was primarily due to lower clinical trial and manufacturing costs associated with the development of mupadolimab.

The net loss for the three months ended September 30, 2023 was $6.0 million compared to a net loss of $14.8 million for the same period in 2022. Total stock compensation expense for the three months ended September 30, 2023 was $0.5 million compared to $0.7 million for the same period in 2022 and the non-cash loss from Corvus’ equity method investment in Angel Pharmaceuticals was $0.9 million for the three months ended September 30, 2023 compared to $2.7 million in the same period in 2022.

Conference Call Details
Corvus will host a conference call and webcast today, Tuesday, November 7, 2023, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the third quarter 2023 financial results. The conference call can be accessed by dialing 1-855-327-6837 (toll-free domestic) or 1-631-891-4304 (international) or by clicking on this link for instant telephone access to the event. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.

About Corvus Pharmaceuticals
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company pioneering the development of ITK inhibition as a new approach to immunotherapy for a broad range of cancer and immune diseases. The Company’s lead product candidate is soquelitinib, an investigational, oral, small molecule drug that selectively inhibits ITK. Corvus plans to initiate a Phase 3 registrational clinical trial for soquelitinib in patients with relapsed peripheral T cell lymphoma. Its other clinical-stage candidates are being developed for a variety of cancer indications. For more information, visit www.corvuspharma.com.

About Soquelitinib
Soquelitinib (CPI-818) is an investigational small molecule drug given orally designed to selectively inhibit ITK (interleukin-2-inducible T cell kinase), an enzyme that is expressed predominantly in T cells and plays a role in T cell and natural killer (NK) cell immune function. The immunologic effects of soquelitinib lead to what is known as Th1 skewing and is made possible by the high selectivity of soquelitinib for ITK. Research on soquelitinib’s mechanism of action suggests that it has the potential to control differentiation of normal T helper cells and enhance immune responses to tumors by augmenting the generation of cytotoxic killer T cells and the production of cytokines that inhibit cancer cell survival. Soquelitinib has also been shown to prevent T cell exhaustion, a major limitation of current immunotherapy and CAR-T therapies. Optimal doses of soquelitinib have been shown to affect T cell differentiation and induce the generation of Th1 helper cells while blocking the development of both Th2 and Th17 cells and production of their secreted cytokines. Th1 T cells are required for immunity to tumors, viral infections and other infectious diseases. Th2 and Th17 helper T cells are involved in the pathogenesis of many autoimmune and allergic diseases. The Company believes the inhibition of specific molecular targets in T cells may be of therapeutic benefit for patients with cancers, including solid tumors, and in patients with autoimmune and allergic diseases. Based on interim results from a Phase 1/1b clinical trial in patients with refractory T cell lymphomas, which demonstrated tumor responses in very advanced, refractory, difficult to treat T cell malignancies, the Company plans to initiate a registrational Phase 3 clinical trial of soquelitinib in patients with relapsed peripheral T cell lymphoma (PTCL).

About Peripheral T Cell Lymphoma
Peripheral T cell lymphoma is a heterogeneous group of malignancies accounting for about 10% of non-Hodgkin’s lymphomas (NHL) in Western populations, reaching 20% to 25% of NHL in some parts of Asia and South America. The most common subtypes are PTCL-not otherwise specified (PTCL-NOS) and T follicular helper cell lymphoma. First line treatment for these diseases is typically combination chemotherapy, however, approximately 75% of patients either do not respond or relapse within the first two years. Patients in relapse are treated with various chemotherapy agents but have poor overall outcomes with median progression-free survival in the three to four month range and overall median survival of six to 12 months. There are no approved drugs in relapsed PTCL based on randomized trials.

PTCL is a disease of mature helper T cells that express ITK, often containing numerous genetic mutations and frequently associated with viral infection. Most often the malignant cells of PTCL express a Th2 phenotype.

About Ciforadenant
Ciforadenant (CPI-444) is an investigational small molecule, oral, checkpoint inhibitor designed to disable a tumor’s ability to subvert attack by the immune system by blocking the binding of adenosine to immune cells present in the tumor microenvironment. Adenosine, a metabolite of ATP (adenosine tri-phosphate), is produced within the tumor microenvironment where it may bind to the adenosine A2A receptor present on immune cells and block their activity. Ciforadenant has been shown to block the immunosuppressive effects of myeloid cells present in tumors and preclinical studies published in 2018 demonstrated synergy with combinations of anti PD1 and anti-CTLA4 antibodies.

About Mupadolimab
Mupadolimab (CPI-006) is an investigational, potent humanized monoclonal antibody that is designed to react with a specific site on CD73. In preclinical studies, it has demonstrated immunomodulatory activity resulting in activation of lymphocytes, induction of antibody production from B cells and effects on lymphocyte trafficking. While there are other anti-CD73 antibodies and small molecules in development for treatment of cancer, such agents react with a different region of CD73. Mupadolimab is designed to react with a region of the molecule that acts to stimulate B cells and block production of immunosuppressive adenosine. Mupadolimab is being studied in combination with pembrolizumab in a Phase 1b/2 clinical trial in patients with advanced head and neck cancers and in patients with NSCLC that have failed chemotherapy and anti-PD(L)1 therapy. It is postulated that the activation of B cells will enhance immunity within the tumors of these patients, leading to improved clinical outcomes.