On November 13, 2023 Delcath Systems, Inc. (Nasdaq: DCTH) ("Delcath" or the "Company"), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported business highlights and financial results for the third quarter ended September 30, 2023 (Press release, Delcath Systems, NOV 13, 2023, View Source [SID1234637535]).

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Recent Business Highlights

Received approval from the US Food and Drug Administration (FDA) for HEPZATO KIT (melphalan)/Hepatic Delivery System) as a liver-directed treatment for adult patients with metastatic uveal melanoma (mUM) with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation;
Raised approximately $35 million through the exercise of all the Tranche A warrants issued as part the previously announced March 29, 2023, financing with up to an additional $25 million available upon the achievement of $10 million in quarterly revenue;
Announced publication by independent investigators of a retrospective comparative study of CHEMOSAT Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP) and Selective Internal Radiation Therapy (SIRT) citing a statistically significant difference in median overall survival with 301 days for SIRT and 516 days for PHP;
Announced that independent investigators at Leiden University recruited 55 of 76 planned patients in the CHOPIN trial studying the impact of CHEMOSAT and immune checkpoint inhibitor combination therapy in the treatment of metastatic uveal melanoma and expect the trial to be fully enrolled mid-2024. An interim futility analysis conducted in September resulted in the independent data monitoring committee recommending continuing the study without modification; and
Partnered with seven clinical treatment sites to satisfy the initial training requirements to be able to treat their first commercial patient, subject to proctor availability and the sites’ value analysis committee approvals, upon the planned launch in January.
"The Company has been preparing for a January launch of HEPZATO KIT by building our commercial team and engaging with potential treating centers," said Gerard Michel, Chief Executive Officer of Delcath. Mr. Michel added, "We have been encouraged by the reception that HEPZATO KIT has received from the medical oncologist community and are confident that by the end of 2024 at least 15 centers will be actively treating metastatic uveal melanoma patients with HEPZATO KIT."

Third Quarter 2023 Results

Total revenue for the three months ended September 30, 2023, was approximately $0.4 million, compared to $0.9 million for the prior year period, from our sales of CHEMOSAT in Europe.

Research and development expenses for the quarter were $4.7 million, compared to $4.1 million in the prior year quarter. The increase in R&D expense is primarily due to completing clinical trial activities and expenses related to the FDA inspection and other requests in advance of the approval received for HEPZATO.

Selling, general and administrative expenses for the quarter increased to $6.2 million, compared to $4.8 million in the prior year quarter primarily relating to activities to prepare for a commercial launch. The increase in fair value warrant liability adjustment during the quarter ended September 20, 20223 was a result of our final valuation for the Tranche A warrants exercised and outstanding Tranche B warrants.

On November 13, 2023 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported third quarter financial results and provided a business update (Press release, Cyclacel, NOV 13, 2023, View Source [SID1234637534]).

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"Fadraciclib continues to show good tolerability and anticancer activity as a single agent," said Spiro Rombotis, President and Chief Executive Officer. "After analyzing the genomic profile of several Phase 1 patients with clinical benefit, we have identified mutational and molecular patterns that may be predictive of clinical activity across broad cancer types. If confirmed, these findings may be relevant in the upcoming Phase 2 part of our 065-101 study. In our Phase 1/2 study of plogosertib good tolerability and anticancer activity as a single agent has been observed in multiple patients with various solid tumors. Preclinical data continues to suggest that plogosertib may work through an epigenetic mechanism and that certain mutational biomarkers may identify patients with sensitive tumors. We look forward to presenting data from these two programs and their mechanisms in the coming months."

"In the 065-101 study of oral fadraciclib, our CDK2/9 inhibitor, as monotherapy, we are completing dose escalation level 6A with six patients and expect to select the recommended Phase 2 dosing schedule shortly. We are encouraged by the observations of anticancer activity and the related patient genomic profiles," said Mark Kirschbaum, M.D., Chief Medical Officer. "We believe that fadraciclib’s inhibition of CDK2 and CDK9 may be superior to either CDK2 or CDK9 alone. Importantly, we have been able to give fadraciclib orally with repeat dosing which has led to transient suppression of anti-apoptosis proteins without hematological toxicity. In the 140-101 study of oral plogosertib, our PLK1 inhibitor as a single agent, we are recruiting patients at dose level 5. The anticancer activity observed at low levels of continuous exposure may be due to plogosertib’s novel epigenetic mechanism which we are continuing to investigate. If confirmed, we intend to design clinical studies that could exploit these findings."

Key Milestones

· Report final data from dose escalation stage and RP2D determination from the 065-101 study of oral fadraciclib in patients with advanced solid tumors and lymphoma
· First patient dosed with oral fadraciclib in Phase 2 proof-of-concept stage of 065-101 study in patients with advanced solid tumors and lymphoma
· Report Phase 1 data from 140-101 study of oral plogosertib in patients with advanced solid tumors and lymphoma
· Disclose novel epigenetic mechanism of action of plogosertib

Financial Highlights

As of September 30, 2023, cash equivalents totaled $5.9 million, compared to $18.3 million as of December 31, 2022. Net cash used in operating activities was $12.2 million for the nine months ended September 30, 2023 compared to $15.7 million for the same period of 2022. The Company estimates that its available cash will fund currently planned programs through the end of 2023. The operating plan includes discretionary expenditures, which if not incurred could extend cash runway into the second quarter of 2024.

Research and development (R&D) expenses were $5.2 million for the three months ended September 30, 2023, as compared to $4.4 million for the same period in 2022. R&D expenses relating to fadraciclib were $3.6 million for the three months ended September 30, 2023, as compared to $2.5 million for the same period in 2022 due to due to increased costs associated with manufacture scale up and introduction of the tablet form. R&D expenses related to plogosertib were $1.5 million for the three months ended September 30, 2023, as compared to $1.7 million for the same period in 2022.

General and administrative expenses for the three months ended September 30, 2023 were $1.6 million, as compared to $2.1 million for the same period in 2022 due to non-recurring professional fees of $0.4 million in the prior period.

Total other income, net, for the three months ended September 30, 2023, was $0.1 million compared to an income of $0.4 million for the same period of the previous year.

United Kingdom research & development tax credits for the three months ended September 30, 2023 were $0.6 million compared to $1.0 million for the same period of the previous year due to taxation legislative changes that took effect in April 2023 and reduced the amount of tax credit that could be claimed. Research & development tax credits are directly correlated to qualifying research and development expenditure.

Net loss for the three months ended September 30, 2023, was $6.1 million, compared to $5.1 million for the same period in 2022.

Conference call information:

Call: (800) 245-3047 / international call: (203) 518-9765

Archive: (800) 688-7036 / international archive: (402) 220-1346

Code for live and archived conference call is CYCCQ323. Webcast link

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On November 13, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the third quarter ended September 30, 2023, and recent corporate highlights (Press release, Checkpoint Therapeutics, NOV 13, 2023, View Source [SID1234637533]).

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"The January 3, 2024, action date for our Biologics License Application ("BLA") for cosibelimab is fast-approaching, and we continue to work closely with the U.S. Food and Drug Administration ("FDA") in completing their review," said James Oliviero, President and Chief Executive Officer of Checkpoint. "During the third quarter, pivotal trial results for cosibelimab in metastatic cutaneous squamous cell carcinoma ("cSCC") were published in the peer-reviewed Journal for ImmunoTherapy of Cancer, which further supports the efficacy and safety of cosibelimab. We also reported longer-term data from our pivotal trials in both locally advanced and metastatic cSCC that demonstrate a deepening of response over time with substantially higher complete response rates. We firmly believe that cosibelimab’s clinical profile, which includes a unique dual mechanism of action and favorable safety profile, should position the product, upon its potential launch next year, as the preferred immunotherapy of oncologists, particularly for the large number of difficult-to-treat cSCC patients who continue to suffer poor outcomes with currently available treatments."

Recent Corporate Highlights:

Checkpoint submitted a BLA to the FDA seeking approval of cosibelimab in January 2023. In March 2023, Checkpoint announced the FDA accepted the BLA filing for cosibelimab and set a Prescription Drug User Fee Act ("PDUFA") goal date of January 3, 2024. The FDA has indicated that an advisory committee meeting to discuss the application is not planned.
In July 2023, Checkpoint announced new, longer-term data for cosibelimab from its pivotal studies in locally advanced and metastatic cSCC. These results demonstrate a deepening of response with cosibelimab treatment over time, resulting in substantially higher complete response rates than previously reported. Furthermore, responses continue to remain durable over time.
Also in July 2023, Checkpoint completed a registered direct offering priced at-the-market under Nasdaq rules for total gross proceeds of $10 million.
In October 2023, Checkpoint announced the exercise of previously issued warrants for $11.13 million in gross proceeds.
Also in October 2023, Checkpoint announced the publication of results from the multicenter, multiregional, pivotal trial evaluating cosibelimab in patients with metastatic cSCC in the Journal for ImmunoTherapy of Cancer (JITC), the peer-reviewed, online journal of the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). The paper, entitled, "Efficacy and Safety of Cosibelimab, an Anti–PD-L1 Antibody, in Metastatic Cutaneous Squamous Cell Carcinoma" (doi:10.1136/jitc-2023-007637), describes safety and efficacy results from 78 patients with metastatic cSCC enrolled at clinical sites in eight countries.
Financial Results:

Cash Position: As of September 30, 2023, Checkpoint’s cash and cash equivalents totaled $1.8 million, compared to $7.4 million at June 30, 2023 and $12.1 million at December 31, 2022, a decrease of $5.6 million for the quarter and a decrease of $10.3 million for the first nine months of 2023. Subsequent to the end of the third quarter, Checkpoint raised approximately $11.13 million of gross proceeds from the exercise of previously issued warrants.
R&D Expenses: Research and development expenses for the third quarter of 2023 were $5.5 million, compared to $8.9 million for the third quarter of 2022, a decrease of $3.4 million. Research and development expenses for the third quarters of 2023 and 2022 both included $0.3 million of non-cash stock expenses.
G&A Expenses: General and administrative expenses for the third quarter of 2023 were $2.2 million, compared to $1.8 million for the third quarter of 2022, an increase of $0.4 million. General and administrative expenses for the third quarter of 2023 included $0.6 million of non-cash stock expenses, compared to $0.5 million for the third quarter of 2022.
Net Loss: Net loss attributable to common stockholders for the third quarter of 2023 was $5.7 million, or $0.29 per share, compared to a net loss of $10.6 million, or $1.20 per share, in the third quarter of 2022. Net loss for the third quarter of 2023 included $0.9 million of non-cash stock expenses, compared to $0.8 million for the third quarter of 2022.

On November 13, 2023 Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company focused on discovering and developing medicines that are transformational for patients, reported financial results and business highlights for the third quarter ended September 30, 2023 (Press release, Centessa Pharmaceuticals, NOV 13, 2023, View Source [SID1234637531]).

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"We continue to execute and achieve key milestones across our pipeline of potential transformative medicines for patients with unmet needs," said Saurabh Saha MD PhD, Chief Executive Officer of Centessa. "We’re making good progress enrolling our registrational studies for SerpinPC in hemophilia B, with or without inhibitors, and are excited to share new data from the ongoing Phase 2a study of SerpinPC during a poster presentation at ASH (Free ASH Whitepaper) in December. We also continue to advance our novel LockBody technology platform with LB101, a PD-L1xCD47 LockBody, which is in an ongoing Phase 1/2a clinical trial for the treatment of solid tumors."

Dr. Saha continued, "Momentum continues to build with our orexin agonist development program. The preclinical data we recently shared at the World Sleep Congress show that ORX750 is a highly potent and selective novel orexin receptor 2 (OX2R) agonist that closely mimics the function of the endogenous peptide. We believe these data support a potential best-in-class profile for ORX750 for the treatment of narcolepsy and other sleep-wake disorders. We are focused on rapidly moving ORX750 through IND-enabling studies, obtaining IND clearance and initiating clinical development with the goal of sharing clinical proof of concept data in sleep-deprived healthy volunteers in 2024. In addition, we are exploring follow-up molecules for potential expansion opportunities into a range of sleep-wake disorders and broader neurological indications."Recent Highlights
•In October, the Company announced a set of new preclinical data from in vivo and in vitro studies of its investigational, novel OX2R agonist, ORX750, that support a potential best-in-class profile for the treatment of narcolepsy and other sleep-wake disorders. The preclinical data were presented recently at the World Sleep Congress and are also available within a recorded webcast at View Source
•In November, the Company announced that new data from the third year (Part 5) of the ongoing Phase 2a study of SerpinPC, an investigational subcutaneously administered novel inhibitor of activated protein C (APC), for the treatment of hemophilia, has been accepted for a poster presentation at ASH (Free ASH Whitepaper) on December 10, 2023. The presentation will include efficacy and safety data from an additional 52-weeks of continuous treatment with a subcutaneous injection of SerpinPC in subjects with hemophilia.
•In October, the Company announced the dosing of the first subject in its registrational PRESent-3 clinical study of SerpinPC for the treatment of hemophilia B with inhibitors. The Company initiated dosing in its registrational PRESent-2 clinical study of SerpinPC for the treatment of hemophilia B without inhibitors in July.
•In August, the Company announced LB206, a PD-L1xCD3 LockBody, as a development candidate and shared preclinical data which demonstrated single agent regressions of large tumors in a difficult-to-treat mouse xenograft model.

Anticipated Upcoming Program Milestones
•Hemophilia (SerpinPC) – The registrational PRESent-2 and PRESent-3 studies are ongoing. Data from Part 5 of the ongoing Phase 2a study accepted for poster presentation at ASH (Free ASH Whitepaper) on December 10, 2023.
•Solid Tumors (LockBody Technology Platform)
◦PD-L1xCD47 LockBody LB101 – Phase 1/2a first-in-human clinical study is ongoing.
◦PD-L1xCD3 LockBody LB206 – LB206 is a development candidate.
•Narcolepsy and Other Sleep-Wake Disorders (ORX750) – The Company is focused on rapidly advancing ORX750 through IND-enabling studies, obtaining IND clearance and initiating clinical development with the goal of sharing clinical proof of concept data in sleep-deprived healthy volunteers in 2024.

Where applicable, the Company plans to provide updates on preclinical programs as they advance toward clinical studies.

Third Quarter 2023 Financial Results
•Cash, Cash Equivalents and Short-term Investments: $281.3 million as of September 30, 2023, which the Company expects will fund operations into 2026, without drawing on the remaining available tranches under the Oberland credit facility.
•Research & Development Expenses: $28.2 million for the third quarter ended September 30, 2023, compared to $36.7 million for the third quarter ended September 30, 2022.
•General & Administrative Expenses: $12.0 million for the third quarter ended September 30, 2023, compared to $12.3 million for the third quarter ended September 30, 2022.
•Net Loss Attributable to Ordinary Shareholders: $38.6 million for the third quarter ended September 30, 2023, compared to $53.9 million for the third quarter ended September 30, 2022.

On November 13, 2023 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the third quarter ended September 30, 2023 and provided other recent corporate updates (Press release, Celcuity, NOV 13, 2023, View Source [SID1234637530]).

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"Despite the important role the PI3K/mTOR pathway plays in tumor types such as breast and prostate cancer, the available therapies that target this pathway have only reported limited improvements in outcomes for patients. Development of an optimized PI3K/mTOR inhibitor, like gedatolisib, thus represents, we believe, one of the most important opportunities to improve the standard of care in these cancers. With our Phase 3 program in HR+/HER2- advanced breast cancer, and our newly initiated Phase 1b/2 program in metastatic castration resistance prostate cancer, we are hoping to eventually impact over 200,000 patients globally," said Brian Sullivan, CEO and Co-Founder of Celcuity. "The equity financing we recently closed further supports these programs and extends our cash runway into mid-2026."

Third Quarter 2023 Business Highlights and Other Recent Developments

● The VIKTORIA-1 Phase 3 trial remains on track to provide initial data and analysis of the PIK3CA wild type patient sub-group in the second half of 2024 and data for the PIK3CA mutated patient sub-group in the first half of 2025.

○ The Phase 3 VIKTORIA-1 clinical trial is now recruiting patients at nearly 220 sites in 23 countries in North and South America, Europe, and Asia.

○ VIKTORIA-1 is evaluating gedatolisib in combination with fulvestrant, an endocrine therapy, with and without palbociclib, a CDK4/6 inhibitor, in adults with HR+, HER2- advanced breast cancer.

● The CELC-G-201 Phase 1b/2 clinical trial evaluating gedatolisib in combination with Nubeqa (darolutamide) for the treatment of metastatic castration resistant prostate cancer (mCRPC) is on track to activate trial sites in the first quarter of 2024 and to report initial data in the first half of 2025.

○ The U.S. Food and Drug Administration (FDA) cleared an Investigational New Drug (IND) submission that enables Celcuity to proceed with a trial to evaluate gedatolisib in combination with darolutamide in August 2023.

○ We entered into a clinical trial collaboration and supply agreement with Bayer AG in August 2023 to provide Nubeqa (darolutamide) at no cost.

○ The Phase 1b/2 trial will enroll up to 54 patients with mCRPC whose disease progressed after treatment with an androgen receptor inhibitor.

● Celcuity closed a private placement of equity that resulted in net proceeds of approximately $50 million in October 2023.

Third Quarter 2023 Financial Results

Unless otherwise stated, all comparisons are for the third quarter ended September 30, 2023, compared to the third quarter ended September 30, 2022.

Total operating expenses were $18.9 million for the third quarter of 2023, compared to $10.6 million for the third quarter of 2022. Research and development (R&D) expenses were $17.5 million for the third quarter of 2023, compared to $9.6 million for the third quarter of 2022. Of the approximately $7.9 million increase in R&D expenses, $7.5 million was due to an increase in expenses related to the VIKTORIA-1 Phase 3 clinical trial, and $0.4 million was related to increased employee-related expenses.

General and administrative expenses were $1.4 million for the third quarter of 2023, compared to $1.0 million for the third quarter of 2022. Employee-related expenses accounted for $0.3 million of the increase. The remaining $0.1 million increase resulted from professional fees and other expenses associated with being a public company.

Net loss for the third quarter of 2023 was $18.4 million, or $0.83 loss per share, compared to a net loss of $10.9 million, or $0.75 loss per share, for the third quarter of 2022. Non-GAAP adjusted net loss for the third quarter of 2023 was $17.3 million, or $0.78 loss per share, compared to non-GAAP adjusted net loss for the third quarter of 2022 of $9.5 million, or $0.63 loss per share. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the third quarter of 2023 was $12.7 million, compared to $9.3 million for the third quarter of 2022. At September 30, 2023, Celcuity reported cash, cash equivalents and short-term investments of $133.9 million.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 8:00 a.m. ET today to discuss the third quarter financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-877-407-0784 or1-201-689-8560. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=7d2827005c. A replay of the webcast will be available on the Celcuity website following the live event.