CORMEDIX INC. TO PRESENT AT THE CANTOR FITZGERALD GLOBAL HEALTHCARE CONFERENCE

On September 26, 2023 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening conditions and diseases, reported that management will be giving a company presentation at the Cantor Fitzgerald Global Healthcare Conference being held in New York City on September 26 – 28, 2023 (Press release, CorMedix, SEP 26, 2023, View Source [SID1234635408]).

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Cantor Fitzgerald Global Healthcare Conference
Date: Thursday, September 28, 2023
Time: 8:35 a.m. EDT
Webcast: Webcast Link

Avalo Therapeutics Successfully Eliminates $35 Million Debt Paving the Way for Future Growth and Innovation

On September 26, 2023 Avalo Therapeutics (Nasdaq: AVTX) reported the payoff of the remainder of its $35 million debt owed to Horizon Technology Finance Corporation (Nasdaq: HRZN) (Press release, Avalo Therapeutics, SEP 26, 2023, View Source [SID1234635407]). This significant milestone not only signifies the company’s steadfast commitment to financial stability but also paves the way for future accelerated growth and progress toward executing its ambitious plans to advance its most promising drug candidates, including its anti-LIGHT mAb (quisovalimab or AVTX-002) and its BTLA agonist fusion protein (AVTX-008).

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Dr. Garry A. Neil, MD, Chief Executive Officer, and Chairman of the Board at Avalo Therapeutics, expressed his enthusiasm about this crucial financial achievement, stating, "Eliminating our $35 million debt is a testament to the dedication and hard work of our entire team. It is a pivotal moment for Avalo, signaling our readiness to embrace new opportunities and transform the lives of patients with immunological diseases and addressing unmet medical needs. With a strengthened balance sheet, the company is well-positioned to pursue collaborations, expand research efforts, and bring innovative treatments to market. Our lead asset, quisovalimab, is a potential first-in-class fully human mAb against an important immunological target and has already demonstrated clinical proof of concept in both acute and chronic inflammatory diseases. Additionally, we believe AVTX-008, our BTLA agonist fusion protein, has potential to be a best-in-class molecule. It addresses an increasingly recognized and important target for a variety of immune diseases as evidenced by Gilead’s $405 million acquisition of MiroBio. Our highly skilled and capable team are executing well against our plan. We can now turn our attention to moving our development programs forward as well as looking for new opportunities to augment our very promising pipeline."

Dr. Neil continued, "We’d like to thank our colleagues at our lender, Horizon Technology Finance, who worked professionally, collaboratively and constructively with us to achieve this milestone."

Gerald A. Michaud, President of Horizon Technology Finance Corporation, said, "We were very impressed with the focus and professionalism that Garry and his management team showed in paying off this debt in full and ahead of schedule in a challenging market environment for biotechnology. Avalo created and executed a coherent and investable strategy that included eliminating non-essential costs, streamlining the pipeline on their most highly valued assets, operational excellence and executing multiple business development transactions that brought in significant non-dilutive funding. We are proud of our role in helping promising emerging companies, like Avalo, achieve their financial goals and wish Avalo Therapeutics much future success."

Termination of Material Definitive Agreement

On September 26, 2023, Caribou Biosciences, Inc. (the "Company") received notice from AbbVie Manufacturing Management Unlimited Company ("AbbVie") that AbbVie has elected to terminate the Collaboration and License Agreement, as amended, by and between the Company and AbbVie, dated February 9, 2021 (the "Agreement") (Filing, 8-K, Caribou Biosciences, SEP 26, 2023, View Source [SID1234635406]). AbbVie exercised its right to terminate the Agreement for convenience, and this decision was based on AbbVie’s strategic focus and was unrelated to Caribou’s performance under the Agreement or the data generated to date. By mutual agreement of the Company and AbbVie, termination of the Agreement will be effective October 25, 2023.

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Under the terms of the Agreement, the Company was conducting certain preclinical research, development, and manufacturing activities for two AbbVie programs, each program comprising one or more collaboration allogeneic CAR-T products directed toward the single cancer target or target combination, using the Company’s Cas12a chRDNA genome-editing and CAR-T cell therapy technologies. AbbVie has elected not to advance these two programs and, effective upon termination of the Agreement, all licenses granted thereunder will immediately terminate.

This summary of the terms of the Agreement is qualified in its entirety by reference to the Agreement, which was filed as Exhibits 10.1, 10.2, and 10.3 to the Company’s Annual Report on 10-K for the year ended December 31, 2022.

Cardiff Oncology Announces Positive Clinical Data in Pancreatic Cancer and Small Cell Lung Cancer, including Single-Agent Activity from Onvansertib Monotherapy

On September 26, 2023 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, reported positive clinical data with onvansertib monotherapy and combination therapy in our ongoing trials in metastatic pancreatic ductal adenocarcinoma (mPDAC) and small cell lung cancer (SCLC), as well as plans for a mPDAC first-line investigator-initiated trial (IIT) of the combination of onvansertib plus standard-of-care (SoC) (Press release, Cardiff Oncology, SEP 26, 2023, View Source [SID1234635405]).

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"We are excited that the data released from these trials, in two challenging cancers with low survival rates, expands the opportunity for onvansertib beyond our lead program in RAS-mutated mCRC," said Mark Erlander, Ph.D., Chief Executive Officer of Cardiff Oncology. "In pancreatic cancer, the strength of the data provides a clear rationale for a first-line trial using onvansertib in combination with standard of care, which we believe provides the greatest opportunity for a positive impact on patients. In small cell lung cancer, we are encouraged to observe single-agent activity with onvansertib monotherapy in this difficult-to-treat extensive stage refractory setting."
mPDAC Phase 2 CRDF-001 trial: 19% ORR and 5.0-month mPFS

Data from the ongoing Phase 2 open-label trial of onvansertib combined with nanoliposomal irinotecan, leucovorin, and 5-FU in patients with second-line mPDAC demonstrated an objective response rate (ORR) of 19% (4 of 21 evaluable patients; 1 confirmed PR, 3 waiting for confirmatory scan) and median progression-free survival (mPFS) of 5.0 months as of the data cutoff of September 13, 2023. Historical control trials in similar patient populations have shown an ORR of 7.7% and mPFS of 3.1 months with SoC.

mPDAC biomarker discovery trial: decrease in clinically-validated tumor biomarkers from onvansertib monotherapy
The investigator-initiated biomarker discovery trial is exploring the impact of onvansertib 10-day monotherapy on tumors in mPDAC patients, and is currently enrolling at the Oregon Health & Science University (OHSU) Knight Cancer Institute. Two patients have been enrolled to date. One patient demonstrated an 86% decrease in Ki67, a well-established biomarker of tumor proliferation, and a 28% decrease in CA 19-9, a clinically-used biomarker to monitor treatment response.

"Serum carbohydrate antigen 19-9 is the most extensively studied and validated serum biomarker in PDAC, which provides a clinically meaningful surrogate for response to treatment. We are encouraged by the ability of onvansertib to provide an approximately 30% reduction in this biomarker with only 10 days of monotherapy in a refractory setting," said Fairooz Kabbinavar, MD, Chief Medical Officer of Cardiff Oncology. "We will continue to explore onvansertib in the first-line mPDAC investigator-initiated trial at the OHSU Knight Cancer Institute."

Update in Clinical Development Plan for mPDAC

The next trial in mPDAC will be a new Phase 2 investigator-initiated trial at OHSU Knight Cancer Institute in mPDAC in the first-line setting. There are two cohorts in this trial. In cohort 1, patients will receive the combination of onvansertib with SoC (Gemzar + Abraxane). In cohort 2, patients will receive 10 days of onvansertib monotherapy followed by onvansertib + SoC to identify biomarkers that predict response to onvansertib.

SCLC Phase 2 Investigator-Initiated Trial

The ongoing Phase 2 trial of onvansertib monotherapy in patients with relapsed extensive stage SCLC who have received up to two prior therapies is currently enrolling patients at the University of Pittsburgh Medical Center. An examination of the safety data from the first six patients by the institutional review board confirmed the trial can continue to enroll as planned. Preliminary efficacy data in evaluable patients will be discussed on the company conference call.

Conference Call and Webcast

Cardiff Oncology will host a corresponding conference call and live webcast at 5:00 p.m. ET/2:00 p.m. PT on September 26, 2023. Individuals interested in listening to the live conference call may do so by using the webcast link in the "Investors" section of the company’s website at www.cardiffoncology.com. A webcast replay will be available in the investor relations section on the company’s website for 30 days following the completion of the call.

Candel Therapeutics Reports Encouraging Initial Survival Data from Phase 2 Clinical Trial of CAN-2409 in Non-Small Cell Lung Cancer

On September 26, 2023 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing viral immunotherapies to help patients fight cancer, reported updated activity data from its ongoing, open-label, phase 2 clinical trial of CAN-2409 plus valacyclovir in combination with continued immune checkpoint inhibitor (ICI) treatment in patients with non-resectable, stage III/IV non-small cell lung cancer (NSCLC), who have an inadequate response to front line anti-PD(L)1 therapy (Press release, Candel Therapeutics, SEP 26, 2023, View Source [SID1234635404]). These patients historically have had an expected median overall survival of 10-13 months (Reckamp K et al. J Clin Onc 2022;40:2295-2306). The aim of the CAN-2409 immunotherapy antitumor strategy is to raise the tail on the survival curve by increasing the number of long survivors beyond 10-13 months.

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In 2022, the Company presented data from this phase 2 clinical trial where patients who received two administrations of CAN-2409 plus prodrug demonstrated: 1) increased infiltration of CD8+ cytotoxic T cells in the tumor microenvironment, systemic expansion of effector T cells and increased soluble granzyme B levels in peripheral blood, 2) favorable changes in the trajectory of tumor progression, 3) decreased tumor size of target lesions in most patients, and 4) reduced size of uninjected tumor lesions (Aggarwal C et al. Abstract #9037 ASCO (Free ASCO Whitepaper) June 2022 and Aggarwal C et al. Candel Virtual R&D Day, December 2022). These data were further confirmed in the current update.

Highlights as of August 1, 2023 data cutoff, include:


As of data cutoff (August 1, 2023), 40 patients across Cohort 1 (stable disease at enrollment; n=5) and Cohort 2 (progressive disease at enrollment; n=35) were evaluable, as they received two courses of CAN-2409 + valacyclovir and completed the 12-week treatment window.

While overall survival is not yet mature, Candel has observed an encouraging number of long survivors, which the Company believes that CAN-2409 may induce a new state of functional immunosurveillance and durable disease control in a subset of the patients.

Of the 40 evaluable patients, 15 patients have lived ≥ 12 months; of these, 10 patients have lived > 18 months, of whom 70% (7/10) were alive as of last follow up. All 4 patients (100%) with OS > 24 months were alive at last follow up, with the longest reaching 31.7 months (data cutoff August 1, 2023).

An additional 18 out of the 40 evaluable patients are also alive but have not yet reached 12 months of follow up.

Notably, many patients treated with CAN-2409 have had long survival (≥ 12 months) despite having disease features generally associated with advanced disease and reduced likelihood to benefit from ICI therapy, such as low or negative PD-L1 expression, including:

Amongst patients alive ≥ 12 months with known PD-L1 status (14/15), 93% had negative or low PD-L1 score (<1 or between 1-49).

Advanced disease with stage IV in 73% (11/15), lymph node involvement in 73% (11/15), pleural effusion in 40% (6/15), bone metastases in 27% (4/15), adrenal metastases in 20% (3/15), brain metastases in 13% (2/15), liver metastases in 7% (1/15), involvement of 3 or more organs in 13% (2/15), and ECOG performance status 1 in 40% (6/15).

There was a significant increase observed in activated central memory, effector-memory, effector T cells, and NK cells after CAN-2409 treatment. These include CD8+Ki67+IFNg+ T cells, CD8+ granzyme B+Ki67+ T cells, CD56+CD16+granzyme B+ NK cells, and gd+ T cells. Candel also observed an increase in B memory cells after CAN-2409 treatment.

Candel observed an increase in effector/cytotoxic T cells and NK cells in peripheral blood after the second CAN-2409 administration associated with improved survival (≥ 12 months).

Candel continued to observe a favorable safety / tolerability profile after CAN-2409 treatment in NSCLC. There were no dose limiting toxicities or grade 4 or greater treatment related adverse events. Grade 3 treatment related adverse
events were reported in < 10% of patients receiving at least one dose of CAN-2409 (safety population), which Candel believes compares favorably to current standard of care options.

Candel expects to share topline overall survival data for Cohort 2 in the second quarter of 2024, assuming mature data at that time.
"We are very encouraged by the initial survival data, particularly for Cohort 2 which enrolled patients whose disease progressed despite receiving anti-PD(L)1 treatment. This patient population represents a major unmet need, since median survival following progression is typically less than 12 months with available treatments," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "Importantly, 93% of patients with long survival were low or negative for PD-L1 expression, which we believe confirms the potential of CAN-2409 to convert patients with highly immunosuppressive tumor microenvironments, unresponsive to ICI treatment, into long term survivors. These early data support the promise and potential of CAN-2409 to provide a long-term survival tail in some of the most difficult to treat NSCLC cases."

Dr. Tak continued, "Our approach is patient-centric. The proposed treatment regimen for CAN-2409 with prodrug is approximately 12 weeks, and patients can continue their anti-PD(L)1 therapy at local treatment centers, reducing treatment burden. The immediate goal is to provide a treatment benefit through disease stabilization via the addition of CAN-2409. The ultimate goal is to extend patient survival with improved quality of life. These data support a survival benefit in individual patients, which continues to be associated with a favorable safety and tolerability profile, particularly when compared to standard of care taxane-based chemotherapy."

"The latest biomarker research performed by Candel, PACT, CIMAC and the National Cancer Institute further supports and expands previous findings," said Francesca Barone, MD, PhD, Chief Scientific Officer of Candel. "We demonstrated for the first time the potential of CAN-2409 to engage the humoral arm of the immune system, broadening the scope of the antitumoral immune response. The correlations between early changes in key effector immune populations after CAN-2409 treatment and survival suggest the possibility to use early biological readouts as predictors of clinical response."

"Although ICIs have transformed initial therapy in NSCLC and extended life for many patients, there still remains a serious unmet need, especially in patients with low levels of PD-L1 expression," said Charu Aggarwal, MD, MPH, Associate Professor for Lung Cancer Excellence, Perelman School of Medicine, University of Pennsylvania, and Co-Principal Investigator for the phase 2 clinical trial. "Most patients progress following ICI treatment and options after progression are often associated with toxicity and small treatment benefit. The report of today’s initial promising data on survival gives us an insight into the potential of CAN-2409 to produce a sustained anti-tumor immune response and potentially extend the lives of patients living with advanced NSCLC. I look forward to the maturation of these overall survival data and expected readout in Q2 2024."

About CAN-2409

CAN-2409, Candel’s most advanced viral immunotherapy candidate, is an investigational off-the-shelf replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s specific tumor and induce an individualized, systemic immune response against the disease. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells. The intra-tumoral administration results in the release of tumor-specific neoantigens in the microenvironment. At the same time, the adenoviral serotype 5 capsid protein elicits a strong pro-inflammatory signal in the tumor microenvironment. This is designed to create the optimal conditions to induce an individualized and specific CD8+ T cell mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity. Because of its versatility, CAN-2409 has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and ICI have previously been shown in several preclinical and clinical settings. Furthermore, more than 950 patients have been dosed with CAN-2409 with a favorable tolerability profile to date, supporting the potential for combination with other therapeutic strategies without inordinate concern of overlapping adverse events. Currently, Candel is evaluating the effects of treatment with CAN-2409 in NSCLC, borderline resectable pancreatic cancer, and localized, non-metastatic prostate cancer in ongoing clinical trials. The U.S. Food and Drug Administration granted Fast Track designation for CAN-2409 plus valacyclovir in combination with pembrolizumab in order to improve survival or delay progression in patients with stage III/IV NSCLC who are resistant to first line anti-PD(L)1 therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy.