On December 18, 2024 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, reported the presentation of two data sets through two abstracts showcasing pelareorep’s potential in difficult-to-treat gastrointestinal cancers were accepted and will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in San Francisco January 23-25, 2025 (Press release, Oncolytics Biotech, DEC 18, 2024, View Source [SID1234649196]).

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Tom Heineman, M.D., Ph.D., Oncolytics’ Chief Medical Officer, said, "We are enthusiastic about pelareorep’s applicability across multiple gastrointestinal cancer indications, including pancreatic and anal cancer. Pelareorep engages patients’ immune systems to help make commonly used chemotherapies and checkpoint inhibitors, such as atezolizumab, more effective in fighting cancer. This offers the promise of delaying disease progression and improving survival in patients with these devasting diseases. Given the versatility of pelareorep, we see multiple clinical and regulatory options for bringing this promising medicine to patients."

Abstract Number: 6
Title: GOBLET platform study: Preliminary safety and tumor response results for the relapsed anal carcinoma cohort in patients treated with pelareorep and atezolizumab.
Presentation Type: Poster
Session Title: Poster Session C: Cancers of the Colon, Rectum, and Anus
Session Date and Time: January 25, 2025, 7:00 – 7:55 a.m. PT

Abstract Number: 730
Title: GOBLET study: Results of the safety run-in for first-line metastatic pancreatic ductal adenocarcinoma (PDAC) patients treated with pelareorep + modified FOLFIRINOX +/- atezolizumab.
Presentation Type: Poster
Session Title: Poster Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Session Date and Time: January 24, 2025, 11:30 a.m. – 1:00 p.m. PT

Abstracts will be published on the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium website at 5:00 p.m. ET on January 21, 2025.

On December 18, 2024 Cara Therapeutics, Inc. (Nasdaq: CARA) and Tvardi Therapeutics, Inc. ("Tvardi"), a privately held, clinical-stage biopharmaceutical company focused on the development of novel, oral, small molecule therapies targeting STAT3 to treat fibrosis-driven diseases, reported that the companies have entered into a definitive merger agreement to combine in an all-stock transaction (the "Merger") (Press release, Tvardi Therapeutics, DEC 18, 2024, View Source [SID1234649197]).

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Under the terms of the agreement, Tvardi will merge with a wholly owned subsidiary of Cara. Upon completion of the Merger, pre-Merger Cara Therapeutics stockholders are expected to own approximately 17.0% of the combined company and pre-Merger Tvardi Therapeutics investors are expected to own approximately 83.0% of the combined company, in each case, prior to adjustment from the issuance of the shares in the recently completed Tvardi financing and assuming Cara has net cash at closing of between $22.875 million and $23.125 million. The percentage of the combined company that pre-merger Cara stockholders and pre-merger Tvardi stockholders will own upon the closing of the merger is subject to further adjustment if Cara’s net cash balance falls outside of the range. Upon completion of the Merger, the combined company is expected to operate under the name Tvardi Therapeutics, Inc. and trade on Nasdaq under the ticker symbol "TVRD".

Imran Alibhai, Ph.D., Chief Executive Officer of Tvardi Therapeutics, stated, "As we approach meaningful value inflection points next year, including two Phase 2 readouts of our lead program in idiopathic pulmonary fibrosis, followed by the readout in our hepatocellular carcinoma program, this merger, the recently completed financing, and becoming a publicly traded company give us access to the critical funding required to further advance our promising pipeline programs that address significant unmet needs. I am grateful to the Cara Board, leadership team, and shareholders who share our vision of Tvardi that is well-positioned to introduce effective, new treatment options to patients suffering from serious, chronic, fibrosis-driven diseases."

"We are very excited to enter into this merger agreement with Tvardi and combine our financial resources with their expertise in STAT3 inhibition," added Christopher Posner, President and Chief Executive Officer of Cara Therapeutics. "Our management and our Board of Directors thoroughly explored numerous strategic alternatives and believe that this merger with Tvardi is in the best interests of our stockholders and provides them with the opportunity to meaningfully participate in a company treating fibrosis-driven diseases in an innovative way."

Tvardi has recently completed an approximately $28 million private financing from a syndicate of new and existing institutional investors. With the cash from both companies at closing and the proceeds of this financing, the combined company is expected to have sufficient cash to fund its operating expenses and capital expenditure requirements into the second half of 2026, past the anticipated Phase 2 readouts in the second half of 2025.

KORSUVA/KAPRUVIA Asset Sale:
Concurrent with the entry into the merger agreement with Tvardi, Cara also entered into an asset purchase agreement with Vifor Fresenius Medical Care Renal Pharma, Ltd. ("CSL Vifor"), a company jointly owned by Fresenius Medical Care and by the CSL Vifor business unit of the CSL Group. Pursuant to such asset purchase agreement, at the consummation of the transaction, Cara will sell to CSL Vifor and CSL Vifor will acquire from Cara certain assets and rights to the development, manufacture and commercialization of Korsuva/Kapruvia (difelikefalin) as well as certain associated liabilities (the "Asset Disposition") for a purchase price of $900,000 (subject to certain adjustments with respect to inventory). Additionally, pursuant to the Asset Purchase Agreement, at the consummation of the Asset Disposition, Cara has agreed to pay CSL Vifor $3,000,000 to compensate CSL Vifor for the estimated incremental future expenses to be incurred by CSL Vifor as a result of the transfer of the assets to be acquired and the liabilities to be assumed by it in connection with the Asset Disposition.

The Asset Disposition is subject to certain conditions to closing, including the consummation of the merger with Tvardi substantially contemporaneously with the Asset Disposition.

Tvardi’s Pipeline of STAT3 Inhibitors:
The combined company will focus on advancing Tvardi’s pipeline of novel, oral, small molecule therapies targeting STAT3 to treat fibrosis-driven diseases with significant unmet need, including its lead candidate, TTI-101, which is in a Phase 2 trial for idiopathic pulmonary fibrosis (IPF) and a Phase 1b/2 trial for hepatocellular carcinoma (HCC). STAT3 is a highly validated, yet historically undruggable, transcription factor, which is a central catalyst in fibrosis-driven diseases.

TTI-101: TTI-101 is an orally bioavailable, small-molecule inhibitor of signal transducer and activator of transcription 3 (STAT3), a transcription factor whose upregulation and activation acts as a catalyst across critical pathways associated with fibrosis-driven diseases. TTI-101’s differentiated mechanism of action is designed to inhibit STAT3 to address the unmet need in fibrosis-driven diseases, without interfering with its other essential biological functions. TTI-101 has shown a robust pharmacokinetic profile, potency in inhibiting STAT3 activation and efficacy in animal models of fibrosis-driven diseases. In addition, in clinical trials performed to date, oral dosing with TTI-101 lowered levels of activated STAT3 in tumor tissue, was generally well-tolerated, and led to clinical responses in HCC and other tumor types.

The REVERTIPF ongoing clinical trial is evaluating the safety and efficacy of TTI-101 alone or in addition to nintedanib (OFEV) in patients suffering from IPF. The clinical trial is testing two different doses of TTI-101 compared to placebo using a Phase 2, randomized, double-blind, placebo-controlled design and is being conducted in the United States. Unblinded data from the REVERTIPF study is anticipated to be reported in the second half of 2025. ClinicalTrials.gov ID: NCT05671835

The REVERTLIVER CANCER ongoing clinical trial is evaluating the safety and efficacy of TTI-101 across three cohorts of patients with HCC: alone or in combination with standard of care treatments pembrolizumab (Keytruda) or atezolizumab (Tecentriq) and bevacizumab (Avastin or biosimilars Vegzelma, Alymsys, Zirabev, and Mvasi). The clinical trial is a Phase 1b/2 open-label study design being conducted in the United States. Preliminary topline data from the REVERTLIVER CANCER study is anticipated in the second half of 2025. ClinicalTrials.gov ID: NCT05440708

TTI-109: TTI-109, Tvardi’s second product candidate, is an oral, small-molecule, which is structurally related to, yet chemically distinct from, TTI-101 and is designed to enhance the ability to target STAT3. An IND application for TTI-109’s first human study is expected in the first half of 2025.

Management and Organization:
Following the Merger, the combined company will be headquartered in Houston, Texas and will be led by Tvardi’s CEO, Imran Alibhai, Ph.D., and other members of the Tvardi management team. The combined company’s board of directors will be comprised of six directors from Tvardi’s Board of Directors and one director from Cara’s Board of Directors.

About the Proposed Merger:
The transaction has been approved by the Boards of Directors of both companies and is expected to close in the first half of 2025, subject to certain closing conditions, including, among other things, approval by the stockholders of each company, the effectiveness of a registration statement to be filed with the SEC to register the shares of Tvardi common stock to be issued in connection with the Merger, Cara having a minimum amount of net cash as of the closing, and other customary closing conditions. In connection with the Merger, directors and officers of Cara and directors, officers and certain stockholders of Tvardi have executed support agreements, pursuant to which they have agreed to vote all their shares of capital stock in favor of the Merger.

Advisors:
Piper Sandler & Co. is serving as exclusive financial advisor to Cara Therapeutics. Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, P.C. is serving as legal counsel to Cara Therapeutics. Cooley LLP and Goodwin Procter LLP are serving as legal counsel to Tvardi.

Conference Call and Webcast:
The management teams of both companies will host an investor conference call and webcast today, December 18th, at 8:30am ET, to discuss the proposed Merger.

On December 18, 2024 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported preliminary clinical data for the triplet combination of avutometinib and sotorasib plus defactinib in the RAMP 203 Phase 1/2 study in KRAS G12C mutant advanced non-small cell lung cancer (NSCLC) (Press release, Verastem, DEC 18, 2024, View Source [SID1234649198]). No dose-limiting toxicities (DLTs) have been observed in the triplet combination. RAMP 203 continues to progress, with additional enrollment expected and an interim update is planned to be presented at a medical meeting in the second half of 2025.

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"We continue to make progress across our pipeline to develop novel therapies alone or in synergistic combinations that have the potential to improve outcomes in RAS/MAPK pathway-driven cancers. Defactinib, our oral FAK inhibitor, has been an important addition to multiple clinical trials with avutometinib to address key resistance mechanisms in parallel pathway signaling," said Dan Paterson, chief executive officer at Verastem Oncology. "Recently, we added defactinib to the combination of avutometinib and sotorasib in our RAMP 203 trial. Preliminary data for the triplet combination have shown a generally favorable tolerability profile and encouraging initial anti-tumor activity. We look forward to progressing enrollment and evaluating the safety and efficacy of the triplet combination in treating KRAS G12C mutant non-small cell lung cancer."

RAMP 203 Clinical Update

As of a November 21, 2024, data cutoff, three patients whose cancer previously progressed on a G12C inhibitor have been treated with the triplet combination of sotorasib 960 mg administered daily on a continuous schedule and avutometinib 3.2 mg twice-weekly (BIW) plus defactinib 200 mg twice-daily (BID). Avutometinib and defactinib are administered on a three out of four weeks schedule.​ Two of the three patients demonstrated initial tumor reductions of at least 20% at the first scan. As of the data cutoff, all three patients remain on treatment.​ With no DLTs observed in the first triplet combination cohort, the Company anticipates the enrollment of additional patients to the triplet combination prior to presenting the data at a medical meeting next year.​

As previously reported, the doublet combination of avutometinib with sotorasib has completed enrollment (n=28) in the G12C inhibitor treatment-naïve Stage 1 Part B cohort.​ The KRAS G12C inhibitor prior-treated Stage I Part B cohort is still enrolling patients and is anticipated to complete enrollment in early 2025. Patients in both cohorts continue to be followed for safety and efficacy to determine if observed efficacy supports expanded enrollment. The Company plans to complete enrollment and evaluate the safety and efficacy of the triplet combination, before expanding either of the doublet cohorts.

"We are encouraged by the initial data from the triplet combination of avutometinib and sotorasib plus defactinib in the RAMP 203 trial, which shows early evidence of tumor reductions for patients who have limited treatment options," said John Hayslip, M.D., chief medical officer at Verastem Oncology. "While the data matures for the doublet combination across cohorts, we are now focused on completing the enrollment in the triplet combination, guided by preclinical data that indicates that the addition of a FAK inhibitor increases the anti-tumor efficacy of avutometinib plus sotorasib in KRAS G12C mutant NSCLC models, and tumors that progress on a G12C-inhibitor treatment can be made to respond again upon treatment with a FAK inhibitor plus avutometinib. As planned, the triplet combination builds on the experience from the RAMP 201 study in recurrent low-grade serous ovarian cancer, where there was a clear advantage to adding defactinib. Based on the RAMP 201 results, we did an assessment of our clinical programs and made the decision to add defactinib to almost all our studies."

About RAMP 203

RAMP 203 is a Phase 1/2, multicenter, open label, dose evaluation/expansion study evaluating the efficacy and safety of avutometinib and sotorasib with or without defactinib in patients with KRAS G12C mutant non-small cell lung cancer (NSCLC) who have not been previously treated with a KRAS G12C inhibitor as well as in patients who have been previously treated with a KRAS G12C inhibitor (NCT05074810). RAMP 203 is being conducted in collaboration with Amgen.

About the Avutometinib and Defactinib Combination

Avutometinib is an oral RAF/MEK clamp that potentially inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors.

Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion. FAK activation has been shown to mediate resistance to multiple anti-cancer agents including RAF and MEK inhibitors.

Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK driven tumors as part of its Raf And Mek Program or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097;ENGOT-ov81/NCRI) (NCT06072781) an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC).

Verastem completed its rolling New Drug Application (NDA) submission to the to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy, in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810). Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024. RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award. FDA granted Orphan Drug Designation to the avutometinib and defactinib combination for the treatment of pancreatic cancer.

On December 18, 2024 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS), a commercial-stage biotechnology company, reported initial clinical results from its ongoing KYANITE-1 study evaluating inhaled KB707 in patients with solid tumors of the lung (Press release, Krystal Biotech, DEC 18, 2024, View Source [SID1234649199]). KB707 administered via inhalation demonstrated early evidence of monotherapy activity that was most pronounced in patients with advanced non-small cell lung cancer (NSCLC), where an objective response rate (ORR) of 27% and disease control rate (DCR) of 73% were observed as of data cut-off on December 6, 2024.

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"The inhaled local delivery of cytokine that maximizes efficacy and limits systemic toxicity is truly innovative," said Wen Wee Ma, MBBS, Vice Chair of Research and Director of the Novel Cancer Therapeutics Center at Cleveland Clinic. "To see potential benefit as a monotherapy in NSCLC patients who have progressed after standard of care treatments is very encouraging and provides much needed hope."

KYANITE-1 is an ongoing, open-label, multicenter, dose escalation and expansion study evaluating inhaled KB707 for the treatment of solid tumors of the lung. Treatments of either 108 PFU or 109 PFU of KB707 were evaluated in dose escalation, following which 109 PFU was selected for dose expansion. Frequency of KB707 administration has been consistent throughout dose escalation and expansion, with patients receiving KB707 via inhalation once weekly for the first three weeks, then once every three weeks. Trial objectives include evaluation of safety, tolerability, and tumor response measured using RECIST v1.1 criteria. Additional details of the KYANITE-1 study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.

The first patient in KYANITE-1 was dosed on April 17, 2024. A total of 37 patients were enrolled and received at least one dose of inhaled KB707, including 17 patients with a diagnosis of advanced NSCLC. All patients had malignant lesions in the lung at baseline.

Inhaled KB707 has been safe and generally well tolerated to date in this diverse, heavily pre-treated patient population with advanced disease, and amenable to administration in an outpatient setting. Treatment-emergent adverse events have been predictable and consistent with both the underlying disease and known adverse event profiles of interleukin-2 and interleukin-12. The majority of treatment-related adverse events have been mild to moderate in severity and transient, with no Grade 4 or 5 adverse events observed.

Clinical activity observed to date in the KYANITE-1 study has shown the most therapeutic benefit in patients with advanced NSCLC. As of the data cut-off, 11 NSCLC patients were evaluable for response with at least one radiographic scan and RECIST v1.1 evaluation. Patients included in the analysis were heavily pre-treated with 4 median lines of prior therapy and all had received at least one line of prior immunotherapy. In this NSCLC patient analysis cohort, an ORR of 27%, with three partial responses, has been achieved. DCR to date has been 73% with 7 out of 11 patients still remaining on treatment. Duration of treatment for patients included in the analysis ranged from 10.3 to 33.3 weeks as of data cut-off.

In addition to preliminary evidence of abscopal effect and treatment benefit outside of the lung, treatment responses in lesions of the lung were especially notable. Among the same 11 evaluable NSCLC patients, the ORR in target lung lesions specifically was 36%, with three partial responses and one complete response, and DCR was 82%.

"Signals of monotherapy activity with inhaled KB707, although early, are an exciting milestone for our program and highlight the significant potential of our vectorized cytokine approach in the treatment of difficult cancers," said Suma Krishnan, President of Research and Development of Krystal Biotech. "These data add to a rapidly growing clinical dataset, generated across multiple programs and patient populations, demonstrating that our HSV-1 platform can safely and repeatedly deliver functional genetic material to the lung and impact the course of disease. We are excited about the implications for our platform and the prospect of delivering meaningful clinical benefit to patients suffering from rare and serious lung diseases."

Based on positive initial results in monotherapy, the Company has amended the KYANITE-1 protocol to add two cohorts evaluating inhaled KB707 for the treatment of advanced NSCLC in combination with either anti-programmed cell death protein 1 (PD-1) therapy or anti-PD-1 therapy and chemotherapy. No patients have been enrolled in the combination expansion cohorts to date.

The Company expects to disclose detailed and updated results of KYANITE-1 at future scientific conference(s).

About IL-2, IL-12, and KB707

IL-2 and IL-12 are secreted cytokines with complementary functions promoting cell-mediated immunity in humans. Both IL-2 and IL-12 have been shown to elicit anti-tumor immune responses in preclinical or clinical models and have been extensively studied for their potential in cancer immunotherapy. Despite promising signs of efficacy, it has proven difficult to effectively harness IL-2 and IL-12 for therapeutic benefit, as systemic administration is often poorly tolerated, and their inherently short half-lives necessitate high dose levels and extremely frequent dose intervals. KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 directly to a patient’s tumor(s) and promote systemic immune-mediated tumor clearance. KB707 targets solid tumors that are accessible via intratumoral injection or inhalation.

On December 18, 2024 Applied DNA Sciences, Inc. (NASDAQ:APDN) ("Applied DNA" or the "Company"), a leader in PCR-based DNA technologies, reported that the State Institute for Drug Control of the Czech Republic (SÚKL) approved an application for a Phase I clinical trial of an investigational CD123-specific autologous CAR T-cell therapy by the Institute of Hematology and Blood Transfusion (ÚHKT/Eng: IHBT) in Prague for the treatment of relapsed and/or refractory acute myeloid leukemia (AML) (Press release, Applied DNA Sciences, DEC 18, 2024, View Source;id=331355&p=2353954&I=1206939-c7Z3G6f3m8 [SID1234649200]). UHKT-CAR123-01 utilizes Applied DNA’s synthetic DNA, Linea DNA, as a critical component in its manufacture.

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AML is a hematologic malignancy with a high rate of treatment failure for which current treatment options are often restricted to palliative approaches. Novel emerging methods leveraging advancements in genetic medicines, such as CAR T-cell therapies, can potentially improve outcomes of patients after relapse but have been difficult to establish for clinical use due to high costs and long manufacturing times predominantly attributable to the use of viral vectors.

UHKT-CAR123 seeks to address these issues by generating CD123-specific CAR T-cells in a non-viral workflow utilizing Linea DNA to reduce manufacturing costs and timelines. Preclinical data showed that ÚHKT’s Linea DNA-empowered non-viral workflow resulted in the rapid production of substantial and cost-efficient CAR T-cell yields with high potency[1].

"The use of Linea DNA illustrates our innovative approach to finding new and best-in-class treatments for patients with relapsed or refractory AML," stated Dr. Jan Vydra, principal investigator of the UHKT-CAR123 clinical trial.

Added Pavel Otáhal, Ph.D., scientific project leader at ÚHKT, "The Linea DNA platform enables the very rapid abiotic production of expression vectors usable for highly effective electroporation-based CAR-T manufacturing compared to plasmid-based vectors. The decreases in complexities and costs of developing autologous CAR-T technologies offer an innovative approach for the rapid clinical testing of novel types of CAR-T products. This is an incredible milestone for ÚHKT and one that we could not have achieved without the commitment of the Applied DNA team."

Applied DNA CEO Dr. James A. Hayward, said, "We congratulate ÚHKT on their progress into the clinic. Their accomplishment is also a significant milestone for Linea DNA as we look to support additional customers expected to initiate clinical trials in calendar 2025."

About Linea DNA

Linea DNA is an enzymatically produced, linear DNA manufactured by the Company’s proprietary, large-scale polymerase chain reaction ("PCR") based manufacturing platform, the Linea DNA platform. As an alternative to plasmid-based DNA, Linea DNA can be used to manufacture of a wide range of nucleic acid-based therapies and in vitro diagnostics, including mRNA therapies, DNA vaccines, cell and gene therapies, and molecular and genetic diagnostic tests.