On April 26, 2024 Medigene reported its first quarterly report (Presentation, MediGene, APR 26, 2024, View Source [SID1234642454]).

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On April 26, 2024 Arbele, a clinical-stage biotechnology company focused on the development of novel immunotherapies targeted for advanced gastrointestinal cancers, reported the positive recommendation from the second meeting of the Data Monitoring Committee (DMC) to continue the A001 Phase I clinical trial evaluating cabotamig (ARB202) in patients with advanced gastrointestinal (GI) cancers without modification to the trial protocol (Press release, ARBELE, APR 26, 2024, View Source [SID1234642400]). The DMC, composed of a group of independent experts, arrived at this recommendation after review of the interim safety data of patients enrolled in the A001 trial.

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This safety assessment was based on the review of safety data from 18 patients, including patients that have been treated with multiple doses of cabotamig. This positive recommendation confirms tolerability profile of cabotamig is consistent with published safety data of T-cells engagers in other indications.

"We are greatly indebted to the courageous subjects who have participated in this early study of cabotamig that have allowed us better understand the safety profile while optimizing the dosing of this promising therapy" said Dr Dennis Wong, Chief Medical Officer at Arbele.

About A001 Trial

The A001 trial is a multi-center, open label, FIH continuous study designed to evaluate the safety, tolerability, PK, PD, and anti-tumor activity of cabotamig administered intravenously in patients with unresponsive advanced GI malignancies expressing CDH17. The first part of the study consists of a dose-escalation stage (single ascending doses (SAD) Phase Ia) enrolling approximately 28 patients, and then a multiple dose ascending/ranging study (MAD).

Except for colorectal cancer patients, all other patients are screened for tumors expressing CDH17 using TibTech proprietary automated tissue diagnostic platform. If CDH17 is expressed, patients will be eligible to enter screening to be dosed. Colorectal cancer patients may be eligible without CDH17 marker testing.

For more information about A001, visit View Source

About Cabotamig

ARB202 is a first-in-class bispecific antibody based on Arbele’s patented CDH17 biomarker. It targets both CDH17 on GI cancer as well as CD3 on T cells. The unique differential binding affinities of ARB202 toward CDH17 and CD3 allows it to have high specificity and cytotoxicity, while avoiding the "on-target" normal CDH17 expressing cells. Preclinical data showed that ARB202 can facilitate T-cell attachment to cancer cells, activation and release of IL-2, thereby demonstrating target engagement activation and cytotoxicity.

About CDH17

Cadherin-17 (CDH17) is an adhesion molecule that binds to self and integrin α2β1. Its expression in humans is limited to gastrointestinal tissue, most studied in the adherens junctions along with E-cadherin on the basal lateral surface of colon epithelial cells. Its expression is tightly regulated and believed to be involved in the tight control of solutes between epithelial cells in the gut which prevents the movement of water and proteins into the lumen. Its aberrant expression at the cell surface in GI neoplastic cells has been well documented. CDH17 has been shown to be involved in cancer progression and is associated with poor prognosis. The soluble form, sCDH17 was previously found to increase with tumor staging and decrease with tumor debulking. Given CDH17 is expressed de novo or overexpressed at abnormally levels in GI cancers including CRC, GEJ, CCA, and PDAC, it provides a way to target T-cells to GI malignant cells within a suppressive tumor micro-environment.

On April 26, 2024 Ractigen Therapeutics, a leader in the development of small activating RNA (saRNA) therapeutics, reported that the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational New Drug (IND) application for RAG-01, a groundbreaking saRNA therapy targeting non-muscle invasive bladder cancer (NMIBC) (Press release, Ractigen, APR 26, 2024, View Source [SID1234642399]). This approval facilitates the launch of U.S. clinical trials, following the successful initiation of a Phase I trial in Australia.

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RAG-01 is a pioneering therapy in bladder cancer treatment, representing the first of its kind to specifically target and activate the p21 tumor suppressor gene. This gene is critical in regulating cell cycle progression and is a key component in stopping the growth of cancer cells. By activating p21, RAG-01 offers a targeted approach to potentially curb the progression of NMIBC, a prevalent form of bladder cancer.

"FDA IND approval for RAG-01 is a major achievement for Ractigen and a significant advancement for saRNA technology worldwide," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "This first-in-class saRNA therapy harnesses the power of RNAa to target the p21 gene, offering a promising new option for patients with limited treatment choices. This approval validates the potential of RAG-01 as a leading saRNA therapy and strengthens our position as innovators in RNA-based treatments."

About RAG-01: RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. Traditionally considered "undruggable," p21 presents a unique opportunity for saRNA-based targeted activation. The drug, delivered through intravesical instillation using Ractigen’s proprietary LiCO delivery technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models. Currently, the Phase I clinical trial of RAG-01 in Australia has successfully enrolled and dosed the first three patients. Its development marks a significant stride in RNAa based therapies, addressing the unmet needs of NMIBC patients.

About NMIBC: NMIBC represents 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. RAG-01’s development is a significant step towards addressing this substantial unmet need in bladder cancer therapy.

About LiCO: LiCO, Ractigen’s proprietary extra-hepatic delivery system, enables the delivery of duplex RNA into a variety of tissues and organs which are hard to reach by conventional approach. It supports multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal, providing profound and durable activity. Its versatility and effectiveness have been validated in many preclinical studies, marking it as a cornerstone in Ractigen’s therapeutic arsenal.

About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.

On April 26, 2024 Citius Pharmaceuticals Inc. (Nasdaq: CTXR) ("Citius" or the "Company"), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, reported that it has entered into definitive agreements for the purchase of an aggregate of 21,428,574 shares of its common stock and accompanying warrants to purchase up to an aggregate of 21,428,574 shares of its common stock, at a purchase price of $0.70 per share and accompanying warrant in a registered direct offering (Press release, Citius Pharmaceuticals, APR 26, 2024, View Source [SID1234642398]). The warrants will have an exercise price of $0.75 per share, will be exercisable six months from the date of issuance, and will expire five years from the initial exercise date. The closing of the offering is expected to occur on or about April 30, 2024, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The aggregate gross proceeds to the Company from the offering are expected to be approximately $15 million, before deducting the placement agent fees and other offering expenses payable by the Company. Citius currently intends to use the net proceeds from the offering for general corporate purposes, including pre-clinical and clinical development of our product candidates and working capital and capital expenditures.

The securities described above are being offered pursuant to a "shelf" registration statement (File No. 333-277319) filed with the Securities and Exchange Commission ("SEC") on February 23, 2024 and declared effective on March 1, 2024. The offering is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. The prospectus supplement and the accompanying prospectus relating to the securities being offered will be filed with the SEC and be available at the SEC’s website at www.sec.gov. Electronic copies of the prospectus supplement and the accompanying prospectus relating to the securities being offered may also be obtained, when available, by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by telephone at (212) 856-5711 or e-mail at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On April 26, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported that it has received a $15 million grant from the California Institute for Regenerative Medicine (CIRM) to support the clinical development of ALLO-316, an AlloCAR T investigational product targeting CD70 in development for the treatment of advanced or metastatic renal cell carcinoma (RCC) (Press release, Allogene, APR 26, 2024, View Source [SID1234642396]).

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"CAR T has transformed the treatment of hematologic malignancies but there remains a significant opportunity to apply this innovation to solid tumors," said Zachary Roberts, M.D., Ph.D., Executive Vice President, Research & Development and Chief Medical Officer of Allogene. "We believe this CIRM award validates the remarkable inroads we have made in our TRAVERSE trial to date and the therapeutic potential ALLO-316 has for patients with advanced RCC who have failed standard therapies. We look forward to advancing this trial with the added support of this grant and are grateful for the recognition from the CIRM reviewers of the potential for ALLO-316 to make a difference for patients."

Metastatic RCC is the most common kidney cancer globally and there are limited options for treatment after treatments with checkpoint blockers and targeted therapy have failed. It is a disease in need of innovation as current therapies are based on a few mechanistic targets and complete response rates are low. The five-year survival rate for patients with advanced kidney cancer is less than 17%1.

The grant will support the ongoing Phase 1 TRAVERSE trial which assesses safety, tolerability and preliminary efficacy of ALLO-316 in advanced RCC that has progressed despite standard therapy. Initial data from the TRAVERSE trial, presented at AACR (Free AACR Whitepaper) 2023, showed promising response rates and early anti-tumor activity with deepening responses over time in participants with a marked unmet medical need. In the TRAVERSE trial, ALLO-316 has demonstrated the potency of the Dagger technology, which selectively eliminates CD70 positive, alloreactive host immune cells, thus delaying or preventing premature rejection of AlloCAR T cells by the patient’s immune system. ALLO-316 has shown marked expansion and persistence both in preclinical experiments and in clinical trial patients, even when combined with comparatively less-intense lymphodepletion regimens. The intent of this grant will be to facilitate completion of the Phase 1 portion of the trial, including expansion of clinical sites to increase access for diverse patient populations. Additionally, the grant will support translational and clinical analyses to inform a recommended Phase 2 regimen.

"This clinical study has the potential to demonstrate the value of Chimeric Antigen Receptor (CAR) T cell therapy in solid cancers such as kidney cancer with a high unmet medical need," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.

Details on a potentially cornerstone safety algorithm discovered during the initial portion of the Phase 1 TRAVERSE trial, which may facilitate expanded use of CAR Ts in solid tumors, is planned for a publication in Q2 2024. A more comprehensive data update from the ongoing trial is planned for later in 2024.

About ALLO-316 (TRAVERSE)
ALLO-316, an AlloCAR T investigational product targets CD70, which is highly expressed in renal cell carcinoma (RCC). CD70 is also selectively expressed in several cancers, creating the potential for ALLO-316 to be developed across a variety of both hematologic malignancies and solid tumors. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic clear cell RCC. In March 2022, the U.S. Food and Drug Administration granted Fast Track Designation (FTD) based on the potential of ALLO-316 to address the unmet need for patients with difficult to treat RCC who have failed standard RCC therapies.