On April 16, 2024 Ferring Pharmaceuticals reported start up of new studies in the United States (U.S.) for three new ADSTILADRIN in BLadder cancEr (ABLE) clinical trials in patients with non-muscle invasive bladder cancer (NMIBC) with plans to expand select clinical trials outside of the U.S. later this year (Press release, Ferring Pharmaceuticals, APR 16, 2024, View Source [SID1234642117]). ADSTILADRIN was approved by the U.S. Food and Drug Administration (FDA) in December 2022 for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors.

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"ADSTILADRIN is the foundation of Ferring’s leadership mission in the uro-oncology therapeutic category and our ambition is for it to become the new standard of care and the backbone therapy for patients across the NMIBC disease spectrum," said Bipin Dalmia, Senior Vice President, Global Head, Uro-Oncology Franchise, Ferring Pharmaceuticals. "Embarking on a broad research and geographic expansion program is yet another demonstration of our commitment to the uro-oncology community, together with our significant manufacturing investments that recently enabled full availability of ADSTILADRIN to every appropriate U.S. patient who needs it."

The addition of three new ABLE clinical trials to the existing multi-year ADSTILADRIN studies program will expand the body of evidence for the intravesical non-replicating gene therapy:

ABLE-22: A Phase 2, randomized, multi-center, open label clinical study to evaluate the safety and efficacy of ADSTILADRIN alone or in combination with chemotherapy (gemcitabine and docetaxel) or immunotherapy (pembrolizumab) in adult patients with high-grade BCG-unresponsive NMIBC.
ABLE-32: A Phase 3b randomized, controlled clinical study to evaluate the safety and efficacy of ADSTILADRIN versus observation in patients with intermediate-risk NMIBC.
ABLE-42: A Phase 4, multi-center, open-label clinical study to evaluate the efficacy of retreatment with ADSTILADRIN in previously-treated patients with high-grade BCG-unresponsive NMIBC with CIS with or without papillary tumors who had not responded to the product at the first three-month assessment.
"ADSTILADRIN is an established monotherapy treatment option for patients with BCG-unresponsive NMIBC disease, an area where patients’ treatment options have been severely limited for decades," said Pierre-Yves Berclaz, M.D., Ph.D., Executive Vice President and Chief Science and Medical Officer, Ferring Pharmaceuticals. "These new trials underscore Ferring’s commitment to create solutions for patients by enlarging the body of evidence for this novel nonreplicating gene therapy."

The ADSTILADRIN clinical trial program includes two additional studies, the first being ABLE–41, a U.S. Real-World Evidence (RWE) Study announced in September 2023 and the second being a bridging study in Japan. The ABLE-22, ABLE-32 and ABLE-42 clinical trials announced today are yet another milestone for this comprehensive research program.

Additional ongoing studies in the ADSTILADRIN clinical trial program include:

ABLE-41 (NCT06026332): An ongoing, non-interventional RWE study following NMIBC patients aged 18 years or older with high-risk, BCG-unresponsive NMIBC who are being treated with ADSTILADRIN in a clinical setting and had not previously received this therapy in a clinical trial. The study is exploring early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting in these patients. The first patient was enrolled in September 2023.
Japan Phase 3B Study (000381): Single arm bridging study of high grade, BCG-unresponsive NMIBC patients.
Healthcare providers in the U.S. can learn more about the ADSTILADRIN clinical trial program by calling 1-888-FERRING (888-337-7464) and selecting option number one or emailing [email protected].

About ADSTILADRIN

ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high quantities of interferon alfa-2b protein, a naturally-occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).1

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.2 In the United States, bladder cancer is the seventh most common cancer, fourth among men3-4, and it is estimated that there will be approximately 83,190 new cases of bladder cancer in the U.S. in 2024.5 Historically, 75% of bladder cancer presents as NMIBC.6 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.5 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).7

INDICATION

ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.

On April 16, 2024 Calidi Biotherapeutics, Inc. (NYSE American: CLDI or "Calidi"), a clinical-stage biotechnology company developing a new generation of targeted immunotherapies, reported the pricing of its "reasonable best efforts" public offering of 15,197,500 shares of common stock (or pre-funded warrants in lieu thereof) and accompanying Series A Common Warrants, Series B Unit Warrants, with each unit consisting of one share of common stock and a Series B-1 Common Warrant and Series C Unit Warrants, with each unit consisting of one share of common stock and a Series C-1 Common Warrant (or common stock equivalents in lieu thereof) at an effective combined price of $0.40 per share and common warrants for aggregate gross proceeds of approximately $6.1 million, before deducting placement agent fees and other offering expenses (Press release, Calidi Biotherapeutics, APR 16, 2024, View Source [SID1234642116]). The common warrants will have an exercise price of $0.60 per share, and the Series A Common Warrants, Series B Unit Warrant and Series C Unit Warrant will be exercisable immediately. The common warrants will expire in five years (with respect to the Series A Common Warrant, the Series B-1, Warrant and the Series C-1 Common Warrant), twelve months (with respect to the Series B common warrants) and four months (with respect to the Series C common warrants) from the issuance date.

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The closing of the offering is expected to occur on or about April 18, 2024, subject to the satisfaction of customary closing conditions. The Company currently intends to use a portion of the net proceeds of this offering for working capital and general corporate purposes, and pre-clinical and clinical trials subject to the actual amount of proceeds received.

Ladenburg Thalmann & Co. Inc. is acting as sole placement agent in connection with the offering.

The securities described above are being offered pursuant to a registration statement on Form S-1 (File No. 333-276741) previously filed with the Securities and Exchange Commission (SEC) which became effective on April 15, 2024. The offering is being made only by means of a prospectus forming part of the effective registration statement. Copies of the preliminary prospectus and, when available, copies of the final prospectus, relating to the offering may be obtained on the SEC’s website located at View Source Electronic copies of the final prospectus relating to the offering may be obtained, when available, from Ladenburg Thalmann & Co. Inc., 640 Fifth Avenue, 4th Floor, New York, New York 10019, or by telephone at (212) 409-2000, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other 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 other jurisdiction.

On April 16, 2024 SCG Cell Therapy (SCG) and the Agency for Science, Technology and Research (A*STAR) reported the launch joint laboratories for cellular immunotherapies (Press release, SCG Cell Therapy, APR 16, 2024, View Source [SID1234642115]). This collaboration, at a combined funding of close to S$30 million supported under Singapore’s Research, Innovation and Enterprise 2025 Plan (RIE2025), aims to advance the development of induced pluripotent stem cell (iPSC) technology to produce novel cell therapies that meet Good Manufacturing Practice (GMP) standards. The collaboration will also establish a talent development programme to train the next generation of experts in this field, in accordance with current GMP and regulatory requirements.

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The research and application of new technologies are essential for addressing growing healthcare needs and maintaining long-term sustainability. However, turning laboratory innovations into practical clinical solutions poses significant challenges. These often involve developing manufacturing processes, validating analytical methods, and implementing automation and digitalisation to guarantee the stability and scalability of products.

The joint laboratories, established at SCG’s GMP facility and A*STAR’s research facility, leverage SCG’s and A*STAR’s proprietary technologies to develop scalable GMP-grade iPSC and therapeutic products. SCG contributes its specialised, automated cell therapy manufacturing technologies, while A*STAR brings its unique monoclonal antibody assets, iPSC banks, and expertise in process scaling and analytics.

This collaboration bridges the expertise between public sector research and development (R&D) and industry, consolidating resources from SCG Cell Therapy and A*STAR’s Bioprocessing Technology Institute (BTI) and Institute of Molecular and Cell Biology (IMCB) to advance innovative R&D towards GMP manufacturing. Additionally, it immerses researchers in the rigorously controlled GMP environment, facilitating the progression from research to clinical application.

"Cellular immunotherapies herald a new era of regenerative medicine, offering hope for patients with cancers and other serious illnesses. As a key player in T cell receptor (TCR) T cell therapeutics, SCG has developed in-house cGMP manufacturing capabilities to supply high-quality cell therapy products to patients. Through this first-of-its-kind joint collaboration with A*STAR, we bring together A*STAR’s advanced iPSC technology and bioprocessing capabilities with our expertise in GMP cell therapy manufacturing and clinical development, furthering our mission to provide affordable off-the-shelf cell therapy treatment options to patients", said Christy Ma, Chief Strategy Officer of SCG Cell Therapy.

"The discovery of iPSCs has revolutionised regenerative medicine, offering the potential for standardised, off-the-shelf cell therapies. Through this collaboration with SCG Cell Therapy, we aim to accelerate the translation of iPSC research into clinically viable therapies and strengthen Singapore’s position as a global leader in cell therapy innovation. By leveraging our complementary expertise and resources, the joint labs will not only advance iPSC technology for scalable, GMP-compliant cell therapy production but also serve as a platform for nurturing the next generation of talent in this transformative field," said Prof Koh Boon Tong, Executive Director, A*STAR’s BTI.

About iPSC

Induced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from adult cells. The iPSC technology was pioneered by Shinya Yamanaka’s lab in Kyoto, Japan, who showed in 2006 that the introduction of four specific genes encoding transcription factors could convert adult cells into pluripotent stem cells. He was awarded the 2012 Nobel Prize along with Sir John Gurdon "for the discovery that mature cells can be reprogrammed to become pluripotent". Pluripotent stem cells hold great promise in the field of regenerative medicine. Because they can propagate indefinitely, as well as give rise to every other cell type in the body (such as neurons, heart, pancreatic and liver cells), they represent a single source of cells that could be used to replace those lost to damage or disease.

On April 16, 2024 Kumquat Biosciences, a leader in translating breakthrough science into first-in-class therapeutics, reported a strategic and exclusive collaboration with Takeda to develop and commercialize a novel immuno-oncology small molecule inhibitor as a mono- and/or combination-therapy (Press release, Kumquat Biosciences, APR 16, 2024, View Source [SID1234642114]).

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Under the terms of the agreement, Kumquat granted Takeda an exclusive, global, and royalty bearing license to develop and commercialize a selected small molecule inhibitor. Subject to Kumquat’s option, Takeda will assume and fund all development and commercialization activities beyond the phase 1 trial activities led by Kumquat. Kumquat will receive up to $130 million in near-term payments and potentially more than $1.2 billion if all future clinical, regulatory, and commercial milestones are achieved during the term of the agreement plus tiered royalties on potential net sales of any commercial products resulting from this license.

"The advancement of this immuno-oncology small molecule program speaks to our ability to thrive on the challenges of drugging tough therapeutic targets of great potential," said Yi Liu, Chief Executive Officer of Kumquat. "We are proud of our persistent and innovative breakthrough, and we are thrilled to collaborate with Takeda, who shares our vision and strategy for realizing the benefit of small molecule-based transformative I/O treatments. This collaboration empowers this I/O program to advance to the clinic quickly and holds potential to benefit a broad population of cancer patients."

"We are excited to collaborate with Kumquat, an accomplished team with deep insights into this challenging target and therapeutic space," said P.K. Morrow, the Head of the Oncology Therapeutic Area Unit at Takeda. "This collaboration aligns with our mission to advance a cutting-edge pipeline focused on maximizing the benefit of the immune system to address the continued unmet needs of cancer patients. We look forward to working with Kumquat to accelerate the development of this exciting asset."

On April 16, 2024 Biomica Ltd., a clinical-stage biopharmaceutical company developing innovative microbiome-based therapeutics and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), reported that it will present preliminary data from its Phase 1 study of BMC128, in combination with immune checkpoint inhibitor (ICI) immunotherapy, in refractory patients with either non-small cell lung cancer (NSCLC), melanoma or renal cell carcinoma (RCC), who previously progressed on immunotherapy, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Biomica, APR 16, 2024, View Source [SID1234642113]). The conference will take place at McCormick Place in Chicago, Illinois, and will also be available virtually from May 31 to June 4, 2024.

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Presentation Details:

Title: Preliminary results from a First-in-Human (FIH), open-label Phase 1 study with BMC128, a rationally designed live bacterial consortium, in combination with nivolumab.
Session Type: Poster Session
Abstract Number: 8631
Date and Time: June 3, 2024, 1:30 PM – 4:30 PM (CDT)

Dr. Elran Haber, CEO of Biomica, stated: "We look forward to presenting our preliminary data at ASCO (Free ASCO Whitepaper), and to provide initial safety and efficacy results from our ongoing Phase 1 POC trial evaluating BMC-128 in combination with nivolumab in refractory patients. These results mark significant progress, laying the groundwork for the next phase in our clinical development process".

Additional information about the trial, can be found at: View Source (ClinicalTrials.gov Identifier: NCT05354102).

About BMC128:
BMC128 is a rationally designed microbial consortium identified and selected through a detailed functional microbiome analysis using PRISM, a proprietary high-resolution microbiome analysis platform powered by Evogene’s MicroBoost AI tech-engine.
Developed as a Live Bacterial Product (LBP), BMC128 is an LBP consortium comprised of four unique bacterial strains, natural inhabitants of the human intestinal tract, that harbor specific functional capabilities with the potential to enhance immunological therapeutic responses and facilitate anti-tumor immune activity through multiple biological processes.
Rationally-designed consortia are multi-strain products designed to restore diversity and specific functionality to a host’s microbial community with individually selected, cultured bacteria.