On September 3, 2024 Primrose Bio, a leading biotechnology company specializing in technologies that improve the manufacturing of next-generation therapeutics, and ExPLoRNA Therapeutics, a pioneering biotechnology company advancing mRNA technologies, vaccines, and therapeutics, reported a strategic partnership aimed at jointly developing and marketing products for mRNA medicines (Press release, ExploRNA Therapeutics, SEP 3, 2024, View Source [SID1234646291]).

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The landscape of mRNA medicines is evolving, marked by an expanding pipeline of drug candidates, a diverse range of targeted therapeutic indications, and advancements in drug design and delivery. Consequently, the pharmaceutical industry is confronted with pressing challenges in manufacturing. To effectively address these challenges, innovative solutions are necessary.

Primrose Bio’s expertise in engineering high-performance RNA polymerases (Prima RNApols) enhances both the yield and fidelity of mRNA synthesis, effectively reducing double-stranded RNA (dsRNA) and lowering costs. This advancement meets the rigorous manufacturing demands for a wide range of mRNA-based applications in therapeutics and vaccines.

ExPLoRNA Therapeutics specializes in innovations of mRNA as a therapeutic platform, focusing on novel cap analogs vital for successful protein production. Their technology significantly increases protein production compared to standard cap analogs, limits double-stranded RNA formation, and brings down IVT cost, offering promising advancements in therapeutic effectiveness.

Together, Primrose Bio and ExPLoRNA will co-develop and offer RNA polymerases and capping compounds, co-optimized to enhance mRNA quality, manufacturing performance, and therapeutic safety and efficacy. This partnership provides mRNA drug developers with superior manufacturing solutions and innovative design options through advanced capping chemistry.

"We are excited to work with ExPLoRNA to bring industry-leading mRNA cap analogs to the market and pairing them with Primrose Bio’s RNA polymerases," said Helge Zieler, CEO of Primrose Bio. "This partnership will result in broader manufacturing and design options for companies developing mRNA therapeutics and vaccines, facilitating access to the most efficient capping chemistry available."

"Through joining forces, we’ll advance the mRNA as a modality from the first applications to the next generation," said Jacek Jemielity, CEO of ExPLoRNA Therapeutics. "We aim at creating the best synergies between our cap analogs and novel polymerases to surpass current manufacturing process and what is achievable with mRNA today."

On September 3, 2024 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported that Richard A. Miller, M.D., president and chief executive officer, and Jeffrey Arcara, chief business officer, will conduct one-on-one meetings with investors and present a corporate overview at the H.C. Wainwright 26th Annual Global Investment Conference, which is being held in New York from September 9-11, 2024 (Press release, Corvus Pharmaceuticals, SEP 3, 2024, View Source [SID1234646290]). The Company’s presentation will be on Tuesday, September 10 at 12:00pm ET.

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A webcast of the presentation will be available live and for 90 days following the event. The webcast may be accessed via the investor relations section of the Corvus website.

On September 3, 2024 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported several updates from the company’s ongoing registration-directed clinical trials of its potential best-in-class KIT mutant inhibitor, bezuclastinib (Press release, Cogent Biosciences, SEP 3, 2024, View Source [SID1234646288]).

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Patient enrollment is now complete in Cogent’s Phase 3 PEAK trial evaluating bezuclastinib in combination with sunitinib for the treatment of patients with gastrointestinal stromal tumors (GIST). Based on strong global patient interest, a total of 413 patients were enrolled in the study. In addition, Cogent recently completed a pre-planned interim futility analysis, and the Independent Data Monitoring Committee (IDMC) recommended continuing the PEAK study without modification. This pre-specified analysis was based on an assessment of progression-free survival (PFS) as determined by independent central review and did not include the option for early stopping due to efficacy. 

Separately, based on significant patient interest in the ongoing SUMMIT trial in nonadvanced systemic mastocytosis (NonAdvSM), Cogent also announced today that it expects to complete enrollment in this study during Q1 2025, approximately three months earlier than originally projected.

"We are excited to announce these important updates to the PEAK and SUMMIT studies today," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "Strong continued interest from patients around the world to participate in our bezuclastinib trials has allowed us to accelerate development and surpass our original enrollment timelines. Completing enrollment in our Phase 3 PEAK trial of bezuclastinib and sunitinib for second-line GIST patients several months ahead of schedule represents a significant milestone for the program and we are extremely grateful to the patients, families, caregivers, advocacy groups and clinical investigators for their participation in, and support of, the PEAK trial." 

PEAK is a randomized, open-label, global Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib alone in GIST patients previously treated with imatinib. The primary endpoint of the trial is median progression free survival (mPFS). PEAK is a registration study intended to support a New Drug Application (NDA) in GIST. 

SUMMIT is a randomized, blinded, global, registration-directed clinical trial evaluating bezuclastinib vs. placebo in NonAdvSM patients. The primary endpoint of the trial is mean improvement in patient symptoms measured at 24 weeks. SUMMIT is intended to be a registrational study designed to support a New Drug Application (NDA) in NonAdvSM. 

Appointment of Darara Dibabu as Vice President of Marketing
In addition to the updates to PEAK and SUMMIT trials, Cogent announced today that Mr. Dibabu has joined Cogent as the VP of Marketing. Mr. Dibabu has 25 years of experience in the biopharmaceutical industry, most recently as the Global Brand Lead of TUKYSA at Pfizer and SeaGen, where he led the global launch and marketing strategy of the product for metastatic breast cancer patients. Previously, he served in various roles of increasing responsibility at Seagen, Bayer and Merck. Mr. Dibabu holds a bachelor’s degree in Biology from the University of Southern California. In connection with Mr. Dibabu joining the company, he was granted an "inducement" equity award in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market.  The award was approved by the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, as an inducement material to Mr. Dibabu’s employment. The award consists of nonqualified options to purchase 100,000 shares of Cogent common stock with a 10-year term, at an exercise price of $10.74 per share, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of Mr. Dibabu’s employment and the remainder vesting in equal monthly installments over the subsequent 36 months, provided Mr. Dibabu’s remains employed through each such vesting date.

On September 3, 2024 Circle Pharma, a clinical-stage biopharmaceutical company dedicated to discovering and developing cell-permeable macrocycles as a new class of therapies, reported the successful closing of a $90 million Series D financing round, which includes the conversion of a convertible note (Press release, Circle Pharma, SEP 3, 2024, View Source;utm_medium=rss&utm_campaign=circle-pharma-closes-90-million-series-d-financing-to-advance-innovative-oral-macrocycle-therapies [SID1234646287]). The financing was led by The Column Group with participation from new and existing investors, including Nextech Invest and Euclidean Capital.

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The proceeds from the financing will be used to fund the clinical development of CID-078, Circle Pharma’s first-and-only-in-class cyclin A/B RxL inhibitor, and to support the development of the company’s portfolio of discovery programs built with its MXMO macrocycle platform.

"We are grateful for the continued support of such a strong syndicate of investors as we advance our pioneering work in macrocycle therapeutics," said David Earp, JD, Ph.D., CEO of Circle Pharma. "This financing not only enables us to progress CID-078 through critical stages of clinical development but also allows us to further advance our pipeline of innovative therapies targeting cancer and other serious diseases."

With this latest round of funding, Circle Pharma is well positioned to advance its mission of creating effective treatments for cancer and other serious illnesses.

On September 3, 2024 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported a scientific article in Science Advances suggesting that TALEN-edited MUC1 CAR T-cells could be a potential treatment option for advance-stage triple negative breast cancer (TNBC) patients with limited therapeutic options (Press release, Cellectis, SEP 3, 2024, View Source [SID1234646286]).

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Globally, breast cancer continues to be the most prevalent malignancy in women. Among all subtypes, triple-negative breast cancer (TNBC) stands out as the most aggressive form with high metastatic potential and poor survival rates.

Despite a few emerging targeted therapies under investigation, surgery, chemotherapy and radiation therapy continue to be the standard of care, and their success remains limited. As an alternative, Chimeric Antigen Receptor (CAR) T-cell therapies could hold promise for advance-stage TNBC patients as tumor-associated MUC1 antigen is overexpressed in a large number of patients thus offering a distinct target for treatment.

In this article, Cellectis described its multi-layered CAR T-cell engineering strategy using TALEN and synthetic biology to multi-armor CAR T-cells with synergistic functionalities to overcome the immunosuppressive tumor microenvironment (TME) of solid tumors. With this strategy, Cellectis demonstrates enhanced cytotoxic activity of MUC1 CAR T-cells armored with PD1KO, tumor-specific IL12 release and TGFBR2KO attributes, all of them catered towards the TNBC TME, in intravenous and intratumoral mouse models.

"Complexity of solid tumors decreases the efficacy of CAR T-cell therapies. With this pre-clinical study, we showed that TALEN-mediated multiplex editing can support CAR T-cells in effectively mounting an anti-tumor response to clear breast tumors, and that we can further decrease the dose of the treatment by injecting the CAR T cells intratumorally while still treating distant tumors. This innovative approach also allowed us to discover an unexpected cooperation between the edits in increasing safety, highlighting the potential capabilities of multiplex editing" said Piril Erler, PhD, Scientist II at Cellectis.

Importantly, intratumoral treatment effectively reduced local and distant tumors of large size using low doses of multi-armored MUC1 CAR T-cells. This pre-clinical data suggests that the benefits of antigen recognition are maintained at distant sites and highlights the potential to address metastasis with local administration.