On July 5, 2023 Cardinal Health (NYSE: CAH) reported plans to release fourth-quarter and year-end financial results for its fiscal year 2023 on August 15, prior to the opening of trading on the New York Stock Exchange (Press release, Cardinal Health, JUL 5, 2023, View Source [SID1234633050]). The company will webcast a discussion of these results beginning at 8:30 a.m. Eastern.

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To access the webcast and corresponding slide presentation, visit Cardinal Health’s Investor Relations page. No access code is required. Presentation slides and a webcast replay will be available on the Investor Relations page for 12 months.

On July 5, 2023 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation (TPD) science to develop a new generation of small-molecule medicines and transform how disease is treated, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug (IND) application for CFT8919, an orally bioavailable BiDAC degrader designed to be potent and selective against EGFR L858R for non-small cell lung cancer (NSCLC) patients (Press release, C4 Therapeutics, JUL 5, 2023, View Source [SID1234633049]).

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This milestone marks C4T’s fourth IND clearance from its proprietary TORPEDO platform. In May 2023, C4T and Betta Pharmaceuticals entered into an exclusive licensing agreement for the development and commercialization of CFT8919 in Greater China, including Hong Kong SAR, Macau SAR and Taiwan. In China, approximately 693,000 patients were diagnosed with NSCLC in 2020 and approximately 40% of these cases are driven by the EGFR mutation. The L858R mutation is the second most common EGFR mutation, found in approximately 40% of NSCLC patients with EGFR mutations in China. Betta Pharmaceuticals is responsible for preparing and submitting a Clinical Trial Application to the National Medical Products Administration in China and plans to commence a first-in-human clinical trial of CFT8919 in China. C4T expects to initiate clinical trial activities outside Greater China following the completion of Betta Pharmaceuticals’ Phase 1 dose escalation study in Greater China.

About CFT8919
CFT8919 is an orally bioavailable allosteric BiDAC degrader that is designed to be potent and selective against EGFR bearing an oncogenic L858R mutation. In preclinical studies, CFT8919 is active in in vitro and in vivo models of L858R driven non-small cell lung cancer. Importantly, in preclinical studies, CFT8919 retains full activity against additional EGFR mutations that confer resistance against approved EGFR inhibitors including L858R-C797S, L858R-T790M, and L858R-T790M-C797S. In 2023, C4T and Betta Pharmaceuticals entered into an exclusive licensing agreement for the development and commercialization of CFT8919 in Greater China, including Hong Kong SAR, Macau SAR and Taiwan.

On July 5, 2023 Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, reported that it has concluded an instructive and successful End-of-Phase-2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA). As a result of the meeting, the company confirms that it is in agreement with the FDA on key elements of the randomized Phase 3 segment of RINGSIDE (Press release, Ayala Pharmaceuticals, JUL 5, 2023, View Source [SID1234633048]). The Agency accepted the Company’s selection of the 1.2 mg once daily dose being evaluated in the currently-enrolling Phase 3 and the completed and proposed clinical pharmacology plan. As previously agreed with the FDA on a seamless Phase 2/3 design, enrollment in the Phase 3 segment of RINGSIDE commenced in November 2022, and is continuing globally as planned, with target enrollment of 156 patients.

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"We are grateful for the FDA’s continued support and guidance, and very pleased to have reached agreement with the Agency on these items," said Ken Berlin, President and Chief Executive Officer of Ayala. "Supported by the clinical data generated from studies to date, we believe AL102 has the potential to be a best-in-class gamma secretase inhibitor and may offer a promising new treatment option to patients with unresectable, recurrent or progressive desmoid tumors. There are currently no approved therapies for these rare tumors. Current standards of care, which include off-label chemotherapy, radiation and tyrosine kinase inhibitors, are often poorly tolerated and offer inconsistent efficacy. We look forward to completing enrollment of the Phase 3 segment of this important study."

The Phase 3 segment of the RINGSIDE study is a double-blind, multi-center trial enrolling up to 156 patients with progressive disease, randomized between AL102 1.2mg dosed once daily or placebo. The primary endpoint is progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), and patient-reported Quality of Life (QOL) measures.

AL102 received Fast Track designation from the U.S. FDA for the treatment of progressing desmoid tumors, a rare disease with no currently approved treatments.

About RINGSIDE

The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial, with a seamless design, allowing Ayala to continue to Phase 3 without concluding the Phase 2 segment. The Phase 2 segment of the study (Part A) evaluated the efficacy, safety, tolerability, and tumor volume by MRI after 16 weeks of AL102 in patients with desmoid tumors. It enrolled 42 patients and evaluated 3 doses of AL102. Phase 3 of RINGSIDE (Part B) is a double-blind, placebo-controlled, clinical trial enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once-daily to placebo. For more information on the RINGSIDE Phase 2/3 study of AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282 (RINGSIDE).

About Desmoid Tumors

Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root, and chest wall, or other parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to surgical morbidity and a high rate of recurrence post-surgery. There are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

About AL102

AL102 is an investigational small molecule gamma secretase inhibitor (GSI) that is designed to potently and selectively inhibit Notch 1, 2, 3 and 4, and is currently being evaluated in the Phase 2/3 RINGSIDE clinical studies in patients with progressing desmoid tumors. AL102 is designed to inhibit the expression of Notch gene targets by blocking the final cleavage step by the gamma secretase required for Notch activation. Ayala obtained an exclusive, worldwide license to develop and commercialize AL102 from Bristol-Myers Squibb Company in November 2017. AL102 was granted U.S. FDA Fast Track Designation for the treatment of DT.

On July 4, 2023 pHion Therapeutics reported a joint Innovate UK award with Queen’s University Belfast (Press release, pHion Therapeutics, JUL 4, 2023, View Source [SID1234638968]). The £1million grant will fund a 24-month project to develop a multi-antigenic therapeutic vaccine for prostate cancer. This next generation vaccine is possible because of pHion’s innovative technology which delivers the mRNA into antigen-presenting cells in stealth mode. The company’s proprietary RALA platform uses a peptide as the ‘taxi’ to deliver the mRNA cargo, and because the peptide is not recognised as foreign, no adverse response is triggered, and the mRNA trains the immune cells to attack the cancer.

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The project is a collaboration between founder and CEO of pHion Therapeutics, Professor Helen McCarthy, and Dr Niamh Buckley, both from the School of Pharmacy at Queen’s. The £1million grant was awarded in the recent Innovate UK Biomedical Catalyst 2022 Round 2: Industry-led Research & Development funding competition.

Prostate Cancer accounts for 7.3% of all cancers worldwide (1.4 million cases) and 27.8% of all cancers in the UK. The 5-year survival rate for the most aggressive form, metastatic Castrate Resistant Prostate Cancer (CRPC), is only 28.9% in the UK; and the incidence is also set to rise by a further 12% by 2035. Worldwide, a recent Global Cancer Observatory (GCO) study has predicted a 60% increase to ~2.3 million new cases and 740,000 deaths by 2040.

As Professor Helen McCarthy explains: ‘Given the likely trajectory for this aggressive disease, there is an urgent need to develop a next generation vaccine that can reduce mortality rates. Our vision is to begin large-scale toxicology studies by the end of the project and, from there, to progress to clinical trials."

"The investment from Innovate UK will support growth in the global use of pHion’s unique technology, and further strengthen the UK’s position in the emerging gene therapy market."

On July 4, 2023 OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE) reported that the United States Patent and Trademark Office has granted a new patent protecting Tedopi, a therapeutic cancer vaccine treating HLA-A2 positive patients after secondary resistance to PD-1/PD-L1 immune checkpoint inhibitor treatment (Press release, OSE Immunotherapeutics, JUL 4, 2023, View Source [SID1234633043]). The new patent further improves the unique value proposition of Tedopi and provides protection until year 2037 in the US.

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This US patent recognises the innovation of a multiepitope combination (all peptides included in Tedopi) administered after failure to PD-1/PD-L1 immune checkpoint inhibitor in HLA-A2 positive non-small cell lung cancer (NSCLC) patients. This further protection for Tedopi, OSE Immunotherapeutics’ most advanced late-stage asset, adds significant value to the Company’s product portfolio.

Nicolas Poirier, CEO of OSE Immunotherapeutics, said: "We are very pleased to expand our patent portfolio internationally with this new US patent strengthening the protection rights for Tedopi in the significant US market. This patent represents an additional milestone in the product’s clinical development based on the first Phase 3 positive results in non-small cell lung cancer after checkpoint inhibitor escape in secondary resistance. These data show a significant overall survival benefit, an improved quality of life and a better safety profile versus chemotherapy. The next confirmatory Phase 3 trial under preparation in second line treatment will address the same high unmet medical need. Tedopi presents a differentiated mechanism of action activating tumor specific T cells after acquired resistance of immune checkpoint inhibitor."

This patent family, focused on the same targeted population, has been filed internationally in other territories and has already been granted previously in Japan.

This population in second line treatment after failure to PD-1/PD-L1 inhibitor treatment, targeted with Tedopi, is estimated to be up to 100,000 patients per year in 7 major markets across the US, Europe, China and Japan. This estimate is based on HLA-A2-positive patients accounting for about 45% of all NSCLC patients, as well as the large and growing use of anti-PD-1/PD-L1 therapies and their failure rate.