On November 18, 2024 European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) reported to have adopted a positive opinion recommending the approval of Sarclisa in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM) who are ineligible for autologous stem cell transplant (ASCT) (Press release, Sanofi, NOV 18, 2024, View Source [SID1234648476]). A final decision is expected in the coming months.

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Dietmar Berger, M.D., Ph.D.

Chief Medical Officer, Global Head of Development at Sanofi

"The positive CHMP opinion is an important step forward for people with transplant-ineligible newly diagnosed multiple myeloma for whom effective front-line therapy may improve long-term outcomes. If approved, this Sarclisa-based combination could establish a new standard-of-care treatment approach for patients in the EU, helping to address a critical care gap in multiple myeloma treatment, and reinforcing Sarclisa’s potential as the anti-CD38 therapy of choice."

In September 2024, the US Food and Drug Administration (FDA) approved Sarclisa in combination with VRd for the treatment of adult patients with NDMM who are not eligible for ASCT, representing the first global approval for Sarclisa in the first line setting. In addition, the FDA granted orphan drug exclusivity for Sarclisa in the approved indication.

Sarclisa is currently approved in two indications for the treatment of certain adult patients with relapsed or refractory MM in more than 50 countries, including the US and EU.

First positive global phase 3 study combining anti-CD38 therapy with VRd to significantly improve PFS versus VRd alone in transplant-ineligible NDMM supports CHMP decision

The positive CHMP opinion is based on data from the IMROZ phase 3 study, which was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2024 annual meeting, European Hematology Association (EHA) (Free EHA Whitepaper) 2024 meeting, and published in The New England Journal of Medicine. IMROZ is the first global phase 3 study of a CD38 monoclonal antibody in combination with standard-of-care VRd to significantly improve progression-free survival (PFS) versus VRd alone. The safety and tolerability of Sarclisa observed was consistent with the established safety profile of Sarclisa and VRd with no new safety signals.

About Sarclisa

Sarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US FDA.

Currently Sarclisa is approved in more than 50 countries, including the US and EU, across two indications; Sarclisa is approved under an additional indication in the US. In Europe, based on the ICARIA-MM phase 3 study, Sarclisa is approved in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the European Union for patients with MM who have received at least 1 prior therapy. In the US, Sarclisa is also approved in combination with VRd as a first line treatment option for adult patients with NDMM who are not eligible for ASCT, based on the IMROZ phase 3 study.

Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. In addition, the company is evaluating a subcutaneous administration method for Sarclisa in clinical studies. The safety and efficacy of Sarclisa has not been evaluated by any regulatory authority outside of its approved indications and methods of delivery.

In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers.

On November 18, 2024 Cancer Targeted Technology (CTT), a privately-held Seattle-based biotechnology firm focusing on cancer diagnostics and therapeutics, reported that the National Cancer Institute awarded the Phase I portion ($400K) of the fast track Phase I/II ($2.4M) Small Business Innovation Research (SBIR) grant to develop a new drug to treat metastatic prostate cancer (Press release, Cancer Targeted Technology, NOV 18, 2024, View Source [SID1234648491]). The grant focuses on a promising new prodrug, CTT2274, that targets Prostate-Specific Membrane Antigen (PSMA) on prostate cancer and is designed to release a toxic drug, MMAE, within the cell that takes up the prodrug. PSMA is over-expressed on prostate cancer and expression increases as the cancer metastasizes and becomes castrate resistant. CTT’s unique phosphoramidate-based agents, bind irreversibly to PSMA and unlike other agents targeting PSMA, this distinctive mode of binding enhances uptake and internalization by tumor cells, leading to increased accumulation of the therapeutic payload and improved efficacy.

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Studies to date using CTT2274 treatment of mice bearing human prostate tumors have shown remission of tumor growth and an overall increase in survival. In addition, because of the prodrug release within the tumor cells, safety of the drug, at doses that are effective at inhibiting or reversing tumor growth, is excellent. In the Phase I portion of the grant, to be completed in Q2 2025, CTT will conduct additional non-clinical efficacy studies and manufacturing optimization. In Phase II of the grant, to be completed in Q2, 2026, CTT will conduct additional manufacturing and safety assessments necessary to advance CTT2274 to an Investigational New Drug (IND) application. These IND studies will support the initial clinical trial in metastatic prostate cancer planned for 2026.

"CTT2274 has a unique structure and linker that maximizes tumor uptake and allows for release of the chemotherapeutic drug only within the tumor cell thus minimizing potential side effects from the chemotherapy. No other prodrug like this is being developed for prostate cancer and CTT2274 holds great promise as a future treatment for men suffering from prostate cancer." stated Dr. Beatrice Langton-Webster, CEO of CTT and Principal Investigator on the grant. Visit CTT’s website at View Source to learn more about CTT2274.

On November 18, 2024 Alentis Therapeutics ("Alentis"), the clinical-stage biotechnology company developing treatments for Claudin-1 positive (CLDN1+) tumors and organ fibrosis, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ALE.P02 for the treatment of advanced or metastatic CLDN1+ squamous cancers irrespective of the organ of origin (Press release, Alentis Therapeutics, NOV 18, 2024, View Source [SID1234648462]). ALE.P02 is an investigational antibody-drug conjugate (ADC) targeting CLDN1, developed for CLDN1+ squamous cancers including but not limited to lung, head and neck, cervical and esophageal cancers.

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"We are encouraged by the FDA’s recognition of ALE.P02’s potential as a treatment for CLDN1+ squamous cancers," said Roberto Iacone, Chief Executive Officer of Alentis Therapeutics. "It reflects the importance of advancing ALE.P02 through clinical development, and it brings us one step closer to providing a new treatment option for patients."

Fast Track designation is designed to expedite the development and review of drugs that treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

"We have set out to develop ALE.P02 in the most rational way, following CLDN1 science and the clinical understanding of squamous cancers. The FDA’s support in this endeavor is certainly motivating," added Luigi Manenti, Chief Medical Officer of Alentis. "We are optimistic about the potential of ALE.P02 and look forward to the start of our first-in-human clinical trial in Q1 2025."

"Squamous cancers of various origin have been shown to overexpress CLDN1, making ALE.P02 a promising ADC to address the unmet medical needs of these patients," said Tony Mok Professor of Clinical Oncology at the Chinese University of Hong Kong. "CLDN1 is an exciting new target for ADCs and Alentis has been the frontrunner in developing anti-CLDN1 therapeutics."

About ALE.P02
ALE.P02 is a first-in-class ADC designed by linking a tubulin inhibitor, a potent cancer drug, to our antibody that specifically targets a unique CLDN1 epitope exposed on cancer cells. This combination could be a powerful new tool to fight the many squamous cancers that overexpress CLDN1 with less toxicity than traditional cancer drugs. The IND application for ALE.P02 to commence a Phase 1/2 clinical trial in advanced or metastatic CLDN1+ squamous solid tumors was recently cleared by the FDA.

On November 18, 2024 SK Biopharmaceuticals, a biotech company, reported that it has entered into an agreement with the Korea Institute of Radiological and Medical Sciences (KIRAMS), Korea’s premier research institution dedicated to the study and advancement of radiological and medical sciences, to collaborate on discovering and developing preclinical radiopharmaceutical drug candidates (Press release, SK biopharmaceuticals, NOV 18, 2024, View Source [SID1234648479]).

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This marks the first collaborative research agreement in which both sides will aim to discover radiopharmaceutical compounds and investigate novel oncological treatments, using actinium-225 (225Ac), an alpha-particle emitting radioisotope that selectively kills cancer cells. The research for radiopharmaceutical therapy (RPT) focuses on this application that has been gaining attention for its high potential in nuclear medicine.

SK Biopharmaceuticals has already initiated its 225Ac-based research as it secured a supply of the radioisotope from TerraPower Isotopes, a subsidiary of TerraPower, a nuclear innovation company whose investors include SK Biopharmaceuticals’ parent SK Inc. and Bill Gates.

SK Biopharmaceuticals and KIRAMS will seek to submit an Investigational New Drug application by 2027, as they leverage each other’s resources to optimize their drug discovery efforts. SK Biopharmaceuticals said it could significantly reduce the time and cost of new drug development, with KIRAMS’ researchers, facilities and equipment using the radioisotope, enabling the company to further accelerate in securing and expanding its RPT pipeline and capability.

The agreement is in line with SK Biopharmaceuticals’ so-called "RPT Roadmap" recently introduced to become a global leading RPT player by 2027, as the company will fortify its RPT business by discovering new drug compounds, extending supply and production capacities, and developing a tech platform, via strengthened internal assets and strategic partnerships.

In addition to the 225Ac supply agreement aligned with its roadmap, the company has in-licensed SKL35501 (FL-091) radiopharmaceutical compound targeting neurotensin receptor 1 (NTSR1) solid tumors.

Jinkyung Lee, President of KIRAMS, stated, "Through this joint research agreement, we aim to take a leading role in developing innovative radiopharmaceutical therapeutics, in alignment with the Ministry of Science and ICT’s strategic initiative to drive advancements in Radiation Biology. We look forward to contributing to the growth of the domestic radiopharmaceutical industry in close collaboration with SK Biopharmaceuticals."

Donghoon Lee, CEO and President of SK Biopharmaceuticals, said "This agreement is one of key steps in enhancing SK Biopharmaceuticals’ research capabilities. We will seek to spur SK Biopharmaceuticals to become a leading global RPT player through strengthened ties."

On November 18, 2024 GRAIL, Inc. (Nasdaq: GRAL), a healthcare company whose mission is to detect cancer early when it can be cured, reported that the first patient has been tested for eligibility with the investigational GRAIL Non-Small Cell Lung Cancer (NSCLC) ctDNA Assay in the global TROPION-Lung12 Phase 3 study evaluating adjuvant treatment regimens in patients with Stage I adenocarcinoma NSCLC (Press release, Grail, NOV 18, 2024, View Source [SID1234648480]). The study is sponsored by AstraZeneca (LSE/STO/Nasdaq:AZN) in collaboration with Daiichi Sankyo (TSE: 4568).

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The study, which is being conducted under an FDA-approved Investigational Device Exemption application, held by GRAIL, leverages GRAIL’s targeted methylation platform to detect ctDNA. With GRAIL’s blood-only approach, tissue analysis and bespoke panel development are not required, enabling simple integration into pharmaceutical clinical trial workflows. In TROPION-Lung12, patients will be screened with the GRAIL assay prior to surgery to inform eligibility for post-surgery randomization to an adjuvant treatment regimen (NCT06564844). Assay performance was previously reported in the Journal of Thoracic Oncology and presented at the 2023 North America Conference on Lung Cancer.

"We’re excited to continue our strategic collaboration with AstraZeneca with the use of our novel assay in the TROPION-Lung12 study. We hope this study will further demonstrate the potential of GRAIL’s Methylation Platform to enhance patient selection for cancer treatment," said Harpal Kumar, President, International Business & Biopharma, at GRAIL. "GRAIL’s ctDNA detection approach, which does not require tumor tissue, has the potential to offer oncologists a rapid, accessible method to help refine patients’ diagnostic and prognostic profiles for better guided cancer therapy. This is among the first times a ctDNA assay has been used in a clinical trial of early-stage lung cancer patients to identify those most likely to benefit from further treatment. As such, we hope this approach could provide substantial additional benefit for patients diagnosed with Stage 1 lung cancer."

"In TROPION-Lung12, screening for ctDNA is intended to identify the patients at an increased risk of disease recurrence after surgery and thus most likely to benefit from adjuvant therapy," said Cristian Massacesi, MD, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca. "The novel strategy we are deploying in this trial illustrates our commitment to both detect cancer earlier and use those early insights to enable more personalized treatment decisions for the benefit of patients."

In 2022, GRAIL announced a broad strategic collaboration with AstraZeneca to develop and commercialize companion diagnostic (CDx) assays for use with AstraZeneca’s therapies. GRAIL is committed to leveraging its blood-based methylation testing for patient care by developing fit-for-purpose diagnostics to enable precision oncology strategies with biopharma partners.