On September 11, 2023 Cellares, the first Integrated Development and Manufacturing Organization (IDMO) dedicated to clinical and industrial-scale cell therapy manufacturing, and Lyell Immunopharma (NASDAQ: LYEL), a clinical‑stage T-cell reprogramming company advancing a diverse pipeline of cell therapies for patients with solid tumors, reported Lyell will evaluate Cellares’ automated manufacturing platform, the Cell Shuttle, through Cellares’ Technology Adoption Partnership (TAP) program (Press release, Lyell Immunopharma, SEP 11, 2023, View Source [SID1234635069]). As part of the collaboration, the companies have agreed on a proof-of-concept technology transfer process for the manufacture of Lyell’s LYL797 CAR T-cell therapy, using the Cell Shuttle.

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CAR T-cell therapy is a personalized immunotherapy that takes a patient’s own T cells and modifies them to recognize and kill cancer cells that have a specific biomarker. LYL797 is an investigational CAR T-cell therapy in development for the treatment of solid tumors that express ROR1, a protein present on the surface of various solid tumors. LYL797 is enhanced with Lyell’s novel genetic and epigenetic reprogramming technologies designed to generate highly tumor-reactive, longer-lasting functional T cells. Lyell is enrolling patients with triple-negative breast cancer and non-small cell lung cancer in a Phase 1 clinical trial evaluating LYL797.

"We are excited to work with Cellares to evaluate their innovative automated manufacturing processes as part of our overall manufacturing strategy to efficiently, rapidly, and cost-effectively scale manufacturing capacity for our CAR T-cell product candidates for future clinical trials and potential commercialization," said Stephen Hill, Chief Operating Officer of Lyell. "We are impressed with the progress Cellares has made with their manufacturing capabilities, and the commitment and vision of the Cellares team to apply these technologies to help deliver new and potentially transformative therapies to patients."

Cellares’ TAP program offers cell therapy developers a swift and low-risk pathway to embrace the company’s automated manufacturing technology for their pipeline products. Lyell is utilizing this program to assess the automated manufacturing process and generate data that validates the Cell Shuttle’s viability as a manufacturing option for CAR T-cell therapies. Cellares partners with prominent cell therapy developers through its TAP program to integrate the Cell Shuttle as a GMP manufacturing solution in both clinical and commercial stages at their IDMO Smart Factories.

"By integrating LYL797 into our TAP program, we seek to demonstrate the ability to seamlessly adapt Lyell’s CAR T-cell manufacturing process to our Cell Shuttle platform," said Fabian Gerlinghaus, CEO of Cellares. "This collaboration represents a significant step towards fulfilling our vision of accelerating access to life-saving cell therapies, reducing process failure rates, and meeting total patient demand through the efficient utilization of the Cell Shuttle platform at global scale."

Cellares’ innovative manufacturing technology transforms autologous and allogeneic cell therapy processes, covering nearly 90% of cell therapy modalities. Through their TAP program, Cellares can facilitate the automation and tech transfer of manual processes onto the Cell Shuttle manufacturing platform in just six months. This program allows cell therapy developers to seamlessly integrate their processes onto a Cell Shuttle at any stage of development – from pre-clinical to post-regulatory approval. With automation, standardization, and software-defined manufacturing (SDM), subsequent tech transfers become instant to any other Cell Shuttle into any IDMO Smart Factory worldwide.

Please visit cellares.com/partnering/ to learn more about the TAP program and request a meeting with a business development representative.

On September 11, 2023 Laminar Pharma reported that the U.S. Food & Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to LAM561 (idroxioleic acid, sodium) an investigational drug candidate for patients with Pediatric-Type Diffuse High Grade Glioma (pdHGG) (Press release, Laminar Pharma, SEP 11, 2023, View Source [SID1234635068]).

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"Pediatric patients who develop Pediatric-Type Diffuse High Grade Glioma face a significant unmet need with limited treatment options, presenting differential characteristics from adult patients with high grade glioma that need to be considered during their treatment" said Dr. Victoria Llado, Chief Scientific Officer of the US subsidiary, Laminar Pharma Inc. "The Rare Pediatric Disease Designation highlights the serious and life-threatening manifestations of this rare disease in the pediatric population and supports our mission to provide LAM561 as a potential new treatment option for patients suffering from Pediatric-Type Diffuse High Grade Glioma."

The RPD program is intended to encourage the development of new drugs for the treatment of rare pediatric diseases. RPD Designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for LAM561 for the treatment of pdHGG is approved by the FDA, Laminar Pharma might be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application.

LAM561 is a synthetic fatty acid which has previously been granted with the designation of Orphan Drug/Medicinal Product for the treatment of malignant glioma in adult and pediatric patients by the FDA and the European of Medicines Agency (EMA), It also received Fast Track designation from the FDA in October 2022 for the treatment of glioblastoma. The clinical development of LAM561 includes two successfully completed studies to determine its safety and tolerability in adult patients (a Phase I/IIa in patients with advanced solid tumors and a Phase Ib study in patients with newly diagnosed glioblastoma in combination with the standard of care) and other two clinical trials that are being currently conducted: A Phase IIb/III clinical study for the treatment of adult patients with newly diagnosed glioblastoma in combination with standard of care in Europe; and a Phase I/IIa study to determine the safety, tolerability and potential clinical activity in pediatric patients with advanced solid tumors (including malignant glioma) in the US, as part of a pediatric program initiated by Laminar in accordance with its commitment with this population.

On September 11, 2023 Immunocore Holdings Plc (Nasdaq: IMCR), a commercial-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious diseases and autoimmune conditions, reported that management will present at the following investor conferences in September (Press release, Immunocore, SEP 11, 2023, View Source [SID1234635067]):

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H.C. Wainwright Healthcare Conference
Fireside Chat: Monday, September 11, 2023, at 2:30 p.m. EDT
Morgan Stanley Healthcare Conference
Fireside Chat: Tuesday, September 12, 2023, at 5:30 p.m. EDT
Baird Healthcare Conference
1×1 and small group meetings: Wednesday, September 13, 2023
2023 Cantor Global Healthcare Conference
Fireside Chat: Thursday, September 28, 2023, at 8:00 a.m. EDT

Where relevant, the presentations will be webcast live and can be accessed by visiting ‘Events’, under ‘News & Events’, via the ‘Investors’ section of Immunocore’s website at www.immunocore.com. Following the events, a replay of the presentations will be made available for a limited time.

On September 11, 2023 Moderna, Inc. (NASDAQ: MRNA, "Moderna") and Immatics N.V. (NASDAQ: IMTX, "Immatics"), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, reported a strategic research and development collaboration to pioneer novel and transformative therapies for cancer patients with high unmet medical need (Press release, Immatics, SEP 11, 2023, View Source [SID1234635065]). This broad multi-platform collaboration will leverage the deep scientific expertise and core operational capabilities of both companies, combining Immatics’ TCR platform with Moderna’s cutting-edge mRNA technology, and span various therapeutic modalities including bispecifics, cell therapy and cancer vaccines.

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The strategic R&D collaboration between Moderna and Immatics focuses on three pillars:

Applying Moderna’s mRNA technology for in vivo expression of Immatics’ next-generation, half-life extended TCR bispecifics (TCER) targeting cancer-specific HLA-presented peptides.
Enabling the discovery and development of novel mRNA-based cancer vaccines by leveraging Moderna’s deep knowledge of mRNA science and customized information from Immatics’ wealth of tumor and normal tissue data included in the target discovery platform XPRESIDENT and its bioinformatics and AI platform XCUBE.
Evaluating Immatics’ IMA203 TCR-T therapy targeting PRAME in combination with Moderna’s PRAME mRNA-based cancer vaccine. The collaboration contemplates conducting preclinical studies and a Phase 1 clinical trial evaluating the safety and efficacy of the combination with the objective of further enhancing IMA203 T cell responses.

"We are excited to embark on this strategic collaboration with Immatics, a pioneer in developing innovative cancer immunotherapies. This partnership presents a groundbreaking opportunity to leverage our mRNA technology alongside Immatics’ TCR platform, potentially diversifying and augmenting the way we approach cancer treatment. We believe this collaboration will accelerate the development of novel oncology therapies and bring us one step closer to providing significant benefits for patients with high unmet medical needs," said Rose Loughlin, Ph.D., Moderna’s Senior Vice President for Research and Early Development.

"We are thrilled to join forces with Moderna in our quest to pioneer innovative and transformative therapies to combat cancer. We believe Immatics’ cancer target and TCR platforms, along with Moderna’s cutting-edge mRNA technology, represent a powerful combination that has the potential to deliver meaningful benefits to cancer patients," said Toni Weinschenk, PhD, Chief Innovation Officer at Immatics. "The rapid advancement of our first 2 TCER programs into the clinic, with additional TCER compounds fueling our pre-clinical pipeline, underscores our commitment to develop innovative therapeutics. We are confident that we can explore the optimal delivery of TCER molecules through this collaboration to maximize clinical benefit in a broad patient population," added Carsten Reinhardt, MD, PhD, Chief Development Officer of Immatics.

About the Collaboration
Under the terms of the agreement, Immatics will receive an upfront payment of $120 million. Immatics will also receive research funding and is eligible to receive development, regulatory, and commercial milestone payments that could exceed $1.7 billion. Immatics is also eligible to receive tiered royalties on global net sales of TCER products and certain vaccine products that are commercialized under the agreement. Under the agreement, Immatics has an option to enter into a global profit and loss share arrangement for the most advanced TCER.

Moderna will lead the clinical development and commercialization of cancer vaccines and TCER therapeutics resulting from the collaboration. Immatics will be responsible for conducting the preclinical studies and a potential Phase 1 clinical trial investigating IMA203 TCR-T in combination with the PRAME mRNA vaccine to further enhance IMA203 T cell responses. Each party will retain full ownership of its investigational PRAME compound, and the parties will fund the clinical study on a cost sharing basis.

Within the collaboration, preclinical activities conducted by Immatics will be managed by the Immatics Discovery Unit, a recently created internal division at Immatics integrating its technology platforms into one interdisciplinary team focused on all early-stage preclinical pipeline and collaboration programs.

The collaboration is subject to customary antitrust clearance in the United States.

On September 11, 2023 GSK plc (LSE/NYSE: GSK) reported that the Ministry of Health, Labour and Welfare (MHLW), Japan, has accepted for review a new drug application (NDA) for momelotinib, a potential new medicine with a differentiated mechanism of action that may address the significant medical needs of myelofibrosis patients, especially those with anaemia (Press release, GlaxoSmithKline, SEP 11, 2023, View Source [SID1234635064]). The NDA is based on data from the pivotal phase III trials SIMPLIFY-1 and MOMENTUM.

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Myelofibrosis is a blood cancer that can lead to splenomegaly (enlarged spleen); constitutional symptoms such as fatigue, night sweats, and bone pain; and severely low blood counts, including anaemia and thrombocytopenia.[1],[2],[3] About 70% of patients diagnosed with primary myelofibrosis and about half of patients diagnosed with secondary myelofibrosis in Japan have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease.[4],[5],[6],[7],[8],[9] Patients who are transfusion dependent have a poor prognosis and shortened survival.[10],[11],[12],[13],[14],[15],[16],[17],[18]

Momelotinib is not currently approved in any market.

About momelotinib

Momelotinib has a differentiated mechanism of action, with inhibitory ability along three key signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type I (ACVR1).[19],[20],[21],[22] Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly.[19],[21],[22] Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anaemia.[19],[20],[21],[22]

About myelofibrosis

Myelofibrosis is a rare blood cancer that disrupts the body’s normal production of blood cells as a result of dysregulated JAK-signal transducer and activator of transcription protein signalling. The clinical hallmarks of myelofibrosis are progressive splenomegaly (enlarged spleen), anaemia and debilitating symptoms attributable to ineffective hematopoiesis and excessive production of proinflammatory cytokines.[23] Myelofibrosis patients with anaemia require additional supportive care, including transfusions, and have poor outcomes.[24],[25] Myelofibrosis affects approximately 1 in 500,000 people worldwide, with up to 5,000 patients impacted in Japan.[10],[19],[26],[27]

About the pivotal SIMPLIFY-1 clinical trial

SIMPLIFY-1 was a multicentre randomised, double-blind, phase III study that compared the safety and efficacy of momelotinib to ruxolitinib in patients with myelofibrosis who had not received prior treatment with a JAK inhibitor. SIMPLIFY-1 met its primary endpoint, demonstrating non-inferiority of momelotinib to ruxolitinib in spleen volume response (reduction by 35% or greater), and substantial improvements in transfusion independence rates.[28],[29]

About the pivotal MOMENTUM clinical trial

MOMENTUM was a global, randomised, double-blind phase III clinical trial of momelotinib (MMB) versus danazol (DAN) in patients with myelofibrosis who were symptomatic and anaemic and had been previously treated with an approved JAK inhibitor. The MOMENTUM trial met all its primary and key secondary endpoints, with respect to splenic response, constitutional symptoms and transfusion independence. Results from the 24-week treatment period were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and subsequently published in The Lancet.[22] ,[30]