On August 19, 2025 Kelonia Therapeutics, Inc., a biotech company revolutionizing in vivo gene delivery, reported that the first patient has been dosed in the inMMyCAR study, a Phase 1 clinical trial evaluating KLN-1010, a novel in vivo gene therapy that generates anti-BCMA CAR-T cells, in patients with relapsed and refractory multiple myeloma (Press release, Kelonia Therapeutics, AUG 19, 2025, View Source [SID1234655389]).

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"KLN-1010 offers hope to multiple myeloma patients who can’t access today’s CAR-T cell therapies," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. "This first-in-human dosing marks a major milestone toward a future where CAR-T benefits can be delivered without preparative chemotherapy or bespoke manufacturing delays, and at virtually any hospital around the world. Beginning our Phase 1 inMMyCAR study is not only pivotal for patients, but for the entire field. It’s the starting point for transforming CAR-T cell therapies with our in vivo Gene Placement System (iGPS) technology."

Professor Simon Harrison, MBBS, MRCP(UK), FRCPath(UK), FRACP, Ph.D., Director of the Centre of Excellence in Cellular Immunotherapy at the Peter MacCallum Cancer Centre, Melbourne, Australia and inMMyCAR Lead Investigator, added, "Multiple myeloma can be a challenging disease to treat as most patients experience relapse after initial treatment, and many become resistant to currently available therapies. In vivo CAR-T cell therapies would be transformative for these patients, providing rapid access to potentially life-saving treatments. We’re at a pivotal moment, in which we’re taking important steps to make such therapies a reality in Australia through highly novel clinical trials, and I’m proud to play a role in ushering in these innovative therapies."

inMMyCAR is a multi-center Phase 1, open-label, dose-escalation clinical trial designed to assess the safety and preliminary efficacy of a single dose of KLN-1010. The first patient was dosed at Royal Prince Alfred Hospital, Sydney, Australia, by inMMyCAR Investigator, Professor Joy Ho, MB.BS. (Hons), D.Phil (Oxon), FRACP, FRCPA, FFSc (RCPA).

"Dosing the first patient in the inMMyCAR Phase 1 study is a tremendous milestone and marks significant advancement towards bringing KLN-1010 to patients who need it most," said Professor Ho. "I and my team are proud to have achieved this proficiently. As an in vivo CAR-T cell therapeutic candidate, KLN-1010 has the potential to deliver the full promise of CAR-T cell therapies without the complex manufacturing requirements, lengthy production timelines and toxic lymphodepleting chemotherapy that ex vivo CAR-T cell therapies require. We believe it has the potential to revolutionize CAR-T therapy in myeloma."

About inMMyCAR

inMMyCAR is a Phase 1, open-label, dose-escalation clinical trial designed to assess the safety, tolerability, pharmacology and preliminary efficacy of a single dose of KLN-1010 in up to 40 patients. The primary endpoints are incidence and severity of treatment-emergent adverse events (TEAEs), including dose limiting toxicities (DLTs), and to establish the recommended Phase 2 dose of KLN-1010. KLN-1010 has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA). This Phase 1 clinical trial marks the first time KLN‑1010 will be evaluated in humans. Additional information and study site information may be found on clinicaltrials.gov (NCT07075185).

About Relapsed and Refractory Multiple Myeloma

Multiple myeloma is a hematologic malignancy characterized by the proliferation of plasma cells in the bone marrow, leading to bone destruction, anemia, renal dysfunction, and immunosuppression. It is driven by complex genetic and epigenetic alterations that promote malignant cell survival and resistance to apoptosis. Relapsed and refractory multiple myeloma is characterized by clonal evolution, drug resistance, and increased disease heterogeneity, heightening the need for accessible, personalized therapeutic strategies.

About KLN-1010

KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR‑T treatments, KLN‑1010 is administered to patients via direct transfusion and is designed to generate durable CAR‑T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.

On August 19, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported that the China National Medical Products Administration (NMPA) has granted Innovative Medical Device Designation for Tumor Treating Fields (TTFields) for patients with pancreatic cancer based on the positive results from the Phase 3 PANOVA-3 trial (Press release, Zai Laboratory, AUG 19, 2025, View Source [SID1234655388]). The Innovative Medical Device Designation allows Zai Lab to take advantage of an expedited approval procedure for TTFields that offers opportunities for the NMPA to prioritize the allocation of review resources to expedite the regulatory review and approval process.

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"We are excited that TTFields has been granted the Innovative Medical Device Designation, a status that offers expedited registration and priority review by the NMPA. This designation also allows us to submit the application in China before approval in the country of origin," said Rafael Amado, M.D., President, Head of Global Research and Development at Zai Lab. "Pancreatic cancer remains one of the most challenging cancers to treat globally, with approximately 134,000 new cases diagnosed annually in China alone. We are on track to submit for regulatory approval in China in the second half of 2025 and look forward to collaborating closely with the NMPA throughout the review process."

The Phase 3 PANOVA-3 trial evaluated the use of TTFields therapy concomitantly with gemcitabine and nab-paclitaxel as a first-line treatment for unresectable, locally advanced pancreatic adenocarcinoma compared to gemcitabine and nab-paclitaxel alone. The trial met its primary endpoint, demonstrating a statistically significant improvement in median overall survival for patients treated with TTFields. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively).

In August 2019, the NMPA granted Innovative Medical Device Designation for Optune in China for the treatment of newly diagnosed and recurrent glioblastoma (GBM). Zai Lab subsequently submitted the regulatory application in September 2019 and received approval in May 2020. Optune is a registered trademark of Novocure GmbH, and Zai Lab markets Optune under license from Novocure GmbH.

About PANOVA-3

PANOVA-3 is an international prospective, randomized, open-label, controlled Phase 3 clinical trial designed to test the efficacy and safety of TTFields therapy used concomitantly with gemcitabine and nab-paclitaxel, as a first-line treatment for locally advanced pancreatic adenocarcinoma. Patients were randomized to receive either TTFields therapy concomitant with gemcitabine and nab-paclitaxel or gemcitabine and nab-paclitaxel alone.

The primary endpoint is overall survival. Secondary endpoints include progression-free survival, local progression-free survival, objective response rate, one-year survival rate, quality of life, pain-free survival, puncture-free survival, resectability rate, and toxicity.

The PANOVA-3 trial enrolled 571 patients who were randomized 1:1 and followed for a minimum of 18 months.

About Pancreatic Cancer in China

Pancreatic cancer is one of the most common and deadliest cancers globally. In China, there were an estimated 134,374 new cases and 131,203 cancer deaths in 2022, and it is the sixth leading cause of cancer mortality in China1. Pancreatic cancer has a 5-year survival rate of less than 10%, making it the malignancy with the lowest survival rate in China 2.

The patients with locally advanced, unresectable pancreatic cancer are no longer operable, so chemotherapy with or without radiation is the only treatment option, with a median overall survival only nine to twelve months.

1 Xia C, Dong X, Li H et al. Cancer statistics in China and United States, 2022: profiles, trends, and determinants. Chin Med J (Engl) 2022; 135: 584-590.

2 Hu JX, Zhao CF, Chen WB et al. Pancreatic cancer: A review of epidemiology, trend, and risk factors. World J Gastroenterol 2021; 27: 4298-4321.

About Tumor Treating Fields

Tumor Treating Fields (TTFields) is a cancer therapy that uses electric fields that exert physical forces to kill cancer cells via a variety of mechanisms. TTFields do not significantly affect healthy cells because they have different properties (including division rate, morphology, and electrical properties) than cancer cells. These multiple, distinct mechanisms work together to target and kill cancer cells. Due to these multi mechanistic actions, TTFields therapy can be added to cancer treatment modalities in approved indications and demonstrates enhanced effects across solid tumor types when used with chemotherapy, radiotherapy, immune checkpoint inhibitors, or targeted therapies in preclinical models. TTFields therapy provides clinical versatility that has the potential to help address treatment challenges across a range of solid tumors.

To learn more about TTFields therapy and its multifaceted effects on cancer cells, visit tumortreatingfields.com.

On August 19, 2025 VantAI, the leader in generative AI for proximity drug discovery, and Halda Therapeutics, the pioneer of RIPTAC (Regulated Induced Proximity Targeting Chimeras) therapeutics, reported a strategic research collaboration worth over $1 billion in total potential value, inclusive of upfront and milestone payments across multiple targets (Press release, Halda Therapeutics, AUG 19, 2025, View Source [SID1234655387]). This landmark alliance combines two category-defining platforms to accelerate the discovery and development of selective proximity-based therapies across cancer and immunology indications. The agreement includes upfront payments, research support, success-based development and commercial milestones, and tiered royalties on net sales, with a path to expand the collaboration over time.

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Under the partnership, VantAI will leverage its Neo-1 foundation model and NeoLink high-throughput structural proteomics platform to rapidly identify and validate novel, context-specific target–effector pairs. These pairs—across both oncology and immunology—will feed directly into Halda’s proprietary RIPTAC development pipeline, which harnesses a distinctive "hold-and-kill" mechanism to achieve potent, cell-selective effects in disease-relevant tissues. By pairing Halda’s validated modality with VantAI’s AI-driven rational drug design engine, the partnership is poised to deliver the next generation of selective, proximity-based therapeutics tailored to disease-specific signatures.

"Induced proximity is poised to unlock a new and exciting chapter of medicine, including but also beyond protein degradation," said Zachary Carpenter, Co-Founder and CEO of VantAI. "Halda is leading this next wave, already demonstrating its potential in the clinic. Together, we’re advancing a new generation of RIPTACs—leveraging the unique properties of this modality to address previously inaccessible targets, with the potential to meaningfully impact patients underserved by current treatment options."

"VantAI’s proprietary platform enables the systematic rewiring of protein interactions with precision," said Christian Schade, President and CEO of Halda Therapeutics. "By pairing Halda’s novel RIPTAC modality with VantAI’s AI-driven rational drug design engine, the partnership will complement platform discovery efforts and will help deliver the next generation of selective, proximity-based therapeutics tailored to disease-specific signatures." Halda’s lead candidate, HLD-0915, is currently advancing through Phase 1/2 clinical trials in metastatic castration-resistant prostate cancer (mCRPC).

On August 19, 2025 Incyte (Nasdaq:INCY) reported that it will present at the following investor conferences during the month of September (Press release, Incyte, AUG 19, 2025, View Source [SID1234655386]):

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Cantor Global Healthcare Conference on Wednesday, September 3, 2025 at 8:00 am (EDT)
Well Fargo Healthcare Conference on Thursday, September 4, 2025 at 8:45 am (EDT)
Morgan Stanley 23rd Annual Global Healthcare Conference on Tuesday, September 9, 2025 at 10:00 am (EDT)
The presentations will be webcast live and can be accessed at Investor.Incyte.com and will be available for replay for 30 days.

On August 19, 2025 Twist Bioscience Corporation (NASDAQ: TWST), a core mid-cap growth and value equity, in the life sciences segment of the health care sector, reported the Twist Oncology DNA Comprehensive Genomic Profiling (CGP) Panel, a customizable, research-use-only solution designed to empower users to identify genomic alterations for a broad set of tumors, guide targeted therapy development and support clinical and translational oncology research (Press release, Twist Bioscience, AUG 19, 2025, View Source [SID1234655385]).

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"We designed this panel to break the status quo in genomic profiling by offering laboratories a powerful alternative to locked, one-size-fits-all solutions," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "With our customizable content, flexible workflow, and compatibility with multiple sequencing platforms, Twist’s Oncology DNA CGP Panel puts scientific control back in the hands of researchers and clinicians, particularly for those who need a more cost-effective solution compared to existing CGP products or a more efficient option than smaller panels."

The Twist Oncology DNA CGP Panel was designed to serve a wide range of research groups including regional clinical labs, academic medical centers, and biopharma partners. The panel composition incorporates clinical research and includes a comprehensive and updated biomarker list covering 562 genes that incorporate all major tumor types. The panel’s coverage spans genomic alterations and genomic signatures and can detect base substitutions, insertions and deletions (indels), copy number variations (CNVs), gene rearrangements as well as measure tumor mutational burden (TMB) and microsatellite instability (MSI). This is paired with Twist high quality and easy-to-use library preparation and target enrichment workflows. Twist is collaborating with several analytics companies to provide a complete solution from sequencing to analysis.