On August 28, 2024 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and genetic testing, reported that members of its leadership team will present at the Morgan Stanley 22nd Annual Global Healthcare Conference on Wednesday, Sept. 4 at 2:35 pm ET | 11:35 am PT in New York City (Press release, Natera, AUG 28, 2024, View Source [SID1234646178]).

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A live webcast and audio archive of the event may be accessed through the investor relations section of the Natera website at investor.natera.com. A replay of the event will be available shortly following the conference.

On August 28, 2024 Bayer and NextRNA Therapeutics, a biotechnology company focused on developing transformative medicines to address long non-coding RNA (lncRNA)-driven diseases, reported that they have entered into a collaboration and license agreement to develop small molecule therapeutics targeting lncRNAs in oncology (Press release, Bayer, AUG 28, 2024, View Source [SID1234646177]). lncRNAs represent a vast class of therapeutic targets that recruit RNA-binding proteins (RBPs) to drive pathological processes across diseases. Disrupting lncRNA-RBP interactions with small molecules represents an innovative approach to develop a new class of therapeutic agents.

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Bayer and NextRNA will collaborate to jointly advance two oncology programs in high unmet need indications. The first program is a lncRNA-targeting small molecule program currently in early preclinical development at NextRNA. For the second program, NextRNA will prosecute lncRNA targets that its platform has already identified, and Bayer will have the option to select one target for joint development.

"With NextRNA’s exceptional expertise and lncRNA platform, we aim to advance novel small molecule therapeutics against a new class of targets in oncology," said Juergen Eckhardt, M.D., Head of Business Development and Licensing at Bayer’s Pharmaceuticals Division. "This partnership further adds to our mission to build one of the most transformative and diversified oncology pipelines in the industry."

Under the agreement, Bayer gains access to NextRNA’s differentiated approach to inhibit the function of lncRNAs by disrupting the interaction between lncRNAs and RBPs with small molecules. NextRNA’s proprietary platform combines its computational engine NextMap with deep lncRNA biology expertise and a diverse set of biochemical, biophysics, and chemistry capabilities.

"We are excited to partner with Bayer, a recognized leader in life sciences and oncology innovation," said Dominique Verhelle, PhD, MBA, NextRNA Co-Founder and CEO. "This collaboration recognizes lncRNAs as an exciting target class and confirms NextRNA’s position as both a leader in this space and a partner-of-choice for companies seeking to develop transformative small molecule therapeutics across disease areas. We look forward to working closely with the Bayer team to advance first-in-class cancer therapies while continuing to build our pipeline in oncology and neuroscience."

Under the terms of the agreement, NextRNA will receive up to $547 million for both programs, including upfront and near-term milestone payments, research funding, and development and commercial milestone payments, as well as tiered royalties on net sales.

On August 28, 2024 Foundation Medicine, Inc. reported a collaboration to develop Foundation Medicine’s RNA platform as a companion diagnostic for Merus N.V.’s (Nasdaq: MRUS) bispecific antibody zenocutuzumab (Zeno) to treat patients with neuregulin 1 fusion (NRG1+) cancer (Press release, Foundation Medicine, AUG 28, 2024, View Source [SID1234646176]).

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Zeno is a Biclonics that utilizes the Merus Dock & Block mechanism to inhibit the neuregulin/HER3 tumor-signaling pathway in solid tumors with NRG1 fusions (NRG1+ cancer). Through its unique mechanism of binding to HER2 and potently blocking the interaction of HER3 with its ligand NRG1, Zeno has the potential to be particularly effective against NRG1+ cancer. In preclinical studies, Zeno potently inhibits HER2/HER3 heterodimer formation thereby inhibiting oncogenic signaling pathways, leading to inhibition of tumor cell proliferation and blocking tumor cell survival.

In May the U.S. Food and Drug Administration (FDA) accepted under priority review a Biologics License Application for Zeno in patients with NRG1+ non-small cell lung (NSCLC) and NRG1+ pancreatic (PDAC) cancer. The FDA has also granted Breakthrough Therapy Designation (BTD) to Zeno for the treatment of patients with advanced unresectable or metastatic NRG1+ pancreatic cancer following progression with prior systemic therapy or who have no satisfactory alternative treatment options. Additionally, the FDA has granted BTD to Zeno for the treatment of patients with advanced unresectable or metastatic NRG1+ NSCLC, following progression with prior systemic therapy.

NRG1 gene fusions are rare and have been identified in patients with different types of solid tumors, including NSCLC, pancreatic cancer, gallbladder cancer, renal cell carcinoma, bladder cancer, ovarian cancer, breast cancer, neuroendocrine tumor, sarcoma, and colorectal cancer.1 NRG1 fusions are unique cancer drivers that create oncogenic chimeric ligands rather than the more widely described chimeric receptors (NTRK, RET, ROS1, ALK, and FGFR fusions).2

While DNA sequencing with optimized targeting can detect fusions, Foundation Medicine’s RNA platform can provide another layer of sophisticated fusion detection in 318 genes through RNA sequencing. It also has expanded capabilities for research use to offer gene expression reporting of over 1,500 genes to support biomarker discovery. Foundation Medicine is the only company with FDA-approved CDx indications for fusion biomarkers using CGP tests, including approved claims using tissue or liquid biopsy for ALK, ROS1, RET, NTRK1/2/3, and FGFR2.3

"We are excited to partner with Merus on this RNA companion diagnostic opportunity to provide fusion detection through next-generation sequencing," says Troy Schurr, Chief Biopharma Business Officer at Foundation Medicine. "We anticipate this innovative molecular information will help detect more NRG1 fusions and provide healthcare providers with important information to inform their care decisions for patients."

On August 28, 2024 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported the strategic reprioritization of its research and development activities to focus its efforts on the advancement of its portfolio of clinical-stage oncology programs (Press release, Repare Therapeutics, AUG 28, 2024, View Source [SID1234646175]). With multiple upcoming clinical milestones and potential near-term registration-enabling studies, the Company is streamlining its operations to focus on the advancement of its lunresertib, camonsertib, RP-1664 and RP-3467 programs while materially reducing the scale of its preclinical research and discovery activities.

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"We acknowledge today the extraordinary contributions and productivity of our discovery team, who have enabled the development of our deep, innovative clinical portfolio. In our mission to rapidly develop new, practice-changing therapies, we will more fully dedicate our resources to our most promising and advanced precision oncology programs to maximize value for patients and for our shareholders," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "We remain on track to report data from our MYTHIC dose expansion trial evaluating lunresertib in combination with camonsertib in patients with ovarian and endometrial cancers in the fourth quarter of 2024, with the potential to begin a registrational trial in 2025."

As part of this strategic refocus, Repare plans to reduce its overall workforce by approximately 25%, with a majority of the headcount reductions from the Company’s preclinical group. Repare expects total non-recurring cash payments of approximately $1.5 million to $2.0 million in the third quarter of 2024 associated with the workforce reduction, and expects to generate annual savings of approximately $15.0 million that will extend its cash runway into the second half of 2026, while aggressively pursuing the further development of its clinical portfolio.

"I want to thank all of our impacted Repare colleagues who have contributed to the pioneering research and innovation, some for more than seven years, to significantly advance Repare in its mission to deliver novel medicines for patients in need," continued Segal.

Clinical Programs and Upcoming Milestones:

Lunresertib (RP-6306): First-in-class, oral small molecule inhibitor of PKMYT1

Repare expects to report data from the ongoing MYTHIC dose expansion clinical trial of lunresertib and camonsertib at the recommended Phase 2 dose (RP2D) in patients with platinum-resistant ovarian and endometrial cancers harboring CCNE1 amplification or FBXW7 or PPP2R1A mutations in the fourth quarter of 2024, with the potential to begin a registrational trial in 2025.
Repare is evaluating lunresertib in combination with Debio 0123, a highly selective, brain-penetrant, clinical WEE1 inhibitor, in Module 4 of the ongoing MYTHIC trial in patients with advanced solid tumors harboring CCNE1 amplification or FBXW7 or PPP2R1A deleterious alterations. Repare expects to report initial data from Module 4 of the MYTHIC trial in 2025.
Repare also recently reported positive data from the MINOTAUR trial of lunresertib and FOLFIRI showing promising efficacy and duration of therapy in the heavily pretreated population with tumors that harbor CCNE1 amplification and FBXW7 mutation alterations that warrant further development.
Camonsertib (RP-3500): Potential best-in-class oral small molecule inhibitor of ATR

Repare is evaluating camonsertib as a monotherapy in the ongoing non-small cell lung cancer (NSCLC) expansion of the Phase 2 TRESR clinical trial. Camonsertib has demonstrated a promising signal of prolonged progression free survival in patients with ATM-mutated NSCLC in the TRESR trial. Repare expects to report initial data from the TRESR trial in 2025.
RP-1664: First-in-class, highly selective, oral inhibitor of PLK4

Repare is evaluating RP-1664 as a monotherapy in the Phase 1 LIONS clinical trial in adult and adolescent patients with TRIM37-high solid tumors. After evaluating safety in the LIONS trial, the Company expects to rapidly advance RP-1664 into a Phase 1/2 trial in pediatric patients with high risk, recurrent neuroblastoma, where the patients have a high prevalence of TRIM37-altered tumors.
RP-3467: Potential best-in-class Polθ ATPase inhibitor

Repare expects to initiate a Phase 1 dose-finding clinical trial of RP-3467 in the fourth quarter of 2024.

On August 28, 2024 Harbour BioMed ("HBM", or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery, development, and commercialization of novel antibody therapeutics focusing on oncology and immunology, reported its interim results for the six months ended June 30, 2024 (Press release, Harbour BioMed, AUG 28, 2024, View Source [SID1234646174]).

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Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, commented, "In the first half of 2024, Harbour BioMed remained profitable, demonstrating the Company’s remarkable resilience and adaptability in challenging market conditions. Our global operations continue to open new avenues for future growth. Our outstanding performance has validated the feasibility of the Two-Engine strategy, driven by Harbour Therapeutics and Nona Biosciences. We are confident in our ability to enhance value creation and drive sustainable growth by fully unlocking the tremendous potential of our core innovation engine."

The Company reported revenues of US$23.7 million for the first half of 2024, achieving an overall profit of approximately US$1.4 million, with a solid cash balance exceeding US$183 million.

Harbour Therapeutics: Robust Portfolio and Differentiated Pipeline

The Company has a robust and diversified pipeline with potentially differentiated drug candidates in immuno-oncology and inflammatory and immunology diseases. Key products include HBM9161, HBM4003, HBM9378 and HBM1020. The Company aims to deliver at least one IND submission generated from the discovery engine each year.

Batoclimab (HBM9161) is a novel, fully human anti-FcRn (neonatal fragment crystallizable receptor) monoclonal antibody which has the potential to be a breakthrough treatment option for a wide range of autoimmune diseases. In June 2023, the National Medical Products Administration (NMPA) of China accepted the BLA of batoclimab for the treatment of gMG, while in December 2023, the Company voluntarily planned to include additional long-term safety data and re-submitted the BLA for batoclimab to the NMPA in June 2024, which was shortly accepted in July 2024. According to the analysis of the Open-Label extension clinical trial up to November 2023, the data demonstrated consistent efficacy and safety of batoclimab in long-term disease management. Its clinical results were published in JAMA Neurology in March 2024.

Batoclimab received the "Breakthrough Therapy Certificate" from the NMPA in 2021 and achieved a positive outcome in the proof-of-concept study for treating Chinese gMG patients in July 2021. The positive topline results of its Phase III clinical trial were announced in March 2023.

In October 2022, the Company entered into an agreement with NBP Pharma, a wholly owned subsidiary of the CSPC Group, to co-develop batoclimab in Greater China. The Company believes that the collaboration with CSPC Group enables the Company to optimize the market potential and advance the clinical development of batoclimab, so as to further maximize the value of batoclimab in Greater China.

Porustobart (HBM4003) is a next-generation, fully human anti-CTLA-4 antibody targeting cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). It is the first internally developed molecule generated from HCAb Harbour Mice platform. Porustobart is the first fully human heavy chain only anti CTLA-4 antibody entered into clinical development worldwide, and has favourable properties compared with conventional anti-CTLA-4 antibodies in pre-clinical settings. Notably, porustobart exhibits unique, favourable properties including significant Treg cell depletion and optimized pharmacokinetics for improved safety. While increasing the potential to selectively deplete intratumoral Treg cells via enhanced antibody-dependent cellular cytotoxicity (ADCC) strategy, the Company believes porustobart will be able to break the significant immune-suppressive barrier of anti-cancer immunotherapies in solid tumors. Porustobart has great potential to overcome the efficacy and toxicity bottleneck of the current CTLA-4 therapies, positioning it as a key product in cancer immunotherapy.

The Company has implemented a global development plan for porustobart across multiple solid tumor types, with adaptive treatment designs. Positive data on efficacy and safety have been read out from the monotherapy trial targeting advanced solid tumors, as well as the trials of combination treatment with PD-1 inhibitor for melanoma, NEN, and HCC. In January 2024, patient enrolment for combination therapy with a PD-1 inhibitor for advanced colorectal carcinoma was initiated.

HBM9378 is a fully human monoclonal antibody targeting thymic stromal lymphopoietin (TSLP), developed from H2L2 Harbour Mice platform. It inhibits the TSLP-mediated signalling pathway by blocking the interaction between TSLP and its receptor. TSLP plays important roles in DC cell maturation, T helper 2 (Th2) cell polarization, and inflammation, particularly in both eosinophilic and non-eosinophilic inflammation asthma. HBM9378 features fully human sequences, which reduce the risk of immunogenicity and improve bioavailability compared to other TSLP-targeting competitors. Its optimized long half-life and outstanding biophysical properties provide dosing and formulation benefits.

HBM9378 has entered the clinical development stage in collaboration with Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (Kelun-Biotech). In February 2022, HBM9378 received IND approval from the NMPA for the treatment of severe asthma and initiated the Phase I trial in China. In October 2023, HBM9378 completed the Phase I clinical trial, and the initiation of its Phase II clinical trial is now ongoing. The Company is also preparing the IND application for a second indication, chronic obstructive pulmonary disease (COPD), for HBM9378.

HBM1020 is a first-in-class, fully human monoclonal antibody generated from Harbour Mice platform, targeting B7H7/HHLA2. As a newly discovered member of the B7 family, B7H7/HHLA2 expression is found non-overlapping with PD-L1 expression in multiple tumor types, suggesting it may play a key role for tumor cells to escape immune surveillance beyond PD-L1 mechanisms. HBM1020 is the first product targeting B7H7/HHLA2 in clinical stage globally. With its excellent product design and target features, the Company believes that HBM1020 has significant potential to address unmet medical needs in the treatment of solid tumors, particularly in patients with low PD-L1 expression and those resistant to PD-(L)1 therapies. The Company initiated a Phase I clinical trial in the U.S. in May 2023 and has since completed multiple dose levels. The latest progress of HBM1020 will be presented at the ESMO (Free ESMO Whitepaper) Congress 2024.

Engaged in the discovery and development of differentiated antibody therapeutics in the areas of immuno-oncology and immunology, the Company are also exploring and developing multiple programs, including novel and challenging antibody therapeutics across various disease areas.

In oncology field, alongside monoclonal antibodies (mAbs) such as HBM1022 (CCR8) and HBM9014 (a LIFR-targeting mAb), the Company is also generating bispecific antibodies from the HBICE platform, featuring innovative designs and differentiated mechanisms such as HBM7020 (BCMAxCD3), HBM9027 (PD-L1xCD40), and HBM7004 (B7H4xCD3). In addition, leveraging the advantages of the Harbour Mice platform and utilizing the XDC platform, the Company is exploring more therapeutic modalities in oncology, including HBM9033 (a MSLN-targeted ADC) and other ADC/RDC programs in early stages.

In the inflammatory and immunology field, the Company has built a robust preclinical pipeline, encompassing bispecific and multi-specific antibody programs in targeting Type 2 pathways and for other inflammatory and immunology conditions.