On October 9, 2024 Arda Therapeutics, a pioneer in targeted cell depletion therapies for chronic diseases, reported the successful completion of a $43 million Series A financing round (Press release, Arda Therapeutics, OCT 9, 2024, View Source [SID1234647120]). The round was led by existing investor Andreessen Horowitz (a16z Bio + Health), with participation from Two Sigma Ventures, RV Invest, Eli Lilly and Company, GV, Biovision Ventures, Valhalla Ventures, Indicator Ventures, Alumni Ventures, LifeLink Ventures, Mana Ventures, Gaingels and ExitFund.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

For decades, the dominant approach in drug development has been to modulate individual proteins and signaling pathways to ameliorate disease. While this strategy has yielded some success, it often leads to limited efficacy and incremental gains, particularly for complex chronic diseases. Arda Therapeutics is pursuing a novel alternative by depleting the cells that drive disease rather than modulating the activity of the proteins they produce.

"Arda is at the forefront of a paradigm shift in treating chronic diseases," said Adam Freund, Ph.D., founder and CEO of Arda Therapeutics. "By focusing on the cells at the core of disease, we can develop therapies that are not only more effective, but also have the potential to fundamentally change patient outcomes. With drug approval rates declining and efficacy improvements stalling, Arda’s strategy to target cells—not pathways—offers a transformative shift in how chronic diseases are treated. With the support of our Series A investors, Arda is well positioned to progress our lead programs toward the clinic and expand our platform to tackle even more disease areas."

Arda’s sophisticated single-cell-based discovery engine identifies the specific pathogenic cells responsible for disease and their surface markers with unprecedented precision. This approach enables the development of targeted biologics that selectively eliminate harmful cells while preserving healthy tissue, leading to significantly greater efficacy and fewer side effects compared to conventional treatments. Arda’s platform has broad applicability across a wide range of diseases, including fibrotic conditions such as pulmonary fibrosis, as well as autoimmune and metabolic disorders.

"The field of oncology therapeutics has been focused on tumor cell clearance as a mechanism of action for decades. Many of our most potent cancer medicines – chemotherapy, CAR-T cells, antibody drug conjugates, T cell engagers, radiopharmaceuticals – fundamentally depend on our ability to identify pathogenic, or bad actor, cell populations and then target them precisely for killing," said Vineeta Agarwala, M.D., Ph.D., general partner at Andreessen Horowitz. "Arda is taking the ‘oncology toolbox’ outside of oncology. Applying the lessons learned from oncology, the Arda team is leveraging cutting-edge, single-cell biology and a deep understanding of the biology of diseases outside cancer to boldly extend the cell clearance paradigm to the treatment of chronic diseases."

Scott Turner, Ph.D., appointed to Chief Scientific Officer

In addition to the financing, Arda announced the appointment of Scott Turner, Ph.D., as chief scientific officer. Scott joins Arda after serving as chief scientific officer of Pliant Therapeutics, where he led the creation of the company’s fibrosis discovery platform and biomarker research, culminating in a successful Phase 2a study for Bexotegrast, an anti-fibrotic therapy. At Pliant, Scott promoted the use of single-cell data to dissect drug mechanisms of action and played a critical role in developing innovative fibrosis models and biomarker strategies that advanced several clinical programs. His leadership helped secure over $500 million in financing, including an IPO. Scott received a B.A. from UC Santa Cruz and a Ph.D. from UC Berkeley.

"I’m thrilled to be joining Arda at such an exciting time," said Dr. Turner. "The team has built an impressive platform for targeting pathogenic cells with precision, which holds tremendous potential for addressing the unmet needs in chronic disease. I look forward to working closely with the team to advance our lead programs toward the clinic and develop innovative therapies that can make a meaningful impact on patients’ lives."

"We are very excited to have Scott on the team," said Rèmi Laberge, Ph.D., co-founder and chief technology officer of Arda. "His deep expertise in fibrosis drug discovery will be instrumental as we drive our programs forward. I look forward to collaborating with him to push the capabilities of our cell depletion platform to the next level."

On October 9, 2024 Alpha Fusion, Inc. (CEO: Sunao Fujioka, headquartered in Kita-ku, Osaka), reported that it has raised a total of ¥1.02 billion through a Series B funding round (Press release, Alpha Fusion, OCT 9, 2024, View Source [SID1234647119]). The round was led by SBI Investment Co., Ltd. and OSAKA University Venture Capital, with participation from several new investors. This funding will enable Alpha Fusion to accelerate its research and development efforts, enhance its supply chain, and deliver Japan’s cutting-edge cancer therapies to the global market as quickly as possible.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Alpha Fusion Leading the World in Astatine-based Drug Discovery

Alpha Fusion is a pioneering startup dedicated to bringing "Targeted Alpha Therapy (TAT) using Astatine (At-211)" to cancer patients worldwide. As a leader in the clinical development of Astatine-based therapeutics, Alpha Fusion is advancing its pipeline and building a highly efficient supply chain using cyclotrons in collaboration with partners across the globe. TAT is highly anticipated to become a foundational drug discovery platform in oncology, offering the potential to create numerous novel treatments.

In the past year, several major pharmaceutical companies in Europe and the U.S. have acquired startups developing Actinium (Ac-225) -based radiopharmaceuticals, signaling a surge of interest in this field. Astatine (At-211) is garnering attention for its short half-life, which anticipates clinical safety, its halogen properties that allow it to be directly labeled into low-mid sized-molecule ligands, its ease of sourcing raw material, and the simplicity of its production using cyclotrons.

Business Progress and the Impact of Series B Funding

Since our Series A funding round in May 2023, Alpha Fusion has made significant strides, as detailed below:

1.1st Pipeline (Thyroid Cancer, Phase 1 ongoing): Progress in high-dose administration, approaching the determination of recommended dosages.

2. 2nd Pipeline (Prostate Cancer, Phase 1 ongoing): Successful completion of the world’s first administration of [211At]PSMA-5 to a patient in June.

3. Additional Pipelines: Multiple pipeline developments underway through both in-house R&D and collaborations.

July 2023: Initiated joint research with QST Chiba for tumor-specific targeting.
August 2023: Began joint research with QST Takasaki/Niigata University on novel Astatine-labeling platform technologies.
4. Supply Chain: Progress in manufacturing preparations for clinical trial drugs both domestically and internationally, shaping the future of our supply chain.

First half of 2024: Completion of Osaka University’s TAT Cyclotron building to enable efficient Astatine production.
First half of 2024: Set up GMP facilities for clinical trial drug production in Japan and advanced collaboration with overseas CDMOs.
5. Intellectual Property: Received patent approvals covering manufacturing methods, materials, and applications related to our pipeline.

6. Research Centers: Expanded domestic research operations, including a new center in Tsukuba (SakuLab-Tsukuba), in addition to Osaka University’s research hub.

7. Team Expansion: Strengthened the advisory board and enhanced functional capabilities critical to pharmaceutical development (e.g., supply chain, CMC, quality assurance, clinical development, regulatory affairs).

With this funding, we will accelerate our R&D efforts and prepare for company-sponsored clinical trials, enhance our supply chain for GMP-grade clinical trial drug production domestically and internationally, and further expand our team. We are committed to demonstrating the unique advantages of Astatine-based drug discovery with tangible business results and clinical evidence.

Investors in Series B Round (in no particular order):

New Investors:

SBI Investment Co., Ltd.
Mitsui Mining & Smelting Co., Ltd.
Kobe University Capital
Joyo Capital Partners
Existing Investors (Follow-on Investment):

OSAKA University Venture Capital
D3 LLC
JGC Corporation

On October 9, 2024 HotSpot Therapeutics, Inc., a biotechnology company pioneering the discovery and development of oral, small molecule allosteric therapies targeting regulatory sites on proteins referred to as "natural hotspots," reported that it will present preclinical data from the Company’s mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) CARD11-BCL10-MALT1 (CBM) signalosome glue program highlighting its potential NF-kB-driven solid tumors in a poster presentation at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium, taking place October 23-25, 2024, in Barcelona, Spain (Press release, HotSpot Therapeutics, OCT 9, 2024, View Source [SID1234647118]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Presentation details are as follows:

Title: Targeting the CBM signalosome with a MALT1 scaffolding inhibitor for treatment of NFkB driven solid tumors
Poster Session: Molecular Targeted Agents
Session Date and Time: Wed., Oct. 23, 2024, 12:00-19:00 CEST
Abstract Number: 105

On October 9, 2024 BridGene Biosciences, a leader in the discovery of small molecule drugs for traditionally "hard-to-drug" targets, reported it will present three groundbreaking posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024 Annual Meeting in Barcelona, on October 25, 2024 (Press release, Bridgene Biosciences, OCT 9, 2024, View Source [SID1234647116]). The posters will showcase the company’s latest advancements in the development of covalent inhibitors for key oncogenic targets, including cMyc, ADAR1, and PAX8, each representing promising new treatment avenues for various cancers.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"These three presentations represent significant strides in our mission to develop novel therapeutics for cancer patients," said Ping Cao, Ph.D., Co-Founder and CEO of BridGene Biosciences. "By focusing on these high-value, historically hard-to-drug targets, we aim to create first-in-class therapies that address critical gaps in cancer treatment. Our platform continues to unlock new possibilities in drug discovery, and we are excited to share these advancements with the scientific community at AACR (Free AACR Whitepaper)."

BridGene’s work on cMyc, titled "Discovery of a Covalent Inhibitor Targeting the Undruggable Oncogene cMyc," focuses on overcoming the challenges posed by its intrinsically disordered structure, which has historically rendered the protein hard-to-drug. Through its proprietary Isobaric Mass Tagged Affinity Characterization (IMTAC) platform, BridGene identified two hit compounds, which effectively inhibit cMyc signaling. These inhibitors showed potent activity in reducing transcriptional function and offering a potential therapeutic strategy for cancers driven by cMyc amplification, which accounts for nearly one-third of all tumors.

In addition to its work on cMyc, BridGene will present research titled "Discovery of a Novel Covalent ADAR1 Inhibitor," focusing on a novel covalent inhibitor targeting ADAR1, an enzyme that plays a critical role in RNA editing and immune suppression in cancer. By inhibiting ADAR1, BridGene’s compounds, could be expected to enhance the efficacy of immune checkpoint blockade therapies. This research highlights the potential for ADAR1 inhibitors to reshape the tumor microenvironment, paving the way for more effective immunotherapies.

BridGene’s third poster presentation, "Discovery of a Novel Covalent PAX8 Inhibitor," will unveil a covalent inhibitor of PAX8, a transcription factor linked to poor prognosis in renal cell carcinoma and ovarian cancer. Utilizing its IMTAC platform, BridGene discovered a compound that disrupts the DNA binding activity of PAX8. This discovery opens the door to new therapeutic strategies for cancers with elevated PAX8 expression, which have traditionally been difficult to target.

On October 9, 2024 Nanjing Leads Biolabs Co., Ltd. ("Leads Biolabs") reported that Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation to LBL-024, an anti-PD-L1/4-1BB bispecific antibody independently developed by Leads Biolabs with global intellectual property rights, for the treatment of the patients with advanced extrapulmonary neuroendocrine carcinoma (EP-NEC) who have progressed after receiving two or more lines of chemotherapy (Press release, Nanjing Leads Biolabs, OCT 9, 2024, View Source [SID1234647115]). The Breakthrough Therapy Designation awarded to LBL-024 stems from its remarkable clinical efficacy and safety profile in patients with advanced EP-NEC who failed in second-line and above chemotherapy. The current clinical data demonstrate that LBL-024 monotherapy has more than doubled both the Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatments for this disease.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Breakthrough Therapy refers to innovative or improved drugs that treat life-threatening conditions or significantly impact quality of life, where no effective treatment exist, or there is sufficient evidence that the therapy offers clear advantages over existing treatment options. In July 2012, the U.S. Food and Drug Administration (FDA) pioneered the "Breakthrough Therapy" review process, complementing fast track, accelerated approval, and priority review mechanisms. By accelerating the approval process and fostering closer collaboration, Breakthrough Therapy Designation facilitates faster development and market access for drugs with significant clinical potential. Historically, FDA data suggests that Breakthrough Therapy Designation can reduce time-to-market by approximately 2.5 years on average.

In July 2020, CDE officially introduced the Breakthrough Therapy Designation concept in China. Products granted Breakthrough Therapy Designation status will receive closer guidance and various forms of support to ensure that patients are provided with new treatment options as soon as possible.

Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, said "According to historical data from the China Drug Review Annual Report, only a few products are eligible for Breakthrough Therapy Designation, and even fewer have been approved. The designation for LBL-024 is another proof of the robust R&D capabilities of Leads Biolabs, and it is also a major victory for Leads Biolabs’ insistence on focusing on the first-in-class product R&D strategy for the global market. Leads Biolabs will vigorously pursue the global clinical development of LBL-024, with the anticipation of offering more effective treatment options for patients with EP-NEC at the earliest opportunity."

Dr. Xiaoqiang Kang, founder, chairman and CEO of Leads Biolabs, said "The Breakthrough Therapy granted by the CDE prioritizes candidates that demonstrate exceptional clinical data, differentiated targets, and therapies with a high degree of efficacy certainty. This Designation represents the highest threshold and most prestigious qualification within China’s pharmaceutical regulatory system, functioning as a crucial benchmark for uncovering valuable new drug projects. Leads Biolabs has consistently embraced differentiated innovation, and we eagerly anticipate the timely market introduction of LBL-024 as a novel immunotherapy, following the groundbreaking successes of anti-PD-1/PD-L1, CTLA-4, and LAG-3."

About LBL-024

LBL-024 is a tetravalent bispecific antibody that simultaneously targets PD-L1 and 4-1BB, serving dual functions: blocking the immunosuppressive PD-1/PD-L1 pathway, and selectively co-stimulating 4-1BB in the tumor microenvironment to enhance immune responses. The dual functions of LBL-024—lifting PD-1/PD-L1 immune inhibition and intensifying 4-1BB modulated T cell activation—synergistically enhance the anti-tumor immune response.

About EP-NEC

Neuroendocrine carcinoma (NEC) is a class of poorly differentiated, high-grade neuroendocrine neoplasms (NENs), which originate in the diffuse neuroendocrine cell system and can occur in many different sites. NEC can be categorized into pulmonary NEC and EP-NEC. EP-NEC exhibits similar highly aggressive and metastatic characteristics to small cell lung cancer (SCLC). Most patients are diagnosed at a later stage or already have distant metastases, resulting in rapid disease progression and a poor prognosis.

Currently, the primary first-line treatment for advanced EP-NEC involves platinum-based chemotherapy, achieving an overall response rate (ORR) of approximately 30% to 50%, with a median overall survival (mOS) of only approximately one year. Treatment options for advanced EP-NEC are limited, and there are no standard treatment strategies for patients who progress beyond second-line chemotherapy. These underscore the urgency to develop novel therapeutic approaches.