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 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality, reported that it has engaged Philippe Pultar, MD as his senior medical advisor to support Aprea with developing and advancing APR-1051, Aprea’s potential best in class WEE1 inhibitor (Press release, Aprea, OCT 9, 2024, View Source [SID1234647103]).

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!

Dr. Pultar is a seasoned pharmaceutical executive with extensive experience in oncology, including the development of a WEE1 inhibitor (azenosertib) from early to late-stage clinical development. Dr. Pultar has extensive experience in clinical development within both large and early-stage pharmaceutical companies. Engaging Dr. Pultar aligns with Aprea’s commitment to provide its WEE1 inhibitor program with all the necessary resources, including the best available professional talent and expertise, to succeed. Dr. Pultar was most recently employed at Zentalis Pharmaceuticals where he played a key role in the strategy and execution of the global clinical development of azenosertib, a WEE1 inhibitor.

Patient enrollment is currently ongoing in the Phase 1 ACESOT-1051 study, designed to assess the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of single-agent APR-1051 in advanced solid tumors harboring certain cancer-associated gene alterations. Aprea intends to provide an update on the progress of this clinical study by year end 2024. The WEE1 program is part of Aprea’s portfolio of DDR-targeted therapeutics aimed to deliver precision medicine solutions that ensure the right patients receive the most effective Aprea treatment, with the goals of improving outcomes and reducing treatment resistance.

"WEE1 inhibition is a promising therapeutic approach in oncology and my prior experience has given me a thorough understanding of biomarker driven clinical studies, the development and regulatory landscape as well as the likely attributes for a successful therapeutic," said Dr. Pultar. "Aprea has a great opportunity to be a leader in this space. I am quite impressed by the progress the team has made advancing APR-1051, which has a differentiated profile, and is supported by compelling pre-clinical data. I am very excited to be associated with this program and I believe APR-1051 has the potential to be best in class. I look forward to working with the talented scientists at Aprea with the goals of advancing its WEE1 program and bringing new treatments to patients battling difficulty-to-treat cancers."

"Dr. Pultar is a highly capable scientific leader with a track record of successfully leading programs through late-stage development and regulatory approval," said Oren Gilad, Ph.D., President and CEO of Aprea. "Given the recent initiation of our Phase 1 ACESOT-1051 clinical trial evaluating our promising WEE1 inhibitor, APR-1051, we are excited to bring on a high caliber advisor such as Dr. Pultar. Dr. Pultar’s prior experience in managing multiple clinical trials for a WEE1 inhibitor is particularly relevant and we hope to leverage his expertise as we advance our own program and maximize the therapeutic potential of APR-1051. His addition to the team is part of our broader strategy to bring in exceptional talent with the experience and skills to successfully advance our programs."

Dr. Nadeem Mirza will be stepping down as Chief Medical Officer effective as of October 9, 2024, to pursue other professional endeavors but will remain with the Company through a date not later than December 13, 2024, to ensure a smooth leadership transition.

About Philippe Pultar, MD

Dr. Pultar brings over 17 years of extensive experience in early and late-stage clinical development. From 2020 to 2023 he was Vice President, Clinical Development at Zentalis Pharmaceuticals where he was responsible for the strategy and execution of the global clinical development of azenosertib, working closely with co-development partners GSK, Pfizer, and Zentera for China. He initiated seven Phase 1 and Phase 2 trials with azenosertib as single agent and in combination with chemotherapy and targeted agents across multiple indications. Prior to Zentalis, he spent seven years at Novartis Oncology (2013 to 2020), including as Senior Clinical Development Medical Director. He was appointed Medical Lead in rare diseases and was assigned to New Drug Application (NDA) activities for Isturisa (osilodrostat). He led the writing and review of key EU and US NDA documents for Isturisa, resulting in its approval with broad indication in Cushing’s syndrome with limited post-approval commitments. He was also Medical Lead for Phase 2, 3 and 4 trials on Isturisa, Signifor (pasireotide) and Odomzo (sonidegib). Prior to Novartis he was employed in senior clinical development roles at Agennix AG (2011 to 2013) and ImClone Systems (2007 to 2011). Earlier in his career, he held positions at GPC Biotech, ALTANA Pharma, REDEON and MEDICERCLE.

Dr. Pultar earned his medical thesis from Université de Médecine de Poitiers, France and practiced as a physician for several years. His foundational education includes Medical Studies and Residency at Université de Médecine de Poitiers, providing him with a solid academic background to complement his extensive practical experience in clinical development.

On October 9, 2024 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported four accepted abstracts at the 2024 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics being held October 23-25, 2024, in Barcelona, Spain (Press release, Arcus Biosciences, OCT 9, 2024, View Source [SID1234647121]). The data being presented include a growing body of evidence supporting the potential of casdatifan as a best-in-class HIF-2a inhibitor for the treatment of ccRCC.

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!

The oral presentation will highlight data from the approximately 30 patients in the 100mg daily monotherapy expansion cohort of ARC-20, a Phase 1/1b study evaluating casdatifan in late-line ccRCC. It will include data on safety and efficacy, including objective response rate and rate of primary progression, as well as other data to assess the depth and duration of responses. The presentation will also highlight data from the 50mg monotherapy expansion cohort of approximately 30 patients in the same setting.

"We are thrilled to be presenting the first clinical efficacy data from the ARC-20 study for our HIF-2a inhibitor, casdatifan, in an oral plenary session, as well as two additional posters that further highlight the differentiation of casdatifan in ccRCC and the therapeutic opportunities in other tumor types," said Terry Rosen, Ph.D., chief executive officer of Arcus. "These data support a potential best-in-class profile, and we are rapidly advancing a differentiated development program for casdatifan, including the planned initiation of our first Phase 3 study in the first half of 2025."

Arcus is pursuing a broad development program in both the first-line and post-anti-PD-1 settings with differentiated combinations to maximize the opportunity for casdatifan in ccRCC. In addition to the monotherapy cohorts of ARC-20, the study is also enrolling a cohort to evaluate casdatifan in combination with cabozantinib, a VEGFR tyrosine kinase inhibitor, which is intended to support the initiation of Arcus’s first Phase 3 study, PEAK-1, evaluating casdatifan in combination with cabozantinib versus cabozantinib monotherapy in patients with metastatic ccRCC who have previously received anti-PD-1 therapy. The primary endpoint will be progression-free survival with a key secondary endpoint of overall survival. Arcus also recently announced a clinical collaboration as part of its first-line strategy in advanced first-line ccRCC to evaluate casdatifan in combination with volrustomig, an investigational PD-1/CTLA-4 bispecific antibody.

Investors may dial in to the conference call at +1 (404) 975-4839 (local) or +1 (833) 470-1428 (toll-free), using Conference ID: 595409 on Thursday, October 24, 2024, at 5:00 AM PT / 8:00 AM ET. To access the live webcast and accompanying slide presentation, please visit the "Investors & Media" section of the Arcus Biosciences website at www.arcusbio.com. A replay will be available following the live event.

Four Accepted Abstracts Will Be Presented

Study

Title

Abstract Number

Session Type & Title

Session Date & Time

Casdatifan (HIF-2a Inhibitor)

ARC-20

Casdatifan in Patients (pts) with Previously Treated Clear Cell Renal Cell Cancer (ccRCC) and Other Solid Tumors; Preliminary Results From ARC-20: A Phase 1, Open-Label Dose Escalation and Expansion Study

4

Proffered Papers: Advancing patient care through novel clinical trials – Oral Plenary Session 3

10/24/2024, 10:54 AM – 11:06 AM CEST

AB521 (Casdatifan) Potently and Selectively Inhibits Hypoxia-Inducible Factor 2 Alpha (HIF-2α) Dependent Pro-Tumorigenic Activity

91

Molecular Targeted Agents

10/23/2024, 12:00 PM – 7:00 PM CEST

ARC-20

Clinical Pharmacokinetic/Pharmacodynamic (PK/PD) Relationship for Casdatifan (AB521), a Small Molecule Inhibitor of HIF-2α, Confirms Best-in-class Potential in Treatment of Renal Cell Carcinoma

51

Molecular Targeted Agents

10/23/2024, 12:00 PM – 7:00 PM CEST

AB801 (AXL Inhibitor)

ARC-26

AB801, a Potent and Highly Selective Clinical Stage AXL Inhibitor, Sensitizes Tumors to Standard of Care Therapies

119

New Drugs

10/23/2024, 12:00 PM – 7:00 PM CEST

On October 9, 2024 Akiram Therapeutics reported that it has developed 177Lu-AKIR001, a new type of targeted radioimmunotherapy (Press release, Akiram Therapeutics, OCT 9, 2024, View Source [SID1234647279]). The therapy holds the potential to become a first-in-class treatment in multiple cancer types, including anaplastic and iodine-refractory thyroid cancer, head and neck squamous cell carcinoma, and non-small cell lung cancer. The drug is composed of a target recognition molecule to which therapeutic radioactivity is coupled for effect on tumor cells.

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!

The presentations at the EANM Congress will focus on the development and optimization of the drug in preparation for upcoming clinical trials. Specifically, the cGMP production process, which ensures the drug can be manufactured according to high-quality standards for clinical studies, will be highlighted.

The presentation on the cGMP development of 177Lu-AKIR001 has been selected as one of the Top-Rated Oral Presentations at the congress:

Title: cGMP development of the 177Lu-AKIR001 for clinical translation in first-in-human studies of CD44v6 expressing cancer patients
Presentation No.: OP-082
Date & Time: October 20, 9:45 AM, Hall X1-X4
Speaker: Klas Bratteby

Additionally, several other key research findings on 177Lu-AKIR001 will be showcased as posters, including:

177Lu-AKIR001 inhibits growth of pancreatic tumour xenografts
Poster No.: EP-0100
Presented by: Amanda Gustafsson
Fractionation of 177Lu-DOTA-AKIR001 results in increased curative rates and favorable hematopoietic toxicities compared to single high dose
Poster No.:EP-0992
Presented by: Anja Mortensen
Biodistribution and in vivo efficacy of 161Tb-labeled AKIR001, a novel anti-CD44v6 antibody
Poster No.: EP-0991
Presented by: Anja Mortensen
"We are thrilled to see the results of our academic collaborations presented at the EANM Congress. 177Lu-AKIR001 has shown great preclinical potential to transform the treatment of CD44v6-expressing tumors. We are looking forward to starting phase 1 studies later this fall, with the goal of offering new, targeted treatment options for patients with hard-to-treat cancers," says Marika Nestor, CEO of Akiram Therapeutics. "The selection of the cGMP production presentation as one of the top-rated talks is a clear recognition of the promising research, and we are excited to share the results with the international scientific community."

EANM – The European Association of Nuclear Medicine
The annual EANM Congress is one of the premier global platforms where leading experts and industry representatives from around the world gather to discuss advancements and future trends in nuclear medicine. Read more: View Source

About Akiram’s Drug Candidate
Developed through antibody phage display and affinity maturation targeting the CD44v6 cancer marker, 177Lu-AKIR001 combines the radiation component lutetium-177 with a targeted molecule. Preclinical studies have demonstrated its potential as a promising, first-in-class radiopharmaceutical therapy for cancers with high CD44v6 expression.

On October 9, 2024 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through development of potentially best-in-class IL-2 therapeutics, reported that pharmacokinetic data for evaluation of AU-007 in the Phase 2 portion of its Phase 1/2 clinical trial will be presented at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics (Press release, Aulos Bioscience, OCT 9, 2024, View Source [SID1234647104]). AU-007 is a human IgG1 monoclonal antibody designed using artificial intelligence to harness the power of interleukin-2 (IL-2) to eradicate solid tumors in patients with unresectable locally advanced or metastatic cancers. The EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) symposium is being held October 23-25, 2024, in Barcelona, Spain.

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!

"With the Phase 1 trial of AU-007 completing the dose escalation and expansion phase, we are pleased to share the pharmacokinetic data used to determine dose selection of AU-007 for the Phase 2 portion of the trial," said Aron Knickerbocker, Aulos Bioscience’s president and chief executive officer. "We’re excited by the continued progress and distinct findings with our investigational therapy in the clinic, and we are grateful to the clinicians and patients who participate in this important study."

Details of the poster presentation are as follows:

Poster Number and Title: PB452: Determination of the phase 2 dose of AU-007, an AI-designed human monoclonal antibody that redirects IL-2 to T effector cells
Abstract: 464
Session: New therapies in immuno oncology
Session Date and Time: Friday, October 25, 2024, 9:00 a.m.-3:00 p.m. CEST

The poster will be presented in the Exhibition Hall at the Centre de Convencions Internacional de Barcelona (CCIB).

About AU-007
AU-007 is a computationally designed, human IgG1 monoclonal antibody that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 leverages IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by effector T cells, from binding to trimeric receptors on regulatory T cells while still allowing IL-2 to bind and expand effector T cells and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to CD25-containing receptors on eosinophils, as well as vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

To learn more about the AU-007 Phase 1/2 clinical trial program, including study locations in the United States and Australia, please visit ClinicalTrials.gov (identifier: NCT05267626), www.solidtumorstudy.com (U.S.) and www.solidtumourstudy.com (Australia).