On August 14, 2024 Accutar Biotechnology, Inc., a biotechnology company focusing on artificial intelligence (AI)-enabled drug discovery, reported that the US Food and Drug Administration (FDA) has granted the investigation of AC699 a Fast Track designation for the treatment of patients with estrogen receptor (ER)-positive, human epidermal growth factor receptor-2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer with disease progression on or after at least 1 line of endocrine-based therapy (Press release, Accutar Biotechnology, AUG 14, 2024, View Source;Breast-Cancer [SID1234645936]). AC699 is an investigational orally bioavailable, chimeric degrader of estrogen receptor (ER) α currently in a Phase 1 trial.

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ER-positive/HER2-negative subtype is the most common subtype of breast cancer (~70%), and mutations in the estrogen receptor 1 (ESR1) gene are common (20-40%) among ER-positive/HER2-negative patients who received endocrine therapy in the metastatic setting. AC699 has demonstrated objective response rate (ORR) of 50% in patients with an ESR1 mutation, in an ongoing Phase 1 trial, presented at ASCO (Free ASCO Whitepaper) 2024.

"Receiving Fast Track designation for AC699 from the FDA highlights their recognition of the serious and life-threatening nature of this malignancy, the critical unmet medical needs not fully addressed by existing therapies, and the potential of AC699 to fill in the gap," said Jie Fan, Ph.D., Chief Executive Officer of Accutar Biotechnology, Inc. "We look forward to working closely with the FDA to optimize and expedite the development program."

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of novel drugs intended to treat serious conditions and address significant unmet medical needs. Companies that receive Fast Track designation are eligible for more frequent meetings and communications with the FDA during clinical development and potentially accelerated approval and priority review, if relevant criteria are met. For more information on Fast Track Designation, please visit the FDA’s website at View Source

About AC699 and the Phase 1 Study (AC699-001)

AC699 is an investigational orally bioavailable, chimeric degrader of estrogen receptor (ER) α. In preclinical studies, AC699 has demonstrated potent and selective protein degradation of ERα wildtype and mutants with favorable pharmacological properties, as well as promising anti-tumor activities in ER-positive animal tumor models.

The purpose of the Phase 1 multi-center, open-label study is to assess the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of AC699 treatment in patients with ER-positive / HER2-negative locally advanced or metastatic breast cancer (NCT05654532). Additional information on this clinical trial can be found on www.clinicaltrials.gov.

On August 14, 2024 Oncopeptides reported its second quarter 2024 results (Presentation, Oncopeptides, AUG 14, 2024, View Source [SID1234646759]).

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On August 14, 2024 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, reported its financial results for the second quarter ended June 30, 2024, and recent business highlights (Press release, Achilles Therapeutics, AUG 14, 2024, View Source [SID1234645884]).

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"During the second quarter we shared interim Phase I/IIa data from our ongoing CHIRON and THETIS TIL-based cNeT clinical trials and established an important research collaboration with Arcturus Therapeutics to explore the use of clonal neoantigens in second-generation personalized mRNA cancer vaccines", said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. "Our insights into the factors that drive durable engraftment and immune evasion led us to add an additional cohort in the CHIRON and THETIS trials to evaluate cNeT persistence and clinical activity in patients with enhanced host conditioning (EHC). These findings, along with the cancer vaccine research collaboration, continue to illustrate the potential value of our platform, including the unparalleled capability of PELEUS to select tumor targets with the highest potential for immune system recognition in a variety of modalities including TIL, neoantigen vaccines, ADCs, and TCR-T therapies.

Our financial position remains strong with more than $95 million in cash, which we expect to support operations through 2025, including the completion of the ongoing Phase I/IIa trials and the Arcturus collaboration."

Clinical and Business Updates
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Announced research collaboration with Arcturus Therapeutics to explore second-generation mRNA cancer vaccines

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Combines Achilles’ AI-powered, tumor-targeting technology with Arcturus’ self-amplifying mRNA platform
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Combined technologies have the potential to generate potent and durable T cell responses in pre-clinical IND-enabling studies 
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Provided an interim Phase I/IIa update on the use of clonal neoantigen reactive T cells (cNeT) in advanced NSCLC and melanoma
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Dosed first EHC patients in CHIRON and THETIS, with the first three EHC patients showing improved cNeT persistence and engraftment
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Delivered 10 products containing over 100 million cNeT and five containing over one billion cNeT
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Matilde Saggese, MD, has been appointed as Interim Chief Medical Officer. Dr. Saggese has served as Vice President and Medical Director of Achilles Therapeutics since March 2021.

Financial Highlights
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Cash and cash equivalents: Cash and cash equivalents were $95.1 million as of June 30, 2024, as compared to $131.5 million as of December 31, 2023. The Company believes that its cash and cash equivalents are sufficient to fund its planned operations through 2025.
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Research and development (R&D) expenses: R&D expenses were $13.6 million for the second quarter ended June 30, 2024, compared to $13.8 million for the second quarter ended June 30, 2023. The decrease was primarily driven by decreased activity in THETIS and lower personnel costs, partially offset by increased activity in CHIRON.
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General and administrative (G&A) expenses: G&A expenses were $4.2 million for the second quarter ended June 30, 2024, compared to $4.3 million for second quarter ended June 30, 2023. This decrease was primarily driven by lower personnel costs and lower insurance premiums.
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Net loss: Net loss for the second quarter ended June 30, 2024 was $16.4 million or $0.41 per share compared to $16.8 million or $0.42 per share for the second quarter ended June 30, 2023.

2024 Focus

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Clinical Data: Report clinical activity and translational science data from patients in CHIRON and THETIS, evaluating the benefit of EHC, with a meaningful data update expected in the second half of 2024
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Arcturus Collaboration: Ongoing evaluation of best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines (PCVs) targeting clonal neoantigens with the potential to generate IND-enabling data

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Translational Science: Leverage the Company’s unique bioinformatics platform to better understand the drivers associated with clinical responses
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Manufacturing Development: Continue VELOS and PELEUS development to optimize cNeT dose and functionality

On August 14, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Deltacel (KB-GDT-01), the Company’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy (Press release, Kiromic, AUG 14, 2024, View Source [SID1234645921]). The designation was awarded for KB-GDT-01 in combination with low-dose radiation therapy for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) who have progressed on at least two lines of standard of care therapy including platinum-based chemotherapy, immune checkpoint inhibitors and targeted therapy to improve progression-free survival and overall survival. Deltacel is currently being evaluated in the Deltacel-01 Phase 1 study in patients with stage 4 NSCLC who have failed to respond to standard therapies.

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Fast Track designation is designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need. The designation allows for more frequent communication with the FDA, potential priority review, and a rolling submission of Biologics License Application or New Drug Application.

"Receipt of Fast Track designation is a significant milestone for Kiromic and underscores the potential of Deltacel to address the urgent needs of patients with advanced solid tumors," said Pietro Bersani, Chief Executive Officer of Kiromic BioPharma. "We are encouraged by the FDA’s recognition of our innovative approach and are committed to the clinical development of Deltacel. Fast Track designation will enable us to work more closely with the FDA as we complete Deltacel-01 and advance this promising therapy into later stage studies."

The Fast Track designation follows recent positive data from the ongoing Deltacel-01 clinical trial, in which Deltacel has demonstrated a favorable safety profile and preliminary clinical efficacy in NSCLC patients. Kiromic expects to activate a fifth clinical trial site on August 30th.

About Deltacel-01

In Kiromic’s open-label Phase 1 clinical trial, titled "Phase 1 Trial Evaluating the Safety and Tolerability of Gamma Delta T Cell Infusions in Combination With Low Dose Radiotherapy in Subjects With Stage 4 Metastatic Non-Small Cell Lung Cancer" (NCT06069570), patients with stage 4 NSCLC will receive two intravenous infusions of Deltacel with four courses of low-dose, localized radiation over a 10-day period. The primary objective of the Deltacel-01 trial is to evaluate safety, while secondary measurements include objective response, progression-free survival, overall survival, time to progression, time to treatment response and disease control rates.

About Deltacel

Deltacel (KB-GDT-01) is an investigational gamma delta T-cell (GDT) therapy currently in the Deltacel-01 Phase 1 trial for the treatment of stage 4 metastatic NSCLC. An allogeneic product consisting of unmodified, donor-derived gamma delta T cells, Deltacel is the leading candidate in Kiromic’s GDT platform. Deltacel is designed to exploit the natural potency of GDT cells to target solid cancers, with an initial clinical focus on NSCLC, which represents about 80% to 85% of all lung cancer cases. Data from two preclinical studies demonstrated Deltacel’s favorable safety and efficacy profile when it was combined with low-dose radiation.

On August 14, 2024 Novocure (NASDAQ: NVCR) reported its participation in the upcoming International Association for the Study of Lung Cancer (IASLC) 2024 World Conference on Lung Cancer (WCLC) from September 7 – 10, 2024 in San Diego, California (Press release, NovoCure, AUG 14, 2024, View Source [SID1234645937]). Novocure will take part in presentations and symposia throughout the event and will exhibit several posters exploring the use of Tumor Treating Fields (TTFields) therapy in the treatment of lung cancer, including a new post-hoc analysis of data from the LUNAR trial in metastatic non-small cell lung cancer (NSCLC).

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"We are eager to share these exciting new analyses with the lung cancer community," said Nicolas Leupin, MD, Novocure’s Chief Medical Officer. "TTFields therapy offers significant potential for the treatment of this prevalent disease. With multiple regulatory reviews underway, there is no better time to examine the promise of TTFields use in the treatment of NSCLC."

The new post-hoc analysis evaluated survival data from the phase 3 LUNAR clinical trial, as well as a data from a simulation study, to assess the effect of body mass index (BMI) on overall survival (OS) and the feasibility of delivering TTFields at a therapeutic intensity to patients of varying BMIs. Investigators did not identify a difference in OS benefit between patients with a BMI <25 kg/m2 compared a BMI >25 kg/m2. Additionally, data from the simulation-based study suggests TTFields can be delivered to the lungs in therapeutic dose and a corresponding clinical benefit observed in patients across a range of BMIs.

The LUNAR trial was designed to evaluate the use of TTFields therapy together with standard systemic therapies for the treatment of metastatic non-small cell lung cancer, following progression on or after platinum-based therapy. The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful extension in OS for patients treated with TTFields and standard systemic therapies, as well as a pronounced extension in OS for patients randomized to receive physician’s choice immune checkpoint inhibitor together with TTFields. Novocure has submitted these data to regulatory agencies for approval and anticipates treating patients in late 2024.

Novocure’s presence at the 2024 WCLC will include:

Sunset Seminar: Women in Thoracic Oncology, hosted by Women Leaders in Oncology (WLO), sponsored by Novocure, on September 8, 2024, at 6:00 p.m. UTC-7
Impact of BMI on TTFields in Patients with mNSCLC: Post-hoc Analysis from the Phase 3 LUNAR Study and Simulation Model Data. Poster P1.098B.04 displayed in the Exhibit Hall on September 8, 2024, at 12:00 p.m. UTC-7
Treatment of Non-Small Cell Lung Carcinoma (NSCLC) Cells With Tumor Treating Fields (TTFields) and DNA-Dependent Protein Kinase (PK) Inhibitors. E-Poster EP.03F.04
Tumor Treating Fields (TTFields) Induce Pro-Inflammatory Phenotype Skewing of Macrophages. E-Poster EP.03G.03

ABOUT TUMOR TREATING FIELDS THERAPY

Tumor Treating Fields (TTFields) are 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 multimechanistic 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 inhibition, 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 Tumor Treating Fields therapy and its multifaceted effect on cancer cells, visit tumortreatingfields.com.

ABOUT LUNAR

LUNAR tested the safety and effectiveness of TTFields therapy when used together with either an immune checkpoint inhibitor (ICI) or docetaxel for the treatment of patients diagnosed with metastatic NSCLC following progression on or after the use of platinum-based therapy. Patients randomized to receive TTFields therapy together with standard therapies (n=137) demonstrated median overall survival (OS) of 13.2 months compared to 9.9 months in patients treated with standard therapies alone (n=139). Patients randomized to receive TTFields therapy and physician’s choice ICI (n=66) demonstrated a median OS of 18.5 months versus a median OS of 10.8 months in patients treated with an ICI alone (n=68; HR=0.63; P=0.03). Patients randomized to receive TTFields therapy and docetaxel (n=71) had a positive survival trend with a median OS of 11.1 months vs 8.7 months in patients treated with docetaxel alone (n=71). TTFields therapy was well-tolerated with no added systemic toxicities and few grade 3 (no grade 4 or 5) device-related adverse events.