On September 30, 2024 Valerio Therapeutics reported its half-year 2024 results (Press release, Valerio Therapeutics, SEP 30, 2024, https://valeriotx.com/wp-content/uploads/2024/09/EN_-HY-financial-report-consolidated-Update-Crowe-HAF-v09262024-Formatted-signed.pdf [SID1234647056]).

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On September 30, 2024, Gritstone bio, Inc. (the "Company") reported interim data from the Phase 2 portion of its randomized, open-label Phase 2/3 study evaluating its personalized cancer vaccine, GRANITE (GRTC901/GRT-R902), in combination with immune checkpoint blockade for patients with front-line metastatic microsatellite stable colorectal cancer ("MSS-CRC") (Press release, Gritstone Bio, SEP 30, 2024, View Source [SID1234646976]).

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Key Findings from Interim Phase 2 Data in MSS-CRC

Data cut as of August 19, 2024

104 patients were randomized 1:1 in the study: 69 patients (39 GRANITE arm, 30 control arm) are included in the treated analysis below. Demographics and clinical characteristics were balanced between arms (stage, sidedness, presence of liver metastases), with the vast majority (80%) of patients having liver metastases in the treated analysis. Thirty-five patients did not advance to study treatment after oxaliplatin, most commonly due to withdrawing consent (n=15), disease progression (n=8), and other reasons (n=12) (12 in GRANITE arm; 23 in control arm).

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Interim data demonstrated an emerging progression-free survival ("PFS") benefit to all GRANITE recipients (study not statistically powered for PFS)

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21% relative risk reduction of progression or death with GRANITE vs. standard of care ("SOC") control in all treated population (HR=0.79 [95% CI, 0.42-1.s])

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33% (13/39) GRANITE and 23% (7/30) of control patients remain on study and free of progression

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Last circulating tumor DNA ("ctDNA") assessment is below the assay limit of quantitation in 12/13 GRANITE and 4/7 control patients

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Clinical benefit was most notable in patients with low disease burden (defined as patients with ctDNA equal to or below the trial population median value at study entry)

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38% relative risk reduction of progression or death with GRANITE vs. SOC control with low ctDNA subgroup (HR=0.62 [95% CI, 0.23-1.70])

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Low baseline ctDNA is a likely prognostic and predictive factor

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Immune data were consistent with clinical activity

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Functional neoantigen-specific T cells were observed in all 16/16 GRANITE patients tested by ELISPOT

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Association of PFS and peak ex vivo ELISPOT responses was apparent, suggesting that ex vivo ELISPOT may be a surrogate for PFS

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GRANITE demonstrated a favorable safety and tolerability profile

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No patients discontinued study treatment due to an adverse event ("AE")

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Common AEs were the mild systemic and local effects associated with any potent vaccine, i.e. transient flu-like illness

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One treatment-related serious AE (fatigue) occurred in the GRANITE arm (patient continued GRANITE treatment without recurrence upon recovery)

The Company plans to review the PFS data with the U.S. Food and Drug Administration in the coming months and agree on next steps to advance GRANITE, including a potential Phase 2 or 3 trial using ctDNA levels as eligibility criteria.

On September 30, 2024 Cartherics Pty Ltd ("Cartherics" or "Company"), a biotechnology company developing immune cell therapies for the treatment of cancer and other diseases, reported that it has successfully raised well over its target AU$15M in an oversubscribed financing round (Press release, Cartherics, SEP 30, 2024, View Source [SID1234646960]).

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This funding will support the clinical trial for CTH-401, the Company’s lead cell therapy for ovarian cancer, and expand its pipeline to include other diseases.

Cartherics’ Chief Executive Officer, Prof. Alan Trounson AO commented: "The successful capital raising, in times of scant investment support in biotechnology, is welcome and further supports confidence in the company for the delivery of effective therapies in ovarian cancer and other difficult diseases."

CTH-401 is the only natural killer (NK) cell product currently under development that incorporates a chimeric antigen receptor (CAR) that targets the adenocarcinoma specific antigen, TAG-72 – a well-validated tumour marker, widely expressed in a range of solid tumours, including ovarian, gastric, colorectal, prostate and pancreatic cancers.

Cartherics has demonstrated that CTH-401 is very effective in killing ovarian cancer cells in both tissue culture and animal models, with initiation of the first clinical trial planned for next year.

Cartherics’ Chairman, Bob Moses said: "We are eager to start the CTH-401 clinical trial, building on promising preclinical results. This milestone reflects our commitment to innovative ovarian cancer treatments and our investors’ confidence in our vision to improve patient outcomes and drive growth."

On September 30, 2024 Valerio Therapeutics S.A. (Euronext Growth Paris: ALVIO), a clinical-stage biotechnology company leading the way in the development of innovative DNA Decoy therapeutics, reported the acquisition of Emglev Therapeutics, a spinoff of Institut Curie, one of France’s leading cancer centers (Press release, Valerio Therapeutics, SEP 30, 2024, View Source [SID1234646959]).

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Valour Bio has been established as a wholly owned subsidiary of Valerio Therapeutics to focus on discovering single domain antibodies (sdAbs) as drug and radio conjugates, bispecific T-cell engagers, blocking and binding sdAbs, or CAR-T sdAb drug candidates for multiple therapeutic areas.

Emglev’s proprietary synthetic sdAb libraries overcome the current barriers of conventional antibody discovery by providing unique targets and selecting humanized or fully human sdAbs entirely in vitro. These sdAbs are validated for a wide array of therapeutic applications, offering a distinct advantage in antibody development.

Animal free sdAb screening strategies exploit highly diverse synthetic libraries and functional screening, enabling the identification of validated lead binders in less than two months. Since Emglev’s antibody scaffold is already humanized or derived from human variable region of the heavy chain (VH), there is no need for further humanization, thereby streamlining the development process and reducing the risk of immunogenicity and loss of affinity.

Emglev’s sdAb technology has broad applications in several therapeutic areas such as inflammatory disorders, autoimmune diseases and cancer, but it also allows for the development of various treatment modalities including sdAb drug and radio-conjugates, bispecific T-cell engagers, blocking and binding sdAb or CAR-T sdAb drugs to name a few. Pre-clinical proof-of-concept of the sdAb technology has been obtained using CAR-T sdAb, BiTE sdAb and sdAb-drug conjugate, highlighting the ability of Emglev to rapidly and efficiently discover tailored antibody fragments with enhanced properties. Added to the expertise brought by Valerio Therapeutics, this positions Valour Bio with a versatile and promising platform for developing new best-in-class or first-in-class therapies for a wide range of diseases.

The acquisition is structured through a sale of shares paid in cash and a contribution in kind of Emglev shares against Valour Bio shares. As a result, shareholders of Emglev became shareholders of Valour Bio.

Dr. Shefali Agarwal, President & Chief Executive Officer of Valerio Therapeutics and CEO of Valour Bio, stated:

"The acquisition of Emglev Therapeutics through our newly formed subsidiary Valour Bio reinforces our commitment to bring innovative therapeutics to patients. We are excited to advance this next-generation technology developed by the accomplished scientists at Emglev and we look forward to the novel treatments this platform may yield like sdAb drug and radio -conjugates, bispecific T-cell engagers, blocking and binding sdAb or CAR-T sdAb drugs. Furthermore, we believe that the versatility of this platform can not only target a wide array of antigens but potentially address current limitations of antibody-based therapeutics such as tissue penetration, immunogenicity and ease of manufacturing. Following this acquisition, Valour Bio will focus on optimizing the therapeutic use of single-domain antibodies, focusing on developing treatments for severe and life-threatening diseases, including but not limited to autoimmune and inflammatory diseases as well as cancers."

Christelle Masdeu, CEO of Emglev Therapeutics, commented:

"We are excited to enter into this joint effort with Valerio Therapeutics. As a shareholder of Valour Bio now holding the Emglev platform, we remain committed to investing our energy, expertise and passion to develop unique treatments addressing unmet medical needs. The synergy created by combining Emglev’s proprietary synthetic single-domain antibody platform with Valerio’s distinctive preclinical and clinical development expertise perfectly aligns with our mission to deliver transformative therapies that make a meaningful difference in patients’ lives."

Dr. Sandrine Moutel, Emglev’s co-founder and head of the Antibody facility at Institut Curie and Dr. Franck Perez, Emglev’s co-founder and a member of its scientific board, director of the Cell Biology and Cancer Unit at Institut Curie, stated jointly:

"As founding members of Emglev, we are delighted to enter into this agreement which represents the culmination of tireless work by a dedicated team of scientists. Our team has created a platform that can generate therapeutics including CAR-Ts, T-cell engagers, binders, and bispecifics sdAbs demonstrating wide and versatile applicability to many diseases. This new chapter allows us to bring our vision of single-domain antibodies one step closer to the clinic and we are looking forward to beginning this journey with Valerio Therapeutics."

Cécile Campagne, Director of Institut Curie’s Technology Transfer Office, declared:

"We are proud of the path taken by Emglev Therapeutics, a start-up that has emerged from Institut Curie’s incubation program, which is moving forward at full speed! The acquisition of Emglev, less than two years after its creation, by Valour Bio, a subsidiary of Valerio Therapeutics which is a listed company, reflects the quality of Institut Curie’s innovations and the importance of putting them at the service of the creation of companies that will bring these cutting-edge technologies to all patients."

To this end, Valerio Therapeutics will provide Valour Bio with services in terms of scientific know-how in drug development, company support and equipment against financial compensation.

Given the potential of the technology and depth of the market potential, Valour Bio is confident that it will find the needed financial resources to support the company’s growth. The acquisition and operation of Emglev will enable new scientific and financial synergies to be created within the Valerio Group, with the first benefits for Valerio Therapeutics and Valour Bio expected to materialize in Q1 2025.

On September 30, 2024 Indivumed Therapeutics reported significant changes in the molecular characteristics of tumor samples within as little as 10 minutes after surgical removal (Press release, Indivumed Therapeutics, SEP 30, 2024, View Source [SID1234646957]). The findings underline the importance of rapid snap freezing of tissue samples to reliably capture cancer biology and reveal novel drug targets.

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Publishing in Cell Death & Disease, the study analyzed the impact of cold ischemia time (the time it takes to preserve surgically removed tissue) on more than 1,800 tumor and matched normal tissue samples across four cancer types: colorectal, liver and two subtypes of lung cancer.

Multi-omics analysis was used to compare gene expression, proteins, and phosphoproteins in samples with cold ischemia times ranging from less than 10 to more than 25 minutes. Significant differences emerged in the expected patterns of gene and protein activity in samples with longer cold ischemia times compared with those frozen within less than 10 minutes.

Senior Data Scientist at Indivumed Therapeutics, Silvia von der Heyde, said, "We already knew that the molecular expression profile in tissue samples changes the longer they are outside the body, but we were surprised to see the extent of the differences after just ten minutes. This misleading picture of cancer biology has a significant impact on drug discovery, leading researchers to waste time and money on false leads or simply not seeing potentially important targets for novel therapeutics."

Through its Global Clinical Network partners, Indivumed Therapeutics has gathered an unrivaled biobank of many thousands of high-quality tumor and normal tissue samples, collected and rapidly frozen within an average of 10 minutes cold ischemia time according to the company’s strict protocols.

"We’re proud to build on this unique resource, using the potential of bioinformatics, biomathematics, and cell biology to identify, validate, and characterize novel oncology drug targets." says Jobst Landgrebe, Head of R&D and Chief Technology Strategist at Indivumed Therapeutics.

This paper validates the original vision of Indivumed’s founder and CEO Hartmut Juhl, who recognized that the best way to find genuine targets for novel cancer drugs is to get as close to the molecular reality of the disease as possible.

Juhl says, "I am beyond excited to see my vision from 22 years ago reflected in our work today. Our long-standing commitment to sample and data quality is finally paying off and contributing to the search for new treatments that will bring forward the promise of effective cancer medicine for all the patients out there."