On September 16, 2024 CEL-SCI Corporation (NYSE American: CVM) reported new data from its concluded Phase 3 study of Multikine (Leukocyte Interleukin, Injection)* that were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress on Saturday, September 14, 2024 in a poster titled "Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy" (Press release, Cel-Sci, SEP 16, 2024, View Source [SID1234646685]). This data is highly relevant to CEL-SCI’s 212 patient confirmatory Registration Study which has received the U.S. Food and Drug Administration’s (FDA) go-ahead and is currently under preparation.

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Summary of Phase 3 Study: Multikine-treated patients who were recommended treatment of surgery and radiotherapy had a nearly 4-year survival benefit over control group

As previously reported, CEL-SCI’s completed Phase 3 study of 923 patients showed that newly diagnosed head and neck cancer patients who were deemed at low risk for recurrence after surgery (and therefore recommended to receive only radiotherapy after surgery) had a median overall survival (OS) benefit of 46.5-months, almost 4-years, over control patients. However, patients who were deemed to be high risk for recurrence after surgery (and therefore recommended to have chemotherapy added to the radiotherapy after surgery) showed no survival benefit.

Upcoming FDA Confirmatory Registration Study

Since the completed Phase 3 study showed clear survival benefit for some, but not all of the patients, the FDA requested that CEL-SCI conduct a confirmatory Registration Study focusing on the patients who showed the best survival benefit. Based on the data, CEL-SCI determined this target population to be patients with newly diagnosed locally advanced primary head and neck cancer with no lymph node involvement and with low PD-L1 tumor expression. Applying these selection criteria to the completed Phase 3 study of 923 patients resulted in the target population (n=114) having a 73% survival at 5 years vs a 45% survival at 5 years for the control patients, log rank p=0.0015. The hazard ratio was an exceptional 0.34, with a 95% confidence interval upper limit of 0.65; Wald p=0.0012 and achieving a 66% reduction in the overall risk of death.

Summary of New Data Presented at ESMO (Free ESMO Whitepaper)

The new data presented at ESMO (Free ESMO Whitepaper) includes a further analysis of the 114 patients in the completed Phase 3 study who met these target population selection criteria and form the basis for the confirmatory study. Specifically, the new analysis focused on those patients who were deemed low risk for recurrence (recommended to be given only radiotherapy – but no chemotherapy, per National Comprehensive Cancer Network "NCCN" guidelines) following surgery (n=79) as opposed to the selected patients who were deemed high risk for recurrence and who were recommended to have chemotherapy added to their treatment following surgery per the same guidelines (n=35).

While the overall survival benefit was clear and statistically significant (log rank p=0.0015) for the entire target population (n=114), the 79 patients who were recommended to receive only radiotherapy benefited to an even greater degree from pre-surgery treatment with Multikine than the group of 114 as a whole. This target low risk population (n=38) had a 5-year overall survival of 82.6% when treated with Multikine vs. 47.3% when treated with standard of care alone (n=41), without overlap in their respective 95% confidence intervals. More recent analysis for the target low risk population (n=79) showed a hazard ratio of 0.27 (95% CI [0.12, 0.64], Wald p=0.0027) achieving a 73% reduction in overall risk of death.

Management Commentary

"The additional data presented this weekend at ESMO (Free ESMO Whitepaper) 2024 provides further evidence that we have identified the target population that has the greatest survival benefit from Multikine, and that our study criteria can select for these patients upon diagnosis, before surgery," stated CEL-SCI CEO Geert Kersten. "It makes sense that Multikine, an immunotherapy, provides even greater benefit to patients who are not scheduled to receive chemotherapy following surgery, given the known detriments of chemotherapy on the immune system. Seeing more clearly than ever that patients who were not recommended chemotherapy benefited the most begs the question: What if, through better diagnostic technology such as the PET scan, which we will be using in the confirmatory study, resulting in better patient selection, we could treat only those patients who are supposed to be treated with radiotherapy alone, and not chemotherapy? The data presented at ESMO (Free ESMO Whitepaper) is clear. This would lead to even better 5-year survival, 82% instead of 73%."

CEL-SCI’s CSO Eyal Talor, Ph.D. commented, "The criteria we developed for selecting these locally advanced head and neck cancer patients clearly showed that when patients were treated with Multikine before surgery, they demonstrated an overall survival advantage over control irrespective of whether these patients were characterized as being at low- or high-risk for recurrence following surgery. With this new analysis we also saw that patients selected by these criteria who are deemed low risk for recurrence post-surgery have a further improved survival outcome with a hazard ratio of 0.27, which even is better than the already exceptional hazard ratio of 0.34 seen for the overall selected population."

The data were presented at ESMO (Free ESMO Whitepaper) 2024 by the study’s co-author József Tímár MD, PhD, DSc, Professor Department of Pathology, Forensic and Insurance Medicine at Semmelweis University in Budapest, Hungary. Dr. Timar served as the Director of the Central Pathology Laboratory for CEL-SCI’s IT-MATTERS Phase 3 study. With 174 peer reviewed studies published, Dr. Timar is a founding editor, editor in chief, or a member of the editorial board of four oncology journals. He is the recipient of a dozen honors and awards for excellence in cancer research and teaching.

On September 16, 2024 Debiopharm, a privately-owned, Swiss-based, biopharmaceutical company aiming to establish tomorrow’s standard-of-care to cure cancer and infectious diseases, reported the signing of a partnership with WhiteLab Genomics (WLG), a Paris and Boston-based biotech company specializing in Artificial Intelligence for Genomic Medicine R&D (Press release, Debiopharm, SEP 16, 2024, View Source [SID1234646684]). This collaboration aims to achieve two key objectives. The first is to identify cancer-specific receptors that are overexpressed on the surface of cancer cells. The second is to find lead candidates capable of binding to these receptors. Once identified, these agents will be attached to the surface of lipid nanoparticles (LNPs) to enhance active tissue targeting.

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Lipid nanoparticles (LNPs) have emerged as a gold standard vector for drug delivery with the potential to transform the oncology landscape, particularly the genomic medicine field, through their high delivery efficacy. One of the challenges with LNPs is their tendency to accumulate in non-target organs like the liver, which limits their distribution to the desired site. To maximize LNPs potential, WLG is set to deploy its AI-powered platform to explore innovative in silico approaches for enhancing the targeted delivery capabilities of LNPs, creating new opportunities for therapeutic applications.

As a key investor in WLG, Debiopharm has consistently supported the company’s innovative approaches and cutting-edge solutions through Debiopharm’s Innovation Fund. This collaboration provides a significant opportunity to validate the potential of WLG’s AI platform and strengthen their ongoing relationship.

"WhiteLab’s innovative AI platform represents a significant advancement in the field of targeted cancer therapy. By improving the specificity and delivery of lipid nanoparticles, we aim to provide more effective treatments for patients suffering from hormonal cancers," said David Del Bourgo, CEO co-founder of WLG. "Our collaboration with a leading player such as Debiopharm further demonstrates our focus on developing cutting-edge solutions that have the potential to transform cancer treatments."

"As a company specialized in drug development, we know the hardships of the field and therefore we recognize the added value of AI powered platforms like the WhiteLab Genomics’ platform applied to the early stages of drugs R&D. We look forward to fully exploiting the potential of AI based solutions to develop drugs, de-risk assets, de-risk any toxicity, and ensure that we bring the most efficacious and personalized drugs to patients." said Betrand Ducrey, CEO of Debiopharm

On September 16, 2024 IMMUNOPRECISE ANTIBODIES LTD. (the "Company" or "IPA") (NASDAQ: IPA), an artificial intelligence-driven biotherapeutic research and technology company, reported financial results for the first quarter of its 2025 fiscal year ("FY25"), which ended July 31, 2024. All numbers are expressed in Canadian dollars, unless otherwise noted (Press release, ImmunoPrecise Antibodies, SEP 16, 2024, View Source [SID1234646683]).

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"Our commitment to end-to-end in silico engineering of antibodies for challenging targets has delivered exceptional results. By harnessing our advanced computational capabilities, we’ve not only accelerated our R&D process, but also significantly reduced costs compared to traditional technologies," stated Dr. Jennifer Bath, President and CEO. "Notably, two of our three wet lab locations reported impressive double-digit revenue growth this quarter, underscoring the effectiveness of our strategy. Our third site focused efforts on rebuilding its off-the-shelf inventory, a move designed to support future sales of higher-margin products. This balanced approach of driving immediate growth while strategically positioning for future profitability demonstrates our commitment to long-term value creation. Our innovative antibody development techniques, combined with astute operational decisions, reinforce our competitive edge in this dynamic market. We’re confident that this multifaceted strategy will continue to drive sustainable growth and enhance shareholder value in the quarters to come."

Business Highlights and Corporate Update

ImmunoPrecise continues to execute its growth strategy, focusing on scaling its AI-driven antibody drug discovery platforms, expanding its global client base, and optimizing operational efficiencies. The Company’s comprehensive approach to antibody discovery and development is consistently yielding tangible outcomes. In the quarter ending July 31, 2024, ImmunoPrecise recorded revenue of $5.3 million, with two of its three sites delivering impressive double-digit growth rates year-over-year. While IPA’s protein manufacturing site experienced a year-over-year decline this quarter, this was primarily due to an exceptionally strong performance in the previous year and rebuilding off-the-shelf inventory of higher-margin products. ImmunoPrecise’s balanced approach of driving immediate growth while strategically positioning for the future underscores its commitment to long-term value creation and reinforces its competitive edge in the dynamic antibody market.

BioStrand is driving revenue growth through its LENSai platform, offering fee-for-service solutions while developing commercial products tailored to IPA’s extensive client base of over 600 companies. Recently, BioStrand achieved a significant breakthrough by developing and optimizing antibodies entirely through computer simulations, targeting a protein within the Tumor Microenvironment (TME). This achievement is particularly noteworthy given the TME’s intricate nature and the lack of prior structural data on the target. Despite these formidable challenges, our team successfully navigated the complexities of end-to-end in silico antibody development in this demanding context. Building on this AI-driven success, BioStrand is now accelerating the rollout of advanced in silico antibody engineering solutions, positioning the company at the forefront of computational drug discovery and potentially opening new revenue streams in this high-growth market segment.

BioStrand is also making progress in developing applications for the AWS Marketplace. For context, the AWS Marketplace is a global platform that enables partners to leverage Amazon Web Services’ technologies, programs, and expertise to build and deliver cutting-edge solutions. This marketplace provides BioStrand with enhanced visibility to a broader customer base, opening new opportunities for growth and collaboration while ensuring the delivery of high-quality, AI-driven SaaS solutions.

Talem Therapeutics and BioStrand have partnered to enhance Talem’s lead therapeutic assets using BioStrand’s LENSai software suite, which includes over 80 in silico applications. This collaboration aims to increase the data quality and value of these assets. By leveraging LENSai to refine existing lead candidates, the Company expects to improve their market readiness and commercial potential. The Company plans to soon release more details on these enhanced assets, demonstrating LENSai’s utility in antibody engineering and its impact on asset value.

First Quarter FY25 Financial Results

Revenue: Total revenue was $5.3 million, compared to revenue of $5.7 million in fiscal year 2024 ("FY24") Q1. Project revenue generated $4.9 million, including projects using IPA’s proprietary B Cell Select platform and IPA’s proprietary LENSai platform, compared to $5.2 million in FY24 Q1. Product sales and cryostorage revenue were $0.4 million, compared to $0.5 million in FY24 Q1.
Research & Development (R&D) Expenses: R&D expenses were $1.6 million, compared to $0.9 million in FY24 Q1, with the increase reflecting increased expenditures related to the build of the Company’s LENSai platform.
Sales & Marketing (S&M) Expenses: S&M expenses were $0.7 million, compared to $1.1 million in FY24 Q1. The reduction in sales and marketing expenses reflect lower share-based expense and cost savings from our synergistic sales efforts across our comprehensive AI-enhanced antibody discovery and development services.
General & Administrative (G&A) Expenses: G&A expenses were $4.2 million, compared to $4.0 million in FY24 Q1.
Net Loss: Net loss of $4.0 million, or $(0.15) per share on a basic and diluted basis, compared to a net loss of $3.4 million or $(0.14) on a basic and diluted basis in FY24 Q1.
Liquidity: Cash totaled $4.0 million as of July 31, 2024, compared to $6.8 million as of July 31, 2023.
Conference Call and Webcast Details

The Company will host a live conference call and webcast to discuss these results and provide a corporate update on Monday, September 16, 2024, at 10:30AM ET.

The conference call will be webcast live and available for replay via a link provided in the Events section of the Company’s IR pages at View Source

***Participant Dial-In Details***

Participants call one of the allocated dial-in numbers (below) and advise the Operator of either the Conference ID 3224490 or Conference Name.

USA / International Toll +1 (646) 307-1963
USA – Toll-Free (800) 715-9871
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Canada – Toll-Free (800) 715-9871

On September 16, 2024 Cellworks Group Inc., a leader in Personalized Therapy Decision Support and Precision Drug Development, reported results from a study using the Cellworks Platform to predict homologous recombination deficiency (HRD) and the effectiveness of PARP inhibitors (PARPi) in real-world cohorts of patients with ovarian, pancreatic, prostate, and triple-negative breast cancers (TNBC) (Press release, Cellworks, SEP 16, 2024, View Source [SID1234646682]). This research highlights the potential of the Cellworks mechanistic biosimulation model to go beyond BRCA mutation status and provide more comprehensive predictions of patient response to PARPi therapies.

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Results from the study were showcased in a poster presentation titled, Use of Biosimulation to Predict Homologous Recombination Deficiency and PARPi Benefit in Patients with Ovarian, Pancreatic, Prostate and Triple Negative Breast Cancers, as part of the ESMO (Free ESMO Whitepaper) Congress 2024 held in Barcelona, Spain from September 13-17, 2024.

"PARP inhibitors have become a standard treatment for cancers characterized by homologous recombination deficiency," said Professor Daniel Palmer, Molecular and Clinical Cancer Medicine, University of Liverpool, and Principal Investigator of the study. "However, current HRD tests do not capture all patients who might benefit from PARPi, often focusing primarily on BRCA status. By using Cellworks personalized therapy biosimulation in this study, we produced an HRD classifier that was predictive of PARPi benefit in patients with wild-type BRCA. This is an important step towards using biosimulation to identify wild-type BRCA patients who may benefit from PARPi therapy."

"Biosimulation incorporates multiple levels of genomic, transcriptomic, and protein regulation, capturing the molecular interactions of key homologous recombination components," said Dr. Michael Castro, Cellworks Chief Medical Officer. "This study utilized Cellworks mechanistic biology model along with a patient’s tumor-based genomic profile to identify dysregulations in HR signaling pathways and predict differential responses to PARPi. Through this approach, we can capture patients with HRD but lacking BRCA1 or BRCA2 mutations who can benefit from PARPi therapy, leading to more personalized and effective treatment strategies."

Study Design

Cellworks computational biosimulation was performed on four real-world retrospective cohorts from The Cancer Genome Atlas (TCGA), including ovarian, pancreatic, prostate, and triple-negative breast cancer patients. The model output, representing key HR pathways, was used to develop a classifier that distinguishes HRD by comparing BRCA wild-type (WT) ovarian cancer patients (n=32) to BRCA-mutated patients (n=187). This locked classifier was then prospectively validated in independent sets of ovarian, pancreatic, and prostate cancer patients (n=336, 428, 189 respectively). Efficacy scores, based on biosimulated composite cell growth in response to the PARP inhibitor Olaparib, were evaluated in relation to the predicted HRD status in BRCA wild-type patients.

Study Results

The HRD classifier produced through Cellworks biosimulation was significantly associated with BRCA status across all four validation sets, demonstrating strong predictiveness for BRCA status in ovarian (AUC = 0.863, p < 0.001), pancreatic (AUC = 0.759, p = 0.002), prostate (AUC = 0.717, p < 0.001), and TNBC (AUC = 0.88, p < 0.001) cancers. Additionally, in all four cancer types, predicted PARPi efficacy was significantly higher in BRCA wild-type patients identified as HRD (ovarian p = 0.026, prostate p < 0.001, pancreatic p < 0.001, TNBC p < 0.001).

The Cellworks Platform

The Cellworks Platform performs computational biosimulation of protein-protein interactions, enabling in silico modeling of tumor behavior using comprehensive genomic data. This allows for the evaluation of how personalized treatment strategies interact with the patient’s unique tumor network. Multi-omic data from an individual patient or cohort is used as input to the in silico Cellworks Computational Biology Model (CBM) to generate a personalized or cohort-specific disease model. The CBM is a highly curated mechanistic network of 6,000+ human genes, 30,000 molecular species and 600,000 molecular interactions. This model along with associated drug models are used to biosimulate the impact of specific compounds or combinations of drugs on the patient or cohort and produce therapy response predictions. The Cellworks CBM has been tested and applied against various clinical datasets with results provided in over 125 presentations and publications with global collaborators.

On September 16, 2024 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer and rare diseases, reported that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to P-BCMA-ALLO1, an investigational stem cell memory T cell (TSCM)-based allogeneic CAR-T cell therapy in Phase 1/1b clinical development for the treatment of patients with relapsed/refractory multiple myeloma (Press release, Poseida Therapeutics, SEP 16, 2024, View Source [SID1234646681]).

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RMAT designation includes all the benefits of the Fast Track and Breakthrough Therapy designation programs, including early interactions with the FDA. Poseida’s RMAT application was evaluated based on encouraging early data from its ongoing Phase 1 study of P-BCMA-ALLO1, which demonstrated P-BCMA-ALLO1’s potential to offer promising efficacy, safety profile and rapid ‘off-the-shelf’ patient access.

"The RMAT designation for P-BCMA-ALLO1, our lead program, is based on impressive early clinical data from our ongoing Phase 1 study and further validates its potential to address the unmet needs of patients with relapsed/refractory multiple myeloma," said Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics. "Importantly, our data has shown clinical responses in very sick, refractory patients, including those that have received prior BCMA-targeted therapies. With both RMAT and Orphan Drug designations for P-BCMA-ALLO1, we look forward to working closely with the FDA as we continue to advance this next-generation, off-the shelf allogeneic CAR-T therapy, including the recently initiated Phase 1b portion of the trial."

The Company will report new clinical data from the P-BCMA-ALLO1 Phase 1 study in an oral session at the 21st International Myeloma Society Annual Meeting, which is being held in Rio de Janeiro from September 25-28, 2024. Additional clinical updates are planned for the second half of 2024, subject to coordination with Roche, which has a strategic collaboration with Poseida covering multiple investigational allogeneic CAR-T therapies targeting blood cancers, including P-BCMA-ALLO1.

The RMAT designation is a program under the 21st Century Cures Act that is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such disease or condition.

RMAT designation includes all Breakthrough Therapy designation features, including early interactions to discuss any potential surrogate or intermediate endpoints. RMATs may be eligible for accelerated approval based on previously agreed-upon surrogate or intermediate endpoints that are reasonably likely to predict long-term clinical benefit, or reliance upon data obtained from a meaningful number of sites, including through expansion to additional sites, as appropriate.

About P-BCMA-ALLO1
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate licensed to Roche targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. This allogeneic program includes a VH-based binder that targets BCMA and clinical data presented at ASH (Free ASH Whitepaper) in December 2023 support the Company’s belief that T stem cell (TSCM)-rich allogeneic CAR-Ts have the potential to offer effective, safe, and reliable treatment addressing unmet needs in multiple myeloma. The FDA has granted P-BCMA-ALLO1 Regenerative Medicine Advanced Therapy (RMAT) designation for adult patients with relapsed/refractory multiple myeloma after three or more prior lines of therapies including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and anti-CD38 antibody in addition to Orphan Drug designation for multiple myeloma. Additional information about the Phase 1/1b study is available at www.clinicaltrials.gov using identifier: NCT04960579.