On February 14, 2022 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended December 31, 2021, as well as key clinical and corporate developments (Press release, Cel-Sci, FEB 14, 2022, View Source [SID1234608087]).

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Clinical and Corporate Developments include:

During the first fiscal quarter, CEL-SCI completed the commercial scale build out of its dedicated current Good Manufacturing Practice (cGMP) facility in which it manufactures its investigational immunotherapy Multikine (Leukocyte Interleukin, Injection)*. The construction is designed to ensure the facility will be compliant with all U.S. Food and Drug Administration’s (FDA) and European cGMP regulations. Production capacity has been doubled to meet anticipated market demand for Multikine once it receives regulatory approval.
Based on the results of its pivotal Phase 3 study, CEL-SCI intends to file a Biologic License Application (BLA) with the FDA for approval of the Multikine treatment regimen in locally advanced primary squamous cell carcinoma of the head and neck in patients scheduled to receive surgery and radiation as their primary treatments.
CEL-SCI’s trial was conducted in over 20 countries in which marketing clearance applications may also be filed subsequent to FDA filing and/or approval.
The head and neck cancer patients who are scheduled to receive surgery and radiation as their first treatments have not seen a marked improvement in their treatment outcome in decades. This is clearly an unmet medical need. The study showed great improvement in survival with no noted safety issues for these patients. The number of patients who could benefit from this treatment annually is large, estimated at about 211,000 globally.
As of December 31, 2021, CEL-SCI had $37.1 million in cash and cash equivalents.
"Our current focus is in three primary areas, all of which are essential steps towards drug approval. First, we are assembling the clinical documentation required to obtain FDA approval of a Biologic Product License which will allow commercial marketing of Multikine, second, we are preparing and submitting our compelling data to peer review publications and third, on the production side, we are preparing the facility for FDA inspection as part of the Biologic Establishment License Application process. We look forward to providing updates on these essential steps as we reach milestones," stated CEL-SCI CEO, Geert Kersten.

CEL-SCI reported a net loss available to common shareholders of approximately $8.8 million for the quarter ended December 31, 2021 versus a net loss of approximately $8.0 million for the quarter ended December 31, 2020.

During the quarter ended December 31, 2021, CEL-SCI completed a major upgrade of its leased manufacturing facility to prepare for the potential commercial production of Multikine. Total costs of this upgrade were approximately $11.1 million, of which the landlord of the property agreed to finance $2.4 million. The landlord financing is being repaid through increased lease payments over the remaining term of the lease.

On February 14, 2022 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported an update on PharmaCyte’s activities to lift the U.S. Food and Drug Administration’s (FDA) clinical hold on PharmaCyte’s treatment for locally advanced, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, FEB 14, 2022, View Source [SID1234608086]). After submission of an initial Investigational New Drug Application (IND), the FDA requested additional studies and information as a prerequisite for approval of PharmaCyte’s IND. A number of additional studies and assays have already been completed; several others are quite lengthy and are underway or are slated to begin soon. As each study and assay is completed, the results are being compiled and will make up PharmaCyte’s complete IND submission package to the FDA.

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, explained, "Given our treatment is a biologic and our technology is a one of a kind live-cell encapsulation, we completely understand the FDA’s need for more information. We are confident that with the data we’re producing in our additional studies and assays that the FDA will grant us an open IND just as the FDA granted us the Orphan Drug Designation for our treatment for LAPC.

"Last June, we provided an update on efforts being made by our team of regulatory and scientific experts that are addressing the FDA’s requirements to have the clinical hold lifted. Today, we would like to update that list and explain where we are in the process of delivering our updated IND package to the FDA. We have brought additional regulatory and scientific experts onboard the team, and we continue to engage closely with leading Contract Research Organizations (CROs) and our partner Austrianova to ensure a successful IND submission.

"We also want to make it clear that PharmaCyte and its service providers are under the same constraints as everyone else globally with supply chain issues, Covid related delays, and late delivery of materials that are needed to complete studies and assays required by the FDA. This also applies to the manufacturing of empty capsules and encapsulated live cells that are necessary to complete many of these required studies and assays. Moreover, many of the laboratories that are conducting our studies and assays are being met with long delays in receiving the consumables needed to conduct the specific studies for which they are responsible. So, we’re at the mercy of what is a global problem, which obviously makes it more difficult to offer any accurate timelines for completion of the FDA required assays and studies."

Below is the list of items on which PharmaCyte has been working, including updates on those tests previously reported.

Additional Regulatory Expertise Added to IND Team – In addition to its established team of experts, PharmaCyte has retained Biologics Consulting as a fresh set of regulatory eyes to perform a regulatory "Gap Analysis" and to assist with PharmaCyte’s IND submission. Biologics Consulting is a full-service regulatory and product development consulting firm for biologics, pharmaceuticals and medical devices and has personnel with extensive FDA experience. Although it took a lengthy amount of time to onboard Biologics Consulting, this should augment PharmaCyte’s ability to submit an acceptable IND to the FDA.
Stability Studies on PharmaCyte’s Clinical Trial Product – PharmaCyte has now successfully completed a product stability study after 3, 6, 9, 12 and 18-months of storage frozen at -80C on PharmaCyte’s clinical trial product known as CypCaps, including container closure integrity testing for certain timepoints. The next time point in this ongoing stability study will be at 24 months of product stability of the CypCaps. This 24-month time point analysis is ready to commence, and data will be available in the coming weeks.
Additional Studies Requested by the FDA – PharmaCyte has designed and commenced various additional studies requested by the FDA, including a stability study on the cells from its Master Cell Bank (MCB) used to make the CypCaps. PharmaCyte is already at the 3-year stability timepoint for the cells from its MCB.
Determination of the Exact Sequence of the Cytochrome P450 2B1 Gene – PharmaCyte has completed the determination of the exact sequence of the cytochrome P450 2B1 gene inserted at the site previously identified on chromosome 9 using state-of-the-art nanopore sequencing, a cutting edge, unique and scalable technology that permits real-time analysis of long DNA fragments. The result of this analysis of the sequence data confirmed that the genes are intact.
Biocompatibility Studies – PharmaCyte has designed and commenced 8 biocompatibility studies, 6 of which have been completed successfully. The remaining 2 studies are underway. Those studies are the Acute Systemic Toxicity Study of Empty Cellulose Sulphate Capsules in Mice and the Skin Sensitization Study of Empty Cellulose Sulphate Capsules in Guinea Pigs. To enable these studies to be performed, Austrianova manufactured and delivered an additional 400 syringes of empty capsules. Some of the data being generated will also be used to demonstrate comparability with the CypCaps successfully used in two earlier clinical trials for pancreatic cancer.
Micro-Compression and Swelling Assays – This project is underway. The project is developing and optimizing two reproducible methods for testing and confirming the physical stability and integrity of the CypCaps produced under GMP. These studies required the acquisition of new equipment by Austrianova as well as validation and integration into Austrianova’s Quality Control laboratory.
Break Force and Glide Testing – PharmaCyte is in the process of developing a protocol to measure whether the syringe, attached to the catheter when used to expel the capsules, will still have a break and glide force that is within the specification that PharmaCyte has established. PharmaCyte will set this specification based on the syringe/plunger manufacturer’s measured break and glide forces, or alternatively, accepted ranges for glide forces routinely used in the clinic.
CypCaps Capsules Compatibility with the Syringe and Other Components of the Microcatheter Delivery System – PharmaCyte has commenced studies designed to show that CypCaps are not in any way adversely affected by the catheters used by interventional radiologists to deliver them into a patient. Compatibility data is being generated to demonstrate that the quality of the CypCaps is maintained after passage through the planned microcatheter systems.
CypCaps Capsules and Cell Viability after Exposure to Radiological Contrast Medium – PharmaCyte has designed and commenced a project to test the effect of the exposure of CypCaps to two routinely used types of contrast medium that interventional radiologists use to implant the CypCaps in a patient. The contrast medium is used to visualize the blood vessels during implantation of the CypCaps.
Master Drug File Information- Austrianova is providing additional detailed confidential information to the FDA on the manufacturing process, including information on the improvements and advancements made to the product since the last clinical trials were conducted with respect to reproducibility and safety. However, Austrianova has not changed the overall physical characteristics of the CypCaps. PharmaCyte is supporting Austrianova financially in this work.
Additional Documentation Requested by the FDA – PharmaCyte is in the process of updating its documentation including extending its discussion on immunological aspects of its LAPC treatment.
Pig Study – Finally, the Company has designed a study in pigs to address biocompatibility and long-term implantation and dispersion of the CypCaps. This animal study will complement the positive data already available from the previous human clinical trials showing the safety of CypCaps implantation in human patients.

On February 14, 2022 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported that it will announce financial results for the fourth quarter and full year of 2021 along with recent corporate updates on February 28, 2022 (Press release, Mirati, FEB 14, 2022, View Source [SID1234608084]). During a conference call at 4:30 p.m. ET / 1:30 p.m. PT on February 28, company executives will provide company updates and review financial results.

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Investors and the general public are invited to listen to a live webcast of the call at the "Investors and Media" section on Mirati.com or by dialing the U.S. toll free +1 313-209-4906 or international +1 877-502-9276, confirmation code: 6820152. A replay of the call will be available approximately 2 hours after the event has ended at the same website or by dialing in the U.S. toll free 1+ 719-457-0820 or international +1 888-203-1112, confirmation code: 6820152.

On February 14, 2022 Veracyte, Inc. (Nasdaq: VCYT) reported that new data validating the clinical utility of the company’s Decipher Prostate Genomic Classifier for guiding treatment selection in men with intermediate-risk prostate cancer will be presented Thursday, February 17, at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (ASCO GU) Symposium (Poster #M1) (Press release, Veracyte, FEB 14, 2022, View Source [SID1234608083]). The data, from the randomized, Phase 3 NRG/RTOG 0126 study, confirm initial findings that the Decipher Prostate Genomic Classifier is a prognostic biomarker that can help physicians and patients make personalized treatment decisions in the intermediate-risk setting.

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"These findings represent the first high-level evidence of any genomic classifier in intermediate-risk prostate cancer and demonstrate that the Decipher 22-gene biomarker significantly improves prognostic performance across numerous clinically meaningful endpoints," said Daniel Spratt, M.D., chair of Radiation Oncology at University Hospitals Seidman Cancer Center and professor and chair of the Department of Radiation Oncology at Case Western Reserve University School of Medicine, and lead investigator for the study.

Prostate cancer deemed "intermediate risk" by the National Comprehensive Cancer Network (NCCN) Guidelines for Prostate Cancer is the most heterogenous of all risk groups in prostate cancer, and there are a wide variety of treatment options available. The Decipher Prostate Genomic Classifier is a prognostic biomarker that provides additional information about the aggressiveness of individual patients’ cancer to help physicians more accurately categorize their risk and select appropriate treatments. The study conducted by Dr. Spratt and colleagues confirms findings from prior Phase 2 studies and validates for the first time the test’s performance in men with intermediate-risk disease using multi-center Phase 3 trial data.

To assess the prognostic performance of the Decipher Prostate test in the intermediate-risk, post-biopsy setting, researchers utilized patient biopsy samples from the phase 3, randomized NRG/RTOG 0126 trial. This study enrolled men with intermediate-risk prostate cancer, and then compared clinical outcomes following randomization to two different doses of radiation therapy (70.2 Gy vs 79.2 Gy) without hormone therapy. Researchers generated classifier data for each of 215 biopsy samples, then linked the data with clinical outcomes assessing multiple oncologic and survival endpoints. Patients were followed for a median of 12.8 years.

The findings, which will be presented for the first time at ASCO (Free ASCO Whitepaper) GU this week, show that the Decipher Prostate test was independently prognostic for all clinical endpoints, including disease progression (sub-distribution hazard ratio [sHR] 1.12, 95%CI 1.0-1.26, p=0.04), biochemical failure (sHR 1.22, 95%CI 1.1-1.37, p<0.001), distant metastasis (sHR 1.28, 95%CI 1.06-1.55, p=0.01), prostate cancer-specific mortality (sHR 1.45, 95%CI 1.2-1.76, p<0.001), distant metastasis-free survival (sHR 1.12, 95%CI 1.03-1.23, p=0.009), and overall survival (sHR 1.11, 95%CI 1.01-1.21, p=0.03).

Men in the study with disease classified as Decipher "high-risk" had worse 10-year oncologic and survival outcomes compared to those whose disease was classified as Decipher "low-risk." Based on this finding, men with NCCN intermediate-risk prostate cancer whose disease is classified as Decipher "high-risk" may have improved outcomes with treatment intensification (i.e., the addition of hormone therapy), while men with disease classified as Decipher "low-risk" may have favorable outcomes with radiotherapy alone.

The study also evaluated – within Decipher risk groups – the clinical impact of receiving lower- vs. higher-dose radiation. Among patients with lower Decipher risk scores, the difference in 10-year distant metastasis rates between those who received 70.2Gy vs. 79.2Gy was low (5%). However, among those with higher Decipher scores, the difference was 26%, suggesting that men whose disease is Decipher "high-risk" receive more benefit from the higher dose of radiation than their "low-risk" counterparts.

"This is an important study that provides further evidence for Decipher Prostate and demonstrates how this test could help physicians better personalize treatment for their patients with intermediate-risk prostate cancer," said Elai Davicioni, Ph.D., Veracyte’s senior vice president of Scientific and Clinical Operations, Urologic Cancers.

Informing Treatment in Asian Men with Prostate Cancer

In a second ASCO (Free ASCO Whitepaper) GU Symposium presentation scheduled for this Thursday (Poster #M4), researchers will share data from a study that evaluated the clinical utility of the Decipher Prostate Genomic Classifier in Asian men with prostate cancer, as well as the genomic differences between prostate cancer in Asian vs. Caucasian men enrolled in a 80,829-patient cohort. In a retrospective analysis of Asian men treated at the National Cancer Centre of Singapore, researchers found that Decipher "high-risk" scores were significantly associated with worse metastasis-free survival (hazard ratio 5.22, 95% CI:1.08–25.3, p=0.04). Additionally, the study found substantial differences in tumor biology between Asian and North American men with prostate cancer.

"This is the first report on the performance of the Decipher Prostate test for predicting aggressive disease in Asian men. We commend the researchers in Singapore for their efforts to investigate this question and to add to the existing body of evidence supporting Decipher’s validity in men of African and European descent," said Dr. Davicioni. "The findings from this study could help inform personalized treatment recommendations for Asian men with prostate cancer, and, more broadly, deepen our understanding of tumor biology that could benefit all men with the disease."

On February 14, 2022 2seventy bio, Inc. (NASDAQ: TSVT) reported that Steven Bernstein, M.D. will join the company effective today and assume the role of chief medical officer (CMO) (Press release, 2seventy bio, FEB 14, 2022, View Source [SID1234608082]). Also announced today, Susan Abu-Absi, Ph.D. has been appointed chief technology & manufacturing officer at 2seventy bio.

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"We are thrilled to welcome Steve to the 2seventy team as we bring our next-generation cell therapy product engine to the clinic. He brings both experience as a treating heme/oncology physician as well as extensive translational clinical expertise, including a deep knowledge of T-cell biology and immunology," said Nick Leschly, chief kairos officer. "I’m also pleased to share the promotion of Dr. Susan Abu-Absi to chief technology & manufacturing officer, leading 2seventy’s manufacturing and supply chain organization. Susan has already made significant contributions during her time at bluebird and now 2seventy as we continue to build both our collaborative manufacturing network and in-house capabilities. We look forward to both Susan and Steve’s contributions toward the development and delivery of transformational cell therapies for people living with cancer."

"I am excited to join 2seventy during the company’s first full year and am inspired by their patient-driven mission to outmaneuver cancer and provide people with the chance for more time. As a treating physician, I have witnessed firsthand the devastating impact that cancer can have and understand how precious time is to all of us," said Steven Bernstein, M.D., chief medical officer. "I look forward to applying my experience in early clinical development to support the team in evolving the treatment landscape in cell therapy to make a meaningful impact on the lives of patients, their families and care partners."

Steven Bernstein, M.D., has more than 25 years of experience as a treating physician, translational scientist, and pharmaceutical executive. Prior to joining 2seventy, Dr. Bernstein served as Chief Translational Officer at Turnstone Biologics. Before that, he served as Chair, Immuno-Oncology Translational Research and Development, and Head, Integrated Science, at Bristol-Myers Squibb (BMS). Prior to BMS, he was an academic physician scientist most recently at the University of Rochester’s James P. Wilmot Cancer Institute, where he was Professor of Medicine, Co-Director of the Hematological Malignancy and Lymphoma Biology Programs and Co-Principal Investigator of the University of Rochester/Arizona Cancer Center NCI SPORE grant in lymphoma. Dr. Bernstein was a member of the Lymphoma Working Group of SWOG, and the Scientific Advisory Boards of both the Lymphoma Research Foundation and the Leukemia and Lymphoma Society. He has a B.A. from Brandeis University and a M.D. from New York University School of Medicine.

Susan Abu-Absi, Ph.D. has over 20 years of experience in biologics and cell & gene therapy process development and manufacturing. She most recently served as our Senior Vice President and Head of Technical Development & Operations at 2seventy bio. Previously, Susan led the oncology manufacturing organization at bluebird bio, having joined in 2017 as Head of Process and Analytical development and advancing through roles of increasing responsibility during her tenure. Prior to joining bluebird, she spent more than ten years in the Global Product Development & Supply organization at Bristol Myers Squibb supporting clinical and commercial biologics drug substance manufacturing at internal sites and at contract manufacturers. Before BMS, Susan was a member of the Process Sciences organization at Bayer Healthcare in Berkeley, CA. Susan has led several teams developing the manufacturing processes, analytical methods and CMC packages for license applications and has directly contributed to the approval of seven products, including Abecma, Zynteglo, and Skysona. Susan earned a Ph.D. in Chemical Engineering from the University of Minnesota, where she was a National Science Foundation Fellow, and a B.S. in Chemical Engineering, Summa Cum Laude with Honors, from the University of Toledo (Toledo, OH).