On April 1, 2021 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, announced today that it has successfully completed the twelve-months product stability testing required by the U.S. Food and Drug Administration (FDA) for its CypCaps final clinical trial product (Press release, PharmaCyte Biotech, APR 1, 2021, View Source [SID1234577508]). This product will be used in the company’s planned clinical trial in locally advanced, inoperable pancreatic cancer upon the FDA lifting the clinical hold on PharmaCyte’s Investigational New Drug Application (IND).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the completed twelve-month stability study, "We are pleased that our CypCaps product successfully completed all of the required stability tests and that the product has now shown itself to be stable and active after being stored for 1 year at -80oC. The study will continue in order to determine the maximum shelf life of the CypCaps product. It should be noted that this data is exceptional for a product of this kind."

As reported previously, the ongoing stability study is designed to determine the shelf life of the Cell-in-a-Box encapsulated cell product, CypCaps, stored frozen at -80oC. Upon analysis after 12 months in storage at -80oC, the unfrozen CypCaps product passed all of the specified tests, including cell viability, enzyme activity and cell potency as well as pH, label check, capsule appearance, and integrity. This twelve-month data, as well as all future longer-term time points of the shelf life analyses, such as the next time point to be evaluated after 18-months of storage at -80oC, will be reported to the FDA. This ongoing stability study was initiated prior to the submission of the company’s IND to the FDA, and the information and data will form part of the package of information together with data from additional studies requested by the FDA.

In a survey of European biotech/biomedical companies developing advanced therapeutic medicinal products (i.e., products containing living cells like CypCaps), shelf life issues were reported to be one of the major challenges that such products face (ten Ham et al., 2018, Molecular Therapy: Methods & Clinical Development vol. 11, pp121-130). Laboratory data on non-GMP produced encapsulated cells have suggested that the shelf life of Cell-in-a-Box products stored at -80oC is even longer than 1 year.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On April 1, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it has received PRIority MEdicines (PRIME) designation from the European Medicines Agency (EMA) for AUTO1, the company’s CAR T cell therapy being investigated in the ongoing FELIX Phase 1b/2 study in relapsed / refractory (r/r) adult B-Acute Lymphocytic Leukemia (ALL) (Press release, Autolus, APR 1, 2021, View Source [SID1234577507]).

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"We are pleased to have received PRIME designation for AUTO1 as it will accelerate the review of a promising therapy targeting unmet medical need," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "The designation comes soon after we presented compelling activity and safety data from the ALLCAR Phase 1 clinical trial at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. We believe AUTO1 could change standard of care by offering a potentially curative therapy for r/r ALL."

About PRIME
The PRIME program aims to optimize development plans and speed up evaluation of medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. The PRIME designation is awarded by the EMA to promising medicines that target an unmet medical need. To be eligible and accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data coupled with non-clinical data. Through the PRIME program, the EMA offers enhanced support to medicine developers including early interaction and dialogue, and a pathway for accelerated evaluation by the agency. The program is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible.

On April 1, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage company focused on DNA-based immunotherapy and next-generation vaccines, reported that a poster highlighting the Company’s ongoing Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer was presented last week at the Virtual Annual Meeting on Women’s Cancer, sponsored by the Society of Gynecologic Oncology (Press release, Celsion, APR 1, 2021, View Source [SID1234577506]). The poster, titled "A Phase I/II Study Evaluating Intraperitoneal GEN-1 in Combination with Neoadjuvant Chemotherapy [NACT] in Patients with Newly Diagnosed Advanced Epithelial Ovarian Cancer (EOC)," can be viewed here.

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GEN-1 is Celsion’s DNA-mediated interleukin-12 (IL-12) immunotherapy designed using TheraPlas, its proprietary, synthetic, non-viral nanoparticle delivery system platform. The poster was presented by Premal Thaker, MD, Study Chair of the OVATION 2 Study and Professor of Obstetrics and Gynecology, Director of Gynecological Oncology Clinical Research, Division of Gynecologic Oncology, Washington University School of Medicine. Additional authors were R.W. Holloway, L. Kuroki, S. E. DePasquale, W.H. Bradley, A. ElNaggar, M.C. Bell, R.P. Rocconi, A. Bregar, M.D. Indermaur, C. Gunderson, B. Pothuri, R. Agajanian, D. Warshal, D. Provencher, M. McHale, V. John, M. Bergman, S. Lau, L. Musso, K. Anwer, N. Borys and C.A. Leath III.

"Currently advanced ovarian cancer has a low survival rate and a lack of effective therapies. PARP inhibitors have made an important contribution in a subset of patients. For the majority of patients there is hope that an immunotherapy such as GEN-1 will provide a valuable new treatment option to improve both the quality of life and the life expectancy for these women," commented Dr. Thaker. "OVATION 2 Study data now show R0 resections in 14 of 17 patients, or 82%, in the GEN-1 + NACT arm, compared with seven of 12 patients, or 58%, in the NACT alone arm. We view R0 resections as a good predictor of survival, and R0 resections in the GEN-1 + NACT arm are encouraging thus far," Dr. Thaker added.

The poster describes the OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

The Company recently announced that it has enrolled approximately 40% of the anticipated 110 patients to be enrolled into the OVATION 2 Study. To date, 29 patients have had their interval debulking surgery with the following results:

14 of 17, or 82%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed.
7 of 12 patients, or 58%, of patients in the control arm had an R0 resection.
This interim data represents a 41% improvement in R0 resection rates for GEN-1- patients compared with control arm patients and is consistent with the reported improvement in resection scores noted in the encouraging Phase I OVATION 1 Study, the manuscript of which has been submitted for publication in a peer-reviewed journal.
Celsion also recently announced that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration (FDA). This designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

About the Virtual Annual Meeting on Women’s Cancer

The Society of Gynecologic Oncology (SGO) 2021 Annual Meeting on Women’s Cancer will be a fully virtual meeting, allowing participants to access high-quality content and engage remotely from around the world in a platform that will be designed explicitly for this meeting. SGO is working hard to provide a great virtual experience with the latest gynecologic cancer research and education you have come to expect at the Annual Meeting on Women’s Cancer. All education sessions will be recorded and available through the virtual platform for attendees who register for the meeting by March 25, 2021.

On April 1, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that its corporate partner HanX Biopharmaceuticals has enrolled three patients in the second dosing cohort of its Phase 1 study with ON 123300 in HR+ HER2- metastatic breast cancer and other advanced relapsed/refractory cancers in China (Press release, Onconova, APR 1, 2021, View Source [SID1234577505]).

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The HanX trial has enrolled six patients to date in two cohorts and may continue to enroll patients with advanced relapsed/refractory cancer at increasing doses with three to six patients per dose until the recommended Phase 2 dose is identified. To date, patients have been dosed at the 40 mg and 80 mg dosage levels. HanX recently opened a third site, in Shanghai, for the conduct of the study.

"We are encouraged that the HanX Phase 1 study is proceeding as planned, and look forward to the identification of a recommended Phase 2 dose to move into later-stage trials. The third cohort in this trial with 120 mg of ON 123300 is expected to begin enrollment next; depending on the incidence of dose limiting toxicities, if any, at the 80 mg cohort," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova Therapeutics. "The HanX study trial design calls for dosing on days 1-21 of each 28-day cycle, compared with our planned Phase 1 study in the U.S. that will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally as monotherapy at increasing doses starting at 40 mg daily for continuous 28-day cycles. We are preparing to begin our U.S. study in the second quarter of 2021."

"ON 123300 is a multi-kinase inhibitor in addition to targeting CDK 4/6, which we believe presents an innovative approach to treating advanced cancers including HR+ HER2- metastatic breast cancer that is, or has become, resistant to commercial CDK 4/6 inhibitors. Beyond metastatic breast cancer, we believe ON 123300 may present an innovative approach to treating other cancers including mantle cell lymphoma, multiple myeloma, advanced colorectal cancer, hepatocellular carcinoma and inoperable glioblastoma," concluded Dr. Fruchtman.

In December 2017, Onconova entered into an agreement with HanX Biopharmaceuticals for the development, registration, and commercialization of ON 123300 in Greater China. The agreement included a licensing fee, future potential milestone payments, and royalties on sales. Onconova retains rights to ON 123300 in the rest of the world outside of Greater China.

On April 1, 2021 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform oncology drug discovery and development, reported a collaboration with Johns Hopkins Pediatric Oncology Division of The Sidney Kimmel Comprehensive Cancer Center and Dr. Eric Raabe, M.D., Ph.D. focused on Lantern’s drug candidate LP-184 in the area of brain tumors, and specifically in Atypical Teratoid Rhabdoid Tumors ("ATRT"), an ultra-rare and fast-growing cancerous tumor of the brain that presents primarily in children (Press release, Lantern Pharma, APR 1, 2021, View Source [SID1234577504]).

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"As we enriched our RADR A.I. platform for additional cancer indications, we began to discover common molecular pathways that drive response to our drug candidate, LP-184, across multiple additional CNS cancers," stated Panna Sharma, President and CEO of Lantern Pharma. "Chief among these newly identified CNS cancers was ATRT, an ultra-rare and fast-growing cancerous tumor of the brain that presents primarily in children with no effective therapies. The urgency of directing LP-184 towards helping children battle this particularly aggressive cancer was self-evident, as was the opportunity to collaborate with the Johns Hopkins’ pediatric oncologist, Dr. Eric Raabe, who has devoted his career to studying pediatric brain cancers, including ATRT."

Rhabdoid tumors (RTs) can emerge in the brain, kidneys, liver and all compartments of the central nervous system ("CNS"). Approximately 66% of RTs occur in the CNS and are called ATRTs. ATRTs predominantly affect infants and young children, with up to 15% of ATRTs arising in the brain. Incidence of ATRT is between 1.4 and 3.0 per million, and the survival rate is between 10% and 15% depending on the age at diagnosis. Pediatric brain cancer is the second-leading cause of pediatric cancer death with the incidence rate growing at ~2.7% per year in the United States.

Dr. Eric Raabe, M.D., Ph.D., is assistant professor of oncology in the Division of Pediatric Oncology at Johns Hopkins and a co-principal investigator at the Pacific Pediatric Neuro-Oncology Consortium. A physician-scientist, Dr. Raabe has devoted his career to the pursuit of treatment options for the most high-risk pediatric brain cancers, including ATRT where Dr. Raabe uses a unique and highly curated panel of cell lines and xenografts in preclinical studies for drug development and research. These models have had extensive molecular and genomic profiling including biomarker studies to help better understand the ATRT and other related CNS cancers.

Over 90% of cases of ATRT are caused by a mutation which drives a partial or whole loss of chromosome 22, resulting in the inactivation of the SMARCB1 gene (Switch/sucrose nonfermentable [SWI/SNF] related, Matrix-associated, Actin-dependent Regulator of Chromatin, subfamily B1). SMARCB1 is a protein encoding and tumor suppressor gene which drives downstream production of the SMARCB1 protein and other SWI/SNF protein subunits which are thought to act as tumor suppressors. While ATRT is diagnosed with standard immunochemistry staining to detect loss of the respective protein(s), no standard of care currently exists for ATRT and ATRT in the brain is typically unresectable. Treatment options are typically limited to only chemotherapy agents since radiotherapy is not advised in children.

"To support the discovery and development of innovative medicines that may help children diagnosed with rare diseases, the U.S. FDA has created a Rare Pediatric Disease Designation. We believe that the rarity of incidence of ATRT in the U.S and its prevalence in children supports the potential for LP-184 to qualify in the future for a possible grant by the US. FDA for a Rare Pediatric Disease Designation for use of LP-184 for ATRT," continued Mr. Sharma. "Moreover, if we are successful in receiving a Rare Pediatric Disease Designation, we believe LP-184, if it receives ultimate approval, may possibly qualify for the granting by the U.S. FDA of a Rare Pediatric Disease Priority Review Voucher ("PRV"). We believe the award of a PRV would represent a significant value enhancing milestone for Lantern Pharma."

Lantern Pharma plans on continuing to use RADR to potentially uncover and develop other indications in brain and CNS cancers where LP-184 has the potential to show efficacy.