On April 19, 2022 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing first-in-class allogeneic gamma delta chimeric antigen receptor (CAR) T cell therapies for cancer, reported the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead program ADI-001, an investigational therapy targeting CD20 for the potential treatment of relapsed or refractory B-cell Non-Hodgkin’s lymphoma (NHL) (Press release, Adicet Bio, APR 19, 2022, View Source [SID1234612507]).

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ADI-001 is currently being evaluated in an ongoing dose escalation Phase 1 study evaluating the safety and tolerability of ADI-001 for the potential treatment of NHL. The Fast Track Designation was granted based on ADI-001’s potential to address an unmet need within the adult NHL patient population.

"Fast Track Designation represents an important milestone in the clinical development of ADI-001," said Chen Schor, President and Chief Executive Officer of Adicet Bio. "We believe ADI-001 is unique in that it is designed to target malignant B cells by leveraging the innate and adaptive receptors found naturally on gamma delta T cells with the added benefit of an engineered anti CD20 CAR. We remain optimistic about the potential of our program and look forward to reporting additional data from the Phase 1 trial of ADI-001 in the first half of 2022."

Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need.

On April 19, 2022 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm,’ reported a peer-reviewed publication in ClinicoEconomics and Outcomes Research outlining the potential clinical and health economic benefits of lenzilumab, if authorized or approved for use in the United Kingdom (Press release, Humanigen, APR 19, 2022, View Source [SID1234612506]).

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"As COVID-19 continues to place significant burden on the National Health Service ("NHS"), this paper demonstrates there is an opportunity to realize significant cost savings for healthcare systems of the UK while improving outcomes for patients. As a variant-agnostic treatment, lenzilumab may offer both a clinically effective and cost-effective option against current and emerging variants," said Adrian Kilcoyne, M.D., Chief Medical Officer, Humanigen, the lead author of the publication.

The publication demonstrated, in all cases, lenzilumab plus SOC improved all specified clinical outcomes compared with SOC alone. Additionally, patient selection, utilizing CRP<150 mg/L as a biomarker, optimized both clinical and economic outcomes. The observed cost savings are mainly driven by fewer bed days, days on invasive mechanical ventilation and ICU days.

The greatest per-patient cost savings were for patients aged <85 years, CRP <150 mg/L, and receiving remdesivir of £10,427 (net savings of £3,127 after expected lenzilumab acquisition costs); and for Black patients with CRP <150 mg/L of £17,277 (net savings of £9,977).

"During these unprecedented and challenging times, we are preparing to commercialize lenzilumab, if authorized or approved, as a single day treatment and a potential driver of clinical and economic value to patients and the healthcare system," said Edward Jordan, Chief Commercial Officer, Humanigen.

This peer-reviewed publication highlights the significant costs of treating hospitalized COVID-19 patients and the economic benefits of potentially improving survival without ventilation, reducing ventilator use, hospital days and ICU days which may be associated with adding lenzilumab to standard of care.

Lenzilumab is an investigational product and is not approved or authorized in any country.

About Lenzilumab

Lenzilumab is a proprietary Humaneered first-in-class monoclonal antibody that has been proven to neutralize GM-CSF, a cytokine of critical importance in the hyperinflammatory cascade, sometimes referred to as cytokine release syndrome, or cytokine storm, associated with COVID-19 and other indications. Lenzilumab binds to and neutralizes GM-CSF, potentially improving outcomes for patients hospitalized with COVID-19. Humanigen believes that GM-CSF neutralization with lenzilumab also has the potential to reduce the hyper-inflammatory cascade known as cytokine release syndrome common to chimeric antigen receptor T-cell (CAR-T) therapy and acute Graft versus Host Disease (aGvHD).

In CAR-T, lenzilumab successfully achieved the pre-specified primary endpoint at the recommended dose in a Phase 1b study with Yescarta in which the overall response rate was 100% and no patient experienced severe cytokine release syndrome or severe neurotoxicity. Based on these results, Humanigen plans to test lenzilumab in a randomized, multicenter, potentially registrational, Phase 3 study ("SHIELD") to evaluate its efficacy and safety when combined with Yescarta and Tecartus CAR-T therapies in non-Hodgkin lymphoma. Lenzilumab will also be tested to assess its ability to prevent and/or treat aGvHD in patients undergoing allogeneic hematopoietic stem cell transplantation.

A study of lenzilumab is also underway for patients with chronic myelomonocytic leukemia (CMML) exhibiting RAS pathway mutations. This study builds on evidence from a Phase 1 study, conducted by Humanigen, that showed RAS mutations are associated with hyper-proliferative features, which may be sensitive to GM-CSF neutralization.

On April 19, 2022 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported that dosing of the first patient in the Company’s streamlined, multi-cohort Phase 1/2 study of oral CYC140 in patients with advanced solid tumors and lymphomas (Press release, Cyclacel, APR 19, 2022, View Source [SID1234612505]).

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"With the start of this trial, Cyclacel is now enrolling patients across three registration-directed studies to evaluate safety and efficacy of our two lead drug candidates, fadraciclib and CYC140, for the treatment of various solid tumors, lymphomas and leukemias," said Spiro Rombotis, Cyclacel’s President and Chief Executive Officer. "As with the fadraciclib studies, the Phase 1 stage of this trial will determine an optimal dosing regimen of oral CYC140 and provide insights with respect to safety, tolerability and clinical activity. The Phase 2 stage will enroll up to seven cohorts by histology and also a basket cohort. We expect initial data from this trial during the first half of 2023. We also look forward to reporting initial data from the fadraciclib clinical study in advanced solid tumors during the first half of 2022."

"Overexpression of PLK1 is known to be important in many types of cancer," said Mark Kirschbaum M.D., Senior Vice President and Chief Medical Officer. "We have optimized the properties of CYC140 to fit its apoptosis-driven mechanism, including a short half-life and differentiated structural and biological properties, compared to other PLK1 inhibitors in development. In preclinical studies CYC140 has demonstrated promising activity in multiple solid tumors and leukemias. The study has initially opened at City of Hope and MD Anderson Cancer Center with more sites to join later on. In addition to patients with certain PLK1 over-expressing tumors, the study will enroll patients with MYC amplified and KRAS-mutated cancers in which PLK1 inhibition may be effective. If successful, CYC140 may provide new treatment options for patients with advanced solid tumors or lymphomas."

"As a key regulator of cell mitosis, PLK1 plays an integral role in prolonged survival of many cancer cells, including p53(-) and KRAS mutant genotypes," said Miguel Villalona-Calero, M.D., co-leader of the Development Cancer Therapeutics Program and Professor, Department of Medical Oncology & Therapeutics Research at City of Hope, one of the largest cancer research and treatment organizations in the United States. "The totality of preclinical evidence suggests that CYC140 has significant potency and single-agent activity. This novel agent warrants clinical investigation across multiple solid tumors and lymphomas."

The Phase 1/2 registration-directed trial, designated CYC140-101, uses a streamlined design and will first determine in a dose escalation stage the recommended Phase 2 dose (RP2D) for single-agent CYC140. Once RP2D has been established, the trial will immediately enter into proof-of-concept, cohort stage, using a Simon 2-stage design. In this stage CYC140 will be administered to patients in up to 7 mechanistically-relevant cohorts including patients with bladder, breast, colorectal (including KRAS mutant), hepatocellular and biliary tract, and lung cancers (both small cell and non-small cell), as well as lymphomas. An additional basket cohort will enroll patients with biomarkers relevant to the drug’s mechanism, including MYC amplified tumors. The protocol allows for expansion of individual cohorts based on response which may allow acceleration of the clinical development and registration plan for CYC140.

About Polo-like kinase 1 (PLK1) and CYC140

Polo-like kinase 1 (PLK1) is a serine/threonine kinase that plays a central role in cell division or mitosis. PLK1 is an important regulator of the DNA damage cell cycle checkpoint, mitotic entry and exit, spindle formation and cytokinesis, or cell separation into daughter cells. Cancer cells in general, and in particular KRAS mutated and p53(-) cells, are very sensitive to PLK1 depletion. In contrast normal cells with intact cell cycle checkpoints are less sensitive. Pharmacological inhibition of PLK1 in cancer cells blocks proliferation by prolonged mitotic arrest and induces onset of apoptotic death of such cells.

CYC140 is a novel, small molecule, selective and potent PLK1 inhibitor. It has demonstrated impressive efficacy in human tumor xenografts at nontoxic doses. Cyclacel’s translational biology program supports the development of CYC140 in solid tumors and leukemias. CYC140 was designed with improved pharmaceutical properties over earlier clinical-stage PLK inhibitors. Recent data suggest that PLK1 inhibition may be effective in KRAS-mutated metastatic colorectal cancer. PLK1 overexpression correlates with poor patient prognosis in several tumors, including esophageal, gastric, leukemia, lung, ovarian, and squamous cell cancers, as well as MYC-amplified cancers.

On April 19, 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 that the empty capsule material that comprises its pancreatic cancer clinical trial product candidate does not cause systemic toxicity (Press release, PharmaCyte Biotech, APR 19, 2022, View Source [SID1234612504]).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "Another important study has been conducted and concluded. This time the study evaluated the potential toxicity of the capsule component of our CypCaps clinical trial product candidate. We are pleased to announce that there was no evidence of toxicity in this animal study in any of the parameters examined and that the study confirms previous data that the capsule material is inert."

The study, which was performed by a third-party Contract Research Organization, involved the ISO compliant testing of an extract of empty cellulose sulphate capsules provided by Austrianova for potential acute systemic toxicity in mice, according to ISO 10993-11: 2017(E), an FDA recognized consensus standard. Upon intraperitoneal injection of capsule extract, the mice were observed for clinical signs of toxicity at 30 to 40 min, 1 hour, 2 hours and 4 hours post dosing on day 1. On days 2, 3, 7 and 14, all of the animals were observed once daily for clinical signs and twice daily for mortality.

The body weight of the mice was recorded prior to administration of the capsule extract on day 1 and on days 2, 3, 4, 7 and 14 during the observation period. At the end of observation period, all the animals were examined for signs of toxicity. The analyses revealed that none of the mice died or showed any clinical signs of toxicity or gross pathological changes as compared to control mice. Moreover, no treatment related changes were noted in body weight and percent change in body weight with respect to day 1 values and all animals revealed a normal increase in body weight during the observation period.

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 19, 2022 Kite, a Gilead Company (Nasdaq: GILD), reported the U.S. Food and Drug Administration (FDA) has approved commercial production at the company’s new CAR T-cell therapy manufacturing facility in Frederick, Maryland (Press release, Kite Pharma, APR 19, 2022, View Source [SID1234612503]). The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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The Maryland site joins Kite’s existing manufacturing facilities in Southern California and Amsterdam, Netherlands to form the largest, dedicated in-house cell therapy manufacturing network in the world, spanning process development, vector manufacturing, clinical trial production and commercial product manufacturing. With today’s announcement, and through a variety of optimization efforts across Kite’s global CAR T-cell therapy manufacturing network, Kite estimates network capacity will be increased by 50%, enabling more patients to be served now and in the future.

"The FDA approval of our Maryland site marks an important milestone within our global CAR T-cell therapy manufacturing network, and will enable us to significantly expand our production capacity and further strengthen our ability to meet the needs of people living with difficult-to-treat blood cancers," said Christi Shaw, Chief Executive Officer of Kite. "Manufacturing is central to every decision we make at Kite. Our teams hold patients’ cells in their hands every day, which could mean the difference between getting a chance to live or possibly losing their battle with cancer. It must be fast and extremely high quality to give patients the best outcome."

CAR T-cell therapies are individually manufactured for each patient using their own T-cells extracted from their white blood cells. The patient’s T-cells are sent to Kite’s manufacturing facilities where they are modified with a Chimeric Antigen Receptor (CAR) to recognize, attack and destroy their cancer cells. Once the individualized therapy is created for a patient, the cells are carefully preserved, packed and sent back to the hospital to be infused back into the patient. Unlike most cancer treatments, CAR T-cell therapy is a one-time treatment, available through authorized treatment centers (ATCs), or hospitals, that have experience with CAR T-cell therapy. Kite therapies are available at over 275 ATCs around the world, including more than 110 leading cancer hospitals in the U.S.

Kite began construction of the 275,000 square foot facility in Maryland on 20 acres in 2019. The site has unfinished space to add future capacity, which will allow Kite to accommodate potential new scientific and technological advances in the field of cell therapy. The new Maryland facility is purpose-built for cell therapy, incorporating learnings from across the Kite manufacturing network as well as incorporating automation for some previously manual processes, allowing more therapies to be produced to meet growing patient demand.

"As the global leader in cell therapy, it is important to keep innovating as manufacturing in cell therapy is unlike anything else in the biologics or pharmaceutical space – each production run is made to order for one patient – and we do it at scale, with a 96% success rate in returning our specialized treatments on time and to specification," said Charles Calderaro, Kite’s Global Head of Technical Operations. "Manufacturing quality, reliability, and speed are critically important to us as we know patients are waiting. Our median cycle time is industry leading at 16 days in the U.S., from apheresis to finished product, which includes many processes that are heavily regulated for safety."

The company is invested in the future of the cell therapy workforce and anticipates having more than 400 employees working at the Maryland site by the end of 2022. The site is also committed to training and developing the region’s cell therapy talent through partnerships with local academic institutions as well as community and government organizations.

Kite’s manufacturing and process development expertise in cell therapy make it a partner of choice, and this expertise is a key component of partnership pipeline deals for the next generation of cell therapies, including previously announced deals with Appia Bio and Shoreline Biosciences.