On March 2, 2022 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that among the large and rapidly growing stem cell therapeutics sector, the Company’s cancer stem cell technology, PRP, offers renewed hope to achieve a total victory in the fight against cancer (Press release, Propanc, MAR 2, 2022, View Source [SID1234609365]). As stem cell therapeutics continue to evolve in a wide range of indications, from tissue regeneration, to even curing HIV, the discovery of cancer stem cells (CSCs) and their critical role in enabling solid tumors to spread and recur throughout the body, has led to companies looking to target these cells as a way to treat and prevent metastatic cancer.

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Cancer stem cells are resistant to standard treatments because they can lie dormant for long periods, then migrate to other organs and trigger explosive tumor growth, causing the patient to relapse. Eighty percent of cancers are from solid tumors and metastasis is the main cause of patient death. PRP is designed to target and eradicate cancer stem cells not killed by radiation or chemotherapy. By treating solid tumors with PRP, the tumor loses the ability to generate new cells and the tumor disappears, with no option to form a metastatic tumor elsewhere.

The Company’s scientific researchers have published data confirming that PRP regulates up to 4 relevant pathways, TGFβ, Hippo, Wnt, and Notch, related to cancer spread and metastasis of CSCs. That cascade of reactions disrupts CSC characteristics that leads to tumor invasion into surrounding tissues. PRP interferes with the signals that the primary tumor sends to other tissues to prepare the pre-metastatic niche.

"PRP is a targeted, CSC therapy for the treatment and prevention of metastatic cancer, which I believe can help achieve a total victory in the fight against metastatic cancer from solid tumors," said Dr Julian Kenyon, M.B.Ch.B., M.D., Propanc’s Chief Scientific Officer. "We have been working with cancer stem cells for several years and our preclinical studies demonstrate that PRP has a significant effect on suppressing these cells, which are the main drivers of cancers. I am impressed in the lab, looking down through the microscope at pancreatic CSCs, then adding PRP, and many disappear. That was really remarkable and is likely to put us ahead of the competition. This is highly significant because suppressing CSCs reduces the risk of tumor recurrence, clinically."

Mr. James Nathanielsz, Propanc’s Chief Executive Officer said, "Through scientific research, our discoveries provide evidence that our technology can control the spread of cancer. As a result of international borders reopening since the pandemic, we plan to visit our joint research partners in Spain, as well as undertake a road show in the US, advancing our company’s partnerships as we prepare for a First-In-Human study in advanced cancer patients."

On March 2, 2022 Verrica Pharmaceuticals Inc. (Verrica) (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, Verrica Pharmaceuticals, MAR 2, 2022, View Source [SID1234609381]).

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"2022 is poised to be an exciting year for Verrica as we prepare to potentially launch VP-102 this summer for the treatment of molluscum, a disease affecting an estimated six million patients with no approved treatments, representing a significant market opportunity," said Ted White, Verrica’s President and Chief Executive Officer. "In addition, in keeping with our mission to develop treatments for the most significant unmet needs in medical dermatology, we are rapidly advancing LTX-315, a novel immunotherapy for the treatment of non-melanoma skin cancers. The first patient is expected to be dosed in the Phase 2 trial evaluating LTX-315 in basal cell carcinoma in the first quarter of 2022."

Business Highlights and Recent Developments

VP-102

On November 29, 2021, Verrica announced that it resubmitted the New Drug Application (NDA) for VP-102 for the treatment of molluscum contagiosum (molluscum) to the U.S. Food and Drug Administration (FDA). On December 15, 2021, Verrica announced that the FDA acknowledged that Verrica’s resubmitted NDA was complete and assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 24, 2022.
LTX-315

Verrica expects to dose the first patient in the Phase 2 clinical trial of LTX-315 for the treatment of basal cell carcinoma in the first quarter of 2022. LTX-315 is a potentially first-in-class oncolytic peptide immunotherapy in development as a non-surgical treatment option for non-melanoma skin cancers. The Phase 2 trial is a three-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, pharmacokinetics, and efficacy of LTX-315 when administered intratumorally to adults with biopsy-proven basal cell carcinoma. The study is expected to enroll approximately 66 adult subjects with a histological diagnosis of basal cell carcinoma in at least one eligible target lesion.
Corporate Highlights

On March 1, 2022, Verrica amended its existing $40 million credit facility led by Silicon Valley Bank. The amendment provides Verrica increased financial flexibility by extending the interest-only payment period and reducing the minimum cash Verrica is required to maintain on its balance sheet.
Financial Results

Fourth Quarter 2021 Financial Results

Verrica reported a net loss of $9.5 million for the fourth quarter of 2021, compared to a $13.0 million loss for the same period in 2020.
Research and development expenses were $3.4 million in the fourth quarter of 2021, compared to $2.3 million for the same period in 2020. The increase was primarily attributable to higher Chemistry, Manufacturing, and Controls (CMC).
General and administrative expenses were $5.1 million in the fourth quarter of 2021, compared to $9.8 million for the same period in 2020. The decrease was primarily due to higher stock-based compensation expense recorded in December 2020, which includes $4.8 million of stock-based compensation expense related to the modification of a stock award to a former executive partially offset by an increase in expenses due to increased headcount, an increase in insurance, and other operating costs.
Full Year 2021 Financial Results

Verrica recognized license revenues of $12.0 million for the year ended December 31, 2021 related to the Collaboration and License Agreement (Torii Agreement) with Torii Pharmaceutical Col, Ltd (Torii). There were no license revenues recognized in 2020.
Research and development expenses were $15.9 million for the year ended December 31, 2021, compared to $15.7 million for the same period in 2020. The increase was primarily attributable to a one-time $2.3 million milestone payment to Lytix Biopharma AS upon the achievement of a regulatory milestone for LTX-315 and increased compensation costs related to higher headcount and increased clinical costs related to Verrica’s development of VP-102 for molluscum partially offset by decreased CMC costs related to Verrica’s development of VP-102 for molluscum contagiosum
General and administrative expenses were $27.0 million for the year ended December 31, 2021, compared to $24.5 million for the same period in 2020. The increase was primarily a result of expenses related to increased headcount, an increase in insurance, professional fees and other operating costs, and an increase in expenses related to pre-commercial activities for VP-102. The increase was partially offset by a decrease in stock-based compensation costs, which includes $4.8 million of stock-based compensation expense recorded in December 2020 related to the modification of a stock award to a former executive.
For the year ended December 31, 2021, net loss on a GAAP basis was $35.1 million, or $1.30 per share, compared to a net loss of $42.7 million, or $1.71 per share, for the same period in 2020.
For the year ended December 31, 2021, non-GAAP net loss was $27.6 million, or $1.02 per share, compared to a non-GAAP net loss of $31.9 million, or $1.28 per share, for the same period in 2020.
As of December 31, 2021, Verrica had aggregate cash, cash equivalents, and marketable securities of $70.4 million. Verrica believes that its existing cash, cash equivalents, and marketable securities as of December 31, 2021 will be sufficient to support planned operations into the third quarter of 2022.
Non-GAAP Financial Measures

In evaluating the operating performance of its business, Verrica’s management considers non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude stock-based compensation charges and non-cash interest expense that are required by GAAP. Verrica believes that non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share provides useful information to both management and investors by excluding the effect of certain non-cash expenses and items that Verrica believes may not be indicative of its operating performance, because either they are unusual and Verrica does not expect them to recur in the ordinary course of its business, or they are unrelated to the ongoing operation of the business in the ordinary course. Non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share should be considered in addition to results prepared in accordance with GAAP, but should not be considered a substitute for, or superior to, GAAP results. Non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share have been reconciled to the nearest GAAP measure in the tables following the financial statements in this press release.

About VP-102

Verrica’s lead product candidate, VP-102, is a proprietary drug-device combination product that contains a GMP-controlled formulation of cantharidin (0.7% w/v) delivered via a single-use applicator that allows for precise topical dosing and targeted administration. VP-102 is currently under U.S. Food and Drug Administration (FDA) review and could potentially be the first product approved by the FDA to treat molluscum contagiosum — a common, highly contagious skin disease that affects an estimated six million people in the United States, primarily children. If approved, VP-102 will be marketed in the United States under the conditionally accepted brand name YCANTH. In addition, Verrica has successfully completed a Phase 2 study of VP-102 for the treatment of common warts and a Phase 2 study of VP-102 for the treatment of external genital warts.

About Molluscum Contagiosum (Molluscum)

There are currently no FDA-approved treatments for molluscum, a highly contagious viral skin disease that affects approximately six million people — primarily children — in the United States. Molluscum is caused by a pox virus that produces distinctive raised, skin-toned-to-pink-colored lesions that can cause pain, inflammation, itching and bacterial infection. It is easily transmitted through direct skin-to-skin contact or through fomites (objects that carry the disease like toys, towels or wet surfaces) and can spread to other parts of the body or to other people, including siblings. The lesions can be found on most areas of the body and may carry substantial social stigma. Without treatment, molluscum can last for an average of 13 months, and in some cases, up to several years.

On March 2, 2022 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported that Noreen Roth Henig, M.D., Chief Medical Officer, will participate on the Kidney Disease/Fibrosis Panel at the Cowen 42nd Annual Health Care Conference (Press release, Kezar Life Sciences, MAR 2, 2022, View Source [SID1234609397]). The panel will be held on Wednesday, March 9, 2022, at 2:10 p.m. ET.

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A live webcast of the panel will be available on the "Events & Presentations" section of the Company’s website at View Source A replay will be available on the Kezar website for 90 days following the event.

On March 2, 2022 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LBT4) systems are implicated, reported the appointment of Rachelle Jacques as President and Chief Executive Officer of Akari Therapeutics (Press release, Akari Therapeutics, MAR 2, 2022, View Source [SID1234609415]). Ms. Jacques will also join the company’s Board of Directors. Her appointment begins at the end of March 2022.

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Outgoing Chief Executive Officer, Clive Richardson, will continue to serve the Company, supporting Rachelle to accelerate business development and ensuring a smooth transition.

"I am very pleased to be able to welcome Rachelle as CEO of Akari Therapeutics. She has considerable biotechnology experience including a proven track record of bringing advanced therapies for rare diseases to the market," said Ray Prudo, M.D., Akari Therapeutics’ founder and Chairman of the company’s Board of Directors. "With her combined experience in C5 inhibition, immunology and rare diseases, Rachelle has a deep understanding of the potential of Akari’s pipeline. This, together with her strategic experience, affords a springboard to advance our Phase 3 pipeline through potential regulatory approval and towards commercialization, as well as and bring forward our earlier stage pipeline. I very much look forward to working with Rachelle who will be based in the Boston area."

"It’s an exciting time at Akari and I look forward to building on the robust research and development work that has already been completed to advance nomacopan, a unique drug differentiated by its dual mode of action which has the potential to deliver benefits beyond standard complement inhibition," stated Ms. Jacques. "Our pipeline, including two late-stage pivotal programs, has tremendous potential for patients with significant unmet needs and we are working with urgency to realize that promise for them."

Before this appointment, Ms. Jacques served as Chief Executive Officer of Enzyvant Therapeutics Inc., a commercial-stage biotechnology company developing transformative regenerative therapies for rare diseases. Prior to Enzyvant, she served as the Senior Vice President and Global Complement Franchise Head at Alexion Pharmaceuticals, Inc., where she was responsible for global franchise strategy development and execution of the C5 complement inhibitors, eculizumab and ravulizumab, across the therapeutic areas of hematology, nephrology and neurology. She was Vice President of U.S. Hematology Marketing at Baxalta Inc. and then Shire plc, following Shire’s acquisition of Baxalta in 2016. At Baxalta, she served as Vice President of Business Operations after its spinoff from Baxter International Inc. Ms. Jacques held multiple leadership positions at Baxter, including Vice President of Finance, U.S. BioScience Business. Earlier in her career, Ms. Jacques served in various roles at Dow Corning Corporation, including operational management positions in the U.S., Europe, and China.

Ms. Jacques serves on the boards of directors of uniQure N.V. (Nasdaq: QURE) and Corbus Pharmaceuticals (Nasdaq: CRBP). She is co-chair of the Alliance for Regenerative Medicine (ARM) Tissue Engineering & Biomaterials Committee and is a founding member of the ARM Action for Equality Task Force. Ms. Jacques received her B.A. in business administration from Alma College.

On March 2, 2022 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported study results that further demonstrate the potential for use of memory-like cytokine-enriched natural killer (m-ceNK) cells for the treatment of cancer (Press release, ImmunityBio, MAR 2, 2022, View Source [SID1234609366]). ImmunityBio has successfully scaled the process of generating over 20 billion m-ceNK cells from a single extraction of white blood cells from a donor or patient, and reports promising clinical data supporting the ongoing development of memory NK cells for the treatment of both liquid and solid tumors of all types.

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In a Phase 2 study sponsored by Washington University School of Medicine (NCT02782546), white blood cells retrieved from HCT donors to treat acute myeloid leukemia (AML) patients, were stimulated with cytokines (IL-12, -15, and -18) to form memory-like NK cells, and were re-infused to the patients, who also received ImmunityBio’s IL-15 superagonist Anktiva (N-803). The cytokine-stimulated NK cells safely augmented a leading treatment for AML-—HLA-haploidentical hematopoietic cell transplantation. The data showed that the cytokine-stimulated NK cells were persistent, showed increased production of interferon-gamma and functionality, and led to a complete response in 87% of patients after Day 28. The results were recently published in a peer-reviewed article ("Hematopoietic cell transplantation donor-derived memory-like NK cells functionally persist after transfer into patients with leukemia") in Science Translational Medicine.

NK cells are innate immune cells that are under investigation as a cell therapy for multiple types of cancer. A challenge of NK cell-based therapies, however, is ensuring that the cells persist and remain functional long-term. This study showed that memory-like NK cells derived from the same donor as the hematopoietic cell transplantation (HCT) persisted for at least two months after transplantation and were highly functional ex vivo. Together, these findings support the use of memory NK cells as a component of HCT for leukemia.

"This important study adds to the growing body of data that shows the cancer-fighting ability of NK cells can be enhanced by stimulating expansion and persistence of these cells," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "The study also showed that stimulated memory NK cells are effective with only a single infusion when given along with Anktiva support. To facilitate this approach, ImmunityBio has successfully scaled cytokine enrichment and expansion of m-ceNK cells, so cancer patients can receive multiple infusions of these potent and persistent m-ceNK cells generated from a single donation of white blood cells."

About Memory Cytokine-Enhanced NK Cells (m-ceNK):
ImmunityBio has successfully enriched and expanded NK cells obtained from peripheral blood of donors using a technique called apheresis to generate NK cells with a memory-like phenotype, which exhibit both high cytotoxicity and increased interferon-gamma production. These m-ceNK cells can be generated from an individual donor for autologous cell therapy, but have also been generated as an allogeneic product from cord blood. In addition to the high potential for enhanced efficacy, m-ceNK cells can be infused easily in an outpatient setting.

ImmunityBio has developed a novel method of m-ceNK cells production that yields multiple clinical-dose forms from a single apheresis (white blood cell collection) using the company’s proprietary NANT 001 Bioreactor (GMP-in-a-Box), thereby alleviating pressures on supply of starting material. An optimized cryopreservation protocol for maximum shelf-life and potency upon recovery was also established, a necessity for any off-the-shelf product. ImmunityBio is leveraging their ability to generate m-ceNK cells with potent cytotoxicity, increased IFN-gamma production, proliferative capacity, activation surface markers and memory response to establish a propriety method for generation, expansion, and cryopreservation of these cells for autologous use.

The QUILT 3.076 Study of Cryopreserved M-ceNK cells
Cryopreserved m-ceNK cells in combination with Anktiva (N-803) will be tested in a 2-part Phase 1 study (NCT04898543) designed to evaluate safety in subjects with locally advanced or metastatic solid tumors; solid tumors comprise approximately 90% of adult cancers and 40% of all cancers in children, according to the American Cancer Society. The study will compare the quantity and characteristics of m-ceNK cells collected and cytokine-enriched from newly diagnosed patients who have not received prior treatment to m-ceNK cells generated from patients who have received at least two prior treatments for their cancer.

The study consists of two cohorts and there will be 10 participants in each cohort. Cohort 1 includes participants with newly-diagnosed, high-risk solid tumors who have not received prior treatment; and cohort 2 includes participants with relapsed/refractory (r/r) solid tumors who have progressive disease after receiving ≥ 2 prior therapies. Participants will be enrolled in the two cohorts simultaneously.

Participants in Cohort 1 will participate in apheresis collection of lymphocytes (part A) and will not receive any investigational therapy in this study.
Participants in Cohort 2 will undergo an apheresis collection of lymphocytes (part A) prior to receiving 4 infusions of M-ceNK on days 1, 8, 15 and 22 along with N-803 on days 1 and 15.