FLARE THERAPEUTICS APPOINTS AMIT RAKHIT, M.D. AS PRESIDENT AND CHIEF EXECUTIVE OFFICER

July 25, 2022 Flare Therapeutics, a biotechnology company targeting transcription factors to discover precision medicines for cancer and other diseases, reported the appointment of Amit Rakhit, M.D., MBA, as President and Chief Executive Officer (Press release, , AUG 25, 2022, View Source [SID1234618750]). Dr. Rakhit has over 20 years of life sciences leadership experience and joins at a pivotal time as Flare transitions into a clinical-stage company. He succeeds interim CEO Abbie Celniker, Ph.D., a partner at Third Rock Ventures, who will continue to serve as Chair of the Company’s Board of Directors.

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"Flare’s pioneering approach to drugging transcription factors stands to profoundly impact medical practice globally for people living with cancer and other difficult to treat diseases. I am excited to join such a dynamic and experienced leadership team at this important inflection point for the company," said Dr. Rakhit. "The team’s swift progress in identifying dozens of novel druggable pockets, or ‘switch sites’, within therapeutic transcription factor targets of interest and rapid advancement of the lead precision oncology program both inspired my belief in Flare and compelled me to join to help catapult the company into its next stage of growth."

"Dr. Rakhit’s experience includes a rare mix of business acumen, clinical development expertise and passion for leading innovation in new treatment modalities that can benefit patients," said Dr. Celniker. "His leadership and experience will be extremely valuable in driving the strategy forward as we transition our first precision oncology program into the clinic and grow our pipeline of discovery targets in multiple new therapeutic indications."

For more than two decades, Dr. Rakhit has served in key leadership, clinical and operational roles in biotechnology and pharmaceutical companies spanning diverse therapeutic areas including rare disease, neurology, oncology and cardiology. Most recently, he has focused on launching and growing start-up biotechnology companies, overseeing clinical development and commercialization strategy in roles as CEO of Sporos BioVentures and President and Chief Medical Officer of Ovid Therapeutics. Earlier in his career, he was Senior Vice President and Head of Worldwide Medical Affairs at Biogen, Inc. where he led global external collaborations with key stakeholders and executed post-marketing development strategies for the launch and commercialization of key products in rare disease, including Spinraza, and in neurology. Prior to Biogen, he held multiple roles of increasing seniority in global clinical development and medical affairs at Bristol-Myers Squibb, including spearheading global initiatives for Plavix. Dr. Rakhit holds an M.D. from Tufts University School of Medicine and dual MBAs from Columbia University and London Business School. He completed his fellowship in pediatric cardiology at Harvard Medical School and was previously on staff at Boston Children’s Hospital.

About Flare Therapeutics
Flare Therapeutics is a biotechnology company pioneering a new therapeutic space with a novel approach to decipher the biology of transcription factors to develop small molecule medicines. Based on insights from the seminal work of its scientific founders, Flare’s team has uncovered ‘switch sites,’ druggable regions that are key targets for transcription factor regulation, to address mutations that cause disease. Flare’s drug discovery efforts to target switch sites has rapidly advanced, resulting in an emerging pipeline of drug programs that address well-validated transcription factors, initially focused on precision oncology with future potential in neurology, rare genetic disorders, immunology and inflammation. Flare Therapeutics was launched in 2021 and is backed by founding investor Third Rock Ventures, as well as Boxer Capital, Nextech Invest, Casdin Capital, Invus Financial Advisors and Eventide Asset Management. For more information, please visit www.flaretx.com.

Iovance Biotherapeutics Initiates Biologics License Application (BLA) Submission for Lifileucel in Advanced Melanoma

On August 25, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, reported that it has initiated a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for lifileucel, a tumor infiltrating lymphocyte (TIL) therapy, in patients with advanced (unresectable or metastatic) melanoma who progressed on or after prior anti-PD-1/L1 therapy, and if BRAF mutation positive, also prior BRAF or BRAF/MEK inhibitor therapy (Press release, Iovance Biotherapeutics, AUG 25, 2022, View Source [SID1234618645]). There are no FDA approved therapies in this treatment setting.

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Initiating our rolling BLA submission for lifileucel is a significant step towards our goal to deliver the first individualized, one-time cell therapy for melanoma patients with significant unmet need. In parallel, we are executing our on-boarding and personnel training at authorized treatment centers, education and awareness initiatives, internal capacity planning, and launch readiness activities to prepare for commercialization. The FDA is supportive of our regulatory approach, and we look forward to continuing this collaboration throughout the submission and review process."

A rolling BLA allows Iovance to submit portions of the BLA to the FDA on an ongoing basis, which enables the FDA to begin review as early as possible while documents are received. Iovance expects to complete the BLA submission in the fourth quarter of 2022. The rolling BLA submission and eligibility for priority review are benefits available under the FDA’s guidance on expedited programs for serious conditions. The FDA previously granted a regenerative medicine advanced therapy (RMAT) designation for lifileucel in advanced melanoma.

"Lifileucel represents hope and a new treatment for thousands of people with advanced melanoma who have very limited options after they progress on available standard of care," said Kyleigh LiPira, CEO, Melanoma Research Foundation. "Cell immunotherapies are revolutionizing cancer treatment, and we are excited about the potential for the first FDA-approved TIL cell therapy for the treatment of melanoma, which helps us take another step towards finding a cure."

The BLA submission for lifileucel is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced melanoma. Iovance plans to present additional results from the C-144-01 trial at a medical meeting later this year.

Regen BioPharma Develops Novel Means of Using CAR-T cell Immunotherapy to Attack Solid Tumors

On August 25, 2022 Regen BioPharma, Inc. (OTC PINK: RGBP) and (OTC PINK: RGBPP) reported development of a novel immunotherapy for treatment of solid tumors (Press release, Regen BioPharma, AUG 25, 2022, View Source [SID1234618684]). Current cellular approaches to kill cancer involve administration of a specific type of modified T cell called "chimeric antigen receptor" (CAR) T cell . Despite significant progress being made in using CAR-T cells to treat leukemia, little progress has been made in solid tumors such as those found in lung, brain, breast, prostate and colon. It is believed that solid tumors possess an abnormal "microenvironment" which physically prevents T cells from entering the tumor, as well as inactivating T cells that do manage to enter. The microenvironment consists of surrounding blood vessels, immune cells, fibroblasts, signaling molecules and an extracellular matrix. The solid tumor is surrounded by its microenvironment and constantly interacts with it.

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The current approach developed by the Company, for which a provisional patent application has been submitted, involves initial treatment of the tumor microenvironment with cells of the innate immune system called CAR-M and CAR-NK cells. These cells, which also begin to attack the tumor, are able to "normalize" the tumor microenvironment, thus allowing CAR-T cells to enter and attack the tumor.

"The company believes that taking this two-step approach – first softening up the target with CAR-M and CAR-NK cells and then bringing in the heavy artillery (CAR-T cells) will allow solid tumors to be successfully targeted by these cell therapies, thus greatly expanding the CAR-T market and ultimately saving people’s lives," said Dr. David Koos, CEO and Chairman of the Company.

Alpha Tau Medical Announces Second Quarter 2022 Financial Results and Provides Corporate Update

On August 25, 2022 Alpha Tau Medical Ltd. (Nasdaq: DRTS and DRTSW), ("Alpha Tau" or the "Company"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported second quarter 2022 financial results and provided a corporate update (Press release, Alpha Tau Medical, AUG 25, 2022, View Source [SID1234618700]).

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"We have made significant progress by gaining conditional FDA approval, whose conditions have since been satisfied, for our pivotal trial IDE in SCC as well as treating our first patient in a prostate cancer feasibility trial. We’ve seen overwhelming interest from leading clinical sites across the U.S. in participating in our upcoming pivotal trial in SCC, and look forward to initiating that trial shortly. While making clinical progress, we are thoughtfully expanding our pipeline from superficial cancers that only require a temporary Alpha DaRT implant, to more invasive internal tumors like liver and prostate, and then finally to ones such as pancreatic cancer and GBM with the greatest unmet need. We’ve made great strides in GBM in large animal studies, on the back of receipt of Breakthrough Device Designation for recurrent GBM from the FDA," said Alpha Tau CEO Uzi Sofer. "In tandem, we have been preparing for global commercialization efforts by expanding our manufacturing capabilities alongside our expanded radioactive licenses in Israel and in the U.S., as well as educating the medical community on our technology via hands-on experience in our trials."

Recent Corporate Highlights:

Approval by the U.S. Food and Drug Administration (FDA) for an Investigational Device Exemption (IDE) application to initiate our multi-center pivotal study for the treatment of recurrent cutaneous Squamous Cell Carcinoma (SCC) using the Alpha DaRT. The clinical study has been approved to enroll up to 86 patients at up to 20 institutions in the U.S., in addition to any sites that may be added outside the U.S., and will focus on patients with recurrent cutaneous SCC who have failed at least first line standard of care therapy and are not indicated for another curative standard of care therapy.

First patient treated in a feasibility study evaluating the Alpha DaRT as a neoadjuvant therapy in patients with prostate cancer. This is the Company’s first patient treated with Alpha DaRT in an internal organ.

Alpha Tau was granted an active radioactive license for use and possession of Thorium-228 and Radium-224, radioactive materials which are utilized in the production of the Alpha DaRT sources, in the main manufacturing floor of the Company’s production facility in Jerusalem by the Israeli Ministry of Environmental Protection. This license should enable the Company to achieve a nearly 3x increase in production capacity, to yield approximately 90,000 Alpha DaRT sources per year.

First patients treated with Alpha DaRT in a French multicenter trial for skin cancer, which is currently being conducted at six cancer centers in France, on patients with malignant cutaneous lesions (including SCC, BCC, lentigo maligna melanoma, and carcinosarcoma), and is evaluating two cohorts: (1) newly diagnosed patients (up to 49 subjects), and (2) patients with locally recurrent disease (36 subjects). The primary effectiveness endpoint is the assessment of the overall response rate using RECIST criteria, 9 to 11 weeks after Alpha DaRT source insertion. Dr. Pascal Pommier is the principal investigator of this study.

Held an investor KOL event featuring Professor Michael Zelefsky and Dr. Mark D’Andrea to review clinical data and user experience with Alpha DaRT and to preview potential new indications for the technology. The event was held in New York on July 18, 2022 and a replay is available here and on the Alpha Tau website in the Investors section.
Upcoming Anticipated 2022 Milestones

Expecting first patient treated in U.S. multi-center pivotal trial in recurrent cutaneous SCC in the second half of 2022.
Targeting recruitment in the Canadian feasibility trial in pancreatic tumors to begin in the fourth quarter of 2022.
Planned submission of Alpha DaRT pivotal trial results in head and neck SCC to Japan’s regulatory authority, PMDA, in the coming months for marketing approval.
Targeting Health Canada approval for initiation of liver cancer feasibility trial by the end of 2022.
Financial results for the second quarter ended June 30, 2022

R&D expenses for the quarter ended June 30, 2022 were $5.4 million, compared to $3.0 million for the same period in 2021, primarily due to increased R&D headcount, costs associated with our U.S. multi-center pivotal study and other clinical studies, and increased share-based compensation costs.

Marketing expenses for the quarter ended June 30, 2022 were $0.1 million, compared to $0.1 million for the same period in 2021.

G&A expenses for the quarter ended June 30, 2022 were $2.4 million, compared to $0.4 million for the same period in 2021, primarily due to increased professional fees (including D&O insurance), share-based compensation, and costs (including bonuses) associated with our financing transaction in the first quarter of 2022.

Financial income, net, for the quarter ended June 30, 2022 was $6.0 million, compared to financial expense, net of $3.5 million for the same period in 2021, primarily due to the remeasurement of warrants.

For the quarter ended June 30, 2022, the Company had a net loss of $2.0 million, or ($0.03) per share, compared to a loss of $7.0 million, or ($0.17) per share, in the same period in 2021.

Balance Sheet Highlights

As of June 30, 2022, the Company had cash and cash equivalents, restricted cash and short term deposits in the amount of $112.8 million, compared to $31.9 million on December 31, 2021. The Company expects that this cash balance will be sufficient to fund operations for at least two years.

About Alpha DaRT

Alpha DaRT (Diffusing Alpha-emitters Radiation Therapy) is designed to enable highly potent and conformal alpha-irradiation of solid tumors by intratumoral delivery of radium-224 impregnated sources. When the radium decays, its short-lived daughters are released from the sources and disperse while emitting high-energy alpha particles with the goal of destroying the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT aims to mainly affect the tumor, and to spare the healthy tissue around it.

Starpharma annual report and full year financial results

On August 25, 2022 Starpharma (ASX: SPL, OTCQX: SPHRY) reported its annual report and financial results for the year ended 30 June 2022 (Press release, Starpharma, AUG 25, 2022, View Source;mc_eid=bf52dd3418 [SID1234618667]).

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Download Annual Report 2022.

Financial Results

Strong cash position with a balance of $49.9M as at 30 June 2022
Revenue up 128% to $4.9M (FY21: $2.2M) on the rollout of VIRALEZE
Reported loss down 18% to $16.2M (FY21: $19.7M)
Receipt of $7.7M R&D tax incentive
Highlights

Two new DEP partnerships with leading, global pharmaceutical companies, Genentech and Merck & Co., Inc.
Sales of VIRALEZE have significantly increased in FY22, following new product launches in Vietnam and Italy and relaunch in the UK by LloydsPharmacy
Positive interim efficacy findings reported in the prostate cancer cohort of the Phase 2 DEP cabazitaxel trial showing efficacy signals in 100% of evaluable patients
Three clinical-stage internal DEP oncology assets advancing well, with encouraging efficacy signals observed
AstraZeneca’s partnered DEP product, AZD0466: additional indication (non-Hodgkin’s lymphoma) added and recruitment initiated in a global Phase 1/2 clinical trial; additional trial sites opened for the initial Phase 1/2 leukemia trial
VIRALEZE demonstrated high levels of protection against Omicron in an in vivo challenge model as well as impressive antiviral effects against influenza in laboratory studies and outperformed other antiviral agents used in marketed nasal sprays
Starpharma CEO, Dr Jackie Fairley, commented on the full year results:

"Starpharma has achieved many significant milestones across our business during FY22, including significant increases in revenue and the execution of new and valuable corporate partnerships, despite the ongoing challenges presented by the global pandemic.

"Starpharma was very pleased to sign two new DEP partnerships with leading, global companies – Genentech and Merck & Co., Inc – while our most advanced partner, AstraZeneca, further expanded the global clinical program for their DEP product, AZD0466, adding a new cancer type (non-Hodgkin’s lymphoma) and significantly increasing the number of trial sites worldwide. These new AZD0466 developments are particularly exciting for Starpharma, given the expanded market potential, and together with our new DEP partnerships, signify the growing momentum and value in our DEP platform.

"During the year, Starpharma’s internal DEP products continued to yield impressive responses in our Phase 2 oncology trials, including significant tumour shrinkage and stable disease in some of the most common and deadly cancers. We were pleased to report the positive interim findings from the prostate cancer cohort of Starpharma’s DEP cabazitaxel trial in which efficacy signals were observed in all evaluable patients treated with our DEP version of the leading prostate cancer drug cabazitaxel.

"Starpharma’s marketed products have reached more people this year than ever before, with sales of VIRALEZE significantly increasing following successful product launches in Vietnam and Italy, and relaunch in the UK through LloydsPharmacy. In parallel with commercial activities for VIRALEZE, we have continued to test the efficacy of SPL7013, the antiviral agent in VIRALEZE, against important respiratory viruses, including multiple variants of SARS-CoV-2 and influenza. SPL7013 has demonstrated consistent potent activity against a broad spectrum of respiratory viruses and has outperformed other marketed products when tested head-to-head.

"Looking ahead, Starpharma is well positioned for growth, with a strong balance sheet, growing sales revenue, an expanding portfolio of DEP products and valuable corporate DEP partnerships. Starpharma continues to champion its Environment, Social and Governance (ESG) pillars by creating important products that have the potential to make a significant contribution to the health and wellbeing of patients around the world."

Key activities

Partnered DEP Programs

Signed and commenced a second DEP Research Agreement with MSD (Merck & Co., Inc.), building on our DEP partnership in the innovative and valuable research area of antibody drug conjugates (ADCs).

Signed and commenced an exploratory DEP Research Agreement with Genentech, which was expanded within six months of the initial agreement to include an additional DEP program.

Under Starpharma’s DEP licence with AstraZeneca, the global clinical program for AZD0466 continued to advance with multiple new sites opening and commencement of a new clinical trial in an additional cancer type – non-Hodgkin’s lymphoma (NHL).

Starpharma also continued to progress its DEP program with Chase Sun, which involves the development of a DEP anti-infective product for Chase Sun.

Starpharma continues to pursue further partnering opportunities for its DEP drug delivery platform and active commercial discussions are underway in a number of research areas including DEP radiotheranostics.

Internal DEP Programs

Starpharma’s Phase 2 clinical trial of DEP cabazitaxel continues to recruit well with 70 patients enrolled to date. During the year, Starpharma reported positive interim findings from the prostate cancer cohort of this trial, where 100% of evaluable patients treated with DEP cabazitaxel demonstrated one or more efficacy signals. This trial continues recruitment of patients with ovarian and gastroesophageal cancers, following observation of encouraging efficacy signals in these tumour types, thereby expanding the market potential for DEP cabazitaxel.

The DEP irinotecan Phase 2 clinical trial continues to progress well, with 83 patients now enrolled. Efficacy signals including prolonged tumour shrinkage and reductions in tumour markers have been observed in multiple tumour types, including colorectal, breast, ovarian, pancreatic, lung, and oesophageal cancers. Enrolment of patients in the combination arm for DEP irinotecan in combination with 5-FU + leucovorin (a commonly used combination treatment regimen in colorectal cancer) has now commenced.

The clinical program for DEP docetaxel has enrolled 72 patients to date across the monotherapy and combination arms. Encouraging efficacy signals such as prolonged stable disease and significant tumour shrinkage have been observed in heavily pre-treated patients with lung, pancreatic, oesophageal, cholangiocarcinoma and gastric cancers.

Manufacture of DEP gemcitabine is now complete in readiness for Starpharma to commence a Phase 1/2 clinical trial, with planned clinical trial sites in the UK and Australia. Preparations for trial commencement are well advanced, with the clinical research organisation and site selection processes, regulatory and ethics preparations nearing completion.

Starpharma also continues to deepen its pipeline of DEP assets by actively progressing a number of its own internal programs in areas including DEP radiotheranostics and DEP ADCs.

Marketed Products

Starpharma signed sales and distribution arrangements for VIRALEZE broad-spectrum antiviral nasal spray with commercial partners in Italy (ADMENTA Italia Group) and Vietnam (Health Co), and an agreement with Etqan & Nazahah for nine countries in the Middle East.

VIRALEZE was launched in Vietnam and Italy and relaunched in the UK through LloydsPharmacy.

Starpharma continued its scientific collaboration with The Scripps Research Institute to test VIRALEZE and SPL7013 against a range of respiratory viruses, including multiple variants of SARS-CoV-2[1] (Omicron and Delta) and influenza.

VIRALEZE demonstrated excellent protection against infection with the highly transmissible SARS-CoV-2 Omicron variant in a stringent in vivo viral challenge model[2]. These in vivo findings build on the in vitro findings reported by Starpharma earlier in the financial year, which showed that VIRALEZE achieved the maximal possible reduction of virus infectivity against the Omicron variant of SARS-CoV-2 in laboratory-based antiviral and virucidal assays.

The broad-spectrum activity of VIRALEZE was further demonstrated with impressive results for SPL7013, in VIRALEZE, against influenza A and B. SPL7013 achieved more than 90% reduction in viral infectivity of both influenza A and B viruses within one minute. SPL7013 also demonstrated irreversible virucidal properties against both types of influenza virus and outperformed other antiviral agents used in marketed nasal sprays.

VIRALEZE is registered in more than 30 countries and is available in pharmacies, retail outlets and online in a number of countries. Sales of VIRALEZE have significantly increased in FY22.

Starpharma continued to pursue registration and commercialisation for VIRALEZE in multiple other countries, with regulatory submissions in progress and active commercial discussions underway. In Australia, the review by the TGA for the nasal spray application as a medical device is ongoing.

Regulatory approvals for VivaGel BV were achieved in the Middle Eastern countries, Bahrain and Qatar, and pre-launch marketing activities have commenced. Starpharma’s marketing partner, Mundipharma, is also progressing further launches of VivaGel BV in Asia and registrations in other countries across Asia and the Middle East.

An important publication for VivaGel BV was achieved in the highly regarded peer-reviewed European journal, Archives of Gynecology & Obstetrics. This publication will support marketing activities and importantly, the inclusion of the product in clinical management guidelines for BV.

Starpharma’s partner, Okamoto, launched a new VivaGel condom range in Japan, under the brand name Pure Marguerite. The range is being distributed through major retail chains in Japan. Okamoto has also commenced regulatory processes for the VivaGel condom in additional countries in Asia.

Corporate

Ms Lynda Cheng was appointed as an independent non-executive director on 1 August 2021. Ms Cheng has more than 25 years of experience as a finance executive including more than 15 years at Visy Industries/Pratt Holdings and 10 years in investment banking.

Dr Jeff Davies, former CSL executive, was appointed as an independent non-executive director on 1 April 2022, bringing over 35 years of biopharmaceutical industry experience to Starpharma’s Board.

Financials

Starpharma concluded FY22 in a strong financial position with a cash balance $49.9 million. Net operating cash outflows for the year were $13.2 million (FY21: $14.8 million). Receipts from customers and grants totalled $13.0 million, including receipts from customers of $4.8 million (FY21: $2.4 million) and R&D tax incentive of $7.7 million.

Revenue was up 128% to $4.9 million for the year, with $4.7 million for VIRALEZE and VivaGel product sales, royalty, and research revenue from commercial partners. Interest income was $0.2 million for the year. Other income of $0.3 million, consisted of grant funding awarded by the Australian Government’s Medical Research Future Fund to expedite development and commercialisation of VIRALEZE (FY21: $0.9 million).

The loss after tax of $16.2 million (FY21: $19.7 million) includes expensing of all research and development expenditure and IP costs across the portfolio. The decreased loss compared to the prior year reflects the combination of higher sales and partner revenue and lower R&D expenditure due to the completion of VIRALEZE development and the stage and progression of DEP internal clinical programs. The reduced loss reflects a favourable foreign exchange movement of $0.9 million over the prior year, primarily on foreign currency held.

Download ASX Announcement: Starpharma annual report and full year financial results (PDF, 8MB)

[1] SARS-CoV-2 is the virus that causes COVID-19

[2] The study used the K18-hACE2 mouse model, which is an in vivo humanised mouse model that expresses the human angiotensin converting enzyme (hACE2) receptor, the receptor used by SARS-CoV-2 to infect cells in the human nasal cavity and respiratory tract.