Neuren reports 2022 full-year results

On February 23, 2023 Neuren Pharmaceuticals (ASX: NEU) reported its full-year financial results for 2022 (Press release, Neuren, FEB 24, 2023, View Source;[email protected] [SID1234627651]).

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Neuren CEO Jon Pilcher commented: "The substantial progress that was made in 2022 across the whole business leaves Neuren very well placed for the transforming catalysts that have been anticipated to crystallise in 2023. We are now approximately two weeks away from the first of those – the FDA PDUFA action date for trofinetide in Rett syndrome. The Neuren team is excited about the year ahead and the prospects for both trofinetide and NNZ-2591."

Commentary on events and outlook

Trofinetide for Rett syndrome

In December 2021 Neuren’s partner for trofinetide in North America, Acadia Pharmaceuticals (Nasdaq: ACAD), announced positive top-line results from the pivotal, Phase 3 Lavender study evaluating the efficacy and safety of trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome. The 12-week placebo-controlled study demonstrated a statistically significant improvement over placebo for both co-primary endpoints. In September 2022 the US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for trofinetide to treat Rett syndrome in adults and pediatric patients two years of age or older, that was submitted in July by Acadia. The FDA granted a Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of 12 March 2023. The FDA also informed Acadia that they were not planning to hold an Advisory Committee meeting. If approved, trofinetide will be the first drug for the treatment of Rett syndrome. The trofinetide program has Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA.

In October 2022, Neuren received from Acadia a milestone payment of US$10 million following the acceptance of the NDA for review by the FDA. If the NDA is approved by the FDA, Neuren expects to earn revenue in 2023 for Rett syndrome in the US alone of A$104 million plus royalties. The next potential milestone payment to Neuren would be US$40 million (A$57 million at an assumed exchange rate of 0.70), payable following the first commercial sale of trofinetide in the United States. Subsequently, Neuren is eligible to receive ongoing doubledigit percentage royalties on net sales of trofinetide in North America, plus milestone payments of up to US$350 million (A$500 million) on achievement of a series of four thresholds of total annual net sales, plus one third of the market value of a Rare Pediatric Disease Priority Review Voucher if awarded by the FDA upon approval of the NDA, with the one third share estimated by Neuren as US$33 million (A$47 million). No royalties or similar costs are payable by Neuren to third parties, which means that Neuren’s revenue from Acadia will flow through to pre-tax profit.

Acadia has exclusive rights to develop and commercialize trofinetide in North America, which is fully funded by Acadia. Neuren retains all rights to trofinetide for all countries outside North America and has a fully paid-up, irrevocable licence for use in those countries to all data generated by Acadia. Neuren has received strong interest for potential commercial partnerships and discussions are advancing under a process to secure the optimum outcome.

NNZ-2591 for multiple neurodevelopmental disorders

Neuren is developing NNZ-2591 for four serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Phase 2 clinical trials are currently ongoing in children with each of Angelman, Phelan-McDermid and Pitt Hopkins syndromes and in preparation for Prader-Willi syndrome. All four programs have been granted Orphan Drug designation by the FDA. The estimated number of potential patients being targeted across these four disorders is more than five times larger than Rett syndrome. Neuren retains all global rights to NNZ-2591.

In July and August 2022, Neuren announced the commencement of Phase 2 clinical trials of NNZ-2591 for Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome, after receiving in March 2022 approval from the FDA for Investigational New Drug (IND) applications to conduct the trials. In December 2022, Neuren submitted an IND application to the FDA for approval to proceed with a Phase 2 trial in Prader-Willi syndrome and received approval from the FDA in January 2023.

The open label Phase 2 trials are each enrolling up to 20 children to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.  All subjects receive NNZ-2591 as an oral liquid dose twice daily, with escalation in two stages up to the target dose during the first 6 weeks of treatment, subject to independent review of safety and tolerability data.

The current trials are enrolling subjects in three age groups.  Safety and tolerability data in the oldest age group must be independently reviewed before proceeding with dosing in the second age group and then safety and tolerability data in the second age group must be independently reviewed before proceeding with dosing in the youngest age group.

The study begins with 4 weeks of observation to thoroughly examine baseline characteristics prior to treatment, against which safety and efficacy are assessed for each child. This is followed by the treatment period of 13 weeks.  A follow-up assessment is made 2 weeks after the end of treatment. 

Phelan-McDermid Pitt Hopkins Angelman Subjects Up to 20, aged 3 to 12 Up to 20, aged 3 to 17 Up to 20, aged 3 to 17 Number of sites 4 (US) 5 (US) 3 (Australia) www.clinicaltrials.gov NCT05025241 NCT05025332 NCT05011851

In December 2022, Neuren announced that in the Phelan-McDermid syndrome trial and in the Angelman syndrome trial, the first subject in the oldest age group had completed the treatment period of 13 weeks, with a good safety and tolerability profile. Each subject was successfully escalated up to the target dose following safety and tolerability reviews by an independent data and safety monitoring committee (DSMC). No serious adverse events were reported and no dose modifications were required. Most of the adverse events reported were mild and not considered to be related to study drug. There were no clinically relevant observations in safety laboratory measurements or cardiac tests.

The overall aim of these first clinical trials in patients is to expedite the generation of data that will enable the subsequent trials to be designed as registration trials. The four trials will likely complete at different times, with a series of top-line results announcements anticipated, commencing with Phelan-McDermid syndrome in H2 2023.

In order to accelerate the overall development plan, in parallel with conducting the Phase 2 trials Neuren is executing additional development work required for Phase 3 development. This includes non-clinical toxicity studies to support longer clinical trials and commercial use of the product, as well as optimisation of the drug product and drug substance manufacturing arrangements.

Neuren is well funded from current cash reserves to execute the Phase 2 trials and Phase 3 preparation, notwithstanding the anticipated material cash flows from trofinetide.

Financials

Profit after tax for the year ended 31 December 2022 was A$0.2 million compared with a loss of A$7.8 million in 2021. Revenue of A$14.6 million was received under the licence agreement with Acadia (2021: nil) and foreign exchange gains were A$1.2 million (2021: A$0.4 million). These were offset by an increase of A$3.2 million in research and development costs, due to higher expenditures in 2022 for the NNZ-2591 Phase 2 clinical trials and the foundational work to prepare for Phase 3 development of NNZ-2591 across multiple indications. There was also an increase in corporate and administrative costs of A$1.5 million, mainly due to share-based payments and higher employee benefits expense, reflecting some expansion for the NNZ-2591 program. In addition, a loss of A$0.7 million on the fair value of outstanding forward contracts to sell Australian dollars and buy US dollars was recognised at 31 December 2022. Prudent control of expenditure continues to be an important principle in the Group’s operations and financing.

Cash reserves at 31 December 2022 were $40.2 million (2021: $36.8 million). Net cash received from operating activities was $3.6 million, compared with net cash used in operating activities of $10.0 million in 2021. The increase of $13.6 million was due to the receipt of the first milestone payment from Acadia of $15.9 million (2021: nil), offset by higher payments for employees and directors of $2.8 million (2021: $1.8 million) and a lower receipt under the R&D Tax Incentive program of $1.4 million (2021: $2.5 million). Net cash from financing activities for 31 December 2022 was nil, compared with $22.2 million in 2021 from the issue of new ordinary shares in a share placement and share purchase plan.

The large increase in market capitalization during the year resulted in Neuren being promoted in September into the S&P/ASX 300 index.

BullFrog AI Partners with J. Craig Venter Institute to Develop Colorectal Cancer Therapeutic

On February 23, 2023 BullFrog AI Holdings, Inc. (NASDAQ:BFRG; BFRGW) ("Bullfrog AI" or the "Company"), a digital technology company using machine learning to usher in a new era of precision medicine reported a co-development arrangement with the J. Craig Venter Institute (JCVI), a world-leading institution in genomics research and innovation, for the design and validation of a synthetic HSV-1 (herpes simplex type 1) virus particle targeting colorectal cancer (Press release, Bullfrog AI, FEB 23, 2023, View Source [SID1234635367]).

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"This project leverages our bfLEAP artificial intelligence and machine learning platform to optimize oncolytic HSV-1 particle design for cancer-cell-specific targeting, reduced toxicity, and therapeutic payload," said BullFrog AI’s founder and CEO, Vin Singh. "Our objective with this collaboration is to generate a novel class of targeted colorectal cancer therapeutics with potential for a best-in-class toxicity profile, while providing a clear demonstration of our ability to advance development of new medicines using artificial intelligence."

"The JCVI has been at the forefront of synthetic biology for more than 25 years, and we’ve long stated that the knowledge, tools, and techniques developed have broad use. It is gratifying seeing their application in such an impactful area of human health, and we look forward to working with BullFrog AI," remarked J. Craig Venter, Ph.D., chairman, CEO, and founder of JCVI.

"We are excited to partner with Bullfrog AI. The combination of artificial intelligence and engineered biology approaches has the potential to rapidly develop safer precision cancer therapeutics," added Sanjay Vashee, Ph.D., professor and director of the JCVI, Rockville, Maryland campus.

According to the American Cancer Society, colorectal cancer is the third most common cancer diagnosed in both men and women in the U.S., excluding skin cancers, and is the third leading cause of cancer-related deaths in the U.S.

Moderna Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Updates

On February 23, 2023 Moderna, a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported financial results and provided business updates for the fourth quarter and fiscal year 2022 (Press release, Moderna Therapeutics, FEB 23, 2023, View Source [SID1234630404]).

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"2022 was another impressive year for Moderna, with over $19 billion in revenue and significant clinical breakthroughs across our portfolio. We continue to provide our Omicron-targeting bivalent vaccines worldwide, with the latest real-world evidence highlighting the continued protection of our vaccines against hospitalization and death," said Stéphane Bancel, Chief Executive Officer of Moderna. "Our infectious disease platform continues to progress with positive Phase 3 data in RSV for older adults. We are investing to scale Phase 3 manufacturing for personalized cancer vaccines so that we can run several Phase 3 studies simultaneously. With planned R&D investments of $4.5 billion for the year, I am excited about the new medicines we believe we will bring to patients in the coming few years."

Moderna is also announcing that Juan Andres, currently President, Strategic Partnerships and Enterprise Expansion, will be retiring from the Company in May after more than six years.

"Juan has played a tremendous role since joining Moderna in 2017. Juan served as Chief Technical Operations and Quality Officer and led our manufacturing from an early-stage clinical development company to a commercial company. In 2020 and 2021, Juan did a historic job with his team to scale Moderna for a global commercial launch, during a pandemic. It is unbelievable that he led the team from having made less than 100,000 doses across our entire portfolio in 2019 to more than 800 million doses in 2021 of our COVID-19 vaccine globally, all during a pandemic," said Mr. Bancel. "Very few manufacturing leaders could have led such an achievement during such a challenging time. We, and the hundreds of millions of people across the globe who received the Moderna COVID-19 vaccine, owe Juan our gratitude."

Recent Progress Includes:
Respiratory Vaccines
Approved and Phase 3 trials
Spikevax Bivalent BA.4/BA.5 authorized under EUA in the U.S. as a booster dose for all age groups from 5 months.
RSV vaccine in older adults (mRNA-1345) met its primary efficacy endpoint and received Breakthrough Therapy Designation from FDA. mRNA-1345 demonstrated vaccine efficacy of 83.7% against RSV lower respiratory tract disease, defined by 2 or more symptoms, and 82.4% with 3 or more symptoms in older adults. mRNA-1345 was generally well-tolerated, with no safety concerns identified by the Data Safety Monitoring Board (DSMB). Based on these results, Moderna expects to submit a Biologics License Application (BLA) for mRNA-1345 to the FDA in the first half of 2023. The pediatric Phase 1 trial of mRNA-1345 is fully enrolled.
Flu (mRNA-1010) interim analysis from Phase 3 immunogenicity and safety trial demonstrated mRNA-1010 achieved superiority on seroconversion rates for A/H3N2 and A/H1N1, superiority on geometric mean titer ratios for A/H3N2, and non-inferiority on geometric mean titer ratios for A/H1N1. Non-inferiority was not met for seroconversion rates, and geometric mean titer ratios for the influenza B/Victoria- and B/Yamagata-lineage strains. mRNA-1010 was found to be generally well-tolerated.
The ongoing mRNA-1010 Phase 3 efficacy study (P302) conducted in the Northern Hemisphere has accrued more than 200 PCR-confirmed cases. The first per-protocol interim analysis of efficacy is expected to be reviewed by an independent DSMB before the end of the first quarter.

Latent Vaccines
Phase 3 trials
CMV vaccine (mRNA-1647) pivotal Phase 3 study, known as CMVictory, is ongoing, with enrollment more than 40% complete. CMV vaccine candidate mRNA-1647 adolescent trial dosed its first participants

Early clinical
First participants, adults 50 years of age or older, dosed in Phase 1/2 trial of varicella-zoster virus (VZV) candidate mRNA-1468. VZV causes chickenpox, commonly affecting children and young adults, and shingles in adults.

Therapeutics
Immuno-oncology
Personalized cancer vaccine (PCV) Phase 2 evaluating mRNA-4157/V940 in combination with KEYTRUDA , Merck’s anti-PD-1 therapy, as adjuvant treatment for patients with high-risk melanoma reported positive topline data, showing a 44% reduction of recurrence or death versus Keytruda alone.
• The companies have received Breakthrough Therapy Designation for mRNA-4157/V940.
• As previously announced on October 12, Merck exercised its option to jointly develop and commercialize mRNA-4157/V940 pursuant to the terms of its existing Collaboration and License Agreement, and Moderna received $250 million from Merck in the fourth quarter in connection with the option exercise. The companies plan to discuss results with regulatory authorities, initiate a Phase 3 study in adjuvant melanoma in 2023, and rapidly expand to additional tumor types, including non-small cell lung cancer (NSCLC).

Rare diseases
The Phase 1/2 Paramount study of propionic acidemia (PA) candidate (mRNA-3927) is enrolling patients in cohort 5 (0.9mg/kg). Encouraging clinical benefit has been observed to date and all eligible participants continue to opt-in to the Open Label Expansion. Next step is dose selection for the expansion arm of the Phase 1/2 study.
Moderna’s partner, Vertex, initiated Phase 1 trial for VX-522, an mRNA therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung disease for those who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. VX-522 is Moderna’s first inhaled mRNA to enter the clinic.

Cardiovascular disease
Phase 1B trial of heart failure treatment candidate, mRNA-0184 was initiated. mRNA-0184 is designed to produce the naturally occurring cardioprotective hormone relaxin.

Moderna now has 48 programs1 in development across 45 development candidates, of which 38 are currently in active clinical trials. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 140 peerreviewed publications.

Fourth Quarter and Full Year 2022 Financial Results
Revenue: Total revenue for the fourth quarter of 2022 was $5.1 billion, compared to $7.2 billion in the same period in 2021, mainly due to a decrease in sales of the Company’s COVID-19 vaccines. Product sales for the fourth quarter of 2022 were $4.9 billion, a decrease of 30% compared to the same period in 2021, primarily driven by lower sales volume, compared to overall higher demand in the prior year and the related manufacturing ramp up in the fourth quarter of 2021.

COVID-19 vaccine sales. Cost of sales, as a percentage of product sales, increased by 25 percentage points, from 14% in the same period in 2021. The increase was driven by increased royalties, a charge of $297 million for inventory write-downs related to COVID-19 products that have exceeded or are expected to exceed their approved shelf-lives prior to being used, a loss on firm purchase commitments and related cancellation charges of $281 million, and an expense for unutilized manufacturing capacity and related contract manufacturing organization charges of $376 million. These charges, other than royalties, are driven by costs associated with surplus production capacity, overall lower demand and a shift to our most recent Omicron-targeting COVID-19 bivalent booster, mRNA-1273.222

Research and Development Expenses: Research and development expenses for the fourth quarter of 2022 increased by 87% to $1.2 billion, in comparison to the same quarter of 2021. The growth in spending was mainly due to an increase in clinical trial-related expenses, largely driven by increased clinical development activities, particularly with respect to our RSV, seasonal flu and CMV programs. The growth was also driven by the acquisition of a Priority Review Voucher and an increase in personnelrelated costs due to increased headcount to support our increased research and development efforts.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for the fourth quarter of 2022 increased by 87% to $375 million, in comparison to the same quarter of 2021. The growth in spending was primarily due to increased external and marketing spend, driven by commercial activities in support of the Company’s marketed products and build-out.

Effective Tax Rate: The effective tax rate was 11% for the fourth quarter of 2022, remained relatively flat, compared to 10% for the same period in 2021.

Net Income: Net income decreased by 70% to $1.5 billion in the fourth quarter of 2022, compared to the same period in 2021. Earnings Per Share: Diluted EPS decreased by 68% to $3.61 in the fourth quarter of 2022, compared to the same period in 2021. Full Year 2022 Revenue: Total revenue was $19.3 billion for the full year 2022, compared to $18.5 billion in 2021. Total revenue increased in 2022, primarily due to increased sales of the Company’s COVID-19 vaccines. Product sales for 2022 were $18.4 billion, an increase of 4%, compared to 2021, primarily driven by a higher average selling price due to customer mix. Cost of Sales: Cost of sales was $5.4 billion, or 29% of the product sales for 2022, including third-party royalties of $1.1 billion. Cost of sales, as a percentage of product sales, increased by 14 percentage points, from 15% in 2021. The increase was mainly due to a charge of $1.3 billion for inventory writedowns related to COVID-19 products that have exceeded or are expected to exceed their approved shelf-lives prior to being used, a loss on firm purchase commitments and related cancellation charges of $725 million, and an expense for unutilized manufacturing capacity and related contract manufacturing organization charges of $776 million. These charges are driven by overall lower demand, in particular from low-income countries, a shift in product demand to our Omicron-targeting COVID-19 bivalent boosters and costs associated with surplus production capacity. Research and Development Expenses: Research and development expenses increased by 65% to $3.3 billion for 2022, compared to 2021. The growth in spending in 2022 was mainly due to increases in clinical trial expenses, clinical manufacturing expenses, personnel-related costs, and consulting and outside services, largely driven by the Company’s late-stage clinical studies for the RSV, seasonal flu and CMV vaccine programs, as well as continued development of the Company’s pipeline. Selling, General and Administrative Expenses: Selling, general and administrative expenses increased by 100% to $1.1 billion for 2022, compared to 2021. The growth in spending in 2022 was mainly due to increases in consulting and outside services, marketing expense, and personnel-related costs, primarily attributable to the Company’s continued corporate expansion, particularly in the commercial area and to a lesser extent, in support functions. Provision for Income Taxes: The effective tax rate was 13% for 2022, compared to 8% for 2021. The increase in 2022 was primarily due to the benefit recorded in 2021 related to the release of the valuation allowance on the majority of our deferred tax assets. Net Income: Net income decreased by 31% to $8.4 billion for the full year 2022, compared to 2021. Earnings Per Share: Diluted EPS decreased by 29% to $20.12 for the full year 2022, compared to 2021. Cash Position: Cash, cash equivalents and investments as of December 31, 2022 and December 31, 2021 were $18.2 billion and $17.6 billion, respectively. Net Cash Provided By Operating Activities: Net cash provided by operating activities was $5.0 billion for the full year 2022, compared to $13.6 billion for the same period in 2021. Net cash provided by operating activities decreased in 2022, primarily attributable to revenue recognized from deferred revenue in excess of customer deposits received and increased income tax payments, partially offset by higher collection of receivables. Cash Used for Purchases of Property, Plant and Equipment: Cash used for purchases of property and equipment was $400 million for the full year 2022, compared to $284 million for the same period in 2021. The increase was primarily driven by the Company’s continued business expansion of its manufacturing and research facilities. Cash Used for Repurchases of Common Stock: Cash used for repurchases of common stock was $3.3 billion for the full year 2022. Moderna did not conduct share repurchases prior to the fourth quarter of 2021. From the end of the third quarter of 2021 to the end of the fourth quarter of 2022, the Company repurchased 27 million shares, reducing the number of common shares outstanding from 405 million to 385 million, more than offsetting 7 million shares of common stock issued in connection with equity compensation over this period. 2023 Financial Framework Advance Purchase Agreements (APAs): The Company has COVID vaccine sales of approximately $5 billion currently contracted for 2023 delivery, with potential additional sales opportunities in the United States (endemic private and government markets), Europe, Japan, and other key markets. The Company expects product sales in the first half of 2023 of approximately $2.0 billion. Cost of Sales: Cost of sales for the full year are expected to be in the range of 35-40% of sales, which includes approximately 5% of sales for combined royalties to Cellscript and NIAID. Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses: Full-year expenses are expected to be approximately $6.0 billion, with approximately $4.5 billion in R&D. Income Tax Provision: The Company expects a negligible provision for income taxes in 2023. Capital Expenditures: The Company expects capital investments for 2023 of approximately $1.0 billion. Share Repurchase Program The $3 billion share repurchase program announced in February 2022 was completed early in the fourth quarter 2022. The Company has commenced repurchases from the additional $3 billion program announced in August 2022, and currently has $2.8 billion remaining under this latest authorization. Corporate Updates • Continued Growth: Moderna had approximately 3,900 employees as of December 31, 2022, compared to approximately 2,700 employees as of December 31, 2021. • Company Accolades o Moderna named a Top Employer by Science and Science Careers for an eighth consecutive year. o Moderna named Top Employer by BioSpaces Best Places to Work for Second Consecutive Year. • Executive Committee Update • Juan Andres, President, Strategic Partnerships has announced that he will retire from the Company in May 2023. • Moderna previously announced that Jerh Collins joined the Company in October as Chief Technical Operations and Quality Officer. • On December 7, Brad Miller was appointed Chief Information Officer of Moderna. At the same time, Marcello Damiani, who was serving in the role as Chief Digital Officer, announced that he would retire from the Company after seven and a half years. Key 2023 Investor and Analyst Event Dates • Vaccines Day: April 11 • R&D Day: September 13

Investor Call and Webcast Information Moderna will host a live conference call and webcast at 8:00 a.m. ET on Thursday, February 23, 2023. To access the live conference call via telephone, please register at the link below. Once registered, dial-in numbers and a unique pin number will be provided. A live webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website. • Telephone: https://register.vevent.com/register/BI7d95b0c10db64ef48ce2960a272f006a • Webcast: View Source The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.

Quarterly Report Q4 2022

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Theralase® Phase II NMIBC Clinical Study (Interim) Data Presented at the ASCO GU Cancer Symposium

On February 23, 2023 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDCs") and their associated drug formulations, used to safely and effectively destroy various cancers, bacteria and viruses, reported that its Phase II clinical study ("Study II") interim data was recently presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Genito Urinary ("GU") Cancer Symposium via a moderated poster presentation (Press release, Theralase, FEB 23, 2023, View Source [SID1234628197]).

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Study II has been designed to treat patients diagnosed with Bacillus Calmette-Guérin ("BCG")-Unresponsive, Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") (with or without resected papillary Ta/T1 disease) with a patented investigational study drug (TLD-1433) (Trade Name: Ruvidar), a ruthenium based PDC intravesically instilled into a patient’s bladder, then subsequently activated by a proprietary investigational study device (TLC-3200), a green (520 nm) laser system equipped with fiber-optic light emitters and detectors.

Girish Kulkarni M.D., Ph.D., FRCSC, Divisions of Urology and Surgical Oncology, Department of Surgery, Princess Margaret Cancer Centre, University Health Network, Professor, University of Toronto, (Toronto, Ontario, Canada), lead principal investigator of Study II stated, "Patients with BCG-Unresponsive CIS NMIBC have historically had limited treatment options other than bladder removal surgery to deal with this disease. I am encouraged by the positive interim results of the light-activated, intravesical study drug TLD-1433 (Trade Name: Ruvidar) currently under investigation by Theralase in Study II. The interim analysis of the clinical data collected from Study II to date supports that early results show complete response rates in 53% of patients evaluated at 90 days and 28 % of patients evaluated at 450 days. Based on the clinical data presented to date, Rudivar could represent a viable treatment option with an acceptable safety profile.

I look forward to enrolling and treating additional patients, alongside the other clinical study sites involved in Study II, to complete this study’s enrollment."

Arkady Mandel MD, Ph.D., D.Sc., Interim Chief Executive Officer and Chief Scientific Officer, Theralase stated, "The high complete response rates and duration of this complete response without serious adverse events, directly related to the study drug or study device, indicates that intravesical Ruvidar is a promising alternative to existing therapies and compares favorably to other approved therapies; including: valrubicin, pembrolizumab and Adstiladrin. Ruvidar, as an intravesical monotherapy, has the potential to be introduced into mainstream medical practice, subject to regulatory approval, based on its one to two treatment methodology, high efficacy and high safety profile. One day we hope that Ruvidar will become the organ-sparing solution that is a game-changer for both patients and physicians."

The poster presented at the ASCO (Free ASCO Whitepaper) GU Cancer Symposium can be found on the Company’s website at www.theralase.com/ASCO_Poster.

About ASCO (Free ASCO Whitepaper)

Founded in 1964, the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) is the world’s leading professional organization for physicians and oncology professionals caring for people with cancer.

About Study II

Study II utilizes the therapeutic dose of TLD-1433 (0.70 mg/cm2) activated by the proprietary TLC-3200 (90 J/cm2) medical laser system. Study II is focused on enrolling and treating approximately 100 to 125 BCG-Unresponsive NMIBC CIS patients in up to 20 clinical study sites located in Canada and the United States.

About TLD-1433 (Ruvidar TM)

TLD-1433 is a patented PDC with 12 years of published peer reviewed preclinical research and is currently under investigation in Study II.

The trade name Ruvidar was selected by the Company, as Ru is the element symbol for Ruthenium, a rare transition metal belonging to the platinum group, which the Theralase PDC is based upon, vita is Latin for life and dar is Russian for gift; hence, roughly translated, "Ruthenium, the gift of life".