On February 21, 2024 Aleta Biotherapeutics (Aleta), a clinical stage, immuno-oncology company with a CAR T-Cell Engager (CTE) platform that enables cell cancer therapies to work more effectively, and Cancer Research UK’s Centre for Drug Development, reported the first patient was dosed in a Phase 1/2 clinical trial (Press release, Aleta Biotherapeutics, FEB 21, 2024, View Source [SID1234640353]). This trial is evaluating the Company’s first-in-class biologic CAR T-Cell Engager, ALETA-001, for the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19-targeting CAR T-cell therapy.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cancer Research UK’s Centre for Drug Development is sponsoring and conducting the Phase 1/2 clinical trial of ALETA-001, the lead agent in Aleta’s portfolio.

"It is very exciting and meaningful to have ALETA-001 now in the clinic. Clinical evaluation of ALETA-001 is a key milestone toward a much-needed treatment option for the many cancer patients whose CD19-targeted CAR T-cell therapies ultimately stop working. ALETA-001 restores and increases the effectiveness with which CAR T-cells can kill cancer cells, and we believe that it will enable more patients to successfully benefit from cell therapies," stated Dr. Paul Rennert, President and Chief Scientific Officer, Aleta Biotherapeutics.

The ALETA-001 Phase 1/2 clinical trial is an open-label, dose-expansion trial which will evaluate safety and tolerability, pharmacokinetic and pharmacodynamic effects, and early signals of clinical efficacy of ALETA-001 as a single agent. Chief Investigator Dr. Sridhar Chaganti is leading the trial from University Hospital Birmingham NHS Foundation Trust in Birmingham, UK, and will enroll patients who are relapsed/refractory following treatment with available CD19-targeting CAR T-cell therapies. For more information on this trial, please visit clinicaltrials.gov (NCT06045910)

Dr. Lars Erwig, Cancer Research UK’s Director of Drug Development, said, "A significant number of patients with blood cancers unfortunately relapse following CD19-directed CAR T-cell therapy. ALETA-001 is being developed to provide patients with these cancers a better chance for a successful treatment outcome. At Cancer Research UK, we are very excited to study the clinical potential of ALETA-001 to transform a patient’s treatment journey – which can possibly be lifesaving in many cases."

"Aleta-001 is a uniquely designed new molecule with a novel mechanism of action, coating tumor cells densely with the target antigen, thereby stimulating the tumor killing ability of activated CAR T-cells," commented Dr. Sridhar Chaganti, Chief Investigator, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK. "This is an important new strategy being investigated to improve outcomes for patients with relapsed or refractory lymphomas who experience disease progression after CAR T-cell therapy and have a poor prognosis."

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead clinical stage program, ALETA-001, was designed specifically for the treatment of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy and are at risk of treatment failure. Developed to improve the effectiveness of CD19-directed CAR T-cell therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell, ALETA-001 contains the CD19 target protein which is further linked to an CD20 antibody domain. This allows CD19+/CD20+ cancer cells to be easily recognized and killed by CD19-directed CAR T-cells that were previously administered and are already circulating within a patient.

Aleta previously secured landmark clinical support and funding from Cancer Research UK for the ALETA-001 Phase 1/2 clinical trials, and ALETA-001 has received a UK Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is granted to medicines that address life-threatening or seriously debilitating conditions, and where there exists a significant patient or public health need.

On February 21, 2024 Oncoinvent AS, a clinical stage company advancing alpha emitter therapy across a variety of solid cancers, reported that members of management will present at the Evercore ISI 2024 Emerging Biotech Conference on Wednesday, February 28, 2024 at 11:20 a.m. ET (Press release, Oncoinvent, FEB 21, 2024, View Source [SID1234640352]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On February 21, 2024 Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a clinical-stage biopharmaceutical company advancing novel therapies in immunology and oncology by targeting myeloid cell inhibitory receptors, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IO-202 for the treatment of chronic myelomonocytic leukemia (CMML) (Press release, Immune-Onc Therapeutics, FEB 21, 2024, View Source [SID1234640351]). IO-202 received Fast Track Designation for treatment of relapsed or refractory CMML in 2023. In addition, Fast Track and Orphan Drug Designations for IO-202 were granted by the FDA for the treatment of acute myeloid leukemia (AML) in 2022 and 2020, respectively.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to Leukocyte Immunoglobulin-Like Receptor subfamily B4 (LILRB4) and serves as a targeted therapy with broad potential in blood cancers, autoimmune and inflammatory diseases. IO-202 in combination with azacitidine (AZA) is currently in a Phase 1 dose expansion clinical trial (NCT04372433), enrolling newly diagnosed patients with CMML who have not received any hypomethylating agents (HMA). A dose escalation trial of IO-202 has been completed, showing clinical efficacy in relapsed or refractory AML and CMML, either as a monotherapy or in combination with AZA. IO-202 has been shown to be well tolerated in all patients treated to date.

"Although current therapeutic options for CMML can improve a patient’s quality of life, there is a high unmet need for effective disease-modifying approaches that are potentially curative," said Charlene Liao Ph.D., chief executive officer and board chair of Immune-Onc. "We are very proud that the FDA has granted IO-202 Orphan Drug Designation for the treatment of CMML. We look forward to continued collaborations with our investigators and the FDA as we work to bring this potentially important therapy to patients with hard-to-treat blood cancers."

The FDA grants Orphan Drug Designation to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including exemption of FDA application fees and tax credits for qualified clinical testing, and conveys seven years of marketing exclusivity for a drug approved to treat an orphan disease in the United States.

ABOUT CHRONIC MYELOMONOCYTIC LEUKEMIA (CMML)

CMML is a rare form of blood cancer, occurring in 4 of every 1,000,000 people in the United States each year, or about 1,100 annual cases1. CMML is characterized by the presence of a high monocyte count (>1×109/L peripheral monocytes with monocytes ≥ 10% of white blood count) along with dysplastic features in the bone marrow2. Current FDA approved therapies for CMML are all hypomethylating agents, including azacitidine, that attempt to control CMML without enhancement to overall survival.

ABOUT LILRB4 (also known as ILT3)

LILRB4, also known as ILT3, is an immune-modulatory transmembrane protein found on monocytes and monocyte-derived cells. LILRB4 is expressed on certain hematologic cancer cells, such as myelomonocytic leukemia blasts, and on certain pathogenic cells involved in autoimmunity and inflammatory processes.

About IO-202

IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to LILRB4. IO-202 is a humanized IgG1 antibody with Fc effector function to kill LILRB4hi cells via antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). As such, IO-202 is a targeted therapy with broad potential in both blood cancers and autoimmune and inflammatory diseases.

IO-202 has completed the dose escalation part of the first-in-human, multicenter, open-label Phase 1 study in the U.S., and the data was presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in 2023. This Phase 1 trial has advanced to the dose expansion stage to evaluate IO-202 in combination with azacitidine (NCT04372433) in patients with newly diagnosed chronic myelomonocytic leukemia (CMML) who have not received any hypomethylating agents (HMA).

The U.S. Food and Drug Administration granted IO-202 Fast Track Designations for treatment for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) and for the treatment of patients with relapsed or refractory chronic myelomonocytic leukemia (CMML). The FDA has also granted Orphan Drug Designations to IO-202 for the treatment of AML and for the treatment of CMML.

On February 21, 2024 Intensity Therapeutics, Inc. (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported that Lewis H. Bender, President and Chief Executive Officer, will present a company overview at the 2024 BIO CEO & Investor Conference on Monday, February 26, 2024, at the New York Marriott Marquis at 11:45am ET in the Uris Room (Press release, Intensity Therapeutics, FEB 21, 2024, View Source;investor-conference-302066806.html [SID1234640350]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mr. Bender will also host in-person one-on-one meetings during the event. For more information about the 2024 BIO CEO & Investor Conference, please visit View Source

On February 21, 2024 Photocure ASA (OSE: PHO) reported Hexvix/Cysview revenues of NOK 114.2 million in the fourth quarter of 2023, an increase of 20% over the prior-year period (Q4 2022: NOK 94.9 million), and EBITDA of NOK 29.9 million (NOK -16.9 million) following solid business development for the Company (Press release, PhotoCure, FEB 21, 2024, View Source [SID1234640349]). Total revenues increased 37% in the fourth quarter of 2023 compared to Q4 2022, including a milestone payment from Asieris related to the license agreement for Cevira. Photocure has an ambition to deliver 40-70 new and upgraded Saphira tower installations in 2024, consolidated product revenue growth of 6% to 9% (constant currency), and positive EBITDA excluding business development expenses.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Hexvix/Cysview revenue increased 20% year-over-year in the fourth quarter, and we delivered EBITDA of NOK 29.9 million. Our U.S. business grew despite the ongoing phase down of flexible BLC. In Europe, we continued to see positive developments with sales growth in Germany and strong signs of traction in high-potential underpenetrated territories, known as our Priority Growth Markets. Additionally, our partner Asieris announced acceptance of their new drug application for Hexvix in China, and that they plan to present the positive Phase III results for Cevira at the EUROGIN 2024 HPV Congress in March," says Dan Schneider, President & Chief Executive Officer of Photocure.

Photocure reported total group revenues of NOK 142.5 million in the fourth quarter of 2023 (NOK 104.2 million), and EBITDA* of NOK 29.9 million (NOK -16.9 million), driven by a combination of unit volume growth, price increases and a benefit from foreign exchange. Hexvix/Cysview revenues ended at NOK 114.2 million in the quarter (Q4 2022: NOK 94.9 million). EBIT was NOK 22.5 million (NOK -23.1 million) and the cash balance at the end of the period was NOK 259.5 million.

At the end of the fourth quarter of 2023, the installed base of rigid blue light cystoscopy (BLC) systems in the U.S. was 352, an increase of 17% or 51 towers since the fourth quarter of 2022. 2023 marked the highest number of rigid cystoscopes placed during any year since Photocure launched BLC in the U.S. Saphira blue light towers now represent 39% of the installed base of U.S. rigid towers.

"We continue to expect a large order of new rigid towers to come through, however, timing of the order is now anticipated in the second quarter of this year. We believe deployment of these towers has potential to significantly expand access to blue light cystoscopy for more physicians and patients in the U.S. In addition, our commercial team in North America remains focused on increasing Cysview kit usage in existing accounts, accelerating adoption in accounts using Saphira for the first time, reactivating low users or inactive accounts, and expanding BLC usage by leveraging the positive clinical results from studies reported in 2023," Schneider adds.

Photocure believes that the benefits of Blue Light Cystoscopy with Hexvix/Cysview offering superior detection and management of bladder cancer will continue to be adopted and become the standard of care. For 2024, the Company anticipates new and upgraded Saphira blue light tower installations in the U.S. in the range of 40 to 70, consolidated product revenue growth of 6% to 9% in constant currency, and positive EBITDA excluding business development expenses.

"This year, we are planning for further growth and are well-positioned for new opportunities. We expect that the installed base of rigid towers in the U.S. will continue to expand, augmented by a large potential order anticipated next quarter. The technology upgrade cycle in blue light cystoscopy is expected to continue with the anticipated launch of Olympus’ new HD blue light system later this year. Also key to long term success is our strategy to reintroduce flexible BLC in high definition so that we can further develop the large surveillance market worldwide," Schneider continues, and concludes:

"The Citizen’s Petition to reclassify BLC equipment from Class 3 to Class 2 in the U.S. is a potential regulatory action that could change the game by creating an accelerated approval process that would enable equipment manufacturers to access the U.S. market quickly and expand BLC use by meeting the demands of the market. Our partner Asieris continues to advance its licensed and partnered programs: Cevira and Hexvix in China, respectively, which are both expected to generate significant additional revenue through regulatory and future sales milestones. With these initiatives and more, we look forward to the coming year. We remain focused on driving value for patients, our customers and our shareholders."

Please find the full financial report and presentation enclosed.

EBITDA* and other alternative performance measures (APMs) are defined and reconciled to the IFRS financial statements as a part of the APM section of the fourth quarter 2023 financial report on page 24.

The quarterly report and presentation will be published at 08:00 CET and will be publicly available at www.photocure.com. Dan Schneider, CEO and Erik Dahl, CFO, will host a live webcast at 14:00 CET.

The presentation will be held in English and questions can be submitted throughout the event. The streaming event is available through https://channel.royalcast.com/landingpage/hegnarmedia/20240221_5/

The presentation is scheduled to conclude at 14:45 CET.