On August 9, 2023 Georgiamune Inc., a privately held, clinical stage biotechnology company, reported the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for GIM-122, a dual-functioning monoclonal antibody (Press release, Georgiamune, AUG 9, 2023, View Source [SID1234634109]). Furthermore, the company successfully completed a $75M Series A financing, positioning Georgiamune as a promising new biotechnology company in the fields of oncology and autoimmune diseases.

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The clearance of Georgiamune’s first IND represents a significant milestone for the company as it embarks on its first clinical trial in oncology. GIM-122, a first-in-class dual-functioning monoclonal antibody, targets a highly novel mechanism discovered through the distinguished academic work of Georgiamune’s Founder and Chief Executive Officer Dr. Samir Khleif and developed by the company to overcome immunotherapy resistance.

Georgiamune will initiate a first-in-human, open-label, phase 1/2 dose-escalation with enrichment and dose expansion study to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics and antitumor activity of GIM-122 as a single agent in adults with advanced solid malignancies who have failed treatment with a checkpoint inhibitor. Georgiamune plans to initiate its phase 1/2 clinical trial in the second half of 2023 and continue development of its extensive first-in-class pipeline for cancer and autoimmune diseases with high unmet needs.Dr. Khleif stated, "Georgiamune is a discovery and early clinical development biotech company focused on transforming immunotherapy by reprogramming the immune system for the treatment of cancer and autoimmune diseases. The acceptance of our first IND is a testament to the hard work and dedication of our talented team, and brings us closer to fulfilling our mission of transforming cancer patient care."

In addition to the IND acceptance, Georgiamune has successfully closed an oversubscribed $75M Series A financing round, securing substantial financial support from leading biotech investors and venture capital firms. General Catalyst and Parker Institute for Cancer Immunotherapy (PICI) co-led the round. Mubadala Capital, Alexandria Venture Investments, Catalio Capital Management, CJNV BioVenture, and Verition Fund Management also participated.

"Georgiamune’s therapeutic approach to unlock and reprogram the immune system seeks to enable patients to recover from cancer or autoimmune diseases, and is a remarkable example of leveraging nature’s mechanisms for improved healthcare solutions, a core part of our Health Assurance thesis," said Deep Nishar, Managing Director at General Catalyst. "We are proud to support Georgiamune in getting its novel therapies to patients worldwide, in what we believe will help people live higher quality and longer lives."

"PICI is proud to support Georgiamune in bringing this novel technology to cancer patients worldwide," said John Connolly, PhD, Chief Scientific Officer at PICI. "Georgiamune’s approach of leveraging fundamental discoveries in next generation checkpoint blockade to develop novel immuno-oncology treatments is directly in line with PICI’s mission to accelerate breakthrough therapies to patients."

"Georgiamune’s innovative therapeutic approach targets critical challenges in immunotherapy, adeptly tuning the immune system to reestablish equilibrium in cancer and autoimmune conditions and presenting a promising way forward for patients with high unmet needs," said Ayman AlAbdallah, Partner at Mubadala Capital. "We are proud to partner with the Georgiamune team as they emerge from a period of intense research and development and advance several groundbreaking preclinical and clinical programs."

On August 9, 2023 Researchers at City of Hope, one of the largest cancer research and treatment organizations in the United States and a leading research center for diabetes and other life-threatening illnesses, reported that they have been awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel Phase 1 clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia and severe aplastic anemia (Press release, City of Hope, AUG 9, 2023, View Source [SID1234634108]).

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Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel Phase 1 clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia and severe aplastic anemia. (Photo credit: City of Hope)
Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel Phase 1 clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia and severe aplastic anemia. (Photo credit: City of Hope)
Beckman Research Institute of City of Hope was awarded two-thirds of the $50 million in this latest round of CIRM awards, the highest of any entity in the state.

"These CIRM awards are a testament to City of Hope’s commitment to the advancement of gene therapies for the treatment of human diseases," said John D. Carpten, Ph.D., City of Hope’s chief scientific officer, Irell & Manella Cancer Center Director’s Distinguished Chair and Morgan & Helen Chu Director’s Chair of the Beckman Research Institute. "Through our tireless pursuit of best-in-class research and innovation, we believe our work at City of Hope will benefit patients living with HIV or impacted by cancer, and we thank CIRM for its support to help make this a reality."

City of Hope is a leader in gene therapies, including bone marrow and blood stem cells transplants. As one of the highest volume transplant centers in the nation and the largest in California, more than 18,000 transplants have been performed at City of Hope since it became one of the first cancer centers in the U.S. to perform the therapy in 1976. City of Hope and other institutions also developed a pretransplant treatment for older adults and other patients that resulted in the fifth patient in the world — and the oldest — to achieve long-term remission for leukemia and HIV. (The patient had also received stem cells from a donor with a rare genetic mutation that makes people resistant to most HIV strains.)

Potential bispecific CAR T therapy for HIV

City of Hope is at the forefront of another cellular therapy known as chimeric antigen receptor therapy (CAR) T cell therapy, which uses a patient’s own immune system to fight cancer and has the potential to target other diseases, such as HIV.

More than 80 CAR T and other immune effector cell trials have been conducted to date at City of Hope and over 1,200 patients have been treated with CAR T therapy, either in clinical trials, which may use CARs developed by City or Hope, or Food & Drug Administration-approved CAR T cell therapies.

"City of Hope made a major advancement when our transplant team helped Paul Edmonds, globally known as the ‘City of Hope patient,’ go into remission for both HIV and leukemia," said John A. Zaia, M.D., the Aaron D. Miller and Edith Miller Chair for Gene Therapy at City of Hope. "We are hoping to evaluate a CAR T therapy for HIV in a Phase 1 clinical trial so that one day more people with HIV might be able to achieve long-term HIV remission."

CIRM awarded Zaia $11.3 million for the clinical trial. Up to 12 healthy people with HIV will be treated in this first-in-human study performed at City of Hope and the University of California San Diego. The investigators include John Baird, M.D., Angelo Cardoso, M.D., Ph.D., Joycelynne Palmer, Ph.D., and Xiuli Wang, Ph.D., at City of Hope and colleagues at UCSD.

Zaia’s team will test a novel approach City of Hope scientists developed where a patient’s immune T cells are engineered to target both the HIV antigen gp120 and a common virus that infects nearly all persons living with HIV, namely cytomegalovirus. The researchers’ aim is for these bispecific HIV-CAR T cells to persist and eliminate HIV-infected cells so that antiviral drugs are no longer needed.

Trial to focus on a difficult blood cancer to treat

CIRM awarded a City of Hope team led by Elizabeth Budde, M.D., Ph.D., nearly $12 million to develop and conduct a first-in-human Phase 1 immunotherapy trial for acute myeloid leukemia (AML), which has the highest mortality rate of all blood cancers.

"It is critical to find more effective therapies for patients whose AML has relapsed," said Budde, a City of Hope associate professor in the Department of Hematology & Hematopoietic Cell Transplantation. "We are hopeful that this novel therapy will eventually enable City of Hope and other institutions to offer a new treatment for patients whose AML has returned. We are very grateful for the recognition and support from CIRM."

Most AML patients who go into remission after one treatment will develop the disease again and will then need a blood stem cell transplant from a donor. Even after a transplant, patients can still relapse, leaving them with few treatment options.

The CIRM-funded trial will be the first to use T cells, a type of immune cell, from a healthy donor who has donated stem cells for the AML patient to undergo a transplant. These cells will be reengineered in a laboratory to express CD33CARs, or special receptors on a cell’s surface.

These CARs will target the CD33 protein, which is found in more than 80% of AML cases. T cells harboring the CD33CARs will also be multiplied to millions and then infused back into the patient. The CARs are living cells and expected to multiply inside a patient’s body and recognize and kill the cancer cells.

Before receiving the CAR T cell therapy, patients will receive chemotherapy to prepare their bodies to accept the CAR T cells.

The trial, which will enroll up to 18 AML patients who have relapsed after receiving a transplant, will evaluate the CAR T therapy’s safety and its effectiveness in eliminating leukemia cells.

City of Hope’s David Horne, Ph.D., Marissa Del Real, Ph.D., Karam Sandhu, M.D., and Anthony Stein, M.D., are co-investigators on this project.

Finding a better treatment for severe aplastic anemia

A City of Hope research team received $9.05 million from CIRM to start a clinical trial using a novel blood stem cell transplantation procedure for severe aplastic anemia, a rare bone marrow disease that can turn into blood cancer and is currently difficult to treat. If successful, the treatment could also be used for other autoimmune diseases, such as treatment-resistant type 1 diabetes.

Currently, a blood stem cell transplant from a related or unrelated donor whose human leukocyte antigen (HLA) genes match a patient’s HLA is the most effective treatment for severe aplastic anemia. But it is difficult to find unrelated donor matches for people of color due to a limited number of donors from various racial and ethnic groups. Transplants can also cause severe side effects, particularly in older patients.

Defu Zeng, Ph.D., a City of Hope professor with the Department of Immunology & Theranostics within the Arthur Riggs Diabetes & Metabolism Research Institute, has led research on the potential new therapy, which involves induction of mixed chimerism in adult patients. Mixed chimerism refers to transplanting blood-forming stem cells from a healthy, half-matched family member donor into a patient who has received a milder, less toxic chemotherapy treatment free of radiation that removes some, but not all, of the patient’s diseased bone marrow stem cells.

The Phase 1 trial is expected to start later this year and will enroll up to six patients.

"City of Hope has extensive expertise performing bone marrow and stem cell transplants for patients with blood cancers and other diseases, and we are consistently looking for ways to improve the process and effectively treat more patients with transplants, regardless of any barriers," said Ryotaro Nakamura, City of Hope’s Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation in the Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, and the trial’s principal investigator. "We are hopeful that this will provide another treatment option for patients with this devastating disease and thankful for CIRM’s funding."

City of Hope has treated patients with sickle cell diseases using this novel therapy in a clinical trial also funded by CIRM.

On August 9, 2023 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported that its President and CEO, Robert E. Hoffman will present in the Emerging Growth Conference on Wednesday, August 9, 2023 at 12:00 pm ET (Press release, Kintara Therapeutics, AUG 9, 2023, View Source [SID1234634107]).

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On August 9, 2023 Photocure ASA (OSE: PHO) reported Hexvix/Cysview revenues of NOK 115.9 million in the second quarter of 2023 (Q2 2022: NOK 99.9 million), and an EBITDA of NOK 23.4 million (NOK 1.4 million). The Company reiterates its 2023 annual guidance of new Saphira blue light tower installations in the U.S. expected in the range of 65 to 75, anticipated consolidated product revenue growth above 20%, and positive EBITDA excluding business development spending (Press release, PhotoCure, AUG 9, 2023, View Source [SID1234634106]).

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"In the second quarter of 2023, Hexvix/Cysview revenue increased 16% year over year driven by higher unit sales in the U.S., price increases and foreign exchange. We generated over NOK 23 million in EBITDA, including a positive contribution from our commercial business and a milestone payment from Asieris related to the clinical development of Cevira. During the second quarter, we delivered 4% sequential unit sales growth in North America and 6% in Europe, demonstrating continued positive traction in the region," says Dan Schneider, President & Chief Executive Officer of Photocure.

Photocure reported total group revenues of NOK 144.3 million in the second quarter of 2023 (NOK 100.6 million), and an EBITDA* of NOK 23.4 million (NOK 1.4 million). Hexvix/Cysview revenues ended at NOK 115.9 million in the quarter (Q2 2022: NOK 99.9 million), and the total group revenues includes a milestone payment related to the development of Cevira by Asieris of NOK 26.9 million. The EBIT was NOK 16.8 million (NOK -4.6 million), and the cash balance at the end of the period was NOK 258.9 million. Photocure paid off the balance of its term debt during the second quarter of 2023.

At the end of the second quarter of 2023, the total installed base of rigid blue light cystoscopes (BLC) in the U.S. was 327, an increase of 21% or 56 towers since the same period in 2022. These figures do not include the 69 flexible cystoscopy towers that were installed in the U.S. as of December 31, 2022. In early 2023, Karl Storz announced that it will no longer sell flexible BLC equipment for use in the surveillance setting and will cease servicing existing units when the availability of replacement parts has been exhausted, anticipated in late 2023. Despite Karl Storz’ decision to discontinue its flexible BLC equipment, surveillance of bladder cancer patients with flexible BLC remains a strategic priority for Photocure, and the Company plans to pursue initiatives to restore this specialized equipment in the U.S. and in international markets.

"Offsetting the loss in flexible BLC equipment, the installed base of rigid blue light capital equipment expanded once again, with 13 new Saphira towers installed in Q2. Eight new placements and five upgrades were installed prior to Karl Storz’s recent promotional discount program which went into effect in July and shifted the timing of some tower installations into the third quarter. In July alone, 40 quotes were issued for new Saphira systems, and so far in Q3, 12 purchase orders have been received. We believe that this momentum will result in a strong second half of the year for new tower placements," Schneider adds.

Photocure reiterates its guidance for 2023 and continues to expect new Saphira blue light tower installations in the range of 65 to 75, consolidated product revenue growth above 20%, and positive EBITDA excluding business development spending.

"I remain confident in Photocure’s future and our ability to bring Hexvix/Cysview to more patients and establish Blue Light Cystoscopy as standard of care. The new technology cycle of high-definition blue light equipment from multiple manufacturers is expected to significantly increase the use of Hexvix/Cysview. We continue to await information from Karl Storz and the FDA regarding the Citizen’s Petition to re-classify BLC as a Class 2 device, which we believe will enable additional equipment manufacturers to enter the U.S. market and build critical mass behind BLC. Given our prior success in creating and accelerating the use of BLC in the large untapped bladder cancer surveillance market, we are pursuing a strategy to reintroduce the technology in the near to intermediate future. For the remainder of the year, we will focus on driving sales in the rigid cystoscopy setting, and we look forward to several milestone events including the anticipated Phase 3 results for Hexvix in China and Cevira with our partner Asieris," Schneider concludes.

Photocure also announced today that Jan H. Egberts, M.D. has decided to step down from his position as Chairperson of the Company’s Board of Directors due to increased time commitments to his personal companies. Dr. Egberts will leave the Board in September 2023, and the Nomination Committee has initiated a search for a new Chairperson.

Robert Blatt, Chair of Photocure’s Nomination Committee, said, "I would like to express my gratitude to Jan, who has made important contributions to Photocure during his more than six years as Chairperson of the Board. His dedication and medical experience will be missed".

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 second quarter and first half 2023 financial report on page 23.

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

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/hegnarmedia/#!/hegnarmedia/20230809_1

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

On August 9, 2023 Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for genetically defined cancers with alterations in mTOR pathway genes, reported a corporate update and announced financial results for the second quarter of 2023 (Press release, Aadi Bioscience, AUG 9, 2023, View Source [SID1234634105]).

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"We are seeing continued growth in FYARRO sales and are pleased with the positive feedback we are receiving from the treatment community," said Scott Giacobello, Interim CEO and President and CFO of Aadi. "The PRECISION1 trial is progressing well, and we are looking forward to providing results of an interim analysis on 40 patients with appropriate follow-up before the end of the year. We expect to complete enrollment as planned in the spring of 2024."

"With PRECISION1 on track, we are excited to announce the expansion of our pipeline to further investigate mTOR pathway inhibition in endometrial cancer and NETs," continued Giacobello. "We are encouraged by the positive feedback received from clinicians, and we believe our preclinical data supporting these programs is promising. We look forward to harnessing the unique pharmacology of nab-sirolimus, both in combination and as monotherapy, to provide enhanced therapeutic benefit in these indications with meaningful patient populations and high unmet need."

Second Quarter 2023 Updates and Recent Operational Highlights

Interim analysis from PRECISION1 expected before the end of 2023. An interim analysis from the tumor agnostic PRECISION1 trial on 40 patients with appropriate follow-up is on track with data expected before the end of 2023. Enrollment continues to be well-balanced between the two arms, and as previously reported, more than 15 discrete tumor types have been enrolled, supporting the thesis that TSC1 and TSC2 alterations occur broadly across different solid tumors.
FYARRO net product sales were $6.2 million in the second quarter, reflecting growth of approximately 6% over Q1 2023 and 80% over the prior year quarter.
Planned initiation of Phase 2 combination trial in endometrial cancer. The Company is initiating a Phase 2 trial investigating the combination of nab-sirolimus with letrozole for the treatment of advanced or recurrent endometrioid-type endometrial cancer (EEC). This is a Phase 2 open-label, multi-institutional study to evaluate the efficacy and safety of nab-sirolimus and letrozole in patients with advanced or recurrent endometrioid endometrial carcinoma. Prior clinical studies with mTOR inhibitors and letrozole in endometrial cancer patients have yielded promising results. In preclinical models, intravenous nab-sirolimus demonstrates significantly higher tumor growth inhibition, intra-tumoral drug accumulation, and greater mTOR target suppression compared with oral inhibitors. The Company is exploring whether the combination of nab-sirolimus with endocrine therapy may produce synergistic anti-tumor activity in patients with EEC. Initiation of this trial is expected in the fourth quarter of 2023.
Planned initiation of Phase 2 study in neuroendocrine tumors (NETs). The Phase 2 study is a multicenter, open-label, single-arm trial that is evaluating adult patients with functional or non-functional, well-differentiated, locally advanced unresectable or metastatic NETs of the GI tract, lung, or pancreas who have received no more than two prior lines of therapy. In preclinical animal models, nab-sirolimus demonstrated improved target suppression relative to similar weekly doses of sirolimus and everolimus, supporting further exploration of nab-sirolimus in NETs. Initiation of this trial is expected in the fourth quarter of 2023.
Initiated Phase 1/2 trial in KRASG12C in collaboration with Mirati Therapeutics. The first patient has been dosed in a Phase 1/2 trial evaluating the combination of adagrasib with nab-sirolimus in this collaborative study. The open-label Phase 1/2 trial is intended to determine the optimal dose and recommended Phase 2 dose in patients with KRASG12C mutant solid tumors.
Multiple posters presented at ASCO (Free ASCO Whitepaper) Annual Meeting 2023. Aadi presented a company-sponsored TIP update from the PRECISION1 Phase 2 study and combination data of nab-sirolimus and pazopanib (PAZO) from an ongoing Investigator Initiated Trial at ASCO (Free ASCO Whitepaper) 2023. The posters are available on the investor relations page of the Aadi website at www.aadibio.com.
Second Quarter 2023 Financial Results

Total revenue resulting from sales of FYARRO for the quarter ended June 30, 2023, was $6.2 million. This compares to the prior year period of $3.4 million.
Cash, cash equivalents and short-term investments as of June 30, 2023, were $134.9 million as compared to $172.6 million as of December 31, 2022, which is expected to fund operations into 2025 based on current plans.
Net loss for the three months ended June 30, 2023, was $18.0 million as compared to $18.3 million for the three months ended June 30, 2022.
Conference Call Information

The Aadi management team is hosting a conference call and webcast today at 8:30 am ET (5:30 am PT) to provide a corporate update and discuss results for the second quarter 2023.

Participants may access a live webcast of the call on the "Investors & News" page of the Aadi Bioscience website at aadibio.com. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company’s website for at least 30 days.

About FYARRO

FYARRO is an mTOR inhibitor indicated for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

About the PRECISION1 Trial

The PRECISION1 trial is a multi-center, open-label, tumor-agnostic registrational clinical trial of nab-sirolimus. This tumor agnostic study will evaluate approximately 60 mTOR inhibitor naïve patients in each of two independent study arms, or approximately 120 in total, comprised of patients with solid tumors harboring pathogenic inactivating alterations in either TSC1 or TSC2 genes. In November 2021, the FDA granted Fast Track designation to evaluate nab-sirolimus for this patient population.

nab-Sirolimus 100 mg/m2 is given weekly intravenously over 30 minutes on Days 1 and 8 of each 21-day cycle. The primary endpoint is overall response rate per independent radiographic review (IRR) using RECIST v1.1. Other endpoints include duration of response, time to response, progression-free survival by IRR, overall survival, patient-reported quality of life, and safety.