Alpha Tau Medical to Present at the Emerging Growth Conference on August 9th, 2023

On August 2, 2023 Alpha Tau Medical Ltd. ("Alpha Tau", or the "Company") (NASDAQ: DRTS, DRTSW), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported that Raphi Levy, Chief Financial Officer, will present at the Emerging Growth Conference on August 9th, 2023 (Press release, Alpha Tau Medical, AUG 2, 2023, View Source [SID1234633637]).

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Event: Emerging Growth Conference
Date: Wednesday, August 9th, 2023
Time: 11:25-11:55 a.m. ET
Location: Virtual

If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available on EmergingGrowth.com and on the Emerging Growth YouTube Channel, View Source

Allogene Therapeutics Reports Second Quarter 2023 Financial Results and Business Update

On August 2, 2023 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported a corporate update and announced financial results for the quarter ended June 30, 2023 (Press release, Allogene, AUG 2, 2023, View Source [SID1234633636]).

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"The last few months have been exciting for cell therapy as the transformative potential of an allogeneic CAR T product becomes more evident. Within this framework, we are thrilled that our off-the-shelf CD19 AlloCAR T data continues to demonstrate what I believe to be both first-in-class and best-in class promise in hematological cancers," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "We remain focused on advancing the industry’s first potentially pivotal allogeneic CAR T trials in order to enable more patients to receive cell therapy."

Pipeline Updates

ALLO-501A: Anti-CD19 AlloCAR T Program
The Company is enrolling patients in the industry’s first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A across sites in the United States and Canada. The European Medicines Agency (EMA) recently approved the ALPHA2 Clinical Trial Application (CTA) and patient enrollment in Europe is expected to begin in Q3 2023 and in Australia by year-end.

The single-arm ALPHA2 trial in relapsed/refractory (R/R) large B cell lymphoma (LBCL) utilizes a single dose of ALLO-501A (120 million CAR+ cells) following lymphodepletion with FCA90 (fludarabine, 30 mg/m2; cyclophosphamide 300 mg/m2; and ALLO-647 30 mg, daily for 3 days). This trial will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. The primary endpoint is overall response rate (ORR), and the key secondary endpoint is duration of response (DoR). Patients may receive treatment as an outpatient at the investigator’s discretion. The Company expects to complete enrollment in 1H 2024 with the first data readout by the end of 2024.

Long-term follow up data from the Phase 1 ALPHA/ALPHA2 trials in LBCL was presented at both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting with an encore presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, and the International Conference on Malignant Lymphoma (ICML) Lugano in June 2023. The Phase 1 trials enrolled heavily pre-treated patients with a median of three prior lines of therapy. Data from 33 CAR T-naïve LBCL patients receiving Alloy cell product including 12 patients treated with the Phase 2 regimen, are the first to demonstrate the potential for an allogeneic CAR T product to induce complete responses at rates and durability similar to approved autologous therapies. Treatment with ALLO-501/501A was generally well tolerated with no incidence of Grade 3 or greater cytokine release syndrome, and no cases of immune effector cell-associated neurotoxicity syndrome or graft versus host disease. Cytopenia and infections were manageable and comparable to the experience with autologous CAR T cell therapies in patients with r/r LBCL.

The EXPAND trial is also underway to support licensure of ALLO-647, the Company’s anti-CD52 monoclonal antibody used in conjunction with standard low-dose FC (fludarabine, 30 mg/m2 and cyclophosphamide 300 mg/m2, daily for 3 days) lymphodepletion regimens to control premature rejection of AlloCAR T cells by the patient’s immune system. At the ASCO (Free ASCO Whitepaper) and Lugano data presentations, the Company’s proprietary lymphodepletion strategy was shown to promote robust AlloCAR T cell expansion and best-in-class longest persistence without incurring significant changes in infectious or immunosuppressive complications as compared to autologous CAR T therapies.

The EXPAND trial, which is designed to demonstrate the contribution of ALLO-647 to the standard low dose FC lymphodepletion, will enroll approximately 70 patients with r/r LBCL who will be randomized to lymphodepletion with FCA90 (which includes 90 mg of ALLO-647) versus FC alone before receiving a single 120 million cell dose of ALLO-501A. The primary endpoint of the study is progression free survival (PFS).

ALLO-316: Anti-CD70 AlloCAR T Program
The ongoing Phase 1 dose escalation TRAVERSE study is enrolling patients with advanced or metastatic renal cell carcinoma (RCC) who have progressed on standard therapies including an immune checkpoint inhibitor and a VEGF-targeting therapy. Initial data from TRAVERSE were presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) conference in April and demonstrated the potential of an allogeneic CAR T product to treat CD70 expressing RCC. In this trial, ALLO-316 showed early anti-tumor activity with deepening responses over time and a trend toward greater tumor shrinkage in patients with higher levels of CD70 expression.

The Dagger effect, which is a feature of ALLO-316, enables ALLO-316 CAR T cells to target and eliminate alloreactive host immune cells, thereby mitigating potential premature rejection of AlloCAR T cells by the patient’s immune system. Translational results shared at AACR (Free AACR Whitepaper) suggest this unique immunomodulatory effect of ALLO-316 contributed to robust AlloCAR T cell expansion and persistence, and clinical remissions.

The TRAVERSE trial is now deploying an investigational in vitro companion diagnostic (IVD) assay designed to prospectively assess CD70 expression levels in patients. Dose escalation in the TRAVERSE trial is expected to be completed in 2023.

Based on preclinical results demonstrating the ability to combine anti-CD19 and other AlloCARs with the Dagger technology, the Company intends to explore this approach to potentially enhance the activity of next generation AlloCAR T products candidates, including those that target other hematological and solid tumors.

ALLO-715: Anti-BCMA AlloCAR T Program
The Company previously presented ALLO-715 Phase 1 data from the UNIVERSAL trial which was the first study to demonstrate that an allogeneic anti-BCMA CAR T could produce response rates in multiple myeloma similar to an approved autologous CAR T therapy. As treatments in multiple myeloma advance, the Company is evaluating manufacturing process improvement across its BCMA candidates to achieve an improved competitive profile.

Second Quarter Financial Results

The Company had $544.5 million in cash, cash equivalents, and investments as of June 30, 2023, which includes net proceeds of approximately $87.9 million raised in the second quarter from an at-the market (ATM) equity financing facility. Based on current expectations, the Company expects its cash runway to fund operations into 2H 2025.
Research and development expenses were $62.0 million for the second quarter of 2023, which includes $6.9 million of non-cash stock-based compensation expense.
General and administrative expenses were $18.5 million for the second quarter of 2023, which includes $9.7 million of non-cash stock-based compensation expense.
Net loss for the second quarter of 2023 was $78.0 million, or $0.53 per share, including non-cash stock-based compensation expense of $16.6 million.
2023 Financial Guidance

As previously reported, the Company expects a decrease in cash, cash equivalents, and investments of approximately $230 million in 2023. GAAP Operating Expenses are expected to be approximately $340 million, including estimated non-cash stock-based compensation expense of approximately $80 million. These estimates exclude any impact from potential business development activities.

Conference Call and Webcast Details

Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

ACCENT Trial Recruitment Update

On August 2, 2023 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), reported an update on progress in the ongoing Phase 1b/2a ACCENT clinical trial in first-line patients with advanced pancreatic cancer (Press release, Amplia Therapeutics, AUG 2, 2023, View Source;[email protected] [SID1234633606]).

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The current Phase 1b stage of the ACCENT trial is designed to test ascending doses of AMP945 in patients, given in combination with gemcitabine and nab-paclitaxel. The dose-escalation stage will cease, and a recommended dose for the Phase 2a stage of the trial identified, when either two or more dose-limiting toxicities (DLTs) are observed and/or when the pharmacokinetic and pharmacodynamic profiles are predicted to be optimised.

We are currently dosing the third cohort of patients in the dose-escalation phase of the trial. At this time 12 patients have been dosed across the 3 cohorts and have completed their first full cycle of treatment (28 days). Notably all patients, in consultation with their physicians, elected to stay on the study drug after completion of the first cycle of treatment. A DLT signal was reported in one patient in the current cohort, and consistent with the trial protocol, we expanded this third cohort with an additional three patients. We are now pleased to report the final patient in this cohort has been recruited and began dosing yesterday. Importantly, all other patients in this cohort have completed their first full 28-day cycle and continue to remain on drug at this dose. In line with the study protocol, we will continue to collect the data for this expanded cohort to determine whether further doseescalation is warranted. The combined data will be reviewed by the Safety Committee once all patients in this cohort have been dosed for a complete 28-day cycle.

Amplia’s CEO and Managing Director Dr Chris Burns commented: "While the cohort expansion has resulted in a delay for the complete data from this cohort to be reviewed by the safety committee, we continue to be encouraged by the data reported from all trial sites. We are grateful for the patients who are participating in the trial, and I look forward to reporting the outcomes from the Safety Committee meeting next month."

About the ACCENT Trial

The protocol for the ACCENT trial is entitled ‘A Phase 1b/2a, Multicentre, Open Label Study of the Pharmacokinetics, Safety and Efficacy of AMP945 in Combination with Nab-paclitaxel and Gemcitabine in Pancreatic Cancer Patients’.

The trial is a single-arm open label study conducted in two stages. The first, Phase 1b stage of the trial, will determine an optimal dose of AMP945 by assessing the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of AMP945 when dosed in combination with gemcitabine and nab-paclitaxel (Abraxane) in first-line patients with advanced pancreatic cancer.

The second, Phase 2a, stage of the trial is designed to perform an assessment of the optimal dose of AMP945, in combination with gemcitabine and nab-paclitaxel, with the primary endpoint being Objective Response Rate (ORR). Further endpoints will assess efficacy by other means as well as safety and tolerability. More information about the ACCENT trial, including a list of participating sites, can be found via our website and at ClinicalTrials.gov under the identifier NCT05355298. The Company will provide further updates on the trial as recruitment proceeds.

This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics.

OSE Immunotherapeutics Announces New Public Grant to Support Innovation and Research Programs in the Field of RNA Therapeutics

On August 1, 2023 OSE Immunotherapeutics reported that the Company has received €200,000 grant innovation funding from the French Government and Region Pays de la Loire as part of the "France 2030 investment plan" operated by Bpifrance (Press release, OSE Immunotherapeutics, AUG 1, 2023, View Source [SID1234646942]).

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Nicolas Poirier, Chief Executive Officer of OSE Immunotherapeutics, comments:
"We thank the French government, the "Region Pays de la Loire" and Bpifrance to help OSE in developing next future generation immunotherapies based on the unique advantages that RNA Therapeutics could offer as a novel first-in-class modality. Exciting progress is being made to explore and define new frontiers of science in RNA-based medicines paving the way for RNA therapeutic-based immunotherapies targeting diseases that cannot be treated with other conventional drug groups. OSE is building a first-in-kind research platform at the intersection of Antibody Engineering, Data Science, Artificial Intelligence (AI) regularly used by the OSE Team and adding now novel RNA Therapeutics and RNA Delivery methods recently patented by OSE to continue to develop next-generation immunotherapy medicines modulating immune cell responses in the field of immuno-inflammation and immuno-oncology. I thank warmly the OSE R&D team moving fast in this new emerging RNA era generating exciting opportunities for future transformative medicines".

Renaissance Pharma launches today and announces the in-licensing of Hu14.18K322A (Hu14.18) from St. Jude Children’s Research Hospital for the treatment of High-Risk Neuroblastoma

On August 1, 2023 Renaissance Pharma Ltd., ("Renaissance" or "the Company"), a rapidly emerging company focused on the development of life changing therapies in paediatric rare disease reported the signing of an exclusive license agreement with St. Jude Children’s Research Hospital ("St. Jude") for Hu14.18, a humanised antibody in development by St. Jude for the treatment of newly diagnosed high-risk neuroblastoma (Press release, Pharma, AUG 1, 2023, View Source [SID1234642049]).

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Neuroblastoma represents 8-10% of all childhood cancers 1, is the second most common cancer in children and is the most common cancer in children under one year of age2. 50% of neuroblastoma patients have high-risk (HRNB) disease which has an overall survival of ~50% over 5 years3. Each year more than 500 HRNB patients are diagnosed in Europe and 300 in the US. The current standard of care includes multiple cycles of chemotherapy, surgery, radiotherapy, stem cell transplantation and anti-GD2 monoclonal antibody (mAb) treatment.

Hu14.18 is an anti GD2 humanised mAb. A novel Phase II trial incorporating Hu14.18 into induction therapy and additionally, within post-consolidation therapy, demonstrated outstanding patient outcomes with OS of 86.0% and EFS of 73.7%4. Within the patient population, where the antibody was used in all patients prior to consolidation and maintenance, 66.7% achieved a Partial Response or better after the first two chemo-immunotherapy induction cycles, and 96.8% achieved a Partial Response or better at the end of induction. Data from the successful Phase II study was published in the Journal of Clinical Oncology in December 2021 and will soon reach maturity in regard to its 5-year OS readouts5.

Renaissance Pharma, launched today, is led by Executive Chairman, Lee Morley; Chief Executive Officer, Simon Ball; Chief Medical Officer, Dr Jonathan Morgan; and Chief Technical Officer, Debra Nevin. The Company’s founding team members are highly experienced with extremely relevant and proven track records across orphan disease states, antibody technologies, oncology and paediatric therapeutic areas.

Under the terms of the licensing agreement, Renaissance Pharma has secured exclusive development, manufacturing and commercialisation rights to US, Canada, Europe, China, Japan and Turkey.

Renaissance will now pursue interaction with the FDA, EMA and other Regulatory Agencies internationally to configure the optimal path for Hu14.18’s submission.

Lee Morley, Executive Chairman of Renaissance Pharma, said: "We couldn’t be more motivated by the prospect of bringing Hu14.18 to patients. The Phase II data as published in the Journal of Oncology is breathtaking and I am certain that the neuroblastoma community will be as excited as we are when considering the potential significance of this breakthrough treatment. Our responsibility is now to continue the journey that St. Jude so professionally began. Our intent is simple, to get Hu14.18 into the hands of treating physicians as swiftly as possible so the potential benefits Hu14.18 offers can be passed on to the patients that so desperately need them. This responsibility begins immediately."

Simon Ball, CEO of Renaissance Pharma, said: "It is rare to have the opportunity to make such a significant improvement in a treatment paradigm; with Hu14.18, we have just that opportunity. Renaissance Pharma now has its full attention on bringing this humanised antibody to market as expeditiously as possible. The collaboration with St. Jude marks the intention of Renaissance and we must now play our part in making the product available."

"It is exciting and deeply meaningful to see how our clinical efforts to develop Hu14.18 may now have the potential to positively impact patients with high-risk neuroblastoma around the world," said Sara M. Federico, MD, St. Jude Department of Oncology.