On May 13, 2024 Applied DNA Sciences, Inc. (NASDAQ: APDN) (Applied DNA), a leader in PCR-based DNA technologies, and the Institute of Hematology and Blood Transfusion (ÚHKT/IHBT) reported that an abstract relating to the development of a fully enzymatic, non-viral manufacturing workflow to enable the rapid and cost-effective production of clinical-grade (GMP) CAR T-cell therapies has been accepted for presentation at the prestigious European Hematology Association (EHA) (Free EHA Whitepaper) 2024 Hybrid Congress to be held in Madrid, Spain from June 13 – 16, 2024 (Press release, Applied DNA Sciences, MAY 13, 2024, View Source [SID1234643131]). Accepted abstracts will be available on the Congress’ website starting on May 14, 2024. This current study extends the successful results previously published on the virus-free preparation of CD19-specific CAR T-cells against refractory B cell malignancies utilizing Applied DNA’s Linea DNA.

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The current study was designed to address the high cost of conventional CAR T-cell therapies, which is primarily due to conventional manufacturing processes that utilize difficult-to-manufacture viral vectors. By generating CD123-specific CAR T-cells in a non-viral workflow via piggyBac transposons made from Linea DNA at substantial and cost-efficient yields with no risk of antibiotic-resistance gene transfer, we believe the study could offer a promising solution for the rapid manufacture of CAR T-cell therapies. IHBT is currently seeking SÚKL-EMA approval to initiate clinical trials using its Linea DNA-enabled CD123-specific CAR T-cells in patients with refractory acute myeloid leukemia.

Details of the oral presentation:
Abstract Title: "Linear DNA Platform For The Non-Viral Point-Of-Care Production Of Car-T Cells"
Abstract Number: P1722
Presenting Author: Dr. Pavel Otáhal

About the Linea DNA and Linea IVT Platforms
The Linea DNA platform is a completely cell-free DNA production platform founded on Applied DNA’s long-standing expertise in the large-scale enzymatic production of DNA. Capable of producing DNA in quantities ranging from milligrams to grams, the Linea DNA platform can produce high-fidelity DNA constructs ranging from 100bp to 20kb in size. The DNA produced via the Linea DNA platform is free of the adventitious DNA sequences found in other sources of DNA, is rapidly scalable, and provides for simple chemical modification of DNA constructs.

The Linea IVT platform combines DNA IVT templates manufacturing via the Linea DNA platform with a proprietary Linea RNAP to enable mRNA and sa-mRNA manufacturers to produce what Applied DNA believes to be better mRNA faster, with advantages over conventional mRNA production, including: 1) the elimination of plasmid DNA as a starting material; 2) the prevention or reduction of double-stranded DNA (dsRNA) contamination; and 3) simplified mRNA production workflows.

Learn more about the Linea DNA and Linea IVT platforms: LineaRx, an Applied DNA Sciences company

On May 13, 2024 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 its first patient with liver cancer metastases has been treated in a feasibility and safety study of Alpha DaRT at the McGill University Health Center in Montreal, Canada (Press release, Alpha Tau Medical, MAY 13, 2024, View Source [SID1234643130]).

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The trial seeks to recruit up to 10 patients who are eligible for a two-staged hepatectomy to resect liver metastases of colorectal cancer. Specifically, in the first stage, as many metastatic tumors as possible will be surgically removed in a non-curative intervention, while Alpha DaRT sources will be inserted into a separate tumor in the second liver lobe, and in the second stage, after allowing for the resected liver to regenerate naturally, the portion of the liver with the Alpha DaRT sources will be surgically removed.

The study primarily aims to examine the feasibility of delivering Alpha DaRT sources into the liver metastases, as well as the safety of utilizing the Alpha DaRT sources in the liver by assessing the frequency and severity of related adverse events. The study also aims to examine Alpha DaRT’s efficacy in terms of radiological response, assessed after insertion of the Alpha DaRT sources, and via pathological response, assessed after the second stage of surgery. Additional information about the trial can be found at View Source

Alpha Tau CEO Uzi Sofer commented, "The start of this trial marks a big milestone towards addressing the urgent need for new therapeutic options in treating patients with liver metastases. This trial is part of our overall strategy to showcase the broad use and potential of the Alpha DaRT in other hard-to-treat indications such as cancers of the brain, lung, vulva and pancreas. We would like to thank Dr. Peter Metrakos and his team at the McGill University Health Center for enrolling and treating the first patient in this highly significant feasibility and safety trial."

Peter Metrakos, MD, CM, FACS, FRCSC, Professor of Surgery, Pathology and Anatomy and Cell Biology, Director of Hepato-Pancreato-Bilary Surgery and Senior Scientist, McGill University Health Centre -Research Institute, Cancer Research Program, Department of Surgery at McGill University, and the principal investigator of the trial, commented "Liver Metastases associated with colorectal cancer is of major concern and a leading cause of patient mortality with limited effective treatment options. We are encouraged by the early results of Alpha DaRT treatment in other tumor types and are hopeful that the Alpha DaRT treatment will offer better outcomes to these patients with such a challenging disease."

Robert Den MD, Alpha Tau CMO commented, "While we continue to accrue patients with cutaneous recurrent SCC to the ongoing pivotal ReSTART trial, we also look to expand the use of the Alpha DaRT technology to high unmet medical need indications in internal organs such as liver metastases. This trial is particularly exciting for us in light of previous preclinical work that has demonstrated the ability of Alpha DaRT to generate an observed systemic immune response when used to treat a colorectal tumor model, and we hope to better understand the effect of Alpha DaRT on liver metastases and immune cell populations in human patients. We look forward to the preliminary results of this trial, which we hope will further our goal of advancing the use of Alpha DaRT across a range of indications and helping patients worldwide."

This clinical study is the next phase of a multi-year research collaboration program between the McGill University Research Center and the Alpha Tau Medical research team. This collaboration started with translation experiments using orthotopic animal models that mimic human liver colorectal metastatic disease, showing a reduction in tumor growth rate when using Alpha DaRT compared to control and changes in the hepatic immune microenvironment.

About Alpha DaRT

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

On May 13, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported the pricing of an underwritten offering of 37,931,035 shares of its common stock at a price of $2.90 per share (Press release, Allogene, MAY 13, 2024, View Source [SID1234643129]). The gross proceeds from this offering are expected to be approximately $110 million, before deducting the underwriting discount and commissions and estimated offering expenses payable by Allogene.

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The offering is anchored by mutual funds and large institutional investors and includes leading healthcare specialists as well as certain members of the Allogene Board of Directors and Executive Management Team. The offering is expected to close on or about May 16, 2024, subject to customary closing conditions.

Goldman Sachs & Co. LLC is acting as sole book-running manager for the offering.

The securities described above are being offered by Allogene pursuant to a shelf registration statement (including a prospectus) filed on March 14, 2024 with the Securities and Exchange Commission (SEC), which was declared effective on April 25, 2024. A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available for free on the SEC’s website at View Source Copies of the final prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained from: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, or by telephone at (866) 471-2526, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On May 13, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported corporate updates, and announced financial results for the quarter ended March 31, 2024 (Press release, Allogene, MAY 13, 2024, View Source [SID1234643128]).

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"We are very proud of the progress we’ve made across our portfolio, in particular the transformative potential of our pivotal ALPHA3 trial with cema-cel which is expected to readout in 2026. Securing the EU and UK rights reinforces our conviction in the cema-cel program," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "We will continue to focus all of our resources on advancing these core programs and believe we are well-positioned to change the CAR T treatment landscape for the benefit of patients."

Key Corporate Updates

Expansion of CD19 Oncology Program Rights
The Company has obtained development and commercialization oncology rights to all EU Member States and the United Kingdom (Extended Territory) for its CD19-directed allogeneic cell therapy products from Servier.

These new expanded rights, combined with the U.S. rights already owned by the Company, substantially increase the potential total market opportunity from more than $6 billion in the U.S. alone to more than $9.5 billion in the combined U.S. and Extended Territory. It is expected that the future multibillion-dollar revenue potential for cema-cel now could increase by 50% with these expanded rights. The Company also has the ability to obtain the development and commercialization rights for cema-cel in Japan and China in the future at no additional cost, subject to Allogene demonstrating the resources to pursue those markets.

Program Updates

Cema-Cel: Pivotal Phase 2 ALPHA3 1L Consolidation Trial in Large B Cell Lymphoma (LBCL)
The Company continues to focus on the development of its investigational product cemacabtagene ansegedleucel, or cema-cel (previously known as ALLO-501A), as part of the first line (1L) treatment plan for LBCL patients who are at risk of relapse following 1L chemoimmunotherapy. The ALPHA3 trial will be conducted in a wide array of cancer treatment centers, including community cancer centers where most earlier line patients seek care.

This innovative trial will identify patients at high risk for relapse after 1L treatment by utilizing a novel and highly accurate test for minimal residual disease, or MRD. This investigational test is being developed by our partners Foresight Diagnostics and aims to offers improved prediction over existing methods for future relapse after completion of 1L treatment. ALPHA3 takes advantage of the allogeneic attributes of cema-cel. With off-the-shelf availability and convenience, cema-cel will be administered as a one-time infusion immediately upon detection of MRD at the completion of six cycles of R-CHOP or other standard 1L chemoimmunotherapy regimen. The outcome of this consolidation treatment could potentially improve the cure rate and uniquely position cema-cel to become the standard "7th cycle" of frontline treatment available to all eligible patients with MRD.

Start-up activities for the ALPHA3 trial are ongoing with a planned study initiation in mid-2024. This randomized study will enroll approximately 240 patients and is designed to demonstrate a meaningful improvement in event free survival (EFS) in patients treated with cema-cel relative to patients who receive the current standard of care (observation). Efficacy analyses are expected to occur in 2026 and will include the Independent Data Safety Monitoring Board (IDSMB) interim EFS analysis in 1H 2026 and the data readout of the primary EFS analysis in 2H 2026 with a Biologics License Application (BLA) submission targeted for 2027.

Cema-Cel: Phase 1 Trial in Chronic Lymphocytic Leukemia (CLL)
Enrollment is ongoing in the relapsed/refractory (r/r) CLL cohort of the Phase 1 ALPHA2 trial of cema-cel. While recent approval of an autologous CD19 CAR T therapy has been a positive step for patients with r/r CLL T cell dysfunction, and high circulating leukemia burden often found in patients with CLL make the isolation of functional T cells for autologous CAR T manufacturing difficult. This trial has been driven by investigator enthusiasm for an allogeneic CAR T to potentially boost the curative power of CAR T.

Initial data readout from the Phase 1 ALPHA2 CLL cohort (n=12) is projected by year-end 2024 with a Phase 2 pivotal study expected in 2025.

ALLO-329: CD19/CD70 Dual CAR with Dagger Technology in Autoimmune Disease (AID)
The Company has applied its deep understanding of CAR T research and development to design next-generation allogeneic CAR T investigational products that could sustain the scale of the AID market while also meeting the unique requirements for these patients.

ALLO-329, the Company’s CRISPR-based AlloCAR T investigational product for AID, incorporates the Dagger technology which is intended to eliminate the need for lymphodepletion while targeting CD19+ B-cells and CD70+ activated T-cells, both of which are likely to play a role in AID.

The Company plans to file an Investigational New Drug (IND) application in Q1 2025 and expects to have proof-of-concept by YE 2025.

ALLO-316: TRAVERSE Trial in Renal Cell Carcinoma (RCC)
In April 2024, the Company announced a $15 million grant from the California Institute for Regenerative Medicine (CIRM) to support the ongoing TRAVERSE trial with ALLO-316 in RCC.

The Company has developed and implemented a diagnostic and treatment algorithm in the TRAVERSE trial that may mitigate the treatment-associated hyperinflammatory response without compromising the CAR T function needed to eradicate solid tumors. This builds upon the field’s understanding of how certain drugs can act as a "safety key" to mitigate hyperinflammatory response without compromising CAR T function or efficacy.

Details on this potentially cornerstone discovery in the Phase 1 TRAVERSE trial are planned for release Q2 2024. A Phase 1 data update from approximately 20 patients with CD70 positive RCC is planned by YE 2024.

2024 First Quarter Financial Results
•Research and development expenses were $52.3 million for the first quarter of 2024, which includes $3.8 million of non-cash stock-based compensation expense.
•General and administrative expenses were $17.3 million for the first quarter of 2024, which includes $8.1 million of non-cash stock-based compensation expense.
•Net loss for the first quarter of 2024 was $65.0 million, or $0.38 per share, including non-cash stock-based compensation expense of $11.9 million.
•The Company had $397.3 million in cash, cash equivalents, and investments as of March 31, 2024.

Based on the cash runway as of March 31, 2024, the Company expects its cash runway to fund operations into 2026. The Company expects a decrease in cash, cash equivalents, and investments of approximately $200 million in 2024. GAAP

Operating Expenses are expected to be approximately $300 million, including estimated non-cash stock-based compensation expense of approximately $60 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.

About Cemacabtagene Ansegedleucel (Previously Known as ALLO-501A) Cemacabtagene ansegedleucel, or cema-cel is a next generation anti-CD19 AlloCAR T investigational product for the treatment of large B cell lymphoma (LBCL). The ALPHA3 pivotal Phase 2 trial in first line (1L) consolidation for the treatment of LBCL is expected to begin mid-2024. In June 2022, the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to cema-cel in third line (3L) r/r LBCL.

On May 13, 2024 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported its financial results for the first quarter ending March 31, 2024 (Press release, Akoya Biosciences, MAY 13, 2024, View Source [SID1234643127]).

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Business Highlights

● Revenue was $18.4 million in the first quarter of 2024, compared to $21.4 million in the prior year period; a decrease of 14%. Reagents and services revenue continued to increase.
● Akoya announced the establishment of a new Manufacturing Center of Excellence in Marlborough, Massachusetts to scale internal reagent manufacturing to meet the accelerating demand for reagents.
● Akoya’s partner Acrivon Therapeutics presented initial positive Phase 2b clinical data for the ACR-368 therapeutic in patients positive for the ACR-368 OncoSignature Assay in ovarian and endometrial cancer, deployed on the PhenoImager HT platform.
● Akoya and NeraCare, a leading developer of laboratory tests for the prognosis of melanoma patients, announced an exclusive partnership to enable personalized therapy selection for early-stage melanoma patients at high risk of relapse and death.
● Akoya and Shanghai KR Pharmtech announced that the KR-HT5 instrument, based on the PhenoImager HT, has received premarket approval from China’s National Medical Products Administration (NMPA).

"While we made meaningful progress in advancing both our operational and clinical objectives, our first quarter results fell short of expectations due to three main factors. First, systemic pressure on capital expenditures persisted. Second, certain pharmaceutical partner lab services revenue were deferred to the second half of 2024 due to revised clinical trial milestones. Third, the completion and launch of our fully operational Manufacturing Center of Excellence temporarily impacted reagent fulfillment times, delaying instrument purchases," said Brian McKelligon, CEO of Akoya Biosciences. "With the industry’s leading installed base, normalization of pharmaceutical partner revenue in the second half of 2024, and now resolved reagent availability issues, we believe that our foundational initiatives will lead us back to strong top-line growth and achievement of our profitability objectives."

First Quarter 2024 Financial Results

● Revenue was $18.4 million in the first quarter of 2024, compared to $21.4 million in the prior year period; a decrease of 14%. Reagents and services revenue continued to increase.
● For the first quarter of 2024, reported gross margin was 46% while non-GAAP adjusted gross margin was 57% when excluding the write off from discontinued legacy instruments. Both GAAP and non-GAAP adjusted gross margin were 57% for the first quarter of 2023.
● For the first quarter of 2024, operating expenses were $30.0 million and non-GAAP operating expenses were $25.6 million when excluding an impairment charge for facility consolidation and restructuring associated with a reduction in force in January. Both GAAP and non-GAAP operating expenses were $29.7 million for the first quarter of 2023.
● For the first quarter of 2024, loss from operations was $21.6 million and non-GAAP loss from operations was $15.2 million, excluding the items noted above. Both GAAP and non-GAAP loss from operations were $17.4 million for the first quarter of 2023.
● Ended the quarter with an instrument installed base of 1,213 (354 PhenoCyclers, 859 PhenoImagers), a year-over-year increase of 22% compared to an installed base of 992 in the prior year period (273 PhenoCyclers, 719 PhenoImagers).
● 1,307 total publications citing Akoya’s technology as of March 31, 2024, compared to 860 total publications in the prior year period: an increase of 52%.
● $61.6 million of cash, cash equivalents and marketable securities as of March 31, 2024.

2024 Financial Outlook

Akoya is updating its revenue outlook for the full year 2024 while maintaining its commitment to achieving operating cash flow breakeven by year end. The Company now expects full year 2024 revenue to be in the range of $104.0-$112.0 million.

Webcast and Conference Call Details

Akoya will host a conference call today, May 13, 2024, at 5:00 p.m. Eastern Time to discuss its first quarter 2024 financial results. Investors interested in listening to the conference call are required to register online. A live webcast of the conference call will be available on the "Investors" section of the Company’s website at View Source The webcast will be archived on the website following the completion of the call for three months.