ATL-001

– On track to dose 15-20 patients with higher dose cNeT by year-end 2023 – – Clinical and translational science data update from the ongoing Phase I/IIa trials in NSCLC and melanoma expected in Q1 2024 with additional data available in mid-2024 – – Strong cash position of $140 million supports operations through 2025 – LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, today announced its financial results for the quarter ended September 30, 2023, and recent business highlights. “We are pleased with progress across the scientific field that continues to demonstrate the importance of neoantigens as the most attractive targets in solid-tumor oncology. We believe that our proprietary capability to identify the most immunogenic clonal neoantigens, recently shared at ESGCT and SITC, puts us in a strong position with our clonal neoantigen-reactive T cell (cNeT) therapy and potentially other neoantigen targeting approaches,” said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. “Our Phase I/IIa clinical trials evaluating cNeT therapy for the treatment of advanced NSCLC (CHIRON) and metastatic malignant melanoma (THETIS) continue to progress and we are on track to dose patients in line with our previous guidance. We look forward to sharing our next clinical and translational data update in Q1 2024.” Recent Highlights Presented posters (Abstract 437, Abstract 1312) at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting highlighting a significant dose boost of reactive CD8 and CD4 cNeT from the optimized VELOSTM manufacturing platform and the use of the PELEUS™ AI-powered bioinformatics platform for the identification and validation of immunogenic clonal tumor neoantigens.Presented posters (P459, P485, P507) at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress highlighting the favorable cNeT phenotypic properties and polyfunctionality critical for precision adoptive cell therapies and the expansion of cNeT from the blood of patients with cancer.Delivered an oral presentation at the mRNA Cancer Vaccines Summit highlighting neoRanker™, a new AI-based tool for the selection of immunogenic clonal tumor neoantigens for personalized therapies.Delivered oral presentations at the 5th Annual TIL Therapies Summit highlighting optimized manufacturing of AI-powered precision TIL therapies for solid cancer, and the potency and fitness of cNeT.Presented posters (P319, P407, P518) at the Seventh International Cancer Immunotherapy Conference (CICON23) highlighting a precision adoptive cell therapy process based on expansion of cNeT from blood of cancer patients. Financial Highlights Cash and cash equivalents: Cash and cash equivalents were $140.1 million as of September 30, 2023, as compared to $173.3 million as of December 31, 2022. The Company anticipates that its cash and cash equivalents are sufficient to fund its planned operations through 2025.Research and development (R&D) expenses: R&D expenses were $14.7 million for the third quarter ended September 30, 2023, an increase of $4.1 million compared to $10.6 million for the third quarter ended September 30, 2022. The increase was primarily driven by increased activity related to our ongoing clinical trials and overall R&D.General and administrative (G&A) expenses: G&A expenses were $4.4 million for the third quarter ended September 30, 2023, a decrease of $1.0 million compared to $5.4 million for the third quarter ended September 30, 2022. This decrease was primarily driven by lower professional fees, personnel costs and facilities spend. Net loss: Net loss for the third quarter ended September 30, 2023, was $16.7 million or $0.42 per share compared to $12.5 million or $0.32 per share for the third quarter ended September 30, 2022. About Achilles Therapeutics Achilles is a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with advanced non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens. Forward Looking Statements This press release contains express or implied forward-looking statements that are based on our management's belief and assumptions and on information currently available to our management. Forward-looking statements in this press release include, but are not limited to, statements regarding the timing of the Company’s clinical and translational data updates and the Company’s beliefs about recent data updates, and expectations related to the Company’s operating expenses and capital expense requirements. Although we believe that the expectations reflected in these forward-looking statements are reasonable, these statements relate to future events or our future operational or financial performance, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. The forward-looking statements in this press release represent our views as of the date of this press release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should therefore not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release. For further information, please contact: Investors:Meru AdvisorsLee M. Sternlstern@meruadvisors.com Media:ICR ConsiliumSukaina Virji, Tracy Cheung, Emmalee Hoppe+44 (0) 203 709 5000achillestx@consilium-comms.com ACHILLES THERAPEUTICS PLC Condensed Consolidated Balance Sheets (Unaudited)(in thousands, except share and per share amounts)(expressed in U.S. Dollars, unless otherwise stated) September 30, December 31, 2023 2022 ASSETS Current assets: Cash and cash equivalents $140,138 $173,338 Prepaid expenses and other current assets 15,354 23,242 Total current assets 155,492 196,580 Non-current assets: Property and equipment, net 9,648 12,399 Operating lease right of use assets 7,013 8,081 Deferred tax assets 185 251 Restricted cash 33 33 Other assets 2,967 3,014 Total non-current assets 19,846 23,778 TOTAL ASSETS $175,338 $220,358 LIABILITIES AND SHAREHOLDERS’ EQUITY Current liabilities: Accounts payable $5,352 $5,187 Income taxes payable - 326 Accrued expenses and other liabilities 8,209 8,292 Operating lease liabilities-current 4,620 4,188 Total current liabilities 18,181 17,993 Non-current liabilities: Operating lease liabilities-non-current 2,731 4,388 Other long-term liability 941 933 Total non-current liabilities 3,672 5,321 Total liabilities 21,853 23,314 Commitments and contingencies (Note 12) Shareholders’ equity: Ordinary shares, £0.001 par value; 40,782,948 and 40,932,727 shares authorized, issued and outstanding at September 30, 2023 and December 31, 2022, respectively 54 54 Deferred shares, £92,451.851 par value, one share authorized, issued and outstanding at September 30, 2023 and December 31, 2022 128 128 Additional paid in capital 413,815 408,844 Accumulated other comprehensive loss (19,190) (21,695)Accumulated deficit (241,322) (190,287)Total shareholders’ equity 153,485 197,044 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $175,338 $220,358 ACHILLES THERAPEUTICS PLC Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited)(in thousands, except share and per share amounts)(expressed in U.S. Dollars, unless otherwise stated) Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 OPERATING EXPENSES: Research and development $14,712 $10,597 $42,354 $38,387 General and administrative 4,384 5,437 13,387 17,162 Total operating expenses 19,096 16,034 55,741 55,549 Loss from operations (19,096) (16,034) (55,741) (55,549)OTHER INCOME, NET: Other income 2,389 3,599 4,692 8,499 Total other income, net 2,389 3,599 4,692 8,499 Loss before provision for income taxes (16,707) (12,435) (51,049) (47,050)Benefit (Provision) for income taxes 24 (41) 14 (70)Net loss (16,683) (12,476) (51,035) (47,120)Other comprehensive income: Foreign exchange translation adjustment (5,289) (18,147) 2,505 (45,126)Comprehensive loss $(21,972) $(30,623) $(48,530) $(92,246)Net loss per share attributable to ordinary shareholders—basic and diluted $(0.42) $(0.32) $(1.28) $(1.20)Weighted average ordinary shares outstanding—basic and diluted 40,066,922 39,313,764 39,900,910 39,104,866

[20-September-2023] NetworkNewsWire Editorial Coverage NEW YORK, Sept. 20, 2023 /PRNewswire/ -- More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Pro >) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer ("ES-SCLC") who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases. Four decades after the Orphan Drug Act was passed, hundreds of 'orphan' drugs have been approved for use. Genprex's REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation. With recent ODD and FTD designations, Genprex is on the verge of commencing patient treatment in small cell lung cancer in Q4 2023. Click here to view the custom infographic of the Genprex editorial. Launching Point for Revolution More than 30 million Americans struggle with rare diseases. A comprehensive study published by the National Library of Medicine (NLM) states that the 1983 passage of the Orphan Drug Act (ODA), which created the Orphan Drug Designation Program, represented a launching point for a rare-disease drug-development revolution for these patients. "Drug development for rare diseases can be commercially risky, and prior to this legislation, only approximately two drugs per year had been approved by the U.S. Food and Drug Administration for rare diseases," the report observed. "Now, four decades later, hundreds of 'orphan' drugs have been approved for use in the 7,000­–10,000 diseases and conditions that are considered rare." Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases. "Additionally, 882 of those designations resulted in at least one FDA approval for use in 392 rare diseases," the report continued. "Much of this development has been concentrated in oncology as seven of the top ten most designated and approved diseases were rare cancers. Based on our study, we can conclude that around 5% of rare diseases have an FDA-approved drug and up to 15% of rare diseases have at least one drug that has been developed and shown promise in their treatment, diagnosis or prevention. Funding of basic and translational science for rare-disease drug development should continue in order to bring therapies to the millions of affected patients who remain without treatment options." Bringing Hope As the NLM article observed, there is a growing recognition of the potential of gene therapies to transform cancer care. Genprex (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, is among those companies leveraging this trend. The company is currently working in both the non-small cell lung cancer (NSCLC) and the small cell lung cancer (SCLC) space, where its REQORSA Immunogene Therapy (quratusugene ozeplasmid) has gained both Orphan Drug Designation and Fast Track Designation (FTD). "We are excited to receive Orphan Drug Designation from the FDA for REQORSA for patients with SCLC," said Rodney Varner, Genprex president, chair and CEO. "This FDA Orphan Drug Designation in combination with our recently received FDA Fast Track designation underscores the great need for better treatment options for patients with ES (extensive-stage)-SCLC and non-small cell lung cancer (NSCLC). We look forward to initiating the Acclaim-3 clinical trial expected in the fourth quarter of 2023 in order to bring hope of an effective new therapy to patients suffering with this life-limiting cancer." Meeting the SCLC Need In the United States, lung cancer is the most lethal cancer in both men and women, with the American Cancer Society estimating that almost 130,000 people will die this year alone. SCLC comprises approximately 14% of all lung cancers. Limited stage SCLC (LS-SCLC) accounts for approximately 33% of all SCLC and is potentially curable with a combination of cytotoxic chemotherapy and radiation therapy. However, extensive stage (ES-SCLC), which comprises approximately 66% of all SCLC, is not considered curable. There is an unmet need for more effective maintenance therapy options for ES-SCLC, as well as for better combination treatments for SCLC overall. This is the space Genprex is looking to fill with its REQORSA Immunogene Therapy. The company received the FTD approval for REQORSA Immunogene Therapy in combination with Genentech Inc.'s Tecentriq(R). Genentech is the U.S.-based unit of Swiss pharma giant Roche. With ES-SCLC, cancer has spread from one lung to the other, or to other parts of the body. Genprex will be combining its drug with Genentech's Tecentriq to focus on this type of cancer during the upcoming Acclaim-3 clinical trial. The phase 1/2 open-label, dose escalation and clinical response study will evaluate REQORSA in combination with Tecentriq in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex has also been granted FTD approval for two other indications of REQORSA Immunogene Therapy: REQORSA in combination with Tagrisso for NSCLC in patients who have progressed after Tagrisso treatment, and REQORSA in combination with Keytruda for NSCLC in patients who have progressed after Keytruda treatment. Transforming Lives, Redefining Treatments Genprex operates in the dynamic fields of gene therapy, specifically targeting cancer and diabetes. With a focus on developing groundbreaking treatments for patients, the company's market is characterized by the urgent need for innovative solutions. The cancer and diabetes sectors are vast and rife with unmet medical needs, offering significant opportunities for pioneering therapies that can transform lives and redefine treatment paradigms. Genprex's potential for growth is underscored by its promising pipeline of advanced gene therapies in development. REQORSA is the company's lead candidate, which it hopes will revolutionize the treatment of lung cancers. With the recent ODD and FTD designations, along with a robust clinical-development program, Genprex is poised to make a significant impact in the market. Elevating Innovation Finding solutions in the rare disease sector can be challenging but certainly fulfilling as companies research, explore and strive to help those who need it the most. Add the incentives that come with an Orphan Drug Designation, and those efforts can become even more rewarding for these companies. Roche Holding AG ADR (OTCQX: RHHBY), through Genentech, has received several Orphan Drug Designations. In addition to atezolizumab for the treatment of adult patients with ES-SCLC, Genentech most recently received Orphan Drug Designation for pralsetinib in the treatment of certain types of solid tumors. The ODD designation covers the use of pralsetinib in metastatic RET fusion-positive solid tumors. According to the terms, Roche has exclusive rights to commercialize the treatment outside the U.S. except for Greater China. Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), a global biopharmaceutical company dedicated to developing life-changing medicines for people with serious diseases, has also developed a drug for the SCLC market. Its Zepzelca(TM) (lurbinectedin), which was developed in collaboration with PharmaMar, was granted Orphan Drug Designation in 2018, and in 2020, it received accelerated FDA approval based on overall response rate (ORR) and duration of response. ALX Oncology Holdings Inc. (NASDAQ: ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, received ODD from both the FDA and the European Commission for its evorpacept, a next-generation CD47 blocker. The ODD indication includes both gastric cancer and gastroesophageal junction adenocarcinoma. "Receiving orphan drug designation from both the EC and the FDA represents a significant regulatory achievement for ALX Oncology and signifies the growing recognition of evorpacept as a potential new drug to improve clinical outcomes in patients with GC," said ALX Oncology chief medical officer Sophia Randolph, MD., PhD. Achilles Therapeutics PLC (NASDAQ: ACHL) is a biopharmaceutical company developing precision T cell therapies that target clonal neoantigens, or protein markers unique to each individual. Lead indications, including advanced NSCLC and recurrent metastatic melanoma, were selected for their high mutational burden, high level of T cell infiltration and high unmet medical need. Deaths from non-small cell lung cancer outnumber those resulting from breast cancer, colon cancer and prostate cancer combined, and there are limited approved treatment options for lung cancer and melanoma patients whose disease has not responded well to other therapies. In its March 25, 2021, SEC filing, the company noted that it "may in the future seek orphan drug designation for ATL001 and any future product candidates." For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress. When a company such as Genprex, which truly is committed to innovation and patient-centric approaches, receives the designation, it becomes an even more distinctive differentiator. As investors explore opportunities in the biotech sector, Genprex stands out with its cutting-edge therapies, potential market disruption and dedication to transforming lives through innovative science. For more information about Genprex Inc., please visit Genprex. About NetworkNewsWire NetworkNewsWire ("NNW") is a specialized communications platform with a focus on financial news and content distribution for private and public companies and the investment community. 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