Latest news with #RegenerativeMedicineAdvancedTherapy
Time Business News
5 days ago
- Business
- Time Business News
Pioneering Technologies in Regenerative Medicine
Regenerative medicine is a branch of medical science that aims to repair, replace, and regenerate damaged tissues or organs to bring them back to normal function. Stem cell research and regenerative technologies are creating new opportunities for treatment and accelerating growth in the regenerative medicine market. The growing prevalence of chronic disease, increasing funding, and technology advances in gene therapy, an increasing aging population, and more favorable regulations are major contributors to the growing share and speed of development of the regenerative medicine. Key Growth Drivers and Opportunities Rising Number of Aging Population: With the global aging phenomenon, there will be increased incidences of chronic and degenerative diseases such as osteoarthritis, cardiovascular ailments, neurodegenerations, and musculo-skeletal problems, very much in demand for advanced treatment options. Regenerative medicine comprising stem cell therapy, gene therapy, and tissue engineering, among others, presents viable and sustainable alternatives in contrast to conventional treatments such as medication and surgery, which provide temporary relief. Given that regenerative medicine addresses the actual cause of tissue and organ damage, it significantly enhances the quality of life and decreases long-term care burdens, serving as the prime growth driver for the regenerative medicine. Challenges The regenerative medicine is limited by high treatment costs, difficult regulatory approvals, not enough long-term clinical data, and problems in big manufacturing and making everything the same which can block easy acceptance and availability. Innovation and Expansion Beam Therapeutics' BEAM-302 has granted RMAT status by the FDA for Alpha-1 Antitrypsin Deficiency In May 2025, The U.S. Food and Drug Administration (FDA) has designated BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor, as Regenerative Medicine Advanced Therapy (RMAT). This designation is intended to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). Beam Therapeutics Inc. is a biotechnology company that develops precision genetic medicines through base editing. There is a substantial unmet need for efficient treatments that may address the whole range of symptoms for AATD, an inherited genetic illness that affects the lungs and/or liver and causes early onset emphysema and liver disease. The FDA Gives BrainChild Bio's CAR T Therapy for Pediatric Brain Tumors RMAT Status In May 2025, The U.S. Food and Drug Administration (FDA) has designated the investigational B7-H3 targeting autologous CAR T-cell therapy as a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This news was released by BrainChild Bio, Inc., a clinical-stage biotechnology company that is developing CAR T-cell therapies to treat tumors in the central nervous system (CNS). The FDA Designates MeiraGTx's AAV-GAD Gene Therapy as an RMAT for Parkinson's disease In May 2025, AAV-GAD has been designated by the U.S. Food and Drug Administration (FDA) as Regenerative Medicine Advanced Therapy (RMAT) for the treatment of Parkinson's disease that is not sufficiently controlled with anti-Parkinsonian drugs. This announcement was made today by MeiraGTx Holdings plc, a vertically integrated, clinical-stage genetic medicines company. This RMAT was granted after the FDA received favorable results from three clinical trials showing the advantages of AAV-GAD when given as a single stereotactic infusion to the brain's subthalamic nucleus. Inventive Sparks, Expanding Markets Key players in the regenerative medicine market are R&D Systems, Inc., Moderna, Novartis AG and others. These key players are focusing on increasing the research and development of advanced therapies and innovative strategies for 3D bioprinting advancement, and more acquisitions to enhance product offerings through collaborations will be projected to drive the target market. About Author: Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business. TIME BUSINESS NEWS
Yahoo
24-06-2025
- Business
- Yahoo
Carisma Therapeutics, OrthoCellix enter definitive merger agreement
Carisma Therapeutics (CARM) and OrthoCellix, a wholly-owned subsidiary of Ocugen (OCGN), announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the development of OrthoCellix's NeoCart technology for the treatment of knee articular cartilage defects and plans to initiate a U.S. Food and Drug Administration-endorsed Phase 3 clinical trial for NeoCart. OrthoCellix is developing NeoCart as an autologous cartilage implant technology utilizing patient cells to repair articular cartilage defects of the knee. The novel platform merges a fortified 3D scaffold and patented bioprocessing technology to grow chondrocytes-the cells responsible for maintaining cartilage health-to produce adolescent-like cartilage at the time of implant. NeoCart has the potential to accelerate healing and reduce pain by creating a similar, functional joint surface to help patients return to normal activities and prevent complications associated with articular cartilage damage. OrthoCellix anticipates launching its Phase 3 clinical trial by the end of 2025. Previously, NeoCart received Regenerative Medicine Advanced Therapy designation and concurrence from the FDA on a single, confirmatory Phase 3 clinical trial to enable submission of a Biologics License Application. Under the terms of the merger agreement, OrthoCellix will merge with and into a wholly-owned subsidiary of Carisma, with OrthoCellix continuing as a wholly-owned subsidiary of Carisma and the surviving company of the Merger. Carisma will issue to the pre-merger OrthoCellix stockholder shares of Carisma common stock as merger consideration in exchange for the cancellation of shares of capital stock of OrthoCellix. Carisma also expects to enter into subscription agreements for a private financing with Ocugen and other select investors, which is expected to close concurrently with the completion of the merger, to enable the combined company to complete the Phase 3 trial of NeoCart without any additional cost or investment from Ocugen. In connection with the closing of the proposed transactions, Carisma stockholders will be issued contingent value rights representing the right to receive certain payments from proceeds received by the combined company, if any, related to Carisma's pre-transaction legacy assets. Under the terms of the merger agreement, upon the closing of the proposed transactions and after giving effect to the contemplated $25M concurrent financing, OrthoCellix's stockholder and the other participants in the concurrent financing are expected to own approximately 90% of the combined company, and existing Carisma stockholders are expected to own approximately 10% of the combined company, each on a fully diluted basis. The percentage of the combined company that each company's former stockholders will own after completion of the merger is subject to adjustment based on Carisma's net cash at the closing and the proceeds from the concurrent financing, among other adjustments, in each case as described in the merger agreement. Upon the closing of the proposed transactions, 'Carisma Therapeutics Inc.' is expected to be renamed 'OrthoCellix, Inc.' and trade on the Nasdaq Capital Market under the ticker symbol 'OCLX.' The transaction has been unanimously approved by the board of directors of both companies and is expected to close in the second half of 2025, subject to customary closing conditions, including approvals by the stockholders of each company and the effectiveness of a registration statement to be filed with the Securities and Exchange Commission to register the shares of Carisma common stock to be issued in connection with the merger. In connection with the companies' entry into the merger agreement, directors and officers of Carisma and OrthoCellix's stockholder have executed support agreements, pursuant to which they have agreed to vote all of their shares of capital stock in favor of the merger or the issuance of Carisma equity in the merger, as applicable. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Disclaimer & DisclosureReport an Issue Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
17-06-2025
- Business
- Yahoo
Lyell Immunopharma Announces Positive New Clinical Data Demonstrating High Rates of Durable Complete Responses from the Phase 1/2 Trial of LYL314 for the Treatment of Aggressive Large B-cell Lymphoma
LYL314 demonstrated robust clinical responses, with an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting (N = 25) 71% of patients with complete response remained in complete response at ≥ 6 months Manageable safety profile appropriate for outpatient administration with no Grade ≥ 3 cytokine release syndrome and low rates of Grade ≥ 3 ICANS with rapid resolution Pivotal single-arm PiNACLE trial is underway in CAR T-naïve patients with large B-cell lymphoma treated in the third- or later-line setting Lyell to host an investor webcast at 8:00 AM ET today SOUTH SAN FRANCISCO, Calif., June 17, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing next-generation CAR T-cell therapies for patients with cancer, today announced positive new clinical data from the multi-center Phase 1/2 trial of LYL314, including data from patients with large B-cell lymphoma (LBCL) treated in the third- or later-line (3L+) setting. LYL314 is an autologous dual-targeting CD19/CD20 chimeric antigen receptor (CAR) T-cell product candidate with Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA that is in development for patients with relapsed and/or refractory (R/R) LBCL. In patients treated in the 3L+ setting (N = 25), LYL314 continued to demonstrate robust clinical responses, with an 88% overall response rate and a 72% complete response rate. Of the 3L+ patients who achieved a complete response, 71% remained in complete response at ≥ 6 months. The single-arm pivotal PiNACLE trial, a seamless expansion of the Phase 1/2 trial of patients with R/R LBCL being treated in the 3L+ setting, is underway. 'Based on the LYL314 data to be presented at the International Conference on Malignant Lymphoma and my personal experience treating patients in the clinical trial, I believe that LYL314 has the potential to provide differentiated benefit for patients with relapsed/refractory large B-cell lymphoma in both the complete response rate and durability of response,' stated Akil Merchant, MD, Associate Professor and Co-Director of the Lymphoma Program at the Samuel Oschin Cancer Center, Cedars-Sinai Medical Center, Los Angeles, CA, and an investigator in the Phase 1/2 clinical trial. 'We look forward to completing enrollment in the ongoing single-arm pivotal trial evaluating LYL314 in patients in the third- or later-line setting.' Fifty-one CAR T-naive patients with R/R LBCL received LYL314 as of April 15, 2025 (the data cutoff date for the presentation). The efficacy evaluable population consisted of 36 patients with Day 84 assessments or prior disease progression or death. Patient demographics and baseline disease characteristics were consistent with high-risk patient populations: median ages of 65 and 69 years in the 3L+ and 2L, respectively, 41% of 3L+ and 65% of 2L patients had Stage IV disease at trial entry, and 47% of 3L+ and 82% of 2L patients had primary refractory disease. There were 49 patients who received the recommended Phase 2 dose of 100 x 106 CAR T cells; two patients received a dose of 300 x 106 CAR T cells. CD19/CD20 screening was not required prior to enrollment. In efficacy-evaluable 3L+ patients, with a median follow up of 9 months (N = 25): The overall response rate was 88% (22/25 patients), with 72% (18/25) of patients achieving a complete response 71% (10/14) of patients with complete response remained in complete response at ≥ 6 months In initial data from efficacy-evaluable 2L patients, with a median follow up of 5 months (N = 11): The overall response rate was 91% (10/11 patients), with 64% (7/11) achieving a complete response 100% (7/7) of patients with complete response were in complete response at last assessment, including 3/3 at ≥ 6 months In patients with primary refractory disease, a difficult to treat population, 70% (7/10) achieved a complete response These patients had high-risk features, including primary refractory disease (91%), stage IV disease (64%), and older age (27% > 75 years; median age 73 years) In 51 patients, including patients from both the 3L+ and the 2L cohorts, a manageable safety profile appropriate for outpatient administration was observed. No Grade ≥ 3 and low rates of Grade 1 (22%) or Grade 2 (35%) cytokine release syndrome (CRS) were reported. Immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 6% (Grade 1), 2% (Grade 2), and 14% (Grade ≥ 3) of patients. The median time to complete resolution of all reports of ICANS was 5 days, with rapid improvement (median of 2 days) to Grade 2 or lower with standard therapy. No deaths were related to LYL314 administration. LYL314 demonstrated robust expansion with a time to peak of 10 days (N = 51). The final drug product contained the desired CD62L-positive naïve T-cell phenotype (median, 95%). Rapid and durable depletion of B cells was demonstrated through month 6 and up to the month 12 assessment using a highly sensitive and robust method. 'Based on these robust data, and our recent End-of-Phase 1 meeting with the FDA, we have initiated PiNACLE, a single-arm pivotal trial of LYL314 in patients with large B-cell lymphoma in the third- or later-line setting and remain on track to initiate a pivotal trial to evaluate LYL314 in the second-line setting by the beginning of 2026,' said Lynn Seely, MD, Lyell's President and Chief Executive Officer. The data will be presented on Wednesday, June 18, 2025 in an oral session at the International Conference on Malignant Lymphoma in Lugano, Switzerland by Akil Merchant, MD, Associate Professor and Co-Director of the Lymphoma Program at the Samuel Oschin Cancer Center, Cedars-Sinai Medical Center, Los Angeles, CA, and will be available as a presentation in the Investors' section of the Company's website. Conference Call Details Lyell's management will host an investor conference call and webcast beginning at 8:00 AM ET today. The Webcast registration link can be accessed here. A replay of the event and presentation materials will be archived on the Investor page of the Lyell Website following the end of the event. About LYL314 LYL314 (formerly IMPT-314) is a next-generation dual-targeting CD19/CD20 CAR T-cell product candidate designed to increase complete response rates and prolong the duration of the responses as compared to the approved CD19‑targeted CAR T-cell therapies for the treatment of large B-cell lymphoma. LYL314 is designed with an 'OR' logic gate to target B cells that express either CD19, CD20 or both. LYL314 is manufactured to produce a CAR T-cell product with higher proportions of naïve and central memory T cells through a proprietary process that enriches for CD62L-expressing cells. This manufacturing process is designed to generate CAR T cells with enhanced antitumor activity. LYL314 has received Regenerative Medicine Advanced Therapy (RMAT) designation, as well as Fast Track Designation, from the U.S. Food and Drug Administration for the treatment of patients with relapsed and/or refractory aggressive B-cell lymphoma in the third- or later-line setting. About the PiNACLE Trial PiNACLE is a single-arm pivotal trial of LYL314, 100 x 106 CAR T cells, in patients with large B-cell lymphoma treated in the third- or later-line setting. The trial is expected to enroll approximately 120 patients with relapsed and/or refractory diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, high grade B-cell lymphoma, grade 3B follicular lymphoma, or transformed follicular lymphoma who have not previously received CAR T-cell therapy. Patients may be treated with LYL314 in either the inpatient or outpatient setting. The primary endpoint of the trial is the overall response rate. More information about the PiNACLE trial can be found on (NCT05826535) here. About Lyell Lyell is a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with hematologic malignancies and solid tumors. To realize the potential of cell therapy for cancer, Lyell utilizes a suite of technologies to endow CAR T cells with attributes needed to drive durable tumor cytotoxicity and achieve consistent and long-lasting clinical responses, including the ability to resist exhaustion, maintain qualities of durable stemness and function in the hostile tumor microenvironment. The Lyell LyFE Manufacturing Center™ has commercial launch capability and can manufacture more than 1,200 CAR T-cell doses at full capacity. To learn more, please visit Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding: the potential clinical benefits and therapeutic potential of LYL314 for patients with R/R LBCL, including its potential to increase complete response rates and prolong the duration of responses as compared to approved CD19-targeted CAR T-cell therapies for the treatment of LBCL; the potential benefits, if any, from the RMAT and Fast Track designations from the FDA for the treatment of patients with R/R LBCL in the 3L+ setting; expectations around enrollment and timing of Lyell's ongoing single-arm pivotal trial evaluating LYL314 in patients in the 3L+setting and the initiation of a pivotal trial to evaluate LYL314 in the 2L setting; the potential of Lyell's manufacturing process to generate CAR T cells with enhanced antitumor activity; the sufficiency of the capacity of LyFE to manufacture drug supply for Lyell's ongoing and planned pivotal trials and through potential commercial launch; Lyell's anticipated progress, business plans, business strategy and clinical trials; Lyell's advancement of its pipeline, technology platform and research, development and clinical capabilities; and other statements that are not historical fact. These statements are based on Lyell's current plans, objectives, estimates, expectations and intentions, are not guarantees of future performance and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, but are not limited to, risks and uncertainties related to: the potential for results from clinical trials to differ from nonclinical, early clinical, preliminary or expected results; Lyell's limited experience as a company in enrolling and conducting clinical trials and lack of experience in completing clinical trials; significant adverse events, toxicities or other undesirable side effects associated with Lyell's product candidates; Lyell's ability to initiate or progress clinical trials on the anticipated timelines, if at all; RMAT and Fast Track designations may not actually lead to faster development, regulatory review or approval process, and does not assure ultimate FDA approval; the significant uncertainty associated with Lyell's product candidates ever receiving any regulatory approvals; Lyell's ability to obtain, maintain or protect intellectual property rights related to its product candidates; the complexity of manufacturing cellular therapies and Lyell's ability to manufacture and supply its product candidates for its clinical trials; implementation of Lyell's strategic plans for its business and product candidates; the sufficiency of Lyell's capital resources and need for additional capital to achieve its goals; the effects of macroeconomic conditions, including the effects of disruption between the U.S. and its trading partners due to tariffs or other policies, and any geopolitical instability; potential changes to U.S. drug pricing, including the potential for 'most-favored nations' pricing limitations; and other risks, including general economic conditions and regulatory developments, not within our control; and other risks described under the heading 'Risk Factors' in Lyell's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the Securities and Exchange Commission on May 13, 2025. Forward-looking statements contained in this press release are made as of this date, and Lyell undertakes no duty to update such information except as required under applicable law. Contact: Ellen RoseSenior Vice President, Communications and Investor Relationserose@ while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
12-06-2025
- Business
- Yahoo
Lyell Immunopharma Reports Business Highlights and Financial Results for the First Quarter 2025
Presenting new clinical data from Phase 1/2 multi-center clinical trial of LYL314, a next-generation dual‑targeting CD19/CD20 CAR T-cell product candidate for the treatment of relapsed and/or refractory large B‑cell lymphoma at the 18th International Conference on Malignant Lymphoma. Received Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for LYL314 for the treatment of relapsed and/or refractory diffuse large B-cell lymphoma in the third- or later-line setting. LYL314 clinical supply now manufactured at Lyell's LyFE Manufacturing Center™, following successful technology transfer and clearance by the FDA of an Investigational New Drug Amendment. Remain on track to initiate a pivotal trial of LYL314 in the third- or later-line setting in mid-2025 and expect to initiate a pivotal trial in the second-line setting by early 2026. Cash, cash equivalents and marketable securities of $330.1 million as of March 31, 2025 support advancing pipeline into 2027 through multiple clinical milestones. SOUTH SAN FRANCISCO, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with cancer, today reported financial results and business highlights for the first quarter ended March 31, 2025. Lyell's lead clinical program, LYL314 (formerly known as IMPT-314), is an autologous CD19/CD20 dual-targeting CAR T-cell product candidate under evaluation in a Phase 1/2 trial enrolling patients with relapsed and/or refractory large B-cell lymphoma (LBCL). LYL314 was recently granted RMAT designation by the United States FDA in recognition of its potential to address significant unmet needs in patients with aggressive LBCL in the third- or later-line setting. 'We are pleased with the progress we are making with our LYL314 clinical development strategy and look forward to presenting new clinical data from patients with aggressive large B-cell lymphoma who have not previously received CAR T-cell therapy at the International Conference on Malignant Lymphoma in Lugano, Switzerland in June,' said Lynn Seely, M.D., President and CEO of Lyell. 'Based on promising clinical data, Lyell remains on track to initiate two pivotal programs for LYL314: one for patients in the third- or later-line setting by mid-2025 and a second for patients in the second-line setting by early 2026. In addition, our LyFE Manufacturing Center in Bothell, Washington is now manufacturing the LYL314 clinical supply following completion of a successful technology transfer and FDA clearance of an IND amendment.' First Quarter Updates and Recent Business Highlights Lyell is advancing a pipeline of next-generation CAR T-cell product candidates. Its lead program, LYL314, is in Phase 1/2 clinical development for relapsed and/or refractory LBCL and its preclinical programs target solid tumor indications. Lyell's programs target cancers with large unmet need with substantial patient is an autologous CAR T-cell product candidate with a true 'OR' logic gate to target B cells that express either CD19 or CD20 with full potency and that is manufactured with a process that enriches for CD62L-positive cells to generate more naïve and central memory CAR T cells with enhanced stemlike features and antitumor activity. LYL314 is currently being evaluated in a multi-center, open-label trial that is enrolling patients in the third- or later-line and second-line settings who have not previously received CAR T-cell therapy. The trial is designed to evaluate the tolerability and clinical benefit of LYL314 in patients with relapsed and/or refractory LBCL and determine a recommended Phase 2 dose. The FDA has granted LYL314 RMAT and Fast Track designations for the treatment of relapsed and/or refractory diffuse LBCL in the third- or later-line setting. RMAT provides all the benefits of the Fast Track and Breakthrough Therapy designation programs and enables increased frequency of communications with the FDA on the development of LYL314. New clinical data from the Phase 1/2 multi-center clinical trial of LYL314 in patients with relapsed and/or refractory LBCL will be presented at the 18th International Conference on Malignant Lymphoma in June, including more mature data from patients treated in the third- or later-line setting and initial data from patients treated in the second-line setting. The data will be highlighted in an oral presentation titled 'LYL314, a CD19/CD20 CAR T-cell candidate enriched for CD62L+ stem-like cells, achieves high rates of durable complete responses in R/R large B-cell lymphoma' to be presented on June 18th at 5:40 pm CEST. Initial clinical data in 23 patients treated with LYL314 from the ongoing Phase 1/2 clinical trial were presented at the American Society for Hematology 2024 Annual Meeting on December 9, 2024. The efficacy evaluable population consisted of 17 patients with relapsed and/or refractory LBCL in the third- or later-line setting who had not previously received a CAR T-cell therapy prior to LYL314 administration. The overall response rate was 94% (16/17) of patients, and 71% (12/17) of patients achieved a complete response by three months. The median follow up was 6.3 months (range 1.2 – 12.5 months) and 71% of patients experienced a response at last follow-up. In the safety evaluable population of 23 patients, no event of Grade 3 or greater cytokine release syndrome was reported. Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 13% (3/23) of patients with a median time to ICANS resolution of 5 days, and rapid improvement to Grade 2 or lower with standard therapy. LYL314 clinical supply is now manufactured at the LyFE Manufacturing Center in Bothell, Washington following successful technology transfer and clearance by the FDA of an IND amendment. LyFE is a state-of-the-art cell therapy manufacturing facility with the capacity to provide drug supply for Lyell's ongoing and planned pivotal trials and through potential commercial launch, with a capacity over 1,000 CAR T-cell therapy doses per year. A pivotal trial in the third- or later-line setting is expected to be initiated in mid-2025 in patients with relapsed and/or refractory LBCL who have not previously received CAR T-cell therapy. More mature data from the ongoing Phase 1/2 trial in the second-line setting are expected to be presented in late-2025. A pivotal trial in the second-line setting is expected to be initiated by early 2026 in patients with relapsed and/or refractory LBCL who have not previously received CAR T-cell therapy. Preclinical Pipeline, Technologies and Manufacturing Protocols Lyell is advancing next-generation fully-armed CAR T-cell product candidates, each including multiple technologies, designed to overcome T-cell exhaustion and lack of durable stemness, as well as immune suppression within the hostile tumor microenvironment. The first IND for a fully-armed CAR T-cell product candidate with an undisclosed target for solid tumors is expected in 2026. An abstract titled 'Engineered T Cells Combining Stackable Reprogramming Technologies Enable Durable Anti‑tumor Activity in Xenograft Solid Tumors' has been accepted for an oral presentation at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), on May 16, 2025, in New Orleans, LA. The presentation will highlight preclinical data demonstrating that engineered T cells enhanced with c-Jun overexpression, a chimeric co-stimulatory receptor and localized cytokine signaling technologies exhibit potent tumor cell killing in vitro, T-cell viability in vitro and in vivo without cytokine support and effective durable antitumor activity in preclinical solid tumor models. An abstract titled 'Optimizing Stim-R™, a synthetic stimulatory agent, for feeder-free tumor-infiltrating lymphocyte manufacturing' was presented at the American Association for Cancer Research Annual Meeting 2025. The presented work demonstrated that Stim-R, Lyell's customizable synthetic T-cell activation reagent designed to emulate natural antigen presentation, has the potential to overcome scalability limitations in tumor-infiltrating lymphocyte manufacturing processes by providing a synthetic replacement for feeder cells. First Quarter 2025 Financial Results Lyell reported a net loss of $52.2 million for the first quarter ended March 31, 2025, compared to a net loss of $60.7 million for the same period in 2024. The $8.5 million decrease in net loss was primarily driven by $13.0 million in impairment expenses recognized in the prior year period that did not occur in 2025, partially offset by lower interest income of $3.0 million primarily driven by lower interest rates in 2025, coupled with lower cash equivalent and marketable securities balances. Non‑GAAP net loss, which excludes stock-based compensation, non-cash expenses related to the change in the estimated fair value of success payment liabilities and certain non-cash investment gains and charges, increased to $46.3 million for the first quarter ended March 31, 2025, compared to $37.5 million for the same period in 2024 due primarily to increased personnel costs and lower interest income in 2025. GAAP and Non-GAAP Operating Expenses Research and development (R&D) expenses were $43.4 million for the first quarter ended March 31, 2025, compared to $43.2 million for the same period in 2024. The increase in first quarter 2025 R&D expenses of $0.3 million was primarily due to a $3.1 million increase in personnel expenses, due primarily to severance expenses resulting from Lyell's 2025 workforce reduction related to the closure of the West Hills manufacturing facility acquired as part of Lyell's acquisition of ImmPACT in 2024, partially offset by reductions in collaboration agreement and leasehold improvement depreciation costs. Non‑GAAP R&D expenses, which exclude non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities for the first quarter ended March 31, 2025 were $41.1 million compared to $38.9 million for the same period in 2024. General and administrative (G&A) expenses were $14.0 million for the first quarter ended March 31, 2025, respectively, compared to $13.5 million for the same period in 2024. The increase in first quarter 2025 G&A expenses of $0.6 million was primarily driven by an increase of $2.4 million in personnel‑related expenses due to higher headcount associated with Lyell's acquisition of ImmPACT and severance expenses related to the West Hills facility closure, partially offset by $1.7 million in decreased stock-based compensation expense due to a decrease in the value of awards granted. Non‑GAAP G&A expenses, which exclude non-cash stock‑based compensation, for the first quarter ended March 31, 2025 were $10.4 million, compared to $8.1 million for the same period in 2024. The $2.3 million increase in first quarter 2025 non-GAAP G&A expenses was primarily driven by increased headcount and severance expenses. A discussion of non-GAAP financial measures, including reconciliations of the most comparable GAAP measures to non‑GAAP financial measures, is presented below under 'Non-GAAP Financial Measures.' Cash, cash equivalents and marketable securities Cash, cash equivalents and marketable securities as of March 31, 2025 were $330.1 million compared to $383.5 million as of December 31, 2024. Lyell believes that its cash, cash equivalents and marketable securities balances will be sufficient to meet working capital and capital expenditure needs into 2027. About Lyell Immunopharma, Inc. Lyell is a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with hematologic malignancies and solid tumors. To realize the potential of cell therapy for cancer, Lyell utilizes a suite of technologies to endow CAR T cells with attributes needed to drive durable tumor cytotoxicity and achieve consistent and long-lasting clinical responses, including the ability to resist exhaustion, maintain qualities of durable stemness and function in the hostile tumor microenvironment. To learn more, please visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding: Lyell's plans to present new LYL314 clinical data from the ongoing Phase 1/2 trial of LYL314; the anticipated benefits of RMAT designation for LYL314; the sufficiency of the capacity of LyFE to manufacture drug supply for Lyell's ongoing and planned pivotal trials and through potential commercial launch; Lyell's initiation of pivotal trials in 2025 and 2026 for LYL314; expectations around enrollment and the timing of additional clinical data from Lyell's Phase 1/2 trials for LYL314; timing of Lyell's submission of a new IND in 2026 for a CAR T-cell product candidate with an undisclosed target for solid tumors; Lyell's anticipated progress, business plans, business strategy and clinical trials; Lyell's advancement of its pipeline and its research, development and clinical capabilities; the potential clinical benefits and therapeutic potential of Lyell's product candidates; the advancement of Lyell's technology platform; Lyell's expectation that its financial position and cash runway will support advancement of its pipeline through multiple clinical milestones and meet working capital and capital expenditure needs into 2027; and other statements that are not historical fact. These statements are based on Lyell's current plans, objectives, estimates, expectations and intentions, are not guarantees of future performance and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, but are not limited to, risks and uncertainties related to: the complexity of manufacturing cellular therapies, which subjects us to a multitude of manufacturing risks, any of which could substantially increase our costs, delay our programs or limit supply of our product candidates; RMAT designation may not actually lead to faster development, regulatory review or approval process, and does not assure ultimate FDA approval; the effects of macroeconomic conditions, including the effects of disruption between the U.S. and its trading partners due to tariffs or other policies, any geopolitical instability and actual or perceived changes in interest rates and economic inflation; Lyell's ability to submit planned INDs or initiate or progress clinical trials on the anticipated timelines, if at all; Lyell's limited experience as a company in enrolling and conducting clinical trials, and lack of experience in completing clinical trials; the nonclinical profiles of Lyell's product candidates or technology not translating in clinical trials; the potential for results from clinical trials to differ from nonclinical, early clinical, preliminary or expected results; significant adverse events, toxicities or other undesirable side effects associated with Lyell's product candidates; the significant uncertainty associated with Lyell's product candidates ever receiving any regulatory approvals; Lyell's ability to obtain, maintain or protect intellectual property rights related to its product candidates; implementation of Lyell's strategic plans for its business and product candidates; the sufficiency of Lyell's capital resources and need for additional capital to achieve its goals; and other risks, including those described under the heading 'Risk Factors' in Lyell's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission (SEC) on March 11, 2025, and Lyell's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, being filed with the SEC today. Forward-looking statements contained in this press release are made as of this date, and Lyell undertakes no duty to update such information except as required under applicable law. Lyell Immunopharma, Selected Consolidated Financial Data(in thousands) Statement of Operations Data: Three Months Ended March 31, 2025 2024 Revenue $ 7 $ 3 Operating expenses: Research and development(1) 43,447 43,174 General and administrative 14,046 13,494 Other operating income, net (119 ) (1,090 ) Total operating expenses 57,374 55,578 Loss from operations (57,367 ) (55,575 ) Interest income, net 3,862 6,819 Other income, net(1) 1,310 1,090 Impairment of other investments — (13,001 ) Total other income (loss), net 5,172 (5,092 ) Net loss $ (52,195 ) $ (60,667 ) (1) As of October 1, 2024, the Company's success payment liability was recognized at fair value as Stanford had provided the requisite service obligation to earn the potential success payment consideration. The change in the estimated fair value of Stanford success payment liabilities in the first quarter of 2025 was recognized within other income, net in the Condensed Consolidated Statements of Operations and Comprehensive Loss. The change in the estimated fair value of Stanford success payment liabilities in the first quarter of 2024 was recognized within research and development expenses in the Condensed Consolidated Statements of Operations and Comprehensive Loss. The change in the estimated fair value of Fred Hutch success payment liabilities was recognized within other income, net in the Condensed Consolidated Statements of Operations and Comprehensive Loss. Balance Sheet Data: As of March 31, As of December 31, 2025 2024 Cash, cash equivalents and marketable securities $ 330,126 $ 383,541 Property and equipment, net $ 44,195 $ 48,200 Total assets $ 429,798 $ 490,859 Total stockholders' equity $ 336,521 $ 382,824 Non-GAAP Financial Measures To supplement our financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), we present non-GAAP net loss, non-GAAP R&D expenses and non-GAAP G&A expenses. Non‑GAAP net loss and non-GAAP R&D expenses exclude non-cash stock-based compensation expense and non-cash expenses related to the change in the estimated fair value of success payment liabilities from GAAP net loss and GAAP R&D expenses. Non-GAAP net loss further adjusts non‑cash investment gains and charges, as applicable. Non‑GAAP G&A expenses exclude non-cash stock-based compensation expense from GAAP G&A expenses. We believe that these non‑GAAP financial measures, when considered together with our financial information prepared in accordance with GAAP, can enhance investors' and analysts' ability to meaningfully compare our results from period to period, and to identify operating trends in our business. We have excluded stock-based compensation expense, changes in the estimated fair value of success payment liabilities, and non-cash investment gains and charges from our non‑GAAP financial measures because they are non-cash gains and charges that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. We also regularly use these non‑GAAP financial measures internally to understand, manage and evaluate our business and to make operating decisions. These non-GAAP financial measures are in addition to, and not a substitute for or superior to, measures of financial performance prepared in accordance with GAAP. In addition, these non‑GAAP financial measures have no standardized meaning prescribed by GAAP and are not prepared under any comprehensive set of accounting rules or principles and, therefore, have limits in their usefulness to investors. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business. Lyell Immunopharma, Reconciliation of GAAP to Non-GAAP Net Loss(in thousands) Three Months Ended March 31, 2025 2024 Net loss - GAAP $ (52,195 ) $ (60,667 ) Adjustments: Stock-based compensation expense 6,024 9,155 Change in the estimated fair value of success payment liabilities (125 ) 968 Impairment of other investments — 13,001 Net loss - Non-GAAP(1) $ (46,296 ) $ (37,543 ) (1) There was no income tax effect related to the adjustments made to calculate non-GAAP net loss because of the full valuation allowance on our net deferred tax assets for all periods presented. Lyell Immunopharma, Reconciliation of GAAP to Non-GAAP Research and Development Expenses(in thousands) Three Months Ended March 31, 2025 2024 Research and development - GAAP $ 43,447 $ 43,174 Adjustments: Stock-based compensation expense (2,388 ) (3,792 ) Change in the estimated fair value of success payment liabilities(1) — (525 ) Research and development - Non-GAAP $ 41,059 $ 38,857 (1) As of October 1, 2024, the Company's success payment liability was recognized at fair value as Stanford had provided the requisite service obligation to earn the potential success payment consideration. The change in the estimated fair value of Stanford success payment liabilities in the first quarter of 2025 was recognized within other income, net in the Condensed Consolidated Statements of Operations and Comprehensive Loss. The change in the estimated fair value of Stanford success payment liabilities in the first quarter of 2024 was recognized within research and development expenses in the Condensed Consolidated Statements of Operations and Comprehensive Loss. The change in the estimated fair value of Fred Hutch success payment liabilities was recognized within other income, net in the Condensed Consolidated Statements of Operations and Comprehensive Loss. Lyell Immunopharma, Reconciliation of GAAP to Non-GAAP General and Administrative Expenses(in thousands) Three Months Ended March 31, 2025 2024 General and administrative - GAAP $ 14,046 $ 13,494 Adjustments: Stock-based compensation expense (3,636 ) (5,363 ) General and administrative - Non-GAAP $ 10,410 $ 8,131 Contact:Ellen RoseSenior Vice President, Communications and Investor Relationserose@
Business Wire
06-06-2025
- Business
- Business Wire
OS Therapies Submits Request for Regenerative Medicine Advanced Therapy (RMAT) Designation to U.S. FDA for OST-HER2 in the Prevention of Metastases in Recurrent, Fully-Resected, Lung Metastatic Pediatric Osteosarcoma
NEW YORK--(BUSINESS WIRE)-- OS Therapies Inc. (NYSE-A: OSTX) ('OS Therapies' or 'the Company'), a clinical-stage cancer immunotherapy and antibody drug conjugate biotechnology company, today announced it has submitted a request for Regenerative Medicine Advanced Therapy (RMAT) Designation to U.S. FDA for OST-HER2 in the prevention of metastases in recurrent, fully-resected, lung metastatic pediatric osteosarcoma. RMAT designations are granted to sponsors with regenerative medicine therapies for serious or life-threatening conditions and provide sponsors with various benefits, including eligibility for an accelerated Biologics License Application (BLA) review. OST-HER2 has already received Rare Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD) and Fast Track Designation (FTD) for osteosarcoma from the U.S. FDA. If OST-HER2 receives a conditional BLA via Accelerated Review prior to September 30, 2026, the Company will become eligible to receive a Priority Review Voucher (PRV) that it intends to immediately sell. The most recent publicly disclosed PRV sale, valued at $155 million, occurred in May 2025. The Company is awaiting feedback by mid-June 2025 from a Type D meeting with FDA regarding the statistical analysis plan to be used in an End of Phase 2 meeting for OST-HER2 in the prevention of metastases in recurrent, fully-resected, lung metastatic pediatric osteosarcoma. Upon receipt of the Type D Meeting feedback, the Company intends to promptly request the End of Phase 2 meeting with FDA in which it will be seek agreement to allow it to begin a rolling BLA submission in the third quarter of 2025. The grant of the RMAT designation in the third quarter of 2025 complements the company's parallel efforts in other major markets, including Europe and the United Kingdom, where the company plans to seek EMA PRIME Designation and Conditional Market Access (CMA) applications. In parallel to regulatory engagement and market access planning for OST-HER2, the Company is preparing for the late stage clinical development of other pipeline candidates. As such, the Company is well positioned for sustained growth across multiple therapeutic modalities. About OS Therapies OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for osteosarcoma and other solid tumors. OST-HER2, the Company's lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food & Drug Administration and Fast-Track and Orphan Drug designations from the U.S. FDA and European Medicines Agency. The Company has demonstrated positive data in its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma demonstrating statistically significant benefit in the 12-month event free survival (EFS) primary endpoint of the study. The Company anticipates submitting a BLA to the U.S. FDA for OST-HER2 in osteosarcoma in 2025 and, if approved, would become eligible to receive a Priority Review Voucher that it could then sell. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the U.S. Department of Agriculture for the treatment of canines with osteosarcoma. In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) and Drug Conjugates (DC), known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone Si-Linker and Conditionally Active Payload (CAP) technology, enabling the delivery of multiple payloads per linker. For more information, please visit Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of the federal securities laws. These forward-looking statements and terms such as "anticipate," "expect," "intend," "may," "will," "should" or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. OS Therapies cautions readers that forward-looking statements are based on management's expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially, including, but not limited to the approval of OST-HER2 by the U.S. FDA and other risks and uncertainties described in 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations' in the Company's most recent Annual Report on Form 10-K and other subsequent documents the Company files with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
