On February 13, 2025 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2024 and provided financial guidance (Press release, Blueprint Medicines, FEB 13, 2025, View Source [SID1234650245]).

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"We are entering 2025 in the strongest position we have ever been in as a company with a focus on driving growth and innovation through operational excellence. AYVAKIT is a medicine that is changing patients’ lives, and the SM market is larger and growing faster than we originally expected. Our 2025 guidance of $680 to $710 million represents robust growth, putting AYVAKIT firmly on track to achieve $2 billion in revenue by 2030. And with the larger potential SM franchise peak opportunity of $4 billion, we have a plan to drive innovation in the treatment of SM with elenestinib as we move beyond symptom control to disease modification in the HARBOR registration study, building a durable SM franchise that we anticipate will grow throughout the next decade," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our next potential blockbuster opportunity, BLU-808, is also coming into focus with the healthy volunteer data reported last month. BLU-808 demonstrated a best-in-class profile for an oral wild-type KIT inhibitor enabling our differentiated approach to modulating mast cell activity across thousands of patients with mast-cell-mediated allergic and inflammatory diseases. BLU-808 offers us the opportunity to find the right balance of efficacy and tolerability to realize true pipeline-in-a-medicine potential. Altogether, our portfolio represents a unique set of assets, targeting fundamental mast cell biology, that will drive durable and diversified growth over the next decade with significant operating leverage."

Fourth Quarter 2024 Highlights and Recent Progress

Mast cell disorders

● Achieved AYVAKIT net product revenues of $479.0 million, including $421.8 million in the US and $57.1 million ex-US, representing 135 percent growth year-over-year. In the fourth quarter of 2024, AYVAKIT achieved $144.1 million, including $124.1 million in the US and $20.0 million ex-US.
● Blueprint estimates the peak revenue opportunity for the company’s SM franchise is $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030.
● Presented AYVAKIT data at the 2024 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, showing a significant survival benefit in treatment-naïve patients with advanced SM, and sustained improvement in bone density for advanced SM patients with low bone mass at baseline. View the presentations here.
● Reported in January 2025 results from the Phase 1 healthy volunteer study of BLU-808, a wild-type KIT inhibitor, showing a differentiated profile that enables the evaluation of tunable dosing strategies. BLU-808 was well-tolerated at all doses tested, showed consistent pharmacokinetics supporting once daily oral dosing, and achieved dose-dependent reductions in tryptase exceeding 80 percent. These positive data support initiation of multiple proof-of-concept studies planned in 2025.

Corporate

● Presented the company’s 2025 corporate outlook and strategy to drive continued growth at the 43rd Annual J.P. Morgan Healthcare Conference. View the press release here.
● Following closing of the IDRx acquisition by GSK, Blueprint anticipates approximately $80 million in gross proceeds from an equity stake in IDRx.
● Announced the appointment of Sherwin Sattarzadeh to Chief Business Officer, overseeing business development, portfolio leadership and project management, and alliance management. His previous roles over a decade of service at Blueprint include Head of Regulatory Affairs, Chief of Staff to the Chief Executive Officer, and Senior Vice President of Strategic Operations. He succeeds Helen Ho, Ph.D. who is departing the company to pursue another career opportunity.

2025 Financial Guidance

Blueprint reported it anticipates approximately $680 million to $710 million in global AYVAKIT net product revenues for all approved indications in 2025. The midpoint of this range represents 45 percent year-over-year revenue growth. In 2024, Blueprint reduced cash burn by more than 50 percent and expects further reduction in 2025 as the company continues to invest in advancing prioritized programs, balancing investing in innovation with financial discipline. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will maintain a durable capital position to enable the company to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following milestones in the first half of 2025:

● Present 14 abstracts at the 2025 American Academy of Allergy, Asthma and Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress, including:
o Three-year safety and efficacy data from the PIONEER trial of AYVAKIT.
o Bone density improvements shown by AYVAKIT in the PIONEER trial.
o Phase 1 healthy volunteer data for BLU-808, a highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line results reported in January 2025.
● Initiate two proof-of-concept studies of BLU-808 in patients with chronic spontaneous urticaria/chronic inducible urticaria, and allergic rhinitis/allergic conjunctivitis.

Fourth Quarter and Year End 2024 Results

● Revenues: Revenues were $146.4 million for the fourth quarter of 2024, including $144.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.2 million in collaboration revenues. Revenues for the year ended December 31, 2024 were $508.8 million, including $479.0 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $29.9 million in collaboration and license revenues. Blueprint Medicines recorded $72.0 million and $249.4 million in revenues in the fourth quarter and year ended December 31, 2023, respectively.
● Cost of Sales: Cost of sales was $7.4 million for the fourth quarter of 2024 and $20.2 million for the year ended December 31, 2024, as compared to $0.3 million and $8.5 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to higher sales to our collaboration and other partners and an increase in product sales volume.
● R&D Expenses: Research and development expenses were $83.7 million for the fourth quarter of 2024 and $341.4 million for the year ended December 31, 2024, as compared to $97.5 million and $427.7 million for the fourth quarter and year ended December 31, 2023, respectively. This decrease was primarily due to operational efficiency across our portfolio as we executed across our top priority programs, and the timing of manufacturing of clinical trial materials. Research and development expenses also included $11.7 million in stock-based compensation expenses for the fourth quarter of 2024 and $47.5 million in stock-based compensation for the year ended December 31, 2024.
● SG&A Expenses: Selling, general and administrative expenses were $96.5 million for the fourth quarter of 2024 and $359.3 million for the year ended December 31, 2024, as compared to $79.3 million and $295.1 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $16.6 million in stock-based compensation expenses for the fourth quarter of 2024 and $61.5 million in stock-based compensation for the year ended December 31, 2024.
● Net Loss: Net loss was $(50.0) million for the fourth quarter of 2024 and $(67.1) million for the year ended December 31, 2024, or a diluted net loss per share of $(0.79) and diluted net loss per share of $(1.07), respectively, as compared to a net loss of $(110.9) million for the fourth quarter of 2023 and a net loss of $(507.0) million for the year ended December 31, 2023, or a diluted net loss per share of $(1.82) and a diluted net loss per share of $(8.37), respectively.
● Cash Position: As of December 31, 2024, cash, cash equivalents and investments were $863.9 million, as compared to $767.2 million as of December 31, 2023.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss fourth quarter and full year 2024 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international), and referring to conference ID 349846. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in two upcoming investor conferences:

● 45th Annual Cowen Health Care Conference on Monday, March 3, 2025 at 1:10 p.m. ET.
● H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference on Thursday, March 27, 2025 at 12:00 p.m. ET.

Live webcasts of the presentations will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source Replays of the webcasts will be archived on the Blueprint Medicines website for 30 days following the presentations.

On February 13, 2025 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported the receipt of newly issued patents in the United States and New Zealand, and updated investors on the extent of its global intellectual property portfolio (Press release, Bio-Path Holdings, FEB 13, 2025, View Source [SID1234650244]).

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Bio-Path received Notice of Allowance from the United States Patent and Trademark Office for U.S. Patent No. 17/339,366 titled, "P-ethoxy nucleic acids for STAT3 inhibition." The New Zealand Intellectual Property Office has granted Patent No. 741793 titled, "P-ethoxy nucleic acids for liposomal formulation." These new patents build on earlier patents granted that protect the platform technology for DNAbilize, the Company’s novel RNAi nanoparticle drugs.

"We are proud of our growing global intellectual property portfolio as it safeguards our technology, serves as a deterrent to would-be competitors and creates value around our core competencies," said Peter Nielsen, Chief Executive Officer of Bio-Path. "These efforts are designed to protect our investments and advance our clinical programs to bring new medicines to patients suffering with obesity and cancer. Importantly, our newly issued U.S. patent provides additional intellectual property protection around our STAT3 program, where we intend to advance into non-small cell lung cancer."

Bio-Path continues to expand its intellectual property portfolio by filing patent applications that are applicable to its technology and business strategy. Bio-Path’s patent portfolio currently includes seven issued patents in the U.S. and 61 issued patents in foreign jurisdictions, providing protection in 26 countries. The Company has three additional pending patent applications in the U.S. and five additional allowed patent applications in foreign jurisdictions.

On February 13, 2025 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported an update from the Company’s ongoing Phase 1/1b clinical trial of BP1001-A in solid tumor patients and reports continued patient progress from the Company’s ongoing Phase 2 triple combination study of prexigebersen in Acute Myeloid Leukemia (AML) (Press release, Bio-Path Holdings, FEB 13, 2025, View Source [SID1234650243]).

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"These continued positive responses mark a significant milestone for Bio-Path as they suggest our DNAbilize platform technology has the potential to produce multiple drug candidates capable of target-specific protein inhibition for over-expressed, disease-causing gene products," said Peter H. Nielsen, President and Chief Executive Officer of Bio-Path. "As previously reported in August 2024, we were thrilled to see that our first patient treated with the higher dose (90 mg/m2) in our Phase 1/1b study of BP1001-A who has shown tumor regression and stable disease continued successful treatment through a tenth treatment cycle. We believe this is significant, particularly considering the heavily pretreated and fragile patient population involved."

"This elderly female patient with gynecologic cancer had previously been treated with multiple lines of chemotherapy along with multiple surgeries for her disease, and only now is showing positive clinical results with BP1001-A treatment. Importantly, we are not seeing the onerous side effects typically seen in patients with advanced solid tumors being treated with standard chemotherapies," continued Mr. Nielsen.

"In addition, we previously noted extended treatment durability in two elderly patients in our Phase 2 triple combination study of prexigebersen, venetoclax and decitabine in AML patients. We are pleased to report that each of these patients remain in complete remission after two years of treatment. These ongoing positive outcomes underscore the potential for prexigebersen to treat fragile AML patients for extended periods. We are particularly pleased with these results, as elderly AML patients are typically unable to tolerate intensive chemotherapy and thus experience very poor clinical outcomes," concluded Mr. Nielsen.

Solid Tumor Patient Response in Second, Higher Dose Cohort – Previously, Bio-Path reported its first patient in the second dose cohort in its Phase 1/1b advanced solid tumor clinical trial experienced a positive response that may signal that this analog of prexigebersen has potential as a new treatment for advanced solid tumors. The patient appears to be doing well on study after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through six cycles of treatment. Moreover, it appears that these positive outcomes may have contributed to allowing her to continue with rigorous exercise and improved quality of life.

As of January 2025, this patient continues doing well on treatment, recently completing nine cycles and is now in her tenth treatment cycle.

The dose finding portion of the Phase 1/1b trial is comprised of BP1001-A monotherapy with no accompanying chemotherapy. This clinical trial of BP1001-A in patients with advanced or recurrent solid tumors has successfully completed the initial prescribed dose in the first cohort of 60 mg/m2 and began enrollment in the higher dose cohort of 90 mg/m2. The Phase 1b portion of the study is expected to commence after completion of three planned BP1001-A monotherapy dose level cohorts and is intended to assess the safety and efficacy of BP1001-A in combination with paclitaxel in patients with recurrent ovarian or endometrial tumors. Phase 1b studies are also expected to be opened in combination with gemcitabine in late-stage pancreatic cancer.

AML Patients Demonstrate Extended Treatment Durability – Previously, Bio-Path reported two patients were identified in the Phase 2 clinical trial treating AML patients who demonstrated continued treatment durability. As of January 2025, both these patients are receiving treatment and are continuing to do well. The first patient is an elderly female who had received 16 cycles of treatment over 21 months when first reported on. She continues on study having received 20 cycles over 26 months and she remains in complete remission. The second patient is an elderly male who had received 12 cycles of treatment over 14 months when first reported on. He continues on study having received 16 cycles over 20 months and remains in complete remission. Both patients are being treated with the triple combination of prexigebersen, decitabine and venetoclax.

On February 13, 2025 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in PCR-based DNA technologies, reported financial results for its first quarter of fiscal 2025 ended December 31, 2024 (Press release, Applied DNA Sciences, FEB 13, 2025, View Source [SID1234650241]). The Company’s Form 10-Q, once filed, can be viewed on the SEC Filings page of its Investor Relations website.

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Recent Corporate and Operational Updates:

Corporate:

In December 2024, the Company announced a strategic restructuring of its business operations, including the potential divestiture of its DNA Tagging and Security Products and Services ("DNA Tagging") business segment.
Today, the Company announced its exit from its DNA Tagging business segment and completion of a workforce reduction of approximately 20% of its total headcount related primarily to employees within its DNA Tagging segment. The workforce reduction will result in an approximate 13% reduction in annual payroll costs, offset by one-time separation costs totaling approximately $300,000, which are expected to be recorded in the quarter ending March 31, 2025. The Company will continue to service certain of its existing DNA Tagging customer contracts.
LineaRx (Therapeutic DNA Production and Services subsidiary)

The buildout of the Company’s initial GMP facility located in Stony Brook, New York ("Site 1"), was completed on January 31, 2025, and certified for commercial operation with ISO 7-compliant with ISO 5-compliant workspaces. As currently configured, Site 1 enables the enzymatic manufacture of Linea DNA IVT templates used in the production of mRNA clinical trial materials:
The initial projected manufacturing capacity of Site 1 is approximately ten grams per annum, which supports potential annual revenues in the range of $10 million to $30 million, depending on product mix[1].
Site 1 gives the Company the new ability to service clients across all customer product stages with relevant grades of DNA: RUO for research and development; GLP for discovery and early pre-clinical studies; GMP for late pre-clinical studies and clinical phases.
The Company is in the late stage of process development with a U.S.-based therapeutics developer. If development is completed successfully, the Company anticipates its receipt of a first GMP order for IVT templates in the quarter ending June 30, 2025.
The Company’s Linea DNA platform secured its first-in-human clinical validation with Linea DNA used as a critical component in the manufacture of a CAR-T therapy under a Czech Republic State Institute for Drug Control-approved Phase I clinical trial sponsored by the Institute of Hematology and Blood Transfusion (UHKT). The trial represents the first instance of regulatory consent of Linea DNA’s use in a clinical setting and demonstrates Linea DNA’s ability to empower faster manufacturing timelines than conventional plasmid DNA-based therapy development.
Applied DNA Clinical Labs (MDx Testing Services subsidiary)

Completed certifications necessary to expand the TR8 PGx testing service to all S. States that recognize New York’s CLEP/CLIA certification for genetic testing. Samples collected from outside of New York State will be tested at Applied DNA’s diagnostic laboratory in Stony Brook.
Management Commentary
"Our first quarter performance reflects the implementation of a strategic restructuring to support our growth through our synthetic DNA manufacturing strategy," stated Dr. James A. Hayward, Chairman and CEO of Applied DNA. "We are taking difficult but necessary steps to optimize our corporate structure to lower our cash burn rate and stabilize our financial position to ensure our ability to execute against near-term operational goals."

"As we move through fiscal 2025, we are focused on commercializing the DNA production capacity of our recently certified GMP Site 1 facility," concluded Dr. Hayward. "We are preparing for initial orders of clinical grade materials, the acquisition of which we believe will validate the economics of our proprietary low-CAPEX approach to enzymatic DNA production in front of an industry that is actively seeking cell-free, synthetic alternatives to traditional pDNA production processes. We believe our capacity for the GMP production of DNA in an economical, fast, and scalable manner to advance the rapid development of genetic medicines is a unique competitive advantage in the marketplace and is the lynchpin to our future success."

First Quarter Fiscal 2025 Financial Highlights

Total revenues: $1.2 million, an increase of 34% compared to $891 thousand for the first quarter of fiscal 2024.
Operating loss: $3.0 million, compared to an operating loss of $3.8 million for the first quarter of fiscal 2024.
Net loss: $2.7 million, compared to a net loss of $1.1 million for the first quarter of fiscal 2024.
Adjusted EBITDA: improved to negative $2.9 million, compared to negative $3.2 million for the first quarter of fiscal 2024.
Cash and cash equivalents as of December 31, 2024: $9.3 million, which includes $5.7 million of net proceeds (after deducting placement agent fees and other offering expenses) from the registered direct offering that closed on October 31, 2024.
February 13 Investor Update Conference Call Information

The Company will hold a conference call and webcast to update investors on its restructuring and GMP roadmap on February 13, 2025, at 4:30 PM ET. To participate in the conference call, please follow the instructions below. While every attempt will be made to answer investors’ questions on the Q&A portion of the call, not all questions may be answered.

To participate, please ask to be joined to the ‘Applied DNA Sciences’ call:

Domestic callers (toll free): 844-887-9402
International callers: 412-317-6798
Canadian callers (toll free): 866-605-3852
Live and replay of webcast: link
Telephonic replay (available 1 hour following the conclusion of the live call through February 20, 2025):

Domestic callers (toll free): 1-877-344-7529
Canadian callers (toll free): 1-855-669-9658
Participant Passcode: 7896562
An accompanying slide presentation that will be embedded in the webcast can be accessed under ‘News & Events’ tab and ‘Company Events’ section of the Applied DNA investor relations website at View Source

On February 13, 2025 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 publication of data from its Phase 1 ALPHA and ALPHA2 clinical studies of cemacabtagene ansegedleucel (cema-cel; formerly ALLO-501/A) in relapsed/refractory (R/R) large B-cell lymphoma (LBCL) as a Rapid Communication in the Journal of Clinical Oncology (Press release, Allogene, FEB 13, 2025, View Source [SID1234650240]). These results represent the largest dataset of LBCL patients treated with an allogeneic CAR T product and, with a minimum of two years of follow-up, the longest follow-up to date.

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"Publication of the Phase 1 ALPHA/ALPHA2 trials in R/R LBCL mark a landmark moment for the field. These findings represent the most robust allogeneic CAR T experience yet presented and show, for the first time, that an "off-the-shelf" CAR T can induce durable complete remissions in a large fraction of patients with heavily pretreated LBCL," said Frederick L. Locke, MD, Chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center and Research Institute (Tampa, FL). "These peer-reviewed results highlight how cema-cel development is on the cutting edge of lymphoma care, particularly with the ALPHA3 trial targeting only those patients who are MRD positive at the end of first-line treatment. If successful, ALPHA3 and cema-cel could transform the treatment paradigm for newly diagnosed patients."

"With multiple patients in ongoing complete remissions beyond four years, the lingering question of whether an allogeneic CAR T could deliver durable responses has now been answered," said Zachary Roberts, M.D., Ph.D., Executive Vice President, Research and Development and Chief Medical Officer of Allogene. "Furthermore, these results provide potent evidence supporting the use of CAR T in patients with low disease burden and the unique opportunity for the ALPHA3 trial to achieve something novel in this disease – predict and intervene before relapse. Opportunities to redefine the standard of care in oncology are rare, but if successful, ALPHA3 has the potential to achieve precisely that."

Key Findings from the Publication
The ALPHA/ALPHA2 studies were single-arm, multicenter, open-label, Phase 1 trials. As of the data cutoff date (September 26, 2024), 87 heavily pretreated patients with R/R non-Hodgkin lymphoma (NHL) were treated in the ALPHA/ALPHA2 studies between May 2019 and September 2022. In total, 33 CD19 CAR T-naive patients with R/R LBCL received cema-cel/ALLO-501 manufactured with the process selected for use in pivotal studies and were the focus of this publication.

Overall Response Rate (ORR) and Complete Response (CR) Rate: ORR and CR rates in the ALPHA/ALPHA2 trials were consistent with those observed with approved autologous CD19 CAR T cell products for patients with R/R LBCL after two or more lines of systemic therapy. All treatment regimens studied demonstrated clinical benefit. The selected Phase 2 regimen (fludarabine/cyclophosphamide lymphodepletion with 90 mg of ALLO-647 (FCA90) followed by a single dose of CAR+ cells) yielded the highest ORR and CR of 67% and 58%, respectively.
Durability of Response (DOR): Patients who achieved a CR had excellent outcomes with a median DOR, PFS (progression free survival) and OS of 23.1 months, 24 months, and not reached, respectively. For patients receiving the selected Phase 2 regimen, median DOR was 23.1 months and median OS was not reached.
Safety Profile: The overall safety profile, including incidence of cytopenias and infections, was manageable and consistent with that of approved autologous CD19 CAR T cell therapies. There were no dose-limiting toxicities, graft-versus-host disease (GvHD), immune effector cell-associated neurotoxicity syndrome (ICANS), or high-grade cytokine release syndrome (CRS). The most common any-grade treatment emergent adverse events (TEAE) (≥25%) were neutropenia (85%), anemia (67%), thrombocytopenia (58%), infusion-related reactions (IRRs; 58%), fatigue (52%), and pyrexia (49%), nausea (39%), lymphopenia (36%), hypotension (36%), peripheral edema (33%), decreased white blood cell count (30%), CMV reactivation (30%), decreased appetite (30%), chills (30%), and hypoxia (27%).
Time to Treatment: The median time to start of treatment was two days from study enrollment. In contrast, autologous CAR T cell products require wait times often longer than 1 month despite incremental advancements in manufacturing and supply chains.
Potential in Low Disease Burden Settings
A growing body of evidence indicates that treatment with CAR T at times when the disease burden is low leads to improved safety and efficacy outcomes and this study reported similar findings. Among patients with baseline tumor burden <1000 mm² or normal lactate dehydrogenase (LDH) levels prior to treatment, a blood test that indicates low disease activity, the CR rate was 100% (6/6) and 82% (9/11), respectively. These CR rates in this subpopulation support cema-cel as a promising therapeutic option in patients with minimum residual disease (MRD), the population currently being studied in the ALPHA3 trial.

Foundation for the ALPHA3 Trial
These results serve as a foundation for the ongoing ALPHA3 trial, which is evaluating cema-cel as a consolidation therapy in LBCL patients who are in remission following 1L treatment but remain positive for minimal residual disease (MRD) as detected by an ultrasensitive ctDNA based blood test, using Foresight Diagnostics’ investigational CLARITY powered by PhasED-Seq. These patients have extremely low disease burden, a key subgroup who demonstrated excellent disease outcomes in the ALPHA/ALPHA2 trials.

The groundbreaking randomized controlled ALPHA3 trial, initiated in June 2024, is the first to evaluate CAR T treatment as part of 1L consolidation treatment regimen for LBCL patients who achieve remission but test positive for MRD following initial therapy. The ALPHA3 trial is designed to predict and intervene before relapse. Cema-cel is administered only to patients at high risk for relapse as a one-time consolidation dose before disease recurs.

About Cemacabtagene Ansegedleucel (cema-cel)
Cemacabtagene ansegedleucel, or cema-cel, is a next generation anti-CD19 AlloCAR T investigational product for the treatment of large B cell lymphoma (LBCL). In June 2022, the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to cema-cel in r/r LBCL. The ALPHA3 pivotal Phase 2 trial in first line (1L) consolidation for the treatment of LBCL launched in June 2024. Allogene has oncology rights to cema-cel in the US, EU and UK with options for rights in China and Japan.

About the ALPHA3 Trial
Over 60,000 patients are expected to be treated for LBCL annually in the US, the EU and the UK. While first line (1L) R-CHOP or other chemoimmunotherapy is effective for most patients, approximately 30% will relapse and require subsequent treatment. The current standard of care (SOC) after 1L treatment has been simply to "watch and wait" to see if the disease relapses. The pivotal Phase 2 ALPHA3 study takes advantage of cema-cel as a one-time, off-the-shelf treatment that can be administered immediately upon discovery of MRD following six cycles of R-CHOP or other chemoimmunotherapy, positioning it to become the standard "7th cycle" of frontline treatment available to all eligible patients with MRD.