On May 13, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Sellas Life Sciences, MAY 13, 2025, View Source [SID1234652989]).

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"We are very encouraged by the strong momentum across our pipeline," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The positive overall survival data in cohort 3 from the ongoing Phase 2 trial of SLS009 in r/r AML, showing a median OS that exceeds all historical benchmarks by over 3 times, further underscores the transformative potential of SLS009 for many underserved patients. In parallel, our new preclinical findings demonstrating the ability of SLS009 to overcome TP53-driven resistance, along with the promising clinical efficacy from the ongoing Phase 2, give us renewed optimism for patients across different genetic AML mutations. We look forward to presenting further data on SLS009 at ASCO (Free ASCO Whitepaper), highlighting its preclinical efficacy in ASXL1-mutated colorectal cancer lines. With the full topline Phase 2 data of SLS009 anticipated soon, and the final analysis of our Phase 3 pivotal REGAL trial of GPS in AML expected later this year, we are well-positioned for an exciting and meaningful 2025."

Recent Corporate Highlights:

Announced Positive Overall Survival in Cohort 3 from the Ongoing Phase 2 Trial of SLS009 in r/r AML: The data demonstrated that patients with AML-Myelodysplasia-Related Changes (AML-MRC) achieved a mOS of 8.9 months, while all relapsed or refractory to venetoclax-based regimens patients receiving 30 mg BIW achieved a mOS of 8.8 months, far surpassing the historical benchmark of 2.5 months. In addition, the therapy demonstrated a 67% ORR in patients with AML-MRC and 46% in all evaluable patients, significantly exceeding the targeted 20% ORR. The trial continues with full data and FDA regulatory path feedback expected in 1H 2025.

Presented Preclinical Data Highlighting Efficacy of SLS009 in TP53 Mutated AML at the 2025 AACR (Free AACR Whitepaper) Conference: Preclinical data suggest that SLS009 can induce apoptosis downstream of p53 by targeting critical proteins such as MCL-1 and survivin, regardless of p53 status. Immunoblot analysis reveals near-complete removal of these proteins in treated cells within 8 hours of exposure to SLS009. Furthermore, the treatment reduced TP53-mutated leukemia cell populations by up to 97% in combination with azacitidine–venetoclax, and by up to 80% as monotherapy.

Preclinical Efficacy of SLS009 in ASXL1 Mutated Colorectal Cancer to be Showcased at ASCO (Free ASCO Whitepaper) 2025: The poster, entitled, In vitro efficacy of CDK9 inhibitor tambiciclib (SLS009) in ASXL1 mutated colorectal cancer cell lines, will be presented on Monday, June 2, 2025, 1:30 PM-4:30 PM CDT.

Announced Positive Outcome of Interim Analysis for Phase 3 REGAL Trial of GPS in AML: The interim futility, efficacy, and safety analysis was designed to assess whether the therapy is safe, demonstrates potential efficacy, and merits continuation. The IDMC’s review supports the continuation of the study according to its original protocol. Based on this positive evaluation, GPS has shown preliminary signals of effectiveness, allowing the trial to advance toward completion. Fewer than 50% of enrolled patients were confirmed deceased after the median follow-up of 13.5 months, indicating a median survival of over 13.5 months in the trial vs. a historical median survival of 6 months for conventional therapy, as reported in a similar Phase 2 study. The next and final analysis will be conducted once 80 events (deaths) are reached, further determining the potential of GPS in addressing the needs of AML patients. SELLAS anticipates that 80 events will be reached this year.

Announced Promising Data from Phase 2a Trial of SLS009 in Combination with Zanubrutinib in DLBCL: The trial, conducted and funded by GenFleet Therapeutics (Shanghai), Inc. ("Genfleet"), was an open-label single-arm multicenter Phase 2a study in China evaluating SLS009 in combination with BTK inhibitor, Brukinsa (zanubrutinib) in r/r DLBCL. The results showed an overall response rate (ORR) of 67%, more than double the expected ORR of zanubrutinib alone. Among responders, one achieved complete response (CR), while three had partial response (PR) with target lesion shrinkages of 89%, 78%, and 56%, respectively. As of the last follow-up, after the median of 4.6 (range: 1.4 – 7.4) months follow-up, median overall survival (OS) was not reached, and 6 out of 9 patients were alive. GenFleet will determine the next steps in development around lymphoma as SELLAS’ focus remains on AML and spliceosome–chromatin mutations, including ASXL1 mutations.

PIVOT – Received Preliminary Data for Pediatric Acute Lymphoblastic Leukemia (ALL) Patients Derived Xenografts (PDX): The experiment conducted and funded by the National Institute of Health (NIH) through the PIVOT program included 27 patient-derived ALL tumors from pediatric patients. Tumors were xenografted in mice in two groups: vehicle control arm and SLS009 arm. Mice were treated with a fractionated dose once per week for 6 consecutive weeks. The treatment was well tolerated. For all models, median survival was approximately tripled in the SLS009 arm compared to the vehicle control arm. SLS009 demonstrated delayed progression in 25/27 (93%) models and more than 2 times longer time to progression in 15/27 (56%) of ALL models. In addition, there were complete responses (CR) in 2 models, and in one of the two models, CR was maintained after the treatment had been completed until the end of the study (4 months). Among 7 KMT2A rearranged models, time to progression was extended in all 7 models, and in 6/7 (86%) time to progression was more than doubled.

Raised $25.0 Million of Gross Proceeds from a Registered Direct Offering Priced At-the-Market under Nasdaq Rules: On January 28, 2025, SELLAS announced the closing of a $25 million registered direct offering with a single healthcare-focused institutional investor before deducting placement agent’s fees and related offering expenses. The net proceeds from the offering strengthens the Company’s financial position and will be used for working capital purposes and general corporate procedures, including the purchase of any pending or future acquisitions.

Financial Results for the First Quarter 2025:

R&D Expenses: Research and development expenses for the quarter ended March 31, 2025, were $3.2 million, compared to $5.1 million for the same period in 2024. The $1.9 million decrease was primarily due to decreases in clinical trial expenses, manufacturing costs, and clinical drug supply purchases, and clinical and regulatory consulting costs primarily driven by the completion of enrollment in the REGAL study in the first quarter of 2024.

G&A Expenses: General and administrative expenses for the first quarter of 2025 were $2.9 million, as compared to $4.5 million for the same period in 2024. The $1.6 million decrease was primarily attributable to a decrease in personnel related expenses driven by the initial recognition of a one-time severance charge during the three months ended March 31, 2024, and a decrease in headcount, and decreases in professional fees and other general and administrative expenses.

Net Loss: The net loss was $5.8 million for the first quarter of 2025, or a basic and diluted loss per share of $0.07, as compared to a net loss of $9.6 million for the first quarter of 2024, or a basic and diluted loss per share of $0.21.

Cash Position: As of March 31, 2025, cash and cash equivalents totaled approximately $28.4 million. Subsequent to March 31, 2025, the Company received $4.0 million in proceeds from the exercise of warrants.

On May 13, 2025 Orchard Therapeutics, a Kyowa Kirin company, reported a publication on long-term safety and efficacy outcomes for Lenmeldy (atidarsagene autotemcel) in the treatment of early-onset metachromatic leukodystrophy (MLD) (Press release, Orchard Theraputics, MAY 13, 2025, View Source [SID1234653172]). The manuscript, titled, "Long-term effects of atidarsagene autotemcel for metachromatic leukodystrophy" (F. Fumagalli, V. Calbi, A. Aiuti, et. al.) was published in the April 24 issue of NEJM.

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MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease that affects approximately one in 100,000 live births. It is caused by an error in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. These children eventually deteriorate into a vegetative state, which may require 24-hour intensive care, and the majority pass away within five years of symptom onset, creating an enormous emotional and financial burden on the family. Lenmeldy is the only approved therapy intended to correct the underlying cause of MLD for eligible patients in the United States (U.S.). It is known as Libmeldy in Europe.

The FDA approval of Lenmeldy is based on data from pediatric patients with early-onset MLD enrolled in two single-arm, open-label clinical studies or treated under European expanded access frameworks, who received a one-time administration of the hematopoietic stem cell (HSC) gene therapy and compared with natural history data from untreated patients. The NEJM publication details clinical outcomes of children treated in these studies. All treated patients were administered HSC gene therapy and subsequently monitored at Ospedale San Raffaele in Milan, Italy. The primary endpoint was severe motor impairment-free survival (sMFS), defined as the interval from birth to the first occurrence of loss of locomotion and loss of sitting without support (Gross Motor Function Classification-MLD [GMFC-MLD] Level ≥ 5) or death.

With more than 12 years of follow-up in the earliest treated patients (median 6.76 years) and more than 250 patient years, treatment with Lenmeldy significantly extended overall survival and resulted in the preservation of motor function and cognitive skills in most late infantile MLD patients past ages at which untreated patients showed severe cognitive and motor impairments. Lenmeldy also resulted in the preservation of motor function and cognitive skills in some early juvenile MLD patients which is not expected when compared to untreated patients.

Treatment with Lenmeldy was well-tolerated, with no treatment-related serious adverse events. Most adverse events were associated with busulfan conditioning or background disease. The most common non-laboratory adverse reactions (incidence ≥ 10%) were: febrile neutropenia (85%), stomatitis (77%), respiratory tract infections (54%), rash (33%), device related infections (31%), other viral infections (28%), pyrexia (21%), gastroenteritis (21%), and hepatomegaly (18%). The most common laboratory abnormalities were: elevated D-dimer (67%), neutropenia (28%), and elevated liver enzymes (23%). Please see below for additional details and Important Safety Information.

"Lenmeldy represents a significant step forward in the treatment of MLD, a cruel and ultimately fatal disease for which there were previously no approved treatment options beyond supportive and end-of-life care," said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. "These compelling results, which encompass more than a cumulative 250 years of patient experience, continue to demonstrate the ability of Lenmeldy to preserve motor and cognitive function in eligible children with MLD particularly when treatment is administered prior to the onset of symptoms."

Dr. Gaspar continued, "These long-term results, coupled with the homogenous, predictable and precipitous decline and eventual death observed in the natural history cohort, underscores the urgent need to enable timely and accurate diagnosis and intervention through the proliferation of universal newborn screening for MLD."

Summary of Participation at ASGCT (Free ASGCT Whitepaper) 2025

The company also announced six presentations (three oral and three posters) will be featured at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting taking place May 13-17 in New Orleans.

Featured data include several accepted abstracts and presentations detailing clinical, biochemical and other functional outcomes from across the company’s commercial- and clinical-stage neurometabolic portfolio in metachromatic leukodystrophy (MLD), the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), and mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A.

Company researchers will also present data derived from its efforts to develop an automated manufacturing process for HSC gene therapy production and in developing an HSC gene therapy approach for the treatment of another lysosomal storage disorder.

Finally, Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics, will give an invited talk on Friday, May 16 at 8:00 a.m. CDT, titled "Developing and delivering hematopoietic stem cell gene therapies to patients with rare neurometabolic diseases," which will explore key insights into the development and delivery of one-time treatments for rare neurometabolic diseases and beyond.

Details of the oral presentations are as follows (all times in CDT):

Title: Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD): Interim analysis of a Phase III trial
Date/Time: Wednesday, May 14 at 2:45 p.m.
Presenter: Valeria Calbi
Title: Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD)
Date/Time: Friday, May 16 at 1:30 p.m.
Presenter: Valeria Calbi
Title: Extensive detoxification and favorable effects on systemic clinical outcomes after Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type I-Hurler (OTL-203)
Date/Time: Friday, May 16 at 2:00 p.m.
Presenter: Giulia Consiglieri
Details of the poster presentations are as follows (all times in CDT):

Title: Toward the development of an automated manufacturing process for hematopoietic stem cell gene therapies
Date/Time: Wednesday, May 14 from 5:30 to 7:00 p.m.
Presenter: Vasileios Paraskevas
#1302
Title: Anti-SGSH antibodies following hematopoietic stem cell (HSC) gene therapy in MPSIIIA patients neither impact engraftment of genetically modified HSC nor interfere with multi-compartment substrate reduction
Date/Time: Thursday, May 15 from 5:30 to 7:00 p.m.
Presenter: Brian Bigger
#1513
Title: Correction of Glycogen Accumulation in Muscle, Heart and CNS in a Pre-Clinical Model of Hematopoietic Stem Cell Gene Therapy for Pompe Disease
Date/Time: Thursday, May 15 from 5:30 to 7:00 p.m.
Presenter: Piv Sagoo
#1514
About MLD
MLD is a rare and life-threatening inherited disease of the body’s metabolic system estimated to occur in approximately one in every 100,000 live births based on existing literature. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity, and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50 percent and 44 percent at 10 years for juvenile patients.i

About Lenmeldy / Libmeldy
Lenmeldy (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the U.S. for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

For additional details about Lenmeldy, please refer to the full Prescribing Information.

In Europe, Lenmeldy is known as Libmeldy, where it has been approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA). For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

The program was originated by and developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

INDICATION

LENMELDY (atidarsagene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Thrombosis and Thromboembolic Events:

Treatment with LENMELDY may increase the risk of thrombosis and thromboembolic events. A child with PSEJ MLD died after experiencing a left hemisphere cerebral infarction secondary to a thrombotic event in a large blood vessel approximately 1 year after treatment with LENMELDY. Evaluate the risk factors for thrombosis prior to and after LENMELDY infusion according to best clinical practice.

Encephalitis:
Treatment with LENMELDY may increase the risk of encephalitis. A child with ESEJ developed a serious event of encephalitis after treatment with LENMELDY. The etiology of this event is unclear but attribution to LENMELDY cannot be ruled out. Treatment with LENMELDY may trigger a relapsing-remitting pattern of disease progression. No other events related to encephalitis have been reported during the clinical development of LENMELDY. Monitor children for signs or symptoms of encephalitis after LENMELDY treatment.

Serious Infection:
In the period between start of conditioning and within 1 year after LENMELDY treatment, severe Grade 3 infections occurred in 39% of all children (21% bacterial, 5% viral, 5% bacterial and viral or bacterial and fungal, and 8% unspecified). Grade 3 febrile neutropenia developed within 1 month after LENMELDY infusion in 82% of children. In the event of febrile neutropenia, monitor for signs and symptoms of infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated. Monitor children for signs and symptoms of infection after myeloablative conditioning and LENMELDY infusion and treat appropriately. Administer prophylactic antimicrobials according to best clinical practice.

Veno-Occlusive Disease:

Three children (8%) treated in clinical trials of LENMELDY developed veno-occlusive disease (VOD) with one Grade 4 SAE and two Grade 3 AEs. None of these three events met Hy’s Law criteria. Monitor children for signs and symptoms of VOD including liver function tests in all children during the first month after LENMELDY infusion. Consider prophylaxis for VOD with anti-thrombotic agents based on risk factors for VOD and best clinical practice.

Delayed Platelet Engraftment (DPE):
DPE has been observed with LENMELDY treatment. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in children with prolonged thrombocytopenia. In clinical trials of LENMELDY, 4 (10%) children had delayed platelet engraftment after day 60 (range day 67-109), with 3 children requiring platelet transfusions until engraftment occurred. Patients should be informed of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding until platelet engraftment and recovery are achieved.

Neutrophil Engraftment Failure:
There is a potential risk of neutrophil engraftment failure after treatment with LENMELDY. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a child treated with LENMELDY, provide rescue treatment with the unmanipulated back-up collection of CD34+ cells.

Insertional Oncogenesis:
There is a potential risk of LVV-mediated insertional oncogenesis after treatment with LENMELDY. Children treated with LENMELDY may develop hematologic malignancies and should be monitored life-long. Monitor for hematologic malignancies with a complete blood count (with differential) annually and integration site analysis as warranted for at least 15 years after treatment with LENMELDY. In the event that a malignancy occurs, contact Orchard Therapeutics at 1-888-878-0185 for reporting and to obtain instructions on collection of samples for testing.

Hypersensitivity Reactions:
The dimethyl sulfoxide (DMSO) in LENMELDY may cause hypersensitivity reactions, including anaphylaxis which is potentially life-threatening and requires immediate intervention. Hypersensitivity including anaphylaxis can occur in children with and without prior exposure to DSMO. Monitor for hypersensitivity reactions during infusion and after infusion.

Anti-Retroviral Use:
Children should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization, or for the expected duration of time needed for the elimination of the medications. Anti-retroviral medications may interfere with the manufacturing of LENMELDY. If a child requires antiretrovirals for HIV prophylaxis, initiation of LENMELDY treatment should be delayed until confirmation of a negative test for HIV.

Interference With Serology Testing:
Due to the likelihood of a false-positive test for HIV, children who have received LENMELDY should not be screened for HIV infection using a PCR-based assay.

USE IN SPECIFIC POPULATIONS

Females and Males of Reproductive Potential

Pregnancy Testing
As a precautionary measure, a negative serum pregnancy test must be confirmed prior to the start of mobilization, and reconfirmed prior to conditioning procedures, and before administration of LENMELDY in females of childbearing potential.

Contraception

Consult the Prescribing Information of the mobilization and conditioning agents for information on the need for effective contraception. Males capable of fathering a child and females of childbearing age should use an effective method of contraception from start of mobilization through at least 6 months after administration of LENMELDY.

Infertility
There are no data on the effects of LENMELDY on fertility.

Data are available on the risk of infertility with myeloablative conditioning. In clinical trials of LENMELDY, seven children (50% of females) developed ovarian failure. Advise children of the option to cryopreserve semen or ova before treatment, if appropriate.

For additional safety information, please see the full Prescribing Information.

On May 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 corporate updates and announced financial results for the quarter ended March 31, 2025 (Press release, Allogene, MAY 13, 2025, View Source [SID1234652953]).

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"We are executing on a strategy that is grounded in science, shaped by real-world insights, and supported by a platform purpose-built to scale," said David Chang, M.D., Ph.D., President, Chief Executive Officer, and Co-Founder of Allogene. "From transforming the role of CAR T in first line LBCL through ALPHA3 to breaking new ground in autoimmune disease with ALLO-329 to showing what’s possible in solid tumors with ALLO-316, we are leading where others have yet to go. The experience we’ve gained from our trials, the commitment from our partners, and the unmatched efficiencies of our allogeneic platform reinforce my confidence that our approach is not only working but also defining the future of cell therapy. We have never been clearer about our path or more certain about the value we can deliver to patients and the field."

Program Updates
Cema-Cel: Pivotal Phase 2 ALPHA3 1L Consolidation Trial in LBCL
The ALPHA3 trial evaluating cema-cel as 1L consolidation therapy for LBCL remains a cornerstone of Allogene’s clinical strategy. The trial now includes nearly 50 activated sites across the U.S., spanning both community cancer centers and leading academic institutions. The trial uniquely identifies high-risk patients using Foresight CLARITY powered by PhasED-Seq, an Investigational Use Only (IUO) MRD test, and randomizes approximately 240 patients to assess whether early intervention with cema-cel improves event-free survival (EFS) compared to observation, which is the current standard of care.

As the first study of its kind, ALPHA3 has delivered valuable real-world insights. Since launching in mid-2024, Allogene has partnered closely with sites to refine patient identification strategies; educated patients on their relapse risk and opportunity for additional therapy based on MRD status; and navigated expected and unforeseen challenges. Chief among these has been industry-wide factors that have delayed site readiness to initiate screening activities for three months or more after activation. As a result, the milestone for lymphodepletion regimen selection and futility analysis has been shifted by approximately two quarters and is now expected in the first half of 2026.

The momentum behind ALPHA3 however has been tangible and significant, driven by a strong partnership between Allogene and its clinical sites. Several sites have proactively collaborated with the Company to develop and share best practices, fostering a coordinated network. An important measure of success is the identification of patients early in 1L treatment who are interested in the trial and consenting them for MRD testing at completion of therapy. This metric is now being consistently met as earlier introduction to the trial has increased the proportion of screening-eligible patients advancing to MRD testing and, if positive, agreeing to randomization. With the earliest activated sites now screening regularly and newly activated sites implementing best practices immediately, we have now consented over 250 patients for MRD screening, with nearly half of these consents occurring in the last three months. Meanwhile, growing international interest has prompted expansion of the trial footprint outside the U.S., a move expected to accelerate enrollment further and strengthen trial execution.

The Company will intentionally hold off on projecting additional milestones until after the first milestone for lymphodepletion regimen selection and futility analysis. Recognizing the strategic significance of this inflection point and guided by stakeholder feedback, the Company is re-evaluating what data will be appropriate to share at the time of the lymphodepletion announcement.

ALLO-329: CD19/CD70 Dual CAR with Dagger Technology in AID
ALLO-329 offers a novel approach to treating autoimmune diseases as the first allogeneic CD19/CD70 dual CAR T product specifically designed to target CD19+ B-cells and CD70+ activated T-cells, both of which are key players in autoimmune diseases. The investigational product utilizes CRISPR-based site-specific integration and incorporates the Company’s clinically validated Dagger technology, which aims to reduce or eliminate the need for lymphodepletion, believed to be a potentially significant obstacle to the broader adoption of CAR T therapies in autoimmune indications.

The Phase 1 RESOLUTION basket trial in rheumatology will begin in mid-2025. The trial will include patients with systemic lupus erythematosus, including lupus nephritis, idiopathic inflammatory myopathies, and systemic sclerosis. The innovative design of the RESOLUTION trial will include two distinct lymphodepletion arms: one using a dose of cyclophosphamide alone and another that eliminates lymphodepletion entirely. The Company has shifted timing for its first update to this program to 1H 2026 to allow for both biomarker and clinical proof-of-concept data.

ALLO-316: TRAVERSE Trial in RCC
ALLO-316 is the only allogeneic CAR T therapy to show potential in solid tumors. Enrollment has completed in the Phase 1b cohort, which is evaluating the safety and efficacy of ALLO-316 at DL2 (80M CAR T cells) in patients with heavily pretreated advanced or metastatic RCC. The Company will present updated data from the Phase 1b cohort in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 1, 2025, in Chicago, Illinois.

2025 First Quarter Financial Results

Research and development expenses were $50.2 million for the first quarter of 2025, which includes $5.0 million of non-cash stock-based compensation expense.
General and administrative expenses were $15.0 million for the first quarter of 2025, which includes $7.1 million of non-cash stock-based compensation expense.
Net loss for the first quarter of 2025 was $59.7 million, or $0.28 per share, including non-cash stock-based compensation expense of $12.2 million.
The Company had $335.5 million in cash, cash equivalents, and investments as of March 31, 2025.
In recognition of the evolving macroeconomic environment and the importance of preserving capital, the Company has taken steps to optimize its operations and extend the financial runway. Strategic cost-realignment efforts have been implemented to prioritize high-impact, value-generating programs, which include the clinical advancement of cema-cel in the ALPHA3 trial and ALLO-329 in the RESOLUTION trial. Operational savings have been achieved through a tactical reduction in manufacturing operations, leveraging prior infrastructure investments and the inherent efficiencies of the Company’s allogeneic CAR T platform, while preserving core capabilities. These actions have extended the cash runway into the second half of 2027, allowing the Company to strengthen its ability to weather market uncertainty as it executes clinical programs through key milestones. As a result of these efforts, new guidance for 2025 is an expected decrease in cash, cash equivalents, and investments of approximately $150 million. GAAP Operating Expenses are now expected to be approximately $230 million, including estimated non-cash stock-based compensation expense of approximately $45 million. These estimates exclude any impact from potential business development activities.

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

On May 13, 2025 Instil Bio, Inc. ("Instil") (Nasdaq: TIL), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel therapies, reported its first quarter 2025 financial results and provided a corporate update (Press release, Instil Bio, MAY 13, 2025, View Source [SID1234652970]).

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Recent Highlights:

ImmuneOnco’s Phase 1b/2 trial of AXN-2510/IMM2510 in combination with chemotherapy in 1L NSCLC in China is ongoing and ImmuneOnco anticipates initial clinical data from first-line NSCLC patients in 2H 2025.
U.S. clinical study of AXN-2510/IMM2510 in combination with chemotherapy in 1L NSCLC anticipated to commence before the end of 2025: Instil anticipates initiating a U.S. clinical trial of AXN-2510/IMM2510 in combination with chemotherapy for 1L NSCLC patients before the end of 2025, assuming the necessary regulatory approvals are obtained.
First Quarter 2025 Financial and Operating Results:

As of March 31, 2025, Instil had $111.8 million in total cash, cash equivalents, restricted cash, marketable securities and long-term investments, which consisted of $15.4 million in cash and cash equivalents, approximately $1.0 million in restricted cash, $88.3 million in marketable securities and $7.2 million in long-term investments, compared to $115.1 million in total cash, cash equivalents, restricted cash and marketable securities, which consisted of $8.8 million in cash and cash equivalents, $1.8 million in restricted cash, and $104.5 million in marketable securities, as of December 31, 2024. Instil expects that its cash, cash equivalents, marketable securities and long-term investments as of March 31, 2025 will enable it to fund its current operating plan beyond 2026.

Research and development expenses were $5.4 million for the three months ended March 31, 2025, compared to $7.3 million for the three months ended March 31, 2024.

General and administrative expenses were $9.1 million for the three months ended March 31, 2025, compared to $12.4 million for the three months ended March 31, 2024.

Restructuring and impairment charges were $16.1 million for the three months ended March 31, 2025, compared to $4.3 million for the three months ended March 31, 2024.

Net loss per share, basic and diluted were $4.32 for the three months ended March 31, 2025, compared to $3.74 for the three months ended March 31, 2024. Non-GAAP net loss per share, basic and diluted were $1.32 for the three months ended March 31, 2025, compared to $2.39 for the three months ended March 31, 2024.

Note Regarding Use of Non-GAAP Financial Measures

In this press release, Instil has presented certain financial information that has not been prepared in accordance with U.S. generally accepted accounting principles ("GAAP"). These non-GAAP financial measures include non-GAAP net loss and non-GAAP net loss per share, which are defined as net loss and net loss per share, respectively, excluding non-cash stock-based compensation expense and restructuring and impairment charges. Instil believes that these non-GAAP financial measures, when considered together with the GAAP figures, can enhance an overall understanding of Instil’s financial performance. The non-GAAP financial measures are included with the intent of providing investors with a more complete understanding of Instil’s operating results. In addition, these non-GAAP financial measures are among the indicators Instil’s management uses for planning purposes and to measure Instil’s performance. These non-GAAP financial measures should be considered in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The non-GAAP financial measures used by Instil may be calculated differently from, and therefore may not be comparable to, non-GAAP financial measures used by other companies. Please refer to the below reconciliation of these non-GAAP financial measures to the comparable GAAP financial measures.

On May 13, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended March 31, 2025 and provided a corporate update (Press release, Tempest Therapeutics, MAY 13, 2025, View Source [SID1234652990]).

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"The amezalpat program continues to produce data that reinforce its potential as a cancer therapy, most recently in a presentation at AACR (Free AACR Whitepaper) showing that amezalpat reduced immunosuppression and activated the immune system to attack tumors. We were pleased to present these data that elucidate one part of the amezalpat mechanism of action and support the positive randomized Phase 2 data, including the benefit seen in patients with markers of immune resistance," said Stephen Brady, president and chief executive officer of Tempest. "We are actively engaged in exploring strategic alternatives to advance our promising clinical-stage programs and maximize stockholder value and, given the data, continue to have strong conviction in the potential of our oncology portfolio to drive meaningful impact for patients facing cancer."

Recent Highlights

Amezalpat (TPST-1120) (clinical PPARα antagonist):
Reported new data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting supporting the immune component of amezalpat’s dual mechanism of action and reinforcing its potential as a novel cancer treatment.
Granted both Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) for amezalpat for the treatment of patients with HCC.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist):
Granted Orphan Drug designation by the FDA to treat patients with FAP.
Received a "Study May Proceed" letter from the FDA for the Phase 2 trial for the treatment of FAP.
This trial, run by CP-CTNet and financially supported by the NCI’s Division of Cancer Prevention, underscores the urgent need for innovative cancer prevention strategies in high-risk patient populations. The Phase 2 study is expected to begin in 2025.
Corporate:
Announced (i) plans to explore a full range of strategic alternatives to advance the company’s promising clinical-stage programs and maximize stockholder value and (ii) a reduction in force that was completed on April 30, 2025.
Using cash on hand, the company repaid $3.5 million in full satisfaction of Loan and Security Agreement with Oxford Finance LLC in April 2025.

Financial Results

First Quarter 2025

Tempest ended the quarter with $21.5 million in cash and cash equivalents, compared to $30.3 million on December 31, 2024. The decrease was primarily due to cash used in operating activities, offset by proceeds of $1.5 million from the at-the-market offering program.
Net loss and net loss per share for the quarter were $10.9 million and $3.16, respectively, compared to $7.9 million and $4.62, respectively, for the same period in 2024.
Research and development expenses for the quarter were $7.6 million compared to $4.3 million for the same period in 2024. The $3.3 million increase was primarily due to an increase in costs incurred from engaging contract research and manufacturing organizations in preparation for our pivotal Phase 3 trial of amezalpat for the treatment of first-line HCC.
General and administrative expenses for the quarter were $3.3 million compared to $3.6 million for the same period in 2024. The $0.3 million decrease was primarily due to a decrease in consulting services.