On November 24, 2025 Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, reported that its first results from the ongoing inMMyCAR study, a Phase 1 clinical trial evaluating KLN-1010, a novel in vivo gene therapy that generates anti-BCMA CAR-T cells in patients with relapsed and refractory multiple myeloma, will be presented in a late-breaking oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2025 Annual Meeting in Orlando, Florida.

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The late-breaking abstract features results from the first three patients treated with KLN-1010. All patients achieved minimal residual disease (MRD) negativity at month 1 that persisted through three months in the patient with the longest follow up. CAR-T cell expansion and persistence of memory CAR-T cells occurred without the use of lymphodepleting chemotherapy, apheresis, or ex vivo cell manufacturing.

"KLN-1010 is beginning to show the extraordinary clinical outcomes that may be possible with in vivo CAR-T therapy — early, deep responses from a single infusion without the barriers that limit access to traditional CAR-T treatments," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. "These initial data point to a potentially powerful medicine while also providing clear clinical validation of our iGPS platform; enabling a growing number of partnered programs as well as our wholly owned pipeline that includes KLN-1010. We are encouraged by these first-in-human results and looking forward to sharing additional details from the study with the scientific and medical communities at the ASH (Free ASH Whitepaper) Annual Meeting."

"In these early patients, we are seeing both rapid MRD-negative responses and persistent memory-phenotype CAR-T cells, a combination that has been strongly prognostic for durable clinical benefit with existing CAR-T approaches," said Simon Harrison, MBBS, MRCP(UK), FRCPath(UK), FRACP, Ph.D., Director of the Centre of Excellence in Cellular Immunotherapy at the Peter MacCallum Cancer Centre and lead author of the late-breaking abstract. "Achieving these outcomes without lymphodepleting chemotherapy or CAR-T cell manufacturing underscores the potential of this in vivo approach to fundamentally expand access to CAR-T therapy for patients with relapsed and refractory multiple myeloma."

Oral Presentation Details:

Minimal residual disease (MRD)-negative outcomes following a novel, in vivo gene therapy generating anti–B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells in patients with relapsed and refractory multiple myeloma (RRMM): Preliminary results from inMMyCAR, the first-in-human Phase 1 study of KLN-1010

Date: Tuesday, December 9, 2025, 7:30 – 9:00 AM EST

Location: West Hall D2 (Orange County Convention Center)

Session Title: Late-Breaking Abstracts Session

About inMMyCAR

inMMyCAR is a Phase 1, open-label, dose-escalation clinical trial designed to assess the safety, tolerability, pharmacology and preliminary efficacy of a single dose of KLN-1010 in up to 40 patients. The primary endpoints are incidence and severity of treatment-emergent adverse events (TEAEs), including dose limiting toxicities (DLTs), and to establish the recommended Phase 2 dose of KLN-1010. KLN-1010 has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA). This Phase 1 clinical trial marks the first time KLN‑1010 will be evaluated in humans. Additional information and study site information may be found on clinicaltrials.gov (NCT07075185).

About Relapsed and Refractory Multiple Myeloma

Multiple myeloma is a hematologic malignancy characterized by the proliferation of plasma cells in the bone marrow, leading to bone destruction, anemia, renal dysfunction, and immunosuppression. It is driven by complex genetic and epigenetic alterations that promote malignant cell survival and resistance to apoptosis. Relapsed and refractory multiple myeloma is characterized by clonal evolution, drug resistance, and increased disease heterogeneity, heightening the need for accessible, personalized therapeutic strategies.

About KLN-1010

KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR‑T treatments, KLN‑1010 is administered to patients via direct transfusion and is designed to generate durable CAR‑T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.

(Press release, Kelonia Therapeutics, NOV 24, 2025, View Source [SID1234660923])

On November 24, 2025 CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a clinical-stage oncology company advancing leronlimab, a first-in-class humanized monoclonal antibody targeting the CCR5 receptor with therapeutic potential across multiple indications, including metastatic triple-negative breast cancer (mTNBC) and colorectal cancer (mCRC), reported that Dr. Milana V. Dolezal, Clinical Associate Professor, Medicine Oncology at Stanford University School of Medicine, will present a poster at the prestigious San Antonio Breast Cancer Symposium (SABCS), being held December 9-12, 2025, at the Henry B. Gonzalez Convention Center in San Antonio, Texas.

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Currently, mTNBC is associated with a poor prognosis. The efficacy of immune checkpoint inhibitors (ICIs) is reduced in patients with mTNBC who have low levels of PD-L1[1]. A recent review of CytoDyn’s prior oncology trials suggests that treatment with leronlimab was associated with an increase in levels of PD-L1, and that when combined with an ICI or preceded by treatment with an ICI, it may improve survival in patients with mTNBC[2].

"Our strategy to use leronlimab in a combined approach with ICIs continues to demonstrate great promise," said Jacob Lalezari, M.D., CEO of CytoDyn. "At SABCS 2025, we look forward to sharing details of a group of patients with mTNBC who were treated with leronlimab and different commercially available PD-L1/PD-1 immune checkpoint inhibitors who continue to demonstrate sustained long-term remission, including 3 individuals with no current evidence of disease."

Details of the poster presentation are as follows:

Title: Prolonged survival following PD-L1/PD-1 immune checkpoint inhibitor therapy after leronlimab-induced PD-L1 upregulation on cancer-associated macrophage-like cells and circulating tumor cells in patients with metastatic or locally advanced triple-negative breast cancer

Presenter: Dr. Milana V. Dolezal, MD, MSci, Clinical Associate Professor, Medicine Oncology at Stanford University School of Medicine

Poster ID: PS5-02-30

Date/Time: December 12, 2025, 12:30 PM–2:00 PM CST

Location: Exhibit Hall

A copy of the presentations will be made available on CytoDyn’s website under the Publications & Posters section after it is presented at the symposium.

(Press release, CytoDyn, NOV 24, 2025, View Source [SID1234660892])

On November 24, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that it will webcast its presentations at two investor conferences in December. The presentations will feature a business overview and update by Steve Harr, Sana’s President and Chief Executive Officer.

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Sana will present at Citi’s 2025 Global Healthcare Conference at 2:30 p.m. ET on Tuesday, December 2, 2025.
Sana will present at the 8th Annual Evercore Healthcare Conference at 2:35 p.m. ET on Wednesday, December 3, 2025.
The webcasts will be accessible on the Investor Relations page of Sana’s website at View Source A replay of each presentation will be available at the same location for 30 days following the conference.

(Press release, Sana Biotechnology, NOV 24, 2025, View Source [SID1234660908])

On November 24, 2025 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technologies to its Tumor Activated T Cell Engager (TRACTr), Tumor Activated Immunomodulator (TRACIr), and Adaptive Immune Response Modulator (ARM) platforms, reported it will host a virtual event on Monday, December 1, 2025, at 4:30 PM ET.

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David Campbell, Ph.D., President & Chief Executive Officer will provide an update on JANX007 primarily focused on Phase 1a dose escalation data and secondarily on Phase 1b expansion data in adult subjects with metastatic castration-resistant prostate cancer (mCRPC).

A live analyst question and answer session will follow the formal presentation. To register for the event, please click here.

Janux’s TRACTr, TRACIr and ARM Pipeline

Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with mCRPC. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. Janux is also advancing additional CD3-based TRACTr and CD28-based TRACIr programs for future clinical development, including a PSMA-TRACIr for use in combination with our PSMA-TRACTr JANX007, and a TROP2-TRACTr for the treatment of TROP2+ solid tumors. Janux is advancing its first ARM platform program candidate, a CD19-ARM for the potential treatment of autoimmune diseases toward clinical trials. Janux is also generating a number of additional TRACTr, TRACIr and ARM programs for potential future development.

(Press release, Janux Therapeutics, NOV 24, 2025, View Source [SID1234660924])

On November 24, 2025 Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) ("Day One" or the "Company"), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported three-year results from the OJEMDA pivotal Phase 2 FIREFLY-1 trial in an oral presentation at the 30th Annual Meeting & Education Day of the Society for Neuro-Oncology .

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New data from the ongoing FIREFLY-1 trial (Arm 1), with an updated median study duration of 40.6 months (data cutoff June 6, 2025), are described below. Primary trial results, including trial eligibility, patient demographics, efficacy and safety have been previously reported (Kilburn, et al. Nature Medicine 2024).

"We are excited by these updated three-year data showing that patients taking tovorafenib were able to spend meaningful time off therapy, with the option to retreat as needed," said Elly Barry, MD, Chief Medical Officer of Day One. "These findings highlight the potential for a treatment approach to help support patients through the long-term course of their disease and further support our view that tovorafenib has the potential to become the second line standard of care in pLGG."

In 76 evaluable patients from Arm 1, 44 (58%) completed 26 or more cycles of treatment (approximately 24 months). Amongst the key primary endpoints, the overall response rate was 53% (40/76), the median duration of response was 19.4 months (95% CI [13.8-27.2]), and the time to response was 5.4 months (range [1.6-17.5]). The pre-specified secondary study endpoint of Progression Free Survival was evaluated by Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma. The median was 16.6 (10.9–22.0) months.

Exploratory analyses were also undertaken to further assess the clinical impact of tovorafenib in the same study population. Among these analyses:

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The median time-to-next-treatment (defined as the time from the date of the first dose of tovorafenib to the start of the first subsequent anticancer treatment, or date of death) was 42.6 months (95% CI [36.7-NE]).

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39 patients entered a treatment-free observation period:

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77% (30/39) were treatment-free for a minimum of 12 months.

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Median treatment-free interval, measured from the end of tovorafenib primary treatment to the start of the next subsequent anticancer treatment or death, was not reached.

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Tumor rebound was minimal in the first 6 months off therapy, with 31% of patients experiencing a ≥25% increase in tumor size from the last scan prior to the last dose.

•
Eight patients received retreatment with tovorafenib:

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The median retreatment duration was 9 months (all patients remained on therapy at the time of data cutoff).

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The median maximum percentage change in tumor reduction was –38.3%.

"These three-year data showed that patients were able to maintain disease control during extended periods off therapy, with the option to reinitiate tovorafenib treatment if clinically indicated," said Dr. Cassie Kline, Director of Clinical Research in the Division of Neuro-Oncology at the Children’s Hospital of Philadelphia. "This approach has the potential to offer patients and their families meaningful time away from treatment."

In this updated three-year analysis, no new safety signals were identified. Grade 3 or higher adverse events most commonly reported (≥ 5% of patients) were decreased growth velocity, anemia, blood creatine phosphokinase increased, maculopapular rash, alanine aminotransferase increased.

Tovorafenib is approved by the U.S. Food and Drug Administration for the treatment of pediatric patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement.

About tovorafenib

Tovorafenib (known as OJEMDATM in the U.S.) is a Type II RAF kinase inhibitor mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. Tovorafenib is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based, in part, on response rate and duration of response according to multiple response assessment criteria: Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO LGG) criteria, and Response Assessment for Neuro-Oncology Low-Grade Glioma (RANO LGG) criteria. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib is under evaluation as a therapy for patients aged 6 months to 25 years with pLGG harboring BRAF fusion or rearrangement, or BRAF V600 mutation requiring front-line treatment (Phase III FIREFLY-2/LOGGIC).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

For more information, please visit www.ojemda.com.

About Pediatric Low-Grade Glioma

Pediatric low-grade glioma (pLGG) is the most common brain tumor with an estimated US incidence of 1,100 and Europe incidence of 700 children per year who are eligible for front-line systemic therapy.i, ii BRAF is the gene most commonly altered in pLGG, which include two primary types of BRAF alterations – a BRAF gene fusion and BRAF point mutation. These BRAF alterations account for >50% of pLGG cases worldwide and until now there were no approved treatments for people with pLGG driven by BRAF fusions.i, iii

Pediatric low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact their life over the long term, and may include muscle weakness, loss of vision, and difficulty speaking. This type of tumor has a high risk of progression, and many children with pLGG require long-term treatment. While most children with pLGG survive their cancer, children who do not achieve a complete resection following surgery may face years of increasingly aggressive treatment.

About FIREFLY-1

FIREFLY-1 is evaluating tovorafenib as once-weekly monotherapy in patients aged 6 months to 25 years with relapsed or progressive pLGG harboring a known activating BRAF alteration. The trial is being conducted in collaboration with the Pediatric Neuro-Oncology Consortium (PNOC). The pivotal and ongoing Phase 2 FIREFLY-1 study evaluated the safety and efficacy of tovorafenib in 137 relapsed or refractory BRAF-altered pLGG patients, who had received at least one line of prior therapy, across two study arms. Arm 1 (n=77) was used for the efficacy analyses and Arm 2 provided safety data for an additional 60 patients, initiated to enable access to tovorafenib once Arm 1 had fully recruited.

At the time of data cutoff on May 10, 2024, the major efficacy outcome measure was overall response rate (ORR) of 53% (40/76), determined by blinded independent central review based on RAPNO-LGG (Response Assessment in Pediatric Neuro-Oncology) criteria. Median duration of response (DOR) was 18 months (12.0, 22.8) and median time to response (TTR) was 5.4 months (range 1.6, 17.5). The most common adverse reactions (≥30%) were rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection. Grade 3 or higher adverse reactions (≥5%) were rash, viral infection, and hemorrhage. Additional information about FIREFLY-1 may be found at ClinicalTrials.gov, using Identifier NCT04775485.

About the Pediatric Neuro-Oncology Consortium

The Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with study sites within the United States, Canada, Europe and Australia dedicated to bringing new therapies to children and young adults with brain tumors.

(Press release, Day One, NOV 24, 2025, View Source [SID1234660893])