On March 13, 2025 SOTIO Biotech, a clinical-stage biopharmaceutical company owned by PPF Group, reported it will be exercising its option, under its license and option agreement with Synaffix B.V. ("Synaffix"), a Lonza company (SWX: LONN), to obtain a license to Synaffix’s technology to develop two bispecific antibody-drug conjugate (ADC) programs (Press release, SOTIO, MAR 13, 2025, View Source [SID1234651142]).

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The exercised option will enable SOTIO to advance two bispecific candidates, designed by its in-house research team, that have the potential to become first-in-class and/or best-in-class treatments for solid tumors. SOT112 is a bispecific ADC with potential applicability in several major cancer indications, while SOT113 demonstrates promise for precision targeting of prostate cancer. Both programs leverage the selection of highly attractive target pairs and are now in the lead nomination stage.

The bispecific antibody candidates were identified by SOTIO’s research team and will now be developed under SOTIO’s license and option agreement with Synaffix, which enables SOTIO to combine its proprietary antibodies with Synaffix’s GlycoConnect, HydraSpace, and potent linker-payloads of the toxSYN platform to research, develop, manufacture, and commercialize ADC products.

"The promising preclinical data on SOT112 and SOT113 have reinforced our confidence in their potential, and we are excited to advance these candidates within our first-in-class and best-in-class ADC portfolio," said Martin Steegmaier, Ph.D., chief scientific officer of SOTIO. "Bispecific ADCs represent a groundbreaking frontier in oncology, offering enhanced tumor-targeting precision and the potential to overcome the challenges of heterogeneity in solid tumor target expression. Both advancements align with our mission to develop safer and more effective treatments for solid tumors."

Peter van de Sande, head of Synaffix, commented: "We are pleased that SOTIO, recognizing the versatility and robustness of our clinically validated platform, has chosen to expand its use of our ADC technology to two additional targets. ADCs are a key part of our offering, and our technology is primed to support this next stage of innovation in targeted cancer therapeutics. We look forward to seeing these promising candidates advance toward the clinic."

Under the terms of the agreement, Synaffix is eligible to receive an upfront payment with the potential for milestone payments, plus single-digit royalties on net sales. SOTIO will be responsible for research, development, manufacturing, and commercialization of the ADC products, and Synaffix will closely support SOTIO’s research activities and be responsible for the manufacturing of components that are specifically related to its proprietary GlycoConnect, HydraSpace, and linker-payloads of the toxSYN technologies.

On March 13, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported U.S. Urology Partners, one of the nation’s largest independent providers of urology and related specialty services, is one of the first providers to participate in ImmunityBio’s Expanded Access Program (EAP) for recombinant Bacillus Calmette-Guérin (rBCG) to address the current shortage of TICE BCG in the U.S (Press release, ImmunityBio, MAR 13, 2025, View Source [SID1234651127]).

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"The shortage of BCG has real consequences, especially in many rural communities across the country where supply shortages have impacted the treatment of patients with bladder cancer," said Christopher M. Pieczonka, M.D., Chief Executive Officer and Director of Clinical Research, Associated Medical Professionals, an affiliate of U.S. Urology Partners. "I’m pleased U.S. Urology is at the forefront of offering rBCG and would like to thank ImmunityBio for their initiative to seek and secure authorization from the FDA for expanded access to this vital BCG alternative."

Supply shortages of TICE BCG in the U.S. have become a significant impediment to the treatment of bladder cancer patients. In a recent Sermo survey of 100 U.S. urologists, 57 percent indicated they were unable to treat patients in the last 12 months due to a lack of access to TICE BCG. Providers who are unable to source TICE BCG are able to access rBCG through the ImmunityBio EAP.

"It is gratifying to see the rapid uptake and interest in the rBCG Expanded Access Program, which was authorized by the FDA just recently," added Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer of ImmunityBio. "Participation by a major national provider like U.S. Urology Partners is a testament to the critical importance of rBCG and the need for an alternative supply of this essential medicine."

"This is an incredibly exciting opportunity to expand the BCG supply to allow more patients to receive the full course of their cancer treatment, saving bladders and extending lives," said Dr. Eugene B. Cone, Co-Director of Clinical Research, Urology of Indiana, an affiliate of U.S. Urology.

The Serum Institute of India, ImmunityBio’s collaboration partner, is the world’s largest manufacturer of vaccines by volume. In bladder cancer clinical trials in Europe, the recombinant BCG vaccine has demonstrated potent immunogenicity with CD8+ and CD4+ T cell stimulation and improved safety compared to earlier BCG strains and formulations. Clinical research organization, Anova Enterprises, Inc. (Anova) is helping ImmunityBio manage the rBCG EAP.

"Constrained BCG supply has improved stewardship of a limited resource but has come at a high cost to our patients," said Dr. Chad Reichard, Co-Director of Clinical Research, Urology of Indiana. "rBCG represents a long overdue potential alternative but does not abrogate responsibility to ongoing diligence and discipline in BCG delivery."

About Recombinant BCG (rBCG)

BCG is a benign bacterium originally developed as a live vaccine against tuberculosis (TB). It is based on the well-known Mycobacterium bovis (M. bovis) Bacillus Calmette-Guérin (BCG) strain. It has been in use since 1921 and administered to more than 4 billion individuals worldwide. BCG given via intravesical instillation (delivery to the bladder via a catheter) has been the standard of care for patients with non-muscle invasive bladder cancer (NMIBC) since 1977. BCG induces an immune response in the bladder in proximity to the cancer cells, leading to clearance of the cancer in many patients.

Two gene modifications have been implemented in rBCG to improve its immunogenicity and safety in comparison to earlier strains and formulations of BCG. Recombinant rBCG has completed Phase 1/2 human clinical studies in Europe as an immunotherapy in patients with NMIBC. The findings from those studies demonstrate rBCG is well-tolerated when administered intravesically with a safety profile similar to placebo, and reduced rates of adverse events observed in earlier strains and formulations of BCG.

Supportive clinical data of rBCG as a TB vaccine are available from four clinical trials. Two studies in healthy adult volunteers and one Phase IIa study in healthy newborn infants were performed with rBCG. Additionally, a Phase II clinical trial was conducted with rBCG in HIV-unexposed and HIV-exposed, BCG-naïve newborn infants for clinical bridging. Clinical trials have also been conducted to assess the effect of rBCG vaccination on TB recurrence and on the susceptibility or severity of respiratory diseases during the severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2) pandemic.

BCG is one of the most widely used vaccines worldwide. However, because BCG is a biologic drug that uses benign bacteria, it is more complicated to manufacture than many other types of drugs. Serum Institute of India is the largest manufacturer of BCG vaccines in the world, while Merck & Co., based in New Jersey, currently is the only manufacturer of BCG (TICE BCG) in the U.S.

ImmunityBio has been awarded multiple patents covering the composition and methods of use for the combination of BCG plus ANKTIVA in bladder cancer (US 11,173,191 Β2; US 11,679,144 Β2; US 11,890,323 B2).

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

On March 13, 2025 OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage biotechnology company advancing immunotherapies and targeted drug conjugates for cancer treatment, reported that it has been awarded a presentation slot at the MIB Agents Factor Osteosarcoma Conference to be held June 26-28, 2025 in Salt Lake City, Utah (Press release, OS Therapies, MAR 13, 2025, View Source [SID1234651143]). Key data will be presented from the Company’s Phase 2b clinical trial of OST-HER2 in the prevention of recurrent, fully resected, lung metastatic osteosarcoma. This data will be compared with a newly created regulatorily compliant synthetic control group comprised of case matched controls that will be used for the Company’s US Food & Drug Administration (FDA) OST-HER2 Biologics License Application (BLA) submission. Equivalent submissions with international regulatory authorities will be made in order to gain commercial marketing authorization. MIB Agents FACTOR is the most important annual osteosarcoma conference bringing together the leading osteosarcoma researchers, clinicians, patient families, osteosarcoma survivors, patients, and bereaved parents to "Make It Better" (MIB) for those battling this deadly cancer.

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The Company reiterates its intention to submit a BLA with FDA for OST-HER2 in osteosarcoma in the second quarter of 2025, positioning it to receive approval in the fourth quarter of 2025. OST-HER2 has received Rare & Pediatric Disease Designation (RPDD) from the FDA and Fast Track and Orphan Drug Designations from the FDA and European Medicines Agency (EMA). If OST-HER2’s BLA submission is approved by the FDA before September 30, 2026, the Company will be granted a Priority Review Voucher (PRV) that it will be able to sell to a third party. The most recent PRV transaction occurred last month when Zevra sold its PRV to an undisclosed third party for $150 million.

On March 13, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported financial results and business highlights for the fourth quarter and full-year ended December 31, 2024 (Press release, In8bio, MAR 13, 2025, View Source [SID1234651128]).

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William Ho, Chief Executive Officer and co-founder of IN8bio, commented, "In 2024, IN8bio made significant progress advancing its pipeline of gamma-delta T cell therapies and positioning the Company for long-term success. The INB-100 program continues to demonstrate 100% long term durable response rates as of January 17, 2025, reinforcing its potential to help treat and maintain remissions in high-risk AML patients." Mr. Ho continued, "We have also disclosed our novel INB-600 platform, which we believe has the potential to reshape the T cell engager landscape by potentially improving both the durability of response and safety compared to existing CD3 targeting TCE therapies. Throughout 2025, we remain focused on driving innovation, advancing our clinical programs and delivering value to patients and our stakeholders."

Key Highlights

Announced INB-600 Platform & INB-619, a Novel Preclinical Gamma-Delta TCE

Introduced INB-619, a proprietary next generation gamma-delta TCE targeting CD19 for potential use in oncology and autoimmune diseases (March 2025)
Demonstrated potent B-cell depletion and sustained gamma-delta T cell expansion in preclinical studies, offering commercial and scientific advantages by potentially improving durability and safety over existing CD3 targeting TCE therapies
Demonstrated Additional Clinical Progress with INB-100 (Allogeneic Gamma-Delta T Cell Therapy for AML & Leukemias) for High-Risk AML

Presented new Phase 1 data showing durable remissions with 100% of treated AML patients remaining relapse-free (February 2025)
Observed favorable safety profile with no cytokine release syndrome (CRS) or neurotoxicity (ICANs) reported to date
Presented expanded clinical data at 2025 Tandem Meetings reinforcing potential of INB-100 to significantly reduce post-transplant relapse in high-risk AML patients relative to historical real-world data
Presented Continued Durable Remissions in Phase 1 Trial of INB-200 (Autologous, Drug-Resistant Gamma-Delta T Cell Therapy for Glioblastoma)

Presented updated data in a plenary oral presentation at the 2024 Society for Neuro-Oncology (SNO) Annual Meeting, showing the majority of patients exceeded their expected progression free survival based on age and tumor status
Highlighted potential long-term benefits of INB-200. As of October 18, 2024, five patients remained alive, three patients had returned to work, and one IDH-mutant, grade 4 glioma patient remained progression free for an impressive 40.5 months post treatment
Strategically Optimized Pipeline and Launched Operational Efficiency Initiatives

Paused enrollment in the Phase 2 INB-400 glioblastoma program – despite compelling data – to focus resources while exploring partnership and alternative funding opportunities
Implemented cost-saving measures to extend cash runway and prioritize pipeline development
Raised approximately $16.6 million in gross proceeds through various equity offerings, including through our at-the-market ("ATM") offering program and private placements, during 2024 through February 2025, extending the Company’s cash runway into March 2026
Upcoming Anticipated Pipeline Milestones and Important Events

INB-100: For the potential treatment of high-risk leukemias including AML

Accelerating patient enrollment in expansion cohort of ongoing Phase 1 clinical trial including the inclusion of an additional investigational center
Aim to complete enrollment into the expansion cohort in 2025. Guidance from the U.S. Food and Drug Administration (FDA) confirmed relapse-free survival (RFS) in AML patients as an acceptable primary endpoint for a potential pivotal randomized control trial
Expect to present updated clinical data in the second half of 2025
Advancing preparations for potential registrational Phase 2 trial with possible IND submission anticipated in 2026 based on receipt of additional funding
INB-200 and INB-400 (Autologous Gamma-Delta T Cell Therapy for Glioblastoma & Solid Tumors):

Continue to evaluate early clinical data for signs of efficacy and durability
Report additional findings at upcoming medical meetings in 2025
Seek additional funding sources to advance allogeneic product for glioblastoma and other solid tumors
INB-600 and INB-619 (Gamma-Delta T Cell Engager Targeting CD19 – Oncology & Autoimmune Diseases):

Present preclinical data evaluating potency, expansion potential, and safety profile at medical meetings in the second quarter of 2025
Plan to advance towards IND-enabling studies with additional funding to support future clinical development
Exploring partnership and collaboration options to accelerate development of the platform
Fourth Quarter and Full Year 2024 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $3.8 million for the three months ended December 31, 2024, compared with $4.5 million for the comparable prior year period. R&D expenses were $17.2 million for the year ended December 31, 2024, compared with $16.8 million in the prior year. The change was primarily due to increased clinical trial-related activities for the INB-100 and INB-400 programs and was partially offset by a decrease in personnel-related costs and facility-related and other expenses, in each case as a result of the Company’s pipeline prioritization announced in September 2024.
General and administrative (G&A) expenses: G&A expenses were $2.6 million for the three months ended December 31, 2024, compared with $3.1 million for the comparable prior year period. G&A expenses were $12.6 million for the year ended December 31, 2024, compared with $13.5 million in the prior year. The change was primarily due to cost savings related to director and officer insurance premiums, professional services, and personnel-related costs, partially offset by an increase in legal and consulting expenses.
Severance and related charges: Severance and related charges were $1.1 million for the year ended December 31, 2024, compared with zero in the prior year. These were one-time costs related to the September 2024 workforce reduction, including non-cash stock-based compensation expense and severance payments.
Net loss: The company reported a net loss of $6.4 million, or $0.08 per basic and diluted common share, for the three months ended December 31, 2024, compared with a net loss of $7.6 million, or $0.22 per basic and diluted common share, for the comparable prior year period. For the year ended December 31, 2024, net loss was $30.7 million, or $0.57 per basic and diluted common share, compared with a net loss of $30.0 million, or $1.00 per basic and diluted common share, for the prior year.
Cash position: As of December 31, 2024, the Company had cash of $11.1 million as of December 31, 2024, compared with $21.3 million as of December 31, 2023. Subsequently, in February 2025, the Company sold common stock under its ATM offering program and had Series C warrants exercised for aggregate net proceeds of $4.1 million.

On March 13, 2025 Exicure, Inc. (Nasdaq: XCUR) reported that the Australian Patent Office has issued Patent No. 2018388302, titled "GPCR Heteromer Inhibitors and Uses Thereof (Press release, Exicure, MAR 13, 2025, View Source [SID1234651144])." This patent covers the company’s innovative combination approach to cancer treatment, specifically targeting CXCR4 and GPCRx.

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The newly granted patent supports Exicure’s ongoing Phase 2 clinical trial (NCT05561751), which evaluates the combination of GPC-100 and propranolol in multiple myeloma patients. This method aims to improve hematopoietic stem cell mobilization by co-targeting CXCR4 and ADRB2, thereby enhancing the efficacy of CXCR4 inhibitors like GPC-100.

"This issuance reinforces the exclusivity of our lead clinical program," said Andy Yoo, CEO of Exicure, Inc. "By co-targeting CXCR4 and ADRB2, we can significantly enhance the potency of CXCR4 inhibitors like GPC-100. This opens new possibilities for these inhibitors to become more effective therapies, and our well-devised patent portfolio ensures strong intellectual property protection for this proprietary approach."

This patent family has already been granted in the United States, Japan, and Taiwan, with applications pending in other key jurisdictions.