On June 3, 2026 SOTIO Biotech, a clinical-stage biopharmaceutical company owned by PPF Group, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SOT106 for the treatment of osteosarcoma, reinforcing its potential as a targeted therapy for this high unmet need population.

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SOT106 is a next-generation antibody-drug conjugate (ADC) targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumor associated stroma. The program is designed to deliver an early and actionable clinical signal, with expansion potential across multiple solid tumors. Preclinical data demonstrate strong anti-tumor activity in both soft tissue and osteosarcoma models and favorable tolerability supporting a high therapeutic index. SOTIO expects to initiate a first-in-human clinical trial of SOT106 in the second half of 2026.

"Orphan Drug Designation for SOT106 underscores both the urgent need for new treatment options in osteosarcoma and the strength of our ADC platform," said Radek Spisek, M.D., Ph.D., chief executive officer of SOTIO. "Osteosarcoma is a devastating disease that has seen little therapeutic innovation over the past four decades. Treatment continues to rely on intensive chemotherapy regimens associated with significant toxicities and limited long-term benefit. We are encouraged by this recognition from the FDA and look forward to advancing SOT106 into the clinic later this year."

Sarcomas are a diverse group of cancers arising in bones and soft tissues and comprising more than 70 distinct subtypes. Their rarity and biological heterogeneity have made therapeutic innovation challenging, including the development of targeted approaches such as ADCs. Patients today are primarily treated with a combination of surgery, radiation and/or chemotherapy, yet outcomes remain poor for patients with aggressive, recurrent or metastatic disease. This need is especially clear in osteosarcoma, the most common bone cancer in children and adolescents, where a significant number of patients require amputation of the affected limb.

ODD provides several incentives to support the development of therapies for rare diseases and areas of high unmet need, including the potential for seven years of market exclusivity upon approval, waivers of certain regulatory fees, and enhanced interaction with and guidance from the U.S. FDA throughout the development process.

(Press release, SOTIO, JUN 3, 2026, View Source [SID1234666432])

On June 3, 2026 Ingenix, the AI and biology company built around a single question – "What would it take for AI to truly understand biology?" – reported a €13m seed-extension funding round led by Sofinnova Partners, with participation from Inovo VC and OTB VC.

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The funding will scale development of Ingenix’s Biological Reasoning Engine and broaden its work with pharma and biotech partners through the Qualified Access Program, launching today.

The dominant approach in AI for drug development, which trains larger models on larger datasets assuming biology will yield to scale, faces a structural problem. Biology is profoundly complex, fundamentally non-linguistic, and runs across modalities and biological scales. No single model, however large, can capture that on its own. Moreover, no single company holds all the data, across every modality and biological scale, to train one. Ingenix has built a different architecture, Modality Fusion, which integrates best-in-class models across modalities and biological scales, then reasons across their representations directly. The Biological Reasoning Engine is what Modality Fusion enables.

"This funding lets us extend the Biological Reasoning Engine to the partners and questions where it can do the most useful work," said Piotr Surma, CEO and co-founder of Ingenix. "We built Ingenix on the conviction that biology needs an AI architecture designed for biology and not a general-purpose model retrofitted to it. The early results have been stronger than we forecast, and we’re excited to extend the Engine to a select number of partners through the Qualified Access Program."

"It is no longer enough to just build models," added Simon Turner, Partner at Sofinnova Partners. "Ingenix is building the reasoning layer, the part that actually connects the biology, the chemistry, and the clinical data into something a scientist can interrogate and act on. That’s the hard bit, and that’s where the value compounds. We’re thrilled to back a team that gets that."

The Engine in Action: ADC Payload Prioritization

In a recent engagement, Ingenix applied its Biological Reasoning Engine to a dual-payload ADC prioritization problem for an oncology biotech which had thousands of possible payload configurations but lacked the experimental capacity to test them.

The Engine produced 15 candidate combinations. Under blind expert review by the biotech’s translational science team, the predictions broke down as follows:

5 were publicly known hypotheses.
2 were supported in existing literature but not widely cited.
3 had been confirmed by the biotech through internal experiments but never published and never disclosed to Ingenix.
5 were novel hypotheses not previously considered by the biotech team. Of these, the biotech flagged 3 as actionable candidates.
The double-payload ADC space is too new for any AI system to have seen meaningful training data on it. The engine reached its predictions by reasoning from first principles about the underlying biology, rather than by pattern-matching against prior examples.

In short, insights that had taken the biotech several years of research and millions of euros to develop were accomplished by the Engine in a matter of minutes.

Applications to the Qualified Access Program are now open at ingenix.ai/qap.

(Press release, Ingenix, JUN 3, 2026, View Source [SID1234666417])

On June 3, 2026, Abeona Therapeutics Inc. (the "Company") reported to have participated in a pre-Investigational New Drug application ("Pre-IND") meeting with the U.S. Food and Drug Administration ("FDA") regarding ABO-701, a recently licensed radically novel engineered T-cell therapy, targeting Prostate-Specific Membrane Antigen to treat prostate cancer. While official minutes of the meeting have not yet been received, we believe the meeting was constructive and continues to target submission of an IND application for ABO-701 in 2027, consistent with our previously stated timeline.

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We can provide no assurance that the FDA will not require additional studies, data, or information before accepting an IND submission for ABO-701, or that any IND submission, if submitted, will be accepted by the FDA or result in authorization to commence clinical trials. Our development plans remain subject to ongoing evaluation and may be revised based on, among other things, feedback received from the FDA, results of preclinical studies, manufacturing considerations, and other factors.

(Press release, Abeona Therapeutics, JUN 3, 2026, View Source [SID1234666436])

On June 3, 2026 ADC Therapeutics SA (NYSE: ADCT) reported topline data from its Phase 3 LOTIS-5 confirmatory trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with rituximab in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL). ZYNLONTA plus rituximab achieved statistical significance on the trial’s primary endpoint of progression-free survival (PFS) and demonstrated no detrimental effect on the key secondary efficacy endpoint of overall survival (OS). In addition, a higher complete response (CR) rate and duration of CRs (DoCR) were observed with ZYNLONTA plus rituximab. Overall, treatment emergent adverse event (TEAE) rates were similar between arms. Similar rates of overall Grade ≥3 TEAEs greater than 5% were observed across both arms, with hematologic TEAEs higher in the control arm and infection, hepatotoxicity, and edema/effusion higher in the test arm. Serious adverse events (SAEs), TEAEs leading to study drug withdrawal, and Grade 5 events were higher in the test arm, with the majority of Grade 5 TEAEs in the test arm occurring in patients aged 75 years or older.

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"In the context of a positive study, based on the totality of the data, we plan to discuss the benefit-risk profile of this combination with the U.S. FDA as we prepare for the planned supplemental Biologics License Application (sBLA) filing," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We would like to extend our thanks to the patients, investigators, and clinical teams who contributed to this important trial."

The LOTIS-5 trial is a randomized, open‐label, two‐arm, multicenter study evaluating ZYNLONTA plus rituximab versus the standard immunochemotherapy rituximab gemcitabine‐oxaliplatin (R‐GemOx), for the treatment of r/r DLBCL after one or more lines of systemic therapy. The study met the primary endpoint of PFS (per independent review committee) with statistical significance (HR = 0.73; p-value = 0.008 two sided), with a median PFS of 6.1 months for ZYNLONTA plus rituximab vs 4.7 months for R-GemOx. Overall survival showed no detrimental effect with ZYNLONTA plus rituximab compared to the control arm (HR = 0.96, impacted by the earlier use and a higher rate of new anti-lymphoma treatment switching in the control arm). Overall response rate (ORR) was 58.1% vs. 45.2%, CR rate was 39.5% vs. 26.7%, median duration of response (DOR) was 9.2 months vs. 7.7 months, and median DoCR was 16.8 months vs. 12.3 months for ZYNLONTA plus rituximab compared to R-GemOx, respectively. Of patients achieving CR, 48.5% vs. 16.7% remained in CR at 24 months in favor of ZYNLONTA plus rituximab. Of note, results in North America were consistent with the overall study results.

Overall, TEAE rates were similar between arms (98.5% vs. 97.5%). Higher rates of SAEs were seen in the test arm (49.0% vs. 34.5%). Grade ≥3 TEAEs observed in > 5% of patients were hematologic (40.7% vs. 59.4%), followed by infection/infestations (24.5% vs. 15.7%), then hepatotoxicity (17.2% vs. 8.1%) and oedema/effusion (7.4% vs. 0.5%) when comparing ZYNLONTA plus rituximab to R-GemOx. A higher rate of Grade 5 TEAEs was observed in the ZYNLONTA plus rituximab arm (27 pts/13.2%) vs. R-GemOx (9 pts/4.6%). Of note, the majority of Grade 5 TEAEs in the test arm occurred in patients aged 75 years or older. Higher rates of TEAEs leading to any drug withdrawal occurred in the ZYNLONTA plus rituximab arm (25.5% vs. 9.1%). In this study, the TEAE reporting window was defined as 105 days after the last dose of study treatment or the start of new anticancer therapy, whichever is earlier. The rates of TEAEs in this study were impacted by the longer overall TEAE observation time in the test vs. control arm. This difference is primarily driven by a higher rate of and earlier switching to subsequent therapies in the control arm.

"LOTIS-5 was designed to address a clear unmet need in r/r DLBCL in patients who cannot access or who progress on a CAR-T or other complex therapies," said Mehdi Hamadani, MD, Professor of Medicine, Associate Director of Clinical Research, Section Chief of Hematologic Malignancies at Medical College of Wisconsin and principal investigator for the trial. "Based on these results, I believe this combination may provide an additional option in treating relapsed or refractory DLBCL."

"Based on these topline results from LOTIS-5, we look forward to discussing next steps for this combination of ZYNLONTA plus rituximab with the U.S. FDA," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "We intend to conduct a pre-sBLA meeting in August and are preparing for a planned sBLA submission in the fourth quarter of 2026."

In addition, the Company will continue to evaluate a broad range of value maximizing alternatives, including but not limited to near-term cost reduction initiatives.

For more information about LOTIS-5, please visit View Source (identifier: NCT04384484).

Conference Call Details
ADC Therapeutics management will host a conference call and live audio webcast to discuss the LOTIS-5 results today at 4:30 p.m. EDT. To access the conference call, please register here. Registrants will receive the dial-in number and unique PIN. It is recommended that you join 10 minutes before the event, though you may pre-register at any time. A live webcast of the call will be available under "Events & Presentations" in the Investors section of the ADC Therapeutics website at ir.adctherapeutics.com. The archived webcast will be available for 30 days following the call.

(Press release, ADC Therapeutics, JUN 3, 2026, View Source [SID1234666418])

On June 3, 2026 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported the publication in the New England Journal of Medicine of results from the ASCERTAIN-V Phase 1/2 clinical trial, which evaluated the first all-oral regimen of decitabine-cedazuridine plus venetoclax in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The study demonstrated favorable response rates and survival with expected myelosuppressive effects.1

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Previously presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting and the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, the results suggest this first all-oral regimen may provide outcomes consistent with existing hypomethylating agent–venetoclax–based approaches for patients with newly diagnosed AML who cannot receive intensive induction therapy.

Nearly 23,000 people in the U.S. are expected to be diagnosed with AML, a cancer of the blood and bone marrow, in 2026. 2 More than half of those patients will likely be deemed ineligible for intensive induction chemotherapy because of factors such as older age (75 or older) or poor health. 3 For these patients, the standard treatment often combines oral venetoclax with parenteral hypomethylating agents, requiring frequent injections or infusions and an average of 12.9 days per month engaging in healthcare visits. 4

Oral decitabine-cedazuridine is a hypomethylating agent that has demonstrated oral bioavailability and equivalent pharmacokinetic exposure to intravenous decitabine. 5

"We are committed to advancing therapies that improve the lives of patients, their families and their caregivers," said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. "An all-oral regimen for AML could help reduce the significant time and cost burden associated with treatment in hospitals or infusion centers."

The ASCERTAIN-V trial evaluated the first all-oral regimen of oral decitabine-cedazuridine plus venetoclax in 189 patients (Phase 1, n=30; Phase 2a, n=58; Phase 2b, n=101). After an initial ramp-up period, patients were treated in 28-day cycles, receiving decitabine-cedazuridine on days one through five and venetoclax daily. Key results include complete response rates, durable remissions and survival, and a safety profile consistent with hypomethylating agent-venetoclax regimens.

In the Phase 2b of ASCERTAIN-V:
The complete response rate was achieved in 47% of patients (95% CI: 36-57%). The composite of complete response or complete response with incomplete hematologic recovery was 63% (95% CI: 53-73%).
The median time to marrow blasts of less than 5% was 28 days (range 22 to 57), and the median time to a complete response or complete response with incomplete hematologic recovery was 35 days (range 27 to 58).
Among patients achieving complete response, 80% (95% CI: 64%, 90%) had an ongoing response at six months and 75% (95% CI: 57%, 87%) at 12 months.
Median overall survival was 15.5 months (95% CI 7.6-not estimable). The 30- and 60-day mortality rates were 3% and 10%, respectively. The median follow-up period was 11.2 months.
Serious adverse events were reported in 84% of patients, consistent with known hypomethylating agent–venetoclax regimens. Common related Grade ≥3 adverse events were anemia (30%), neutropenia (26%) and febrile neutropenia (25%).

No drug–drug interactions were observed between decitabine-cedazuridine and venetoclax.
In the Phase 2b of ASCERTAIN-V, decitabine-cedazuridine and venetoclax dosing was modified once leukemic blast clearance was achieved as measured by bone marrow morphology assessments. Serious adverse events and febrile neutropenia decreased as venetoclax dosing days decreased with each treatment cycle. This information potentially supports the need to explore a treatment strategy of an initial induction followed by dose-adjusting ongoing cycles to enhance tolerability and minimize cytopenia-related complications in patients who have reached remission.

"The positive results of the trial — response rates, safety, pharmacokinetics and tolerability data — suggest that an all-oral regimen could potentially become a meaningful alternative to existing treatment protocols that involve parenteral hypomethylating agents for patients with AML who can’t undergo intensive induction chemotherapy," said Gail J. Roboz, M.D., the paper’s lead author, a professor of medicine, director of the Clinical and Translational Leukemia Program and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a hematologist oncologist at NewYork-Presbyterian/Weill Cornell Medical Center in New York City. "An all-oral treatment regimen is a highly anticipated addition to current treatment options for patients with AML."

About Decitabine and Cedazuridine Fixed-Dose Combination
This product is an orally administered, fixed dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine,6 an inhibitor of cytidine deaminase.7 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over five days.

(Press release, Taiho, JUN 3, 2026, View Source [SID1234666419])