On May 8, 2025 iOnctura, a clinical-stage biopharmaceutical company combating neglected and hard-to-treat cancers, reported it has dosed the first patient in the randomized Phase I/II study investigating lead asset roginolisib in combination with dostarlimab with or without docetaxel, in patients with advanced non-small cell lung cancer (NSCLC) (Press release, iOnctura, MAY 8, 2025, View Source [SID1234652782]).

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NSCLC is the most common type of lung cancer. Lung cancer is the leading cause of cancer death globally, accounting for approximately 1 in 5 of all cancer deaths[1].

Anti-programmed death-ligand 1 (PD-L1) or anti-programmed cell death protein 1 (PD1) immunotherapy is a standard therapy for NSCLC patients with no actionable mutations in the first or second lines of therapy, with or without chemotherapy. However, treatment is often of limited duration as the cancer cells develop resistance to the treatment. Rebalancing the immune system via PI3Kδ inhibition is thought to re-invigorate anti-tumour immune cells and thus is a promising mechanism to overcome resistance to current immunotherapies[2].

Michele Maio, MD, PhD, Professor of Medical Oncology at the University of Siena and Director of the Center for Immuno-Oncology at the University Hospital of Siena (Italy), Primary Coordinating Investigator of the Phase I/II study said: ‘There is a significant lack of treatment options for NSCLC patients who have progressed on immunotherapy and chemotherapy. We will be investigating whether the combination of roginolisib with dostarlimab and +/- chemotherapy can be given safely and may provide a novel treatment option in patients who no longer respond to their current treatment.’

Emerging clinical and translational biomarker data supports the hypothesis that combining roginolisib with an anti-PD-L1/PD1 agent, with or without docetaxel, may prevent or reverse drug resistance in NSCLC and may show synergistic anti-tumor immune activity without significant addition of toxicity. Firstly, the combination of anti-PD1 and roginolisib had an anti-tumor effect in vitro[3]. Secondly, ex vivo evaluation of NSCLC samples from patients responding and failing to immunotherapy showed that roginolisib rebalanced the composition of immune cells by inhibiting regulatory T-cells and enhancing CD8+ T cell killing cells3. Finally, roginolisib augmented the cytotoxicity of chemotherapy and immunotherapy3.

Dostarlimab is a PD-1-blocking antibody approved in the US in combination with chemotherapy for certain patients with endometrial cancer, as well as a single agent for certain patients with dMMR tumours that have progressed on or following prior treatment. It has also demonstrated clinical activity in combination with chemotherapy in NSCLC[4]. Under a supply agreement with iOnctura, GSK will supply dostarlimab for use in the trial. iOnctura will retain worldwide rights to roginolisib.

The Phase I/II open-label, randomized, parallel-arm PULMO-01 study (NCT06879717) will assess roginolisib in combination with dostarlimab, with or without docetaxel. The study will enrol approximately 45 patients who have progressed on standard-of-care immune checkpoint therapy. It will investigate the safety of the combination and the proportion of patients with a reduction in peripheral blood regulatory T cells in each arm.

Roginolisib is an allosteric modulator of PI3Kδ, widely recognized as a ‘master switch’ of cancer. Inhibition of PI3Kδ unleashes a multi-pronged anti-tumor and immune response to combat the tumor[5]. Roginolisib has demonstrated an unprecedented clinical profile in solid and liquid cancers[6], and is currently being investigated in a randomized Phase II clinical trial in uveal melanoma. A separate Phase I/II study in myelofibrosis is being initiated.

On May 8, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the third patient in Cohort 4 of its Phase 1a clinical trial has received their initial dose of TTX-MC138 (Press release, TransCode Therapeutics, MAY 8, 2025, View Source [SID1234652781]). All cohorts have enrolled at least three patients who have been dosed with TTX-MC138 at least once. The Safety Review Committee monitoring the clinical trial unanimously approved opening the fourth cohort based on its review of available safety and pharmacokinetic (PK) data. Additionally, the Safety Review Committee approved expanded enrollment in Cohort 3 to obtain additional safety data. To date, 15 patients have received at least one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8 mg/kg to 4.8 mg/kg. Three patients have been treated in the expanded enrollment.

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Ten patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each treatment cycle every 28 days, and may remain on study absent any significant safety observations or disease progression. Two patients who have remained on study the longest have received so far seven doses of TTX-MC138 over approximately seven months and have demonstrated stable disease. The 10 patients currently on study have shown no disease progression. Further, no significant safety or dose limiting toxicities have been reported in any of the trial’s 15 patients. Analysis of PK activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, preliminary PK and pharmacodynamic (PD) data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates miR-10b target engagement at 24 hours post-infusion.

The observed tolerability profile and the available PK/PD results thus far support advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial.

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

About the Trial

TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types and at a certain dose level selected based on preliminary results from the dose-escalation phase.

Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

On May 8, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported financial results for the first quarter ended March 31, 2025, and provided recent corporate progress (Press release, Whitehawk Therapeutics, MAY 8, 2025, View Source [SID1234652780]).

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"By uniting novel approaches to tumor targeting with next wave technologies, our goal at Whitehawk Therapeutics is to efficiently deliver improved ADC therapies for patients with difficult-to-treat cancers. In the first quarter of 2025, we made meaningful progress in advancing our portfolio toward the clinic and remain on track to bring all three assets to IND by mid-2026, with the first IND filed in Q4 this year," said Dave Lennon, President and CEO of Whitehawk Therapeutics. "Our assets target PTK7, MUC16 and SEZ6 – which we believe have the potential for broad clinical impact comparable to well-established targets like HER2 and TROP2, but with less competition and greater opportunity for differentiation. Coupled with our TOPO1-based leading next wave platform, we are confident in the strength of our strategy and remain committed to advancing with urgency and purpose."

Recent Operational Highlights:

Relaunched as Whitehawk Therapeutics marking evolution into ADC company. Launched with a three-asset portfolio consisting of clinically validated tumor targets that are upregulated in high-potential cancer indications, including lung and ovarian. These assets are engineered to produce minimal off-target toxicity, with a higher therapeutic index and greater stability than first-generation predecessors.
Completed strategic transactions. Closed $100 million PIPE financing and the divestiture of Aadi Subsidiary, Inc. ("Aadi Sub") to Kaken Pharmaceuticals ("Kaken") for $102.4 million, including specified purchase price adjustments. Kaken assumed ownership of Aadi Sub on March 25, 2025, including the Aadi name, trademark and the FYARRO business.
First Quarter 2025 Financial Results:

Cash, cash equivalents and short-term investments as of March 31, 2025, were $231.1 million as compared to $47.2 million as of December 31, 2024.
After completion of transaction-related payments, including the payment of the upfront fees under the Wuxi ADC agreement, we expect to have cash and cash equivalents of approximately $185 million, which we anticipate will fund operations into 2028 based on current plans.
Total revenue for the quarter ended March 31, 2025, was $7.1 million, resulting from sales of FYARRO through March 25, 2025, the closing date of the divestiture to Kaken.
Net income for the three months ended March 31, 2025, was $73.0 million, including a gain of $87.4 million on the sale of Aadi Sub, as compared to a net loss of $18.3 million for the three months ended March 31, 2024. Excluding the gain on the sale of Aadi Sub, net loss for the first quarter of 2025 was $14.4 million.

On May 8, 2025 Aptevo Therapeutics Inc. (Nasdaq:APVO), a clinical-stage biotechnology company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported a program update for APVO711, a dual mechanism bispecific utilizing the PD-L1 arm to block the PD-1/PD-L1 pathway and the CD40 arm to enhance T cell priming through activation of the stimulatory receptor CD40 on antigen presenting cells (Press release, Aptevo Therapeutics, MAY 8, 2025, View Source [SID1234652779]). This update highlights the depth and scientific versatility of Aptevo’s existing pipeline and reinforces the Company’s ability to advance differentiated candidates for solid tumors with significant unmet need. Currently in preclinical development, APVO711 reflects the potential of Aptevo’s platform-based strategy to generate bispecific therapeutics with novel mechanisms and broad clinical relevance.

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"With APVO711, we’re advancing an innovative dual-mechanism approach to immunotherapy-one that not only boosts the immune system through PD-L1 blockade but also facilitates T-cell priming via CD40 activation. This is important in the development of new therapeutics for cancers that have proven resistant to standard checkpoint therapies. We believe APVO711 has the potential to unlock deeper, more sustained responses, and ultimately expand the reach of immunotherapy to patients who need it most," said Marvin White, President and CEO of Aptevo.

About APVO711

Novel Mechanism: APVO711 is a bispecific antibody targeting PD-L1 × CD40-combining checkpoint inhibition with immune activation in a single molecule.

Designed for Potency: Unlike traditional checkpoint inhibitors, APVO711 simultaneously blocks immune suppression and primes antigen presenting cells to trigger robust T-cell responses.

Targeting the Unmet Need: Developed to address a wide variety of tumor types and augment the anti-tumor responses achieved with PD-1/PD-L1 blockade alone.

Pipeline Synergy: Represents a key part of Aptevo’s novel immuno-oncology platform, with strong potential for combination strategies across solid tumors.

Momentum Building: Preclinical studies demonstrate dual anti-cancer functionality.

"APVO711 represents a novel immunotherapy approach – by targeting both PD-L1 and CD40, this bispecific candidate is designed to block tumor-driven immune suppression while simultaneously priming T cells for a stronger, more durable anti-tumor response. In a landscape where many patients fail to respond to checkpoint inhibitors alone, APVO711 offers a rational strategy to expand the benefits of immunotherapy to a broader range of cancers and patients," said Michelle H. Nelson, Ph.D., Director of Immunobiology at Aptevo Therapeutics.

On May 7, 2025 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three months ended March 31, 2025, and provided operating and partner program updates (Press release, OmniAb, MAY 8, 2025, View Source [SID1234652778]).

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"We have started the year with robust deal flow, including both platform and asset-based deals. Our business remains strong as our diversified pipeline of partner programs is progressing with recent and expected new clinical entrants and data readouts," said Matt Foehr, Chief Executive Officer of OmniAb. "Today we announced the xPloration Partner Access Program for OmniAb partners, enhancing the scalability of our technology platforms and creating new business opportunities that we believe will be accretive to both earnings and cash flow in short- and long-term. This initiative furthers our mission to push the frontiers of discovery technologies along with our focus on creating value for our partners and our stakeholders. As we look ahead, our 2025 outlook remains on track with our commitment to running an efficient and leverageable business."

First Quarter 2025 Financial Results

Revenue for the first quarter of 2025 was $4.2 million, compared with $3.8 million for the same period in 2024, with the increase primarily due to the recognition of a $1.0 million Phase 1 milestone payment and higher license fees, partially offset by lower service revenue.

Research and development expense was $12.6 million for the first quarter of 2025, compared with $14.6 million for the same period in 2024, with the decrease primarily due to lower share-based compensation expense and lower external expenses associated with our small-molecule ion channel programs and technology development. General and administrative expense was $7.9 million for the first quarter of 2025, compared with $8.3 million for the same period in 2024, with the decrease primarily due to lower legal fees and share-based compensation expense.

Net loss for the first quarter of 2025 was $18.2 million, or $0.17 per share, compared with a net loss of $19.0 million, or $0.19 per share, for the same period in 2024.

As of March 31, 2025, OmniAb had cash, cash equivalents and short-term investments of $43.6 million.

2025 Financial Guidance

OmniAb affirms guidance for 2025 revenue to be in the range of $20 million to $25 million, and revises operating expense guidance to be in the range of $85 million to $90 million from the previous range of $90 million to $95 million. In addition, OmniAb continues to expect 2025 cash use to be lower than cash use in 2024. Cash use in 2024 was $38.9 million, excluding the 2024 ATM issuance. The 2025 full year effective tax rate is expected to be approximately 0%.

First Quarter 2025 and Recent Business Highlights

During the first quarter of 2025, OmniAb entered into three new platform license agreements including the Wyss Institute at Harvard University, Takis Biotech S.r.l. and Orion Corporation.

OmniAb entered into a research collaboration and license agreement with Orion Corporation to discover and generate an antibody-based compound for a specific ion-channel target. Under the terms of the agreement, OmniAb will receive an upfront payment of $250,000 and is eligible to receive service payments. OmniAb is also eligible to receive development, regulatory and commercialization milestone payments totaling to over $55 million. OmniAb will receive low- to mid-single digit tiered royalties on net sales, should the program reach commercialization.

As of March 31, 2025, the Company had 95 active partners and 378 active programs, including 33 OmniAb-derived programs in clinical development or being commercialized.

Post-quarter close, OmniAb entered into an asset-based sale with Angelini Pharma for a small molecule Kv7.2 program. OmniAb will receive an upfront payment of $3 million, and potential milestones of over $170 million and royalties.

In addition, OmniAb launched the offering of xPloration to existing partners through a Partner Access Program. xPloration is a high-throughput single B-cell screening instrument that leverages machine learning and artificial intelligence to address challenges in primary B-cell screening with traditional methods, such as limited antibody diversity and lengthy processes. We believe it offers a competitive edge over current market offerings for B-cell screening with unmatched screening throughput, superior hit recovery, exceptional ease-of-use and reliability. OmniAb will showcase xPloration at the 21st Annual PEGS Boston Summit, taking place May 12-16 at the Omni Boston Hotel at the Seaport.

First quarter 2025 and recent partner and business highlights include the following:

IMVT-1402

Immunovant announced that potentially registrational trials for IMVT-1402 are currently enrolling patients in four indications: myasthenia gravis (MG), chronic inflammatory demyelinating polyneuropathy (CIDP), Graves’ disease (GD) and difficult-to-treat rheumatoid arthritis.
A fifth potentially registrational trial for Sjogren’s disease is planned to begin in the summer of 2025. Additionally, a proof-of-concept study has been initiated in a sixth indication, cutaneous lupus erythematosus.
Batoclimab

Immunovant announced positive study results for batoclimab in MG and CIDP. The pivotal study in MG met its primary endpoint, showing a change from baseline in the Myasthenia Gravis Activities of Daily Living score in the acetylcholine receptor antibody positive population at 12 weeks. The 680mg dose arm showed a 5.6-point improvement with a 74% mean immunoglobulin G (IgG) reduction, while the 340mg dose arm showed a 4.7-point improvement with a 64% mean IgG reduction.
Initial CIDP results from Period 1, following standard-of-care washout, demonstrated a mean improvement in the adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) disability score of 1.8 across study arms. An 84% responder rate (with response defined as an aINCAT improvement ≥1) was observed among all patients whose IgG was reduced by ≥70%.
In both batoclimab studies, deeper IgG reductions correlated with better clinical outcomes across a range of assessments and timepoints.
Immunovant expects to announce additional data for batoclimab in GD, including six-month remission data, this summer. Additionally, top-line results for batoclimab from potentially registrational Phase 3 trials in thyroid eye disease are expected in the second half of 2025.
Sugemalimab

CStone Pharmaceuticals announced the submission of a Type II variation application to the European Medicines Agency (EMA) for sugemalimab, seeking approval for the treatment of unresectable stage III non-small cell lung cancer (NSCLC) in patients who have not progressed following platinum-based chemoradiotherapy. This is CStone’s second regulatory submission for sugemalimab to the EMA, following its 2024 approval for metastatic NSCLC.
TEV-53408

Teva Pharmaceuticals initiated a Phase 2 trial of TEV-53408 in adults with celiac disease. The primary efficacy objective is to assess the ability of TEV-53408 to attenuate gluten-induced enteropathy. Additional objectives include the safety assessment of TEV-53408.
RNDO- 564

Rondo Therapeutics published preclinical data for RNDO-564, a CD28 x Nectin-4 bispecific antibody for bladder cancer, in theJournal of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper).In vitrostudies demonstrated that RNDO-564 enhanced T-cell activation and cytotoxicity against Nectin-4 positive tumor cells. The antibody demonstrated significant tumor regression in tumor-bearing mouse models, both alone and with an immune checkpoint inhibitor. Favorable pharmacokinetic and tolerability profiles were observed in non-human primates.
OmniAb recently appointed Philip J. Gotwals, Ph.D., and Steve Crouse to its Board of Directors. Dr. Gotwals, with 30 years of biopharmaceutical experience in R&D, business development, product development, and therapeutic area strategy, along with Mr. Crouse, who brings over 20 years of expertise in life sciences sales and marketing, product development, business development, and general management, will help advance the Company’s strategic initiatives.

OmniAb reported that partners presented data on nine OmniAb derived molecules at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, held April 25-30. These presentations showcased clinical trial designs, as well as data across various preclinical and clinical studies.

The Company also expects that multiple partners will be presenting data from programs developed with OmniAb technology at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 30 – June 3, 2025.

Conference Call and Webcast

OmniAb management will host a conference call with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 549 8228 using the conference ID 96760. Slides, as well as the live and replay webcast of the call, are available at View Source