On May 15, 2025 RyboDyn, Inc., a biotechnology company pioneering first-in-class immunotherapies targeting the dark proteome, reported the expansion of its strategic collaboration with Moffitt Cancer Center, a global leader in cancer care and research (Press release, RyboDyn, MAY 15, 2025, View Source [SID1234653191]). The partnership aims to accelerate the development of immunotherapies targeting novel, cancer-specific proteins identified through RyboDyn’s proprietary sequencing and AI-driven target discovery platform, RyboCypher.

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Earlier this year, RyboDyn reported the identification of a cryptic class of proteins derived from overlooked RNA species—called Dark Targets. These novel antigens, invisible to standard detection methods, represent a new and largely untapped frontier for oncology drug development. Through its expanded collaboration with Moffitt, RyboDyn will validate the presence and therapeutic potential of these Dark Targets across a wide array of tumor types, leveraging Moffitt’s uniquely annotated tumor repository—one of the largest and most comprehensive in the world. "Moffitt’s tumor bank is a critical resource in validating novel targets across a diverse set of cancer patients," said Dr. Alex Jaeger, PhD, assistant member of the Molecular Oncology Department at Moffitt Cancer Center. "The ability to correlate molecular signals with deep clinical metadata significantly accelerates both discovery and translational development."

Initial studies from the collaboration have confirmed that select Dark Targets are consistently presented on tumors—but not in healthy tissues—de-risking this novel class of antigens and providing a path toward scalable, tumor-specific therapies. "The fact that our lead targets are consistently presented across patient samples—yet absent in healthy tissues, gives us high confidence in their relevance," said Corey Dambacher, PhD, President, CTO, and Co-founder of RyboDyn.

These data support RyboDyn’s core hypothesis: while not all Dark Targets are cancer-specific, a distinct subset is uniquely and consistently presented on tumor cells—making them highly compelling candidates for therapeutic development. "We’re not building a personalized medicine platform—we’re designing therapies that combine precision with broad reach," said Imad Ajjawi, PhD, MBA, CEO and Co-founder of RyboDyn. "By focusing on conserved targets with tumor-specific presentation, we’re unlocking new opportunities for scalable, off-the-shelf therapies in oncology."

"This collaboration reflects our shared commitment to advancing the next generation of cancer immunotherapies," said Dr. Jaeger. "By combining Moffitt’s clinical and translational expertise with RyboDyn’s novel discovery platform, we’re accelerating progress toward more precise and effective treatments."

On May 15, 2025 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results and business highlights for the first quarter of 2025 and shared recent operational progress (Press release, Abeona Therapeutics, MAY 15, 2025, View Source [SID1234653156]).

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"ZEVASKYN’s approval just a few weeks ago is a landmark achievement for recessive dystrophic epidermolysis bullosa patients and signifies Abeona’s transition to a commercial-stage cell and gene therapy company," said Vish Seshadri, Chief Executive Officer of Abeona. "We are rapidly advancing the launch of ZEVASKYN and building positive momentum. Patients can now start their treatment journey with Lurie Children’s activated as our initial treatment center ready to identify patients and our scheduling system is operational. In addition, we are collecting patient registrations through our patient support program, Abeona Assist, and have entered into agreements with commercial payer groups ensuring broad access to ZEVASKYN."

Recent Developments

ZEVASKYN FDA approval, commercial launch progress and new data

● FDA approval of first-in-class RDEB therapy: On April 28, 2025, the U.S. FDA approved ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB. There is no cure for RDEB and ZEVASKYN is the only FDA-approved product to treat RDEB wounds with a single application.
● Activation of first ZEVASKYN Qualified Treatment Center (QTC): Following approval, ZEVASKYN is now commercially available in the U.S. after the activation of Ann & Robert H. Lurie Children’s Hospital of Chicago, a top-ranked hospital, as the first of five QTCs for ZEVASKYN. The first patient is expected to be treated in the third quarter of 2025. Abeona expects to activate all five QTCs by the end of 2025.
● High interest from patients and caregivers: Since approval, approximately 30 patients and caregivers have started registering in the Abeona Assist patient services program.
● Increasing enthusiasm from healthcare professionals: New data from two posters were presented at the 2025 Society for Investigative Dermatology (SID) Annual Meeting. One poster presentation details the potential progenitor cell populations within ZEVASKYN that may contribute to long-term wound closure and persistent COL7A1 expression observed after a single treatment. A second poster presentation details the absence of insertional oncogenesis and replication competent retrovirus in clinical and pre-clinical experience with ZEVASKYN.

● Securing broad patient access: Abeona has executed value-based agreements with several commercial payer groups representing dozens of downstream plans and approximately 100 million commercially-insured lives. In addition, Abeona is in active discussions with multiple commercial and government payers to further expand ZEVASKYN access to eligible patients in the U.S.

Key corporate updates

● Secured non-dilutive capital: Abeona entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease PRV for gross proceeds of $155 million upon the closing of the transaction. Abeona was awarded the PRV following the FDA approval of ZEVASKYN.

"The proceeds from our PRV sale fully fund our operations for over two years, extending our runway through our projected ZEVASKYN-driven profitability in early 2026," said Joe Vazzano, Chief Financial Officer of Abeona. "This robust financial footing, achieved even before ZEVASKYN revenues, eliminates the need for additional capital to reach this crucial commercial milestone."

Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $84.5 million as of March 31, 2025, before accounting for the proceeds pending the close of the PRV sale. As of December 31, 2024, cash, cash equivalents, restricted cash and short-term investments totaled $98.1 million.

Research and development spending for the three months ended March 31, 2025 was $9.9 million, compared to $7.2 million for the same period of 2024. The increase was primarily due to increased headcount related to scale-up of manufacturing capacity in preparation for the planned ZEVASKYN commercial launch and pre-clinical development work. General and administrative expenses were $9.8 million for the three months ended March 31, 2025, compared to $7.1 million for the same period of 2024. The increase was primarily due to increased headcount associated with the planned launch of ZEVASKYN.

Net loss was $12.0 million for the first quarter of 2025, or $0.24 loss per common share. Net loss in the first quarter of 2024 was $31.6 million, or $1.16 loss per common share.

Conference Call Details

The Company will host a conference call and webcast on Thursday, May 15, 2025, at 8:30 a.m. ET, to discuss the financial results and corporate progress. To access the call, dial 877-545-0523 (U.S. toll-free) or 973-528-0016 (international) and Entry Code: 292299 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at View Source The archived webcast replay will be available for 30 days following the call.

On May 15, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that the Phase 3 KEYNOTE-B96 trial, also known as ENGOT-ov65, met its primary endpoint of progression-free survival (PFS) for the treatment of patients with platinum-resistant recurrent ovarian cancer whose tumors expressed PD-L1 and in all comers (Press release, Merck & Co, MAY 15, 2025, View Source [SID1234653174]). The study also met a secondary endpoint of overall survival (OS) in patients whose tumors express PD-L1. The study is evaluating KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in combination with chemotherapy (paclitaxel) with or without bevacizumab for these patients. The trial is continuing and OS for the full study population will be evaluated at a future analysis.

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At pre-specified interim analyses conducted by an independent Data Monitoring Committee, the KEYTRUDA-based regimen demonstrated a statistically significant and clinically meaningful improvement in PFS regardless of PD-L1 status compared to placebo plus chemotherapy with or without bevacizumab. The study also showed a statistically significant and clinically meaningful improvement in OS in patients whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1) compared to placebo plus chemotherapy with or without bevacizumab. The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified. Results will be presented at an upcoming medical meeting and shared with regulatory authorities worldwide.

"This marks the first time a KEYTRUDA-based regimen has shown the ability to help certain patients with platinum-resistant ovarian cancer live longer, and the first time an immune checkpoint inhibitor-based regimen has demonstrated an overall survival benefit in ovarian cancer," said Dr. Gursel Aktan, vice president, global clinical development, Merck Research Laboratories. "The positive results from this trial add to the growing body of evidence supporting the potential benefit of KEYTRUDA across gynecological cancers, including this difficult-to-treat form of ovarian cancer for which patients are in need of new options."

KEYTRUDA is not approved to treat ovarian cancer (see selected KEYTRUDA indications in the U.S. below). LYNPARZA (olaparib), which is being jointly developed and commercialized by AstraZeneca and Merck, has three approved ovarian cancer indications in the U.S.: in first-line maintenance treatment of BRCA-mutated advanced ovarian cancer, following complete or partial response to first-line platinum-based chemotherapy; in first-line maintenance treatment of HRD-positive advanced ovarian cancer in combination with bevacizumab, following complete or partial response to first-line platinum-based chemotherapy; and in maintenance treatment of BRCA-mutated recurrent ovarian cancer, following complete or partial response to platinum-based chemotherapy. For each of these indications, patients are selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA (see indications for LYNPARZA below). In 2024, Merck announced the initiation of the Phase 2/3 REJOICE-Ovarian01 trial evaluating raludotatug deruxtecan (R-DXd), an investigational potential first-in-class CDH6 directed DXd antibody-drug conjugate discovered by Daiichi Sankyo and being jointly developed with Merck, in patients with platinum-resistant ovarian cancer.

About KEYNOTE-B96/ENGOT-ov65

KEYNOTE-B96, also known as ENGOT-ov65, is a randomized, double-blind Phase 3 trial (ClinicalTrials.gov, NCT05116189) sponsored by Merck and conducted in collaboration with the European Network for Gynecologic Oncology Trial (ENGOT) groups investigating KEYTRUDA in combination with chemotherapy (paclitaxel) with or without bevacizumab compared to placebo plus chemotherapy with or without bevacizumab for the treatment of platinum-resistant recurrent ovarian cancer. The primary endpoint is PFS, and OS is a key secondary endpoint. The trial enrolled an estimated 643 patients who were randomized to receive:

KEYTRUDA (400 mg intravenously every six weeks for approximately two years) plus paclitaxel with or without bevacizumab, or;
placebo plus paclitaxel with or without bevacizumab.
About ovarian cancer

Ovarian cancer often begins in the fallopian tubes or on the outer surface of the ovaries. It is the third most common gynecologic malignancy and seventh most common cancer in women worldwide. Globally, there were approximately 324,603 patients diagnosed with ovarian cancer and about 206,956 deaths from the disease in 2022. In the U.S., it is estimated there will be approximately 20,890 patients diagnosed with ovarian cancer and about 12,730 deaths from the disease in 2025. The primary aim of first-line treatment is to delay disease progression for as long as possible with the intent to achieve long-term remission.

On May 15, 2025 The START Center for Cancer Research ("START"), the world’s largest early-phase community-based oncology site network, reported the dosing of the first U.S. participant in Moderna’s Phase 1 study evaluating mRNA-4106, a novel Pan-Tumor Antigen Therapy candidate, in patients with advanced or metastatic solid tumors (Press release, Moderna Therapeutics, MAY 15, 2025, View Source [SID1234653192]).

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The first dose was administered at START San Antonio by Dr. Amita Patnaik who shared, "mRNA represents a bold new frontier in cancer immunotherapy, and we’re proud to provide our patients access to this innovative treatment as part of a first-in-human trial. At START, our mission is to ensure that scientific breakthroughs translate into real-world options for patients—this study is a powerful example of that commitment."

The Phase 1 trial (ClinicalTrials.gov identifier: NCT06880549) will evaluate the safety, pharmacodynamics, immunogenicity and preliminary efficacy of mRNA-4106 administered alone and in combination with checkpoint inhibitor therapy. mRNA-4106’s multivalent approach aims to broaden treatment options for patients with cancer, advancing the field beyond single-targeted immunotherapies.

"This milestone reflects the core of who we are at START – accelerating the next generation of cancer therapies by bringing them directly to patients in the communities where they live," said Nick Slack, MBE, Chairman and CEO of START. "We’re proud to support Moderna in advancing this innovation into the clinic, reinforcing our commitment to faster, more accessible early phase trials that expand opportunity and impact for patients and sponsors alike."

"With mRNA-4106, we sought to design an inclusive therapy that encodes for antigens commonly shared across patients and tumor types. We are thrilled to be able to bring this medicine to trial participants, and in partnership with forward-thinking site networks like START, further showcase the promise of mRNA to transform cancer care," said Dr. Rose Loughlin, Executive Vice President of Research at Moderna.

START’s participation in this trial reinforces its leadership in accelerating access in transformative therapies, delivering on its mission of "Hope Through Access" for patients worldwide.

On May 15, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX), a biotechnology company developing novel Antibody Drug Conjugates (ADCs) with immuno-oncology payloads for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2025 and provided a corporate update (Press release, Akari Therapeutics, MAY 15, 2025, View Source [SID1234653157]).

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"We remain laser focused on becoming a key player in the ADC space and advancing our novel ADC platform built around immuno-oncology payloads and our lead asset AKTX-101, an ADC targeting Trop2 with our immuno-oncology payload, PH1. We continue to develop and execute a clear path forward for our ADC pipeline and support these efforts with ongoing activities and building the team that we believe positions us for success in the near and long term," commented Abizer Gaslightwala, President and Chief Executive Officer of Akari. "In particular, we were pleased to recently welcome Mark Kubik, a seasoned leader in the Antibody Drug Conjugate space, as Head of Business Development, Oncology and believe his expertise will be invaluable as we continue to advance our novel ADC platform technology."

Leveraging its innovative payload platform, the Company is advancing a pipeline of potentially first-in-class, best-in-class ADC candidates across a wide range of cancer tumor targets. These initial candidates have shown significant tumor-killing activity in preclinical models with the ability to robustly activate the immune system to drive durable, and sustained outcomes.

Upcoming Expected Value-Driving Milestones

Novel ADC’s With Immuno-Oncology Payloads

•
Anticipate presenting preclinical data showing that a proof-of-concept ADC with PH1 payload exhibits robust immuno-oncology activity, at a scientific conference in second half of 2025.
•
Complete additional preclinical studies for novel PH1 payload exploring activity in prostate cancer cell lines.
•
Explore preclinical activity for AKTX-101 in different solid tumor indications including lung, as single agent and in combination with other approved agents.
•
Continue to focus on operational excellence and efficient capital allocation to advance novel payload ADC platform.
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Ongoing efforts to seek strategic partners for research collaborations on PH1 immuno-oncology payload across customized tumor targets. Continued discussions with partners on advancing AKTX-101 ADC (Trop2/PH1 payload) through additional IND-enabling activities.

Non-Core Asset Out Licensing

•
Continue efforts to out-license non-core assets across inflammation, ophthalmology, and rare diseases as a source of non-dilutive capital to invest into ADC platform.

Summary of Financial Results for First Quarter 2025

The net loss from operations for the three months ended March 31, 2025 was approximately $3.7 million compared to $5.6 million for the same period in 2024.

The Company reported research and development expenses of $0.8 million for the three months ended March 31, 2025 compared to approximately $2.3 million for the same period in 2024. The decrease was primarily due to our decision to suspend our HSCT-TMA clinical stage program with nomacopan in May 2024.

General and administrative expenses were approximately $2.7 million for the three months ended March 31, 2025 compared to approximately $3.7 million for the same period in 2024. The decrease was primarily due to (i) decreases in legal and professional fees (primarily related to the Merger) and (ii) a decrease in directors’ and officers’ insurance.

As of March 31, 2025, the Company had cash of approximately $2.6 million. The net proceeds from the Company’s March 2025 offering, after deducting placement agent fees and other offering expenses, were approximately $6.0 million, of which $4.0 million was received in April 2025.