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RedHill’s Opaganib Receives FDA Rare Pediatric Disease Designation for Neuroblastoma in Addition to Current Orphan Drug Designation

On June 9, 2026 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to opaganib for treatment of neuroblastoma (NB).

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"Receiving a cancer diagnosis is always distressing, but when it involves your child, it becomes profoundly devastating. There is an ongoing necessity to explore new alternatives that can augment treatment and enhance results for neuroblastoma, the most prevalent cancer in infants. In data from models of high-risk NB (HRNB), presented at AACR (Free AACR Whitepaper) 2026, we saw positive effects of opaganib as a potential add-on to chemotherapy, showing an ability to directly destabilize a key oncogenic driver of neuroblastoma and other solid tumors, n-Myc, through increased ceremide production enhancing programmed cancer cell death (apoptosis)," said Dr. Mark Levitt, Chief Scientific Officer at RedHill. "This rare pediatric disease designation, supported by data from NB and other preclinical oncology models, along with a clinically demonstrated safety and tolerability profile adds to our belief that opaganib holds promise for improving outcomes in treating pediatric NB. We aim to further advance development following ongoing discussions with Penn State University and the Beat Childhood Cancer consortium."

The FDA grant of rare pediatric disease designation to opaganib provides for a Priority Review Voucher (PRV), subject to certain conditions, and with opaganib’s current neuroblastoma orphan drug designation also allows for the potential for seven years’ marketing exclusivity, if approved, accelerated development and review times, FDA Prescription Drug User Fee Act (PDUFA) application fee waivers and tax credits. The neuroblastoma market is expected to be valued at approximately $3.5 billion in 2032.

Opaganib is a novel, potentially broad acting, oral, small molecule sphingosine kinase-2 (SPHK2) selective inhibitor drug with demonstrated safety & efficacy profiles. It is in development for multiple oncology, viral (including Ebola virus disease (EVD), inflammatory and diabetes and obesity-related indications.

About Neuroblastoma

Neuroblastoma is a type of cancer most commonly affecting babies and young children. While rare, neuroblastoma is the most common infancy cancer with ~5,500 global pediatric cases per year in children aged 0–14. It accounts for 10% of childhood cancers and 15% of pediatric cancer-related deaths in the U.S.5,6. Around 750 children in the United States are diagnosed with neuroblastoma each year7. Approximately half of all neuroblastoma patients have high risk (HRNB) disease which has an overall five-year survival of ~50%8.

Neuroblastoma originates from immature nerve cells (neuroblasts), and most often forms in the adrenal glands – small organs that sit on top of the kidneys – but can also start in nerve cells in the abdomen, chest, neck, or pelvis. The exact cause of neuroblastoma is not well understood, but genetic mutations and abnormalities are known to play a role. Some cases may be linked to genetic syndromes or family history, although most occur sporadically without a clear inherited pattern.

About Opaganib (ABC294640)

Opaganib is a proprietary first-in-class investigational, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor drug. Potentially broad-acting, it is in development for multiple oncology, viral (including Ebola virus disease), inflammatory, metabolic (diabetes and obesity) and additional indications.

Opaganib’s suggested mechanism of action, published in the journal Drug Design, Development and Therapy, is host-directed and potentially broad-acting and is expected to maintain its effect against emerging viral variants. Opaganib is thought to work through the inhibition of multiple pathways, the induction of autophagy and apoptosis, and disruption of viral replication, through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SPHK2, DES1 and GCS).

Opaganib has received Orphan Drug designation from the FDA for the treatment of neuroblastoma and cholangiocarcinoma. A Bayer-supported 80-patient placebo-controlled randomized Phase 2 study is ongoing to evaluate the efficacy of opaganib in combination with Bayer’s darolutamide in men with metastatic castrate-resistant prostate cancer (mCRPC), testing the potentially enhancing effect of opaganib in patients with a poor prognosis9. Opaganib also has a Phase 1 chemoradiotherapy study protocol ready for FDA-IND submission.

Opaganib has demonstrated its safety and tolerability profile in more than 470 participants in multiple clinical studies and expanded access use, including a large global Phase 2/3 study in hospitalized patients with moderate to severe COVID-19, published in Microorganisms.

(Press release, RedHill Biopharma, JUN 9, 2026, View Source [SID1234666513])

GT Medical Technologies Completes Oversubscribed $100 Million Series E Equity Financing to Advance Commercial Treatment for Patients with Operable Brain Tumors

On June 9, 2026 GT Medical Technologies, Inc., a medical device company dedicated to improving the lives of patients with brain tumors, reported the closing of an oversubscribed $100 millon Series E equity financing. The financing was led by new investor Viking Global Investors with participation from key existing investors, including MVM Partners, Gilde Healthcare, Evidity Health Capital, Medtech Venture Partners and FemHealth Ventures.

"This financing is validation of GammaTile’s potential to be a standard of care treatment for operable brain tumors and will accelerate our ability to bring this important therapy to even more patients," said Per Langoe, Chief Executive Officer at GT Medical Technologies.

The funds will be used to accelerate investments across commercial and operational initiatives following the release of the final data from the Company’s ROADS randomized controlled trial (RCT) last month. The ROADS RCT studied the use of GammaTile, the Company’s FDA-cleared bioresorbable radiotherapy implant, in patients with newly diagnosed brain metastases. The ROADS RCT data was selected for oral presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) conference and demonstrated statistical superiority for GammaTile relative to standard of care; GammaTile reduced the risk of tumor recurrence and death at 12 months by 93% and 41%, respectively.1

Proceeds from the financing will also support continued expansion of GT Medical Technologies’ recently initiated BRIDGES RCT in patients with newly diagnosed glioblastomas (GBMs).

"We are excited to lead this financing and to work with GT to unlock the potential of GammaTile therapy," said Jason Rostovsky, a Principal at Viking Global Investors. "GT Medical Technologies’ technology has demonstrated profound clinical impact, and we look forward to supporting the GT Medical Technologies team in improving the treatment algorithm for patients with resectable brain tumors."

"An oversubscribed financing of this magnitude and the strength of our syndicate provides significant optionality for the Company, allowing us to accelerate the next phase of our growth and make meaningful clinical and operational investments for the future," added James Leech, Chief Financial & Strategy Officer at GT Medical Technologies.

GammaTile is an innovative form of radiation therapy placed at the time of brain tumor removal surgery, delivering immediate, targeted radiation to the tumor resection site when cancer cells are at their lowest residual levels. Unlike conventional approaches that require delays between surgery and the initiation of radiation therapy to allow for wound healing, GammaTile eliminates this treatment gap by starting treatment immediately. By delivering immediate, concentrated radiation directly at the tumor site, GammaTile maximizes the treatment’s effectiveness against remaining cancer cells and reduces the risk of regrowth.

ROADS RCT data presented at the 2026 ASCO (Free ASCO Whitepaper) conference demonstrated that the 12-month rate of tumor surgical bed recurrence was dramatically lower with GammaTile at 1.3% compared with standard of care at 15.4% (HR:0.07, p=0.012).1 Surgical bed recurrence-free survival, defined as the time from surgery to either tumor recurrence or death from any cause, whichever occurred first, was significantly improved with GammaTile, with the median time to an event not reached compared with standard of care at 10.9 months (HR: 0.48, P=0.002).1 Additionally, 24-month overall survival was 61.7% for GammaTile compared with 35.7% for standard of care (HR: 0.59, p=0.032).

(Press release, GT Medical Technologies, JUN 9, 2026, View Source [SID1234666512])

Imugene’s Azer-cel Granted FDA Fast Track Designation for CLL/SLL and MZL

On June 9, 2026 Imugene Limited (ASX:IMU), a clinical-stage immuno-oncology company, reported that the US Food and Drug Administration (FDA) has granted Fast Track Designation to azer-cel (azercabtagene zapreleucel) for two indications: relapsed or refractory Chronic Lymphocytic Leukaemia / Small Lymphocytic Lymphoma (CLL/SLL) and relapsed or refractory Marginal Zone Lymphoma (MZL).

The FDA’s Fast Track Designation is designed to facilitate the development and expedite the review of drugs that treat serious or life-threatening conditions and meet an unmet medical need. Benefits include more frequent meetings with the FDA to discuss development plans, the option for rolling review of regulatory submissions, and potential eligibility for Accelerated Approval and Priority Review upon meeting the relevant criteria.

Azer-cel is an off-the-shelf, CD19-directed CAR T-cell therapy engineered to overcome the logistical constraints of autologous CAR T therapies, which typically require three to six weeks to manufacture from a patient’s own cells. By using pre-manufactured donor T-cells, azer-cel can be ready to administer within days. For patients with relapsed or refractory disease who have exhausted standard treatment options, that speed is clinically significant.

Chronic Lymphocytic Leukaemia / Small Lymphocytic Lymphoma (CLL/SLL)

CLL/SLL is one of the most common blood cancers in adults. Many patients experience disease progression despite multiple prior treatments. Clinical data from the Phase 1b basket study demonstrates a 100% ORR in the CAR T-naive CLL/SLL cohort, in patients who had received a median of three or more prior lines of therapy. Azer-cel’s activity in CAR T-naive patients is significant in demonstrating the potential of an off-the-shelf allogeneic approach to deliver responses in patients who have not previously received cellular therapy.

Marginal Zone Lymphoma (MZL)

MZL is an indolent but incurable B-cell lymphoma. Patients who relapse following standard therapies including anti-CD20 chemoimmunotherapy have limited remaining options. Updated clinical data from the Phase 1b basket study, which post-dates the ASCO (Free ASCO Whitepaper) data cut, shows an 83% ORR in MZL, based on five of six evaluable patients responding, including four complete responses, in patients who had received a median of two or more prior lines of therapy.

Leslie Chong, Managing Director and CEO of Imugene, commented: "FDA Fast Track Designation for both CLL/SLL and MZL reflects the meaningful clinical activity we are seeing with azer-cel across multiple B-cell malignancies. For patients who have exhausted standard treatment options in these indications, we believe azer-cel represents a genuinely promising approach, and this designation will support closer engagement with the FDA as we advance the program."

(Press release, Imugene, JUN 9, 2026, View Source [SID1234666511])

Summit Therapeutics Announces Proposed Public Offering of Common Stock

On June 9, 2026 Summit Therapeutics Inc. (Nasdaq: SMMT) ("Summit," "we," or the "Company") reported that it has commenced an underwritten public offering of $500.0 million of shares of its common stock. All of the shares in the proposed offering are being offered by Summit. In addition, Summit intends to grant the underwriters a 30-day option to purchase up to an additional $75.0 million of shares of its common stock at the public offering price, less underwriting discounts and commissions. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Summit intends to use the net proceeds from the proposed offering, together with its existing cash, cash equivalents, to fund the research and development of its lead product candidate, ivonescimab, and for working capital and other general corporate purposes.

J.P. Morgan, Goldman Sachs & Co. LLC and Citigroup are acting as joint book-running managers for the proposed offering.

The securities described above are being offered by Summit pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was previously filed with the Securities and Exchange Commission (SEC) and which became automatically effective on June 9, 2026. A preliminary prospectus supplement and accompanying base prospectus relating to and describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying base prospectus may also be obtained, when available, from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by email at [email protected] and [email protected]; Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at (866) 471-2526, or by email at [email protected]; or Citigroup, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (800) 831-9146.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, Summit Therapeutics, JUN 9, 2026, View Source [SID1234666510])

Zetagen Therapeutics Presents Phase 2a Results at ASCO for Investigational ZetaMet™ (Zeta BC 003) in Metastatic Breast Cancer Patients with Lytic Bone Lesions

On June 9, 2026 Zetagen Therapeutics, a clinical-stage biopharmaceutical company developing novel therapies for primary and metastatic breast cancer, reported preliminary topline results from its Phase 2a clinical study evaluating ZetaMet (Zeta BC 003) in subjects with metastatic breast cancer (MBC) with lytic bone lesions.

In this open-label study, no skeletal-related events (SREs) or fractures were observed, no treatment-emergent adverse events (AEs) or serious adverse events (SAEs) were reported, and cessation of tumor activity was noted in treated vertebral bodies. Zeta BC 003 is an investigational product that has received Breakthrough Designation from the U.S. Food and Drug Administration (FDA) and has not been approved by the FDA or any regulatory authority.

"We are encouraged by the preliminary findings from this Phase 2a study, which provide important clinical observations on the investigational use of ZetaMet in patients with metastatic breast cancer involving bone," said Joe C. Loy, President & CEO of Zetagen Therapeutics. "These results also highlight a major achievement for our team, overcoming longstanding industry challenges in intratumoral administration by developing proprietary carriers which deliver compounds that demonstrate solubility and localized bio-adhesion."

The preliminary findings were presented by Dr. Bryan Margulies, Chief Scientific Officer, and Joe C. Loy, President & CEO, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Monday, June 2, 2026. Abstract: View Source

Study Overview
The open-label Phase 2a study (ZGMBC; NCT05280067), conducted at the University of British Columbia, enrolled 10 subjects with a mean age of 52, representing multiple MBC subtypes:

HR+ (n=6)
HR+/HER2+ (n=2)
HER2+/HR– (n=1)
TNBC (n=1)
Five subjects had breakthrough lytic lesions despite prior bisphosphonate therapy. One subject died due to pleural edema, unrelated to study treatment. Across the 10 subjects, 11 target lesions received a single intratumoral injection of Zeta BC 003 under sedation. Four adjacent, non-injected lesions were also evaluated.

Study Context
Historical literature reports SRE rates of approximately 53% (Parke et al., Journal Oncologist, 2018) in metastatic breast cancer with bone involvement under conventional therapy, with SREs, particularly fractures, associated with a reduction in overall survival of approximately 4.8 months (Saad et al., Journal of the National Cancer Institute, 2004).

Observed Study Findings
No SREs or fractures reported
Cessation of tumor activity within treated vertebral bodies
Therapeutic spread observed to adjacent, untreated lesions within the same vertebral body
No treatment-emergent AEs or SAEs
Mean bone defect volume decreased 65.4% at Day 84 (±20.5%; p=0.0003)
Mean bone defect volume decreased 84.1% at Day 180 (±13.1%; p<0.0001)
Pain scores (NRS) decreased by 4.16 points (p<0.05)
Opioid use (MED) decreased 33–67% among opioid-treated subjects
Spinal stability (SINS) improved 18.5% (p<0.05)
Quality of life improved:
PCS increased 24.2%
MCS increased 12.1%
While cross-study comparisons have inherent limitations, the absence of AEs and SREs in this Phase 2a study provides supportive information for continued investigation.

These observations are also consistent with two published Expanded Access case reports (seven lesions, 2-year follow-up; Palma et al., Pain Management, 2023), which similarly demonstrated absence of SREs, cessation of tumor activity, neo-trabecular bone formation, and therapeutic spread within treated vertebral bodies.

(Press release, Zetagen Therapeutics, JUN 9, 2026, View Source [SID1234666509])