On September 30, 2026 Rznomics Inc., a biopharmaceutical company specializing in the development of RNA-based gene therapies, reported that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on the Orphan Medicinal Product Designation (OMPD) for RZ-001 (taspitimagene advec) for the treatment of glioma.

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RZ-001 is an adenovirus serotype 5-vectored gene therapy built on Rznomics’ proprietary RNA trans-splicing ribozyme platform. It targets human telomerase reverse transcriptase (hTERT) mRNA—overexpressed in 80–90% of human cancers—and replaces the target RNA with a therapeutic RNA encoding the cytotoxic herpes simplex virus thymidine kinase (HSV-TK), thereby selectively inducing apoptosis in cancer cells.

This recommendation was grounded in a Phase 1/2a interim clinical data from patients with recurrent glioblastoma (GBM), a subtype of glioma and a highly aggressive WHO Grade IV brain cancer associated with a 1-year survival rate of less than 25%. Despite existing authorized therapies in Europe, the COMP determined that RZ-001 has sufficient potential to provide a "significant benefit".

RZ-001 is currently being evaluated in a Phase 1/2a clinical trial in South Korea (IND clearance in the U.S.) for patients with hTERT-positive recurrent GBM, and has been administered to 10 patients enrolled in clinical trials and to 3 patients under the U.S. FDA’s Expanded Access Program.

"This positive opinion from the EMA COMP shows that RZ-001 goes beyond meeting basic prevalence criteria by delivering meaningful, evidence-backed clinical advantages over current standard-of-care treatments for glioma," said Bomi Kim, Head of the Drug Development Center at Rznomics. "We will continue to advance our global regulatory and clinical development roadmap."

"Following our FDA RMAT designation for hepatocellular carcinoma (HCC) in May 2026, this European regulatory recognition further strengthens the therapeutic versatility and commercial potential of our RNA editing platform," added Dr. Seong-Wook Lee, CEO of Rznomics. "We are committed to expanding RZ-001’s target indications and accelerating global business development opportunities."

Following formal designation by the European Commission, Rznomics will be eligible for key regulatory and commercial incentives, including up to 10 years of market exclusivity, EMA protocol assistance, and reductions in regulatory and filing fees associated with European marketing applications.

About Glioblastoma (GBM)
Glioblastoma is the most common and aggressive primary malignant brain tumor in adults, classified as a WHO Grade IV tumor. Characterized by rapid progression, high recurrence rates, and resistance to standard therapies, recurrent glioblastoma remains an area of significant unmet medical need with limited effective therapeutic options available globally.

About Orphan Medicinal Product Designation in the European Union
Orphan Medicinal Product Designation in the European Union is granted to medicinal products for the treatment of rare, serious, or life-threatening conditions and provides regulatory incentives, including ten years of market exclusivity following approval, which may be extended to twelve years in certain circumstances.

How RZ-001 (taspitimagene advec) Works
RZ-001 utilizes Rznomics’ proprietary RNA trans-splicing ribozyme technology. The ribozyme embedded in RZ-001 selectively recognizes and cleaves hTERT mRNA expressed in cancer cells, replacing it with an RNA strand containing a suicide gene (HSV-TK) derived from the herpes simplex virus. Subsequently, the orally administered antiviral prodrug valganciclovir, is converted to ganciclovir and further activated by HSV-TK, generating cytotoxic metabolites that inhibit viral and cellular DNA synthesis and selectively induce apoptosis in cancer cells.

(Press release, Rznomics, SEP 30, 2026, View Source [SID1234671195])

On September 30, 2026 Phrontline Biopharma, a clinical-stage biotechnology company developing next-generation antibody-drug conjugates (ADCs), reported that the first patient has been dosed in its global, first-in-human Phase I/II study of TJ102, an investigational FRα (FOLR1) × CDH6 bispecific dual-payload ADC, in patients with advanced or metastatic ovarian cancer and other solid tumors. The study is being conducted under an IND cleared by the U.S. FDA and a clinical trial application approved by China’s NMPA.

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"Dosing the first patient with TJ102 is an important milestone for Phrontline," said Zhaoyuan "Tony" Chen, Ph.D., Chief Executive Officer of Phrontline Biopharma. "TJ102 combines dual tumor targeting with two mechanistically distinct, potency-balanced payloads in a single, defined molecule. Our DLP1 platform attaches a pre-assembled dual-payload linker to the antibody in one step, supporting scalable manufacturing and consistent product quality."

The open-label, multicenter study (TJ102-I/II-001-G; NCT07778498) will assess safety, tolerability, pharmacokinetics, immunogenicity and preliminary antitumor activity, with dose escalation followed by dose-optimization and expansion cohorts to select recommended dose(s) for further development.

"This trial tests the central hypothesis behind TJ102: that targeting both FRα and CDH6 while co-delivering two distinct payloads may overcome the antigen heterogeneity and resistance that limit single-target, single-payload ADCs," said Martín Sebastian Olivo, MD, MSc, Chief Medical Officer of Phrontline Biopharma. "Our immediate priority is to rigorously characterize safety, pharmacokinetics and early signs of clinical activity."

About TJ102

TJ102 is an investigational IgG1 bispecific ADC targeting FRα and CDH6, two antigens expressed in ovarian and other solid tumors. Its DLP1 linker-payload is a branched, protease-cleavable linker carrying a topoisomerase I inhibitor and a potency-balanced microtubule inhibitor. Four DLP1 units are conjugated to each antibody in a single step, yielding a defined 4 + 4 payload composition (drug-to-antibody ratio of 8). TJ102 has not been approved by any regulatory authority, and its safety and efficacy have not been established.

(Press release, Phrontline Biopharma, SEP 30, 2026, View Source [SID1234671194])

On September 30, 2026 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported operational highlights and financial results for the six months ended June 30, 2026.

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Commercial portfolio update:

$12 million upfront payment to Ferring for the acquisition of commercialization rights to Rebyota and Clenpiq, fully funded by the $18 million received upfront for the Talicia divestment
Transactions strengthen liquidity, provide potential growth and expansion opportunities and enable addition of complementary revenue-generating products
RedHill’s lean commercial infrastructure supports delivery of significant synergies with capacity for further portfolio extension
Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "We have executed on two major transactions that unlock significant value and fuel our capacity for growth. The acquisition of commercial rights to Rebyota and Clenpiq, funded by sale of Talicia, improves our liquidity position and provides us with control to drive increased revenues, identify additional complementary revenue-generating products and support ongoing development of our assets. We are now focused on the rapid and smooth transition of Rebyota and Clenpiq into RedHill, utilizing our lean and efficient commercial team, which is already well positioned and deeply embedded in the U.S. GI market."

Mr. Ben-Ashercontinued: "Looking forward to the second half of 2026 and beyond, we also aim to progress our R&D development projects, which provide compelling opportunities in areas of significant unmet need, including multiple oncology indications, Crohn’s disease and other GI-associated conditions and Ebola virus disease (EBOV). In oncology, opaganib has been granted the U.S. Food and Drug Administration’s rare pediatric disease designation for neuroblastoma, with potential to receive an accompanying Priority Review Voucher. Furthermore, new preclinical data supportive of opaganib add-on therapy in neuroblastoma and triple negative breast cancer was presented at AACR (Free AACR Whitepaper), while recruitment continues into the Bayer-supported Phase 2 study of opaganib and darolutamide in advanced castrate resistant prostate cancer. Progress has also been made on key aspects of our Phase 2-ready Crohn’s program with RHB-204, potentially the first ever in a wholly MAP-positive population, with in vitro assay data demonstrating RHB-204’s comparable MAP killing to RHB-104. Meanwhile, RHB-102 continues its late-stage development for oncology support and GI indications including GLP-1/GIP-associated GI intolerance."

Commercial focus:

Following the divestment of RedHill’s 70% stake in Talicia for $18 million in upfront cash, plus up to $35 million in potential worldwide net sales milestone payments, the Company has acquired for an upfront cash payment of $12 million, plus future milestones and tiered royalties, the exclusive global and U.S. commercialization rights to Rebyota and Clenpiq, respectively, two synergistic, revenue-generating, FDA-approved GI drugs that together generated approximately $37.5 million in 2025 net sales under Ferring.

With a growing medical need, the potential for expanded indications and territories, and an experienced and efficient commercial infrastructure that is highly established within the GI market, these two new assets provide RedHill with a stronger commercial engine for revenue growth, in both the U.S. and other new territories, with potential for further added value through acquisition of additional complementary revenue-generating products.

Rebyota:

Rebyota is a proprietary, first and only single-dose FDA-approved Fecal Microbiota Transplant (FMT) for prevention of recurrent Clostridioides difficile (C. diff) infection (rCDI) in individuals 18 years of age and older following antibiotic treatment for rCDI. FDA-approved in 2022, it generated approximately $16.9 million in U.S. net sales in 2025 through its clinical profile, broad coverage and established customer base.

C. diff(CDI) infection is a distressing and life-threatening condition, being the most common cause of healthcare-associated (HA) diarrhea in developed countries1. An estimated 3.6 million cases of CDI occur globally each year, including approximately half a million cases in the U.S. 30,000 Americans die every year due to CDI2, with a global mortality rate of 1-in-10, rising to almost 1-in-3 in high-risk settings1. In the U.S., up to approximately 165,000 CDI cases result in one or more recurrences3, indicating the need for use of prevention strategies.

Rebyota’s one-time administration, which requires no fasting, bowel prep or extended treatment timeframe, has demonstrated more than a 70% success rate at preventing rCDI4. An aging population and accompanying increasing hospital-acquired vulnerabilities will likely increase this need. A streamlined ordering and reimbursement process is already in place for physicians, and broad and improving U.S. payer coverage means that 93% of lives have coverage including 43% with coverage after 1st recurrence5.

Rebyota, which was granted, Breakthrough Therapy and Orphan Drug designations, with exclusivities and other patent protections that could run to 2036, provides multiple growth opportunities, including recent approval in Canada. The existing approvals also provide the potential for additional approvals in other territories.

Clenpiq:

Clenpiq is a ready-to-drink, low-volume bowel preparation on the U.S. market. In 2025, it delivered $20.6 million in net sales, with minimal promotion under Ferring. Colonoscopy is the cornerstone of GI practice, with more than 15 million colonoscopies performed in the U.S. annually6. This number is rising, with follow-up needed after noninvasive screening tests that yield a positive result, an aging population, and guidelines that recommend testing at age 45 instead of age 50, contributing to an increased need for colonoscopy.

Bowel preparation quality drives outcomes and cost, but it is not a pleasant process and is often poorly performed – driving the need for repeated procedures. As such the benefits of a simplified, ready-to-drink, low-volume bowel preparation are evident. Clenpiq has broad commercial and government payer coverage making its benefits readily accessible to patients: More than 108 million lives (63%) with unrestricted (no prior approval) have commercial coverage, including 57 million (33%) with preferred position. 23 million lives (41%) of Medicare Part D lives have unrestricted access to Clenpiq, including 12 million (21%) lives with preferred position5.

Focused and advanced R&D pipeline – first half of 2026 highlights:

Opaganib granted U.S. Food and Drug Administration (FDA) rare pediatric disease designation for neuroblastoma, the most prevalent cancer in infants, providing potential for an accompanying Priority Review Voucher (PRV)
New preclinical data showed positive effects of opaganib as potential add-on therapy in models of neuroblastoma (NB) and triple-negative breast cancer (TNBC). Two sets of data presented at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting indicate that:
– Opaganib may enhance the therapeutic efficacy of the oxaliplatin + doxorubicin (OXDOX) chemotherapy combination in high-risk NB by directly destabilizing n-Myc, a key oncogenic driver of neuroblastoma and other solid tumors, through increased ceremide production enhancing programmed cell death (apoptosis) in cancer cells7
– Pre-treatment with opaganib, followed by low-dose diABZI treatment, potentiated the downstream STING-mediated effects and may augment anti-tumor immunity in TNBC, which has the poorest prognosis of the breast cancer subtypes8
– Opaganib Ebola (Bundibugyo, BDBV) development next steps and potential partnership discussions ongoing with African Medicines Agency, Ugandan Ministry of Health and Mbarara University of Science and Technology (MUST), alongside liaison with the Democratic Republic of Congo (DRC) Scientific Advisory Committee
In Crohn’s disease, new RHB-204in vitro data, from both spot and phage assays, demonstrated comparable MAP killing to RHB-1049. RHB-204 achieved these results with lower doses of key active ingredients enhancing potential for reduced toxicity and side effects, supporting an innovative FDA-aligned Phase 2-ready study design allowing for a smaller sample size, lower study costs and faster time to completion
Financial results for the six months ended June 30, 2026 (Unaudited) 10

Following the transfer of the Talicia commercial operations to Talicia Holding Inc. ("THI") in October 2025, Talicia’s operations are presented as "discontinued operations" in RedHill’s financial statements. The comparison below is therefore presented on a continuing operations basis, with first half of 2025 comparative figures re-presented on the same basis. RedHill’s ongoing economic participation in the Talicia franchise during the period is reflected separately as its share of loss of the joint venture. RedHill sold its entire 70% interest in THI to Apotex in August 2026.

Net Revenues: No revenues were recognized in the first half of 2026, compared to $0.3 million in the first half of 2025, which was generated from the Hyloris license agreement for RHB-102 (Bekinda).

Research and Development Expenses for the first half of 2026 were $1.0 million, unchanged from the first half of 2025. During the first half of 2026, the Company continued preclinical work related to RHB-204 and opaganib in Ebola.

General, Administrative and Business Development Expenses for the first half of 2026 were $2.5 million, compared to $2.7 million for the first half of 2025. The decrease was primarily attributable to lower professional services expenses and the allocation of certain costs to THI following the transfer of Talicia commercial operations.

Share of Loss of Joint Venture for the first half of 2026 was $0.7 million, representing RedHill’s share of THI’s net loss for the period. THI recorded net revenues of $1.3 million and a net loss of $1.0 million for the first half of 2026.

Operating Loss for the first half of 2026 was $4.1 million, compared to $3.4 million for the first half of 2025. The increase was mainly attributable to the share of THI’s loss and the absence of license revenues in the first half of 2026, partially offset by lower general, administrative and business development expenses.

Financial Expenses, net for the first half of 2026 were $1.6 million, compared to Financial Income, net of $0.5 million for the first half of 2025. Financial expenses in the first half of 2026 mainly reflected warrant-related issuance costs and fair value adjustments on derivative financial instruments.

Net Loss from Continuing Operations for the first half of 2026 was $5.7 million, compared to $2.9 million for the first half of 2025. The increase was mainly attributable to the share of THI’s loss and higher net financial expenses, partially offset by lower general, administrative and business development expenses.

Net Loss from Discontinued Operations for the first half of 2026 was $0.4 million, compared to $1.2 million for the first half of 2025. The decrease was primarily attributable to the transfer of the Talicia commercial operations to THI.

Net Loss for the first half of 2026 was $6.1 million, compared to $4.1 million for the first half of 2025.

Total Assets as of June 30, 2026, were $26.3 million, compared to $25.3 million as of December 31, 2025. The increase was mainly attributable to higher cash and other receivables, partially offset by a decrease in the investment in THI.

Total Liabilities as of June 30, 2026, were $27.4 million, compared to $21.1 million as of December 31, 2025. The increase primarily reflected derivative liabilities associated with the June 2026 private placement and higher accrued expenses and other liabilities.

Net Cash Used in Operating Activities for the first half of 2026 was $4.6 million, compared to $5.0 million for the first half of 2025.

Net Cash Provided by Financing Activities for the first half of 2026 was $5.4 million, primarily reflecting net proceeds from the June 2026 private placement, compared to $3.3 million for the first half of 2025, primarily generated through the Company’s equity offerings11.

Cash Balance as of June 30, 2026, was $5.3 million.

(Press release, RedHill Biopharma, SEP 30, 2026, View Source [SID1234671193])

On September 30, 2026 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal immunotherapies to improve disease outcomes for patients with cancer, reported the presentation of new biomarker data from its completed phase 3 clinical trial of aglatimagene besadenovec (aglatimagene) in patients with localized intermediate- to high-risk prostate cancer at the American Society for Radiation Oncology (ASTRO) Annual Meeting 2026.

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The Company used digitized hematoxylin and eosin-stained prostate biopsy samples collected at baseline and after treatment with either aglatimagene or placebo, from patients who had completed the 3-injection course (n=647) in the randomized, placebo-controlled phase 3 study (NCT01436968). AI-enabled digital pathology analysis performed on tumor biopsies evaluable for digital analysis (622 pre-treatment and 427 post-treatment collected more than two years after completion of radiation), demonstrates that aglatimagene plus external beam radiation therapy (EBRT) significantly increased intratumoral lymphocyte infiltration and remodeled the tumor microenvironment compared with placebo plus EBRT. Among those patients with residual tumor, aglatimagene increased lymphocyte infiltration within tumor regions and enhanced lymphocyte-tumor engagement.

Key Highlights from ASTRO 2026 Presentation:

The Company previously reported that two years after treatment, 80% of evaluable aglatimagene patients (n=167) had negative prostate biopsies versus 63% of control patients (n=62) (p=0.0018)
Aglatimagene significantly increased overall lymphocytic fraction compared to control (p=0.048), with both arms showing immune activation from radiation therapy (p<0.001) (calculated in all digital-pathology evaluable biopsies; 622 pre-treatment biopsies and 427 post-treatment biopsies analyzed)
In paired biopsies from patients with residual tumor (n=108; aglatimagene n=62, control n=46), aglatimagene significantly increased intratumoral lymphocyte fraction (p=0.006), lymphocyte-tumor enrichment (p=0.003), and lymphocyte-tumor mixing (p<0.001)

"While radiation therapy alone drives immune infiltration into prostate tumors, these data support that aglatimagene produced a qualitatively distinct immune response," said Francesca Barone, M.D., Ph.D., Chief Scientific Officer of Candel Therapeutics. "The significantly greater lymphocyte infiltration and sustained lymphocyte-tumor engagement which we observed, more than two years after treatment, supports durable immune remodeling rather than transient inflammation. This mechanistic insight may help explain the clinical improvements in disease-free survival and pathologic outcomes we reported in the phase 3 trial."

"These tissue-level findings provide important biological support for the clinical benefit demonstrated in our phase 3 trial," said Paul Peter Tak, M.D., Ph.D., FMedSci, President and Chief Executive Officer of Candel. "We believe the convergence of sustained immune remodeling, improved pathologic response, and a clinically meaningful improvement in disease-free survival provides a compelling and coherent picture of aglatimagene’s activity in localized prostate cancer as we advance toward our planned Biologics License Application submission."

The data was presented by Dr. Barone during the Genitourinary Cancer session at ASTRO 2026 on September 29, 2026, and the poster is titled "Quantitative digital pathology defines immune activation following aglatimagene besadenovec immunotherapy in localized prostate cancer" (Abstract LBA 30).

About aglatimagene besadenovec

Aglatimagene besadenovec (aglatimagene), Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s tumor. After intratumoral administration, HSV-tk enzyme activity results in conversion of prodrug (valacyclovir) into deoxyribonucleic acid (DNA)-incorporating nucleotide analogs, leading to immunogenic cell death in cells exhibiting DNA damage and proliferating cells, with subsequent release of a variety of tumor (neo)antigens in the tumor microenvironment. At the same time, the adenoviral serotype 5 capsid proteins promote inflammation through the induction of expression of pro-inflammatory cytokines, chemokines, and adhesion molecules. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. Aglatimagene has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several nonclinical and clinical settings. More than 1,000 patients have been dosed with aglatimagene in clinical trials with a favorable tolerability profile to date, supporting the potential for use with standard of care, when indicated. Aglatimagene is currently not approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority for any use.

(Press release, Candel Therapeutics, SEP 30, 2026, View Source [SID1234671192])

On September 30, 2026 Genentech, a member of the Roche Group (SIX: RO, ROP; OTCQX: RHHBY), reported that detailed results from the evERA Breast Cancer study, showing investigational giredestrant in combination with everolimus significantly improved progression-free survival (PFS) versus standard endocrine therapy plus everolimus, were published in The New England Journal of Medicine. The evERA trial is evaluating giredestrant, an investigational oral selective estrogen receptor degrader (SERD), in combination with everolimus in people with estrogen receptor (ER)-positive locally advanced or metastatic breast cancer who had disease progression or recurrence following treatment with a cyclin-dependent kinase (CDK)4/6 inhibitor and endocrine therapy.

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"Despite advancements with CDK4/6 inhibitor treatment in the first-line setting, many people with advanced breast cancer will eventually experience disease progression as their tumors become resistant to endocrine therapy," said Dr. Erica L. Mayer, medical oncologist at Dana-Farber Cancer Institute. "The evERA trial demonstrated that the giredestrant combination significantly delayed disease progression, potentially offering a promising all-oral treatment option in a setting where there remains a need for additional treatments to improve patient outcomes."

In the ESR1-mutated population, the median PFS was 10.0 months with giredestrant plus everolimus compared with 5.5 months in the comparator arm (stratified hazard ratio [HR]=0.38, 95% CI: 0.27-0.54, p<0.001). In the intent-to-treat (ITT) population, the median PFS was 8.8 months compared with 5.5 months in the giredestrant and comparator arms, respectively (HR=0.56, 95% CI: 0.44-0.71, p<0.001). Overall survival (OS) data were immature at the time of analysis, but a clear positive trend has been observed in the ITT (HR=0.69, 95% CI: 0.47-1.00) and ESR1-mutated populations (HR=0.62, 95% CI: 0.38-1.02). Follow-up for OS will continue to the next analysis. Adverse events for the giredestrant combination were manageable and consistent with the known safety profiles of the individual medicines. No unexpected safety findings were observed, including no photopsia and low rates of bradycardia.

"The evERA results represent an important step forward for people with ER-positive advanced breast cancer and underscore the potential for giredestrant to help address the challenge of disease progression in this setting," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "Together with the positive Phase III lidERA results in early breast cancer, these findings reinforce the potential of giredestrant to redefine care in both metastatic and early disease settings."

Additional evERA analyses were presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, showing that giredestrant plus everolimus prolongs PFS2 (time from randomization to disease progression following second-line therapy) and chemotherapy-free survival compared with standard endocrine therapy, suggesting that the clinical benefit may be sustained beyond initial progression.

Hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer accounts for approximately 70% of breast cancer cases. Resistance to endocrine therapies, particularly in the post-CDK inhibitor setting, increases the risk of disease progression and is associated with poor outcomes.

evERA was the first positive Phase III readout for giredestrant, followed by the lidERA Breast Cancer study in the early-stage setting. Based on these evERA data, the U.S. Food and Drug Administration (FDA) accepted Genentech’s New Drug Application (NDA) for giredestrant in combination with everolimus for the treatment of people with ER-positive, HER2-negative, ESR1-mutated locally advanced or metastatic breast cancer whose disease has recurred during or after completing endocrine-based adjuvant therapy, or who have progressed on at least one endocrine-based regimen in the metastatic setting. The FDA is expected to decide on the application by December 18, 2026. The FDA also accepted an NDA under Priority Review for giredestrant as an adjuvant treatment for adults with ER-positive, HER2-negative, stage I, II and III breast cancer based on the lidERA results. The FDA has set a Prescription Drug User Fee Act goal date of November 30, 2026.

Genentech’s expanding giredestrant clinical development program spans distinct treatment settings and lines of therapy, reflecting our commitment to deliver innovative medicines to as many people with HR-positive breast cancer as possible.

About the evERA Breast Cancer study

evERA Breast Cancer [NCT05306340] is a Phase III, randomized, open-label, multicenter study evaluating the efficacy and safety of giredestrant in combination with everolimus versus standard endocrine therapy in combination with everolimus in people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer who have had previous treatment with cyclin-dependent kinase (CDK)4/6 inhibitor and endocrine therapy, either in the adjuvant or locally advanced/metastatic setting.

The co-primary endpoints are investigator-assessed progression-free survival in the intention-to-treat and ESR1-mutated populations, defined as the time from randomization to the time when the disease progresses or a patient dies from any cause. In the post-CDK inhibitor setting, up to 40% of people with ER-positive disease have ESR1 mutations. Key secondary endpoints include overall survival, objective response rate, duration of response, clinical benefit rate and safety.

About giredestrant

Giredestrant is an investigational, oral, potent next-generation selective estrogen receptor degrader and full antagonist.

Giredestrant is designed to block estrogen from binding to the estrogen receptor (ER), triggering its breakdown (known as degradation) and stopping or slowing down the growth of cancer cells.

Giredestrant has an extensive clinical development program including five company-sponsored Phase III clinical trials to address the needs of patients across multiple treatment settings and lines of therapy:

Giredestrant versus standard endocrine therapy as adjuvant treatment in ER-positive, human epidermal growth factor receptor 2 (HER2)-negative early-stage breast cancer (lidERA Breast Cancer; NCT04961996)
Giredestrant plus everolimus versus standard endocrine therapy plus everolimus in ER-positive, HER2-negative, locally advanced or metastatic breast cancer (evERA Breast Cancer; NCT05306340)
Giredestrant plus palbociclib versus letrozole plus palbociclib in ER-positive, HER2-negative, endocrine-sensitive, recurrent locally advanced or metastatic breast cancer (persevERA Breast Cancer; NCT04546009)
Giredestrant plus investigator’s choice of a cyclin-dependent kinase (CDK)4/6 inhibitor versus fulvestrant plus a CDK4/6 inhibitor in ER-positive, HER2-negative advanced breast cancer resistant to adjuvant endocrine therapy (pionERA Breast Cancer; NCT06065748)
Giredestrant plus dual HER2 blockade versus dual HER2 blockade in ER-positive, HER2-positive locally advanced or metastatic breast cancer (heredERA Breast Cancer; NCT05296798)

About hormone receptor (HR)-positive breast cancer

Globally, the burden of breast cancer continues to grow, with 2.3 million women diagnosed and 670,000 dying from the disease every year. Breast cancer remains the number one cause of cancer-related deaths amongst women, and the second most common cancer type.

HR-positive, human epidermal growth factor receptor 2-negative breast cancer accounts for approximately 70% of breast cancer cases and the majority are diagnosed in the early-stage. A defining feature of HR-positive breast cancer is that its tumor cells have receptors that attach to hormones like estrogen, which can contribute to tumor growth.

Despite treatment advances, HR-positive breast cancer remains particularly challenging to treat due to its biological complexity. Patients often face the risk of disease progression, treatment side effects and resistance to endocrine therapy. There is an urgent need for more effective treatments that can delay clinical progression and reduce the burden of treatment on people’s lives.

(Press release, Genentech, SEP 30, 2026, https://www.gene.com/media/press-releases/15132/2026-09-30/genentechs-giredestrant-combination-sign [SID1234671191])