ENHERTU® Demonstrated Highly Statistically Significant and Clinically Meaningful Improvement in Invasive Disease-Free Survival Versus T-DM1 in DESTINY-Breast05 Phase 3 Trial in Patients with High-Risk Early Breast Cancer Following Neoadjuvant Therapy




ENHERTU® Demonstrated Highly Statistically Significant and Clinically Meaningful Improvement in Invasive Disease-Free Survival Versus T-DM1 in DESTINY-Breast05 Phase 3 Trial in Patients with High-Risk Early Breast Cancer Following Neoadjuvant Therapy

• Second positive phase 3 trial of Daiichi Sankyo and AstraZeneca’s ENHERTU in HER2 positive early breast cancer reinforces its potential to become a foundational treatment option in curative-intent setting
• Results from the DESTINY-Breast05 and DESTINY-Breast11 trials will be presented at ESMO 2025 in a Presidential Symposium
• Plans for regulatory submissions are underway

TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Positive topline results from a planned interim analysis of the DESTINY-Breast05 phase 3 trial showed ENHERTU^® (trastuzumab deruxtecan) demonstrated a highly statistically significant and clinically meaningful improvement in invasive disease-free survival (IDFS) versus trastuzumab emtansine (T-DM1) in patients with HER2 positive early breast cancer with residual invasive disease in the breast or axillary lymph nodes after neoadjuvant treatment and high risk of disease recurrence. This is the second positive phase 3 trial of ENHERTU in the HER2 positive early breast cancer setting following positive results from the DESTINY-Breast11 phase 3 neoadjuvant trial earlier this year.

Overall survival (OS) was not mature at the time of this planned interim analysis and will be assessed at a subsequent analysis.

ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN).

Currently, approximately half of patients with HER2 positive early breast cancer have residual disease following neoadjuvant treatment, putting them at an increased risk of disease recurrence.^1-7 Despite receiving additional treatment in the post-neoadjuvant setting, some patients still ultimately experience tumor progression to metastatic disease. ^8,9 New treatment options are needed in the early breast cancer setting to help reduce the likelihood of disease progression and improve long-term outcomes for more patients.^10,11

“In patients with early breast cancer with residual disease following neoadjuvant treatment, it is critical to optimize treatment as this represents the last opportunity to prevent progression to metastatic disease,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “The results of DESTINY-Breast05 demonstrate that treatment with ENHERTU following surgery increases the length of time patients are able to live free of invasive disease compared to the existing standard of care, potentially offering patients with HER2 positive early breast cancer a new treatment approach in this curative-intent setting.”

“This landmark trial is the first to directly compare ENHERTU and T-DM1 in early breast cancer and the results clearly show that ENHERTU delivers superior outcomes, indicating that it may be a better option for patients with high risk HER2 positive early breast cancer in the post-neoadjuvant setting,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology Hematology R&D, AstraZeneca. “These results from DESTINY-Breast05, coupled with DESTINY-Breast11, underscore our commitment to moving ENHERTU into early-stage HER2 positive breast cancer where patients can achieve sustained long-term outcomes, increasing the opportunity for cure.”

The safety profile of ENHERTU observed in DESTINY-Breast05 was consistent with its known profile with no new safety concerns identified.

Data from DESTINY-Breast05 (Abstract #LBA1) and DESTINY-Breast11 (Abstract #291O) will be presented during Presidential Symposium I on Saturday, October 18 at the upcoming 2025 European Society for Medical Oncology (#ESMO25) Congress. The DESTINY-Breast05 data also will be shared with global regulatory authorities.

DESTINY-Breast05 was conducted in collaboration with the National Surgical Adjuvant Breast and Bowel Project Foundation (NSABP), the German Breast Group (GBG), Arbeitsgemeinschaft Gynäkologische Onkologie (AGO-B) and SOLTI Breast Cancer Research Group.

About DESTINY-Breast05

DESTINY-Breast05 is a global, multicenter, randomized, open-label, phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus T-DM1 in patients with HER2 positive primary breast cancer with residual invasive disease in breast or axillary lymph nodes following neoadjuvant therapy and a high risk of recurrence. High risk of recurrence was defined as presentation with inoperable cancer (prior to neoadjuvant therapy) or pathologically positive axillary lymph nodes following neoadjuvant therapy.

The primary endpoint of DESTINY-Breast05 is investigator-assessed IDFS. IDFS is defined as the time from randomization until first recurrence, distant recurrence or death from any cause. The key secondary endpoint is investigator-assessed disease-free survival. Other secondary endpoints include OS, distant recurrence-free interval, brain metastases-free interval and safety.

DESTINY-Breast05 enrolled 1,635 patients in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.

About HER2 Positive Early Breast Cancer

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.¹² More than two million breast cancer cases were diagnosed in 2022, with more than 665,000 deaths globally.¹²

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors including breast cancer.¹³ HER2 protein overexpression may occur as a result of HER2 gene amplification and is often associated with aggressive disease and poor prognosis in breast cancer.¹⁴ Approximately one in five cases of breast cancer are considered HER2 positive.¹⁵

Currently, approximately half of patients with HER2 positive early breast cancer have residual disease following neoadjuvant treatment, putting them at an increased risk of disease recurrence.^1-7 Despite receiving additional treatment in the post-neoadjuvant setting, some patients still ultimately experience tumor progression to metastatic disease.^8,9 Once patients are diagnosed with metastatic disease, the five-year survival rate drops from nearly 90% to approximately 30%.¹⁶ New treatment options are needed in the early breast cancer setting to help reduce the likelihood of disease progression and improve long-term outcomes for more patients.^10,11

About ENHERTU

ENHERTU (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

ENHERTU (5.4 mg/kg) is approved in more than 85 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (immunohistochemistry [IHC] 3+ or in-situ hybridization (ISH)+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

ENHERTU (5.4 mg/kg) is approved in more than 85 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

ENHERTU (5.4 mg/kg) is approved in more than 45 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 low (IHC 1+ or IHC 2+ / ISH-) or HER2 ultralow (IHC 0 with membrane staining) breast cancer, as determined by a locally or regionally approved test, that has progressed on one or more endocrine therapies in the metastatic setting based on the results from the DESTINY-Breast06 trial.

ENHERTU (5.4 mg/kg) is approved in more than 60 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 and/or DESTINY-Lung05 trials. Continued approval in China and the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (6.4 mg/kg) is approved in more than 70 countries/regions worldwide for the treatment of adult patients with locally advanced or metastatic HER2 positive (IHC 3+ or IHC 2+/ISH+) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric06 trials. Continued approval in China for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (5.4 mg/kg) is approved in more than 10 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02 trials. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About the ENHERTU Clinical Development Program

A comprehensive global clinical development program is underway evaluating the efficacy and safety of ENHERTU as a monotherapy or in combination or sequentially with other cancer medicines across multiple HER2 targetable cancers.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and DATROWAY^® in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and DATROWAY.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and DATROWAY, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

ENHERTU U.S. Important Safety Information

Indications

ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with:

• Unresectable or metastatic HER2-positive (IHC 3+ or ISH positive) breast cancer who have received a prior anti-HER2-based regimen either:
  • In the metastatic setting, or
  • In the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy

• Unresectable or metastatic:
  • Hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, that has progressed on one or more endocrine therapies in the metastatic setting
  • HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer, as determined by an FDA-approved test, who have received a prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy

• Unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
  

• Locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH positive) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen

• Unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Contraindications

None.

Warnings and Precautions

Interstitial Lung Disease / Pneumonitis

Severe, life-threatening, or fatal interstitial lung disease (ILD), including pneumonitis, can occur in patients treated with ENHERTU. A higher incidence of Grade 1 and 2 ILD/pneumonitis has been observed in patients with moderate renal impairment. Advise patients to immediately report cough, dyspnea, fever, and/or any new or worsening respiratory symptoms. Monitor patients for signs and symptoms of ILD. Promptly investigate evidence of ILD. Evaluate patients with suspected ILD by radiographic imaging. Consider consultation with a pulmonologist. For asymptomatic ILD/pneumonitis (Grade 1), interrupt ENHERTU until resolved to Grade 0, then if resolved in ≤28 days from date of onset, maintain dose. If resolved in >28 days from date of onset, reduce dose 1 level. Consider corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥0.5 mg/kg/day prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), permanently discontinue ENHERTU. Promptly initiate systemic corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥1 mg/kg/day prednisolone or equivalent) and continue for at least 14 days followed by gradual taper for at least 4 weeks.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, ILD occurred in 12% of patients. Median time to first onset was 5.5 months (range: 0.9 to 31.5). Fatal outcomes due to ILD and/or pneumonitis occurred in 0.9% of patients treated with ENHERTU.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, ILD occurred in 10% of patients. Median time to first onset was 2.8 months (range: 1.2 to 21).

Neutropenia

Severe neutropenia, including febrile neutropenia, can occur in patients treated with ENHERTU. Monitor complete blood counts prior to initiation of ENHERTU and prior to each dose, and as clinically indicated. For Grade 3 neutropenia (Absolute Neutrophil Count [ANC] <1.0 to 0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then maintain dose. For Grade 4 neutropenia (ANC <0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then reduce dose by 1 level. For febrile neutropenia (ANC <1.0 x 10⁹/L and temperature >38.3º C or a sustained temperature of ≥38º C for more than 1 hour), interrupt ENHERTU until resolved, then reduce dose by 1 level.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, a decrease in neutrophil count was reported in 65% of patients. Nineteen percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 22 days (range: 2 to 939). Febrile neutropenia was reported in 1.2% of patients.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, a decrease in neutrophil count was reported in 72% of patients. Fifty-one percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 16 days (range: 4 to 187). Febrile neutropenia was reported in 4.8% of patients.

Left Ventricular Dysfunction

Patients treated with ENHERTU may be at increased risk of developing left ventricular dysfunction. Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including ENHERTU. Assess LVEF prior to initiation of ENHERTU and at regular intervals during treatment as clinically indicated. Manage LVEF decrease through treatment interruption. When LVEF is >45% and absolute decrease from baseline is 10-20%, continue treatment with ENHERTU. When LVEF is 40-45% and absolute decrease from baseline is 20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF of 20% is confirmed, permanently discontinue ENHERTU. Permanently discontinue ENHERTU in patients with symptomatic congestive heart failure. Treatment with ENHERTU has not been studied in patients with a history of clinically significant cardiac disease or LVEF <50% prior to initiation of treatment.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, LVEF decrease was reported in 4.6% of patients, of which 0.6% were Grade 3 or 4.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, no clinical adverse events of heart failure were reported; however, on echocardiography, 8% were found to have asymptomatic Grade 2 decrease in LVEF.

Embryo-Fetal Toxicity

ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of ENHERTU. Advise females of reproductive potential to use effective contraception during treatment and for 7 months after the last dose of ENHERTU. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for 4 months after the last dose of ENHERTU.

Additional Dose Modifications

Thrombocytopenia
For Grade 3 thrombocytopenia (platelets <50 to 25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then maintain dose. For Grade 4 thrombocytopenia (platelets <25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then reduce dose by 1 level.

Adverse Reactions

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

The pooled safety population reflects exposure to ENHERTU 5.4 mg/kg intravenously every 3 weeks in 2233 patients in Study DS8201-A-J101 (NCT02564900), DESTINY-Breast01, DESTINY-Breast02, DESTINY-Breast03, DESTINY-Breast04, DESTINY-Breast06, DESTINY-Lung01, DESTINY-Lung02, DESTINY-CRC02, and DESTINY-PanTumor02. Among these patients, 67% were exposed for >6 months and 38% were exposed for >1 year. In this pooled safety population, the most common (≥20%) adverse reactions, including laboratory abnormalities, were decreased white blood cell count (73%), nausea (72%), decreased hemoglobin (67%), decreased neutrophil count (65%), decreased lymphocyte count (60%), fatigue (55%), decreased platelet count (48%), increased aspartate aminotransferase (46%), increased alanine aminotransferase (44%), increased blood alkaline phosphatase (39%), vomiting (38%), alopecia (37%), constipation (32%), decreased blood potassium (32%), decreased appetite (31%), diarrhea (30%), and musculoskeletal pain (24%).

HER2-Positive Metastatic Breast Cancer

DESTINY-Breast03
The safety of ENHERTU was evaluated in 257 patients with unresectable or metastatic HER2-positive breast cancer who received at least 1 dose of ENHERTU 5.4 mg/kg intravenously once every 3 weeks in DESTINY-Breast03. The median duration of treatment was 14 months (range: 0.7 to 30) for patients who received ENHERTU.

Serious adverse reactions occurred in 19% of patients receiving ENHERTU. Serious adverse reactions in >1% of patients who received ENHERTU were vomiting, ILD, pneumonia, pyrexia, and urinary tract infection. Fatalities due to adverse reactions occurred in 0.8% of patients including COVID-19 and sudden death (1 patient each).

ENHERTU was permanently discontinued in 14% of patients, of which ILD/pneumonitis accounted for 8%. Dose interruptions due to adverse reactions occurred in 44% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose interruption were neutropenia, leukopenia, anemia, thrombocytopenia, pneumonia, nausea, fatigue, and ILD/pneumonitis. Dose reductions occurred in 21% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose reduction were nausea, neutropenia, and fatigue.

The most common (≥20%) adverse reactions, including laboratory abnormalities, were nausea (76%), decreased white blood cell count (74%), decreased neutrophil count (70%), increased aspartate aminotransferase (67%), decreased hemoglobin (64%), decreased lymphocyte count (55%), increased alanine aminotransferase (53%), decreased platelet count (52%), fatigue (49%), vomiting (49%), increased blood alkaline phosphatase (49%), alopecia (37%), decreased blood potassium (35%), constipation (34%), musculoskeletal pain (31%), diarrhea (29%), decreased appetite (29%), headache (22%), respiratory infection (22%), abdominal pain (21%), increased blood bilirubin (20%), and stomatitis (20%).

Contacts

Media Contacts:

Global/US:
Jennifer Brennan

Daiichi Sankyo

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

Japan:
Daiichi Sankyo Co., Ltd.

DS-PR_jp@daiichisankyo.com

Investor Relations Contact:
DaiichiSankyoIR_jp@daiichisankyo.com

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PureTech Presents New Data from Phase 2b Open-Label Extension Study of Deupirfenidone (LYT-100), Further Supporting Strong and Durable Efficacy and Potential to Serve as New Standard of Care in IPF




PureTech Presents New Data from Phase 2b Open-Label Extension Study of Deupirfenidone (LYT-100), Further Supporting Strong and Durable Efficacy and Potential to Serve as New Standard of Care in IPF

Patients who switched from placebo or pirfenidone to deupirfenidone in the open-label extension study achieved stabilization of lung function with favorable tolerability

Findings further substantiate safety and efficacy results from the randomized, placebo- and active-controlled 26-week trial and highlight opportunity to address patients inadequately served by current therapies

Regulatory engagement underway; update on Phase 3 trial design expected in Q4 2025

BOSTON–(BUSINESS WIRE)–PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced new data from the open-label extension (OLE) of its Phase 2b ELEVATE IPF trial of deupirfenidone (LYT-100) in people living with idiopathic pulmonary fibrosis (IPF). These new results showed that participants who completed 26 weeks of placebo or pirfenidone treatment in the randomized portion of the trial and then switched to deupirfenidone for an additional 26 weeks in the OLE achieved stabilization of lung function. These findings, delivered in a late-breaking oral presentation at the 2025 European Respiratory Society (ERS) Congress in Amsterdam, Netherlands, highlight the potential for deupirfenidone to become a new standard of care for the treatment of IPF.

“The blinded portion of the ELEVATE trial challenged the perspective that the biggest opportunity for new therapies in IPF is improved safety, by showing that treatment with deupirfenidone 825 mg three times a day can achieve lung function stabilization with favorable tolerability. The initial 52-week extension data then raised the bar by demonstrating that this effect with deupirfenidone was durable,” said Argyrios E. Tzouvelekis, M.D., Ph.D., University of Patras, Greece, and presenting investigator at ERS 2025. “Now we are seeing that two additional patient cohorts who experienced lung function decline in Part A of the trial achieved stabilization once switched to deupirfenidone. These findings reinforce that the blinded results with deupirfenidone are reproducible and support the potential for benefit in patients transitioning from standard of care.”

ELEVATE IPF, a global, randomized, double-blind, active- and placebo-controlled Phase 2b trial, achieved its primary endpoint and demonstrated a statistically significant and clinically meaningful reduction in lung function decline at 26 weeks with deupirfenidone 825 mg three times a day (TID) compared to placebo (Part A). As previously announced, participants treated with deupirfenidone 825 mg TID experienced a slower rate of lung function decline, as measured by change from baseline of Forced Vital Capacity (FVC), at 26 weeks versus those who were treated with pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks. Following the completion of the blinded portion of the trial, 170 participants (more than 90%) enrolled in the OLE (Part B). Those who remained on deupirfenidone 825 mg TID maintained a robust treatment effect and experienced an overall FVC decline of -32.8 mL over the 52-week period,¹ which is similar to the expected natural decline in lung function in healthy older adults over that time (approximately -30.0 mL to -50.0 mL).²

The new results presented at ERS provide additional evidence from participants who initially received placebo or pirfenidone for 26 weeks during Part A and then switched to deupirfenidone for 26 weeks in Part B. Those who switched from placebo to deupirfenidone 825 mg TID (n=17) had a mean change in FVC of +20.0 mL (placebo switch cohort), while those who switched from pirfenidone to deupirfenidone 825 mg TID (n=16) had a mean change in FVC of -23.1 mL (pirfenidone switch cohort).³ These results provide further evidence from two additional patient cohorts that deupirfenidone may stabilize the decline in lung function to that expected of healthy older adults and suggest a potential benefit for patients transitioning from standard of care to deupirfenidone.

Deupirfenidone continued to be well tolerated at both doses studied in Part B after six months of treatment, and the safety profile remained consistent with Part A. Additional analyses shared at ERS included a summary of the most common treatment-emergent adverse events (TEAEs) in the OLE, defined as occurring in at least 10% of participants in either treatment group. As of May 9, 2025, the most common TEAEs were nausea (8.4% vs. 11.5%), dyspepsia (14.5% vs. 12.6%), upper respiratory infections (16.9% vs. 17.2%), and cough (10.8% vs. 4.6%) for deupirfenidone 550 mg TID (n=83) and deupirfenidone 825 mg TID (n=87), respectively.

“The ELEVATE trial has been designed and executed to provide one of the most rigorous Phase 2 evaluations of a potential therapy in IPF,” said Sven Dethlefs, Ph.D., Chief Executive Officer of Celea Therapeutics, the PureTech Founded Entity created to advance deupirfenidone. “The reproducibility we’re seeing across blinded and extension data, and now in switch cohorts, gives us strong confidence in the robustness of the findings. We believe deupirfenidone has the potential to become a new standard of care in IPF, and we are actively engaging with regulators to finalize the Phase 3 trial design and expect to share an update in the fourth quarter.”

About Deupirfenidone (LYT-100)

Deupirfenidone (LYT-100) is in development as a potential new standard of care for the treatment of idiopathic pulmonary fibrosis (IPF). It is a deuterated form of pirfenidone, which – along with nintedanib – is one of the two FDA-approved treatments for IPF. Both approved therapies offer only modest efficacy in slowing lung function decline, largely due to tolerability challenges that limit the ability to achieve higher doses that could significantly improve patient outcomes. These limitations have contributed to low treatment uptake and poor adherence, with approximately 25% of people with IPF in the U.S. ever receiving either drug.⁴ Despite this, combined peak global sales exceed $5 billion, representing a significant market opportunity in IPF and other fibrotic lung diseases. ⁵

Deupirfenidone may overcome these limitations. In the global Phase 2b ELEVATE IPF trial, deupirfenidone demonstrated the potential to stabilize lung function decline over at least 26 weeks as a monotherapy while maintaining a favorable safety and tolerability profile. Initial data from an ongoing open-label extension study suggest that this effect may be sustained through at least 52 weeks. These findings support the potential for deupirfenidone to offer a meaningful advance for people living with this progressive and deadly disease. Beyond IPF, deupirfenidone may also address multiple underserved fibrotic conditions, including progressive fibrosing interstitial lung diseases.

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue that leads to a steady decline in lung function. Median survival following diagnosis is estimated to be two to five years, and currently there is no cure.⁶

About Celea Therapeutics

Celea Therapeutics is dedicated to advancing transformative treatments for people with serious respiratory diseases. Drawn from the Latin word for “sky,” the name reflects the company’s mission to rise above the status quo and deliver therapies that change lives. The company’s lead program, deupirfenidone (LYT-100), is a Phase 3-ready therapeutic candidate with the potential to set a new standard of care for idiopathic pulmonary fibrosis (IPF) and other fibrotic lung diseases.

Celea was founded by PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a biotherapeutics company dedicated to giving life to science. PureTech’s innovative R&D model drives the creation of Founded Entities like Celea, enabling the advancement of highly promising medicines to patients in a capital-efficient manner. For more information, please visit www.celeatx.com and www.puretechhealth.com.

About PureTech Health

PureTech Health is a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value. We do this through a proven, capital-efficient R&D model focused on opportunities with validated pharmacology and untapped potential to address significant patient needs. This strategy has produced dozens of therapeutic candidates, including three that have received U.S. FDA approval. By identifying, shaping, and de-risking these high-conviction assets, and scaling them through dedicated structures backed by external capital, we accelerate their path to patients while creating sustainable value for shareholders.

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

Cautionary Note Regarding Forward-Looking Statements

This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements that relate to continued development of and regulatory interactions related to deupirfenidone, the potential of deupirfenidone in IPF and other indications, our expectations around our therapeutic candidates and approach towards addressing major diseases, our plans to advance our programs and deliver on our milestones, our future plans, prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption “Risk Factors” in our Annual Report on Form 20-F for the year ended December 31, 2024 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

PureTech
Public Relations

publicrelations@puretechhealth.com
Investor Relations

IR@puretechhealth.com

UK/EU Media
Ben Atwell, Rob Winder

+44 (0) 20 3727 1000

puretech@fticonsulting.com

US Media
Justin Chen

jchen@tenbridgecommunications.com

Savara Presents New Data From the Phase 3 IMPALA-2 Clinical Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at the European Respiratory Society (ERS) Congress 2025




Savara Presents New Data From the Phase 3 IMPALA-2 Clinical Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at the European Respiratory Society (ERS) Congress 2025

– The Beneficial Clinical Effects of Molgramostim Were Similar in Patients with aPAP Regardless of Disease Severity Defined by DLco% –

– Changes in DLco%, the Primary Endpoint in IMPALA-2, Were Significantly Correlated with Changes in Measures of Quality of Life, Patient Functionality, and Surfactant Burden in Patients with aPAP –

– Savara’s Partner, TrilliumBiO, Presented Data on the Development of a Dried Blood Spot Test to Aid in the Diagnosis of aPAP –

LANGHORNE, Pa.–(BUSINESS WIRE)–Savara Inc. (the “Company”) (Nasdaq: SVRA), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced additional analyses from its pivotal Phase 3 IMPALA-2 clinical trial of molgramostim in aPAP were presented as poster presentations at the ERS Congress 2025 in Amsterdam, The Netherlands.

ERS 2025 Posters

Savara

Title: Efficacy of Inhaled Molgramostim According to Severity of Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Presenter: Cormac McCarthy, M.D., Ph.D., FRCPI, Associate Professor of Medicine at the University College Dublin (UCD) and Consultant Respiratory Physician, St. Vincent’s University Hospital, Dublin, Ireland

Poster Number: PA3026

Poster Summary:

• Prespecified analyses were conducted to determine if the beneficial clinical effects of molgramostim in IMPALA-2, a Phase 3 clinical trial, were similar in patients with aPAP based on their disease severity as measured by hemoglobin-adjusted percent predicted diffusing capacity of the lungs for carbon monoxide, or DLco% (≤50% or >50%) at randomization
• Molgramostim demonstrated improvement compared with placebo on the primary endpoint, change in DLco% from baseline to Week 24, in patients with aPAP regardless of disease severity
• Molgramostim significantly improved measures of pulmonary gas transfer (DLco%), respiratory health-related quality of life (St. George’s Respiratory Questionnaire [SGRQ] Total and Activity scores), and patient functionality (exercise capacity expressed as peak metabolic equivalents [METs]) compared with placebo in both subgroups of patients with DLco% values of ≤50% or >50% at randomization

Title: Relationship Between Pulmonary Gas Transfer, Respiratory Health-related Quality of Life (HRQoL), Exercise Capacity, and Surfactant Burden in Autoimmune Pulmonary Alveolar Proteinosis (PAP)

Presenter: Francesco Bonella, M.D., Ph.D., Head of the Center for Interstitial and Rare Lung Diseases and Assistant Professor, Ruhrlandklinik University Hospital, Essen, Germany

Poster Number: PA3027

Poster Summary:

• DLco% (a measure of pulmonary gas transfer) was chosen as the primary endpoint in the IMPALA-2 Phase 3 clinical trial because it is a standardized measure of pulmonary gas transfer widely used in clinical practice
• Previous research has shown that changes in DLco% correlate with changes in aPAP disease severity (i.e., surfactant accumulation/burden) and may predict the need for whole-lung lavage
• Post-hoc correlation analyses of IMPALA-2 data were conducted to evaluate the relationship between DLco% and measures of HRQoL, patient functionality, and surfactant burden in patients with aPAP
• Significant negative correlations were observed between DLco% and measures of respiratory HRQoL (SGRQ Total and Activity scores; higher scores indicate worse quality of life), as well as DLco% and surfactant accumulation (ground-glass opacity scores; lower scores indicate less surfactant) at Week 24. Significant positive correlations were observed between DLco% and patient functionality (exercise capacity expressed as peak METs; higher scores indicate improved exercise capacity) at Weeks 24 and 48
• Results further support the clinical relevance of changes in DLco% in aPAP; changes in DLco% are associated with changes in clinical outcomes and the extent of pulmonary pathology, making it a clinically meaningful measure in this rare lung disease

TrilliumBiO

Title: Development of a Dried Blood Spot Assay for the Detection of GM-CSF Autoantibodies to Aid in the Diagnosis of Autoimmune Pulmonary Alveolar Proteinosis

Presenter: Eagappanath Thiruppathi, Ph.D., Director of Test and Method Development, TrilliumBiO and Joannah Kim, Vice President of Development and Operations, TrilliumBiO

Poster Number: PA3025

Poster Summary:

• The study was conducted to develop and validate a novel particle-based flow cytometry assay using dried blood spot (DBS) samples for detection of quantitative GM-CSF autoantibody levels to aid in the accurate and timely diagnosis of aPAP
• A strong linear correlation was observed between GM-CSF autoantibody levels measured in serum from venipuncture and those measured using serum-derived dried blood spot samples
• The novel assay demonstrated high precision and sensitivity for the detection of GM-CSF autoantibodies in dried blood spot samples. This approach offers a practical and convenient diagnostic tool to help confirm or rule-out aPAP, representing a significant advancement in the detection of this rare and serious lung disease

For more details about the ERS Congress please visit their website.

The posters are available on the Congresses & Publications page of the Company’s corporate website.

About the IMPALA-2 Trial

IMPALA-2 is a global, pivotal, Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg self-administered once daily by inhalation with matching placebo in patients with aPAP. The trial is being conducted at 43 clinical trial sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and countries in Europe, including Turkey. The primary efficacy assessment was diffusing capacity of the lungs for carbon monoxide (DLco%), a gas transfer measure, and the primary endpoint was change from baseline to Week 24 in percent predicted DLco%, with a secondary endpoint of change from baseline to Week 48 in percent predicted DLco%. Three additional secondary efficacy variables evaluated clinical measures of direct patient benefit: St. George’s Respiratory Questionnaire (SGRQ) Total score, SGRQ Activity score, and exercise capacity using a treadmill test, with each endpoint measured at Weeks 24 and 48. The primary time point for efficacy assessments was at Week 24; however, efficacy was assessed through Week 48 to evaluate durability of effect. Safety was assessed through Week 48. All patients who completed the 48-week double-blind treatment period continued into a 96-week open-label period during which molgramostim 300 mcg is administered once daily.

About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas transfer, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant.

About Savara

Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via a proprietary investigational eFlow^® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of molgramostim. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com and LinkedIn.

Contacts

Media and Investor Relations Contact

Savara Inc.

Temre Johnson, Executive Director, Corporate Affairs

ir@savarapharma.com

Genmab to Acquire Merus, Expanding Late-Stage Pipeline and Accelerating into a Wholly Owned Model




Genmab to Acquire Merus, Expanding Late-Stage Pipeline and Accelerating into a Wholly Owned Model

Company Announcement

• Genmab to acquire Merus for USD 97.00 per share in an all-cash transaction representing a transaction value of approximately USD 8.0 billion
• Proposed acquisition adds petosemtamab, a late-stage asset with two Breakthrough Therapy Designations, to Genmab’s portfolio
• Transaction anticipated to be accretive to EBITDA by end of 2029
• Genmab to host a conference call today at 1:00 PM CEST / 12:00 PM BST / 7:00 AM EDT

COPENHAGEN, Denmark & UTRECHT, Netherlands–(BUSINESS WIRE)–Genmab A/S (Nasdaq: GMAB) and Merus N.V. (Nasdaq: MRUS) announced today that they have entered into a transaction agreement pursuant to which Genmab intends to acquire all the shares of Merus, a clinical-stage biotechnology company with its late-stage breakthrough therapy asset petosemtamab, which is in Phase 3 development, for USD 97.00 per share in an all-cash transaction representing a transaction value of approximately USD 8.0 billion. The transaction has been unanimously approved by the Boards of Directors of both companies. A wholly owned subsidiary of Genmab (“Purchaser”) will commence a tender offer for 100% of Merus’ common shares, which is anticipated to close by early in the first quarter of 2026.

The proposed acquisition of Merus is expected to meaningfully accelerate Genmab’s shift to a wholly owned model, expanding and diversifying the company’s revenue, driving sustained growth into the next decade and contributing to Genmab’s evolution into a biotechnology leader. The addition of petosemtamab, Merus’ lead asset, to Genmab’s promising late-stage pipeline is a compelling strategic fit with Genmab’s portfolio and aligns with Genmab’s expertise in antibody therapy development and commercialization in oncology. Following the closing of the transaction, Genmab will have four proprietary programs expected to drive multiple new drug launches by 2027.

“The proposed acquisition of Merus clearly aligns with our long-term strategy. It has the potential to significantly accelerate our evolution into a global biotechnology leader by providing durable growth for the company well into the next decade,” said Jan van de Winkel, Ph.D., President and Chief Executive Officer of Genmab. “Petosemtamab has the potential to be a transformational therapy for patients living with head and neck cancer. With our proven track record of success, both in clinical development and in commercialization, we are confident that we will be able to unlock the promise of petosemtamab.”

“We are excited for the opportunity to join Genmab, a leader in antibody therapeutics, to further develop and bring petosemtamab to patients. Our two companies have a rich history of innovation with multiple approvals in the field of multispecific antibodies. We believe Genmab has the right vision and experience to advance petosemtamab in recurrent/metastatic head and neck cancer and beyond,” said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. “I’m immensely proud of the Merus team who have pioneered our foundational platform technologies to make better medicines and who have demonstrated – with an approved product and a product candidate, petosemtamab, in registrational studies – an ability to deliver on our promise to close in on cancer.”

Petosemtamab is an EGFRxLGR5 bispecific antibody with the potential to be both first- and best-in-class in head and neck cancer. It has been granted two Breakthrough Therapy Designations (BTD) by the U.S. Food and Drug Administration (FDA) for first- and second-line plus head and neck cancer indications. Of note, compelling Phase 2 data was presented at the American Society for Clinical Oncology (ASCO) 2025 Annual Meeting showing both an overall response rate and median progression free survival that were substantially higher than standard of care.

Merus is currently running two Phase 3 trials in first- and second/third line head and neck cancer, with topline interim readout of one or both trials anticipated in 2026. Based on Genmab’s experience in late-stage development and excellence in commercial execution, Genmab anticipates the potential for the initial launch of petosemtamab in 2027, subject to clinical results and regulatory approvals. Genmab also intends to broaden and accelerate petosemtamab’s development with potential expansion into earlier lines of therapy. Following its initial anticipated approval, Genmab believes that petosemtamab will be accretive to EBITDA with at least one-billion-dollar annual sales potential by 2029, with multi-billion-dollar annual revenue potential thereafter.

Details of the Transaction and Financing

Under the transaction agreement, Purchaser, a wholly owned subsidiary of Genmab, will commence a tender offer for all the outstanding common shares of Merus. Following the closing of the tender offer, Merus and Genmab will effect a series of transactions resulting in Genmab owning 100% of the common shares of Merus (or a successor entity). Depending on the structure of the back end transactions, Merus shareholders that do not tender their shares into the tender offer will either receive the same consideration for their common shares as the common shares tendered into the tender offer (subject to applicable withholding taxes) or a fair price for their common shares determined by a Dutch court in statutory buy-out proceedings. The closing of the tender offer is subject to the satisfaction of customary closing conditions for similar transactions, including a minimum acceptance condition of at least 80% of Merus’ common shares (which threshold may be reduced to 75% unilaterally by Genmab if all other closing conditions are satisfied), approval by Merus’ shareholders of resolutions relating to Merus’ post-closing governance and the back end transactions at Merus’ extraordinary shareholders meeting to be held for that purpose, and completion of the relevant works councils consultation processes.

The USD 97.00 per common share purchase price payable in the tender offer represents a premium of approximately 41% over Merus’ closing stock price on September 26, 2025, of USD 68.89 and approximately 44% over Merus’ 30-day volume weighted average price of USD 67.42.

The transaction is not subject to a financing condition. Consideration is expected to be funded through a combination of cash on hand and approximately $5.5 billion of non-convertible debt financing. Genmab has obtained a funding commitment from Morgan Stanley Senior Funding, Inc. for this amount.

The financing package includes a meaningful portion of prepayable debt, in line with Genmab’s commitment to deleveraging with a target of gross leverage <3x within two years after the closing of the proposed transaction. Today’s news does not impact Genmab’s financial guidance for the full year 2025, last issued on August 7, 2025. Genmab will provide its financial outlook for the full year 2026 in conjunction with its full year 2025 earnings report in February 2026.

PJT Partners and Morgan Stanley & Co. International plc are acting as joint financial advisors to Genmab and A&O Shearman and Kromann Reumert as its legal advisors.

Jefferies LLC is acting as financial advisor to Merus and Latham & Watkins and NautaDutilh as its legal advisors.

Conference Call Details

Genmab will hold a conference call to discuss the transaction today, September 29, 2025 at 1:00 PM CEST / 12:00 PM BST / 7:00 AM EDT. To join the call please use the following registration link https://register-conf.media-server.com/register/BI65be2c038b9b42dbb064dfc843b6a478. Registered participants will receive an email with a link to access dial-in information as well as a unique personal PIN. A live and archived webcast of the calls and relevant slides will be available at https://www.genmab.com/investor-relations.

About Genmab

Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab’s vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines^®.

Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X.

About Merus

Merus is an oncology company developing innovative full-length human bispecific and trispecific antibody therapeutics, referred to as Multiclonics^®. Multiclonics^® are manufactured using industry standard processes and have been observed in preclinical and clinical studies to have several of the same features of conventional human monoclonal antibodies, such as long half-life and low immunogenicity. For additional information, please visit Merus’ website, LinkedIn and Bluesky.

Additional Information

The tender offer for the common shares (“Common Shares”) of Merus referenced in this announcement has not yet commenced. This announcement is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell Common Shares or any other securities, nor is it a substitute for the tender offer materials that Genmab and Purchaser will file or cause to be filed with the Securities and Exchange Commission (the “SEC”) upon the commencement of the tender offer. This communication may be deemed to be solicitation material in respect of the EGM Proposals (defined below). At the time the tender offer is commenced, Genmab and Purchaser will file or cause to be filed with the SEC a tender offer statement on Schedule TO (the “Tender Offer Statement”), and Merus will file with the SEC a solicitation/recommendation statement on Schedule 14D-9 (the “Solicitation/Recommendation Statement”), in each case, with respect to the tender offer. Merus also intends to file with the SEC a proxy statement on Schedule 14A in connection with an extraordinary general meeting of Merus’ shareholders, at which Merus’ shareholders will vote on certain proposed resolutions (the “EGM Proposals”) in connection with the transactions referenced herein, and will mail the definitive proxy statement and a proxy card to each shareholder of Merus entitled to vote at the extraordinary general meeting. THE TENDER OFFER STATEMENT (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS), THE SOLICITATION/RECOMMENDATION STATEMENT AND THE PROXY STATEMENT WILL CONTAIN IMPORTANT INFORMATION. SHAREHOLDERS OF MERUS ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS OF COMMON SHARES SHOULD CONSIDER BEFORE MAKING ANY DECISION WITH RESPECT TO THE TENDER OFFER OR MAKING ANY VOTING DECISION. The tender offer for Common Shares will be made only pursuant to the Offer to Purchase, the Letter of Transmittal and related documents filed as a part of the Tender Offer Statement. The Tender Offer Statement (including the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents), as well as the Solicitation/Recommendation Statement, will be made available to all holders of Common Shares at no expense to them. The Tender Offer Statement and the Solicitation/Recommendation Statement will be made available for free at the SEC’s website at www.sec.gov. Copies of the documents filed by Genmab or Purchaser with the SEC will also be available free of charge on Genmab’s website at https://www.genmab.com/investor-relations or by contacting Genmab’s investor relations department at ir@genmab.com. Copies of the documents filed by Merus with the SEC will also be available free of charge on Merus’ website at https://ir.merus.nl/ or by contacting Merus’ investor relations department at s.spear@merus.nl. In addition, shareholders of Merus may obtain free copies of the tender offer materials by contacting the information agent for the tender offer that will be named in the Tender Offer Statement.

Participants in the Solicitation

Merus and certain of its directors, executive officers and other members of management and employees may be deemed to be participants in soliciting proxies from its shareholders in connection with the proposed back end transactions. Information regarding the persons who may, under the rules of the SEC, be considered to be participants in the solicitation of Merus’ shareholders in connection with the proposed back end transactions will be set forth in Merus’ definitive proxy statement for its extraordinary general meeting at which the EGM Proposals will be submitted for approval by Merus’ shareholders. You may also find additional information about Merus’ directors and executive officers in Merus’ Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the SEC on February 27, 2025 (as amended) and Merus’ Definitive Proxy Statement for its 2025 annual general meeting of shareholders, which was filed with the SEC on April 24, 2025.

This Company Announcement contains forward looking statements. The words “believe,” “expect,” “anticipate,” “intend” and “plan” and similar expressions identify forward looking statements. Statements in this Company Announcement that are forward looking may include, but are not limited to, statements regarding: the benefits and potential effects of the proposed transaction; the development plan, regulatory approval, data release timing, commercial launch timing and revenue potential of petosemtamab; the expected timing of the closing of the proposed transaction; and Genmab’s expectations regarding financing the proposed transaction, de-levering and timing of new drug launches. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, the occurrence of any event, change or other circumstance that could give rise to the right of Genmab or Merus or both of them to terminate the transaction agreement, including circumstances requiring a party to pay the other party a termination fee pursuant to the transaction agreement; the failure to obtain applicable regulatory approvals or clearances or Merus shareholder approval in a timely manner or otherwise; the risk that the proposed transaction may not close in the anticipated timeframe or at all due to one or more of the other closing conditions to the proposed transaction not being satisfied or waived; the risk that there may be unexpected costs, charges or expenses resulting from the proposed transaction; risks related to the ability of Genmab to successfully integrate Merus’ business with Genmab’s existing businesses and achieve the expected benefits of the proposed transaction within the expected timeframes or at all and the possibility that such integration may be more difficult, time consuming or costly than expected; risks that the proposed transaction disrupts Genmab’s or Merus’ current plans and operations; risks related to disruption of each company’s management’s time and attention from ongoing business operations due to the proposed transaction; continued availability of capital and financing and rating agency actions; the risk that any announcements relating to the proposed transaction could have adverse effects on the market price of Genmab’s and/or Merus’ securities or operating results; the risk that the proposed transaction and its announcement could have an adverse effect on the ability of Genmab and Merus to retain and hire key personnel, and to maintain relationships with their respective business partners and on their respective operating results and businesses generally; risks typically associated with conducting clinical trials, including the risk that additional clinical trials testing Merus’ products may not be successful; the risk that Merus’ products may not be approved on expected timelines or at all; the risk of litigation that could be instituted against Genmab or its directors, managers or officers and/or regulatory actions related to the proposed transaction, including the effects of any outcomes related thereto; risks related to unpredictable and severe or catastrophic events, including but not limited to acts of terrorism, war or hostilities, cyber-attacks, or the impact of any pandemic, epidemic or outbreak of an infectious disease in the United States or worldwide on Genmab’s and/or Merus’ business, financial condition and results of operations, as well the response thereto by each company’s management; and other business effects, including the effects of industry, market, economic, political or regulatory conditions. Also, actual results or performance of Genmab and Merus may differ materially from any future results or performance expressed or implied by such statements for a number of additional reasons as described in Genmab’s and Merus’ respective filings with the SEC, including those included in Genmab’s most recent Annual Report on Form 20-F, which is available at www.genmab.com and www.sec.gov, and those included in Merus’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which is available at https://merus.nl/ and www.sec.gov. Neither Genmab nor Merus undertakes any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab^®; the Y-shaped Genmab logo^®; Genmab in combination with the Y-shaped Genmab logo^®; HuMax^®; DuoBody^®; HexaBody^®; DuoHexaBody^®, HexElect^® and KYSO^®.

Multiclonics^®, Biclonics^®, Triclonics^® and ADClonics^® are registered trademarks of Merus N.V.

Contacts

Genmab Contacts:

Marisol Peron, Senior Vice President, Global Communications & Corporate Affairs

T: +1 609 524 0065; E: mmp@genmab.com

Andrew Carlsen, Vice President, Head of Investor Relations

T: +45 3377 9558; E: acn@genmab.com

Merus Contacts:
Sherri Spear, Senior Vice President, Investor Relations and Strategic Communications

T: 617-821-3246; E: s.spear@merus.nl

Kathleen Farren, Director Investor Relations and Corporate Communications

T: 617-230-4165; E: k.farren@merus.nl

Virometix Announces Completion of Enrollment in Phase I Trial of V-212 — a Fully Synthetic, Serotype-Independent Vaccine Candidate Against Streptococcus Pneumoniae




Virometix Announces Completion of Enrollment in Phase I Trial of V-212 — a Fully Synthetic, Serotype-Independent Vaccine Candidate Against Streptococcus Pneumoniae

SCHLIEREN, Switzerland–(BUSINESS WIRE)–Virometix AG, a Swiss clinical-stage biotechnology company pioneering fully synthetic vaccines, today announced that it has successfully completed enrollment in the Phase I clinical trial of V-212, a peptide-based, serotype-independent vaccine candidate targeting Streptococcus pneumoniae infections.

“Completing enrollment in this Phase I trial marks a significant milestone for V-212,” said Anna Sumeray, CEO of Virometix. “This fully synthetic, serotype-independent vaccine candidate is designed to advance our mission of delivering scalable, safe, and broad-spectrum protection against pneumococcal disease, while addressing the current limitations of existing PCV approaches. Through our collaboration with CEVAC, we are well-positioned to deliver high-quality Phase I data, with topline results anticipated in the first quarter of 2026.”

Prof. Isabel Leroux-Roels, Principal Investigator at CEVAC, added, “We are proud to collaborate with Virometix on this first-in-human study of V-212. Pneumococcal infections remain a major global health challenge, underscoring the urgent need for next-generation vaccines with broader and more durable protection. V-212’s fully synthetic, serotype-independent approach is highly innovative, and we look forward to advancing the clinical evaluation of this important candidate.”

About Virometix and the V-212 Program

Virometix develops structure-based, fully synthetic nanoparticle vaccines designed to elicit targeted, robust, and durable immune responses. Its proprietary Synthetic Virus-Like Particle (SVLP) platform employs conformational synthetic peptide mimetics displayed on self-assembling lipopeptidic nanoparticles that include built-in adjuvant elements, including T-helper epitopes and Toll-like receptor (TLR) ligands—eliminating dependence on biological components and simplifying manufacturing.

V-212, the lead pneumococcal vaccine candidate, is specifically engineered as a serotype-independent, peptide-based immunogen. Multiple conserved antigenic epitopes from key Streptococcus pneumoniae surface proteins are synthesized and conjugated to SVLP nanoparticles, aiming to induce broad immunity across diverse serotypes—addressing the limitations of current conjugate vaccines.

Preclinical studies have demonstrated robust, long-lasting immunogenicity in mouse and rabbit models. V-212 prevented lethal sepsis in a serotype 3 challenge, inhibited bacterial dissemination into blood, and reduced pulmonary burden. It also conferred protection against serotype 8 infections. Moreover, antisera elicited by V-212 recognized multiple pneumococcal serotypes, including non-PCV-13 types, underscoring its serotype-independent potential.

Phase I Trial Design and Enrollment Highlights

• Study ID: NCT06975319 (VMX-SPN-212-001)
• Design: A randomized, double-blind, placebo-controlled, first-in-human, Phase I trial in healthy adult volunteers.
• Participants: A total of 60 healthy subjects aged 18–45 years have been enrolled.
• Collaboration: The trial is being conducted in collaboration with CEVAC (Centre for Vaccinology) at Ghent University Hospital, a leading European clinical trial unit with extensive expertise in vaccine development.
• Dosing Regimen: Subjects receive three intramuscular injections of either V-212 or placebo, across low, medium, and high dose groups to assess safety, tolerability, and immunogenicity.
• Primary Objective: Evaluate safety and tolerability across dose levels.
• Secondary Objective: Assess immunogenicity to identify an optimal dose for subsequent studies.
• Next Milestone: Topline safety and immunogenicity data are expected in Q1 2026. 

About Virometix

Virometix AG is a privately held Swiss biotechnology company developing a new generation of fully synthetic vaccines to generate targeted and protective immune responses against infectious diseases and cancer. There is a considerable medical need for vaccines to combat infectious as well as a number of chronic human diseases, including cancer. Rational molecular design, chemical synthesis and Virometix’ proprietary “Synthetic Virus-Like Particle” platform technology allow for the rapid production and optimization of vaccine candidates with the potential to demonstrate superior properties in terms of safety, efficacy, ease and cost of manufacturing and stability. Learn more at www.virometix.com

Forward-Looking Statements

This release contains forward-looking statements regarding the clinical development of V-212. Trial outcomes, timelines, and future steps involve inherent risks and uncertainties.

Contacts

Media & Investor Contact
For further inquiries, please contact:

Virometix AG

Wagistrasse 14

8952 Schlieren, Switzerland

+41 43 433 86 60

Email: press@virometix.com

Veracyte Announces that Decipher-Enabled Biomarker Predicts Hormone Therapy Benefit in Men with Recurrent Prostate Cancer




Veracyte Announces that Decipher-Enabled Biomarker Predicts Hormone Therapy Benefit in Men with Recurrent Prostate Cancer

Findings from the first prospective validation trial for biomarker presented at ASTRO 2025

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–$VYCT #ASTRO25—Veracyte, Inc. (Nasdaq: VCYT), a leading genomic diagnostics company, announced that new data from the prospective, randomized integral biomarker BALANCE trial (NCT03371719) finds that the PAM50 molecular signature predicts which patients with recurrent prostate cancer benefit from hormone therapy with apalutamide in addition to salvage radiation therapy. The prostate PAM50 biomarker is currently available for Research Use Only on the Decipher GRID (Genomic Resource for Intelligent Discovery) research tool.

The new findings were shared today by Daniel Spratt, M.D., University Hospitals Seidman Cancer Center, Case Western Reserve University, in a podium presentation at ASTRO 2025, the annual meeting of the American Society for Radiation Oncology, being held in San Francisco.

“Our findings mark the first time, to my knowledge, that a predictive biomarker has been validated in a prospective, biomarker-driven, randomized trial in non-metastatic prostate cancer,” said Dr. Spratt. “Thus, this is an unprecedented advancement for patients who can be more-precisely selected to receive hormone therapy or forego the treatment and the potential side effects.”

For the study, 295 men with recurrent, non-metastatic prostate cancer following prostate-removal surgery were randomly assigned to salvage radiation therapy with a placebo or apalutamide for 6 months. The PAM50 biomarker was a key stratification variable to ensure each arm had a similar proportion of luminal B and non-luminal B subtypes. They were followed for a median of 5 years during which they were evaluated for biochemical failure, which is a rise in levels of prostate-specific antigen (PSA) post treatment—an early sign of salvage therapy failure. Among the 127 men with luminal B molecular subtype tumors (as determined by the PAM50 signature), 72% of those taking apalutamide did not experience biochemical failure, as compared to the 54% rate in the placebo group [HR 0.45 (80% CI 0.29-0.68), p=0.0062]. In the non-luminal B subset, there was no difference between those taking apalutamide versus placebo (70% vs 71%) [HR 0.95 (80% CI 0.65-1.41), p=0.44].

“These results from NRG GU006 represent the highest level of evidence to support routine biomarker testing in recurrent prostate cancer patients planned to receive secondary radiotherapy,” Dr. Spratt added. “With such a strong difference in the metastasis-free survival response to hormone therapy between luminal B and non-luminal-B tumors, the use of the predictive PAM50 biomarker is a game changer to help personalize treatment for men with recurrent prostate cancer beyond merely prognostic tools.”

The PAM50 signature is the third biomarker—assessed through the whole-transcriptome-based Decipher platform—that a major study has shown predicts benefit from hormone therapy, radiation therapy or chemotherapy. Another trial—PREDICT-RT—recently completed enrollment two years early and is evaluating the Decipher Prostate test’s ability to predict benefit of combined hormone therapy (ADT and apalutamide) concurrent with radiation in patients with high-risk prostate cancer at initial diagnosis.

“Prostate cancer, like all cancers, is a disease of the genome,” said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. “Our Decipher GRID tool uniquely enables researchers to better pinpoint adverse molecular features that are associated with poor outcomes. This can ultimately lead to more-personalized care for each patient based on their tumor’s unique molecular make-up. We are proud to partner with the world’s leading prostate cancer researchers to help uncover insights that can change the trajectory of care for each individual patient and also help deliver the next generation of prostate cancer diagnostics.”

The BALANCE trial results are among 9 Decipher-focused abstracts being presented at the ASTRO 2025 conference. More information can be found here.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients across the full spectrum of prostate cancer. The test is performed on biopsy or surgically resected samples and conveys the aggressiveness of the cancer. For patients with localized or regional prostate cancer, the Decipher score indicates a patient’s risk of metastasis, helping to determine treatment timing and intensity. For patients with metastatic prostate cancer, the Decipher score indicates the likelihood of cancer progression and survival benefit with treatment intensification. Armed with this information, physicians can better personalize their patients’ care. The Decipher Prostate test’s performance and clinical utility has been demonstrated in over 90 studies involving more than 200,000 patients. It is the only gene expression test to achieve “Level I” evidence status and inclusion in the risk-stratification table in the most recent NCCN^® Guidelines* for prostate cancer. More information about the Decipher Prostate test can be found here.

About Veracyte

Veracyte (Nasdaq: VCYT) is a global diagnostics company whose vision is to transform cancer care for patients all over the world. We empower clinicians with the high-value insights they need to guide and assure patients at pivotal moments in the race to diagnose and treat cancer. Our Veracyte Diagnostics Platform delivers high-performing cancer tests that are fueled by broad genomic and clinical data, deep bioinformatic and AI capabilities, and a powerful evidence-generation engine, which ultimately drives durable reimbursement and guideline inclusion for our tests, along with new insights to support continued innovation and pipeline development. For more information, please visit www.veracyte.com or follow us on LinkedIn or X (Twitter).

About Decipher GRID

The Decipher GRID database includes more than 250,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis. More information about Decipher GRID can be found here.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to our statements regarding the use of the predictive PAM50 biomarker to help personalize treatment for men with recurrent prostate cancer beyond merely prognostic tools and the belief that this is a game changer, and expectations that our Decipher GRID tool uniquely enables researchers to better pinpoint adverse molecular features that are associated with poor outcomes; that this can ultimately lead to more-personalized care for each patient based on their tumor’s unique molecular make-up; and that this can help uncover insights that can change the trajectory of care for each individual patient and also help deliver the next generation of prostate cancer diagnostics. Forward-looking statements can be identified by words such as: “appears,” “anticipate,” “intend,” “plan,” “expect,” “believe,” “should,” “may,” “will,” “enable,” “positioned,” “offers,” “designed,” “ultimately,” and similar references to future periods. Actual results may differ materially from those projected or suggested in any forward-looking statements. These statements involve risks and uncertainties, which could cause actual results to differ materially from our predictions, and include, but are not limited to the potential impact the Veracyte Diagnostics Platform can have on scientific advancements in cancer and, in turn, patient care. Additional factors that may impact these forward-looking statements can be found under the caption “Risk Factors” in our Annual Report on Form 10-K filed on February 28, 2025. Copies of these documents, when available, may be found in the Investors section of our website at https://investor.veracyte.com. These forward-looking statements speak only as of the date hereof and, except as required by law, we specifically disclaim any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise.

Veracyte, the Veracyte logo, and Decipher are registered trademarks of Veracyte, Inc., and its subsidiaries in the U.S. and selected countries.

* National Comprehensive Cancer Network. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

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