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Alkermes to Participate in the 46th Annual Goldman Sachs Global Healthcare Conference

On June 2, 2025 Alkermes plc (Nasdaq: ALKS) reported that management will participate in a fireside chat presentation at the Goldman Sachs 46th Annual Global Healthcare Conference on Monday, June 9, 2025 at 10:00 a.m. EDT (3:00 p.m. BST) (Press release, Alkermes, JUN 2, 2025, View Source [SID1234653594]). The live webcast may be accessed under the Investors tab on www.alkermes.com and will be archived for 14 days.

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June 2, 2025: MaaT Pharma Advances Toward Commercialization And Submits Marketing Authorization Application to the European Medicines Agency (EMA) for Xervyteg® (MaaT013) in Acute Graft-versus-Host Disease

On June 2, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported the submission of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead drug candidate MaaT013, under the registered brand name of Xervyteg (Press release, MaaT Pharma, JUN 2, 2025, View Source [SID1234653591]). If approved, the Marketing Authorization would establish Xervyteg as the first microbiota therapeutic approved by the EMA, and the first one globally for a hematology indication. Xervyteg would also be the first approved therapy for the treatment of acute Graft-versus-Host Disease including gastro-intestinal involvement (GI-aGvHD) following 2 prior lines of systemic therapy.

"Submitting our MAA to the EMA marks a major regulatory milestone for MaaT Pharma and a meaningful advancement for patients with refractory aGvHD—a life-threatening complication of stem cell transplantation with no approved therapies," said Hervé Affagard, Co-founder and CEO of MaaT Pharma. "We are now closer to providing a much-needed treatment option and remain deeply committed to advancing immunomodulating microbiota technologies in hemato-oncology, where new solutions are urgently needed."

While advancing toward the commercialization of Xervyteg (if approved) in Europe, MaaT Pharma is also actively exploring strategic partnerships to ensure broad access in timely fashion. The Company has active discussions with experienced partners who share its mission of delivering meaningful advancements to patients.

In parallel to the MAA submission, MaaT Pharma continues to provide access to Xervyteg in Europe and the U.S. through its Early Access Program (EAP)1 for patients with aGvHD and other indications. In 2024, physician demand for Xervyteg under the EAP (n=107) increased by 75% compared to 2023, driven by growing adoption across Europe and in the U.S. In France where the EAP first started, MaaT Pharma has captured 25% of the addressable market on a yearly basis in 2024. Overall, this position reflects the increasing recognition of Xervyteg as a valuable treatment option for GI-aGvHD patients.

aGvHD is the most severe complication of allogeneic stem cell transplantation, a standard-of-care treatment with curative intent offered to patients with blood cancers and some non-malignant hematological conditions. aGvHD refractoriness to current treatments is frequently encountered and severely impacts prognosis. In particular, patients with aGvHD failing both steroid and ruxolitinib typically exhibit a dismal prognosis, with a median survival of 28 days and 85% mortality at one year (Abedin et al 2021). Currently, no therapy is approved for third-line aGvHD, underscoring the urgent need for innovative therapies capable of improving survival and quality of life.

The MAA is supported by positive results from the Pivotal ARES study, a single-arm, open-label, multicenter European study evaluating the efficacy and safety of Xervyteg in GI-aGvHD as third-line therapy in 66 patients. Notably, the study met its primary endpoint, achieving a gastrointestinal overall response rate (GI-ORR) of 62% at Day 28, significantly exceeding the expected 38% response rate, and an overall response rate across all organs of 64% at Day 28. Among responding patients at Day 28, the majority exhibited full resolution of GvHD clinical manifestations (i.e., complete response), an important finding predictive of durable control of aGvHD clinical manifestations over time. The 12-month probability of survival was 54% (vs 15% Abedin et al, 2021). Importantly, patients who exhibited gastrointestinal response at Day 28 had a significantly better probability of survival than non-responders (67% vs 28% respectively, p <0.0001), indicating that Xervyteg-mediated aGvHD control is associated with a remarkable survival benefit. Additional secondary endpoints, including overall survival, will become available in late H2 2025. The Company also integrated supporting safety and efficacy data from 186 aGvHD patients2 treated under its ongoing EAP, which aligns with the positive topline results of the ARES trial and further supports Xervyteg’s strong efficacy and favorable safety profile in aGvHD.

The safety and tolerability of Xervyteg has been monitored by an independent Data Safety Monitoring Board (DSMB). In March 2025, the DSMB reviewed the overall safety of the trial (after all patients completed Day 28 visit or were discontinued earlier) and confirmed that "given the remarkable efficacy results, the study results show an acceptable safety profile and a favourable benefit /risk ratio". The DSMB members will continue to review safety on an ongoing basis until the 1-year follow-up.

The EMA will review the application under the centralized marketing authorization procedure and potentially a marketing authorization could be granted in H2 2026. This centralized procedure means that a single marketing authorization application can be submitted to the EU, and if granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway.

DATROWAY® Continues to Show Promising Tumor Responses as Part of Combination Regimens in Patients with Early and Advanced Non-Small Cell Lung Cancer

On June 1, 2025 Daiichi Sankyo reported results from three trials continue to demonstrate the potential of DATROWAY (datopotamab deruxtecan) in combination with various immunotherapies to improve outcomes in patients with non-small cell lung cancer (NSCLC) across multiple stages of the disease (Press release, Daiichi Sankyo, JUN 2, 2025, View Source [SID1234653562]). These results from TROPION-Lung02, TROPION-Lung04 and NeoCOAST-2 were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (#ASCO25) Annual Meeting.

DATROWAY is a specifically engineered TROP2 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).

"Patients with non-small cell lung cancer have limited treatment options and often experience disease progression due to the aggressive nature of the disease," said Benjamin Levy, MD, Clinical Director, Medical Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Medicine. "The safety and efficacy data from these trials and the exploratory QCS analysis from TROPION-Lung02 support the potential of DATROWAY to become an important medicine to use in combination with various immunotherapies to improve outcomes for patients across multiple stages of lung cancer."

"These data presented at ASCO (Free ASCO Whitepaper) continue to reinforce the potential for DATROWAY to become an important part of immunotherapy-based combination regimens for the treatment of certain patients with non-small cell lung cancer," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "We look forward to further evaluation of these combinations through our robust clinical development program in order to determine how DATROWAY may help address unmet needs of patients with lung cancer."

"The DATROWAY combination data at ASCO (Free ASCO Whitepaper), including results with our own durvalumab and rilvegostomig as well as pembrolizumab, support the combinability of this medicine and its potential to change treatment expectations across stages of lung cancer," said Cristian Massacesi, MD, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca. "Further, the results from the TROPION-Lung02 exploratory biomarker analysis offer additional evidence that the more precise measurement of TROP2, as enabled by our computational pathology platform, can help identify patients with non-small cell lung cancer more likely to respond to DATROWAY."

DATROWAY plus pembrolizumab with or without platinum-based chemotherapy show consistent tumor responses as first-line treatment of advanced NSCLC
Final results from the TROPION-Lung02 phase 1b trial of DATROWAY plus Merck’s (known as MSD outside of the United States and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab) with or without platinum chemotherapy in patients with advanced NSCLC without actionable genomic alterations were featured during an oral presentation (#8501) on Sunday, June 1.In 42 patients receiving first-line doublet DATROWAY plus pembrolizumab, an objective response rate (ORR) of 54.8% (95% confidence interval [CI]: 38.7-70.2) was observed. In 54 patients receiving first-line triplet DATROWAY plus pembrolizumab and platinum chemotherapy, an ORR of 55.6% (95% CI: 41.4-69.1) was observed. This analysis included patients enrolled during the dose escalation phase of the trial, where 4.8% and 40.7% of patients treated with the doublet and triplet regimens, respectively, received DATROWAY at a dose of 4 mg/kg versus 6 mg/kg. Median treatment duration was 9.7 months for patients receiving the doublet regimen and 5.8 months for those receiving the triplet regimen.

The safety profiles of the doublet and triplet regimens of DATROWAY in TROPION-Lung02 were consistent with previous analyses. Grade 3 or higher treatment-related adverse events (TRAEs) occurred in 40.5% and 55.6% of patients receiving the doublet and triplet regimens, respectively. The most common grade 3 or higher TRAEs occurring in 5% or more of patients treated with the doublet regimen were increased amylase (14%) and stomatitis (5%). The most common grade 3 or higher TRAEs occurring in 5% or more of patients treated with the triplet regimen were decreased neutrophil count (15%), neutropenia (13%), anemia (13%), increased amylase (9%), fatigue (6%) and nausea (6%). Two (4.8%) grade 3 interstitial lung disease (ILD) events in patients treated with the doublet regimen and one (1.9%) grade 3 ILD event in patients treated with the triplet regimen were adjudicated as drug-related by an independent committee.

In TROPION-Lung02, patients across six cohorts received DATROWAY plus pembrolizumab (doublet) or DATROWAY plus pembrolizumab and chemotherapy (triplet). As of data cut-off of April 29, 2024, 96 patients received either the doublet (n=42) or triplet (n=54) combination as first-line therapy.

Summary of TROPION-Lung02 First-Line Efficacy Results

Efficacy Measure

Doublet

Triplet

Overall
(n=42)

PD-L1<50%
(n=30)

PD-L1≥50%
(n=5)

Overall
(n=54)

PD-L1<50%
(n=40)

PD-L1≥50%
(n=10)

Confirmed ORR,i,ii %

(95% CI)

54.8%

(38.7–70.2)

53.3%

(34.3–71.7)

100%

(47.8–100)

55.6%

(41.4–69.1)

55%

(38.5-70.7)

60%

(26.2–87.8)

CR, %

2.4%

3.3%

3.7%

2.5%

10%

PR, %

52.4%

50%

100%

51.9%

52.5%

50%

SD, %

33%

PD, %

DCR, % (n)iii

(95% CI)

88.1% (37)

(74.4–96.0)

96.7% (29)

(82.8–99.9)

100% (5)

(47.8–100)

88.9% (48)

(77.4–95.8)

87.5% (35)

(73.2–95.8)

90% (9)

(55.5–99.7)

Median DoR,

(months) (95% CI)

20.1 months

(9.7–NE)

12 months

(8.0–NE)

(5.5–NE)

13.7 months

(5.7–NE)

14.6 months

(5.3–NE)

(4.1–NE)

Median PFS, (months) (95% CI)

11.2 months

(8.2–21.3)

11.1 months

(7.2–13.3)

(8.3–NE)

6.8 months

(5.5–11.1)

6.4 months

(5.5–13.2)

6.8 months

(0.8–NE)

CI, confidence interval; CR, complete response; DCR, disease control rate; DoR, duration of response; NA, not available; NE, not estimable; ORR, objective response rate; PD, progressive disease; PFS, progression-free survival; PR, partial response; SD, stable disease

iORR is CR+ PR

iiAs assessed by investigator

iiiProportion of patients with confirmed CR + confirmed PR + SD.

Tissue samples collected from patients in TROPION-Lung02 were analyzed retrospectively using quantitative continuous scoring (QCS), AstraZeneca’s proprietary computational platform. Tumors were considered biomarker positive if ≥75% of tumor cells exhibited a normalized membrane ratio (NMR) below a predetermined value (≤0.56), indicating a greater proportion of TROP2 in the cytoplasm than on the membrane. Results from this exploratory analysis showed that TROP2-NMR biomarker positivity was associated with a trend toward prolonged progression free survival (PFS) in patients treated with the doublet (hazard ratio [HR]: 0.50; 95% CI: 0.19-1.29) and triplet (HR: 0.67; 95% CI: 0.33-1.36) regimens, and a trend toward prolonged overall survival in patients treated with the doublet (HR: 0.35; 95% CI: 0.07-1.72) and triplet (HR: 0.71; 95% CI: 0.31-1.59) regimens compared to the TROP2-NMR biomarker negative population.

DATROWAY plus rilvegostomig show encouraging activity in first-line treatment of advanced NSCLC
First results from cohort 5 of the TROPION-Lung04 phase 1b trial, presented during a poster session (#8521) on Saturday, May 31, showed DATROWAY plus AstraZeneca’s PD-1/TIGIT bispecific antibody rilvegostomig as a first-line treatment demonstrated an ORR of 57.5% (95% CI: 40.9-73.0), including one complete response (CR) and 22 partial responses (PRs) in 40 patients with advanced or metastatic NSCLC. A disease control rate (DCR) of 95% (95% CI: 83.1-99.4) was seen. Objective responses were observed across both squamous (45.5%; 95% CI: 16.7-76.6) and nonsquamous (62.1%; 95% CI: 42.3-79.3) histologies and all PD-L1 expression levels. Median duration of response (DoR) was 5.8 months (4.5-not evaluable [NE]).

The safety profile of DATROWAY and rilvegostomig was consistent with the known safety profile of each agent with no new safety signals identified. Grade 3 or higher treatment-emergent adverse events (TEAEs) occurred in 60% of patients. The most common grade 3 or greater TEAEs were pneumonia (10%), pneumonitis (7.5%), anemia (2.5%), decreased appetite (2.5%), increased amylase (2.5%), musculoskeletal pain (2.5%), rash (2.5%) and stomatitis (2.5%). Three (7.5%) grade 2 ILD events and two (5%) grade 3 ILD events were adjudicated as drug-related by an independent committee.

Cohort 5 of TROPION-Lung04 enrolled patients with untreated advanced NSCLC without actionable genomic alterations. As of data cut-off of October 24, 2024, 40 patients received DATROWAY plus rilvegostomig with a median treatment duration of 5.1 months. DATROWAY treatment was ongoing in 19 patients and rilvegostomig treatment was ongoing in 20 patients.

Daiichi Sankyo and AstraZeneca are evaluating DATROWAY plus rilvegostomig as first-line treatment for patients with advanced or metastatic nonsquamous NSCLC with PD-L1 ≥50% and without actionable genomic alterations in the TROPION-Lung10 phase 3 trial.

DATROWAY plus durvalumab and chemotherapy demonstrate encouraging pathological response rates in patients with early-stage resectable NSCLC
Final results from Arm 4 of the NeoCOAST-2 phase 2 platform trial evaluating neoadjuvant DATROWAY plus AstraZeneca’s anti-PD-L1 therapy IMFINZI (durvalumab) and single-agent platinum chemotherapy were presented during a poster session (#8046) on Saturday, May 31 and showed the combination demonstrated a pathologic complete response (pCR) rate of 35.2% (95% CI: 22.7-49.4) and a major pathologic response (mPR) rate of 63% (95% CI: 48.7-75.7). This was numerically higher than response rates shown by other combination regimens evaluated in Arm 1 and Arm 2 of NeoCOAST-2; however, the trial was not powered to make direct statistical comparisons between arms. These results, along with results from these other two arms of NeoCOAST-2, were simultaneously published in Nature Medicine.

The safety profile of DATROWAY, durvalumab and single-agent platinum chemotherapy was consistent with the known safety profile of each agent with no new safety signals identified. Feasibility of surgery was maintained with this arm of NeoCOAST-2 relative to the standard of care. Grade 3 or higher TEAEs occurred in 24.1% of patients in the neoadjuvant period.

About TROPION-Lung02
TROPION-Lung02 is an ongoing global, open-label, six-cohort phase 1b trial evaluating the safety and efficacy of DATROWAY at two dose levels (4 mg/kg and 6 mg/kg) in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab; 200 mg) with or without four cycles of platinum chemotherapy (carboplatin or cisplatin) in patients with previously untreated or pretreated locally advanced or metastatic NSCLC without actionable genomic alterations (e.g., EGFR, ALK, ROS1, NTRK, BRAF, RET, MET or other known AGAs). Participants with tumors that harbor KRAS mutations are eligible for this study.

The primary endpoints of TROPION-Lung02 are dose-limiting toxicities and treatment-emergent adverse events. Secondary endpoints include ORR, DoR, PFS as assessed by investigator, overall survival, pharmacokinetics and anti-drug antibodies for DATROWAY and pembrolizumab.

TROPION-Lung02 is one of three clinical trials along with the phase 3 TROPION-Lung07 and TROPION-Lung08 trials in a collaboration and supply agreement between Daiichi Sankyo, AstraZeneca and Merck (known as MSD outside of the United States and Canada) to evaluate the combination of DATROWAY and pembrolizumab.

TROPION-Lung02 enrolled 145 patients in Asia, Europe and North America. For more information visit ClinicalTrials.gov.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About TROPION-Lung04
TROPION-Lung04 is an ongoing global, open-label, 15-cohort phase 1b trial evaluating the safety and efficacy of DATROWAY (4 mg/kg or 6 mg/kg) in combination with immunotherapy (durvalumab, rilvegostomig or volrustomig) with or without up to four cycles of carboplatin in patients with advanced or metastatic NSCLC without actionable genomic alterations. Participants with tumors that harbor KRAS mutations are eligible for this study. Patients enrolled in the cohorts evaluating durvalumab were previously untreated or had received one or fewer lines of systemic chemotherapy without concomitant immunotherapy.

The primary endpoints of TROPION-Lung04 are safety and tolerability. Secondary endpoints include ORR, DCR, duration of response and progression-free survival as assessed by investigator.

Rilvegostomig is AstraZeneca’s PD-1/TIGIT bispecific antibody. The TIGIT component of rilvegostomig is derived from the clinical-stage anti-TIGIT antibody, COM902, developed by Compugen Ltd. (Nasdaq/TASE: CGEN).

TROPION-Lung04 will enroll more than 370 patients in Asia, Europe and North America. For more information visit ClinicalTrials.gov.

About NeoCOAST-2
NeoCOAST-2 is a global, randomized, multicenter, open-label, multi-arm phase 2 platform trial conducted by AstraZeneca evaluating the efficacy and safety of durvalumab in multiple novel combinations, before and after surgery, in patients with resectable, early-stage (stage IIA-IIIB) NSCLC.

The dual primary endpoints of NeoCOAST-2 are antitumor activity of neoadjuvant treatment assessed by pCR and the safety and tolerability of neoadjuvant and adjuvant treatment. Secondary endpoints include event-free survival, disease-free survival and ORR as determined by investigator using RECIST version 1.1, OS, feasibility of surgery, and mPR determined by central blinded independent pathologist review.

NeoCOAST-2 will enroll approximately 630 patients in Asia, Europe and North America. For more information visit ClinicalTrials.gov.

About Non-Small Cell Lung Cancer
Nearly 2.5 million lung cancer cases were diagnosed globally in 2022.1 NSCLC is the most common type of lung cancer, accounting for about 87% of cases.2 While most NSCLC cases are diagnosed in the advanced setting, between 25 to 30% of diagnoses occur in the early stage of the disease.3,4

Despite improvements in treatment for early-stage NSCLC, patients may experience disease recurrence even after complete tumor resection with or without treatment with adjuvant therapy.5,6,7 For patients with advanced NSCLC without actionable genomic alterations, immunotherapy with or without platinum-based chemotherapy is the standard first-line treatment. While these medicines have improved outcomes in the first-line metastatic setting, most patients experience disease progression.8,9,10

TROP2 is a protein broadly expressed in the majority of NSCLC tumors.11 There is currently no TROP2 directed ADC approved in the U.S. for the treatment of lung cancer.

About DATROWAY
DATROWAY (datopotamab deruxtecan; datopotamab deruxtecan-dlnk in the U.S. only) is a TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, DATROWAY is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. DATROWAY is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

DATROWAY is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results from the TROPION-Breast01 trial.

About the DATROWAY Clinical Development Program
A comprehensive global clinical development program is underway with more than 20 trials evaluating the efficacy and safety of DATROWAY across multiple cancers, including NSCLC, triple negative breast cancer and HR positive, HER2 negative breast cancer. The program includes eight phase 3 trials in lung cancer and five phase 3 trials in breast cancer evaluating DATROWAY as a monotherapy and in combination with other anticancer treatments in various settings.

Sanofi to acquire Blueprint Medicines, expanding portfolio in rare immunological disease and adding early-stage pipeline in immunology

On June 2, 2025 Sanofi and Blueprint Medicines Corporation (Blueprint), a US-based, publicly traded biopharmaceutical company specializing in systemic mastocytosis (SM), a rare immunological disease, and other KIT-driven diseases, reported to have entered into an agreement under which Sanofi will acquire Blueprint (Press release, Blueprint Medicines, JUN 2, 2025, View Source [SID1234653560]).

The acquisition includes a rare immunology disease medicine, Ayvakit/Ayvakyt (avapritinib), approved in the US and the EU, and a promising advanced and early-stage immunology pipeline. Furthermore, Blueprint’s established presence among allergists, dermatologists, and immunologists is expected to enhance Sanofi’s growing immunology pipeline.

Ayvakit/Ayvakyt is the only approved medicine for advanced and indolent systemic mastocytosis (ASM & ISM), a rare immunology disease, which is characterized by the accumulation and activation of aberrant mast cells in bone marrow, skin, the gastrointestinal tract, and other organs. The acquisition will also bring elenestinib, a next-generation medicine for SM, as well as BLU-808, a highly selective and potent oral wild-type KIT inhibitor that has the potential to treat a broad range of diseases in immunology.

Under the terms of the acquisition, Sanofi will pay $129.00 per share in cash at closing, representing an equity value of approximately $9.1 billion. Blueprint shareholders also will receive one non-tradeable contingent value right (CVR) which will entitle the holder to receive two potential milestone payments of $2 and $4 per CVR for the achievement, respectively, of future development and regulatory milestones for BLU-808. The total equity value of the transaction, including potential CVR payments, represents approximately $9.5 billion on a fully diluted basis.

Paul Hudson
CEO, Sanofi
"The proposed acquisition of Blueprint Medicines represents a strategic step forward in our rare and immunology portfolios. It enhances our pipeline and accelerates our transformation into the world’s leading immunology company. This acquisition is fully aligned with our strategic intent to strengthen our existing therapeutic areas, to bring relevant and differentiated medicines to patients and to secure attractive returns to our shareholders. It complements recent acquisitions of early-stage medicines that remain our main field of interest. Sanofi still retains a sizeable capacity for further acquisitions. We are excited to welcome Blueprint’s talented people and we look forward to chasing the miracles of science together. This makes sense for science, for both companies, for healthcare professionals, and – most of all – for patients."

Kate Haviland
CEO, Blueprint Medicines
"Since our founding, Blueprint Medicines has worked at the intersection of scientific innovation and operational excellence. I’m incredibly proud of the medical innovations our people have created and delivered to patients. We have translated our unique scientific understanding of mast cell biology into a portfolio of important therapies including Ayvakit – the first and only medicine approved to treat the root cause of systemic mastocytosis – and worked collaboratively with communities to improve standards of care and patient outcomes. With this agreement, we begin our next chapter with Sanofi, whose exceptional leadership in rare disease and immunology and proven ability to solve medical challenges at scale stand to accelerate our joint mission to bring life-changing medicines to many more patients around the world."

Mast cells play an important role in immune responses and are typically found in tissues that encounter the external environment, such as the skin, lungs, and gastrointestinal tract. Upon activation, mast cells release pro-inflammatory molecules such as histamines and proteases. Systemic mastocytosis is a rare immunologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients’ quality of life. The symptoms that patients with SM experience can include anaphylaxis, bone disease, gastrointestinal distress and skin lesions. ISM represents the majority of SM cases.

Ayvakit achieved net revenues of $479 million in 2024 and nearly $150 million in Q1 2025, representing year-on-year growth of more than 60 percent over Q1 2024. The oral medicine is a potent and selective inhibitor of activated KIT and PDGFRA mutant kinases. In certain diseases, mutations in KIT and PDGFRA force protein kinases into an increasingly active state and Ayvakit/Ayvakyt is designed to bind and inhibit these proteins.

Elenestinib is a next-generation, potent and highly selective KIT D816V inhibitor with limited central nervous system penetration. The oral investigational ISM medication is the subject of HARBOR, a phase 2/3 study (clinical study identifier: NCT04910685). The ongoing, randomized, double-blind, placebo-controlled study is designed to evaluate the efficacy and safety of elenestinib plus symptom-directed therapy in patients with ISM and smoldering SM.

BLU-808 is an investigational oral, highly potent and selective wild-type KIT inhibitor that was developed leveraging Blueprint’s expertise in mast cell biology. Wild-type KIT plays a central role in mast cell activation, which is implicated in a broad range of inflammatory diseases.

Transaction terms and financial considerations
Under the terms of the merger agreement, Sanofi will commence a cash tender offer to acquire all outstanding shares of Blueprint for $129.00 per share in cash, reflecting a total equity value of approximately $9.1 billion. In addition, Blueprint’s shareholders will receive one non-tradeable CVR per Blueprint share with two potential milestone payments as follows:

$2 per share, conditioned upon the achievement of a clinical development milestone for BLU-808, and
$4 per share, conditioned upon the achievement of a regulatory milestone for BLU-808.
The upfront offer price represents a premium of approximately 27% over the closing price of Blueprint on May 30, 2025 and a premium of approximately 34% over the 30 trading days volume weighted average price (VWAP) of Blueprint as of May 30, 2025. Together with the CVR, the premium is approximately 33% over the closing price on May 30, 2025 and approximately 40% over the 30 trading days VWAP.

The consummation of the tender offer is subject to customary closing conditions, including the tender of a number of shares of Blueprint common stock representing at least a majority of the outstanding shares of Blueprint common stock, the receipt of required regulatory approvals, and other customary conditions.

If the tender offer is successfully completed, a wholly owned subsidiary of Sanofi will merge with and into Blueprint and all of the outstanding Blueprint shares that are not tendered in the tender offer will be converted into the right to receive the same $129.00 per share in cash and one CVR per share offered to Blueprint shareholders in the tender offer. Sanofi plans to finance the transaction with a combination of cash on hand and proceeds from new debt. The tender offer is not subject to any financing condition. Subject to the satisfaction or waiver of customary closing conditions, Sanofi currently expects to complete the acquisition in the third quarter of 2025. The acquisition will not have a significant impact on Sanofi’s financial guidance for 2025. It is immediately accretive to gross margin and accretive to business operating income and EPS after 2026.

Conference call for investors and analysts
Sanofi will host a conference call for investors and analysts at 08:30 CEST today. A presentation will be available for download from the Investor Relations section of sanofi.com before the conference call starts.

The call will be held on Zoom with the following access details:
View Source
Webinar ID: 979 9146 5119
Passcode: 801394

About Ayvakit
Ayvakit (avapritinib) is the first and only medicine approved by the US Food and Drug Administration (FDA) to treat the root cause of SM. It was FDA approved for the treatment of advanced SM in June 2021 and indolent SM in May 2023. It now is indicated in adults with ISM, adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. The medicine is approved in the EU as Ayvakyt for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. Globally, the medicine is approved for one or more indications in 16 countries, including China where it is marketed by CStone Pharmaceuticals, paying tiered percentage royalties on sales.

Novartis Pluvicto™ demonstrates statistically significant and clinically meaningful rPFS benefit in patients with PSMA-positive metastatic hormone-sensitive prostate cancer

On June 2, 2025 Novartis reported topline results from a pre-specified interim analysis of the Phase III PSMAddition trial. The trial met its primary endpoint with a statistically significant and clinically meaningful benefit in radiographic progression-free survival (rPFS) with a positive trend in overall survival (OS) in patients with prostate-specific membrane antigen (PSMA)-positive metastatic hormone-sensitive prostate cancer (mHSPC) treated with radioligand therapy (RLT), Pluvicto (lutetium (177Lu) vipivotide tetraxetan), in combination with standard of care (SoC) versus SoC alone (Press release, Novartis, JUN 2, 2025, View Source [SID1234653556]). In PSMAddition, the SoC is a combination of androgen receptor pathway inhibitor (ARPI) therapy and androgen deprivation therapy (ADT)3.

Almost all mHSPC patients ultimately progress to metastatic castration-resistant prostate cancer (mCRPC)4. There is a need for additional treatment options with novel mechanisms of action that further delay progression, prolong OS and improve disease control compared to the current SoC, while showing a favorable safety and tolerability profile.

"The progression from metastatic hormone-sensitive prostate cancer to castration-resistant disease remains a formidable challenge that can profoundly impact the survival of patients," said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer at Novartis. "These results further strengthen our confidence in Pluvicto as a PSMA-targeted radioligand therapy. Following the recent FDA approval based on the PSMAfore trial in metastatic castration-resistant prostate cancer, these data suggest using it in an earlier disease setting could advance care and address a significant unmet need for hormone-sensitive prostate cancer patients."

This is the third positive read-out for Pluvicto in a Phase III trial, following the VISION and PSMAfore studies5,6. Results from PSMAddition in mHSPC show potential for treatment in an earlier setting with Pluvicto, which was recently granted US Food and Drug Administration (FDA) approval for earlier use in mCRPC, based on results from PSMAfore1,2. Novartis is harnessing the innovation of world-class scientists, strategic partnerships and one of the industry’s most competitive pipelines to explore the potential of new, targeted therapies and precision medicine platforms to address the greatest unmet needs in prostate cancer.

Data will be presented at an upcoming medical meeting and, based on FDA feedback, will be submitted for regulatory review in the second half of the year.

About PSMAddition study
PSMAddition (NCT04720157) is a Phase III, open-label, prospective, 1:1 randomized study comparing the efficacy and safety of Pluvicto in combination with SoC (ARPI + ADT) vs. SoC alone in adult patients with PSMA-positive mHSPC3. Patients randomized to the SoC alone arm are allowed to crossover to receive Pluvicto, upon confirmation of radiographic progression by blinded independent review committee (BIRC) and per the discretion of the treating physician3. The primary endpoint is rPFS, defined as the time to radiographic progression by PCWG3-modified RECIST V1.1 (as assessed by BIRC) or death3. The key secondary endpoint of OS is defined as time to death due to any cause3.

About Pluvicto (INN: lutetium (177Lu) vipivotide tetraxetan)
Pluvicto is an intravenous RLT that combines a targeting compound (a ligand) with a therapeutic radionuclide (a radioactive particle, in this case lutetium-177)5,7. After administration into the bloodstream, Pluvicto binds to PSMA-expressing target cells, including prostate cancer cells that express PSMA, a transmembrane protein5,7. Once bound, energy emissions from the radioisotope damage the target cells and nearby cells, disrupting their ability to replicate and/or triggering cell death7. Pluvicto is the only PSMA-targeted agent approved for PSMA-positive mCRPC and is the first targeted RLT to demonstrate a clinical benefit for patients with PSMA-positive mHSPC1. Novartis is investigating Pluvicto in earlier stages of disease, including oligometastatic prostate cancer (PSMA-DC, NCT05939414).