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bluebird bio Announces Completion of Acquisition by Carlyle and SK Capital

On June 2, 2025 bluebird bio (NASDAQ: BLUE) ("bluebird"), a pioneer in gene therapies for severe genetic diseases, reported the completion of its sale to funds managed by global investment firms Carlyle (NASDAQ: CG) and SK Capital Partners, LP ("SK Capital") (Press release, bluebird bio, JUN 2, 2025, View Source [SID1234653597]). With the closing of the transaction, bluebird’s common stock has ceased trading and will no longer be publicly listed. Carlyle and SK Capital have provided significant primary capital to support and scale bluebird’s commercial delivery of gene therapies for patients with sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy.

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David Meek, who became Chief Executive Officer of bluebird at close, said, "Today marks the beginning of a new era for bluebird as its go-forward financial backing and leadership team will better enable all stakeholders to realize the full potential of our revolutionary therapies. Historically, bluebird has excelled as a scientific innovator and should be very proud of the many achievements it has delivered to patients. Our vision is to further that legacy of scientific excellence while improving the commercial execution of our approved products to rapidly expand access to lifechanging gene therapies."

"We are excited to back bluebird in partnership with SK Capital. We believe providing bluebird the necessary funding along with the new leadership team will help bluebird realize its full potential," said Joe Bress, Carlyle Partner and Global Co-Head of Healthcare. Bali Muralidhar, Co-Managing Partner and Chief Investment Officer & COO of Abingworth, Carlyle’s life sciences investment franchise, added, "There is an incredible opportunity to bring bluebird’s groundbreaking therapies to more patients in need, and we look forward to advancing bluebird in its mission."

"SK Capital looks forward to partnering with David and his team as well as Carlyle to scale bluebird’s pioneering gene therapies that can make a lifechanging difference for patients around the world," said Aaron Davenport, Managing Director at SK Capital, adding, "We believe our deep collective experience in manufacturing and commercializing therapies can help drive the next chapter of bluebird’s growth."

Incoming Team Bolsters Commercial Gene Therapy Experience

The company’s momentum is reinforced by a deeply experienced management team, led by CEO David Meek. David brings more than 30 years of leadership in life sciences, including as CEO of Mirati Therapeutics and Ipsen. David is joined by Tom Klima as Chief Commercial & Operating Officer, Debasish Roychowdhury, M.D., as Chief Medical Officer, Wendy DiCicco as Chief Financial Officer, and Ellen Forest as Chief People Officer. Additional details are available at View Source

From Scientific Breakthroughs to Delivery at Scale

With the transaction now closed, bluebird is prioritizing expanding its manufacturing infrastructure, streamlining the patient journey, supporting treatment centers, and strengthening its payer partnerships. The acquisition provides the strategic and financial backing needed to meet rising demand and drive commercial and operational excellence across the organization.

"bluebird has demonstrated what’s possible through effective gene therapy," David added. "Now we will build the ecosystem to ensure every patient who needs these therapies can access them."

BioNTech and Bristol Myers Squibb Announce Global Strategic Partnership to Co-Develop and Co-Commercialize Next-generation Bispecific Antibody Candidate BNT327 Broadly for Multiple Solid Tumor Types

On June 2, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech") and Bristol Myers Squibb (NYSE: BMY, "BMS") reported that the companies have entered into an agreement for the global co-development and co-commercialization of BioNTech’s investigational bispecific antibody BNT327 across numerous solid tumor types (Press release, BioNTech, JUN 2, 2025, View Source [SID1234653596]). Under the agreement, BioNTech and BMS will work jointly to broaden and accelerate the development of this clinical candidate.

BioNTech’s BNT327, a next-generation bispecific antibody candidate targeting PD-L1 and VEGF-A, is currently being evaluated in multiple ongoing trials with more than 1,000 patients treated to date, including global Phase 3 trials with registrational potential evaluating BNT327 as first-line treatment in extensive stage small cell lung cancer ("ES-SCLC") and non-small cell lung cancer ("NSCLC"). A global Phase 3 trial evaluating the candidate in triple negative breast cancer ("TNBC") is planned to start by the end of 2025. Preliminary data from ongoing trials underscore the potential for combining anti-PD-L1 and anti-VEGF-A – two well-established therapeutic targets – into a single molecule to deliver synergistic clinical benefits for patients across multiple tumor types.

Under the terms of the agreement, the companies will jointly develop and commercialize BNT327, including the development of BNT327 as monotherapy and in combination with other products. Both companies have the right to independently develop BNT327 in further indications and combinations, including combinations of BNT327 with proprietary pipeline assets.

"We believe BNT327 has the potential to become a foundational immuno-oncology backbone, moving beyond single-mechanism checkpoint inhibitors and expanding into multiple solid-tumor indications. Our collaboration with BMS, a pioneering leader in immuno-oncology, aims to accelerate and broadly expand BNT327’s development to fully realize its potential," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "Our focus remains on advancing high-impact, pan-tumor programs and combination strategies in oncology, with BNT327 complementing our antibody-drug conjugate programs and mRNA-based immunotherapies. We are dedicated to delivering truly transformative options for patients in need."

"Our deep experience and expertise in advancing and delivering groundbreaking immuno-oncology medicines positions BMS well to collaboratively realize the potential of BNT327, an asset with significant potential for transforming the standard of care for patients with solid tumors," said Christopher Boerner, Ph.D., Board Chair and CEO of Bristol Myers Squibb. "The science behind BNT327 and its leading clinical position in multiple hard-to-treat tumor types, further bolsters our pursuit of novel mechanisms and multiple modalities in oncology, and enhances our growth trajectory. We are impressed by the innovation that BioNTech has achieved to date, and we look forward to partnering to accelerate existing clinical trials and time to market, while expanding the number of potential indications."

BMS will pay BioNTech $1.5 billion in an upfront payment and $2 billion total in non-contingent anniversary payments through 2028. These tax-deductible charges will be recorded as Acquired IPR&D Expense when incurred, with the $1.5 billion being incurred in Q2. In addition, BioNTech will be eligible to receive up to $7.6 billion in additional development, regulatory and commercial milestones. BioNTech and BMS will share joint development and manufacturing costs on a 50:50 basis, subject to certain exceptions. Global profits/losses will be equally shared between BioNTech and BMS.

About BNT327
BNT327 is a novel investigational bispecific antibody combining two complementary, validated mechanisms in oncology into one single molecule. BNT327 combines PD-L1 checkpoint inhibition aimed at restoring T cells’ ability to recognize and destroy tumor cells with the neutralization of VEGF-A. The blocking of VEGF-A is aimed at reversing the tumor’s immuno-suppressive effect in its microenvironment and cutting off the blood and oxygen supply that feeds tumor cells (anti-angiogenesis effect), with the intention of preventing the tumor from growing and proliferating. BNT327 may be differentiated via its mechanism of action of targeting PD-L1 on tumor cells to localize anti-VEGF activity within the tumor microenvironment, aiming to enhance therapeutic precision and minimize systemic exposure. A treatment with BNT327 is intended to help normalize blood vessels at the tumor site, improving delivery and potential effectiveness of combination therapies. This targeted vascular remodeling positions BNT327 as a potential backbone therapy across a wide range of solid tumors.1,2

More than 1,000 patients have been treated with BNT327 in clinical trials to date. More than 20 clinical trials are currently ongoing or planned to evaluate BNT327 either as a monotherapy or in combination with other treatment modalities targeting different oncogenic pathways in more than 10 solid tumor indications. Multiple global trials are ongoing or planned to start in 2025, including three global clinical trials with registrational potential in first-line small cell lung cancer ("SCLC"), first-line non-small cell lung cancer ("NSCLC") and first-line triple-negative breast cancer ("TNBC"). Additional trials will explore combining BNT327 and BioNTech’s proprietary antibody-drug conjugate candidates ("ADCs"). If successfully developed and approved, BioNTech aims to use this bispecific antibody candidate as a next-generation immuno-oncology ("IO") backbone in combination with other treatment modalities targeting a broad range of cancer indications.

Anixa Biosciences Announces Completion of Enrollment in Phase 1 Trial of Breast Cancer Vaccine

On June 2, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported the completion of enrollment in the Phase 1 clinical trial of its breast cancer vaccine (Press release, Anixa Biosciences, JUN 2, 2025, View Source [SID1234653595]). This novel vaccine, invented at Cleveland Clinic, is also being developed in partnership with Cleveland Clinic and the Phase 1 trial is fully funded by a grant from the U.S. Department of Defense (DoD).

The vaccine is designed to stimulate the immune system to target breast cancer before it can recur or develop. A total of 35 women have received the vaccine in the trial, including 26 in the triple-negative breast cancer ("TNBC") group, four in the prevention group, and five in the pembrolizumab group. These participants represent three distinct patient cohorts:

TNBC Group: Women who have completed treatment for triple-negative breast cancer, are currently cancer-free, and are at risk of recurrence.
Prevention Group: Women who are cancer-free but carry genetic mutations that place them at high risk of developing breast cancer and have elected to undergo preventative mastectomy to lower their risk.
Pembrolizumab (Keytruda) Group: Women who are receiving pembrolizumab in a post-operative setting. These women are receiving the vaccine concurrently with pembrolizumab.
The final patient visits are scheduled for August 2025. Once completed, the final study report will be submitted to the Department of Defense. A Clinical Study Report (CSR) will then be prepared for submission to the U.S. Food and Drug Administration. As part of the continued development process, the Investigational New Drug (IND) application will be transferred from Cleveland Clinic to Anixa.

Anixa and Cleveland Clinic plan to submit for presentation all trial data at a major upcoming scientific meeting.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, commented, "We are very encouraged by the data we are seeing from this trial. Preliminary results show that our breast cancer vaccine is well tolerated, with more than 70% of patients demonstrating protocol-defined immune responses. These encouraging findings are guiding our planning for Phase 2 trials, which will include discussions with the FDA, protocol development, manufacturing, and clinical site selection. While cancer vaccines have traditionally faced significant hurdles, our approach is aimed at a novel target that has not been previously explored in this context. We believe this could represent a new paradigm in immuno-oncology. The breast cancer market, particularly for triple-negative breast cancer and genetically high-risk populations, continues to face a major unmet need. Our vaccine may offer a unique, immunologic pathway for both prevention and treatment."

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.

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.