On February 21, 2025 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird") reported that it has entered into a definitive agreement to be acquired by funds managed by global investment firms Carlyle (NASDAQ: CG) and SK Capital Partners, LP ("SK Capital") in collaboration with a team of highly experienced biotech executives (Press release, bluebird bio, FEB 21, 2025, View Source [SID1234650448]). David Meek, former CEO of Mirati Therapeutics and Ipsen, is expected to become CEO of bluebird upon closing. Carlyle and SK Capital will provide bluebird primary capital to scale bluebird’s commercial delivery of gene therapies for patients with sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy.

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Under the terms of the agreement, bluebird stockholders will receive $3.00 per share in cash and a contingent value right per share, entitling the holder to a payment of $6.84 in cash per contingent value right if bluebird’s current product portfolio achieves $600 million in net sales in any trailing 12-month period prior to or ending on December 31, 2027, for a potential total value of up to $9.84 per share in cash, subject to the tender of a majority of the outstanding shares of bluebird, receipt of applicable regulatory approvals, and other customary closing conditions. bluebird’s Board of Directors (the "bluebird Board") unanimously approved the agreement and recommends that stockholders tender their shares. Following a comprehensive review of bluebird’s strategic alternatives, including meeting with more than 70 potential investors and partners over a period of five months, and a third and final denial by the Federal Drug Administration of bluebird’s appeal for a priority review voucher, the bluebird Board determined that, absent a significant infusion of capital, bluebird is at risk of defaulting on its loan covenants. The bluebird Board has decided that this transaction is the only viable solution to generate value for stockholders. Additional details on the process will be available in bluebird’s Solicitation/Recommendation Statement on Schedule 14D-9, which will be filed with the U.S. Securities and Exchange Commission ("SEC").

"For more than a decade, bluebird has been at the forefront of gene therapy, delivering groundbreaking treatments to patients facing life-threatening genetic diseases," said Andrew Obenshain, current CEO of bluebird. "However, as our financial challenges mounted, it became clear that securing the right strategic partner was critical to maximizing value for our stockholders and ensuring the long-term future of our therapies. After an extensive review process, this acquisition represents the best path forward – maximizing value for stockholders and bringing significant capital, commercial expertise, and a commitment to provide more patients the opportunity to benefit from potentially transformative gene therapies."

David Meek commented, "bluebird is built on an extraordinary legacy of scientific breakthroughs, and we are committed to unlocking its full potential for patients. With the backing of Carlyle and SK Capital, we will bring the capital and commercial capabilities needed to accelerate and expand patient access to bluebird’s life-changing gene therapies."

"Carlyle’s healthcare and Abingworth teams have significant experience investing in biopharma and are excited about what lies ahead for bluebird. We look forward to working with David and SK Capital to drive bluebird’s future growth and mission of delivering its therapies to improve patient outcomes," said Joe Bress, Carlyle Partner and Global Co-Head of Healthcare. Bali Muralidhar, Partner and Chief Investment Officer & COO of Abingworth, Carlyle’s life sciences investment franchise, added, "Over the past decade, we have tracked and been impressed by bluebird’s success in researching and developing breakthrough gene therapies for large, unmet medical needs. Joining forces with Carlyle enables us to collaborate in supporting companies like bluebird in commercializing their innovations for patients."

Aaron Davenport, Managing Director at SK Capital, commented, "SK Capital has deep experience in the life sciences sector. We have long admired bluebird’s scientific leadership, dedicated focus on severe genetic diseases, and track record of successful product development and launch. We are excited to partner with David and Carlyle to invest in and accelerate the delivery of bluebird’s pioneering gene therapies to needing patients."

Transaction Details

Under the terms of the agreement, bluebird stockholders will receive $3.00 per share in cash and a contingent value right per share, entitling the holder to a payment of $6.84 in cash per contingent value right if bluebird’s current product portfolio achieves $600 million in net sales in any trailing 12-month period prior to or ending on December 31, 2027.

The transaction is expected to close in the first half of 2025, subject to the tender of a majority of the outstanding shares of bluebird, receipt of applicable regulatory approvals, and other customary closing conditions. bluebird has also entered into amendments to its loan agreement with Hercules Capital, Inc. to facilitate adequate liquidity to position it to maintain operations through the closing.

Upon completion of the transaction, bluebird will become a privately held company, and shares of bluebird common stock will no longer be listed on any public market.

Leerink Partners is acting as bluebird’s financial advisor, and Latham & Watkins LLP is serving as legal counsel to bluebird. Bourne Partners is acting as financial advisor to Carlyle and SK Capital, and Wachtell, Lipton, Rosen & Katz, Kirkland & Ellis LLP, and Orrick, Herrington & Sutcliffe are serving as legal advisors to Carlyle and SK Capital.

On February 20, 2025 Oxcia reported that the FDA (the United States Food and Drug Administration ) has granted ODD (orphan drug designation) to OXC-101 as a potential treatment option for patients with AML (acute myeloid leukemia) (Press release, Oxcia, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=oxc-101-has-been-granted-odd-status-by-the-fda-in-aml [SID1234650416]).

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OXC-101 is Oxcia’s lead clinical candidate, a first-in class mitotic MTH1 inhibitor with a unique dual mechanism of action. In short, OXC-101, fights cancer by taking advantage of one of the Achilles heels of cancer cells – the high endogenous oxidative stress and DNA damage. In preclinical studies, OXC-101 significantly prolongs survival and reduces tumor growth in AML disease models.

Clinical Phase 1 data in advanced hematological cancer patients is reassuring. Oxcia is now performing an expansion trial, a phase 1/2 study in a subpopulation (relapsed/recurrent AML), in combination with one of the standard treatments, idarubicin. The aim is to confirm the preliminary efficacy previously shown in AML to support further clinical development in a pivotal phase 2 trial that can be the base for regulatory fast-track approval.

Oxcia AB was in the beginning of 2023 awarded a grant of 3 million SEK by Swelife and Medtech4Health for this study. To ensure the recruitment of patients with this rare AML disease in a cost- and time-efficient manner, we are now adding additional clinical sites. Rigshospitalet in Denmark will be enrolled shortly. In addition, Bulgaria and Serbia will be added during 2025.

"We are delighted with the FDA’s decision to grant orphan drug designation to OXC-101 for AML. This is a significant milestone and underscores the significant unmet need for novel medicines and the unique approach by OXC-101. We believe OXC-101 holds potential to greatly improve treatment for patients who suffer from AML", says Ulrika Warpman Berglund, Oxcia’s CEO. We are presently preparing an application for ODD also with EMA (European Medicines Agency). Early engagement with the FDA and EMA is pivotal.

In the study we are also integrating precision medicine screening to help identify responders/non-responders. Precision medicine screening is a novel way of personalizing treatment for patients and thus identifying the best treatment strategy for individuals reducing unsuccessful treatments with unnecessary suffering and lack of effect.

Briefly about AML

Acute myeloid leukemia (AML) is an aggressive blood cancer associated with infection, anemia and bleeding. It is the most common acute leukemia in adults, accounting for more than 80% of the cases. In 2021, there were 162,200 incident cases of AML worldwide, and the number is forecasted to increase to 169,000 incident cases by 2027 (Datamonitor). Even though the overall survival rate among AML patients has been increasing in the past decades, it remains poor with an overall 5-year survival rate of 20% in selected subpopulations.

AML is a very heterogenous disease, which explains why classical intensive chemotherapy, constituting of a cytarabine /anthracycline backbone, remains the standard of care (SoC). The medical need is high. Although we have seen recent approvals for biomarker led AML subsets, the majority of patients are still in need of well-tolerated and effective novel therapies.

Briefly about Orphan Drug Designation

The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent a life-threatening rare disease or condition that affects fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

On February 20, 2025 OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage biotechnology company advancing immunotherapies and targeted drug conjugates for cancer treatment, reported that it received a Notice of Allowance from the United States Patent & Trademark Office (USPTO) that a patent will be issued covering the manufacturing methods required for the OST-HER2 commercial product (Press release, OS Therapies, FEB 20, 2025, View Source [SID1234650434]). The USPTO granted a Patent Term Adjustment of 572 days, providing market exclusivity for the OST-HER2 commercial drug product into 2040. OS Therapies is preparing to initiate discussions with the United States Food & Drug Administration (FDA) following the successful treatment phase of its Phase 2b clinical trial in the prevention of recurrent, resected, lung metastatic osteosarcoma with a view towards submitting a Biologics Licensing Application (BLA) and gaining conditional or accelerated FDA approval in 2025. The Company recently completed a $7.1 million financing in January 2025 and has sufficient capital into mid-2026.

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OST-HER2 has already received rare pediatric disease (RPDD), fast-track (FTD) and orphan drug (ODD) designations from the US FDA for osteosarcoma. The Company intends to (1) focus on gaining FDA BLA approval for OST-HER2 in osteosarcoma in late 2025, (2) sell the Priority Review Voucher (PRV) it would receive from the FDA BLA approval prior to the September 30, 2026 PRV deadline to a larger pharmaceutical company at prevailing market prices (most recent PRV sale transaction was $150 million), (3) commercialize OST-HER2 in osteosarcoma and (4) then expand the clinical development of OST-HER2 into breast cancer and other larger solid tumor indications for additional revenue potential.

OST-HER2 has successfully completed a Phase 1 trial in adult patients with HER2 overexpressing cancers, primarily breast cancer patients. Preclinical breast cancer results with OST-HER2 showed:

78% reduction in tumor size (3mm for OST-HER2 treated vs. 14mm for control arm) in FVB/N HER2 transgenic mouse model of breast cancer treatment at day 75
33% prevention of breast cancer in OST-HER2 treated mice vs. 0% prevention of breast cancer in FVB/N HER2 transgenic model of breast cancer prevention at week 50
20% reduction of tumor size for OST-HER2 plus HER2-targeted antibody vs. HER2-targeted antibody alone Tg tumor regression model of breast cancer at day 42
65% reduction cellular concentration of metastatic cells for OST-HER2-treated mice compared with controls in brain metastasis model of primary breast cancer
The osteosarcoma treatment market was estimated at $1.2 billion in 2022 according to Data Bridge Market Research. The Company believes the market opportunity for OST-HER2 in the prevention of lung metastases in osteosarcoma is over $500 million. The breast cancer treatment market was estimated at $29.2 billion in 2023 and expected to grow to $53.7 billion by 2030 according to Grandview Research. The Company believes the market opportunity for OST-HER2 in the treatment of breast cancer exceeds $1 billion.

On February 20, 2025 Oxford Vacmedix (OVM) reported the first patient being treated with new extended dosing of OVM-200, at the prestigious Sarah Cannon Research Institute in London (Press release, Oxford Vacmedix, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=first-patient-ovm-200-cancer-vaccine-extended-dosing [SID1234650417]). The extended dosing protocol was first suggested by the clinical investigators in the trial, following the excellent safety record seen in Phase 1a. The new regime will allow up to 11 vaccinations of OVM-200 over a six-month period and has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA). OVM-200 is a new therapeutic cancer vaccine developed using OVM’s novel recombinant overlapping peptide (ROP) platform. It targets survivin, a protein overexpressed by cancer cells, which prevents them being attacked by the body’s immune system.

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The Phase I trial of OVM-200 is focused on safety and on establishing an immune response in patients with three tumour types – non small cell lung cancer (NSCLC), prostate cancer and ovarian cancer. It is being run at four sites in the UK including the Sarah Cannon Institute and University College Hospital (UCH) in London, the Churchill hospital of the Oxford University Hospitals Foundation Trust (OUHFT) and the Christie NHS Foundation Trust in Manchester. The first part of the trial, Phase 1a, has been completed and has shown both excellent safety and a strong immune response. The Chief Investigator for the trial is Professor Martin Forster, based at UCH. This trial is both the first time OVM-200 has been used in people and also the first time any ROP-based vaccine has been tested in the clinic.

William Finch, CEO of Oxford Vacmedix, said:

The ROP technology has been developed from an initial concept in the laboratory to now being tested as a treatment for critically ill patients. We see the potential benefits of a vaccination approach both in stimulating the body’s immune system to attack the cancer and also, in future trials, enhancing the efficacy of other immune oncology agents. This Phase I trial is a first step towards having effective cancer vaccines.

Dr Anja Williams Principal Investigator at the Sarah Cannon Research Institute UK, added:

It is a privilege to work with Oxford Vacmedix on this innovative vaccine programme for patients with lung, prostate, and ovarian cancer. We are very pleased with the results to date and strongly believe that vaccine treatments will play a major role in future cancer treatments. Extending the dosing will maximise the potential benefits of the vaccine.

On February 20, 2025 Incyte (Nasdaq:INCY) and Genesis Therapeutics, Inc. reported that the companies have entered into a strategic collaboration focused on the research, discovery and development of novel small molecule medicines, with an initial focus on collaboration targets selected by Incyte (Press release, Incyte, FEB 20, 2025, View Source [SID1234650435]).

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Genesis is pioneering generative and predictive artificial intelligence (AI) technologies to help create therapeutics for challenging targets. Utilizing Genesis’ proprietary AI platform, GEMS (Genesis Exploration of Molecular Space), the partnership will pursue the discovery and optimization of small molecule compounds for the collaboration targets. Incyte is granted exclusive rights for potential clinical development and commercialization of collaboration products.

"As a leader in pharmaceutical innovation, Incyte is continually seeking new technologies that can transform how new medicines are discovered and developed," said Pablo J. Cagnoni, M.D., President and Head of Research and Development at Incyte. "Partnering with Genesis Therapeutics presents a unique opportunity to leverage their AI technologies to accelerate the discovery of breakthrough small molecules for high-impact targets in our pipeline."

"AI has the potential to redefine how we discover small molecule medicines, and our team is at the forefront of this revolution," said Evan Feinberg, Ph.D., Founder and Chief Executive Officer of Genesis. "We are pleased to establish this world-class partnership to combine our GEMS AI platform with Incyte’s deep expertise and track record in drug discovery and development, with the shared goal of advancing critical treatments for patients with severe diseases."

Terms of the Agreement

Under the terms of the agreement, Genesis will receive an upfront payment of $30 million. Genesis and Incyte have agreed to collaborate on two initial targets, and Incyte will have the option to nominate an additional target for a predetermined fee. If all milestones are achieved, Genesis is eligible to receive up to $295 million in development, regulatory and commercial milestone payments per target. Genesis is also eligible to receive tiered royalties on sales of any collaboration products, once approved.