Omidubicel (Gamida Cell)

LONDON--(BUSINESS WIRE)--Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a cell therapy pioneer working to turn cells into powerful therapeutics, announced positive interim study results for omidubicel, a stem cell transplant therapy, in treating Severe Aplastic Anemia (SAA). The findings were shared by Dr. Ronit Simantov, Chief Medical and Scientific Officer of Gamida Cell, during a presentation at the 2025 Association for the Advancement of Blood & Biotherapies (AABB) Annual Meeting. Interim results from the study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), were highly encouraging, with 13 of the 14 patients (92.9%) reaching rapid neutrophil recovery, with a median recovery time of 7 days. The disease-free survival rate, as well as overall survival, was 92.3%, comparing favorably with outcomes from patients who receive transplants from matched donors. Additionally, no severe (Grade III–IV) graft-versus-host disease (GvHD) or chronic GvHD was observed, and only 14% of patients experienced moderate (Grade II) GvHD. Dr. Simantov commented: “Gamida Cell has long been dedicated to developing breakthrough therapies for patients with significant unmet medical needs. The results of this study demonstrate omidubicel, with a 92.3% disease-free survival, can offer a real option for patients with severe aplastic anemia who do not have a matched donor, expanding curative transplant access for patients. We are focused on working towards the FDA’s priority review to bring this therapy to patients and their physicians with a PDUFA target date of December 10, 2025.” The open-label, single-center study was designed to evaluate whether a single transplanted omidubicel unit could achieve rapid and durable blood cell recovery in patients with Severe Aplastic Anemia who lacked a matched donor. The study of omidubicel transplantation used a reduced intensity conditioning regimen and included 14 patients (median age 22 years) whose diseases had not responded to standard therapies. This study, supplemented with additional data, forms the basis for the company’s submission to the U.S. Food and Drug Administration (FDA) for approval with a Prescription Drug User Fee Act (“PDUFA”) target action date of December 10, 2025. About Severe Aplastic Anemia Severe Aplastic Anemia (SAA) is a rare, life-threatening hematologic disease characterized by bone marrow failure and low circulating blood cells. Bone marrow stem cell transplant may be curative for SAA, but there is a significant unmet need for patients who do not have an available matched sibling donor. Gamida Cell is advancing omidubicel to help bridge that gap. About Ayrmid Ltd. and Gamida Cell Ayrmid Ltd. is the parent company of Gamida Cell Inc., a cell therapy pioneer working to turn cells into powerful therapeutics. Gamida Cell Inc. currently has two FDA approved products on the market in the US, namely Omisirge (please see the current full Prescribing Information, including boxed warning, here) and APHEXDA (please see the current full Prescribing Information here). Gamida Cell operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram.

Ayrmid's new offer values bluebird bio at about $45 million upfront. A rival bidder has emerged to acquire struggling gene therapy specialist bluebird bio.Ayrmid has offered to buy bluebird for $4.50 apiece upfront plus a one-time contingent value right (CVR) of $6.84 per share tied to a sales milestone, bluebird said Friday.The upfront tag is 50% higher than the $3-per-share selling price bluebird has previously penned with Carlyle and SK Capital Partners. That private equity duo’s buyout offer also includes a $6.84-per-share CVR.For now, bluebird’s board has not changed its mind, and the company remains bound by the original merger agreement. But it’s willing to look at the new unsolicited nonbinding written proposal.“Consistent with its fiduciary duties, the bluebird Board of Directors is carefully reviewing the Ayrmid proposal in consultation with its legal and financial advisors,” the Massachusetts biopharma said Friday. A higher price would be good news for bluebird, as the Carlyle-SK offer valued the biotech at a discount, which highlights the challenge the company has faced in commercializing its gene therapies. Bluebird’s cheap sale cast a dark shadow over the entire gene therapy field, which is undergoing an industrywide reckoning.Per bluebird’s merger agreement with the private equity group, the company’s board, on the condition of not breaching its non-solicitation obligations, may withdraw its recommendation for the deal if a more attractive offer emerges. In that case, bluebird’s board must first provide the Carlyle-SK group a right to make counterproposals, according to a securities filing.If bluebird eventually endorses a rival bid, it must pay a termination fee of $1.5 million, which the Ayrmid offer would more than cover. While the original deal values bluebird at about $29 million, the new offer is worth about $45 million upfront.According to bluebird, it had previously approached Ayrmid to gauge its interest in a takeover, but Ayrmid didn’t submit any proposal to bluebird as part of that process. U.K. entity Ayrmid is known as the parent company of Gamida Cell, which is a cell therapy company using a nicotinamide technology to create allogeneic cell products for blood cancers. Gamida sells Omisirge, an FDA-approved allogeneic hematopoietic progenitor cell therapy for use in patients 12 years and older with hematologic malignancies who are set to receive an umbilical cord blood transplantation.