Author: Ayrmid Pharma Ltd

Motixafortide alone and in combination with natalizumab can support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle

Key Highlights

• Motixafortide alone and in combination with natalizumab (VLA-4 inhibitor) led to robust hematopoietic stem cell (HSC) mobilization predicting 16.9 million and 19.6 million cells/kg collected in a single apheresis session.

• Motixafortide alone and in combination with natalizumab mobilized 2.7 and 2.8 fold greater HSCs, relative to plerixafor

• Results support the use of motixafortide as an effective single-agent mobilizer of HSCs for people with sickle cell disease enabling accelerated access to gene therapies

• Data from study shows that motixafortide was safe and well tolerated

• Findings presented at ASH 2025

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SOURCE: Ayrmid Pharma Ltd

View the original press release on ACCESS Newswire

New data presented at the 2025 ASH demonstrates 94% disease free and overall survival rate

Omisirge approved by FDA as First Cell Therapy to Treat Severe Aplastic Anemia

Key Highlights of Data Presented at ASH:

• 95% of patients achieved rapid neutrophil recovery

• Median time to neutrophil recovery: 8 days

• Immune recovery post-transplant was rapid

• 94% disease-free and overall survival

• No cases of BMT-CTN severe acute graft-versus-host disease (GVHD), or chronic GVHD observed

• Omisirge approved by the FDA on December 5, 2025 for SAA

DUBLIN, IRELAND AND LONDON, UK = / ACCESS Newswire / December 8, 2025 / Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a leader in cell therapy innovation today announced further positive interim clinical results for Omisirge (Omidubicel-onlv), its advanced stem cell transplant therapy for Severe Aplastic Anemia (SAA). The data were presented by Dr. George Aue of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), at the American Society of Hematology (ASH) 2025 Annual Meeting, taking place from Dec 6-9 in Orlando FL.

The ongoing open-label, single-center study (NCT03173937|17-H-0091), led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), demonstrated highly encouraging results. Among 19 patients (median age 20 years) whose disease had not responded to standard therapies, 18 (95%) achieved rapid neutrophil recovery with a median time of 8 days. Both disease-free survival and overall survival were 94%. Importantly, only 16% of patients experienced (BMT-CTN Grade II) acute GvHD and no cases of severe (BMT-CTN Grade III-IV) acute GVHD or chronic GVHD were observed.

Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH) commented: “The results of the ongoing study are extremely encouraging and indicate a significant advancement in the treatment options for patients with a high unmet medical need. The SAA patients in the study were high risk but had significantly better than expected outcomes with remarkably fast and high rates of neutrophil engraftment. This was achieved with low rates of mild acute GVHD and no chronic GVHD, meaning patients experienced a rapid return to normal life.”

Dr. Ronit Simantov, Chief Medical and Scientific Officer of Ayrmid commented: “These results highlight the significant potential of Omisirge as a transplant option for patients with SAA given the rapid and sustained recovery of blood counts observed. Omisirge potentially expands transplant access for patients who otherwise have limited options. We remain deeply committed to advancing transformative therapies for patients with serious unmet medical needs.”

Omisirge is now approved for treatment in adults and pediatric patients 6 years of age and older with SAA following reduced intensity conditioning. The FDA approval of Omisirge was based on data from the 17-H-0091 study.

About Severe Aplastic Anemia

Severe Aplastic Anemia is a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. Stem cell transplantation offers a potential cure; however, many patients lack a matched sibling donor. Ayrmid is advancing therapies to address this unmet medical need.

About Ayrmid Ltd. and Gamida Cell

Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed 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.

Contacts: Media, Investors / Business Development: bd@ayrmid.com

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit www.rns.com.

SOURCE: Ayrmid Pharma Ltd

View the original press release on ACCESS Newswire

Key Highlights

• Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies

• 90% (9 of 10) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing

• 86% (6 of 7) of patients had previously failed to collect sufficient cells with plerixafor

• Results support the use of motixafortide as an effective single-agent mobilizer of HSC for people with sickle cell disease for gene therapies

• Findings presented at ASH 2025

LONDON, UK / ACCESS Newswire / December 6, 2025 / Ayrmid, Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., today announced encouraging real-world data on the use of motixafortide, a long-acting CXCR4 inhibitor licensed by Ayrmid under the brand name APHEXDA^®, for mobilizing HSCs in patients with sickle cell disease undergoing gene therapy. The results were presented at the 2025 American Society of Hematology (ASH) Annual Meeting, taking place from Dec 6-9 in Orlando FL.

Motixafortide is currently FDA approved in combination with filgrastim (G-CSF) for stem cell mobilization in multiple myeloma. The product’s effectiveness and pharmacologic profile have prompted growing interest in its potential use for sickle cell disease, where collecting adequate stem cells remains a significant barrier to gene therapy. Many patients do not mobilize enough cells with standard approaches, limiting access to curative treatment.

Researchers from five treatment centers evaluated the real-world use of motixafortide in ten patients aged 14-50. Each patient underwent one or more collection cycles over two or more days to obtain the number of stem cells required for gene therapy manufacturing.

Among the seven patients who previously failed plerixafor, motixafortide led to a substantial improvement in stem cell mobilization, enabling six of the seven to successfully collect enough cells to progress to gene therapy manufacturing. Manufacturing or infusion of a gene therapy product is underway, with two patients already transplanted and demonstrating appropriate engraftment.

Dr. John Manis, Director Transfusion Medicine Service, Associate Professor of Pathology, Harvard Medical School, commented: “The approval of gene therapies for sickle cell disease has opened the door to transformative outcomes, but inadequate stem cell collection has significantly delayed manufacturing and prevented some patients from starting treatment. These findings suggest that motixafortide may help overcome this challenge, and with more studies, expand access to potentially life-changing therapies.”

About Sickle Cell Disease
Sickle cell disease, also known as sickle cell anemia, is a severe inherited blood disorder caused by a genetic mutation that leads to misshapen, rigid red blood cells. These cells can obstruct blood flow, causing sudden episodes of severe pain, known as pain crises, and leading to life-threatening complications. Ayrmid is dedicated to advancing therapies that help individuals living with sickle cell disease manage their symptoms and improve their quality of life.

About Ayrmid Ltd. and Gamida Cell
Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed 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.

Contacts: Media, Investors / Business Development: bd@ayrmid.com

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit www.rns.com.

SOURCE: Ayrmid Pharma Ltd.

View the original press release on ACCESS Newswire

Study demonstrated early and sustained hematopoietic recovery and transfusion independence

Omisirge is a new and novel transplant option for patients with SAA

Additional manufacturing capacity for Omisirge will be established in the U.S.

Represents a supplementary FDA approval for Omisirge

DUBLIN, IE AND LONDON, UK / ACCESS Newswire / December 5, 2025 / Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a leader in cell therapy innovation announces that it has received U.S. Food and Drug Administration (“FDA”) approval for Omisirge (Omidubicel-onlv) for the treatment of Severe Aplastic Anemia (SAA).

This approval is based on the ongoing open-label, single-center study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), which demonstrated highly encouraging results including:

• Median time to neutrophil recovery: 11 days

• 86% of patients had early and sustained neutrophil recovery at 100 days

• Immune recovery post-transplant was rapid

• 86% of patients achieved red blood cell transfusion independence

• No cases of BMT-CTN severe acute graft-versus-host disease (GVHD), or chronic GVHD observed

• 92% disease-free and overall survival rate

Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH) commented: “The approval of Omisirge is a significant step forward in the treatment options available for patients with a high unmet medical need. Patients in the study with aplastic anemia were high risk, but had significantly better than expected outcomes and demonstrated remarkably fast and high rates of neutrophil engraftment. This was achieved with low rates of mild acute GVHD and no chronic GVHD meaning the patients achieved a rapid return to a normal life.”

Dr. Joe Wiley, Ayrmid Chairman and Chief Executive Officer commented: “Alongside its initial FDA approval in hematological malignancy in 2023, Omisirge is a new and novel transplant option for the many SAA patients who do not have a matched related donor. This approval is potentially transformative for patients living with SAA, given the rapid and sustained recovery of blood counts observed. At Ayrmid, we remain deeply committed to advancing innovative therapies to help patients with serious unmet medical needs.”

To meet the expected demand from SAA patients, Ayrmid has a strategic partnership in place with RoslinCT for the production of Omisirge in the U.S. alongside existing manufacturing facilities. The technology transfer and commercial manufacturing of Omisirge will take place in RoslinCT’s state-of-the-art, purpose-built cell therapy CGMP manufacturing facility in Hopkinton, MA. beginning in 2027.

About Severe Aplastic Anemia
Severe Aplastic Anemia is a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. Stem cell transplantation offers a potential cure; however, many patients lack a matched sibling donor. Ayrmid is advancing therapies to address this unmet medical need.

About Ayrmid Ltd. and Gamida Cell
Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed 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.

Contacts: Media, Investors / Business Development: bd@ayrmid.com

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit www.rns.com.

SOURCE: Ayrmid Pharma Ltd.

View the original press release on ACCESS Newswire