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2025-05-31-accounts

NAITBABIES ANNUAL REPORT 2025

News and events from

1 June 2024– 31 May 2025

Fetal and neonatal alloimmune thrombocytopenia - FNAIT

Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

Reference and administrative details of the charity and its Trustees

Charity name: Naitbabies Other names we are known by: NAIT babies Charity number: 1161698 Principle address: 13 Redinnick Terrace Penzance Cornwall, TR18 4HR, England

Members of the CIO are also the trustees who administer the charity. Thea Palmer Chair Michelle Minshall Treasurer Stacy Corke Secretary Andrea Palmer Trustee

Treasurer : Michelle Minshall Minshall & Co Bridge House Nantwich Cheshire, CW5 7JX

Independent examiner: Mr Neill Hallam FCCA Crane and Johnston Chartered Certified Accountants 11 Alverton Terrace Penzance Cornwall, TR18 4JH

Bankers : HSBC, 1 Green Market Penzance Cornwall, TR18 2SD

Type of governing document : Constitution How the charity is constituted : Charitable Incorporated Organisation (CIO) Foundation Date of governing document : 15 May 2015

Website :

www.naitbabies.org

1 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

Naitbabies are members of:

Genetic Alliance UK

Rare Disease UK

NORD USA National Organization for Rare Disorders

Mission Statement

Our mission is to be a strong voice for parents, children and families who have been diagnosed with or suspect that they may have Fetal and neonatal alloimmune thrombocytopenia - FNAIT.

Objectives and Activities

The objects of the charity as set out in the CIO’s constitution are:

The relief of sickness and the preservation and protection of good health of parents, children and their families and carers who have or are affected by the severe bleeding disorder neonatal alloimmune thrombocytopenia – FNAIT; in particular by the provision of support, advocacy and practical assistance as the trustees shall think fit;

To carry out, or to provide funds to support research into FNAIT, its causes, treatment and prevention and publish the useful results of such research; and

To advance the education of the public, in particular those involved in the diagnosis, treatment and care of those suffering from FNAIT.

Summary of the main activities undertaken for the public benefit in relation to these objects.

Naitbabies are a small Charitable Incorporated Organisation run by families whose children have been affected by the severe bleeding disorder FNAIT – Fetal and neonatal alloimmune thrombocytopenia.

Registered in the UK we are the only organisation for this disorder. We provide information to the public in general.

We run an international FNAIT Parents Support Group and advocate for parents worldwide. To date we have over 1,200 members.

We support research into FNAIT and have contact with an expert medical panel of doctors who have a special interest in this devastating disease.

Watch our FNAIT video here

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

What is FNAIT?

Fetal and neonatal alloimmune thrombocytopenia FNAIT (NAIT FMAIT) results from incompatibility between mother and baby for platelet-specific antigens that have been inherited from the father and are absent in mother.

The maternal immune response may make antibodies to destroy her baby’s platelets which she sees as foreign. Platelet destruction may cause bleeding into all major organs e.g. the stomach, spinal cord and lungs. The most feared is bleeding into the brain known as intracranial haemorrhage - ICH. Babies are at serious risk of death or permanent neurological impairment such as epilepsy, cortical blindness, cerebral palsy, precocious puberty, motor and cognitive delays and sensory processing disorders.

What are platelets?

3 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

Petechial rash result from areas of haemorrhage into the dermis.

NAIT is analogous to the red blood cell disease Haemolytic Disease of the Fetus and Newborn – HDFN, more commonly known as Rhesus disease.

Routine antenatal care has included screening of all expectant mothers for HDFN since the early 1970’s. No country carries out antenatal screening for NAIT although it has been known about since the 1950’s and is very well documented. Rarer diseases are also screened for.

Treatment is available for mothers who have developed antibodies against their unborn baby’s platelets and the success rate is high. If subsequent pregnancies are not treated they may also be at risk.

JUNE 2024

FNAIT – Mental health

Naitbabies began an important FNAIT mental health research project in collaboration with Professor J B Bussel, Weill Cornell Medical University, New York, USA.

6 June - Pharmaceutical Company Janssen Research & Development, LLC, have registered the clinical trial study FREESIA-1. Nipocalimab is an investigational, monoclonal antibody that aims to selectively block the FcRn receptor to reduce levels of circulating immunoglobulin G (IgG) antibodies, including autoantibodies and alloantibodies. Nipocalimab is also known as M281

FREESIA-1 represents the first placebo-controlled, randomized, multicentre clinical trial

investigating the efficacy and safety of Nipocalimab, a non-invasive antenatal therapy, for the treatment of alloimmunized pregnant individuals at standard risk for FNAIT. ClinicalTrials.gov ID NCT06449651

Study Details | A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) | ClinicalTrials.gov

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

11 June - Professor Thijs W de Vos, Leiden Medical University, Netherlands has received the H.C.J.M. van Dijk prize at the 2024 NVB-TRIP Symposium for ‘’the best dissertation of 2023-2024 within the field’’ Entitled ” Fetal and Neonatal Alloimmune Thrombocytopenia: The proof of the pudding is in the eating” his dissertation can be read on this link htps://books.gildeprint.nl/thesis/586840-deVos/17/

JULY 2024

OUR NEW TRUSTEE Rachel Walker celebrated the run up to her 50[th] birthday by taking part in 50 park runs for NAIT babies!! Raising a magnificent £724!!!!

Rachel has three children, two of whom have been affected by FNAIT. Her first born, a son Otto, suffers from cerebral palsy and epilepsy. As there is no screening yet for FNAIT his intracranial haemorrhages were not picked up before his birth.

Rachel and husband Jamie have a 50% chance of every pregnancy being affected, due to Jamie’s platelet type being ‘HPA-1a/1b’, which makes him heterozygous for HPA-1.

Out of two further pregnancies, both girls, one was not affected and one was. Rachel was treated for FNAIT with weekly infusions of IVIG and her affected daughter had a normal platelet count at birth.

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

AUGUST 2024

8 August - Latest news from Rallybio, Connecticut, USA, Clinical Stage pharmaceutical company developing an FNAIT prophylaxis RLYB212:

– On Track to Initiate RLYB212 Phase 2 Dose Confirmation Trial in Pregnant Women at Higher Risk of FNAIT in 4Q 2024 –

PDF Version

SEPTEMBER 2024

27 – 29 September. ESPGIXVII

Three trustees attended the Seventeenth European Symposium on Platelet and Granulocyte Immunobiology held in Ede, The Netherlands.

https://sanquinacademy.nl/17th-european-symposium-on-platelet-and-granulocyteimmunobiology-espgi/

OCTOBER/NOVEMBER 2024

RALLYBIO’s Amanda Hayward, Head of Global Business Development presented their research, in partnership with Health Lumen, on quantifying the proportion of women at risk of an FNAIT pregnancy in diverse populations in the United States at NORD24. Using genetic data, researchers found that nearly 20,000 pregnancies in the U.S. are at higher risk for FNAIT each year—far more than previously estimated.

The study highlights that non-white populations also carry significant FNAIT risk, challenging past assumptions. These findings make a compelling case for universal screening of pregnant women for FNAIT risk, regardless of race or ethnicity.

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

Medical University, New York, USA.

‘’Analysis of Screening for Autism Spectrum Disorders in Children Affected By FNAIT with and without Intra-Cranial Hemorrhage’’

Katherine A. Knightly, Margaret H. McKelvy, James B. Bussel, Emilie Vander Haar, Eleanore A. McFarland, Thea D. Palmer, Stephanie V. Volpe, Stacy Corke.

Published in the American Society of Hematology ‘Blood’ Journal. https://doi.org/10.1182/blood-2024-205475

DECEMBER 2024

3 December – NAIT babies AGM

7 – 10 December – ASH, the 66th American Society of Hematology Annual Meeting and Exposition took place on December 7-10, 2024, in San Diego, California. An oral and abstract poster was presented of the research paper in the ASH Blood journal.

Analysis of Screening for Autism Spectrum Disorders in Children Affected By Fnait with and without Intra-Cranial Hemorrhage | Blood | American Society of Hematology

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

NAIT babies published research ‘’ Medical Problems of Mothers of Children with Fetal-Neonatal Alloimmune Thrombocytopenia (FNAIT): Autoimmunity and Psychological Symptoms’’ can be found on this link https://ash.confex.com/ash/2024/webprogram/Paper203685.html

9 December - Johnson & Johnson announce FREESIA-3 Clinical Trial ‘’Nipocalimab v IVIG’’, being held in the United Kingdom and other European countries.

Scientific name: ‘’Multicenter, open-label, randomised study of Nipocalimab or IVIG in pregnancies at risk of fetal and neonatal alloimmune thrombocytopenia (FREESIA-3)’’.

Principal investigator: Dr Katie Morris, Birmingham Women’s Hospital. UK Study participating centres:

Birmingham Women's Hospital, Mindelsohn Way, Edgbaston, Birmingham, B15 2TG

Liverpool Women’s Hospital, Crown Street, Liverpool, L8 7SS

Queen Charlotte’s and Chelsea Hospital, Du Cane Road, London , W12 0HS

United Kingdom link: https://www.isrctn.com/ISRCTN17841362 ClinicalTrials.gov number NCT06533098

Secondary identifying numbers: 80202135FNAIT3003, CPMS 62590

JANUARY 2025

10 January - Naitbabies were invited to the first NHSBT ‘’Plasma for Medicines’’ meeting. The UK are now producing their own plasma for medicines, which had previously been purchased from the USA and the EU.

February 2025

11 February - The clinical-stage biotechnology company rallybio.com have dosed the first participant in the Phase 2 clinical trial program RLYB212. The company expect pharmacokinetic and safety data from the second trimester in the second quarter of 2025, with pharmacokinetic and safety data at the time of delivery expected in the third quarter of 2025.

Read the full news release - https://investors.rallybio.com/news-releases/news-releasedetails/rallybio-announces-initiation-dosing-rlyb212-phase-2-clinical

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

28 February – Rare Disease Day 2025

March/April

25 – 31 March - FNAIT Annual Awareness week

2 April – At the invitation of Doctor Mike Desborough, Honorary Consultant Haematologist, NHS Blood and Transplant, Thea and Andrea Palmer, Trustees at Naitbabies presented at the first NHSBT UK National FNAIT Meeting held at Pembroke College, Oxford University.

8 April - Rallybio LLC announced they are to discontinue development of their RLYB212 Program. This was based on pharmacokinetic (PK) data from the Phase 2 clinical trial’ demonstrating the inability of the RLYB212 dose regimen to achieve predicted target concentrations, as well as the minimum target concentration required for efficacy.

Rallybio to Discontinue Development of RLYB212 for Prevention of FNAIT | Rallybio

11 April – Naitbabies research article has been published in the British Journal of Haematology. ‘’Fetal–neonatal alloimmune thrombocytopenia: Mothers are affected too’’ http://doi.org/10.1111/bjh.20112

April 2025 - Rising to the challenge: an international Delphi consensus study on fetal and neonatal alloimmune thrombocytopenia.

https://doi.org/10.1016/S2352-3026(25)00029-8

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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.

May

14 May NHSBT National Blood Week – media partners.

31 May – Naitbabies End of Year.

Many thanks to everyone for reading our review and for your continued support:

SAVING BABIES ONE TREATMENT AT A TIME!!!!

Naitbabies.org

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Charitable Incorporated Organisation (Foundation}.

Registered in England and Wales No.1161698.

Charity registration number: 1161698

Naitbabies.org

Annual Report and Financial Statements

for the Year Ended 31 May 2025

Naitbabies.org

Contents

Contents
Reference and Administrative Details 1
Trustees' Report 2 to 3
Independent Examiner's Report 4
Receipts & Payment Account 5
Statement of Assets & Liabilities 6
Notes to the Financial Statements 7

Naitbabies.org

Reference and Administrative Details

Charity Registration Number

1161698

Trustees

Y D Palmer A Palmer S Corke M Minshall R Walker

Principal Office

13 Redinnick Terrace Penzance Cornwall TR18 4HR

Independent Examiner

Mr Neil Hallam FCCA Crane & Johnston Chartered Certified Accountants 11 Alverton Terrace Penzance Cornwall TR18 4JH

Bankers

HSBC Penzance 1 Green Market Penzance Cornwall TR18 2SD

Page 1

Naitbabies.org

Trustees' Report

The trustees present the annual report together with the financial statements of the charity for the year ended 31 May 2025.

Objectives and activities

Objects and aims

The objects of the charity are as follows:

  1. The relief of sickness and the preservation and protection of good health of parents, children and their families and carers who have or are affected by the genetic disorder 'Neonatal Alloimmune Thrombocytopenia' (also known as NAIT or FNAIT). In particular by the provision of support, advocacy and practical assistance as the trustees shall think fit;

  2. To carry out, or to provide funds to support research into NAIT/FNAIT, its causes, treatment and prevention and publish the useful results of such research; and

  3. To advance the education of the public, in particular those involved in the diagnosis, treatment and care of those suffering from NAIT/FNAIT.

Activities

Naitbabies.org is a non-profit making organisation that has been created to raise the knowledge and awareness of a severe bleeding disorder many have never heard of, neonatal alloimmune thrombocytopenia or NAIT.

On 15 May 2015 Naitbabies became a registered charity and is now a Charitable Incorporated Organisation (CIO). Its registered number with Charity Commission is '1161698'.

Mission statement

Our mission is to be a strong voice for parents, children and families who have been diagnosed with, or suspect that they might have the genetic disorder neonatal alloimmune thrombocytopenia. We support research into NAIT/FNAIT, its causes, treatment and prevention.

Trustees and officers

The trustees and officers serving during the year and since the year end were as follows:

Trustees:

Y D Palmer

A Palmer

S Corke

M Minshall

R Walker

Page 2

Naitbabies.org

Trustees' Report (continued)

The annual report was approved by the trustees of the charity on 20 August 2025 and signed on its behalf by:

Page 3

Naitbabies.org

Independent Examiner's Report to the trustees of Naitbabies.org

I report to the trustees on my examination of the accounts of Naitbabies.org for the year ended 31 May 2025.

Responsibilities and basis of report

As the charity trustees of Naitbabies.org you are responsible for the preparation of the accounts in accordance with the requirements of the Charities Act 2011 (‘the Act’).

I report in respect of my examination of the Naitbabies.org's accounts carried out under section 145 of the 2011 Act and in carrying out my examination I have followed all the applicable Directions given by the Charity Commission under section 145(5)(b) of the Act.

Independent examiner’s statement

I have completed my examination. I confirm that no material matters have come to my attention in connection with the examination giving me cause to believe that in any material respect:

  1. accounting records were not kept in respect of Naitbabies.org as required by section 130 of the Act; or

  2. the accounts do not accord with those records.

I have no concerns and have come across no other matters in connection with the examination to which attention should be drawn in this report in order to enable a proper understanding of the accounts to be reached.

...................................... Neil Hallam FCCA Crane & Johnston Chartered Certified Accountants 11 Alverton Terrace Penzance Cornwall TR18 4JH

21 August 2025

Page 4

Naitbabies.org

Receipts & Payment Account for the Year Ended 31 May 2025

Total
Total
2025
2024
£
£
Receipts:
Receipts from charitable activities
Donations 2,094
3,072
Fundraising 28
134
Interest receivable 885
819
Total Receipts
3,007
4,025
Payments:
Costs of charitable activities
Accountancy (414)
(396)
Advertising and PR -
(65)
Bank charges (150)
(155)
Conferences and training (641)
(1,664)
Equipment expensed -
(342)
Insurance (149)
(123)
PC consumables and software -
(217)
Stationery and printing (29)
(77)
Subscriptions (496)
(421)
Sundry (6)
(16)
Telephone (193)
(193)
Travel and subsistence (1,987)
(492)
Total Payments
(4,065)
(4,161)
Net (payments)/receipts
(1,058)
(136)
Reconciliation of cash funds
Total cash funds brought forward
48,851
48,987
Total cash funds carried forward
47,793
48,851

Page 5

Naitbabies.org

(Registration number: 1161698) Statement of Assets & Liabilities as at 31 May 2025

2025
2024
£
£
Cash Funds
Cash at bank and in hand 47,793
48,851
Unrestricted income funds
Unrestricted funds 47,793
48,851
Total funds
47,793
48,851
Assets retained for the CIO's own use
(estimated written down value)
2025
2024
Note
£
£
Computer equipment & other equipment - unrestricted funds -
-
Computer equipment & other equipment purchased during
theyear - unrestricted funds -
-
-
-
Liabilities
Amounts relating to but not included in the accounts.
2025
2024
Note
£
£
Accountancy fee
438
415

The financial statements were approved by the trustees, and authorised for issue on 20 August 2025 and signed on their behalf by:

Page 6

Naitbabies.org

Notes to the Financial Statements for the Year Ended 31 May 2025

1 Accounting policies

Basis of preparation

The accounts have been prepared on the receipts and payments basis.

Naitbabies.org meets the definition of a public benefit entity under FRS 102. The accounts (financial statements) have been prepared under the historical cost convention with items recognised at cost or transaction value unless otherwise stated in the relevant note(s) to these accounts.

Receipts

Receipts are the total amounts received by the charity for goods and services provided to the public. Any donations have been included in the accounts when received.

Payments

Expenditure is included on a paid basis, including any VAT which cannot be recovered.

2 Trustees & Secretary remuneration and expenses

No trustees (or any persons connected with them) have received any remuneration from the charity during the year.

Two trustees (2024: no trustees) were reimbursed expenses from the charity during the year totalling £334 (2024: £Nil).

3 Guarantees

The trustees confirm, in accordance with the Charitable Incorporated Organisations (General) Regulations 2012, that at the year ending the CIO did not have any outstanding guarantees to third party nor any debts secured on assets of the CIO.

Page 7