BOOST Pharma – Treating the untreatable
BOOST Pharma is based on many years of collaborative research from Karolinska Institute in Stockholm with the focus on a novel cell therapy treatment for Osteogenesis Imperfecta. The research teams of associate professor Cecilia Götherström and professor Magnus Westgren have shown that treatment with BOOST cells, called BP-1, greatly enhanced the quality of life for patients suffering from this otherwise extremely debilitating disease.
Osteogenesis Imperfecta
Brittle Bone Disease
The greatest wish of every parent, every family, is to have their children grow up with a bright future, the ability to pursue their dreams, and life in perfect, or at the very least modest, health.
Unfortunately, for parents with children suffering from Osteogenesis Imperfecta, this is not the case. This devastating inherited disease, which is usually abbreviated as OI, leaves patients bound to a life filled with pain, fear and disability. This disease is due to mutations, often in the collagen gene, that cause collagen to either not be present in sufficient amounts or not be of sufficient quality.
Patients have
- Very fragile bones, resulting in many bone fractures, especially during the early years of life
- Deformities of long bones, spinal curvature, and deformed ribs
- Pain, fatigue, hearing loss, impaired pulmonary function
There are very few therapies available and those that exist, such as physiotherapy, rodding surgery, and bisphosphates (BPs), are merely palliative and fail to reduce the frequency of fractures. Generally, OI sufferers have an almost normal life span with severe disabilities due to bone defects and hundreds of painful bone fractures, especially during the first years of life, causing irreversible damage.
OI affects currently more than 56.000 people in the United States of America (USA) and the European Union (EU) alone, while 1 out of 15,000 newborns worldwide is diagnosed with OI.
There is no cure, no treatment. To date, there is no EMA and FDA approved therapy.
BOOST Pharma was founded based on science from Karolinska Institutet, Sweden, a world-leader in cell therapy research. BOOST Pharma is developing a cell therapy treatment, called BP-1, for Osteogenesis Imperfecta (OI) using Mesenchymal Stem Cells (MSC).
Technology
BOOST Pharma is developing a first-in-class therapy to treat Osteogenesis Imperfecta (OI). This therapy, BP-1, is based on novel Mesenchymal Stem Cells (MSC) with high bone-forming capabilities.
In mice models, BP-1 has shown that cell therapy leads to higher calcium deposition, higher alkaline phosphatase activity, and a high ectopic bone formation.
Once injected, cells will migrate to the bone of patients with OI, where they will engraft and start bone formation. BOOST Pharma obtained human proof-of-concept for BP-1 after four children with Type III and IV OI were treated; the children have been followed for years up to adolescence.
BP-1 shows great promise for the effectiveness of treating children with OI: the children treated followed their own growth curve, had increased lengthwise growth compared to contemporary OI patients and showed a significant reduction of bone fractures. The cells are considered to be safe with no adverse reactions and no immune responses towards the donor MSC.
BP-1 therapy starts already at the prenatal stage, when OI is first diagnosed, or as early as possible after the child is born. By treating this early, BOOST Pharma is addressing the disease at the earliest possible stage, thereby increasing the treatment benefits for the patient in later years, such as strong bones and possibly improved lung function. A typical treatment scheme for BP-1, such as used in the Phase I/II BOOSTB4 trial, are infusions of every 4 month for one year, i.e. directly after the diagnosis for OI has been made. Patients will be monitored to see, if they follow their normal growth curve and they are monitored for occurence of bone fractures.
BOOST Pharma Corporate Milestones
- Mar 2020: First patient dosed with BP-1 in Phase I/II BOOSTB4 trial
- Dec 2021: Orphan Drug Designation in EU
- May 2022: Orphan Drug Designation in USA
- Oct 2022: FDA’s Rare Paediatric Disease (RPD) Designation
- Jan 2023: Final dosing BP-1 in Phase I/II BOOSTB4 trial
- May 2024: Investors Industrifonden and KD joined
- Sep 2024: BP-1 positive topline results in Phase I/II BOOSTB4 trial
- Nov 2024: Positive pIND meeting outcome with FDA
The next steps for BOOST Pharma are mainly to prepare for the pivotal Phase III trial with BP-1 for OI Type III/IV children. We have ongoing fundraising activities to support these developments.
Clinical Trials
The two clinical studies – one in Europe (BOOSTB4) and one in India (BOOST2B) – were funded under a Horizon 2020 academic grant, Vinnova, and the Swedish Research Council. Both of these clinical trials started in the first half of 2020 and positive results were announced for the BOOSTB4 trial in September 2024.
The BOOSTB4 Phase I/II clinical trial is a multi-centre, open-label, and multiple doses for safety and efficacy of postnatal or pre-and/or postnatal dosing of BP-1. The treatment regime is four doses of allogeneic MSC every 4th month during the duration of the trials. The inclusion age is ≤18 months.
The BOOST2B Phase I/II clinical trial is a single-center, open-label, and multiple doses for safety and efficacy postnatal dosing. In this trial, older children will be treated (1-4 years of age). We also are examining another type of administration: direct injection into the bone, in addition to intravenous injection.
Team
Evert Kueppers
Corporate Development - MBA & M.Sc. Biomedical Sciences
Serial Biotech CEO ao. Pieris Pharmaceuticals (NASDAQ:PIRS)
25 yrs ops leadership, incl.R&D restructuring
Raised over 100M€ (US/EU)
Built three ventures
Lilian Walther Jallow
Co-founder & CDO - PhD
Cell therapy drug development
CMC/RA experience
Former Sci. Secretary Swedish Pharmaceutical Society
ATMP Sweden Board member
Over 40 publications in peer-reviewed papers
Tony Hall
Chief Medical Officer - MB BS BSc AKC MFPM(Dis)
Former TA Head Orphan Diseases at Mereo Biopharma, former CMO Healx & Med. Dir. Prosensa
Strong network within OI patients groups
Co-founder & Trustee of rare diseases charity Beacon
Fredrik Lehmann
CMC Advisor - PhD
20+ years of experience in the life science industry. Acted as CEO, Head of Research and CMC
Venture Partner Industrifonden
Board of Directors
Ingelise Saunders
Chairman & CEO - M.Sc. Pharm.
More than 35 years experience from biotech and pharmaceutical industry at C-level positions as well as chairman/member of boards both in SME and large pharma companies
Magnus Westgren
Co-founder and Member of BoD and SAB - MD & PhD
Senior Professor at Karolinska Institutet. Established fetal therapies in Sweden and performed the first prenatal MSC transplantation in the world
John Öhd
Member of BoD - MD PhD
Board representative for Karolinska Development, Sweden
Jonathan Ilicki
Member of BoD - MD MSc. MBA
Board representative for Industrifonden, Sweden
Scientific Advisors
Cecilia Gotherstrom
Co-founder and SAB - PhD
Associate professor for stem cell science at Karolinska Institutet. Author of numerous publications on mesenchymal stem cell therapy for OI.
Jörg Oliver Semler
Member of SAB - MD & PhD
One of the world’s foremost OI experts. Head of the Department of Skeletal Dysplasias and associate professor of pediatrics at University Hospital in Cologne. Chair of the medical advisory board of the national and the European patient organization for OI
Willem E. Fibbe
Member of SAB - MD & PhD
Chairman of the Department of Immunohematology and Blood Transfusion at Leiden University. An expert within Stem Cell Therapies and has vast experience with clinical program using Advanced Therapy Medicinal Products (ATMP) for a variety of clinical indications
Publications
BOOSTB4 PHASE I/II: THE CLINICAL TRIAL PROTOCOLL:
Sagar et al. An exploratory open-label multi-centre Phase I/II trial evaluating the safety and efficacy of postnatal or prenatal and postnatal administration of…. BMJ Open (2024). Jun 4;14 (6):e079767.
Case report on stem cell engraftment in OI patient:
Le Blanc, Katarina et al. Fetal mesenchymal stem-cell engraftment in bone after in utero transplantation in a patient with severe osteogenesis imperfecta. Transplantation vol. 79,11 (2005): 1607-14. doi:10.1097/01.tp.0000159029.48678.93
Review article on prenatal stem cell therapies:
Ekblad-Nordberg Å, Walther-Jallow L, Westgren M, Götherström C. Prenatal stem cell therapy for inherited diseases: Past, present, and future treatment strategies. Stem Cells Transl Med. 2020;9(2):148-157. doi:10.1002/sctm.19-0107
Review article on stem cell therapy for OI:
Götherström C, Walther-Jallow L. Stem Cell Therapy as a Treatment for Osteogenesis Imperfecta. Curr Osteoporos Rep. 2020 Aug;18(4):337-343. doi: 10.1007/s11914-020-00594-3. PMID: 32710427; PMCID: PMC7419362.
About BOOST Pharma
BOOST Pharma is based on years of collaborative research from Karolinska Institute in Stockholm with a focus on novel cell therapy treatment for Osteogenesis Imperfecta.